Despite multiple existing conventional synthetic disease-modifying antirheumatic drug (csDMARD) and biologic DMARD (bDMARD) options, many patients with rheumatoid arthritis (RA) do not respond adequately to treatment. In an exciting development, a recent phase 2 study by Tuttle and colleagues examined a novel treatment approach in RA: stimulation of the programmed cell death protein 1 (PD-1) inhibitor pathway. PD-1 is a checkpoint inhibitor receptor whose activation reflects T-cell activation and may play a role in synovitis and extra-articular inflammation. Blocking PD-1 in cancer therapy has been associated with an increase in inflammatory arthritis. In this 12-week study, RA disease activity was analyzed in patients randomly assigned to two different monthly intravenous doses of peresolimab or placebo. Of note, a large majority of participants were seropositive for rheumatoid factor (RF) or cyclic citrullinated peptide (CCP). Patients receiving the 700-mg dose of peresolimab had a better American College of Rheumatology (ACR) 20 response than did those receiving placebo (71% vs 42%), but not a better ACR50 or ACR70 response; the 300-mg dose was not better than placebo. Although reported adverse events were similar in all three groups, with a short timeframe it would be difficult to address concerns about cancer risk. Though this novel treatment is exciting, a larger and longer-term trial is necessary to address this concern as well as potentially tease out risk factors (including age or other immunosuppression) in this susceptible group.

Two other studies examined use of a much older csDMARD therapy, hydroxychloroquine (HCQ), in Brazilian patients with RA. Bredemeier and colleagues looked at the effects of HCQ on adverse events as well as the persistence of bDMARD/targeted synthetic DMARD (tsDMARD) therapy in over 1300 patients with RA. Using the BiobadaBrasil registry of patients starting their first bDMARD or Janus kinase (JAK) inhibitor, they looked at effects of combination therapy with HCQ during the treatment course of up to six bDMARD or JAK inhibitors. At baseline, patients prescribed antimalarial therapy had shorter RA duration and began treatment earlier, perhaps due to patient or physician preferences regarding starting "milder" antimalarial medication earlier or due to use of "triple therapy" with methotrexate and sulfasalazine. Of interest, patients receiving antimalarial therapy had a lower incidence of adverse events, especially serious infections, but no effect on cardiovascular events was seen despite HCQ's perceived beneficial effects on thrombotic risk and cholesterol profile. Patients receiving HCQ were also more likely to persist in their course of bDMARD or JAK inhibitor therapy, though the effect size seems relatively small. As the focus in this study was on adverse effects, the authors' analysis of the effects on antimalarials on the persistence of therapy was not detailed.

Lin and colleagues also looked at the effects of HCQ in patients with older-onset RA with respect to mortality risk. Using data from the electronic health records of a hospital in Taiwan, mortality-associated risk factors were evaluated in 980 patients with RA diagnosed at >60 years. Male sex, current smoking status, and cancer status were all associated with mortality, whereas HCQ use was associated with reduced mortality (hazard ratio 0.30). In contrast to the registry study mentioned above, patients receiving HCQ had a lower risk for cardiovascular events, hyperlipidemia, diabetes, and chronic kidney disease. Interaction with cancer was less clear due to lower number of patients. Of interest, use of cyclosporine, leflunomide, and a bDMARD was associated with higher mortality risk. The source and true relevance of the potential risk reduction in this study is not clear because of the lack of prospective data, but combined with the information above, this study suggests that the benefits of HCQ use should not be discounted in patients with RA.

Despite multiple existing conventional synthetic disease-modifying antirheumatic drug (csDMARD) and biologic DMARD (bDMARD) options, many patients with rheumatoid arthritis (RA) do not respond adequately to treatment. In an exciting development, a recent phase 2 study by Tuttle and colleagues examined a novel treatment approach in RA: stimulation of the programmed cell death protein 1 (PD-1) inhibitor pathway. PD-1 is a checkpoint inhibitor receptor whose activation reflects T-cell activation and may play a role in synovitis and extra-articular inflammation. Blocking PD-1 in cancer therapy has been associated with an increase in inflammatory arthritis. In this 12-week study, RA disease activity was analyzed in patients randomly assigned to two different monthly intravenous doses of peresolimab or placebo. Of note, a large majority of participants were seropositive for rheumatoid factor (RF) or cyclic citrullinated peptide (CCP). Patients receiving the 700-mg dose of peresolimab had a better American College of Rheumatology (ACR) 20 response than did those receiving placebo (71% vs 42%), but not a better ACR50 or ACR70 response; the 300-mg dose was not better than placebo. Although reported adverse events were similar in all three groups, with a short timeframe it would be difficult to address concerns about cancer risk. Though this novel treatment is exciting, a larger and longer-term trial is necessary to address this concern as well as potentially tease out risk factors (including age or other immunosuppression) in this susceptible group.

Two other studies examined use of a much older csDMARD therapy, hydroxychloroquine (HCQ), in Brazilian patients with RA. Bredemeier and colleagues looked at the effects of HCQ on adverse events as well as the persistence of bDMARD/targeted synthetic DMARD (tsDMARD) therapy in over 1300 patients with RA. Using the BiobadaBrasil registry of patients starting their first bDMARD or Janus kinase (JAK) inhibitor, they looked at effects of combination therapy with HCQ during the treatment course of up to six bDMARD or JAK inhibitors. At baseline, patients prescribed antimalarial therapy had shorter RA duration and began treatment earlier, perhaps due to patient or physician preferences regarding starting "milder" antimalarial medication earlier or due to use of "triple therapy" with methotrexate and sulfasalazine. Of interest, patients receiving antimalarial therapy had a lower incidence of adverse events, especially serious infections, but no effect on cardiovascular events was seen despite HCQ's perceived beneficial effects on thrombotic risk and cholesterol profile. Patients receiving HCQ were also more likely to persist in their course of bDMARD or JAK inhibitor therapy, though the effect size seems relatively small. As the focus in this study was on adverse effects, the authors' analysis of the effects on antimalarials on the persistence of therapy was not detailed.

Lin and colleagues also looked at the effects of HCQ in patients with older-onset RA with respect to mortality risk. Using data from the electronic health records of a hospital in Taiwan, mortality-associated risk factors were evaluated in 980 patients with RA diagnosed at >60 years. Male sex, current smoking status, and cancer status were all associated with mortality, whereas HCQ use was associated with reduced mortality (hazard ratio 0.30). In contrast to the registry study mentioned above, patients receiving HCQ had a lower risk for cardiovascular events, hyperlipidemia, diabetes, and chronic kidney disease. Interaction with cancer was less clear due to lower number of patients. Of interest, use of cyclosporine, leflunomide, and a bDMARD was associated with higher mortality risk. The source and true relevance of the potential risk reduction in this study is not clear because of the lack of prospective data, but combined with the information above, this study suggests that the benefits of HCQ use should not be discounted in patients with RA.

Despite multiple existing conventional synthetic disease-modifying antirheumatic drug (csDMARD) and biologic DMARD (bDMARD) options, many patients with rheumatoid arthritis (RA) do not respond adequately to treatment. In an exciting development, a recent phase 2 study by Tuttle and colleagues examined a novel treatment approach in RA: stimulation of the programmed cell death protein 1 (PD-1) inhibitor pathway. PD-1 is a checkpoint inhibitor receptor whose activation reflects T-cell activation and may play a role in synovitis and extra-articular inflammation. Blocking PD-1 in cancer therapy has been associated with an increase in inflammatory arthritis. In this 12-week study, RA disease activity was analyzed in patients randomly assigned to two different monthly intravenous doses of peresolimab or placebo. Of note, a large majority of participants were seropositive for rheumatoid factor (RF) or cyclic citrullinated peptide (CCP). Patients receiving the 700-mg dose of peresolimab had a better American College of Rheumatology (ACR) 20 response than did those receiving placebo (71% vs 42%), but not a better ACR50 or ACR70 response; the 300-mg dose was not better than placebo. Although reported adverse events were similar in all three groups, with a short timeframe it would be difficult to address concerns about cancer risk. Though this novel treatment is exciting, a larger and longer-term trial is necessary to address this concern as well as potentially tease out risk factors (including age or other immunosuppression) in this susceptible group.

Two other studies examined use of a much older csDMARD therapy, hydroxychloroquine (HCQ), in Brazilian patients with RA. Bredemeier and colleagues looked at the effects of HCQ on adverse events as well as the persistence of bDMARD/targeted synthetic DMARD (tsDMARD) therapy in over 1300 patients with RA. Using the BiobadaBrasil registry of patients starting their first bDMARD or Janus kinase (JAK) inhibitor, they looked at effects of combination therapy with HCQ during the treatment course of up to six bDMARD or JAK inhibitors. At baseline, patients prescribed antimalarial therapy had shorter RA duration and began treatment earlier, perhaps due to patient or physician preferences regarding starting "milder" antimalarial medication earlier or due to use of "triple therapy" with methotrexate and sulfasalazine. Of interest, patients receiving antimalarial therapy had a lower incidence of adverse events, especially serious infections, but no effect on cardiovascular events was seen despite HCQ's perceived beneficial effects on thrombotic risk and cholesterol profile. Patients receiving HCQ were also more likely to persist in their course of bDMARD or JAK inhibitor therapy, though the effect size seems relatively small. As the focus in this study was on adverse effects, the authors' analysis of the effects on antimalarials on the persistence of therapy was not detailed.

Lin and colleagues also looked at the effects of HCQ in patients with older-onset RA with respect to mortality risk. Using data from the electronic health records of a hospital in Taiwan, mortality-associated risk factors were evaluated in 980 patients with RA diagnosed at >60 years. Male sex, current smoking status, and cancer status were all associated with mortality, whereas HCQ use was associated with reduced mortality (hazard ratio 0.30). In contrast to the registry study mentioned above, patients receiving HCQ had a lower risk for cardiovascular events, hyperlipidemia, diabetes, and chronic kidney disease. Interaction with cancer was less clear due to lower number of patients. Of interest, use of cyclosporine, leflunomide, and a bDMARD was associated with higher mortality risk. The source and true relevance of the potential risk reduction in this study is not clear because of the lack of prospective data, but combined with the information above, this study suggests that the benefits of HCQ use should not be discounted in patients with RA.

After a median follow-up of 21.6 mo, the dalpiciclib group demonstrated a significantly longer median progression-free survival (PFS) compared with the placebo group (30.6 mo vs 18.2 mo; stratified hazard ratio [HR] 0.51; 95% CI 0.38-0.69; P < .0001). Overall, the dalpiciclib group demonstrated a manageable safety profile, although a higher percentage of grade 3/4 adverse events was noted with dalpiciclib than with placebo (90% vs 12%), as expected. Overall survival data for this CDK4/6 inhibitor are yet to come. These results suggest that dalpiciclib in combination with endocrine therapy is an alternative treatment for this group of patients, especially in countries where the traditionally approved CDK4/6 inhibitors (palbociclib, ribociclib, and abemaciclib) are not available.

The optimal sequencing of endocrine therapy (ET) after progression on CDK4/6 inhibitor–based therapy remains a challenge. In the phase 2 MAINTAIN trial, 119 patients (all of whom had HR+/HER2- metastatic breast cancer and who progressed on ET and CDK4/6 inhibitors) were randomly assigned to receive a different ET (fulvestrant or exemestane) from the previous ET they had received plus either the CDK4/6 inhibitor ribociclib or placebo. In the study by Kalinksky and colleagues, at a median follow-up of 18.2 mo, a significant improvement in PFS was observed in the switched ET-plus-ribociclib group compared with the switched ET-plus-placebo group (HR 0.57; P = .006). The phase 2 MAINTAIN trial is the first randomized trial to show the benefit of a CDK4/6 inhibitor after progression on another CDK4/6 inhibitor. It is important to note that the majority of patients in the MAINTAIN study previously received palbociclib in the first-line setting, which in recent studies has been demonstrated to be inferior to other CDK4/6 inhibitors. Therefore, it is important to confirm whether this will hold true upon progression from ribociclib or abemaciclib in the first-line setting. In addition, more data are needed to compare this approach with other ET treatment options, such as phosphoinositide 3-kinases inhibitors and oral selective estrogen receptor degraders.

There are several options for adjuvant radiation therapy for early-stage breast cancer. A meta-analysis of 14 randomized controlled trials and six comparative observational studies assessed the efficacy of whole breast irradiation (WBI) compared with partial breast irradiation (PBI) in 17,234 adults with early-stage breast cancer. Results of this meta-analysis showed that PBI was not significantly different from WBI, with similar rates of ipsilateral breast recurrence  at 5 years (relative risk [RR] 1.34; 95% CI 0.83-2.18) and 10 years (RR 1.29; 95% CI 0.87-1.91), although patients undergoing PBI reported fewer acute adverse events  compared with patients undergoing WBI (incidence rate ratio [IRR] 0.53; 95% CI 0.31-0.92) and acute grade ≥2 adverse events (IRR 0.21; 95% CI 0.07-0.62). These findings support using PBI as the adjuvant radiotherapy modality for select patients with favorable-risk early-stage breast cancer.

Another meta-analysis looked at assessing the survival benefit of adding CDK4/6 inhibitors to standard ET in older patients with advanced breast cancer. The study included 10 trials with 1985 older patients with advanced ER+ breast cancer who received ET with or without CDK4/6 inhibitors. The findings showed that adding CDK4/6 inhibitors to ET (letrozole or fulvestrant) significantly reduced the mortality risk by 21% (HR 0.79; 95% CI 0.69-0.91) and progression risk by 41% (HR 0.59; 95% CI 0.51-0.69) in older patients (age ≥ 65 years) with advanced breast cancer. Grade 3-4 neutropenia and diarrhea were similar in older patients. This study supports the use of CDK4/6 inhibitors as a reasonable treatment modality for older patients. More studies dedicated to the geriatric population are needed to help elaborate on the efficacy and tolerability of such agents in this population.

The phase 3 National Surgical Adjuvant Breast and Bowel Project B-42 (NSABP B-42) trial evaluated the role of extended letrozole therapy in postmenopausal breast cancer patients who were disease-free after 5 years of aromatase inhibitor–based therapy. The study included 3966 postmenopausal women with stage I-IIIA HR+ breast cancer who were randomly assigned to receive letrozole or placebo for 5 more years. After a median follow-up of 10.3 years, letrozole significantly improved disease-free survival (10-year absolute benefit 3.4%; HR 0.85; P = .01) compared with placebo, although there were no differences noted in overall survival between the groups (HR 0.97, P = .74). Furthermore, letrozole significantly reduced the breast cancer–free interval (HR 0.75, ,P = .003) and distant recurrence (HR 0.72, P = .01). There were no notable differences in toxicity, particularly rates of osteoporotic fractures and arterial thrombotic events, between the groups. Extended therapy with aromatase inhibitors beyond 5 years can be considered for select patients with early-stage breast cancer. Careful consideration of risks and benefits is needed to make these recommendations. 

