Bone Marrow Transplant for Thalassemia

What is a Bone Marrow Transplant?

As much complex is the interior of a human being, more is its need for every small little cell to perform its bit. Everybody knows about the red and white blood cells or the fighter cells of the body, which is required to fight infections and carry oxygen. These cells are produced by the bone marrow.

If due to any infection or disease bone marrow does not function properly, another healthy bone marrow from a donor or self is put in the patient. This procedure is known as a bone marrow transplant.

When it is performed?

The procedure is performed in cases like:

  • Leukemia
  • Lymphoma
  • Sickle cell anemia
  • Severe aplastic anemia
  • Fanconi anemia
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Pure red cell aplasia
  • Amegakaryocytosis/congenital thrombocytopenia
  • Severe Combined Immunodeficiency (SCID)
  • Wiskott-Aldrich Syndrome (WAS)
  • Beta thalassemia major
  • Krabbe disease (GLD)
  • Hurler syndrome
  • Adrenoleukodystrophy (ALD)
  • Metachromatic Leukodystrophy (MLD)
  • Myelodysplastic syndromes and myeloproliferative disorders
  • Multiple myeloma and other plasma cell disorders
  • Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders

Thalassemia is a genetic blood ailment in which the body makes abnormal or inadequate hemoglobin. Hemoglobin is a protein pigment found in red blood cells that carries oxygen. Here a large number of red blood cells are destroyed causing anemia.

Who performs a Bone Marrow Transplant?

An Oncosurgeon.

Preparing for the Bone Marrow Transplant in Thalassemia:

There are 2 major types of Bone Marrow Transplant –

  1. Autologous transplants – Using a person’s stem cells
  2. Allogeneic transplants – Using donor’s stem cells

Even after diagnosis, several tests are performed to finalize the type of stem cells needed. The procedure is similar to blood transfusion and the area of the hospital where the transplant is done is away from routine OPD / IPD to prevent infections.

The procedure may take up to a week therefore seems like a good idea to pick a hospital close to the house.

The patient at a bone marrow transplant center is categorized into 3 classes depending on the factors affecting their outcome. These factors are:

  • Age
  • Adequacy of chelation
  • Presence of liver fibrosis
  • Presence of hepatomegaly

Diagnosis of Thalassemia:

  • Blood test-complete blood count (CBC)
  • Hemoglobin tests to define of thalassemia type alpha or beta

How a Bone Marrow Transplant is performed:

Bone marrow or Hematopoietic cell transplantation is carried out is assistance with high-dose chemotherapy to eradicate thalassemia-producing cells in the bone marrow, those of which are then replaced with healthy donor cells. The donor is the person who is a Human-Leukocyte Antigen (HLA) match with the patient, usually an identical sibling. As young is the patient the better will be the outcome.

Your cells if used are harvested and kept in a stem cell bank before chemotherapy session starts.

The cells are often collected from both hipbones through a needle under anesthesia.

While collecting donor stem cells, they are injected with medications so that stem cells move from bone marrow to blood from where they are collected through a drip to a machine. This machine separates white blood (containing stem cells) cells from the rest of the blood.

When the patient is transfused, a central line is introduced through the chest directly into the heart so that the stem cell goes to the heart and the entire body including bone marrows. Here, they establish and begin to propagate. Similar sessions are carried out another couple of times to ensure success. The central line stays intact until all sessions are complete.

What to expect after a Bone Marrow Transplant:

The patient is closely monitored for any growth of white blood cells which means the treatment was successful. It takes about 10 and 28 days after the initial transplant for completing sessions and 3 months to a year to fully recover.

Side-effects of Bone Marrow Transplant:

  • Drop-in blood pressure
  • Headache
  • Nausea
  • Pain
  • Shortness of breath
  • Chills
  • Fever

Complications of the Procedure:

  • Graft-versus-host disease where donor cells attack your body
  • Graft failure – when transplanted cells don’t produce new cells
  • Bleeding in the lungs, brain, and other parts of the body
  • Cataract characterized by clouding in the lens of the eye
  • Damage to vital organs
  • Early menopause
  • Anemia – when the body doesn’t produce enough red blood cells
  • Infections
  • Nausea, diarrhea, or vomiting
  • Mucositis – inflammation, and soreness in the mouth, throat, and stomach

Post-procedure care:

  • Avoid excess iron
  • Eat a healthy diet
  • Avoid infections

As complicated as this procedure sounds the thalassemia-free overall survival rate of an HLA-matched sibling stem cell transplantation patient is 85% to 90% which is significant.

Dr. Suparno Chakrabarti, Senior Consultant & HOD – Bone Marrow Transplant, Haemato Oncology, Oncology, Dharamshila Narayana Superspeciality Hospital, Delhi

Narayana Health

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