New Treatments may Offer Hope for Transfusion-Dependent Thalassemia Patients

New medication will decrease the need for blood transfusions in thalassemia patients, and eventually improve bone-marrow transplant outcomes, according to an expert, speaking ahead of World Thalassemia Day.// Thalassemia 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.

Rabi Hanna M.D., Director of Pediatric Blood & Marrow Transplants at which was approved by the FDA towards the end of last year, as it is the first medication to treat thalassemia directly. It can help reduce 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.

The new drug is 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 Dr. Hanna, a clinical trial called BELIEVE has shown that it can reduce the need for blood transfusions by up to 50% in some patients.

He adds that while the drug is not in itself a cure, it can enhance outcomes in curative bone marrow transplantations, which have also become more widely accessible in recent years thanks to a new transplant approach. Dr. Hanna was among the pioneers of the haploidentical bone marrow transplant that allows for non-identical-HLA (human leukocyte antigen) donors, and which he performed successfully on a young Emirati patient in 2016.

He explains 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.

Advertisement

Whereas previously only 25% of thalassemia patients could find a match for bone marrow donation, with the newer method, the donated cells only need 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 relatives.

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.

Advertisement

Transplant success story

Among the first patients to receive the newer type of transplant was Dubai-based Emirati Hussein Alblooshi, with the assistance of the UAE 8.5% of Emiratis are believed to be carriers of thalassemia genes.

The operation was performed by Dr. Hanna and his team at Cleveland Clinic 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, having visited a thalassemia center in Dubai earlier this year to talk to patients about the procedure and what it entails.

“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. I live a completely normal life now, and want to let others know they can too,” he says.

Dr. Hanna adds: “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.”

Source-Medindia