Medical Oncology and Hematology, Princess Margaret Cancer Centre, University of Toronto, Toronto, Ontario, Canada.
Department of Biostatistics, Princess Margaret Cancer Centre, University of Toronto, Toronto, Ontario, Canada.
Transplant Cell Ther. 2021 Jul;27(7):600.e1-600.e8. doi: 10.1016/j.jtct.2021.03.027. Epub 2021 Mar 30.
Despite the curative potential of allogeneic hematopoietic cell transplantation (HCT) for myelofibrosis (MF), a significant number of patients with MF do not undergo HCT. Factors influencing treatment preferences in these patients have not been well studied. This study was conducted to identify patient-, disease-, and donor-related factors influencing the decision regarding HCT in patients with MF. A secondary objective was to compare survival between patients who elected upfront HCT and those who opted for nontransplantation therapy. We conducted a retrospective chart review amongst patients meeting criteria for transplant indication, evaluating clinical characteristics, treatment preferences, and outcomes. Of the 183 study eligible patients age <70 years, 129 (70%) developed an HCT indication. Age >60 years was significantly associated with higher rates of HLA-typing refusal (13 of 72 versus 1 of 44; P = .02). Caucasian ethnicity was significantly associated with an increased rate of identifying well-matched donors compared with non-Caucasian ethnicity (75% versus 48%; P = .02). Of the 69 patients with well-matched donors, 34 (49%) preferred to not pursue upfront HCT despite an indication for transplantation. Patient preference for nontransplantation therapies was the most common reason for declining HCT. We did not find any difference in survival between patients pursuing upfront HCT and those opting for nontransplantation therapies, although more patients in the HCT arm were in remission at the last follow-up. Patients of Caucasian ethnicity were significantly more likely than non-Caucasian patients to identify a well-matched donor. Despite availability of a well-matched donor, a significant proportion of MF patients with an indication for transplantation do not pursue HCT. Patient age, donor type, and patient preference play major roles in the selection of upfront HCT. Although a survival difference was not observed between upfront HCT versus non-transplant therapy, more patients in the HCT arm were in remission at the last follow-up.
尽管同种异体造血细胞移植（HCT）对骨髓纤维化（MF）具有治疗潜力，但仍有相当一部分 MF 患者未接受 HCT。尚未对影响这些患者治疗偏好的因素进行很好的研究。本研究旨在确定影响 MF 患者 HCT 决策的患者、疾病和供体相关因素。次要目标是比较选择 upfront HCT 和非移植治疗的患者的生存情况。我们对符合移植指征的患者进行了回顾性图表审查，评估了临床特征、治疗偏好和结果。在 183 名年龄<70 岁的研究合格患者中，129 名（70%）出现了 HCT 指征。年龄>60 岁与 HLA 配型拒绝率较高显著相关（72 例中有 13 例，44 例中有 1 例；P=0.02）。与非白种人相比，白种人明显更有可能找到匹配良好的供体（75%比 48%；P=0.02）。在 69 名有匹配良好供体的患者中，尽管有移植指征，但 34 名（49%）患者仍不愿立即接受 HCT。患者对非移植治疗的偏好是拒绝 HCT 的最常见原因。尽管 upfront HCT 组的患者在最后一次随访时更有可能处于缓解状态，但我们未发现接受 upfront HCT 和选择非移植治疗的患者之间的生存差异。与非白种人相比，白种人患者更有可能找到匹配良好的供体。尽管有匹配良好的供体，但仍有相当一部分有移植指征的 MF 患者不接受 HCT。患者年龄、供体类型和患者偏好在 upfront HCT 的选择中起着重要作用。尽管 upfront HCT 与非移植治疗之间未观察到生存差异，但在最后一次随访时，HCT 组的更多患者处于缓解状态。