After a median follow-up of 21.6 mo, the dalpiciclib group demonstrated a significantly longer median progression-free survival (PFS) compared with the placebo group (30.6 mo vs 18.2 mo; stratified hazard ratio [HR] 0.51; 95% CI 0.38-0.69; P < .0001). Overall, the dalpiciclib group demonstrated a manageable safety profile, although a higher percentage of grade 3/4 adverse events was noted with dalpiciclib than with placebo (90% vs 12%), as expected. Overall survival data for this CDK4/6 inhibitor are yet to come. These results suggest that dalpiciclib in combination with endocrine therapy is an alternative treatment for this group of patients, especially in countries where the traditionally approved CDK4/6 inhibitors (palbociclib, ribociclib, and abemaciclib) are not available.

The optimal sequencing of endocrine therapy (ET) after progression on CDK4/6 inhibitor–based therapy remains a challenge. In the phase 2 MAINTAIN trial, 119 patients (all of whom had HR+/HER2- metastatic breast cancer and who progressed on ET and CDK4/6 inhibitors) were randomly assigned to receive a different ET (fulvestrant or exemestane) from the previous ET they had received plus either the CDK4/6 inhibitor ribociclib or placebo. In the study by Kalinksky and colleagues, at a median follow-up of 18.2 mo, a significant improvement in PFS was observed in the switched ET-plus-ribociclib group compared with the switched ET-plus-placebo group (HR 0.57; P = .006). The phase 2 MAINTAIN trial is the first randomized trial to show the benefit of a CDK4/6 inhibitor after progression on another CDK4/6 inhibitor. It is important to note that the majority of patients in the MAINTAIN study previously received palbociclib in the first-line setting, which in recent studies has been demonstrated to be inferior to other CDK4/6 inhibitors. Therefore, it is important to confirm whether this will hold true upon progression from ribociclib or abemaciclib in the first-line setting. In addition, more data are needed to compare this approach with other ET treatment options, such as phosphoinositide 3-kinases inhibitors and oral selective estrogen receptor degraders.

There are several options for adjuvant radiation therapy for early-stage breast cancer. A meta-analysis of 14 randomized controlled trials and six comparative observational studies assessed the efficacy of whole breast irradiation (WBI) compared with partial breast irradiation (PBI) in 17,234 adults with early-stage breast cancer. Results of this meta-analysis showed that PBI was not significantly different from WBI, with similar rates of ipsilateral breast recurrence  at 5 years (relative risk [RR] 1.34; 95% CI 0.83-2.18) and 10 years (RR 1.29; 95% CI 0.87-1.91), although patients undergoing PBI reported fewer acute adverse events  compared with patients undergoing WBI (incidence rate ratio [IRR] 0.53; 95% CI 0.31-0.92) and acute grade ≥2 adverse events (IRR 0.21; 95% CI 0.07-0.62). These findings support using PBI as the adjuvant radiotherapy modality for select patients with favorable-risk early-stage breast cancer.

Another meta-analysis looked at assessing the survival benefit of adding CDK4/6 inhibitors to standard ET in older patients with advanced breast cancer. The study included 10 trials with 1985 older patients with advanced ER+ breast cancer who received ET with or without CDK4/6 inhibitors. The findings showed that adding CDK4/6 inhibitors to ET (letrozole or fulvestrant) significantly reduced the mortality risk by 21% (HR 0.79; 95% CI 0.69-0.91) and progression risk by 41% (HR 0.59; 95% CI 0.51-0.69) in older patients (age ≥ 65 years) with advanced breast cancer. Grade 3-4 neutropenia and diarrhea were similar in older patients. This study supports the use of CDK4/6 inhibitors as a reasonable treatment modality for older patients. More studies dedicated to the geriatric population are needed to help elaborate on the efficacy and tolerability of such agents in this population.

The phase 3 National Surgical Adjuvant Breast and Bowel Project B-42 (NSABP B-42) trial evaluated the role of extended letrozole therapy in postmenopausal breast cancer patients who were disease-free after 5 years of aromatase inhibitor–based therapy. The study included 3966 postmenopausal women with stage I-IIIA HR+ breast cancer who were randomly assigned to receive letrozole or placebo for 5 more years. After a median follow-up of 10.3 years, letrozole significantly improved disease-free survival (10-year absolute benefit 3.4%; HR 0.85; P = .01) compared with placebo, although there were no differences noted in overall survival between the groups (HR 0.97, P = .74). Furthermore, letrozole significantly reduced the breast cancer–free interval (HR 0.75, ,P = .003) and distant recurrence (HR 0.72, P = .01). There were no notable differences in toxicity, particularly rates of osteoporotic fractures and arterial thrombotic events, between the groups. Extended therapy with aromatase inhibitors beyond 5 years can be considered for select patients with early-stage breast cancer. Careful consideration of risks and benefits is needed to make these recommendations. 

After a median follow-up of 21.6 mo, the dalpiciclib group demonstrated a significantly longer median progression-free survival (PFS) compared with the placebo group (30.6 mo vs 18.2 mo; stratified hazard ratio [HR] 0.51; 95% CI 0.38-0.69; P < .0001). Overall, the dalpiciclib group demonstrated a manageable safety profile, although a higher percentage of grade 3/4 adverse events was noted with dalpiciclib than with placebo (90% vs 12%), as expected. Overall survival data for this CDK4/6 inhibitor are yet to come. These results suggest that dalpiciclib in combination with endocrine therapy is an alternative treatment for this group of patients, especially in countries where the traditionally approved CDK4/6 inhibitors (palbociclib, ribociclib, and abemaciclib) are not available.

The optimal sequencing of endocrine therapy (ET) after progression on CDK4/6 inhibitor–based therapy remains a challenge. In the phase 2 MAINTAIN trial, 119 patients (all of whom had HR+/HER2- metastatic breast cancer and who progressed on ET and CDK4/6 inhibitors) were randomly assigned to receive a different ET (fulvestrant or exemestane) from the previous ET they had received plus either the CDK4/6 inhibitor ribociclib or placebo. In the study by Kalinksky and colleagues, at a median follow-up of 18.2 mo, a significant improvement in PFS was observed in the switched ET-plus-ribociclib group compared with the switched ET-plus-placebo group (HR 0.57; P = .006). The phase 2 MAINTAIN trial is the first randomized trial to show the benefit of a CDK4/6 inhibitor after progression on another CDK4/6 inhibitor. It is important to note that the majority of patients in the MAINTAIN study previously received palbociclib in the first-line setting, which in recent studies has been demonstrated to be inferior to other CDK4/6 inhibitors. Therefore, it is important to confirm whether this will hold true upon progression from ribociclib or abemaciclib in the first-line setting. In addition, more data are needed to compare this approach with other ET treatment options, such as phosphoinositide 3-kinases inhibitors and oral selective estrogen receptor degraders.

There are several options for adjuvant radiation therapy for early-stage breast cancer. A meta-analysis of 14 randomized controlled trials and six comparative observational studies assessed the efficacy of whole breast irradiation (WBI) compared with partial breast irradiation (PBI) in 17,234 adults with early-stage breast cancer. Results of this meta-analysis showed that PBI was not significantly different from WBI, with similar rates of ipsilateral breast recurrence  at 5 years (relative risk [RR] 1.34; 95% CI 0.83-2.18) and 10 years (RR 1.29; 95% CI 0.87-1.91), although patients undergoing PBI reported fewer acute adverse events  compared with patients undergoing WBI (incidence rate ratio [IRR] 0.53; 95% CI 0.31-0.92) and acute grade ≥2 adverse events (IRR 0.21; 95% CI 0.07-0.62). These findings support using PBI as the adjuvant radiotherapy modality for select patients with favorable-risk early-stage breast cancer.

Another meta-analysis looked at assessing the survival benefit of adding CDK4/6 inhibitors to standard ET in older patients with advanced breast cancer. The study included 10 trials with 1985 older patients with advanced ER+ breast cancer who received ET with or without CDK4/6 inhibitors. The findings showed that adding CDK4/6 inhibitors to ET (letrozole or fulvestrant) significantly reduced the mortality risk by 21% (HR 0.79; 95% CI 0.69-0.91) and progression risk by 41% (HR 0.59; 95% CI 0.51-0.69) in older patients (age ≥ 65 years) with advanced breast cancer. Grade 3-4 neutropenia and diarrhea were similar in older patients. This study supports the use of CDK4/6 inhibitors as a reasonable treatment modality for older patients. More studies dedicated to the geriatric population are needed to help elaborate on the efficacy and tolerability of such agents in this population.

The phase 3 National Surgical Adjuvant Breast and Bowel Project B-42 (NSABP B-42) trial evaluated the role of extended letrozole therapy in postmenopausal breast cancer patients who were disease-free after 5 years of aromatase inhibitor–based therapy. The study included 3966 postmenopausal women with stage I-IIIA HR+ breast cancer who were randomly assigned to receive letrozole or placebo for 5 more years. After a median follow-up of 10.3 years, letrozole significantly improved disease-free survival (10-year absolute benefit 3.4%; HR 0.85; P = .01) compared with placebo, although there were no differences noted in overall survival between the groups (HR 0.97, P = .74). Furthermore, letrozole significantly reduced the breast cancer–free interval (HR 0.75, ,P = .003) and distant recurrence (HR 0.72, P = .01). There were no notable differences in toxicity, particularly rates of osteoporotic fractures and arterial thrombotic events, between the groups. Extended therapy with aromatase inhibitors beyond 5 years can be considered for select patients with early-stage breast cancer. Careful consideration of risks and benefits is needed to make these recommendations. 

An automated artificial intelligence (AI) model trained to read electroencephalograms (EEGs) in patients with suspected epilepsy is just as accurate as trained neurologists, new data suggest.

Known as SCORE-AI, the technology distinguishes between abnormal and normal EEG recordings and classifies irregular recordings into specific categories crucial for patient decision-making.

“SCORE-AI can be used in place of experts in underprivileged areas, where expertise is missing, or to help physicians to preselect or prescore recordings in areas where the workload is high – we can all benefit from AI,” study investigator Sándor Beniczky, MD, PhD, said in a JAMA Neurology podcast.

Dr. Beniczky is professor of clinical neurophysiology at Aarhus University in Denmark.

The findings were published online in JAMA Neurology.
 

Gaining a foothold

Increasingly, AI is gaining a foothold in medicine by credibly addressing patient queries and aiding radiologists.

To bring AI to EEG interpretation, the researchers developed and validated an AI model that was able to assess routine, clinical EEGs in patients with suspected epilepsy.

Beyond using AI to distinguish abnormal from normal EEG recordings, the researchers wanted to train the new system to classify abnormal recordings into the major categories that are most relevant for clinical decision-making in patients who may have epilepsy. The categories included epileptiform-focal, epileptiform-generalized, nonepileptiform-focal, and nonepileptiform-diffuse abnormalities.

The researchers trained the learning model using Standardized Computer-based Organized Reporting of EEG (SCORE) software.

In the development phase, the model was trained using more than 30,490 anonymized and highly annotated EEG recordings from 14,100 men (median age, 25 years) from a single center. The recordings had an average duration of 31 minutes and were interpreted by 17 neurologists using standardized criteria. If an EEG recording was abnormal, the physicians had to specify which abnormal features were present.

SCORE-AI then performed an analysis of the recordings based on input from the experts.

To validate the findings, investigators used two independent test datasets. The first dataset consisted of 100 representative routine EEGs from 61 men (median age, 26 years), evaluated by 11 neurologists from different centers.

The consensus of these evaluations served as the reference standard. The second dataset comprised nearly 10,000 EEGs from a single center (5,170 men; median age, 35 years), independently assessed by 14 neurologists.
 

Near-perfect accuracy

When compared with the experts, SCORE-AI had near-perfect accuracy with an area under the receiver operating characteristic (AUROC) curve for differentiating normal from abnormal EEG recordings of 0.95.

SCORE-AI also performed well at identifying generalized epileptiform abnormalities (AUROC, 0.96), focal epileptiform abnormalities (AUROC, 0.91), focal nonepileptiform abnormalities (AUROC, 0.89), and diffuse nonepileptiform abnormalities (AUROC, 0.93).

In addition, SCORE-AI had excellent agreement with clinicians – and sometimes agreed with individual experts more than the experts agreed with one another.

When Dr. Beniczky and team tested SCORE-AI against three previously published AI models, SCORE-AI demonstrated greater specificity than those models (90% vs. 3%-63%) but was not as sensitive (86.7%) as two of the models (96.7% and 100%).

One of the study’s limitations was the fact that SCORE-AI was developed and validated on routine EEGs that excluded neonates and critically ill patients.

In the future, Dr. Beniczky said on the podcast, the team would like to train SCORE-AI to read EEGs with more granularity, and eventually use only one single channel to record EEGs. At present, SCORE-AI is being integrated with Natus Neuro, a widely used EEG equipment system, the investigators note.

In an accompanying editorial, Jonathan Kleen, MD, PhD, and Elan Guterman, MD, said, “The overall approach taken ... in developing and validating SCORE-AI sets a standard for this work going forward.”

Dr. Kleen and Dr. Guterman note that the technological gains brought about by SCORE-AI technology “could offer an exciting prospect to improve EEG availability and clinical care for the 50 million people with epilepsy worldwide.”
 

A version of this article originally appeared on Medscape.com.

An automated artificial intelligence (AI) model trained to read electroencephalograms (EEGs) in patients with suspected epilepsy is just as accurate as trained neurologists, new data suggest.

Known as SCORE-AI, the technology distinguishes between abnormal and normal EEG recordings and classifies irregular recordings into specific categories crucial for patient decision-making.

“SCORE-AI can be used in place of experts in underprivileged areas, where expertise is missing, or to help physicians to preselect or prescore recordings in areas where the workload is high – we can all benefit from AI,” study investigator Sándor Beniczky, MD, PhD, said in a JAMA Neurology podcast.

Dr. Beniczky is professor of clinical neurophysiology at Aarhus University in Denmark.

