Department of Pediatrics, Division of Hematology-Oncology-BMT, Emory University School of Medicine, Atlanta, Georgia.
Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, Atlanta, Georgia.
JAMA Netw Open. 2020 May 1;3(5):e206742. doi: 10.1001/jamanetworkopen.2020.6742.
Bone marrow transplant (BMT) is a potentially curative treatment for sickle cell disease (SCD). Patient and caregiver attitudes toward BMT for SCD and the willingness to accept risks of BMT vary, but these attitudes are not well understood.
To understand patient and caregiver perceptions of and attitudes toward BMT for SCD and decision-making about BMT.
DESIGN, SETTING, AND PARTICIPANTS: Qualitative study of interview transcripts from a convenience sample. Transcripts were from adults with SCD and caregivers of patients with SCD recruited from national and regional SCD conferences, symposia, and sickle cell clinics in 2 cities. Interview transcripts were used from the needs assessment phase to develop a patient-decision aid in 2013 to 2014 (group 1) and from the baseline point in 2015 to 2016 (group 2) of the parent trial, a randomized clinical trial of adults and caregivers of patients with SCD to evaluate the effectiveness of a patient decision aid.
Participant perspectives on decision-making regarding BMT for SCD.
Fifty-seven transcripts from adults with SCD and 50 transcripts from caregivers of patients with SCD were included. Median (interquartile range [IQR]) age of adults with SCD was 34 (21-50) years in group 1 and 30 (23-38) years in group 2. The median (IQR) age of caregivers was 42.5 (31-52) years in group 1 and 41 (35-46.5) years in group 2. Most transcripts from adults with SCD (75.0% in group 1 and 72.4% in group 2) and caregivers of patients with SCD (76.7% in group 1 and 85.0% in group 2) were from female participants. Bone marrow transplant was perceived as a treatment option associated with serious risks. Reported attitudes toward BMT occurred on a continuum ranging from unfavorable to favorable. Participants reported serious decisional dilemma regarding BMT for SCD. Most participants expressed interest in learning about BMT or curative treatments.
This qualitative study found a continuum in attitudes toward BMT for SCD and highlights the complexity of decision-making in BMT for SCD. Patients and families with SCD expressed an interest in learning about BMT. Future prospective studies of patient decision-making regarding BMT, especially in the context of emerging curative and novel disease-modifying therapies for SCD, are warranted.
骨髓移植（BMT）是治疗镰状细胞病（SCD）的一种潜在治愈方法。患者和护理人员对 SCD 进行 BMT 的态度以及对 BMT 风险的接受意愿各不相同，但这些态度尚未得到充分理解。
了解患者和护理人员对 SCD 进行 BMT 的看法和态度，以及对 BMT 的决策。
设计、地点和参与者：从便利样本的访谈记录中进行定性研究。记录来自全国和地区 SCD 会议、研讨会以及 2 个城市的 SCD 诊所招募的 SCD 患者的成年患者和护理人员。访谈记录用于 2013 年至 2014 年（第 1 组）开发患者决策辅助工具的需求评估阶段，以及 2015 年至 2016 年（第 2 组）父母试验的基线点，这是一项评估成年人和 SCD 患者护理人员的随机临床试验患者决策辅助工具的有效性。
患者对 SCD 进行 BMT 的决策的看法。
第 1 组和第 2 组分别纳入了 57 份 SCD 成年患者的转录本和 50 份 SCD 患者护理人员的转录本。SCD 成年患者的中位（四分位距 [IQR]）年龄分别为第 1 组的 34（21-50）岁和第 2 组的 30（23-38）岁。护理人员的中位（IQR）年龄分别为第 1 组的 42.5（31-52）岁和第 2 组的 41（35-46.5）岁。大多数 SCD 成年患者（第 1 组的 75.0%和第 2 组的 72.4%）和 SCD 患者护理人员（第 1 组的 76.7%和第 2 组的 85.0%）的转录本来自女性参与者。骨髓移植被视为一种伴有严重风险的治疗选择。报告的 BMT 态度在从不利到有利的连续体上有所不同。参与者报告了 SCD 骨髓移植的严重决策困境。大多数参与者表示有兴趣了解 BMT 或治愈性治疗。
这项定性研究发现 SCD 骨髓移植的态度呈连续变化，并强调了 SCD 骨髓移植决策的复杂性。SCD 患者和家属表示有兴趣了解 BMT。未来需要对 SCD 患者关于 BMT 的决策进行前瞻性研究，特别是在新兴的治愈性和新型疾病修正治疗 SCD 的背景下。
