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Baricitinib may benefit patients with Aicardi-Goutières syndrome
CHARLOTTE, N.C. – Scores on a novel AGS scale improved, and skin and liver complications resolved in children with AGS who received treatment with baricitinib, according to results presented at the annual meeting of the Child Neurology Society.
AGS is caused by various heritable disorders of the innate immunity that result in excessive interferon production. AGS characteristically manifests as an early-onset encephalopathy that causes intellectual and physical disability, but patients may have a wide range of clinical phenotypes. The disease may involve the skin, liver, lungs, heart, and other organs, as well as the brain.
A multisystem disorder
“The neurologic features, while they are the most compelling for us, are really only the tip of the iceberg,” said Adeline Vanderver, MD, program director of the leukodystrophy center, and the Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics at Children’s Hospital of Philadelphia. “Nearly every single organ system in the body is affected, from either direct interferon injury or from a secondary vasculopathy related to the interferonopathy.”
Dr. Vanderver presented results from the compassionate use study, which assessed whether the JAK inhibitor baricitinib (Olumiant) may decrease interferon signaling in AGS and limit the morbidity of the disease.
The phase 1, open-label trial “included compassionate use of baricitinib in AGS under the argument that these children did not have time to wait for approval of the drug,” said Dr. Vanderver. In 2018, the Food and Drug Administration approved baricitinib for moderate to severe rheumatoid arthritis in adults with an inadequate response to methotrexate.
The phase 1 trial in AGS included 35 patients with mutation-defined AGS and evidence of inflammatory disease that could be targeted by JAK inhibition. The trial population was 36% female. The average age of disease onset was 0.8 years, and patients’ average age at treatment was 6.1 years. The investigators assessed safety and laboratory data every 3 months and conducted clinical assessments every 6 months.
The heterogeneity of AGS phenotypes within families and across genotypes makes treatment trials in this disorder a challenge, Dr. Vanderver said. Outcome measures may have ceiling or floor effects that fail to capture the range of severity of AGS symptoms. Dr. Vanderver and colleagues developed a novel AGS scale to capture the scope of neurologic function in patients with AGS
.
When the researchers applied the AGS scale to a historical cohort of patients, most had stable scores about 6 months after disease onset. “After the first 6 months of the disease, the disease tends to be much more static, as the children have sustained significant neurologic injury,” Dr. Vanderver said.
They applied the novel AGS scale post hoc as an exploratory endpoint in the phase 1 trial. In addition, parents recorded information in a diary about skin involvement, irritability, seizures, and fever. “Over time, we see a reduction, although not always a statistically significant reduction, in symptom burden,” Dr. Vanderver said. The AGS clinical diary scores reflect “what the parents were telling us – that they felt like their children were feeling better during treatment,” she said.
Several patients had skin conditions that improved with treatment. One patient with dermatitis or eczema had the skin abnormality resolve within 3 days. A patient with full-body panniculitis began healing for the first time after about a month of treatment. Seasonal variations and dose adjustments led to fluctuations in some of the skin conditions. Nevertheless, the results suggested significant improvement in skin manifestations in patients with AGS, Dr. Vanderver said.
Patients generally had stable AGS scale scores in the year before treatment, although a couple of patients who were closer to disease onset had precipitous decline in neurologic function, she said. “We had a statistically significant increase in that scale of neurologic function in our patients during the period of the study, even in patients who had sometimes had years of disease duration,” said Dr. Vanderver.
Dr. Vanderver cautioned that she does not want to overstate the changes in function. Patients with AGS may have less potential for recuperation, compared with patients with other conditions. “A child with significant disruptive CNS disease may not recuperate normal functioning,” Dr. Vanderver said, “but it can be clinically meaningful to families if children start having better head control, smile, communicate, even if they might not regain all their motor milestones.”
In addition, a small subset of patients who had potentially life threatening liver complications from the disease experienced rapid normalization and improvement of liver function. “This blockade can be important not just for neurologic function but also to maintain normal physiologic homeostasis of other organs that are affected by the interferonopathy,” Dr. Vanderver said.
Interferon signaling scores decreased in the days after starting treatment and subsequently leveled out.
Serious adverse events that occurred during the trial, such as hospitalizations, were attributable to AGS. One child died from unrecognized pulmonary hypertension, which is now known to be a complication of AGS but was not at the time.
Harnessing a side effect
The most significant and recurrent laboratory abnormality was thrombocytosis. “That is a known complication of this family of drugs that in many cases allowed us to improve previous treatment-resistant thrombocytopenia, so we kind of like that side effect in most cases, but in two cases it did ... result in dose adjustments, although we never had to stop the medication for that.”
The study offers proof of principle that AGS is treatable, Dr. Vanderver said. A phase 2 trial is enrolling patients closer to disease onset. Early treatment of AGS may remain a challenge until there is newborn screening for the disease, she said.
Dr. Vanderver receives grant and in-kind support for translational research without personal compensation from Eli Lilly, Takeda, Illumina, Biogen, Homology, and Ionis. In addition, Dr. Vanderver serves on the scientific advisory boards of the European Leukodystrophy Association and the United Leukodystrophy Foundation, as well as in an unpaid capacity for Takeda, Ionis, Biogen, and Illumina.
Eli Lilly provided support for the phase 1 study. In addition, the study received support from the AGS Association Americas Family Foundation, National Human Genome Research Institute, National Institute of Neurological Disorders and Stroke, and the Children’s Hospital of Philadelphia Research Institute.
SOURCE: Vanderver A et al. CNS 2019. Abstract PL1-6.
CHARLOTTE, N.C. – Scores on a novel AGS scale improved, and skin and liver complications resolved in children with AGS who received treatment with baricitinib, according to results presented at the annual meeting of the Child Neurology Society.
AGS is caused by various heritable disorders of the innate immunity that result in excessive interferon production. AGS characteristically manifests as an early-onset encephalopathy that causes intellectual and physical disability, but patients may have a wide range of clinical phenotypes. The disease may involve the skin, liver, lungs, heart, and other organs, as well as the brain.
A multisystem disorder
“The neurologic features, while they are the most compelling for us, are really only the tip of the iceberg,” said Adeline Vanderver, MD, program director of the leukodystrophy center, and the Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics at Children’s Hospital of Philadelphia. “Nearly every single organ system in the body is affected, from either direct interferon injury or from a secondary vasculopathy related to the interferonopathy.”
Dr. Vanderver presented results from the compassionate use study, which assessed whether the JAK inhibitor baricitinib (Olumiant) may decrease interferon signaling in AGS and limit the morbidity of the disease.
The phase 1, open-label trial “included compassionate use of baricitinib in AGS under the argument that these children did not have time to wait for approval of the drug,” said Dr. Vanderver. In 2018, the Food and Drug Administration approved baricitinib for moderate to severe rheumatoid arthritis in adults with an inadequate response to methotrexate.
The phase 1 trial in AGS included 35 patients with mutation-defined AGS and evidence of inflammatory disease that could be targeted by JAK inhibition. The trial population was 36% female. The average age of disease onset was 0.8 years, and patients’ average age at treatment was 6.1 years. The investigators assessed safety and laboratory data every 3 months and conducted clinical assessments every 6 months.
The heterogeneity of AGS phenotypes within families and across genotypes makes treatment trials in this disorder a challenge, Dr. Vanderver said. Outcome measures may have ceiling or floor effects that fail to capture the range of severity of AGS symptoms. Dr. Vanderver and colleagues developed a novel AGS scale to capture the scope of neurologic function in patients with AGS
.
When the researchers applied the AGS scale to a historical cohort of patients, most had stable scores about 6 months after disease onset. “After the first 6 months of the disease, the disease tends to be much more static, as the children have sustained significant neurologic injury,” Dr. Vanderver said.
They applied the novel AGS scale post hoc as an exploratory endpoint in the phase 1 trial. In addition, parents recorded information in a diary about skin involvement, irritability, seizures, and fever. “Over time, we see a reduction, although not always a statistically significant reduction, in symptom burden,” Dr. Vanderver said. The AGS clinical diary scores reflect “what the parents were telling us – that they felt like their children were feeling better during treatment,” she said.
Several patients had skin conditions that improved with treatment. One patient with dermatitis or eczema had the skin abnormality resolve within 3 days. A patient with full-body panniculitis began healing for the first time after about a month of treatment. Seasonal variations and dose adjustments led to fluctuations in some of the skin conditions. Nevertheless, the results suggested significant improvement in skin manifestations in patients with AGS, Dr. Vanderver said.
Patients generally had stable AGS scale scores in the year before treatment, although a couple of patients who were closer to disease onset had precipitous decline in neurologic function, she said. “We had a statistically significant increase in that scale of neurologic function in our patients during the period of the study, even in patients who had sometimes had years of disease duration,” said Dr. Vanderver.
Dr. Vanderver cautioned that she does not want to overstate the changes in function. Patients with AGS may have less potential for recuperation, compared with patients with other conditions. “A child with significant disruptive CNS disease may not recuperate normal functioning,” Dr. Vanderver said, “but it can be clinically meaningful to families if children start having better head control, smile, communicate, even if they might not regain all their motor milestones.”
In addition, a small subset of patients who had potentially life threatening liver complications from the disease experienced rapid normalization and improvement of liver function. “This blockade can be important not just for neurologic function but also to maintain normal physiologic homeostasis of other organs that are affected by the interferonopathy,” Dr. Vanderver said.
Interferon signaling scores decreased in the days after starting treatment and subsequently leveled out.
Serious adverse events that occurred during the trial, such as hospitalizations, were attributable to AGS. One child died from unrecognized pulmonary hypertension, which is now known to be a complication of AGS but was not at the time.
Harnessing a side effect
The most significant and recurrent laboratory abnormality was thrombocytosis. “That is a known complication of this family of drugs that in many cases allowed us to improve previous treatment-resistant thrombocytopenia, so we kind of like that side effect in most cases, but in two cases it did ... result in dose adjustments, although we never had to stop the medication for that.”
The study offers proof of principle that AGS is treatable, Dr. Vanderver said. A phase 2 trial is enrolling patients closer to disease onset. Early treatment of AGS may remain a challenge until there is newborn screening for the disease, she said.
Dr. Vanderver receives grant and in-kind support for translational research without personal compensation from Eli Lilly, Takeda, Illumina, Biogen, Homology, and Ionis. In addition, Dr. Vanderver serves on the scientific advisory boards of the European Leukodystrophy Association and the United Leukodystrophy Foundation, as well as in an unpaid capacity for Takeda, Ionis, Biogen, and Illumina.
Eli Lilly provided support for the phase 1 study. In addition, the study received support from the AGS Association Americas Family Foundation, National Human Genome Research Institute, National Institute of Neurological Disorders and Stroke, and the Children’s Hospital of Philadelphia Research Institute.
SOURCE: Vanderver A et al. CNS 2019. Abstract PL1-6.
CHARLOTTE, N.C. – Scores on a novel AGS scale improved, and skin and liver complications resolved in children with AGS who received treatment with baricitinib, according to results presented at the annual meeting of the Child Neurology Society.
AGS is caused by various heritable disorders of the innate immunity that result in excessive interferon production. AGS characteristically manifests as an early-onset encephalopathy that causes intellectual and physical disability, but patients may have a wide range of clinical phenotypes. The disease may involve the skin, liver, lungs, heart, and other organs, as well as the brain.
A multisystem disorder
“The neurologic features, while they are the most compelling for us, are really only the tip of the iceberg,” said Adeline Vanderver, MD, program director of the leukodystrophy center, and the Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics at Children’s Hospital of Philadelphia. “Nearly every single organ system in the body is affected, from either direct interferon injury or from a secondary vasculopathy related to the interferonopathy.”
Dr. Vanderver presented results from the compassionate use study, which assessed whether the JAK inhibitor baricitinib (Olumiant) may decrease interferon signaling in AGS and limit the morbidity of the disease.
The phase 1, open-label trial “included compassionate use of baricitinib in AGS under the argument that these children did not have time to wait for approval of the drug,” said Dr. Vanderver. In 2018, the Food and Drug Administration approved baricitinib for moderate to severe rheumatoid arthritis in adults with an inadequate response to methotrexate.
The phase 1 trial in AGS included 35 patients with mutation-defined AGS and evidence of inflammatory disease that could be targeted by JAK inhibition. The trial population was 36% female. The average age of disease onset was 0.8 years, and patients’ average age at treatment was 6.1 years. The investigators assessed safety and laboratory data every 3 months and conducted clinical assessments every 6 months.
The heterogeneity of AGS phenotypes within families and across genotypes makes treatment trials in this disorder a challenge, Dr. Vanderver said. Outcome measures may have ceiling or floor effects that fail to capture the range of severity of AGS symptoms. Dr. Vanderver and colleagues developed a novel AGS scale to capture the scope of neurologic function in patients with AGS
.
When the researchers applied the AGS scale to a historical cohort of patients, most had stable scores about 6 months after disease onset. “After the first 6 months of the disease, the disease tends to be much more static, as the children have sustained significant neurologic injury,” Dr. Vanderver said.
They applied the novel AGS scale post hoc as an exploratory endpoint in the phase 1 trial. In addition, parents recorded information in a diary about skin involvement, irritability, seizures, and fever. “Over time, we see a reduction, although not always a statistically significant reduction, in symptom burden,” Dr. Vanderver said. The AGS clinical diary scores reflect “what the parents were telling us – that they felt like their children were feeling better during treatment,” she said.
Several patients had skin conditions that improved with treatment. One patient with dermatitis or eczema had the skin abnormality resolve within 3 days. A patient with full-body panniculitis began healing for the first time after about a month of treatment. Seasonal variations and dose adjustments led to fluctuations in some of the skin conditions. Nevertheless, the results suggested significant improvement in skin manifestations in patients with AGS, Dr. Vanderver said.
Patients generally had stable AGS scale scores in the year before treatment, although a couple of patients who were closer to disease onset had precipitous decline in neurologic function, she said. “We had a statistically significant increase in that scale of neurologic function in our patients during the period of the study, even in patients who had sometimes had years of disease duration,” said Dr. Vanderver.
Dr. Vanderver cautioned that she does not want to overstate the changes in function. Patients with AGS may have less potential for recuperation, compared with patients with other conditions. “A child with significant disruptive CNS disease may not recuperate normal functioning,” Dr. Vanderver said, “but it can be clinically meaningful to families if children start having better head control, smile, communicate, even if they might not regain all their motor milestones.”
In addition, a small subset of patients who had potentially life threatening liver complications from the disease experienced rapid normalization and improvement of liver function. “This blockade can be important not just for neurologic function but also to maintain normal physiologic homeostasis of other organs that are affected by the interferonopathy,” Dr. Vanderver said.
Interferon signaling scores decreased in the days after starting treatment and subsequently leveled out.
Serious adverse events that occurred during the trial, such as hospitalizations, were attributable to AGS. One child died from unrecognized pulmonary hypertension, which is now known to be a complication of AGS but was not at the time.
Harnessing a side effect
The most significant and recurrent laboratory abnormality was thrombocytosis. “That is a known complication of this family of drugs that in many cases allowed us to improve previous treatment-resistant thrombocytopenia, so we kind of like that side effect in most cases, but in two cases it did ... result in dose adjustments, although we never had to stop the medication for that.”
The study offers proof of principle that AGS is treatable, Dr. Vanderver said. A phase 2 trial is enrolling patients closer to disease onset. Early treatment of AGS may remain a challenge until there is newborn screening for the disease, she said.
Dr. Vanderver receives grant and in-kind support for translational research without personal compensation from Eli Lilly, Takeda, Illumina, Biogen, Homology, and Ionis. In addition, Dr. Vanderver serves on the scientific advisory boards of the European Leukodystrophy Association and the United Leukodystrophy Foundation, as well as in an unpaid capacity for Takeda, Ionis, Biogen, and Illumina.
