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
myeloma.org

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Table of Contents
Introduction
5
What is Multiple Myeloma?
6
The Stages of Multiple Myeloma
7
Background Rationale for Use of High-Dose
Chemotherapy and Blood Stem Cell
Transplant or Rescue
8
Types of Stem Cell Transplant
10
How Stem Cell Transplant is Used
as a Part of Myeloma Therapy
11
What are the Benefits of High-Dose
Chemotherapy with Blood Stem Cell Rescue? 13
Practical Steps in Considering Stem Cell
Transplant as a Treatment Option
14
How Stem Cells are Collected
16
Administering High-Dose Chemotherapy
21
Preventing Infection
23
Engraftment and Recovery
24
Am I a Candidate for an
Autologous Transplant?
25
Transplants and Clinical Trials
27
Psychosocial Issues
29
Questions and Answers about Stem Cell
Transplantation
29
Questions for the Doctor
35
About the IMF
39
Glossary
42
Bibliography
47
©2011, International Myeloma Foundation, North Hollywood, California (v11)

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Introduction
This booklet explains important details about the
transplantation 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
since the introduction of novel therapy
approaches in the last decade? 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.
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What is Multiple Myeloma?
The Stages of Multiple Myeloma
Multiple myeloma (also known as myeloma
and plasma cel myeloma) is a cancer of the
Stage I (low cel mass)
600 bil ion
myeloma cel s*
immunoglobulin-producing plasma cel s found
Al of the fol owing:
in the bone marrow. It is a cancer that involves
· Hemoglobin value >10 g/dL
the immune system. The cancerous plasma
· Serum calcium value normal or <10.5 mg/dL
cel s, or myeloma cel s, rarely enter the blood
· Bone X-ray, normal bone structure (scale 0)
stream. The myeloma cel s accumulate in the
or solitary bone plasmacytoma only
bone marrow, causing the fol owing:
· Low M-component production rates
IgG value <5.0 g/dL
n Disruption of normal bone marrow function,
IgA value <3.0 g/dL
most commonly causing anemia (a low
· Urine light chain M-component
level of red blood cel s in the bloodstream),
on electrophoresis <4 g/24h
although reduction in white blood cel and
Stage I
platelet counts can also occur
(intermediate cel mass)
600 to 1,200
Fit ing neither stage I nor stage I I
bil ion
n Damage to bone surrounding accumulated
myeloma cel s*
myeloma cel s
Stage I I (high cel mass)
>1,200 bil ion
n Release of an abnormal protein, monoclonal
One or more of the fol owing:
myeloma cel s*
protein (M protein), into the blood stream
· Hemoglobin value <8.5 g/dL
and/or urine
· Serum calcium value >12 mg/dL
· Advanced lytic bone lesions (scale 3)
n Suppression of normal immune function,
observed as reduced levels of normal immu-
· High M-component production rates
noglobulins and increased susceptibility to
IgG value >7.0 g/dL
infection.
IgA value >5.0 g/dL
· Urine light chain M-component
Myeloma cel s can also grow in the form of local-
on electrophoresis >12 g/24h
ized tumors or plasmacytomas. Plasmacytomas
Subclassification (either A or B)
may be single or multiple and either medul ary
· A: relatively normal renal function
(confined within bone marrow and bone) or
(serum creatinine value) <2.0 mg/dL
extramedul ary (outside of the bone). When
· B: abnormal renal function
there are multiple plasmacytomas inside or out-
(serum creatinine value) >2.0 mg/dL
side bone, this condition is also cal ed multiple
Examples:
myeloma.
Stage IA (low cel mass with normal renal function)
Stage I IB (high cel mass with abnormal renal
function)
*myeloma cel s in the whole body
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A new prognostic factor system cal ed the
n High-dose
melphalan seriously dam-
International Staging System (ISS) was intro-
ages normal stem cel s. High-dose mel-
duced in 2005. It is based upon the levels of
phalan is a very ef ective treatment for
two blood proteins: beta-2 microglobulin (2M)
myeloma, but can also permanently damage
and albumin; the levels of these proteins predict
normal blood stem cel s. High dosages of
overal outcome with myeloma treatment.
melphalan can be especial y helpful in eradi-
cating myeloma cel s from the bone marrow.
STAGE
CRITERIA
To circumvent the problem of simultaneous
Stage 1
Serum 2 microglobulin <3.5 mg/L
severe damage to and potential destruction
Serum albumin 3.5 g/dL
of normal blood stem cel s in the bone mar-
Stage 2
Serum 2 microglobulin < 3.5 mg/L
row, techniques for col ecting and saving
and serum albumin < 3.5 g/dL
normal blood stem cel s before administering
or
the melphalan have been developed.
Serum 2 microglobulin 3.5 - 5.5 mg/L
n Stem cel s can be col ected (harvested) and
Stage 3
Serum 2 microglobulin >5.5 mg/L
infused after treatment to replace those
damaged by treatment. Normal blood stem
Multiple myeloma is a serious cancer, but it is
cel s are col ected or "harvested" from the
treatable. Many patients experience a series
patient or donor before administration of the
of responses, relapses, and remissions. New
melphalan. The harvested normal blood stem
treatments may extend the survival for patients
cel s are returned to the blood circulation by
diagnosed with multiple myeloma.
a process similar to blood transfusion. By a
seeding process, the stem cel s pass from the
Background Rationale for Use of
circulation back into the bone marrow where
High-Dose Chemotherapy and Blood
they divide and grow to repopulate the
Stem Cell Transplant or Rescue
normal bone marrow space. Approximately
n Myeloma cel s and normal blood stem
cel s are in the same bone marrow micro-
environment. As myeloma cel s build up in
the bone marrow, they become intermixed
with normal blood stem cel s responsible for
the production of normal red and white cel s
as wel as platelets. Any drugs reaching the
bone marrow microenvironment can there-
fore damage both the myeloma cel s and the
normal blood stem cel s.
