NursingLink

A Breakthrough With Sickle Cell

Virginian Pilot May 12 2008 Stephanie Desmon/The Baltimore Sun - Doctors at Johns Hopkins Hospital say Pamila Newton, who had just months to live, is one of the first adults in the world to be cured of sickle cell disease and the first using an experimental bone marrow transplant that could cure thousands like her who have been told they will never get better. Sixteen months ago she was in excruciating pain. She was on 15 pills a day, all heavy narcotics. She was bleeding regularly and needed daily transfusions of platelets. 80,000 Americans and millions around the world suffer from theis debilitating and usually fatal disease, prominent among among blacks and hispanics. What makes the Hopkins procedure different is that it allows patients to receive bone marrow from a donor who is not an exact match-a longtime obstacle to healing large numbers of people. Sickle cell is an inherited blood disorder that affects red blood cells. Normally round, the cells become C shaped like sickles and pile up on one another, keeping them from properly supplying oxygen to the body. The patients, prone to infection and serious pain, typically live only into their 40s. Until now, few have been cured and there has been little progress in developement of new treatments. Only one drug has been approved for treating sickle cell. Painkillers and antibiotics help patients live longer, but nothing has addressed the core problem of the genetic defect. Dr. Robert Brodsky and his colleagues say they hope to change that. They are trying to enroll 25 patients in a clinical trial. Still the procedure is not without risk. When performed on patients with leukemia and other diseases, it has a mortality rate of 16 %. "We may shorten peoples' lives, but this is their only chance for a cure" Dr. Brodsky said. "Would you offer this to every sickle cell patient? Absolutely not. But the patient who is having organ-threatening and life-threatening complications from the disease should be able to make that decision". Critics say the risk associated with the Hopkins' procedure is too high, even for a small trial. They say most sickle cell patients aren't likely to die immediately without a bone marrow transplant. Such transplants have been used to treat sickle cell disease for 20 years, but almost all of the 200 cured have been children. The treatments rely on high doses of chemicals that knock out the patient's own marrow before the transplant and are so toxic that adults with sickle cell-induced organ damage would be unlikely to survive. Brodsky says his team's procedure is less toxic. They no longer believe they have to destroy as much of the patient's marrow as they once did, so they administer just enough chemotherapy to suppress the immune system. That dose keeps patients from rejecting the new marrow without harming their organs. This change allows transplants for adults as well as children.One transplant obstacle has been finding a perfect bone marrow match. A full sibling's marrow provides the best chance, but there's only a 25% chance that even a full sibling wll be a match. Because sickle cell is inherited, siblings also might have the disease. That leaves about a 10% chance that a patient will find a donor. Brodsky's procedure requires just a half match, meaning children and parents of the patient could be suitable donors. Brodsky said the biggest problem in promoting his knew procedure may be the cost. Most insurance companies won't cover experimental treatment for genetic disorders. The procedure has been a life saver for Pamela Newton. Her mother was the marrow donor. Two months after the transplant, Newton began to feel better. Slowly the pain dissipated. She took her last Oxycontin in April.

---