Use of Rescue Therapy with IVIG or Cyclophosphamide in Juvenile Myositis.

Abstract

This review aims to present the literature available to date on the use of intravenous immunoglobulin and cyclophosphamide for juvenile patients with inflammatory myopathies, to evaluate the strength of the evidence so far for both these medications, and to reach conclusions about their efficacy. Juvenile idiopathic inflammatory myopathies, mainly represented by juvenile dermatomyositis (JDM), are rare diseases but quite debilitating for the patients. JDM is an autoimmune condition with predominantly muscle and skin involvement but also systemic features affecting the cardiovascular, respiratory, and gastrointestinal systems. The mainstay therapy is based on corticosteroids and methotrexate, but often other therapeutic alternatives are sought for patients with severe or refractory disease. The rarity of these conditions makes research for new medications even more challenging. Innovative trial designs or statistical methods can be used to emulate a randomized study and investigate drug effectiveness. Despite the lack of Level I evidence on the use and efficacy of intravenous immunoglobulin and cyclophosphamide, their use is advocated by a substantial number of case reports and case series as well as analyses using marginal structural models.