The findings were published online in JAMA Neurology.
 

Gaining a foothold

Increasingly, AI is gaining a foothold in medicine by credibly addressing patient queries and aiding radiologists.

To bring AI to EEG interpretation, the researchers developed and validated an AI model that was able to assess routine, clinical EEGs in patients with suspected epilepsy.

Beyond using AI to distinguish abnormal from normal EEG recordings, the researchers wanted to train the new system to classify abnormal recordings into the major categories that are most relevant for clinical decision-making in patients who may have epilepsy. The categories included epileptiform-focal, epileptiform-generalized, nonepileptiform-focal, and nonepileptiform-diffuse abnormalities.

The researchers trained the learning model using Standardized Computer-based Organized Reporting of EEG (SCORE) software.

In the development phase, the model was trained using more than 30,490 anonymized and highly annotated EEG recordings from 14,100 men (median age, 25 years) from a single center. The recordings had an average duration of 31 minutes and were interpreted by 17 neurologists using standardized criteria. If an EEG recording was abnormal, the physicians had to specify which abnormal features were present.

SCORE-AI then performed an analysis of the recordings based on input from the experts.

To validate the findings, investigators used two independent test datasets. The first dataset consisted of 100 representative routine EEGs from 61 men (median age, 26 years), evaluated by 11 neurologists from different centers.

The consensus of these evaluations served as the reference standard. The second dataset comprised nearly 10,000 EEGs from a single center (5,170 men; median age, 35 years), independently assessed by 14 neurologists.
 

Near-perfect accuracy

When compared with the experts, SCORE-AI had near-perfect accuracy with an area under the receiver operating characteristic (AUROC) curve for differentiating normal from abnormal EEG recordings of 0.95.

SCORE-AI also performed well at identifying generalized epileptiform abnormalities (AUROC, 0.96), focal epileptiform abnormalities (AUROC, 0.91), focal nonepileptiform abnormalities (AUROC, 0.89), and diffuse nonepileptiform abnormalities (AUROC, 0.93).

In addition, SCORE-AI had excellent agreement with clinicians – and sometimes agreed with individual experts more than the experts agreed with one another.

When Dr. Beniczky and team tested SCORE-AI against three previously published AI models, SCORE-AI demonstrated greater specificity than those models (90% vs. 3%-63%) but was not as sensitive (86.7%) as two of the models (96.7% and 100%).

One of the study’s limitations was the fact that SCORE-AI was developed and validated on routine EEGs that excluded neonates and critically ill patients.

In the future, Dr. Beniczky said on the podcast, the team would like to train SCORE-AI to read EEGs with more granularity, and eventually use only one single channel to record EEGs. At present, SCORE-AI is being integrated with Natus Neuro, a widely used EEG equipment system, the investigators note.

In an accompanying editorial, Jonathan Kleen, MD, PhD, and Elan Guterman, MD, said, “The overall approach taken ... in developing and validating SCORE-AI sets a standard for this work going forward.”

Dr. Kleen and Dr. Guterman note that the technological gains brought about by SCORE-AI technology “could offer an exciting prospect to improve EEG availability and clinical care for the 50 million people with epilepsy worldwide.”
 

A version of this article originally appeared on Medscape.com.

An automated artificial intelligence (AI) model trained to read electroencephalograms (EEGs) in patients with suspected epilepsy is just as accurate as trained neurologists, new data suggest.

Known as SCORE-AI, the technology distinguishes between abnormal and normal EEG recordings and classifies irregular recordings into specific categories crucial for patient decision-making.

“SCORE-AI can be used in place of experts in underprivileged areas, where expertise is missing, or to help physicians to preselect or prescore recordings in areas where the workload is high – we can all benefit from AI,” study investigator Sándor Beniczky, MD, PhD, said in a JAMA Neurology podcast.

Dr. Beniczky is professor of clinical neurophysiology at Aarhus University in Denmark.

The findings were published online in JAMA Neurology.
 

Gaining a foothold

Increasingly, AI is gaining a foothold in medicine by credibly addressing patient queries and aiding radiologists.

To bring AI to EEG interpretation, the researchers developed and validated an AI model that was able to assess routine, clinical EEGs in patients with suspected epilepsy.

Beyond using AI to distinguish abnormal from normal EEG recordings, the researchers wanted to train the new system to classify abnormal recordings into the major categories that are most relevant for clinical decision-making in patients who may have epilepsy. The categories included epileptiform-focal, epileptiform-generalized, nonepileptiform-focal, and nonepileptiform-diffuse abnormalities.

The researchers trained the learning model using Standardized Computer-based Organized Reporting of EEG (SCORE) software.

In the development phase, the model was trained using more than 30,490 anonymized and highly annotated EEG recordings from 14,100 men (median age, 25 years) from a single center. The recordings had an average duration of 31 minutes and were interpreted by 17 neurologists using standardized criteria. If an EEG recording was abnormal, the physicians had to specify which abnormal features were present.

SCORE-AI then performed an analysis of the recordings based on input from the experts.

To validate the findings, investigators used two independent test datasets. The first dataset consisted of 100 representative routine EEGs from 61 men (median age, 26 years), evaluated by 11 neurologists from different centers.

The consensus of these evaluations served as the reference standard. The second dataset comprised nearly 10,000 EEGs from a single center (5,170 men; median age, 35 years), independently assessed by 14 neurologists.
 

Near-perfect accuracy

When compared with the experts, SCORE-AI had near-perfect accuracy with an area under the receiver operating characteristic (AUROC) curve for differentiating normal from abnormal EEG recordings of 0.95.

SCORE-AI also performed well at identifying generalized epileptiform abnormalities (AUROC, 0.96), focal epileptiform abnormalities (AUROC, 0.91), focal nonepileptiform abnormalities (AUROC, 0.89), and diffuse nonepileptiform abnormalities (AUROC, 0.93).

In addition, SCORE-AI had excellent agreement with clinicians – and sometimes agreed with individual experts more than the experts agreed with one another.

When Dr. Beniczky and team tested SCORE-AI against three previously published AI models, SCORE-AI demonstrated greater specificity than those models (90% vs. 3%-63%) but was not as sensitive (86.7%) as two of the models (96.7% and 100%).

One of the study’s limitations was the fact that SCORE-AI was developed and validated on routine EEGs that excluded neonates and critically ill patients.

In the future, Dr. Beniczky said on the podcast, the team would like to train SCORE-AI to read EEGs with more granularity, and eventually use only one single channel to record EEGs. At present, SCORE-AI is being integrated with Natus Neuro, a widely used EEG equipment system, the investigators note.

In an accompanying editorial, Jonathan Kleen, MD, PhD, and Elan Guterman, MD, said, “The overall approach taken ... in developing and validating SCORE-AI sets a standard for this work going forward.”

Dr. Kleen and Dr. Guterman note that the technological gains brought about by SCORE-AI technology “could offer an exciting prospect to improve EEG availability and clinical care for the 50 million people with epilepsy worldwide.”
 

A version of this article originally appeared on Medscape.com.

As a general practitioner with a specialist interest in diabetes, I am increasingly diagnosing younger people living with type 2 diabetes and obesity. Sadly, my youngest patient living with type 2 diabetes and obesity is only in her early 20s.
 

In fact, in England, there are now more people under the age of 40 years living with type 2 diabetes than type 1 diabetes. These younger individuals tend to present with very high hemoglobin A1c levels; I am routinely seeing double-digit A1c percentage levels in my practice. Indeed, the patient mentioned above presented with an A1c of more than 13%.

The lifetime cardiometabolic risk of individuals like her is considerable and very worrying: Younger adults with type 2 diabetes often have adverse cardiometabolic risk profiles at diagnosis, with higher body mass indices, marked dyslipidemia, hypertension, and abnormal liver profiles suggesting nonalcoholic fatty liver disease. The cumulative impact of this risk profile is a significant impact on quality and quantity of life. Evidence tells us that a younger age of diagnosis with type 2 diabetes is associated with an increased risk for premature death, especially from cardiovascular disease.

Early treatment intensification is warranted in younger individuals living with type 2 diabetes and obesity. My patient above is now on triple therapy with metformin, a sodium-glucose cotransporter 2 (SGLT2) inhibitor, and a glucagonlike peptide–1 (GLP-1) receptor agonist. I gave her an urgent referral to my local weight management service for weight, nutritional, and psychological support. I have also issued her a real-time continuous glucose monitoring (rt-CGM) device: Whilst she does not meet any current U.K. criteria for using rt-CGM, I feel that the role of CGM as an educational tool for her is invaluable and equally important to her pharmacologic therapies. We are in desperate need of effective pharmacologic and lifestyle interventions to tackle this epidemic of cardiometabolic disease in the young.

I attended the recent ADA 2023 congress in San Diego, including the presentation of the SURMOUNT-2 trial data. SURMOUNT-2 explored the efficacy and safety of the dual GLP-GIP agonist tirzepatide for weight management in patients with obesity and type 2 diabetes. Tirzepatide was associated with significant reductions in weight (average weight loss, 14-16 kg after 72 weeks) and glycemia (2.1% reduction in A1c after 72 weeks), as well as reductions in clinically meaningful cardiometabolic risk factors, including systolic blood pressure, liver enzymes, and fasting non–HDL cholesterol levels. The overall safety profile of tirzepatide was also reassuring and consistent with the GLP-1 class. Most adverse effects were gastrointestinal and of mild to moderate severity. These adverse effects decreased over time.

These results perfectly position tirzepatide for my younger patients like the young woman mentioned above. The significant improvements in weight, glycemia, and cardiometabolic risk factors will not only help mitigate her future cardiometabolic risk but also help the sustainability of the U.K.’s National Health System. The cost of diabetes to the NHS in the United Kingdom is more than 10% of the entire NHS budget for England and Wales. More than 80% of this cost, however, is related not to the medications and devices we prescribe for diabetes but to the downstream complications of diabetes, such as hospital admissions for cardiovascular events and amputations, as well as regular hospital attendance for dialysis for end-stage kidney disease.

There is no doubt, however, that modern obesity medications such as semaglutide and tirzepatide are expensive, and demand has been astronomical. This demand has been driven by private weight-management services and celebrity influencers, and has resulted in major U.K.-wide GLP-1 shortages.

This situation is tragically widening health inequalities, as many of my patients who have been on GLP-1 receptor agonists for many years are unable to obtain them. I am having to consider switching therapies, often to less efficacious options without the compelling cardiorenal benefits. Furthermore, the GLP-1 shortages have prevented GLP-1 initiation for my other high-risk younger patients, potentially increasing future cardiometabolic risk.

There remain unanswered questions for tirzepatide: What is the durability of effect of tirzepatide after 72 weeks (that is, the trial duration of SURMOUNT-2)? Crucially, what is the effect of withdrawal of tirzepatide on weight loss maintenance? Previous evidence has suggested weight regain after discontinuation of a GLP-1 receptor agonist for obesity. This, of course, has further financial and sustainability implications for health care systems such as the NHS.

Finally, we are increasingly seeing younger women of childbearing age with or at risk for cardiometabolic disease. Again, my patient above is one example. Many of the therapies we use for cardiometabolic disease management, including GLP-1 receptor agonists and tirzepatide, have not been studied, and hence have not been licensed in pregnant women. Therefore, frank discussions are required with patients about future family plans and the importance of contraception. Often, the significant weight loss seen with GLP-1 receptor agonists can improve hormonal profiles and fertility in women and result in unexpected pregnancies if robust contraception is not in place.

Tirzepatide has yet to be made commercially available in the United Kingdom, and its price has also yet to be set. But I already envision a clear role for tirzepatide in my treatment armamentarium. I will be positioning tirzepatide as my first injectable of choice after oral treatment escalation with metformin and an SGLT2 inhibitor in all my patients who require treatment intensification – not just my younger, higher-risk individuals. This may remain an aspirational goal until supply chains and cost are defined. There is no doubt, however, that the compelling weight and glycemic benefits of tirzepatide alongside individualized lifestyle interventions can help improve the quality and quantity of life of my patients living with type 2 diabetes and obesity.

Dr. Fernando is a general practitioner near Edinburgh. He reported receiving speaker fees from Eli Lilly and Novo Nordisk..

A version of this article first appeared on Medscape.com.

As a general practitioner with a specialist interest in diabetes, I am increasingly diagnosing younger people living with type 2 diabetes and obesity. Sadly, my youngest patient living with type 2 diabetes and obesity is only in her early 20s.
 

In fact, in England, there are now more people under the age of 40 years living with type 2 diabetes than type 1 diabetes. These younger individuals tend to present with very high hemoglobin A1c levels; I am routinely seeing double-digit A1c percentage levels in my practice. Indeed, the patient mentioned above presented with an A1c of more than 13%.

The lifetime cardiometabolic risk of individuals like her is considerable and very worrying: Younger adults with type 2 diabetes often have adverse cardiometabolic risk profiles at diagnosis, with higher body mass indices, marked dyslipidemia, hypertension, and abnormal liver profiles suggesting nonalcoholic fatty liver disease. The cumulative impact of this risk profile is a significant impact on quality and quantity of life. Evidence tells us that a younger age of diagnosis with type 2 diabetes is associated with an increased risk for premature death, especially from cardiovascular disease.

Early treatment intensification is warranted in younger individuals living with type 2 diabetes and obesity. My patient above is now on triple therapy with metformin, a sodium-glucose cotransporter 2 (SGLT2) inhibitor, and a glucagonlike peptide–1 (GLP-1) receptor agonist. I gave her an urgent referral to my local weight management service for weight, nutritional, and psychological support. I have also issued her a real-time continuous glucose monitoring (rt-CGM) device: Whilst she does not meet any current U.K. criteria for using rt-CGM, I feel that the role of CGM as an educational tool for her is invaluable and equally important to her pharmacologic therapies. We are in desperate need of effective pharmacologic and lifestyle interventions to tackle this epidemic of cardiometabolic disease in the young.

I attended the recent ADA 2023 congress in San Diego, including the presentation of the SURMOUNT-2 trial data. SURMOUNT-2 explored the efficacy and safety of the dual GLP-GIP agonist tirzepatide for weight management in patients with obesity and type 2 diabetes. Tirzepatide was associated with significant reductions in weight (average weight loss, 14-16 kg after 72 weeks) and glycemia (2.1% reduction in A1c after 72 weeks), as well as reductions in clinically meaningful cardiometabolic risk factors, including systolic blood pressure, liver enzymes, and fasting non–HDL cholesterol levels. The overall safety profile of tirzepatide was also reassuring and consistent with the GLP-1 class. Most adverse effects were gastrointestinal and of mild to moderate severity. These adverse effects decreased over time.