Eli Lilly provided support for the phase 1 study. In addition, the study received support from the AGS Association Americas Family Foundation, National Human Genome Research Institute, National Institute of Neurological Disorders and Stroke, and the Children’s Hospital of Philadelphia Research Institute.
SOURCE: Vanderver A et al. CNS 2019. Abstract PL1-6.
REPORTING FROM CNS 2019
Celiac disease may underlie seizures
CHARLOTTE, N.C. – , according to a retrospective chart review presented at the annual meeting of the Child Neurology Society. Associations between celiac disease and seizures may have implications for screening and treatment, said study author Shanna Swartwood, MD, a fellow in the department of pediatric neurology at University of Utah in Salt Lake City.
“Screening for [celiac disease] early in patients with epilepsy, specifically with temporal EEG findings and intractable epilepsy, is warranted given the improvement of seizure burden that may result from exclusion of gluten from the diet,” said Dr. Swartwood and colleagues.
About 10% of patients with celiac disease have clinical neurologic manifestations, such as seizures. To characterize features of epilepsy in a pediatric population with celiac disease and to examine the effect of a gluten-free diet on seizure burden, Dr. Swartwood and colleagues reviewed patients treated at Primary Children’s Hospital in Salt Lake City since 2002. They identified patients with ICD-10 codes for seizures or epilepsy and celiac disease and reviewed 187 charts in all.
In all, 40 patients with seizures had biopsy-proven celiac disease, and 22 had a diagnosis of celiac disease based on the presence of antibodies. Among those with biopsy-proven celiac disease, 43% had intractable seizures. Among those with antibody-positive celiac disease, 31% had intractable seizures.
Among patients with intractable epilepsy, seizure onset preceded the diagnosis of celiac disease by an average of 5 years. For patients with nonintractable epilepsy, the first seizure occurred 1 year before the celiac disease diagnosis on average, but some patients received a celiac disease diagnosis first.
Focal seizures with secondary generalization and generalized tonic clonic seizures were the most common seizure types in this cohort. Epileptiform activity most often was seen in the temporal lobe. While other studies in patients with celiac disease have found occipital epileptiform activity to be the most common, only one patient in this cohort had activity in that location, Dr. Swartwood noted.
Patients with intractable seizures who adhered to a gluten-free diet “had a fairly robust response in terms of seizure improvement,” she said. Seizure improvement, including a decrease in seizure frequency or a decrease in antiepileptic medication dosage, occurred in seven of nine patients in the biopsy-proven group and in two of three patients in the antibody-positive group who adhered to a gluten-free diet and had intractable seizures. One patient was able to stop antiepileptic medication, and one patient had a complete resolution of seizure activity.
The researchers plan to further study the relationship between celiac disease and epilepsy, including whether various HLA subtypes of celiac disease correlate with seizures, said coinvestigator Cristina Trandafir, MD, PhD, assistant professor of pediatric neurology at University of Utah.
The chart review included relatively few patients with limited data. Nevertheless, the results suggest that there may be “substantial lag time” from first seizure to celiac disease diagnosis and that “earlier diagnosis and earlier placement on a gluten-free diet may be beneficial,” Dr. Swartwood said. Celiac disease may be asymptomatic, and screening for celiac disease with a blood test may make sense for patients with intractable seizures, she said.
The researchers had no relevant disclosures.
CHARLOTTE, N.C. – , according to a retrospective chart review presented at the annual meeting of the Child Neurology Society. Associations between celiac disease and seizures may have implications for screening and treatment, said study author Shanna Swartwood, MD, a fellow in the department of pediatric neurology at University of Utah in Salt Lake City.
“Screening for [celiac disease] early in patients with epilepsy, specifically with temporal EEG findings and intractable epilepsy, is warranted given the improvement of seizure burden that may result from exclusion of gluten from the diet,” said Dr. Swartwood and colleagues.
About 10% of patients with celiac disease have clinical neurologic manifestations, such as seizures. To characterize features of epilepsy in a pediatric population with celiac disease and to examine the effect of a gluten-free diet on seizure burden, Dr. Swartwood and colleagues reviewed patients treated at Primary Children’s Hospital in Salt Lake City since 2002. They identified patients with ICD-10 codes for seizures or epilepsy and celiac disease and reviewed 187 charts in all.
In all, 40 patients with seizures had biopsy-proven celiac disease, and 22 had a diagnosis of celiac disease based on the presence of antibodies. Among those with biopsy-proven celiac disease, 43% had intractable seizures. Among those with antibody-positive celiac disease, 31% had intractable seizures.
Among patients with intractable epilepsy, seizure onset preceded the diagnosis of celiac disease by an average of 5 years. For patients with nonintractable epilepsy, the first seizure occurred 1 year before the celiac disease diagnosis on average, but some patients received a celiac disease diagnosis first.
Focal seizures with secondary generalization and generalized tonic clonic seizures were the most common seizure types in this cohort. Epileptiform activity most often was seen in the temporal lobe. While other studies in patients with celiac disease have found occipital epileptiform activity to be the most common, only one patient in this cohort had activity in that location, Dr. Swartwood noted.
Patients with intractable seizures who adhered to a gluten-free diet “had a fairly robust response in terms of seizure improvement,” she said. Seizure improvement, including a decrease in seizure frequency or a decrease in antiepileptic medication dosage, occurred in seven of nine patients in the biopsy-proven group and in two of three patients in the antibody-positive group who adhered to a gluten-free diet and had intractable seizures. One patient was able to stop antiepileptic medication, and one patient had a complete resolution of seizure activity.
The researchers plan to further study the relationship between celiac disease and epilepsy, including whether various HLA subtypes of celiac disease correlate with seizures, said coinvestigator Cristina Trandafir, MD, PhD, assistant professor of pediatric neurology at University of Utah.
The chart review included relatively few patients with limited data. Nevertheless, the results suggest that there may be “substantial lag time” from first seizure to celiac disease diagnosis and that “earlier diagnosis and earlier placement on a gluten-free diet may be beneficial,” Dr. Swartwood said. Celiac disease may be asymptomatic, and screening for celiac disease with a blood test may make sense for patients with intractable seizures, she said.
The researchers had no relevant disclosures.
CHARLOTTE, N.C. – , according to a retrospective chart review presented at the annual meeting of the Child Neurology Society. Associations between celiac disease and seizures may have implications for screening and treatment, said study author Shanna Swartwood, MD, a fellow in the department of pediatric neurology at University of Utah in Salt Lake City.
“Screening for [celiac disease] early in patients with epilepsy, specifically with temporal EEG findings and intractable epilepsy, is warranted given the improvement of seizure burden that may result from exclusion of gluten from the diet,” said Dr. Swartwood and colleagues.
About 10% of patients with celiac disease have clinical neurologic manifestations, such as seizures. To characterize features of epilepsy in a pediatric population with celiac disease and to examine the effect of a gluten-free diet on seizure burden, Dr. Swartwood and colleagues reviewed patients treated at Primary Children’s Hospital in Salt Lake City since 2002. They identified patients with ICD-10 codes for seizures or epilepsy and celiac disease and reviewed 187 charts in all.
In all, 40 patients with seizures had biopsy-proven celiac disease, and 22 had a diagnosis of celiac disease based on the presence of antibodies. Among those with biopsy-proven celiac disease, 43% had intractable seizures. Among those with antibody-positive celiac disease, 31% had intractable seizures.
Among patients with intractable epilepsy, seizure onset preceded the diagnosis of celiac disease by an average of 5 years. For patients with nonintractable epilepsy, the first seizure occurred 1 year before the celiac disease diagnosis on average, but some patients received a celiac disease diagnosis first.
Focal seizures with secondary generalization and generalized tonic clonic seizures were the most common seizure types in this cohort. Epileptiform activity most often was seen in the temporal lobe. While other studies in patients with celiac disease have found occipital epileptiform activity to be the most common, only one patient in this cohort had activity in that location, Dr. Swartwood noted.
Patients with intractable seizures who adhered to a gluten-free diet “had a fairly robust response in terms of seizure improvement,” she said. Seizure improvement, including a decrease in seizure frequency or a decrease in antiepileptic medication dosage, occurred in seven of nine patients in the biopsy-proven group and in two of three patients in the antibody-positive group who adhered to a gluten-free diet and had intractable seizures. One patient was able to stop antiepileptic medication, and one patient had a complete resolution of seizure activity.
The researchers plan to further study the relationship between celiac disease and epilepsy, including whether various HLA subtypes of celiac disease correlate with seizures, said coinvestigator Cristina Trandafir, MD, PhD, assistant professor of pediatric neurology at University of Utah.
The chart review included relatively few patients with limited data. Nevertheless, the results suggest that there may be “substantial lag time” from first seizure to celiac disease diagnosis and that “earlier diagnosis and earlier placement on a gluten-free diet may be beneficial,” Dr. Swartwood said. Celiac disease may be asymptomatic, and screening for celiac disease with a blood test may make sense for patients with intractable seizures, she said.
The researchers had no relevant disclosures.
REPORTING FROM CNS 2019
Pediatric epilepsy surgery may improve cognition and behavior
CHARLOTTE, NC – according to a study presented at the annual meeting of the Child Neurology Society. The presence of comorbidities such as mood disorders and autism may influence the likelihood of perceived improvement, whereas the type of surgery may not.
“The parents and the families of the patients perceive that, even if the patients are not completely seizure free, the behavior and cognitive outcomes are better if there is some sort of seizure improvement,” said Trishna Kantamneni, MD, director of pediatric epilepsy at UC Davis in Sacramento.
To assess behavioral and cognitive outcomes following pediatric epilepsy surgery and to identify factors that predict improvement, Dr. Kantamneni and colleagues at the Cleveland Clinic Epilepsy Center retrospectively reviewed 126 patients younger than 18 years who underwent epilepsy surgery for medically refractory epilepsy during 2009-2016.
The primary outcome measure was the Impact of Childhood Neurologic Disability Scale (ICNDS), a parent-reported scale that assesses the behavior, cognition, and physical or neurologic disability of children with epilepsy. Parents completed the ICNDS preoperatively and at 6, 12, and 24 months after surgery. The researchers constructed separate linear mixed effects models to identify predictors of postoperative changes in ICNDS score.
Of the 126 patients, 62.7% were male, the median duration of epilepsy was 4.7 years, and 69.8% were seizure-free at the 2-year follow-up. Postoperative ICNDS scores were available for 103 patients at 6 months and for 54 patients at 24 months.
Before surgery, the average total ICNDS score was 55.7. At 6 months after surgery, the average score was 34.6, and at 24 months, it was 32.1, representing significant improvement from baseline.
In addition, behavior, cognition, and epilepsy subscores also improved post operatively, and the improvement persisted through 24 months. ICNDS scores significantly improved “even in patients who were not seizure-free after surgery,” by an average of about 22 points, the researchers said.
The absence of comorbid autism, cognitive impairment, and global developmental impairment and the absence of anxiety, depression, and ADHD were predictors of improved total ICNDS scores. Tumor pathology and being seizure free at 2 years also predicted improved scores. Duration and type of epilepsy, the number of antiepileptic drugs that patients were taking before surgery, and lobe of surgery were not predictive of improved ICNDS scores.
Dr. Kantamneni had no relevant disclosures.
SOURCE: Kantamneni T et al. CNS 2019, Abstract 51.
CHARLOTTE, NC – according to a study presented at the annual meeting of the Child Neurology Society. The presence of comorbidities such as mood disorders and autism may influence the likelihood of perceived improvement, whereas the type of surgery may not.
“The parents and the families of the patients perceive that, even if the patients are not completely seizure free, the behavior and cognitive outcomes are better if there is some sort of seizure improvement,” said Trishna Kantamneni, MD, director of pediatric epilepsy at UC Davis in Sacramento.
To assess behavioral and cognitive outcomes following pediatric epilepsy surgery and to identify factors that predict improvement, Dr. Kantamneni and colleagues at the Cleveland Clinic Epilepsy Center retrospectively reviewed 126 patients younger than 18 years who underwent epilepsy surgery for medically refractory epilepsy during 2009-2016.
The primary outcome measure was the Impact of Childhood Neurologic Disability Scale (ICNDS), a parent-reported scale that assesses the behavior, cognition, and physical or neurologic disability of children with epilepsy. Parents completed the ICNDS preoperatively and at 6, 12, and 24 months after surgery. The researchers constructed separate linear mixed effects models to identify predictors of postoperative changes in ICNDS score.
Of the 126 patients, 62.7% were male, the median duration of epilepsy was 4.7 years, and 69.8% were seizure-free at the 2-year follow-up. Postoperative ICNDS scores were available for 103 patients at 6 months and for 54 patients at 24 months.
Before surgery, the average total ICNDS score was 55.7. At 6 months after surgery, the average score was 34.6, and at 24 months, it was 32.1, representing significant improvement from baseline.
In addition, behavior, cognition, and epilepsy subscores also improved post operatively, and the improvement persisted through 24 months. ICNDS scores significantly improved “even in patients who were not seizure-free after surgery,” by an average of about 22 points, the researchers said.
The absence of comorbid autism, cognitive impairment, and global developmental impairment and the absence of anxiety, depression, and ADHD were predictors of improved total ICNDS scores. Tumor pathology and being seizure free at 2 years also predicted improved scores. Duration and type of epilepsy, the number of antiepileptic drugs that patients were taking before surgery, and lobe of surgery were not predictive of improved ICNDS scores.
Dr. Kantamneni had no relevant disclosures.
SOURCE: Kantamneni T et al. CNS 2019, Abstract 51.
CHARLOTTE, NC – according to a study presented at the annual meeting of the Child Neurology Society. The presence of comorbidities such as mood disorders and autism may influence the likelihood of perceived improvement, whereas the type of surgery may not.
“The parents and the families of the patients perceive that, even if the patients are not completely seizure free, the behavior and cognitive outcomes are better if there is some sort of seizure improvement,” said Trishna Kantamneni, MD, director of pediatric epilepsy at UC Davis in Sacramento.
To assess behavioral and cognitive outcomes following pediatric epilepsy surgery and to identify factors that predict improvement, Dr. Kantamneni and colleagues at the Cleveland Clinic Epilepsy Center retrospectively reviewed 126 patients younger than 18 years who underwent epilepsy surgery for medically refractory epilepsy during 2009-2016.
The primary outcome measure was the Impact of Childhood Neurologic Disability Scale (ICNDS), a parent-reported scale that assesses the behavior, cognition, and physical or neurologic disability of children with epilepsy. Parents completed the ICNDS preoperatively and at 6, 12, and 24 months after surgery. The researchers constructed separate linear mixed effects models to identify predictors of postoperative changes in ICNDS score.
Of the 126 patients, 62.7% were male, the median duration of epilepsy was 4.7 years, and 69.8% were seizure-free at the 2-year follow-up. Postoperative ICNDS scores were available for 103 patients at 6 months and for 54 patients at 24 months.
Before surgery, the average total ICNDS score was 55.7. At 6 months after surgery, the average score was 34.6, and at 24 months, it was 32.1, representing significant improvement from baseline.
In addition, behavior, cognition, and epilepsy subscores also improved post operatively, and the improvement persisted through 24 months. ICNDS scores significantly improved “even in patients who were not seizure-free after surgery,” by an average of about 22 points, the researchers said.
The absence of comorbid autism, cognitive impairment, and global developmental impairment and the absence of anxiety, depression, and ADHD were predictors of improved total ICNDS scores. Tumor pathology and being seizure free at 2 years also predicted improved scores. Duration and type of epilepsy, the number of antiepileptic drugs that patients were taking before surgery, and lobe of surgery were not predictive of improved ICNDS scores.
Dr. Kantamneni had no relevant disclosures.
SOURCE: Kantamneni T et al. CNS 2019, Abstract 51.
REPORTING FROM CNS 2019
Delay in EEG monitoring associated with increased seizure duration in pediatric refractory status epilepticus
CHARLOTTE, NC – , according to a multicenter study that was presented at the annual meeting of the Child Neurology Society. Delays in initiating EEG monitoring are associated with longer seizure duration in this patient population.
