Download Myelofibrosis Fact Sheet

Myelofibrosis Fact Sheet | 1
Myelofibrosis Fact Sheet
What is myelofibrosis (MF)?
MF is a rare, life-threatening blood cancer where bone marrow, the tissue within bones that produces
red blood cells, white blood cells and platelets, is replaced by scar tissue1,2. This results in abnormal,
decreased blood cell production1,2.
MF is classified under a group of related blood cancers known as myeloproliferative neoplasms (MPNs)3.
Of the three main types of MPNs, MF has the worst prognosis3. The underlying cause of MF is not fully
known, however, more than 80% of patients have mutations that disrupt the JAK/STAT signaling
pathway, which may explain the development of the disease4.
Enlarged Spleen
In MF, bone marrow does not function properly, causing other organs
to take over the production of blood cells, including the spleen, which
often becomes enlarged5.
What are the symptoms and complications of MF?
The signs, symptoms and complications associated
with MF result from an inability of bone marrow to
produce blood cells effectively. MF can take a heavy
toll on patients, with debilitating symptoms that can
significantly impact quality of life6.
One hallmark complication associated with the
disease occurs when the spleen compensates
for the decreased blood cell production and becomes
enlarged5.
Symptoms and complications of MF may
include2:
 Fatigue/Weakness
 Bone/Joint Pain
 Shortness of Breath
 Abdominal Pain
 Fever/Night Sweats
 Weight Loss
 Itchy Skin
How is MF diagnosed?
MF can be diagnosed at any age, but is more commonly seen in adults from 50 to 80 years old7. The
disease can be challenging to diagnose since symptoms often don’t appear until later stages8.
A physician may consider a diagnosis of MF when a routine medical examination shows an enlarged
spleen and abnormal blood counts8. A combination of laboratory tests including a complete blood count
(CBC) and bone marrow aspiration and biopsy can help verify the absence or presence
of abnormal blood cells and/or bone marrow fibrosis, key characteristics of MF, to help confirm
the disease9.
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February 2016
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Myelofibrosis Fact Sheet | 2
What treatments are available for MF?
For most people, MF treatment does not cure the disease.
Rather, the goal of treatment is to prolong survival, reduce
spleen size, relieve symptoms, improve quality of life and
reduce the risk of complication10. It is important that patients
receive the best available treatment early in the course of
their disease to ensure that they achieve the most optimal
outcome11. If left untreated, patients with MF can experience
a significantly higher disease burden and may have a lower
life expectancy than patients who receive treatment and the
disease can progress, eventually becoming fatal12,13.
Common types of treatments include8:
Targeted-drug therapy: Use of a targeted-drug agent to address factors that play a role in the development of
MF, specifically the dysregulation of the JAK/STAT pathway10.
Cytoreductive therapy: This treatment helps to decrease high platelet and white blood cell counts and treat
complications of an enlarged spleen and other MF symptoms. Patients who take this treatment alone can
experience worsening symptoms or become resistant to or intolerant of treatment 5.
Bone marrow or stem cell transplant: The only potential curative treatment option presented to younger
patients whose disease state is more advanced. Because this treatment option involves high levels of
chemotherapy or radiation therapy, it is accompanied with harmful side effects and not recommended for older
patients.
Splenectomy (removal of the spleen): Procedure carried out to reduce severe pain and the feeling of
fullness experienced by patients with MF who have an enlarged spleen. Risks associated with splenectomy
include infection and potential long term cardiovascular complications.
Radiotherapy: This procedure is useful for a small number of patients to reduce spleen size and help alleviate
other MF-related symptoms. Radiation of the spleen is an option if a splenectomy cannot be performed on a
patient.
Patients should discuss with their hematologist the available treatments that best address their individual
circumstances14.
References:
1. Dictionary of Cancer Terms. National Cancer Institute. US National Institutes of Health. Available at: http://www.cancer.gov/dictionary. Accessed February 2016.
2. National Center for Biotechnology Information, US National Library of Medicine, National Institutes of Health. Myelofibrosis. Available at:
http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001558/. Accessed February 2016.
3. Zimmerman M et al. Myeloproliferative Disorders and Myelofibrosis. Am J Manag Care 2012 May; (3 Spec No.): SP131-3.
4. Cazzola M, Kralovics R. From Janus Kinase 2 to Calreticulin: The Clinically Relevant Genomic Landscape of Myeloproliferative Neoplasms. Blood. 2014 Jun
12;123(24):3714-9.
5. Cervantes F. How I treat splenomegaly in myelofibrosis. Blood Cancer J. Oct 2011; 1(10): e37.
6. Mesa RA, Schwagera S, Radia D, et al. The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and
symptomatic response to treatment in myelofibrosis. Leuk Res. 2009;33:1199-1203.
7. Leukemia & Lymphoma Society. Idiopathic myelofibrosis. Available at:
http://www.lls.org/#/diseaseinformation/myeloproliferativediseases/idiopathicmyelofibrosis/. Accessed February 2016.
8. National Cancer Institute. Myeloproliferative Neoplasm Treatments. Available at: http://www.cancer.gov/types/myeloproliferative/patient/chronic-treatmentpdq#section/_234. Accessed February 2016.
9. National Cancer Institute. Myelodysplastic/ Myeloproliferative Neoplasms Treatment. Available at:
http://www.cancer.gov/types/myeloproliferative/patient/mds-mpd-treatment-pdq. Accessed February 2016.
10. The Merck Manuals. Primary Myelofibrosis. Accessed at: http://www.merckmanuals.com/professional/hematology-and-oncology/myeloproliferativedisorders/primary-myelofibrosis. Accessed February 2016.
11. Cancer.net. When the First Treatment Doesn’t Work. Available at: http://www.cancer.net/navigating-cancer-care/how-cancer-treated/when-first-treatmentdoesnt-work. Accessed February 2016.
12. Mesa RA, Shields Alan, Hare Thomas, et al. Progressive burden of myelofibrosis in untreated patients: Assessment of outcomes in patients randomized to
placebo in the COMFORT-I study. Leuk Res. 2013 May 17; 37: 911– 916.
13. Tefferi A, Lasho TL, Jimma T, et al. One thousand patients with primary myelofibrosis: The mayo clinic experience. Mayo Clin Proc. 2012;87(1):25-33.
14. Leukemia & Lymphoma Society. Myelofibrosis. Available at: http://www.lls.org/myeloproliferative-neoplasms/myelofibrosis. Accessed February 2016.
Novartis Pharma AG
CH-4002 Basel Switzerland
© Novartis 2016
February 2016
G-INC-1132891