Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study
J. inborn errors metab. screen
;
5: e170008, 2017. tab, graf
Artículo
en Inglés
|
LILACS-Express
| LILACS
| ID: biblio-1090939
ABSTRACT
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.
Texto completo:
Disponible
Índice:
LILACS (Américas)
Tipo de estudio:
Ensayo Clínico Controlado
Idioma:
Inglés
Revista:
J. inborn errors metab. screen
Asunto de la revista:
Medicina Cl¡nica
/
Patologia
Año:
2017
Tipo del documento:
Artículo
/
Documento de proyecto
País de afiliación:
Bélgica
/
Canadá
/
Alemania
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Irlanda
/
Estados Unidos
/
Reino Unido
Institución/País de afiliación:
Alberta Children's Hospital/CA
/
Baylor College of Medicine/US
/
BioMarin Pharmaceutical Inc./US
/
Ismar Healthcare/BE
/
Montreal Children's Hospital/CA
/
New York University School of Medicine/US
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Northwestern University Feinberg School of Medicine/US
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UCSF/US
/
University College Dublin/IE
/
University Medical Center Hamburg-Eppendorf/DE
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