These results perfectly position tirzepatide for my younger patients like the young woman mentioned above. The significant improvements in weight, glycemia, and cardiometabolic risk factors will not only help mitigate her future cardiometabolic risk but also help the sustainability of the U.K.’s National Health System. The cost of diabetes to the NHS in the United Kingdom is more than 10% of the entire NHS budget for England and Wales. More than 80% of this cost, however, is related not to the medications and devices we prescribe for diabetes but to the downstream complications of diabetes, such as hospital admissions for cardiovascular events and amputations, as well as regular hospital attendance for dialysis for end-stage kidney disease.

There is no doubt, however, that modern obesity medications such as semaglutide and tirzepatide are expensive, and demand has been astronomical. This demand has been driven by private weight-management services and celebrity influencers, and has resulted in major U.K.-wide GLP-1 shortages.

This situation is tragically widening health inequalities, as many of my patients who have been on GLP-1 receptor agonists for many years are unable to obtain them. I am having to consider switching therapies, often to less efficacious options without the compelling cardiorenal benefits. Furthermore, the GLP-1 shortages have prevented GLP-1 initiation for my other high-risk younger patients, potentially increasing future cardiometabolic risk.

There remain unanswered questions for tirzepatide: What is the durability of effect of tirzepatide after 72 weeks (that is, the trial duration of SURMOUNT-2)? Crucially, what is the effect of withdrawal of tirzepatide on weight loss maintenance? Previous evidence has suggested weight regain after discontinuation of a GLP-1 receptor agonist for obesity. This, of course, has further financial and sustainability implications for health care systems such as the NHS.

Finally, we are increasingly seeing younger women of childbearing age with or at risk for cardiometabolic disease. Again, my patient above is one example. Many of the therapies we use for cardiometabolic disease management, including GLP-1 receptor agonists and tirzepatide, have not been studied, and hence have not been licensed in pregnant women. Therefore, frank discussions are required with patients about future family plans and the importance of contraception. Often, the significant weight loss seen with GLP-1 receptor agonists can improve hormonal profiles and fertility in women and result in unexpected pregnancies if robust contraception is not in place.

Tirzepatide has yet to be made commercially available in the United Kingdom, and its price has also yet to be set. But I already envision a clear role for tirzepatide in my treatment armamentarium. I will be positioning tirzepatide as my first injectable of choice after oral treatment escalation with metformin and an SGLT2 inhibitor in all my patients who require treatment intensification – not just my younger, higher-risk individuals. This may remain an aspirational goal until supply chains and cost are defined. There is no doubt, however, that the compelling weight and glycemic benefits of tirzepatide alongside individualized lifestyle interventions can help improve the quality and quantity of life of my patients living with type 2 diabetes and obesity.

Dr. Fernando is a general practitioner near Edinburgh. He reported receiving speaker fees from Eli Lilly and Novo Nordisk..

A version of this article first appeared on Medscape.com.

As a general practitioner with a specialist interest in diabetes, I am increasingly diagnosing younger people living with type 2 diabetes and obesity. Sadly, my youngest patient living with type 2 diabetes and obesity is only in her early 20s.
 

In fact, in England, there are now more people under the age of 40 years living with type 2 diabetes than type 1 diabetes. These younger individuals tend to present with very high hemoglobin A1c levels; I am routinely seeing double-digit A1c percentage levels in my practice. Indeed, the patient mentioned above presented with an A1c of more than 13%.

The lifetime cardiometabolic risk of individuals like her is considerable and very worrying: Younger adults with type 2 diabetes often have adverse cardiometabolic risk profiles at diagnosis, with higher body mass indices, marked dyslipidemia, hypertension, and abnormal liver profiles suggesting nonalcoholic fatty liver disease. The cumulative impact of this risk profile is a significant impact on quality and quantity of life. Evidence tells us that a younger age of diagnosis with type 2 diabetes is associated with an increased risk for premature death, especially from cardiovascular disease.

Early treatment intensification is warranted in younger individuals living with type 2 diabetes and obesity. My patient above is now on triple therapy with metformin, a sodium-glucose cotransporter 2 (SGLT2) inhibitor, and a glucagonlike peptide–1 (GLP-1) receptor agonist. I gave her an urgent referral to my local weight management service for weight, nutritional, and psychological support. I have also issued her a real-time continuous glucose monitoring (rt-CGM) device: Whilst she does not meet any current U.K. criteria for using rt-CGM, I feel that the role of CGM as an educational tool for her is invaluable and equally important to her pharmacologic therapies. We are in desperate need of effective pharmacologic and lifestyle interventions to tackle this epidemic of cardiometabolic disease in the young.

I attended the recent ADA 2023 congress in San Diego, including the presentation of the SURMOUNT-2 trial data. SURMOUNT-2 explored the efficacy and safety of the dual GLP-GIP agonist tirzepatide for weight management in patients with obesity and type 2 diabetes. Tirzepatide was associated with significant reductions in weight (average weight loss, 14-16 kg after 72 weeks) and glycemia (2.1% reduction in A1c after 72 weeks), as well as reductions in clinically meaningful cardiometabolic risk factors, including systolic blood pressure, liver enzymes, and fasting non–HDL cholesterol levels. The overall safety profile of tirzepatide was also reassuring and consistent with the GLP-1 class. Most adverse effects were gastrointestinal and of mild to moderate severity. These adverse effects decreased over time.

These results perfectly position tirzepatide for my younger patients like the young woman mentioned above. The significant improvements in weight, glycemia, and cardiometabolic risk factors will not only help mitigate her future cardiometabolic risk but also help the sustainability of the U.K.’s National Health System. The cost of diabetes to the NHS in the United Kingdom is more than 10% of the entire NHS budget for England and Wales. More than 80% of this cost, however, is related not to the medications and devices we prescribe for diabetes but to the downstream complications of diabetes, such as hospital admissions for cardiovascular events and amputations, as well as regular hospital attendance for dialysis for end-stage kidney disease.

There is no doubt, however, that modern obesity medications such as semaglutide and tirzepatide are expensive, and demand has been astronomical. This demand has been driven by private weight-management services and celebrity influencers, and has resulted in major U.K.-wide GLP-1 shortages.

This situation is tragically widening health inequalities, as many of my patients who have been on GLP-1 receptor agonists for many years are unable to obtain them. I am having to consider switching therapies, often to less efficacious options without the compelling cardiorenal benefits. Furthermore, the GLP-1 shortages have prevented GLP-1 initiation for my other high-risk younger patients, potentially increasing future cardiometabolic risk.

There remain unanswered questions for tirzepatide: What is the durability of effect of tirzepatide after 72 weeks (that is, the trial duration of SURMOUNT-2)? Crucially, what is the effect of withdrawal of tirzepatide on weight loss maintenance? Previous evidence has suggested weight regain after discontinuation of a GLP-1 receptor agonist for obesity. This, of course, has further financial and sustainability implications for health care systems such as the NHS.

Finally, we are increasingly seeing younger women of childbearing age with or at risk for cardiometabolic disease. Again, my patient above is one example. Many of the therapies we use for cardiometabolic disease management, including GLP-1 receptor agonists and tirzepatide, have not been studied, and hence have not been licensed in pregnant women. Therefore, frank discussions are required with patients about future family plans and the importance of contraception. Often, the significant weight loss seen with GLP-1 receptor agonists can improve hormonal profiles and fertility in women and result in unexpected pregnancies if robust contraception is not in place.

Tirzepatide has yet to be made commercially available in the United Kingdom, and its price has also yet to be set. But I already envision a clear role for tirzepatide in my treatment armamentarium. I will be positioning tirzepatide as my first injectable of choice after oral treatment escalation with metformin and an SGLT2 inhibitor in all my patients who require treatment intensification – not just my younger, higher-risk individuals. This may remain an aspirational goal until supply chains and cost are defined. There is no doubt, however, that the compelling weight and glycemic benefits of tirzepatide alongside individualized lifestyle interventions can help improve the quality and quantity of life of my patients living with type 2 diabetes and obesity.

Dr. Fernando is a general practitioner near Edinburgh. He reported receiving speaker fees from Eli Lilly and Novo Nordisk..

A version of this article first appeared on Medscape.com.

A new scientific statement from the American Heart Association focuses on equity in cardio-oncology care and research.

A “growing body of evidence” suggests that women and people from underrepresented patient groups experience disproportionately higher cardiovascular effects from new and emerging anticancer therapies, the writing group, led by Daniel Addison, MD, with the Ohio State University, Columbus, pointed out.

For example, women appear to be at higher risk of immune checkpoint inhibitor–related toxicities, whereas Black patients with cancer face up to a threefold higher risk of cardiotoxicity with anticancer therapies.

With reduced screening and delayed preventive measures, Hispanic patients have more complex heart disease, cancer is diagnosed at later stages, and they receive more cardiotoxic regimens because of a lack of eligibility for novel treatments. Ultimately, this contributes to a higher incidence of treatment complications, cardiac dysfunction, and adverse patient outcomes for this patient group, they write.

Although no studies have specifically addressed cardio-oncology disparities in the LGBTQIA+ population, such disparities can be inferred from known cardiovascular disease and oncology disparities, the writing group noted.

These disparities are supported by “disparately high” risk of death after a cancer diagnosis among women and individuals from underrepresented groups, even after accounting for socioeconomic and behavioral patterns, they pointed out.

The scientific statement was published online in Circulation.
 

Evidence gaps and the path forward

“Despite advances in strategies to limit the risks of cardiovascular events among cancer survivors, relatively limited guidance is available to address the rapidly growing problem of disparate cardiotoxic risks among women and underrepresented patient populations,” the writing group said.

Decentralized and sporadic evaluations have led to a lack of consensus on the definitions, investigations, and potential optimal strategies to address disparate cardiotoxicity with contemporary cancer immunotherapy, as well as biologic and cytotoxic therapies, they noted.

They said caution is needed when interpreting clinical trial data about cardiotoxicity and in generalizing the results because people from diverse racial and ethnic groups have not been well represented in many trials.

The writing group outlined key evidence gaps and future research directions for addressing cardio-oncology disparities, as well as strategies to improve equity in cardio-oncology care and research.

These include the following:

• Identifying specific predictive factors of long-term cardiotoxic risk with targeted and immune-based cancer therapies in women and underrepresented populations.
• Investigating biological mechanisms that may underlie differences in cardiotoxicities between different patient groups.
• Developing personalized cardioprotection strategies that integrate biological, genetic, and social determinant markers.
• Intentionally diversifying clinical trials and identifying optimal strategies to improve representation in cancer clinical trials.
• Determining the role of technology, such as artificial intelligence, in improving cardiotoxicity disparities.

“Conscientiously leveraging technology and designing trials with outcomes related to these issues in practice (considering feasibility and cost) will critically accelerate the field of cardio-oncology in the 21st century. With tangible goals, we can improve health inequities in cardio-oncology,” the writing group said.

The research had no commercial funding. No conflicts of interest were reported.

A version of this article originally appeared on Medscape.com.

A new scientific statement from the American Heart Association focuses on equity in cardio-oncology care and research.

A “growing body of evidence” suggests that women and people from underrepresented patient groups experience disproportionately higher cardiovascular effects from new and emerging anticancer therapies, the writing group, led by Daniel Addison, MD, with the Ohio State University, Columbus, pointed out.

For example, women appear to be at higher risk of immune checkpoint inhibitor–related toxicities, whereas Black patients with cancer face up to a threefold higher risk of cardiotoxicity with anticancer therapies.

With reduced screening and delayed preventive measures, Hispanic patients have more complex heart disease, cancer is diagnosed at later stages, and they receive more cardiotoxic regimens because of a lack of eligibility for novel treatments. Ultimately, this contributes to a higher incidence of treatment complications, cardiac dysfunction, and adverse patient outcomes for this patient group, they write.

Although no studies have specifically addressed cardio-oncology disparities in the LGBTQIA+ population, such disparities can be inferred from known cardiovascular disease and oncology disparities, the writing group noted.

These disparities are supported by “disparately high” risk of death after a cancer diagnosis among women and individuals from underrepresented groups, even after accounting for socioeconomic and behavioral patterns, they pointed out.

The scientific statement was published online in Circulation.
 

Evidence gaps and the path forward

“Despite advances in strategies to limit the risks of cardiovascular events among cancer survivors, relatively limited guidance is available to address the rapidly growing problem of disparate cardiotoxic risks among women and underrepresented patient populations,” the writing group said.

Decentralized and sporadic evaluations have led to a lack of consensus on the definitions, investigations, and potential optimal strategies to address disparate cardiotoxicity with contemporary cancer immunotherapy, as well as biologic and cytotoxic therapies, they noted.

They said caution is needed when interpreting clinical trial data about cardiotoxicity and in generalizing the results because people from diverse racial and ethnic groups have not been well represented in many trials.

The writing group outlined key evidence gaps and future research directions for addressing cardio-oncology disparities, as well as strategies to improve equity in cardio-oncology care and research.

These include the following:

• Identifying specific predictive factors of long-term cardiotoxic risk with targeted and immune-based cancer therapies in women and underrepresented populations.
• Investigating biological mechanisms that may underlie differences in cardiotoxicities between different patient groups.
• Developing personalized cardioprotection strategies that integrate biological, genetic, and social determinant markers.
• Intentionally diversifying clinical trials and identifying optimal strategies to improve representation in cancer clinical trials.
• Determining the role of technology, such as artificial intelligence, in improving cardiotoxicity disparities.

“Conscientiously leveraging technology and designing trials with outcomes related to these issues in practice (considering feasibility and cost) will critically accelerate the field of cardio-oncology in the 21st century. With tangible goals, we can improve health inequities in cardio-oncology,” the writing group said.

The research had no commercial funding. No conflicts of interest were reported.

A version of this article originally appeared on Medscape.com.

A new scientific statement from the American Heart Association focuses on equity in cardio-oncology care and research.

A “growing body of evidence” suggests that women and people from underrepresented patient groups experience disproportionately higher cardiovascular effects from new and emerging anticancer therapies, the writing group, led by Daniel Addison, MD, with the Ohio State University, Columbus, pointed out.

For example, women appear to be at higher risk of immune checkpoint inhibitor–related toxicities, whereas Black patients with cancer face up to a threefold higher risk of cardiotoxicity with anticancer therapies.

