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Anabasum shows promise in treating skin-predominant dermatomyositis, systemic sclerosis
SAN DIEGO – Results from two phase 2 studies of the investigational agent anabasum provide evidence supporting its safety and efficacy in patients with refractory skin-predominant dermatomyositis or diffuse cutaneous systemic sclerosis.
Anabasum (JBT-101) is a nonimmunosuppressive, synthetic, oral cannabinoid receptor type 2 agonist being developed by Norwood, Mass.–based Corbus Pharmaceuticals. It works by triggering resolution of innate immune responses, said Barbara White, MD, a rheumatologist who is chief medical officer for Corbus. “It restores homeostasis, gets rid of inflammation, turns off active fibrotic processes, and helps clear bacteria if it is present – all without immunosuppression,” she explained in an interview at the annual meeting of the American College of Rheumatology. “A drug that would restore homeostasis in the setting of an ongoing immune response has the potential to be helpful in multiple autoimmune diseases.”
The mean age of the study participants was 53 years and most were white. Even though 19 of the patients were on immunosuppressants at baseline, both cohorts had mean CDASI scores in the 33-35 range, which can be considered severe.
The investigators, led by Victoria P. Werth, MD, of the University of Pennsylvania, Philadelphia, reported that anabasum-treated subjects experienced a medically meaningful improvement in mean CDASI scores, with a reduction of at least 5 points at all visits after week 4 and reaching –9.3 at the end of the study, compared with –3.7 points in the placebo group (P = .02). They also found that 56% of subjects in the anabasum group had a 10-point reduction or more in CDASI score, compared with only 18% in the placebo group (P = .09). In addition, no subjects in the anabasum group developed skin erosions during the active dosing period, compared with 36% of subjects in the placebo group (P = .05).
The researchers also observed that, compared with subjects in the placebo group, those in the anabasum group had greater improvement in patient-reported global skin disease and overall disease assessments, skin symptoms (including photosensitivity and itch), fatigue, sleep, interference with activities, pain, and physical function. No serious, severe, or unexpected adverse events occurred in the anabasum group. Adverse events included dizziness, dyspepsia, headache, and increased appetite.
There is not a Food and Drug Administration or European Medicines Agency–approved drug for refractory skin-predominant dermatomyositis, “so this is encouraging,” Dr. White said.
Dr. Spiera reported that patients in the anabasum group had greater improvement in ACR CRISS, compared with placebo-treated subjects over 16 weeks (P = .044). They also had greater improvement and less worsening in individual CRISS core measures, including modified Rodnan Skin Score, Patient Global Assessment, Physician Global Assessment, and the Health Assessment Questionnaire Disability Index. Patient-reported outcomes of systemic sclerosis skin symptoms, itch, and the Patient-Reported Outcomes Measurement Information System–29 (PROMIS-29) domains of physical function, pain interference, and sleep also improved for the anabasum group, compared with the placebo group (P less than .05 for all).
An analysis of paired skin biopsies before and after treatment with the assessor blinded to treatment assignment demonstrated that patients treated with anabasum were more likely to show improvement in fibrosis and inflammation and less likely to show worsening than were those treated with placebo, consistent with what was observed clinically. In a related poster presented at the meeting, gene expression analysis from specimens before and after treatment also revealed that anabasum treatment (as opposed to treatment with placebo) was associated with changes relevant to pathways involved in fibrosis and inflammation.
“This is the first double-blind, randomized, placebo controlled trial in diffuse cutaneous systemic sclerosis to demonstrate a clinical benefit using the CRISS as an endpoint, with a drug that was safe and well tolerated in the trial,” Dr. Spiera said. “These results bring hope to patients and their physicians that anabasum may be an effective drug for systemic sclerosis where currently there are no proven treatments.”
Both studies were sponsored by Corbus, and the dermatomyositis study was also sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Some of the investigators in both studies were employees of Corbus. Dr. Spiera reported receiving research support from Corbus and many other pharmaceutical companies. He also is a consultant to Roche-Genentech, GlaxoSmithKline, Boehringer Ingelheim, and CSL Behring. He is a member of the Rheumatology News editorial advisory board.
SAN DIEGO – Results from two phase 2 studies of the investigational agent anabasum provide evidence supporting its safety and efficacy in patients with refractory skin-predominant dermatomyositis or diffuse cutaneous systemic sclerosis.
Anabasum (JBT-101) is a nonimmunosuppressive, synthetic, oral cannabinoid receptor type 2 agonist being developed by Norwood, Mass.–based Corbus Pharmaceuticals. It works by triggering resolution of innate immune responses, said Barbara White, MD, a rheumatologist who is chief medical officer for Corbus. “It restores homeostasis, gets rid of inflammation, turns off active fibrotic processes, and helps clear bacteria if it is present – all without immunosuppression,” she explained in an interview at the annual meeting of the American College of Rheumatology. “A drug that would restore homeostasis in the setting of an ongoing immune response has the potential to be helpful in multiple autoimmune diseases.”
The mean age of the study participants was 53 years and most were white. Even though 19 of the patients were on immunosuppressants at baseline, both cohorts had mean CDASI scores in the 33-35 range, which can be considered severe.
The investigators, led by Victoria P. Werth, MD, of the University of Pennsylvania, Philadelphia, reported that anabasum-treated subjects experienced a medically meaningful improvement in mean CDASI scores, with a reduction of at least 5 points at all visits after week 4 and reaching –9.3 at the end of the study, compared with –3.7 points in the placebo group (P = .02). They also found that 56% of subjects in the anabasum group had a 10-point reduction or more in CDASI score, compared with only 18% in the placebo group (P = .09). In addition, no subjects in the anabasum group developed skin erosions during the active dosing period, compared with 36% of subjects in the placebo group (P = .05).
The researchers also observed that, compared with subjects in the placebo group, those in the anabasum group had greater improvement in patient-reported global skin disease and overall disease assessments, skin symptoms (including photosensitivity and itch), fatigue, sleep, interference with activities, pain, and physical function. No serious, severe, or unexpected adverse events occurred in the anabasum group. Adverse events included dizziness, dyspepsia, headache, and increased appetite.
There is not a Food and Drug Administration or European Medicines Agency–approved drug for refractory skin-predominant dermatomyositis, “so this is encouraging,” Dr. White said.
Dr. Spiera reported that patients in the anabasum group had greater improvement in ACR CRISS, compared with placebo-treated subjects over 16 weeks (P = .044). They also had greater improvement and less worsening in individual CRISS core measures, including modified Rodnan Skin Score, Patient Global Assessment, Physician Global Assessment, and the Health Assessment Questionnaire Disability Index. Patient-reported outcomes of systemic sclerosis skin symptoms, itch, and the Patient-Reported Outcomes Measurement Information System–29 (PROMIS-29) domains of physical function, pain interference, and sleep also improved for the anabasum group, compared with the placebo group (P less than .05 for all).
An analysis of paired skin biopsies before and after treatment with the assessor blinded to treatment assignment demonstrated that patients treated with anabasum were more likely to show improvement in fibrosis and inflammation and less likely to show worsening than were those treated with placebo, consistent with what was observed clinically. In a related poster presented at the meeting, gene expression analysis from specimens before and after treatment also revealed that anabasum treatment (as opposed to treatment with placebo) was associated with changes relevant to pathways involved in fibrosis and inflammation.
“This is the first double-blind, randomized, placebo controlled trial in diffuse cutaneous systemic sclerosis to demonstrate a clinical benefit using the CRISS as an endpoint, with a drug that was safe and well tolerated in the trial,” Dr. Spiera said. “These results bring hope to patients and their physicians that anabasum may be an effective drug for systemic sclerosis where currently there are no proven treatments.”
Both studies were sponsored by Corbus, and the dermatomyositis study was also sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Some of the investigators in both studies were employees of Corbus. Dr. Spiera reported receiving research support from Corbus and many other pharmaceutical companies. He also is a consultant to Roche-Genentech, GlaxoSmithKline, Boehringer Ingelheim, and CSL Behring. He is a member of the Rheumatology News editorial advisory board.
SAN DIEGO – Results from two phase 2 studies of the investigational agent anabasum provide evidence supporting its safety and efficacy in patients with refractory skin-predominant dermatomyositis or diffuse cutaneous systemic sclerosis.
Anabasum (JBT-101) is a nonimmunosuppressive, synthetic, oral cannabinoid receptor type 2 agonist being developed by Norwood, Mass.–based Corbus Pharmaceuticals. It works by triggering resolution of innate immune responses, said Barbara White, MD, a rheumatologist who is chief medical officer for Corbus. “It restores homeostasis, gets rid of inflammation, turns off active fibrotic processes, and helps clear bacteria if it is present – all without immunosuppression,” she explained in an interview at the annual meeting of the American College of Rheumatology. “A drug that would restore homeostasis in the setting of an ongoing immune response has the potential to be helpful in multiple autoimmune diseases.”
The mean age of the study participants was 53 years and most were white. Even though 19 of the patients were on immunosuppressants at baseline, both cohorts had mean CDASI scores in the 33-35 range, which can be considered severe.
The investigators, led by Victoria P. Werth, MD, of the University of Pennsylvania, Philadelphia, reported that anabasum-treated subjects experienced a medically meaningful improvement in mean CDASI scores, with a reduction of at least 5 points at all visits after week 4 and reaching –9.3 at the end of the study, compared with –3.7 points in the placebo group (P = .02). They also found that 56% of subjects in the anabasum group had a 10-point reduction or more in CDASI score, compared with only 18% in the placebo group (P = .09). In addition, no subjects in the anabasum group developed skin erosions during the active dosing period, compared with 36% of subjects in the placebo group (P = .05).
The researchers also observed that, compared with subjects in the placebo group, those in the anabasum group had greater improvement in patient-reported global skin disease and overall disease assessments, skin symptoms (including photosensitivity and itch), fatigue, sleep, interference with activities, pain, and physical function. No serious, severe, or unexpected adverse events occurred in the anabasum group. Adverse events included dizziness, dyspepsia, headache, and increased appetite.
There is not a Food and Drug Administration or European Medicines Agency–approved drug for refractory skin-predominant dermatomyositis, “so this is encouraging,” Dr. White said.
Dr. Spiera reported that patients in the anabasum group had greater improvement in ACR CRISS, compared with placebo-treated subjects over 16 weeks (P = .044). They also had greater improvement and less worsening in individual CRISS core measures, including modified Rodnan Skin Score, Patient Global Assessment, Physician Global Assessment, and the Health Assessment Questionnaire Disability Index. Patient-reported outcomes of systemic sclerosis skin symptoms, itch, and the Patient-Reported Outcomes Measurement Information System–29 (PROMIS-29) domains of physical function, pain interference, and sleep also improved for the anabasum group, compared with the placebo group (P less than .05 for all).
An analysis of paired skin biopsies before and after treatment with the assessor blinded to treatment assignment demonstrated that patients treated with anabasum were more likely to show improvement in fibrosis and inflammation and less likely to show worsening than were those treated with placebo, consistent with what was observed clinically. In a related poster presented at the meeting, gene expression analysis from specimens before and after treatment also revealed that anabasum treatment (as opposed to treatment with placebo) was associated with changes relevant to pathways involved in fibrosis and inflammation.
“This is the first double-blind, randomized, placebo controlled trial in diffuse cutaneous systemic sclerosis to demonstrate a clinical benefit using the CRISS as an endpoint, with a drug that was safe and well tolerated in the trial,” Dr. Spiera said. “These results bring hope to patients and their physicians that anabasum may be an effective drug for systemic sclerosis where currently there are no proven treatments.”
Both studies were sponsored by Corbus, and the dermatomyositis study was also sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Some of the investigators in both studies were employees of Corbus. Dr. Spiera reported receiving research support from Corbus and many other pharmaceutical companies. He also is a consultant to Roche-Genentech, GlaxoSmithKline, Boehringer Ingelheim, and CSL Behring. He is a member of the Rheumatology News editorial advisory board.
AT ACR 2017
Key clinical point: Anabasum had a favorable safety profile and was well tolerated in patients with either refractory skin-predominant dermatomyositis or diffuse cutaneous systemic sclerosis.
Major finding: Treatment with anabasum resulted in greater improvement in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) score at 12 weeks, compared with placebo (–9.3 vs. –3.7 points, respectively; P = .02) and also in the ACR Combined Response Index in Systemic Sclerosis (CRISS) at 16 weeks, compared with placebo (P = .044).
Study details: Two 12-week, phase 2 studies of anabasum in patients with dermatomyositis or systemic sclerosis.
Disclosures: The studies were funded by Corbus, and the dermatomyositis study was additionally supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Dr. Spiera reported receiving research support from Corbus and many other pharmaceutical companies. He is a consultant to Roche-Genentech, GlaxoSmithKline, Boehringer Ingelheim, and CSL Behring. Some of the investigators in both studies were employees of Corbus.
Race may transcend social, geographical parameters in lupus mortality
SAN DIEGO – Blacks with systemic lupus erythematosus (SLE) who share the same social and geographic contexts as whites with the disease were disproportionately more likely to die young and to show severe patterns of mortality, according to a study of death certificate data.
“One of the most salient aspects of the epidemiology of lupus is the predilection of the disease for women and racial minorities,” lead study author Titilola Falasinnu, PhD, said at the annual meeting of the American College of Rheumatology.
