目的 分析感恩拓延-建构理论在血友病患儿家庭护理中的应用效果。方法 对2021年1月—2022年1月期间在河南省儿童医院治疗的116例血友病患儿进行随机数表法分组,两组各58例。对照组给予常规临床干预,观察组给予感恩拓延-建构理论干预。比较两组患儿的负性情绪评分、家庭功能评分、自我意识评分、功能独立性评分、感恩问卷评分。结果 干预后观察组的焦虑性情绪障碍筛查表（SCARED）、儿童抑郁障碍自评量表（DSRSC）评分、各项家庭功能评分均低于对照组,干预后观察组的各项自我意识评分、各项功能独立性评分、感恩问卷评分均高于对照组,差异统计学有意义（P<0.05）。结论 感恩拓延-建构理论干预能够在一定程度上改善血友病患儿的负性情绪、家庭功能、自我意识、独立性及感恩水平。

Objective To analyze the application effect of gratitude extension-construction theory in family nursing of children with haemophilia．Methods A total of 116 children with haemophilia from January 2021 to January 2022 were randomly divided into two groups,58 children in each group．The control group received routine clinical intervention,while the observation group received gratitude extension-construction theory intervention．The negative emotional scores,family function scores,self-awareness scores,functional independence scores and gratitude questionnaire scores between the two groups were compared．Results After intervention,the SCARED,DSRSC,and family function scores of the observation group were lower than those of the control group．The self-awareness score,functional independence score,and gratitude questionnaire score of the observation group were higher than those of the control group,and the differences were significant（P<0.05）．Conclusions Gratitude extension-construction theory intervention can improve the negative emotion,family function,self-consciousness,independence and gratitude level of children with haemophilia to a certain extent．

血友病是一种由于X染色体上凝血因子基因突变所致的遗传性出血性疾病,目前主要的治疗方法是凝血因子替代疗法。但长期频繁的注射用药往往导致患者依从性差,容易产生抑制性抗体,从而影响治疗效果。虽然现在延长半衰期的新型凝血因子药物、人源化双特异性抗体以及抗组织因子途径抑制剂单克隆抗体等用于疾病治疗,在给药方式和作用持续时间上已有很大进步,但它们仍无法治愈血友病。因此,以疾病根治为重要目标的基因治疗被设计出来,近年来受到了广泛的关注。该文介绍了血友病基因治疗的原理、基因治疗载体的选择、基因治疗预处理方案,总结了现阶段基因治疗临床应用的安全性和有效性;最后讨论基因治疗目前存在的问题以及未来发展方向。

Hemophilia is a genetic bleeding disorder resulting from mutations in coagulation factor genes on the X chromosome．The mainstay of current treatment is coagulation factor replacement therapy．However,frequent and long-term injections often lead to poor patient compliance,easy inhibitor development,and compromised therapeutic efficacy．Despite advancements in delivery methods and prolonged action of novel agents such as extended half-life coagulation factor concentrates,humanized bispecific antibodies,and anti-tissue factor pathway inhibitor monoclonal antibodies,these approaches still fall short of curing hemophilia．Consequently,gene therapy,aiming for disease eradication,has garnered significant attention in recent years．This review delves into the principles of gene therapy,the selection of gene therapy vectors,and gene therapy preconditioning regimens．It summarizes the safety and efficacy of gene therapy in current clinical applications and discusses challenges and future directions in this field．