Understanding
Stem Cell
Transplant
International Myeloma Foundation
12650 Riverside Drive, Suite 206
North Hol ywood, CA 91607 USA
Telephone:
800-452-CURE (2873)
(USA & Canada)
818-487-7455
Fax: 818-487-7454
TheIMF@myeloma.org
www.myeloma.org
03/07
Table of Contents
Introduction
5
What is Multiple Myeloma?
5
The Stages of Multiple Myeloma
6
Background Rationale for Use of High-Dose
Chemotherapy and Blood Stem Cell
Transplant or Rescue
8
Types of Stem Cell Transplant
9
How Stem Cell Transplant is Used
as a Part of Myeloma Therapy
10
What are the Benefits of High-Dose
Chemotherapy with Blood Stem Cell Rescue? 12
Practical Steps in Considering Stem Cell
Transplant as a Treatment Option
14
How Stem Cells are Collected
15
Administering High-Dose Chemotherapy
20
Preventing Infection
23
Engraftment and Recovery
24
Am I a Candidate for an
Autologous Transplant?
25
Transplants and Clinical Trials
26
Psychosocial Issues
28
Questions and Answers about Stem Cell
Transplantation
29
Questions for the Doctor
35
About the IMF
39
Glossary
42
Bibliography
47
©2007, International Myeloma Foundation, North Hollywood, California
Introduction
This booklet explains important details about
the transplant of blood stem cel s. Questions
addressed include:
n What are blood stem cel s?
n Why are blood stem cel s col ected and used
for transplant?
n What are the benefits and risks of high-dose
chemotherapy with blood stem cel rescue as
part of the treatment for myeloma?
n What is the role of high-dose chemotherapy
versus novel therapies? Can they be used in
combination?
The booklet is meant to provide you with gen-
eral information only. It is not meant to replace
the advice of your doctor, nurse, or other
healthcare practitioners. Your healthcare team
can answer specific questions related to your
personal treatment plan. Al words that appear
in bold type are defined in the glossary at the
back of this booklet.
What is Multiple Myeloma?
Multiple myeloma (also known as myeloma
and plasma cel myeloma) is a cancer of the
immunoglobulin-producing plasma cel s found
in the bone marrow. It is a cancer that involves
the immune system. The cancerous plasma
cel s, or myeloma cel s, rarely enter the blood
stream. The myeloma cel s accumulate in the
bone marrow, causing the fol owing:
n Disruption of normal bone marrow function,
most commonly causing anemia (a low
level of red blood cel s in the bloodstream),
4
5
although reduction in white blood cel and
Stage II (intermediate cell mass): An inter-
platelet counts can also occur
mediate stage between stage I and stage I I.
n Damage to bone surrounding accumulated
Stage III (high cell mass): More
myeloma cel s
advanced disease. One or more of the
fol owing are present:
n Release of an abnormal protein, monoclonal
protein (M protein), into the blood stream
n Anemia
and/or urine
n A high level of calcium in the blood
n Suppression of normal immune function,
n More than 3 areas of advanced lytic bone
observed as reduced levels of normal immu-
lesions (destructive holes in the bones)
noglobulins and increased susceptibility to
n A high level of M protein in the blood or
infection
urine
Myeloma cel s can also grow in the form of local-
A new prognostic factor system cal ed the
ized tumors or plasmacytomas. Plasmacytomas
International Staging System (ISS) was recently
may be single or multiple and either medul ary
introduced. It is based upon the levels of two
(confined within bone marrow and bone) or
blood proteins: beta-2 microglobulin (ß2M) and
extramedul ary (outside of the bone). When
albumin; the levels of these proteins predict
there are multiple plasmacytomas inside or out-
overal outcome with myeloma treatment.
side bone, this condition is also cal ed multiple
myeloma.
Stage I (best outcome)
n Serum albumin 3.5 g/dl
The Stages of Multiple Myeloma
n ß2M of < 3.5 mg/l
Confronted with a diagnosis of multiple myelo-
Stage II
ma, a doctor (usual y a hematologist/oncolo-
n Serum albumin and ß
gist) must determine the stage of the disease.
2M both < 3.5 or
Disease staging wil help determine which
n Serum ß2M between 3.5 and 5.5 mg/l
parts of the body have been af ected and how
Stage III
severely. This wil al ow the doctor to decide
n Has more elevated serum ß2M of 5.5mg/l
upon the best treatment options.
Multiple myeloma is a serious cancer, but it is
Stage I (low cell mass): Early disease. The
very treatable. Many patients experience a
bone structure appears normal or close to nor-
series of responses, relapses, and remissions.
mal on x-ray images; the number of red blood
New treatments may extend the average sur-
cel s and amount of calcium in the blood are
vival of 5 years or more for patients diagnosed
normal or close to normal; and the amount of
with multiple myeloma. Patients with myeloma
M protein is very low.
can live over 10 years; some live over 20 years.
6
7
circulation back into the bone marrow where
Background Rationale for Use of
they divide and grow to repopulate the
High-Dose Chemotherapy and Blood
normal bone marrow space. Approximately
Stem Cell Transplant or Rescue
3648 hours after administering the melpha-
n Myeloma cel s and normal blood stem
lan, the blood and tissue levels of melphalan
cel s are in the same bone marrow micro-
are very low and do not harm the new stem
environment. As myeloma cel s build up in
cel growth. This whole process of harvest
the bone marrow, they become intermixed
and re-infusion at the best time is cal ed
with normal blood stem cel s responsible for
"stem cel transplant."
the production of normal red and white cel s
as wel as platelets. Any drugs reaching the
Types of Stem Cell Transplant
bone marrow microenvironment can there-
n Autologous stem cel transplant. Stem cel s are
fore damage both the myeloma cel s and the
harvested from a myeloma patient fol owing
normal blood stem cel s.
initial therapy and re-infused after high-dose
melphalan therapy has been administered.
n High-dose melphalan seriously damag-
This is the most common type of stem cell
es normal stem cel s. High-dose melpha-
transplant. The procedure can be performed
lan is a very ef ective treatment against
once (single autotransplant) or twice (double
myeloma, but can also permanently damage
or tandem transplant).
normal blood stem cel s. High dosages of
melphalan can be especial y helpful in eradi-
n Syngeneic stem cel transplant. Stem cel s
cating myeloma cel s from the bone marrow.
are harvested from an identical twin. In this
To circumvent the problem of simultaneous
case, the stem cel s from the identical twin
severe damage to and potential destruction
are infused after high-dose therapy, which
of normal blood stem cel s in the bone mar-
can be melphalan or other agents.
row, techniques for col ecting and saving
normal blood stem cel s before administering
the melphalan have been developed.
