Table 3.

Overview of outcomes related to BMT platform.

	Patients (n=20)
Graft failure	2 (10%)
Primary graft failure	1 (5%)
Secondary graft failure	1 (5%)
1-year transplant-related mortality	2 (10%)
Acute GVHD
Any grade	4 (20%)
Grade II-IV	3 (15%)
Grade III-IV	1 (5%)
Chronic GVHD	0
Relapse of pre-BMT lymphoproliferative disorder/lymphoma, n=6	0
Sinusoidal obstructive syndrome&	1 (5%)
Idiopathic pneumonia syndrome	1 (5%)
Post-BMT donor CD3+ T-lymphocyte infusion, n (%)	4 (20%)
For mixed chimerism or slow engraftment	3 (15%)
For CMV disease	1 (5%)
Second transplant, n (%)	1 (5%)
Freedom from transfusion, n=18	18 (100%)
Freedom from post-BMT immunosuppression, n=18	17 (94%)
Freedom from Ig replacement (n=13 survivors on pre-BMT Ig replacement)	10 (77%)
Evidence of phenotype reversal among those with unknown genetic defect, n=3	3 (100%)
Evidence of phenotype reversal among patients with significantly mixed chimerism, n=2	2 (100%)

Abbreviations: BMT, bone marrow transplantation; GVHD, graft-versus-host disease; CMV, cytomegalovirus; Ig, immunoglobulin

^&P10 developed sinusoidal obstructive syndrome without reversal of flow on ultrasound or fulfillment of Baltimore criteria, but histologically proven on day +22 liver biopsy performed for hyperbilirubinemia that additionally showed nodular regenerative hyperplasia (presumed to have been present pre-BMT due to prior 6-mercaptopurine treatment), cholestasis of sepsis, and mild hemosiderosis.