Myelodysplastic syndromes (MDS) - pediatric
Transplant advances and outcomes
Pediatric myelodysplastic syndromes (MDS) account for less than 5% of childhood hematological malignancies with an annual incidence of 2 to 4 cases per million in children.
Evaluation for an underlying germline predisposition is particularly important diagnosing and managing pediatric MDS. Allogeneic HCT remains the mainstay of treatment for most forms of pediatric MDS and the only curative option. [1]
Recommended timing for transplant consultation in pediatric MDS
- At diagnosis for all subtypes
Juvenile myelomonocytic leukemia (JMML)
Juvenile myelomonocytic leukemia (JMML) is the most common pediatric myelodysplastic/myeloproliferative disorder, although it is rare overall. Its overall incidence is about 1-2 cases per million people, with a median age of 1.8 years at presentation. Allogeneic HCT is the only known cure for JMML. [2,3]
Recommended timing for transplant consultation in JMML
- At diagnosis
Support for parents considering HCT for their child
NMDPSM works as an extension of hematology/oncology practices to address the needs of parents beyond their child's clinical care. The NMDP Patient Support Center team provides tailored information and assistance to parents from their child's diagnosis through recovery after HCT. There is no cost to parents or the hematology/oncology practice.
Parents can receive:
- Step-by-step guidance from an NMDP patient navigator who specializes in HCT
- Emotional and practical support
- Navigation in their language from bilingual NMDP navigators and social workers (English and Spanish) and interpreter services
- Financial assistance to help pay for out-of-pocket costs before or after their child's transplant
- Education in multiple languages and formats, including videos for children in English and Spanish
- Peer support from parents who have been through transplant with their child
References
- Schwartz JR, Ma J, Lamprecht T, et al. The genomic landscape of pediatric myelodysplastic syndromes. Nat Commun. 2017;8(1):1557. Access
- National Cancer Institute: Juvenile myelomonocytic leukemia (PDQ®)–health professional version. Accessed July 18, 2025. Access
- Gupta AK, Meena JP, Chopra A, et al. Juvenile myelomonocytic leukemia-A comprehensive review and recent advances in management. Am J Blood Res. 2021;11(1):1–21. Access
- NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF
- National Comprehensive Cancer Network. Myelodysplastic syndromes. (Version 1.2023). Access