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36 to 48 hours after administering the
n "Mini" or non-myeloablative al ogeneic trans-
melphalan, the blood and tissue levels of
plant is a newer and safer procedure than
melphalan are very low and do not harm
ful al ogenic transplant. It involves the use of
the new stem cel growth. This whole process
reduced intensity chemotherapy in combina-
of harvest and re-infusion at the best time is
tion with a donor stem cel transplant.
cal ed "stem cel transplant."
n Matched Unrelated Donor (M.U.D.) stem cel
transplant. Stem cel s are harvested from a
Types of Stem Cell Transplant
non-family member. In this case, the stem
n Autologous stem cel transplant. Stem cel s
cel s are rarely a 100% tissue (HLA) match.
are harvested from a patient with myelo-
Hence the term "mismatch" is frequently used
ma fol owing initial therapy and re-infused
in this situation.
after high-dose melphalan therapy has been
administered. This is the most common type
How Stem Cell Transplant is Used as
of stem cel transplant. The procedure can
a Part of Myeloma Therapy
be performed once (single autotransplant) or
n Fol owing diagnosis, several options are
twice (double or tandem transplant).
available for initial or front-line therapy.
n Syngeneic stem cel transplant. Stem cel s
Typical frontline regimens currently
are harvested from an identical twin. In this
utilized are:
case, the stem cel s from the identical twin
· Thalomid® or Revlimid® plus
are infused after high-dose therapy, which
dexamethasone
can be melphalan or other agents.
· VELCADE® plus dexamethasone
n Al ogeneic stem cel transplant. Stem cel s
· Various combinations incorporating an
are harvested from a family member who is
anthracycline (e.g., Adriamycin® or
not an identical twin, but is wel matched by
DOXIL®) plus VELCADE®
tissue (HLA) typing. Again, the stem cel s are
· Cytoxan®, an alkylating agent, can also
infused after the high-dose therapy.
be used as part of the initial approach
in combination with VELCADE® or
Revlimid®.
Ful details of these treatments are discussed in
other publications of the International Myeloma
Foundation.
n In general, stem cel transplant is a potential
option for al myeloma patients upon comple-
tion of frontline therapy. However, since trans-
plant is an intensive approach, patients over
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the age of 65 years and/or those with other
What are the Benefits of
medical conditions may not be able to tolerate
High-Dose Chemotherapy
the procedure and/or may run the risk of more
serious complications. If stem cel transplant is
with Blood Stem Cell Rescue?
considered to be a potential option, the most
n Further improvement in the level of response
important caution is to avoid use of melphalan
achieved with frontline therapy is a major
by mouth prior to stem cel harvesting, since this
advantage of high-dose therapy with stem cel
can lead to damage of normal bone marrow
transplant. Over half the time, partial respons-
stem cel s. Thus, avoiding melphalan initial y
es wil be improved to either VGPR (very
and keeping al options open is the most com-
good partial response, with 90% myeloma
monly recommended strategy. Conversely, if
protein reduction) or CR (complete response,
stem cel transplant can never be an option or is
with disappearance of measurable myeloma
not preferred, for whatever reason, melphalan
protein level).
pil s as a part of initial therapy can be a simple
and very ef ective treatment.
n Enhanced benefit in patients who have
already experienced VGPR or CR. With the
n Stem cel s are harvested and transplant is
advent of more frequent VGPR or CR with novel
performed after initial or frontline therapy.
frontline therapies, the added benefit of high-
This means that treatment is used to achieve
dose therapy in this set ing is coming under
response and at least some degree of remis-
closer scrutiny. High-dose chemotherapy has
sion before proceeding to therapy with high-
conferred statistical y significant benefit fol ow-
dose melphalan and blood stem cel rescue.
ing traditional chemotherapy induction using,
Two major points about frontline therapy are:
for example, VAD chemotherapy. However,
· Initial therapy for 3 to 6 months should
novel therapy combinations can produce high
be with drugs that do not damage normal
levels of VGPR and CR. The additional ben-
blood stem cel s.
efit of high-dose therapy for a patient who
· Ideal y, response to induction therapy
has already achieved VGPR or CR is under
should provide a >50% reduction in myelo-
investigation.
ma protein levels and/or other indicators
n Enhanced response without the necessity
of active myeloma prior to the col ection
of maintenance. A particular benefit of high-
of normal blood stem cel s. However, even
dose therapy is that added response can
lesser degrees of response may be suf icient
occur within a few weeks of the procedure. If
to al ow safe and ef ective stem cel col ec-
CR or VGPR occur, then such patients can be
tion to be performed.
fol owed and monitored without the absolute
need for ongoing maintenance anti-myeloma
therapy. Patients undergoing high-dose therapy
also tend to be in remission longer and thus
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to have a longer period before retreatment is
approach to treatment, it is an important
required. Thus, the potential ongoing toxicity,
time to seek a second opinion before going
inconvenience, and expense of maintenance
ahead with a frontline strategy.
can be avoided. However, depending upon the
STEP TWO
patient's individual details, including the results
n Proceed with initial or frontline therapy
of chromosome testing, maintenance therapy
to bring the myeloma under control and
and/or other (consolidation) therapy may be
achieve an initial response.
recommended after transplant.
n Make sure to avoid melphalan or other
n Potential benefit with double or tandem
therapy that may reduce the success of nor-
transplantation. If CR or VGPR are not
mal blood stem cel harvesting. Radiation
achieved with a single autologous transplant,
therapy to the pelvis, for example, can
then a second autologous transplant can be
reduce stem cel reserves and should be
of ered. Continuing in the at empt to achieve
avoided if possible.
VGPR with the second transplant does appear
STEP THREE
to confer benefit.
n Assess the response to treatment with each
n Significance of achieving CR or VGPR. It has
cycle of therapy (usual y every 3 to 4 weeks).
been general y accepted that patients achiev-
n After 3 to 4 cycles of treatment, more com-
ing bet er response such as CR or VGPR have
plete re-evaluation is recommended, includ-
bet er outcomes (versus, for example, partial
ing bone marrow testing plus x-ray/scans as
response [PR]). However, further studies are
needed to determine the level of response.
required. Having a durable response at a par-
ticular level, whether that is a simple PR (50%
STEP FOUR
improvement), VGPR (90%) or CR (100%), is
n Review with the physician the pros and cons
more important than the level of the response
of stem cel transplant (and/or stem cel
in itself. Response lasting 2 years is particu-
harvesting without immediate transplant).
larly beneficial. The relative benefit of stable
disease at the PR, VGPR, or CR level is under
further study.