Dr. Falasinnu, a postdoctoral fellow in Stanford (Calif.) University’s department of health research and policy, and her associates examined SLE mortality across eight groups of race-county combinations published in 2006 and known as the “Eight Americas” (PLoS Med. 2006 Sep 12;3[9]:e260). This seminal work, which has been validated across multiple disease states, jointly characterized race, socioeconomic status, and geographical location in relation to health disparities in the United States to demonstrate the most important factors accounting for these disparities within the Eight Americas.
For the current analysis, Dr. Falasinnu and her associates were most interested in America 6 (black middle America), America 7 (Southern low-income rural blacks), and America 8 (high-risk urban blacks). “The question we wanted to ask is whether, on average, poorer individuals have more severe SLE mortality experiences, compared with richer individuals in the black community,” she said. “What happens when you hold race constant and you vary socioeconomic indices?” Using death certificate data from the National Center for Health Statistics Multiple Causes of Death database, they identified SLE-related deaths between 2003 and 2014. Next, they compared these data with mortality statistics from each of the Eight Americas.
In all, there were nearly 25,000 SLE-related deaths, of which 85% were female. More than one-third of deaths occurred among those aged 45-64 years, and the mean age at death was 57 years. More than half of deaths (64%) occurred among whites, and 31% among blacks. Among SLE decedents, northern rural whites in America 2 had the lowest mortality rates. Blacks in America 6, 7, and 8 had the highest mortality, yet there were no significant differences in the death rates among those three groups. “We found that in general, blacks were three times more likely to die with SLE, compared with those in America 3 [middle America],” Dr. Falasinnu said.
Asians, Native Americans, and blacks with SLE died at an average age of 48-49 years, regardless of their social context, while Northern whites had the highest life expectancy: an average age of 69 years. They also found that 17% of SLE deaths in America 2 occurred before the age of 50 years, compared with more than 50% in Americas 6, 7, and 8. The most frequently reported associated causes of death were cardiovascular disease (about 50% of all SLE-related deaths) and kidney manifestations (about 20% of all SLE-related deaths). Compared with those in America 3, racial minorities had a 23%-53% higher risk of infections, a 5%-64% higher risk of kidney disease, a 7%-23% lower risk of cardiovascular disease, and a 20%-52% lower risk of cancers.
“Although blacks inhabited three vastly different geographical and social contexts, SLE mortality parameters did not vary among socially advantaged and disadvantaged black Americas,” Dr. Falasinnu concluded. “Blacks sharing the same social and geographical contexts as whites were disproportionately more likely to die young and exhibit patterns of mortality associated with active SLE disease.”
She acknowledged certain limitations of the study, including that differences in the degree of underreporting on death certificates across racial groups could bias the results. “The Eight Americas framework does not allow for evaluation of ethnicity,” she added. “We were also unable to examine causes for the disparities in SLE mortality. One could argue that there are a lot of other social factors that are likely race related that are not necessarily captured by the Eight Americas. Also, as with most epidemiological studies, we cannot rule out the role that ecological fallacy may play where the population average may not be appropriate in estimating an individual’s risk of mortality.”
One of the study coauthors reported receiving partial salary support through the Dr. Elaine Lambert Lupus Foundation via the John and Marcia Goldman Foundation and previously receiving salary support through a lupus-related grant from the Genomics Institute of the Novartis Research Foundation. The other coauthors reported having no relevant disclosures.
[email protected]
SAN DIEGO – Blacks with systemic lupus erythematosus (SLE) who share the same social and geographic contexts as whites with the disease were disproportionately more likely to die young and to show severe patterns of mortality, according to a study of death certificate data.
“One of the most salient aspects of the epidemiology of lupus is the predilection of the disease for women and racial minorities,” lead study author Titilola Falasinnu, PhD, said at the annual meeting of the American College of Rheumatology.
Dr. Falasinnu, a postdoctoral fellow in Stanford (Calif.) University’s department of health research and policy, and her associates examined SLE mortality across eight groups of race-county combinations published in 2006 and known as the “Eight Americas” (PLoS Med. 2006 Sep 12;3[9]:e260). This seminal work, which has been validated across multiple disease states, jointly characterized race, socioeconomic status, and geographical location in relation to health disparities in the United States to demonstrate the most important factors accounting for these disparities within the Eight Americas.
For the current analysis, Dr. Falasinnu and her associates were most interested in America 6 (black middle America), America 7 (Southern low-income rural blacks), and America 8 (high-risk urban blacks). “The question we wanted to ask is whether, on average, poorer individuals have more severe SLE mortality experiences, compared with richer individuals in the black community,” she said. “What happens when you hold race constant and you vary socioeconomic indices?” Using death certificate data from the National Center for Health Statistics Multiple Causes of Death database, they identified SLE-related deaths between 2003 and 2014. Next, they compared these data with mortality statistics from each of the Eight Americas.
In all, there were nearly 25,000 SLE-related deaths, of which 85% were female. More than one-third of deaths occurred among those aged 45-64 years, and the mean age at death was 57 years. More than half of deaths (64%) occurred among whites, and 31% among blacks. Among SLE decedents, northern rural whites in America 2 had the lowest mortality rates. Blacks in America 6, 7, and 8 had the highest mortality, yet there were no significant differences in the death rates among those three groups. “We found that in general, blacks were three times more likely to die with SLE, compared with those in America 3 [middle America],” Dr. Falasinnu said.
Asians, Native Americans, and blacks with SLE died at an average age of 48-49 years, regardless of their social context, while Northern whites had the highest life expectancy: an average age of 69 years. They also found that 17% of SLE deaths in America 2 occurred before the age of 50 years, compared with more than 50% in Americas 6, 7, and 8. The most frequently reported associated causes of death were cardiovascular disease (about 50% of all SLE-related deaths) and kidney manifestations (about 20% of all SLE-related deaths). Compared with those in America 3, racial minorities had a 23%-53% higher risk of infections, a 5%-64% higher risk of kidney disease, a 7%-23% lower risk of cardiovascular disease, and a 20%-52% lower risk of cancers.
“Although blacks inhabited three vastly different geographical and social contexts, SLE mortality parameters did not vary among socially advantaged and disadvantaged black Americas,” Dr. Falasinnu concluded. “Blacks sharing the same social and geographical contexts as whites were disproportionately more likely to die young and exhibit patterns of mortality associated with active SLE disease.”
She acknowledged certain limitations of the study, including that differences in the degree of underreporting on death certificates across racial groups could bias the results. “The Eight Americas framework does not allow for evaluation of ethnicity,” she added. “We were also unable to examine causes for the disparities in SLE mortality. One could argue that there are a lot of other social factors that are likely race related that are not necessarily captured by the Eight Americas. Also, as with most epidemiological studies, we cannot rule out the role that ecological fallacy may play where the population average may not be appropriate in estimating an individual’s risk of mortality.”
One of the study coauthors reported receiving partial salary support through the Dr. Elaine Lambert Lupus Foundation via the John and Marcia Goldman Foundation and previously receiving salary support through a lupus-related grant from the Genomics Institute of the Novartis Research Foundation. The other coauthors reported having no relevant disclosures.
[email protected]
SAN DIEGO – Blacks with systemic lupus erythematosus (SLE) who share the same social and geographic contexts as whites with the disease were disproportionately more likely to die young and to show severe patterns of mortality, according to a study of death certificate data.
“One of the most salient aspects of the epidemiology of lupus is the predilection of the disease for women and racial minorities,” lead study author Titilola Falasinnu, PhD, said at the annual meeting of the American College of Rheumatology.
Dr. Falasinnu, a postdoctoral fellow in Stanford (Calif.) University’s department of health research and policy, and her associates examined SLE mortality across eight groups of race-county combinations published in 2006 and known as the “Eight Americas” (PLoS Med. 2006 Sep 12;3[9]:e260). This seminal work, which has been validated across multiple disease states, jointly characterized race, socioeconomic status, and geographical location in relation to health disparities in the United States to demonstrate the most important factors accounting for these disparities within the Eight Americas.
For the current analysis, Dr. Falasinnu and her associates were most interested in America 6 (black middle America), America 7 (Southern low-income rural blacks), and America 8 (high-risk urban blacks). “The question we wanted to ask is whether, on average, poorer individuals have more severe SLE mortality experiences, compared with richer individuals in the black community,” she said. “What happens when you hold race constant and you vary socioeconomic indices?” Using death certificate data from the National Center for Health Statistics Multiple Causes of Death database, they identified SLE-related deaths between 2003 and 2014. Next, they compared these data with mortality statistics from each of the Eight Americas.
In all, there were nearly 25,000 SLE-related deaths, of which 85% were female. More than one-third of deaths occurred among those aged 45-64 years, and the mean age at death was 57 years. More than half of deaths (64%) occurred among whites, and 31% among blacks. Among SLE decedents, northern rural whites in America 2 had the lowest mortality rates. Blacks in America 6, 7, and 8 had the highest mortality, yet there were no significant differences in the death rates among those three groups. “We found that in general, blacks were three times more likely to die with SLE, compared with those in America 3 [middle America],” Dr. Falasinnu said.
Asians, Native Americans, and blacks with SLE died at an average age of 48-49 years, regardless of their social context, while Northern whites had the highest life expectancy: an average age of 69 years. They also found that 17% of SLE deaths in America 2 occurred before the age of 50 years, compared with more than 50% in Americas 6, 7, and 8. The most frequently reported associated causes of death were cardiovascular disease (about 50% of all SLE-related deaths) and kidney manifestations (about 20% of all SLE-related deaths). Compared with those in America 3, racial minorities had a 23%-53% higher risk of infections, a 5%-64% higher risk of kidney disease, a 7%-23% lower risk of cardiovascular disease, and a 20%-52% lower risk of cancers.
“Although blacks inhabited three vastly different geographical and social contexts, SLE mortality parameters did not vary among socially advantaged and disadvantaged black Americas,” Dr. Falasinnu concluded. “Blacks sharing the same social and geographical contexts as whites were disproportionately more likely to die young and exhibit patterns of mortality associated with active SLE disease.”
She acknowledged certain limitations of the study, including that differences in the degree of underreporting on death certificates across racial groups could bias the results. “The Eight Americas framework does not allow for evaluation of ethnicity,” she added. “We were also unable to examine causes for the disparities in SLE mortality. One could argue that there are a lot of other social factors that are likely race related that are not necessarily captured by the Eight Americas. Also, as with most epidemiological studies, we cannot rule out the role that ecological fallacy may play where the population average may not be appropriate in estimating an individual’s risk of mortality.”
One of the study coauthors reported receiving partial salary support through the Dr. Elaine Lambert Lupus Foundation via the John and Marcia Goldman Foundation and previously receiving salary support through a lupus-related grant from the Genomics Institute of the Novartis Research Foundation. The other coauthors reported having no relevant disclosures.
[email protected]
AT ACR 2017
Key clinical point: Blacks sharing the same social and geographical contexts as whites were more likely to die young and exhibit patterns of mortality associated with active SLE disease.
Major finding: Blacks in three race-geographic contexts were about three times more likely than whites in middle America to die with SLE.
Study details: An analysis of nearly 25,000 SLE-related deaths from the National Center for Health Statistics Multiple Causes of Death database.
Disclosures: One of the study coauthors reported receiving partial salary support through the Dr. Elaine Lambert Lupus Foundation via the John and Marcia Goldman Foundation and previously receiving salary support through a lupus-related grant from the Genomics Institute of the Novartis Research Foundation. The other coauthors reported having no relevant disclosures.
Obesity linked to pain, fatigue in SLE
SAN DIEGO – A new study offers a double message about the potential impact of obesity on systemic lupus erythematosus (SLE) in women: Excess pounds are linked to a higher risk of patient-reported outcomes such as pain and fatigue, and body mass index may be an appropriate tool to study weight issues in this population.
Researchers found “a strong relationship between body composition and worse outcomes,” Sarah Patterson, MD, a fellow in rheumatology at the University of California, San Francisco, and the lead study author, said at the annual meeting of the American College of Rheumatology.
For the new study, Dr. Patterson and her colleagues analyzed findings from surveys of 148 participants in the Arthritis Body Composition and Disability study. All participants were women with a verified SLE diagnosis.
About two-thirds of the sample were white, 14% were Asian, and 13% were African American. The average age was 48 years, the average disease duration was 16 years, and 45% took glucocorticoids.
Researchers used two measurements of obesity: BMI of 30 kg/m2 or greater and fat mass index (FMI) of 13 kg/m2 or greater.
They calculated FMI with data collected via whole dual x-ray absorptiometry. Of the participants, 32% and 30% met criteria for obesity under FMI and BMI definitions, respectively.
Researchers also collected survey data regarding measurements of disease activity, depressive symptoms, pain and fatigue.
The study authors controlled their results to account for factors such as age, race, and prednisone use. They found that those defined as obese via FMI had more disease activity and depression than did nonobese women: 14.8 versus 11.5, P = .010, on the Systemic Lupus Activity Questionnaire scale, and 19.8 versus 13.1, P = .004, on the Center for Epidemiologic Studies Depression scale.
On two other scales of pain and fatigue, obese patients scored lower – a sign of worse status – compared with nonobese women: 38.7 versus 44.2, P = .004, on the Short Form 36 (SF-36) Health Survey pain subscale and 39.6 versus 45.2, P = .010, on the SF-36 vitality subscale. The researchers reported similar findings when using BMI to assess obesity.
