In the 5-year follow-up of patients diagnosed with Juvenile Myelomonocytic Leukemia (JMML) in a single center; reveal treatment and survival analyzes

In this study, clinical and laboratory data of 12 JMML patients followed in Ankara Pediatric Hematology Hospital and Ankara City Hospital Pediatric Hematology Clinics between 2015-2020 were analyzed retrospectively.

The median age at diagnosis was 1.7 years (0.23-5.7). Monosomy 7, 4 PTPN11, 2 NRAS, 4 KRAS, 1 CBL mutations were detected in 2 of the patients. Hematopoietic stem cell transplantation was performed in 8 of the patients. Before transplantation, 7 patients had received a median of 4 cycles of azacitidine treatment. The mean time from diagnosis to transplantation was 15 months (1-29 months). The 5-year overall survival at median 15-month follow-up was 50%.

With hypomethylating agents and HSCT, survival in JMML improves compared to historical control groups. However, further multicenter prospective studies are needed to prevent long-term mortality and morbidity.