New thalassaemia cure gives hope to patients in UAE

Thalassaemia is a debilitating genetic disease that requires a patient to undergo frequent blood transfusions.

 Just two and half years ago, 17-year-old Hussein Alblooshi had to worry about blood transfusion constantly.
Today, he is living the full life like any other teenager in Dubai - playing sports, spending time with friends and family, doing homework, and dreaming of his future as an engineer or doctor.
Hussein's breakthrough came in 2016, with the opportunity to undergo a radical new treatment, after a childhood spent visiting medical facilities around the globe in the hope of finding a cure.
With this new procedure - a haplo-identical bone marrow transplant, carried out at Cleveland Clinic in the US - there was a potential to cure thalassaemia with minimal risks or long-term side effects.
Thalassaemia is a debilitating genetic disease that requires a patient to undergo frequent blood transfusions and held the risk of fatal complications.
It is common in people of Middle Eastern descent, and around 8.5 per cent of UAE citizens are believed to be carrying the defective gene.
On International Thalassaemia Day marked on Wednesday, Hussein has a simple message for others with the disease: "Never give up hope. I truly believe that everyone has a chance to be cured as nothing is impossible."
Hussein's doctor, Dr Rabi Hanna, director of paediatric bone marrow transplantation at Cleveland Clinic Children's Hospital, explained that "in traditional transplants, the donor and recipient cells need to be genetically matched".
This means only around 25 per cent of patients would be able to receive a bone marrow transplant, Dr Hanna said.
"But in the newer haplo-identical transplant, the donated cells only need to match half of the recipient's important genes. This broadens the scope of possible donors tremendously. Now, almost every child will have a suitable donor, whether it's their mum or dad, or a half-sibling."
After preconditioning chemotherapy and medication, Hussein underwent the procedure at Cleveland Clinic in November 2016, using the bone marrow donated by his brother Suhail, then aged 11. After the transplant, Hussein had additional immuno-suppression medications to avoid the rejection of the marrow.
Dr Hanna said that besides being more accessible, the new procedure offers better outcomes.
"In the old days, we didn't offer bone marrow transplants widely because of the risk of Graft-versus-Host Disease," he explained.
Graft-versus-Host Disease (GvHD) is a complication where donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, prompting them to attack the body.
"Thanks to the techniques and protocol we use, there is little risk of GvHD, so within six to nine months, we are able to stop immune suppression medicines and the patients can go back and live a normal life," said Dr Hanna.
Hussein is doing just that.
"Before the procedure, I was on heavy medication, and had to wake up at night to take the drugs," he said. "Now, I have stopped all medications because I do not need them anymore."
He also used to have painful blood transfusions monthly, and the constant need for medical care affected his education.
"I used to miss lots of classes because of the doctor appointments and because I felt tired and exhausted most of the time. Now, I attend my school regularly and I do not miss anything."
His mother, Farida, said the disease had an impact on the whole family's life due to anxiety over the condition. The extensive travelling to seek treatment also took them away from Hussein's other siblings, who frequently had to stay with a family member.
"I had so many sleepless nights. I had to check on Hussein every night to make sure he was well, I was constantly living in fear for his life," she said.
Beta thalassaemia major can be fatal due to the extensive blood transfusions needed. Over time, an influx of iron-containing haemoglobin can lead to a build-up of iron in the body, resulting in heart, liver and hormone problems.
For Dr Hanna, being able to change the lives of patients, such as Hussein, has a 'very personal' aspect. When he was in school, one of his friends died because of thalassaemia.
"He was a really smart kid. Knowing that now we can offer a cure to almost everyone, I feel it is my duty to make people aware of this."
Understanding thalassaemia
>A debilitating genetic disease that requires a patient to undergo frequent blood transfusions
>Can be fatal due to the extensive blood transfusions needed
>Common in people of Middle Eastern descent
>8.5% of UAE citizens are believed to be carrying the defective gene
Treatment
Traditional transplant
>Donor and recipient cells need to be genetically matched
>Only 25% of patients can get the right bone marrow
New method:
Haplo-identical transplant
>Donated cells only need to match half of the recipient's important genes
>Broadens the scope of possible donors tremendously
>Almost every child will have a suitable donor
>Donors can be their mum or dad, or even a half-sibling
asmaalizain@khaleejtimes.com