It’s not clear why obesity and lupus may be linked, Dr. Patterson said, though she noted that inflammation is a shared factor. “People with lupus have arthritis and chronic pain, so there may be this vicious feedback cycle with hindrances to be able to live healthy lifestyles,” she added.
The study has limitations, including that the sample is largely white, while lupus is more common among minority women. In addition, the study does not include underweight patients or track patients over time. “It will be important to look at obesity and patient-reported outcomes to determine whether weight loss results in better outcomes,” Dr. Patterson said.
The study does provide an extra benefit by suggesting that BMI is not an inferior tool to measure the effects of obesity in the SLE population, Dr. Patterson said. BMI has been criticized as a misleading measurement of obesity. But the BMI and FMI measures produced similar results in this study. “That’s really good news in a way for the practicalities of using this information,” she said.
But FMI may still be a better measurement of obesity in the general population, where BMI may be more likely to be thrown off by high muscle mass.
It may seem obvious that obesity is linked to worse lupus outcomes, but rheumatologist Bryant England, MD, of the University of Nebraska, Omaha, said that this research is noteworthy because it highlights the importance of focusing on obesity in the clinic.
Rheumatologists shouldn’t leave obesity to primary care physicians but instead confront it themselves, said Dr. England, who moderated a discussion of new research at an ACR annual meeting press conference. But he cautioned that prudence is especially important when talking about obesity with lupus patients because they may be sensitive about medication-related weight gain.
Dr. Patterson and the other study authors reported having no relevant disclosures. Dr. England also reported no relevant disclosures. The study was funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
SAN DIEGO – A new study offers a double message about the potential impact of obesity on systemic lupus erythematosus (SLE) in women: Excess pounds are linked to a higher risk of patient-reported outcomes such as pain and fatigue, and body mass index may be an appropriate tool to study weight issues in this population.
Researchers found “a strong relationship between body composition and worse outcomes,” Sarah Patterson, MD, a fellow in rheumatology at the University of California, San Francisco, and the lead study author, said at the annual meeting of the American College of Rheumatology.
For the new study, Dr. Patterson and her colleagues analyzed findings from surveys of 148 participants in the Arthritis Body Composition and Disability study. All participants were women with a verified SLE diagnosis.
About two-thirds of the sample were white, 14% were Asian, and 13% were African American. The average age was 48 years, the average disease duration was 16 years, and 45% took glucocorticoids.
Researchers used two measurements of obesity: BMI of 30 kg/m2 or greater and fat mass index (FMI) of 13 kg/m2 or greater.
They calculated FMI with data collected via whole dual x-ray absorptiometry. Of the participants, 32% and 30% met criteria for obesity under FMI and BMI definitions, respectively.
Researchers also collected survey data regarding measurements of disease activity, depressive symptoms, pain and fatigue.
The study authors controlled their results to account for factors such as age, race, and prednisone use. They found that those defined as obese via FMI had more disease activity and depression than did nonobese women: 14.8 versus 11.5, P = .010, on the Systemic Lupus Activity Questionnaire scale, and 19.8 versus 13.1, P = .004, on the Center for Epidemiologic Studies Depression scale.
On two other scales of pain and fatigue, obese patients scored lower – a sign of worse status – compared with nonobese women: 38.7 versus 44.2, P = .004, on the Short Form 36 (SF-36) Health Survey pain subscale and 39.6 versus 45.2, P = .010, on the SF-36 vitality subscale. The researchers reported similar findings when using BMI to assess obesity.
It’s not clear why obesity and lupus may be linked, Dr. Patterson said, though she noted that inflammation is a shared factor. “People with lupus have arthritis and chronic pain, so there may be this vicious feedback cycle with hindrances to be able to live healthy lifestyles,” she added.
The study has limitations, including that the sample is largely white, while lupus is more common among minority women. In addition, the study does not include underweight patients or track patients over time. “It will be important to look at obesity and patient-reported outcomes to determine whether weight loss results in better outcomes,” Dr. Patterson said.
The study does provide an extra benefit by suggesting that BMI is not an inferior tool to measure the effects of obesity in the SLE population, Dr. Patterson said. BMI has been criticized as a misleading measurement of obesity. But the BMI and FMI measures produced similar results in this study. “That’s really good news in a way for the practicalities of using this information,” she said.
But FMI may still be a better measurement of obesity in the general population, where BMI may be more likely to be thrown off by high muscle mass.
It may seem obvious that obesity is linked to worse lupus outcomes, but rheumatologist Bryant England, MD, of the University of Nebraska, Omaha, said that this research is noteworthy because it highlights the importance of focusing on obesity in the clinic.
Rheumatologists shouldn’t leave obesity to primary care physicians but instead confront it themselves, said Dr. England, who moderated a discussion of new research at an ACR annual meeting press conference. But he cautioned that prudence is especially important when talking about obesity with lupus patients because they may be sensitive about medication-related weight gain.
Dr. Patterson and the other study authors reported having no relevant disclosures. Dr. England also reported no relevant disclosures. The study was funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
SAN DIEGO – A new study offers a double message about the potential impact of obesity on systemic lupus erythematosus (SLE) in women: Excess pounds are linked to a higher risk of patient-reported outcomes such as pain and fatigue, and body mass index may be an appropriate tool to study weight issues in this population.
Researchers found “a strong relationship between body composition and worse outcomes,” Sarah Patterson, MD, a fellow in rheumatology at the University of California, San Francisco, and the lead study author, said at the annual meeting of the American College of Rheumatology.
For the new study, Dr. Patterson and her colleagues analyzed findings from surveys of 148 participants in the Arthritis Body Composition and Disability study. All participants were women with a verified SLE diagnosis.
About two-thirds of the sample were white, 14% were Asian, and 13% were African American. The average age was 48 years, the average disease duration was 16 years, and 45% took glucocorticoids.
Researchers used two measurements of obesity: BMI of 30 kg/m2 or greater and fat mass index (FMI) of 13 kg/m2 or greater.
They calculated FMI with data collected via whole dual x-ray absorptiometry. Of the participants, 32% and 30% met criteria for obesity under FMI and BMI definitions, respectively.
Researchers also collected survey data regarding measurements of disease activity, depressive symptoms, pain and fatigue.
The study authors controlled their results to account for factors such as age, race, and prednisone use. They found that those defined as obese via FMI had more disease activity and depression than did nonobese women: 14.8 versus 11.5, P = .010, on the Systemic Lupus Activity Questionnaire scale, and 19.8 versus 13.1, P = .004, on the Center for Epidemiologic Studies Depression scale.
On two other scales of pain and fatigue, obese patients scored lower – a sign of worse status – compared with nonobese women: 38.7 versus 44.2, P = .004, on the Short Form 36 (SF-36) Health Survey pain subscale and 39.6 versus 45.2, P = .010, on the SF-36 vitality subscale. The researchers reported similar findings when using BMI to assess obesity.
It’s not clear why obesity and lupus may be linked, Dr. Patterson said, though she noted that inflammation is a shared factor. “People with lupus have arthritis and chronic pain, so there may be this vicious feedback cycle with hindrances to be able to live healthy lifestyles,” she added.
The study has limitations, including that the sample is largely white, while lupus is more common among minority women. In addition, the study does not include underweight patients or track patients over time. “It will be important to look at obesity and patient-reported outcomes to determine whether weight loss results in better outcomes,” Dr. Patterson said.
The study does provide an extra benefit by suggesting that BMI is not an inferior tool to measure the effects of obesity in the SLE population, Dr. Patterson said. BMI has been criticized as a misleading measurement of obesity. But the BMI and FMI measures produced similar results in this study. “That’s really good news in a way for the practicalities of using this information,” she said.
But FMI may still be a better measurement of obesity in the general population, where BMI may be more likely to be thrown off by high muscle mass.
It may seem obvious that obesity is linked to worse lupus outcomes, but rheumatologist Bryant England, MD, of the University of Nebraska, Omaha, said that this research is noteworthy because it highlights the importance of focusing on obesity in the clinic.
Rheumatologists shouldn’t leave obesity to primary care physicians but instead confront it themselves, said Dr. England, who moderated a discussion of new research at an ACR annual meeting press conference. But he cautioned that prudence is especially important when talking about obesity with lupus patients because they may be sensitive about medication-related weight gain.
Dr. Patterson and the other study authors reported having no relevant disclosures. Dr. England also reported no relevant disclosures. The study was funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
AT ACR 2017
Key clinical point:
Major finding: Obese women with SLE had more disease activity than did nonobese women (14.8 versus 11.5, P = .010).
Data source: An analysis of 148 SLE patients (65% white, mean age 48, about 31% obese) with obesity measured by body mass index or fat mass index.
Disclosures: The study authors reported having no relevant disclosures. The National Institute of Arthritis and Musculoskeletal and Skin Diseases funded the study.
VIDEO: A challenging case of lichen planus
LAS VEGAS – For challenging cases of oral or cutaneous lichen planus, bullous pemphigoid, or lupus, Miriam S. Bettencourt, MD, recommends thinking outside the box and considering off-label treatments.
At the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar, Dr. Bettencourt discussed such cases, including a series of patients with oral lichen planus who improved with apremilast, an oral phosphodiesterase 4 inhibitor approved for psoriasis.
In a video interview at the meeting, she described one of those patients, a 73-year-old woman with mouth ulcers who was diagnosed with oral lichen planus. Multiple topical and oral therapies proved unsuccessful, and her condition was eventually controlled with apremilast, and the patient is doing well, “with occasional flares,” said Dr. Bettencourt, of the University of Nevada, Las Vegas.
She described this case in her annual presentation at the meeting, titled “Great Cases From the Las Vegas Dermatology Society.”
Dr. Bettencourt disclosed relationships with multiple companies including AbbVie, Aclaris, Celgene, IntraDerm, Pfizer, Promium, Sun Pharma, and Valeant.
SDEF and this news organization are owned by the same parent company.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
LAS VEGAS – For challenging cases of oral or cutaneous lichen planus, bullous pemphigoid, or lupus, Miriam S. Bettencourt, MD, recommends thinking outside the box and considering off-label treatments.
At the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar, Dr. Bettencourt discussed such cases, including a series of patients with oral lichen planus who improved with apremilast, an oral phosphodiesterase 4 inhibitor approved for psoriasis.
In a video interview at the meeting, she described one of those patients, a 73-year-old woman with mouth ulcers who was diagnosed with oral lichen planus. Multiple topical and oral therapies proved unsuccessful, and her condition was eventually controlled with apremilast, and the patient is doing well, “with occasional flares,” said Dr. Bettencourt, of the University of Nevada, Las Vegas.
She described this case in her annual presentation at the meeting, titled “Great Cases From the Las Vegas Dermatology Society.”
Dr. Bettencourt disclosed relationships with multiple companies including AbbVie, Aclaris, Celgene, IntraDerm, Pfizer, Promium, Sun Pharma, and Valeant.
SDEF and this news organization are owned by the same parent company.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
LAS VEGAS – For challenging cases of oral or cutaneous lichen planus, bullous pemphigoid, or lupus, Miriam S. Bettencourt, MD, recommends thinking outside the box and considering off-label treatments.
At the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar, Dr. Bettencourt discussed such cases, including a series of patients with oral lichen planus who improved with apremilast, an oral phosphodiesterase 4 inhibitor approved for psoriasis.
In a video interview at the meeting, she described one of those patients, a 73-year-old woman with mouth ulcers who was diagnosed with oral lichen planus. Multiple topical and oral therapies proved unsuccessful, and her condition was eventually controlled with apremilast, and the patient is doing well, “with occasional flares,” said Dr. Bettencourt, of the University of Nevada, Las Vegas.
She described this case in her annual presentation at the meeting, titled “Great Cases From the Las Vegas Dermatology Society.”
Dr. Bettencourt disclosed relationships with multiple companies including AbbVie, Aclaris, Celgene, IntraDerm, Pfizer, Promium, Sun Pharma, and Valeant.
SDEF and this news organization are owned by the same parent company.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
AT SDEF LAS VEGAS DERMATOLOGY SEMINAR
Study highlights disparities in U.S. lupus mortality
Mortality from systemic lupus erythematosus has declined since 1968 in the United States, but not as markedly as rates of death from other causes, according to a study in Annals of Internal Medicine.
Furthermore, systemic lupus erythematosus (SLE) mortality varied significantly by sex, race, and geographic region, noted Eric Y. Yen, MD, and his associates at the University of California, Los Angeles. Mortality from SLE was highest among females, black individuals, and those who lived in the South. Multivariable analyses confirmed that sex, race, and geographic region were independent risk factors for SLE mortality and that race modified relationships among SLE mortality, sex, and geographic region.
Between 1968 and 2013, there were 50,249 deaths from SLE and more than 100.8 million deaths from other causes in the United States, the researchers said. Mortality from other causes continuously dropped over the study period, but SLE mortality dropped only between 1968 and 1975 before rising continuously for 24 years. Only in 1999 did SLE mortality begin to fall again. Consequently, the ratio of SLE mortality to mortality from other causes rose by 34.6% overall between 1968 and 2013, and rose by 62.5% among blacks and by 58.6% among southerners.
After the researchers accounted for age, sex, race or ethnicity, and geographic region, the risk of death from SLE dropped significantly during 2004 through 2008, compared with 1999 through 2003, and declined even more between 2009 and 2013. Female sex, racial or ethnic minority status, residing in the South or West, and being older than 65 years all independently increased the risk of dying from SLE.
