Understanding
Thalidomide
Therapy
International Myeloma Foundation
12650 Riverside Drive, Suite 206
North Hol ywood, CA 91607 USA
Telephone:
800-452-CURE
(USA & Canada)
818-487-7455
Fax: 818-487-7454
TheIMF@myeloma.org
www.myeloma.org
Table of Contents
Introduction
5
What Is Thalidomide?
6
Is Thalidomide the Same
as Chemotherapy?
7
Who Can Benefit
From Thalidomide Therapy?
7
How Does Thalidomide Work?
9
What Are the Possible Side Effects
of Thalidomide?
10
Who Should Not Take Thalidomide?
15
How Is Thalidomide Given?
16
Can Thalidomide Be Taken
With Other Cancer Treatments?
16
Will Insurance Cover the
Cost of Thalidomide?
17
A Final Note
17
About the IMF
18
Glossary
21
©2011, International Myeloma Foundation, North Hollywood, California (IV11)
Introduction
Using thalidomide to treat myeloma* is
an idea that emerged in the 1990s, but
thalidomide itself has been studied for many
decades. Much has been learned about
how thalidomide works in the treatment of
dif erent diseases and how its side effects can
be treated. Additional y, we now understand
how important it is to prevent women who
may be pregnant from being exposed to
thalidomide. Thalidomide was first approved
in the US for the treatment of erythema
nodosum leprosum, an inflammatory
condition seen in some patients with leprosy.
Thalidomide is also approved in combination
with dexamethasone for the treatment of
patients with newly diagnosed multiple
myeloma. Many patients with myeloma have
benefited from this therapy. Thalidomide is
also being investigated in combination with
other agents for the treatment of myeloma.
This booklet is intended to provide a basic
understanding of thalidomide therapy:
n What thalidomide is and how it works
to treat myeloma
n How to handle thalidomide safely
n What side ef ects might be expected
while taking thalidomide
n How doctors can minimize side ef ects
n How thalidomide may be used with
other therapies.
*Words appearing in bold are defined in the glossary at the end of the booklet.
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The information in this booklet may be useful
that are helpful, such as slowing or stopping
not only to patients, but also to friends, fam-
the growth of new blood vessels, cal ed
ily, loved ones, and other caregivers. Please
angiogenesis. Today, a program cal ed
remember that myeloma, like al cancers, is
the System for Thalidomide Education and
a complicated disease. Specific questions
Prescribing Safety (S.T.E.P.S.®) helps to
about treatment should be addressed by a
ensure that every ef ort is made to use the
doctor or nurse.
drug safely.
What is Thalidomide?
Is Thalidomide the Same as
Thalidomide is a drug that was first used in
Chemotherapy?
the late 1950s in Europe for the treatment of
Chemotherapy works by kil ing cel s that
morning sickness. It was later withdrawn from
are dividing rapidly. These cel s include
use when it was reported that the drug pro-
cancer cel s as wel as some normal cel s
duced severe, life-threatening birth defects.
in the body. Hair loss, nausea and vomit-
Today, the medical community has a bet er
ing, and gastric upset are common side
understanding of this drug and how it works.
ef ects that occur, because some healthy
Thalidomide is classified as an immuno-
cel s are affected by chemotherapy.
modulatory agent, which means it af ects
Thalidomide is not considered a form of
the levels of certain chemicals in the body
chemotherapy. It is instead considered a
that control the activity of cel s. We know that
new kind of treatment, because it can af ect
thalidomide can produce many other ef ects
the levels of certain proteins that the body
normal y uses to control the activity of cel s.
Who Can Benefit
from Thalidomide Therapy?
Today, thalidomide is approved both for the
treatment of erythema nodosum leprosum
and in combination with dexamethasone for
newly diagnosed myeloma.
Clinical trials have shown that thalidomide
is active against myeloma and can produce
lasting complete or partial responses, as
wel as disease stabilization. In these trials,
thalidomide has been found to be ef ective
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and 17-20 every 28 days. Selection of the
appropriate treatment and dosage, however
is made on a case-by-case basis. The ideal
daily dose of thalidomide is under investi-
gation. In some cases, lower doses have
been found to be ef ective alone and in
combination.
Response to thalidomide therapy takes time.
General y, improvement in the disease is
seen after about 3 months of treatment;
however, improvements have been noted as
early as 2 weeks and as late as 8 months.
Once a response is achieved, the physician
wil determine if ongoing, or maintenance,
therapy is needed and appropriate. The
in patients with dif erent stages of myeloma,
presence or absence of side ef ects, wil , of
including:
course, influence this decision. It is important
to note, however, that not everyone who
n Patients with newly diagnosed myeloma
takes thalidomide wil have a response, and
n Patients who have not responded to other
other therapies may be considered.
treatments
n Patients in whom myeloma has returned
How Does Thalidomide Work?
after initial successful treatment .