Practical Steps in Considering Stem
Cell Transplant as a Treatment Option
STEP ONE
n Confirm the diagnosis of active myeloma that
requires anti-myeloma treatment.
n If there is any doubt about the diagnosis or
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n If 50% response (PR: 50% reduction in
Methods of Col ecting Stem Cel s from the
myeloma protein level in blood and/or
Blood Stream (Peripheral Blood Stem Cel s
urine) is achieved, stem cel harvesting can
[PBSC])
be planned if the patient and the physician
There are three main methods for col ecting
concur that this is the best approach. If there
stem cel s: 1) giving growth factors alone,
is no plan for harvest and/or transplant, a
2) giving growth factors with chemotherapy,
plan for ongoing maintenance or fol ow-up
or 3) giving growth factors with a mobilizing
treatment is required.
agent.
n If there is < 50% response, then other
1. Giving growth factors with a mobilizing agent.
therapy may be required before proceeding
to transplant.
Growth factors are drugs that stimulate blood
stem cel s both to grow and to be released
"Questions and Answers" about stem cel trans-
into the blood stream. There are red cel
plant as wel as "Questions to Ask the Doctor"
and white cel growth factors. These medica-
about the potential procedure are listed later in
tions are administered subcutaneously (under
the brochure.
the skin). Growth factors are often used for
patients receiving chemotherapy to hasten their
How Stem Cells are Collected
white and red cel count recovery. The white
Blood stem cel s are located in the bone mar-
cel growth factors (Neupogen®, Neulasta®,
row. Until about twenty years ago, the only
Leukine®) used in high doses stimulate the
way to col ect these stem cel s was to have the
release of stem cel s from the bone marrow into
patient or donor receive a general anesthetic
the bloodstream. This process is cal ed "mobili-
and undergo as many as fifty to one hundred
zation." The injections are given daily for three
bone marrow aspirations from the back of the
or more days. Stem cel s are usual y col ected
pelvic bone to remove enough bone marrow
on the fourth or fifth day after starting the injec-
and stem cel s to use for future transplant. This
tions. The col ections and injections wil contin-
was obviously painful, invasive, and inconve-
ue daily until suf icient stem cel s are obtained.
nient. The discovery that stem cel s could be col-
lected from the bloodstream by giving a patient
or donor injections of stem cel growth factors
("colony-stimulating factors ­ CSFs") such as
Neupogen®, Neulasta®, or Leukine® to trigger
the release of bone marrow stem cel s into the
bloodstream was a major breakthrough. With
refinements over the years, this has become the
standard method. It is rarely necessary to use
the old method of direct bone marrow harvest-
ing from the pelvic bone.
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2. Using chemotherapy plus growth factors.
increases the number of stem cel s that can be
Chemotherapy with growth factors may also
col ected.
be used to release stem cel s from the bone
The Col ection or Harvesting Procedure
marrow into the bloodstream. The doctor wil
In medical language, the harvesting is cal ed
explain why it may or may not be appropriate
apheresis or leukapheresis ­ literal y the
to use chemotherapy in addition to growth fac-
removal of white cel s from the blood stream.
tors. The doctor wil explain the chemotherapy
Apherisis is a procedure whereby blood from
that wil be administered to mobilize the blood
the patient or donor passes through a special
stem cel s and its potential benefits and side
machine that separates (using a centrifuge
ef ects. Fol owing chemotherapy for stem cel
technique) and then removes stem cel s. The
mobilization, a white cel growth factor is given
rest of the blood is immediately returned to the
by injection under the skin daily for approxi-
patient or donor. Compared to direct bone mar-
mately ten days. This procedure is therefore lon-
row harvesting, this is a remarkably simple and
ger and much more intensive than using growth
pain-free procedure.
factors alone. The patient or someone who
agrees to be responsible may be taught how
Apheresis/Leukapheresis: Prior to the start of
to give the growth factor injection so that it can
apheresis, a thin flexible plastic tube cal ed a
be administered at home. Some patients may
catheter is inserted through the skin and into a
receive their injections at a clinic or hospital,
vein so that blood can be taken out. The cath-
or they may receive them at home from a visit-
eter is usual y inserted into the chest just below
ing nurse. Once the number of stem cel s in the
the col arbone. Insertion of the catheter is usu-
blood stream is high enough, they wil be col-
al y done as an outpatient procedure, and only
a local anesthetic is necessary. The site where
lected over two to five days, while the patient is
the catheter enters the skin may be sore for a
stil receiving the growth factor injections.
few days; the discomfort may be relieved with
3. Using a mobilization agent plus growth factors.
medications like acetaminophen (Tylenol®). The
Mozobil® (plerixafor) was approved by the
catheter may be kept in place for several weeks
FDA in 2008 as a stem cel mobilizer. It is
because it can be used to give chemotherapy
used in combination with growth factors to
after stem cel s have been col ected. Sometimes
release stem cel s into the blood so they can
the same catheter is used during the transplant
be col ected and used for transplant in patients
procedure as wel . The apheresis procedure
with myeloma (as wel as patients with non-
wil last 3 to 4 hours each day for 1 to 5 days.
Hodgkin's lymphoma). Patients are treated with
Apheresis is usual y done as an outpatient
growth factors for 4 days prior to receiving
procedure.
Mozobil®. Mozobil® is injected subcutaneously
The most common side ef ects experienced dur-
11 hours before the planned stem cel col ec-
ing apheresis are slight dizziness and tingling
tion for up to 4 consecutive days. Mozobil®
sensations in the hands and feet. Less common
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side ef ects include chil s, tremors, and muscle
checked in each daily col ection and the num-
cramps. These side ef ects are temporary and
ber tal ied. The stem cel col ection process con-
are caused by changes in the volume of the
tinues daily until the planned number of stem
patient's blood as it circulates in and out of
cel s is col ected ­ usual y 1 to 4 days. Some
the apheresis machine, as wel as by blood
transplant centers check the number of CD34+
thinners added to keep the blood from clot ing
cel s BEFORE starting leukapheresis to make
during apheresis.
certain there wil be a good col ection that day.
