Pharmacy Medical News about Anemia, Sickle Cell

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Investigational drugs in sickle cell anemia
Expert Opinion on Investigational Drugs, 10/05/09

Kotiah SD et al. – Sickled RBCs undergo intravascular hemolysis and accelerated erythropoesis. The hallmarks of this disease are shortened RBC survival and vaso–occlusive crises. For the past ten years, the pathophysiology of this disease has been better elucidated and has led to significant improvements in the standard of care. Vaso–occlusion is now understood to be a complex event that involves abnormal interactions between RBCs, leukocytes, endothelial cells and the coagulation pathways.

Exclusive Author Commentary
Samir K Ballas, 10/07/09

The hallmark of sickle cell disease is the acute recurrent attacks of painful crises that often require treatment in the Emergency room and hospital with relatively large amounts of opioids (also referred to as narcotics). Besides the severe pain these crises may be associated with infection, organ damage, stroke, acute chest syndrome, severe anemia or sudden death. Although cure could be achieved in some children and young adults, management of sickle cell disease continues to be primarily palliative. Search for a drug that aborts or prevents the crises has been a major goal since 1910 when the disease was first discovered in the USA. The advent of antibiotics, blood transfusion, newborn screening, vaccination and supportive care has improved the quality of life of some patients. Hydroxyurea (also refereed to as Hydroxycarbamide) has been shown to improve the quality of life of some patients and to decrease the frequency of painful crises, acute chest syndrome, and the need for blood transfusion, the morbidity and mortality associated with sickle cell disease. Unfortunately not all patients respond to hydroxyl urea. Dr. Kotiah and I have reviewed the availability of drugs that are available for the treatment of sickle cell disease. This review, we hope, will give interested investigators insights in designing new drugs that meet the criteria for conducting controlled randomized clinical trials.

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