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
1/06
Notes
Table of Contents
My Health Care Team
Introduction
5
Doctor______________________ Phone _____________________
What Is Thalidomide?
6
Nurse ______________________ Phone _____________________
Is Thalidomide the Same
Hospital_____________________ Main Phone__________________
as Chemotherapy?
6
Other ______________________ Phone _____________________
Emergency Contacts
Who Can Benefit
From Thalidomide Therapy?
7
__________________________ Phone _____________________
__________________________ Phone _____________________
How Does Thalidomide Work?
9
Other Medications I Am Taking
What Are the Possible Side Effects
____________________________________________________
of Thalidomide?
10
____________________________________________________
____________________________________________________
Who Should Not Take Thalidomide?
15
Thalidomide Dosing Schedule
How Is Thalidomide Given?
16
Dose (eg, 100 mg) _________________________________________
Number of Capsules (eg, 2 x 50 mg) ______________________________
Can Thalidomide Be Taken
With Other Cancer Treatments?
17
Schedule: ® Evening
® Every Other Day
® Other ____________
Important Reminders When Taking Thalidomide
Will Insurance Cover the
____________________________________________________
Cost of Thalidomide?
17
____________________________________________________
A Final Note
18
____________________________________________________
About the IMF
19
____________________________________________________
____________________________________________________
Glossary
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The use of thalidomide for the treatment of myeloma is currently under investigation in clinical
trials. This booklet is meant to provide the patient with general information on thalidomide treat-
ment. It is not part of, nor is it meant to substitute for any part of, the System for Thalidomide
Education and Prescribing Safety (S.T.E.P.S.®) program. All patients must be advised of, agree to,
and comply with the requirements of the S.T.E.P.S. ® program in order to receive thalidomide.
©2006, International Myeloma Foundation, North Hollywood, California
Introduction
Using thalidomide to treat myeloma* is a
relatively new idea, 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 is currently approved for the
treatment of erythema nodosum leprosum, an
inflammatory condition seen in some patients
with leprosy. However, thalidomide is active-
ly being investigated for the treatment of
myeloma. Many patients with myeloma have
benefited from this therapy.
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.
The information in this booklet may be useful
not only to patients, but also to friends, family,
loved ones, and other caregivers. Please
*Words appearing in italics are defined in the glossary at the end of the booklet.
4
5
remember that myeloma, like al cancers, is a
because some healthy cel s are af ected by
complicated disease. Specific questions about
chemotherapy. Thalidomide is not consid-
treatment should be addressed by a doctor
ered a form of chemotherapy. It is instead
or nurse.
considered a new kind of treatment, because
it can af ect the levels of certain proteins that
WHAT IS THALIDOMIDE?
the body normal y uses to control the activity
Thalidomide is a drug that was first used in
of cel s.
the late 1950s in Europe for the treatment
of morning sickness. It was later withdrawn
WHO CAN BENEFIT FROM THALIDOMIDE
from use when it was reported that the
THERAPY?
drug produced severe, life-threatening birth
Today, thalidomide is approved for the
defects.
treatment of erythema nodosum leprosum.
However, thalidomide has also been studied
Today, the medical community has a bet er
in a number of other diseases, including
understanding of this drug and how it works.
cancer.
Thalidomide is classified as an immuno-
modulatory agent, which means it af ects the
Clinical trials have shown that thalidomide
levels of certain chemicals in the body that
is active against myeloma and can produce
control the activity of cel s. We know that tha-
lasting complete or partial responses, as
lidomide can produce many other ef ects that
wel as disease stabilization. In these trials,
are helpful, such as slowing or stopping the
growth of new blood vessels, cal ed angio-
genesis. Today, a program cal ed the
System for Thalidomide Education and
Prescribing Safety (S.T.E.P.S.®) helps to
ensure that every ef ort is made to use the
drug safely.
IS THALIDOMIDE THE SAME AS
CHEMOTHERAPY?
Chemotherapy works by kil ing cel s that
are dividing. These cel s include cancer cel s
as wel as some normal cel s in the body.
Hair loss, nausea and vomiting, and gastric
upset are common side ef ects that occur,
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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. It is important to note,
however, that not everyone who takes tha-
lidomide wil have a response, and other
therapies may be considered.
