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
Article
in English
|
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.
Full text:
Available
Index:
LILACS (Americas)
Type of study:
Controlled clinical trial
Language:
English
Journal:
J. inborn errors metab. screen
Journal subject:
Medicina Cl¡nica
/
Patologia
Year:
2017
Type:
Article
/
Project document
Affiliation country:
Belgium
/
Canada
/
Germany
/
Ireland
/
United States
/
United kingdom
Institution/Affiliation country:
Alberta Children's Hospital/CA
/
Baylor College of Medicine/US
/
BioMarin Pharmaceutical Inc./US
/
Ismar Healthcare/BE
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Montreal Children's Hospital/CA
/
New York University School of Medicine/US
/
Northwestern University Feinberg School of Medicine/US
/
UCSF/US
/
University College Dublin/IE
/
University Medical Center Hamburg-Eppendorf/DE
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