The basics
Published by the International Myeloma Foundation (UK) August 2000
9 Gayfield Square
Edinburgh
AL
EH1 3NT
Tel: (44) 0131 557 3332
Freephone Support Line: 0800 980 3332
Fax: (44) 0131 556 9720
E-mail: TheIMF@myeloma.org.uk
Website: www.myeloma.org.uk
Amyloidosis
0104
Charity Registration Number: SC 026116
332
Company Number: 190563
l.0131eT
The International Myeloma Foundation (UK)
UK.
is affiliated to the International Myeloma Foundation
Edinburgh,
Consultants,
Design
Kirkhill
Design:
Contents
p 1
Introduction
p 2
What is Amyloidosis?
p 3
AL Amyloidosis
p 5
AL Amyloidosis and Myeloma
p 6
Common features of Amyloidosis at time of presentation
p 6
Specific symptoms
p 7
Other common symptoms
p 8
Initial diagnosis
p 9
Investigations to confirm diagnosis
p 12
Prognostic factors
p 12
Sub types of AL Amyloidosis
p 13
Treatment
p 18
Other treatments
p 19
Treatment directed at relieving symptoms
p 19
Research and future treatment options
p 20
What results should one expect from treatment?
p 21
How should one choose among available treatments?
p 23
Conclusion
p 25
Self-help
p 26
What can I do to help myself?
p 29
The Future
p 31
Other useful organisations
p 33
Terms and Definitions
Introduction
AL Amyloidosis is a relatively rare disease. Most patients
have never heard of Amyloidosis at the time of their
diagnosis. Initial reactions tend to be a combination of
fear of the unknown and confusion about all the new
information and terminology that they are hearing.
Information in libraries or on the internet is frequently
more confusing and can often be misleading. Much
medical information is highly technical and not relevant
to the patient's needs.
This handbook is intended to improve the situation, to provide a basic
understanding of the disease sufficient to allow patients to make informed
decisions about treatment choices. This handbook's aim is to supplement
the information given by the doctor. Caregivers, family and friends may also
find this information useful.
1
AL Amyloidosis is potentially a very serious condition, left untreated it is life
AL Amyloidosis
threatening. Although there is no treatment that can cure AL Amyloidosis
In some forms of Amyloidosis the cause is secondary to another disease.
there are different effective treatment options that in many cases can lead
However in the case of AL Amyloidosis it is the Amyloidosis which is the
to a full and durable remission. Patients have gone on to have full and
primary cause and it is not associated with another disease, hence AL
active lives. With increasing research the overall outlook for AL Amyloidosis
Amyloidosis used to be called primary Amyloidosis. It is the most
is steadily improving. Knowing more about the disease and understanding
commonly diagnosed form of systemic Amyloidosis. Systemic means that
what can be done to help reduces anxieties and makes it easier to come
it is throughout the body system. In rare cases AL Amyloidosis can be
to terms with the diagnosis.
localised.
What is Amyloidosis?
Typically AL Amyloidosis affects people in their 5th and 6th decades of life,
but people in their 20's and 30's have been diagnosed. AL Amyloidosis is
Amyloidosis is a condition where amyloid is produced. Amyloid is an
not contagious or inherited.
abnormal protein that may be deposited in any of the body's tissues or
organs.
AL Amyloidosis is a plasma cell disorder. Plasma cells are normally present
in the bone marrow and are a critical part of the body's immune system.
The term Amyloidosis describes a group of disorders, it is not a single
We need plasma cells to survive, they are responsible for making
disease. There are over a dozen different types of Amyloidosis, each being
antibodies, also called immunoglobulins, which are proteins involved in the
associated with the deposition of a certain type of protein. The three most
body's defence against infection. Normally after they have served their
common are AL Amyloidosis (primary systemic), AA Amyloidosis
function these antibodies are broken down and recycled by the body.
(secondary systemic), and hereditary Amyloidosis (ATTR).
This booklet focuses on AL Amyloidosis.
