Table 2.
Changes from baseline to last visit in predefined outcomes
| Baseline mean (SD) | Last visit mean (SD) | Mean change (95% CI) | P value | |
|---|---|---|---|---|
| Primary outcome | ||||
| T3, nmol/L (n = 67) | 4.58 (1.11) | 1.66 (0.69) | −2.92 (−3.23 to −2.61) | <0.0001 |
| Secondary outcomes | ||||
| Anthropometric parameters and heart rate | ||||
| Body weight, kg (n = 58) | 17.8 (12.1) | 23.6 (14.5) | 5.7 (4.2 to 7.2) | |
| Weight-for-age Z-score (n = 58) | −2.81 (1.94) | −2.64 (1.81) | 0.17 (−0.18 to 0.53) | 0.3263 |
| Δ Weight-for-age—predicted weight-for-age Z-score (n = 55) | 0.07 (1.83) | 0.79 (1.92) | 0.72 (0.36 to 1.09) | 0.0002 |
| Height, cm (n = 44) | 101 (21) | 116 (23) | 15 (12 to 19) | |
| Height-for-age Z-score (n = 44) | −1.84 (1.77) | −1.92 (1.51) | −0.09 (−0.50 to 0.32) | 0.6705 |
| Δ Height-for-age—predicted height-for-age Z-score (n = 43) | −0.44 (1.38) | 0.14 (1.41) | 0.58 (0.12 to 1.05) | 0.0139 |
| Weight-for-height Z-score (n = 44) | −2.02 (2.49) | −1.50 (2.44) | 0.52 (−0.35 to 1.39) | 0.2358 |
| Heart rate, bpm (n = 48) | 113 (21) | 97 (20) | −17 (−24 to −10) | <0.0001 |
| Heart rate-for-age Z-score (n = 48) | 1.59 (0.89) | 0.96 (1.01) | −0.64 (− 0.98 to −0.29) | 0.0005 |
| Thyroid function tests | ||||
| TSH, mU/L (n = 62)* | 3.32 (2.30) | 0.95 (0.73) | −2.38 (−2.98 to −1.77) | <0.0001 |
| Free T4, pmol/L (n = 64) | 9.5 (2.3) | 3.4 (1.6) | −6.1 (−6.7 to −5.4) | <0.0001 |
| T4, nmol/L (n = 63) | 54.2 (11.8) | 18.1 (9.8) | −36.1 (−39.5 to −32.7) | <0.0001 |
| Peripheral markers | ||||
| Sex hormone-binding globulin, nmol/L (n = 48) | 245 (99) | 209 (92) | −36 (−57 to −16) | 0.0008 |
| Creatinine, µmol/L (n = 47) | 32 (11) | 39 (13) | 7 (6 to 9) | <0.0001 |
| Creatine kinase, U/L (n = 47)* | 110 (87) | 128 (80) | 18 (−8 to 45) | 0.2166 |
All outcomes were assessed in all patients who received Triac treatment longer than the mean time to optimal dose (5.0 months; N = 64). Data are mean. Body weight-for-age Z-scores were calculated using TNO growth calculator and heart rate-for-age Z-scores were calculated using the Boston Z-score calculator. Abbreviations: bpm, beats per minute; T3, triiodothyronine; T4, thyroxine; TSH, thyroid-stimulating hormone.
*TSH and creatine kinase concentrations were log-transformed to ensure a normal distribution before paired t tests were done (nontransformed means [SD] and mean changes [95% CI] are presented for the sake of interpretability).