Although the South had the highest SLE mortality among whites, the West had the highest SLE mortality among all other races and ethnicities, the investigators determined. Previous research has identified pockets of increased SLE mortality in Alabama, Arkansas, Louisiana, and New Mexico, and has shown that poverty is a stronger predictor of SLE mortality than race, they noted. “Geographic differences in the quality of care of patients with lupus nephritis have also been reported, with more patients in the Northeast receiving standard-of-care medications,” they wrote. “Interactions between genetic and non-genetic factors associated with race/ethnicity and geographic differences in environment, such as increased sunlight exposure, socioeconomic factors, and access to medical care, might also influence SLE mortality.”
The National Institutes of Health, the Lupus Foundation of America, and the Rheumatology Research Foundation funded the study. The investigators reported having no conflicts of interest.
Mortality from systemic lupus erythematosus has declined since 1968 in the United States, but not as markedly as rates of death from other causes, according to a study in Annals of Internal Medicine.
Furthermore, systemic lupus erythematosus (SLE) mortality varied significantly by sex, race, and geographic region, noted Eric Y. Yen, MD, and his associates at the University of California, Los Angeles. Mortality from SLE was highest among females, black individuals, and those who lived in the South. Multivariable analyses confirmed that sex, race, and geographic region were independent risk factors for SLE mortality and that race modified relationships among SLE mortality, sex, and geographic region.
Between 1968 and 2013, there were 50,249 deaths from SLE and more than 100.8 million deaths from other causes in the United States, the researchers said. Mortality from other causes continuously dropped over the study period, but SLE mortality dropped only between 1968 and 1975 before rising continuously for 24 years. Only in 1999 did SLE mortality begin to fall again. Consequently, the ratio of SLE mortality to mortality from other causes rose by 34.6% overall between 1968 and 2013, and rose by 62.5% among blacks and by 58.6% among southerners.
After the researchers accounted for age, sex, race or ethnicity, and geographic region, the risk of death from SLE dropped significantly during 2004 through 2008, compared with 1999 through 2003, and declined even more between 2009 and 2013. Female sex, racial or ethnic minority status, residing in the South or West, and being older than 65 years all independently increased the risk of dying from SLE.
Although the South had the highest SLE mortality among whites, the West had the highest SLE mortality among all other races and ethnicities, the investigators determined. Previous research has identified pockets of increased SLE mortality in Alabama, Arkansas, Louisiana, and New Mexico, and has shown that poverty is a stronger predictor of SLE mortality than race, they noted. “Geographic differences in the quality of care of patients with lupus nephritis have also been reported, with more patients in the Northeast receiving standard-of-care medications,” they wrote. “Interactions between genetic and non-genetic factors associated with race/ethnicity and geographic differences in environment, such as increased sunlight exposure, socioeconomic factors, and access to medical care, might also influence SLE mortality.”
The National Institutes of Health, the Lupus Foundation of America, and the Rheumatology Research Foundation funded the study. The investigators reported having no conflicts of interest.
Mortality from systemic lupus erythematosus has declined since 1968 in the United States, but not as markedly as rates of death from other causes, according to a study in Annals of Internal Medicine.
Furthermore, systemic lupus erythematosus (SLE) mortality varied significantly by sex, race, and geographic region, noted Eric Y. Yen, MD, and his associates at the University of California, Los Angeles. Mortality from SLE was highest among females, black individuals, and those who lived in the South. Multivariable analyses confirmed that sex, race, and geographic region were independent risk factors for SLE mortality and that race modified relationships among SLE mortality, sex, and geographic region.
Between 1968 and 2013, there were 50,249 deaths from SLE and more than 100.8 million deaths from other causes in the United States, the researchers said. Mortality from other causes continuously dropped over the study period, but SLE mortality dropped only between 1968 and 1975 before rising continuously for 24 years. Only in 1999 did SLE mortality begin to fall again. Consequently, the ratio of SLE mortality to mortality from other causes rose by 34.6% overall between 1968 and 2013, and rose by 62.5% among blacks and by 58.6% among southerners.
After the researchers accounted for age, sex, race or ethnicity, and geographic region, the risk of death from SLE dropped significantly during 2004 through 2008, compared with 1999 through 2003, and declined even more between 2009 and 2013. Female sex, racial or ethnic minority status, residing in the South or West, and being older than 65 years all independently increased the risk of dying from SLE.
Although the South had the highest SLE mortality among whites, the West had the highest SLE mortality among all other races and ethnicities, the investigators determined. Previous research has identified pockets of increased SLE mortality in Alabama, Arkansas, Louisiana, and New Mexico, and has shown that poverty is a stronger predictor of SLE mortality than race, they noted. “Geographic differences in the quality of care of patients with lupus nephritis have also been reported, with more patients in the Northeast receiving standard-of-care medications,” they wrote. “Interactions between genetic and non-genetic factors associated with race/ethnicity and geographic differences in environment, such as increased sunlight exposure, socioeconomic factors, and access to medical care, might also influence SLE mortality.”
The National Institutes of Health, the Lupus Foundation of America, and the Rheumatology Research Foundation funded the study. The investigators reported having no conflicts of interest.
FROM ANNALS OF INTERNAL MEDICINE
Key clinical point: Mortality from systemic lupus erythematosus has declined since 1968, but not as markedly as rates of death from other causes.
Major finding: The ratio of SLE mortality to mortality from other causes rose by nearly 35% between 1968 and 2013.
Data source: Analyses of the Centers for Disease Control and Prevention’s National Vital Statistics System and CDC WONDER.
Disclosures: The National Institutes of Health, the Lupus Foundation of America, and the Rheumatology Research Foundation funded the study. The investigators reported having no conflicts of interest.
British Society of Rheumatology issues first U.K. lupus guideline
From diagnosing, assessing, and managing common manifestations of nonrenal lupus, such as skin rashes and arthritis, through dealing with less common but potentially more serious problems such as kidney disease, the guideline aims to help everyone involved in the management of patients with SLE to give the best, evidenced-based care.
U.K.-specific guidance
The British Society for Rheumatology’s (BSR) guideline is the first to specifically cover lupus management in the United Kingdom, and it builds on existing European League Against Rheumatism (EULAR) guidance published almost a decade ago (Ann Rheum Dis. 2008;67:195-205), and more recent EULAR/European Renal Association–European Dialysis and Transplant Association (Ann Rheum Dis. 2012;71:1771-82) and American College of Rheumatology (Arthritis Care Res. 2012;64:797-808) recommendations on managing lupus nephritis (LN).
The BSR’s full guideline provides a summary of the EULAR/ERA-EDTA’s LN guidelines, and the degree to which their new guidelines concur.
As with all BSR guidelines, the recommendations have been developed by a multidisciplinary team. This included academic and consultant rheumatologists and nephrologists, rheumatology trainees, a primary care physician, a clinical nurse specialist, a patient representative, and a lay member. This should make the guideline relevant to anyone who may come across someone with SLE, including primary care physicians, dermatologists, and emergency medicine practitioners.
“These recommendations are based on the literature review covering the diagnosis, assessment, monitoring, and treatment of mild, moderate, and severe lupus, including neuropsychiatric disease,” the guideline authors stated. They noted that the reason they looked only at nonrenal disease was because the EULAR/ERA-EDTA recommendations for LN have been published close to the time that work was started on the guideline. Each of the recommendations the multidisciplinary team devised was carefully graded and the degree to which members of the team agreed with each recommendation was calculated.
Diagnosis recommendations
One of the key recommendations regarding the diagnosis of SLE is that a combination of clinical features and at least one relevant immunologic irregularity needs to be present. Blood tests, including serologic marker tests, should be performed if there is clinical suspicion of lupus.
Another recommendation on diagnosis is that if antinuclear antibodies are absent, then it is unlikely that the patient has lupus. This is because around 95% of SLE patients will test positive for antinuclear antibodies. Antiphospholipid antibodies should be tested in all patients with lupus at baseline, according to the guideline.
Monitoring recommendations
“Patients with SLE should be monitored on a regular basis for disease manifestations, drug toxicities, and comorbidities,” Dr. Gordon and her associates advised in one of the recommendations on monitoring patients. In another, they wrote that those with active disease need reviewing at least every 1-3 months, which should include evaluation of patients’ blood pressure, urine, renal function, anti-dsDNA antibodies, complement, a full blood count, and liver function tests.
It is also important to monitor patients for the presence of antiphospholipid antibodies, which are associated with thrombotic events, and it is always important to be on the lookout for comorbidities such as atherosclerotic disease and manage modifiable risk factors such as hypertension. The guideline does not go into detail about managing all of the potential complications of lupus, however, as these are covered by other national guidelines.
Treatment recommendations
Guidance on treatment is separated into how to treat patients with mild, moderate, and severe SLE. The guideline does not cover topical or systemic treatment for isolated cutaneous lupus, nor does it look at how to manage pediatric patients, the authors noted. General guidance is given on how to treat patients, and specific dosing regimens are beyond the scope of the guidelines.
The recommendations encourage the use of a variety of treatments to try to ensure less reliance on the use of steroids to control symptoms. The guideline authors noted that only hydroxychloroquine, corticosteroids, and belimumab are currently licensed treatments for lupus in the United Kingdom.
For mild disease, the disease-modifying antirheumatic drugs hydroxychloroquine and methotrexate are suggested, as are nonsteroidal anti-inflammatory drugs. If prednisolone is used, then it should be in low doses (7.5 mg or less per day). Patients should be encouraged to use sunscreen and sun avoidance to protect them against ultraviolet-induced skin lesions.
For moderate disease, higher steroid doses may be needed and immunosuppressives might be warranted, and in refractory cases, monoclonal antibody treatment may be necessary.
For severe disease, thorough investigation is essential to exclude other possible causes of any renal or neuropsychiatric manifestations. Immunosuppressive treatment is recommended, with biologic therapies considered on a case-by-case basis. Intravenous immunoglobulin and plasmapheresis may also be an option in certain patients.
Key standards of care
As general standards of care, Dr. Gordon and her coauthors wrote that “lupus patients should be referred to a physician with experience in managing lupus who can confirm the diagnosis, assess the level of disease activity, and provide advice on treatment and monitoring of the disease, its complications, and side effects of therapy.”
Furthermore, a multidisciplinary team approach is needed, and if a treatment is not helping get patients into a state of low disease activity within an expected time frame, “it is important to consider adherence to treatment and adjusting the therapy to reduce the accumulation of chronic damage.” It is also recommended that patients receive personalized advice, written information, and education about the disease and its drug treatment.
No specific funding was received from any organizations in the public, commercial, or not-for-profit sectors to carry out the work described in the guideline.
Dr. Gordon has undertaken consultancies and received honoraria from Bristol-Myers Squibb, Eli Lilly, GlaxoSmithKline, MedImmune, Merck Serono, Parexel, Roche, and UCB. She has been a member of the speakers bureau for GlaxoSmithKline, UCB, and Eli Lilly. She also has received research grant support from Aspreva/Vifor Pharma in the past and currently receives it from UCB.
From diagnosing, assessing, and managing common manifestations of nonrenal lupus, such as skin rashes and arthritis, through dealing with less common but potentially more serious problems such as kidney disease, the guideline aims to help everyone involved in the management of patients with SLE to give the best, evidenced-based care.
U.K.-specific guidance
The British Society for Rheumatology’s (BSR) guideline is the first to specifically cover lupus management in the United Kingdom, and it builds on existing European League Against Rheumatism (EULAR) guidance published almost a decade ago (Ann Rheum Dis. 2008;67:195-205), and more recent EULAR/European Renal Association–European Dialysis and Transplant Association (Ann Rheum Dis. 2012;71:1771-82) and American College of Rheumatology (Arthritis Care Res. 2012;64:797-808) recommendations on managing lupus nephritis (LN).
The BSR’s full guideline provides a summary of the EULAR/ERA-EDTA’s LN guidelines, and the degree to which their new guidelines concur.
As with all BSR guidelines, the recommendations have been developed by a multidisciplinary team. This included academic and consultant rheumatologists and nephrologists, rheumatology trainees, a primary care physician, a clinical nurse specialist, a patient representative, and a lay member. This should make the guideline relevant to anyone who may come across someone with SLE, including primary care physicians, dermatologists, and emergency medicine practitioners.
“These recommendations are based on the literature review covering the diagnosis, assessment, monitoring, and treatment of mild, moderate, and severe lupus, including neuropsychiatric disease,” the guideline authors stated. They noted that the reason they looked only at nonrenal disease was because the EULAR/ERA-EDTA recommendations for LN have been published close to the time that work was started on the guideline. Each of the recommendations the multidisciplinary team devised was carefully graded and the degree to which members of the team agreed with each recommendation was calculated.
Diagnosis recommendations
One of the key recommendations regarding the diagnosis of SLE is that a combination of clinical features and at least one relevant immunologic irregularity needs to be present. Blood tests, including serologic marker tests, should be performed if there is clinical suspicion of lupus.
Another recommendation on diagnosis is that if antinuclear antibodies are absent, then it is unlikely that the patient has lupus. This is because around 95% of SLE patients will test positive for antinuclear antibodies. Antiphospholipid antibodies should be tested in all patients with lupus at baseline, according to the guideline.
