Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Department of Haematology, Ampang Hospital, Selangor, Malaysia.
Eur J Haematol. 2018 Sep;101(3):305-317. doi: 10.1111/ejh.13099. Epub 2018 Jul 4.
Ruxolitinib is the first approved drug for treatment of myelofibrosis, but its impact of outcome after allogeneic stem cell transplantation (ASCT) is unknown.
We reported on 159 myelofibrosis patients (pts) with a median age of 59 years (r: 28-74) who received reduced intensity ASCT between 2000 and 2015 in eight German centers from related (n = 23), matched (n = 86) or mismatched (n = 50) unrelated donors. Forty-six (29%) patients received ruxolitinib at any time point prior to ASCT. The median daily dose of ruxolitinib was 30 mg (range 10-40 mg) and the median duration of treatment was 4.9 months (range 0.4-39.1 months).
Primary graft failure was seen in 2 pts (4%) in the ruxolitinib and 3 (2%) in the non-ruxolitinib group. Engraftment and incidence of acute GVHD grade II to IV and III/IV did not differ between groups (37% vs 39% and 19% vs 28%, respectively), nor did the non-relapse mortality at 2 years (23% vs 23%). A trend for lower risk of relapse was seen in the ruxolitinib group (9% vs 17%, P = .2), resulting in a similar 2 year DFS and OS (68% vs 60% and 73% vs 70%, respectively). No difference in any outcome variable could be seen between ruxolitinib responders and those who failed or lost response to ruxolitinib.
These results suggest that ruxolitinib pretreatment in myelofibrosis patient does not negatively influence outcome after allogeneic stem cell transplantation.
芦可替尼是首个获批用于治疗骨髓纤维化的药物,但它对异基因造血干细胞移植(allo-SCT)后结局的影响尚不清楚。
我们报告了 159 例骨髓纤维化患者(pts),中位年龄为 59 岁(范围 28-74 岁),他们于 2000 年至 2015 年间在德国的 8 个中心接受了来自亲缘(n=23)、匹配(n=86)或不匹配(n=50)无关供者的减低强度 allo-SCT。46(29%)例患者在 allo-SCT 前的任何时间点接受了芦可替尼治疗。芦可替尼的中位日剂量为 30mg(范围 10-40mg),中位治疗时间为 4.9 个月(范围 0.4-39.1 个月)。
芦可替尼组有 2 例(4%)出现原发性移植物失败,非芦可替尼组有 3 例(2%)出现。两组间嵌合状态、急性移植物抗宿主病Ⅱ-Ⅳ级和Ⅲ/Ⅳ级以及非复发死亡率的发生率无差异(分别为 37% vs 39%和 19% vs 28%,2 年时分别为 23% vs 23%)。芦可替尼组复发风险较低(9% vs 17%,P=0.2),导致 2 年无进展生存(DFS)和总生存(OS)率相似(分别为 68% vs 60%和 73% vs 70%)。芦可替尼应答者与未应答或芦可替尼治疗失败者在任何结局变量上均无差异。
这些结果表明,芦可替尼预处理对骨髓纤维化患者allo-SCT 后的结局没有负面影响。