Understanding
Thalidomide
Therapy
Table of Contents
Introduction
5
What Is Thalidomide?
6
Is Thalidomide the Same
as Chemotherapy?
6
Who Can Benefit From
Thalidomide Therapy?
6
What Are the Possible Side Effects of
Thalidomide?
8
Who Should Not Take Thalidomide?
11
How Is Thalidomide Given?
11
Can I Take Thalidomide With
Other Cancer Treatments?
12
Will Insurance Cover the Cost
The use of thalidomide for the treatment of multiple
of Thalidomide?
12
myeloma is currently under investigation in clinical trials.
This booklet is meant to provide the patient with general
information on thalidomide treatment. It is not part of, nor
A Final Note
13
is it meant to substitute for any part of, the S.T.E.P.S.TM
program. All patients must be advised of, agree to, and
comply with the requirements of the S.T.E.P.S.TM program
in order to receive thalidomide.
Glossary
15
Sponsored by an unrestricted educational grant from
Celgene Corporation.
About the IMF
20
©2001, International Myeloma Foundation
North Hollywood, California
Introduction
Using thalidomide to treat myeloma* is a new
idea, but thalidomide itself has been studied for
many decades. Much has been learned about
how thalidomide works in the treatment of
different diseases, and how its side effects can
be treated. Additionally, 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, a condition
seen in some patients with leprosy. However,
thalidomide is actively being investigated for the
treatment of multiple myeloma. Many patients
with myeloma have benefited from this therapy.
This booklet is intended to provide you with
a basic understanding of thalidomide therapy:
What thalidomide is, and how it works to
treat myeloma
How to handle thalidomide safely
What side effects you might expect while
taking thalidomide
How your doctor can minimize side effects
How thalidomide may be used with other
therapies
You may want to share this information with
friends, family, or loved ones who are helping
you during treatment. Please remember that
myeloma, like all cancers, is a complicated
disease. Speak with your doctor or nurse if you
have specific questions about your treatment.
*All words that appear in italics are defined in the glossary at the back of this booklet.
4
5
What Is Thalidomide?
thalidomide is approved for the treatment of
Thalidomide is a drug that was first used in the
erythema nodosum leprosum, an inflammatory
late 1950s in Europe for the treatment of morning
condition seen in some patients with leprosy.
sickness. It was later withdrawn from use when
Thalidomide is also being studied in a number of
it was reported that the drug produced severe,
other diseases, including cancer. Cancer cells,
life-threatening birth defects.
like normal cells, need to get nutrients and oxygen
Today, we have a better understanding of this
from the blood in order to survive and multiply.
drug and how it works. Thalidomide is classified as
Some tumors send chemicals into the body that
an immunomodulatory agent. That means it affects
can trigger the formation of new blood vessels.
the levels of certain chemicals in the body that
As more blood vessels grow into the tumor, it
control the activity of cells. We know that
can become larger. It is thought that one way
thalidomide can produce many other effects that
thalidomide may help to limit tumor growth is by
are helpful, such as slowing or stopping the growth
hindering new blood vessel growth within tumors.
of new blood vessels, called angiogenesis. Every
Clinical trials have already shown that
effort is made to ensure that the drug is used safely.
thalidomide is active against myeloma, and can
produce lasting complete or partial responses,
Is Thalidomide the Same
as well as disease stabilization. It is not yet
as Chemotherapy?
known if thalidomide is working through its
Chemotherapy works by killing cells that are
effect on blood vessels, or if it has a direct effect
dividing. This includes cancer cells, as well as
on myeloma cells. Additional trials are
some normal cells in the body. Hair loss, nausea
underway to identify how thalidomide can be
and vomiting, and gastric upset are common
used in combination with chemotherapy to
side effects that occur because healthy cells are
further improve responses, and possibly
affected by chemotherapy. Thalidomide is not
increase survival.
considered to be a form of chemotherapy.
