GVHD is not diminished in congenital acid sphingomyelinase deficiency, Niemann-Pick A/B disease
| Graft/disease . | N . | Probability . | |
|---|---|---|---|
| Acute GVHD, grades 2-4, at day 100 after HCT (95% CI) . | Chronic GVHD at 3 y after HCT (95% CI) . | ||
| MSD BM | |||
| Niemann-Pick | 13 | 30% (9%-55%) | 12% (6%-49%) |
| Other IEM* | 202 | 22% (16%-28%) | 9% (6%-14%) |
| URD BM + CB | |||
| Niemann-Pick | 11 | 18% (3%-44%) | None† |
| Other IEM* | 543 | 39% (34%-43%) | 20% (17%-24%) |
| Total | |||
| Niemann-Pick | 24 | 25% (10%-43%) | 13% (3%-30%) |
| Other IEM* | 745 | 34% (31%-37%) | 16% (13%-19%) |
| Graft/disease . | N . | Probability . | |
|---|---|---|---|
| Acute GVHD, grades 2-4, at day 100 after HCT (95% CI) . | Chronic GVHD at 3 y after HCT (95% CI) . | ||
| MSD BM | |||
| Niemann-Pick | 13 | 30% (9%-55%) | 12% (6%-49%) |
| Other IEM* | 202 | 22% (16%-28%) | 9% (6%-14%) |
| URD BM + CB | |||
| Niemann-Pick | 11 | 18% (3%-44%) | None† |
| Other IEM* | 543 | 39% (34%-43%) | 20% (17%-24%) |
| Total | |||
| Niemann-Pick | 24 | 25% (10%-43%) | 13% (3%-30%) |
| Other IEM* | 745 | 34% (31%-37%) | 16% (13%-19%) |
N indicates number of cases; CI, confidence interval; BM, bone marrow; CB, cord blood; IEM, inborn errors of metabolism; MSD, matched sibling donor; and URD, unrelated donor.
Unrelated donor-recipient matching at human leukocyte antigens (HLAs) for BM recipients included 2 cases of 6/6 antigen-matched and 1 case of 5/6 antigen-matched. After typing at the HLA-C locus became available, 1 transplantation was performed that was 6/8 antigen-matched (mismatch at the HLA-B locus, and allele mismatch at the HLA-C locus). For CB transplants, 1 was 6/6, 5 were 5/6, and 1 was 4/6 antigen-matched.
Similarly aged patients with IEM from the CIBMTR database who underwent transplantation between 1987 and 2007.
No patient developed chronic GVHD.