Monitoring recommendations
“Patients with SLE should be monitored on a regular basis for disease manifestations, drug toxicities, and comorbidities,” Dr. Gordon and her associates advised in one of the recommendations on monitoring patients. In another, they wrote that those with active disease need reviewing at least every 1-3 months, which should include evaluation of patients’ blood pressure, urine, renal function, anti-dsDNA antibodies, complement, a full blood count, and liver function tests.
It is also important to monitor patients for the presence of antiphospholipid antibodies, which are associated with thrombotic events, and it is always important to be on the lookout for comorbidities such as atherosclerotic disease and manage modifiable risk factors such as hypertension. The guideline does not go into detail about managing all of the potential complications of lupus, however, as these are covered by other national guidelines.
Treatment recommendations
Guidance on treatment is separated into how to treat patients with mild, moderate, and severe SLE. The guideline does not cover topical or systemic treatment for isolated cutaneous lupus, nor does it look at how to manage pediatric patients, the authors noted. General guidance is given on how to treat patients, and specific dosing regimens are beyond the scope of the guidelines.
The recommendations encourage the use of a variety of treatments to try to ensure less reliance on the use of steroids to control symptoms. The guideline authors noted that only hydroxychloroquine, corticosteroids, and belimumab are currently licensed treatments for lupus in the United Kingdom.
For mild disease, the disease-modifying antirheumatic drugs hydroxychloroquine and methotrexate are suggested, as are nonsteroidal anti-inflammatory drugs. If prednisolone is used, then it should be in low doses (7.5 mg or less per day). Patients should be encouraged to use sunscreen and sun avoidance to protect them against ultraviolet-induced skin lesions.
For moderate disease, higher steroid doses may be needed and immunosuppressives might be warranted, and in refractory cases, monoclonal antibody treatment may be necessary.
For severe disease, thorough investigation is essential to exclude other possible causes of any renal or neuropsychiatric manifestations. Immunosuppressive treatment is recommended, with biologic therapies considered on a case-by-case basis. Intravenous immunoglobulin and plasmapheresis may also be an option in certain patients.
Key standards of care
As general standards of care, Dr. Gordon and her coauthors wrote that “lupus patients should be referred to a physician with experience in managing lupus who can confirm the diagnosis, assess the level of disease activity, and provide advice on treatment and monitoring of the disease, its complications, and side effects of therapy.”
Furthermore, a multidisciplinary team approach is needed, and if a treatment is not helping get patients into a state of low disease activity within an expected time frame, “it is important to consider adherence to treatment and adjusting the therapy to reduce the accumulation of chronic damage.” It is also recommended that patients receive personalized advice, written information, and education about the disease and its drug treatment.
No specific funding was received from any organizations in the public, commercial, or not-for-profit sectors to carry out the work described in the guideline.
Dr. Gordon has undertaken consultancies and received honoraria from Bristol-Myers Squibb, Eli Lilly, GlaxoSmithKline, MedImmune, Merck Serono, Parexel, Roche, and UCB. She has been a member of the speakers bureau for GlaxoSmithKline, UCB, and Eli Lilly. She also has received research grant support from Aspreva/Vifor Pharma in the past and currently receives it from UCB.
From diagnosing, assessing, and managing common manifestations of nonrenal lupus, such as skin rashes and arthritis, through dealing with less common but potentially more serious problems such as kidney disease, the guideline aims to help everyone involved in the management of patients with SLE to give the best, evidenced-based care.
U.K.-specific guidance
The British Society for Rheumatology’s (BSR) guideline is the first to specifically cover lupus management in the United Kingdom, and it builds on existing European League Against Rheumatism (EULAR) guidance published almost a decade ago (Ann Rheum Dis. 2008;67:195-205), and more recent EULAR/European Renal Association–European Dialysis and Transplant Association (Ann Rheum Dis. 2012;71:1771-82) and American College of Rheumatology (Arthritis Care Res. 2012;64:797-808) recommendations on managing lupus nephritis (LN).
The BSR’s full guideline provides a summary of the EULAR/ERA-EDTA’s LN guidelines, and the degree to which their new guidelines concur.
As with all BSR guidelines, the recommendations have been developed by a multidisciplinary team. This included academic and consultant rheumatologists and nephrologists, rheumatology trainees, a primary care physician, a clinical nurse specialist, a patient representative, and a lay member. This should make the guideline relevant to anyone who may come across someone with SLE, including primary care physicians, dermatologists, and emergency medicine practitioners.
“These recommendations are based on the literature review covering the diagnosis, assessment, monitoring, and treatment of mild, moderate, and severe lupus, including neuropsychiatric disease,” the guideline authors stated. They noted that the reason they looked only at nonrenal disease was because the EULAR/ERA-EDTA recommendations for LN have been published close to the time that work was started on the guideline. Each of the recommendations the multidisciplinary team devised was carefully graded and the degree to which members of the team agreed with each recommendation was calculated.
Diagnosis recommendations
One of the key recommendations regarding the diagnosis of SLE is that a combination of clinical features and at least one relevant immunologic irregularity needs to be present. Blood tests, including serologic marker tests, should be performed if there is clinical suspicion of lupus.
Another recommendation on diagnosis is that if antinuclear antibodies are absent, then it is unlikely that the patient has lupus. This is because around 95% of SLE patients will test positive for antinuclear antibodies. Antiphospholipid antibodies should be tested in all patients with lupus at baseline, according to the guideline.
Monitoring recommendations
“Patients with SLE should be monitored on a regular basis for disease manifestations, drug toxicities, and comorbidities,” Dr. Gordon and her associates advised in one of the recommendations on monitoring patients. In another, they wrote that those with active disease need reviewing at least every 1-3 months, which should include evaluation of patients’ blood pressure, urine, renal function, anti-dsDNA antibodies, complement, a full blood count, and liver function tests.
It is also important to monitor patients for the presence of antiphospholipid antibodies, which are associated with thrombotic events, and it is always important to be on the lookout for comorbidities such as atherosclerotic disease and manage modifiable risk factors such as hypertension. The guideline does not go into detail about managing all of the potential complications of lupus, however, as these are covered by other national guidelines.
Treatment recommendations
Guidance on treatment is separated into how to treat patients with mild, moderate, and severe SLE. The guideline does not cover topical or systemic treatment for isolated cutaneous lupus, nor does it look at how to manage pediatric patients, the authors noted. General guidance is given on how to treat patients, and specific dosing regimens are beyond the scope of the guidelines.
The recommendations encourage the use of a variety of treatments to try to ensure less reliance on the use of steroids to control symptoms. The guideline authors noted that only hydroxychloroquine, corticosteroids, and belimumab are currently licensed treatments for lupus in the United Kingdom.
For mild disease, the disease-modifying antirheumatic drugs hydroxychloroquine and methotrexate are suggested, as are nonsteroidal anti-inflammatory drugs. If prednisolone is used, then it should be in low doses (7.5 mg or less per day). Patients should be encouraged to use sunscreen and sun avoidance to protect them against ultraviolet-induced skin lesions.
For moderate disease, higher steroid doses may be needed and immunosuppressives might be warranted, and in refractory cases, monoclonal antibody treatment may be necessary.
For severe disease, thorough investigation is essential to exclude other possible causes of any renal or neuropsychiatric manifestations. Immunosuppressive treatment is recommended, with biologic therapies considered on a case-by-case basis. Intravenous immunoglobulin and plasmapheresis may also be an option in certain patients.
Key standards of care
As general standards of care, Dr. Gordon and her coauthors wrote that “lupus patients should be referred to a physician with experience in managing lupus who can confirm the diagnosis, assess the level of disease activity, and provide advice on treatment and monitoring of the disease, its complications, and side effects of therapy.”
Furthermore, a multidisciplinary team approach is needed, and if a treatment is not helping get patients into a state of low disease activity within an expected time frame, “it is important to consider adherence to treatment and adjusting the therapy to reduce the accumulation of chronic damage.” It is also recommended that patients receive personalized advice, written information, and education about the disease and its drug treatment.
No specific funding was received from any organizations in the public, commercial, or not-for-profit sectors to carry out the work described in the guideline.
Dr. Gordon has undertaken consultancies and received honoraria from Bristol-Myers Squibb, Eli Lilly, GlaxoSmithKline, MedImmune, Merck Serono, Parexel, Roche, and UCB. She has been a member of the speakers bureau for GlaxoSmithKline, UCB, and Eli Lilly. She also has received research grant support from Aspreva/Vifor Pharma in the past and currently receives it from UCB.
FROM RHEUMATOLOGY
Abatacept shows potential in refractory myositis
Abatacept could be a new treatment option for people with adult dermatomyositis and polymyositis refractory to conventional treatment, a small, randomized pilot study suggests.
The investigators, led by first author Anna Tjärnlund, PhD, of the Karolinska Institute in Stockholm, noted that the health-related quality of life for people with dermatomyositis (DM) and polymyositis (PM) is low, compared with the general population.
“The majority of commonly used drugs are not approved for myositis, and only [a] few randomized, controlled trials (RCTs) have been performed in this patient group. Thus, there is an unmet need for new therapies for these patients,” they wrote (Ann Rheum Dis. 2017 Oct 9. doi: 10.1136/annrheumdis-2017-211751).
According to the researchers, muscle biopsies of people with DM and PM show a predominance of T cells in inflammatory infiltrates, suggesting a role for T cells in the disease process. Abatacept (Orencia), a fully human fusion protein of CTLA-4 and the Fc portion of human IgG1 that inhibits the co-stimulation of T cells, has been shown in several case reports to have beneficial effects in myositis, but no RCT has been done.
Abatacept is approved by the Food and Drug Administration for the treatment of moderately to severely active rheumatoid arthritis, moderately to severely active polyarticular juvenile idiopathic arthritis, and active psoriatic arthritis in adults.
The aim of the current phase 2b pilot study was to investigate the efficacy and safety of abatacept in a randomized trial with a delayed start in one arm. The researchers randomized 19 patients with DM or PM with refractory disease to receive either immediate active treatment (n = 10) with intravenous abatacept (dosed according to body weight) or a 3-month delayed start (n = 9). Patients who weighed less than 60 kg received 500 mg abatacept, those who weighed 60-100 kg received 750 mg, and those with body weight greater than 100 kg received 1,000 mg.
The primary endpoint was the number of responders defined by the International Myositis Assessment and Clinical Studies (IMACS) Group definition of improvement (relative improvement of 20% or greater in three of any six core set measures, with no more than two core set measures worsening by 25% or more) after 6 months of treatment.
The researchers saw improvements in the active treatment arm, compared with the delayed-start arm. At 3 months, five patients in the active treatment arm were responders, compared with one patient in the delayed treatment arm. For example, the active treatment groups improved by a mean of 2.5 points on the Manual Muscle Testing–8 (one of the individual components of the IMACS core set), compared with –4.9 in the delayed treatment arm (P = .0375).
At 6 months, an intent-to-treat analysis revealed that 8 out of 19 patients responded (2 with DM, 6 with PM) and reached the definition of improvement, with the remaining patients classified as nonresponders.
In patients who had before and after muscle biopsies, the expression of anti-inflammatory Foxp3+ regulatory T cells was significantly greater after abatacept treatment, the researchers reported. They noted that they had previously seen a decrease in the number of Foxp3+ cells in the tissues of patients with myositis on treatment with glucocorticoids.
“This difference could be related to the different treatment targets as since tissue-resident Foxp3+ regulatory T cells have been implicated in muscle repair and regeneration.”
Overall, 36 adverse events were reported during the study. Eight were considered related to abatacept, of which four were considered “mild” and the remaining four “moderate.”
The researchers concluded that although their study was not powered to confirm efficacy, treatment with abatacept was “clinically efficacious in a subgroup of patients with DM or PM and has an acceptable safety profile in refractory patients.”
They cautioned that treatment with abatacept might provide a new treatment option in PM/DM, but it needs to be investigated in randomized, placebo-controlled trials in larger patient populations.
The study was funded by grants from Bristol-Myers Squibb, the Börje Dahlin Foundation, the Swedish Research Council, the Swedish Rheumatism Association, and the King Gustaf V 80-Year Foundation. Two authors reported receiving research grants from Bristol-Myers Squibb and one serves as an advisory board consultant to the company.
Abatacept could be a new treatment option for people with adult dermatomyositis and polymyositis refractory to conventional treatment, a small, randomized pilot study suggests.
The investigators, led by first author Anna Tjärnlund, PhD, of the Karolinska Institute in Stockholm, noted that the health-related quality of life for people with dermatomyositis (DM) and polymyositis (PM) is low, compared with the general population.
“The majority of commonly used drugs are not approved for myositis, and only [a] few randomized, controlled trials (RCTs) have been performed in this patient group. Thus, there is an unmet need for new therapies for these patients,” they wrote (Ann Rheum Dis. 2017 Oct 9. doi: 10.1136/annrheumdis-2017-211751).
According to the researchers, muscle biopsies of people with DM and PM show a predominance of T cells in inflammatory infiltrates, suggesting a role for T cells in the disease process. Abatacept (Orencia), a fully human fusion protein of CTLA-4 and the Fc portion of human IgG1 that inhibits the co-stimulation of T cells, has been shown in several case reports to have beneficial effects in myositis, but no RCT has been done.
Abatacept is approved by the Food and Drug Administration for the treatment of moderately to severely active rheumatoid arthritis, moderately to severely active polyarticular juvenile idiopathic arthritis, and active psoriatic arthritis in adults.