HOW DOES THALIDOMIDE WORK?
Although scientists are stil trying to under-
stand exactly how thalidomide fights cancer,
thalidomide is known to work on 2 important
levels. First, thalidomide is believed to boost
thalidomide has been found to be ef ective
the body's immune response to cancer.
in patients with dif erent stages of myeloma,
Second, it helps block the blood supply of
including:
cancerous tumors. Cancer cel s, like normal
n
cel s, need to get nutrients and oxygen from
Patients with newly diagnosed myeloma
the blood to survive and multiply. Some
n Patients who have not responded to other
tumors send chemicals into the body that can
treatments
trigger the formation of new blood vessels.
n Patients in whom myeloma has returned
As more blood vessels grow into the tumor, it
after initial successful treatment .
can become larger. It is thought that one way
thalidomide may help to limit tumor growth
Additional y, thalidomide has been successful
is by hindering new blood vessel growth
in treating myeloma either when given alone
within tumors.
or when given in combination with the drug
dexamethasone, a type of steroid. Selection
Thalidomide is also believed to act in several
of an appropriate treatment is made on a
other ways against myeloma, including tar-
case-by-case basis. The ideal daily dose of
geting the myeloma cel s and the molecules
thalidomide is under investigation. In some
that al ow them to grow. However, these
cases, low doses have been found to be
exact ef ects are not clear and scientists are
ef ective alone and in combination.
actively studying them.
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WHAT ARE THE POSSIBLE SIDE EFFECTS OF
to a health-care professional. Prescription
THALIDOMIDE?
medications and over-the-counter products that
are being taken should also be reported.
The most common side ef ects associated
with thalidomide are:
Drowsiness
n
Thalidomide often causes feelings of drowsi-
Drowsiness feelings of sleepiness or
ness. These methods may help relieve this
fatigue
side ef ect:
n Peripheral neuropathy tingling or numb-
n Taking thalidomide at bedtime
ness in the arms, hands, legs, and/or feet
n
n
Avoiding use of other drugs that may cause
Dizziness sensation of unsteadiness
drowsiness while taking thalidomide
n Constipation delayed or infrequent pas-
n At the discretion of a doctor or nurse,
sage of hardened feces
taking other drugs to help al eviate
n Rash an eruption on the skin
drowsiness
n Leukopenia a low level of white blood
n Avoiding alcohol.
cel s.
Situations in which drowsiness may be a
Other side effects have been reported,
problem should be avoided. Mental and
although infrequently. Any side ef ects a
physical abilities needed to perform danger-
patient experiences while receiving treatment
ous tasks, such as driving a car, may be
should be discussed with a doctor or nurse as
impaired.
soon as possible. In addition, any changes in
overal health or wel -being should be reported
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
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
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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.
Dizziness
Dizziness may occur while taking thalido-
mide. Sit ing up and waiting a few minutes
before get ing out of bed may help reduce
dizziness.
Constipation
Constipation may occur during treatment
with thalidomide; however, constipation
is rarely severe. Prevention is the key to
management.
These strategies may help al eviate constipa-
tion:
Rash
In some cases, a rash may develop while
n Drinking at least 8 glasses of fluid daily
taking thalidomide. A mild rash (red or dis-
n Adding plenty of dietary fiber every morn-
colored skin, with or without raised bumps)
ing, such as prune juice, apple juice,
usual y begins on the trunk and spreads
and bran
to the arms and legs. Mild rashes may be
n Exercising
relieved in the fol owing ways:
n At the recommendation of a doctor
n At the recommendation of a doctor or
or nurse, taking stool softeners and
nurse, taking antihistamines and topical
laxatives.
corticosteroids
If constipation becomes severe, the dose of
n To al eviate dry skin, using Calendra
thalidomide may be lowered or temporarily
lotion, cocoa but er cream, oatmeal
discontinued.
soap, Eucerin® cream, and Acid Mantle®
cream.