Although scientists are stil trying to under-
Additional y, thalidomide has been success-
stand exactly how thalidomide fights cancer,
ful in treating myeloma either when given
thalidomide is believed to work on two impor-
alone or when given in combination with
tant levels. First, thalidomide is believed to
the drug dexamethasone, a type of steroid.
boost the body's immune response to cancer.
According to the prescribing information on
Second, it helps block the blood supply of
the package insert for thalidomide (brand
cancerous tumors. Cancer cel s, like normal
name in many countries: Thalomid®), the
cel s, need to obtain nutrients and oxygen
standard dose is 200mg administered oral y
from the blood to survive and multiply. Some
once daily with water, preferably at bed-
tumors send chemicals into the body that can
time and at least 1 hour after the evening
trigger the formation of new blood vessels.
meal. The dose of dexamethasone is 40mg
As more blood vessels grow into the tumor, it
daily administered oral y on days 1-4, 9-12,
can become larger. It is thought that one way
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thalidomide may help to limit tumor growth
n Rash an eruption on the skin
is by hindering new blood vessel growth
n Leukopenia a low level of white blood
within tumors.
cel s.
Thalidomide is also believed to act in several
Other side effects have been reported,
other ways against myeloma, including tar-
although infrequently. Any side ef ects a
geting the myeloma cel s and the molecules
patient experiences while receiving treatment
that al ow them to grow. However, these
should be discussed with a doctor or nurse as
exact ef ects are not clear and scientists are
soon as possible. In addition, any changes in
actively studying them.
overal health or wel -being should be reported
to a health-care professional. Prescription
What Are the Possible Side Effects
medications and over-the-counter products
that are being taken should also be reported.
of Thalidomide?
The most common side ef ects associated
Drowsiness
with thalidomide are:
Thalidomide often causes feelings of
drowsiness. These methods may help relieve
n Drowsiness feelings of sleepiness or
this side ef ect:
fatigue
n Taking thalidomide at bedtime
n Peripheral neuropathy tingling or numb-
ness in the arms, hands, legs, and/or feet
n Avoiding use of other drugs that may cause
drowsiness while taking thalidomide
n Dizziness sensation of unsteadiness
n At the discretion of a doctor or nurse,
n Constipation delayed or infrequent
taking other drugs to help al eviate
passage of hardened feces
drowsiness
n Avoiding alcohol.
Situations in which drowsiness may be a
problem should be avoided. Mental and
physical abilities needed to perform danger-
ous tasks, such as driving a car, may be
impaired.
Peripheral neuropathy
Impairment of the nerves in the extremities
(hands, arms, legs, feet) is known as periph-
eral neuropathy. This side ef ect can be mild,
causing tingling in the hands and feet; more
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rarely, it can be severe and painful. It typi-
cal y occurs after a long period of taking tha-
lidomide, but it can sometimes occur sooner.
These strategies may help al eviate symptoms
of peripheral neuropathy:
n Walking and other forms of exercising
n Avoiding tight shoes and socks with
elastic
n At the discretion of a doctor, reducing the
dose of thalidomide
n At the discretion of a doctor or nurse,
taking additional medications.
A physician should be notified if any symp-
toms of peripheral neuropathy occur. If side
ef ects are severe, thalidomide therapy may
need to be stopped altogether.
n At the recommendation of a doctor
Dizziness
or nurse, taking stool softeners and
Dizziness may occur during treatment with
laxatives.
thalidomide. Sit ing up and waiting a few
If constipation becomes severe, the dose of
minutes before standing or get ing out of bed
thalidomide may be lowered or temporarily
may help reduce dizziness.
discontinued.
Constipation
Rash
Constipation may occur during treatment
In some cases, a rash may develop while
with thalidomide; however, constipation
taking thalidomide. A mild rash (red or dis-
is rarely severe. Prevention is the key to
colored skin, with or without raised bumps)
management.
usual y begins on the trunk and spreads
These strategies may help al eviate
to the arms and legs. Mild rashes may be
constipation:
relieved in the fol owing ways:
n Drinking at least 8 glasses of fluid daily
n At the recommendation of a doctor or
n Adding plenty of dietary fiber every morn-
nurse, taking antihistamines and topical
ing, such as prune juice, apple juice,
corticosteroids
and bran
n To al eviate dry skin, using Calendra
n Exercising
lotion, cocoa but er cream, oatmeal soap,
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Eucerin® cream, or Acid Mantle® cream.
of thalidomide may have to be changed or
the treatment may need to be interrupted.
