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Fever, uncontrolled bleeding, or severe breathlessness need emergency care today. With almost no white cells, infection can become life-threatening within hours. Bleeding from very low platelets is equally urgent.

Home  /  Treatments  /  Bone marrow transplant  /  Aplastic anaemia

Aplastic anaemia

The bone marrow stops producing blood cells. Red cells, white cells and platelets all fall together, leaving the patient anaemic, vulnerable to infection and at risk of bleeding.

One practical warning matters more than anything else on this page, and it is easily missed: do not accept blood transfusions from family members if transplant is being considered.

In young patients
Transplant first
Better than drug treatment where a sibling matches
Critical warning
No family transfusions
They can cause the transplant to be rejected
Drug option
Immunosuppressive therapy
For those without a donor
Time in India
3 to 4 months
For transplant, including recovery
The condition

What aplastic anaemia is

The bone marrow's stem cells are destroyed or suppressed, and production of all three blood cell lines fails. The marrow, examined under the microscope, is largely empty of blood-forming tissue and replaced by fat.

In most cases the cause is immune — the body's own T cells attack the marrow stem cells. Some cases follow certain drugs, viral hepatitis, benzene exposure, or pregnancy. A proportion, particularly in children, are inherited conditions such as Fanconi anaemia, which must be excluded because they change treatment entirely.

Severity is graded by the blood counts, particularly the neutrophil and platelet counts and the reticulocyte count. Severe and very severe aplastic anaemia are treated urgently, because without treatment the mortality from infection and bleeding is high.

This is one of the conditions where transplant is not a last resort but a first-line treatment. In a young patient with a fully matched sibling, transplant offers a substantially better chance of long-term cure than immunosuppressive drugs, which frequently produce only partial responses and carry a risk of later relapse or transformation.

Symptoms

Symptoms and warning signs

Common symptoms
  • Fatigue, breathlessness and pallor from anaemia
  • Easy bruising, nosebleeds and bleeding gums
  • Tiny red spots on the skin, particularly on the legs
  • Frequent or persistent infections
  • Mouth ulcers and sore throat that will not settle
  • Heavy menstrual bleeding
  • In inherited forms, short stature, thumb or kidney abnormalities and skin pigmentation
Warning signs of an emergency
  • Fever of any degree
  • Bleeding that will not stop, or blood in stool or urine
  • Severe headache with vomiting — possible bleeding into the brain
  • Breathlessness at rest
  • Confusion or drowsiness
  • Rapidly falling blood counts

Do not accept blood transfusions from family members

This sounds counterintuitive and it is genuinely important. Transfusing blood from a relative exposes the patient's immune system to that relative's tissue antigens, which can lead to sensitisation and make a later transplant from that same relative fail. Families offering to donate blood are acting out of love, and the effect is the opposite of what they intend. If transplant is being considered — and in a young patient with siblings it usually should be — insist on blood from unrelated volunteer donors, and ask for it to be leucodepleted and irradiated. Tell every doctor treating the patient about this.

Diagnosis

How it is diagnosed

Blood counts suggest it; the marrow examination confirms it and excludes the alternatives.

Initial tests

  • Full blood count with reticulocyte count — all three lines low, with a low reticulocyte count showing the marrow is not responding
  • Bone marrow aspirate and trephine biopsy — the decisive test, showing an empty marrow
  • Cytogenetics — excludes myelodysplastic syndrome, which can look similar
  • Viral screening — hepatitis, HIV and others

The deciding tests

  • HLA typing of patient and all full siblings — urgent, because transplant is first-line in young patients
  • Chromosome breakage testing — for Fanconi anaemia, essential in children and young adults, because it changes the conditioning regimen completely
  • Flow cytometry for PNH clone — commonly present and affects prognosis
  • Telomere length testing — where an inherited bone marrow failure syndrome is suspected

Send the marrow report and the counts over time

We need the bone marrow biopsy report, full blood counts with reticulocytes showing the trend, cytogenetics, and — critically — whether Fanconi anaemia has been excluded in any patient under about forty. Also tell us how many transfusions have been given and whether any came from family members, because that materially affects transplant planning.

Options

Treatment options

In a young patient with a matched sibling, transplant comes first rather than last.

First choice

Matched sibling donor transplant

For younger patients with severe disease and a fully matched sibling, transplant is the treatment of choice rather than a fallback. It offers a high chance of long-term cure, whereas immunosuppressive therapy frequently produces incomplete responses, relapse, or later transformation to myelodysplasia. HLA typing the siblings is therefore urgent, not optional.

