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Article Abstract
Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric myeloproliferative disease, and newly diagnosed patients frequently cannot tolerate hematopoietic stem cell transplantation (HSCT) at diagnosis due to their poor condition. This retrospective analysis aimed to explore the short-term effect of decitabine-dominant therapy on improving the condition of JMML patients before HSCT. The subjects were 10 JMML patients. All patients were treated with decitabine after low-dose chemotherapy with an interval of 4 weeks before bridging to HSCT. The median treatment course was 3 cycles, and the overall response rate (ORR) was 70.0% after one cycle and 71.4% after three cycles. White blood cell (WBC) and monocyte counts were significantly lower after treatment, and spleen volume was also lower, though not significantly lower. The 12 month progression-free survival rate (PFS) was 80.0 ± 12.6%. Decitabine-dominant therapy was beneficial for reducing tumor burden and improving clinical condition.
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| http://dx.doi.org/10.1007/s12185-022-03457-y | DOI Listing |
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Short-term efficacy of decitabine-based therapy in JMML: a retrospective study from a single center in China.
Int J Hematol
January 2023
Division of Pediatric Blood Diseases Center, State Key Laboratory of Experimental Hematology, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, National Clinical Research Center for Blood Diseases, Chinese Academy of Medical Sciences and Peking Union Medical Co
Article Synopsis
- - Juvenile myelomonocytic leukemia (JMML) is a serious type of blood cancer in children, and patients often can't undergo critical treatment like stem cell transplants due to their fragile health at diagnosis.
- - This study examined the effects of decitabine-based therapy on 10 JMML patients, finding that after three treatment cycles, 71.4% showed improvement, alongside significant reductions in white blood cell and monocyte counts.
- - After one year, the study reported an 80% progression-free survival rate, suggesting decitabine therapy effectively helps reduce cancer burden and enhance patient condition prior to transplant.