NLM Director’s Comments Transcript
Hope for

Adult Severe Sickle Cell Disease:

08/11/2014

Photo: Courtesy of
the National Library of Medicine.

Greetings from the National Library of Medicine and MedlinePlus.gov

Regards to all our listeners!

I'm Rob Logan, Ph.D. senior staff U.S. National Library of Medicine for Donald Lindberg, M.D, the Director of the U.S. National Library of Medicine.

Here is what's new this week in MedlinePlus.

Bone marrow transplants reversed adult severe sickle cell disease in a small but encouraging study recently published (with an accompanying editorial) in the Journal of the American Medical Association.

The study found a bone marrow transplant successfully reversed severe sickle cell disease in 26 of 30 adults in a trial supervised by the National Heart, Lung, and Blood Institute. All participants had genetically matched marrow donors.

Fifteen of the 30 study participants (with severe sickle cell disease) also were able to stop taking medications that prevent the rejection of a bone marrow transplant within a year after the procedure. The findings suggest for the first time that bone marrow transplants may reverse severe sickle cell disease in adults as well as children.

The study’s senior author told the New York Times (and we quote): ‘This is what we hoped for’ (end of quote).

The transplant begins with treatment that uses chemotherapy and radiation to destroy some bone marrow before replacing it with healthy donor marrow cells. Unlike the prior, successful children’s research, the participants in the adult study deliberately retained some of their prior bone marrow in order to reduce the needed amount of donor marrow.

The transplant procedure was recommended only after the use of existing drug treatments failed to reverse sickle cell disease.

In an accompanying editorial, two physicians from the Washington University School of Medicine, explain sickle cell disease affects about 100,000 Americans, mostly African-Americans, and is found globally. The editorial’s authors add sickle cell disease is a genetic condition that damages the hemoglobin that carries oxygen in red blood cells. This can block blood flow through the veins and can result in anemia, pain, and organ damage.

While the editorial’s authors explained the study’s findings are based on a small sample and required genetically matched donors (who sometimes are unavailable), the authors noted the study suggests the current practice of limiting bone marrow transplants to children should be reconsidered.

The editorial’s authors conclude (and we quote): ‘In a population of relatively older adults with sickle cell disease, these findings offer hope’ (end of quote).

Meanwhile, a helpful overview of sickle cell disease is provided by the Mayo Foundation for Medical Education and Research in the ‘start here’ section of MedlinePlus.gov’s sickle cell anemia health topic page. The National Heart, Lung, and Blood Institute also provides a website about the treatment of sickle cell anemia in the ‘treatment’ section of MedlinePlus.gov’s sickle cell anemia health topic page.

MedlinePlus.gov’s sickle cell anemia health topic page additionally provides links to the latest pertinent journal research articles, which are available in the ‘journal articles’ section. Links to clinical trials that may be occurring in your area are available in the ‘clinical trials’ section. You can sign up to receive updates about sickle cell disease as they become available on MedlinePlus.gov.

To find MedlinePlus.gov’s sickle cell anemia health topic page type ‘sickle ‘s….i….c….k….l….e’ cell’ in the search box on MedlinePlus.gov’s home page, then, click on ‘sickle cell anemia (National Library of Medicine).’ NLM also has an article on bone marrow transplants that can be found by typing ‘bone marrow transplant’ in the search box on MedlinePlus.gov’s home page.

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A disclaimer — the information presented in this program should not replace the medical advice of your physician. You should not use this information to diagnose or treat any disease without first consulting with your physician or other health care provider.

It was nice to be with you. I look forward to meeting you here next week.