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Review
. 2024 Oct 30;16(4):698-713.
doi: 10.3390/hematolrep16040067.

Treatment Strategies Used in Treating Myelofibrosis: State of the Art

Massimo Martino  1   2 , Martina Pitea  1   2 , Annalisa Sgarlata  1   2 , Ilaria Maria Delfino  1   2 , Francesca Cogliandro  1   2 , Anna Scopelliti  1   2 , Violetta Marafioti  1   2 , Simona Polimeni  1   2 , Gaetana Porto  1   2 , Giorgia Policastro  1   2 , Giovanna Utano  1   2 , Maria Pellicano  1   2 , Giovanni Leanza  3 , Caterina Alati  1
Affiliations
Review

Treatment Strategies Used in Treating Myelofibrosis: State of the Art

Massimo Martino et al. Hematol Rep. .

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.

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Conflict of interest statement

The authors declare no conflicts of interest.

Figures

Figure 1
Figure 1
Defining ruxolitinib failure in clinical practice.
Figure 2
Figure 2
Management of patient candidates for allogeneic stem cells transplantation.
Figure 3
Figure 3
Myelofibrosis: how to choose second-line therapy.
See this image and copyright information in PMC

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