• 主题
高级搜索  >
历史搜索 清空历史
首页> 美国卫生研究院文献>Molecular Genetics and Metabolism Reports >Pilot study of the safety and effect of adalimumab on pain physical function and musculoskeletal disease in mucopolysaccharidosis types I and II
【2h】

Pilot study of the safety and effect of adalimumab on pain physical function and musculoskeletal disease in mucopolysaccharidosis types I and II

机译：阿达木单抗对I型和II型黏多糖贮积病的疼痛身体功能和肌肉骨骼疾病的安全性和作用的初步研究

代理获取
本网站仅为用户提供外文OA文献查询和代理获取服务，本网站没有原文。下单后我们将采用程序或人工为您竭诚获取高质量的原文，但由于OA文献来源多样且变更频繁，仍可能出现获取不到、文献不完整或与标题不符等情况，如果获取不到我们将提供退款服务。请知悉。
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Mucopolysaccharidosis I and II are lysosomal storage disorders that, despite treatment with hematopoietic cell transplantation (HCT) and/or enzyme replacement therapy (ERT), continue to cause significant skeletal abnormalities leading to pain, stiffness, physical dysfunction, and short stature. Tumor necrosis factor – alpha (TNF-α) is elevated in individuals with MPS I and II and associated with pain and physical dysfunction. Therefore, we evaluated the safety and effects of the TNF-α inhibitor adalimumab in patients with MPS I and II in a 32-week, randomized, double blind, placebo-controlled, crossover study of adalimumab at a dose of 20 mg (weight 15–<30 kg) or 40 mg (weight ≥ 30 kg) administered subcutaneously every other week or saline placebo for 16 weeks. Participants were evaluated at baseline, week 16, and week 32 with the Children's Health Questionnaire – Parent Form 50 (CHQ-PF50), the Pediatric Pain Questionnaire (PPQ), range-of-motion (ROM) measurements, anthropometry, six-minute walk test (6MWT), hand dynamometer, and laboratory evaluations for safety. The primary outcome was safety and primary efficacy outcome was bodily pain (BP) measured by the CHQ-PF50. Two subjects, one with MPS I and one with MPS II, completed the study. Adalimumab was well tolerated and there were no serious adverse events. Standardized BP scores for age and gender were higher (i.e. less pain) at the end of the treatment versus placebo phase for both subjects. Subject #1 became unblinded during treatment due to skin erythema. Behavior measured by both CHQ-PF50 and parental report improved during treatment compared to placebo in both subjects. ROM improved by > 5° in seven of eight joints in Subject #1 and five of eight joints in Subject #2 (range 7.0° to 52.8°). There was no change in the PPQ, 6MWT, or hand dynamometer. Data from this small pilot study suggest that treatment with adalimumab is safe, tolerable, and may improve ROM, physical function, and possibly pain, in children with MPS I or II. However, additional clinical trials are needed before this therapy should be recommended as part of clinical care.
机译：粘多糖贮积病I和II是溶酶体贮积病，尽管使用了造血细胞移植（HCT）和/或酶替代疗法（ERT）进行了治疗，但仍继续导致严重的骨骼异常，从而导致疼痛，僵硬，身体机能障碍和身材矮小。患有MPS I和II的个体中的肿瘤坏死因子–α（TNF-α）升高，并伴有疼痛和身体机能障碍。因此，在一项为期20周，剂量为20 mg（体重15的阿达木单抗）的32周随机双盲安慰剂对照，交叉研究中，我们评估了TNF-α抑制剂阿达木单抗对MPS I和II患者的安全性和疗效。 –每隔一周皮下注射– <30 kg）或40 mg（体重≥30 kg）或生理盐水安慰剂治疗16周。使用儿童健康调查表-父母表格50（CHQ-PF50），儿童疼痛调查表（PPQ），运动范围（ROM）测量，人体测量学，六分钟，在基线，第16周和第32周对参与者进行评估步行测试（6MWT），测功机和安全性实验室评估。主要结果是安全性，主要功效结果是通过CHQ-PF50测量的身体疼痛（BP）。两名受试者完成了这项研究，其中一名参加MPS I，另一名参加MPS II。阿达木单抗耐受性好，没有严重的不良事件。与受试者的安慰剂阶段相比，治疗结束时年龄和性别的标准化BP评分更高（即疼痛更少）。受试者＃1在治疗期间因皮肤红斑而失明。与安慰剂相比，通过CHQ-PF50和父母报告测量的行为在治疗过程中均得到改善。受试者1的八个关节中的七个关节和受试者2的八个关节中的五个关节的ROM提高了> 5°（范围7.0°至52.8°）。 PPQ，6MWT或手持测功机没有变化。这项小型先导研究的数据表明，对MPS I或II患儿，使用阿达木单抗治疗是安全，可耐受的，并且可以改善ROM，身体功能，甚至可能改善疼痛。但是，在建议将该疗法作为临床护理的一部分之前，还需要进行其他临床试验。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
代理获取

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号