Perfetto Jessica, Lewandowski Laura B, Wahezi Dawn M, Ogega Vanessa, Ahimbisibwe Joan, Webb Kate, Scott Christiaan, Migowa Angela
Hassenfeld Children's Hospital at NYU Langone, New York, NY, USA.
Lupus Genomics and Global Health Disparities Unit, Systemic Autoimmunity Branch, National Institute of Arthritis and Musculoskeletal and Skin Diseases, NIH, Bethesda, MD, USA.
Pediatr Rheumatol Online J. 2025 Jan 27;23(1):10. doi: 10.1186/s12969-024-01009-8.
There are limited studies of juvenile dermatomyositis (JDM) in low and middle-income countries (LMIC). Many demonstrate delays to care, high prevalence of severe manifestations, and high mortality. Given the disease-associated damage with JDM, understanding JDM in Africa further is critical. Our objectives are to understand the burden of JDM in Africa and provider access to diagnostic tools and therapy through survey methodology.
A survey (available in English and French) was distributed via WhatsApp to 363 total members of the African League of Associations for Rheumatology (AFLAR; n = 233) and Paediatric Society of the African League Against Rheumatism (PAFLAR; n = 130) from November 2022-January 2023. Topics included respondent specialty, number of JDM patients followed, severe manifestations, and available diagnostic tools and medications (with and without considering cost).
Forty-three (12%) of the 363 providers who received the survey started it. Among the 43 who started the survey, 37 (86%) provided consent and manage JDM patients; of these 37 providers, 4 (11%) partially and 16 (43%) fully completed the survey. Most were adult and/or pediatric rheumatologists (n = 19; 95%). Respondents represented all 5 African regions and described 216 children with JDM within the last 10 years. There was high prevalence of calcinosis (as high as 100%) and interstitial lung disease (ILD) (as high as 32%); mortality rates in Kenya (6/42; 14%) and Zambia (2/7; 29%) exceeded the 1-3% mortality reported in studies of high-income countries. Thirteen of 27 diagnostic tools and medications were accessible to ≤ 50% of respondents after considering cost, mostly in Northern or Southern Africa (9/13; 69%). Despite being cost-free, disease assessment tools and physical exam to assess calcinosis were not reported as universally available or accessible.
This is the first study to explore experiences of providers caring for children with JDM in Africa. Respondents identified 216 children with JDM seen within the last 10 years, exceeding the 196 children with JDM reported within the last 25 years but likely still underestimating prevalence. Our findings align with reports of severe manifestations and poor outcomes in African children with JDM. Access to many diagnostics and medications is limited, and differences in accessibility parallel regional healthcare disparities. The potential differences in JDM severity warrant systematic study and highlight the need to include patients and providers from LMIC in collaborative research efforts.
中低收入国家（LMIC）对青少年皮肌炎（JDM）的研究有限。许多研究表明，患者在就医方面存在延误，严重表现的患病率高，死亡率也高。鉴于JDM会造成与疾病相关的损害，进一步了解非洲的JDM至关重要。我们的目标是通过调查方法了解非洲JDM的负担以及医疗服务提供者获取诊断工具和治疗方法的情况。
2022年11月至2023年1月期间，通过WhatsApp向非洲风湿病协会联盟（AFLAR；n = 233）和非洲抗风湿病联盟儿科学会（PAFLAR；n = 130）的363名成员分发了一份调查问卷（有英文和法文版本）。调查主题包括受访者的专业、随访的JDM患者数量、严重表现以及可用的诊断工具和药物（包括考虑成本和不考虑成本的情况）。
363名收到调查问卷的医疗服务提供者中，有43名（12%）开始填写问卷。在开始填写问卷的43名提供者中，37名（86%）表示同意并负责管理JDM患者；在这37名提供者中，4名（11%）部分完成了问卷，16名（43%）完全完成了问卷。大多数是成人和/或儿科风湿病学家（n = 19；95%）。受访者来自非洲所有5个地区，并描述了过去10年内216例JDM患儿的情况。钙质沉着症（高达100%）和间质性肺病（ILD）（高达32%）的患病率很高；肯尼亚（6/42；14%）和赞比亚（2/7；29%）的死亡率超过了高收入国家研究报告的1%-3%的死亡率。在考虑成本后，27种诊断工具和药物中有13种只有不到50%的受访者能够获得，其中大部分在北非或南非（9/13；69%）。尽管疾病评估工具和用于评估钙质沉着症的体格检查是免费的，但并未报告它们普遍可用或可及。
这是第一项探索非洲照顾JDM患儿的医疗服务提供者经历的研究。受访者确认在过去10年内共诊治了216例JDM患儿，超过了过去25年报告的196例JDM患儿，但患病率可能仍被低估。我们的研究结果与关于非洲JDM患儿严重表现和不良预后的报告一致。许多诊断方法和药物的可及性有限，可及性的差异与地区医疗差距并行。JDM严重程度的潜在差异值得进行系统研究，并突出了将中低收入国家的患者和医疗服务提供者纳入合作研究工作的必要性。
