FS14 Myelofibrosis Facts I page 4
Myelofibrosis Facts
l Decreased serum levels of albumin, total cholesterol and
high-density lipoprotein (HDL).
Because there are several other blood diseases, such as
leukemia and lymphoma that can cause marrow brosis,
to conrm a diagnosis of MF, the following tests may also
be performed.
l Imaging tests—Ultrasound tests may be used to
determine the size of the spleen. Magnetic resonance
imaging (MRI) tests may be used to identify changes in
the bone marrow that indicate MF.
l Bone marrow examination—Bone marrow biopsy and
aspiration ndings can conrm a diagnosis of MF. In
these procedures (usually done together), a small amount
of bone and marrow is removed and then examined under
a microscope by a hematopathologist (a doctor
specializing in evaluating cells, tissues and organs to
diagnose disease). Studying genetic components of the
bone marrow cells can show mutations in the cells that
may help eliminate other types of bone marrow disorders.
For example, the absence of the Philadelphia
chromosome or the BCR/ABL translocation can rule out
chronic myeloid leukemia (CML). A blood or bone
marrow sample may also be used for a test called a
“karyotype.” In this test, a microscope is used to examine
the size, shape and number of chromosomes in a sampling
of cells. e results of the karyotype may be helpful in
making certain treatment decisions.
l Gene tests—A sample of blood or bone marrow may be
analyzed in a laboratory to look for gene mutations, such
as the JAK2 mutation, present in 50 to 60 percent of
MF patients.
Treatment Planning
Some doctors use a prognostic scoring system to plan a
risk-adapted treatment strategy for a patient, which might
include observation only; Jaka;investigational therapies
(e.g., other JAK inhibitors, pomalidomide); drug therapy;
allogeneic stem cell transplantation or reduced-intensity
allogeneic stem cell transplantation; splenectomy; or
radiation therapy. One model is e Dynamic International
Prognostic Scoring System (DIPSS) Plus, which classies
risk as low (no risk factors), intermediate-1 (one risk factor),
intermediate-2 (2 or 3 risk factors) and high (4 or more
risk factors). e independent risk factors include
thrombocytopenia, anemia, need for red blood cell
transfusion, age above 65 years, leukocytosis (high white
blood cell count), unfavorable karyotype, circulating blast
cells and constitutional symptoms (e.g., weight loss greater
than 10 percent in the previous year, unexplained fever or
excessive sweating persisting for greater than one month).
Every patient’s medical situation is dierent and should be
evaluated individually by an oncologist who specializes in
treating blood cancers. It is important for you and members
of your medical team to discuss all treatment options,
including treatments being studied in clinical trials.
For more information about choosing a doctor or a
treatment center, see the free LLS publication Choosing a
Blood Cancer Specialist or Treatment Center.
Treatment
Currently, there is no drug therapy that can cure MF.
An allogeneic stem cell transplant (see Stem Cell Transplantation
on page 5) is the only potential cure for MF. e procedure is
risky in older MF patients, who may also have other health
problems, so allogeneic stem cell transplantation is usually
appropriate only for a small subset of younger patients,
typically less than 5 percent of patients with MF.
Patients who are symptom-free and do not have signs of
anemia, an enlarged spleen or other complications are
generally not treated. Some people remain stable and
symptom-free for many years. However, these patients need
to be monitored closely through regular medical checkups
and examinations to detect any signs and symptoms of
disease progression.
Drug erapies
l Janus-associated kinase (JAK) inhibitors—is drug class
inhibits enzymes called “JAK1” and “JAK2,” which are
involved in the production of blood cells.
Ruxolitinib (Jaka
TM
), given by mouth, is the rst JAK
inhibitor and currently the only drug approved by the
FDA to treat symptoms and signs of MF, including an
enlarged spleen, night sweats, itching and bone or muscle
pain. It is indicated for treatment of patients with
intermediate- or high-risk myelobrosis, including
primary myelobrosis, post polycythemia vera
myelobrosis and post essential thrombocythemia
myelobrosis. e most common side eects aecting the
blood cells are thrombocytopenia (a decrease below the
normal number of platelets) and anemia. Other common
side eects include bruising, dizziness and headache.
Patients should be aware that after discontinuation of
Jaka, myelobrosis signs and symptoms are expected to
return.ere have been isolated cases of patients
discontinuing Jaka during acute intervening illnesses
after which the patient’s clinical course continued to
worsen. It has not been established whether
discontinuation of therapy contributed to the clinical
course of these patients. When discontinuing Jaka
therapy for reasons other than thrombocytopenia, gradual
tapering of the dose of Jaka may be considered.