Kröger N, Bornhäuser M, Stelljes M, Pichlmeier U, Trenschel R, Schmid C, Arnold R, Martin H, Heinzelmann M, Wolschke C, Meyer R G, Bethge W, Kobbe G, Ayuk F, Gökbuget N, Hölzer D, Zander A, Beelen D
Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Department of Hematology/Oncology, University Hospital Dresden, Dresen, Germany.
Bone Marrow Transplant. 2015 Dec;50(12):1503-7. doi: 10.1038/bmt.2015.202. Epub 2015 Sep 14.
TBI-based preparative regimens are considered as standard conditioning therapy for allogeneic stem cell transplantation (AHSC) in patients with ALL. We investigated toxicity and efficacy of a non-TBI-based regimen consisting of treosulfan, etoposide and cyclophosphamide for ALL within a prospective study. Major inclusion criteria were CR and non-eligibility for TBI. Fifty patients with a median age of 46.5 years (range, 18-64) were included. Donors were HLA-identical sibling (n=8), matched (n=42) or mismatched (n=10) unrelated. The toxicity was moderate, resulting in a cumulative incidence of non-relapse mortality (NRM) at 1 year of 8% (90% confidence interval: 2-15%). Acute GvHD grade II-IV and grade III/IV was noted in 53% and 14%, respectively. Chronic GvHD at one year was seen in 41%. After a median follow-up of 24 months the cumulative incidence of relapse was 36% (90% confidence interval: 24-48) and 51% (90% confidence interval: 37-65) at 1 and 2 years, respectively. The estimated 2-year disease-free and overall survivals were 36 and 48%, respectively. Treosulfan, etoposide and cyclophosphamide followed by AHSC has a favorable toxicity profile with low NRM and therefore represents a potential alternative regimen for ALL in 1. CR (NCT00682305).
基于全身照射（TBI）的预处理方案被认为是急性淋巴细胞白血病（ALL）患者异基因干细胞移植（AHSC）的标准预处理疗法。我们在一项前瞻性研究中调查了由曲奥舒凡、依托泊苷和环磷酰胺组成的非TBI方案对ALL的毒性和疗效。主要纳入标准为完全缓解（CR）且不适合进行TBI。纳入了50例患者，中位年龄为46.5岁（范围18 - 64岁）。供者为HLA全相合的同胞（n = 8）、配型相合（n = 42）或配型不相合（n = 10）的无关供者。毒性为中度，1年时非复发死亡率（NRM）的累积发生率为8%（90%置信区间：2 - 15%）。分别有53%和14%的患者出现II - IV级和III/IV级急性移植物抗宿主病（GvHD）。1年时慢性GvHD的发生率为41%。中位随访24个月后，1年和2年时复发的累积发生率分别为36%（90%置信区间：24 - 48）和51%（90%置信区间：37 - 65）。估计2年无病生存率和总生存率分别为36%和48%。曲奥舒凡、依托泊苷和环磷酰胺序贯AHSC具有良好的毒性特征，NRM较低，因此代表了ALL在1. CR时的一种潜在替代方案（NCT00682305）。
