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Aplastic anaemia
Information for patients
This leaflet explains aplastic anaemia and how it is treated. If you have any
queries or concerns, please speak to the specialist team caring for you.

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What is aplastic anaemia (AA)?
It is a rare but serious blood disorder in which your bone marrow
fails to produce enough blood cells.

What causes AA?

There are two types of AA – acquired and inherited – and the causes
of each are different.

Acquired: this is the most common type of AA. You develop

the condition and are not born with it. Acquired AA is usually
‘idiopathic’, which means we do not know the cause.

Inherited: you can inherit a faulty gene, which causes you to

develop AA later in life. Inherited AA is rare. There are a number of
types, including Fanconi anaemia. If you are born with this faulty
gene, you are most likely to develop AA between the ages of 10
and 15 or over 60.

How does my body make blood?

Your blood cells are made from special cells called stem cells. Stem
cells grow into all the different blood cells – red and white blood
cells and platelets – that your body needs.

Stem cells are made by your bone marrow, a spongy material found
in all the long bones in your body, such as your thigh, hip and breast
bones. It is your blood cell production factory.
Normal Severe aplastic
anaemia

Bone Marrow
Figure 1: A normal bone marrow
sample (trephine)
Figure 2: A bone marrow sample
Figure 1 Figure 2
lacking cells, which suggests AA.

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What do the different types of blood cells do?
• Red blood cells carry oxygen around your body.
• White blood cells do several things, depending on their type:
neutrophils fight bacterial infections, while lymphocytes help fight
viruses such as chicken pox and measles.
• Platelets stop you from bleeding and bruising.

What happens if I have AA?

Your bone marrow becomes damaged and starts to produce fewer
blood cells and platelets. Your immune system, which normally
monitors your body and helps it to fight infections and bugs,
then starts to attack your bone marrow because it thinks there
is something wrong with it, or that it is ‘foreign’ and not part of
your body. It stops your bone marrow from healing and working
normally.

Treatment for AA aims to slow down – or ‘dampen’ – your immune

system, to allow your bone marrow to heal and start producing
blood cells normally again.

What are the symptoms of AA?

If your bone marrow does not produce enough blood cells, you may
have a number of symptoms.

• A low number of red blood cells causes anaemia. This leads to

low energy levels, headaches, and shortness of breath when
you are doing moderate exercise such as going up stairs or light
work/activity. You can sometimes have palpitations, where you
notice your heartbeats and your heart may feel like it is pounding,
fluttering or beating irregularly for a few seconds or minutes. If
your anaemia is severe, you may even have chest pain. You usually
need regular blood transfusions to increase the number of red
cells in your blood and improve your energy levels.

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• A low number of white blood cells – especially neutrophils, which
fight bacterial infections – makes you more prone to fevers,
bacterial and fungal infections, and sometimes serious infections
which means you have to be admitted to hospital. Common
infections include:
o a sore throat that will not go away or keeps coming back
o skin infections
o chest infections.
• A low number of platelets can make you bruise and bleed more
easily. You often need platelet transfusions to help prevent
bleeding and bruising. Common signs include:
o bleeding gums, particularly during or after brushing your teeth
o nose bleeds
o periods that are heavy or last longer than usual
o blood blisters in your mouth
o bruised skin, sometimes when you have not knocked yourself
o a rash made up of small red spots known as ‘petechiae’ or
‘purpura’, mostly on your legs.

Figure 1 Figure 2

Some examples of bleeding caused by low platelets

Figure 1: Bruised skin on arms/hands
Figure 2: Blood blisters in the mouth

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How do you measure the severity of AA?
The degree to which your bone marrow fails can vary from mild to
very severe. This means that some people have more severe AA than
others and become more ill.

AA can be divided into three groups: non-severe aplastic anaemia

(NSAA), severe aplastic anaemia (SAA) and very severe aplastic
anaemia (VSAA). The blood counts you can expect with each
grouping are shown in the table on page 6.

Knowing the number of cells in your bone marrow, as well as

how low your blood counts are when you are diagnosed, is very
important because it helps doctors to work out the severity of your
AA and the best treatment.

Sometimes the symptoms show slowly and get worse over time
because your ability to produce stem cells gradually declines and
your degree of bone marrow failure changes.

How do you diagnose AA?

You will have a number of tests. These are mainly to make sure you
do not have any other condition that could be causing your low
blood counts.

Blood tests
We take samples of your blood to test for AA and other conditions.
They include:
• a full blood cell count, including checking the number of young
blood cells (reticulocytes)
• checking your iron, vitamin B12 and folate levels to make sure
your anaemia is not caused by nutritional problems
• screening for autoantibodies

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• checking your chromosomes for certain inherited types of AA,
such as Fanconi anaemia
• checking to see how well your liver is working and for viruses
such as hepatitis A, B and C, HIV, Epstein-Barr virus (EBV) and
parvovirus
• screening for a rare blood condition called paroxysmal nocturnal
haemoglobinuria (PNH).

