Nonmalignant disease cohort: patient, disease, and transplantation characteristics for children age 0-18 y who underwent first allogeneic HCT for nonmalignant disease in the United States from 2006-2015 stratified by neighborhood poverty
| Characteristic . | n (%) . | P . | ||
|---|---|---|---|---|
| Total . | Low-poverty neighborhood* . | High-poverty neighborhood* . | ||
| No. of patients | 1696 (100) | 1468 (87) | 228 (13) | |
| No. of centers | 88 | 84 | 59 | |
| Follow-up of survivors, mo | 72 | 73 | 71 | |
| Median | ||||
| Range | 3-136 | 3-136 | 11-122 | |
| Age at transplantation, y | .93 | |||
| <4 | 828 (49) | 719 (49) | 109 (48) | |
| 4-9 | 475 (28) | 409 (28) | 66 (29) | |
| 10-18 | 393 (23) | 340 (23) | 53 (23) | |
| Male sex | 1049 (62) | 901 (61) | 148 (65) | .31 |
| Race | <.001 | |||
| White | 1118 (66) | 1003 (68) | 115 (50) | |
| Black | 332 (20) | 250 (17) | 82 (36) | |
| Other | 168 (10) | 150 (10) | 18 (8) | |
| Missing | 78 (4) | 65 (4) | 13 (6) | |
| Ethnicity | <.001 | |||
| Not Hispanic | 1324 (78) | 1179 (80) | 145 (64) | |
| Hispanic | 344 (20) | 264 (18) | 80 (35) | |
| Missing | 28 (<1) | 25 (2) | 3 (1) | |
| Insurance type | <.001 | |||
| Private | 1020 (60) | 936 (64) | 84 (37) | |
| Medicaid | 597 (35) | 465 (32) | 132 (58) | |
| Uninsured | 15 (< 1) | 14 (<1) | 1 (<1) | |
| Missing | 64 (4) | 53 (4) | 11 (5) | |
| Performance score before HCT | .89 | |||
| ≥80 | 1527 (90) | 1323 (90) | 204 (89) | |
| <80 | 107 (6) | 91 (6) | 16 (7) | |
| Missing | 62 (4) | 54 (4) | 8 (4) | |
| Disease | .005 | |||
| Severe aplastic anemia | 297 (18) | 257 (18) | 40 (18) | |
| Inherited abnormality of erythrocytes or platelets | 446 (26) | 383 (26) | 63 (28) | |
| SCID and other immune system disorders | 492 (29) | 422 (29) | 70 (31) | |
| Inherited disorders of metabolism or osteopetrosis | 270 (16) | 251 (17) | 19 (8) | |
| Histiocytic disorders | 191 (11) | 155 (11) | 36 (16) | |
| Donor graft type | .59 | |||
| Cord blood | 733 (43) | 634 (43) | 99 (43) | |
| HLA-identical sibling BM | 311 (18) | 264 (18) | 47 (21) | |
| HLA-identical sibling PB | 13 (<1) | 13 (<1) | 0 | |
| Other related BM | 72 (4) | 62 (4) | 10 (4) | |
| Other related PB | 51 (3) | 44 (3) | 7 (3) | |
| Well-matched unrelated BM | 310 (18) | 272 (19) | 38 (17) | |
| Well-matched unrelated PB | 51 (3) | 47 (3) | 4 (2) | |
| Partially matched unrelated BM | 101 (6) | 89 (6) | 12 (5) | |
| Partially matched unrelated PB | 29 (2) | 24 (2) | 5 (2) | |
| Mismatched unrelated BM/PB | 25 (1) | 19 (1) | 6 (3) | |
| Donor age (unrelated only), y | .51† | |||
| Median | 30 | 30 | 31 | |
| Range | 19-60 | 18-59 | 19-54 | |
| Sex match (donor/recipient) | .45 | |||
| Male/male | 341 (20) | 287 (20) | 54 (24) | |
| Male/female | 202 (12) | 182 (12) | 20 (9) | |
| Female/male | 258 (15) | 222 (15) | 36 (16) | |
| Female/female | 162 (10) | 143 (10) | 19 (8) | |
| Cord blood/male | 450 (27) | 392 (27) | 58 (25) | |
