Drug Topics - May 5, 2008 - (Page 4) 4 DRUG TOPICS MAY 5, 2008 www.drugtopics.com Sickle cell disease: The hunt is on for a cure Kathy Hitchens, Pharm.D., MSBA Drug treatments are still elusive for this disorder, which affects primarily African Americans. S ickle cell disease (SCD) is DT CAPSULE life-defining. Those with this inherited blood disease never know when a crisis will disrupt their lives. They never know how long SCD is going to interfere with daily living. In many respects SCD determines an individual’s quality of life, level of career success, personal finances, and perhaps even longevity—although over the past 30 years improved medical care has significantly improved the lifespan of individuals with SCD. Progress toward a cure for SCD is moving at an Most SCD patients are not drug-seekers but are inadequately treated for pain. Sickle cell disease at a glance W hat is sickle cell disease (SCD)? SCD is a genetic disease characterized by the presence of a hemoglobin variant—hemoglobin S—that shortens the lifespan of red blood cells (~16 days instead of the normal 120) and allows red blood cells to change shape (sickle), stick together, and block small blood vessels. This leads to tissue ischemia causing different types of complications. What are problems associated with SCD? • SCD can lead to kidney failure, pulmonary problems, joint destruction, end organ damage. To address the needs of SCD patients, “We did something in Atlanta that was unique,” Platt said. “We built a specific emergency room (ER) just for them [SCD patients]. They do need ER care. They can have catastrophic complications that present with pain. Atlanta Photo: Getty Images/Aldon • Many patients experience chronic pain. Opioid therapy may be routine. • Acute pain crisis usually require large doses of pain medication. • Pseudoaddiction is a problem related to SCD. Patients claim to need stronger pain medication or more frequent dosing. Many healthcare professionals interpret this as drug-seeking behavior, but for most SCD patients, it represents inadequately treated pain. • Some analgesics mask fever. Not masking fever is important because fever is a sign of infection that needs to be evaluated and treated. • Immunosuppression predisposes patients to life-threatening illnesses. Until age six, all children should be on a prophylactic penicillin regimen. • Even very young children are at risk of strokes. • Frequent blood transfusions lead to iron overload. Chelation therapy is essential. • Hydroxyurea has a narrow therapeutic index and can be toxic. excruciatingly slow pace, hampered by a low public awareness and limited research funding. While some individuals have been cured by bone marrow transplant, for most that is out-of-reach. The only new drug therapy to impact SCD in the last decade is deferasirox (Exjade, Novartis), an iron chelator. Niprisan (Hemoxin, Xechem International, Inc.), a phytopharmaceutical compound approved for treatment of SCD by Nigeria’s National Agency for Food & Drug Administration and Control, has caught the interest of many SCD patients. It has not been evaluated by the U.S. Food & Drug Administration, although it has been granted orphan drug status. SCD afflicts about 80,000 African Americans in the United States. In addition, more than 2.5 million Americans (primarily African Americans) genetically carry the sickle cell trait. SCD is not limited to African Americans; in some parts of the United States a significant number of Hispanic Americans have SCD too. SCD is not common, but neither is it rare. However, health professionals don’t seem to know much about it. Commented Willarda V. Edwards, M.D., MBA, president and COO of the Sickle Cell Disease Association of America (SCDAA), “M.D.s don’t know a lot about it.” That many medical professionals know little about SCD bodes ill for patients who are vulnerable to a variety of disease complications. (See sidebar). “Patients are often isolated, stigmatized, and don’t know where to go to obtain the medical care they need,” said Allan Platt, PA-C Co-Coordinator, Career Masters Program, Emory University Physician Assistant Program. ER for SCD patients http://www.drugtopics.com
Table of Contents Feed for the Digital Edition of Drug Topics - May 5, 2008 Drug Topics - May 5, 2008 Contents Don't Get Caught in Fed's Fraud and Abuse Dragnets Sickle Cell Disease: The Hunt is On for a Cure Latest News Roundup Drug Topics - May 5, 2008 Drug Topics - May 5, 2008 - Contents (Page 1) Drug Topics - May 5, 2008 - Contents (Page 2) Drug Topics - May 5, 2008 - Don't Get Caught in Fed's Fraud and Abuse Dragnets (Page 3) Drug Topics - May 5, 2008 - Sickle Cell Disease: The Hunt is On for a Cure (Page 4) Drug Topics - May 5, 2008 - Sickle Cell Disease: The Hunt is On for a Cure (Page 5) Drug Topics - May 5, 2008 - Latest News Roundup (Page 6)
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