medical advisor and board member
What’s new in Sickle Cell Disease?
Sickle cell disease is one of the most common diseases in the world, but there is much to be learned about this illness. Clinical investigators are making progress in developing ways to treat and possibly cure sickle cell anemia.
Some sickle cell patients may require chronic transfusion therapy, which predisposes them to iron overload in the body. This iron overload leads to body damage over time.
In an effort to avoid chronic transfusion therapy and its side effects, investigators are studying how best to use the medication hydroxyurea. This oral medication increases the content of fetal hemoglobin, which makes the red blood cells less likely to sickle, avoiding many of the side effects of sickle cell disease. Investigators are currently studying if patients with stroke or those with abnormal trans cranial Doppler studies can be safely switched from chronic transfusion therapy to hydroxyurea. Other investigators are studying how patients are doing after they take hydroxyurea for several years to see if there are any long term side effects and what the benefits are to the patients.
Bone marrow transplantation is the only curative therapy for sickle cell anemia. When bone marrow from a matched brother or sister is used, cure rates approach 90%. However, most patients do not have a matched relative to be the donor. Therefore, there are new efforts to increase the donor pool to include unrelated volunteer donors and expand cord blood banking. There is a new clinical trial sponsored by the Bone Marrow Transplant Clinical Trials Network using unrelated donors to treat high risk patients with sickle cell disease. Persons interested in enlisting as a volunteer donor should go to the web site of the National Marrow Donor Program at www.marrow.org. persons interested in donating their child’s cord blood at the time of delivery should discuss this option with the obstetrician. Those interested in considering clinical trials in Florida should contact Dr. Gregory Hale at All Children’s Hospital in St. Petersburg, Florida.
Gene therapy is an active area of study, but not immediately ready for patients yet. Researchers are studying methods to insert normal hemoglobin genes into bone marrow cells to correct the sickle cell hemoglobin
This is an exciting time for researchers, patients and families with sickle cell anemia. Science is finding new ways to treat sickle cell anemia and improve the quality of life for patients and their families.
Dr. Gregory Hale