Yokoyama Tadafumi, Inoue Natsumi, Sakumura Naoto, Tasaki Yuko, Wada Taizo
Department of Pediatrics, Kanazawa University, Ishikawa, JPN.
Cureus. 2024 Jun 15;16(6):e62425. doi: 10.7759/cureus.62425. eCollection 2024 Jun.
Juvenile dermatomyositis (JDM) patients who test positive for the antimelanoma differentiation-associated gene 5 (MDA5) antibody have a poor prognosis because of rapidly progressing interstitial lung disease (ILD). However, agreement on the best treatment for this condition remains elusive. We encountered a 13-year-old girl with anti-MDA5 antibody-positive JDM who presented with arthritis and was already showing signs of ILD when she was admitted to the hospital. While cyclophosphamide (CY) is commonly used, it can cause gonadal disorders and other complications when administered to adolescent females. Consequently, we chose multitarget therapy, which includes tacrolimus and mycophenolate mofetil. Her ILD and skin symptoms gradually improved, and she was able to maintain remission and avoid CY administration for three years. We conducted a thorough literature review to determine the efficacy and safety of multitarget therapy for anti-MDA5 antibody-positive DM and JDM. Multitarget therapy shows promise as a potentially effective and relatively safe treatment. The ability to avoid CY, which is especially important for adolescent patients concerned about fertility preservation, highlights a significant benefit of this multitarget therapy for anti-MDA5 antibody-positive DM and JDM patients.
抗黑色素瘤分化相关基因5（MDA5）抗体检测呈阳性的青少年皮肌炎（JDM）患者，由于间质性肺病（ILD）进展迅速，预后较差。然而，对于这种疾病的最佳治疗方法仍未达成共识。我们遇到一名13岁抗MDA5抗体阳性的JDM女孩，她有关节炎症状，入院时已出现ILD迹象。虽然环磷酰胺（CY）是常用药物，但给青春期女性使用时可能会导致性腺疾病和其他并发症。因此，我们选择了包括他克莫司和霉酚酸酯的多靶点治疗。她的ILD和皮肤症状逐渐改善，并且能够维持缓解状态，三年来未使用CY。我们进行了全面的文献综述，以确定多靶点治疗对抗MDA5抗体阳性的皮肌炎和JDM的疗效和安全性。多靶点治疗有望成为一种潜在有效且相对安全的治疗方法。避免使用CY的能力，这对关注生育保留的青少年患者尤为重要，凸显了这种多靶点治疗对抗MDA5抗体阳性的皮肌炎和JDM患者的显著益处。
