Table 7.
Outcomes of hematopoietic stem cell transplantation in patients with Hurler syndrome and adrenoleukodystrophy: comparison of our data with published reports
| Author | Center or group | Study years | Donor source (no. patients) | Median follow-up, y | OS after first transplantation, % | Engraftment, % | Patients achieving high (> 90%) donor chimerism, % |
|---|---|---|---|---|---|---|---|
| Hurler syndrome | |||||||
| Bolens et al45 | EBMT | 1994-2004 | R-BM/PBSC (49), R-CB (3), U-CB (20), U-BM/PBSC (70) | 3.3 | 57 | ||
| Peters et al5 | 14 centers | 1989-1994 | U-BM (40) | 1.3 | 49 at 2 y | 63 | |
| Peters et al6 | 13 centers | 1983-1995 | R-M-BM (carrier (13); normal (15)) | 7.3 | 75 at 5 y | 85 | 54.0 |
| R-MM-BM (carrier (23); normal (3)) | 4.6 | 53 at 5 y | 61 | 62.0 | |||
| Souillet et al46 | Lyon, France, single center | 1986-2001 | U-BM (15) | 4.7 | 82 | 3 of 17 autologous recovery or rejection | 47.1 |
| R-BM (10); R-CB (2) | 4.7 | 90 | 4 of 12 autologous recovery or rejection | 33.3 | |||
| Current study | Duke University | 1995-2007 | U-CB (45) | 5.8 | 77.3 at 1 y and 74.5 at 5 y | 88.9 | 88.9 |
| Adrenoleukodystrophy | |||||||
| Peters et al7 | 43 centers | 1982-1999 | R-M-BM (33); R-MM-BM (9) | 3.1 | 64 | 93 | |
| U-BM (40); U-CB (12) | 3.1 | 53 | 80 | ||||
| Current study | Duke University | 1995-2007 | U-CB (13) | 6 | 76.9 at 1 y and 69.1 at 5 y | 84.6 | 84.6 |
EBMT indicates European Group for Blood and Marrow Transplantation (Barcelona, Spain); R-BM, related bone marrow; R-M-BM, related matched bone marrow; R-MM-BM, related mismatched bone marrow; R-PBSC, related peripheral blood stem cell; R-CB, related cord blood; U-CB, unrelated cord blood; and U-BM, unrelated bone marrow.