n Stem cel s can be col ected (harvested) and
infused after treatment to replace those
damaged by treatment. Normal blood stem
cel s are col ected or "harvested" from the
patient or donor before administration of the
melphalan. The harvested normal blood stem
cel s are returned to the blood circulation by
a process similar to blood transfusion. By a
seeding process, the stem cel s pass from the
8
9
n Al ogeneic stem cel transplant. Stem cel s
n In general, stem cel transplant is a potential
are harvested from a family member who is
option for al myeloma patients upon comple-
not an identical twin, but is wel matched by
tion of frontline therapy. However, since trans-
tissue (HLA) typing. Again, the stem cel s are
plant is an intensive approach, patients over
infused after the high-dose therapy.
the age of 65 years and/or those with other
medical conditions may not be able to tolerate
n "Mini" or non-myeloablative al ogeneic trans-
the procedure and/or may run the risk of more
plant is a newer and safer procedure than
serious complications. If stem cel transplant is
ful al ogenic transplant. It involves the use of
considered to be a potential option, the most
reduced intensity chemotherapy in combina-
important caution is to avoid use of melphalan
tion with an al ogeneic stem cel transplant.
by mouth prior to stem cel harvesting, since this
n Matched Unrelated Donor (M.U.D.) stem cell
can lead to damage of normal bone marrow
transplant. Stem cel s are harvested from a
stem cel s. Thus, avoiding melphalan initial y
non-family member. In this case, the stem
and keeping al options open is the most com-
cel s are rarely a 100% tissue (HLA) match.
monly recommended strategy. Conversely, if
Hence the term "mismatch" is frequently used
stem cel transplant can never be an option or is
in this situation.
not preferred, for whatever reason, melphalan
pil s as a part of initial therapy can be a simple
How Stem Cell Transplant is Used as
and very ef ective treatment.
a Part of Myeloma Therapy
n Stem cel s are harvested and transplant is
n Fol owing diagnosis, several options are
performed after initial or frontline therapy.
available for initial or front-line therapy.
This means that treatment is used to achieve
Typical frontline regimens currently
response and at least some degree of remis-
utilized are:
sion before proceeding to therapy with high-
dose melphalan and blood stem cel rescue.
· Thalidomide plus dexamethasone
· Dexamethasone alone
· Various dexamethasone combinations
incorporating an anthracycline (e.g.,
Adriamycin® or Doxil® as part of VAD or
VDD), Velcade®, or more recently Revlimid®
combinations. Cytoxan® can also be used
as part of the initial approach.
Ful details of these treatments are discussed in
other publications of the International Myeloma
Foundation.
10
11
Major details include:
dose therapy for a patient who has already
· Initial therapy for 36 months with drugs
achieved VGPR or CR is under investigation.
that do not damage normal blood stem
n Enhanced response without the necessity
cel s.
of maintenance. A particular benefit of high-
· Ideal y, response is achieved with > 50%
dose therapy is that added response can
reduction in myeloma protein levels and/or
occur within a few weeks of the procedure. If
other indicators of active myeloma prior to
CR or VGPR occur, then such patients can be
the col ection of normal blood stem cel s.
fol owed and monitored without the absolute
However, even lesser degrees of response
need for ongoing maintenance anti-myeloma
may be suf icient to al ow safe and ef ective
therapy. Patients undergoing high-dose therapy
stem cel col ection to be performed.
also tend to be in remission longer and thus
to have a longer period before retreatment is
What are the Benefits of
required. Thus, the potential ongoing toxicity,
High-Dose Chemotherapy with Blood
inconvenience, and expense of maintenance
Stem Cell Rescue?
can be avoided. However, depending upon
the individual details, including chromosome
n Further improvement in the level of response
testing, maintenance therapy and/or other
achieved with frontline therapy is a major
(consolidation) therapy may be recommended
advantage of high-dose therapy with stem cell
after transplant.
transplant. Over half the time, partial responses
wil be improved to either VGPR (very good
n Potential benefit with double or tandem trans-
partial response, with 90% myeloma protein
plantation. If CR or VGPR are not achieved
reduction) or CR (complete response, with dis-
with a single autologous transplant, then a
appearance of measurable myeloma protein
second autologous (or an alternate transplant
level).
such as "mini al ogeneic" [see above]) can be
of ered. Continuing in the at empt to achieve
n Enhanced benefit in patients who have
VGPR with the second transplant does appear
already achieved VGPR or CR. With the advent
to confer benefit.
of more frequent VGPR or CR with novel front-
line therapies, the added benefit of high-dose
n Significance of achieving CR or VGPR. It has
therapy in this set ing is coming under closer
been general y accepted that patients achiev-
scrutiny. High-dose chemotherapy has con-
ing bet er response such as CR or VGPR have
ferred statistical y significant benefit fol owing
bet er outcomes (versus, for example, partial
traditional chemotherapy induction using, for
response [PR]). However, further studies are
example, VAD chemotherapy. However, novel
required. Having a durable response at a par-
therapy combinations can produce high levels
ticular level, whether that is a simple PR ( 50%
of VGPR and CR. The additional benefit of high-
improvement), VGPR ( 90%) or CR (100%), is
12
13
more important than the level of the response
STEP FOUR
in itself. Response lasting 2 years is particu-
n Review with the physician the pros and cons
larly beneficial. The relative benefit of stable
of stem cel transplant (and/or stem harvest-
disease at the PR, VGPR, or CR level is under
ing without immediate transplant).
further study.
n If 50% response (PR: 50% reduction in
myeloma protein level in blood and/or
Practical Steps in Considering Stem
urine) is achieved, stem cel harvesting can
Cell Transplant as a Treatment Option
be planned if it is agreed to proceed. If there
STEP ONE
is no plan for harvest and/or transplant, a
n
plan for ongoing maintenance or fol ow-up
Confirm the diagnosis of active myeloma that
treatment is required.
requires anti-myeloma treatment.
n If there is < 50% response, then other therapy
n If there is any doubt about the diagnosis or
may be required before proceeding to trans-
approach to treatment, it is an important
plant.
time to seek a second opinion before going
ahead with a frontline strategy.
"Questions and Answers" about stem cel trans-
plant as wel as "Questions to Ask the Doctor"
STEP TWO
about the potential procedure are listed later in
n Proceed with initial or frontline therapy
the brochure.
to bring the myeloma under control and
achieve an initial response.
How Stem Cells are Collected
n Make sure to avoid melphalan or other
Blood stem cel s are located in the bone mar-
therapy that may reduce the success of nor-
row. Until about 20 years ago, the only way to
mal blood stem cel harvesting. Radiation
col ect these stem cel s was to have the patient
therapy to the pelvis, for example, can
or donor receive a general anesthetic and
reduce stem cel reserves and should be
avoided if possible.
