An assessment of the continuing medical education needs of US physicians in the management of patients with beta thalassemia

Abstract

Patients with beta thalassemia are benefitting from longer life expectancies, highlighting the importance of appropriate transition from pediatric to adult care. Data are limited regarding continuity of care and adult hematologists' management of patients with beta thalassemia. We conducted a survey of practicing US hematologists to identify practice gaps, attitudes, and barriers to optimal patient management among US-practicing hematologists. A total of 42 responses were collected, with 19 (45%) practicing at a beta thalassemia center of excellence (CoE). Nearly 90% of CoE physicians said they had a transition protocol or plan in place versus 30% of non-CoE physicians. Most physicians said parents should remain actively involved in medical visits. Adherence was rated as the most important patient education topic during transition. The most significant barrier cited was patient reluctance to transition away from pediatric care. Physicians in CoEs as compared with non-CoE physicians reported greater knowledge of beta thalassemia and familiarity with butyrates, gene therapy, and luspatercept. Highly rated topics for beta thalassemia-focused CME activities included management of complications and clinical trial updates. These findings suggest practice gaps and barriers to optimal care in the transition from pediatric to adult care, the ongoing management of adult patients, knowledge of the disease state, and familiarity with emerging treatments. Differences CoE vs non-CoE physician responses suggest variations in knowledge, practice, and attitudes that may be helpful in tailoring CME activities to different learner audiences. The small sample size used in some sub-analyses may not be representative of all hematologists treating beta thalassemia patients.

Parental barriers in preventing recurrent thalassemia in children: A qualitative study

“…The costs associated with thalassemia treatment are often significant and go beyond medical treatment, including transportation costs for routine care, nutritional supplements, and loss of income due to frequent absences from work. In this study, many parents expressed concern about the ongoing financial burden, which is compounded by the lack of insurance coverage or financial support from the government (Behdani et al, 2015;Stacy et al, 2021). Previous research has identified that access to affordable healthcare is a determining factor in the successful management of pediatric chronic diseases (Stacy et al, 2021).…”

The patient perspective: The struggles of living with thalassemia as an adult

“…The key is helping adult providers gain confidence in the nuanced care of adult patients through continuing education 21 . There are several easily accessible, on‐demand webinars and grand rounds covering various topics related to thalassemia management offered by the Cooley's Anemia Foundation, the Thalassemia International Federation as well as continuing medical courses sponsored by industry partners.…”

“…As a result, patients are compelled to advocate for their needs as the doctors tasked with their care may not understand their transfusion requirements, the nuances of chelation therapy, or the inherent risk of comorbidities. [20][21][22] Balancing the normal challenges of adulthood with the demands of living with a rare genetic blood disorder is difficult enough without the added struggle of trying to find a provider who is competent in our care. 23 Since I outgrew my pediatrician (who was also my pediatric hematologist), I have not had a consistent primary care provider (PCP).…”

“…Many adult hematologists are unfamiliar with both the hypertransfusion regimen that has become the standard of care for people with thalassemia major and the importance of chelation. As a result, patients are compelled to advocate for their needs as the doctors tasked with their care may not understand their transfusion requirements, the nuances of chelation therapy, or the inherent risk of comorbidities 20–22 …”

“…20 The key is helping adult providers gain confidence in the nuanced care of adult patients through continuing education. 21 There are several easily accessible, on-demand webinars and grand rounds covering various topics related to thalassemia management offered by the Cooley's Anemia Foundation, the Thalassemia International Federation as well as continuing medical courses sponsored by industry partners. In addition, the American Society of Hematology, in partnership with the France Foundation, is now offering a free online program entitled "All Grown Up: Ensuring Effective Care Transitions in Beta-thalassemia" specifically geared toward improving the transition process and preparing providers to manage adult patients.…”