Paediatric Rheumatology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, WC1N 3JH, UK.
Division of Infection and Immunity, University College London, London, WC1E 6BT, UK.
Curr Rheumatol Rep. 2021 Feb 8;23(2):13. doi: 10.1007/s11926-020-00974-9.
Juvenile dermatomyositis (JDM) is a rare autoimmune disease characterised by muscle and skin involvement. Calcinosis is a debilitating complication of JDM which is difficult to treat and may cause long-term morbidity. The purpose of this review is to provide an update for the treatment of JDM-associated calcinosis based on previously published studies.
Evidence-based studies are lacking for the management of calcinosis, and current treatment modalities have been largely based on case reports, case series, cohort studies, limited controlled studies and anecdotal clinical experience. The use of early aggressive therapy for resistant cases is strongly suggested to halt persistent disease activity which may help in reducing steroid use and their associated complications. Recent insights into disease pathogenesis, myositis-specific antibodies and genetic associations have led to identification of novel therapeutic targets such as Janus kinase (JAK) 1/2. Different treatment regimens with variable outcomes are in use for the treatment of refractory calcinosis; nevertheless, the level of evidence is not sufficient to propose specific guidelines. Recently, JAK 1/2 inhibitors have shown to be effective as an emerging therapeutic option highlighting that translational and clinical research is crucial to develop targeted treatment for JDM-associated calcinosis.
幼年特发性皮肌炎（JDM）是一种罕见的自身免疫性疾病，其特征为肌肉和皮肤受累。钙质沉着症是 JDM 的一种使人虚弱的并发症，难以治疗，可能导致长期发病。本文的目的是根据以往的研究，就 JDM 相关钙质沉着症的治疗提供最新信息。
钙质沉着症的治疗缺乏循证医学证据，目前的治疗方法主要基于病例报告、病例系列、队列研究、有限对照研究和经验性临床观察。强烈建议对耐药病例采用早期积极治疗以阻止持续的疾病活动，这可能有助于减少类固醇的使用及其相关并发症。最近对疾病发病机制、肌炎特异性抗体和遗传相关性的深入了解，导致了新型治疗靶点的出现，如 Janus 激酶（JAK）1/2。目前正在使用不同的治疗方案来治疗难治性钙质沉着症，但其疗效不一；然而，目前的证据水平还不足以提出具体的治疗指南。最近，JAK 1/2 抑制剂已被证明是一种有效的治疗选择，这突显了转化和临床研究对于开发 JDM 相关钙质沉着症的靶向治疗至关重要。
