Patient, disease, and transplantation characteristics for 249 children with severe T-cell deficiencies who received either UCBT or MMRDT
| . | UCBT (n = 74) . | MMRDT (n = 175) . | P . |
|---|---|---|---|
| Median age at transplantation, mo (range) | 6.4 (1-41) | 6.5 (1-35) | .87 |
| Median time from diagnosis to transplantation, d (range) | 55 (17-523) | 46 (4-485) | .06 |
| Male sex, n (%) | 42 (57%) | 112 (64%) | .28 |
| Year of transplantation, n (%) | |||
| 1995-2000 | 26 (35%) | 87 (50%) | .03 |
| 2001-2005 | 48 (65%) | 88 (50%) | |
| Diagnosis, n (%) | |||
| SCID T−B* | 36 (49%) | 62 (36%) | .01 |
| Undefined T−B− | 24 | 29 | |
| RAG1 or RAG2 deficiency | 2 | 23 | |
| ADA deficiency | 7 | 3 | |
| Reticular dysgenesis | 3 | 7 | |
| SCID T−B+ | 23 (31%) | 90 (51%) | |
| Undefined T−B+ | 7 | 38 | |
| Gamma-chain deficiency | 15 | 39 | |
| JAK3 deficiency | 1 | 13 | |
| Omenn syndrome | 15 (20%) | 23 (13%) | |
| Pretransplantation characteristics, events/available data, n (%) | |||
| Failure to thrive | 39/69 (57%) | 76/167 (46%) | .12 |
| Chronic diarrhea | 28/66 (42%) | 59/167 (35%) | .31 |
| Respiratory impairment | 36/68 (53%) | 84/166 (51%) | .75 |
| Infections | |||
| Viral | 23/69 (33%) | 83/164 (51%) | .02 |
| Bacterial | 21/69 (30%) | 59/128 (36%) | .42 |
| Fungal | 12/69 (17%) | 37/127 (23%) | .38 |
| Mycobacterial | 8/69 (12%) | 13/164 (8%) | .37 |
| P jiroveci | 8/69 (12%) | 28/164 (17%) | .29 |
| Transplantation characteristics, n (%) | |||
| Cell source | |||
| BM | 99 (56%) | ||
| PBSCs | 68 (39%) | ||
| BM + PBSCs | 8 (5%) | ||
| UCB | 74 (100%) | ||
| No. of HLA mismatches | |||
| 0 | 21 (28%) | ||
| 1 | 29 (39%) | 15 (9%) | |
| 2 or 3 | 24 (33%) | 160 (91%) | |
| Conditioning regimen | |||
| No conditioning | 7 (10%) | 30 (17%) | .04 |
| Myeloablative (busulfan-based) | 46 (62%) | 81 (46%) | |
| Reduced-intensity | 20 (27%) | 64 (37%) | |
| ATG alone | 5 | 6 | |
| Cy ± other | 2 | 10 | |
| Bu based (< 8 mg/Kg) | 7 | 40 | |
| Other | 6 | 8 | |
| Missing data | 1 (1%) | ||
| Use of ATG or other mAbs | 36 (49%) | 47 (27%) | .001 |
| GVHD prophylaxis | |||
| None | 1 (1.3%) | 119 (68%) | |
| CsA-based | 71 (96%) | 48 (27%) | |
| Other | 1 (1.3%) | 2 (1%) | |
| Missing data | 1 (1.3%) | 6 (4%) | |
| Median follow-up, mo (range) | 83 (5-162) | 58 (3-157) |
| . | UCBT (n = 74) . | MMRDT (n = 175) . | P . |
|---|---|---|---|
| Median age at transplantation, mo (range) | 6.4 (1-41) | 6.5 (1-35) | .87 |
| Median time from diagnosis to transplantation, d (range) | 55 (17-523) | 46 (4-485) | .06 |
| Male sex, n (%) | 42 (57%) | 112 (64%) | .28 |
| Year of transplantation, n (%) | |||
| 1995-2000 | 26 (35%) | 87 (50%) | .03 |
| 2001-2005 | 48 (65%) | 88 (50%) | |
| Diagnosis, n (%) | |||
| SCID T−B* | 36 (49%) | 62 (36%) | .01 |
| Undefined T−B− | 24 | 29 | |
| RAG1 or RAG2 deficiency | 2 | 23 | |
| ADA deficiency | 7 | 3 | |
| Reticular dysgenesis | 3 | 7 | |
| SCID T−B+ | 23 (31%) | 90 (51%) | |
| Undefined T−B+ | 7 | 38 | |
| Gamma-chain deficiency | 15 | 39 | |
| JAK3 deficiency | 1 | 13 | |
| Omenn syndrome | 15 (20%) | 23 (13%) | |
| Pretransplantation characteristics, events/available data, n (%) | |||
| Failure to thrive | 39/69 (57%) | 76/167 (46%) | .12 |
| Chronic diarrhea | 28/66 (42%) | 59/167 (35%) | .31 |
| Respiratory impairment | 36/68 (53%) | 84/166 (51%) | .75 |
| Infections | |||
| Viral | 23/69 (33%) | 83/164 (51%) | .02 |
| Bacterial | 21/69 (30%) | 59/128 (36%) | .42 |
| Fungal | 12/69 (17%) | 37/127 (23%) | .38 |
| Mycobacterial | 8/69 (12%) | 13/164 (8%) | .37 |
| P jiroveci | 8/69 (12%) | 28/164 (17%) | .29 |
| Transplantation characteristics, n (%) | |||
| Cell source | |||
| BM | 99 (56%) | ||
| PBSCs | 68 (39%) | ||
| BM + PBSCs | 8 (5%) | ||
| UCB | 74 (100%) | ||
| No. of HLA mismatches | |||
| 0 | 21 (28%) | ||
| 1 | 29 (39%) | 15 (9%) | |
| 2 or 3 | 24 (33%) | 160 (91%) | |
| Conditioning regimen | |||
| No conditioning | 7 (10%) | 30 (17%) | .04 |
| Myeloablative (busulfan-based) | 46 (62%) | 81 (46%) | |
| Reduced-intensity | 20 (27%) | 64 (37%) | |
| ATG alone | 5 | 6 | |
| Cy ± other | 2 | 10 | |
| Bu based (< 8 mg/Kg) | 7 | 40 | |
| Other | 6 | 8 | |
| Missing data | 1 (1%) | ||
| Use of ATG or other mAbs | 36 (49%) | 47 (27%) | .001 |
| GVHD prophylaxis | |||
| None | 1 (1.3%) | 119 (68%) | |
| CsA-based | 71 (96%) | 48 (27%) | |
| Other | 1 (1.3%) | 2 (1%) | |
| Missing data | 1 (1.3%) | 6 (4%) | |
| Median follow-up, mo (range) | 83 (5-162) | 58 (3-157) |
Myeloablative conditioning regimen in these patients consisted of Bu (> 8 mg/kg) and Cy (200 mg/kg total dose); no irradiation was used.
RAG indicates recombination activating gene; ADA, adenosine deaminase; PBSC, peripheral blood stem cell; UCB, umbilical cord blood; ATG, anti-thymocyte globulin; Bu, busulfan; Cy, cyclophosphamide; and CsA, cyclosporine A.
This form of SCID is characterized by the complete absence of T and B lymphocytes (T−B−).