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13
Rashes often resolve spontaneously after
Leukopenia
about 10 to 14 days of treatment. Some
Thalidomide can sometimes cause a decrease
rashes are a potential y serious reaction to
in white blood cel s. This condition is cal ed
thalidomide treatment. Rare reactions include
leukopenia. Because of this possibility, blood
Stevens-Johnson syndrome and toxic epider-
tests need to be done regularly. If the white
mal necrolysis (TEN). Systems of Stevens-
blood cel count becomes too low, the dose
Johnson syndrome include persistent fever,
of thalidomide may have to be changed or
rash, blisters, or red splotches on the skin
the treatment may need to be interrupted.
and blisters in the mouth, eyes, ears, nose,
WHO SHOULD NOT TAKE THALIDOMIDE?
and genital area. TEN is characterized by
If thalidomide is taken during pregnancy, it
blistering and peeling of large sections of
can cause severe birth defects or death to an
skin.
unborn baby. Thalidomide should never be
A doctor should be contacted immediately
used by women who are pregnant or who
if a fever and/or drop in blood pressure
could become pregnant while taking the
occur.
drug. Thalidomide may be detected in male
sperm. Therefore, both men and women are
required to fol ow strict rules for birth control
while taking thalidomide.
Only physicians and pharmacists who are
registered with the special program cal ed
S.T.E.P.S.® can prescribe or dispense tha-
lidomide. Physicians and pharmacists may
register with the S.T.E.P.S.® program by cal -
ing Celgene Corporation, the manufacturer
of thalidomide, 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
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n Women of childbearing age must have
CAN THALIDOMIDE BE TAKEN WITH OTHER
pregnancy tests every week during the first
CANCER TREATMENTS?
month of thalidomide therapy and monthly
Yes, thalidomide can be taken alone or in
afterwards (every 2 weeks for women with
combination with chemotherapy, radiation
irregular menstrual cycles)
therapy, or biologic treatments. A doctor wil
n Women of childbearing age must receive
advise on the appropriate treatment for each
contraceptive counseling and use 2
individual.
methods of birth control 4 weeks before,
WILL INSURANCE COVER THE COST OF
during, and at least 4 weeks after complet-
THALIDOMIDE?
ing therapy
Insurance coverage varies. Individual insur-
n Men who are sexually involved with
ance companies can provide information
women of childbearing age must use a
regarding thalidomide coverage. Celgene
latex condom during and at least 4 weeks
Corporation also of ers a therapy assis-
after completing thalidomide therapy.
tance program. For more information
about this program, cal 888-4-CELGENE
HOW IS THALIDOMIDE GIVEN?
(888-423-5436).
Thalidomide is available as a capsule. The
dose, or number of capsules to be taken
every day, wil be determined by whether
thalidomide is being given alone or in com-
bination with other drugs. How the drug is
tolerated by the body wil also determine
the dose.
The dose may be gradual y increased over
time. A gradual increase ensures the most
ef ective dose is given as safely as possible.
If side ef ects occur, a doctor or nurse should
be notified immediately. The dose may need
to be lowered, or even discontinued, if the
side ef ects are severe. The dose should only
be changed under the direction of a doctor.
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A FINAL NOTE
About the IMF
Thalidomide is an important treatment for a
number of diseases. However, like any drug,
"One person can make a dif erence,
it can cause harm if misused. It is important
Two can make a miracle."
that al advice from health care profession-
Brian D. Novis
als be fol owed while taking this drug. Any
IMF Founder
questions should be promptly addressed by
a doctor or nurse.
Myeloma is a lit le-known, complex, and
often misdiagnosed bone marrow cancer
Questions and concerns regarding thalido-
that at acks and destroys bone. Myeloma
mide treatment may arise once treatment
af ects approximately 75,000 to 100,000
begins. Some of these concerns may be
people in the United States, with more than
about thalidomide itself. Others may be
15,000 new cases diagnosed each year.
about the outcome of treatment and its side
While there is presently no known cure for
ef ects. Stil other concerns may be more
myeloma, doctors have many approaches
emotional or financial in nature.
to help myeloma patients live bet er and
Many resources are available. Concerns
longer.
should be shared with the treatment
The International Myeloma Foundation (IMF)
team so that assistance can be provided.
was founded in 1990 by Brian and Susie
Communication wil help lead to the active
Novis shortly after Brian's myeloma diagno-
management of side ef ects, minimize symp-
sis at the age of 33. It was Brian's dream that
toms, and help al eviate fears and concerns
future patients would have easy access to
during treatment. Involvement in personal
medical information and emotional support
care wil ultimately lead to confidence and a
throughout their bat le with myeloma. He
sense of control regarding treatment choices.
established the IMF with the 3 goals of treat-
ment, education, and research. He sought
to provide a broad spectrum of services for
patients, their families, friends, and health
care providers. Although Brian died 4 years
after his initial diagnosis, his dream didn't.