Neurologists are advised to initiate continuous EEG monitoring rapidly for all cases of pediatric refractory status epilepticus. Little information is available, however, about patterns in the timing of EEG placement. In addition, the relationship between delays in the initiation of continuous EEG and outcomes of refractory status epilepticus are unknown. Dmitry Tchapyjnikov, MD, assistant professor of child neurology at Duke University in Durham, N.C., and colleagues evaluated trends in the time to continuous EEG initiation and examined whether delays are associated with longer seizure duration in children with refractory status epilepticus.
A retrospective analysis of pSERG data
Dr. Tchapyjnikov and colleagues analyzed data from 11 hospitals participating in the Pediatric Status Epilepticus Research Group (pSERG), a prospective, observational cohort. They focused on pediatric patients who were admitted from 2011 to 2017 with refractory status epilepticus, which they defined as a seizure that persisted after treatment with two or more antiseizure medications (ASMs), one of which had to be a nonbenzodiazepine ASM, or a continuous infusion. Eligible patients were between 1 month and 21 years old and had convulsive seizures at onset. Patients who had EEG placement before seizure onset were excluded.
The investigators included in their study 121 patients who had seizure durations of 3 or more hours. Based on an exploratory analysis of various time-point cutoffs, Dr. Tchapyjnikov and colleagues defined delayed continuous EEG placement as placement at more than 5 hours after seizure onset. They used the Kaplan–Meier estimator to assess time to continuous EEG and used covariate-adjusted proportional hazards models to examine the association between delay in continuous EEG placement and seizure duration.
EEG placement overall was delayed
The median time to continuous EEG placement after seizure onset was 9 hours. Approximately 4% of the children had continuous EEG placed within 1 hour, and 74% had it placed within 24 hours.
The investigators found that seizure onset outside the study hospital was associated with a higher likelihood of delayed time to EEG placement. “Females seemed to be more likely to have timely EEG placement,” said Dr. Tchapyjnikov. “I don’t have a physiological explanation for that.” The researchers saw no difference in treatment between patients who had timely EEG placement and those who had delayed EEG placement.
About 68% of children were having seizures at the time of continuous EEG placement. A presumed seizure etiology of CNS infection was associated with a higher likelihood of being in status epilepticus at the time of EEG placement. A history of epilepsy, developmental delay, or home ASM use, however, was associated with a lower likelihood of being in status epilepticus at time of EEG placement.
Dr. Tchapyjnikov’s group found that the 24-hour cumulative probability of seizure resolution was lower among patients who did not have continuous EEG initiation within 5 hours, compared with those who did (56% vs.70%). The association remained significant after the investigators adjusted the data for covariates that were independently associated with 24-hour seizure resolution (hazard ratio, 0.31).
The investigators included in their analysis patients who had seizure resolution before EEG placement, because restricting the analysis to patients who have persistent status epilepticus would have overemphasized the benefits of EEG, according to Dr. Tchapyjnikov. “Looking at the overall hazard ratios is a more conservative way of looking at these data.”
The study was not supported by external funding. Dr. Tchapyjnikov had no relevant disclosures.
CHARLOTTE, NC – , according to a multicenter study that was presented at the annual meeting of the Child Neurology Society. Delays in initiating EEG monitoring are associated with longer seizure duration in this patient population.
Neurologists are advised to initiate continuous EEG monitoring rapidly for all cases of pediatric refractory status epilepticus. Little information is available, however, about patterns in the timing of EEG placement. In addition, the relationship between delays in the initiation of continuous EEG and outcomes of refractory status epilepticus are unknown. Dmitry Tchapyjnikov, MD, assistant professor of child neurology at Duke University in Durham, N.C., and colleagues evaluated trends in the time to continuous EEG initiation and examined whether delays are associated with longer seizure duration in children with refractory status epilepticus.
A retrospective analysis of pSERG data
Dr. Tchapyjnikov and colleagues analyzed data from 11 hospitals participating in the Pediatric Status Epilepticus Research Group (pSERG), a prospective, observational cohort. They focused on pediatric patients who were admitted from 2011 to 2017 with refractory status epilepticus, which they defined as a seizure that persisted after treatment with two or more antiseizure medications (ASMs), one of which had to be a nonbenzodiazepine ASM, or a continuous infusion. Eligible patients were between 1 month and 21 years old and had convulsive seizures at onset. Patients who had EEG placement before seizure onset were excluded.
The investigators included in their study 121 patients who had seizure durations of 3 or more hours. Based on an exploratory analysis of various time-point cutoffs, Dr. Tchapyjnikov and colleagues defined delayed continuous EEG placement as placement at more than 5 hours after seizure onset. They used the Kaplan–Meier estimator to assess time to continuous EEG and used covariate-adjusted proportional hazards models to examine the association between delay in continuous EEG placement and seizure duration.
EEG placement overall was delayed
The median time to continuous EEG placement after seizure onset was 9 hours. Approximately 4% of the children had continuous EEG placed within 1 hour, and 74% had it placed within 24 hours.
The investigators found that seizure onset outside the study hospital was associated with a higher likelihood of delayed time to EEG placement. “Females seemed to be more likely to have timely EEG placement,” said Dr. Tchapyjnikov. “I don’t have a physiological explanation for that.” The researchers saw no difference in treatment between patients who had timely EEG placement and those who had delayed EEG placement.
About 68% of children were having seizures at the time of continuous EEG placement. A presumed seizure etiology of CNS infection was associated with a higher likelihood of being in status epilepticus at the time of EEG placement. A history of epilepsy, developmental delay, or home ASM use, however, was associated with a lower likelihood of being in status epilepticus at time of EEG placement.
Dr. Tchapyjnikov’s group found that the 24-hour cumulative probability of seizure resolution was lower among patients who did not have continuous EEG initiation within 5 hours, compared with those who did (56% vs.70%). The association remained significant after the investigators adjusted the data for covariates that were independently associated with 24-hour seizure resolution (hazard ratio, 0.31).
The investigators included in their analysis patients who had seizure resolution before EEG placement, because restricting the analysis to patients who have persistent status epilepticus would have overemphasized the benefits of EEG, according to Dr. Tchapyjnikov. “Looking at the overall hazard ratios is a more conservative way of looking at these data.”
The study was not supported by external funding. Dr. Tchapyjnikov had no relevant disclosures.
CHARLOTTE, NC – , according to a multicenter study that was presented at the annual meeting of the Child Neurology Society. Delays in initiating EEG monitoring are associated with longer seizure duration in this patient population.
Neurologists are advised to initiate continuous EEG monitoring rapidly for all cases of pediatric refractory status epilepticus. Little information is available, however, about patterns in the timing of EEG placement. In addition, the relationship between delays in the initiation of continuous EEG and outcomes of refractory status epilepticus are unknown. Dmitry Tchapyjnikov, MD, assistant professor of child neurology at Duke University in Durham, N.C., and colleagues evaluated trends in the time to continuous EEG initiation and examined whether delays are associated with longer seizure duration in children with refractory status epilepticus.
A retrospective analysis of pSERG data
Dr. Tchapyjnikov and colleagues analyzed data from 11 hospitals participating in the Pediatric Status Epilepticus Research Group (pSERG), a prospective, observational cohort. They focused on pediatric patients who were admitted from 2011 to 2017 with refractory status epilepticus, which they defined as a seizure that persisted after treatment with two or more antiseizure medications (ASMs), one of which had to be a nonbenzodiazepine ASM, or a continuous infusion. Eligible patients were between 1 month and 21 years old and had convulsive seizures at onset. Patients who had EEG placement before seizure onset were excluded.
The investigators included in their study 121 patients who had seizure durations of 3 or more hours. Based on an exploratory analysis of various time-point cutoffs, Dr. Tchapyjnikov and colleagues defined delayed continuous EEG placement as placement at more than 5 hours after seizure onset. They used the Kaplan–Meier estimator to assess time to continuous EEG and used covariate-adjusted proportional hazards models to examine the association between delay in continuous EEG placement and seizure duration.
EEG placement overall was delayed
The median time to continuous EEG placement after seizure onset was 9 hours. Approximately 4% of the children had continuous EEG placed within 1 hour, and 74% had it placed within 24 hours.
The investigators found that seizure onset outside the study hospital was associated with a higher likelihood of delayed time to EEG placement. “Females seemed to be more likely to have timely EEG placement,” said Dr. Tchapyjnikov. “I don’t have a physiological explanation for that.” The researchers saw no difference in treatment between patients who had timely EEG placement and those who had delayed EEG placement.
About 68% of children were having seizures at the time of continuous EEG placement. A presumed seizure etiology of CNS infection was associated with a higher likelihood of being in status epilepticus at the time of EEG placement. A history of epilepsy, developmental delay, or home ASM use, however, was associated with a lower likelihood of being in status epilepticus at time of EEG placement.
Dr. Tchapyjnikov’s group found that the 24-hour cumulative probability of seizure resolution was lower among patients who did not have continuous EEG initiation within 5 hours, compared with those who did (56% vs.70%). The association remained significant after the investigators adjusted the data for covariates that were independently associated with 24-hour seizure resolution (hazard ratio, 0.31).
The investigators included in their analysis patients who had seizure resolution before EEG placement, because restricting the analysis to patients who have persistent status epilepticus would have overemphasized the benefits of EEG, according to Dr. Tchapyjnikov. “Looking at the overall hazard ratios is a more conservative way of looking at these data.”
The study was not supported by external funding. Dr. Tchapyjnikov had no relevant disclosures.
REPORTING FROM CNS 2019
Does AED prophylaxis delay seizure onset in children with brain tumors?
CHARLOTTE, N.C. – according to research presented at the annual meeting of the Child Neurology Society. Levetiracetam, oxcarbazepine, and phenytoin are the most common initial prophylactic AEDs administered to children with brain tumors, the researchers said.
The literature indicates that between 20% and 35% of children with brain tumors have seizures, and up to half of these patients have seizure as their presenting symptom. Common practice is to prescribe antiseizure medication after a child has had a first seizure, because the risk for recurrence is high. In 2000, the American Academy of Neurology discouraged prophylactic use of AEDs in children, citing a lack of evidence for efficacy. Most of the data that it reviewed, however, came from adults.
Michelle Yun, a medical student at Weill Cornell Medical College, New York, and colleagues used national Medicaid claims data that had been collected between 2009 and 2012 for children with seizures to conduct a retrospective, observational, case-control study. They included children aged 0-20 years with a diagnosis of brain tumor, a seizure diagnosis within 6 months after brain tumor diagnosis, an AED prescription, and 12 continuous months of Medicaid coverage following brain tumor diagnosis in their analysis. The investigators defined seizure prophylaxis as AED prescription within 30 days after brain tumor diagnosis but before a first seizure diagnosis.
The exposure in the study was AED prescription within 45 days of diagnosis, and the outcome was the time to first seizure. Ms. Yun and colleagues also analyzed the most common initial prophylactic AEDs and the proportion of cases with first seizure diagnosis after prophylactic AED discontinuation, which was defined as a treatment gap longer than 30 days. The study covariates included age, sex, race, ethnicity, and medical comorbidities.
In all, 218 children were included in the study; 40 received AED prophylaxis and 26 received it within 45 days of brain tumor diagnosis. Patients with and without AED prophylaxis were well matched on all covariates.
At 1 year, Ms. Yun and colleagues saw no difference in time to first seizure between the two groups. The median time to first seizure was 75 days in the prophylaxis group and 80 days in the no-prophylaxis group. The researchers observed a transient separation between the two groups, however, in the early months after brain tumor diagnosis. When they examined children who had a seizure during the first 6 months of follow-up, the median time to diagnosis of first seizure was 68 days in children with prophylaxis and 34 days in the no-prophylaxis group. The difference between groups was statistically significant. “When we added all the covariates of interest, we found that there was a protective effect in these children with early seizures,” said Ms. Yun.
Among the study limitations that Ms. Yun acknowledged were its observational, retrospective design and its small sample size. Medicaid data themselves are limited, since states do not report them in a uniform manner, and the data do not include much clinical information. “Something that would be helpful is a prospective clinical study,” Ms. Yun concluded.
The Weill Cornell Clinical and Translational Science Center and the American Academy of Neurology provided funding for the study. The Pediatric Epilepsy Research Foundation provided the Medicaid data. Ms. Yun had no relevant disclosures.
SOURCE: Yun M et al. CNS 2019, Abstract PL2-1.
CHARLOTTE, N.C. – according to research presented at the annual meeting of the Child Neurology Society. Levetiracetam, oxcarbazepine, and phenytoin are the most common initial prophylactic AEDs administered to children with brain tumors, the researchers said.
The literature indicates that between 20% and 35% of children with brain tumors have seizures, and up to half of these patients have seizure as their presenting symptom. Common practice is to prescribe antiseizure medication after a child has had a first seizure, because the risk for recurrence is high. In 2000, the American Academy of Neurology discouraged prophylactic use of AEDs in children, citing a lack of evidence for efficacy. Most of the data that it reviewed, however, came from adults.
Michelle Yun, a medical student at Weill Cornell Medical College, New York, and colleagues used national Medicaid claims data that had been collected between 2009 and 2012 for children with seizures to conduct a retrospective, observational, case-control study. They included children aged 0-20 years with a diagnosis of brain tumor, a seizure diagnosis within 6 months after brain tumor diagnosis, an AED prescription, and 12 continuous months of Medicaid coverage following brain tumor diagnosis in their analysis. The investigators defined seizure prophylaxis as AED prescription within 30 days after brain tumor diagnosis but before a first seizure diagnosis.
The exposure in the study was AED prescription within 45 days of diagnosis, and the outcome was the time to first seizure. Ms. Yun and colleagues also analyzed the most common initial prophylactic AEDs and the proportion of cases with first seizure diagnosis after prophylactic AED discontinuation, which was defined as a treatment gap longer than 30 days. The study covariates included age, sex, race, ethnicity, and medical comorbidities.
In all, 218 children were included in the study; 40 received AED prophylaxis and 26 received it within 45 days of brain tumor diagnosis. Patients with and without AED prophylaxis were well matched on all covariates.
At 1 year, Ms. Yun and colleagues saw no difference in time to first seizure between the two groups. The median time to first seizure was 75 days in the prophylaxis group and 80 days in the no-prophylaxis group. The researchers observed a transient separation between the two groups, however, in the early months after brain tumor diagnosis. When they examined children who had a seizure during the first 6 months of follow-up, the median time to diagnosis of first seizure was 68 days in children with prophylaxis and 34 days in the no-prophylaxis group. The difference between groups was statistically significant. “When we added all the covariates of interest, we found that there was a protective effect in these children with early seizures,” said Ms. Yun.
Among the study limitations that Ms. Yun acknowledged were its observational, retrospective design and its small sample size. Medicaid data themselves are limited, since states do not report them in a uniform manner, and the data do not include much clinical information. “Something that would be helpful is a prospective clinical study,” Ms. Yun concluded.
The Weill Cornell Clinical and Translational Science Center and the American Academy of Neurology provided funding for the study. The Pediatric Epilepsy Research Foundation provided the Medicaid data. Ms. Yun had no relevant disclosures.
SOURCE: Yun M et al. CNS 2019, Abstract PL2-1.
CHARLOTTE, N.C. – according to research presented at the annual meeting of the Child Neurology Society. Levetiracetam, oxcarbazepine, and phenytoin are the most common initial prophylactic AEDs administered to children with brain tumors, the researchers said.
The literature indicates that between 20% and 35% of children with brain tumors have seizures, and up to half of these patients have seizure as their presenting symptom. Common practice is to prescribe antiseizure medication after a child has had a first seizure, because the risk for recurrence is high. In 2000, the American Academy of Neurology discouraged prophylactic use of AEDs in children, citing a lack of evidence for efficacy. Most of the data that it reviewed, however, came from adults.
