Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

• Autor(a) principal: Treadwell,Marsha
• Data de Publicação: 2017
• Outros Autores: Harmatz,Paul R., Burton,Barbara K., Mitchell,John J., Muschol,Nicole, Jones,Simon A., Pastores,Gregory M., Lau,Heather A., Sparkes,Rebecca, Sutton,V. Reid, Meesen,Bianca, Haller,Christine A., Shaywitz,Adam J., Gold,Jeffrey I.
• Tipo de documento: Artigo
• Idioma: eng
• Título da fonte: Journal of Inborn Errors of Metabolism and Screening
• Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100803
• topic: Morquio A syndrome; chronic pain; ysosomal storage diseases; galactosamine-6-sulfatase; enzyme replacement therapy; elosulfase alfa

Abstract Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported pain, of 52 weeks of treatment with elosulfase alfa (at a dose of 2.0 or 4.0 mg/kg/week) in patients with Morquio A syndrome (?7 years old). Assessment of pain at baseline revealed that patients (N = 25) had a mean number of pain locations of 5.7, mean pain intensity score of 4.6 (indicative of medium pain), and a mean number of selected pain descriptors of 7.4 words. Treatment with elosulfase alfa improved subjective pain score (reduced to 3.2), pain locations (reduced by a mean of 1 location), and pain descriptor words (reduced to 4.9 words) over 1 year (52 weeks), suggesting that elosulfase alfa can reduce pain in some patients with Morquio A.