2
3
Patients with AL Amyloidosis usually have an abnormal line of plasma cells
Amyloid deposits can build up virtually anywhere in the body or remain
in the bone marrow. For unknown reasons a single population, or clone, of
quite localised. Each patient has a distinct pattern of amyloid deposition
plasma cells grows excessively. Because they result from a single cell this
with predominant involvement of either kidney, heart, liver, spleen, small
is known as a monoclonal plasma cell disorder. In 80% of patients the
intestine, tongue, or nerve. However AL Amyloidosis does not affect the
abnormal growth of plasma cells is very subtle and this would not give rise
brain. The build up of amyloid in tissues gradually causes damage and
to any immediate illness itself. The abnormal plasma cells produce an
illness by interfering with the structure and function of normal tissues.
amyloid forming protein that cannot be broken down by the body. These
Careful evaluation is therefore crucial as a basis for management
proteins then build up in the blood stream. Ultimately they leave the
decisions.
bloodstream and can deposit in tissue as amyloid.
AL Amyloidosis and Myeloma
The protein represents a portion of the antibody molecule called the light-
chain; thus when this material deposits as amyloid the resulting condition is
Although AL Amyloidosis is not considered to be a cancer it is closely
called amyloid light-chain or AL Amyloidosis.
related to a bone marrow cancer called myeloma. 10-15% of myeloma
patients have co-existing AL Amyloidosis most are first diagnosed with
When examined with an electron microscope, amyloid appears as rigid
myeloma. It is unusual though not unknown for patients with AL
fibrils. The material has an unusual structure, namely these proteins
Amyloidosis to progress to myeloma at a later date.
assemble as pleated sheets, much like silk fibres. This feature renders
amyloid exceptionally insoluble.
Although most patients with AL Amyloidosis do not actually have myeloma
but a low-grade cousin of it, the treatment of AL Amyloidosis is often
exactly the same as for myeloma.
4
5
Common features of Amyloidosis at time of presentation
Shortness of breath. Amyloid deposits in the heart can cause the heart to
become unusually stiff. The most common symptom is shortness of
Symptoms vary widely. Each patient has a set of symptoms that is
breath, noticeable even with the slightest exertion. Amyloidosis can affect
particular to the amyloid distribution in their body. However there are a
the electrical system of your heart, causing the heart beat to be disturbed.
number of symptoms that are commonly seen, a patient may be affected
by some or all of the following symptoms.
Nerves can also be affected, giving rise to pain, numbness, and tingling
fingers. This is known as carpel tunnel syndrome. Nerves that control
Non specific symptoms are common and may have been present for
blood pressure can cause dizziness on standing too quickly. Nerves
sometime before diagnosis. Sometimes termed general malaise they
elsewhere can be affected.
include tiredness, weakness, weight loss, and loss of appetite.
Other common symptoms include
Specific symptoms
· Diarrhoea
Swelling of ankles and legs (oedema) is caused by amyloid deposits in the
kidneys affecting their filtering system. This can cause them to leak healthy
· Enlarged tongue (macroglossia)
blood protein into the urine, known as nephrotic syndrome, this causes
· Feeling of fullness in the stomach after eating small amounts.
water to leak out of the blood vessels into the feet and lower legs causing
swelling. For unknown reasons this loss of protein results in
unexplained rises in blood cholesterol. In some cases the amyloid will
cause the kidneys to lose the ability to purify the blood, this is known as
renal failure.
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7
Initial diagnosis
Diagnosis is often made after the patient has symptoms
of organ failure. As the use of blood and urine testing
increases for screening purposes, some cases may be
diagnosed before any major symptoms have emerged.
Thus providing the opportunity to treat the disease
before organ involvement occurs.
Investigations to confirm diagnosis
There are a number of tests that may be done, some to confirm the
diagnosis and others to investigate the extent to which you are affected by
the disease. All of these studies help to create a picture of the exact
condition and which organs are affected in the individual. You may have
some or all of the following tests.