With reduced screening and delayed preventive measures, Hispanic patients have more complex heart disease, cancer is diagnosed at later stages, and they receive more cardiotoxic regimens because of a lack of eligibility for novel treatments. Ultimately, this contributes to a higher incidence of treatment complications, cardiac dysfunction, and adverse patient outcomes for this patient group, they write.

Although no studies have specifically addressed cardio-oncology disparities in the LGBTQIA+ population, such disparities can be inferred from known cardiovascular disease and oncology disparities, the writing group noted.

These disparities are supported by “disparately high” risk of death after a cancer diagnosis among women and individuals from underrepresented groups, even after accounting for socioeconomic and behavioral patterns, they pointed out.

The scientific statement was published online in Circulation.
 

Evidence gaps and the path forward

“Despite advances in strategies to limit the risks of cardiovascular events among cancer survivors, relatively limited guidance is available to address the rapidly growing problem of disparate cardiotoxic risks among women and underrepresented patient populations,” the writing group said.

Decentralized and sporadic evaluations have led to a lack of consensus on the definitions, investigations, and potential optimal strategies to address disparate cardiotoxicity with contemporary cancer immunotherapy, as well as biologic and cytotoxic therapies, they noted.

They said caution is needed when interpreting clinical trial data about cardiotoxicity and in generalizing the results because people from diverse racial and ethnic groups have not been well represented in many trials.

The writing group outlined key evidence gaps and future research directions for addressing cardio-oncology disparities, as well as strategies to improve equity in cardio-oncology care and research.

These include the following:

• Identifying specific predictive factors of long-term cardiotoxic risk with targeted and immune-based cancer therapies in women and underrepresented populations.
• Investigating biological mechanisms that may underlie differences in cardiotoxicities between different patient groups.
• Developing personalized cardioprotection strategies that integrate biological, genetic, and social determinant markers.
• Intentionally diversifying clinical trials and identifying optimal strategies to improve representation in cancer clinical trials.
• Determining the role of technology, such as artificial intelligence, in improving cardiotoxicity disparities.

“Conscientiously leveraging technology and designing trials with outcomes related to these issues in practice (considering feasibility and cost) will critically accelerate the field of cardio-oncology in the 21st century. With tangible goals, we can improve health inequities in cardio-oncology,” the writing group said.

The research had no commercial funding. No conflicts of interest were reported.

A version of this article originally appeared on Medscape.com.

Teenagers with diabetes who use a continuous glucose monitor (CGM) employ a wide variety of alarm settings to alert them when their blood sugar may be too high or too low. But sometimes those thresholds generate too many alarms – which in turn might lead patients to ignore the devices, according to a study presented at the 2023 annual meeting of the Endocrine Society.

“These alarms alert people with diabetes and their caregivers of pending glycemic changes. However, little work has been done studying CGM alarm settings in pediatric clinical populations,” said Victoria Ochs, BS, a medical student at the Indiana University, Indianapolis, who helped conduct the study.

Ms. Ochs and colleagues analyzed 2 weeks of real-time CGM alarm settings from 150 children with diabetes treated at Indiana. Their average age was 14 years; 47% were female, 89% of were White, 9.5% were Black, and 1.5% were Asian. Approximately half the patients used insulin pumps (51%) in addition to the monitoring devices.  

For both alarms that indicated blood sugar was too low or too high, settings among the children often varied widely from thresholds recommended by the University of Colorado’s Barbara Davis Center for Diabetes, Aurora. Those thresholds are 70 mg/dL of glucose for low and 180 mg/dL for high glucose. At Indiana, the median alert level for low was set to 74 mg/dL (range: 60-100), while the median for high was 242 mg/dL (range: 120-400). 

“If we have it set at 100, what exactly is the purpose of that? Is it just to make you more anxious that you’re going to drop low at some point?” asked Cari Berget, MPH, RN, CDE, who specializes in pediatric diabetes at the University of Colorado, speaking of the low blood sugar alarm. Setting this alarm at 70 md/dL instead could lead to concrete action when it does go off – such as consuming carbohydrates to boost blood sugar, she said. 

“Alarms should result in action most of the time,” said Ms. Berget, associate director of Colorado’s PANTHER program, which established the alarm thresholds used in the Indiana study. Alarm setting is not one-size-fits-all, Ms. Berget noted: Some people might want 70 mg/dL to warn of low blood sugar, whereas others prefer 75 or 80 mg/dL. 

As for alerts about hyperglycemia, Ms. Berget said patients often exceed the high range of 180 mg/dL immediately after a meal. Ideally these sugars will subside on their own within 3 hours, a process aided by insulin shots or pumps. Setting a threshold for high blood sugar too low, such as 120 mg/dL, could result in ceaseless alarms even if the person is not at risk for harm.

“If you receive an alarm and there’s no action for you to take, then we need to change how we’re setting these alarms,” Ms. Berget said. She advised parents and children to be thoughtful about setting their CGM alarm thresholds to be most useful to them.

Ms. Ochs said in some cases families have CGM devices shipped directly to their homes and never consult with anyone about optimal alarm settings.

“It would be useful to talk to families about what baseline information they had,” Ms. Ochs told this news organization. “It would be nice to talk to diabetes educators, and I think it would be nice to talk to physicians.”

Ms. Ochs reports no relevant financial relationships. Ms. Berget has consulted for Dexcom and Insulet.
 

A version of this article originally appeared on Medscape.com.

Teenagers with diabetes who use a continuous glucose monitor (CGM) employ a wide variety of alarm settings to alert them when their blood sugar may be too high or too low. But sometimes those thresholds generate too many alarms – which in turn might lead patients to ignore the devices, according to a study presented at the 2023 annual meeting of the Endocrine Society.

“These alarms alert people with diabetes and their caregivers of pending glycemic changes. However, little work has been done studying CGM alarm settings in pediatric clinical populations,” said Victoria Ochs, BS, a medical student at the Indiana University, Indianapolis, who helped conduct the study.

Ms. Ochs and colleagues analyzed 2 weeks of real-time CGM alarm settings from 150 children with diabetes treated at Indiana. Their average age was 14 years; 47% were female, 89% of were White, 9.5% were Black, and 1.5% were Asian. Approximately half the patients used insulin pumps (51%) in addition to the monitoring devices.  

For both alarms that indicated blood sugar was too low or too high, settings among the children often varied widely from thresholds recommended by the University of Colorado’s Barbara Davis Center for Diabetes, Aurora. Those thresholds are 70 mg/dL of glucose for low and 180 mg/dL for high glucose. At Indiana, the median alert level for low was set to 74 mg/dL (range: 60-100), while the median for high was 242 mg/dL (range: 120-400). 

“If we have it set at 100, what exactly is the purpose of that? Is it just to make you more anxious that you’re going to drop low at some point?” asked Cari Berget, MPH, RN, CDE, who specializes in pediatric diabetes at the University of Colorado, speaking of the low blood sugar alarm. Setting this alarm at 70 md/dL instead could lead to concrete action when it does go off – such as consuming carbohydrates to boost blood sugar, she said. 

“Alarms should result in action most of the time,” said Ms. Berget, associate director of Colorado’s PANTHER program, which established the alarm thresholds used in the Indiana study. Alarm setting is not one-size-fits-all, Ms. Berget noted: Some people might want 70 mg/dL to warn of low blood sugar, whereas others prefer 75 or 80 mg/dL. 

As for alerts about hyperglycemia, Ms. Berget said patients often exceed the high range of 180 mg/dL immediately after a meal. Ideally these sugars will subside on their own within 3 hours, a process aided by insulin shots or pumps. Setting a threshold for high blood sugar too low, such as 120 mg/dL, could result in ceaseless alarms even if the person is not at risk for harm.

“If you receive an alarm and there’s no action for you to take, then we need to change how we’re setting these alarms,” Ms. Berget said. She advised parents and children to be thoughtful about setting their CGM alarm thresholds to be most useful to them.

Ms. Ochs said in some cases families have CGM devices shipped directly to their homes and never consult with anyone about optimal alarm settings.

“It would be useful to talk to families about what baseline information they had,” Ms. Ochs told this news organization. “It would be nice to talk to diabetes educators, and I think it would be nice to talk to physicians.”

Ms. Ochs reports no relevant financial relationships. Ms. Berget has consulted for Dexcom and Insulet.
 

A version of this article originally appeared on Medscape.com.

Teenagers with diabetes who use a continuous glucose monitor (CGM) employ a wide variety of alarm settings to alert them when their blood sugar may be too high or too low. But sometimes those thresholds generate too many alarms – which in turn might lead patients to ignore the devices, according to a study presented at the 2023 annual meeting of the Endocrine Society.

“These alarms alert people with diabetes and their caregivers of pending glycemic changes. However, little work has been done studying CGM alarm settings in pediatric clinical populations,” said Victoria Ochs, BS, a medical student at the Indiana University, Indianapolis, who helped conduct the study.

Ms. Ochs and colleagues analyzed 2 weeks of real-time CGM alarm settings from 150 children with diabetes treated at Indiana. Their average age was 14 years; 47% were female, 89% of were White, 9.5% were Black, and 1.5% were Asian. Approximately half the patients used insulin pumps (51%) in addition to the monitoring devices.  

For both alarms that indicated blood sugar was too low or too high, settings among the children often varied widely from thresholds recommended by the University of Colorado’s Barbara Davis Center for Diabetes, Aurora. Those thresholds are 70 mg/dL of glucose for low and 180 mg/dL for high glucose. At Indiana, the median alert level for low was set to 74 mg/dL (range: 60-100), while the median for high was 242 mg/dL (range: 120-400). 

“If we have it set at 100, what exactly is the purpose of that? Is it just to make you more anxious that you’re going to drop low at some point?” asked Cari Berget, MPH, RN, CDE, who specializes in pediatric diabetes at the University of Colorado, speaking of the low blood sugar alarm. Setting this alarm at 70 md/dL instead could lead to concrete action when it does go off – such as consuming carbohydrates to boost blood sugar, she said. 

“Alarms should result in action most of the time,” said Ms. Berget, associate director of Colorado’s PANTHER program, which established the alarm thresholds used in the Indiana study. Alarm setting is not one-size-fits-all, Ms. Berget noted: Some people might want 70 mg/dL to warn of low blood sugar, whereas others prefer 75 or 80 mg/dL. 

As for alerts about hyperglycemia, Ms. Berget said patients often exceed the high range of 180 mg/dL immediately after a meal. Ideally these sugars will subside on their own within 3 hours, a process aided by insulin shots or pumps. Setting a threshold for high blood sugar too low, such as 120 mg/dL, could result in ceaseless alarms even if the person is not at risk for harm.

“If you receive an alarm and there’s no action for you to take, then we need to change how we’re setting these alarms,” Ms. Berget said. She advised parents and children to be thoughtful about setting their CGM alarm thresholds to be most useful to them.

Ms. Ochs said in some cases families have CGM devices shipped directly to their homes and never consult with anyone about optimal alarm settings.

“It would be useful to talk to families about what baseline information they had,” Ms. Ochs told this news organization. “It would be nice to talk to diabetes educators, and I think it would be nice to talk to physicians.”

Ms. Ochs reports no relevant financial relationships. Ms. Berget has consulted for Dexcom and Insulet.
 

A version of this article originally appeared on Medscape.com.

Lucia Agajanian, a 25-year-old freelance film producer in Chicago, doesn’t have a specific primary care doctor, preferring the convenience of visiting a local clinic for flu shots or going online for video visits. “You say what you need, and there’s a 15-minute wait time,” she said, explaining how her appointments usually work. “I really liked that.”

But Olga Lucia Torres, a 52-year-old who teaches narrative medicine classes at Columbia University in New York, misses her longtime primary care doctor, who kept tabs for two decades on her conditions, including lupus and rheumatoid arthritis, and made sure she was up to date on vaccines and screening tests. Two years ago, Torres received a letter informing her that he was changing to a “boutique practice” and would charge a retainer fee of $10,000 for her to stay on as a patient.

“I felt really sad and abandoned,” Ms. Torres said. “This was my PCP. I was like, ‘Dude, I thought we were in this together!’ ”

The two women reflect an ongoing reality: The primary care landscape is changing in ways that could shape patients’ access and quality of care now and for decades to come. A solid and enduring relationship with a primary care doctor – who knows a patient’s history and can monitor new problems – has long been regarded as the bedrock of a quality health care system. But investment in primary care in the U.S. lags behind that of other high-income countries, and America has a smaller share of primary care physicians than most of its European counterparts.

An estimated one-third of all physicians in the U.S. are primary care doctors – who include family medicine physicians, general internists, and pediatricians – according to the Robert Graham Center, a research and analysis organization that studies primary care. Other researchers say the numbers are lower, with the Peterson-KFF Health System Tracker reporting only 12% of U.S. doctors are generalists, compared with 23% in Germany and as many as 45% in the Netherlands.

That means it’s often hard to find a doctor and make an appointment that’s not weeks or months away.

“This is a problem that has been simmering and now beginning to erupt in some communities at a boil. It’s hard to find that front door of the health system,” said Ann Greiner, president and CEO of the Primary Care Collaborative, a nonprofit membership organization.

Today, a smaller percentage of physicians are entering the field than are practicing, suggesting that shortages will worsen over time.

Interest has waned partly because, in the U.S., primary care yields lower salaries than other medical and surgical specialties.

Some doctors now in practice also say they are burned out, facing cumbersome electronic health record systems and limits on appointment times, making it harder to get to know a patient and establish a relationship.

Others are retiring or selling their practices. Hospitals, insurers like Aetna-CVS Health, and other corporate entities like Amazon are on a buying spree, snapping up primary care practices, furthering a move away from the “Marcus Welby, M.D.”-style neighborhood doctor. About 48% of primary care physicians currently work in practices they do not own. Two-thirds of those doctors don’t work for other physicians but are employed by private equity investors or other corporate entities, according to data in the “Primary Care Chartbook,” which is collected and published by the Graham Center.

Patients who seek care at these offices may not be seen by the same doctor at every visit. Indeed, they may not be seen by a doctor at all but by a paraprofessional – a nurse practitioner or a physician assistant, for instance – who works under the doctor’s license. That trend has been accelerated by new state laws – as well as changes in Medicare policy – that loosen the requirements for physician supervisors and billing. And these jobs are expected to be among the decade’s fastest-growing in the health sector.

