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首页> 外文期刊>Journal of Inborn Errors of Metabolism & Screening >Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks MOR-008: A Randomized, Double-Blind, Pilot Study
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Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks MOR-008: A Randomized, Double-Blind, Pilot Study

机译：硫磺酶阿尔法对52周以上Morquio A综合征患者疼痛的影响MOR-008：一项随机双盲试验研究

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摘要

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.
机译：患有黏多糖贮积症（MPS），尤其是Morquio A综合征（MPS IVA）的患者通常会报告严重的疼痛负担。 MOR-008是一项随机，双盲，先导性研究，评估了依洛糖酶α（剂量为2.0或4.0 mg / kg /周）治疗52周的安全性和有效性，包括对患者报告的疼痛的影响。患有Morquio A综合征（≥7岁）的患者。基线时的疼痛评估显示，患者（N = 25）的平均疼痛部位数为5.7，平均疼痛强度评分为4.6（指示中度疼痛），平均选定的疼痛描述数为7.4个字。在一年（52周）内使用elosulfase alfa治疗可改善主观疼痛评分（降低至3.2），疼痛部位（降低1个位置的平均值）和疼痛描述词（降低至4.9个单词），表明elosulfase alfa可以减轻一些Morquio A患者的疼痛

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