Thalassaemia major cure out of reach of most

NST: KUALA LUMPUR: Of the 3,000 thalassaemia major sufferers in Malaysia, only around 100 have been lucky enough to undergo the bone marrow transplant procedure necessary to cure their illness.
In each case, fully-compatible donors were found from within the patient's family.
Thalassaemia Society of University Hospital president Professor Dr Chan Lee Lee said most of the donors were siblings of the thalasemic patient.
In fact, on average, one in four of a thalasemic's siblings is a suitable donor. Parents and unrelated people are very rarely a close enough match.
Dr Chan said only three hospitals in Malaysia can perform the bone marrow transplant procedure: Kuala Lumpur Hospital (HKL), University Malaya Medical Centre (UMMC) and Subang Jaya Medical Centre (SJMC).
"A patient who is to undergo the procedure is admitted (to these hospitals) for at least two to three months. A series of tests is done before the marrow is taken from the donor."
The marrow is extracted from bone that is rich in it, such as the hip bone, using a syringe.
Once that is completed, space must be made in the thalasemic patient's bones before the marrow is transplanted. This is done by placing the marrow in a blood bag and running it into a vein, just like with a blood transfusion.
Dr Chan said the procedure is quite expensive, costing about RM150,000 if conducted at UMMC, and RM500,000 if done at SJMC.
For patients unable to locate a compatible donor within the family, some embark on searching for non-related donors, which is very difficult and rare.
Dr Chan said the 3,000 thalassaemia major patients in Malaysia were still on regular blood transfusion and iron chelation therapy.
She said 97 of these patients had been undergoing oral chelation therapy since 2006, when the pharmaceutical company responsible for manufacturing the drug took in the patients for its clinical trials. The company was still supporting these patients by providing the drug for free.
Dr Chan said she hoped the government would also provide the oral chelation therapy drug so that patients need not undergo the agony of other painful therapies, which included injecting drugs into the bloodstream five times a week for up to 12 hours each time.
One thalassaemia major victim who is now leading a normal life after a bone marrow transplant is Nik Ahmad Hashimi, 8. His donor was none other than his younger brother, Nik Abdullah Ariff, 4.
Nik Ahmad Hashimi was diagnosed with thalassaemia major when he was 21 months old and had the transplant in 2006 at HKL.
His mother, 45-year-old accounts executive Nik Rosenidah Jaafar, said Nik Ahmad Hashimi "came down with fever too often, vomited whatever he ate and he looked yellowish".
He was diagnosed at Universiti Kebangsaan Malaysia Hospital.
"The whole family was screened and all of us were found to be carriers, except for my eldest son," she said. She has four sons and a daughter, aged six to 15.
Nik Ahmad Hashimi began receiving blood transfusions in 2002, and in 2005, he began chelation therapy when he was found to have "iron overloading" - a common side effect of the body not being able to remove the build-up of iron naturally.
In August 2006, it was found that younger brother Nik Abdullah Ariff's bone marrow was a compatible match, and the next month he had the transplant.
Nik Rosenidah said neither she nor her husband had known that they were carriers of the disease until Nik Ahmad Hashimi fell ill.
"I felt so sorry for causing my son to suffer. I kept apologising to him every time I saw him in pain. I definitely would not have conceived if I had known both my husband and I were carriers."
She said the two boys had developed a close bond and they were even starting to look like each other.
"Many people have asked if they are twins. They look so much alike. Even I am surprised at the change," said the relieved mother.