Characteristics of patients with nonmalignant disease who have high-resolution typing categorized by the number of allele-level mismatches at HLA-A, -B, -C, and -DRB1
| Variable . | 8/8, N (%) . | 7/8, N (%) . | 6/8, N (%) . | P . |
|---|---|---|---|---|
| No. of patients | 372 | 191 | 100 | |
| No. of centers | 81 | 63* | 38* | |
| Median patient age, y (range) | 9 (< 1-71) | 10 (< 1-58) | 9 (< 1-54) | .42 |
| Age by decade | .09 | |||
| 0-9 | 192 (52) | 94 (49) | 57 (57) | |
| 10-19 | 84 (23) | 46 (24) | 25 (25) | |
| 20-29 | 33 (9) | 24 (13) | 12 (12) | |
| 30-39 | 38 (10) | 12 (6) | 2 (2) | |
| 40-49 | 8 (2) | 9 (5) | 3 (3) | |
| 50+ | 17 (5) | 6 (3) | 1 (1) | |
| Race | .01 | |||
| White | 292 (78) | 129 (68) | 63 (63) | |
| Black | 18 (5) | 22 (11) | 11 (11) | |
| Asian/Pacific Islander | 14 (4) | 10 (5) | 5 (5) | |
| Hispanic | 44 (12) | 28 (15) | 19 (19) | |
| Native American | 1 (< 1) | 0 | 0 | |
| Other | 3 (1) | 2 (1) | 2 (2) | |
| Sex of recipient | .72 | |||
| Male | 214 (58) | 112 (59) | 62 (62) | |
| Female | 158 (42) | 79 (41) | 38 (38) | |
| Karnofsky score | .49 | |||
| < 90 | 67 (20) | 42 (23) | 24 (25) | |
| ≥ 90 | 270 (80) | 142 (77) | 72 (75) | |
| Disease groups | .89 | |||
| Acquired severe aplastic anemia | 147 (40) | 75 (39) | 35 (35) | |
| Fanconi anemia | 46 (12) | 25 (13) | 16 (16) | |
| Histiocytic disorders | 36 (10) | 17 (9) | 11 (11) | |
| SCIDs and other severe T-cell deficiencies | 15 (4) | 14 (7) | 6 (6) | |
| Wiskott-Aldrich syndrome | 20 (5) | 8 (4) | 6 (6) | |
| Hurler syndrome | 20 (5) | 10 (5) | 3 (3) | |
| Other | 88 (24) | 42 (22) | 23 (23) | |
| CMV matching, donor/recipient | ||||
| Negative/negative | 144 (39) | 46 (24) | 26 (26) | .004 |
| Negative/positive | 99 (27) | 63 (33) | 35 (35) | |
| Positive/negative | 46 (12) | 32 (17) | 21 (21) | |
| Positive/positive | 76 (20) | 47 (25) | 14 (14) | |
| Missing | 7 (2) | 3 (2) | 4 (4) | |
| Time from diagnosis to TX, in mo | 11 (1-318) | 15 (1-340) | 17 (3-186) | .07 |
| Conditioning regimen | .02 | |||
| Myeloablative | 151 (41) | 99 (52) | 51 (51) | |
| Reduced intensity | 132 (35) | 57 (30) | 37 (37) | |
| Nonmyeloblative | 89 (24) | 35 (18) | 12 (12) | |
| GVHD prophylaxis | .0003 | |||
| Ex vivo TCD | 66 (18) | 50 (26) | 36 (36) | |
| No TCD | 306 (82) | 141 (74) | 64 (64) | |
| Graft type | .09 | |||
| Bone marrow | 305 (82) | 153 (80) | 90 (90) | |
| Peripheral blood | 67 (18) | 38 (20) | 10 (10) | |
| ATG given | .18 | |||
| Yes | 256 (69) | 123 (64) | 75 (75) | |
| No | 116 (31) | 68 (36) | 25 (25) | |
| Alemtuzumab given | .89 | |||
| Yes | 42 (11) | 24 (13) | 11 (11) | |
| No | 330 (89) | 167 (87) | 89 (89) | |
| Sex match, donor→recipient | .17 | |||
| Male→male | 144 (39) | 62 (32) | 36 (36) | |
| Male→female | 97 (26) | 45 (24) | 17 (17) | |
| Female→male | 70 (19) | 50 (26) | 26 (26) | |
| Female→female | 61 (16) | 34 (18) | 21 (21) | |
