Theonymfi Doudouliaki1, Charalampia Papadopoulou1,2, Claire T Deakin3,4,5. 1. Department of Rheumatology, Great Ormond Street Hospital NHS Foundation Trust, London, UK. 2. Infection, Immunity and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK. 3. Infection, Immunity and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK. c.deakin@ucl.ac.uk. 4. Centre for Adolescent Rheumatology Versus Arthritis at UCL, UCLH and GOSH, London, UK. c.deakin@ucl.ac.uk. 5. NIHR Biomedical Research Centre at Great Ormond Street Hospital, London, UK. c.deakin@ucl.ac.uk.
Abstract
PURPOSE OF REVIEW: 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. RECENT FINDINGS: 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.
PURPOSE OF REVIEW: 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. RECENT FINDINGS: 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.
Authors: Liza J McCann; Jamie J Kirkham; Lucy R Wedderburn; Clarissa Pilkington; Adam M Huber; Angelo Ravelli; Duncan Appelbe; Paula R Williamson; Michael W Beresford Journal: Trials Date: 2015-06-12 Impact factor: 2.279
Authors: Sarah L Tansley; Stefania Simou; Gavin Shaddick; Zoe E Betteridge; Beverley Almeida; Harsha Gunawardena; Wendy Thomson; Michael W Beresford; Angela Midgley; Francesco Muntoni; Lucy R Wedderburn; Neil J McHugh Journal: J Autoimmun Date: 2017-06-26 Impact factor: 7.094