| Cord blood/female | 283 (17) | 242 (16) | 41 (18) | |
| Recipient CMV | .03 | |||
| Negative | 808 (48) | 711 (48) | 97 (43) | |
| Positive | 834 (49) | 716 (49) | 118 (52) | |
| Missing | 54 (3) | 41 (3) | 13 (6) | |
| Conditioning intensity (myeloablative) | .62 | |||
| TBI | 14 (<1) | 11 (<1) | 3 (1) | |
| Chemotherapy | 885 (52) | 770 (52) | 115 (50) | |
| Reduced-intensity conditioning/NST | 753 (44) | 651 (44) | 102 (45) | |
| No conditioning given | 44 (3) | 36 (2) | 8 (4) | |
| GVHD prophylaxis | .01 | |||
| Ex vivo T-cell depletion + CD34 selection | 128 (8) | 104 (7) | 24 (11) | |
| Postcyclophosphamide + other(s) | 14 (<1) | 13 (<1) | 1 (<1) | |
| Tacrolimus based | 511 (30) | 425 (29) | 86 (38) | |
| Cyclosporin based | 942 (56) | 833 (57) | 109 (48) | |
| Other | 40 (2) | 37 (3) | 3 (1) | |
| Missing | 61 (4) | 56 (4) | 5 (2) | |
| ATG/alemtuzumab | .71 | |||
| ATG + alemtuzumab | 7 (<1) | 7 (<1) | 0 | |
| ATG alone | 992 (58) | 861 (59) | 131 (57) | |
| Alemtuzumab alone | 487 (29) | 422 (29) | 65 (29) | |
| No ATG or alemtuzumab | 201 (12) | 171 (12) | 30 (13) | |
| Missing | 9 (<1) | 7 (<1) | 2 (<1) | |
| Year of transplantation | .79 | |||
| 2006-2008 | 660 (39) | 575 (39) | 85 (37) | |
| 2009-2011 | 516 (30) | 447 (31) | 69 (30) | |
| 2012-2015 | 520 (31) | 446 (30) | 74 (33) | |
| Characteristic . | n (%) . | P . | ||
|---|---|---|---|---|
| Total . | Low-poverty neighborhood* . | High-poverty neighborhood* . | ||
| No. of patients | 1696 (100) | 1468 (87) | 228 (13) | |
| No. of centers | 88 | 84 | 59 | |
| Follow-up of survivors, mo | 72 | 73 | 71 | |
| Median | ||||
| Range | 3-136 | 3-136 | 11-122 | |
| Age at transplantation, y | .93 | |||
| <4 | 828 (49) | 719 (49) | 109 (48) | |
| 4-9 | 475 (28) | 409 (28) | 66 (29) | |
| 10-18 | 393 (23) | 340 (23) | 53 (23) | |
| Male sex | 1049 (62) | 901 (61) | 148 (65) | .31 |
| Race | <.001 | |||
| White | 1118 (66) | 1003 (68) | 115 (50) | |
| Black | 332 (20) | 250 (17) | 82 (36) | |
| Other | 168 (10) | 150 (10) | 18 (8) | |
| Missing | 78 (4) | 65 (4) | 13 (6) | |
| Ethnicity | <.001 | |||
| Not Hispanic | 1324 (78) | 1179 (80) | 145 (64) | |
| Hispanic | 344 (20) | 264 (18) | 80 (35) | |
| Missing | 28 (<1) | 25 (2) | 3 (1) | |
| Insurance type | <.001 | |||
| Private | 1020 (60) | 936 (64) | 84 (37) | |
| Medicaid | 597 (35) | 465 (32) | 132 (58) | |
| Uninsured | 15 (< 1) | 14 (<1) | 1 (<1) | |
| Missing | 64 (4) | 53 (4) | 11 (5) | |
| Performance score before HCT | .89 | |||
| ≥80 | 1527 (90) | 1323 (90) | 204 (89) | |
| <80 | 107 (6) | 91 (6) | 16 (7) | |
| Missing | 62 (4) | 54 (4) | 8 (4) | |
| Disease | .005 | |||
| Severe aplastic anemia | 297 (18) | 257 (18) | 40 (18) | |
| Inherited abnormality of erythrocytes or platelets | 446 (26) | 383 (26) | 63 (28) | |
| SCID and other immune system disorders | 492 (29) | 422 (29) | 70 (31) | |
| Inherited disorders of metabolism or osteopetrosis | 270 (16) | 251 (17) | 19 (8) | |