Michelle Yun, a medical student at Weill Cornell Medical College, New York, and colleagues used national Medicaid claims data that had been collected between 2009 and 2012 for children with seizures to conduct a retrospective, observational, case-control study. They included children aged 0-20 years with a diagnosis of brain tumor, a seizure diagnosis within 6 months after brain tumor diagnosis, an AED prescription, and 12 continuous months of Medicaid coverage following brain tumor diagnosis in their analysis. The investigators defined seizure prophylaxis as AED prescription within 30 days after brain tumor diagnosis but before a first seizure diagnosis.
The exposure in the study was AED prescription within 45 days of diagnosis, and the outcome was the time to first seizure. Ms. Yun and colleagues also analyzed the most common initial prophylactic AEDs and the proportion of cases with first seizure diagnosis after prophylactic AED discontinuation, which was defined as a treatment gap longer than 30 days. The study covariates included age, sex, race, ethnicity, and medical comorbidities.
In all, 218 children were included in the study; 40 received AED prophylaxis and 26 received it within 45 days of brain tumor diagnosis. Patients with and without AED prophylaxis were well matched on all covariates.
At 1 year, Ms. Yun and colleagues saw no difference in time to first seizure between the two groups. The median time to first seizure was 75 days in the prophylaxis group and 80 days in the no-prophylaxis group. The researchers observed a transient separation between the two groups, however, in the early months after brain tumor diagnosis. When they examined children who had a seizure during the first 6 months of follow-up, the median time to diagnosis of first seizure was 68 days in children with prophylaxis and 34 days in the no-prophylaxis group. The difference between groups was statistically significant. “When we added all the covariates of interest, we found that there was a protective effect in these children with early seizures,” said Ms. Yun.
Among the study limitations that Ms. Yun acknowledged were its observational, retrospective design and its small sample size. Medicaid data themselves are limited, since states do not report them in a uniform manner, and the data do not include much clinical information. “Something that would be helpful is a prospective clinical study,” Ms. Yun concluded.
The Weill Cornell Clinical and Translational Science Center and the American Academy of Neurology provided funding for the study. The Pediatric Epilepsy Research Foundation provided the Medicaid data. Ms. Yun had no relevant disclosures.
SOURCE: Yun M et al. CNS 2019, Abstract PL2-1.
REPORTING FROM CNS 2019
PASI-75 with ixekizumab approaches 90% in pediatric psoriasis study
MADRID – The interleukin-17A inhibitor , Kim A. Papp, MD, PhD, reported at the annual congress of the European Academy of Dermatology and Venereology.
The results bode well for an underserved population.
“I think all of us know that there is still a vulnerable population that remains a high-risk population because of the limited number of therapies available for them, and that is children,” said Dr. Papp, a dermatologist and president of Probity Medical Research, Inc., of Waterloo, Ont.
At present, etanercept, one of the earliest biologics to become available, and a relatively less effective one, is the only biologic approved for treatment of pediatric psoriasis. However, Lilly, which sponsored the phase 3 ixekizumab study, has announced that based upon the highly positive findings the company plans to seek Food and Drug Administration approval for an expanded indication for the medication in pediatric psoriasis. The company now markets ixekizumab for the approved indications of treatment of adults with moderate to severe plaque psoriasis, active psoriatic arthritis, or active ankylosing spondylitis.
The 12-week, double-blind, multicenter phase 3 trial known as IXORA-PEDS included 115 pediatric psoriasis patients randomized to weight-based ixekizumab, 30 on weight-based etanercept, and 58 on placebo. At the 12-week mark, everyone was switched to open-label ixekizumab in a long-term extension study. Children weighing less than 25 kg received a 40-mg loading dose of ixekizumab, followed by a maintenance dose of 20 mg by subcutaneous injection every 4 weeks. Patients weighing 25-50 kg got a starting dose of 80 mg, then 40 mg for maintenance therapy. Those who weighed more than 50 kg got the usual adult dosing: a 160-mg loading dose followed by 80 mg every 4 weeks. Etanercept was dosed at 0.8 mg/kg once weekly.
The coprimary endpoints were the proportion of subjects achieving a static Physician’s Global Assessment (sPGA) of 0 or 1 – that is, clear or almost clear skin – at week 12, and the PASI 75 response rate.
An sPGA of 0 or 1 at week 12 was documented in 81% of the ixekizumab group, 11% on placebo, and 40% of etanercept-treated patients, who on average had more severe baseline disease than did the other two groups.
The PASI 75 rate was 89% with ixekizumab, 25% for placebo, and 63% on etanercept. But Dr. Papp indicated that’s too low a bar. “I don’t think PASI 75s are the standard any longer,” he said.
More revealing was the PASI 90 rate: 78% with the IL-17A inhibitor, 5% in placebo-treated controls, and 40% with etanercept.
And then there’s the PASI 100 response rate: 50% with ixekizumab, 2% for placebo, and 17% for etanercept.
“I think this is very telling. I’ll leave it as a tantalizing comment that if one looks at the slope of the curve, it doesn’t yet seem to have reached its plateau at week 12 – and this is very similar to the pattern that we see in the adult population. I don’t have the long-term extension efficacy data, but I am, like you, very interested in seeing where this PASI 100 response rate finally plateaus,” Dr. Papp said.
He did, however, have the combined safety data for the 12-week double-blind phase plus the open-label extension, which he described as essentially the same as the adult experience. Injection-site reactions occurred in 19% of pediatric patients on ixekizumab, but they were generally mild and there were few if any treatment discontinuations for that reason. There was a 2% incidence of Crohn’s disease. Candidiasis and other infections were rare.
Seventy-one percent of the ixekizumab group had at least a 4-point improvement in itch on a 10-point self-rated scale by week 12, as did 20% of placebo-treated controls. A Dermatologic Life Quality Index score of 0 or 1 at week 12, indicative of no or minimal impact of psoriasis on quality of life, was documented in 64% of the ixekizumab group and 23% of controls.
Dr. Papp reported serving as a consultant, investigator, and/or speaker for Lilly and more than three dozen other pharmaceutical companies.
SOURCE: Papp KA. EADV Late breaker.
MADRID – The interleukin-17A inhibitor , Kim A. Papp, MD, PhD, reported at the annual congress of the European Academy of Dermatology and Venereology.
The results bode well for an underserved population.
“I think all of us know that there is still a vulnerable population that remains a high-risk population because of the limited number of therapies available for them, and that is children,” said Dr. Papp, a dermatologist and president of Probity Medical Research, Inc., of Waterloo, Ont.
At present, etanercept, one of the earliest biologics to become available, and a relatively less effective one, is the only biologic approved for treatment of pediatric psoriasis. However, Lilly, which sponsored the phase 3 ixekizumab study, has announced that based upon the highly positive findings the company plans to seek Food and Drug Administration approval for an expanded indication for the medication in pediatric psoriasis. The company now markets ixekizumab for the approved indications of treatment of adults with moderate to severe plaque psoriasis, active psoriatic arthritis, or active ankylosing spondylitis.
The 12-week, double-blind, multicenter phase 3 trial known as IXORA-PEDS included 115 pediatric psoriasis patients randomized to weight-based ixekizumab, 30 on weight-based etanercept, and 58 on placebo. At the 12-week mark, everyone was switched to open-label ixekizumab in a long-term extension study. Children weighing less than 25 kg received a 40-mg loading dose of ixekizumab, followed by a maintenance dose of 20 mg by subcutaneous injection every 4 weeks. Patients weighing 25-50 kg got a starting dose of 80 mg, then 40 mg for maintenance therapy. Those who weighed more than 50 kg got the usual adult dosing: a 160-mg loading dose followed by 80 mg every 4 weeks. Etanercept was dosed at 0.8 mg/kg once weekly.
The coprimary endpoints were the proportion of subjects achieving a static Physician’s Global Assessment (sPGA) of 0 or 1 – that is, clear or almost clear skin – at week 12, and the PASI 75 response rate.
An sPGA of 0 or 1 at week 12 was documented in 81% of the ixekizumab group, 11% on placebo, and 40% of etanercept-treated patients, who on average had more severe baseline disease than did the other two groups.
The PASI 75 rate was 89% with ixekizumab, 25% for placebo, and 63% on etanercept. But Dr. Papp indicated that’s too low a bar. “I don’t think PASI 75s are the standard any longer,” he said.
More revealing was the PASI 90 rate: 78% with the IL-17A inhibitor, 5% in placebo-treated controls, and 40% with etanercept.
And then there’s the PASI 100 response rate: 50% with ixekizumab, 2% for placebo, and 17% for etanercept.
“I think this is very telling. I’ll leave it as a tantalizing comment that if one looks at the slope of the curve, it doesn’t yet seem to have reached its plateau at week 12 – and this is very similar to the pattern that we see in the adult population. I don’t have the long-term extension efficacy data, but I am, like you, very interested in seeing where this PASI 100 response rate finally plateaus,” Dr. Papp said.
He did, however, have the combined safety data for the 12-week double-blind phase plus the open-label extension, which he described as essentially the same as the adult experience. Injection-site reactions occurred in 19% of pediatric patients on ixekizumab, but they were generally mild and there were few if any treatment discontinuations for that reason. There was a 2% incidence of Crohn’s disease. Candidiasis and other infections were rare.
Seventy-one percent of the ixekizumab group had at least a 4-point improvement in itch on a 10-point self-rated scale by week 12, as did 20% of placebo-treated controls. A Dermatologic Life Quality Index score of 0 or 1 at week 12, indicative of no or minimal impact of psoriasis on quality of life, was documented in 64% of the ixekizumab group and 23% of controls.
Dr. Papp reported serving as a consultant, investigator, and/or speaker for Lilly and more than three dozen other pharmaceutical companies.
SOURCE: Papp KA. EADV Late breaker.
MADRID – The interleukin-17A inhibitor , Kim A. Papp, MD, PhD, reported at the annual congress of the European Academy of Dermatology and Venereology.
The results bode well for an underserved population.
“I think all of us know that there is still a vulnerable population that remains a high-risk population because of the limited number of therapies available for them, and that is children,” said Dr. Papp, a dermatologist and president of Probity Medical Research, Inc., of Waterloo, Ont.
At present, etanercept, one of the earliest biologics to become available, and a relatively less effective one, is the only biologic approved for treatment of pediatric psoriasis. However, Lilly, which sponsored the phase 3 ixekizumab study, has announced that based upon the highly positive findings the company plans to seek Food and Drug Administration approval for an expanded indication for the medication in pediatric psoriasis. The company now markets ixekizumab for the approved indications of treatment of adults with moderate to severe plaque psoriasis, active psoriatic arthritis, or active ankylosing spondylitis.
The 12-week, double-blind, multicenter phase 3 trial known as IXORA-PEDS included 115 pediatric psoriasis patients randomized to weight-based ixekizumab, 30 on weight-based etanercept, and 58 on placebo. At the 12-week mark, everyone was switched to open-label ixekizumab in a long-term extension study. Children weighing less than 25 kg received a 40-mg loading dose of ixekizumab, followed by a maintenance dose of 20 mg by subcutaneous injection every 4 weeks. Patients weighing 25-50 kg got a starting dose of 80 mg, then 40 mg for maintenance therapy. Those who weighed more than 50 kg got the usual adult dosing: a 160-mg loading dose followed by 80 mg every 4 weeks. Etanercept was dosed at 0.8 mg/kg once weekly.
The coprimary endpoints were the proportion of subjects achieving a static Physician’s Global Assessment (sPGA) of 0 or 1 – that is, clear or almost clear skin – at week 12, and the PASI 75 response rate.
An sPGA of 0 or 1 at week 12 was documented in 81% of the ixekizumab group, 11% on placebo, and 40% of etanercept-treated patients, who on average had more severe baseline disease than did the other two groups.
The PASI 75 rate was 89% with ixekizumab, 25% for placebo, and 63% on etanercept. But Dr. Papp indicated that’s too low a bar. “I don’t think PASI 75s are the standard any longer,” he said.
More revealing was the PASI 90 rate: 78% with the IL-17A inhibitor, 5% in placebo-treated controls, and 40% with etanercept.
And then there’s the PASI 100 response rate: 50% with ixekizumab, 2% for placebo, and 17% for etanercept.
“I think this is very telling. I’ll leave it as a tantalizing comment that if one looks at the slope of the curve, it doesn’t yet seem to have reached its plateau at week 12 – and this is very similar to the pattern that we see in the adult population. I don’t have the long-term extension efficacy data, but I am, like you, very interested in seeing where this PASI 100 response rate finally plateaus,” Dr. Papp said.
He did, however, have the combined safety data for the 12-week double-blind phase plus the open-label extension, which he described as essentially the same as the adult experience. Injection-site reactions occurred in 19% of pediatric patients on ixekizumab, but they were generally mild and there were few if any treatment discontinuations for that reason. There was a 2% incidence of Crohn’s disease. Candidiasis and other infections were rare.
Seventy-one percent of the ixekizumab group had at least a 4-point improvement in itch on a 10-point self-rated scale by week 12, as did 20% of placebo-treated controls. A Dermatologic Life Quality Index score of 0 or 1 at week 12, indicative of no or minimal impact of psoriasis on quality of life, was documented in 64% of the ixekizumab group and 23% of controls.
Dr. Papp reported serving as a consultant, investigator, and/or speaker for Lilly and more than three dozen other pharmaceutical companies.
SOURCE: Papp KA. EADV Late breaker.
REPORTING FROM THE EADV CONGRESS
Understanding your LGBTQ patients’ needs
NEW ORLEANS – One of the most important things pediatricians can do to support their lesbian, gay, bisexual, transgender (LGBT) and other gender-nonconforming patients is to ask all their patients about their feelings, preferences and experiences when it comes to gender and sexuality, according to Julie Finger, MD, MPH.
It’s equally important not to make assumptions, she told attendees at the annual meeting of the American Academy of Pediatrics. Biology and sexual and gender identity and expression can be very diverse, she said. Specifically, doctors should not assume patients are heterosexual, that bisexuality is a phase, that orientation or attraction translates directly to behavior or vice versa, or that LGBTQ patients have unsupportive families or are engaging in risky behavior. Research suggests LGB youth have slightly higher rates of early sexual debut, sexual activity or multiple partners than straight or uncertain youth, but only marginally so.
Pediatricians also cannot assume a patient’s sexual orientation based on their partner’s gender or determine a patient’s sexual orientation or gender identity based on appearance – or even that either is the same as it was on the previous visit.
What doctors can be sure of is that they do have LGBTQ patients, said Dr. Finger, and assistant professor of clinical pediatrics at Tulane University in New Orleans. According to a 2016 Morbidity and Mortality Weekly Report (2016 Aug 12; 65[9]), about 1 in 10 students in grades 9-12 are a sexual minority. About 2% of respondents identify as gay or lesbian, 6% identify as bisexual and 3% say they aren’t sure.
Knowing the terminology
Dr. Finger defined key terminology regarding gender and sexuality. She first clarified that LGBT is not the full spectrum for sexual orientation. Pansexual (fluid attraction to any sex or gender) and asexual (lack of feeling sexual attraction) can also describe sexuality, and the Q on the end of LGBTQ is often an umbrella term for “queer” or “questioning” that encompasses anyone who fits outside conventional social norms of sexual identity and gender expression.
Sexual behaviors – which include “young men who have sex with men” and “young women who have sex with women” – do not necessarily correspond as one might expect with sexual orientation or identity, which is one’s concept of their romantic or sexual feelings, attractions and desires, again reinforcing the importance of asking patients their identity and preferences.
In terms of gender, a person’s natal or biologic gender is the one assigned people at birth based on their body parts and hormones. Gender identity is a person’s understanding of their own gender, and gender expression refers to how someone acts or presents themselves and communicates their gender within their culture.
Those who identify as “gender nonconforming, genderqueer, gender fluid, or nonbinary” see their gender on a spectrum, not within the binary “male” or “female.” A cisgender person’s gender identity matches both their biological sex assigned at birth and conventional cultural norms, while a transgender person’s gender differs from the sex they were assigned at birth. Transgender women (male to female, MTF) and men (female to men, FTM) go through the process of transition, a time that can occur in weeks or years when they shift from living as one gender to another.
