A new drug recently approved for use in EU countries is expected to provide a leap in the quality of life for patients suffering from iron overload due to blood transfusions, among them 28 Maltese patients suffering from Thalassaemia.
The new drug - manufactured by Novartis - will replace the current daily medication that has to be administered intravenously through a pump over eight hours.
"This proves a great inconvenience for patients," genetic specialist Christian Scerri, one of the doctors running St Luke's Hospital Thalassaemia Clinic, said.
Adherence to the daily regime, he pointed out, drops substantially when a patient reaches his teens because of the impact it has on quality of life. This leads to adverse effects on the patient's health.
Patients with Thalassaemia - an inherited disorder of the red blood cells - need monthly blood transfusions, without which they would die young. But this leads to an iron overload which the body is unable to get rid of.
Over the years the iron tends to get deposited in the vital organs which can lead to heart failure, liver cirrhosis, diabetes and other fatal conditions, Dr Scerri said.
Therefore, patients need medication to help them eliminate the iron. Although the current medication is a life saver, having to be attached to a portable pump for eight hours a day seriously affects the patients' quality of life.
This is why the new medicine, Exjade, is being hailed as a breakthrough by many experts since the daily hours-long inconvenience is replaced by a once-daily drink.
"This should lead to more compliance to the treatment, especially in teenagers," Dr Scerri said. An average of one patient with Thalassaemia is born here every two years.
Although Exjade is more expensive than the existent one - also manufactured by Novartis - more compliance would probably lead to less health problems in patients, and a saving in the long-run. In the last four years two patients in their 20s died partly due to complications of iron overload.
Patients, Dr Scerri said, have shown great interest in the new drug and their expectations that it replaces the current medication at the earliest are very high.
The father of the youngest Maltese patient, a three-month-old baby, is patiently waiting for Exjade to be available.
"Imagine having to stick a needle up his arm everyday; it's heartbreaking," he said. The baby has not yet started treatment but his parents are mentally preparing for it. He admits that he would not have thought twice about enrolling his son in the human trial for Exjade. "Daily injections are not something one aims to do," he said looking at his son.
The Thalassaemia clinic can be contacted between Monday and Friday on 2123 9807 or 2340 2774.