STEP THREE
n Assess the response to treatment with each
cycle of therapy (usual y every 34 weeks).
n After 34 cycles of treatment, more complete
re-evaluation is recommended, including
bone marrow testing plus x-ray/scans as
needed to determine the level of response.
14
15
undergo as many as 50100 bone marrow
or 5th day after starting the injections. The col-
aspirations from the back of the pelvic bone to
lections and injections wil continue daily until
remove enough bone marrow and stem cel s
suf icient stem cel s are obtained.
to use for future transplant. This was obviously
2. Using chemotherapy plus growth factors.
painful, frightening, and inconvenient. The dis-
Chemotherapy with growth factors may also be
covery that stem cel s could be col ected from
used to release stem cel s from the bone marrow
the bloodstream by giving a patient or donor
into the bloodstream. The doctor wil explain
injections of stem cel growth factors such as
why it may or may not be appropriate to use
Neupogen®, Neulasta®, or Leukine® to trigger
chemotherapy in addition to growth factors.
the release of bone marrow stem cel s into the
The doctor wil explain the chemotherapy that
bloodstream was a major breakthrough. With
wil be administered to mobilize the blood stem
refinements over the years, this has become the
cel s and its potential benefits and side ef ects.
standard method. It is rarely necessary to use
Fol owing chemotherapy for stem cel mobiliza-
the old method of direct bone marrow harvest-
tion, a white cel growth factor is given by injec-
ing from the pelvic bone.
tion under the skin daily for approximately ten
Methods of Col ecting Stem Cel s from the
days. This procedure is therefore longer and
Blood Stream (Peripheral Blood Stem Cel s
much more intensive than using growth factors
[PBSC])
alone. The patient or someone who agrees to
There are two main methods for col ecting
be responsible may be taught how to give the
stem cel s: 1) giving growth factors alone or
growth factor injection so that it can be admin-
2) giving growth factors with chemotherapy.
istered at home. Some patients may receive
their injections at the clinic/hospital or from
1. Growth factors alone.
visiting nurses. Once the number of stem cel s
Growth factors are drugs that stimulate blood
in the blood stream is high enough, they wil be
stem cel s both to grow and to be released
col ected over 25 days, while the patient is still
into the blood stream. There are red cel and
receiving the growth factor injections.
white cel growth factors. These medications
are administered subcutaneously (under the
skin). Growth factors are often used for patients
receiving chemotherapy to hasten their white
and red cel count recovery. The white cell
growth factors (Neupogen, Neulasta, Leukine)
used in high doses stimulate the release of stem
cel s from the bone marrow into the blood-
stream. This process is cal ed "mobilization."
The injections are given daily for three or more
days. Stem cel s are usual y col ected on the 4th
16
17
The Col ection or Harvesting Procedure
The most common side ef ects experienced dur-
In medical language, the harvesting is cal ed
ing apheresis are slight dizziness and tingling
apheresis or leukapheresis literal y the
sensations in the hands and feet. Less common
removal of white cel s from the blood stream.
side ef ects include chil s, tremors, and muscle
Apherisis is a procedure whereby blood from
cramps. These side ef ects are temporary and
the patient or donor passes through a special
are caused by changes in the volume of the
machine that separates (using a centrifuge
patient's blood as it circulates in and out of
technique) and then removes stem cel s. The
the apheresis machine, as wel as by blood
rest of the blood is immediately returned to the
thinners added to keep the blood from clot ing
patient or donor. Compared to direct bone mar-
during apheresis.
row harvesting, this is a remarkably simple and
Processing stem cel s: After col ection, the periph-
pain-free procedure.
eral blood (or occasional y direct bone marrow
Apheresis/Leukapheresis: Prior to the start of
material) is taken to the processing laboratory,
apheresis, a thin flexible plastic tube cal ed a
which is usual y located within the hospital or
catheter is inserted through the skin and into a
local blood bank. In the processing laboratory,
vein so that blood can be taken out. The cath-
the bone marrow or blood cel s are prepared
eter is usual y inserted into the chest just below
for freezing (cryopreservation). The stem cel s
the col arbone. Insertion of the catheter is usu-
are mixed with a solution containing the chemi-
al y done as an outpatient procedure, and only
cal DMSO (dimethyl sulfoxide) to prepare the
a local anesthetic is necessary. The site where
stem cel s for freezing. The stem cel s are then
the catheter enters the skin may be sore for a
frozen and stored in liquid nitrogen. The stem
few days; the discomfort may be relieved with
cel s wil be frozen until the time they wil be
medications like acetaminophen (Tylenol®). The
needed for the transplant. They can be stored
catheter may be kept in place for several weeks
frozen for as long as necessary. There is some
because it can be used to give chemotherapy
deterioration with time, but excel ent function of
after stem cel s have been col ected. Sometimes
stem cel s is retained for at least 10 years.
the same catheter is used during the transplant
procedure as wel . During this procedure,
How many stem cel s do I need? Over the years,
blood is col ected through the catheter and
a number of studies have been completed to
processed through a blood-processing machine
determine the number of stem cel s you need to
to remove the stem cel s. The rest of the blood
safely undergo high-dose therapy. The number
is returned through part of the same catheter
of stem cel s is quantified by a special labora-
(the lumen not being used in a double lumen
tory technique cal ed "CD34+ cel analysis by
catheter) or by using a dif erent catheter. The
flow cytometry." A smal sample of the stem cell
apheresis procedure wil last 34 hours each
col ection is tested for the number of CD34+
day for 1 to 5 days. Apheresis is usual y done
cel s in the product. We know that a minimum
as an outpatient procedure.
number of stem cel s to safely complete a trans-
18
19
plant is 2 mil ion CD34+ cel s per kilogram
chemotherapy is to kil myeloma cel s inside
of body weight. The number of CD34+ cel s
the patient's body. The most common type of
is checked in each daily col ection and the
high-dose chemotherapy used to treat myeloma
number tal ied. The stem cel col ection process
is melphalan administered at a dose of 200
continues daily until the planned number of
mil igrams per square meter (mg/m2) of body
stem cel s is col ected usual y 14 days. Some
surface area (size of patient). Depending on
transplant centers check the number of CD34+
the type of myeloma and other factors, some
cel s BEFORE starting leukapheresis to make
patients may receive a second transplant 3 to
certain there wil be a good col ection that day.
6 months after the first transplant (double or
Most transplant physicians col ect enough stem
tandem transplant). A patient should discuss
cel s for two transplants (over 4 mil ion CD34+
with the doctor the pros and cons of more than
cel s per kilogram body weight). In situations
one transplant planned and performed back-
where a suf icient number of blood stem cel s
to-back versus the possibility that the cel s will
cannot be harvested, patients may qualify for
be stored for a potential second transplant at
a compassionate use program of AMD-3100
a later time.
