Immune Disorder HSCT Protocol

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Inclusion Criteria:

• </= 28 years of age
• Performance status >/= 40
• DLCO >/= 40%
• LVEF >/=40% or LVSF >/=26%
• Serum creatinine < 2x ULN
• Liver enzymes </= 5x ULN
• Negative pregnancy test
• Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord)

Exclusion Criteria:

• Known diagnosis of HIV I/II
• Pregnant or breastfeeding
• Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab
• Uncontrolled viral infection within 1 week prior to starting alemtuzumab