Overall, demand for primary care is up, spurred partly by record enrollment in Affordable Care Act plans. All those new patients, combined with the low supply of doctors, are contributing to a years-long downward trend in the number of people reporting they have a usual source of care, be it an individual doctor or a specific clinic or practice.

Researchers say that raises questions, including whether people can’t find a primary care doctor, can’t afford one, or simply no longer want an established relationship.

“Is it poor access or problems with the supply of providers? Does it reflect a societal disconnection, a go-it-alone phenomenon?” asked Christopher F. Koller, president of the Milbank Memorial Fund, a foundation whose nonpartisan analyses focus on state health policy.

For patients, frustrating wait times are one result. A recent survey by a physician staffing firm found it now takes an average of 21 days just to get in to see a doctor of family medicine, defined as a subgroup of primary care, which includes general internists and pediatricians. Those physicians are many patients’ first stop for health care. That runs counter to the trend in other countries, where patients complain of months- or years-long waits for elective procedures like hip replacements but generally experience short waits for primary care visits.

Another complication: All these factors are adding urgency to ongoing concerns about attracting new primary care physicians to the specialty.

When she was in medical school, Natalie A. Cameron, MD, specifically chose primary care because she enjoyed forming relationships with patients and because “I’m specifically interested in prevention and women’s health, and you do a lot of that in primary care.” The 33-year-old is currently an instructor of medicine at Northwestern University, Chicago, where she also sees patients at a primary care practice.

Still, she understands why many of her colleagues chose something else. For some, it’s the pay differential. For others, it’s because of primary care’s reputation for involving “a lot of care and paperwork and coordinating a lot of issues that may not just be medical,” Dr. Cameron said.

The million-dollar question, then, is how much does having a usual source of care influence medical outcomes and cost? And for which kinds of patients is having a close relationship with a doctor important? While studies show that many young people value the convenience of visiting urgent care – especially when it takes so long to see a primary care doctor – will their long-term health suffer because of that strategy?

Many patients – particularly the young and generally healthy ones – shrug at the new normal, embracing alternatives that require less waiting. These options are particularly attractive to millennials, who tell focus groups that the convenience of a one-off video call or visit to a big-box store clinic trumps a long-standing relationship with a doctor, especially if they have to wait days, weeks, or longer for a traditional appointment.

“The doctor I have is a family friend, but definitely I would take access and ease over a relationship,” said Matt Degn, 24, who says it can take two to three months to book a routine appointment in Salt Lake City, where he lives.

Patients are increasingly turning to what are dubbed “retail clinics,” such as CVS’ Minute Clinics, which tout “in-person and virtual care 7 days a week.” CVS Health’s more than 1,000 clinics inside stores across the U.S. treated more than 5 million people last year, Creagh Milford, a physician and the company’s senior vice president of retail health, said in a written statement. He cited a recent study by a data products firm showing the use of retail clinics has grown 200% over the past five years.

Health policy experts say increased access to alternatives can be good, but forgoing an ongoing relationship to a regular provider is not, especially as people get older and are more likely to develop chronic conditions or other medical problems.

“There’s a lot of data that show communities with a lot of primary care have better health,” said Mr. Koller.

People with a regular primary care doctor or practice are more likely to get preventive care, such as cancer screenings or flu shots, studies show, and are less likely to die if they do suffer a heart attack.

Physicians who see patients regularly are better able to spot patterns of seemingly minor concerns that could add up to a serious health issue.

“What happens when you go to four different providers on four platforms for urinary tract infections because, well, they are just UTIs,” posed Yalda Jabbarpour, MD, a family physician practicing in Washington, and the director of the Robert Graham Center for Policy Studies. “But actually, you have a large kidney stone that’s causing your UTI or have some sort of immune deficiency like diabetes that’s causing frequent UTIs. But no one tested you.”

Most experts agree that figuring out how to coordinate care amid this changing landscape and make it more accessible without undermining quality – even when different doctors, locations, health systems, and electronic health records are involved – will be as complex as the pressures causing long waits and less interest in today’s primary care market.

And experiences sometimes lead patients to change their minds.

There’s something to be said for establishing a relationship, said Ms. Agajanian, in Chicago. She’s rethinking her decision to cobble together care, rather than have a specific primary care doctor or clinic, following an injury at work last year that led to shoulder surgery.

“As I’m getting older, even though I’m still young,” she said, “I have all these problems with my body, and it would be nice to have a consistent person who knows all my problems to talk with.”

KFF Health News’ Colleen DeGuzman contributed to this report.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF – an independent source of health policy research, polling, and journalism. Learn more about KFF.

Lucia Agajanian, a 25-year-old freelance film producer in Chicago, doesn’t have a specific primary care doctor, preferring the convenience of visiting a local clinic for flu shots or going online for video visits. “You say what you need, and there’s a 15-minute wait time,” she said, explaining how her appointments usually work. “I really liked that.”

But Olga Lucia Torres, a 52-year-old who teaches narrative medicine classes at Columbia University in New York, misses her longtime primary care doctor, who kept tabs for two decades on her conditions, including lupus and rheumatoid arthritis, and made sure she was up to date on vaccines and screening tests. Two years ago, Torres received a letter informing her that he was changing to a “boutique practice” and would charge a retainer fee of $10,000 for her to stay on as a patient.

“I felt really sad and abandoned,” Ms. Torres said. “This was my PCP. I was like, ‘Dude, I thought we were in this together!’ ”

The two women reflect an ongoing reality: The primary care landscape is changing in ways that could shape patients’ access and quality of care now and for decades to come. A solid and enduring relationship with a primary care doctor – who knows a patient’s history and can monitor new problems – has long been regarded as the bedrock of a quality health care system. But investment in primary care in the U.S. lags behind that of other high-income countries, and America has a smaller share of primary care physicians than most of its European counterparts.

An estimated one-third of all physicians in the U.S. are primary care doctors – who include family medicine physicians, general internists, and pediatricians – according to the Robert Graham Center, a research and analysis organization that studies primary care. Other researchers say the numbers are lower, with the Peterson-KFF Health System Tracker reporting only 12% of U.S. doctors are generalists, compared with 23% in Germany and as many as 45% in the Netherlands.

That means it’s often hard to find a doctor and make an appointment that’s not weeks or months away.

“This is a problem that has been simmering and now beginning to erupt in some communities at a boil. It’s hard to find that front door of the health system,” said Ann Greiner, president and CEO of the Primary Care Collaborative, a nonprofit membership organization.

Today, a smaller percentage of physicians are entering the field than are practicing, suggesting that shortages will worsen over time.

Interest has waned partly because, in the U.S., primary care yields lower salaries than other medical and surgical specialties.

Some doctors now in practice also say they are burned out, facing cumbersome electronic health record systems and limits on appointment times, making it harder to get to know a patient and establish a relationship.

Others are retiring or selling their practices. Hospitals, insurers like Aetna-CVS Health, and other corporate entities like Amazon are on a buying spree, snapping up primary care practices, furthering a move away from the “Marcus Welby, M.D.”-style neighborhood doctor. About 48% of primary care physicians currently work in practices they do not own. Two-thirds of those doctors don’t work for other physicians but are employed by private equity investors or other corporate entities, according to data in the “Primary Care Chartbook,” which is collected and published by the Graham Center.

Patients who seek care at these offices may not be seen by the same doctor at every visit. Indeed, they may not be seen by a doctor at all but by a paraprofessional – a nurse practitioner or a physician assistant, for instance – who works under the doctor’s license. That trend has been accelerated by new state laws – as well as changes in Medicare policy – that loosen the requirements for physician supervisors and billing. And these jobs are expected to be among the decade’s fastest-growing in the health sector.

Overall, demand for primary care is up, spurred partly by record enrollment in Affordable Care Act plans. All those new patients, combined with the low supply of doctors, are contributing to a years-long downward trend in the number of people reporting they have a usual source of care, be it an individual doctor or a specific clinic or practice.

Researchers say that raises questions, including whether people can’t find a primary care doctor, can’t afford one, or simply no longer want an established relationship.

“Is it poor access or problems with the supply of providers? Does it reflect a societal disconnection, a go-it-alone phenomenon?” asked Christopher F. Koller, president of the Milbank Memorial Fund, a foundation whose nonpartisan analyses focus on state health policy.

For patients, frustrating wait times are one result. A recent survey by a physician staffing firm found it now takes an average of 21 days just to get in to see a doctor of family medicine, defined as a subgroup of primary care, which includes general internists and pediatricians. Those physicians are many patients’ first stop for health care. That runs counter to the trend in other countries, where patients complain of months- or years-long waits for elective procedures like hip replacements but generally experience short waits for primary care visits.

Another complication: All these factors are adding urgency to ongoing concerns about attracting new primary care physicians to the specialty.

When she was in medical school, Natalie A. Cameron, MD, specifically chose primary care because she enjoyed forming relationships with patients and because “I’m specifically interested in prevention and women’s health, and you do a lot of that in primary care.” The 33-year-old is currently an instructor of medicine at Northwestern University, Chicago, where she also sees patients at a primary care practice.

Still, she understands why many of her colleagues chose something else. For some, it’s the pay differential. For others, it’s because of primary care’s reputation for involving “a lot of care and paperwork and coordinating a lot of issues that may not just be medical,” Dr. Cameron said.

The million-dollar question, then, is how much does having a usual source of care influence medical outcomes and cost? And for which kinds of patients is having a close relationship with a doctor important? While studies show that many young people value the convenience of visiting urgent care – especially when it takes so long to see a primary care doctor – will their long-term health suffer because of that strategy?

Many patients – particularly the young and generally healthy ones – shrug at the new normal, embracing alternatives that require less waiting. These options are particularly attractive to millennials, who tell focus groups that the convenience of a one-off video call or visit to a big-box store clinic trumps a long-standing relationship with a doctor, especially if they have to wait days, weeks, or longer for a traditional appointment.

“The doctor I have is a family friend, but definitely I would take access and ease over a relationship,” said Matt Degn, 24, who says it can take two to three months to book a routine appointment in Salt Lake City, where he lives.

Patients are increasingly turning to what are dubbed “retail clinics,” such as CVS’ Minute Clinics, which tout “in-person and virtual care 7 days a week.” CVS Health’s more than 1,000 clinics inside stores across the U.S. treated more than 5 million people last year, Creagh Milford, a physician and the company’s senior vice president of retail health, said in a written statement. He cited a recent study by a data products firm showing the use of retail clinics has grown 200% over the past five years.

Health policy experts say increased access to alternatives can be good, but forgoing an ongoing relationship to a regular provider is not, especially as people get older and are more likely to develop chronic conditions or other medical problems.

“There’s a lot of data that show communities with a lot of primary care have better health,” said Mr. Koller.

People with a regular primary care doctor or practice are more likely to get preventive care, such as cancer screenings or flu shots, studies show, and are less likely to die if they do suffer a heart attack.

Physicians who see patients regularly are better able to spot patterns of seemingly minor concerns that could add up to a serious health issue.

“What happens when you go to four different providers on four platforms for urinary tract infections because, well, they are just UTIs,” posed Yalda Jabbarpour, MD, a family physician practicing in Washington, and the director of the Robert Graham Center for Policy Studies. “But actually, you have a large kidney stone that’s causing your UTI or have some sort of immune deficiency like diabetes that’s causing frequent UTIs. But no one tested you.”

Most experts agree that figuring out how to coordinate care amid this changing landscape and make it more accessible without undermining quality – even when different doctors, locations, health systems, and electronic health records are involved – will be as complex as the pressures causing long waits and less interest in today’s primary care market.

And experiences sometimes lead patients to change their minds.

There’s something to be said for establishing a relationship, said Ms. Agajanian, in Chicago. She’s rethinking her decision to cobble together care, rather than have a specific primary care doctor or clinic, following an injury at work last year that led to shoulder surgery.

“As I’m getting older, even though I’m still young,” she said, “I have all these problems with my body, and it would be nice to have a consistent person who knows all my problems to talk with.”

KFF Health News’ Colleen DeGuzman contributed to this report.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF – an independent source of health policy research, polling, and journalism. Learn more about KFF.

Lucia Agajanian, a 25-year-old freelance film producer in Chicago, doesn’t have a specific primary care doctor, preferring the convenience of visiting a local clinic for flu shots or going online for video visits. “You say what you need, and there’s a 15-minute wait time,” she said, explaining how her appointments usually work. “I really liked that.”

But Olga Lucia Torres, a 52-year-old who teaches narrative medicine classes at Columbia University in New York, misses her longtime primary care doctor, who kept tabs for two decades on her conditions, including lupus and rheumatoid arthritis, and made sure she was up to date on vaccines and screening tests. Two years ago, Torres received a letter informing her that he was changing to a “boutique practice” and would charge a retainer fee of $10,000 for her to stay on as a patient.

“I felt really sad and abandoned,” Ms. Torres said. “This was my PCP. I was like, ‘Dude, I thought we were in this together!’ ”

The two women reflect an ongoing reality: The primary care landscape is changing in ways that could shape patients’ access and quality of care now and for decades to come. A solid and enduring relationship with a primary care doctor – who knows a patient’s history and can monitor new problems – has long been regarded as the bedrock of a quality health care system. But investment in primary care in the U.S. lags behind that of other high-income countries, and America has a smaller share of primary care physicians than most of its European counterparts.

An estimated one-third of all physicians in the U.S. are primary care doctors – who include family medicine physicians, general internists, and pediatricians – according to the Robert Graham Center, a research and analysis organization that studies primary care. Other researchers say the numbers are lower, with the Peterson-KFF Health System Tracker reporting only 12% of U.S. doctors are generalists, compared with 23% in Germany and as many as 45% in the Netherlands.

That means it’s often hard to find a doctor and make an appointment that’s not weeks or months away.

“This is a problem that has been simmering and now beginning to erupt in some communities at a boil. It’s hard to find that front door of the health system,” said Ann Greiner, president and CEO of the Primary Care Collaborative, a nonprofit membership organization.

Today, a smaller percentage of physicians are entering the field than are practicing, suggesting that shortages will worsen over time.

Interest has waned partly because, in the U.S., primary care yields lower salaries than other medical and surgical specialties.

Some doctors now in practice also say they are burned out, facing cumbersome electronic health record systems and limits on appointment times, making it harder to get to know a patient and establish a relationship.

Others are retiring or selling their practices. Hospitals, insurers like Aetna-CVS Health, and other corporate entities like Amazon are on a buying spree, snapping up primary care practices, furthering a move away from the “Marcus Welby, M.D.”-style neighborhood doctor. About 48% of primary care physicians currently work in practices they do not own. Two-thirds of those doctors don’t work for other physicians but are employed by private equity investors or other corporate entities, according to data in the “Primary Care Chartbook,” which is collected and published by the Graham Center.

