[Efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II with and without comparison to placebo: systematic review and meta-analysis].

Abstract

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.

• Authors: Taciane Alegra, Dauana Pitano Eizerik, Caio César Silva de Cerqueira, Tiago V Pereira, Alícia Dorneles Dornelles, Ida Vanessa Doederlein Schwartz
• Journal: Cadernos de saude publica (Cad Saude Publica) Vol. 29 Suppl 1 Pg. S45-58 (Nov 2013) ISSN: 1678-4464 [Electronic] Brazil
• Vernacular Title: Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise.
• PMID: 25402250 (Publication Type: Journal Article, Meta-Analysis, Review, Systematic Review)

Chemical References

• Iduronate Sulfatase
• idursulfase

Topics

• Enzyme Replacement Therapy (methods)
• Humans
• Iduronate Sulfatase (adverse effects, therapeutic use)
• Mucopolysaccharidosis II (drug therapy)
• Randomized Controlled Trials as Topic