[Efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II with and without comparison to placebo: systematic review and meta-analysis].
Mucopolysaccharidosis type II (MPS II) is a genetic disease of broad clinical spectrum, characterized by a deficiency of the enzyme iduronate2-sulfatase. The aim of this study was to assess whether enzyme replacement therapy (ERT) with idursulfase (IDS) for MPS II is effective and safe. PubMed/MEDLINE, Embase, LILACS, and Cochrane Library were searched until November 30, 2012. Only five articles met the inclusion criteria (randomized controlled trials - RCTs, or open-label trials/prospective case series including > 5 patients and evaluating relevant outcomes). A meta-analysis was performed for forced vital capacity (FVC; absolute and %) and for distance walked on the 6-minute walking test (6MWT). There was a statistically significant increase, but not clinically relevant, in both variables; an increased risk for development of mild infusion-related reactions and IgG antibodies to IDS were also found. The data suggest that ERT with IDS is safe and has a potential benefit for MPS II patients, but further studies are required.
Genética e Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brasil.
Taciane
T
Alegra
Cardiologia e Ciências Cardiovasculares, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brasil.
Dauana Pitano
DP
Eizerik
Genética e Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brasil.
Caio César Silva
CC
de Cerqueira
Instituto de Educação e Ciências, Hospital Alemão Oswaldo Cruz, São Paulo, Brasil.
Tiago V
TV
Pereira
Faculdade de Medicina, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brasil.
Alícia Dorneles
AD
Dornelles
Genética e Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brasil.
Ida Vanessa Doederlein
IV
Schwartz
D056947:Enzyme Replacement Therapy / Q000379:methods*; D006801:Humans; D007066:Iduronate Sulfatase / Q000009:adverse effects / Q000627:therapeutic use*; D016532:Mucopolysaccharidosis II / Q000188:drug therapy*; D016032:Randomized Controlled Trials as Topic
D007066:Iduronate Sulfatase; C517982:idursulfase