Patients who seek care at these offices may not be seen by the same doctor at every visit. Indeed, they may not be seen by a doctor at all but by a paraprofessional – a nurse practitioner or a physician assistant, for instance – who works under the doctor’s license. That trend has been accelerated by new state laws – as well as changes in Medicare policy – that loosen the requirements for physician supervisors and billing. And these jobs are expected to be among the decade’s fastest-growing in the health sector.

Overall, demand for primary care is up, spurred partly by record enrollment in Affordable Care Act plans. All those new patients, combined with the low supply of doctors, are contributing to a years-long downward trend in the number of people reporting they have a usual source of care, be it an individual doctor or a specific clinic or practice.

Researchers say that raises questions, including whether people can’t find a primary care doctor, can’t afford one, or simply no longer want an established relationship.

“Is it poor access or problems with the supply of providers? Does it reflect a societal disconnection, a go-it-alone phenomenon?” asked Christopher F. Koller, president of the Milbank Memorial Fund, a foundation whose nonpartisan analyses focus on state health policy.

For patients, frustrating wait times are one result. A recent survey by a physician staffing firm found it now takes an average of 21 days just to get in to see a doctor of family medicine, defined as a subgroup of primary care, which includes general internists and pediatricians. Those physicians are many patients’ first stop for health care. That runs counter to the trend in other countries, where patients complain of months- or years-long waits for elective procedures like hip replacements but generally experience short waits for primary care visits.

Another complication: All these factors are adding urgency to ongoing concerns about attracting new primary care physicians to the specialty.

When she was in medical school, Natalie A. Cameron, MD, specifically chose primary care because she enjoyed forming relationships with patients and because “I’m specifically interested in prevention and women’s health, and you do a lot of that in primary care.” The 33-year-old is currently an instructor of medicine at Northwestern University, Chicago, where she also sees patients at a primary care practice.

Still, she understands why many of her colleagues chose something else. For some, it’s the pay differential. For others, it’s because of primary care’s reputation for involving “a lot of care and paperwork and coordinating a lot of issues that may not just be medical,” Dr. Cameron said.

The million-dollar question, then, is how much does having a usual source of care influence medical outcomes and cost? And for which kinds of patients is having a close relationship with a doctor important? While studies show that many young people value the convenience of visiting urgent care – especially when it takes so long to see a primary care doctor – will their long-term health suffer because of that strategy?

Many patients – particularly the young and generally healthy ones – shrug at the new normal, embracing alternatives that require less waiting. These options are particularly attractive to millennials, who tell focus groups that the convenience of a one-off video call or visit to a big-box store clinic trumps a long-standing relationship with a doctor, especially if they have to wait days, weeks, or longer for a traditional appointment.

“The doctor I have is a family friend, but definitely I would take access and ease over a relationship,” said Matt Degn, 24, who says it can take two to three months to book a routine appointment in Salt Lake City, where he lives.

Patients are increasingly turning to what are dubbed “retail clinics,” such as CVS’ Minute Clinics, which tout “in-person and virtual care 7 days a week.” CVS Health’s more than 1,000 clinics inside stores across the U.S. treated more than 5 million people last year, Creagh Milford, a physician and the company’s senior vice president of retail health, said in a written statement. He cited a recent study by a data products firm showing the use of retail clinics has grown 200% over the past five years.

Health policy experts say increased access to alternatives can be good, but forgoing an ongoing relationship to a regular provider is not, especially as people get older and are more likely to develop chronic conditions or other medical problems.

“There’s a lot of data that show communities with a lot of primary care have better health,” said Mr. Koller.

People with a regular primary care doctor or practice are more likely to get preventive care, such as cancer screenings or flu shots, studies show, and are less likely to die if they do suffer a heart attack.

Physicians who see patients regularly are better able to spot patterns of seemingly minor concerns that could add up to a serious health issue.

“What happens when you go to four different providers on four platforms for urinary tract infections because, well, they are just UTIs,” posed Yalda Jabbarpour, MD, a family physician practicing in Washington, and the director of the Robert Graham Center for Policy Studies. “But actually, you have a large kidney stone that’s causing your UTI or have some sort of immune deficiency like diabetes that’s causing frequent UTIs. But no one tested you.”

Most experts agree that figuring out how to coordinate care amid this changing landscape and make it more accessible without undermining quality – even when different doctors, locations, health systems, and electronic health records are involved – will be as complex as the pressures causing long waits and less interest in today’s primary care market.

And experiences sometimes lead patients to change their minds.

There’s something to be said for establishing a relationship, said Ms. Agajanian, in Chicago. She’s rethinking her decision to cobble together care, rather than have a specific primary care doctor or clinic, following an injury at work last year that led to shoulder surgery.

“As I’m getting older, even though I’m still young,” she said, “I have all these problems with my body, and it would be nice to have a consistent person who knows all my problems to talk with.”

KFF Health News’ Colleen DeGuzman contributed to this report.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF – an independent source of health policy research, polling, and journalism. Learn more about KFF.

Nearly 40% of girls and young women in the United States may have iron deficiency, which can lead to fatigue and increase the risk of many health problems, according to a new study. 

Researchers also found that 6 in every 100 of the girls and young women had extremely low iron levels, known as iron-deficiency anemia, which impacts the blood’s ability to carry oxygen throughout the body.

The findings suggest that current screening guidelines for iron levels in girls and women may be flawed, resulting in missed chances to get a simple blood test that can diagnose the easy-to-treat condition. Iron supplements are often prescribed as a treatment.

The study was published in JAMA and included 12 years of data for a total of nearly 3,500 girls and women aged 12-21 years.

In addition to shortness of breath and fatigue, other symptoms of iron deficiency anemia are: 

• Pale skin
• Cold hands and feet
• Feeling dizzy or lightheaded
• Unusual cravings for nonfood items such as ice, dirt, or paper.

The Cleveland Clinic says the most common causes of iron-deficiency anemia are those that involve blood loss, including heavy menstrual periods. The body gets iron from food, and not getting enough iron from food, as can happen from eating a vegan or vegetarian diet, can also lead to deficiency. 

In this latest study, researchers found that young women and girls’ likelihood to have iron deficiency or iron-deficiency anemia were significantly linked to race and ethnicity, poverty status, access to sufficient or quality food (also called food insecurity), and body mass index. Black and Hispanic girls and women were more likely to have iron level problems, compared with White girls and women. Black girls and women were four times more likely to have iron-deficiency anemia, compared with White girls and women.

The authors did not discuss potential causes and suggested further study is needed to identify risk factors of iron deficiency in girls and young women.

A version of this article originally appeared on WebMD.com.

Nearly 40% of girls and young women in the United States may have iron deficiency, which can lead to fatigue and increase the risk of many health problems, according to a new study. 

Researchers also found that 6 in every 100 of the girls and young women had extremely low iron levels, known as iron-deficiency anemia, which impacts the blood’s ability to carry oxygen throughout the body.

The findings suggest that current screening guidelines for iron levels in girls and women may be flawed, resulting in missed chances to get a simple blood test that can diagnose the easy-to-treat condition. Iron supplements are often prescribed as a treatment.

The study was published in JAMA and included 12 years of data for a total of nearly 3,500 girls and women aged 12-21 years.

In addition to shortness of breath and fatigue, other symptoms of iron deficiency anemia are: 

• Pale skin
• Cold hands and feet
• Feeling dizzy or lightheaded
• Unusual cravings for nonfood items such as ice, dirt, or paper.

The Cleveland Clinic says the most common causes of iron-deficiency anemia are those that involve blood loss, including heavy menstrual periods. The body gets iron from food, and not getting enough iron from food, as can happen from eating a vegan or vegetarian diet, can also lead to deficiency. 

In this latest study, researchers found that young women and girls’ likelihood to have iron deficiency or iron-deficiency anemia were significantly linked to race and ethnicity, poverty status, access to sufficient or quality food (also called food insecurity), and body mass index. Black and Hispanic girls and women were more likely to have iron level problems, compared with White girls and women. Black girls and women were four times more likely to have iron-deficiency anemia, compared with White girls and women.

The authors did not discuss potential causes and suggested further study is needed to identify risk factors of iron deficiency in girls and young women.

A version of this article originally appeared on WebMD.com.

Nearly 40% of girls and young women in the United States may have iron deficiency, which can lead to fatigue and increase the risk of many health problems, according to a new study. 

Researchers also found that 6 in every 100 of the girls and young women had extremely low iron levels, known as iron-deficiency anemia, which impacts the blood’s ability to carry oxygen throughout the body.

The findings suggest that current screening guidelines for iron levels in girls and women may be flawed, resulting in missed chances to get a simple blood test that can diagnose the easy-to-treat condition. Iron supplements are often prescribed as a treatment.

The study was published in JAMA and included 12 years of data for a total of nearly 3,500 girls and women aged 12-21 years.

In addition to shortness of breath and fatigue, other symptoms of iron deficiency anemia are: 

• Pale skin
• Cold hands and feet
• Feeling dizzy or lightheaded
• Unusual cravings for nonfood items such as ice, dirt, or paper.

The Cleveland Clinic says the most common causes of iron-deficiency anemia are those that involve blood loss, including heavy menstrual periods. The body gets iron from food, and not getting enough iron from food, as can happen from eating a vegan or vegetarian diet, can also lead to deficiency. 

In this latest study, researchers found that young women and girls’ likelihood to have iron deficiency or iron-deficiency anemia were significantly linked to race and ethnicity, poverty status, access to sufficient or quality food (also called food insecurity), and body mass index. Black and Hispanic girls and women were more likely to have iron level problems, compared with White girls and women. Black girls and women were four times more likely to have iron-deficiency anemia, compared with White girls and women.

The authors did not discuss potential causes and suggested further study is needed to identify risk factors of iron deficiency in girls and young women.

A version of this article originally appeared on WebMD.com.

Abdominal pain has always been among the most common complaints fielded by primary care pediatricians. Much has been written about how we clinicians should respond when one of these patients presents in our office. Obviously, we start with a good history and physical exam and then progress to whatever laboratory or imaging tests we believe will yield the most accurate diagnosis in the shortest amount of time and with the minimum risk to the patient.

However, the number of children complaining of abdominal pain who arrive at clinicians’ offices is but a mere fraction of the youngsters who have shared the complaint with their parents or caregivers. Little has been written about what is going on beneath the surface of this monstrous iceberg of pediatric abdominal pain.

A recent poll commissioned by C.S. Mott Children’s Hospital at the University of Michigan attempts to determine how Doctor Moms and Dads are handling their children’s belly pain complaints on what is truly the frontline of health care. Using a national panel of more than 2,000 parents, the investigators reviewed the responses of more than 1,000 individuals who had at least one child age 3-10.

Seventeen percent of the parents reported that their children complained of abdominal pain at least once a month. Only a bit more than 50% of these parents say they have discussed this frequent pain with their children’s providers. Less than a third of parents reported their children complain of abdominal pain only a few times a year and half the parents responded that their children rarely or never complained of a bellyache.

The survey drilled a little deeper and discovered that for the most part, parents took a thoughtful history and did a reasonably focused physical exam. More than a third of respondents felt “very confident” in their ability to recognize a serious problem. A third of parents reported that they would treat the symptoms with an over-the-counter product.

About a quarter of the parents attributed their children’s complaints to anxiety or to gain attention. In these situations, more than half of the parents said they would talk to the child about his/her concerns and/or suggest relaxation techniques or employ distraction. Only a few would allow the child to stay home from school or miss other activities. In general, it feels like Dr. Moms and Dads in the trenches are doing a pretty good job evaluating, triaging, and managing most children with abdominal pain. At least in my experience, unfortunate outcomes of pediatric abdominal pain as the result of home mismanagement are rare.

This is a nice little survey, but I don’t think it tells us much we haven’t already suspected. What we really want to know more about are those exceedingly rare but avoidable situations when parents have not managed their children’s belly pain well and the results have been tragic. Why did they wait so long to call the physician? What signs did they miss? What symptoms did they ignore or discount? Are there patterns we can better address with education?

Just as in cases of Sudden Unexplained Infant Death, investigating with sensitivity can be extremely difficult. Interviewing parents who are still processing the unexpected death of their child is something that must be done without the slightest hint of assessing blame. Sometimes that is just plain impossible. Fortunately, these cases are rare.

If we are considering launching the study that I have proposed, we must also embark on a parallel study that asks what are the systemic conditions that may have led to the tragic mismanagement of pediatric abdominal pain? When parents have been alert to children’s complaints and appearance and attempted to seek medical care, what impediments did they encounter? Was there a triage nurse or on call physician who didn’t listen, or failed to ask the right questions? Was the emergency room just too busy to allow a proper evaluation? Was there a communication problem? And, of course, there is always the money. Did the parents’ concern about paying for the evaluation blind them to their instinct to call? These are not easy questions to ask ourselves but they must be asked if we wish to bring our failure rate closer to zero and retain the trust of our patients.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Abdominal pain has always been among the most common complaints fielded by primary care pediatricians. Much has been written about how we clinicians should respond when one of these patients presents in our office. Obviously, we start with a good history and physical exam and then progress to whatever laboratory or imaging tests we believe will yield the most accurate diagnosis in the shortest amount of time and with the minimum risk to the patient.

However, the number of children complaining of abdominal pain who arrive at clinicians’ offices is but a mere fraction of the youngsters who have shared the complaint with their parents or caregivers. Little has been written about what is going on beneath the surface of this monstrous iceberg of pediatric abdominal pain.

A recent poll commissioned by C.S. Mott Children’s Hospital at the University of Michigan attempts to determine how Doctor Moms and Dads are handling their children’s belly pain complaints on what is truly the frontline of health care. Using a national panel of more than 2,000 parents, the investigators reviewed the responses of more than 1,000 individuals who had at least one child age 3-10.

Seventeen percent of the parents reported that their children complained of abdominal pain at least once a month. Only a bit more than 50% of these parents say they have discussed this frequent pain with their children’s providers. Less than a third of parents reported their children complain of abdominal pain only a few times a year and half the parents responded that their children rarely or never complained of a bellyache.

The survey drilled a little deeper and discovered that for the most part, parents took a thoughtful history and did a reasonably focused physical exam. More than a third of respondents felt “very confident” in their ability to recognize a serious problem. A third of parents reported that they would treat the symptoms with an over-the-counter product.

