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St. Jude Children’s Research Hospital provides outstanding clinical care and drives innovative research programs in our quest for treatments and, ultimately, cures for children with catastrophic non-malignant hematological diseases. Below are selected highlights from 2011 and 2012.
Novel program helps St. Jude teens with sickle cell disease transition to adult care
Our teen patients with sickle cell disease have access to a new adult-care program designed to improve clinical and social outcomes and ultimately reduce mortality rates among recently transitioned young adults, ages 18 to 25. The program, known as the St. Jude-Methodist Sickle Cell Disease Transition Clinic, mirrors the evidence-based treatment and tailored interventions patients receive in our robust transition program for ages 12 to 18.
St. Jude collaborates on the first NIH-funded international sickle cell disease study
St. Jude is a participating site and the data coordinating center for Sparing Conversion to Abnormal TCD Elevation (SCATE), the first international sickle cell disease study funded by the National Institutes of Health. The Phase III trial includes patients from Brazil, Jamaica and the U.S. Our investigators co-designed the study to determine hydroxyurea’s efficacy in preventing conversion of conditional TCD levels to abnormal in patients with sickle cell disease. Patients with abnormal TCD levels are at increased risk for primary stroke.
Gene therapy achieves early success against hereditary bleeding disorder
Following a single treatment with gene therapy, propensity for bleeding decreased significantly in adults with hemophilia B. Results from this Phase I/II study are the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder.
New England Journal of Medicine, December 2011
Hydroxyurea shown safe and effective for infants and toddlers with sickle cell anemia
A multi-center study led by St. Jude finds that hydroxyurea, commonly used to treat sickle cell anemia in adults and older children, can be safely and effectively used in infants and toddlers. In the BABY HUG trial, hydroxyurea effectively reduced bouts of acute pain and a pneumonia-like illness, cut hospitalization time and eased other symptoms of the disease in these very young patients.
Lancet, May 2011
Hydroxyurea eases inflammation, pneumonia risk for sickle cell patients
Hydroxyurea, which boosts production of fetal hemoglobin in patients with sickle cell anemia, is found to also protect these patients from pneumococcal pneumonia by dampening the production of white blood cells that help drive inflammation and tissue damage.
Blood, February 2012