Gallo Daniela, Nicoli Paolo, Calabrese Chiara, Gaidano Valentina, Petiti Jessica, Rosso Valentina, Signorino Elisabetta, Carturan Sonia, Bot-Sartor Giada, Volpe Gisella, Frassoni Francesco, Saglio Giuseppe, Cilloni Daniela
Department of Clinical and Biological Sciences, University of Turin, Turin, Italy.
Department of Pediatric Hemato-Oncology and Stem Cell and Cellular Therapy Laboratory, Institute G. Gaslini, Largo G Gaslini, Genova.
Cancer Med. 2016 Jul;5(7):1650-3. doi: 10.1002/cam4.735. Epub 2016 May 11.
The Wilms tumor gene WT1 is a useful marker of clonal hematopoiesis and it has been shown to be a good marker of residual disease and it reflects the response to therapy. Although myelofibrosis is characterized by mutations of JAK2 and calreticulin (CALR), these mutations are not useful to monitor response to therapy. In this study we demonstrated that in patients affected by myelofibrosis WT1 correlates with the International Prognostic Scoring System (IPSS) score at diagnosis. Furthermore WT1 is a good marker of response to JAK2 inhibitors especially for patients without blasts and for patients who develop anemia or thrombocytopenia not for progression but as therapy related toxicity. Finally, WT1 transcript reduction can mirror a benefit of therapy on the disease burden. This study demonstrated that WT1 is a good marker for monitoring the response to therapy in patients affected by myelofibrosis.
威尔姆斯瘤基因WT1是克隆性造血的一个有用标志物，并且已被证明是残留疾病的良好标志物，它反映了对治疗的反应。尽管骨髓纤维化以JAK2和钙网蛋白（CALR）突变为特征，但这些突变对监测治疗反应并无用处。在本研究中，我们证明，在骨髓纤维化患者中，WT1与诊断时的国际预后评分系统（IPSS）评分相关。此外，WT1是对JAK2抑制剂反应的良好标志物，特别是对于无原始细胞的患者以及发生贫血或血小板减少症的患者而言，WT1反映的不是疾病进展，而是治疗相关毒性。最后，WT1转录本减少可反映治疗对疾病负担的有益作用。本研究表明，WT1是监测骨髓纤维化患者治疗反应的良好标志物。
