Pediatric Myelofibrosis Medication

Updated: May 12, 2021
  • Author: Trisha Simone Natanya Tavares, MD; Chief Editor: Cameron K Tebbi, MD  more...
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Medication Summary

Treat any underlying disease (eg, rickets) as indicated for the specific disease. The medications listed here have shown some benefit in patients with idiopathic myelofibrosis (IMF). They include the following:

  • Calcitriol

  • Prednisone

  • Methylprednisolone

  • Interferon alfa 2a

  • IV immunoglobulin

  • Thalidomide

  • Lenalidomide

  • Decitabine


Vitamin D Analogs

Class Summary

Myelofibrosis has been described in patients with severe vitamin D deficiency. In addition, some (adult) patients with myelofibrosis associated with essential thrombocythemia or myelomonocytic leukemia, as well as acute (idiopathic) myelofibrosis (IMF), have responded to vitamin D administration. A direct inhibitory effect on platelets has been proposed. However, other studies have not confirmed such a response in patients with IMF.

Calcitriol (Rocaltrol, Calcijex, Vectical)

Calcitriol (ie, 1,25-dihydroxyvitamin D) is the primary active metabolite of vitamin D3. It increases calcium levels by promoting absorption of calcium in the intestines and retention in the kidneys. Doses for myelofibrosis are 5- to 10-fold higher than the physiologic dose.



Class Summary

These agents have immunosuppressive and cytotoxic effects. The mechanism of cytotoxicity is unknown (but apparently mediated through glucocorticoid receptors).


Prednisone is an immunosuppressant for the treatment of autoimmune disorders. It may decrease inflammation by reversing increased capillary permeability and suppressing polymorphonuclear (PMN) cell activity. Prednisone stabilizes lysosomal membranes and also suppresses lymphocyte and antibody production. Its efficacy in some cases of myelofibrosis may reflect an underlying autoimmune defect and/or suppression of a proliferating clone.

Methylprednisolone (Solu-Medrol, Medrol, Depo-Medrol, A-Methapred)

This agent decreases inflammation by suppressing migration of polymorphonuclear leukocytes and reversing increased capillary permeability. Support for this higher-dose therapy in the literature is limited.



Class Summary

Proposed mechanisms of action for these agents are suppression of autoimmunity, enhanced immunoregulation of an abnormal clone, or both.

Interferon alfa-2a (Pegasys)

Interferon alfa-2a is a protein product manufactured by recombinant DNA technology. It acts by modulation of the host's immune response. This treatment has shown long-term efficacy in 1 adolescent patient with an indolent form of myelofibrosis (essentially identical to adult agnogenic myeloid metaplasia with myelofibrosis [AMMM]). Transient responses have been observed in at least 2 adults with acute myelofibrosis.

IV immunoglobulin (Privigen, Hizentra, Octagam, Gammagard)

Response to intravenous immunoglobulin was reported in 1 case of pediatric myelofibrosis, which was associated with autoimmune phenomena.

Thalidomide (Thalomid)

Thalidomide is an immunomodulatory agent that may suppress excessive production of tumor necrosis factor-alpha (ie, TNF-α) and may down-regulate selected cell-surface adhesion molecules involved in leukocyte migration.

Because of concerns regarding teratogenicity, thalidomide can be prescribed only by physicians and dispensed only by pharmacists who are registered with the System for Thalidomide Education and Prescribing Safety (STEPS) program. Patients must participate in ongoing surveys to receive therapy, and only a 28-day supply can be prescribed at a time. Thalidomide is used to improve anemia and decrease blood/platelet transfusions associated with myelofibrosis.

Lenalidomide (Revlimid)

Lenalidomide is indicated for the transfusion-dependent myelodysplastic syndrome (MDS) subtype of the deletion 5q cytogenetic abnormality. It is structurally similar to thalidomide and elicits immunomodulatory and antiangiogenic properties. Lenalidomide inhibits proinflammatory cytokine secretion and increases anti-inflammatory cytokines from peripheral mononuclear blood cells.


Antineoplastic Agents

Class Summary

Antineoplastic agents inhibit cell growth and proliferation.

Decitabine (Dacogen)

Decitabine is a 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 the cellular control of differentiation and proliferation.

Decitabine is indicated for the treatment of MDSs, including previously treated and untreated, de novo, and secondary MDSs of all French-American-British (FAB) subtypes (ie, refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, chronic myelomonocytic leukemia) and International Prognostic Scoring System (IPSS) groups intermediate-1 risk, intermediate-2 risk, and high risk.