Hereditary non-malignant disorders (NMD) such as aplastic anemia and sickle cell disease (SCD) result in morbidity and early mortality. These disorders can be cured by hematopoietic stem cell transplants (HCT). Over 50% of pediatric transplants currently are for NMD indications. The probability of identifying an HLA-matched donor are slim (<25%) especially for patients of mixed or minority ethnicity. Side effects of HCT such as infections, sterility, rejection, mortality, and graft-versus-host disease (GVHD) inhibit use of mismatched donors. This project tests a novel HCT approach using half-matched family donors and targets these barriers to enhance successful HCT.
• Determine overall and disease free survival after familial haploidentical (half-matched) HCT in children with NMD.
• Determine HCT specific outcomes - GVHD, immune recovery, drug pharmacokinetics, cognitive and quality of life evaluations.
• Determine disease related organ functions after HCT for SCD - Specialized serial neuroimaging in collaboration with experts at this center.
Potential impact on child health
Haploidentical family member donors are available to a majority of children with life-threatening disorders cured by transplant. If the novel technique proposed here can successfully eradicate transplant-associated complications after haploidentical HCT, this resource can be routinely utilized for curing childhood genetic disorders and facilitate larger clinical trials.
Sima Bhatt, M.D., Melanie Fields, M.D., Kristin Guilliams, M.D., Allison King, M.D., and Joshua Shimony, M.D.