. Author manuscript; available in PMC: 2021 Dec 12.

Published in final edited form as: Nat Med. 2021 Sep 23;27(10):1818–1824. doi: 10.1038/s41591-021-01505-4

Table 3 |.

Primary and secondary efficacy outcomes, intention-to-treat

	Adjusted* mean change in raw measure		Between-group differences	Adjusted* mean change in normalized measure		Between-group differences	Covariate-adjusted* P value
	Placebo (n = 90)	Valsartan (n = 88)		Placebo (n = 90)	Valsartan (n = 88)
Primary outcome	NA	NA		−0.095	+0.136	+0.231	0.001
Composite z-score	NA	NA		[−0.192, +0.002]	[+0.049, +0.223]	[+0.098, +0.364]	0.001
Components of the primary outcome
Decrease in value indicates improvement
Maximum LV wall	+1.57	+0.40	−1.17	+1.32	+0.16	−1.16	0.06
Thickness^a, mm	[+0.80, +2.35]	[−0.32, +1.12]	[−2.21, −0.14]	[+0.42, +2.21]	[−0.69, +1.00]	[−2.37, +0.06]	0.06
LV mass index^b, g m⁻²	NA	NA	NA	−1.11	−4.32	−3.20	0.12
LV mass index^b, g m⁻²	NA	NA	NA	[−4.04, +1.81]	[−7.32, −1.32]	[−0.81, +7.22]	0.12
LA volume index^b, ml m⁻²	NA	NA	NA	+1.45	+2.47	+1.02	0.59
LA volume index^b, ml m⁻²	NA	NA	NA	[−1.27, +4.17]	[−0.31, +5.25]	[−2.70, +4.74]	0.59
NTproBNP^c, pg ml⁻¹	+84.2	+23.1	−61.0	+0.27	+0.02	−0.25	0.03
NTproBNP^c, pg ml⁻¹	[+23.1, 1+45.2]	[−36.6, +82.9]	[−122.9, +0.9]	[+0.11, +0.43]	[−0.14, +0.18]	[−0.47, −0.03]	0.03
Troponin T^c, ng l⁻¹	+1.18	+1.44	−0.27	+0.11	+0.06	−0.05	0.37
Troponin T^c, ng l⁻¹	[−0.70, +3.05]	[−0.42, +3.30]	[−2.37, +1.84]	[+0.02, +0.20]	[−0.02, +0.14]	[−0.16, +0.06]	0.37
Increase in value indicates improvement
LV end diastolic volume	NA	NA		+0.41	+4.51	+4.10	0.047
Index^d, ml m⁻²	NA	NA		[−2.77, +3.59]	[+1.41, +7.61]	[+0.06, +8.14]	0.047
LV end systolic volume	NA	NA		+2.20	+3.69	+1.49	0.24
Index^b, ml m⁻²	NA	NA		[+0.28, +4.13]	[+1.77, +5.62]	[−1.00, +3.97]	0.24
E′ velocity^d, cm s⁻¹	−0.83	+0.09	+0.92	−0.33	+0.016	+0.35	0.02
E′ velocity^d, cm s⁻¹	[−1.37, −0.29]	[−0.39, +0.57]	[0.24, +1.61]	[−0.57, −0.10]	[−0.19, +0.22]	[+0.06, +0.63]	0.02
S′ velocity^d, cm s⁻¹	−0.15	+0.02	0.17	−0.165	−0.031	+0.13	0.45
S′ velocity^d, cm s⁻¹	[−0.48, +0.17]	[−0.29, +0.33]	[−0.26, +0.61]	[−0.43, +0.10]	[−0.28, +0.21]	[−0.21, +0.48]	0.45
Improvement on any component^e	NA	NA		91%	95%	Unadjusted odds ratio	0.37
						2.05
						[0.59, 7.07]

Results of mixed model linear regressions comparing changes in the outcome between treatment groups, adjusting through random effects for clustering within sites and within families. Data are presented as marginal mean change at year 2 compared to baseline, with pre-specified adjustment for *: baseline value of the outcome (normalized), NYHA class, maximum LV wall thickness, sex, age, pubertal status, body mass index, race (white versus non-white), Hispanic ethnicity, genotype (thick versus thin filament variants), LVEF, systolic blood pressure, beta blocker use and calcium channel blocker use. Between-group difference: valsartan–placebo. P values for secondary outcomes were not adjusted for multiple testing.

Normalized measure is adjusted for BSA, but no adjustment is made in the raw measure.

Normalized measure is indexed to BSA.

Normalized measure was log-transformed, but no adjustment is made in the raw measure.

Normalized measure is adjusted for age, but no adjustment is made in the raw measure.

Unadjusted and based on unimputed data; too few people had no improvements to model. Not applicable (NA) is indicated because only BSA-indexed values of LV mass, LA volume, LV end diastolic volume and LV end systolic volume can be validly combined into the composite z-score, owing to the strong influence of body size on these measures.