Modelling the long-term benefits of tolvaptan therapy on renal function decline in autosomal dominant polycystic kidney disease: an exploratory analysis using the ADPKD outcomes model

Table 2 Model inputs modified in sensitivity and scenario analyses

Modified variables			Modified inputs
Sensitivity analysis
Discontinuation of treatment^a,b			15.30, 6.51, 2.89 and 0.50% in years 1, 2, 3 and 4+, respectively
Replication of ADPKD progression observed in TEMPO 3:4^a			Percentage change in TKV from baseline with placebo: 5.05, 11.49 and 18.85% in years 1, 2 and 3, respectively Annual eGFR slope (mL/min/1. 73m²): − 3.812 with placebo − 2.609 with tolvaptan
Treatment effect based on CKD-Epi equation^a			26.4% reduction in eGFR decline
Scenario analysis
Treatment effect by CKD stage at tolvaptan initiation^a		CKD 1	14.7% reduction in eGFR decline
		CKD 2	31.0% reduction in eGFR decline
		CKD 3	40.5% reduction in eGFR decline
Treatment effect based on CKD-Epi equation	CKD stage at tolvaptan initiation^c	CKD 1	15.5% reduction in eGFR decline
		CKD 2	29.1% reduction in eGFR decline
		CKD 3	31.0% reduction in eGFR decline
	Mayo imaging classification at tolvaptan initiation^d	1C-1E	28.2% reduction in eGFR decline

CKD Chronic kidney disease, CKD-Epi Chronic Kidney Disease Epidemiology Collaboration, eGFR estimated glomerular filtration rate, TKV total kidney volume
^aObserved in TEMPO 3:4 [8, 34]
^bRate extrapolated after year 3
^cTreatment effects reported for CKD 1 to CKD 3 subgroups in TEMPO 3:4 [10]; eGFR was estimated using the CKD-Epi equation [14]
^dTreatment effect (eGFR slope − 2.82 mL/min/1. 73 m2 for tolvaptan versus − 3.93 mL/min/1. 73 m2 in placebo) reported for Mayo subclass 1C-E patients in TEMPO 3:4 [35]; eGFR was estimated using the CKD-Epi equation [14]

ISSN: 1471-2369