Population and HCT characteristics
| Total patient number, n = 145 . | ARTEMIS, n = 69 . | RAG1/2, n = 76 . | P . |
|---|---|---|---|
| Male/female, n | 38/31 | 39/37 | .6 |
| Patients contributed by each center, n (%) | .004 | ||
| University of California, San Francisco | 23 (33) | 9 (12) | |
| Paris Necker | 24 (35) | 42 (55) | |
| Ulm University | 22 (32) | 25 (33) | |
| Pretransplant characteristics | |||
| Transplantation prior to 1995, n (%) | 27 (39) | 18 (24) | .05 |
| Median age in mo at transplantation (range in mo) | 7 (0.5-56) | 7 (0.6-27) | .1 |
| Maternal T-cell engraftment, n (%)* | 19 (28) | 16 (24) | .4 |
| Viral infection prior to transplantation, n (%) | 33 (48) | 35 (46) | .8 |
| Orogenital ulcers, n (%) | 16 (23) | 5 (7) | .006 |
| Omenn syndrome, n (%) | 1 (1.5) | 14 (18) | .001 |
| Transplant characteristics | |||
| Median follow-up (range in mo) | 51 (0.5-333) | 32 (1-333) | .3 |
| Conditioning regimen, n (%) | .06 | ||
| None or immunosuppression only† | 40 (58) | 29 (38) | |
| Low-dose busulfan (8 mg/kg)‡ | 6 (9) | 10 (13) | |
| High-dose busulfan (16 mg/kg)‡ | 23 (33) | 37 (49) | |
| Any alkylator therapy§ | 41 (59) | 47 (62) | .6 |
| Donor, n (%) | .1 | ||
| MSD|| | 9 (13) | 15 (20) | |
| MFD|| | 8 (12) | 10 (13) | |
| MMFD¶ | 8 (12) | 5 (7) | |
| Haploidentical# | 43 (62) | 39 (51) | |
| MUD¶ | 1 (1) | 7 (9) | |
| Additional procedure, n (%) | |||
| Retransplant | 17 (25) | 21 (28) | .7 |
| Boost | 20 (29) | 8 (10) | .005 |
| Total patient number, n = 145 . | ARTEMIS, n = 69 . | RAG1/2, n = 76 . | P . |
|---|---|---|---|
| Male/female, n | 38/31 | 39/37 | .6 |
| Patients contributed by each center, n (%) | .004 | ||
| University of California, San Francisco | 23 (33) | 9 (12) | |
| Paris Necker | 24 (35) | 42 (55) | |
| Ulm University | 22 (32) | 25 (33) | |
| Pretransplant characteristics | |||
| Transplantation prior to 1995, n (%) | 27 (39) | 18 (24) | .05 |
| Median age in mo at transplantation (range in mo) | 7 (0.5-56) | 7 (0.6-27) | .1 |
| Maternal T-cell engraftment, n (%)* | 19 (28) | 16 (24) | .4 |
| Viral infection prior to transplantation, n (%) | 33 (48) | 35 (46) | .8 |
| Orogenital ulcers, n (%) | 16 (23) | 5 (7) | .006 |
| Omenn syndrome, n (%) | 1 (1.5) | 14 (18) | .001 |
| Transplant characteristics | |||
| Median follow-up (range in mo) | 51 (0.5-333) | 32 (1-333) | .3 |
| Conditioning regimen, n (%) | .06 | ||
| None or immunosuppression only† | 40 (58) | 29 (38) | |
| Low-dose busulfan (8 mg/kg)‡ | 6 (9) | 10 (13) | |
| High-dose busulfan (16 mg/kg)‡ | 23 (33) | 37 (49) | |
| Any alkylator therapy§ | 41 (59) | 47 (62) | .6 |
| Donor, n (%) | .1 | ||
| MSD|| | 9 (13) | 15 (20) | |
| MFD|| | 8 (12) | 10 (13) | |
| MMFD¶ | 8 (12) | 5 (7) | |
| Haploidentical# | 43 (62) | 39 (51) | |
| MUD¶ | 1 (1) | 7 (9) | |
| Additional procedure, n (%) | |||
| Retransplant | 17 (25) | 21 (28) | .7 |
| Boost | 20 (29) | 8 (10) | .005 |
Values were compared using a χ2 test for binary variable, and a Kruskal-Wallis nonparametric test for continuous variables.
MFD, matched family donor; MMFD, mismatched family donor; MSD, matched sibling donor; MUD, matched unrelated donor.
Following transplacental transfusion.
Cyclophosphamide or fludarabine with or without serotherapy (antithymocyte globulin, alemtuzumab, anti-LFA-1/anti-CD2) or serotherapy only.
Associated with cyclophosphamide or fludarabine.
Category analyzing all conditioning regimens containing the following alkylating agents including both immunosuppressive and stem cell–toxic regimens: busulfan, treosulfan, thiothepa, melphalan, cyclophosphamide.
No conditioning, no T-cell depletion (except for 3 patients with Omenn syndrome transplanted with conditioning).
No conditioning, no T-cell depletion.
Transplanted with or without conditioning, T-cell depletion.