Rationale for off-label treatments use in primary progressive multiple sclerosis: A review of the literature.

BACKGROUND: Until recently, few therapeutic options, other than symptomatic treatment, were available for patients with primary progressive multiple sclerosis (PPMS). Ocrelizumab is the only approved treatment in this indication, and only since 2017. However, many patients in France are receiving off-label treatments for PPMS, mainly rituximab, mycophenolate mofetil, methotrexate, cyclophosphamide, and azathioprine. OBJECTIVE: To evaluate published data concerning the efficacy of these five treatments frequently used as off-label disease-modifying therapies. METHODS: We reviewed and summarized the studies published in Pubmed since the inception of the database. RESULTS: Evidence from randomized controlled trials is lacking to support the use of these treatments as disease-modifying therapies in PPMS. CONCLUSION: The literature lacks dedicated studies to support the off-label use of these disease-modifying therapies in PPMS. However, some limited data are available in the literature suggesting that the use of rituximab and cyclophosphamide could potentially be of some interest in specific subpopulations.

Alterações cardíacas na anemia falciforme / Cardiac changes in sickle cell anemia

Sao estudadas as alteracoes cardiacas de 10 criancas, de idades entre 2 meses e 7 anos, que tinham diagnostico clinico, laboratorial e anatomopatologico de anemia falciforme.Foram admitidas no hospital por apresentarem crise hemolitico-trombotica provocada por infeccao.Foi observado sopro sistolico na borda esternal esquerda em 5 criancas e, na area mitral, em 3.Na radiografia de torax, registrou-se aumento da area cardiaca em 8 pacientes e sinais de hiperfluxo pulmonar em 2. O eletrocardiograma realizado em 9 casos, mostrou sinais de sobrecarga biventricular em 4; ventricular esquerda em 3 e apenas disturbio da repolarizacao ventricular, em 2.O exame anatomopatologico evidenciou dilatacao das cavidades em 7 pacientes; hipertrofia VE em 2 e foi normal em 1. A microscopia revelou vacuolizacao das fibras em todos os casos, fragmentacao das mesmas e edema interfibrilar em 5, areas de necrose em 3 e degeneracao eosinofilica em 2. Em todos os casos foi notada a presenca de trombembolismo em varios orgaos.Os dados apresentados falam a favor de cardiomiopatia relacionada ao estigma falcemico