Processing stem cel s: After col ection, the
Most transplant physicians col ect enough stem
peripheral blood (or occasional y direct bone
cel s for two transplants (over 4 mil ion CD34+
marrow material) is taken to the processing
cel s per kilogram of weight).
laboratory, which is usual y located within the
Administering High-Dose
hospital or local blood bank. In the processing
Chemotherapy
laboratory, the bone marrow or blood cel s are
prepared for freezing (cryopreservation). The
After the stem cel s are frozen and stored, the
stem cel s are mixed with a solution containing
patient is ready to receive high-dose chemo-
the chemical DMSO (dimethyl sulfoxide) to pre-
therapy. This treatment is designed to destroy
pare the stem cel s for freezing. The stem cel s
myeloma cel s more ef ectively than standard-
are then frozen and stored in liquid nitrogen.
dose chemotherapy. The purpose of high-dose
The stem cel s wil be frozen until the time they
chemotherapy is to kil myeloma cel s inside
wil be needed for the transplant. They can be
the patient's body. The most common type of
stored frozen for as long as necessary. There
high-dose chemotherapy used to treat myeloma
is some deterioration with time, but excel ent
is melphalan administered at a dose of 200
function of stem cel s is retained for at least
mil igrams per square meter (mg/m2) of body
10 years.
surface area (size of patient). Depending on
the type of myeloma and other factors, some
How many stem cel s do I need? Over the years,
patients may receive a second transplant three
a number of studies have been completed to
to six months after the first transplant (double
determine the number of stem cel s you need to
or tandem transplant). A patient should discuss
safely undergo high-dose therapy. The number
with the doctor the pros and cons of more than
of stem cel s is quantified by a special labora-
one transplant planned and performed back-
tory technique cal ed "CD34+ cel analysis by
to-back versus the possibility that the cel s wil
flow cytometry." A smal sample of the stem cel
be stored for a potential second transplant at
col ection is tested for the number of CD34+
a later time.
cel s in the product. We know that a minimum
number of stem cel s to safely complete a trans-
Autologous Stem Cel Transplant or Infusion
plant is 2 mil ion CD34+ cel s per kilogram of
Since high-dose treatment destroys the normal
body weight. The number of CD34+ cel s is
bone marrow in addition to the myeloma cel s,
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the blood stem cel s must be given back to
time in the hospital (or a nearby facility) for
restore the bone marrow. The previously col-
the chemotherapy, transplant, and recovery is
lected stem cel s wil be unfrozen and given
approximately three weeks. Shortly before start-
back, through a catheter, into the bloodstream
ing chemotherapy, patients usual y are given
(as one would receive a blood transfusion) one
large amounts of fluid to prevent dehydration
to two days after administration of the high-
and kidney damage from the chemotherapy.
dose chemotherapy. This procedure is often
Some of the more common side ef ects of
referred to as the transplant. The transplant
chemotherapy include nausea, vomiting, diar-
takes place in the patient's room: it is not a
rhea, mouth sores, skin rashes, hair loss, fever
surgical procedure. The frozen bags of blood
or chil s, and infection. Medications designed
cel s are thawed in a warm water bath, and
to prevent or lessen some of the expected side
then injected into the bloodstream through the
ef ects of treatment are given routinely. Patients
catheter. Upon thawing, the DMSO (freezing
are very closely monitored during and after
agent) evaporates into the air and creates a
the administration of high-dose chemotherapy.
distinct and somewhat unpleasant garlic smel .
Monitoring includes daily weight measurement
Most centers infuse one bag at a time. It usual y
as wel as frequent measurements of blood
takes one to four hours for the infusion. Infused
pressure, heart rate, and temperature.
stem cel s travel through the bloodstream, and
eventual y, to the bone marrow, where they
Preventing Infection
begin to produce new white blood cel s, red
During the first two to three weeks after trans-
blood cel s, and platelets. It takes ten to four-
plantation, the re-infused stem cel s migrate to
teen days for the newly produced blood cel s
the bone marrow and begin the process of
to enter the bloodstream in substantial numbers.
producing replacement blood cel s, a process
Growth factors may again be given to the
cal ed engraftment. Until engraftment of the
patient to speed up this process.
stem cel s takes place, patients are very sus-
In addition to obliterating the bone marrow,
ceptible to developing infections. Even a minor
high-dose chemotherapy can cause other
infection like the common cold can lead to
severe side ef ects, which may require that
serious problems because the body's immune
some patients be admit ed to the hospital for
system is so weakened by the ef ects of the
treatment during this period. (Not al transplant
high-dose chemotherapy. Therefore, special
centers require that patients remain in the
precautions are necessary during recovery.
hospital after the infusion of stem cel s; some
Since the patient's immune system is very
have facilities close by where patients may stay
weak, patients may remain in the hospital until
and be monitored daily at the hospital on an
the white blood cel counts reach a level safe
enough for the patient to be discharged.
out-patient basis, while others al ow patients
who live close to the hospital to sleep at home
To prevent infection, the fol owing supportive
and be monitored at the hospital). The average
care measures may be required:
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· Antibiotics are often prescribed to help pre-
made, symptoms wil resolve, the risk of serious
vent infection.
infections wil be reduced, and transfusions wil
· Visitors should wash their hands and may be
no longer be needed.
asked to wear masks and rubber gloves to
After being discharged from the hospital, a
protect the patient.
patient continues recovery at home for two to
· Fresh fruits, vegetables, and flowers may be
four months. Although patients may be wel
prohibited from the patient's room as these
enough to leave the hospital, their recovery
can carry infection (bacteria and fungi).
wil be far from over. For the first several weeks
· If infection and/or fever occurs (as the result
the patient may be too weak to do much more
of lowered white cel counts), the patient may
than sleep, sit up, and walk a lit le around the
be admit ed to the hospital and be given
house. Frequent visits to the hospital wil be
intravenous antibiotics.
required to monitor progress. Patients usual y
cannot resume normal activities or return to ful -
Engraftment and Recovery
time work for up to three to six months after the
Once the stem cel s have been re-infused, it
transplant, although this varies from individual
wil take about two weeks for blood counts to
to individual.
