Children’s Hospital of Philadelphia – Bone Marrow Failure Syndrome

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The primary objective of this protocol is to expand access for patients who lack a fully HLA
(Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic
hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately
life-threatening disease for which HSCT is indicated. These patients are not eligible for
other Children’s Hospital of Philadelphia Institutional Review Board (IRB) approved
protocols that utilize CliniMACs technology for T depletion.

Rare Disease: