Fouillard L, Labopin M, Gratwohl A, Powles R, Bacigalupo A, Bego G, Chesnel V, Gorin N C
Department of Hematology, Hôpital Saint-Antoine, AP-HP, Paris, France.
Bone Marrow Transplant. 2002 Nov;30(10):637-43. doi: 10.1038/sj.bmt.1703712.
Outcome results of observational databases are frequently criticized as relying on incomplete information from incomplete patient populations. Few data are available to dispute these arguments of selection bias. The European Group for Blood and Marrow transplantation (EBMT) decided to address this question by evaluating the hematopoietic stem cell transplants performed in 1993. A comprehensive survey was launched in an effort to collect informations on all transplants for hematological malignancies performed throughout Europe during the year 1993. The main goals of this effort were to compare the group of spontaneously reported patients with the group of retrospectively solicited patients, and to give an accurate estimate of the outcome of all patients. For the year 1993, the annual EBMT activity survey indicated 6336 transplants performed for hematological malignancies in Europe. A total of 5651 transplants could be analyzed; 2595 were reported spontaneously by the teams (group A) and 3056 were retrieved on solicitation (group B). Patients and transplant characteristics for group A and B were very similar for most parameters with a few exceptions. There was no statistical difference for outcome at 3 years between groups A and B: disease-free survival (DFS) was 45 +/- 1% and 44 +/- 1%, relapse incidence (RI) 41 +/- 1% and 42 +/- 1%, transplant-related mortality (TRM) 23 +/- 1% and 23 +/- 1%, and overall survival (OS) 54 +/- 1% and 55 +/- 1%, respectively, for group A and group B. The real outcome at 3 years for the 5651 patients (group A + group B) transplanted in 1993 was 44 +/- 1%, 41 +/- 1%, 23 +/- 1%, and 54 +/- 1%, for DFS, RI, TRM and OS, respectively. The outcome at 3 years by transplant modality, autologous or allogeneic transplants, and by disease categories showed no difference between groups A and B.
观察性数据库的结果常常受到批评，因为其依赖来自不完整患者群体的不完整信息。几乎没有数据可用于反驳这些关于选择偏倚的观点。欧洲血液和骨髓移植组（EBMT）决定通过评估1993年进行的造血干细胞移植来解决这个问题。开展了一项全面调查，以收集1993年全欧洲所有血液系统恶性肿瘤移植的信息。这项工作的主要目标是比较自发报告患者组和回顾性征集患者组，并准确估计所有患者的结局。对于1993年，EBMT年度活动调查显示欧洲有6336例血液系统恶性肿瘤移植。总共可以分析5651例移植；2595例由各团队自发报告（A组），3056例是在征集后获取的（B组）。A组和B组的患者及移植特征在大多数参数上非常相似，只有少数例外。A组和B组在3年时的结局没有统计学差异：无病生存率（DFS）分别为45±1%和44±1%，复发率（RI）分别为41±1%和42±1%，移植相关死亡率（TRM）分别为23±1%和23±1%，总生存率（OS）分别为54±1%和55±1%。1993年接受移植的5651例患者（A组+B组）3年时的实际结局分别为：DFS为44±1%，RI为41±1%，TRM为23±1%，OS为54±1%。按移植方式（自体或异体移植）和疾病类别划分，A组和B组在3年时的结局没有差异。