9
Tissue biopsies. A biopsy of an affected organ may be done. A biopsy
A SAP scan is used in some hospitals as a diagnostic tool and to
may be taken from the liver, heart, nerves, kidneys or fat. Regardless of the
show where the amyloid deposits are in the body and how much amyloid
organ predominately affected by amyloid deposits, this material is typically
is present. The scan also allows amyloid to be monitored after treatment
present throughout the body. A fat aspirate may be done where a small
has been given. The SAP scan uses a nuclear medicine scanning
amount of fat is removed from under the skin of the abdomen using a
procedure. Scans are done 6 24 hours after an intravenous injection of a
syringe, this is a relatively painless procedure. When seen through a
radiolabelled tracer that binds to the amyloid deposits. This can be used to
microscope with polarised light amyloid fibrils can readily be detected in
ascertain the full extent of amyloid deposition in the body.
Congo red-stained tissue biopsies.
Extensive standard tests will be carried out to ascertain which organs are
A bone marrow sample will be taken to establish the presence of abnormal
involved and the degree to which they are. When a patient is diagnosed
plasma cells. In some patients due to the comparatively small number of
with AL Amyloidosis it is important to evaluate the extent of the disease.
abnormal plasma cells they will not be detected in the biopsy.
These tests may include:
Blood serum and urine immunofixation tests are done to identify the
· Echo cardiograms and ECG's for the heart.
monoclonal protein.
· CT and MRI scans for the liver and kidneys.
· Blood and urine chemistry work ups, which may also detect the amyloid
forming protein.
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Prognostic factors
Treatment
The course of the disease very much varies for each patient. A full
assessment of each patient is important so that an appropriate treatment
programme can be developed. The dominant organ that is involved
influences life expectancy. In addition, patients can have 2 or more organs
involved. Aggressive treatment is much better tolerated by patients without
dominant heart involvement and with only 1 or 2 major systems involved (of
the 4 majors: heart, kidneys, liver/GI, nervous system).
Sub types of AL Amyloidosis
The standard typing of AL Amyloidosis depends on the part of light chain
involved. Thus the sub-type may be lambda or kappa. Lambda appears to
be more prevalent. At present it is not known what differences this might
cause.
12
At present the treatment of AL Amyloidosis is limited.
Chemotherapy
The main approach to treatment is two fold:
Chemotherapy destroys the abnormal (amyloid producing) plasma cells
with the aim of inducing remission or cure. The drugs can be administered
1. Treatment is aimed at reducing or eliminating the bone marrow disorder
either via injection (intravenously) or by mouth (orally). Chemotherapy
i.e. the plasma cells that are responsible for producing the amyloid, thereby
regimens (treatment programmes) generally stretch over a period of
limiting or halting the production of amyloid. These treatments involve the
months. Most often, they are administered on an out patient basis.
use of chemotherapy.
The drugs are administered in cycles giving the patient's immune system
When the production of amyloid is reduced the existing amyloid deposits
and normal bone marrow cells, which are weakened by the chemotherapy,
may slowly regress, alleviating some if not all of the symptoms.
time to recover between cycles.
2. Treatment may be required to alleviate symptoms caused by the
Chemotherapy is said to be effective if it reduces the abnormal blood/urine
deposition and build-up of amyloid in various organs. The treatment in this
protein levels and or the percentage of abnormal plasma cells observed
case is specific to the organ involved.
in the bone marrow. It may be considered successful even if it does not
induce full remission (return to normal levels). There are many chemotherapy
options involving different combinations and dosages of drugs.
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Side Effects of Chemotherapy
Bone Marrow Transplant (BMT) and
Peripheral Blood Stem Cell Transplant (PBSCT)
General side effects of chemotherapy can include hair loss, nausea and
vomiting, increased risk of bruising, bleeding, infections, mouth sores and
Transplants are being evaluated in clinical trials as alternative to
ulcers. With some chemotherapy agents abdominal cramps, constipation,
conventional treatment and as potential cures for AL Amyloidosis. However,
numbness and tingling in the hands or feet may be experienced. Fertility
no type of transplant is definitely curative for Amyloidosis. Typically
may also be affected.
