Table 3.
KM and Cox Regression Models for LoA: Median Age at LoA, Log-Rank p-Values, Cox Hazard Ratios, and p-Values by SPP1-LTBP4 Genotypes and GC Treatment
| Cohort | All Participants |
GC Treated |
GC Untreated |
||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| No. | Median Age at LoA, yr | p, KM Log-Rank | HR (95% CI), Cox p | No. | Median Age at LoA, yr | p, KM Log-Rank | HR (95% CI), Cox p | No. | Median Age at LoA, yr | p, KM Log-Rank | |
| Whole genotyped DNHS cohort, n = 283a | |||||||||||
| SPP1 rs28357094 | 0.048b | 1.22 (0.89–1.68), 0.22 | 0.032b | 1.61 (1.09-2.37), 0.016b | 0.6 | ||||||
| TT | 195 | 13.0 | 150 | 13.9 | 45 | 10.0 | |||||
| TG/GG | 84 | 11.8 | 63 | 12.0 | 21 | 10.0 | |||||
| LTBP4 rs10880 | 0.20 | 0.78 (0.49–1.24), 0.29 | 0.27 | 0.74 (0.44-1.26), 0.27 | — | ||||||
| CC/CT | 242 | 12.0 | 178 | 13.3 | 64 | 10 | |||||
| TT | 32 | 13.9 | 27 | 13.9 | 5 | 9.1 | |||||
| Caucasian cohort, controlled for population stratification by MDS, n = 118a | |||||||||||
| SPP1 rs28357094 | 0.047b | 1.54 (0.93–2.54), 0.09 | 0.07 | 1.85 (1.01–3.38), 0.047b | 0.7 | ||||||
| TT | 81 | 13.9 | 67 | 13.9 | 14 | 10.0 | |||||
| TG/GG | 35 | 12.0 | 26 | 12.0 | 9 | 9.0 | |||||
| LTBP4 rs10880 | 0.024b | 0.49 (0.23–1.07), 0.07 | 0.046b | 0.47 (0.20–1.09), 0.08 | — | ||||||
| CC/CT | 103 | 12.6 | 80 | 13.8 | 23 | 10 | |||||
| TT | 12 | 15.0 | 11 | 16.0 | 1 | N/A | |||||
Total number may not correspond exactly to genotype group number because of a few ungenotyped patients (limited DNA availability).
Statistically significant effect of genotype on LoA.
CI = confidence interval; DNHS = Duchenne Natural History Study; GC = glucocorticoid corticosteroid; GC treated = GC treatment at least 1 year while patient was ambulatory; GC untreated = no or <1 year of GC treatment while patient was ambulatory; HR = hazard ratio for genotype in Cox regression model with GC treatment as a time-varying covariate; KM = Kaplan–Meier survival analysis with log-rank comparison of median age at LoA; LoA = loss of ambulation; MDS = multidimensional scaling; N/A = not available.