Drugs to Treat Sickle Cell Anemia

Definition

Sickle cell anemia is a hereditary form of anemia in which the number of erythrocytes circulating in the blood is so low that it does not guarantee adequate oxygen transport throughout the body. It is called "sickle cell" due to the particular structural shape of red blood cells : these cells are not rounded, biconcave and elastic, but take on a sickle shape, tend to aggregate and are fragile and angular.

Causes

The cause that triggers sickle cell anemia lies in a genetic mutation on the gene responsible for the production of hemoglobin, therefore it is not associated with either food deficiencies (eg iron deficiency anemia) or infections (eg pernicious anemia, which can depend on gastric insults of "H. Pilory). Sickle cell anemia is an autosomal recessive disease.

Symptoms

Pain, headache and respiratory and visual difficulties are the symptoms that characterize sickle cell anemia; more precisely, the pain is often perceived in the back, stomach, chest and bones, and can be of variable entity, lasting a few minutes or last for hours or days. Breathing difficulties often coincide with sporting activities, but can also appear during rest. Sickle cell anemia increases the risk of infections.

The information on Sickle Cell Anemia - Drugs for the Treatment of Sickle Cell Anemia is not intended to replace the direct relationship between a health professional and patient. Always consult your doctor and / or specialist before taking Sickle Cell Anemia - Drugs for the Treatment of. Sickle cell anemia.

Medicines

Unfortunately, there is no drug cure that can ensure complete recovery from sickle cell anemia; however, some drugs can relieve pain and prevent complications associated with the disease.
Bone marrow transplant, on the other hand, is the only therapeutic option that definitively resolves sickle cell anemia, even if this medical procedure is extremely complicated and involves serious risks, including death. It should be emphasized, however, that it is very difficult to find a compatible donor.
Blood transfusions and oxygen supplementation are additional therapeutic procedures useful for alleviating the painful symptoms that accompany sickle cell anemia.

The following are the classes of drugs most used in the therapy against sickle cell anemia, and some examples of pharmacological specialties; it is up to the doctor to choose the active ingredient and the dosage most suitable for the patient, based on the severity of the disease, the state health of the patient and his response to treatment:

Antibiotics: we have seen that sickle cell anemia increases the risk of contracting infections; for this reason, patients affected by it can undertake preventive antibiotic treatment. In particular:

Penicillin G or benzylpenicillin (eg Benzyl B, Benzyl P): can be administered to babies as early as two months after birth; the administration of this antibiotic is always recommended up to 5 years, in order to prevent pneumonia and other more dangerous infections, especially for children. The dosage must always be established by the doctor; indicatively, the recommended dose for the prevention of streptococcal infections is to take 1-5 million units intravenously (loading dose), even several times during the day.

Painkillers (NSAIDs and corticosteroids): since pain is almost constant in all sickle-cell anemia patients, the administration of painkillers (which does not help to combat the triggering cause) is a very important aid to alleviate the symptoms.

Vaccination: since anemia favors infections, especially for affected children, immunization by vaccination is recommended. Consult your doctor.

Urea derivatives (hydroxyurea): the drug is generally used in oncology; however, some sickle cell patients are treated with this active ingredient, both because it relieves the pain associated with the disease and because it decreases the need for blood transfusions. The drug works by stimulating the production of fetal hemoglobin (a type of hemoglobin in newborns that can prevent the development of sickle cells).

Hydroxyurea (eg. Onco-carbide): the dosage of this drug for sickle cell anemia is to take 15mg / kg once a day. It is possible to increase the dose by 5 mg / kg / day every 3 months, up to a maximum 35mg / kg / day. Hydroxyurea can only be used in severe sickle cell anemia and acute painful crises; do not use for long periods, as it seems to favor leukemia or other cancers.

Bronchodilators: sickle cell anemia is often accompanied by more or less intense breathing difficulties (eg asthma), which can be controlled with bronchodilator drugs.

Ipratropium bromide (eg. Atem, Breva): the drug is available in formulations consisting of the active ingredient only, or in association with beta2-agonist drugs. By aerosol, repeat 2 inhalations (36 mcg) 4 times per day (do not exceed 12 inhalations per day). Alternatively, take a single-dose 500 mg vial (nebulized solution), 3-4 times a day. The drug is indicated to treat asthma and severe breathing difficulties triggered by sickle cell anemia.
Isoetarin: belonging to the class of bronchodilator drugs, isoetarin ensures a relaxing action on bronchial smooth muscles.
Theophylline (eg Aminomal Elisir, Diffumal, Respicur): theophylline is a xanthine drug used in therapy to reduce the broncho-constrictor stimulus even in the case of sickle cell anemia. The drug is taken at a loading dose of 5 mg / kg. Consult your doctor.

Integration of folic acid, vitamin E and zinc: in the case of sickle cell anemia, the administration of vitamin B9 and zinc seems to be particularly suitable:

Folic acid (eg. Folina, Fertifol, Folidex): available both as soft capsules of 5 mg of active ingredient (to be taken 1-3 times a day), and as a solution for injection of 15 mg of folic acid (taken once a day , intramuscularly). Vitamin B9 can prevent alterations and damage to blood vessels, which is possible in the context of sickle cell anemia.
Vitamin E (eg. Sursum, Ephynal, Rigentex): Vitamin E, antioxidant par excellence, reduces oxidative stress inside the red blood cells in patients suffering from sickle cell disease and thalassemia. In general, it is recommended to take a dose of active. equal to 200 units (10 ml), orally (oral liquid formulation), once a day.
Zinc (eg Zincometil, Ektogan, ZMA): indicatively, take 50 mg of active ingredient three times a day. The supplementation of zinc allows to decrease the frequency of painful crises in the context of sickle cell anemia.

Pharmacological expectations and hopes for the future: currently, sickle cell anemia is not curable, but researchers are hopeful and new treatments are being tested:

Nitric oxide supplements: by reducing the viscosity of red blood cells and creating a dilation of blood vessels, nitric oxide could prevent the aggregation of sickle cells
Gene therapy: Introducing a healthy gene into the bone marrow (responsible for producing fetal hemoglobin) could rebalance the physiological hemoglobin level.
Deactivation of the defective gene: given that the "sickle cell anemia is caused by an" alteration of a gene, the deactivation of the same could be a solution strategy.
Innovative drugs for the treatment of sickle cell anemia, aimed at increasing the synthesis of fetal hemoglobin, useful for preventing sickle cells from forming.