transplantation involves extremely high dose chemotherapy. The therapy is
so potent that it destroys all the patient's bone marrow. Without the bone
Not everyone will experience all these side effects, this will depend on a
marrow there is no immune system and no ability to manufacture new
number of factors, such as the dose given and the person's general
blood cells so the patient cannot live. The marrow transplant procedure
health. Your doctor will be able to tell you about the side effects that may
replaces the destroyed marrow, rescuing the patient. Thus, the marrow
be caused by the particular chemotherapy you will have. People often have
transplant procedure is a means of administering treatment doses that
preconceptions about chemotherapy, many of which may be false. In
would otherwise prove fatal. It is hoped that by destroying the marrow, all
reality many of the side effects you may have can be minimised or
of the abnormal plasma cells will also be destroyed. The transplanted
alleviated.
marrow is drawn from a genetically matched donor (allogeneic transplant)
or from the patient (autologous transplant). When the patient's own marrow
is used, it can be purged using chemicals or antibodies designed to
remove the abnormal cells before it is transplanted. The transplant might
involve a hospital stay (weeks to months) followed by a period of reduced
activity. Transplants are by far the most aggressive treatment programmes
in use today and they also have the highest level of risk. Transplants have
16
17
only been done relatively recently for AL Amyloidosis thus, though
Treatment directed at relieving symptoms
apparently beneficial for many patients, the long term impact has yet to be
Pain: A whole range of medications and procedures are available to relieve
established.
pain. With currently available pain management strategies no patient should
suffer needlessly. Many centres now have specialised pain management
Other treatments
teams.
If an organ is severely compromised, slowing the production of amyloid
Infection: Antibiotics and vaccines can play a role in preventing and
may not be sufficient.
combating opportunistic infections.
Renal Dialysis may be recommended in some cases where the kidneys
Other Medications: Other medications and supplements should be taken
are no longer able to carry out their job of purging the blood of toxins.
with care when under treatment for AL Amyloidosis. Ideally no additional
Organ transplants have been carried out in a number of cases where the
medications or supplements should be taken without the advice of your
damage caused to organs is considered to be permanent. If a patient has
doctor, even everyday vitamin supplements.
a liver or kidney transplant further treatment will be required to stop the
abnormal plasma cells producing more amyloid and affecting the new
Research and future treatment options
organ.
At present all treatment is aimed at eliminating the abnormal plasma clone.
There is as yet no drug that dissolves or disperses the amyloid deposits.
Much research is being done, and in the near future it is hoped that
treatments will be available that will be capable of promoting amyloid
breakdown and reabsorbtion. Treatments are fast developing and the
outlook for amyloid patients is improving all the time.
18
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What results should one expect from treatment?
How should one choose among available treatments?
1. Stabilising Countering the life threatening affects of the disease that
Treatment recommendations are received when the patient is first
can occur with AL Amyloidosis.
diagnosed or upon discovery of a relapse. Understandably the patient is
upset and often not well informed about the disease or the treatment
2. Palliative Relieving discomfort and increasing the patient's ability to
options. The situation is generally charged with emotion and does not lend
function normally.
itself to complex, technical explanations and careful contemplation of
3. Remission Lessening the severity of the symptoms, slowing or
alternatives. Although it may be important to start treatment soon, the
temporarily arresting the course of the disease.
doctors will understand that you need time and support coming to terms
with the diagnosis and making the decisions about treatment. If you need
4. Cure Achieving a permanent remission (this has rarely, if ever, been
to make a treatment decision the first rule is to stop and think.
achieved and confirmed). To say it another way, treatment is given to make
the patient feel better and function better. It may also control the effects of
Other than crisis intervention to deal with acute life-threatening symptoms
the disease on normal body function, slow the disease down or halt it.
there are few decisions that cannot wait long enough for a second opinion
Remission can last from months to decades. Experimental treatments aim
or personal reflection. Also, certain treatment decisions can rule out future
at a cure, although none has yet been confirmed. When the production of
treatment options. For example, certain types of chemotherapy should not
amyloid is slowed or halted, in many cases the existing amyloid deposits
be used if an autologous bone marrow transplant is likely in the future.
regress, and therefore the patient's symptoms may be alleviated.
