Claudiani Simone, Marktel Sarah, Piemontese Simona, Assanelli Andrea, Lupo-Stanghellini Maria Teresa, Carrabba Matteo, Guggiari Elena, Giglio Fabio, De Freitas Tiago, Marcatti Magda, Bernardi Massimo, Corti Consuelo, Peccatori Jacopo, Lunghi Francesca, Ciceri Fabio
Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milano, Italy.
Hematol Oncol. 2016 Sep;34(3):154-60. doi: 10.1002/hon.2183. Epub 2014 Dec 3.
Allogeneic transplantation is the only potentially curative strategy for myelofibrosis, even in the era of new drugs that so far only mitigate symptoms. The choice to proceed to allogeneic transplantation is based on several variables including age, disease phase, degree of splenomegaly, donor availability, comorbidities and iron overload. These factors, along with conditioning regimen and time to transplantation, may influence the outcome of ASCT. We report 14 patients affected by myelofibrosis with a median age of 57 years (range, 41-76) receiving a treosulfan-fludarabine based reduced toxicity conditioning. Patients (pts) received a stem cell transplantation from an HLA identical (n = 10) or matched unrelated donor (n = 4). All pts had a complete myeloablation followed by engraftment and in 12 out of 13 evaluated pts donor chimerism was 100% at 1 month. In most cases a reduction of splenomegaly and a reduction (or resolution) of bone marrow fibrosis was observed. After a median follow-up of 39 months (range, 3-106), the 3-year probability of overall survival and disease free survival was 54 +/- 14% and 46 +/- 14%, respectively. The cumulative incidence of non-relapse mortality at 2 years was 39 +/- 15%. Causes of non-relapse mortality were: infection (n = 2), GvHD (n = 2) and haemorrhage (n = 1). We can conclude that a treosulfan and fludarabine based conditioning has a potent myeloablative and anti-disease activity although non-relapse mortality remains high in this challenging clinical setting. Copyright © 2014 John Wiley & Sons, Ltd.
异基因移植是骨髓纤维化唯一可能治愈的策略，即使在目前仅能缓解症状的新药时代亦是如此。进行异基因移植的选择基于多个变量，包括年龄、疾病分期、脾肿大程度、供体可用性、合并症和铁过载。这些因素，连同预处理方案和移植时间，可能会影响自体造血干细胞移植（ASCT）的结果。我们报告了14例骨髓纤维化患者，中位年龄57岁（范围41 - 76岁），接受了基于曲奥舒凡-氟达拉滨的低毒性预处理。患者接受了来自人类白细胞抗原（HLA）相同供体（n = 10）或匹配的无关供体（n = 4）的干细胞移植。所有患者均实现了完全骨髓清除并随后植入，在13例评估患者中的12例中，1个月时供体嵌合率为100%。在大多数病例中，观察到脾肿大减轻以及骨髓纤维化减轻（或消退）。中位随访39个月（范围3 - 106个月）后，3年总生存率和无病生存率分别为54±14%和46±14%。2年非复发死亡率的累积发生率为39±15%。非复发死亡原因包括：感染（n = 2）、移植物抗宿主病（GvHD，n = 2）和出血（n = 1）。我们可以得出结论，基于曲奥舒凡和氟达拉滨的预处理具有强大的骨髓清除和抗疾病活性，尽管在这种具有挑战性的临床环境中非复发死亡率仍然很高。版权所有© 2014约翰威立父子有限公司。