Rashes often resolve spontaneously after
about 10 to 14 days of treatment. Some
Who Should Not Take Thalidomide?
rashes are a potential y serious reaction to
If thalidomide is taken during pregnancy, it
thalidomide treatment. Rare reactions include
can cause severe birth defects or death to an
Stevens-Johnson syndrome and toxic epider-
unborn baby. Thalidomide should never be
mal necrolysis (TEN). Symptoms of Stevens-
used by women who are pregnant or who
Johnson syndrome include persistent fever,
could become pregnant while taking the
rash, blisters, or red splotches on the skin and
drug. Thalidomide may be detected in male
blisters in the mouth, eyes, ears, nose, and
sperm. Therefore, both men and women are
genital area. TEN is characterized by blister-
required to fol ow strict rules for birth control
ing and peeling of large sections of skin.
while taking thalidomide.
A doctor should be contacted immediately if
Only physicians and pharmacists who are
a fever and/or drop in blood pressure occur.
registered with the special program cal ed
Leukopenia
S.T.E.P.S.® (as it is known in the US and
Thalidomide can sometimes cause a decrease
in some other countries) can prescribe or
in white blood cel s. This condition is cal ed
dispense thalidomide. In the US, physi-
leukopenia. Because of this possibility, blood
cians and pharmacists may register with
tests need to be done regularly. If the white
the S.T.E.P.S.® program by cal ing Celgene
blood cel count becomes too low, the dose
Corporation, the manufacturer of thalido-
mide, at 888-4-CELGENE (888-423-5436).
Both men and women must agree to fol ow
this program before receiving thalidomide.
To minimize the risk of exposing an unborn
child to thalidomide, the S.T.E.P.S.® program
includes the fol owing elements:
n Patients must provide informed consent,
complete confidential enrol ment, and
complete fol ow-up surveys throughout
treatment
n Women of childbearing age must have
pregnancy tests every week during the first
month of thalidomide therapy and monthly
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afterwards (every 2 weeks for women with
therapy, or biologic treatments. A doctor wil
irregular menstrual cycles)
advise on the appropriate treatment for each
n Women of childbearing age must
individual. Thalidomide is being tested in
receive contraceptive counseling and use
combination with other anti-myeloma agents,
2 methods of birth control 4 weeks before,
for example, with VELCADE® with and with-
during, and at least 4 weeks after complet-
out dexamethasone, and with melphalan
ing therapy
plus prednisone. Details about therapy with
VELCADE® and dexamethasone are dis-
n Men who are sexually involved with
cussed in separate booklets.
women of childbearing age must use a
latex condom during and at least 4 weeks
Will Insurance Cover the Cost
after completing thalidomide therapy.
of Thalidomide?
How is Thalidomide Given?
Insurance coverage varies. Individual insur-
Thalidomide is available as a capsule. The
ance companies can provide information
dose, or number of capsules to be taken
regarding thalidomide coverage. In the US,
every day, will be determined by whether
Celgene Corporation also of ers a therapy
thalidomide is being given alone or in com-
assistance program. For more information
bination with other drugs. How the drug is
about this program, cal Celgene's Patient
tolerated by the body wil also determine
Support Coordinator Program at 800-931-
the dose.
8691.
The dose may be gradual y increased over
A Final Note
time. A gradual increase ensures the most
Thalidomide is an important treatment for a
ef ective dose is given as safely as possible.
number of diseases. However, like any drug,
If side ef ects occur, a doctor or nurse should
it can cause harm if misused. It is important
be notified immediately. The dose may need
that the advice that a patient receives from
to be lowered, or even discontinued, if the
health care professionals be fol owed when
side ef ects are severe. The dose should only
taking thalidomide. Any questions should be
be changed under the direction of a doctor.
promptly addressed with a doctor or nurse.
Questions or concerns regarding treatment
Can Thalidomide Be Taken
may arise once it is started. Some of these
with Other Cancer Treatments?
may be about the drug itself. Others may be
Yes, thalidomide can be taken alone or in
about treatment outcome or side ef ects. Stil
combination with chemotherapy, radiation
others may be emotional, and even financial,
in nature.
16
17
Many resources are available. Good commu-
and healthcare providers. Although Brian
nication with your healthcare providers about
died four years after his initial diagnosis, his
your concerns wil help to actively manage
dream did not. Today, the IMF reaches out
side ef ects, minimize symptoms, and al evi-
to an international membership of more than
ate fears and concerns during treatment. Your
195,000. The IMF was the first organization
involvement in your healthcare wil ultimately
dedicated solely to myeloma, and today it
lead to confidence and a sense of control
remains the largest.
regarding treatment choices.
The IMF provides programs and services to
About the IMF
aid in the research, diagnosis, treatment,
and management of myeloma. The IMF
"One person can make a dif erence,
ensures that no one must brave the myeloma
Two can make a miracle."
bat le alone.
