Table 1.
Participant characteristics by mutation type
| Covariate | Overall N = 47 | Severe mutation N = 31 | Other mutation N = 16 |
|---|---|---|---|
| n (%) | n (%) | n (%) | |
| United Kingdom | 34 (72.3) | 21 (67.7) | 13 (81.2) |
| UMN | 13 (27.7) | 10 (32.3) | 3 (18.8) |
| Female | 18 (38.3) | 12 (38.7) | 6 (37.5) |
| Male | 29 (61.7) | 19 (61.3) | 10 (62.5) |
| Transplant type | |||
| HCT-related marrow | 26 (55.3) | 20 (64.5) | 6 (37.5) |
| HCT-unrelated marrow | 14 (29.8) | 8 (25.8) | 6 (37.5) |
| Cord blood | 7 (14.9) | 3 (9.7) | 4 (25.0) |
| Total body irradiation | 11 (23.4) | 7 (22.6) | 4 (25.0) |
| No GVHD | 15 (31.9) | 9 (29.0) | 6 (37.5) |
| Known GVHD | 18 (38.3) | 13 (41.9) | 5 (31.2) |
| Missing GVHD | 14 (29.8) | 9 (29.0) | 5 (31.2) |
| Engraftment | |||
| <=90% | 9 (19.1) | 8 (25.8) | 1 (6.2) |
| >90% | 33 (70.2) | 20 (64.5) | 13 (81.2) |
| Missing | 5 (10.6%) | 3 (9.7%) | 2 (12.5%) |
| Single transplant | 32 (68.1) | 20 (64.5) | 12 (75.0) |
| Two transplants | 15 (31.9) | 11 (35.5) | 4 (25.0) |
| Vineland version I | 35 (74.5) | 22 (71.0) | 13 (81.2) |
| Vineland version II | 12 (25.5) | 9 (29.0) | 3 (18.8) |
| SES (Hollingshead) | |||
| SES < 4 (middle to upper) | 23 (48.9) | 17 (54.8) | 6 (37.5) |
| SES >=4 (low-mid to low) | 24 (51.1%) | 14 (45.2) | 10 (62.5) |
| Mean (SD) | Mean (SD) | Mean (SD) | |
| Age at time of evaluation (year) | 10.5 (6.8) | 11.4 (7.1) | 8.9 (6.1) |
| Age at transplant (month) | 18.5 (8.2) | 18.8 (8.2) | 17.9 (8.2) |
| Time since transplant (year) | 9.0 (6.7) | 9.8 (7.0) | 7.4 (5.9) |
| IQ | 67.1 (22.3) | 61.1 (19.8) | 78.9 (22.9) |
| Vineland adaptive behavior | |||
| Communication | 70.6 (23.9) | 64.4 (23.2) | 82.7 (21.0) |
| Daily living skills | 63.1 (24.4) | 61.3 (25.6) | 66.6 (22.2) |
| Socialization | 75.9 (20.7) | 72.9 (22.8) | 81.6 (14.8) |
| Adaptive composite | 64.8 (21.6) | 61.4 (21.8) | 71.3 (20.4) |
| CHQ: PHS | 24.1 (15.3) | 25.1 (15.5) | 22.3 (15.1) |
| CHQ: PSS | 45.0 (11.2) | 45.4 (11.6) | 44.1 (10.7) |
Note: HCT hematopoietic cell transplant, GVHD graft versus host disease, SES socioeconomic status (Hollingshead 2-factor), IQ intelligence quotient (from age-appropriate cognitive test), CHQ child health questionnaire, PHS physical summary scale, PSS psychosocial summary scale