Abstract
Primary immunodeficiency patients may develop pulmonary complications & most are ineligible for either lung transplant or BMT due to futility. We report on our first 2 subjects enrolled on NCT01852370 trial receiving tandem BOLT & BMT from the same deceased UNOS donor. We hypothesized: 1) hematopoietic stem cells prepared from organ donor vertebral bodies (VB) would yield greater CD34+ cell dose compared to iliac crest aspirates-with limited by harvest time due to parallel organ recovery teams; 2) could generate durable hematopoiesis & donor derived immunity; 3) persistent mixed chimerism could lead to tolerance (bidirectional) following withdrawal of immunosuppressive drugs (ISD).
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