Division of Hematology and Oncology, SUNY Downstate Health Sciences University, Brooklyn, New York, NY, USA.
GM and TJ are equal first authors.
Cell Transplant. 2021 Jan-Dec;30:9636897211046559. doi: 10.1177/09636897211046559.
Hematopoietic stem cell transplant (HSCT) is the only cure for patients with sickle cell disease (SCD). Although most SCD patients experience progressive end-organ damage and shortened lifespans, not all patients follow the same disease course, tempo, or outcome. Therefore, the dilemma facing physicians is weighing the selection of patients and timing for the procedure against donor type and transplant-related mortality and morbidity that go up with increasing age. On the other hand, the dilemma facing the patients and families is how acceptable HSCT that carries some mortality risks to them. We have analyzed the chronic conditions due to SCD in 449 patients to determine whether SCD-related multiple chronic conditions (MCC) can be risk-stratified to identify the group of patients predicted to not only have shortened lifespans but also functional limitation and poor quality of life so that these at-risk patients can be offered HSCT early and before MCC develops. We identified that the age of onset of the first SCD-related chronic conditions strongly predicted for the risks for disease-related MCC. SCD patients who suffered their first disease-related chronic condition before age 30 years developed MCC at a rate of 19.1 times faster than those at a later age. These patients are therefore high-risk patients and should be offered HSCT early in the course of their disease before multiple organ damage intervenes, even if matched-related donors are not available. This patient selection and timing approach provides a forum for an easy-to-understand and real-time discussion, including the choice of donor type, with SCD patients and families when considering HSCT.
造血干细胞移植（HSCT）是治疗镰状细胞病（SCD）患者的唯一方法。虽然大多数 SCD 患者会经历进行性终末器官损伤和寿命缩短，但并非所有患者都遵循相同的疾病进程、节奏或结果。因此，医生面临的困境是权衡患者的选择和手术时机，以及供体类型和与移植相关的死亡率和发病率，这些因素随着年龄的增长而增加。另一方面，患者和家属面临的困境是他们如何接受带有一定死亡率风险的 HSCT。我们分析了 449 例 SCD 患者的慢性疾病，以确定 SCD 相关的多种慢性疾病（MCC）是否可以进行风险分层，以识别不仅预期寿命缩短，而且存在功能限制和生活质量差的患者群体，以便这些高危患者可以在 MCC 发生之前尽早接受 HSCT。我们发现，首次发生 SCD 相关慢性疾病的年龄强烈预测了疾病相关 MCC 的风险。在 30 岁之前首次发生疾病相关慢性疾病的 SCD 患者，发生 MCC 的风险比年龄较大的患者高 19.1 倍。这些患者是高危患者，应在疾病过程早期，在多个器官损伤介入之前，接受 HSCT，即使没有匹配相关供体。这种患者选择和时机的方法为 SCD 患者和家属提供了一个易于理解和实时的讨论平台，包括在考虑 HSCT 时供体类型的选择。