(Mozobil®), an experimental drug that boosts
Autologous Stem Cel Transplant or
stem cel production.
Infusion
Administering High-Dose
Since high-dose treatment destroys the normal
Chemotherapy
bone marrow in addition to the myeloma cel s,
the blood stem cel s must be given back to
After the stem cel s are frozen and stored, the
restore the bone marrow. The previously col ect-
patient is ready to receive high-dose chemo-
ed stem cel s wil be unfrozen and given back,
therapy. This treatment is designed to destroy
through a catheter, into the bloodstream (as
myeloma cel s more ef ectively than standard-
one would receive a blood transfusion) one to
dose chemotherapy. The purpose of high-dose
two days after administration of the high-dose
chemotherapy. This procedure is often referred
to as the transplant. The transplant takes place
in the patient's room: it is not a surgical pro-
cedure. The frozen bags of bone marrow or
blood cel s are thawed in a warm water bath,
and then injected into the bloodstream through
the catheter. Upon thawing, the DMSO (freez-
ing agent) evaporates into the air and creates
a distinct and somewhat unpleasant garlic
smel . Most centers infuse one bag at a time. It
usual y takes 14 hours for the infusion. Infused
20
21
stem cel s travel through the bloodstream, and
Preventing Infection
eventual y, to the bone marrow, where they
begin to produce new white blood cel s, red
During the first 23 weeks after transplantation,
blood cel s, and platelets. It takes 1014 days
the re-infused stem cel s migrate to the bone
for the newly produced blood cel s to enter the
marrow and begin the process of producing
bloodstream in substantial numbers. Growth
replacement blood cel s, a process cal ed
factors may again be given to the patient to
engraftment. Until engraftment of the stem cel s
speed up this process.
takes place, patients are very susceptible to
developing infections. Even a minor infection
In addition to obliterating the bone marrow,
like the common cold can lead to serious prob-
high-dose chemotherapy can cause other
lems because the body's immune system is so
severe side ef ects, which may require that
weakened by the ef ects of the high-dose che-
some patients be admit ed to the hospital for
motherapy. Therefore, special precautions are
treatment during this period. (Not al transplant
necessary during recovery. Since the patient's
centers require that patients remain in the
immune system is very weak, patients may
hospital after the infusion of stem cel s; some
remain in the hospital until the white blood cell
have facilities close by where patients may stay
counts reach a level safe enough for the patient
and be monitored daily at the hospital on an
to be discharged.
out-patient basis, while others al ow patients
To prevent infection, the fol owing supportive
who live close to the hospital to sleep at home
care measures may be required:
and be monitored at the hospital). The average
time in the hospital (or a nearby facility) for
· Antibiotics are often prescribed to help pre-
vent infection.
the chemotherapy, transplant, and recovery is
approximately 3 weeks. Shortly before starting
· Visitors should wash their hands and may be
chemotherapy, patients usual y are given large
asked to wear masks and rubber gloves to
amounts of fluid to prevent dehydration and
protect the patient.
kidney damage from the chemotherapy. Some
· Fresh fruits, vegetables, and flowers may be
of the more common side ef ects of chemother-
prohibited from the patient's room as these
apy include nausea, vomiting, diarrhea, mouth
can carry infection (bacteria and fungi).
sores, skin rashes, hair loss, fever or chil s, and
· If infection and/or fever occurs (as the result
infection. Medications designed to prevent or
of lowered white cel counts), the patient may
lessen some of the expected side ef ects of
be admit ed to the hospital and be given
treatment are given routinely. Patients are very
intravenous antibiotics.
closely monitored during and after the adminis-
tration of high-dose chemotherapy. Monitoring
Engraftment and Recovery
includes daily weight measurement as wel as
Once the stem cel s have been re-infused, it
frequent measurements of blood pressure, heart
wil take about two weeks for blood counts to
rate, and temperature.
22
23
recover. Many transplant centers wil again use
Am I a Candidate for an Autologous
white blood cel growth factors (Neupogen,
Transplant?
Neulasta, Leukine) after the transplant to help
stimulate the bone marrow to produce normal
A stem cel transplant is a treatment option for
blood cel s. These injections (under the skin) will
many myeloma patients; however, it is not a
continue until the white blood count returns to
cure. It can improve the duration of remission
normal. During this time, red blood cel and/or
and survival. It can also provide a bet er qual-
platelet transfusions may be necessary.
ity of life for most patients. Not al patients
Waiting for the transplanted stem cel s to
with myeloma are candidates for a stem cell
engraft, for blood counts to return to safe levels,
transplant. Many factors must be taken into
and for side ef ects to disappear is often the
consideration. These include factors related to
most dif icult time for both the patient and his
the myeloma itself and patient-related factors.
or her family and friends. During this period
MyElOMa-RElaTEd FacTORS
patients wil feel weak and very fatigued.
· type of myeloma
Having a support network is very important
· disease stage
during this period. Recovery can be like a rol er
coaster ride: one day a patient may feel much
· disease aggressiveness
bet er, only to awake the next day feeling as
· responsiveness to treatment
sick as ever. It is important during this period to
· serum albumin
take things one day at a time. Once new blood
· beta-2 microglobulin
cel s are being made, symptoms wil resolve, the
· chromosome analysis
risk of serious infections wil be reduced, and
PaTiENT-RElaTEd FacTORS
transfusions wil no longer be needed.
· age
After being discharged from the hospital, a
· health status
patient continues recovery at home for two to
· kidney, heart, lung, and liver function
four months. Although patients may be well
· patient preference
enough to leave the hospital, their recovery
wil be far from over. For the first several weeks
We cannot stress enough that myeloma is a
the patient may be too weak to do much more
highly individualized disease. While there are
than sleep, sit up, and walk a lit le around the
similarities between patients, each case has its
house. Frequent visits to the hospital wil be
own distinct characteristics. There wil be testing
required to monitor progress. Patients usual y
to determine how much myeloma there is in
cannot resume normal activities or return to ful -
your body and how aggressive it is. Al of these
time work for up to three to six months after the
variables wil be weighed before determining
transplant, although this varies from individual
whether a transplant is appropriate for you.
to individual.
