Risk Factors and New Methods of Treatment of Myelofibrosis: Transplantation in the Era of JAK Inhibitors

The goal of this educational program is to equip transplant specialists, oncologists, hematologists, and other healthcare professionals involved in the treatment of hematologic malignancies with the up-to-date clinical knowledge and tools they need to best treat their patients with Myelofibrosis.

Myelofibrosis(MF) is a rare myeloproliferative neoplasm (MPN), which are diseases of the blood and bone marrow where an excess number of blood stem cells become platelets, red blood cells and white blood cells. An important feature of MF is the production of too many megakaryocytes, giant cells in the marrow that break up into fragments and produce hundreds to thousands of platelets. This leads to the release of cytokines in the marrow. The cytokines stimulate the development of scar tissue in the marrow, called fibrosis. The platelets’ normal function is to stick to the site of a blood vessel injury and form a clot to seal off the injured blood vessel to stop bleeding. The body makes new platelets to replace used platelets. The megakaryocytes can become so abnormal that platelet production decreases in some patients.

MF occurs mostly in the elderly population, specifically between 60-70 years of age. The prevalence of MF is estimated at between 0.1 to 1 in 100,000 people per year, according to a European study by Moulard et al. Additionally, in 10-15% of MF cases, the disease arises from other myeloproliferative diseases like polycythemia vera (PV) or primary/essential thrombocythemia (ET). When MF follows another myeloproliferative disease, it is secondary MF whereas MF starting on its own is called primary myelofibrosis (PMF).

For most people who have myelofibrosis (MF), there are no obvious risk factors why they developed the disease. The disease starts as one of two other myeloproliferative diseases, either polycythemia vera or primary thrombocythemia, in about 10 percent to 15 percent of people with MF. Physicians do not fully understand the cause of MF. MF results from a mutation in a stem cell in the bone marrow, which leads to uncontrolled blood cell production. Abnormal cell production gradually overtakes production of normal red cells, white cells and platelets. Too few red cells are made, and usually too many platelets and white cells are made. Eventually, there are more abnormal cells in the marrow than there are normal cells.

About 50% of MF patients have a mutation in the JAK2 kinase called V617F. The discovery of the JAK2 mutation in the pathogenesis of MF and other diseases led to the idea that the JAK pathways are good targets for drug therapy in MF. A new class of drugs has been created based on this discovery: oral JAK2 inhibitors. Despite the value shown by use of JAK2 inhibitors, currently, the only treatment for MF that has shown to have curative qualities is hematopoietic stem cell transplantation (HCT). When a patient is diagnosed with PV or ET, it can progress to myelofibrosis or acute myeloid leukemia. However, allogeneic stem cell transplantation (ASCT) can prevent this progression, which involves a transplant from a donor. In some cases, survival rates with ASCT are between 40-60%. ASCT is more effective in certain patients, and the new findings with JAK inhibitors could work well in combination with transplants in eligible patients. More research on this is likely to shed light on this new combination therapy.

Accreditation Statement
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing  Medical Education through the joint providership of The Medical College of Wisconsin and Carden Jennings Publishing. The Medical College of Wisconsin is accredited by the ACCME to provide continuing medical education for physicians.

AMA Credit Designation  Statement
The  Medical College of Wisconsin  designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the  credit  commensurate with  the  extent of their participation in the activity.

Hours  of  Participation for  Allied Health  Care Professionals: The Medical College of Wisconsin designates this activity for up to 1.0 hours of participation for continuing education for allied health professionals.

Commerical Support
This activity was supported by an unrestricted educational grant from Incyte Corporation.

Target Audience

This activity has been developed and is intended for transplant specialists, oncologists, hematologists, and other healthcare professionals involved in the treatment of Myelofibrosis.

Learning Objectives

Upon completion of the program, participants should be able to:
1.Identify the Risk Factors and Optimal Timing of HCT for Myelofibrosis
2.Evaluate the Benefits of Non-transplant Treatment Options
3.Assess the Increased Use of Stem Cell Transplantation in Combination with JAK inhibitor Treatment

Additional information


Linda Caples
Phone Number: 
+1 (414) 955-4900
Course summary
Available credit: 
  • 1.00 AMA PRA Category 1 Credit(s)™
    AMA PRA Category 1 Credit(s)™
  • 1.00 Hours of Participation
    Hours of Participation credit.
Course opens: 
Course expires: 

Vikas Gupta, MD, FRCP, FRCPath
Associate Professor, Department of Medicine
Princess Margaret Cancer Centre
University of Toronto
Toronto, Ontario, Canada

Parameswaran N. Hari, MD, MRCP, MS
Armand Quick – William Stapp Professor of Hematology
Section Head, Hematological Malignancies
Director, Adult Blood and Marrow Transplant Program
Medical College of Wisconsin
Milwaukee, WI

Laura C. Michaelis, MD
Associate Professor of Medicine
Division of Hematology/Oncology
Medical College of Wisconsin
Milwaukee, WI

Editors: John R. Wingard, MD
Price Eminent Scholar and Professor of Medicine Deputy Director for Research, UF Health Cancer Center Director, Bone Marrow Transplant Program

Randy A. Brown, MD
Professor of Medicine Clinical Director, Blood and Marrow Transplant Program, University of Florida Health in the Division of Hematology and Oncology

Off-Label/Investigational  Use
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the Medical College of Wisconsin, Carden Jennings Publishing or Incyte. Before prescribing  any  medication,  physicians should consult primary references and full prescribing information. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings. Further, participants should appraise the information presented critically, and are encouraged to consult appropriate resources for any product or device mentioned in this activity.

CJP Medical  Communications  Disclosure
The employees of CJP Medical Communications have no financial relationships to disclose.

Faculty Disclosures
Consistent with the current Accreditation Council for Continuing Medical Education policy, the CME Provider must be able to show that everyone who is in a position to control the content of an individual educational activity has disclosed all relevant financial relationships. The CME Provider has a mechanism in place to identify and resolve any conflicts of interest discovered in the disclosure process. The presenting faculty members have all made the proper disclosures, and the following relationships are relevant:
John R. Wingard, MD, has no relevant financial relationships to disclose.
Randy A. Brown, MD, has no relevant financial relationships to disclose.
Vikas Gupta, MD, discloses that he has received research funding and honoraria from Novartis and a stipend from Novartis and Incyte.
Parameswaran N. Hari, MD has no relevant financial relationships to disclose.
Laura C. Michaelis, MD discloses that she has received a stipend from Incyte and owns Pfizer stock.

Available Credit

  • 1.00 AMA PRA Category 1 Credit(s)™
    AMA PRA Category 1 Credit(s)™
  • 1.00 Hours of Participation
    Hours of Participation credit.


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