Pediatric Myelodysplasia Medication

Updated: May 17, 2023
  • Author: Natalia Dixon, MD; Chief Editor: Jennifer Reikes Willert, MD  more...
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Medication Summary

Several aspects of the management of pediatric myelodysplasia syndrome (MDS) differ from adult myelodysplasia therapy. Most importantly, the treatment goal must be curative because prolonging life by 5-8 years is not considered successful therapy in a healthy 10-year-old patient as it might in a 90-year-old patient who has other health problems. Therefore, allogeneic HSCT should almost always be the goal for children with myelodysplasia syndrome. However, the timing of HSCT is variable in children depending on symptomatology, disease characteristics, donor availability, and weighing the risks and benefits of transplant with the child’s quality of life.

The relevance of immunomodulating agents in pediatrics is investigational and remains unclear.

Various agents have been used to slow the progression of myelodysplasia syndrome, including low-dose cytosine arabinoside, cladribine (2-CdA), growth factors (erythropoietin, granulocyte colony-stimulating factor [G-CSF]), amifostine, and hydroxyurea. These agents are temporizing at best, and their role has been limited to palliation of myelodysplasia syndrome while a donor search takes place. In adults, decitabine, azacitidine, and lenalidomide (used primarily for 5q-syndrome) can reduce the need for transfusion and, in some cases, delay progression to AML when used in the correct subsets of patients.

Patients who present with a high percentage of marrow blasts and rapidly progressive disease may require chemotherapy while preparing for transplant. Both de novo and therapy-related myelodysplasia syndrome are usually only transiently responsive to conventional chemotherapy, which can be used in an attempt to keep the patient in remission until allogeneic HSCT can be performed. Transplantation from an HLA-matched family donor is optimal, but alternative donors should be considered when an HLA-matched family donor is not available. [40, 41]

Therapy prior to transplant also involves the judicious use of blood products and aggressive infection control because patients are often agranulocytic.


DNA hypomethylating agents

Class Summary

These agents are indicated for myelodysplastic syndrome in adults.

Azacitidine (Vidaza)

Pyrimidine nucleoside analog of cytidine. Interferes with nucleic acid metabolism. Exerts antineoplastic effects by DNA hypomethylation and direct cytotoxicity on abnormal hematopoietic bone marrow cells. Hypomethylation may restore normal function to genes critical for cell differentiation and proliferation. Nonproliferative cells are largely insensitive to azacitidine. Indicated to treat MDS. FDA approved for all 5 MDS subtypes.

Decitabine (Dacogen)

Hypomethylating agent believed to exert antineoplastic effects by incorporating into DNA and inhibiting methyltransferase, resulting in hypomethylation. Hypomethylation in neoplastic cells may restore normal function to genes critical for cellular control of differentiation and proliferation. Indicated for treatment of MDS, including previously treated and untreated, de novo, and secondary MDS of all FAB subtypes (ie, RA, RARS, RAEB, RAEBT, CML) and IPSS groups intermediate-1 risk, intermediate-2 risk, and high risk.


Immunomodulating agents

Class Summary

These agents are indicated for myelodysplasia syndrome in adults.

Lenalidomide (Revlimid)

Indicated for transfusion-dependent MDS subtype of deletion 5q31 cytogenetic abnormality. Structurally similar to thalidomide. Elicits immunomodulatory and antiangiogenic properties. Inhibits proinflammatory cytokine secretion and increases anti-inflammatory cytokines from peripheral blood mononuclear cells. The drug is FDA approved for adult patients with low or intermediate-1 IPSS category who are unlikely to respond to erythropoietin