| HLA-DQB1 matching | .02 | |||
| Matched | 336 (90) | 156 (82) | 81 (81) | |
| Single mismatch | 35 (9) | 32 (17) | 17 (17) | |
| Double mismatch | 1 (< 1) | 3 (2) | 2 (2) | |
| HLA-DPB1 matching | .14 | |||
| Matched | 52 (14) | 20 (11) | 6 (6) | |
| Single mismatch | 210 (56) | 110 (58) | 55 (55) | |
| Double mismatch | 110 (30) | 60 (32) | 39 (39) | |
| Missing HLA-DPB1 typing | 0 | 1 (< 1) | 0 | |
| Median donor age, y (range) | 33 (18-61) | 34 (20-54) | 36 (19-59) | .16 |
| Donor age, y | .05 | |||
| 18-19 | 7 (2) | 0 | 1 (1) | |
| 20-29 | 137 (37) | 63 (33) | 24 (24) | |
| 30-39 | 118 (32) | 66 (35) | 42 (42) | |
| 40-49 | 86 (23) | 55 (29) | 30 (30) | |
| 50 and older | 24 (6) | 7 (4) | 3 (3) | |
| Bone marrow CD34+ dose, ×106/kg | 4.6 (0.8-36.7) | 3.4 (0.5-18.4) | 4.9 (1-18.7) | .04 |
| Bone marrow total nucleated cell dose, ×108/kg | 3.2 (< 1-21.3) | 2.9 (< 1-20.7) | 2.9 (< 1-15.5) | .08 |
| PBSC CD34+ dose, ×106/kg | 8.5 (0.5-36.8) | 7.1 (2.9-28) | 3.7 (2.6-3.8) | .01 |
| PBSC mononucleated cell dose, ×108/kg | 6.0 (0.09-44.5) | 4.9 (0.02-18.6) | 2.5 (0.04-25.2) | .10 |
| Year of transplantation | < .0001 | |||
| 1995-1998 | 49 (13) | 36 (19) | 33 (33) | |
| 1999-2001 | 55 (15) | 40 (21) | 32 (32) | |
| 2002-2004 | 86 (23) | 41 (21) | 16 (16) | |
| 2005-2007 | 182 (49) | 74 (39) | 19 (19) | |
| Median follow-up of survivors, mo | 36 (3-156) | 47 (3-165) | 77 (12-159) | < .0001 |
| Variable . | 8/8, N (%) . | 7/8, N (%) . | 6/8, N (%) . | P . |
|---|---|---|---|---|
| No. of patients | 372 | 191 | 100 | |
| No. of centers | 81 | 63* | 38* | |
| Median patient age, y (range) | 9 (< 1-71) | 10 (< 1-58) | 9 (< 1-54) | .42 |
| Age by decade | .09 | |||
| 0-9 | 192 (52) | 94 (49) | 57 (57) | |
| 10-19 | 84 (23) | 46 (24) | 25 (25) | |
| 20-29 | 33 (9) | 24 (13) | 12 (12) | |
| 30-39 | 38 (10) | 12 (6) | 2 (2) | |
| 40-49 | 8 (2) | 9 (5) | 3 (3) | |
| 50+ | 17 (5) | 6 (3) | 1 (1) | |
| Race | .01 | |||
| White | 292 (78) | 129 (68) | 63 (63) | |
| Black | 18 (5) | 22 (11) | 11 (11) | |
| Asian/Pacific Islander | 14 (4) | 10 (5) | 5 (5) | |
| Hispanic | 44 (12) | 28 (15) | 19 (19) | |
| Native American | 1 (< 1) | 0 | 0 | |
| Other | 3 (1) | 2 (1) | 2 (2) | |
| Sex of recipient | .72 | |||
| Male | 214 (58) | 112 (59) | 62 (62) | |
| Female | 158 (42) | 79 (41) | 38 (38) | |
| Karnofsky score | .49 | |||
| < 90 | 67 (20) | 42 (23) | 24 (25) | |
| ≥ 90 | 270 (80) | 142 (77) | 72 (75) | |
| Disease groups | .89 | |||
| Acquired severe aplastic anemia | 147 (40) | 75 (39) | 35 (35) | |
| Fanconi anemia | 46 (12) | 25 (13) | 16 (16) | |
| Histiocytic disorders | 36 (10) | 17 (9) | 11 (11) | |
| SCIDs and other severe T-cell deficiencies | 15 (4) | 14 (7) | 6 (6) | |
| Wiskott-Aldrich syndrome | 20 (5) | 8 (4) | 6 (6) | |
| Hurler syndrome | 20 (5) | 10 (5) | 3 (3) | |
| Other | 88 (24) | 42 (22) | 23 (23) | |
| CMV matching, donor/recipient | ||||
