Millions worldwide suffer complications from sickle cell disease. SCD is characterized by chronic vaso-occlusive complications, multiple organ dysfunction, and premature death. Current therapies for SCD include supportive care with penicillin, vaccination and narcotics, chronic transfusion, and/or hydroxyurea.
The only curative therapy for SCD is Myeloablative (MA) Allogeneic Stem Cell Transplantation (AlloSCT) from an unaffected HLA matched sibling donor. However, only approximately 15% of SCD patients have such donors. An alternate allogeneic donor source includes unaffected familial haploidentical (FHI) donors. The Haplo SCD Consortium was developed to provide resources to families, patients, and physicians pursuing FHI transplantation for SCD.
To date we have developed a Consortium of five clinical transplant sites, three processing centers, and cores in donor chimerism, anti-HLA antibodies, immunology, cell processing, biostatistics, neuroimaging, neurocognition, quality of life, pulmonary function, pulmonary vascular, radiation therapy, long term effects, hepatic imaging, and endothelial biomarkers. We've also partnered with the Children’s Sickle Cell Foundation and the Sickle Cell Disease Association of Illinois (SCDAI) to help educate patients and their families regarding sickle cell disease and familial haploidentical stem cell transplant.
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