Instead, it is considered a new kind of treatment
because it can affect the levels of certain proteins
that the body normally uses to control the activity
of cells.
Who Can Benefit From Thalidomide
Therapy?
Thalidomide has been shown to slow or stop the
growth of newly forming blood vessels. Today,
6
7
However, drowsiness can carry over into the next
morning. Therefore, situations where 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. Other drugs that cause drowsiness
should be avoided when taking thalidomide.
Alcohol can also increase the drowsiness caused
by thalidomide, and should also be avoided.
Peripheral neuropathy
Damage to the nerves in the extremities (hands,
arms, legs, feet) is known as peripheral neu-
ropathy. This side effect can be mild, causing
tingling in the hands and feet; more rarely, it
can be severe and painful. It typically occurs
after a long period of taking thalidomide, but it
can sometimes occur sooner. Notify your doctor
What Are the Possible Side Effects of
if you feel any tingling or pain in your hands,
Thalidomide?
arms, legs, or feet. The dose of thalidomide may
The most common side effects associated with
need to be lowered, or treatment interrupted,
thalidomide are drowsiness, peripheral neuropathy,
until symptoms get better. For some people who
dizziness, constipation, rash, and leukopenia.
experience severe side effects, thalidomide
Your doctor or nurse will discuss any other side
treatment must be stopped altogether.
effects that you should be aware of, based on
Dizziness
your own health profile.
Some people report dizziness while taking
Always discuss any changes you notice in your
thalidomide. Sitting up and waiting a few
health with your doctor or nurse. Also, make
minutes before getting out of bed may help
sure that your doctor is aware of any other
reduce feelings of dizziness.
prescription medications or over-the-counter
Constipation
products that you may be taking while you are
Some people experience some constipation
on thalidomide.
during treatment with thalidomide; however,
Drowsiness
constipation is rarely severe. Prevention is the
Thalidomide often causes people to feel sleepy.
key to managing constipation. Increasing your
Patients typically take thalidomide at bedtime.
fluid intake and dietary fiber will help to
8
9
minimize the risk of constipation. Your doctor or
done regularly. If the white cell count becomes
nurse may also instruct you on the use of stool
too low, the dose of thalidomide may have to be
softeners or laxatives. If constipation becomes
changed or the treatment interrupted.
severe, your doctor may lower your thalidomide
Who Should Not Take Thalidomide?
dose or discontinue it temporarily.
If thalidomide is taken during pregnancy, it can
Rash
cause severe birth defects or death to an unborn
Some people develop a rash with thalidomide
baby. Thalidomide should never be used by
treatment. It usually begins on the trunk and
women who are pregnant or who could
spreads to the arms and legs. Antihistamines
become pregnant while taking the drug.
and topical corticosteroids may provide relief.
Thalidomide may be detected in male sperm.
Rash often resolves spontaneously after approx-
Therefore, men are also required to follow strict
imately 10 to 14 days of treatment. Some rashes
rules for birth control while taking thalidomide.
are a potentially serious reaction to thalidomide
Only physicians and pharmacists who are
treatment. If you have a fever or drop in blood
registered with a special program called
pressure, please contact your doctor immediately.
S.T.E.P.S.TM (System for Thalidomide Education
Leukopenia
and Prescribing Safety) can prescribe or dis-
Thalidomide can sometimes cause a decrease
pense thalidomide. Physicians and pharmacists
in white blood cells. This condition is called
may register with the S.T.E.P.S.TM program by
leukopenia. That is why blood tests need to be
calling celgene at 1-(888)-4-CELGENE. Both
men and women must agree to follow this
program before receiving thalidomide. Your
doctor or nurse will take you through the entire
S.T.E.P.S.TM program, so that you will know
exactly how to minimize the risk of exposing an
unborn child to thalidomide.
How Is Thalidomide Given?
Thalidomide is available as a capsule. Your
doctor will tell you how many capsules to take
each day. The dose will be determined by
whether thalidomide is being given alone or in
combination with other drugs -- and also by
how your body tolerates the drug.
