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Updated: Feb 5, 2026
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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
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Myelodysplastic syndromes in children
Federica Galaverna1, Annalisa Ruggeri1, Franco Locatelli1,2
1Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome.
Current Opinion in Oncology
|September 18, 2018
Summary
Childhood myelodysplastic syndromes (MDSs) are rare, complex disorders. Hematopoietic stem cell transplantation (HSCT) is the primary treatment, with novel approaches improving outcomes for pediatric MDS patients.
Area of Science:
- Pediatric Hematology
- Oncology
- Genetics
Background:
- Myelodysplastic syndromes (MDSs) are rare in children, presenting unique clinical and biological characteristics.
- Childhood MDS classification includes refractory cytopenia of childhood (RCC), advanced MDS, and therapy-related MDS.
- Germline mutations in genes like GATA2, ETV6, SRP72, and SAMD9/SAMD9-L are increasingly recognized as predisposing factors for pediatric MDS and acute myeloid leukemia.
Purpose of the Study:
- To review the biological, genetic, and clinical features of childhood MDS.
- To provide an updated treatment algorithm for various pediatric MDS subtypes.
Main Methods:
- Review of current literature on childhood MDS.
- Analysis of diagnostic and treatment strategies.
- Focus on classification, genetic associations, and hematopoietic stem cell transplantation (HSCT).
Main Results:
- Childhood MDS subtypes include RCC, advanced, and therapy-related MDS, often linked to inherited bone marrow failure syndromes.
- Allogeneic HSCT from an HLA-identical donor is the preferred treatment for most pediatric MDS cases.
- HSCT is indicated for MDS with excess blasts, therapy-related MDS, and specific RCC subtypes (monosomy 7, complex karyotype, severe neutropenia, transfusion dependence).
Conclusions:
- Treatment for pediatric MDS is subtype-specific.
- Allogeneic HSCT is the cornerstone of therapy, with HLA-haploidentical transplantation offering reduced complications.
- Understanding genetic predispositions and refining HSCT protocols are crucial for improving outcomes in childhood MDS.
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