Patient and graft characteristics (N = 44)
| Variables . | Value . |
|---|---|
| Patient characteristics | |
| Age, median (range), y | 1.7 (0.4-16.6) |
| Weight, median (range), kg | 12 (6-74) |
| Male sex, n (%) | 27 (61) |
| Race, n (%) | |
| White | 29 (66) |
| African American | 8 (18) |
| Hispanic | 3 (7) |
| Asian | 1 (2) |
| Alaska Native | 1 (2) |
| 2 or more races | 2 (5) |
| Diagnosis, n (%) | |
| Krabbe disease | 13 (30) |
| Metachromatic leukodystrophy | 7 (16) |
| Sickle cell disease | 3 (7) |
| Gaucher disease | 2 (5) |
| Hunter syndrome (MPS type II) | 2 (5) |
| MHC class II deficiency | 2 (5) |
| Osteopetrosis | 2 (5) |
| XLP2 | 2 (5) |
| β-thalassemia | 1 (2) |
| Cartilage-hair hypoplasia | 1 (2) |
| Chédiak-Higashi syndrome | 1 (2) |
| Combined immunodeficiency with multiple intestinal atresias | 1 (2) |
| Diamond-Blackfan anemia | 1 (2) |
| GM3 synthase deficiency | 1 (2) |
| Hemophagocytic lymphohistiocytosis | 1 (2) |
| Hurler syndrome (MPS type IH) | 1 (2) |
| Severe congenital neutropenia | 1 (2) |
| Tay-Sachs disease | 1 (2) |
| X-linked adrenoleukodystrophy | 1 (2) |
| CMV serostatus, seropositive | 16 (36) |
| Graft characteristics | |
| HLA match (of 6), n (%) | |
| 6 | 16 (36) |
| 5 | 19 (43) |
| 4 | 9 (21) |
| HLA match (of 8), n (%) | |
| 8 | 8 (18) |
| 7 | 10 (23) |
| 6 | 14 (32) |
| 5 | 10 (23) |
| 4 | 2 (5) |
| TNC/kg, median (range), ×107 | 9.1 (2.3-24.8) |
| CD34/kg, median (range), ×105 | 3.31 (0.92-9.24) |
| Sex mismatch, n (%) | 22 (50) |
| ABO mismatch, n (%) | 28 (64) |
| Variables . | Value . |
|---|---|
| Patient characteristics | |
| Age, median (range), y | 1.7 (0.4-16.6) |
| Weight, median (range), kg | 12 (6-74) |
| Male sex, n (%) | 27 (61) |
| Race, n (%) | |
| White | 29 (66) |
| African American | 8 (18) |
| Hispanic | 3 (7) |
| Asian | 1 (2) |
| Alaska Native | 1 (2) |
| 2 or more races | 2 (5) |
| Diagnosis, n (%) | |
| Krabbe disease | 13 (30) |
| Metachromatic leukodystrophy | 7 (16) |
| Sickle cell disease | 3 (7) |
| Gaucher disease | 2 (5) |
| Hunter syndrome (MPS type II) | 2 (5) |
| MHC class II deficiency | 2 (5) |
| Osteopetrosis | 2 (5) |
| XLP2 | 2 (5) |
| β-thalassemia | 1 (2) |
| Cartilage-hair hypoplasia | 1 (2) |
| Chédiak-Higashi syndrome | 1 (2) |
| Combined immunodeficiency with multiple intestinal atresias | 1 (2) |
| Diamond-Blackfan anemia | 1 (2) |
| GM3 synthase deficiency | 1 (2) |
| Hemophagocytic lymphohistiocytosis | 1 (2) |
| Hurler syndrome (MPS type IH) | 1 (2) |
| Severe congenital neutropenia | 1 (2) |
| Tay-Sachs disease | 1 (2) |
| X-linked adrenoleukodystrophy | 1 (2) |
| CMV serostatus, seropositive | 16 (36) |
| Graft characteristics | |
| HLA match (of 6), n (%) | |
| 6 | 16 (36) |
| 5 | 19 (43) |
| 4 | 9 (21) |
| HLA match (of 8), n (%) | |
| 8 | 8 (18) |
| 7 | 10 (23) |
| 6 | 14 (32) |
| 5 | 10 (23) |
| 4 | 2 (5) |
| TNC/kg, median (range), ×107 | 9.1 (2.3-24.8) |
| CD34/kg, median (range), ×105 | 3.31 (0.92-9.24) |
| Sex mismatch, n (%) | 22 (50) |
| ABO mismatch, n (%) | 28 (64) |
MHC, major histocompatibility complex; MPS, mucopolysaccharidosis; TNC, total nucleated cell; XLP2, X-linked lymphoproliferative syndrome type 2.