Malagola Michele, Polverelli Nicola, Martino Massimo, Patriarca Francesca, Bruno Benedetto, Giaccone Luisa, Grillo Giovanni, Bramanti Stefania, Bernasconi Paolo, De Gobbi Marco, Natale Annalisa, Terruzzi Elisabetta, Olivieri Attilio, Chiusolo Patrizia, Carella Angelo Michele, Casini Marco, Maffini Enrico, Nozzoli Chiara, Mazza Patrizio, Bassi Simona, Onida Francesco, Vacca Adriana, Falcioni Sadia, Luppi Mario, Iori Anna Paola, Pavone Vincenzo, Skert Cristina, Carluccio Paola, Borghero Carlo, Proia Anna, Selleri Carmine, Rubini Vicky, Sacchi Nicoletta, Oldani Elena, Bonifazi Francesca, Ciceri Fabio, Russo Domenico
Department of Clinical and Experimental Sciences, Blood Diseases and Cell Therapies Unit, Bone Marrow Transplant Unit, "Azienda Socio Santiaria Territoriale Spedali Civili" Hospital of Brescia, University of Brescia, Brescia, Italy.
Stem Cell Transplant and Cellular Therapies Unit, "Bianco Melacrino Morelli" Hospital, Reggio Calabria, Italy.
Transplant Direct. 2023 Feb 22;9(3):e1451. doi: 10.1097/TXD.0000000000001451. eCollection 2023 Mar.
The conditioning regimens with different alkylators at different doses can influence the outcome of allogeneic stem cell transplantation (SCT), but conclusive data are missing.
With the aim to analyze real-life allogeneic SCTs performed in Italy between 2006 and 2017 in elderly patients (aged >60 y) with acute myeloid leukemia or myelodysplastic syndrome, we collected 780 first transplants data. For analysis purposes, patients were grouped according to the type of alkylator included in the conditioning (busulfan [BU]-based; n = 618; 79%; treosulfan [TREO]-based; n=162; 21%).
No significant differences were observed in nonrelapse mortality, cumulative incidence of relapse, and overall survival, although in the TREO-based group, we observed a greater proportion of elderly patients ( < 0.001); more active diseases at the time of SCT ( < 0.001); a higher prevalence of patients with either hematopoietic cell transplantation-comorbidity index ≥3 ( < 0.001) or a good Karnofsky performance status ( = 0.025); increased use of peripheral blood stem cells as graft sources ( < 0.001); and greater use of reduced intensity conditioning regimens ( = 0.013) and of haploidentical donors ( < 0.001). Moreover, the 2-y cumulative incidence of relapse with myeloablative doses of BU was significantly lower than that registered with reduced intensity conditioning (21% versus 31%; = 0.0003). This was not observed in the TREO-based group.
Despite a higher number of risk factors in the TREO group, no significant differences were observed in nonrelapse mortality, cumulative incidence of relapse, and overall survival according to the type of alkylator, suggesting that TREO has no advantage over BU in terms of efficacy and toxicity in acute myeloid leukemia and myelodysplastic syndrome.
不同剂量的不同烷化剂预处理方案会影响异基因干细胞移植（SCT）的结果，但确凿数据尚缺。
为分析2006年至2017年期间在意大利对急性髓系白血病或骨髓增生异常综合征老年患者（年龄>60岁）进行的实际异基因SCT情况，我们收集了780例首次移植的数据。为便于分析，根据预处理中所含烷化剂类型将患者分组（白消安[BU]为基础；n = 618；79%；苏消安[TREO]为基础；n = 162；21%）。
在非复发死亡率、复发累积发生率和总生存率方面未观察到显著差异，尽管在以TREO为基础的组中，我们观察到老年患者比例更高（<0.001）；SCT时疾病更活跃（<0.001）；造血细胞移植合并症指数≥3的患者或卡诺夫斯基表现状态良好的患者患病率更高（<0.001或=0.025）；作为移植物来源的外周血干细胞使用增加（<0.001）；以及更多地使用减低强度预处理方案（=0.013）和单倍体相合供者（<0.001）。此外，白消安清髓剂量的2年复发累积发生率显著低于减低强度预处理的发生率（21%对31%；=0.0003）。在以TREO为基础的组中未观察到此现象。
尽管TREO组的危险因素更多，但根据烷化剂类型，在非复发死亡率、复发累积发生率和总生存率方面未观察到显著差异，这表明在急性髓系白血病和骨髓增生异常综合征的疗效和毒性方面，TREO并不优于白消安。
