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Myelofibrosis: an update on drug therapy in 2016
Prithviraj Bose1, Srdan Verstovsek1
1a Department of Leukemia , University of Texas MD Anderson Cancer Center , Houston , TX , USA.
Introduction:
Primary myelofibrosis (PMF) is the least common but the most aggressive of the classic Philadelphia chromosome-negative myeloproliferative neoplasms. Survival is much shorter in PMF than in polycythemia vera (PV) or essential thrombocythemia (ET). Post-PV/ET myelofibrosis (MF) is clinically indistinguishable from PMF and approached similarly. Areas covered: Current pharmacologic therapy of MF revolves around the Janus kinase 1/2 (JAK1/2) inhibitor ruxolitinib, which dramatically improves constitutional symptoms and splenomegaly in the majority of patients, and improves overall survival (OS). However, allogeneic stem cell transplantation remains the only potential cure. Other JAK inhibitors continue to be developed for MF, and momelotinib and pacritinib are in phase III clinical trials. Anemia is common in MF, and initially worsened by ruxolitinib. Momelotinib and pacritinib may prove advantageous in this regard. Current strategies for managing anemia of MF include danazol, immunomodulatory drugs and erythroid stimulating agents, either alone or in combination with ruxolitinib. Expert opinion: A number of other agents, representing diverse drug classes, are in various stages of development for MF. These include newer JAK inhibitors, other signaling inhibitors, epigenetic modifiers, anti-fibrotic agents, telomerase inhibitors, and activin receptor ligand traps (for anemia). Hopefully, these novel therapies will further extend the clinical benefits of ruxolitinib.
Insights
Ruxolitinib improves symptoms and survival in myelofibrosis (MF), but stem cell transplant is the only cure. New JAK inhibitors and other therapies are in development for MF, potentially improving anemia management.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Primary myelofibrosis (PMF) is an aggressive Philadelphia chromosome-negative myeloproliferative neoplasm with shorter survival than polycythemia vera (PV) or essential thrombocythemia (ET).
- Post-polycythemia vera/essential thrombocythemia myelofibrosis (MF) is clinically similar to PMF.
- Ruxolitinib, a Janus kinase 1/2 (JAK1/2) inhibitor, is the current standard pharmacologic therapy for MF, improving symptoms and survival.
Purpose of the Study:
- To review current pharmacologic therapies for myelofibrosis (MF).
- To discuss emerging therapies and management strategies for MF, including anemia.
- To provide an expert opinion on the future of MF treatment.
Main Methods:
- Review of current pharmacologic therapy for MF, focusing on JAK inhibitors.
- Discussion of clinical trials for new agents like momelotinib and pacritinib.
- Analysis of strategies for managing anemia in MF patients.
Main Results:
- Ruxolitinib significantly improves constitutional symptoms and splenomegaly in most MF patients and enhances overall survival.
- Allogeneic stem cell transplantation remains the only curative option for MF.
- Newer JAK inhibitors (momelotinib, pacritinib) and diverse drug classes are in development, offering potential benefits for symptom control and anemia.
Conclusions:
- Ruxolitinib has transformed MF management, but novel therapies are crucial for further progress.
- Emerging agents targeting JAK pathways, fibrosis, and anemia hold promise for improving patient outcomes.
- Future MF treatment will likely involve a combination of established and novel therapies to address diverse patient needs.
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