BCR-ABL negative myeloproliferative neoplasms (MPN) in transformation have a dismal prognosis, and allogeneic transplantation is thought to be the only curative therapeutic option. We retrospectively analyzed 53 molecularly annotated patients treated at Saint Louis Hospital (France), diagnosed between 2008 and 2018 with MPN in transformation. Median age was 65 years and median interval between MPN diagnosis and MPN transformation was 46 months. Median overall survival (OS) of the entire cohort after transformation was 7.1 months. Overall survival (OS) was better for patients treated by hypomethylating agents or by chemotherapy than those treated by best supportive care or single agent targeted therapy: 9.1 vs 1.5 months, p < 0.001. Patients treated by chemotherapy achieved more often complete remission than those treated by hypomethylating agents (68% vs 29%, p = 0.02), and could be transplanted more frequently (59% vs 14%, p = 0.02) but their median OS was similar. We then compared the outcome of transplanted vs. non-transplanted patients using Mantel-Byar's methodology and demonstrated that allogeneic transplantation did not improve survival. In multivariate analysis, independent prognosis factor of survival were Performance status at transformation (p < 0.01), initial treatment by hypomethylating agents or by chemotherapy (p = 0.02), and the ability to achieve complete remission during the follow-up (p < 0.01). In conclusion, indication of transplantation for high-risk MPN should be discussed before transformation since transplantation rescued few patients after transformation.

中文翻译：

---

Ph1阴性骨髓增生性肿瘤是否仍应考虑移植？

转化中的BCR-ABL阴性骨髓增生性肿瘤（MPN）的预后不佳，同种异体移植被认为是唯一的治疗选择。我们回顾性分析了在法国圣路易斯医院接受治疗的53名带有分子注释的患者，这些患者在2008年至2018年之间被诊断为MPN转化。中位年龄为65岁，MPN诊断与MPN转化之间的中位间隔为46个月。转化后整个队列的中位总体生存期（OS）为7.1个月。用次甲基化药物或化学疗法治疗的患者的总生存期（OS）优于采用最佳支持治疗或单一药物靶向治疗的患者：9.1 vs 1.5个月，p <0.001。接受化疗的患者比接受低甲基化药物治疗的患者更容易获得完全缓解（68％vs 29％，p = 0.02），并且可以更频繁地移植（59％vs 14％，p = 0.02），但他们的中位OS相似。然后，我们使用Mantel-Byar方法比较了移植患者和未移植患者的结果，并证明了同种异体移植不能提高生存率。在多变量分析中，生存的独立预后因素是转化时的表现状态（p <0.01），次甲基化剂或化学疗法的初始治疗（p = 0.02），以及随访期间完全缓解的能力（p <0.01）。 ）。结论，