关键词: Bleeding disorders Gene therapy Haematology Haemophilia Multidisciplinary care

来  源:   DOI:10.1159/000538400

Abstract:
BACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript was to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region.
CONCLUSIONS: An expert panel was assembled comprising ten clinical hematologists from Kuwait, Oman, Saudi Arabia, and the UAE. The Delphi methodology was used to obtain a consensus on statements relating to several aspects of hemophilia A. A consensus was reached for all statements by means of an online, anonymized voting system. The consensus statements pertain to screening and diagnosis, treatment approaches, and requirements for the implementation of gene therapy.
CONCLUSIONS: There are significant challenges that hinder the optimal management of hemophilia A in the Gulf region. The consensus statements presented provide specific recommendations to improve diagnostic and treatment approaches, promote multidisciplinary care, and optimize regional data generation and reporting. These statements also delineate the requirements for the establishment of gene therapy centers for hemophilia A in the region.
摘要:
背景:A型血友病在海湾地区是一个重大的健康挑战,它的患病率特别高。该地区有一些与血友病A的管理相关的未满足的需求。本手稿的目的是将未满足的管理需求与背景联系起来,提供优化护理的建议,并规定了在该地区建立基因治疗中心的要求。
结论:由来自科威特的十名临床血液学家组成的专家小组组成,阿曼,沙特阿拉伯,和阿拉伯联合酋长国。德尔菲方法用于就与血友病A的几个方面有关的陈述达成共识。匿名投票系统。共识声明涉及筛查和诊断,治疗方法,以及实施基因治疗的要求。
结论:在海湾地区,存在阻碍A型血友病最佳管理的重大挑战。提出的共识声明为改进诊断和治疗方法提供了具体建议。促进多学科护理,并优化区域数据生成和报告。这些声明还描述了在该地区建立血友病A基因治疗中心的要求。
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