This is NOT to suggest that patients refuse critical treatment.
20
21
However, it is important to ask your doctor which treatment decisions are
Conclusion
critical and which can wait. When the situation permits, take the time to get
To make an informed decision the patient needs to have all the facts.
more than one opinion before beginning a treatment programme.
Much of what is written about AL Amyloidosis is written by doctors and
When talking about treatments, start by understanding the objectives.
researchers to be read by other doctors and researchers. As a result, the
Treatment recommendations typically include multiple components, each
literature tends to be very difficult to read for the patient and other
with different objectives. Often, certain elements of the treatment
concerned lay persons.
programme are more urgent than others and require quicker decisions.
As such, the doctor(s) treating the patient bear the burden of education.
Others aim more at long-term management of the disease, allowing more
Yet doctors must take their cues from the patient and family on how far to
time to decide.
go on this front. Some patients are fiercely curious and want to discuss all
Beyond some tried and true stabilising and palliative treatments there are
aspects of their situation, treatment and prognosis. Others are
very few absolutes in Amyloidosis treatment. For example bone marrow or
overwhelmed and just want to know what to do next.
stem cell transplantation is not an absolute requirement even when feasible
Most doctors are sensitive to this and will vary their approach based on
in a young and otherwise healthy patient. Remission inducing treatments,
what they perceive to be the patient's wishes. Patients can shortcut the
typically chemotherapy programmes, cannot guarantee results. Doctors
process by being very explicit about how deeply they want to get into the
have information on success rates and can use different tests to help
details of the treatment decisions.
choose the programmes that are best suited to the individual. The same
can also be said of bone marrow transplants, which aim at cure.
Treatment decisions are critically important to the survival and quality of life
of the AL Amyloid patient. No matter how comfortable the patient feels with
a doctor, it is generally good practice to get more than one opinion before
proceeding.
22
23
Because the disease is rare, there are a limited number of practitioners and
research/treatment centres specialising in Amyloidosis. Doctors understand
Self-help
this and will be helpful in identifying appropriate specialists and making
referrals. It is very common to seek a second opinion from a specialist at a
research centre and to continue to rely on your local referring physician to
administer and monitor treatment.
Making good decisions on amyloid treatment requires resourcefulness,
careful questioning, serious thought and courage. Most of all it requires
that the patient and his/her support group take charge of the process.
24
What can I do to help myself?
2. Past experience
Start by asking questions, here are a few examples:
Find out how well the treatment has worked for others in similar situations.
Effectiveness is usually measured in many different ways. The questions to
1. Treatment programme
ask here are:
Get a complete description:
How much experience is there with the treatment?
What are the objectives of the treatment?
How many patients have received the treatment?
What exactly is the treatment?
How long have those been followed after their treatment?
Over what period will it be given?
What is the likelihood of achieving a complete or partial remission?
What is involved?
How long have other patient's remissions lasted?
How often must a patient visit a medical facility?
In the event of a relapse, what would the options be, recognising that
Is hospitalisation required or a possibility?
these may change in the interim?
What is the likely impact on the patient's ability to function e.g. work
What are reasonable expectations for relieving symptoms such as bone
and play?
pain, pathological fractures, anaemia, fatigue and hypercalcaemia?
How do people feel before, during and after treatment?
What factors are seen as an influence to outcomes?
How long will they be undergoing treatment?
How long have people who have received the treatment survived?
What are typical recovery time frames?
For newer treatments:
What follow-up or maintenance programmes are required?
How many of the original group of patients are still alive?
26
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3. Find out about side-effects
4. Find out about alternatives
Amyloidosis treatments generally use strong drugs and other measures
Perhaps the most important line of questioning is about alternatives. There
aimed at destroying malignant cells and/or re-balancing body chemistry.
are always alternatives. You need to ask all of these questions for each of
Typically, there are side effects. Some manifest themselves during
the alternatives:
treatment. Others may show up well after the treatment. Questions to ask
What are the alternatives to the treatment recommended?
here are:
What are the relative pros and cons of the alternatives?
What side-effects have been observed in patients receiving the treatment?
