Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers.
Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers.
复制标题
镰状细胞病的基因治疗：患者和护理人员的态度和信息需求。
DOI：
10.1002/pbc.30319
复制
发表时间：
2023
影响因子：
3.2
通讯作者：
Johnson,Liza-Marie
中科院分区：
文献类型：
--
作者：
Sharma,Akshay;Young,Amanda;Carroll,Yvonne;Darji,Himani;Li,Yimei;Mandrell,BelindaN;Nelson,MarquitaN;Owens,CurtisL;Irvine,Mary;Caples,Mary;Jerkins,LaurenP;Unguru,Yoram;Hankins,JaneS;Johnson,Liza-Marie
研究背景镰状细胞病（SCD）是一种遗传性血液病，可导致严重的发病率和早期死亡率。SCD的新疗法，最值得注意的是遗传疗法（GT）和HLA不匹配的供体造血细胞移植，正在临床试验中。虽然这些干预措施具有潜在的治愈性，但它们是SCD最密集的治疗方法之一，并且与严重和改变生活的副作用有关，这些副作用可能在治疗后几年表现出来。很少有人知道知识，信仰，和态度的个人与SCD，或他们的照顾者，对现有的和这些新兴的therapy.MethodsPatients与SCD至少13岁（n= 66）和照顾者（n= 38）进行了调查的知识，态度，和信念周围的治疗SCD。结果只有4.8%的人感到“非常了解”GT SCD，而大多数（63.4%）报告的知识很少。总体而言，受访者的健康知识普及率较低。大多数受访者对GT治疗SCD的安全性以及它是否是一种良好的治疗方法持中立态度（分别为56.7%和58.6%）。只有少数受访者认可GT“不安全”或“不是一种好治疗方法”的观点（分别为5.8%和4.8%）。越来越多的关于GT的知识和同意它是安全的之间存在关联（p= .012）和SCD的良好治疗（p= 0.031）.结论鉴于很少有SCD患者对GT有了解，大多数患者对这种新方法的安全性和实用性持中立态度，随着这些治疗获得监管机构的批准并进入临床，迫切需要文化上适当的以患者为中心的教育。
BackgroundSickle cell disease (SCD) is an inherited blood disorder that results in serious morbidity and early mortality. Novel therapies for SCD, most notably genetic therapies (GTs) and HLA‐mismatched donor hematopoietic cell transplantation, are in clinical trials. While potentially curative, these interventions are some of the most intensive treatments for SCD and are associated with serious and life‐altering side effects, which may manifest several years after treatment. Little is known about knowledge, beliefs, and attitudes of individuals with SCD, or their caregivers, toward existing and these emerging therapies.MethodsPatients with SCD at least 13 years of age (n= 66) and caregivers (n= 38) were surveyed about knowledge, attitudes, and beliefs surrounding treatments for SCD.ResultsOnly 4.8% felt “extremely knowledgeable” about GT for SCD while the majority (63.4%) reported little knowledge. Overall, health literacy was low among respondents. Most respondents had a neutral attitude regarding the safety of GT for SCD, and whether it was a good treatment for the disorder (56.7% and 58.6%, respectively). Only a few respondents endorsed the idea that GT was “unsafe” or “not a good treatment” (5.8% and 4.8%, respectively). There was an association between increasing knowledge about GT and agreement that it is safe (p= .012) and a good treatment for SCD (p= .031).ConclusionsGiven that very few patients with SCD feel knowledgeable about GT and a majority have neutral feelings about the safety and utility of this new approach, culturally appropriate patient‐centered education is urgently needed as these treatments get regulatory approval and proceed to the clinic.