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拉丁美洲重度 A 型血友病患者的肌肉骨骼评估。

Musculoskeletal evaluation in severe haemophilia A patients from Latin America.

机构信息

Unidade de Hemofilia IHTC 'Claudio L. P. Correa', Instituto Nacional de Tecnologia do Sangue, Hemocentro UNICAMP, University of Campinas, Campinas, Brazil.

出版信息

Haemophilia. 2014 Jan;20(1):e63-70. doi: 10.1111/hae.12316.

Abstract

There is a paucity of literature on haemophilia treatment in Latin American countries, a region characterized by rapidly improving systems of care, but with substantial disparities in treatment between countries. The aim of this study was to evaluate the musculoskeletal status of haemophilia patients from Latin America and to examine the relationship between musculoskeletal status and treatment practices across countries. The Committee of Latin America on the Therapeutics of Inhibitor Groups conducted a survey of its member country representatives on key aspects of haemophilia treatment in 10 countries. Musculoskeletal status of patients was obtained during routine comprehensive evaluations between March 2009 and March 2011. Eligible patients had severe haemophilia A (factor VIII <1%) without inhibitors (<0.6 BU mL(-1) ) and were ≥5 years of age. Musculoskeletal status was compared between three groups of countries, based primarily on differences in the availability of long-term prophylaxis. Overall, 143 patients (5-66 years of age) were enrolled from nine countries. In countries where long-term prophylaxis had been available for at least 10 years (Group A), patients aged 5-10 years had significantly better mean World Federation of Hemophilia clinical scores, fewer target joints and fewer affected joints than patients from countries where long-term prophylaxis has been available for about 5 years (Group B) or was not available (Group C). In Latin America, the musculoskeletal status of patients with severe haemophilia without inhibitors has improved significantly in association with the provision of long-term prophylaxis. As more countries in Latin America institute this practice, further improvements are anticipated.

摘要

关于拉丁美洲国家血友病治疗的文献很少,该地区的护理系统正在迅速改善,但各国之间的治疗差距仍然很大。本研究旨在评估来自拉丁美洲的血友病患者的肌肉骨骼状况,并研究各国之间肌肉骨骼状况与治疗实践之间的关系。拉丁美洲抑制剂治疗小组委员会对其成员国代表进行了一项关于 10 个国家血友病治疗关键方面的调查。2009 年 3 月至 2011 年 3 月期间,在常规综合评估期间获得了患者的肌肉骨骼状况。符合条件的患者为严重血友病 A(因子 VIII <1%)且无抑制剂(<0.6 BU mL(-1) ),年龄≥5 岁。根据长期预防治疗可用性的主要差异,将患者分为三组进行比较。总体而言,从九个国家共纳入了 143 名(5-66 岁)患者。在长期预防治疗至少 10 年的国家(A 组)中,5-10 岁的患者的世界血友病联盟临床评分显著更高,目标关节更少,受影响关节更少,而在长期预防治疗使用时间约为 5 年的国家(B 组)或无法获得长期预防治疗的国家(C 组)的患者更少。在拉丁美洲,无抑制剂的严重血友病患者的肌肉骨骼状况随着长期预防治疗的提供而显著改善。随着拉丁美洲更多的国家实施这一实践,预计将进一步改善。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5ec9/4285291/703373f717c7/hae0020-0e63-f1.jpg

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