The aim of the current phase 2b pilot study was to investigate the efficacy and safety of abatacept in a randomized trial with a delayed start in one arm. The researchers randomized 19 patients with DM or PM with refractory disease to receive either immediate active treatment (n = 10) with intravenous abatacept (dosed according to body weight) or a 3-month delayed start (n = 9). Patients who weighed less than 60 kg received 500 mg abatacept, those who weighed 60-100 kg received 750 mg, and those with body weight greater than 100 kg received 1,000 mg.
The primary endpoint was the number of responders defined by the International Myositis Assessment and Clinical Studies (IMACS) Group definition of improvement (relative improvement of 20% or greater in three of any six core set measures, with no more than two core set measures worsening by 25% or more) after 6 months of treatment.
The researchers saw improvements in the active treatment arm, compared with the delayed-start arm. At 3 months, five patients in the active treatment arm were responders, compared with one patient in the delayed treatment arm. For example, the active treatment groups improved by a mean of 2.5 points on the Manual Muscle Testing–8 (one of the individual components of the IMACS core set), compared with –4.9 in the delayed treatment arm (P = .0375).
At 6 months, an intent-to-treat analysis revealed that 8 out of 19 patients responded (2 with DM, 6 with PM) and reached the definition of improvement, with the remaining patients classified as nonresponders.
In patients who had before and after muscle biopsies, the expression of anti-inflammatory Foxp3+ regulatory T cells was significantly greater after abatacept treatment, the researchers reported. They noted that they had previously seen a decrease in the number of Foxp3+ cells in the tissues of patients with myositis on treatment with glucocorticoids.
“This difference could be related to the different treatment targets as since tissue-resident Foxp3+ regulatory T cells have been implicated in muscle repair and regeneration.”
Overall, 36 adverse events were reported during the study. Eight were considered related to abatacept, of which four were considered “mild” and the remaining four “moderate.”
The researchers concluded that although their study was not powered to confirm efficacy, treatment with abatacept was “clinically efficacious in a subgroup of patients with DM or PM and has an acceptable safety profile in refractory patients.”
They cautioned that treatment with abatacept might provide a new treatment option in PM/DM, but it needs to be investigated in randomized, placebo-controlled trials in larger patient populations.
The study was funded by grants from Bristol-Myers Squibb, the Börje Dahlin Foundation, the Swedish Research Council, the Swedish Rheumatism Association, and the King Gustaf V 80-Year Foundation. Two authors reported receiving research grants from Bristol-Myers Squibb and one serves as an advisory board consultant to the company.
Abatacept could be a new treatment option for people with adult dermatomyositis and polymyositis refractory to conventional treatment, a small, randomized pilot study suggests.
The investigators, led by first author Anna Tjärnlund, PhD, of the Karolinska Institute in Stockholm, noted that the health-related quality of life for people with dermatomyositis (DM) and polymyositis (PM) is low, compared with the general population.
“The majority of commonly used drugs are not approved for myositis, and only [a] few randomized, controlled trials (RCTs) have been performed in this patient group. Thus, there is an unmet need for new therapies for these patients,” they wrote (Ann Rheum Dis. 2017 Oct 9. doi: 10.1136/annrheumdis-2017-211751).
According to the researchers, muscle biopsies of people with DM and PM show a predominance of T cells in inflammatory infiltrates, suggesting a role for T cells in the disease process. Abatacept (Orencia), a fully human fusion protein of CTLA-4 and the Fc portion of human IgG1 that inhibits the co-stimulation of T cells, has been shown in several case reports to have beneficial effects in myositis, but no RCT has been done.
Abatacept is approved by the Food and Drug Administration for the treatment of moderately to severely active rheumatoid arthritis, moderately to severely active polyarticular juvenile idiopathic arthritis, and active psoriatic arthritis in adults.
The aim of the current phase 2b pilot study was to investigate the efficacy and safety of abatacept in a randomized trial with a delayed start in one arm. The researchers randomized 19 patients with DM or PM with refractory disease to receive either immediate active treatment (n = 10) with intravenous abatacept (dosed according to body weight) or a 3-month delayed start (n = 9). Patients who weighed less than 60 kg received 500 mg abatacept, those who weighed 60-100 kg received 750 mg, and those with body weight greater than 100 kg received 1,000 mg.
The primary endpoint was the number of responders defined by the International Myositis Assessment and Clinical Studies (IMACS) Group definition of improvement (relative improvement of 20% or greater in three of any six core set measures, with no more than two core set measures worsening by 25% or more) after 6 months of treatment.
The researchers saw improvements in the active treatment arm, compared with the delayed-start arm. At 3 months, five patients in the active treatment arm were responders, compared with one patient in the delayed treatment arm. For example, the active treatment groups improved by a mean of 2.5 points on the Manual Muscle Testing–8 (one of the individual components of the IMACS core set), compared with –4.9 in the delayed treatment arm (P = .0375).
At 6 months, an intent-to-treat analysis revealed that 8 out of 19 patients responded (2 with DM, 6 with PM) and reached the definition of improvement, with the remaining patients classified as nonresponders.
In patients who had before and after muscle biopsies, the expression of anti-inflammatory Foxp3+ regulatory T cells was significantly greater after abatacept treatment, the researchers reported. They noted that they had previously seen a decrease in the number of Foxp3+ cells in the tissues of patients with myositis on treatment with glucocorticoids.
“This difference could be related to the different treatment targets as since tissue-resident Foxp3+ regulatory T cells have been implicated in muscle repair and regeneration.”
Overall, 36 adverse events were reported during the study. Eight were considered related to abatacept, of which four were considered “mild” and the remaining four “moderate.”
The researchers concluded that although their study was not powered to confirm efficacy, treatment with abatacept was “clinically efficacious in a subgroup of patients with DM or PM and has an acceptable safety profile in refractory patients.”
They cautioned that treatment with abatacept might provide a new treatment option in PM/DM, but it needs to be investigated in randomized, placebo-controlled trials in larger patient populations.
The study was funded by grants from Bristol-Myers Squibb, the Börje Dahlin Foundation, the Swedish Research Council, the Swedish Rheumatism Association, and the King Gustaf V 80-Year Foundation. Two authors reported receiving research grants from Bristol-Myers Squibb and one serves as an advisory board consultant to the company.
FROM ANNALS OF THE RHEUMATIC DISEASES
Key clinical point:
Major finding: Of 19 patients, 8 were classified as treatment responders, reaching the IMACS definition of improvement.
Data source: A phase 2b pilot study with a randomized delayed treatment arm.
Disclosures: The study was funded by grants from Bristol-Myers Squibb, the Börje Dahlin Foundation, the Swedish Research Council, the Swedish Rheumatism Association, and the King Gustaf V 80-Year Foundation. Two authors reported receiving research grants from Bristol-Myers Squibb and one serves as an advisory board consultant to the company.
Poverty affects lupus mortality through damage accumulation
The extent of damage caused by lupus appears to be one of the strongest factors that contributes to the higher mortality observed in lupus patients who live in poverty, according to the results of a longitudinal cohort study of patients.
The findings from this analysis of participants in the Lupus Outcomes Study could potentially lead to solutions to reduce mortality of poor patients with systemic lupus erythematosus (SLE) through understanding why they have higher levels of disease damage, said first author Edward Yelin, PhD, of the Philip R. Lee Institute for Health Policy Studies at the University of California, San Francisco, and his colleagues at the university (Arthritis Care Res. 2017 Oct 3. doi: 10.1002/acr.23428).
Previous studies by the researchers have shown that concurrent and persistent poverty are associated with increased damage accumulation, and permanently exiting poverty reduces the level of accumulated damage. Studies by other groups have also revealed that some measures of low socioeconomic status contribute to elevated mortality in patients with SLE. But few studies have explored how poverty leads to increased mortality in SLE patients.
About two-thirds of the patients recruited for the Lupus Outcomes Study, which began in 2003, joined the study through nonclinical sources, such as public service announcements, patient support groups, and word of mouth; the remainder were recruited from academic and community clinical practices. The patients came from 37 states and from urban and rural areas. The investigators contacted participants in annual structured phone interviews that lasted about 45 minutes. The investigators defined poverty as household income at or below 125% of the federal poverty level because most participants were from high-cost urban areas.
The investigators had full information available for 807 of 814 who completed the annual survey in 2009, and these individuals made up the baseline sample for the mortality analysis through 2015. These 807 patients had a mean age of about 50 years and a disease duration of about 17 years; 93% were women, more than one-third were members of racial and ethnic minorities, and 14% met the study’s definition of poverty.
Poor individuals were more likely to be from a racial or ethnic minority (54% vs. 33%), to have a history of smoking (47% vs. 37%, to have a high school education or less (37% vs. 14%), to have never been married (36% vs. 15%), and to have a higher baseline level of disease damage as measured by score on the Brief Index of Lupus Damage (BILD; 2.8 vs. 2.2).
Overall, more poor individuals died during 2009-2015 (12.1% vs. 8.3%), but the difference was not significant. However, the poor died at a mean age of about 50, compared with 64 for individuals who were not poor. Adjustments for age showed that poverty, disease duration, BILD damage score, and having less than a high school education were all associated with higher mortality. But in a full multivariable analysis, only female gender (hazard ratio, 0.43; 95% confidence interval, 0.19-0.94), BILD damage score (1.17/point on 0-18 scale; 95% CI, 1.07-1.29), and physical health status (0.96/point; 95% CI, 0.94-0.98) were significant predictors of subsequent mortality risk, and poverty was no longer a significant predictor of mortality.
While poverty adjusted for age more than doubled the mortality risk (HR, 2.14; 95% CI, 1.18-3.88), much of the association could be attributed to the level of disease damage because once that variable was added to the analysis, poverty was no longer associated with an elevated mortality risk (HR, 1.68; 95% CI, 0.91-3.10). Furthermore, once the investigators took physical and mental health status into account, the risk of mortality associated with poverty was even smaller (HR, 1.20; 95% CI, 0.61-2.36).
“The present analysis indicates that prevention of accumulated damage will attenuate the mortality risk associated with poverty. We know that to achieve the goal of reduced damage requires good medical care in SLE, but that alone is insufficient since medical care accounts for only a small portion of the variance in damage accumulation between the poor and non-poor,” the investigators wrote. “Strategies to reduce disease damage must take into account the provision of high-quality care for the condition as well as the stress associated with poverty and living in neighborhoods with concentrated poverty.”
The research was supported by the Robert Wood Johnson Investigator in Health Policy Award and grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
The extent of damage caused by lupus appears to be one of the strongest factors that contributes to the higher mortality observed in lupus patients who live in poverty, according to the results of a longitudinal cohort study of patients.
The findings from this analysis of participants in the Lupus Outcomes Study could potentially lead to solutions to reduce mortality of poor patients with systemic lupus erythematosus (SLE) through understanding why they have higher levels of disease damage, said first author Edward Yelin, PhD, of the Philip R. Lee Institute for Health Policy Studies at the University of California, San Francisco, and his colleagues at the university (Arthritis Care Res. 2017 Oct 3. doi: 10.1002/acr.23428).
Previous studies by the researchers have shown that concurrent and persistent poverty are associated with increased damage accumulation, and permanently exiting poverty reduces the level of accumulated damage. Studies by other groups have also revealed that some measures of low socioeconomic status contribute to elevated mortality in patients with SLE. But few studies have explored how poverty leads to increased mortality in SLE patients.
About two-thirds of the patients recruited for the Lupus Outcomes Study, which began in 2003, joined the study through nonclinical sources, such as public service announcements, patient support groups, and word of mouth; the remainder were recruited from academic and community clinical practices. The patients came from 37 states and from urban and rural areas. The investigators contacted participants in annual structured phone interviews that lasted about 45 minutes. The investigators defined poverty as household income at or below 125% of the federal poverty level because most participants were from high-cost urban areas.
The investigators had full information available for 807 of 814 who completed the annual survey in 2009, and these individuals made up the baseline sample for the mortality analysis through 2015. These 807 patients had a mean age of about 50 years and a disease duration of about 17 years; 93% were women, more than one-third were members of racial and ethnic minorities, and 14% met the study’s definition of poverty.
Poor individuals were more likely to be from a racial or ethnic minority (54% vs. 33%), to have a history of smoking (47% vs. 37%, to have a high school education or less (37% vs. 14%), to have never been married (36% vs. 15%), and to have a higher baseline level of disease damage as measured by score on the Brief Index of Lupus Damage (BILD; 2.8 vs. 2.2).
Overall, more poor individuals died during 2009-2015 (12.1% vs. 8.3%), but the difference was not significant. However, the poor died at a mean age of about 50, compared with 64 for individuals who were not poor. Adjustments for age showed that poverty, disease duration, BILD damage score, and having less than a high school education were all associated with higher mortality. But in a full multivariable analysis, only female gender (hazard ratio, 0.43; 95% confidence interval, 0.19-0.94), BILD damage score (1.17/point on 0-18 scale; 95% CI, 1.07-1.29), and physical health status (0.96/point; 95% CI, 0.94-0.98) were significant predictors of subsequent mortality risk, and poverty was no longer a significant predictor of mortality.
While poverty adjusted for age more than doubled the mortality risk (HR, 2.14; 95% CI, 1.18-3.88), much of the association could be attributed to the level of disease damage because once that variable was added to the analysis, poverty was no longer associated with an elevated mortality risk (HR, 1.68; 95% CI, 0.91-3.10). Furthermore, once the investigators took physical and mental health status into account, the risk of mortality associated with poverty was even smaller (HR, 1.20; 95% CI, 0.61-2.36).
