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.

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