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) wil
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
with myeloma are candidates for a stem cel
Waiting for the transplanted stem cel s to
transplant. Many factors must be taken into
engraft, for blood counts to return to safe levels,
consideration. These include factors related to
and for side ef ects to disappear is often the
the myeloma itself and patient-related factors.
most dif icult time for patients and their families
and friends. During this period patients wil
MyElOMa-RElaTEd FacTORS
feel weak and very fatigued. Having a support
· type of myeloma
network is very important during this period.
· disease stage
Recovery can be like a rol er coaster ride: one
· disease aggressiveness
day a patient may feel much bet er, only to
· responsiveness to treatment
awake the next day feeling as sick as ever. It is
· serum albumin
important during this period to take things one
· beta-2 microglobulin
day at a time. Once new blood cel s are being
· chromosome analysis
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PaTiENT-RElaTEd FacTORS
example, one could have stem cel s harvested
· age
and saved for a later treatment. This leaves the
· health status
patient open to other more immediate treatment
options. These are things to discuss with the
· kidney, heart, lung, and liver function
doctor. It's important to remember that even if
· patient preference
someone is a good transplant candidate, the
· family situation
ultimate decision about whether or not to have
We cannot stress enough that myeloma is
a transplant is the patient's.
a highly individualized disease. While
there are similarities between patients,
Transplants and Clinical Trials
each patient's disease has its own dis-
A single autologous stem cel transplant is
tinct characteristics. There wil be testing
currently considered the standard of care for
to determine how much myeloma there is in
patients with multiple myeloma. However, there
your body and how aggressive it is. Al of these
are a number of novel approaches that are
variables wil be weighed before determining
being evaluated to try to improve patient out-
whether a transplant is appropriate for you.
comes. These are being conducted as clinical
Therefore, general statements regarding patient
trials. These include the fol owing:
outcomes both during the transplant procedure
A tandem autologous transplant is an approach
and post transplant are inappropriate.
that utilizes two autologous transplants.
When to transplant is also an important con-
Suf icient stem cel s are col ected prior to the
sideration. Most transplant physicians believe
first transplant. Three to six months after the
it is bet er to perform the transplant early in the
first transplant, the patient receives a second
disease course. However, there is no absolute
similar course of high-dose therapy fol owed
clinical data to suggest that transplantation
by infusion of the other half of the stored stem
earlier in the treatment regimen is bet er than
cel s. Preliminary data indicates that tandem
waiting until later. Recent trial results suggest
transplants result in improved disease control
that frontline therapy that includes the anti-
and survival in patients who do not experience
myeloma agents thalidomide, Revlimid®, and/
either VGPR or CR fol owing first autologous
or VELCADE® may result in response rates and
transplant.
duration of response comparable to those of
Sequential autologous transplant fol owed by
stem cel transplant, al owing some patients to
a "mini" (non-myeloablative) al ogeneic trans-
postpone transplant until later in the course of
plant involves high-dose chemotherapy with an
the disease. This is undergoing further investi-
autologous transplant to destroy the majority
gation. Remember, in most cases, unlike a heart
of the myeloma cel s, fol owed 2 to 4 months
at ack, myeloma gives the patient the luxury
later by an al ogeneic mini-transplant to al ow
of time to do some homework and to gather
the donor's immune cel s to destroy any remain-
the information needed to make an informed
ing myeloma cel s. Once the al ogeneic stem
decision about what's right for him or her. For
26
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cel s grow, the donor's immune cel s at ack the
Psychosocial Issues
myeloma. This is a form of immunotherapy.
The risk of this procedure is that the donor's
High-dose chemotherapy and autologous
immune system wil "overreact" and at ack
transplantation can place an enormous stress
more than the myeloma cel s. This reaction is
on patients and families. Physical, psycho-
cal ed "graft-versus-host disease," which can
logical, emotional, and financial stresses can
be very serious and potential y life-threatening.
be overwhelming. Patients and families may
Autologous stem cel transplant fol owed by
experience feelings of anger, depression, and
mini al ogeneic transplant (an "auto-mini al o")
anxiety over an unknown future and a lack
has been studied in the clinical trial set ing in
of control. Support services of ered through
myeloma patients for a number of years. In
the hospital and many other organizations,
2006, an IFM (French myeloma group) study
including myeloma support groups, are very
reported no overal survival or progression-free
important during this time. We urge you to
survival benefit with auto-mini al o vs. tandem
take advantage of these services, or to seek a
auto. In 2007, Bruno et al found improved
referral from your oncologist for psychological
survival for patients receiving auto-mini al vs.
counseling and/or a psychiatric consultation.
tandem auto. At the 2010 American Society
Questions and Answers
of Hematology (ASH) meeting in Orlando, FL,
a large BMT CTN study of tandem autologous
about Stem Cell Transplantation
transplant vs. auto-mini al o (Krishnan et al)
Listed below are some of the questions frequent-
with 710 patients at 43 centers found no dif-
ly asked by people with myeloma who have
ference in progression-free or overal survival.
had or are considering a stem cel transplant.
Given the lack of remission or survival benefit
These questions and other concerns should be
and the increase in treatment-related mortality
discussed with the doctor and members of the
in the auto-mini al o patients, the International
healthcare team before making any final deci-
Myeloma Working Group currently recom-
sions about the patient's treatment plan.
mends that mini al o be performed only in the
context of clinical trials.
Q. Why is a stem cel transplant necessary for
a multiple myeloma patient?
Maintenance therapy is an approach that
A. The transplant procedure al ows the patient
involves lowered doses of anti-myeloma drugs
to receive high doses of chemotherapy to kil
to maintain longer remission and survival after
more myeloma cel s. This therapy is so potent
a transplant. Currently, some of the drugs
that it destroys al of the bone marrow. Without
being evaluated as maintenance therapies in
bone marrow, the body is unable to manufacture
clinical trials include Revlimid®, prednisone,
blood cel s needed to carry oxygen, help blood
VELCADE®, and dexamethasone, alone or in
clot, and defend against infection. Therefore,
combinations.
a stem cel transplant replaces the destroyed
marrow, rescuing the patient from the ef ects of
high-dose chemotherapy.
