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Fever in a child with thalassemia, particularly after splenectomy, needs assessment the same day. Also seek urgent care for breathlessness at rest, palpitations, or severe abdominal pain.

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Thalassemia major

An inherited disorder in which the body cannot make normal haemoglobin. Children need blood transfusions every few weeks for life, and the iron those transfusions leave behind slowly damages the heart, liver and endocrine glands.

Bone marrow transplant is the only treatment that cures thalassemia. Everything else manages it. And the outcome of that transplant depends heavily on how old the child is and how well the iron has been controlled — which is why this page is about acting sooner rather than later.

Clinically reviewed by Dr. Annie Varughese, Consultant Haematologist Clinical oversight Dr. Annie Varughese, MD, FACC Last reviewed August 2026 Editorial and medical review policy
Only cure
Bone marrow transplant
Everything else is lifelong management
Best results
Younger children
Before iron damage accumulates
First step
HLA type the siblings
A blood test, usually possible at home
Time in India
3 to 4 months
Including recovery near the centre
On this page
  1. What it is
  2. Symptoms and warning signs
  3. How it is diagnosed
  4. Treatment options
  5. How the choice is made
  6. How urgent is your case
  7. What to send us
  8. Questions patients ask
The condition

What thalassemia major is

Haemoglobin carries oxygen and is built from globin chains. In beta thalassemia major, the beta chains are absent or severely reduced, so functioning haemoglobin cannot be made. Red cells are destroyed prematurely and severe anaemia appears within the first year or two of life.

It is inherited recessively. Both parents carry the trait, usually without knowing, and each child has a one in four chance of being affected. It is common across the Mediterranean, the Middle East, South Asia and parts of Africa, and considerably more so where marriage between relatives is customary.

Without transplant, treatment means regular blood transfusions every two to four weeks for life. Those transfusions keep the child alive and growing, but each unit of blood deposits iron the body has no way of excreting. Over years that iron accumulates in the heart, liver and endocrine glands.

Iron overload is what ultimately determines the outlook. Heart failure and arrhythmia from cardiac iron are the leading cause of death in poorly chelated patients. Iron chelation drugs remove it, but they must be taken consistently for decades, and adherence is genuinely difficult for children and adolescents.

Symptoms

Symptoms and warning signs

Common features
  • Severe anaemia appearing in the first year or two of life
  • Pallor, tiredness and poor feeding
  • Failure to gain weight and grow normally
  • An enlarged spleen and liver, causing a swollen abdomen
  • Changes in the bones of the face and skull where the marrow expands
  • Delayed puberty and short stature
  • Darkening of the skin from iron deposition
Warning signs of an emergency
  • Fever, particularly after removal of the spleen
  • Breathlessness at rest or on lying flat
  • Palpitations or an irregular pulse
  • Severe abdominal pain
  • Rapidly worsening pallor between transfusions
  • Yellow eyes with dark urine

Transplant results are best in younger, well-chelated children

The strongest predictors of a successful transplant in thalassemia are the child's age, the degree of liver enlargement and scarring, and how consistently iron chelation has been given. Children transplanted young with well-controlled iron have markedly better outcomes than adolescents and adults with accumulated liver and cardiac damage. This is uncomfortable to say to a family who have been managing well for years with transfusions, but it is the truth: the window for the best result narrows with time. If a matched sibling exists, the conversation about transplant should happen early rather than being deferred until problems appear.

Diagnosis

How it is diagnosed

Diagnosis is straightforward; the assessment for transplant is where the detail lies.

Initial tests

  • Full blood count and blood film — showing severe anaemia with small, pale red cells
  • Haemoglobin electrophoresis or HPLC — confirms the diagnosis and the type
  • Genetic testing — identifies the specific mutations, useful for family counselling
  • Serum ferritin — a rough guide to iron loading, tracked over years

The deciding tests

  • HLA typing of the patient and all full siblings — the decisive test for transplant, and it can be done at home
  • Cardiac and liver T2* MRI — measures iron in the heart and liver directly, far more accurately than ferritin
  • Liver assessment — including fibrosis, which strongly influences transplant risk
  • Infection screening — hepatitis B and C from transfusions, and tuberculosis

HLA typing is the document that changes everything

Send the typing results for the patient and every full sibling. If typing has not been done, arranging it in your own country is the single most useful step you can take, and it is a simple blood test. Alongside it send recent haemoglobin levels, transfusion frequency, serum ferritin over time, what chelation the child takes and how consistently, and any cardiac or liver MRI.

Options

Treatment options

Transfusion and chelation keep a child alive. Transplant is what ends the disease.