Therefore, general statements regarding patient
24
25
outcomes both during the transplant procedure
by infusion of the other half of the stored stem
and post transplant are inappropriate.
cel s. Preliminary data indicates that tandem
When to transplant is also an important con-
transplants result in improved disease control
sideration. Most transplant physicians believe
and survival in patients who do not achieve
it is bet er to perform the transplant early in the
either VGPR or CR fol owing first autologous
disease course. However, there is no absolute
transplant.
clinical data to suggest that transplantation
Radiopharmaceuticals (radioactive bone-target-
earlier in the treatment regimen is bet er than
ed therapy) are combined with high-dose che-
waiting until later. Remember, in most cases,
motherapy and autologous stem cel transplant
unlike a heart at ack, myeloma gives the patient
as a means to increase response rates. This
the luxury of time to do some homework and
approach al ows for a two-pronged at ack on
to gather the information needed to make an
the myeloma through high-dose chemotherapy
informed decision about what's right for him
plus a radioactive compound that only at acks
or her. For example, one could have stem cel s
the bone marrow. There is currently a radio-
harvested and saved for a later treatment. This
pharmaceutical agent in myeloma clinical trials:
leaves the patient open to other more imme-
Quadramet (samarium Sm-153 lexidronam).
diate treatment options. These are things to
A "mini" (non-myeloablative) al ogeneic trans-
discuss with the doctor. It's important to remem-
plant involves the use of mild therapy (chemo-
ber that even if someone is a good transplant
therapy and/or radiation therapy) to suppress
candidate, the ultimate decision about whether
the patient's immune system to al ow for donor
or not to have a transplant is the patient's.
stem cel s to grow. This dose of chemotherapy
Transplants and Clinical Trials
does not destroy the bone marrow but does
al ow for the donor's blood cel s and immune
A single autologous stem cel transplant is
system to grow. After the lowered dose of
currently considered the standard of care for
chemotherapy is administered, the patient
patients with multiple myeloma. However, there
receives the donor's stem cel s. Once the al o-
are a number of novel approaches that are
geneic stem cel s grow, the donor's immune cel s
being evaluated to try to improve patient out-
at ack the myeloma. This is a form of immunother-
comes. These are being conducted as clinical
apy. The risk of this procedure is that the donor's
trials. These include the fol owing:
immune system wil "overreact" and at ack more
A tandem autologous transplant is an approach
than the myeloma cel s. This reaction is cal ed
that utilizes two autologous transplants.
"graft-versus-host disease," which can be very
Suf icient stem cel s are col ected prior to the
serious and potential y life threatening.
first transplant. Three to six months after the
Sequential autologous transplant fol owed by
first transplant, the patient receives a second
a mini al ogeneic transplant. Pilot studies using
similar course of high-dose therapy fol owed
sequential transplants have shown promise.
26
27
This involves high-dose chemotherapy with an
Questions and Answers
autologous transplant to destroy the majority of
about Stem Cell Transplantation
the myeloma cel s, fol owed 2 to 4 months later
by an al ogeneic mini-transplant to al ow the
Listed below are some of the questions frequent-
donor's immune cel s to destroy any remaining
ly asked by people with myeloma who have
myeloma cel s. As with a single mini al ogeneic
had or are considering a stem cel transplant.
transplant, there is a risk of graft-versus-host dis-
These questions and other concerns should be
ease, which can be very serious and potential y
discussed with the doctor and members of the
life threatening.
healthcare team before making any final deci-
sions about the patient's treatment plan.
Maintenance therapy is an approach that
involves lowered doses of anti-myeloma drugs
Q. Why is a stem cel transplant necessary for
to maintain longer remission and survival after
a multiple myeloma patient?
a transplant. Currently, some of the drugs
A. The transplant procedure al ows the patient to
being evaluated include thalidomide, Revlimid,
receive high doses of chemotherapy to kil more
prednisone, and dexamethasone, alone or in
myeloma cel s. This therapy is so potent that it
combinations.
destroys al of the bone marrow. Without bone
marrow, the body is unable to manufacture
Psychosocial Issues
blood cel s needed to carry oxygen, help blood
High-dose chemotherapy and autologous
clot, and defend against infection. Therefore,
transplantation can place an enormous stress
a stem cel transplant replaces the destroyed
on patients and families. Physical, psycho-
marrow, rescuing the patient from the ef ects of
logical, emotional, and financial stresses can
high-dose chemotherapy.
be overwhelming. Patients and families may
Q. Am I a candidate for bone marrow or
experience feelings of anger, depression, and
peripheral blood stem cel transplant?
anxiety over an unknown future and a lack
A. Medical experts have yet to arrive at a set of
of control. Support services of ered through
fixed guidelines for selecting patients who will
the hospital and many other organizations,
benefit the most from a transplant. Increasingly
including myeloma support groups, are very
accepted as a part of multiple myeloma
important during this time. We urge you to
treatment protocols, successful transplantation is
take advantage of these services, or to seek a
a function of the patient's age, general physical
referral from your oncologist for psychological
condition, stage of disease, and responsiveness
counseling and/or a psychiatric consultation.
to prior treatments. Only the patient's physician
can provide a patient with the best assessment
of his or her chances for long-term survival.
28
29
Q. Does taking alkylating agents such as
nurses, and other members of the multiple
melphalan, busulfan, and cyclophospha-
myeloma treatment team and learn more
mide (Cytoxan) reduce my suitability for a
about how they approach a transplant. See
transplant?
the room where your transplant wil occur and
A. Alkylating agents are one of the most ef ec-
where you'l be spending your recuperation
tive ways of kil ing myeloma cel s inside the
time. Find out what part of your procedure will
body. However, their prolonged use more
be performed in a clinic or a doctor's of ice
than 4 to 6 months wil reduce the ability to
and what part wil be done in the hospital. You
easily harvest a patient's stem cel s. Therefore,
should be comfortable with the center before
when considering a transplant, a patient should
you begin your transplant.
first discuss the total treatment plan to ensure
Q. If my doctor agrees that a stem cel trans-
that there are as many short-term and long-
plant is an appropriate treatment for my dis-
term treatment options available as possible.
ease, what can I do now to prepare for the
It should be emphasized, however, that col ec-
experience?
tion should ideal y be done before using any
A. The patient can do a lot to get ready for
alkylating agents.
the transplant. By reading this brochure, a
Q. How do I select a transplant center?
patient has already taken the most important
A. A transplant is a complicated medical
step: learning as much as possible about the
procedure that requires an expert team of
procedure. A patient should speak with the
doctors, nurses, social workers, psychologists,
doctor, seek out fel ow survivors, and read as
and al ied health professionals who understand
much as possible, including the publications
the procedure, have performed it success-
and newslet ers from the International Myeloma
ful y many times, and are equipped to respond
Foundation. Patients should ask questions about
when medical and emotional problems arise.
what they've learned and strive to read al the
Today, medical centers that meet these criteria
newest information coming from research. We
can be found throughout the country. Many
suggest that patients bring a tape recorder or
of these centers specialize in treating patients
a friend along to the doctor's of ice so that they
with many dif erent types of cancer. To find the
can give ful at ention to the doctor. Patients
one best suited for patients with multiple myelo-
should share what they know with family and
ma, you should talk with your doctor, other
loved ones so that they wil know what to
multiple myeloma patients, and the International
expect and how they can help in the weeks
Myeloma Foundation.
and months ahead.