About a quarter of the parents attributed their children’s complaints to anxiety or to gain attention. In these situations, more than half of the parents said they would talk to the child about his/her concerns and/or suggest relaxation techniques or employ distraction. Only a few would allow the child to stay home from school or miss other activities. In general, it feels like Dr. Moms and Dads in the trenches are doing a pretty good job evaluating, triaging, and managing most children with abdominal pain. At least in my experience, unfortunate outcomes of pediatric abdominal pain as the result of home mismanagement are rare.

This is a nice little survey, but I don’t think it tells us much we haven’t already suspected. What we really want to know more about are those exceedingly rare but avoidable situations when parents have not managed their children’s belly pain well and the results have been tragic. Why did they wait so long to call the physician? What signs did they miss? What symptoms did they ignore or discount? Are there patterns we can better address with education?

Just as in cases of Sudden Unexplained Infant Death, investigating with sensitivity can be extremely difficult. Interviewing parents who are still processing the unexpected death of their child is something that must be done without the slightest hint of assessing blame. Sometimes that is just plain impossible. Fortunately, these cases are rare.

If we are considering launching the study that I have proposed, we must also embark on a parallel study that asks what are the systemic conditions that may have led to the tragic mismanagement of pediatric abdominal pain? When parents have been alert to children’s complaints and appearance and attempted to seek medical care, what impediments did they encounter? Was there a triage nurse or on call physician who didn’t listen, or failed to ask the right questions? Was the emergency room just too busy to allow a proper evaluation? Was there a communication problem? And, of course, there is always the money. Did the parents’ concern about paying for the evaluation blind them to their instinct to call? These are not easy questions to ask ourselves but they must be asked if we wish to bring our failure rate closer to zero and retain the trust of our patients.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Abdominal pain has always been among the most common complaints fielded by primary care pediatricians. Much has been written about how we clinicians should respond when one of these patients presents in our office. Obviously, we start with a good history and physical exam and then progress to whatever laboratory or imaging tests we believe will yield the most accurate diagnosis in the shortest amount of time and with the minimum risk to the patient.

However, the number of children complaining of abdominal pain who arrive at clinicians’ offices is but a mere fraction of the youngsters who have shared the complaint with their parents or caregivers. Little has been written about what is going on beneath the surface of this monstrous iceberg of pediatric abdominal pain.

A recent poll commissioned by C.S. Mott Children’s Hospital at the University of Michigan attempts to determine how Doctor Moms and Dads are handling their children’s belly pain complaints on what is truly the frontline of health care. Using a national panel of more than 2,000 parents, the investigators reviewed the responses of more than 1,000 individuals who had at least one child age 3-10.

Seventeen percent of the parents reported that their children complained of abdominal pain at least once a month. Only a bit more than 50% of these parents say they have discussed this frequent pain with their children’s providers. Less than a third of parents reported their children complain of abdominal pain only a few times a year and half the parents responded that their children rarely or never complained of a bellyache.

The survey drilled a little deeper and discovered that for the most part, parents took a thoughtful history and did a reasonably focused physical exam. More than a third of respondents felt “very confident” in their ability to recognize a serious problem. A third of parents reported that they would treat the symptoms with an over-the-counter product.

About a quarter of the parents attributed their children’s complaints to anxiety or to gain attention. In these situations, more than half of the parents said they would talk to the child about his/her concerns and/or suggest relaxation techniques or employ distraction. Only a few would allow the child to stay home from school or miss other activities. In general, it feels like Dr. Moms and Dads in the trenches are doing a pretty good job evaluating, triaging, and managing most children with abdominal pain. At least in my experience, unfortunate outcomes of pediatric abdominal pain as the result of home mismanagement are rare.

This is a nice little survey, but I don’t think it tells us much we haven’t already suspected. What we really want to know more about are those exceedingly rare but avoidable situations when parents have not managed their children’s belly pain well and the results have been tragic. Why did they wait so long to call the physician? What signs did they miss? What symptoms did they ignore or discount? Are there patterns we can better address with education?

Just as in cases of Sudden Unexplained Infant Death, investigating with sensitivity can be extremely difficult. Interviewing parents who are still processing the unexpected death of their child is something that must be done without the slightest hint of assessing blame. Sometimes that is just plain impossible. Fortunately, these cases are rare.

If we are considering launching the study that I have proposed, we must also embark on a parallel study that asks what are the systemic conditions that may have led to the tragic mismanagement of pediatric abdominal pain? When parents have been alert to children’s complaints and appearance and attempted to seek medical care, what impediments did they encounter? Was there a triage nurse or on call physician who didn’t listen, or failed to ask the right questions? Was the emergency room just too busy to allow a proper evaluation? Was there a communication problem? And, of course, there is always the money. Did the parents’ concern about paying for the evaluation blind them to their instinct to call? These are not easy questions to ask ourselves but they must be asked if we wish to bring our failure rate closer to zero and retain the trust of our patients.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

This transcript has been edited for clarity.

Those of us in the field of diabetes have long wanted to cure type 1 diabetes, and there are little steps making me feel like this might be a possibility. One of those steps is that a company named Vertex – I’m actually on the steering committee for Vertex in terms of this project – has made beta cells from stem cells. Now, instead of waiting for a cadaveric donor, we can make little beta cells. They started giving them to people in human trials. The Food and Drug Administration has been cautious because it’s new, and I get that.

In the first part of these trials, we could only give half a dose of these beta cells. The doses were determined based on what we know from giving beta-cell transplants from cadaveric donors. We gave half a dose of these stem cell–derived beta cells to two people who were having episodes of severe hypoglycemia.

In patient 1, these beta cells worked incredibly well. He became insulin independent, and now after over a year, he’s basically free of his type 1 diabetes. Patient 2 received half a dose, and she did get some activity of the beta cells, but not enough to achieve insulin independence, so she got a second dose. Shortly after the second dose, she decided she didn’t want to participate in the trial anymore and she was lost to follow-up.

Patient 2 didn’t get the same response as patient 1, but then we moved on to four more patients who got a full dose to start with. Now, there’s a total of six patients. Of those additional four patients, one of them has now been followed for a year. Just like patient 1, he’s off insulin. It’s as though his body has normal beta cells and he’s doing great. For the next three patients, we don’t have enough follow-up data to tell you what’s going to happen to them at a year.

I can tell you that, in all six patients, the beta cells worked. They basically were producing insulin, they had positive C-peptide levels, and it showed that these beta cells work when given to human beings. Now the trial is going to start giving more patients these stem cell–derived beta cells.

One of the things that’s important to realize is that this is a very small sample size, at just six individuals. Even within those six individuals, there was variation in terms of the response to the treatment. Probably, just like with all things in medicine, there will be different doses, different ways in which people do respond, people who get off of insulin completely, and people who may require some ongoing insulin therapy. I have no idea what this is going to look like as we test this in more people.

Everybody did start making C-peptide, they were having an effect of these beta cells, and it was working. We’ll have to see how well it works, how well it works in whom, and how we’re going to be able to use these types of therapies in the future.

In terms of side effects, they were really related to immunosuppression. There were no real surprises, but again, this is a very small sample size.

In summary, I think this is really hopeful. I don’t like to give false hope, but each step of this development process has shown that these beta cells derived from stem cells do seem to work in human beings as native beta cells might. Hopefully, this portends a future of newer therapies in the treatment of people with type 1 diabetes. Thank you.
 

Dr. Peters is professor of medicine at the University of Southern California, Los Angeles, and director of the USC clinical diabetes programs. She has published more than 200 articles, reviews, and abstracts, and three books, on diabetes, and has been an investigator for more than 40 research studies. She has spoken internationally at over 400 programs and serves on many committees of several professional organizations She disclosed ties with Abbott Diabetes Care, AstraZeneca, Becton Dickinson, Boehringer Ingelheim Pharmaceuticals, Dexcom, Eli Lilly, Lexicon Pharmaceuticals, Livongo, MannKind Corporation, Medscape, Merck, Novo Nordisk, Omada Health, OptumHealth, Sanofi, and Zafgen.

A version of this article originally appeared on Medscape.com.

This transcript has been edited for clarity.

Those of us in the field of diabetes have long wanted to cure type 1 diabetes, and there are little steps making me feel like this might be a possibility. One of those steps is that a company named Vertex – I’m actually on the steering committee for Vertex in terms of this project – has made beta cells from stem cells. Now, instead of waiting for a cadaveric donor, we can make little beta cells. They started giving them to people in human trials. The Food and Drug Administration has been cautious because it’s new, and I get that.

In the first part of these trials, we could only give half a dose of these beta cells. The doses were determined based on what we know from giving beta-cell transplants from cadaveric donors. We gave half a dose of these stem cell–derived beta cells to two people who were having episodes of severe hypoglycemia.

In patient 1, these beta cells worked incredibly well. He became insulin independent, and now after over a year, he’s basically free of his type 1 diabetes. Patient 2 received half a dose, and she did get some activity of the beta cells, but not enough to achieve insulin independence, so she got a second dose. Shortly after the second dose, she decided she didn’t want to participate in the trial anymore and she was lost to follow-up.

Patient 2 didn’t get the same response as patient 1, but then we moved on to four more patients who got a full dose to start with. Now, there’s a total of six patients. Of those additional four patients, one of them has now been followed for a year. Just like patient 1, he’s off insulin. It’s as though his body has normal beta cells and he’s doing great. For the next three patients, we don’t have enough follow-up data to tell you what’s going to happen to them at a year.

I can tell you that, in all six patients, the beta cells worked. They basically were producing insulin, they had positive C-peptide levels, and it showed that these beta cells work when given to human beings. Now the trial is going to start giving more patients these stem cell–derived beta cells.

One of the things that’s important to realize is that this is a very small sample size, at just six individuals. Even within those six individuals, there was variation in terms of the response to the treatment. Probably, just like with all things in medicine, there will be different doses, different ways in which people do respond, people who get off of insulin completely, and people who may require some ongoing insulin therapy. I have no idea what this is going to look like as we test this in more people.

Everybody did start making C-peptide, they were having an effect of these beta cells, and it was working. We’ll have to see how well it works, how well it works in whom, and how we’re going to be able to use these types of therapies in the future.

In terms of side effects, they were really related to immunosuppression. There were no real surprises, but again, this is a very small sample size.

In summary, I think this is really hopeful. I don’t like to give false hope, but each step of this development process has shown that these beta cells derived from stem cells do seem to work in human beings as native beta cells might. Hopefully, this portends a future of newer therapies in the treatment of people with type 1 diabetes. Thank you.
 

Dr. Peters is professor of medicine at the University of Southern California, Los Angeles, and director of the USC clinical diabetes programs. She has published more than 200 articles, reviews, and abstracts, and three books, on diabetes, and has been an investigator for more than 40 research studies. She has spoken internationally at over 400 programs and serves on many committees of several professional organizations She disclosed ties with Abbott Diabetes Care, AstraZeneca, Becton Dickinson, Boehringer Ingelheim Pharmaceuticals, Dexcom, Eli Lilly, Lexicon Pharmaceuticals, Livongo, MannKind Corporation, Medscape, Merck, Novo Nordisk, Omada Health, OptumHealth, Sanofi, and Zafgen.

A version of this article originally appeared on Medscape.com.

This transcript has been edited for clarity.

Those of us in the field of diabetes have long wanted to cure type 1 diabetes, and there are little steps making me feel like this might be a possibility. One of those steps is that a company named Vertex – I’m actually on the steering committee for Vertex in terms of this project – has made beta cells from stem cells. Now, instead of waiting for a cadaveric donor, we can make little beta cells. They started giving them to people in human trials. The Food and Drug Administration has been cautious because it’s new, and I get that.

In the first part of these trials, we could only give half a dose of these beta cells. The doses were determined based on what we know from giving beta-cell transplants from cadaveric donors. We gave half a dose of these stem cell–derived beta cells to two people who were having episodes of severe hypoglycemia.

In patient 1, these beta cells worked incredibly well. He became insulin independent, and now after over a year, he’s basically free of his type 1 diabetes. Patient 2 received half a dose, and she did get some activity of the beta cells, but not enough to achieve insulin independence, so she got a second dose. Shortly after the second dose, she decided she didn’t want to participate in the trial anymore and she was lost to follow-up.

Patient 2 didn’t get the same response as patient 1, but then we moved on to four more patients who got a full dose to start with. Now, there’s a total of six patients. Of those additional four patients, one of them has now been followed for a year. Just like patient 1, he’s off insulin. It’s as though his body has normal beta cells and he’s doing great. For the next three patients, we don’t have enough follow-up data to tell you what’s going to happen to them at a year.

I can tell you that, in all six patients, the beta cells worked. They basically were producing insulin, they had positive C-peptide levels, and it showed that these beta cells work when given to human beings. Now the trial is going to start giving more patients these stem cell–derived beta cells.

One of the things that’s important to realize is that this is a very small sample size, at just six individuals. Even within those six individuals, there was variation in terms of the response to the treatment. Probably, just like with all things in medicine, there will be different doses, different ways in which people do respond, people who get off of insulin completely, and people who may require some ongoing insulin therapy. I have no idea what this is going to look like as we test this in more people.

Everybody did start making C-peptide, they were having an effect of these beta cells, and it was working. We’ll have to see how well it works, how well it works in whom, and how we’re going to be able to use these types of therapies in the future.

In terms of side effects, they were really related to immunosuppression. There were no real surprises, but again, this is a very small sample size.

In summary, I think this is really hopeful. I don’t like to give false hope, but each step of this development process has shown that these beta cells derived from stem cells do seem to work in human beings as native beta cells might. Hopefully, this portends a future of newer therapies in the treatment of people with type 1 diabetes. Thank you.
 

Dr. Peters is professor of medicine at the University of Southern California, Los Angeles, and director of the USC clinical diabetes programs. She has published more than 200 articles, reviews, and abstracts, and three books, on diabetes, and has been an investigator for more than 40 research studies. She has spoken internationally at over 400 programs and serves on many committees of several professional organizations She disclosed ties with Abbott Diabetes Care, AstraZeneca, Becton Dickinson, Boehringer Ingelheim Pharmaceuticals, Dexcom, Eli Lilly, Lexicon Pharmaceuticals, Livongo, MannKind Corporation, Medscape, Merck, Novo Nordisk, Omada Health, OptumHealth, Sanofi, and Zafgen.

A version of this article originally appeared on Medscape.com.