Usually appropriate whenSevere or very severe aplastic anaemia in a younger patient with a fully matched sibling.
Option two

Immunosuppressive therapy

Antithymocyte globulin combined with ciclosporin, increasingly with eltrombopag added, suppresses the immune attack on the marrow and allows recovery in a substantial proportion of patients. Response takes months, is often partial, and relapse or later clonal disease can occur. It remains the right choice where no donor exists or the patient is older.

Usually appropriate whenNo matched sibling donor, or an older patient in whom transplant risk is high.
Option three

Haploidentical or unrelated donor transplant

Where immunosuppressive therapy has failed and no matched sibling exists, transplant from a half-matched family member or a registry donor is used. Results have improved considerably and this is now a realistic second-line option rather than a last resort.

Usually appropriate whenFailure of immunosuppressive therapy, with no matched sibling available.
Alongside

Supportive care

Transfusion of red cells and platelets from unrelated donors only, leucodepleted and irradiated. Prompt antibiotics for any fever. Antifungal cover where the neutrophil count is very low. Good supportive care is what keeps patients alive while definitive treatment takes effect.

Usually appropriate whenFrom diagnosis onward, for every patient.
The decision

How the choice is made

Age and severity

Younger patients with severe disease and a matched sibling go straight to transplant. Older patients are more often treated with drugs first.

Is there a matched sibling

This is urgent in aplastic anaemia because it determines first-line treatment, not just a later option.

Has an inherited syndrome been excluded

Fanconi anaemia requires a completely different, much gentler conditioning regimen. Missing it is dangerous, and chromosome breakage testing must be done in younger patients.

If the patient is young with severe disease, we will ask urgently whether siblings have been typed. In this condition that answer changes the entire treatment plan.

Urgency

How urgent is your case

Usually safe to plan travel
  • Counts stable on supportive care
  • Diagnosis established and typing under way
  • No fever or active bleeding
  • Responding to immunosuppressive therapy
Needs local assessment before travel
  • Any fever
  • Active bleeding or very low platelets
  • Severe breathlessness
  • Rapidly falling counts
  • Suspected bleeding into the brain

We will tell you which column you are in

Severe aplastic anaemia with fever or bleeding is a haematological emergency and is treated where the patient is. Transplant planning follows stabilisation.

Next step

What to send us

Photographs taken on your phone are fine. Reports in Arabic, Russian or Bengali are fine — we translate them ourselves.

Most useful

  • Bone marrow aspirate and biopsy report
  • Full blood counts with reticulocytes, over time
  • Cytogenetics results
  • Whether Fanconi anaemia has been excluded

Also helpful

  • HLA typing for patient and siblings
  • Number of transfusions given, and whether any were from family members
  • Viral screening results
  • Details of any immunosuppressive treatment already given
Questions

Questions patients ask

Because transfusing a relative's blood exposes the patient to that relative's tissue antigens, which can sensitise the immune system and cause a later transplant from that same relative to fail. Families offer out of love and it can do real harm. Insist on unrelated volunteer donor blood, leucodepleted and irradiated, and tell every treating doctor that transplant is being considered.

In a young patient with severe disease and a fully matched sibling, no — transplant is the first-line treatment and gives a better chance of long-term cure. Immunosuppressive therapy is the right choice where no matched sibling exists or where the patient is older and transplant risk is higher. This is one of the few conditions where transplant comes first rather than last.

An inherited form of bone marrow failure that can look identical to acquired aplastic anaemia. It matters enormously because these patients cannot tolerate standard transplant conditioning and require a much gentler regimen. Chromosome breakage testing should be done in every younger patient before transplant, and missing it is dangerous.

Severe aplastic anaemia is urgent. Without treatment the mortality from infection and bleeding is high, so supportive care and a treatment decision should not be delayed. HLA typing of siblings should be arranged immediately, because it determines whether the first-line treatment is transplant or drugs.

After successful transplant, relapse of aplastic anaemia is uncommon. After immunosuppressive therapy it is a real possibility, and a proportion of patients later develop myelodysplastic syndrome or paroxysmal nocturnal haemoglobinuria. This difference in durability is a large part of why transplant is preferred in younger patients with a donor.

In most cases no cause is ever identified and the mechanism is immune. Some cases follow specific drugs, viral hepatitis, benzene or other chemical exposure, or pregnancy. A proportion, particularly in children, are inherited. Establishing whether it is inherited matters more than establishing the trigger, because it changes treatment.

Contact

Send us your reports

Send the bone marrow biopsy report and blood counts, and tell us urgently whether siblings have been HLA typed. In this condition that answer decides the first-line treatment.

Your reports go directly to our medical team. We do not share your records with hospitals until you tell us to.

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