While it’s unclear what leads to a person’s sexual orientation – likely a combination of genetic, hormonal and environmental factors—there is no question that sexual orientation is not a “choice,” Dr Finger said. Research has also clarified that one’s sexual orientation does not result from parenting behaviors or a history of sexual abuse.
“But I would urge all of you, instead of focusing on why someone is LGBTQ, to focus on what that means for them in their life,” Dr Finger said. “How is this bearing out in terms of their relationships and their behaviors, and how do they feel about it? How are they being supported by their family or their community, and how is it impacting their lives?”
She cited findings from a Human Rights Campaign survey in 2012 of 10,000 youth aged 13-17, which found that most LGBTQ respondents became aware of their same-sex attraction at 9 years of age, though the average age of disclosures is 16, an improvement from age 21 in the 1980s.
How and what to ask
Although children start becoming conscious of gender at ages 1-2, their sense of gender usually stabilizes by age 4.
“Who should we be screening for gender nonconformity? Quite frankly, all children, because all of them have some gender identity, so we should be asking them about that,” Dr Finger said.
When children are younger, doctors can ask parents about their child’s social interactions, forms of play, dress preferences, and mood. Questions for patients themselves, adapted for their age, might include, “Do you feel more like a girl, boy, neither or both?”, “How would you like to play, cut your hair and dress?” And “What name or pronoun (he or she) fits you?”
While such conversations do not necessarily need to happen annually, doctors should especially ask youth who dress or behave in non–gender-conforming ways or who appear to have mood, behavior or social difficulties.
To understand a patient’s sexuality, ask whether they are attracted to people of their own gender or sex, a different gender or sex, both or all genders or no one, or if they’re not sure yet. Doctors can then ask how comfortable they are with their attraction and whether they have told family members or friends about them.
Sexual behavior questions should be developmentally appropriate and lead to counsel but not judgment, Dr Finger said. Her method, with adjustments for age and development, starts, “There are many way of being sexual or intimate with someone: kissing, hugging and touching, and oral sex, anal sex and vaginal sex. Have you ever had any of these experiences? Which ones? With males or females or both, or other genders?”
Then she gets more specific while remaining sensitive. Doctors can ask younger children if they have held hands or cuddled with someone, if they have kissed someone, or if they have touched another person’s private parts. They can ask teens about oral sex, vaginal sex and anal sex and then gather more details about what parts went where, which helps determine what screenings or treatment options a patient may need or desire.
Doctors can use their judgment about whether to ask questions with parents in the room or not, but as kids grow older, it’s good practice to speak to patients without their caregivers present. Doctors should also explain the rules of confidentiality to their patients and be aware of the risks of “coming out,” including family discord or rejection, problems at school or work, social stigma, bullying and harassment, physical violence and risk-taking behaviors, such as substance use, self-injury and risky sexual behaviors. A HEADSSS screen can help doctors learn if any of these are present.
Making your practice inclusive and welcoming
Fewer than one in five teens who are “out” as LGBTQ have come out to their doctor, Dr Finger cited. Most are out to their friends and classmates, and more than half are out to their family, but teens are less likely to tell their doctors.
Research suggests one reason for this is the fact that pediatricians often don’t ask. One study found that only 20% of pediatricians discussed sexual orientation with their patients (Pediatrics 2010 Apr;125:e741-7). Similarly, only 30% of family physicians brought up sexual orientation, found another study (Fam. Med. 2001 May;33[5]:376-81). The studies found physicians more often discussed condoms, HIV, sexually transmitted infections, abstinence, violence, contraception or, in the case of family physicians, sexual behaviors, and relationships.
But another reason for not being out to doctors is a history of poor experiences. A Lambda Legal Survey in 2009 of 4,916 LGBT respondents found that 8% of LGB and 27% of transgender and gender nonconforming patients had been denied care because of their identity of orientation. Eleven percent said “providers refused to touch them or used excessive precautions,” Dr Finger reported. LGBTQ patients may fear the doctor’s reaction or not keeping their identity confidential. Patients may also have internalized shame or guilt due to societal norms or homophobia, and all these barriers can reduce LGBTQ people’s willingness to seek and access to competent care.
The first step to making LGBTQ patients comfortable in your practice is to confront your own personal biases, Dr Finger said. Understand what they are and that a provider’s discomfort, even unconscious, can be damaging to the patient-provider relationship.
“If you find that this is just not something that you’re going to be comfortable doing, at the very least, I would suggest that you find providers in your area who are comfortable working with this patient population and you refer your patients to them so that they can have a good, trusting patient-provider relationship with somebody who can provide the care that they need,” Dr Finger said.
The next step is creating a safe place with zero tolerance for insensitivity by training staff to be welcoming and inclusive, assuring patients confidentiality, providing support and resources and displaying LGBTQ-affirming materials. These youth need active, visible evidence that the office will be a safe place for them.
Ways pediatricians can communicate an inclusive environment include having gender-neutral restrooms, using “parent” instead of “mother/father” and using forms and EMR prompts with gender-neutral language or multiple options for gender selection.
Screening and LGBTQ patients’ health needs
LGB youth and those who aren’t sure of their sexual orientation tend to have higher rates of substance use, including tobacco, alcohol and illicit drugs, and are more often victims of rape and other sexual violence. Their rates of depressive symptoms, bullying victimization, and suicidality are also significantly higher than in their heterosexual cisgender peers. Homelessness rates are also considerably higher in LGBTQ youth than in heterosexual cisgender youth.
One thing pediatricians can do is work with parents to ensure a patient’s school is meeting their needs. The greater risks LGBTQ youth typically face are mediated by social support, resiliency, supportive friends and family and a supportive school environment, including inclusive curricula and supportive staff.
Lesbian and bisexual women are considerably more at risk for poor sexual or reproductive outcomes, Dr Finger said. Their rates of unplanned pregnancy are double that of straight women, contributing to their higher rates of emergency contraception and abortion. They are also more likely to have more partners (male and females), to have a younger sexual debut and to be forced into sex by a male partner—yet they are far less likely to perceive themselves as at risk for a sexually transmitted infection than their peers.
This patient population therefore may need contraception counseling, including discussing their current methods and reviewing their options, including emergency contraception and possibly an advance prescription. Dr Finger also suggests having male and female condoms available in the office.
Doctors should screen all their female patients, regardless of sexuality, for chlamydia and gonorrhea, and offer routine cervical cancer screening and the HPV vaccine, as recommended by the CDC. They might consider screening for trichomoniasis, bacterial vaginosis, herpes simplex, human papillomavirus and HIV.
For men who have sex with men, the CDC recommends HIV and syphilis serology, urine/pharyngeal/rectal gonorrhea nucleic acid amplification test (NAAT), urine/rectal chlamydia NAAT, and hepatitis C screening for those who are HIV-positive—all at least once a year.
For transgender patients, doctors need to assess their STI- and HIV-related risks based on their current anatomy and sexual behaviors.
Doctors should also consider discussing pre-exposure prophylaxis (PrEP) for any youth at high risk for HIV infection if they are at least 77 pounds (35 kg). Emtricitabine/tenofovir (Truvada, Descovy) reduces the chance of sexually acquired infection by 99%, and infection acquired via drug injection by 74% when taken as prescribed.
Resources
Dr Finger noted a range of resources for LGBTQ youth and their families and providers, including the Family Acceptance Project, Gay and Lesbian Medical Association, Gay, Lesbian and Straight Education Network, GLBTQ Legal Advocates and Defenders (GLAD), Human Rights Campaign, It Gets Better Project, LGBTQ Student Resources and Support, National Center for Lesbian Rights, Parents and Friends of Lesbians and Gays (PFLAG), Safe Schools Coalition and The Trevor Project (concerning suicide risk).
NEW ORLEANS – One of the most important things pediatricians can do to support their lesbian, gay, bisexual, transgender (LGBT) and other gender-nonconforming patients is to ask all their patients about their feelings, preferences and experiences when it comes to gender and sexuality, according to Julie Finger, MD, MPH.
It’s equally important not to make assumptions, she told attendees at the annual meeting of the American Academy of Pediatrics. Biology and sexual and gender identity and expression can be very diverse, she said. Specifically, doctors should not assume patients are heterosexual, that bisexuality is a phase, that orientation or attraction translates directly to behavior or vice versa, or that LGBTQ patients have unsupportive families or are engaging in risky behavior. Research suggests LGB youth have slightly higher rates of early sexual debut, sexual activity or multiple partners than straight or uncertain youth, but only marginally so.
Pediatricians also cannot assume a patient’s sexual orientation based on their partner’s gender or determine a patient’s sexual orientation or gender identity based on appearance – or even that either is the same as it was on the previous visit.
What doctors can be sure of is that they do have LGBTQ patients, said Dr. Finger, and assistant professor of clinical pediatrics at Tulane University in New Orleans. According to a 2016 Morbidity and Mortality Weekly Report (2016 Aug 12; 65[9]), about 1 in 10 students in grades 9-12 are a sexual minority. About 2% of respondents identify as gay or lesbian, 6% identify as bisexual and 3% say they aren’t sure.
Knowing the terminology
Dr. Finger defined key terminology regarding gender and sexuality. She first clarified that LGBT is not the full spectrum for sexual orientation. Pansexual (fluid attraction to any sex or gender) and asexual (lack of feeling sexual attraction) can also describe sexuality, and the Q on the end of LGBTQ is often an umbrella term for “queer” or “questioning” that encompasses anyone who fits outside conventional social norms of sexual identity and gender expression.
Sexual behaviors – which include “young men who have sex with men” and “young women who have sex with women” – do not necessarily correspond as one might expect with sexual orientation or identity, which is one’s concept of their romantic or sexual feelings, attractions and desires, again reinforcing the importance of asking patients their identity and preferences.
In terms of gender, a person’s natal or biologic gender is the one assigned people at birth based on their body parts and hormones. Gender identity is a person’s understanding of their own gender, and gender expression refers to how someone acts or presents themselves and communicates their gender within their culture.
Those who identify as “gender nonconforming, genderqueer, gender fluid, or nonbinary” see their gender on a spectrum, not within the binary “male” or “female.” A cisgender person’s gender identity matches both their biological sex assigned at birth and conventional cultural norms, while a transgender person’s gender differs from the sex they were assigned at birth. Transgender women (male to female, MTF) and men (female to men, FTM) go through the process of transition, a time that can occur in weeks or years when they shift from living as one gender to another.
While it’s unclear what leads to a person’s sexual orientation – likely a combination of genetic, hormonal and environmental factors—there is no question that sexual orientation is not a “choice,” Dr Finger said. Research has also clarified that one’s sexual orientation does not result from parenting behaviors or a history of sexual abuse.
“But I would urge all of you, instead of focusing on why someone is LGBTQ, to focus on what that means for them in their life,” Dr Finger said. “How is this bearing out in terms of their relationships and their behaviors, and how do they feel about it? How are they being supported by their family or their community, and how is it impacting their lives?”
She cited findings from a Human Rights Campaign survey in 2012 of 10,000 youth aged 13-17, which found that most LGBTQ respondents became aware of their same-sex attraction at 9 years of age, though the average age of disclosures is 16, an improvement from age 21 in the 1980s.
How and what to ask
Although children start becoming conscious of gender at ages 1-2, their sense of gender usually stabilizes by age 4.
“Who should we be screening for gender nonconformity? Quite frankly, all children, because all of them have some gender identity, so we should be asking them about that,” Dr Finger said.
When children are younger, doctors can ask parents about their child’s social interactions, forms of play, dress preferences, and mood. Questions for patients themselves, adapted for their age, might include, “Do you feel more like a girl, boy, neither or both?”, “How would you like to play, cut your hair and dress?” And “What name or pronoun (he or she) fits you?”
While such conversations do not necessarily need to happen annually, doctors should especially ask youth who dress or behave in non–gender-conforming ways or who appear to have mood, behavior or social difficulties.
To understand a patient’s sexuality, ask whether they are attracted to people of their own gender or sex, a different gender or sex, both or all genders or no one, or if they’re not sure yet. Doctors can then ask how comfortable they are with their attraction and whether they have told family members or friends about them.
Sexual behavior questions should be developmentally appropriate and lead to counsel but not judgment, Dr Finger said. Her method, with adjustments for age and development, starts, “There are many way of being sexual or intimate with someone: kissing, hugging and touching, and oral sex, anal sex and vaginal sex. Have you ever had any of these experiences? Which ones? With males or females or both, or other genders?”
Then she gets more specific while remaining sensitive. Doctors can ask younger children if they have held hands or cuddled with someone, if they have kissed someone, or if they have touched another person’s private parts. They can ask teens about oral sex, vaginal sex and anal sex and then gather more details about what parts went where, which helps determine what screenings or treatment options a patient may need or desire.
Doctors can use their judgment about whether to ask questions with parents in the room or not, but as kids grow older, it’s good practice to speak to patients without their caregivers present. Doctors should also explain the rules of confidentiality to their patients and be aware of the risks of “coming out,” including family discord or rejection, problems at school or work, social stigma, bullying and harassment, physical violence and risk-taking behaviors, such as substance use, self-injury and risky sexual behaviors. A HEADSSS screen can help doctors learn if any of these are present.
Making your practice inclusive and welcoming
Fewer than one in five teens who are “out” as LGBTQ have come out to their doctor, Dr Finger cited. Most are out to their friends and classmates, and more than half are out to their family, but teens are less likely to tell their doctors.
Research suggests one reason for this is the fact that pediatricians often don’t ask. One study found that only 20% of pediatricians discussed sexual orientation with their patients (Pediatrics 2010 Apr;125:e741-7). Similarly, only 30% of family physicians brought up sexual orientation, found another study (Fam. Med. 2001 May;33[5]:376-81). The studies found physicians more often discussed condoms, HIV, sexually transmitted infections, abstinence, violence, contraception or, in the case of family physicians, sexual behaviors, and relationships.
But another reason for not being out to doctors is a history of poor experiences. A Lambda Legal Survey in 2009 of 4,916 LGBT respondents found that 8% of LGB and 27% of transgender and gender nonconforming patients had been denied care because of their identity of orientation. Eleven percent said “providers refused to touch them or used excessive precautions,” Dr Finger reported. LGBTQ patients may fear the doctor’s reaction or not keeping their identity confidential. Patients may also have internalized shame or guilt due to societal norms or homophobia, and all these barriers can reduce LGBTQ people’s willingness to seek and access to competent care.
The first step to making LGBTQ patients comfortable in your practice is to confront your own personal biases, Dr Finger said. Understand what they are and that a provider’s discomfort, even unconscious, can be damaging to the patient-provider relationship.
“If you find that this is just not something that you’re going to be comfortable doing, at the very least, I would suggest that you find providers in your area who are comfortable working with this patient population and you refer your patients to them so that they can have a good, trusting patient-provider relationship with somebody who can provide the care that they need,” Dr Finger said.
The next step is creating a safe place with zero tolerance for insensitivity by training staff to be welcoming and inclusive, assuring patients confidentiality, providing support and resources and displaying LGBTQ-affirming materials. These youth need active, visible evidence that the office will be a safe place for them.
Ways pediatricians can communicate an inclusive environment include having gender-neutral restrooms, using “parent” instead of “mother/father” and using forms and EMR prompts with gender-neutral language or multiple options for gender selection.
Screening and LGBTQ patients’ health needs
LGB youth and those who aren’t sure of their sexual orientation tend to have higher rates of substance use, including tobacco, alcohol and illicit drugs, and are more often victims of rape and other sexual violence. Their rates of depressive symptoms, bullying victimization, and suicidality are also significantly higher than in their heterosexual cisgender peers. Homelessness rates are also considerably higher in LGBTQ youth than in heterosexual cisgender youth.
One thing pediatricians can do is work with parents to ensure a patient’s school is meeting their needs. The greater risks LGBTQ youth typically face are mediated by social support, resiliency, supportive friends and family and a supportive school environment, including inclusive curricula and supportive staff.
