Patient and parent preferences for characteristics of prophylactic treatment in hemophilia

Figures & data

Table 1 Attribute descriptions and levels by type of hemophilia

Attribute description	Attribute levels
	Hemophilia A	Hemophilia B
Frequency of administration on prophylaxis	Three times a week	Twice a week
	Twice a week	Once a week
	Once a week	Once every 10–14 days
Diluent volume	3 mL	5 mL
	2.5 mL	3 mL
	Either hemophilia A or B
Efficacy: number of breakthrough bleeds while on prophylaxis	One bleed/4 months
	One bleed/6 months
	One bleed/year
	None
Number of vials per infusion	Two vials per infusion
	One vial per infusion
Reconstitution device	Device that connects a prefilled syringe of diluent and a vial of drug
	Dual chamber syringe that contains the diluent and drug in a single device; no assembly required; easy mixing
Manufacturer	The manufacturer of this product is new to hemophilia, and you are unfamiliar with it You are familiar with the manufacturer of this product and the products it offers in hemophilia

Figure 1 Sample treatment option and response scale for rating task.

Table 2 Respondent characteristics by type of hemophilia

Patient characteristics	Hemophilia A		Hemophilia B
	(N=89)		(N=32)
	n (%)		n (%)
Country
Australia	9 (10%)		5 (16%)
Canada	15 (17%)		5 (16%)
US	65 (73%)		22 (69%)
Respondent type
Adult patient	54 (61%)		21 (66%)
Parent of pediatric patient	35 (39%)		11 (34%)
Mean age in years
Adults	33.3		40.4
Pediatric patients	10.5		11.2
Severity
Moderate	3 (3%)		2 (6%)
Severe	86 (97%)		30 (94%)
Treatment paradigm
Prophylaxis		70 (79%)	20 (63%)
On-demand		19 (21%)	12 (38%)
Mean number of bleeds in last year		9.9	12.4

Figure 2 Relative importance of treatment attributes by type of hemophilia.

Table 3 Part-worth utilities of treatment attributes

Attribute and level	Hemophilia A		Hemophilia B
	Utility	SE	Utility	SE
Frequency of administration on prophylaxis
Three times a week	−0.91	0.05	N/A	N/A
Twice a week	−0.00	0.03	−0.58	0.07
Once a week	0.91	0.16	−0.01	0.07
Once every 10–14 days	N/A	N/A	0.60	0.07
Efficacy: number of breakthrough bleeds while on prophylaxis
None	0.47	0.04	0.14	0.06
One per year	0.32	0.03	0.29	0.04
Two per year	−0.24	0.02	−0.10	0.04
Three per year	−0.53	0.05	−0.48	0.10
Number of vials required per infusion
One vial per infusion	0.14	0.02	0.05	0.06
Two vials per infusion	−0.14	0.02	−0.05	0.06
Diluent volume
2.5 mL	−0.03	0.02	N/A	N/A
3 mL	0.03	0.02	0.00	0.05
5 mL	N/A	N/A	0.00	0.05
Reconstitution device
Device that connects a prefilled syringe of diluent and a vial of drug	−0.08	0.02	0.01	0.05
Dual chamber syringe with diluent and drug in a single device	0.08	0.02	−0.01	0.05
Manufacturer
New manufacturer; unfamiliar with it	−0.34	0.03	−0.30	0.06
Familiar with manufacturer and its products	0.34	0.03	0.30	0.06

Abbreviation: SE, standard error of the mean.