DETAILED OUTCOME DATA

APPENDIX 4DETAILED OUTCOME DATA

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Table 13

Secondary Efficacy Variables: Summary of Hierarchical Testing.

Table 14

Change in World Health Organization Functional Class.

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Publisher

Canadian Agency for Drugs and Technologies in Health, Ottawa (ON)

NLM Citation

Riociguat (Adempas): Management of Inoperable Chronic Thromboembolic Pulmonary Hypertension [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2015 Jul. APPENDIX 4, DETAILED OUTCOME DATA.

Table 13Secondary Efficacy Variables: Summary of Hierarchical Testing

Variable^a	Treatment Effect ANCOVA P Value	Shapiro- Wilk Test P Value^b	Stratified Wilcoxon Test P Value	Statistically Significant	Statistically Significant in Hierarchical Testing
6MWD (Primary)	< 0.0001	0.0001	< 0.0001	Yes	Yes
PVR	< 0.0001	0.0001	< 0.0001	Yes	Yes
NT-proBNP	0.0293	0.0001	< 0.0001	Yes	Yes
WHO functional class	–	–	0.0026	Yes	Yes
Time to clinical worsening	0.2180^c	–	0.1724^d	No	No
Borg CR10 Scale^e	–	–	0.0035	Yes	No
EQ-5D questionnaire	0.0002	0.0001	< 0.0001	Yes	No
LPH questionnaire	0.0165	0.0001	0.1220	No	No

: ANCOVA = analysis of covariance; 6MWD = six-minute walk distance; EQ-5D = EuroQol 5-dimensions questionnaire; LPH = Living With Pulmonary Hypertension questionnaire; NT-proBNP = N-terminal prohormone of brain natriuretic peptide; PVR = pulmonary vascular resistance; WHO = World Health Organization.

: Note: P values used to determine statistical significance are given in bold.

a: Variables are ordered in the sequence of testing.

b: Test of normality of the data.

c: Mantel–Haenszel estimate P value for incidence of clinical worsening.

d: Stratified log-rank test P value for time to clinical worsening.

e: Patients enrolled before CHEST-1 analysis plan Amendment 3 used the Modified Borg Dyspnea Scale.

: Source: CHEST-1 Clinical Study Report.⁷

Table 14Change in World Health Organization Functional Class

Visit	WHO Functional Class	Riociguat (n = 173)	Placebo (n = 88)
Baseline	n	▬▬▬▬	▬▬▬▬
	I	▬▬▬▬	▬▬▬▬
	II	▬▬▬▬	▬▬▬▬
	III	▬▬▬▬	▬▬▬▬
	IV	▬▬▬▬	▬▬▬▬
Last Visit	n	▬▬▬▬	▬▬▬▬
	I	▬▬▬▬	▬▬▬▬
	II	▬▬▬▬	▬▬▬▬
	III	▬▬▬▬	▬▬▬▬
	IV	▬▬▬▬	▬▬▬▬
	V^a	▬▬▬▬	▬▬▬▬
Change From Baseline at Last Visit	n	▬▬▬▬	▬▬▬▬
	−2	▬▬▬▬	▬▬▬▬
	−1	▬▬▬▬	▬▬▬▬
	0	▬▬▬▬	▬▬▬▬
	1	▬▬▬▬	▬▬▬▬
	2	▬▬▬▬	▬▬▬▬
	3	▬▬▬▬	▬▬▬▬
P value (stratified Wilcoxon test)	▬▬▬▬

: WHO = World Health Organization.

a: V represented an imputed worst value in case of death.