What are the pros and cons of the alternative treatments versus no
When do they typically occur?
treatment?
How often do they occur i.e. what percentage of patients are affected?
How serious are these effects, are they life threatening, are they painful, are
The Future
they permanent, how long do they last?
The outlook for a person with AL Amyloidosis is continually
Are there treatments for the side effects, do they have side effects?
improving. Researchers continue to look for better ways to
diagnose and treat Amyloidosis, their knowledge about the
disease is growing all the time. Many of these exciting research
projects are being supported by the International Myeloma
Foundation all over the world.
28
29
International Myeloma Foundation (UK)
IMF(UK) are dedicated to improving the quality of life of Myeloma patients
while working towards prevention and a cure. The Foundation provides a
Other useful
number of support, informational and educational services to patients, their
families/carers and health care professionals as well as raising money to
fund important research. These important services are overseen by a
organisations
Management Board which is guided by expert Scientific and Patient
Advisory Boards.
We encourage you to make use of the services the Foundation provides
and hope you will contact us in the future for more information.
30
International Myeloma Foundation
Leukaemia Research Fund
Terms and Definitions
International Headquarters
43 Great Ormond Street
12650 Riverside Drive
London
Albumin: A protein which constitutes more
Ascites: The accumulation of fluid in the
Suite 206
WC1N 3JJ
than half of the blood's serum. Where amyloid
spaces between tissues and organs in the
North Hollywood CA, 91607
Tel: 020 7405 0101
has affected the kidney's filtration system
cavity of the abdomen.
Tel: 00 1 818 487 7455
Fax: 020 7242 1488
albumin may pass into the urine.
Bence Jones Protein: The name used to
Fax: 00 1 818 487 7454
E-mail: lrf@leukaemia.demon.co.uk
Alkylating Agent: A chemotherapeutic agent,
identify immunoglobulin or antibody fragments
E-mail: TheIMF@myeloma.org
such as melphalan, which can react with DNA
(kappa or lambda light chains) present in the
Website: www.myeloma.org
in such a way as to block cell division.
urine.
CancerBACUP
Amyloid: An abnormal protein that may be
Biopsy: The removal of a small sample of
3 Bath Place
deposited in any of the body's tissues or
living tissue for diagnostic purposes.
Rivington Street
organs.
London
Bone Marrow: The soft spongy tissue found in
EC2A 3DR
Amyloidosis: A condition where amyloid is
the centre of bones which form the blood
Office: 020 7696 9003
produced.
cells.
Fax: 020 7696 9002
Anaemia: A below-normal number of red
Bone Marrow Aspiration: The removal, by
Cancer Information Freephone: 0808 800 1234
blood cells.
needle, of fluid and cells from the bone
Cancerlink
marrow.
11-21 Northdown Street
Antibiotics: Drugs used to fight bacterial
London N1 9BN
infections.
Bone Marrow Biopsy: The removal, by needle,
Tel: 020 7833 2818
of a sample of tissue from the bone marrow.
Antibodies: Proteins produced by white blood
Fax: 020 7833 4963
cells to fight against antigens.
Carpal Tunnel Syndrome: A condition where
E-mail: cancerlink@cancerlink.org.uk
there is a compression of the median nerve in
Freephone Cancerlink: 0808 808 0000
Antigens: Any substance that the body
the wrist as the nerve passes through the
regards as foreign or potentially dangerous,
carpal tunnel. Causing pain, numbness, or
which can be as diverse as bacteria and
tingling in the fingers.
pollen grains, against which it produces an
antibody.
Cells: The individual units from which tissues
are formed.
Anti-emetics: Drugs to prevent or minimise
nausea and vomiting.
Clinical: Refers to the treatment of humans, as
opposed to animals or laboratory studies.
Asymptomatic: Without symptoms.