Lesbian and bisexual women are considerably more at risk for poor sexual or reproductive outcomes, Dr Finger said. Their rates of unplanned pregnancy are double that of straight women, contributing to their higher rates of emergency contraception and abortion. They are also more likely to have more partners (male and females), to have a younger sexual debut and to be forced into sex by a male partner—yet they are far less likely to perceive themselves as at risk for a sexually transmitted infection than their peers.
This patient population therefore may need contraception counseling, including discussing their current methods and reviewing their options, including emergency contraception and possibly an advance prescription. Dr Finger also suggests having male and female condoms available in the office.
Doctors should screen all their female patients, regardless of sexuality, for chlamydia and gonorrhea, and offer routine cervical cancer screening and the HPV vaccine, as recommended by the CDC. They might consider screening for trichomoniasis, bacterial vaginosis, herpes simplex, human papillomavirus and HIV.
For men who have sex with men, the CDC recommends HIV and syphilis serology, urine/pharyngeal/rectal gonorrhea nucleic acid amplification test (NAAT), urine/rectal chlamydia NAAT, and hepatitis C screening for those who are HIV-positive—all at least once a year.
For transgender patients, doctors need to assess their STI- and HIV-related risks based on their current anatomy and sexual behaviors.
Doctors should also consider discussing pre-exposure prophylaxis (PrEP) for any youth at high risk for HIV infection if they are at least 77 pounds (35 kg). Emtricitabine/tenofovir (Truvada, Descovy) reduces the chance of sexually acquired infection by 99%, and infection acquired via drug injection by 74% when taken as prescribed.
Resources
Dr Finger noted a range of resources for LGBTQ youth and their families and providers, including the Family Acceptance Project, Gay and Lesbian Medical Association, Gay, Lesbian and Straight Education Network, GLBTQ Legal Advocates and Defenders (GLAD), Human Rights Campaign, It Gets Better Project, LGBTQ Student Resources and Support, National Center for Lesbian Rights, Parents and Friends of Lesbians and Gays (PFLAG), Safe Schools Coalition and The Trevor Project (concerning suicide risk).
NEW ORLEANS – One of the most important things pediatricians can do to support their lesbian, gay, bisexual, transgender (LGBT) and other gender-nonconforming patients is to ask all their patients about their feelings, preferences and experiences when it comes to gender and sexuality, according to Julie Finger, MD, MPH.
It’s equally important not to make assumptions, she told attendees at the annual meeting of the American Academy of Pediatrics. Biology and sexual and gender identity and expression can be very diverse, she said. Specifically, doctors should not assume patients are heterosexual, that bisexuality is a phase, that orientation or attraction translates directly to behavior or vice versa, or that LGBTQ patients have unsupportive families or are engaging in risky behavior. Research suggests LGB youth have slightly higher rates of early sexual debut, sexual activity or multiple partners than straight or uncertain youth, but only marginally so.
Pediatricians also cannot assume a patient’s sexual orientation based on their partner’s gender or determine a patient’s sexual orientation or gender identity based on appearance – or even that either is the same as it was on the previous visit.
What doctors can be sure of is that they do have LGBTQ patients, said Dr. Finger, and assistant professor of clinical pediatrics at Tulane University in New Orleans. According to a 2016 Morbidity and Mortality Weekly Report (2016 Aug 12; 65[9]), about 1 in 10 students in grades 9-12 are a sexual minority. About 2% of respondents identify as gay or lesbian, 6% identify as bisexual and 3% say they aren’t sure.
Knowing the terminology
Dr. Finger defined key terminology regarding gender and sexuality. She first clarified that LGBT is not the full spectrum for sexual orientation. Pansexual (fluid attraction to any sex or gender) and asexual (lack of feeling sexual attraction) can also describe sexuality, and the Q on the end of LGBTQ is often an umbrella term for “queer” or “questioning” that encompasses anyone who fits outside conventional social norms of sexual identity and gender expression.
Sexual behaviors – which include “young men who have sex with men” and “young women who have sex with women” – do not necessarily correspond as one might expect with sexual orientation or identity, which is one’s concept of their romantic or sexual feelings, attractions and desires, again reinforcing the importance of asking patients their identity and preferences.
In terms of gender, a person’s natal or biologic gender is the one assigned people at birth based on their body parts and hormones. Gender identity is a person’s understanding of their own gender, and gender expression refers to how someone acts or presents themselves and communicates their gender within their culture.
Those who identify as “gender nonconforming, genderqueer, gender fluid, or nonbinary” see their gender on a spectrum, not within the binary “male” or “female.” A cisgender person’s gender identity matches both their biological sex assigned at birth and conventional cultural norms, while a transgender person’s gender differs from the sex they were assigned at birth. Transgender women (male to female, MTF) and men (female to men, FTM) go through the process of transition, a time that can occur in weeks or years when they shift from living as one gender to another.
While it’s unclear what leads to a person’s sexual orientation – likely a combination of genetic, hormonal and environmental factors—there is no question that sexual orientation is not a “choice,” Dr Finger said. Research has also clarified that one’s sexual orientation does not result from parenting behaviors or a history of sexual abuse.
“But I would urge all of you, instead of focusing on why someone is LGBTQ, to focus on what that means for them in their life,” Dr Finger said. “How is this bearing out in terms of their relationships and their behaviors, and how do they feel about it? How are they being supported by their family or their community, and how is it impacting their lives?”
She cited findings from a Human Rights Campaign survey in 2012 of 10,000 youth aged 13-17, which found that most LGBTQ respondents became aware of their same-sex attraction at 9 years of age, though the average age of disclosures is 16, an improvement from age 21 in the 1980s.
How and what to ask
Although children start becoming conscious of gender at ages 1-2, their sense of gender usually stabilizes by age 4.
“Who should we be screening for gender nonconformity? Quite frankly, all children, because all of them have some gender identity, so we should be asking them about that,” Dr Finger said.
When children are younger, doctors can ask parents about their child’s social interactions, forms of play, dress preferences, and mood. Questions for patients themselves, adapted for their age, might include, “Do you feel more like a girl, boy, neither or both?”, “How would you like to play, cut your hair and dress?” And “What name or pronoun (he or she) fits you?”
While such conversations do not necessarily need to happen annually, doctors should especially ask youth who dress or behave in non–gender-conforming ways or who appear to have mood, behavior or social difficulties.
To understand a patient’s sexuality, ask whether they are attracted to people of their own gender or sex, a different gender or sex, both or all genders or no one, or if they’re not sure yet. Doctors can then ask how comfortable they are with their attraction and whether they have told family members or friends about them.
Sexual behavior questions should be developmentally appropriate and lead to counsel but not judgment, Dr Finger said. Her method, with adjustments for age and development, starts, “There are many way of being sexual or intimate with someone: kissing, hugging and touching, and oral sex, anal sex and vaginal sex. Have you ever had any of these experiences? Which ones? With males or females or both, or other genders?”
Then she gets more specific while remaining sensitive. Doctors can ask younger children if they have held hands or cuddled with someone, if they have kissed someone, or if they have touched another person’s private parts. They can ask teens about oral sex, vaginal sex and anal sex and then gather more details about what parts went where, which helps determine what screenings or treatment options a patient may need or desire.
Doctors can use their judgment about whether to ask questions with parents in the room or not, but as kids grow older, it’s good practice to speak to patients without their caregivers present. Doctors should also explain the rules of confidentiality to their patients and be aware of the risks of “coming out,” including family discord or rejection, problems at school or work, social stigma, bullying and harassment, physical violence and risk-taking behaviors, such as substance use, self-injury and risky sexual behaviors. A HEADSSS screen can help doctors learn if any of these are present.
Making your practice inclusive and welcoming
Fewer than one in five teens who are “out” as LGBTQ have come out to their doctor, Dr Finger cited. Most are out to their friends and classmates, and more than half are out to their family, but teens are less likely to tell their doctors.
Research suggests one reason for this is the fact that pediatricians often don’t ask. One study found that only 20% of pediatricians discussed sexual orientation with their patients (Pediatrics 2010 Apr;125:e741-7). Similarly, only 30% of family physicians brought up sexual orientation, found another study (Fam. Med. 2001 May;33[5]:376-81). The studies found physicians more often discussed condoms, HIV, sexually transmitted infections, abstinence, violence, contraception or, in the case of family physicians, sexual behaviors, and relationships.
But another reason for not being out to doctors is a history of poor experiences. A Lambda Legal Survey in 2009 of 4,916 LGBT respondents found that 8% of LGB and 27% of transgender and gender nonconforming patients had been denied care because of their identity of orientation. Eleven percent said “providers refused to touch them or used excessive precautions,” Dr Finger reported. LGBTQ patients may fear the doctor’s reaction or not keeping their identity confidential. Patients may also have internalized shame or guilt due to societal norms or homophobia, and all these barriers can reduce LGBTQ people’s willingness to seek and access to competent care.
The first step to making LGBTQ patients comfortable in your practice is to confront your own personal biases, Dr Finger said. Understand what they are and that a provider’s discomfort, even unconscious, can be damaging to the patient-provider relationship.
“If you find that this is just not something that you’re going to be comfortable doing, at the very least, I would suggest that you find providers in your area who are comfortable working with this patient population and you refer your patients to them so that they can have a good, trusting patient-provider relationship with somebody who can provide the care that they need,” Dr Finger said.
The next step is creating a safe place with zero tolerance for insensitivity by training staff to be welcoming and inclusive, assuring patients confidentiality, providing support and resources and displaying LGBTQ-affirming materials. These youth need active, visible evidence that the office will be a safe place for them.
Ways pediatricians can communicate an inclusive environment include having gender-neutral restrooms, using “parent” instead of “mother/father” and using forms and EMR prompts with gender-neutral language or multiple options for gender selection.
Screening and LGBTQ patients’ health needs
LGB youth and those who aren’t sure of their sexual orientation tend to have higher rates of substance use, including tobacco, alcohol and illicit drugs, and are more often victims of rape and other sexual violence. Their rates of depressive symptoms, bullying victimization, and suicidality are also significantly higher than in their heterosexual cisgender peers. Homelessness rates are also considerably higher in LGBTQ youth than in heterosexual cisgender youth.
One thing pediatricians can do is work with parents to ensure a patient’s school is meeting their needs. The greater risks LGBTQ youth typically face are mediated by social support, resiliency, supportive friends and family and a supportive school environment, including inclusive curricula and supportive staff.
Lesbian and bisexual women are considerably more at risk for poor sexual or reproductive outcomes, Dr Finger said. Their rates of unplanned pregnancy are double that of straight women, contributing to their higher rates of emergency contraception and abortion. They are also more likely to have more partners (male and females), to have a younger sexual debut and to be forced into sex by a male partner—yet they are far less likely to perceive themselves as at risk for a sexually transmitted infection than their peers.
This patient population therefore may need contraception counseling, including discussing their current methods and reviewing their options, including emergency contraception and possibly an advance prescription. Dr Finger also suggests having male and female condoms available in the office.
Doctors should screen all their female patients, regardless of sexuality, for chlamydia and gonorrhea, and offer routine cervical cancer screening and the HPV vaccine, as recommended by the CDC. They might consider screening for trichomoniasis, bacterial vaginosis, herpes simplex, human papillomavirus and HIV.
For men who have sex with men, the CDC recommends HIV and syphilis serology, urine/pharyngeal/rectal gonorrhea nucleic acid amplification test (NAAT), urine/rectal chlamydia NAAT, and hepatitis C screening for those who are HIV-positive—all at least once a year.
For transgender patients, doctors need to assess their STI- and HIV-related risks based on their current anatomy and sexual behaviors.
Doctors should also consider discussing pre-exposure prophylaxis (PrEP) for any youth at high risk for HIV infection if they are at least 77 pounds (35 kg). Emtricitabine/tenofovir (Truvada, Descovy) reduces the chance of sexually acquired infection by 99%, and infection acquired via drug injection by 74% when taken as prescribed.
Resources
Dr Finger noted a range of resources for LGBTQ youth and their families and providers, including the Family Acceptance Project, Gay and Lesbian Medical Association, Gay, Lesbian and Straight Education Network, GLBTQ Legal Advocates and Defenders (GLAD), Human Rights Campaign, It Gets Better Project, LGBTQ Student Resources and Support, National Center for Lesbian Rights, Parents and Friends of Lesbians and Gays (PFLAG), Safe Schools Coalition and The Trevor Project (concerning suicide risk).
EXPERT ANALYSIS FROM AAP 2019
Recurrent intussusception rare in young children
NEW ORLEANS – Recurrent intussusception after discharge may be far less common in young children than previously reported, and significant morbidity associated with the condition is rare, results from an analysis of national data show.
“Recurrent intussusception following successful nonoperative reduction has previously been reported with a frequency of 8-12% based on data from individual institutions,” researchers led by Anthony R. Ferrantella, MD, wrote in an abstract presented at the annual meeting of the American Academy of Pediatrics. “Timing of discharge following successful non-operative reduction continues to be debated, but recent studies suggest it is safe to discharge home from the emergency department. However, the practice of hospital admission for an observation period of 24-48 hours following successful nonoperative reduction persists today among many pediatric surgeons.”
In an effort to evaluate readmissions for recurrent intussusception in young children on a large scale, Dr. Ferrantella, a surgery resident at the University of Miami, and his colleagues queried the Nationwide Readmissions Database during 2010-2014 to identify children younger than 5 years of age diagnosed with ileocolic intussusception. They compared the management during index admission and frequency of readmissions for recurrent intussusception up to one year after discharge. They excluded patients lacking procedure data, weighted the results for national estimates, and used chi-square analysis to compare cohorts.
The search yielded 8,289 young children who were diagnosed with ileocolic intussusception during an index admission. Of these, 43% received definitive treatment with nonoperative reduction alone, 42% underwent surgical reduction without bowel resection, and 15% underwent surgery with bowel resection. Among the hospitals where patients were treated, 75% were large, 80% were not-for-profit, and 94% were metropolitan teaching hospitals.
The researchers found that readmission for recurrent intussusception was required for only 4% of patients managed with nonoperative reduction alone, 2% of patients who underwent surgical reduction, and 0% of those who underwent bowel resection. The median time to readmission was 4 days for those managed with nonoperative reduction only and 64 days for those managed with surgery.
Among patients managed with nonoperative reduction alone during index admission, 71% were again managed successfully with nonoperative reduction alone, 24 underwent surgical reduction, and only 5% required bowel resection. No deaths occurred during any readmissions.
The findings “suggest and support the idea that if you can successfully perform a nonoperative reduction on a child that comes in with an intussusception, you can safely discharge them,” Dr. Ferrantella said in an interview. “If you were to keep them in the hospital, the [rate] of recurrences are very low. Even when they do recur, only 30%-40% will happen within the first 24-48 hours, so the majority will not benefit from a hospital admission.”
He acknowledged certain limitations of the analysis, including that the data came from a retrospectively collected database and that he and his colleagues were unable to track readmissions across state lines.
Dr. Ferrantella reported having no financial disclosures.
SOURCE: Ferrantella A. AAP 2019, Section on Surgery session.
NEW ORLEANS – Recurrent intussusception after discharge may be far less common in young children than previously reported, and significant morbidity associated with the condition is rare, results from an analysis of national data show.
“Recurrent intussusception following successful nonoperative reduction has previously been reported with a frequency of 8-12% based on data from individual institutions,” researchers led by Anthony R. Ferrantella, MD, wrote in an abstract presented at the annual meeting of the American Academy of Pediatrics. “Timing of discharge following successful non-operative reduction continues to be debated, but recent studies suggest it is safe to discharge home from the emergency department. However, the practice of hospital admission for an observation period of 24-48 hours following successful nonoperative reduction persists today among many pediatric surgeons.”
