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 1 Patient characteristics of the TEMPO 3:4, TEMPO 4:4 and REPRISE trial populations for use in model validation exercises and illustrative analyses

Trial population	Cohort size (n)	Baseline characteristics, mean (SE)^a				Source
Trial population	Cohort size (n)	Age (years)	Female (%)	eGFR (mL/min/1. 73m²)	TKV (mL)	Source
Model application
TEMPO 3:4 overall cohort	1445	38.7	48.4	81.61	1692.30	[34]
TEMPO 3:4 subgroups:
- CKD 1	502	34.3	52.7	105.69	1353.76	[10]
- CKD 2	689	40.3	49.5	75.00	1712.00	[10]
- CKD 3	248	42.0	36.7	51.34	2323.18	[10]
- CKD 1–3; Mayo subclasses 1C-1E	1285	38.0	46.1	81.29	1760.40	[35]; Otsuka data on file
Model validation
TEMPO 4:4 early-treated cohort^b	557	38.95 (0.29)^c	45.60 (2.11)	81.35 (0.68)^d	1706.00 (37.35)	[11, 36]
REPRISE overall cohort	1370	47.50 (0.22)	50.40 (0.01)	41.00 (0.30)	2026.30 (37.88)	[12, 15]

CKD chronic kidney disease, eGFR estimated glomerular filtration rate, SE standard error, TKV total kidney volume
^aSE derived from standard deviations and patient numbers reported in the original trial publications
^bReported values reflect the characteristics of the TEMPO 4:4 early-treated cohort at TEMPO 3:4 baseline
^cAge of patients at TEMPO 3:4 baseline was assumed to be 3.25 years less than the reported baseline age in TEMPO 4:4, based on the duration of TEMPO 3:4 (3 years) and the off-treatment period between TEMPO 3:4 and TEMPO 4.4 (approximately 3 months)
^dBaseline eGFR was not reported in TEMPO 4:4; thus, it was assumed that baseline eGFR among the matched population was equal to the full TEMPO 3:4 population [8]

ISSN: 1471-2369