Bone Marrow Transplantation

Bone marrow transplantation (BMT) is an option available to the luckier thalassaemics. This is because in thalassaemia, transplant is only carried out if there is a compatible sibling donor available. The sibling who is to be the donor can either be normal or a carrier. This will then render the patient transfusion free if the transplant is successful. The high risk involved is possible death over failure.

In Sarawak General Hospital, once a new thalassaemic is diagnosed, transplant workup is done if the patient has normal or carrier siblings. Transplant workup entails a series of blood test both for the donor as well as the recipient to see whether they match. This blood tests are done free for thalassaemia patients at the Institute for Medical Research in Kuala Lumpur but there is a waiting list

(note: courier charges for handling the blood tests are borne by the parents).

Once the thalassaemia patient and the donor are deemed matched, they go onto the transplant list of the bone marrow center. Although they are one the list, priorities will be given to other urgent cases, such as leukemia patients. For our patients in Sarawak General Hospital, arrangements are usually made with Hospital Kuala Lumpur unless parents request otherwise. There are other centers available, including our neighboring Singapore but it is definitely more expensive.

When to do the Transplant?

Transplant (if it is an option) is preferably performed early before the child receives too many transfusions or develop complications from iron overload. In an older child, the complications associated with transplant increases and together with it increased morbidity and mortality.

In the beginning....

Once these patients are matched, parents will be counseled about the procedure and the risks involved. At the same time, both patient and donor will have to have a series of blood tests and investigations done to make sure they are fit for the transplant. If the child is on desferal parents are informed that they have to optimize desferal use for best results.

A liver biopsy is also required to determine the iron load and also the class of risk associated with transplant.

Chemotherapy

Once there is a slot available, patient together with the parents and donor will be call to the centre. Bone marrow will be harvested from the donor as a backup. At the same sitting, a central line is inserted. This intravenous line is inserted into a big vein and requires a small operation under anesthesia, It is required as it facilitates the giving of chemotherapy, blood product, nutrition, antibiotics, etc. during the acute period post transplant.

About 3-5 days before transplant, some chemotherapy is given to deplete the recipient of its own marrow. After this, his blood count (red, white blood and clotting factors) would start to drop.

On the day of the transplant

On the day of the transplant, the donor is put under anesthesia in the operation theatre and marrow is withdrawn. Once enough marrow is taken, the donor is brought back to the ward. The donor usually has no problems except for some pain and can usually be discharged by the second day with some paracetamol and vitamins.

The marrow taken is processed and infused into the recipient who remains in the ward like a blood transfusion.

If the donor is much older, sometimes stem cells (cells that are required to produce bone marrow) instead of bone marrow are taken from the donor like a blood donation after the donor is given some medication.

During the next 2-3 weeks while waiting for the new stem cells to produce all the blood cells, the patient can be quite sick.

Once the blood counts start to recover, they become febrile and start taking well orally which is usually around the 3rd week, they are discharged to the halfway house if they are from out of Kuala Lumpur. They will continue to receive medications as an outpatient about 3 times a week till 2 months post transplant. They are initially reviewed about twice a week, then weekly till counts show a definite recovery trend. Usually, they can expect to be in Kuala Lumpur for about 2 months.

(Note: They might need Desferal for a short period post transplant depending on their liver biopsy and iron load.)

Post transplant

They will continue to be on cyclosporin for about 6 months. Their immune system remains compromised. We usually advise the patient to wear a mask for 6 months and to remain homebound. If they are school going, usually, they have to miss school during that period of time. Hygiene must be maintained and food usually home-cooked.

The child will need to be reimmunized approximately 1 year post transplant.