Standard care

Regular transfusion and iron chelation

Transfusion every two to four weeks to keep haemoglobin at a level that prevents bone changes and allows normal growth, combined with iron chelation drugs to remove the iron those transfusions deposit. Done well this allows a good quality of life into adulthood. Done inconsistently, iron accumulates and causes the complications that shorten life.

Usually appropriate whenFor every patient, from diagnosis, and continuing until and unless transplant takes place.
Curative

Matched sibling donor transplant

Stem cells from a fully matched brother or sister replace the faulty marrow, and the child then makes normal haemoglobin. It is the definitive cure, with the best results in younger children with limited iron damage. Each full sibling has roughly a one in four chance of matching, which is why family typing comes first.

Usually appropriate whenA fully matched sibling is available, ideally in a younger child with well-controlled iron loading.
Curative

Haploidentical transplant from a parent

A half-matched parent donates. This has opened transplant to the great majority of families who have no matched sibling, and modern techniques have brought results substantially closer to matched sibling transplant. Graft-versus-host disease and graft failure remain somewhat more likely.

Usually appropriate whenNo matched sibling exists, but a healthy parent is available and the child is otherwise a good candidate.
Alongside

Managing the consequences of iron

Intensified chelation before transplant to reduce liver and cardiac iron, endocrine assessment and treatment for growth, thyroid and diabetes problems, and cardiac monitoring. Improving iron status before transplant measurably improves the outcome.

Usually appropriate whenBefore transplant in every patient with significant iron loading, and lifelong in those not transplanted.
The decision

How the choice is made

Is there a matched sibling

This is the first question and it is answered by a blood test that can be done at home.

The child's age and iron status

Younger, better-chelated children do considerably better. Liver size and fibrosis, and cardiac T2* MRI, quantify this.

Can the family commit to three to four months

An abandoned transplant is worse than continued transfusion. This must be settled honestly before starting.

If a matched sibling exists and the child is young, we will urge you not to wait. Every year of accumulated iron makes the transplant harder and the result less certain.

Urgency

How urgent is your case

Usually safe to plan travel
  • Stable on regular transfusion
  • Iron chelation being taken consistently
  • HLA typing complete, planning transplant
  • No active infection
Needs local assessment before travel
  • Fever, especially after splenectomy
  • Breathlessness at rest or heart failure
  • Active hepatitis or tuberculosis
  • Severe untreated iron overload with cardiac involvement
  • Uncontrolled bleeding or severe anaemia

We will tell you which column you are in

A febrile child with thalassemia — particularly one without a spleen — needs same-day assessment locally. Overwhelming infection can develop very quickly in these children.

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

  • HLA typing for the patient and all full siblings
  • Haemoglobin levels and transfusion frequency
  • Serum ferritin results over time
  • Which chelation drug is taken, at what dose, and how consistently

Also helpful

  • Cardiac and liver T2* MRI if performed
  • Liver ultrasound or fibrosis assessment
  • Hepatitis B and C status
  • The child's age, weight and growth chart
Send my reports for review See published cost ranges
Questions

Questions patients ask

Yes. A successful transplant replaces the faulty marrow with donor marrow that makes normal haemoglobin, and the child no longer needs transfusions or chelation. That is a genuine cure rather than a treatment, and it is the reason transplant is worth serious consideration despite its risks.

HLA typing — a blood test taken from the patient and each full sibling. It can almost always be done at a laboratory in your own country. Each full sibling has roughly a one in four chance of matching. This is the first step and it costs very little compared with everything that follows.

Not too late, but the calculation changes. Outcomes are best in younger children with limited iron damage, and by adolescence liver and cardiac iron are usually more significant. Transplant is still performed successfully in older children and adults, with somewhat higher risk. Send the ferritin history and any T2* MRI and we will give you an honest assessment rather than a general answer.

A half-matched parent can donate. Haploidentical transplant has transformed access for thalassemia families, since almost every child has a living parent, and results have improved substantially in recent years. It carries somewhat more risk of graft-versus-host disease and graft failure than a matched sibling transplant.

Because every transfusion leaves iron the body cannot excrete, and it accumulates in the heart, liver and endocrine glands. Cardiac iron is the leading cause of death in poorly chelated patients. Liver iron and fibrosis directly affect how well a child tolerates transplant conditioning. Improving iron status before transplant measurably improves the result.

Yes, for two reasons. HLA typing identifies a potential donor. And testing for thalassemia trait matters for their own future family planning, since two carriers have a one in four chance of an affected child with each pregnancy. Where marriage between relatives is common in the family, this is worth extending more widely.

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Contact

Send us your reports

Send HLA typing for the patient and all siblings if you have it. If not, arranging that blood test at home is the first thing we will recommend.

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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