Reduced need for blood transfusions in thalassemia patients

A new medication is reducing the need for blood transfusions in thalassemia patients and improving bone-marrow transplant outcomes — meaning there is a cure for almost any patient — according to an expert from the Cleveland Clinic in the US.

Thalassemia is a debilitating and potentially fatal genetic disease where the body makes an abnormal form or inadequate amount of haemoglobin — the protein in red blood cells that carries oxygen — and patients need frequent blood transfusions. It is the most common inherited single-gene disorder in the world and occurs most frequently in people from the Middle East, Mediterranean countries, North Africa, India, Central Asia and Southeast Asia.

Now the US FDA has approved the first drug to treat thalassemia directly — a first-in-class medication that enhances erythroid (red blood cell) maturation and reduces the blood transfusion burden, and is given in the form of an injection every three weeks. According to the Cleveland Clinic’s Dr Rabi Hanna, the drug has been shown to reduce the need for blood transfusions by up to 50% in some clinical trial patients.

“It can help reduce the need for blood transfusions and improve patients’ symptoms,” said Dr Hanna, who is Director of Pediatric Blood & Marrow Transplants at Cleveland Clinic Children’s. “Consequently, it reduces the risk of blood transfusion reactions, as well as the risk of iron overload, which can damage organs such as the heart and liver in the long term.”

He added that while the drug is not in itself a cure, it can enhance outcomes in curative bone marrow transplantations. These have become more widely accessible in recent years thanks to a new transplant approach called a haplo-identical bone marrow transplant, which Dr Hanna helped pioneer and which allows for non-identical-HLA (human leukocyte antigen) donors. He explained that the reduction in blood transfusions means the patient has better health prior to the procedure, and also has fewer antibodies that could potentially attack transplanted bone marrow.

“Between the improved overall pre-transplant health from taking the medication and the new approach we take to bone-marrow transplants, almost any patient can be cured,” Dr Hanna said.

Whereas previously only 25% of thalassemia patients could find a match for bone marrow donation, the newer method means the donated cells only need to match half of the recipient’s important genes. This has broadened the scope of possible donors tremendously so that almost every child or young adult will have a suitable donor, such as a parent, half-sibling or other relative.

In addition, the approach uses a reduced-intensity conditioning and post-operative protocol. Patients receive low doses of chemotherapy and immuno-suppressant medication before and after the transplant, and the risks and side effects of the operation are minimal.

Transplant success story

Among the first patients to receive the newer type of transplant was Dubai-based Hussein Alblooshi, with the assistance of the UAE Government and the Cleveland Clinic’s Global Patient Services team. The operation was performed by Dr Hanna and his team at the Cleveland Clinic in 2016, with bone marrow donated by Ablooshi’s brother, Suhail.

The transplant successfully cured Ablooshi’s thalassemia and he no longer takes medication and does not need any blood transfusions. Now 18 and a student at a Dubai college, he is keen to spread awareness about the cure.

“I want to spread the message of hope that there is a cure to all of those going through what I went through — constant tiredness, painful blood transfusions, waking up at night to take medication and all the other disruptive effects,” he said. “I live a completely normal life now, and want to let others know they can too.”

Dr Hanna concluded, “It has been wonderful to keep in touch with Hussein and his family and hear about his progress and his advocacy. He had the procedure a few years ago, before the new medication was approved by the FDA, so the treatment journey is even better for patients now.”

Image credit: ©stock.adobe.com/au/sudok1

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