10
11
Your dose may be gradually increased over
A Final Note
time. This is to make sure you get the most
Thalidomide is an important treatment for a
effective dose as safely as possible.
number of diseases. However, like any drug,
If side effects occur, tell your doctor or nurse
it can cause harm if misused. It is important
immediately. Your dose may need to be low-
to follow all of your doctor's instructions while
ered, or even discontinued for a time, if the side
taking this drug. If you have any questions about
effects are severe. Do not change the amount
thalidomide, or any other part of your treatment,
you are taking without first talking with your
do not hesitate to ask your doctor or nurse.
doctor.
Many patients find that they have a lot of ques-
Remember, not everyone who takes thalidomide
tions and concerns as they adjust to thalidomide
has a response. People who do respond to
treatment. Some of these concerns are about
therapy generally see an improvement at about
thalidomide itself. Others are about the outcome
3 months. However, improvement may be seen
as early as 2 weeks or as late as 8 months. Your
doctor will discuss with you whether therapy
should continue after an improvement is seen.
Can I Take Thalidomide With Other
Cancer Treatments?
Yes, thalidomide is taken alone or in combination
with chemotherapy, radiation therapy, or other
biologic treatments. Your doctor will know
which treatments are best for you.
Will Insurance Cover the Cost
of Thalidomide?
Insurance coverage varies. It is important to
check with your insurance carrier to determine
if your medical insurance covers thalidomide
therapy. Celgene Corporation, the manufacturer
of thalidomide, offers a Therapy Assistance
Program (TAP). For more information about TAP,
call 1-(888)-4-CELGENE (1-888-423-5436).
12
13
Glossary
of treatment and its side effects. Still other
cells: The smallest unit of living organisms. Many cells
concerns may be more emotional or financial
together make up a tissue, and organized tissues make
in nature.
up the organs of the human body.
There are many resources available to you.
chemotherapy: Drugs that are used to kill cancer cells.
Share your concerns with your treatment team
clinical trial: Study in which the effectiveness of a drug
so that they can assist you. By communicating
is tested in a group of patients using a specific treatment
openly with your doctor, you will find that you
protocol.
can play an active part in managing side effects
complete response: When a tumor regresses to such
and minimizing symptoms during treatment. The
an extent that it is undetectable.
more you get involved in your own care, the
disease stabilization: When a tumor stops progress-
more in control you will feel about your treat-
ing, and remains the same size.
ment choices.
erythema nodosum leprosum: A condition that
affects some patients with leprosy, in which areas of the
skin become reddened when small blood vessels become
congested.
immunomodulatory agent: A drug that affects the
level of an immune response (that is, the body's response
to a foreign substance).
inflammatory: Pertaining to a protective response
of the body against injury or disease.
leukopenia: A low level of white blood cells.
myeloma: A cancer composed of cells normally found
in the bone marrow (the blood cell-producing tissues
within the bone).
partial response: When a tumor regresses at least by
half, but does not disappear completely.
peripheral neuropathy: Tingling or numbness in the
arms, hands, legs, and/or feet.
side effects: Unwanted effects caused by a drug.
S.T.E.P.S.TM program: A program designed for doctors,
nurses, pharmacists, and patients to ensure that develop-
ing fetuses are not exposed to thalidomide.
white blood cells: Blood cells that help the body fight
infection.
14
15
Notes
My Health Care Team
Doctor ____________________ Phone ___________
Nurse ____________________ Phone ___________
Hospital ________________ Main Number ___________
Other ____________________ Phone ____________
Emergency Contacts
________________________ Phone ____________
________________________ Phone ____________
Other Medications I Am Taking
___________________________________________
___________________________________________
Thalidomide Dosing Schedule
___________________________________________
___________________________________________
Important Reminders When Taking Thalidomide
___________________________________________
___________________________________________
___________________________________________
16
17
Appointments
Date
Time
Important Notes
Date
Time
Important Notes
18
19
services for patients, their families,
About the IMF
friends, and health care providers.