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29

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Q. Am I a candidate for bone marrow or
the one best suited for patients with multiple
peripheral blood stem cel transplant?
myeloma, you should talk with your doctor, other
A. Medical experts have yet to arrive at a set of
multiple myeloma patients, and the International
fixed guidelines for selecting patients who wil
Myeloma Foundation.
benefit the most from a transplant. Increasingly
Q. What goes on at a transplant center?
accepted as a part of multiple myeloma
treatment protocols, successful transplantation is
A. To understand what goes on at a transplant
a function of the patient's age, general physical
center, we strongly suggest a visit to one or more
condition, stage of disease, and responsiveness
centers. Meet with the staf ­ doctors, nurses,
to prior treatments. Only the patient's physician
and other members of the multiple myeloma
can provide a patient with the best assessment of
treatment team ­ and learn more about how they
his or her chances for long-term survival.
approach a transplant. See the room where your
transplant wil occur and where you'l be spend-
Q. Does taking alkylating agents such as
ing your recuperation time. Find out what part of
melphalan, busulfan, and cyclophospha-
your procedure wil be performed in a clinic or
mide (Cytoxan®) reduce my suitability for a
a doctor's of ice and what part wil be done in
transplant?
the hospital. You should be comfortable with the
A. Alkylating agents are one of the most ef ec-
center before you begin your transplant.
tive ways of kil ing myeloma cel s inside the
Q. If my doctor agrees that a stem cel trans-
body. However, their prolonged use ­ more than
plant is an appropriate treatment for my dis-
4 to 6 months ­ wil reduce the ability to easily
ease, what can I do now to prepare for the
harvest a patient's stem cel s. Therefore, when
experience?
considering a transplant, a patient should first
discuss the total treatment plan to ensure that
A. The patient can do a lot to get ready for the
there are as many short-term and long-term treat-
transplant. By reading this brochure, a patient
ment options available as possible. It should be
has already taken the most important step: learn-
emphasized, however, that col ection should ide-
ing as much as possible about the procedure. A
al y be done before using any alkylating agents.
patient should speak with the doctor, seek out
fel ow survivors, and read as much as possible,
Q. How do I select a transplant center?
including the publications and newslet ers from
A. A transplant is a complicated medical pro-
the International Myeloma Foundation. Patients
cedure that requires an expert team of doc-
should ask questions about what they've learned
tors, nurses, social workers, psychologists, and
and strive to read al the newest information
al ied health professionals who understand the
coming from research. We suggest that patients
procedure, have performed it successful y many
bring a tape recorder or a friend along to the
times, and are equipped to respond when
doctor's of ice so that they can give ful at ention
medical and emotional problems arise. Today,
to the doctor. Patients should share what they
medical centers that meet these criteria can
know with family and loved ones so that they
be found throughout the country. Many of
wil know what to expect ­ and how they can
these centers specialize in treating patients
help in the weeks and months ahead.
with many dif erent types of cancer. To find
30
31

---

The doctor wil perform a series of tests to con-
Q. Can a patient die from the transplant itself?
firm that the patient is wel enough to tolerate
A. Every medical procedure carries risk, and a
the transplant. Al the data gathered on the
transplant for patients with multiple myeloma is
performance of the heart, lungs, kidneys, and
riskier than most. Nonetheless, medical studies
other vital organs wil enable the doctor to com-
have shown that over 95% of patients (usual y
pare the patient's health before and after the
closer to 99%) survive transplant.
procedure. In most cases, the patient won't have
to be hospitalized for these tests since they can
Q. Can the patient relapse after a transplant?
be performed in the doctor's of ice.
A. Yes. Unfortunately, the majority (at least 50%)
of al patients with multiple myeloma experience
Q. What side effects should I anticipate from
a relapse 18 to 36 months after their transplant
the transplant?
is completed.
A. Side ef ects can be expected from every type
of medical treatment, even the use of aspirin.
Q. I've heard a lot about myeloma purging.
Each patient reacts dif erently to chemotherapy
Can it help?
and other drugs given during the transplant. No
A. The process of purging removes myeloma
two patients share exactly the same side ef ect
cel s from peripheral blood taken from the
profile.
patient's body prior to transplant. High-dose
chemotherapy is used to kil myeloma cel s
Patients should therefore seek a transplant cen-
that are within the body. Stem cel selection,
ter where the doctors, nurses, and al ied health
or "purging," is used to remove myeloma cel s
professionals have performed a number of trans-
from the col ected stem cel s prior to the trans-
plants and appear competent to care for each
plant. The goal of this strategy is to reduce the
individual myeloma patient's needs.
number of myeloma cel s both within the patient's
Q. What happens during re-infusion?
body as wel as in the peripheral blood that wil
A. After chemotherapy the patient receives a
be re-infused into the patient. Recent evidence
re-infusion of his or her own stem cel s. The
indicates that this technology is not ef ective in
stem cel s wil be thawed and infused into the
myeloma. Therefore, very few centers currently
patient's catheter either through a syringe or
use stem cel purging for patients with myeloma.
from an intravenous infusion bag. While the
Q. How long wil the transplant patient stay
re-infusion takes place the patient may feel
in the hospital?
warm or lightheaded. The chemical used to
keep the stem cel s fresh has a garlic smel that
A. Patients stay in the hospital for about two
the patient might be able to taste. The oncolo-
to three weeks. The length of stay varies from
gist may re-prescribe or adjust the patient's
patient to patient. Some patients may have sev-
medication to make him or her feel more com-
eral short admissions.
fortable during this procedure.
Q. When wil the stem cel s start to grow again?
A. Stem cel s start to grow back or "engraft"
within ten to fourteen days after re-infusion.