Q. What goes on at a transplant center?
The doctor wil perform a series of tests to
A. To understand what goes on at a transplant
confirm that the patient is wel enough to toler-
center, we strongly suggest a visit to one or
ate the transplant. Al the data gathered on
more centers. Meet with the staf doctors,
the performance of the heart, lungs, kidneys,
30
31
and other vital organs wil enable the doctor to
riskier than most. Nonetheless, medical studies
compare the patient's health before and after
have shown that over 95% of patients (usual y
the procedure. In most cases, the patient won't
closer to 99%) survive transplant.
have to be hospitalized for these tests since they
can be performed in the doctor's of ice.
Q. Can the patient relapse after a transplant?
A. Yes. Unfortunately, the majority (at least 50%)
Q. What side effects should I anticipate from
of al multiple myeloma patients relapse 18 to
the transplant?
36 months after their transplant is completed.
A. Side ef ects can be expected from every
type of medical treatment, even the use of
Q. I've heard a lot about myeloma purging.
aspirin. Each patient reacts dif erently to che-
Can it help?
motherapy and other drugs given during the
A. The process of purging removes myeloma
transplant. No two patients share exactly the
cel s from peripheral blood taken from the
same side ef ect profile.
patient's body prior to transplant. High-dose
chemotherapy is used to kil myeloma cel s
Patients should therefore seek a transplant
that are within the body. Stem cel selection,
center where the doctors, nurses, and al ied
or "purging," is used to remove myeloma cel s
health professionals have performed a number
from the col ected stem cel s prior to the trans-
of transplants and appear competent to care
plant. The goal of this strategy is to reduce the
for each individual myeloma patient's needs.
number of myeloma cel s both within the
Q. What happens during re-infusion?
patient's body as wel as in the peripheral
blood that wil be re-infused into the patient.
A. After chemotherapy the patient receives a
Recent evidence indicates that this technology
re-infusion of his or her own stem cel s. The
is not ef ective in myeloma. Therefore, very
stem cel s wil be thawed and infused into the
few centers currently use stem cel purging for
patient's catheter either through a syringe or
myeloma patients.
from an intravenous infusion bag. While the
re-infusion takes place the patient may feel
Q. How long wil the transplant patient stay in
warm or lightheaded. The chemical used to
the hospital?
keep the stem cel s fresh has a garlic smel that
A. Patients stay in the hospital for about 2 to 3
the patient might be able to taste. The oncolo-
weeks. The length of stay varies from patient to
gist may re-prescribe or adjust the patient's
patient. Some patients may have several short
medication to make him or her feel more com-
admissions.
fortable during this procedure.
Q. When wil the stem cel s start to grow
Q. Can a patient die from the transplant itself?
again?
A. Every medical procedure carries risk, and
A. Stem cel s start to grow back or "engraft"
a transplant for multiple myeloma patients is
within 1014 days after re-infusion.
32
33
Q. What wil the patient's quality of life be after
of their treatment program. Because al drugs,
transplant?
synthetic and natural, interact, and may cre-
A. On average, patients take 3 to 6 months
ate unanticipated side ef ects, patients should
to recover from a transplant. By this time, the
always consult their doctors about their use.
immune system wil once again fight infections
The doctor should be informed of the names
because the bone marrow is producing healthy
of al the alternative and complementary thera-
blood cel s. Hair wil grow back, but the taste
pies being taken so that he or she can adjust
buds might stil be a lit le quirky. Foods that
the regimen accordingly. It is important to note
tasted good before a transplant might not taste
that even seemingly innocuous over-the counter
good now. However, in most cases, patients
drugs, e.g., ibuprofen, may be harmful to a
should be able to return to normal daily activi-
myeloma patient.
ties. It can take as long as a year to recover
normal functioning. Patients and their caregiv-
Questions for the Doctor
ers must take one day at a time. There will
These are questions we suggest be discussed
be bad days and good days, and they won't
with the doctor to provide bet er understanding
necessarily come in that order. Patients should
of the transplant procedure and its ef ects on
prepare themselves to feel dif erently each day
the patient's life. Space is provided for notes:
during the recovery process.
"Am I a candidate for stem cel transplant?"
Q. Should transplant patients expect changes in
their emotions?
________________________________________
A. Yes. Transplant is more than just a medical
________________________________________
procedure. Because it forces the patient to
"What does high-dose chemotherapy with trans-
rely upon the oncologist and other members
plant hope to achieve that can't be achieved by
of the transplant team, as wel as on family
standard chemotherapy?"
and friends, there is often a loss of the sense
of independence and control. Feelings of iso-
________________________________________
lation, depression, and helplessness are com-
________________________________________
mon to transplant patients. Patients and loved
ones should seek assistance from a trained
________________________________________
professional who has experience in counsel-
"What treatment protocols are there at your
ing. Help may also be found through patient
institution and how do you decide which one is
support groups.
right for me?"
Q. What alternative and complementary thera-
________________________________________
pies can be taken during and after transplant?
A. Some patients believe that alternative and
________________________________________
complementary therapies are an important part
________________________________________
34
35
"Does taking alkylating agents such as melpha-
"Wil you be the doctor who provides my
lan, busulfan, and Cytoxan reduce my suitability
ongoing care?"
for a transplant?"
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
"What goes on at a transplant center?"
"How do I select a transplant center?"
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
"If we decide that transplant is an appropriate
"How many transplants has this center
treatment for my disease, what can I do now to
performed for multiple myeloma and what are
prepare for the procedure?"
the success rates?"
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
"When does the transplant procedure begin?"
"How long do patients transplanted in your cen-
________________________________________
ter live after the transplant itself? How does this
compare with national averages?"
________________________________________
________________________________________
"What drugs wil be prescribed for use before,
during, and after the transplant? What do they
________________________________________
do and what are their side effects?"
________________________________________
________________________________________
"Wil you be the doctor who performs the trans-
________________________________________
plant and who are the other members of the
team?"
________________________________________
________________________________________
"How long is the entire treatment cycle, from
preparation for the transplant to recovery?"
________________________________________
________________________________________
________________________________________
________________________________________
36
37
"How long wil I have to be in the hospital? How
About the IMF
often are my fol ow-up visits going to be?"
"One person can make a dif erence,
________________________________________
Two can make a miracle."
________________________________________
Brian D. Novis
________________________________________
IMF Founder
"How wil the transplant procedure affect my
Myeloma is a lit le-known, complex, and often
ability to function? How can I expect to feel dur-
misdiagnosed bone marrow cancer that at acks
ing and after the transplant?"
and destroys bone. Myeloma af ects approxi-
mately 75,000 to 100,000 people in the
________________________________________
United States, with more than 19,000 new
________________________________________
cases diagnosed each year. Although there
is presently no known cure for myeloma, doc-
________________________________________
tors have many approaches to help myeloma
________________________________________
patients live bet er and longer.
