MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

0.5 mg/kg IV over 6 hours on day -9 and 2 mg/kg IV over 4 hours on day -8 and day -7 with premeds and solumedrol through day -2

14.5 mg/kg IV over 1 hour day -6 and -5 50 mg/kg IV over 2 hours with mesna 40 mg/kg IV day +2 and +3

30 mg/m2 IV over 60 minutes days -6 through day -2

See arm description for dosing.

Bone marrow infusion on Day 0

Day +5 through day +100 with goals of 5-10 ug/L (not used for HLA-identical related donors). When used in non-MSD recipients, tapered over 6-8 weeks starting at day +100.

15 mg/kg IV q8h (based upon actual body weight) with the maximum total daily dose not to exceed 3 grams. Day +5 through day 35

Day 60, 100 and 180 (collected during donor BM harvest for graft)

busulfan IV over 3 hours on days -3 and -2 for HLA-mismatched BM recipients only (Arms F and G)