Ye Xingnong, Chen Dan, Zheng Yan, Zhu Xiaoqiong, Fu Junkai, Huang Jian
Department of Hematology, The Fourth Affiliated Hospital, College of Medicine, Zhejiang University, Zhejiang Province, People's Republic of China.
Onco Targets Ther. 2017 Nov 13;10:5425-5428. doi: 10.2147/OTT.S142561. eCollection 2017.
Primary myelofibrosis (PMF) is one of the Philadelphia negative myeloproliferative neoplasms (MPN). The main clinical features are obvious physical symptoms and symptomatic splenomegaly. It may be converse to leukemia and has a shortened life expectancy. Nowadays, the therapy for PMF is aimed at maintaining comfort and there is no curative treatment. PMF with myelodysplastic syndrome (MDS), called MDS/MPN-u, is rare and the treatment is complex. In this study, we want to discuss an effective treatment for MDS/MPN via a case report and literature review.
A female patient was diagnosed with MDS/MPN through bone marrow cytology, immunology, cell genetics, molecular biology, and pathology. She received thalidomide and prednisone as initial treatment. Ten months later, the first-line therapy had failed, she presented with clinically relevant pancytopenia and increased blasts in bone marrow. Because decitabine is one of the first-line treatments for MDS and the patient was frail, she received low-dose decitabine as second-line therapy. Decitabine was administered at 15 mg/m once a week for 3 weeks, in a 4 week cycle. If there was improvement the treatment interval was prolonged.
After one cycle, the blasts in bone marrow were decreased to 0.5%. After four cycles, she felt comfortable and hematological improvement was achieved. The treatment interval was prolonged. After eight cycles, the spleen reduced to 2 cm under the rib, and she achieved complete hematological remission. After ten cycles, the mutation of JAK2/V617F expression was decreased from 60.63% to 0.01%. During the therapy, the patient presented with grade III-IV hematological toxicity after the first two cycles, but there were no side effects after subsequent cycles.
Our research showed that low-dose decitabine may be an effective treatment for MDS/MPN, especially in improving physical symptoms and achieving hematological remission. Besides, it may be possible to reverse positive JAK2 mutation.
原发性骨髓纤维化（PMF）是费城染色体阴性的骨髓增殖性肿瘤（MPN）之一。主要临床特征为明显的身体症状和有症状的脾肿大。它可能会转化为白血病，预期寿命缩短。目前，PMF的治疗旨在维持舒适感，尚无治愈性治疗方法。伴有骨髓增生异常综合征（MDS）的PMF，称为MDS/MPN-u，较为罕见且治疗复杂。在本研究中，我们想通过病例报告和文献综述来探讨一种针对MDS/MPN的有效治疗方法。
一名女性患者通过骨髓细胞学、免疫学、细胞遗传学、分子生物学和病理学被诊断为MDS/MPN。她接受沙利度胺和泼尼松作为初始治疗。十个月后，一线治疗失败，她出现了临床上相关的全血细胞减少和骨髓中原始细胞增加。由于地西他滨是MDS的一线治疗药物之一且患者身体虚弱，她接受低剂量地西他滨作为二线治疗。地西他滨以15mg/m²每周一次，共3周，每4周为一个周期给药。如果有改善则延长治疗间隔。
一个周期后，骨髓中的原始细胞降至0.5%。四个周期后，她感觉舒适，血液学得到改善。治疗间隔延长。八个周期后，脾脏缩小至肋下2cm，她实现了完全血液学缓解。十个周期后，JAK2/V617F表达突变从60.63%降至0.01%。在治疗期间，患者在前两个周期后出现III-IV级血液学毒性，但后续周期没有副作用。
我们的研究表明，低剂量地西他滨可能是治疗MDS/MPN的有效方法，尤其是在改善身体症状和实现血液学缓解方面。此外，它可能有可能逆转JAK2阳性突变。
