Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I

Pediatrics
Lorne A ClarkeGerald F Cox

Abstract

Our goal was to evaluate the long-term safety and efficacy of recombinant human alpha-l-iduronidase (laronidase) in patients with mucopolysaccharidosis I. All 45 patients who completed a 26-week, double-blind, placebo-controlled trial of laronidase were enrolled in a 3.5-year open-label extension study. Mean patient age at baseline was 16 (range: 6-43) years. All patients had attenuated disease (84% Hurler-Scheie, 16% Scheie phenotypes). Clinical, biochemical, and health outcomes measures were evaluated through the extension phase. Changes are presented as the mean +/- SEM. All 40 patients (89%) who completed the trial received at least 80% of scheduled infusions. As shown in earlier trials, urinary glycosaminoglycan levels decreased within the first 12 weeks and liver volume decreased within the first year. Percent predicted forced vital capacity remained stable, with a linear slope of -0.78 percentage points per year. The 6-minute walk distance increased 31.7 +/- 10.2 m in the first 2 years, with a final gain of 17.1 +/- 16.8 m. Improvements in the apnea/hypopnea index (decrease of 7.6 +/- 4.5 events per hour among the patients with significant baseline sleep apnea) and shoulder flexion (increase of 17.4 degrees +/- 3.6 degre...Continue Reading

Citations

Jan 12, 2012·European Journal of Pediatrics·Kristin D'AcoPaige Kaplan
Jun 10, 2010·Journal of Inherited Metabolic Disease·Janet A ThomasGerald F Cox
Jun 10, 2010·Journal of Inherited Metabolic Disease·Wuh-Liang HwuNi-Chung Lee
Jul 7, 2011·Journal of Inherited Metabolic Disease·Lorne Andrew ClarkeJohn Mitchell
Dec 1, 2011·Journal of Inherited Metabolic Disease·Christian J HendrikszUNKNOWN CSP Study Group
Nov 16, 2012·Journal of Inherited Metabolic Disease·Kenneth I BergerDavid M Rapoport
Dec 20, 2012·Bioconjugate Chemistry·Ruben J BoadoWilliam M Pardridge
Oct 22, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Jason A MetcalfKatherine P Ponder
Apr 14, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Kazuhiko MatsuokaKohji Itoh
May 27, 2011·Assay and Drug Development Technologies·Kenneth J ValenzanoElfrida R Benjamin
Jan 11, 2012·Rheumatology·Thomas J A LehmanJoan Keutzer
Jan 11, 2012·Rheumatology·Joseph Muenzer
Jan 11, 2012·Rheumatology·Vassili Valayannopoulos, Frits A Wijburg
Jan 11, 2012·Rheumatology·Robert P Sundel
Dec 10, 2009·Current Opinion in Pediatrics·Robert S Baltimore
Oct 21, 2011·Biochemistry Research International·Maria Francisca CoutinhoSandra Alves
Oct 27, 2009·Pediatric Rheumatology Online Journal·Rolando CimazBernhard Manger
Apr 25, 2012·Orphanet Journal of Rare Diseases·Minke H de RuFrits A Wijburg
Aug 21, 2013·Orphanet Journal of Rare Diseases·Tim A KantersLeona Hakkaart
Sep 24, 2013·Orphanet Journal of Rare Diseases·Shuan-Pei LinChih-Kuang Chuang
May 15, 2013·Orphanet Journal of Rare Diseases·Hsiang-Yu LinShuan-Pei Lin
Jul 11, 2013·Orphanet Journal of Rare Diseases·Sandra D K KingmaNaomi van Vlies
Jun 4, 2011·Genetics and Molecular Biology·Roberto GiuglianiAna Maria Martins
Feb 16, 2013·Genetics and Molecular Biology·Roberto Giugliani
Aug 12, 2014·Pediatric Rheumatology Online Journal·Mercedes O ChanHelen E Foster
Apr 5, 2014·Archives de pédiatrie : organe officiel de la Sociéte française de pédiatrie·S LerouxC Vinceslas
Sep 14, 2012·Annual Review of Genomics and Human Genetics·R J Desnick, E H Schuchman
Nov 5, 2014·Intractable & Rare Diseases Research·Sung Yoon ChoDong-Kyu Jin
Nov 23, 2013·Current Rheumatology Reports·Rolando Cimaz, Francesco La Torre
May 5, 2011·Journal of Inherited Metabolic Disease·María Verónica Muñoz-RojasAna Maria Martins
May 27, 2011·Journal of Inherited Metabolic Disease·Ursula MatteRoberto Giugliani
Mar 29, 2014·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Michael BeckShari Fallet
Oct 1, 2014·Proceedings of the National Academy of Sciences of the United States of America·Christian HindererJames M Wilson
Feb 20, 2013·Journal of Microencapsulation·Valeska Lizzi LagranhaUrsula Matte
Jan 12, 2016·Expert Opinion on Emerging Drugs·Roberto GiuglianiGuilherme Baldo
Jun 19, 2010·Expert Opinion on Emerging Drugs·Michael Beck

❮ Previous
Next ❯

Related Concepts

Related Feeds

Anaphylaxis

Anaphylaxis is a serious allergic reaction that is rapid in onset and may cause death.