"What side effects of my transplant should I
The International Myeloma Foundation (IMF)
anticipate?"
was founded in 1990 by Brian and Susie
________________________________________
Novis shortly after Brian's myeloma diagnosis
at the age of 33. It was Brian's dream that
________________________________________
future patients would have easy access to
________________________________________
medical information and emotional support
throughout their bat le with myeloma. He estab-
________________________________________
lished the IMF with the three goals of treatment,
"What are the risks of the transplant procedure?
education, and research. He sought to provide
Is there a high survival rate for high-dose therapy
a broad spectrum of services for patients and,
with stem cel transplant?
their families, friends, and health- care provid-
ers. Although Brian died four years after his
________________________________________
initial diagnosis, his dream did not. Today, the
________________________________________
IMF reaches out to an international membership
of more than 145,000. The IMF was the first
________________________________________
organization dedicated solely to myeloma, and
________________________________________
today it remains the largest.
The IMF provides programs and services to aid
in the research, diagnosis, treatment, and man-
agement of myeloma. The IMF ensures that no
one must brave the myeloma bat le alone.
38
39
We care for patients today, while working
SUPPORT
toward tomorrow's cure.
MyElOMa HOTliNE: 800-452-cURE (2873)
Toll-free throughout the United States and
How Can the IMF Help You?
Canada, the IMF Hotline is staf ed by trained
PaTiENT EdUcaTiON
information specialists and is in frequent
interaction with members of our Scientific
iNFORMaTiON PackaGE
Advisory Board.
Our free IMF InfoPackTM provides comprehensive
information about myeloma, treatment options,
SUPPORT GROUPS
disease management, and IMF services. It
A worldwide network of more than 100
includes our acclaimed Patient Handbook.
myeloma support groups hold regular meetings
for members of the myeloma community. The
iNTERNET accESS
IMF conducts annual retreats for leaders of
Log on to www.myeloma.org for 24-hour
myeloma support group leaders.
access to information about myeloma, the IMF,
education, and support programs.
RESEaRcH
ONliNE MyElOMa FORUM
BaNk ON a cURE®
Join the IMF Internet Discussion Group at
This DNA bank wil provides genetic data
www.myeloma.org/listserve.html to share your
research in new drug development.
thoughts and experiences.
THE iNTERNaTiONal STaGiNG SySTEM (iSS)
MyElOMa MiNUTETM
This updated staging system for myeloma
Subscribe to this free weekly email news-
enhances physicians' ability to select the most
let er for up-to-the-minute information about
appropriate treatment for each patient.
myeloma.
RESEaRcH GRaNTS
iMF PaTiENT & FaMily SEMiNaRSTM
Leading the world in col aborative research
Meet with leading experts in myeloma treat-
and achieving extraordinary results, the IMF
ment to learn more about recent advances in
Grant Program supports both junior and senior
therapy and research.
researchers working on a broad spectrum of
projects. The IMF has at racted many young
MyElOMa MaTRixTM
investigators into the field of myeloma; they
On our website and in print, this document is a
have remained in the field and are actively
comprehensive guide to drugs in development
for myeloma.
pursuing a cure for this disease.
MyElOMa TOdayTM NEWSlETTER
Our quarterly newslet er is available free of
charge by subscription.
40
41
Glossary
Autologous (autograft) stem cel transplantation: Refers to
stem cel s that are col ected from the patient and are
Alkylating agent: A chemotherapeutic agent such as
given back to the same patient after he or she receives
melphalan (Alkeran) or cyclophosphamide (Cytoxan).
high-dose chemotherapy. Most stem cel transplants in
Alkylating refers to the way in which these agents cross-
myeloma are autologous transplants.
link the DNA of myeloma cel s and block cel division.
Beta-2 microglobulin: A smal protein found in the blood.
Al ogeneic (al ograft) stem cel transplant: Refers to stem cel s
High levels occur in patients with active myeloma. Low or
that are taken from a donor and given to the patient.
normal levels occur in patients with early myeloma and/
Most al ogeneic stem cel transplants are performed
or inactive disease. Approximately 10% of patients have
using donor peripheral blood stem cel s. Conventional
myeloma that does not produce beta-2 microglobulin.
al ogeneic transplants for myeloma patients are rarely
For these patients, beta-2 microglobulin testing cannot
performed in the US due to excessive risk to the patient.
be used to monitor the disease. At the time of relapse,
A newer and, for myeloma, safer technique (discussed
beta-2 microglobulin can increase before there is any
below) is a non-myeloablative or "mini-transplant" that is
change in the myeloma protein level. Therefore, 90%
currently being evaluated in clinical trials. To determine
of the time, beta-2 testing is very useful for determining
if a patient has a potential donor match, a special blood
disease activity.
test cal HLA testing is done. A donor may be a family
Blood stem cel s:
member or may be obtained through a donor registry
Stem cel s, derived from the blood, that
such as the National Marrow Donor Program (NMDP).
result in faster hematologic recovery.
Rarely, donor cel s may be obtained from an umbilical
Bone marrow aspiration: The removal, by needle, of a
cord blood bank.
sample of tissue from the bone marrow.
Anemia: A decrease in the normal number of red blood
Bone marrow transplant: Obtained from the bone marrow
cel s, usual y below 10 g/dl with over 1314 g/dl being
of the patient or the donor. Very few bone marrow trans-
normal. Myeloma in the bone marrow blocks red cell
plants are currently performed due to the availability of
production, thus causing anemia, the symptoms of which
peripheral blood stem cel s. Rarely, bone marrow may
are shortness of breath, weakness, and tiredness.
be col ected for patients who are unable to col ect suf-
ficient number of stem cel s from the peripheral blood.
Apheresis/Leukapheresis: Sometimes cal ed leukapheresis,
apheresis is a procedure in which blood is taken from a
CD34+: This is the laboratory marker used to single out
patient or donor and the portion of the blood containing
and quantify the number of stem cel s in your blood
plasma, white blood cel s, and platelets is separated.
stream. There is a certain minimum number of CD34+
Red blood cel s are transfused back into the donor. The
stem cel s that are required to safely support a transplant
portion containing white blood cel s contains the rare
procedure.
stem cel s.
Chemotherapy: Drugs that are used to kil cancer cel s.
Autologous peripheral blood transplantation: (see Autologous
Creatinine: A smal chemical compound normal y excret-
Stem Cel Transplantation) A procedure in which a
ed by the kidney. If the kidneys are damaged, the serum
patient's blood is col ected by apheresis, stored, and
level of creatinine builds up, resulting in elevated serum
re-infused fol owing high-dose chemotherapy.
creatinine. The serum creatinine test is used to measure
kidney function.
