This retrospective study by the European Society for Blood and Marrow Transplantation analyzed the outcome of 2224 patients with myelofibrosis (MF) who underwent allogeneic stem cell transplantation (allo-SCT) between 2000 and 2014; 781 (35%) underwent myeloablative conditioning (MAC) and 1443 (65%) reduced-intensity conditioning (RIC). Median patient age was 52.9 years (range, 18 to 74 years) and 57.5 years (range, 21 to 76 years) in the MAC and RIC cohorts, respectively. Donor type was similar: matched sibling donors (MAC, 317 [41%]; RIC, 552 [38%]) and unrelated donors (MAC, 464 [59%]; RIC, 891 [62%]). Median time to both neutrophil and platelet (>20 × 109/L) engraftment did not differ between cohorts. Rates of grade II to IV acute GVHD were 28% (MAC) and 31% (RIC; P = NS). Cumulative chronic GVHD rates (limited/extensive) were 22%/27% (MAC) and 19%/31% (RIC; P = .10). Cumulative incidences of nonrelapse mortality (NRM) at 1, 3, and 5 years were 25.5%, 32.2%, and 34.6% (MAC) and 26.3%, 32.8%, and 34.4% (RIC), respectively. There was a trend toward a higher relapse rate with RIC regimens compared with MAC (P = .08); rates at 1, 3, and 5 years were 10.9%, 17.2%, and 20.1% (MAC) and 14%, 19.7%, and 23.2% (RIC), respectively. No significant difference in 5-year probabilities of overall survival (OS) was noted: MAC (53.0%; 95% confidence interval [CI], 49.1% to 56.9%) and RIC (51.0%; 95% CI, 48.3% to 53.7%); P = .78. Regarding the composite end point of GVHD-free/relapse-free survival (GRFS), the unadjusted Kaplan-Meier estimate of 5-year GRFS was 32.4% (95% CI, 29.0% to 36.1%) in the MAC group and 26.1% (95% CI, 23.9% to 28.2%) in the RIC group (P = .001). In the MAC cohort, multivariable analysis confirmed worse OS and NRM with older age (>50 years), using an unrelated donor and a Karnofsky Performance Status of 80 or less. For the RIC cohort, worse OS and NRM were associated with age 60 to 70 years compared with younger recipients, use of a mismatched donor, and poor performance status. In conclusion, although similar OS rates existed for both cohorts overall, this study suggests that MAC should still be used for younger individuals suitable for such an approach due to a trend toward less relapse and an overall suggested advantage of improved GRFS, albeit this should be examined in a more homogeneous cohort. RIC allo-SCT still offers significant survival advantage in the older, fitter MF allograft patient, and optimization to reduce significant relapse and NRM rates is required.

中文翻译：

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骨髓纤维化中强度降低的条件化同种异体造血干细胞移植：欧洲​​血液和骨髓移植学会慢性恶性肿瘤工作组的回顾性研究。

欧洲血液和骨髓移植学会的这项回顾性研究分析了2224例2000年至2014年间接受同种异体干细胞移植（allo-SCT）的骨髓纤维化（MF）患者的结局；781（35％）接受了清髓调节（MAC），1443（65％）进行了低强度调节（RIC）。MAC和RIC研究组的患者中位年龄分别为52.9岁（18至74岁）和57.5岁（21至76岁）。供体类型相似：配对的同胞供体（MAC，317 [41％]； RIC，552，[38％]）和无关的供体（MAC，464 [59％]； RIC，891 [62％]）。两组之间中性粒细胞和血小板植入的中位时间（> 20×109 / L）没有差异。II级至IV级急性GVHD的发生率分别为28％（MAC）和31％（RIC; P = NS）。慢性慢性GVHD累积发生率（有限/广泛）为22％/ 27％（MAC）和19％/ 31％（RIC; P = .10）。在1年，3年和5年时，非复发死亡率（NRM）的累积发生率分别为25.5％，32.2％和34.6％（MAC）和26.3％，32.8％和34.4％（RIC）。与MAC相比，RIC方案有更高的复发率（P = .08）；分别在1年，3年和5年时发生率分别为10.9％，17.2％和20.1％（MAC）和14％，19.7％和23.2％（RIC）。注意到5年总生存率（OS）没有显着差异：MAC（53.0％； 95％置信区间[CI]，从49.1％至56.9％）和RIC（51.0％； 95％CI，从48.3％至53.7 ％）; P ＝ 0.78。关于无GVHD /无复发生存期（GRFS）的复合终点，MAC组5年GRFS的未经调整的Kaplan-Meier估计值为32.4％（95％CI，29.0％至36.1％）和26.1％ （95％CI，23。RIC组为9％至28.2％（P = .001）。在MAC队列中，多变量分析证实年龄较大（> 50岁）的OS和NRM较差，使用的是无关的供体且Karnofsky绩效状态为80或更低。对于RIC队列，与年轻的接受者，使用不匹配的供体和表现不佳相比，年龄在60至70岁之间的OS和NRM较差。总之，尽管总体而言两个队列的OS发生率相似，但这项研究表明，由于复发率较低和GRFS改善的总体优势，MAC应该仍适用于适合这种方法的年轻个体，尽管这应该是可行的。在更同质的队列中进行了研究。RIC同种异体SCT仍然在较老的更适合MF同种异体移植患者中提供显着的生存优势