| Negative/negative | 144 (39) | 46 (24) | 26 (26) | .004 |
| Negative/positive | 99 (27) | 63 (33) | 35 (35) | |
| Positive/negative | 46 (12) | 32 (17) | 21 (21) | |
| Positive/positive | 76 (20) | 47 (25) | 14 (14) | |
| Missing | 7 (2) | 3 (2) | 4 (4) | |
| Time from diagnosis to TX, in mo | 11 (1-318) | 15 (1-340) | 17 (3-186) | .07 |
| Conditioning regimen | .02 | |||
| Myeloablative | 151 (41) | 99 (52) | 51 (51) | |
| Reduced intensity | 132 (35) | 57 (30) | 37 (37) | |
| Nonmyeloblative | 89 (24) | 35 (18) | 12 (12) | |
| GVHD prophylaxis | .0003 | |||
| Ex vivo TCD | 66 (18) | 50 (26) | 36 (36) | |
| No TCD | 306 (82) | 141 (74) | 64 (64) | |
| Graft type | .09 | |||
| Bone marrow | 305 (82) | 153 (80) | 90 (90) | |
| Peripheral blood | 67 (18) | 38 (20) | 10 (10) | |
| ATG given | .18 | |||
| Yes | 256 (69) | 123 (64) | 75 (75) | |
| No | 116 (31) | 68 (36) | 25 (25) | |
| Alemtuzumab given | .89 | |||
| Yes | 42 (11) | 24 (13) | 11 (11) | |
| No | 330 (89) | 167 (87) | 89 (89) | |
| Sex match, donor→recipient | .17 | |||
| Male→male | 144 (39) | 62 (32) | 36 (36) | |
| Male→female | 97 (26) | 45 (24) | 17 (17) | |
| Female→male | 70 (19) | 50 (26) | 26 (26) | |
| Female→female | 61 (16) | 34 (18) | 21 (21) | |
| HLA-DQB1 matching | .02 | |||
| Matched | 336 (90) | 156 (82) | 81 (81) | |
| Single mismatch | 35 (9) | 32 (17) | 17 (17) | |
| Double mismatch | 1 (< 1) | 3 (2) | 2 (2) | |
| HLA-DPB1 matching | .14 | |||
| Matched | 52 (14) | 20 (11) | 6 (6) | |
| Single mismatch | 210 (56) | 110 (58) | 55 (55) | |
| Double mismatch | 110 (30) | 60 (32) | 39 (39) | |
| Missing HLA-DPB1 typing | 0 | 1 (< 1) | 0 | |
| Median donor age, y (range) | 33 (18-61) | 34 (20-54) | 36 (19-59) | .16 |
| Donor age, y | .05 | |||
| 18-19 | 7 (2) | 0 | 1 (1) | |
| 20-29 | 137 (37) | 63 (33) | 24 (24) | |
| 30-39 | 118 (32) | 66 (35) | 42 (42) | |
| 40-49 | 86 (23) | 55 (29) | 30 (30) | |
| 50 and older | 24 (6) | 7 (4) | 3 (3) | |
| Bone marrow CD34+ dose, ×106/kg | 4.6 (0.8-36.7) | 3.4 (0.5-18.4) | 4.9 (1-18.7) | .04 |
| Bone marrow total nucleated cell dose, ×108/kg | 3.2 (< 1-21.3) | 2.9 (< 1-20.7) | 2.9 (< 1-15.5) | .08 |
| PBSC CD34+ dose, ×106/kg | 8.5 (0.5-36.8) | 7.1 (2.9-28) | 3.7 (2.6-3.8) | .01 |
| PBSC mononucleated cell dose, ×108/kg | 6.0 (0.09-44.5) | 4.9 (0.02-18.6) | 2.5 (0.04-25.2) | .10 |
| Year of transplantation | < .0001 | |||
| 1995-1998 | 49 (13) | 36 (19) | 33 (33) | |
| 1999-2001 | 55 (15) | 40 (21) | 32 (32) | |
| 2002-2004 | 86 (23) | 41 (21) | 16 (16) | |
| 2005-2007 | 182 (49) | 74 (39) | 19 (19) | |
| Median follow-up of survivors, mo | 36 (3-156) | 47 (3-165) | 77 (12-159) | < .0001 |
SCID indicates severe combined immune deficiencies; TX, treatment; GVHD, graft-versus-host disease; TCD, T-cell depletion; ATG, antihymocyte globulin; and PBSC, peripheral blood stem cell.
The mismatched transplantations (single or double) were performed by 73 centers.