Table 1.
Characteristics of individual and combined cohorts at study enrollment
| Characteristics Median (IQR) | SMA (n=10) | IF (n=16) | HSCT (n=30) | Combined cohort (n=56) | P-Value |
|---|---|---|---|---|---|
| Age | 9.3 (6, 17.7) | 7.2 (4.6, 9.8) | 13.6 (11.6, 18.4) | 11.61 (7.4, 16.3) | <0.0001 |
| Sex (n, % male) | 5 (50) | 11 (69) | 15 (50) | 31 (55) | NA |
| Weight (kg) | 34.0 (17.7, 40.7) | 19.8 (15.7, 25.6) | 59.6 (34.4, 66.9) | 35.6 (20.6, 60.8) | <0.0001 |
| WAZ | −1.09 (−2.8, 0.3) | −0.91 (−1.1, −0.34) | 0.25 (−0.72, 0.9) | −0.35 (−1.15, 0.5) | 0.04 |
| Height (cm) | 124.7 (112.2, 153.8) | 108.6 (99.3, 122.9) | 159.4 (138.3, 165.3) | 135.9 (114.9, 160.7) | <0.0001 |
| HAZ | −0.82 (−1.6, −0.34) | −1.49 (−2.4, −0.95) | −0.33 (−1.14, 0.49) | −0.82 (−1.7, −0.18) | 0.09 |
| BMI (kg/m2) | 16.2 (15.7, 21.9) | 16.55 (15.3, −0.95) | 21.5 (17.4, 24.1) | 17.76 (16, 23.4) | <0.0001 |
| BMIZ | 0.01 (−2, 1) | 0.37 (−0.44, 1.1) | 0.39 (−0.21, 1.1) | 0.31 (−0.33, 1.1) | 0.63 |
| MAC (cm) | 20 (15.5, 23)* | 16 (15, 19) | 27 (−0.21, 1.1) | 21.5 (16, 28) | <0.0001 |
*
n=9
WAZ- weight for age Z-score; HAZ- height for age z-score; BMIZ- BMI z-score; MAC- Mid-upper Arm Circumference;
SMA- Spinal Muscular Atrophy; IF- intestinal failure; Hematopoietic Stem Cell Transplant.