Gratwohl A, Hermans J, Goldman J M, Arcese W, Carreras E, Devergie A, Frassoni F, Gahrton G, Kolb H J, Niederwieser D, Ruutu T, Vernant J P, de Witte T, Apperley J
Department of Internal Medicine, Kantonsspital, Basel, Switzerland.
Lancet. 1998 Oct 3;352(9134):1087-92. doi: 10.1016/s0140-6736(98)03030-x.
Transplantation of blood or bone-marrow stem cells is the treatment of choice for selected patients with chronic myeloid leukaemia (CML). Transplantation is used with increasing frequency and success, but remains associated with substantial risks of morbidity and mortality. Other treatments with satisfactory short-term outcome are available. For appropriate counselling of patients, a rapid and simple way to assess risk is needed.
Data from 3142 patients (1873 [60%] male, 1269 [40%] female; mean age 34 years, range <1-60 years) treated with allogeneic blood or marrow transplants for CML between 1989 and 1997, reported to the European Group for Blood and Marrow Transplantation (EBMT), were used to develop and test a simple risk score based on previously reported major pretransplant risk factors: histocompatibility, stage of disease at time of transplantation, age and sex of donor and recipient, and time from diagnosis to transplantation. We analysed probabilities of survival, leukaemia-free survival, transplant-related mortality, and relapse incidence with respect to these risk factors.
At the time of analysis, 1922 (61%) of the 3142 patients were alive-1567 (65%) of those with HLA-identical sibling donors and 417 (57%) of those with unrelated donors. 1682 (54%) were alive without relapse. 1220 (39%) patients had died, 1013 (83%) of transplant-related causes, 207 (17%) of relapse. 447 (14%) patients had relapsed. The final scoring system was highly predictive for leukaemia-free survival, survival and transplant-related mortality. Survival at 5 years was 72%, 70%, 62%, 48%, 40%, 18%, and 22% for patients with scores 0, 1, 2, 3, 4, 5, and 6, respectively. Risk of transplant-related mortality was 20%, 23%, 31%, 46%, 51%, 71%, and 73%. Data showed the same trends for HLA-identical sibling transplants and unrelated transplants for transplants done in 1989-93 and 1994-96.
Pretransplant risk factors are cumulative for individual patients with CML having blood or marrow transplantation. A simple system based on five main factors gives adequate risk assessment for counselling of patients and taking decisions.
血液或骨髓干细胞移植是特定慢性髓性白血病（CML）患者的首选治疗方法。移植的使用频率和成功率不断提高，但仍伴有较高的发病和死亡风险。也有其他短期疗效令人满意的治疗方法。为了给患者提供适当的咨询，需要一种快速简便的风险评估方法。
1989年至1997年间，向欧洲血液和骨髓移植组（EBMT）报告的3142例接受异基因血液或骨髓移植治疗CML的患者（1873例[60%]男性，1269例[40%]女性；平均年龄34岁，范围<1 - 60岁）的数据，被用于开发和测试一个基于先前报告的主要移植前风险因素的简单风险评分：组织相容性、移植时疾病分期、供体和受体的年龄及性别，以及从诊断到移植的时间。我们分析了这些风险因素下的生存概率、无白血病生存概率、移植相关死亡率和复发率。
在分析时，3142例患者中有1922例（61%）存活，其中HLA相同的同胞供体患者有1567例（65%），无关供体患者有417例（57%）。1682例（54%）存活且未复发。1220例（39%）患者死亡，1013例（83%）死于移植相关原因，207例（17%）死于复发。447例（14%）患者复发。最终的评分系统对无白血病生存、生存和移植相关死亡率具有高度预测性。评分分别为0、1、2、3、4、5和6的患者5年生存率分别为72%、70%、62%、48%、40%、18%和22%。移植相关死亡风险分别为20%、23%、31%、46%、51%、71%和73%。1989 - 1993年和1994 - 1996年进行的HLA相同的同胞移植和无关移植的数据显示出相同趋势。
移植前风险因素对于接受血液或骨髓移植的CML个体患者是累积性的。一个基于五个主要因素的简单系统可为患者咨询和决策提供充分的风险评估。
