Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC, 20010.
Department of Pediatrics, The George Washington University School of Medicine and Health Sciences, Washington, DC.
Pediatr Blood Cancer. 2015 Jul;62(7):1277-84. doi: 10.1002/pbc.25446. Epub 2015 Mar 23.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only available cure for sickle cell anemia (SCA). HSCT-associated risks are one of many barriers to its widespread use for SCA. The study objective was to assess parent and patient perceptions of HSCT in the era of more widespread knowledge about HSCT for SCA.
We surveyed parents of children with SCA and adolescents with SCA using the standard reference gamble paradigm. Survey responses between Africans (A) and African Americans (AA) and between parents and adolescents were compared.
Seventy-two percent (64/89) of the respondents were willing to accept ≥ 5% risk of mortality, while 57% said they would accept a risk of ≥ 10% of graft versus host disease (GVHD). Twenty-two percent were unwilling to accept any risk of mortality or GVHD. Risk averseness did not differ between A or AA respondents. Fifty-six percent of respondents were willing to accept infertility post-HSCT.
These data suggest that the majority of parents and adolescents are willing to accept the current risks associated with matched sibling HSCT for SCA. However, there continue to be significant numbers of parents and adolescents who are unwilling to accept any risk of HSCT-associated mortality or GVHD.
异基因造血干细胞移植（HSCT）是治疗镰状细胞贫血（SCA）的唯一方法。HSCT 相关风险是其广泛用于治疗 SCA 的众多障碍之一。本研究旨在评估在对 SCA 行 HSCT 有了更广泛了解的时代，父母和患者对 HSCT 的看法。
我们使用标准参考赌局范式对 SCA 患儿的父母和青少年进行了调查。比较了非洲人（A）和非裔美国人（AA）之间以及父母和青少年之间的调查结果。
72%（64/89）的受访者愿意接受≥5%的死亡率风险，而 57%的受访者表示愿意接受≥10%的移植物抗宿主病（GVHD）风险。22%的人不愿意承担任何死亡或 GVHD 风险。A 或 AA 受访者的风险规避程度没有差异。56%的受访者愿意接受 HSCT 后不孕。
这些数据表明，大多数父母和青少年愿意接受目前与同胞 HSCT 相关的治疗 SCA 的风险。然而，仍有相当数量的父母和青少年不愿意接受任何与 HSCT 相关的死亡率或 GVHD 风险。