In an effort to evaluate readmissions for recurrent intussusception in young children on a large scale, Dr. Ferrantella, a surgery resident at the University of Miami, and his colleagues queried the Nationwide Readmissions Database during 2010-2014 to identify children younger than 5 years of age diagnosed with ileocolic intussusception. They compared the management during index admission and frequency of readmissions for recurrent intussusception up to one year after discharge. They excluded patients lacking procedure data, weighted the results for national estimates, and used chi-square analysis to compare cohorts.
The search yielded 8,289 young children who were diagnosed with ileocolic intussusception during an index admission. Of these, 43% received definitive treatment with nonoperative reduction alone, 42% underwent surgical reduction without bowel resection, and 15% underwent surgery with bowel resection. Among the hospitals where patients were treated, 75% were large, 80% were not-for-profit, and 94% were metropolitan teaching hospitals.
The researchers found that readmission for recurrent intussusception was required for only 4% of patients managed with nonoperative reduction alone, 2% of patients who underwent surgical reduction, and 0% of those who underwent bowel resection. The median time to readmission was 4 days for those managed with nonoperative reduction only and 64 days for those managed with surgery.
Among patients managed with nonoperative reduction alone during index admission, 71% were again managed successfully with nonoperative reduction alone, 24 underwent surgical reduction, and only 5% required bowel resection. No deaths occurred during any readmissions.
The findings “suggest and support the idea that if you can successfully perform a nonoperative reduction on a child that comes in with an intussusception, you can safely discharge them,” Dr. Ferrantella said in an interview. “If you were to keep them in the hospital, the [rate] of recurrences are very low. Even when they do recur, only 30%-40% will happen within the first 24-48 hours, so the majority will not benefit from a hospital admission.”
He acknowledged certain limitations of the analysis, including that the data came from a retrospectively collected database and that he and his colleagues were unable to track readmissions across state lines.
Dr. Ferrantella reported having no financial disclosures.
SOURCE: Ferrantella A. AAP 2019, Section on Surgery session.
NEW ORLEANS – Recurrent intussusception after discharge may be far less common in young children than previously reported, and significant morbidity associated with the condition is rare, results from an analysis of national data show.
“Recurrent intussusception following successful nonoperative reduction has previously been reported with a frequency of 8-12% based on data from individual institutions,” researchers led by Anthony R. Ferrantella, MD, wrote in an abstract presented at the annual meeting of the American Academy of Pediatrics. “Timing of discharge following successful non-operative reduction continues to be debated, but recent studies suggest it is safe to discharge home from the emergency department. However, the practice of hospital admission for an observation period of 24-48 hours following successful nonoperative reduction persists today among many pediatric surgeons.”
In an effort to evaluate readmissions for recurrent intussusception in young children on a large scale, Dr. Ferrantella, a surgery resident at the University of Miami, and his colleagues queried the Nationwide Readmissions Database during 2010-2014 to identify children younger than 5 years of age diagnosed with ileocolic intussusception. They compared the management during index admission and frequency of readmissions for recurrent intussusception up to one year after discharge. They excluded patients lacking procedure data, weighted the results for national estimates, and used chi-square analysis to compare cohorts.
The search yielded 8,289 young children who were diagnosed with ileocolic intussusception during an index admission. Of these, 43% received definitive treatment with nonoperative reduction alone, 42% underwent surgical reduction without bowel resection, and 15% underwent surgery with bowel resection. Among the hospitals where patients were treated, 75% were large, 80% were not-for-profit, and 94% were metropolitan teaching hospitals.
The researchers found that readmission for recurrent intussusception was required for only 4% of patients managed with nonoperative reduction alone, 2% of patients who underwent surgical reduction, and 0% of those who underwent bowel resection. The median time to readmission was 4 days for those managed with nonoperative reduction only and 64 days for those managed with surgery.
Among patients managed with nonoperative reduction alone during index admission, 71% were again managed successfully with nonoperative reduction alone, 24 underwent surgical reduction, and only 5% required bowel resection. No deaths occurred during any readmissions.
The findings “suggest and support the idea that if you can successfully perform a nonoperative reduction on a child that comes in with an intussusception, you can safely discharge them,” Dr. Ferrantella said in an interview. “If you were to keep them in the hospital, the [rate] of recurrences are very low. Even when they do recur, only 30%-40% will happen within the first 24-48 hours, so the majority will not benefit from a hospital admission.”
He acknowledged certain limitations of the analysis, including that the data came from a retrospectively collected database and that he and his colleagues were unable to track readmissions across state lines.
Dr. Ferrantella reported having no financial disclosures.
SOURCE: Ferrantella A. AAP 2019, Section on Surgery session.
AT AAP 2016
Wandering is underrecognized, serious problem for autistic children
NEW ORLEANS – Nearly half of all children with autism spectrum disorder wander off from safe supervision at some point in their childhood or adolescence, reported Paul Lipkin, MD, at the annual meeting of the American Academy of Pediatrics.
Though such behavior is developmentally normal in toddlers, it’s rarer for older children to leave a supervised, safe space for a longer period than just running away for a bit, he said.
Far more than an inconvenience, wandering, also called elopement, puts these children at high risk for injury or victimization. In fact, statistics from a survey by the National Autism Foundation suggest that nearly a third of autism-related wandering cases resulted in death or serious enough injury to require medical attention, said Dr. Lipkin, an associate professor of pediatrics at the Kennedy Krieger Institute and Johns Hopkins Medicine in Baltimore.
“Drowning is overwhelmingly the main cause of death in children with autism,” he said, sharing the data from National Autism Association, which relied on parent report and media reports. In that data, 71% of deaths from autistic children who wandered from 2011-2016 were drowning, and of those deaths, 76% of the drownings occurred in a natural body of water or drainage water. At a distant second, 18% of deaths were traffic accidents. The remaining causes were being hit by a train (4%), hypothermia or hyperthermia (3%), falling (1%) or other trauma (3%) (J Autism Dev Disord. 2019 Mar 5. doi: 10.1007/s10803-019-03961-x).
Academic research has found similar statistics to those from the National Autism Association. In one study, 53% of autistic youth who attempted to run off succeeded and were missing long enough to cause safety concerns (Pediatrics. 2012 Nov;130[5]:870-7). Among these youth – representing about a quarter of all families surveyed in the study – the police were called in 31% of cases. In addition, 65% had a “close call” with a traffic injury and 24% had a close call with drowning.
The children wandered off in various settings, including home; another’s home; a store or other public place; or school, daycare or camp. A 2019 study found that 70% of parents reported their children wandering off from home at least once in the past 2 years (J Autism Dev Disorders. 2019 Mar 5; doi: 10.1107/s10803-019-03961-x).
Although most cases occur in children and teens, with the highest rate of death among children aged 5-9, the National Autism Association has received reports of wandering occur throughout autistic people’s lifetime.
Yet this issue doesn’t appear to be on the radar of many pediatricians, and those who are aware of it may not know the best strategies to share with parents to prevent wandering and subsequent injury, Dr Lipkin explained. In one study, only one-third of parents reported receiving guidance from a health provider related to wandering (J Dev Behav Pediatr. 2018 Sep;39[7]:538-46).
That research found that only 10% received advice from a pediatrician or other primary care provider, 12% received advice from a developmental pediatrician or neurologist and 10% received advance from a psychologist or psychiatrist. The largest source of guidance in that study was autism advocacy organizations, whom 22% of parents cited. Others included a teacher or other school staff member (15%), a personal contact (13%), law enforcement (8%) or another source (1%).
Role of the pediatrician
Pediatricians have an important role to play in prevention of elopement, Dr Lipkin said. They can screen autistic patients for wandering and elopement during visits, work with community stakeholders such as schools and law enforcement, advocate for awareness, and provider education and resources for families.
Perhaps the most valuable resource, he said, is the Big Red Safety Box, available from the National Autism Association. This resource, sponsored by more than a half dozen autism advocacy organizations, includes three digital safety toolkits: one for caregivers, one for first responders, and one for teachers. Parents can therefore share the toolkits for first responders and teachers with those respective community members.
Pediatricians can also help families develop a Family Wandering Emergency Plan (FWEP), a template for which is in the Big Red Safety Box. Parents and community members should know the steps to take if someone wanders: Stay calm, call 911, search nearby water first and then implement the FWEP.
It’s first helpful to understand why these youth wander off. In the National Autism Association survey, the most common reasons were to escape an anxious situation, particularly for those with Asperger’s, or simply to run, explore, or go to a favorite place, particularly among those with autism or pervasive developmental disorder-not otherwise specified (PDD-NOS).
Researchers have found similar reasons: 43% of elopement situations occurred when children were trying to escape an anxious situation, 39% left while in a stressful environment, and 24% were in an environment with conflict, found one study (J Autism Dev Disord. 2019 Mar 5. doi: 10.1007/s10803-019-03961-x).
Sensory overload was also a trigger, with 38% of elopements occurring when it was too noisy, and 34% when it was a generally uncomfortable sensory experience. Just over a quarter (27%) of children left when they were understimulated or in a “boring” environment, Dr Lipkin reported. The remaining reason was goal-directed: 27% left to pursue a special interest, 18% sought a place where they enjoyed playing, and 11% were after their favorite food.
Prevention Strategies
Most data about effective strategies to prevent wandering comes from research that relies on parents, Dr Lipkin said. In general, environmental interventions tend to be the most effective, and medication tends to be the least effective.
One study on elopement prevention found that 96% of caregivers use at least some type of intervention, and the vast majority (83%) were using environmental interventions such as dead bolts (51%), latches (49%) and gates (36%). An equal proportion used behavioral services (83%), such as a behavioral psychologist (41%), social stories (40%) or an aide (39%). Just under a third used an ID bracelet or shoe tag (31%), and 19% used GPS trackers, according to Dr. Lipkin.
Although parents reported environmental interventions to be very effective, 68% said they were highly burdensome, though the median cost over 2 years was less than $1,000. The least expensive intervention was home behavioral specialists (when covered by insurance) and school aides, and the most expensive and burdensome – albeit highly effective – was a service animal.
Interventions with the least cost effectiveness included security cameras and GPS trackers, which only 15% of parents reported as being effective.
Although nearly half of parents reported their child had taken any psychiatric medication (48%), only 16% had taken medication explicitly to prevent wandering. Few reported the medication was very effective, however. Among the small number who did (less than 10), lorazepam, diazepam and atomoxetine appeared best.
Teaching children survival skills, as developmentally appropriate and possible, can also help. These include swimming lessons as well as learning how to interact in traffic, knowing their home address, and learning how to navigate around their neighborhood.
Dr. Lipkin no disclosures and used no external funding for this presentation.
NEW ORLEANS – Nearly half of all children with autism spectrum disorder wander off from safe supervision at some point in their childhood or adolescence, reported Paul Lipkin, MD, at the annual meeting of the American Academy of Pediatrics.
Though such behavior is developmentally normal in toddlers, it’s rarer for older children to leave a supervised, safe space for a longer period than just running away for a bit, he said.
Far more than an inconvenience, wandering, also called elopement, puts these children at high risk for injury or victimization. In fact, statistics from a survey by the National Autism Foundation suggest that nearly a third of autism-related wandering cases resulted in death or serious enough injury to require medical attention, said Dr. Lipkin, an associate professor of pediatrics at the Kennedy Krieger Institute and Johns Hopkins Medicine in Baltimore.
“Drowning is overwhelmingly the main cause of death in children with autism,” he said, sharing the data from National Autism Association, which relied on parent report and media reports. In that data, 71% of deaths from autistic children who wandered from 2011-2016 were drowning, and of those deaths, 76% of the drownings occurred in a natural body of water or drainage water. At a distant second, 18% of deaths were traffic accidents. The remaining causes were being hit by a train (4%), hypothermia or hyperthermia (3%), falling (1%) or other trauma (3%) (J Autism Dev Disord. 2019 Mar 5. doi: 10.1007/s10803-019-03961-x).
Academic research has found similar statistics to those from the National Autism Association. In one study, 53% of autistic youth who attempted to run off succeeded and were missing long enough to cause safety concerns (Pediatrics. 2012 Nov;130[5]:870-7). Among these youth – representing about a quarter of all families surveyed in the study – the police were called in 31% of cases. In addition, 65% had a “close call” with a traffic injury and 24% had a close call with drowning.
The children wandered off in various settings, including home; another’s home; a store or other public place; or school, daycare or camp. A 2019 study found that 70% of parents reported their children wandering off from home at least once in the past 2 years (J Autism Dev Disorders. 2019 Mar 5; doi: 10.1107/s10803-019-03961-x).
Although most cases occur in children and teens, with the highest rate of death among children aged 5-9, the National Autism Association has received reports of wandering occur throughout autistic people’s lifetime.
Yet this issue doesn’t appear to be on the radar of many pediatricians, and those who are aware of it may not know the best strategies to share with parents to prevent wandering and subsequent injury, Dr Lipkin explained. In one study, only one-third of parents reported receiving guidance from a health provider related to wandering (J Dev Behav Pediatr. 2018 Sep;39[7]:538-46).
That research found that only 10% received advice from a pediatrician or other primary care provider, 12% received advice from a developmental pediatrician or neurologist and 10% received advance from a psychologist or psychiatrist. The largest source of guidance in that study was autism advocacy organizations, whom 22% of parents cited. Others included a teacher or other school staff member (15%), a personal contact (13%), law enforcement (8%) or another source (1%).
Role of the pediatrician
Pediatricians have an important role to play in prevention of elopement, Dr Lipkin said. They can screen autistic patients for wandering and elopement during visits, work with community stakeholders such as schools and law enforcement, advocate for awareness, and provider education and resources for families.
Perhaps the most valuable resource, he said, is the Big Red Safety Box, available from the National Autism Association. This resource, sponsored by more than a half dozen autism advocacy organizations, includes three digital safety toolkits: one for caregivers, one for first responders, and one for teachers. Parents can therefore share the toolkits for first responders and teachers with those respective community members.
Pediatricians can also help families develop a Family Wandering Emergency Plan (FWEP), a template for which is in the Big Red Safety Box. Parents and community members should know the steps to take if someone wanders: Stay calm, call 911, search nearby water first and then implement the FWEP.
It’s first helpful to understand why these youth wander off. In the National Autism Association survey, the most common reasons were to escape an anxious situation, particularly for those with Asperger’s, or simply to run, explore, or go to a favorite place, particularly among those with autism or pervasive developmental disorder-not otherwise specified (PDD-NOS).
Researchers have found similar reasons: 43% of elopement situations occurred when children were trying to escape an anxious situation, 39% left while in a stressful environment, and 24% were in an environment with conflict, found one study (J Autism Dev Disord. 2019 Mar 5. doi: 10.1007/s10803-019-03961-x).
Sensory overload was also a trigger, with 38% of elopements occurring when it was too noisy, and 34% when it was a generally uncomfortable sensory experience. Just over a quarter (27%) of children left when they were understimulated or in a “boring” environment, Dr Lipkin reported. The remaining reason was goal-directed: 27% left to pursue a special interest, 18% sought a place where they enjoyed playing, and 11% were after their favorite food.
Prevention Strategies
Most data about effective strategies to prevent wandering comes from research that relies on parents, Dr Lipkin said. In general, environmental interventions tend to be the most effective, and medication tends to be the least effective.
One study on elopement prevention found that 96% of caregivers use at least some type of intervention, and the vast majority (83%) were using environmental interventions such as dead bolts (51%), latches (49%) and gates (36%). An equal proportion used behavioral services (83%), such as a behavioral psychologist (41%), social stories (40%) or an aide (39%). Just under a third used an ID bracelet or shoe tag (31%), and 19% used GPS trackers, according to Dr. Lipkin.
Although parents reported environmental interventions to be very effective, 68% said they were highly burdensome, though the median cost over 2 years was less than $1,000. The least expensive intervention was home behavioral specialists (when covered by insurance) and school aides, and the most expensive and burdensome – albeit highly effective – was a service animal.
Interventions with the least cost effectiveness included security cameras and GPS trackers, which only 15% of parents reported as being effective.
