New and Old Treatment Modalities in Primary Myelofibrosis
- 1 November 2007
- journal article
- Published by Ovid Technologies (Wolters Kluwer Health) in The Cancer Journal
- Vol. 13 (6), 377-383
- https://doi.org/10.1097/ppo.0b013e31815a7c0a
Abstract
The treatment of primary myelofibrosis (PMF) remains essentially palliative. Conventional modalities include a wait-and-see approach for asymptomatic patients, oral cytolytic drugs such as hydroxyurea for the hyperproliferative forms of the disease, androgens or erythropoietin for the anemia, and splenectomy in selected patients. These therapeutic modalities improve the patients' quality of life but have no impact on survival. Newer therapies for PMF are currently being used. Antiangiogenic and immunomodulatory drugs such as thalidomide and lenalidomide are associated with frequent side effects but have shown certain efficacy, especially for the anemia and thrombocytopenia. The association of low-dose thalidomide with prednisone has better tolerability, and it is also effective. Tyrosine kinase inhibitors such as imatinib have also been used, but their efficacy is limited. Tipifarnib, a farnesyltransferase inhibitor, has shown certain effects in the anemia. Allogeneic stem cell transplantation (SCT) is the only curative therapy for PMF. Its standard modality has an associated mortality of 30%, and it is indicated in younger patients with high-risk disease or disease resistant to conventional treatment. Reduced-intensity conditioning allogeneic SCT is associated with low mortality while maintaining a curative potential, and until longer follow-up is available, it can be used in patients aged 45–70 years old with high- or intermediate-risk myelofibrosis or myelofibrosis resistant to treatment. Autologous SCT is a palliative measure that can be considered in patients with resistant disease, who lack a suitable donor. Newer immunomodulatory drugs, proteasome inhibitors, hypomethylating agents, and JAK2 inhibitors are currently being tested.Keywords
This publication has 56 references indexed in Scilit:
- Darbepoetin‐alpha for the anaemia of myelofibrosis with myeloid metaplasiaBritish Journal of Haematology, 2006
- Efficacy and tolerability of danazol as a treatment for the anaemia of myelofibrosis with myeloid metaplasia: long‐term results in 30 patientsBritish Journal of Haematology, 2005
- Modern management of myelofibrosisBritish Journal of Haematology, 2005
- Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literatureBritish Journal of Haematology, 2004
- Phase II study of α2 interferon in the treatment of the chronic myeloproliferative disorders (E5487)Cancer, 2003
- Melphalan treatment in patients with myelofibrosis with myeloid metaplasiaBritish Journal of Haematology, 2002
- Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patientsBlood, 2000
- Myelofibrosis With Myeloid Metaplasia: Diagnostic Definition and Prognostic Classification for Clinical Studies and Treatment GuidelinesJournal of Clinical Oncology, 1999
- Treatment of Anemia in Myeloproliferative DisordersArchives of Internal Medicine, 1982
- Agnogenic myeloid metaplasia: a clonal proliferation of hematopoietic stem cells with secondary myelofibrosisBlood, 1978