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Update on the Systemic Treatment of Pediatric Localized Scleroderma

机译：关于儿科局部局部硬皮病的全身治疗更新

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摘要

Juvenile localized scleroderma (jLS) is an orphan disease that can lead to cosmetic disfiguration and orthopedic problems. Two recent publications review the current recommendations regarding diagnosis, assessment, follow up and treatment of pediatric localized scleroderma cases, both of which suggest the Localized Scleroderma Cutaneous Assessment Tool as an important instrument to assess activity and damage. This review focuses on the systemic treatment of jLS. Systemic treatment includes synthetic and biologic disease-modifying antirheumatic drugs. Systemic therapy is indicated if the lesion crosses any joint, or leads to potential cosmetic disfiguration or orthopedic problems. The only controlled trial of systemic treatment has shown the efficacy of methotrexate, which is the first choice of treatment. It appears superior to phototherapy according to a recently published meta-analysis. In case of methotrexate intolerance, mycophenolate mofetil is an option. In case of methotrexate nonresponse, addition of mycophenolate mofetil, tocilizumab or abatacept seems to be effective. Future treatment options derived and extrapolated from adult trials regarding treatment of skin involvement of systemic scleroderma or fibrosis are promising, as the final pathway in the skin seems to be similar in both diseases.
机译：少年本地化硬皮病（JLS）是一种孤儿疾病，可导致美容性破裂和整形外观问题。最近的两个出版物审查了目前关于诊断，评估，后续和治疗儿科局部化硬皮病病例的建议，这两种情况都表明本地化的硬皮病皮肤评估工具是评估活动和损伤的重要仪器。本综述着重于JLS的全身治疗。全身治疗包括合成和生物疾病改性的抗逆素药物。表示病变是否交叉任何关节，或导致潜在的化妆品破义或骨科问题。唯一受控试验的全身治疗试验表明了甲氨蝶呤的疗效，这是一种治疗的首选。根据最近发表的荟萃分析，它看起来优于光疗法。在甲氨蝶呤不耐受的情况下，Mycophenolate Mofetil是一种选择。在甲氨蝶呤非响应的情况下，加入霉霉素Mofetil，Tocolizumab或Abatacept似乎是有效的。未来治疗选项来自成人试验的关于治疗系统性硬皮病或纤维化的皮肤参与的成人试验是有前途的，因为皮肤中的最终途径似乎在这两种疾病中都相似。

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  • 来源
    《Pediatric drugs》 |2019年第6期|共7页
  • 作者

    Foeldvari Ivan;

  • 作者单位

    Schon Klin Hamburg Eilbek Ctr Treatment Scleroderma &

    Uveitis Childhood &

    A Hamburg Ctr Pediat &

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  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类 药学;
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