“The present analysis indicates that prevention of accumulated damage will attenuate the mortality risk associated with poverty. We know that to achieve the goal of reduced damage requires good medical care in SLE, but that alone is insufficient since medical care accounts for only a small portion of the variance in damage accumulation between the poor and non-poor,” the investigators wrote. “Strategies to reduce disease damage must take into account the provision of high-quality care for the condition as well as the stress associated with poverty and living in neighborhoods with concentrated poverty.”
The research was supported by the Robert Wood Johnson Investigator in Health Policy Award and grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
The extent of damage caused by lupus appears to be one of the strongest factors that contributes to the higher mortality observed in lupus patients who live in poverty, according to the results of a longitudinal cohort study of patients.
The findings from this analysis of participants in the Lupus Outcomes Study could potentially lead to solutions to reduce mortality of poor patients with systemic lupus erythematosus (SLE) through understanding why they have higher levels of disease damage, said first author Edward Yelin, PhD, of the Philip R. Lee Institute for Health Policy Studies at the University of California, San Francisco, and his colleagues at the university (Arthritis Care Res. 2017 Oct 3. doi: 10.1002/acr.23428).
Previous studies by the researchers have shown that concurrent and persistent poverty are associated with increased damage accumulation, and permanently exiting poverty reduces the level of accumulated damage. Studies by other groups have also revealed that some measures of low socioeconomic status contribute to elevated mortality in patients with SLE. But few studies have explored how poverty leads to increased mortality in SLE patients.
About two-thirds of the patients recruited for the Lupus Outcomes Study, which began in 2003, joined the study through nonclinical sources, such as public service announcements, patient support groups, and word of mouth; the remainder were recruited from academic and community clinical practices. The patients came from 37 states and from urban and rural areas. The investigators contacted participants in annual structured phone interviews that lasted about 45 minutes. The investigators defined poverty as household income at or below 125% of the federal poverty level because most participants were from high-cost urban areas.
The investigators had full information available for 807 of 814 who completed the annual survey in 2009, and these individuals made up the baseline sample for the mortality analysis through 2015. These 807 patients had a mean age of about 50 years and a disease duration of about 17 years; 93% were women, more than one-third were members of racial and ethnic minorities, and 14% met the study’s definition of poverty.
Poor individuals were more likely to be from a racial or ethnic minority (54% vs. 33%), to have a history of smoking (47% vs. 37%, to have a high school education or less (37% vs. 14%), to have never been married (36% vs. 15%), and to have a higher baseline level of disease damage as measured by score on the Brief Index of Lupus Damage (BILD; 2.8 vs. 2.2).
Overall, more poor individuals died during 2009-2015 (12.1% vs. 8.3%), but the difference was not significant. However, the poor died at a mean age of about 50, compared with 64 for individuals who were not poor. Adjustments for age showed that poverty, disease duration, BILD damage score, and having less than a high school education were all associated with higher mortality. But in a full multivariable analysis, only female gender (hazard ratio, 0.43; 95% confidence interval, 0.19-0.94), BILD damage score (1.17/point on 0-18 scale; 95% CI, 1.07-1.29), and physical health status (0.96/point; 95% CI, 0.94-0.98) were significant predictors of subsequent mortality risk, and poverty was no longer a significant predictor of mortality.
While poverty adjusted for age more than doubled the mortality risk (HR, 2.14; 95% CI, 1.18-3.88), much of the association could be attributed to the level of disease damage because once that variable was added to the analysis, poverty was no longer associated with an elevated mortality risk (HR, 1.68; 95% CI, 0.91-3.10). Furthermore, once the investigators took physical and mental health status into account, the risk of mortality associated with poverty was even smaller (HR, 1.20; 95% CI, 0.61-2.36).
“The present analysis indicates that prevention of accumulated damage will attenuate the mortality risk associated with poverty. We know that to achieve the goal of reduced damage requires good medical care in SLE, but that alone is insufficient since medical care accounts for only a small portion of the variance in damage accumulation between the poor and non-poor,” the investigators wrote. “Strategies to reduce disease damage must take into account the provision of high-quality care for the condition as well as the stress associated with poverty and living in neighborhoods with concentrated poverty.”
The research was supported by the Robert Wood Johnson Investigator in Health Policy Award and grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
FROM ARTHRITIS CARE & RESEARCH
Key clinical point:
Major finding: Poverty adjusted for age more than doubled the mortality risk (HR, 2.14; 95% CI, 1.18-3.88), but poverty was no longer associated with an elevated mortality risk when level of damage was added (HR, 1.68; 95% CI, 0.91-3.10).
Data source: A total of 807 participants in the Lupus Outcomes Study.
Disclosures: The research was supported by the Robert Wood Johnson Investigator in Health Policy Award and grants from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
Survival in lupus patients has plateaued
The major improvement in survival that patients with systemic lupus erythematosus (SLE) have experienced from 1950 to the mid-1990s has plateaued ever since, reported Maria Tektonidou, MD, and her colleagues. The study was published in Annals of the Rheumatic Diseases.
Dr. Tektonidou of National and Kapodistrian University of Athens and her coauthors at the U.S. National Institute of Arthritis and Musculoskeletal and Skin Diseases performed a meta-analysis on studies examining survival in adult and pediatric SLE patients from the 1950s to the mid-1990s. Ultimately, they analyzed 125 adult studies, including 82 from high-income countries and 43 from low- to middle-income countries (LMIC), and 51 pediatric studies, of which 33 were from high-income countries and 18 from LMIC.
In adult studies, researchers found that both high-income and LMIC experienced gradual increases in survival from the 1950s to mid-1990s. After this period of time, the survival estimates stabilized. “In 2008–2016, the 5-year, 10-year, and 15-year survival estimates in high-income countries were 0.95 (95% credible interval, 0.94 to 0.96), 0.89 (0.88 to 0.90) and 0.82 (0.81 to 0.83), respectively” (Ann Rheum Dis. 2017 Aug 9. doi: 10.1136/annrheumdis-2017-211663).
Although there were no data for LMIC prior to 1970, researchers identified survival trends similar to those in high-income countries in more recent years. Over the same time period between 2008 and 2016, “the 5-year, 10-year, and 15-year survival estimates in LMIC were 0.92 (0.91 to 0.93), 0.85 (0.84 to 0.87) and 0.79 (0.78 to 0.81), respectively,” according to the report.
Unlike the steady improvement seen over a 40-year period with adult studies, pediatric SLE patients in high-income countries experienced dramatic increases in survival rates from the 1960s to the 1970s, followed by slower increases in survival rates. The researchers reported that between 2008 and 2016,“the 5-year and 10-year survival estimates from high-income countries were 0.99 (0.98 to 1.00) and 0.97 (0.96 to 0.98), respectively.”
LMIC had significantly worse survival in pediatric SLE patients than did their wealthy counterparts. “Survival persistently lagged [behind] that of high-income countries” between 1980 and 2000. Dr. Tektonidou and her associates found that “5-year and 10-year survival estimates from LMIC were 0.85 (0.83 to 0.88) and 0.79 (0.76 to 0.82), respectively.” Due to the small number of studies reporting 15-year survival rates, this time point was not included in the pediatric analysis.
The researchers also analyzed the cause of death for adult and pediatric SLE patients in both high-income countries and LMIC. High-income countries showed lower rates of SLE-associated deaths over time in adults, although infection-related deaths increased in adults in both high-income countries and LMIC. There were not enough studies and data to assess cause of death in pediatric studies in high-income countries, but pediatric patients in LMIC had an upward trend in SLE-associated deaths.
The Intramural Research Program of the National Institute of Arthritis and Musculoskeletal and Skin Diseases supported the study. The researchers reported having no relevant financial disclosures.
The major improvement in survival that patients with systemic lupus erythematosus (SLE) have experienced from 1950 to the mid-1990s has plateaued ever since, reported Maria Tektonidou, MD, and her colleagues. The study was published in Annals of the Rheumatic Diseases.
Dr. Tektonidou of National and Kapodistrian University of Athens and her coauthors at the U.S. National Institute of Arthritis and Musculoskeletal and Skin Diseases performed a meta-analysis on studies examining survival in adult and pediatric SLE patients from the 1950s to the mid-1990s. Ultimately, they analyzed 125 adult studies, including 82 from high-income countries and 43 from low- to middle-income countries (LMIC), and 51 pediatric studies, of which 33 were from high-income countries and 18 from LMIC.
In adult studies, researchers found that both high-income and LMIC experienced gradual increases in survival from the 1950s to mid-1990s. After this period of time, the survival estimates stabilized. “In 2008–2016, the 5-year, 10-year, and 15-year survival estimates in high-income countries were 0.95 (95% credible interval, 0.94 to 0.96), 0.89 (0.88 to 0.90) and 0.82 (0.81 to 0.83), respectively” (Ann Rheum Dis. 2017 Aug 9. doi: 10.1136/annrheumdis-2017-211663).
Although there were no data for LMIC prior to 1970, researchers identified survival trends similar to those in high-income countries in more recent years. Over the same time period between 2008 and 2016, “the 5-year, 10-year, and 15-year survival estimates in LMIC were 0.92 (0.91 to 0.93), 0.85 (0.84 to 0.87) and 0.79 (0.78 to 0.81), respectively,” according to the report.
Unlike the steady improvement seen over a 40-year period with adult studies, pediatric SLE patients in high-income countries experienced dramatic increases in survival rates from the 1960s to the 1970s, followed by slower increases in survival rates. The researchers reported that between 2008 and 2016,“the 5-year and 10-year survival estimates from high-income countries were 0.99 (0.98 to 1.00) and 0.97 (0.96 to 0.98), respectively.”
LMIC had significantly worse survival in pediatric SLE patients than did their wealthy counterparts. “Survival persistently lagged [behind] that of high-income countries” between 1980 and 2000. Dr. Tektonidou and her associates found that “5-year and 10-year survival estimates from LMIC were 0.85 (0.83 to 0.88) and 0.79 (0.76 to 0.82), respectively.” Due to the small number of studies reporting 15-year survival rates, this time point was not included in the pediatric analysis.
The researchers also analyzed the cause of death for adult and pediatric SLE patients in both high-income countries and LMIC. High-income countries showed lower rates of SLE-associated deaths over time in adults, although infection-related deaths increased in adults in both high-income countries and LMIC. There were not enough studies and data to assess cause of death in pediatric studies in high-income countries, but pediatric patients in LMIC had an upward trend in SLE-associated deaths.
The Intramural Research Program of the National Institute of Arthritis and Musculoskeletal and Skin Diseases supported the study. The researchers reported having no relevant financial disclosures.
The major improvement in survival that patients with systemic lupus erythematosus (SLE) have experienced from 1950 to the mid-1990s has plateaued ever since, reported Maria Tektonidou, MD, and her colleagues. The study was published in Annals of the Rheumatic Diseases.
Dr. Tektonidou of National and Kapodistrian University of Athens and her coauthors at the U.S. National Institute of Arthritis and Musculoskeletal and Skin Diseases performed a meta-analysis on studies examining survival in adult and pediatric SLE patients from the 1950s to the mid-1990s. Ultimately, they analyzed 125 adult studies, including 82 from high-income countries and 43 from low- to middle-income countries (LMIC), and 51 pediatric studies, of which 33 were from high-income countries and 18 from LMIC.
In adult studies, researchers found that both high-income and LMIC experienced gradual increases in survival from the 1950s to mid-1990s. After this period of time, the survival estimates stabilized. “In 2008–2016, the 5-year, 10-year, and 15-year survival estimates in high-income countries were 0.95 (95% credible interval, 0.94 to 0.96), 0.89 (0.88 to 0.90) and 0.82 (0.81 to 0.83), respectively” (Ann Rheum Dis. 2017 Aug 9. doi: 10.1136/annrheumdis-2017-211663).
Although there were no data for LMIC prior to 1970, researchers identified survival trends similar to those in high-income countries in more recent years. Over the same time period between 2008 and 2016, “the 5-year, 10-year, and 15-year survival estimates in LMIC were 0.92 (0.91 to 0.93), 0.85 (0.84 to 0.87) and 0.79 (0.78 to 0.81), respectively,” according to the report.
Unlike the steady improvement seen over a 40-year period with adult studies, pediatric SLE patients in high-income countries experienced dramatic increases in survival rates from the 1960s to the 1970s, followed by slower increases in survival rates. The researchers reported that between 2008 and 2016,“the 5-year and 10-year survival estimates from high-income countries were 0.99 (0.98 to 1.00) and 0.97 (0.96 to 0.98), respectively.”
LMIC had significantly worse survival in pediatric SLE patients than did their wealthy counterparts. “Survival persistently lagged [behind] that of high-income countries” between 1980 and 2000. Dr. Tektonidou and her associates found that “5-year and 10-year survival estimates from LMIC were 0.85 (0.83 to 0.88) and 0.79 (0.76 to 0.82), respectively.” Due to the small number of studies reporting 15-year survival rates, this time point was not included in the pediatric analysis.
The researchers also analyzed the cause of death for adult and pediatric SLE patients in both high-income countries and LMIC. High-income countries showed lower rates of SLE-associated deaths over time in adults, although infection-related deaths increased in adults in both high-income countries and LMIC. There were not enough studies and data to assess cause of death in pediatric studies in high-income countries, but pediatric patients in LMIC had an upward trend in SLE-associated deaths.
