Hemoglobinopathy Treatment Drugs; Help Relieve Pain and Minimize Complications

 

Hemoglobinopathy treatment drugs include hydroxyurea (Siklos, Hydrea, and Droxia), ACE inhibitors, antibiotics, and analgesics. Blood transfusions should be given only when strictly indicated. Hemoglobinopathy is a genetic disorder in which there is an abnormal structure or production of a hemoglobin molecule. This group of disorders includes thalassemias, sickle cell anemia, hemoglobin S-C disease, and hemoglobin C disease. They fall into two main groups: thalassemia syndromes and structural hemoglobin variants (abnormal hemoglobin). Common symptoms of the condition include enlarged spleen, shortness of breath, severe pain, and growth problems in children.

Hemoglobinopathy is a serious condition that can lead to complications in many parts of the body. These include cardiovascular disease and heart attack. Hemoglobinuria, the clinical term for this condition, occurs when the body does not properly use the circulating blood's supply of red blood cells, leading to an accumulation of the substance in the body. This often occurs in the extremities such as the ankles, feet, and legs. The most common symptoms include pain and swelling in the areas where the body normally produces hemoglobin; nausea and vomiting; fatigue; and low energy levels. Moreover, hemoglobinopathy has the potential to spread to the lungs, resulting in a pulmonary embolism, which can be life-threatening.

Hemoglobinopathy treatment drugs include oral medication, such as deferiprone (Ferriprox) or deferasirox (Jadenu and Exjade). Another drug, deferoxamine (Desferal), is given by needle. These drugs are used to treat thalassemia. An individual with thalassemia trait has a normal life expectancy. However, heart complications can make this condition fatal before the age of 30 years. Consuming beetroot, lentils, sweet potatoes, and banana can help reduce symptoms of thalassemia, as these foods have folic acid that can help in producing more red blood cells. Thus, with the increasing prevalence of hemoglobinopathies such as thalassemia, the demand for hemoglobinopathy treatment drugs is also increasing.

According to the National Organization of Rare Disease (NORD), thalassemia is relatively rare in the United States, but is one of the most common autosomal recessive disorders in the world. The incidence of symptomatic cases is estimated to be approximately 1 in 100,000 individuals in the general population.