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Chemotherapy: The treatment of disease by
DNA: (Deoxyribonucleic acid) The genetic
Gene: A unit of genetic material (DNA) that
Kidney Failure: Malfunction of the kidneys due
chemical means.
material which controls hereditary.
carries the directions a cell uses to perform a
to disease or the toxic effects of drugs or
specific function, such as making a given
chemicals. Urine volume may or may not be
Clinical Trials: The procedure in which new
Echo Cardiogram: A test which uses high-
protein.
diminished.
treatments are tested in humans following
frequency sound waves to image the heart
favourable results in laboratory experiments.
and surrounding tissues.
Genetic: Inherited, having to do with
Light Chain: The lighter of two types of chains
information that is passed from parents to
that are found in immunoglobulins. There are
Congestive Heart Failure: A condition where
Electrocardiogram (ECG): Recording of the
their children through DNA.
two types of light chain, kappa and lambda,
ineffective pumping of the heart leads to an
electrical activity of the heart on a moving strip
Amyloidosis may be described as kappa light
accumulation of fluid in the lungs.
of paper.
Haemoglobin: The protein found in red blood
chain or lambda light chain Amyloidosis.
cells that carries oxygen and gives blood its
Congo Red: A dye used for the detection of
Electropheresis: A laboratory test in which a
red colour.
Macroglossia: Enlargement of the tongue.
Amyloidosis through its ability to stain the
patient's serum is subjected to a separation
abnormal amyloid deposits.
technique involving movement in an electric
Hepatic: Pertaining to the liver.
MRI (Magnetic Resonance Imaging): Fine
field. The technique allows the calculation of
resolution pictures of areas of the body
Creatinine Clearance: A sensitive test of
Hepatomegaly: Enlargement of the liver.
the amount of M spike (monoclonal protein)
created by use of magnetic energy rather than
kidney function that requires a 24-hour urine
characteristic of each patient. Used as a tool
Immune Response: The reactions of the
x-ray, useful for soft tissue images.
sample and a blood sample. The test is often
both for diagnosis and monitoring.
immune system to foreign substances.
required to make sure it is safe to give
M Protein: Simply means monoclonal protein
anticancer drugs that may be toxic to the
Erythropoietin: A hormone produced by the
Immune System: The complex group of cells
(or M component/ paraprotein).
kidneys.
kidneys necessary for the production of red
and organs that defend the body against
M Spike: The over production of
blood cells. Where a kidney is damaged
infection and disease.
CT or CAT scan: (Computed Tomography
immunoglobulin protein from a single clone of
injections with synthetic erythropoietin can be
scan or Computed Axial Tomography scan).
plasma cells (monoclonal). The abnormal
helpful. Blood transfusion is another alternative
Immunofixation: A technique used to identify
Detailed pictures of areas of the body created
monoclonal product is called a monoclonal or
especially in an emergency. Synthetic
specific proteins in the blood or urine. It has
by a computer linked to an x-ray machine.
M protein or paraprotein, and can show in
erythropoietin is being used prophylactically
greatest application in the identification (and
monitoring) of monoclonal proteins, such as
serum or urine. Monoclonal or "M spike" refers
Cytotoxic: Destroys cells.
before chemotherapy and as a supportive
to the sharp or spiked pattern which occurs
therapy after chemotherapy to avoid anaemia.
those produced by AL Amyloidosis.
Dexamethasone: A steroid given along with
on protein electrophoresis when an M protein
Immunoglobulins: A family of large protein
other chemotherapy drugs in the treatment of
Gamma Globulin: Proteins in the blood that
is present. In Myeloma and AL Amyloidosis a
molecules, also known as antibodies.
blood disorders.
contain antibodies, part of the body's defence
plasma cell clone gives rise to clonal
against infection.
immunoglobulins or parts of immunoglobulins
Intravenous: Into a vein, a way of injecting
Diuretic: An agent that promotes the excretion
(ie kappa or lambda light chains) which show
drugs.
of urine. Often used in the treatment of
as an M spike in the electrophoresis pattern.
oedema and high blood pressure.
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Monoclonal: A group of identical cells derived
Plasma Cell: Produced by B lymphocyte
Red Cells: One of three major types of cells in
Thrombocytopenia: A reduced level of blood
from a single cell.
(white blood cell) as a response to infection.
the blood. The blood cell which contains
platelets. The normal level is 150-400 000.
