Wajid M Abdul, Gupta Aditya Kumar, Das Gargi, Sahoo Debasish, Meena Jagdish Prasad, Seth Rachna
Division of Pediatric Oncology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi.
Pediatr Hematol Oncol. 2020 Oct;37(7):573-581. doi: 10.1080/08880018.2020.1767244. Epub 2020 May 27.
Juvenile myelomonocytic leukemia(JMML) is a pediatric myeloproliferative disorder. Allogeneic hematopoietic stem cell transplant (HSCT) is the only curative treatment for JMML. Pre-transplant therapy is a matter of controversy, and there are no firm recommendations. Whether chemotherapy is effective in achieving durable remission is questionable. Patients diagnosed as JMML at our center from January-2014 to December-2019 were retrospectively analyzed. All patients treated with at least one cycle of sequential therapy with subcutaneous cytarabine and oral 6-mercaptopurine were further assessed. The total number of patients diagnosed during the study period was 33. Patients were divided into two groups: patients who did not get any chemotherapy ( = 13) and ones who received at least one cycle of chemotherapy(n = 20). Age, total leukocyte count (TLC), monocyte percent, platelet count and spleen size were comparable between the two groups. There was no difference in the overall survival between the two groups, but 6 out of 20 patients showed a response to chemotherapy (2 complete remission, 4 partial remission). Two patients out of 20 underwent hematopoietic stem cell transplant (HSCT). The patients who achieved complete remission received 12 cycles of chemotherapy and have been in follow up for 28 months and 50 months respectively. Our results showed that sequential therapy with 6-mercaptopurine and cytarabine may be offered to patients in whom HSCT is not feasible or as a bridge therapy in those awaiting HSCT. The advantages of this approach include low cost, out-patient management and decreased requirement of blood components. In a subset of patients it may achieve remission.
青少年骨髓单核细胞白血病（JMML）是一种儿童骨髓增殖性疾病。异基因造血干细胞移植（HSCT）是JMML唯一的治愈性治疗方法。移植前治疗存在争议，且没有明确的推荐方案。化疗是否能有效实现持久缓解仍存在疑问。对2014年1月至2019年12月在我们中心诊断为JMML的患者进行了回顾性分析。对所有接受至少一个周期皮下阿糖胞苷和口服6-巯基嘌呤序贯治疗的患者进行了进一步评估。研究期间诊断的患者总数为33例。患者分为两组：未接受任何化疗的患者（n = 13）和接受至少一个周期化疗的患者（n = 20）。两组患者的年龄、总白细胞计数（TLC）、单核细胞百分比、血小板计数和脾脏大小具有可比性。两组患者的总生存率无差异，但20例患者中有6例对化疗有反应（2例完全缓解，4例部分缓解）。20例患者中有2例接受了造血干细胞移植（HSCT）。达到完全缓解的患者接受了12个周期的化疗，并分别随访了28个月和50个月。我们的结果表明，对于无法进行HSCT的患者或等待HSCT的患者作为桥接治疗，可以采用6-巯基嘌呤和阿糖胞苷序贯治疗。这种方法的优点包括成本低门诊管理以及对血液成分的需求减少。在一部分患者中，它可能实现缓解。
