Souillet G, Rey S, Bertrand Y, Pujol M, Pondarré C, Bourgeot J P, Raba M, Eljaafari A, Chapuis F, Manel A M, Philippe N, Gebuhrer L
Paediatrics Immunohaematology and Bone Marrow Transplantation, Hopital Debrousse, Lyon, France.
Bone Marrow Transplant. 2000 Jul;26(1):31-43. doi: 10.1038/sj.bmt.1702460.
The aim of the study was to evaluate the outcome of unrelated bone marrow donor (UBMD) searches initiated for 174 children between 1986 and 1997. Seven patients were registered twice so that a total of 181 UBMD searches took place. At the time of registration, patients suffered from hematological malignancies (n = 121), non-malignant hemopathies (n = 26) and inborn errors (n = 34). Forty-five of the patients (26%) were given transplants from unrelated donors of whom 26 (58%) were HLA-mismatched transplants. Our strategy accepted HLA mismatches at the time of donor selection, using Thymoglobuline as part of the conditioning regimen. Of the 45 patients given unrelated donor transplants, overall survival was 60% at 3 years and concerned 27 patients of whom 14 were from HLA-mismatched donors. Disease-free survival for hematological malignancies was 65% in HLA-matched transplants and 50% in HLA-mismatched transplants. For some patients (16%) urgency led us to use alternative options: non-identical related donor (n = 14), autograft (n = 10), related cord blood transplant (n = 4). For others, UBMD searches were stopped because of favorable evolution (n = 29), death (n = 24), disease progression (n = 22) or other reasons (n = 21). By the end of the follow-up period, 88 patients had died (50%), 75 (43%) are currently alive with or without being transplanted of whom eight are still having active searches and 11 are no longer contactable. In conclusion, in severe disease in children, an immediate transplant from a partially matched donor might be preferable to a prolonged search for a full match. Consequently, this strategy increases the number of patients for whom a suitable donor can be found. We have chosen this option in order not to delay BMT; in so doing we have obtained encouraging results which include high overall survival, low incidence of acute GVHD grade III-IV and low percentage of relapse even in mismatched pairs.
本研究的目的是评估1986年至1997年间为174名儿童启动的无关骨髓供者(UBMD)搜索的结果。7名患者进行了两次登记,因此总共进行了181次UBMD搜索。登记时,患者患有血液系统恶性肿瘤(n = 121)、非恶性血液病(n = 26)和先天性疾病(n = 34)。45名患者(26%)接受了无关供者的移植,其中26名(58%)为HLA配型不合的移植。我们的策略是在供者选择时接受HLA配型不合,使用胸腺球蛋白作为预处理方案的一部分。在45名接受无关供者移植的患者中,3年总生存率为60%,涉及27名患者,其中14名来自HLA配型不合的供者。血液系统恶性肿瘤的无病生存率在HLA配型相合的移植中为65%,在HLA配型不合的移植中为50%。对于一些患者(16%),紧急情况导致我们使用替代方案:非同基因相关供者(n = 14)、自体移植(n = 10)、相关脐血移植(n = 4)。对于其他患者,由于病情好转(n = 29)、死亡(n = 24)、疾病进展(n = 22)或其他原因(n = 21),UBMD搜索被停止。到随访期结束时,88名患者死亡(50%),75名(43%)目前存活,无论是否接受移植,其中8名仍在进行活跃搜索,11名无法再取得联系。总之,在儿童严重疾病中,立即接受部分匹配供者的移植可能比长时间寻找完全匹配的供者更可取。因此,这种策略增加了能够找到合适供者的患者数量。我们选择这个方案是为了不延迟骨髓移植;这样做我们取得了令人鼓舞的结果,包括高总生存率、低III-IV级急性移植物抗宿主病发生率以及即使在配型不合的配对中也有低复发率。