Today the IMF reaches out to an international
membership of more than 125,000. The IMF
was the first organization dedicated solely to
myeloma, and today it remains the largest.
18
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The IMF provides programs and services to
MYELOMA TODAY NEWSLETTER
aid in the research, diagnosis, treatment,
Our quarterly newslet er is available free of
and management of myeloma. The IMF
charge by subscription.
ensures that no one must brave the myeloma
SUPPORT
bat le alone.
MYELOMA HOTLINE: 800-452-CURE (2873)
We care for patients today, while working
Toll-free throughout the United States and
toward tomorrow's cure.
Canada, the IMF Hotline is staf ed by trained
information specialists and is in frequent inter-
How Can the IMF Help You?
action with members of our Scientific Advisory
Board.
PATIENT EDUCATION
INFORMATION PACKAGE
SUPPORT GROUPS
Our free IMF InfoPack provides comprehensive
A worldwide network of more than 100 myelo-
information about myeloma, treatment options,
ma support groups hold regular meetings for
disease management, and IMF services. It
members of the myeloma community. The IMF
includes our acclaimed Patient Handbook.
conducts annual retreats for myeloma support
group leaders.
INTERNET ACCESS
Log on to www.myeloma.org for 24-hour
RESEARCH
access to information about myeloma, the IMF,
BANK ON A CURE ®
education, and support programs.
This DNA bank wil provide genetic data
ONLINE MYELOMA FORUM
research in new drug development.
Join the IMF Internet Discussion Group at
www.myeloma.org/listserve.html to share your
THE INTERNATIONAL STAGING SYSTEM (ISS)
thoughts and experiences.
This updated staging system for myeloma wil
enhance physicians' ability to select the most
MYELOMA MINUTE
appropriate treatment for each patient.
Subscribe to this free weekly email news-
let er for up-to-the-minute information about
RESEARCH GRANTS
myeloma.
Leading the world in col aborative research
and achieving extraordinary results, the IMF
PATIENT & FAMILY SEMINARS
Grant Program supports both junior and senior
Meet with leading experts in myeloma treat-
ment to learn more about recent advances in
researchers working on a broad spectrum of
therapy and research.
projects. The IMF has at racted many young
investigators into the field of myeloma, and
MYELOMA MATRIX
they have remained in the field and are actively
On our website and in print, this document is a
pursuing a cure for this disease.
comprehensive guide to drugs in development
for myeloma.
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Glossary
Appointments
Cells: The smal est unit of living organisms. Many cel s
Date
Time
Important Notes
together make up a tissue, and organized tissues
make up the organs of the human body.
Chemotherapy: Drugs that are used to kil cancer cel s.
Clinical trial: Study in which the ef ectiveness of a drug
is tested in a group of patients using a specific treat-
ment protocol.
Complete response: When a cancer regresses to such an
extent that it is undetectable. For myeloma, a complete
response means that the myeloma protein can no lon-
ger be detected in the blood and/or urine and that the
bone marrow shows no evidence of myeloma.
Disease stabilization: When a tumor stops progressing
and remains the same size.
Erythema nodosum leprosum: A condition that af ects
some patients with leprosy, in which areas of the skin
become reddened when smal blood vessels become
congested.
Immunomodulatory agent: A drug that af ects the level
of an immune response against invading tumor cel s
such as myeloma.
Inflammatory: Pertaining to a protective response of the
body against injury or disease.
Leukopenia: A low level of white blood cel s.
Myeloma: A cancer of bone marrow plasma cel s.
Cancerous plasma cel s are cal ed myeloma cel s.
Partial response: When a cancer regresses at least by
half but does not disappear completely.
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