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Published on: February 17, 2022
Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report
Zhimin Fan1, Nengwen Xu1, Alicia Senin2
1Department of Hematology, Lishui Central Hospital, Lishui, Zhejiang 323400, P.R. China.
Insights
Pediatric myelodysplastic syndrome (MDS), often refractory cytopenia of childhood (RCC), has limited treatment options. Decitabine monotherapy stabilized disease in a child ineligible for stem cell transplant or immunosuppressive therapy, suggesting a new treatment possibility.
Area of Science:
- Pediatric Hematology
- Oncology
- Stem Cell Transplantation
Background:
- Myelodysplastic syndrome (MDS) in children is rare, typically presenting as refractory cytopenia of childhood (RCC).
- Current management for pediatric MDS includes chemotherapy, immunosuppressive therapy (IST), and allogeneic hematopoietic stem cell transplantation (HSCT).
- Limited therapeutic data exists for RCC patients unsuitable for HSCT or IST.
Abstract:
The occurrence of myelodysplastic syndrome (MDS) in children is infrequently documented and most commonly manifests in the form of refractory cytopenia of childhood (RCC). The management of pediatric MDS remains controversial and involves primarily chemotherapy, immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT). However, therapeutic data for patients with RCC who are ineligible for either HSCT or IST are scarce. In the present case, the 8-year-old patient achieved disease stabilization following decitabine monotherapy, thereby suggesting a potential novel therapeutic avenue for children with low-risk chronic MDS or RCC.
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