Kröger Nicolaus, Thiele Jürgen, Zander Axel, Schwerdtfeger Rainer, Kobbe Guido, Bornhäuser Martin, Bethge Wolfgang, Schubert Jörg, de Witte Theo, Kvasnicka Hans Michael
Department of Stem Cell Transplantation, University Hospital Hamburg, Germany.
Exp Hematol. 2007 Nov;35(11):1719-22. doi: 10.1016/j.exphem.2007.08.022.
To investigate the effect of a busulfan/fludarabine-based reduced intensity conditioning followed by allogeneic stem cell transplantation on regression of bone marrow fibrosis in patients with myelofibrosis.
Twenty-four patients (male, n = 16; female, n = 8) with a median age of 52 years (range, 32-63 years) were included. Six patients were transplanted from human leukocyte antigen-identical siblings and 18 patients from matched unrelated donors. Diagnosis was primary myelofibrosis in 18 patients and secondary myelofibrosis in 6 patients; in 4 of them, primary myelofibrosis evolved from polycythemia vera, and in 2 of them from essential thrombocythemia. Using the European Consensus on grading bone marrow fibrosis, all patients had advanced marrow fibrosis MF-2 (n = 13) or MF-3 (n = 11) before allografting According to the Lille Risk Factor Scoring System, patients were classified as low risk (n = 5), intermediate risk (n = 16), or high risk (n = 3).
After stem cell transplantation, a complete (MF-0) or nearly complete (MF-1) regression of bone marrow fibrosis was seen in 59% at day +100, in 90% at day +180, and in 100% at day +360. No correlation between occurrence of acute graft-vs-host disease and fibrosis regression on day +180 was observed.
This study shows that allogeneic stem cell transplantation after reduced-intensity conditioning resulted in rapid regression of bone-marrow fibrosis.
探讨白消安/氟达拉滨减低剂量预处理后行异基因干细胞移植对骨髓纤维化患者骨髓纤维化消退的影响。
纳入24例患者（男性16例，女性8例），中位年龄52岁（范围32 - 63岁）。6例患者接受来自人类白细胞抗原相合同胞的移植，18例患者接受来自匹配无关供者的移植。18例诊断为原发性骨髓纤维化，6例为继发性骨髓纤维化；其中4例原发性骨髓纤维化由真性红细胞增多症演变而来，2例由原发性血小板增多症演变而来。根据欧洲骨髓纤维化分级共识，所有患者在同种异体移植前均有晚期骨髓纤维化MF - 2（13例）或MF - 3（11例）。根据里尔危险因素评分系统，患者分为低危（5例）、中危（16例）或高危（3例）。
干细胞移植后，在第100天59%的患者骨髓纤维化完全（MF - 0）或接近完全（MF - 1）消退，第180天为90%，第360天为100%。未观察到急性移植物抗宿主病的发生与第180天纤维化消退之间存在相关性。
本研究表明，减低剂量预处理后行异基因干细胞移植可使骨髓纤维化迅速消退。
