Beginning early in childhood, patients with sickle cell disease [SCD; a group of genetic haemoglobin disorders characterized by the sickle or HbS mutation (HBB E7V)] are at risk of life-threatening and debilitating health events. Despite the high morbidity and mortality of this disease, haematopoietic cell transplantation (HCT), a curative therapy for SCD, remains underutilized. A variety of factors, including the limited availability of suitable donors, play a role in this trend, but do not fully explain the low frequency with which this therapy is employed. The objective of this study was to identify paediatric haematologists' attitudes about HCT as a treatment option for SCD, and to describe the impact of these attitudes on their practices of discussing HCT with families of children affected by this disease. A nationwide survey of paediatric haematologists in the United States was conducted between February and May 2016. Two hundred and eighty-seven surveys were included in the final analysis (response rate 20%). On average, respondents reported informing 42% of families about HCT as a treatment option (N = 248, 95% confidence interval: 38-46). Clinician attitudes about the cost and safety of HCT were associated with practices of discussing this therapy with families. These findings suggest that clinician attitudes and referral practices may play a role in the underutilization of this therapy in the SCD population.

中文翻译：

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小儿血液学家对造血细胞移植治疗镰状细胞病的态度。

从儿童早期开始，患有镰状细胞病的患者[SCD; 一组以镰刀或HbS突​​变（HBB E7V）为特征的遗传性血红蛋白疾病]危及生命，使人的健康状况恶化。尽管该病的发病率和死亡率高，但造血干细胞移植（HCT）（一种治疗SCD的疗法）仍未得到充分利用。各种因素，包括合适的供体的有限可用性，在这种趋势中起作用，但是不能完全解释采用这种疗法的频率低。这项研究的目的是确定儿科血液学家对HCT作为SCD的治疗选择的态度，并描述这些态度对他们与受此疾病影响的儿童家庭讨论HCT的做法的影响。2016年2月至2016年5月在美国进行了一次全国儿科血液学家调查。最终分析中包括287项调查（答复率为20％）。平均而言，受访者报告说将42％的家庭告知HCT作为治疗选择（N = 248，95％置信区间：38-46）。临床医生对HCT的成本和安全性的态度与与家人讨​​论这种疗法的实践有关。这些发现表明，在SCD人群中，临床医生的态度和转诊做法可能会在该疗法的未充分利用中发挥作用。受访者报告说，有42％的家庭将HCT作为治疗选择（N = 248，95％的置信区间：38-46）。临床医生对HCT的成本和安全性的态度与与家人讨​​论这种疗法的实践有关。这些发现表明，在SCD人群中，临床医生的态度和转诊做法可能会在该疗法的未充分利用中发挥作用。受访者报告说，有42％的家庭将HCT作为治疗选择（N = 248，95％的置信区间：38-46）。临床医生对HCT的成本和安全性的态度与与家人讨​​论这种疗法的实践有关。这些发现表明，在SCD人群中，临床医生的态度和转诊做法可能会在该疗法的未充分利用中发挥作用。