"One person can make a difference,
Although Brian died 4 years after his
Two can make a miracle."
initial diagnosis, his dream didn't. Today
the IMF reaches out to an international
Brian D. Novis
membership of more than 70,000. The
IMF Founder
IMF was the first organization dedicated
Multiple myeloma is a little-known,
solely to multiple myeloma, and today
complex, and often misdiagnosed bone
remains the largest.
marrow cancer that attacks and destroys
The IMF provides programs and services
bone. Myeloma affects approximately
to aid in the research, diagnosis, treat-
75,000 to 100,000 people in the United
ment, and management of myeloma. The
States, with more than 14,500 new
IMF ensures that no one must brave the
cases diagnosed each year. While there
myeloma battle alone. We care for
is presently no known cure for myeloma,
patients today, while working toward
doctors have many approaches to help
tomorrow's cure.
myeloma patients live better and longer.
How Can the IMF Help You?
The International Myeloma Foundation
(IMF) was founded in October 1990 by
(800) 452-CURE
Brian and Susie Novis shortly after
Need to talk? The IMF Hotline, toll-free
Brian's myeloma diagnosis at the age of
throughout the United States and Canada,
provides immediate support and information.
33. It was Brian's dream that future
patients would have easy access to med-
INTERNET ACCESS
ical information and emotional support
At www.myeloma.org, you'll find 24-
throughout their battle with myeloma. He
hour access to in-depth information about
established the IMF with the 3 goals of
multiple myeloma as well as information
treatment, education, and research. He
about the IMF and our education and support
sought to provide a broad spectrum of
programs.
20
21
INFORMATION PACKAGE
PATIENT-TO-PATIENT DIRECTORY
The IMF Info Pack provides comprehensive
The IMF maintains a confidential worldwide
information on treatment options and disease
directory of patients and family members that
management. Available free of charge, the
can help put you in touch with IMFers in your
Info Pack includes The IMF Patient
area.
Handbook and Prof. G.M. Durie's
PATIENT & FAMILY SEMINARS
Concise Review of the Disease and
Each year the IMF holds educational Patient &
Treatment Options as well as information
Family Seminars in cities around the world.
on other IMF publications and services.
These meetings enable patients and their
MYELOMA TODAY
families to learn about the latest developments
Stay in the loop with Myeloma Today, the IMF
in myeloma from a panel of world-renowned
newsletter published 6 times per year.
myeloma experts. The IMF Patient & Family
Myeloma Today covers a broad range of
Seminars are also a great opportunity to meet
topics, offering up-to-date information of use
others touched by myeloma.
to both patients and physicians.
RESEARCH
SUPPORT GROUPS
The IMF research grant program, which funds
The IMF works with a network of more than
both junior and senior investigators, is
50 multiple myeloma support groups from
designed to increase the number of researchers
around the world. To find the support group
working on a cure for myeloma. Created in
nearest you, contact the IMF.
memory of IMF founder Brian D. Novis, the
grants support research into better treatment,
ONLINE MYELOMA FORUM
management, prevention, and ultimately, a cure
The IMF Internet Discussion Group hosts an
for myeloma.
online support group where users can log on
and share thoughts and experiences. Visit
PHYSICIAN SERVICES
www.myeloma.org/listserve.html to
The IMF organizes clinical conferences, expert
access this forum.
research roundtables, and scientific work-
shops to keep physicians and researchers
abreast of advances in the field of myeloma.
Several IMF programs offer continuing medi-
cal education (CME) credit.
22
23
International Myeloma Foundation
12650 Riverside Drive, Suite 206
North Hollywood, CA 91607-3421 USA
Tel:
(800) 452-CURE (US and Canada)
(818) 487-7455
Fax:
(818) 487-7454
TheIMF@myeloma.org
www.myeloma.org
3/01