Department of Pediatric Hematology-Oncology-BMT, Emory University, Atlanta, Georgia.
Aflac Cancer and Blood Disorders, Children's Healthcare of Atlanta, Atlanta, Georgia.
Pediatr Blood Cancer. 2021 Jan;68(1):e28749. doi: 10.1002/pbc.28749. Epub 2020 Oct 8.
Improved outcomes and the availability of clinical trials of hematopoietic cell transplantation (HCT) from alternate donors and genetically modified autologous hematopoietic progenitor cells have expanded the applicability of HCT for sickle cell disease (SCD). To understand the perspective of primary caregivers exploring HCT in the current milieu, we asked the research question "What motivates primary caregivers to decide to consider HCT and to seek, and to attend, an HCT consultation?"
We conducted qualitative interviews with primary caregivers within one week after a consultation for HCT for SCD. Data were analyzed using open and axial coding stages of grounded theory methodology.
We interviewed 29 primary caregivers (26 females, age 29 to 64 [median 42] years). Primary caregivers report of SCD complications in their child included at least one in the last year by 23 (82%), few or none by 8 (28%), and pain on ≥3 days a week by 13 (46%) primary caregivers. Qualitative analysis revealed that primary caregivers, (i) learn about curative options through social networks, social media, and the news media; (ii) seek consultation because of their child's diminished quality of life, recent complications, an imminent major medical decision, or anxiety about future severe complications; and (iii) see gene therapy as a new, less invasive, and more acceptable treatment.
Primary caregivers of children with SCD learn about HCT through social networks, social and news media, and explore HCT as a means to prevent SCD complications and help their child live a normal life.
异体造血细胞移植（HCT）和基因修饰自体造血祖细胞临床试验的结果改善，使 HCT 在镰状细胞病（SCD）中的应用范围扩大。为了了解在当前环境下探索 HCT 的主要照顾者的观点，我们提出了研究问题：“是什么促使主要照顾者决定考虑 HCT，并寻求、参加 HCT 咨询？”
我们在 SCD HCT 咨询后一周内对主要照顾者进行了定性访谈。使用扎根理论方法的开放和轴向编码阶段对数据进行分析。
我们采访了 29 位主要照顾者（26 位女性，年龄 29 至 64 岁[中位数 42]）。主要照顾者报告他们的孩子在过去一年中至少有一次出现 SCD 并发症，其中 23 位（82%）照顾者报告有并发症，8 位（28%）照顾者报告并发症很少或没有，13 位（46%）照顾者报告每周至少有 3 天疼痛。定性分析显示，主要照顾者（i）通过社交网络、社交媒体和新闻媒体了解根治性治疗选择；（ii）由于孩子生活质量下降、近期并发症、即将做出重大医疗决策或对未来严重并发症的焦虑，寻求咨询；（iii）认为基因治疗是一种新的、侵入性更小、更可接受的治疗方法。
SCD 儿童的主要照顾者通过社交网络、社交和新闻媒体了解 HCT，并探索 HCT 作为预防 SCD 并发症和帮助孩子过上正常生活的一种手段。