Although nearly half of parents reported their child had taken any psychiatric medication (48%), only 16% had taken medication explicitly to prevent wandering. Few reported the medication was very effective, however. Among the small number who did (less than 10), lorazepam, diazepam and atomoxetine appeared best.
Teaching children survival skills, as developmentally appropriate and possible, can also help. These include swimming lessons as well as learning how to interact in traffic, knowing their home address, and learning how to navigate around their neighborhood.
Dr. Lipkin no disclosures and used no external funding for this presentation.
NEW ORLEANS – Nearly half of all children with autism spectrum disorder wander off from safe supervision at some point in their childhood or adolescence, reported Paul Lipkin, MD, at the annual meeting of the American Academy of Pediatrics.
Though such behavior is developmentally normal in toddlers, it’s rarer for older children to leave a supervised, safe space for a longer period than just running away for a bit, he said.
Far more than an inconvenience, wandering, also called elopement, puts these children at high risk for injury or victimization. In fact, statistics from a survey by the National Autism Foundation suggest that nearly a third of autism-related wandering cases resulted in death or serious enough injury to require medical attention, said Dr. Lipkin, an associate professor of pediatrics at the Kennedy Krieger Institute and Johns Hopkins Medicine in Baltimore.
“Drowning is overwhelmingly the main cause of death in children with autism,” he said, sharing the data from National Autism Association, which relied on parent report and media reports. In that data, 71% of deaths from autistic children who wandered from 2011-2016 were drowning, and of those deaths, 76% of the drownings occurred in a natural body of water or drainage water. At a distant second, 18% of deaths were traffic accidents. The remaining causes were being hit by a train (4%), hypothermia or hyperthermia (3%), falling (1%) or other trauma (3%) (J Autism Dev Disord. 2019 Mar 5. doi: 10.1007/s10803-019-03961-x).
Academic research has found similar statistics to those from the National Autism Association. In one study, 53% of autistic youth who attempted to run off succeeded and were missing long enough to cause safety concerns (Pediatrics. 2012 Nov;130[5]:870-7). Among these youth – representing about a quarter of all families surveyed in the study – the police were called in 31% of cases. In addition, 65% had a “close call” with a traffic injury and 24% had a close call with drowning.
The children wandered off in various settings, including home; another’s home; a store or other public place; or school, daycare or camp. A 2019 study found that 70% of parents reported their children wandering off from home at least once in the past 2 years (J Autism Dev Disorders. 2019 Mar 5; doi: 10.1107/s10803-019-03961-x).
Although most cases occur in children and teens, with the highest rate of death among children aged 5-9, the National Autism Association has received reports of wandering occur throughout autistic people’s lifetime.
Yet this issue doesn’t appear to be on the radar of many pediatricians, and those who are aware of it may not know the best strategies to share with parents to prevent wandering and subsequent injury, Dr Lipkin explained. In one study, only one-third of parents reported receiving guidance from a health provider related to wandering (J Dev Behav Pediatr. 2018 Sep;39[7]:538-46).
That research found that only 10% received advice from a pediatrician or other primary care provider, 12% received advice from a developmental pediatrician or neurologist and 10% received advance from a psychologist or psychiatrist. The largest source of guidance in that study was autism advocacy organizations, whom 22% of parents cited. Others included a teacher or other school staff member (15%), a personal contact (13%), law enforcement (8%) or another source (1%).
Role of the pediatrician
Pediatricians have an important role to play in prevention of elopement, Dr Lipkin said. They can screen autistic patients for wandering and elopement during visits, work with community stakeholders such as schools and law enforcement, advocate for awareness, and provider education and resources for families.
Perhaps the most valuable resource, he said, is the Big Red Safety Box, available from the National Autism Association. This resource, sponsored by more than a half dozen autism advocacy organizations, includes three digital safety toolkits: one for caregivers, one for first responders, and one for teachers. Parents can therefore share the toolkits for first responders and teachers with those respective community members.
Pediatricians can also help families develop a Family Wandering Emergency Plan (FWEP), a template for which is in the Big Red Safety Box. Parents and community members should know the steps to take if someone wanders: Stay calm, call 911, search nearby water first and then implement the FWEP.
It’s first helpful to understand why these youth wander off. In the National Autism Association survey, the most common reasons were to escape an anxious situation, particularly for those with Asperger’s, or simply to run, explore, or go to a favorite place, particularly among those with autism or pervasive developmental disorder-not otherwise specified (PDD-NOS).
Researchers have found similar reasons: 43% of elopement situations occurred when children were trying to escape an anxious situation, 39% left while in a stressful environment, and 24% were in an environment with conflict, found one study (J Autism Dev Disord. 2019 Mar 5. doi: 10.1007/s10803-019-03961-x).
Sensory overload was also a trigger, with 38% of elopements occurring when it was too noisy, and 34% when it was a generally uncomfortable sensory experience. Just over a quarter (27%) of children left when they were understimulated or in a “boring” environment, Dr Lipkin reported. The remaining reason was goal-directed: 27% left to pursue a special interest, 18% sought a place where they enjoyed playing, and 11% were after their favorite food.
Prevention Strategies
Most data about effective strategies to prevent wandering comes from research that relies on parents, Dr Lipkin said. In general, environmental interventions tend to be the most effective, and medication tends to be the least effective.
One study on elopement prevention found that 96% of caregivers use at least some type of intervention, and the vast majority (83%) were using environmental interventions such as dead bolts (51%), latches (49%) and gates (36%). An equal proportion used behavioral services (83%), such as a behavioral psychologist (41%), social stories (40%) or an aide (39%). Just under a third used an ID bracelet or shoe tag (31%), and 19% used GPS trackers, according to Dr. Lipkin.
Although parents reported environmental interventions to be very effective, 68% said they were highly burdensome, though the median cost over 2 years was less than $1,000. The least expensive intervention was home behavioral specialists (when covered by insurance) and school aides, and the most expensive and burdensome – albeit highly effective – was a service animal.
Interventions with the least cost effectiveness included security cameras and GPS trackers, which only 15% of parents reported as being effective.
Although nearly half of parents reported their child had taken any psychiatric medication (48%), only 16% had taken medication explicitly to prevent wandering. Few reported the medication was very effective, however. Among the small number who did (less than 10), lorazepam, diazepam and atomoxetine appeared best.
Teaching children survival skills, as developmentally appropriate and possible, can also help. These include swimming lessons as well as learning how to interact in traffic, knowing their home address, and learning how to navigate around their neighborhood.
Dr. Lipkin no disclosures and used no external funding for this presentation.
FROM AAP
Urban or rural, children’s gun-related injuries equally deadly
NEW ORLEANS – Accidental firearm-related injuries among children occur more frequently in rural than in urban locations, and nearly 60% of such cases are potentially preventable, results from a single-center study suggest.
Furthermore, these gun injuries carry the same mortality and disability risk.
“Firearm-related injury is an understudied topic,” lead study author Amelia Lucisano, MD, said in an interview in advance of the annual meeting of the American Academy of Pediatrics. “In particular there is a lack of granular level research on firearm-related injury in the population.”
At the meeting, she presented findings from an analysis which set out to investigate the location, preventability, and temporal trends of pediatric firearm-related injury in 184 patients age 18 and younger who were treated in the pediatric trauma program at University of Pittsburgh Medical Center during 2008-2017. Dr. Lucisano, a surgical resident at the university, and her colleagues focused their work on efforts to illustrate the differences and similarities in the demographics, injury-related characteristics, and outcomes between the rural and urban populations of children who are injured by firearms in Southwestern Pennsylvania. They classified the location as rural if the injury occurred outside the region’s central metropolitan county, and classified the injury as potentially preventable if the firearm was not stored securely and was used without permission. Statistical analyses included Wilcoxon rank-sum and chi-square analyses.
Of the 184 children who sustained a firearm-related injury during the study period, 43% occurred in a rural location. Compared with children who were injured in an urban setting, those who were injured in a rural setting were younger (a mean of 13 vs. 14 years; P = 0.0003), were more frequently white (81% vs. 14%; P less than 0.0001), and were more frequently injured by accident (70% vs. 15%; P less than 0.0001). They were also more likely to be injured by rifle or shotgun (24.1% vs. 6.67%; P = 0.001).
The rates of death or disability and lengths of stay did not differ significantly based on location of injury, occurring in 16.5% of rural and 13.3% of urban patients.
Nearly three-quarters of accidental injuries (72%) occurred on the gun-owner’s property and 58% were considered by the researchers to be potentially preventable.
“As expected, rural injuries are more frequently unintentional while urban injuries are more frequently assaults,” Dr. Lucisano said. “However, markers of injury severity and outcomes are equivalent between the groups, meaning that morbidity and mortality of injuries in the rural setting are similar to those in the urban setting.”
She emphasized that while clinician bias may be to consider rural firearm-based injuries as less severe, “our study shows that they carry the same burden of morbidity and mortality as urban injuries and thus should be cared for with the same intensity and anticipation of a possible poor outcome. Furthermore, the large number of potentially preventable injuries among those that were unintentional represents a significant burden of morbidity and mortality that could have been avoided through safer firearm storage. Programs to promote safe firearm storage should be targeted to populations that have high rates of potentially preventable injuries.”
Dr. Lucisano and her colleagues observed that the rates of all forms of firearm-related injury appear to be on the rise in both rural and urban areas: accidental, self-inflicted, and assault, in particular. She acknowledged certain limitations of the study, including its retrospective, single-center design. “We did not capture children who died in the field or who were treated at other hospitals, though as our center is the only pediatric Level 1 trauma center, we capture a large majority of pediatric trauma patients in the region,” she said.
The researchers reported having no disclosures.
SOURCE: Lucisano A. AAP 2019, Section on Surgery program.
NEW ORLEANS – Accidental firearm-related injuries among children occur more frequently in rural than in urban locations, and nearly 60% of such cases are potentially preventable, results from a single-center study suggest.
Furthermore, these gun injuries carry the same mortality and disability risk.
“Firearm-related injury is an understudied topic,” lead study author Amelia Lucisano, MD, said in an interview in advance of the annual meeting of the American Academy of Pediatrics. “In particular there is a lack of granular level research on firearm-related injury in the population.”
At the meeting, she presented findings from an analysis which set out to investigate the location, preventability, and temporal trends of pediatric firearm-related injury in 184 patients age 18 and younger who were treated in the pediatric trauma program at University of Pittsburgh Medical Center during 2008-2017. Dr. Lucisano, a surgical resident at the university, and her colleagues focused their work on efforts to illustrate the differences and similarities in the demographics, injury-related characteristics, and outcomes between the rural and urban populations of children who are injured by firearms in Southwestern Pennsylvania. They classified the location as rural if the injury occurred outside the region’s central metropolitan county, and classified the injury as potentially preventable if the firearm was not stored securely and was used without permission. Statistical analyses included Wilcoxon rank-sum and chi-square analyses.
Of the 184 children who sustained a firearm-related injury during the study period, 43% occurred in a rural location. Compared with children who were injured in an urban setting, those who were injured in a rural setting were younger (a mean of 13 vs. 14 years; P = 0.0003), were more frequently white (81% vs. 14%; P less than 0.0001), and were more frequently injured by accident (70% vs. 15%; P less than 0.0001). They were also more likely to be injured by rifle or shotgun (24.1% vs. 6.67%; P = 0.001).
The rates of death or disability and lengths of stay did not differ significantly based on location of injury, occurring in 16.5% of rural and 13.3% of urban patients.
Nearly three-quarters of accidental injuries (72%) occurred on the gun-owner’s property and 58% were considered by the researchers to be potentially preventable.
“As expected, rural injuries are more frequently unintentional while urban injuries are more frequently assaults,” Dr. Lucisano said. “However, markers of injury severity and outcomes are equivalent between the groups, meaning that morbidity and mortality of injuries in the rural setting are similar to those in the urban setting.”
She emphasized that while clinician bias may be to consider rural firearm-based injuries as less severe, “our study shows that they carry the same burden of morbidity and mortality as urban injuries and thus should be cared for with the same intensity and anticipation of a possible poor outcome. Furthermore, the large number of potentially preventable injuries among those that were unintentional represents a significant burden of morbidity and mortality that could have been avoided through safer firearm storage. Programs to promote safe firearm storage should be targeted to populations that have high rates of potentially preventable injuries.”
Dr. Lucisano and her colleagues observed that the rates of all forms of firearm-related injury appear to be on the rise in both rural and urban areas: accidental, self-inflicted, and assault, in particular. She acknowledged certain limitations of the study, including its retrospective, single-center design. “We did not capture children who died in the field or who were treated at other hospitals, though as our center is the only pediatric Level 1 trauma center, we capture a large majority of pediatric trauma patients in the region,” she said.
The researchers reported having no disclosures.
SOURCE: Lucisano A. AAP 2019, Section on Surgery program.
NEW ORLEANS – Accidental firearm-related injuries among children occur more frequently in rural than in urban locations, and nearly 60% of such cases are potentially preventable, results from a single-center study suggest.
Furthermore, these gun injuries carry the same mortality and disability risk.
“Firearm-related injury is an understudied topic,” lead study author Amelia Lucisano, MD, said in an interview in advance of the annual meeting of the American Academy of Pediatrics. “In particular there is a lack of granular level research on firearm-related injury in the population.”
At the meeting, she presented findings from an analysis which set out to investigate the location, preventability, and temporal trends of pediatric firearm-related injury in 184 patients age 18 and younger who were treated in the pediatric trauma program at University of Pittsburgh Medical Center during 2008-2017. Dr. Lucisano, a surgical resident at the university, and her colleagues focused their work on efforts to illustrate the differences and similarities in the demographics, injury-related characteristics, and outcomes between the rural and urban populations of children who are injured by firearms in Southwestern Pennsylvania. They classified the location as rural if the injury occurred outside the region’s central metropolitan county, and classified the injury as potentially preventable if the firearm was not stored securely and was used without permission. Statistical analyses included Wilcoxon rank-sum and chi-square analyses.
Of the 184 children who sustained a firearm-related injury during the study period, 43% occurred in a rural location. Compared with children who were injured in an urban setting, those who were injured in a rural setting were younger (a mean of 13 vs. 14 years; P = 0.0003), were more frequently white (81% vs. 14%; P less than 0.0001), and were more frequently injured by accident (70% vs. 15%; P less than 0.0001). They were also more likely to be injured by rifle or shotgun (24.1% vs. 6.67%; P = 0.001).
The rates of death or disability and lengths of stay did not differ significantly based on location of injury, occurring in 16.5% of rural and 13.3% of urban patients.
Nearly three-quarters of accidental injuries (72%) occurred on the gun-owner’s property and 58% were considered by the researchers to be potentially preventable.
“As expected, rural injuries are more frequently unintentional while urban injuries are more frequently assaults,” Dr. Lucisano said. “However, markers of injury severity and outcomes are equivalent between the groups, meaning that morbidity and mortality of injuries in the rural setting are similar to those in the urban setting.”
She emphasized that while clinician bias may be to consider rural firearm-based injuries as less severe, “our study shows that they carry the same burden of morbidity and mortality as urban injuries and thus should be cared for with the same intensity and anticipation of a possible poor outcome. Furthermore, the large number of potentially preventable injuries among those that were unintentional represents a significant burden of morbidity and mortality that could have been avoided through safer firearm storage. Programs to promote safe firearm storage should be targeted to populations that have high rates of potentially preventable injuries.”
Dr. Lucisano and her colleagues observed that the rates of all forms of firearm-related injury appear to be on the rise in both rural and urban areas: accidental, self-inflicted, and assault, in particular. She acknowledged certain limitations of the study, including its retrospective, single-center design. “We did not capture children who died in the field or who were treated at other hospitals, though as our center is the only pediatric Level 1 trauma center, we capture a large majority of pediatric trauma patients in the region,” she said.
The researchers reported having no disclosures.
SOURCE: Lucisano A. AAP 2019, Section on Surgery program.
AT AAP 2019