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33

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Q. What wil the patient's quality of life be after
always consult their doctors about their use. The
transplant?
doctor should be informed of the names of al the
A. On average, patients take 3 to 6 months
alternative and complementary therapies being
to recover from a transplant. By this time, the
taken so that he or she can adjust the regimen
immune system wil once again fight infections
accordingly. It is important to note that even
because the bone marrow is producing healthy
seemingly innocuous over-the counter drugs,
blood cel s. Hair wil grow back, but the taste
e.g., ibuprofen, may be harmful to a patient
buds might stil be a lit le quirky. Foods that
with myeloma.
tasted good before a transplant might not taste
good now. However, in most cases, patients
Questions for the Doctor
should be able to return to normal daily activi-
These are questions we suggest be discussed
ties. It can take as long as a year to recover
with the doctor to provide bet er understanding
normal functioning. Patients and their caregivers
must take one day at a time. There wil be bad
of the transplant procedure and its ef ects on
days and good days, and they won't necessar-
the patient's life. Space is provided for notes:
ily come in that order. Patients should prepare
"Am I a candidate for stem cel transplant?"
themselves to feel dif erently each day during the
recovery process.
________________________________________
Q. Should transplant patients expect changes in
________________________________________
their emotions?
________________________________________
A. Yes. Transplant is more than just a medi-
cal procedure. Because it forces the patient to
"What does high-dose chemotherapy with
rely upon the oncologist and other members
transplant hope to achieve that can't be achieved
of the transplant team, as wel as on family
by 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 counseling.
"What treatment protocols are there at your
Help may also be found through patient support
institution and how do you decide which one is
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
________________________________________
of their treatment program. Because al drugs,
synthetic and natural, interact, and may cre-
________________________________________
ate unanticipated side ef ects, patients should
34
35

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"Does taking alkylating agents such as
"Wil you be the doctor who provides my
melphalan, busulfan, and Cytoxan reduce my
ongoing care?"
suitability 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
________________________________________
treatment for my disease, what can I do now to
prepare for the procedure?"
________________________________________
________________________________________
"How many transplants has this center
performed for multiple myeloma and what are
________________________________________
the success rates?"
________________________________________
________________________________________
"When does the transplant procedure begin?"
________________________________________
________________________________________
"How long do patients transplanted in your
center live after the transplant itself? How does
"What drugs wil be prescribed for use before,
this compare with national averages?"
during, and after the transplant? What do they
do and what are their side effects?"
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
"Wil you be the doctor who performs the
transplant and who are the other members of
"How long is the entire treatment cycle, from
the team?"
preparation for the transplant to recovery?"
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
________________________________________
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37

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"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
________________________________________
Myeloma is a lit le-known, complex, and often
"How wil the transplant procedure affect my
misdiagnosed bone marrow cancer that at acks
ability to function? How can I expect to feel
and destroys bone. Myeloma af ects approxi-
during and after the transplant?"
mately 75,000 to 100,000 people in the
United States, with approximately 20,000 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.
________________________________________
The International Myeloma Foundation (IMF)
"What side effects of my transplant should I
was founded in 1990 by Brian and Susie
anticipate?"
Novis shortly after Brian's myeloma diagno-
________________________________________
sis at the age of 33. It was Brian's dream
that future patients would have easy access
________________________________________
to medical information and emotional sup-
________________________________________
port throughout their bat le with myeloma.
He established the IMF with the 3 goals of
________________________________________
treatment, education, and research. He sought
"What are the risks of the transplant procedure?
to provide a broad spectrum of services for
Is there a high survival rate for high-dose therapy
patients, their families, friends, and health
with stem cel transplant?
care providers. Although Brian died 4 years
________________________________________
after his initial diagnosis, his dream didn't.
Today, the IMF reaches out to an interna-
________________________________________
tional membership of more than 195,000.
________________________________________
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
38
39

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management of myeloma. The IMF ensures that
SUPPORT
no one must brave the myeloma bat le alone.
MyElOMa HOTliNE: 800-452-cURE (2873)
Tol -free throughout the United States and Canada,
We care for patients today, while working
the IMF Hotline is staf ed by trained information
toward tomorrow's cure.
specialists and is in frequent interaction with mem-
bers of our Scientific Advisory Board.
How Can the IMF Help?
SUPPORT GROUPS
PaTiENT EdUcaTiON
A worldwide network of more than 100 myeloma
support groups holds regular meetings for mem-
iNFORMaTiON PacKaGE
bers of the myeloma community. The IMF con-
Our free IMF InfoPack provides comprehensive
ducts annual retreats for myeloma support group
information about myeloma, treatment options,
leaders.
disease management, and IMF services. It includes
our acclaimed Patient Handbook.
RESEaRcH
BaNK ON a cURE®
iNTERNET accESS
This DNA bank wil provide genetic data for
Log on to myeloma.org for 24-hour access to
research in new drug development.
information about myeloma, the IMF, education,
and support programs.
iNTERNaTiONal MyElOMa WORKiNG GROUP (iMWG)
IMF's International Myeloma Working Group
ONliNE MyElOMa FORUM
consists of 145 leading myeloma researchers
Join the IMF Internet Discussion Group at www.
from around the world who col aborate on
myeloma.org/listserve.html to share your thoughts
a broad range of myeloma research projects.
and experiences.
With a goal to improve myeloma treatment
options and diagnostic systems, their work focus-
MyElOMa MiNUTE
es on protocols to provide a more durable
Subscribe to this free weekly email newslet er for
remission for myeloma patients while improv-
up-to-the-minute information about myeloma.
ing quality of life, addressing the needs of
PaTiENT & FaMily SEMiNaRS
both myeloma patients and the physicians who
Meet with leading experts in myeloma treatment
treat them.
to learn more about recent advances in therapy
THE iNTERNaTiONal STaGiNG SySTEM (iSS)
and research.
This updated staging system for myeloma wil
enhance physicians' ability to select the most
MyElOMa MaTRiX
appropriate treatment for each patient.
On our website and in print, this document is a
comprehensive guide to drugs in development for
RESEaRcH GRaNTS
myeloma.
Leading the world in col aborative research and
achieving extraordinary results, the IMF Grant
MyElOMa TOday NEWSlETTER
Program supports both junior and senior research-
Our quarterly newslet er is available free of
ers working on a broad spectrum of projects. The
charge by subscription.
IMF has at racted many young investigators into
the field of myeloma who remain in the field and
actively pursue a cure for the disease.
