Treatment adherence in hemophilia

Figures & data

Table 1 Adherence rates with continuous prophylaxis for patients with hemophilia over time

Data source	Type of study	Time frame	Rate of prophylaxis by disease severity (%)
			Severe	Moderate	Mild	Unknown
CDC UDC program^Citation25	Observational prospective	1998–2004	A: 35.3	A: 13.4	A: 0.6	NA
			B: 27.1	B: 6	B: 0.7
			A + B: 34	A + B: 11	A + B: 0.7
CDC UDC program^Citation26	Observational prospective	2005–2009	A + B: 48	A + B: 16.2	A + B: 2.1	A + B: 27.8
Blanchette et al^Citation27	Survey of North American HTC clinicians*	2002	A: 47	A: 13	A: 0	NA
			B: 30	B: 3	B: 0
			A + B: 44.2	A + B: 12.9	A = B
Zappa et al^Citation28	Survey of North American HTC clinicians	2010	A: 58	NA	NA	NA
			B: 51
			A + B: 56.8
ATHN data set^Citation29	Registry	2010–2015	A: 67.6	A: 30.1	A: 4.4	A: 7.5
			B: 60.7	B: 14.1	B: 4.5	B: 6.2

Note:

* Patients aged ≤18 years only.

Abbreviations: CDC, Centers for Disease Control and Prevention; UDC, Universal Data Collection; NA, not applicable; HTC, hemophilia-treatment center; ATHN, American Thrombosis and Hemostasis Network; A, FVIII deficiency; B, FIX deficiency.

Table 2 Patient-reported rates of adherence with prophylaxis

Reference	Data source	Age	Definition of adherence	Reported adherence
^Citation31	Survey	1–18 years	>75% of prescribed infusions	58.8% adherent
^Citation32	Survey	Children and adults	>80% of prescribed infusions	73% adherent
^Citation41	VERITAS-Pro	Adolescents	Score of ≥57	30% adherent
^Citation42	Pharmacy logs	Children and adults	Days supply/days observed ≥60%	Adherence achieved 50% of the time
^Citation36	Infusion logs	Children and adults	>67% weeks of prophylaxis	Children: 26 Adults: 39
^Citation37	Infusion logs	Adults, 18–56 years	Study scoring system to rate adherence with frequency and dosage	Median adherence to frequency: 76% Median adherence to dosage: 93%
^Citation4	Randomized controlled trial, infusion logs	6 months–6 years	Not defined	Mean adherence 96%; 2 of 32 had difficulty with infusion schedule

Table 3 Barriers to prophylaxis adherence and potential strategies for addressing these barriers

Barriers to adherence^Citation28,Citation31,Citation42,Citation45^–^Citation49

Potential strategies to improve adherence^Citation50^–^Citation56

• Patient-related factors  ◦ Health beliefs  ◦ Age • Condition-related factors  ◦ Frequency of bleeding • Treatment-related factors  ◦ Venous access  ◦ Dosing regimen  ◦ Cost  ◦ Perceived costs • Health-care system factors  ◦ Access to hemophilia-treatment center  ◦ Insurance • Socioeconomic factors  ◦ Language  ◦ Acculturation  ◦ Health literacy  ◦ Balancing the child’s care with other family and social needs

• Education (multimedia, peer-to-peer mentors, tailored to patient’s primary language, development, and health literacy)  ◦ Benefits of prophylaxis  ◦ Venous access  ◦ Health-care navigation  ◦ Patient portals in ehealth records • Monitoring  ◦ Logs  ◦ Mobile direct observation • Reminders  ◦ Text messaging  ◦ Phone app • Incentives (age-appropriate)  ◦ Reward charts  ◦ Financial

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