Brian D. Novis
We care for patients today, while working
IMF Founder
toward tomorrow's cure.
Myeloma is a lit le-known, complex, and
How Can the IMF Help You?
often misdiagnosed bone marrow cancer
that at acks and destroys bone. Myeloma
PATIENT EDUCATION
af ects approximately 75,000 to 100,000
INfORmATION PACkAgE
people in the United States, with more than
Our free IMF InfoPackTM provides comprehensive
20,000 new cases diagnosed each year.
information about myeloma, treatment options,
disease management, and IMF services. It includes
Although there is presently no known cure for
our acclaimed Patient Handbook.
myeloma, doctors have many approaches to
help myeloma patients live bet er and longer.
INTERNET ACCEss
Log on to www.myeloma.org for 24-hour access
The International Myeloma Foundation (IMF)
to information about myeloma, the IMF, education,
was founded in 1990 by Brian and Susie
and support programs.
Novis shortly after Brian's myeloma diagno-
myELOmA mANAgERTM PERsONAL CARE AssIsTANTTM
sis at the age of 33. It was Brian's dream
This software program was developed by the
that future patients would have easy access
IMF and is designed specifical y to help patients
to medical information and emotional sup-
and caregivers to capture, display, and store
laboratory test results, and to access important
port throughout their bat le with myeloma.
information. It is available free of charge on the
He established the IMF with the three goals
IMF website at www.myeloma.org. Currently this
of treatment, education, and research. He
program is only compatible with PCs.
sought to provide a broad spectrum of ser-
ONLINE myELOmA fORUm
vices for patients and, their families, friends,
Join the IMF Internet Discussion Group at
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myeloma.org/listserve.html to share your thoughts
REsEARCH gRANTs
and experiences.
Leading the world in col aborative research and
achieving extraordinary results, the IMF Grant
myELOmA mINUTETM
Subscribe to this free weekly email newslet er for
Program supports both junior and senior research-
up-to-the-minute information about myeloma.
ers working on a broad spectrum of projects.
The IMF has at racted many young investigators
Imf PATIENT & fAmILy sEmINARsTM
into the field of myeloma; they have remained
Meet with leading experts in myeloma treatment
in the field and are actively pursuing a cure for
to learn more about recent advances in therapy
this disease.
and research.
myELOmA mATRIxTM
Glossary
On our website and in print, this document is a
Cells: The smal est unit of living organisms. Many cel s
comprehensive guide to drugs in development for
together make up a tissue, and organized tissues
myeloma.
make up the organs of the human body.
myELOmA TODAyTM NEwsLETTER
Chemotherapy: Drugs that are used to kil cancer cel s.
Our quarterly newslet er is available free of
charge by subscription.
Clinical trial: Study in which the ef ectiveness of a drug
is tested in a group of patients using a specific treat-
sUPPORT
ment protocol.
myELOmA HOTLINE: 800-452-CURE (2873)
Complete response: When a cancer regresses to such an
Tol -free throughout the United States and Canada,
extent that it is undetectable. For myeloma, a complete
the IMF Hotline is staf ed by trained information
response means that the myeloma protein can no
specialists and is in frequent interaction with mem-
longer be detected in the blood and/or urine and
bers of our Scientific Advisory Board.
that the bone marrow shows no evidence of myeloma.
sUPPORT gROUPs
Disease stabilization: When a tumor stops progressing
A worldwide network of more than 100 myelo-
and remains the same size.
ma support groups hold regular meetings for
members of the myeloma community. The IMF
Immunomodulatory agent: A drug that af ects the level
conducts annual retreats for leaders of myeloma
of an immune response against invading tumor cel s
support group leaders.
such as myeloma.
Inflammatory: Pertaining to a protective response of the
REsEARCH
body against injury or disease.
BANk ON A CURE®
Leukopenia: A low level of white blood cel s.
This DNA bank wil provides genetic data
research in new drug development.
Myeloma: A cancer of bone marrow plasma cel s.
THE INTERNATIONAL sTAgINg sysTEm (Iss)
Cancerous plasma cel s are cal ed myeloma cel s.
This updated staging system for myeloma enhanc-
Partial response: When a cancer regresses at least by
es physicians' ability to select the most appropri-
half but does not disappear completely.
ate treatment for each patient.
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Peripheral neuropathy: Tingling or numbness in the
hands, arms, legs, and/or feet.
Side effects: Unwanted ef ects caused by a drug.
S.T.E.P.S.® (System for Thalidomide Education and Prescribing
Safety) program: A program designed for doctors,
nurses, pharmacists, and patients to ensure that devel-
oping fetuses are not exposed to thalidomide.
White blood cells: Blood cel s that help the body
fight infection.
22