FDA Approves A New Therapy for Curing Patients Having Rare Thalessemia

The US Food and Drug Administration – FDA – has recently approved a therapy, the first of its kind, to be effective for curing a rare type of thalassemia. The therapy uses Reblozyl (luspatercept – aamt) to treat anemia in grown patients having thalassemia type Beta. These patients require blood transfusions on regular basis.

On receiving regular blood transfusions, the risk of iron overload in the body gets elevated to a much larger extent. This overload can unfortunately result in organ toxicity that can lead to some serious consequences according to the Center for Drug Evaluation and Research of the US Food and Drug Administration.

The approval provides the thalassemic patients with a therapeutic measure that would be helpful in alleviating the amount of blood transfusions. The approval is an example of how a gradual yet regular state of progress of a rare disease and get new medications to the patients early.

Moreover, Beta Thalassemia, which is also called Cooley’s Anemia, is the genetically acquired disorder of the blood that alleviates the production of the oxygen transporting molecule, haemoglobin that contains iron responsible for the whole process occuring in the body.

People having beta thalassemia have extremely low haemoglobin levels because of the deficiency of it, there is a reduction in the supply of oxygen in the body. The resulting condition is anemia, in which the skin becomes pale, sense weakness and has fatigue. Moreover, there are respiratory and other serious complications that come with it.

Supplementary treatment for patients having beta thalassemia usually constitutes of prolong protocols of the chronic transfusions of blood that carry on for life that are important for survival.

Thalassemic patients have an elevated chance of getting abnormal blood clots that can cause heart attack in several cases because of the blockage of blood supply to the heart.

This approval was confirmed through a pilot project in which there was a clinical trial of 336 thalassemia patients having beta thalassemia with a requirement of RBC transfusions. Out of the total number of 336, 112 were categorized in the placebo group.

It was noted that 21 per cent of the thalassemic patients who got Reblozyl were able to experience a reduction of blood transfusions by 33 per cent in comparison to 4.5 per cent of the patients with thalassemia that got a placebo treatment.

The reduction in the transfusion had the meaning that the individual with the disease did not need transfusions by the end of the 12th week of the process during taking Reblozyl.

Some of the common side effects for people that took the medication were bone pain, joint pain, headache, cough, fatigue, diarrhea with pain, and dizziness. The patients might experience episodes of high blood pressure in the treatment process.

The recommendation is that the continuous monitoring of the patient’s blood pressure levels in the treatment process and for the initiation of the anti-hypertensive treatment procedure if needed.

Patients that received Reblozyl medication would have to be continuousy monitored for any blood clots. Moreover, the FDA suggests fertile women to take oral contraception with care during the course of treatment with Reblozyl. Lactating and pregnant women should not go for this type of medication as it might be harmful for the baby.

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