Myelodysplastic syndromes (MDS) - pediatric

Approximately 21,000 people are diagnosed with myelodysplastic syndromes (MDS) in the United States each year, and the incidence increases with age. [1] More MDS patients are now eligible for allogeneic hematopoietic cell transplantation (HCT) than ever before. The development of reduced-intensity conditioning regimens makes HCT—which is the only potentially curative treatment option—more feasible for older patients. [2]

The Centers for Medicare and Medicaid Services (CMS) cover transplants for MDS when performed as part of a Medicare approved clinical study, which allows physicians to offer transplant as an option for older patients. Physicians can enroll Medicare-eligible patients with MDS in a CMS-approved study conducted by the CIBMTR® (Center for International Blood and Marrow Transplant Research®).

Preliminary results from the CIBMTR clinical trial showed that in patients who are eligible for allogeneic HCT, there was no difference in 100-day mortality or overall survival at 2 years for patients age 55-64 years compared to patients age 65 years and older. [3]

Research from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) published in the Journal of Clinical Oncology demonstrates that HCT provides a significant survival benefit for older patients with MDS. BMT CTN 1102 was a multi-center biologic assignment trial comparing reduced-intensity allogeneic HCT to hypomethylating therapy or best supportive care in patients aged 50-75 with advanced MDS. [4]

Myelodysplastic Syndromes – Adult

High-resolution HLA typing is recommended at diagnosis for all patients

• Any intermediate or high IPSS or IPSS-R score
• Any MDS with poor prognostic features, including:
  • Treatment-related MDS
  • Refractory cytopenias
  • Adverse cytogenetics and molecular features
  • Transfusion dependence
  • Failure of hypomethylating agents or chemotherapy
  • Moderate to severe marrow fibrosis

  Myelodysplastic Syndromes – Pediatric

  At diagnosis for all subtypes

  Juvenile Myelomonocytic Leukemia (JMML)

  At diagnosis

1. SEER Cancer Statistics Review 1975-2012: MDS. Website accessed 8 March, 2018. Access
2. Karanes C, Nelson GO, Chitphakdithai P, et al. Twenty years of unrelated donor hematopoietic cell transplantation for adult recipients facilitated by the National Marrow Donor Program. Biol Blood Marrow Transplant. 2008; 14(9, Suppl.): 8-15. Access
3. Atallah E, Horowitz MM, Logan B, et al. Outcome of patients 65 years and older with myelodysplastic syndrome (MDS) receiving allogeneic hematopoietic stem cell transplantation compared to patients 55-64 years of age (abstract). Blood. 2015; 126(23): 193. Access
4. Nakamura R, Saber W, Martens MJ, et al. Biologic assignment trial of reduced-intensity hematopoietic cell transplantation based on donor availability in patients 50-75 years of age with advanced myelodysplastic syndrome. Journal of Clinical Oncology. 2021;39(30):3328-3339. Access
5. NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF
6. National Comprehensive Cancer Network. Myelodysplastic Syndromes. (Version 1.2023). Access