Treatment Strategies Used in Treating Myelofibrosis: State of the Art
- PMID: 39584924
- PMCID: PMC11587016
- DOI: 10.3390/hematolrep16040067
Treatment Strategies Used in Treating Myelofibrosis: State of the Art
Abstract
Background: Current drug therapy for myelofibrosis does not alter the natural course of the disease or prolong survival, and allogeneic stem cell transplantation is the only curative treatment modality. For over a decade, the Janus kinase (JAK) inhibitor ruxolitinib has been the standard of care. More recently, newer-generation JAK inhibitors have joined the ranks of accepted treatment options.
Objectives: The primary goal of treatment is to reduce spleen size and minimize disease-related symptoms. Prognostic scoring systems are used to designate patients as being at lower or higher risk. For transplant-eligible patients, transplant is offered to those with a bridge of a JAK inhibitor; patients who are not eligible for transplant are usually offered long-term therapy with a JAK inhibitor. Limited disease-modifying activity, dose-limiting cytopenias, and other adverse effects have contributed to discontinuation of JAK inhibitor treatment.
Conclusions: Novel JAK inhibitors and combination approaches are currently being explored to overcome these shortcomings. Further research will be essential to establish optimal therapeutic approaches in first-line and subsequent treatments.
Keywords: JAK inhibitors; allogenic stem cell transplantation; fedratinib; momelotinib; myelofibrosis; pacritinib; ruxolitinib.
Conflict of interest statement
The authors declare no conflicts of interest.
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References
-
- Khoury J.D., Solary E., Abla O., Akkari Y., Alaggio R., Apperley J.F., Bejar R., Berti E., Busque L., Chan J.K.C., et al. The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms. Leukemia. 2022;36:1703–1719. doi: 10.1038/s41375-022-01613-1. - DOI - PMC - PubMed
-
- Guglielmelli P., Pacilli A., Rotunno G., Rumi E., Rosti V., Delaini F., Maffioli M., Fanelli T., Pancrazzi A., Pietra D., et al. Presentation and outcome of patients with 2016 WHO diagnosis of prefibrotic and overt primary myelofibrosis. Blood. 2017;129:3227–3236. doi: 10.1182/blood-2017-01-761999. - DOI - PubMed
-
- Rumi E., Boveri E., Bellini M., Pietra D., Ferretti V.V., Sant’Antonio E., Cavalloni C., Casetti I.C., Roncoroni E., Ciboddo M., et al. Clinical course and outcome of essential thrombocythemia and prefibrotic myelofi- brosis according to the revised WHO 2016 diagnostic criteria. Oncotarget. 2017;8:101735–101744. doi: 10.18632/oncotarget.21594. - DOI - PMC - PubMed
-
- Barbui T., Thiele J., Passamonti F., Rumi E., Boveri E., Ruggeri M., Rodeghiero F., d’Amore E.S., Randi M.L., Bertozzi I., et al. Survival and disease progression in essential thrombocythemia are significantly influenced by accurate morphologic diagnosis: An international study. J. Clin. Oncol. 2011;29:3179–3184. doi: 10.1200/JCO.2010.34.5298. - DOI - PubMed
-
- Titmarsh G.J., Duncombe A.S., McMullin M.F., O’Rorke M., Mesa R., De Vocht F., Horan S., Fritschi L., Clarke M., Anderson L.A. How common are myeloproliferative neoplasms? A systematic review and meta-analysis. Am. J. Hematol. 2014;89:581–587. doi: 10.1002/ajh.23690. Erratum in Am. J. Hematol. 2015, 90, 850. - DOI - PubMed
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