Supplementary Material for: Clinical Benefit Derived from Decitabine Therapy for Advanced Phases of Myeloproliferative Neoplasms

Treatment options are limited for patients with advanced forms of myeloproliferative neoplasms (MPN) including blast-phase disease (MPN-BP). Decitabine has frequently been deployed but its efficacy and safety profile are not well described in this population. We retrospectively reviewed 42 patients treated with decitabine either alone or in combination with ruxolitinib at our institution: 16 with MPN-BP, 14 with MPN accelerated-phase (MPN-AP), and 12 with myelofibrosis with high-risk features (MF-HR). The median overall survival (OS) for the MPN-BP patients was 2.6 months, and for those who received ≥2 cycles of decitabine therapy, it was 6.7 months (3.8–29.8). MPN-BP patients with a poor performance status and who required hospitalization at the time of the initiation of decitabine had a dismal prognosis. After a median follow-up of 12.4 months for MPN-AP patients, and 38.7 months for MF-HR patients, the median OS was not reached for either cohort, with 1 and 2 patients alive at 60 months, respectively. The probability of spleen length reduction and transfusion independence within 12 months of initiating decitabine was 28.6 and 23.5%, respectively. The combination of decitabine and ruxolitinib appeared to improve overall survival versus single-agent decitabine (21 and 12.9 months, respectively). Decitabine, alone or in combination with ruxolitinib, appears to have clinical benefit for patients with advanced phases of MPN when initiated early in the disease course prior to the development of MPN-BP.

对于包括急变期骨髓增殖性肿瘤（MPN-BP）在内的晚期骨髓增殖性肿瘤（myeloproliferative neoplasms, MPN）患者，临床治疗选择极为有限。地西他滨（Decitabine）虽常被用于此类患者的治疗，但其在该人群中的疗效与安全性特征尚未得到充分阐明。本研究回顾性分析了本机构42例接受单药或联合芦可替尼（ruxolitinib）治疗的MPN患者，其中16例为MPN急变期（MPN-BP）患者、14例为MPN加速期（MPN-AP）患者、12例为高危骨髓纤维化（myelofibrosis with high-risk features, MF-HR）患者。MPN-BP患者的中位总生存期（median overall survival, OS）为2.6个月；其中接受≥2个周期地西他滨治疗的患者，中位总生存期可达6.7个月（3.8~29.8）。体能状态较差且在启动地西他滨治疗时需住院治疗的MPN-BP患者，预后极差。在对MPN-AP患者进行中位12.4个月随访、对MF-HR患者进行中位38.7个月随访后，两组均未达到中位总生存期，分别有1例和2例患者在60个月时仍存活。在启动地西他滨治疗后的12个月内，患者脾脏长度缩小率与输血不依赖率分别为28.6%与23.5%。与单药地西他滨方案相比，地西他滨联合芦可替尼方案似乎可改善患者总生存期，两组中位OS分别为21个月与12.9个月。无论单药使用还是联合芦可替尼，地西他滨在MPN晚期阶段均展现出临床获益，前提是在疾病进展为MPN-BP之前的病程早期启动治疗。