42
43
Colony-stimulating factor (CSF): Proteins that stimulate the
Monoclonal protein (M protein): An abnormal protein pro-
development and growth of blood cel s. Neupogen,
duced by myeloma cel s which accumulates in and dam-
Neulasta, and Leukine are colony stimulating factors
ages bone marrow. A high level of M-protein indicates
that are used to mobilize stem cel s from the bone mar-
that myeloma cel s are present in large numbers.
row into the bloodstream prior to apheresis. These may
also be used after the transplant to hasten blood count
Myeloablation: The kil ing of bone marrow by radiation or
recovery.
chemotherapy. This term usual y refers to the complete or
near-complete destruction of the bone marrow.
Complete response (CR): CR is the absence of myeloma
protein from the serum and/or urine by standard testing;
Multiple myeloma: A cancer arising from the plasma cel s
absence of myeloma cel s from the bone marrow and/
in the bone marrow. The plasma cel s in patients with
or other areas of myeloma involvement; clinical remis-
multiple myeloma form abnormal antibodies, possibly
sion and improvement of other laboratory parameters to
damaging the bone, bone marrow, and other organs.
normal. CR is not the same thing as a cure.
Peripheral blood stem cel (PBSC): Stem cel s col ected from
Engraftment: The process by which stem cel s in the trans-
the blood. These cel s are similar to stem cel s found in
planted bone marrow or peripheral blood migrate to the
the bone marrow. The term "peripheral" means that the
patient's bone marrow and begin to grow and produce
cel s come from blood outside of the marrow.
new white blood cel s, red blood cel s, and platelets.
Peripheral blood stem cel (PBSC) transplant: Obtained from
Growth factors: Drugs that stimulate blood stem cel s both
the patient or donor bloodstream. Using PBSC for trans-
to grow and to be released into the bloodstream.
plantation al ows for easier and safer col ection of stem
cel s and faster recovery after the transplant than bone
Immune system: The function of a number of related body
marrow transplant.
organs that protect the body from disease organisms,
other foreign bodies, and cancers.
Plasma cel : A type of white blood cel that produces
antibodies.
Immunoglobulin: A protein produced by plasma cel s (a
type of white blood cel ) which helps fight infection. Also
Plasmacytoma: A tumor made up of cancerous plasma
known as an antibody.
cel s.
International Staging System (ISS): the most current stag-
Platelets: Granule-containing cel ular fragments critical
ing system for myeloma, the ISS is the result of the
for blood clot ing and sealing of wounds. Platelets also
col aboration of more than twenty research institutions
contribute to the immune response.
world-wide.
Red blood cel : A blood cel that carries oxygen from the
Lytic bone lesions: Holes in the bone.
lungs throughout the body.
M protein (M spike): Antibodies or parts of antibodies
Remission or response: Remission and response are used
found in unusual y large amounts in the blood or urine
interchangeably. Complete Remission (CR) is the com-
of myeloma patients. M spike refers to the sharp pat ern
mon abbreviation for both. CR is defined as the absence
that occurs on protein electrophoresis when an M protein
of myeloma protein from serum and/or urine by stan-
is present. Synonymous with monoclonal protein and
dard testing, absence of myeloma cel s from the bone
myeloma protein.
marrow and/or other areas of myeloma involvement,
clinical remission, and improvement of other laboratory
parameters to normal.
44
45
Stem cel (hematopoietic stem cel ): Normal (hematopoietic,
Bibliography
or blood-making) stem cel s give rise to normal blood
components, including red cel s, white cel s, and plate-
1. At al M, Harrousseau JL, Stoppa AM, et al. A pro-
lets. These stem cel s are normal y located in the bone
spective, randomized trial of autologous bone marrow
marrow and can be harvested for a transplant.
transplantation and chemotherapy in multiple myeloma.
Intergroupe Francais du Myelome. N Engl J Med.
Stem cel selection: A cel processing technology that is
1996;335:91-97.
used to obtain a stem cel enriched product and thereby
2. Fermand JP, Ravaud P, Chevret S, et al. High-dose
reduce cancer cel s in the transplant. Not used success-
therapy and autologous peripheral blood stem cell
ful y for myeloma patients.
transplantation in multiple myeloma: up-front or rescue
Tandem transplant: A term used to indicate two trans-
treatment? Results of a multicenter sequential randomized
plants. This may be two autologous transplants or an
trial. Blood. 1998;92:3131-3136.
autologous transplant fol owed by an al ogeneic (donor)
3. Barlogie B, Jagannath S. Desikan KR< et al. Total
transplant. Tandem transplants are usual y planned at 3
therapy with tandem transplants for newly diagnosed
to 6 month intervals between transplants.
multiple myeloma. Blood. 1999;93:55-65.
Syngeneic stem cel transplant: Refers to stem cel s that are
4. Child JA, Morgan GJ, Davies FE, et al. High-dose
taken from an identical twin of the patient.
chemotherapy with hematopoietic stem-cel rescue for mul-
tiple myeloma. N Engl J Med. 2003;348:1875-1883.
Transplantation: Stem cel s are used to rescue the patient's
5. At al M, Harousseau JL, Facon T, et al. Single versus
blood-forming potential fol owing a very high-dose che-
double autologous stem-cel transplantation for multiple
motherapy and/or radiation treatment. Transplant is not
myeloma. N Engl J Med. 2003;349:2495-2502.
a treatment but a method of support to make high-dose
chemotherapy treatment possible.
6. Fassas AB, Van Rhee F, Tricot G. Predicting long-term
survival in multiple myeloma patients fol owing autotrans-
Umbilical cord blood transplant: Stem cel s obtained from
plants. Leuk Lymphoma. 2003;44:749-758.
the umbilical cords of newborns. These are frozen and
7. Blade J. Transplantation for multiple myeloma: who,
stored in cord blood banks.
when, how often? High-dose therapy in multiple myelo-
Very good partial response (VGPR): A response that is not
ma. Blood. 2003;15:3469-3477.
quite a complete response (that is, not 100%), but has a
8. Barlogie B, Tricot G, Anaissie E, et al. Thalidomide
90% or greater reduction in serum M-protein.
and menatopoietic-cel transplantation for multiple myelo-
ma. N Engl J Med. 2006;354:1021-30.
White blood cel : One of the three major cel types in the
blood. There are several types of white cel s (i.e., neutro-
9. Cavo M, Baccarani M. The changing landscape of
phils, lymphocytes, and monocytes).
myeloma therapy. N Engl J Med. 2006;354:1076-78.
10. Barlogie B, Tricot G. Complete response in myeloma:
a Trojan horse? Blood. 2006;108:2134.
The IMF would like to thank
Dr. David Vesole and Dr. Brian Durie
for their help in preparing this publication.
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