| Histiocytic disorders | 191 (11) | 155 (11) | 36 (16) | |
| Donor graft type | .59 | |||
| Cord blood | 733 (43) | 634 (43) | 99 (43) | |
| HLA-identical sibling BM | 311 (18) | 264 (18) | 47 (21) | |
| HLA-identical sibling PB | 13 (<1) | 13 (<1) | 0 | |
| Other related BM | 72 (4) | 62 (4) | 10 (4) | |
| Other related PB | 51 (3) | 44 (3) | 7 (3) | |
| Well-matched unrelated BM | 310 (18) | 272 (19) | 38 (17) | |
| Well-matched unrelated PB | 51 (3) | 47 (3) | 4 (2) | |
| Partially matched unrelated BM | 101 (6) | 89 (6) | 12 (5) | |
| Partially matched unrelated PB | 29 (2) | 24 (2) | 5 (2) | |
| Mismatched unrelated BM/PB | 25 (1) | 19 (1) | 6 (3) | |
| Donor age (unrelated only), y | .51† | |||
| Median | 30 | 30 | 31 | |
| Range | 19-60 | 18-59 | 19-54 | |
| Sex match (donor/recipient) | .45 | |||
| Male/male | 341 (20) | 287 (20) | 54 (24) | |
| Male/female | 202 (12) | 182 (12) | 20 (9) | |
| Female/male | 258 (15) | 222 (15) | 36 (16) | |
| Female/female | 162 (10) | 143 (10) | 19 (8) | |
| Cord blood/male | 450 (27) | 392 (27) | 58 (25) | |
| Cord blood/female | 283 (17) | 242 (16) | 41 (18) | |
| Recipient CMV | .03 | |||
| Negative | 808 (48) | 711 (48) | 97 (43) | |
| Positive | 834 (49) | 716 (49) | 118 (52) | |
| Missing | 54 (3) | 41 (3) | 13 (6) | |
| Conditioning intensity (myeloablative) | .62 | |||
| TBI | 14 (<1) | 11 (<1) | 3 (1) | |
| Chemotherapy | 885 (52) | 770 (52) | 115 (50) | |
| Reduced-intensity conditioning/NST | 753 (44) | 651 (44) | 102 (45) | |
| No conditioning given | 44 (3) | 36 (2) | 8 (4) | |
| GVHD prophylaxis | .01 | |||
| Ex vivo T-cell depletion + CD34 selection | 128 (8) | 104 (7) | 24 (11) | |
| Postcyclophosphamide + other(s) | 14 (<1) | 13 (<1) | 1 (<1) | |
| Tacrolimus based | 511 (30) | 425 (29) | 86 (38) | |
| Cyclosporin based | 942 (56) | 833 (57) | 109 (48) | |
| Other | 40 (2) | 37 (3) | 3 (1) | |
| Missing | 61 (4) | 56 (4) | 5 (2) | |
| ATG/alemtuzumab | .71 | |||
| ATG + alemtuzumab | 7 (<1) | 7 (<1) | 0 | |
| ATG alone | 992 (58) | 861 (59) | 131 (57) | |
| Alemtuzumab alone | 487 (29) | 422 (29) | 65 (29) | |
| No ATG or alemtuzumab | 201 (12) | 171 (12) | 30 (13) | |
| Missing | 9 (<1) | 7 (<1) | 2 (<1) | |
| Year of transplantation | .79 | |||
| 2006-2008 | 660 (39) | 575 (39) | 85 (37) | |
| 2009-2011 | 516 (30) | 447 (31) | 69 (30) | |
| 2012-2015 | 520 (31) | 446 (30) | 74 (33) | |
ATG, antithymocyte globulin; CMV, cytomegalovirus; NST, nonmyeloablative stem cell transplantation; SCID, severe combined immunodeficiency; TBI, total-body irradiation.
Neighborhood poverty defined by linkage of a child’s residential ZIP code at the time of HCT to US Census3 and categorized as high-poverty neighborhood (≥20% of persons living below 100% FPL) or low-poverty neighborhood (<20% of persons below 100% FPL).
Kruskal-Wallis test used for hypothesis testing; all others used Pearson χ2 test.