Plasma cells are found in the bone marrow,
haemoglobin and carries oxygen from the
The risk of bleeding increases significantly
Multiple Myeloma: A haematological
connective tissue, and sometimes blood.
lungs to all parts of the body. A low level of
when the platelet level is less than 20 000
malignancy in which a clone of plasma cells
Normal plasma cells produce antibodies to
red cells is called anaemia. Red cell
and becomes even higher with a reduction to
causes bone damage and pain, low blood cell
fight infection In AL Amyloidosis abnormal
production is stimulated by a hormone called
less than 10 000.
counts, increased infections and kidney
plasma cells produce abnormal antibodies
erythropoietin. Erythropoietin is produced by
damage.
Transplantation: Stem cells or bone marrow
which go onto form amyloid. The abnormal
the kidneys.
are used to rescue patient's blood-forming
Nephrotic Syndrome: An abnormal condition
antibodies are the monoclonal protein, or M
Response or Remission: Complete
potential following very high dose
of the kidney that is characterised by low
protein.
Remission or Complete Response (CR):
chemotherapy and/or radiation treatment.
serum albumin, a large amount of protein in
Plasmapheresis: The removal of certain
Remission and response are used
Transplant is not a treatment, but a method of
the urine and swelling (oedema).
proteins from the blood.
interchangeably. CR is the common
support to make high dose treatment
Neutropenia: A reduced level of neutrophils or
abbreviation for both. CR is the absence of
possible.
Platelet: One of three major blood cells, others
white blood cells. There are several types of
amyloid protein from the serum and/or urine
being red and white cells. Platelets plug up
Allogeneic: The person donating the bone
white blood cells, and neutropenia refers to a
by standard testing.
breaks in the blood vessel walls and stimulate
marrow or stem cells is another, not the
reduction in the granulocytes, or neutrophils,
blood clot formation. Platelets are the major
Partial Remission or Partial Response (PR):
patient. Usually donors are HLA identical
necessary to adequately combat bacterial
defence against bleeding.
PR is a level of response less than CR.
family members. HLA refers to the Histo-
infections. Cytoxic chemotherapy has a
compatiability Locus Antigens used for tissue
tendency to induce neutropenia. In contrast
Progression-Free Survival: The improved
Renal: pertaining to the kidneys.
matching. (Unrelated Allogeneic Transplant
lymphocytes which are more important in virus
survival of a patient that can be directly
Spleen: An organ in the abdomen that plays
the person donating is unrelated to the
infections, tend not to be affected by cytotoxic
attributed to the treatment given for the
an important role in immune system activities.
patient).
treatment.
AL Amyloidosis. The term identifies amyloid
It is part of the lymphatic system.
patients who are in complete remission versus
Autologous: The patient donates his/her
Oedema: The presence of abnormally large
those who have had an episode of relapse
SAP Scan: A nuclear medicine scanning
own stem cells or bone marrow, prior to
amounts of fluid in the intercellular tissue
(or progression).
procedure that can be used to ascertain the
treatment, for reinfusion later.
spaces of the body, causing swelling.
full extent of amyloid deposition in the body.
Radiation Therapy: Treatment with high energy
Peripheral Blood Stem Cells (PBSC): Stem
rays to kill abnormal cells.
Stem Cell: A formative cell whose daughter
cells are collected from the circulating blood
cells may give rise to other cell types including
system, not the bone marrow.
red and white blood cells and platelets. Stem
Syngenic: An identical twin donates the
cells are normally located in the bone marrow
bone marrow or stem cells.
and can be harvested for transplant.
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White Cells: One of three major types of cells
in the blood. There are several types of white
cells (i.e. neutrophils, lymphocytes and
monocytes). Neutrophils are necessary to
combat bacterial infection. Neutrophils can
drop to very low levels following
chemotherapy causing neutropenia.
Neutropenia can be prevented or reduced
using a synthetic hormone called G-CSF.
Vaccine: A drug administered for the
prevention or treatment of infectious diseases.
Vitro: In the laboratory.
Vivo: In the body.