AA types Blood counts and bone What this means

marrow cells
Non-severe AA (NSAA) Counts are not as low as Your blood counts are
severe/very severe AA. low but not bad enough
for you to have severe
AA. You may need
transfusions of red blood
cells but not usually
platelets. You are not
usually at risk of infection.
Severe AA (SAA) Bone marrow cells <25%, You have two of the
or 25-50% with less than following:
30% of stem cells in your • a low platelet count –
bone marrow. less than 20 (the normal
range is between 150
You have two out of the and 400)
following three: • a young red blood
• platelets <20 x 109/L cell (reticulocyte)
• reticulocyte <60* x count of less than 25
109/L – the normal range is
• neutrophils <0.5 x between 50 and 150
109/L. • an absolute neutrophil
count of less than
0.5 – the normal range
is between 2 - 6.5.
Very severe AA (VSAA) Same as severe AA but The same as for severe
neutrophils <0.2 x 109/L. AA but your absolute
neutrophil count is very
low, at less than 0.2.
*60x109/L using automated reticulocyte count, 20 x 109/L using manual
reticulocyte count

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Bone marrow tests
We take a sample of your bone marrow from inside one of your
bones – usually the hip bone – using a needle (bone marrow
aspirate) along with a sample of the bone itself (bone marrow
trephine). We test these samples to establish a diagnosis of AA.

How is AA treated?
There are two types of treatment:
(i) Supportive therapy treats the symptoms of AA. If you have a
low red blood cell count you will have blood transfusions and if you
have a low platelet count you will have platelet transfusions. We
also give you antibiotics and antifungal drugs to prevent and treat
infections.

(ii) Definitive therapy aims to get your bone marrow working

again so you do not need blood transfusions, are no longer at risk of
bleeding or infections and can lead a fairly normal life.

It can take a few months – and sometimes longer – to see a

response to definitive therapy. It is important to continue having
regular follow-up appointments with your medical team and
supportive care during this time, as you may still be at risk of
problems with anaemia, bruising, bleeding and infections.

You will need some type of definitive therapy unless you have
non-severe AA and you do not need any blood or platelet
transfusions.

The main types of definitive therapy are:

Immunosuppressive treatment: we can give you drugs to
dampen down your immune system and allow your bone marrow to
heal, start working properly again and make all blood cells.

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The drugs – antithymocyte globulin and cyclosporine – suppress
your faulty immune system and stop it from attacking your stem
cells. This treatment gives you a good chance of long-term survival
and a near-normal to normal quality of life.

Stem cell/Bone marrow transplants: a bone marrow transplant

replaces your empty bone marrow and abnormal immune cells with
stem cells received from a donor, either a matched relative or an
unrelated donor, which then makes the blood cells and also provides
a new immune system.

Not all patients are suitable for a transplant and this treatment is
more successful in children, young adults and those who are under
50 and otherwise fit and well. But this does not mean that if you are
over 50 you cannot have a transplant. This treatment is becoming
more successful and is being assessed in patients in their 60s if they
are otherwise fit and well.

As the risk of complications after transplant increase with age, we

assess each patient individually, based on their general health and
fitness. We will discuss with you in detail all the risks and benefits.

If a matched bone marrow donor is not available, there may be

other methods that can be tried such as using cord blood stem cells
or bone marrow stem cells from a family member who is
half-matched.

Other treatments
Other treatments include anabolic steroids (androgens) and
alemtuzumab (antibody-based therapy). Growth factors for blood
cells such as erythropoietin (Epo) and G-cSF are usually not very
beneficial or effective in the treatment of AA.

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Who do I contact with queries and concerns?
If you have any queries or concerns, please contact the aplastic
anaemia and bone marrow failure team:
Consultant Haematologists
Tel 020 3299 3709 or 020 3299 1039
Clinical Nurse Specialists
Tel 020 3299 7663

Where can I get more information?

Joint AA/MDS/AML Support Group
The AA/MDS Support Group meets three times a year. These
meetings give you a chance to talk to others with rare blood
conditions and find out more. Doctors, nurses and other healthcare
professionals come along to share any new developments and
answer general questions.

For the date of the next meeting, go to the Patient Support section
on the Aplastic Anaemia Trust web site:
www.theaat.org.uk

Leukaemia CARE
www.leukaemiacare.org.uk

This leaflet was written by the following members of King’s College

Hospital NHS Foundation Trust’s Department of Haematological
Medicine: Nana Benson-Quarm, CNS, AA and Bone Marrow Failure
Syndromes; Professor Judith Marsh, Professor of Clinical Haematology,
Lead Consultant; Dr Austin Kulasekararaj, Consultant Haematologist;
and Dr Shreyans Gandhi, Senior Clinical Research Fellow.

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Sharing your information
We have teamed up with Guy’s and St Thomas’ Hospitals in a
partnership known as King’s Health Partners Academic Health
Sciences Centre. We are working together to give our patients the
best possible care, so you might find we invite you for appointments
at Guy’s or St Thomas’. To make sure everyone you meet always has
the most up-to-date information about your health, we may share
information about you between the hospitals.

Care provided by students

We provide clinical training where our students get practical
experience by treating patients. Please tell your doctor or nurse
if you do not want students to be involved in your care. Your
treatment will not be affected by your decision.

PALS
The Patient Advice and Liaison Service (PALS) is a service that offers
support, information and assistance to patients, relatives and
visitors. They can also provide help and advice if you have a concern
or complaint that staff have not been able to resolve for you. The
PALS office is located on the ground floor of the Hambleden Wing,
near the main entrance on Bessemer Road - staff will be happy to
direct you.
Tel: 020 3299 3601
Fax: 020 3299 3626
Email: kch-tr.PALS@nhs.net

You can also contact us by using our online form at

www.kch.nhs.uk/contact/pals

If you would like the information in this leaflet in a different

language or format, please contact PALS on 020 3299 1844.

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Your notes:

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www.kch.nhs.uk Corporate Comms: 0827
PL647.1 February 2015 Review date February 2018