Table 1.

Base-case estimates and ranges used in sensitivity analysis

Variables	Base-case estimate	Range	References	Notes
Clinical probabilities
Bleeding events
Prophylaxis	0.3/mo	2-4/y	23
Hospitalization	0.0175/mo	0.01-0.02/mo	25
Joint complications
Joint bleed	0.39/mo	0.3-0.5/mo	26
Hospitalization	0.10/event	0.05-0.15/mo		Range estimated
Orthopedic procedure	2/lifetime (0.0022/mo)	1-3/lifetime	27
Gene therapy
Success	0.90	0.8-1		Range estimated
Complications	0.01	0.0005-0.002		Range estimated
Death	0.00013/mo	0.0001-0.0005/mo	21	Mean risk for death, 30-40-y-olds (CDC)
Time courses
Bleeding event	2 d	2-4 d	7
Hospitalization	4 d	2-6 d	32	Median hemophilia admission (HCUP)
Joint damage	14 d	7-21 d
Gene therapy complication	4 d	—
Quality-of-life measures (utilities)
Prophylaxis	0.93	0.87-0.93	22, 28
Hospitalization	0.66	0.5-0.7		Based on bleed utility
Bleed	0.66	0.66-0.8	33
Joint damage	0.64	—	30
Gene therapy	1	0.9-1		Assumed utility
Gene therapy complication	0.8	0.7-0.9		Assumed utility
Costs, US$
Prophylaxis	37 759	—	7, 31	Based on 88.7-kg male, 33 IU/kg 3 times weekly
Bleeding event	8 870	4 435-17 740	7	Based on 50 IU/kg/d for bleed duration
Hospitalization for bleeding event	48 603	33 582-63 678	7, 32	Average Medicare reimbursement (HCUP) for hospitalization plus factor at 50 IU/kg/d
Joint damage	61	40-80	33
Orthopedic surgery (knee replacement)	137 461	110 000-138 000	32	Average Medicare reimbursement (HCUP) for knee replacement plus factor
Gene therapy	850 000	10 000-2 100 000	20
Gene therapy failure	800	500-1 000	20

The base-case estimates represent the best estimate for each value. Unless otherwise noted, ranges represent 95% confidence intervals.

CDC, Centers for Disease Control and Prevention.