The Intramural Research Program of the National Institute of Arthritis and Musculoskeletal and Skin Diseases supported the study. The researchers reported having no relevant financial disclosures.
FROM ANNALS OF THE RHEUMATIC DISEASES
Key clinical point:
Major finding: Five-year survival for SLE in adults and children in high-income countries is greater than 0.95.
Data source: Systematic literature review and Bayesian meta-analysis of 171 published cohort studies of survival in SLE patients from 1950 to the present.
Disclosures: The Intramural Research Program of the National Institute of Arthritis and Musculoskeletal and Skin Diseases supported the study. The researchers reported having no relevant financial disclosures.
Alopecia patients share their struggles
SILVER SPRING, MD. – Alopecia areata patients struggle as much, if not more so, with the social and emotional challenges of the disease as with the physical challenges, according to patients and others who spoke at a public meeting on alopecia areata patient-focused drug development.
Alopecia areata affects as many as 6.8 million individuals in the United States, according to the National Alopecia Areata Foundation (NAAF). However, the particulars of alopecia can vary widely from one person to another; some patients experience total hair loss (alopecia universalis), while others retain eyebrows, eyelashes, or some body hair.
The FDA meeting, held on Sept. 11, is part of the agency’s patient-focused drug development initiative. “We wanted to hear the broader patient’s voice,” Theresa M. Mullin, PhD, director of the FDA’s Office of Strategic Programs, said in her opening remarks. Gary Sherwood, communications director for NAAF, said that the meeting was the culmination of a 5-year effort, begun in 2012 when alopecia areata was named as one of 39 disease categories under consideration for such a meeting. “It is too early to know what the exact results will be … but if the past is any indication, they may be significant. The meeting held with psoriasis yielded FDA approval of a treatment previously denied,” he added in an interview.
Two panel presentations featured patients who discussed their experiences with alopecia; each was followed by a discussion period where patients and family members in the audience were invited to share their experiences.
The “Health Effects and Daily Impacts” panel allowed several patients and their family members the opportunity to identify specific issues that may surprise clinicians.
“One thing I learned was how much the patients are bothered by sweating of the scalp; this can affect what type of head covering, hair piece, or hat/helmet they are able to wear, and thus limits activities,” Dr. Marathe continued. “This is not something I had focused on previously. I will be more inclined to ask about sweating and offer treatments, such as scalp botulinum toxin or aluminum chloride now that I have been alerted to this concern. Also, the challenges of facial makeup such as pencil for eyebrows was another thing that the FDA session brought home for me; I’m more inclined to suggest things such as microblading for eyebrows, or to try treatments like latanoprost for eyebrows/lashes.”
The second panel, “Current Approaches to Treatment,” included a different group of patients who shared stories of treatments that had been successful and those that had not. “The patients at the FDA meeting expressed very eloquently what our patients feel – different treatments may work temporarily and then stop working, which leads to a roller coaster of emotions of hope and disappointment,” A. Yasmine Kirkorian, MD, also a dermatologist at Children’s National Health System, said in an interview. “Patients and physicians would be interested in a treatment option with a track record for predictable efficacy with durable and sustained hair regrowth and minimal side effects.”
Dr. Marathe noted that in her experience, those who develop alopecia totalis or universalis at a younger age tend to have more recalcitrant disease. “It is still very hard for me to predict which children will regrow their hair spontaneously, or with topical therapies, versus those with more resistant disease. I hope that continued study will allow us to offer a more realistic prognosis for these patients,” she said.
Discussion after the treatment panel included testimonials from patients who reported successful treatment with tofacitinib (Xeljanz), a Janus kinase inhibitor approved for rheumatoid arthritis, which is not approved for treatment of alopecia.
“I absolutely agree with the focus on JAK inhibitors and increasing our understanding of how they work, as well as what some of the long-term effects are,” said Dr. Marathe. “The better we are able to target the pathogenesis of this condition, the more easily we can treat in a more focused fashion and reduce side effects,” but more clinical trials are needed to determine safety and efficacy for children and teens, she noted.
One of her hesitations in prescribing tofacitinib to her patients is that she cannot provide them with a sense of how long they will need to be on the treatment. “Current data show that the hair growth on the medication is usually lost upon stopping it; the question I still struggle with is whether it is realistic to put a 4- or 5-year-old on a medication that has no estimated or anticipated stop date,” she said.
As for what she offers patients in terms of resources for emotional support, Dr. Kirkorian said the psychosocial aspects of alopecia areata are always discussed at patient visits. “Psychosocial needs vary based on age, personality, and personal philosophy. We offer the gamut of outside resources from local support groups, the National Alopecia Areata Foundation, referral to psychology/psychiatry and, very importantly, referral to Camp Discovery. Children have told us across the board how important and meaningful it was to them to be able to just be themselves around other children who look like them.”
Dr. Marathe and Dr. Kirkorian were attendees at the meeting; they had no relevant disclosures. They are members of the Dermatology News Editorial Advisory Board.
SILVER SPRING, MD. – Alopecia areata patients struggle as much, if not more so, with the social and emotional challenges of the disease as with the physical challenges, according to patients and others who spoke at a public meeting on alopecia areata patient-focused drug development.
Alopecia areata affects as many as 6.8 million individuals in the United States, according to the National Alopecia Areata Foundation (NAAF). However, the particulars of alopecia can vary widely from one person to another; some patients experience total hair loss (alopecia universalis), while others retain eyebrows, eyelashes, or some body hair.
The FDA meeting, held on Sept. 11, is part of the agency’s patient-focused drug development initiative. “We wanted to hear the broader patient’s voice,” Theresa M. Mullin, PhD, director of the FDA’s Office of Strategic Programs, said in her opening remarks. Gary Sherwood, communications director for NAAF, said that the meeting was the culmination of a 5-year effort, begun in 2012 when alopecia areata was named as one of 39 disease categories under consideration for such a meeting. “It is too early to know what the exact results will be … but if the past is any indication, they may be significant. The meeting held with psoriasis yielded FDA approval of a treatment previously denied,” he added in an interview.
Two panel presentations featured patients who discussed their experiences with alopecia; each was followed by a discussion period where patients and family members in the audience were invited to share their experiences.
The “Health Effects and Daily Impacts” panel allowed several patients and their family members the opportunity to identify specific issues that may surprise clinicians.
“One thing I learned was how much the patients are bothered by sweating of the scalp; this can affect what type of head covering, hair piece, or hat/helmet they are able to wear, and thus limits activities,” Dr. Marathe continued. “This is not something I had focused on previously. I will be more inclined to ask about sweating and offer treatments, such as scalp botulinum toxin or aluminum chloride now that I have been alerted to this concern. Also, the challenges of facial makeup such as pencil for eyebrows was another thing that the FDA session brought home for me; I’m more inclined to suggest things such as microblading for eyebrows, or to try treatments like latanoprost for eyebrows/lashes.”
The second panel, “Current Approaches to Treatment,” included a different group of patients who shared stories of treatments that had been successful and those that had not. “The patients at the FDA meeting expressed very eloquently what our patients feel – different treatments may work temporarily and then stop working, which leads to a roller coaster of emotions of hope and disappointment,” A. Yasmine Kirkorian, MD, also a dermatologist at Children’s National Health System, said in an interview. “Patients and physicians would be interested in a treatment option with a track record for predictable efficacy with durable and sustained hair regrowth and minimal side effects.”
Dr. Marathe noted that in her experience, those who develop alopecia totalis or universalis at a younger age tend to have more recalcitrant disease. “It is still very hard for me to predict which children will regrow their hair spontaneously, or with topical therapies, versus those with more resistant disease. I hope that continued study will allow us to offer a more realistic prognosis for these patients,” she said.
Discussion after the treatment panel included testimonials from patients who reported successful treatment with tofacitinib (Xeljanz), a Janus kinase inhibitor approved for rheumatoid arthritis, which is not approved for treatment of alopecia.
“I absolutely agree with the focus on JAK inhibitors and increasing our understanding of how they work, as well as what some of the long-term effects are,” said Dr. Marathe. “The better we are able to target the pathogenesis of this condition, the more easily we can treat in a more focused fashion and reduce side effects,” but more clinical trials are needed to determine safety and efficacy for children and teens, she noted.
One of her hesitations in prescribing tofacitinib to her patients is that she cannot provide them with a sense of how long they will need to be on the treatment. “Current data show that the hair growth on the medication is usually lost upon stopping it; the question I still struggle with is whether it is realistic to put a 4- or 5-year-old on a medication that has no estimated or anticipated stop date,” she said.
As for what she offers patients in terms of resources for emotional support, Dr. Kirkorian said the psychosocial aspects of alopecia areata are always discussed at patient visits. “Psychosocial needs vary based on age, personality, and personal philosophy. We offer the gamut of outside resources from local support groups, the National Alopecia Areata Foundation, referral to psychology/psychiatry and, very importantly, referral to Camp Discovery. Children have told us across the board how important and meaningful it was to them to be able to just be themselves around other children who look like them.”
Dr. Marathe and Dr. Kirkorian were attendees at the meeting; they had no relevant disclosures. They are members of the Dermatology News Editorial Advisory Board.
SILVER SPRING, MD. – Alopecia areata patients struggle as much, if not more so, with the social and emotional challenges of the disease as with the physical challenges, according to patients and others who spoke at a public meeting on alopecia areata patient-focused drug development.
Alopecia areata affects as many as 6.8 million individuals in the United States, according to the National Alopecia Areata Foundation (NAAF). However, the particulars of alopecia can vary widely from one person to another; some patients experience total hair loss (alopecia universalis), while others retain eyebrows, eyelashes, or some body hair.
The FDA meeting, held on Sept. 11, is part of the agency’s patient-focused drug development initiative. “We wanted to hear the broader patient’s voice,” Theresa M. Mullin, PhD, director of the FDA’s Office of Strategic Programs, said in her opening remarks. Gary Sherwood, communications director for NAAF, said that the meeting was the culmination of a 5-year effort, begun in 2012 when alopecia areata was named as one of 39 disease categories under consideration for such a meeting. “It is too early to know what the exact results will be … but if the past is any indication, they may be significant. The meeting held with psoriasis yielded FDA approval of a treatment previously denied,” he added in an interview.
Two panel presentations featured patients who discussed their experiences with alopecia; each was followed by a discussion period where patients and family members in the audience were invited to share their experiences.
The “Health Effects and Daily Impacts” panel allowed several patients and their family members the opportunity to identify specific issues that may surprise clinicians.
“One thing I learned was how much the patients are bothered by sweating of the scalp; this can affect what type of head covering, hair piece, or hat/helmet they are able to wear, and thus limits activities,” Dr. Marathe continued. “This is not something I had focused on previously. I will be more inclined to ask about sweating and offer treatments, such as scalp botulinum toxin or aluminum chloride now that I have been alerted to this concern. Also, the challenges of facial makeup such as pencil for eyebrows was another thing that the FDA session brought home for me; I’m more inclined to suggest things such as microblading for eyebrows, or to try treatments like latanoprost for eyebrows/lashes.”
The second panel, “Current Approaches to Treatment,” included a different group of patients who shared stories of treatments that had been successful and those that had not. “The patients at the FDA meeting expressed very eloquently what our patients feel – different treatments may work temporarily and then stop working, which leads to a roller coaster of emotions of hope and disappointment,” A. Yasmine Kirkorian, MD, also a dermatologist at Children’s National Health System, said in an interview. “Patients and physicians would be interested in a treatment option with a track record for predictable efficacy with durable and sustained hair regrowth and minimal side effects.”
Dr. Marathe noted that in her experience, those who develop alopecia totalis or universalis at a younger age tend to have more recalcitrant disease. “It is still very hard for me to predict which children will regrow their hair spontaneously, or with topical therapies, versus those with more resistant disease. I hope that continued study will allow us to offer a more realistic prognosis for these patients,” she said.
Discussion after the treatment panel included testimonials from patients who reported successful treatment with tofacitinib (Xeljanz), a Janus kinase inhibitor approved for rheumatoid arthritis, which is not approved for treatment of alopecia.
“I absolutely agree with the focus on JAK inhibitors and increasing our understanding of how they work, as well as what some of the long-term effects are,” said Dr. Marathe. “The better we are able to target the pathogenesis of this condition, the more easily we can treat in a more focused fashion and reduce side effects,” but more clinical trials are needed to determine safety and efficacy for children and teens, she noted.
One of her hesitations in prescribing tofacitinib to her patients is that she cannot provide them with a sense of how long they will need to be on the treatment. “Current data show that the hair growth on the medication is usually lost upon stopping it; the question I still struggle with is whether it is realistic to put a 4- or 5-year-old on a medication that has no estimated or anticipated stop date,” she said.
As for what she offers patients in terms of resources for emotional support, Dr. Kirkorian said the psychosocial aspects of alopecia areata are always discussed at patient visits. “Psychosocial needs vary based on age, personality, and personal philosophy. We offer the gamut of outside resources from local support groups, the National Alopecia Areata Foundation, referral to psychology/psychiatry and, very importantly, referral to Camp Discovery. Children have told us across the board how important and meaningful it was to them to be able to just be themselves around other children who look like them.”
Dr. Marathe and Dr. Kirkorian were attendees at the meeting; they had no relevant disclosures. They are members of the Dermatology News Editorial Advisory Board.
AT AN FDA PUBLIC MEETING