Cattaneo Daniele, Vener Claudia, Elli Elena Maria, Bucelli Cristina, Galli Nicole, Cavalca Fabrizio, Auteri Giuseppe, Vincelli Donatella, Martino Bruno, Gianelli Umberto, Palandri Francesca, Iurlo Alessandra
Hematology Division, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Department of Oncology and Hemato-Oncology, University of Milan, 20122 Milan, Italy.
Cancers (Basel). 2022 Apr 1;14(7):1799. doi: 10.3390/cancers14071799.
The 2016 WHO classification recognized pre-fibrotic primary myelofibrosis (pre-PMF) as a distinct entity. Nevertheless, a prognostic model specific for pre-PMF is still lacking. Our aim was to identify the most relevant clinical, histological, and driver mutation information at diagnosis to evaluate outcomes in pre-PMF patients in the real-world setting. We firstly assessed the association between IPSS or DIPSS at diagnosis and response variables in 378 pre-PMF patients. A strict association was observed between IPSS and DIPSS and occurrence of death. Other analyzed endpoints were not associated with IPSS or DIPSS as thrombo-hemorrhagic events at diagnosis or during follow-up, or did not show a clinical plausibility, as transformation into acute leukemia or overt PMF. The only covariates which were significantly associated with death were diabetes and second neoplasia, and were therefore included in two different prognostic settings: the first based on IPSS at diagnosis [class 1 vs. 0, OR (95%CIs): 3.34 (1.85-6.04); class 2 vs. 0, OR (95%CIs): 12.55 (5.04-31.24)], diabetes [OR (95%CIs): 2.95 (1.41-6.18)], and second neoplasia [OR (95%CIs): 2.88 (1.63-5.07)]; the second with DIPSS at diagnosis [class 1 vs. 0, OR (95%CIs): 3.40 (1.89-6.10); class 2 vs. 0, OR (95%CIs): 25.65 (7.62-86.42)], diabetes [OR (95%CIs): 2.89 (1.37-6.09)], and second neoplasia [OR (95%CIs): 2.97 (1.69-5.24)]. In conclusion, our study underlines the importance of other additional risk factors, such as diabetes and second neoplasia, to be evaluated, together with IPSS and DIPSS, to better define prognosis in pre-PMF patients.
2016年世界卫生组织（WHO）分类将纤维化前原发性骨髓纤维化（pre-PMF）视为一种独特的疾病实体。然而，目前仍缺乏针对pre-PMF的特异性预后模型。我们的目的是确定诊断时最相关的临床、组织学和驱动基因突变信息，以评估真实世界中pre-PMF患者的预后。我们首先评估了378例pre-PMF患者诊断时的国际预后评分系统（IPSS）或动态国际预后评分系统（DIPSS）与反应变量之间的关联。观察到IPSS和DIPSS与死亡发生之间存在密切关联。其他分析的终点与IPSS或DIPSS无关联，如诊断时或随访期间的血栓出血事件，或者不具有临床合理性，如转化为急性白血病或明显的原发性骨髓纤维化（PMF）。与死亡显著相关的唯一协变量是糖尿病和第二肿瘤，因此将其纳入两种不同的预后模型：第一种基于诊断时的IPSS[1级与0级，比值比（95%置信区间）：3.34（1.85 - 6.04）；2级与0级，比值比（95%置信区间）：12.55（5.04 - 31.24）]、糖尿病[比值比（95%置信区间）：2.95（1.41 - 6.18）]和第二肿瘤[比值比（95%置信区间）：2.88（1.63 - 5.07）]；第二种基于诊断时的DIPSS[1级与0级，比值比（95%置信区间）：3.40（1.89 - 6.10）；2级与0级，比值比（95%置信区间）：25.65（7.62 - 86.42）]、糖尿病[比值比（95%置信区间）：2.89（1.37 - 6.09）]和第二肿瘤[比值比（95%置信区间）：2.97（1.69 - 5.24）]。总之，我们的研究强调了除IPSS和DIPSS外，评估其他额外风险因素（如糖尿病和第二肿瘤）对于更好地确定pre-PMF患者预后的重要性。
