药物代谢动力学指导下的血友病康复个体化管理——基于改良Delphi法制订的中国专家共识  

Pharmacokinetics Guided Personalized Management of Hemophilia Rehabilitation:A Chinese Consensus Based on Modified Delphi

作　　者：中国血友病协作组 陈丽霞 杨仁池 Hemophilia Treatment Center Collaborative Network of China;CHEN Lixia;YANG Renchi(不详;Department of Rehabilitation Medicine,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China)

机构地区：[1]不详 [2]中国医学科学院北京协和医院康复医学科,北京100730 [3]中国医学科学院血液病医院(中国医学科学院血液学研究所),天津300020

出　　处：《罕见病研究》2025年第1期96-105,共10页Journal of Rare Diseases

基　　金：中国医学科学院医学与健康科技创新工程(2021-I2M-1-003);中央高水平医院临床科研业务费(2022-PUMCH-B-131)。

摘　　要：血友病是一组X染色体连锁隐性遗传性的出血性疾病。血友病患者因关节反复出血,会引发慢性滑膜炎、骨关节病变、肌肉萎缩和关节挛缩等并发症,这些是导致患者功能受限和残疾的主要原因。以功能为核心的康复医学在血友病患者的综合管理中发挥重要作用。由中国血友病协作组遴选的19名共识专家参与本次制订。共识制订采用改良Delphi法,就血友病患者康复方案的制订、药物代谢动力学指导下的个体化管理和骨科围手术期康复治疗的9项建议达成共识,为血友病患者康复治疗的评估和个体化康复管理提供规范化指导意见,并进一步汇总22名康复专家对各类康复治疗场景风险评估的调查结果供临床参考。Hemophilia is a collection of hereditary bleeding disorders that are X-linked recessive.Patients often experience recurrent bleeding in joints,leading to a cascade of complications including chronic synovitis,hemophilic arthropathy,muscle atrophy,and joint contractures.These issues are the primary causes of functional impairment and disabilities in individuals with hemophilia.Rehabilitation focusing on physical function is crucial in the multidisciplinary management of hemophilia.Nineteen experienced consensus experts invited by the Hemophilia Treatment Center Collaborative Network of China participated in the formulation of the Chinese consensus.Utilizing the modified Delphi method,the experts reached a consensus on nine recommendations for the formulation of hemophilia recovery plans,personalized management under the guidance of pharmacokinetics,and postoperative recovery treatment for orthopedic surgery,providing standardized procedures for evaluating and tailoring rehabilitation for hemophilia patients.Additionally,the investigation results of 22 physiotherapists on the risk assessment of various rehabilitation scenarios were further summarized for clinical reference.

关 键 词：血友病 康复治疗 药物代谢动力学 专家共识 

分 类 号：R493[医药卫生—康复医学] R554.1[医药卫生—临床医学]