Department of Hematology, Ankara University Faculty of Medicine, Ankara, Turkey
Balkan Med J. 2023 May 8;40(3):197-204. doi: 10.4274/balkanmedj.galenos.2023.2022-2-32. Epub 2023 Mar 24.
Allogeneic hematopoietic stem cell transplantation is a well-established approach for patients diagnosed with primary myelofibrosis and remains the only potentially curative treatment.
To present the overall outcome of patients with myelofibrosis treated with allogeneic hematopoietic stem cell transplantation.
A retrospective cross-sectional study.
This study is a retrospective analysis of 26 consecutive patients with primary myelofibrosis who underwent transplantation at our center between January 2002 and January 2022. Disease and transplant variables contributing to outcomes were analyzed.
The median age at the time of transplantation was 52.5 (range, 32-63) years and the median time from diagnosis to allogeneic hematopoietic stem cell transplantation was 25 (range, 3.1-156.8) months. Myeloablative conditioning and reduced-intensity conditioning regimens were used in 8 (30.8%) and 18 (69.2%) transplantations, respectively. Neutrophil and platelet engraftment was achieved in 23 patients at a median follow-up of 21.2 months (range, 12 days to 234.8 months). Primary graft failure occurred in 1 of 23 patients (4.3%). Neutrophil and platelet engraftment occurred at a median of 16 (range, 12-39) days and 20 (range, 11-78) days, respectively. Acute graft-versus-host disease was seen in 11 of 22 patients engrafted allografts, of which 7 (31.8%) were grade 3-4 acute graft-versus-host disease. Eight patients (36.4%) developed chronic graft-versus-host disease, and three cases were extensive. Four patients (19%) relapsed after a median of 5.5 months, and three patients received donor lymphocyte infusion. The 3-year overall survival rate of the entire study population was 46.2%. The median overall survival was not reached in the myeloablative conditioning group; however, it was 11.9 months in the reduced-intensity conditioning group (p =0.3). According to the donor graft source, the median overall survival was 0.73 months in mismatched unrelated graft recipients, 12 months in matched sibling donors, and not reached in matched unrelated graft recipients (p = 0.03). The 3-year progression-free survival rate of patients who survived > 100 days was 74.7%. The effect of JAK-2 status, graft source, conditioning regimen or dynamic international prognostic scoring system on progression-free survival was not statistically significant.
Given the poor prognosis of non-transplant recipients and the lack of non-transplant curative approaches, our results support the consideration of allogeneic hematopoietic stem cell transplantation for eligible patients with primary myelofibrosis.
异基因造血干细胞移植是诊断为原发性骨髓纤维化患者的一种成熟治疗方法，仍然是唯一有潜在治愈作用的治疗方法。
介绍接受异基因造血干细胞移植治疗的骨髓纤维化患者的总体结果。
回顾性横断面研究。
本研究是对 2002 年 1 月至 2022 年 1 月在我们中心接受移植的 26 例原发性骨髓纤维化患者的回顾性分析。分析了影响结果的疾病和移植变量。
移植时的中位年龄为 52.5（范围，32-63）岁，从诊断到异基因造血干细胞移植的中位时间为 25（范围，3.1-156.8）个月。8（30.8%）例和 18（69.2%）例患者分别接受了清髓性和减低强度预处理方案。在中位随访 21.2 个月（范围，12 天至 234.8 个月）时，23 例患者获得中性粒细胞和血小板植入。1 例（4.3%）患者发生原发移植物失败。中性粒细胞和血小板植入的中位时间分别为 16（范围，12-39）天和 20（范围，11-78）天。22 例获得移植的患者中有 11 例（50%）发生急性移植物抗宿主病，其中 7 例（31.8%）为 3-4 级急性移植物抗宿主病。8 例（36.4%）患者发生慢性移植物抗宿主病，其中 3 例为广泛性。4 例（19%）患者在中位时间 5.5 个月后复发，3 例患者接受了供者淋巴细胞输注。整个研究人群的 3 年总生存率为 46.2%。清髓性预处理组的中位总生存率未达到；然而，在减低强度预处理组中位总生存率为 11.9 个月（p=0.3）。根据供体移植物来源，在非匹配无关供体受者中中位总生存率为 0.73 个月，在匹配同胞供者中为 12 个月，在匹配无关供体受者中未达到（p=0.03）。在存活>100 天的患者中，3 年无进展生存率为 74.7%。JAK-2 状态、移植物来源、预处理方案或动态国际预后评分系统对无进展生存率的影响无统计学意义。
鉴于非移植受者的预后较差，且缺乏非移植治疗方法，我们的结果支持考虑对原发性骨髓纤维化患者进行异基因造血干细胞移植。