40
41

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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 (2M): A smal protein found in
Al ogeneic (al ograft) stem cel transplant: Refers to stem cel s
the blood. High levels occur in patients with active
that are taken from a donor and given to the patient.
myeloma. Low or normal levels occur in patients with
Most al ogeneic stem cel transplants are performed
early myeloma and/or inactive disease. Approximately
using donor peripheral blood stem cel s. Conventional
10% of patients have myeloma that does not produce
al ogeneic transplants for myeloma patients are rarely
beta-2 microglobulin. For these patients, beta-2 micro-
performed in the US due to excessive risk to the patient.
globulin testing cannot be used to monitor the disease.
A newer and, for myeloma, safer technique (discussed
At the time of relapse, beta-2 microglobulin can increase
below) is a non-myeloablative or "mini-transplant" that is
before there is any change in the myeloma protein level.
currently being evaluated in clinical trials. To determine
Therefore, 90% of the time, beta-2 testing is very useful
if a patient has a potential donor match, a special blood
for determining disease activity.
test cal HLA testing is done. A donor may be a family
Bone marrow:
member or may be obtained through a donor registry
A soft spongy tissue found in most large
bones that produces red and white blood cel s and
such as the National Marrow Donor Program (NMDP).
platelets.
Rarely, donor cel s may be obtained from an umbilical
cord blood bank.
Blood stem cel s: Stem cel s, derived from the blood, that
result in faster hematologic recovery.
Anemia: A decrease in the normal number of red blood
cel s, usual y below 10 g/dL with over 13­14 g/dL
Bone marrow aspiration: The removal, by needle, of a
being normal. Myeloma in the bone marrow blocks red
sample of tissue from the bone marrow.
cel production, thus causing anemia, the symptoms of
which are shortness of breath, weakness, and tiredness.
Bone marrow transplant: Obtained from the bone marrow
of the patient or the donor. Very few bone marrow trans-
Apheresis/Leukapheresis: Sometimes cal ed leukapheresis,
plants are currently performed due to the availability of
apheresis is a procedure in which blood is taken from a
peripheral blood stem cel s. Rarely, bone marrow may
patient or donor and the portion of the blood containing
be col ected for patients who are unable to col ect suf-
plasma, white blood cel s, and platelets is separated.
ficient number of stem cel s from the peripheral blood.
Red blood cel s are transfused back into the donor. The
CD34+: This is the laboratory marker used to single out
portion containing white blood cel s contains the rare
and quantify the number of stem cel s in your blood
stem cel s.
stream. There is a certain minimum number of CD34+
Autologous peripheral blood transplantation: (see Autologous
stem cel s that are required to safely support a transplant
Stem Cel Transplantation) A procedure in which a
procedure.
patient's blood is col ected by apheresis, stored, and
Chemotherapy: Drugs that are used to kil cancer cel s.
re-infused fol owing high-dose chemotherapy.
42
43

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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.
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Stem cel (hematopoietic stem cel ): Normal (hematopoietic,
Bibliography
or blood-making) stem cel s give rise to normal blood
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components, including red cel s, white cel s, and plate-
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lets. These stem cel s are normal y located in the bone
and chemotherapy in multiple myeloma. Intergroupe Francais
marrow and can be harvested for a transplant.
du Myelome. N Engl J Med. 1996;335:91-97.
2. Fermand JP, Ravaud P, Chevret S, et al. High-dose
Stem cel selection: A cel processing technology that is
therapy and autologous peripheral blood stem cel trans-
used to obtain a stem cel enriched product and thereby
plantation in multiple myeloma: up-front or rescue treatment?
reduce cancer cel s in the transplant. Not used success-
Results of a multicenter sequential randomized trial. Blood.
1998;92:3131-3136.
ful y for myeloma patients.
3. Barlogie B, Jagannath S. Desikan KR< et al. Total therapy
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with tandem transplants for newly diagnosed multiple myelo-
taken from an identical twin of the patient.
ma. Blood. 1999;93:55-65.
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5. At al M, Harousseau JL, Facon T, et al. Single versus double
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to 6 month intervals between transplants.
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6. Fassas AB, Van Rhee F, Tricot G. Predicting long-term sur-
Transplantation (transplant): Stem cel s are used to rescue
vival in multiple myeloma patients fol owing autotransplants.
the patient's blood-forming potential fol owing a very
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high-dose chemotherapy and/or radiation treatment.
7. Blade J. Transplantation for multiple myeloma: who, when,
Transplant is not a treatment but a method of support to
how often? High-dose therapy in multiple myeloma. Blood.
make high-dose chemotherapy treatment possible.
2003;15:3469-3477.
8. Barlogie B, Tricot G, Anaissie E, et al. Thalidomide
Umbilical cord blood transplant: Stem cel s obtained from
and hematopoietic-cel transplantation for multiple myeloma.
the umbilical cords of newborns. These are frozen and
N Engl J Med. 2006;354:1021-30.
stored in cord blood banks.
9. Cavo M, Baccarani M. The changing landscape of myelo-
ma therapy. N Engl J Med. 2006;354:1076-78.
Very good partial response (VGPR): A response that is not
10. Barlogie B, Tricot G. Complete response in myeloma: a
quite a complete response (that is, not 100%), but has a
Trojan horse? Blood. 2006;108:2134.
90% or greater reduction in serum M-protein.
11. Kumar S, Giralt S, Stadtmauer EA, et al. Mobilization in
White blood cel : One of the three major cel types in the
myeloma revisited: IMWG consensus perspectives on stem
cel col ection fol owing initial therapy with thalidomide-,
blood. There are several types of white cel s (i.e., neutro-
lenalidomide-, or bortezomib-containing regimens. Blood
phils, lymphocytes, and monocytes).
2009;114:17-29-1735.
12 Barlogie B, At al M, Crowley J, et al. Long-term fol ow-up
of autotransplantation trials for multiple myeloma: update
of protocols conducted by the Intergroupe Francophone du
Myelome, Southwest Oncology Group, and University of
Arkansas for Medical Sciences.
The IMF would like to thank
Dr. David Vesole and Dr. Brian Durie
for their help in preparing this publication.
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