The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators

Shifting Paradigms and Arising Concerns in Severe Hemophilia A Treatment

“…Hence, using an incorrect tool for a particular PK evaluation may lead to unreliable outcomes. [141][142][143] Applications for patient diaries like haemtrack, HaemoAssist 2, smart medication, florio HAEMO, and myPKFiT have been devised to enable patients to record bleeding events and administered doses. These applications can also notify patients when they reach critical factor levels to prevent missed FVIII infusions.…”

“…Depending on PK for bleeding control may be unreliable if the standard of home monitoring is unsatisfactory. 143 PK analysis could be used to recognize patients with extended FVIII half-lives, enabling them to lessen their dosage and/or dosing frequency. This will eventually reduce treatment costs and be more convenient for children, older people, and others with poor venous access.…”

“…Thus, randomized controlled trials with extended follow-up periods are required to compare standard dosing with PK-guided prophylaxis incorporating personalized target levels exceeding 1 IU/dL. 143 Moreover, the need for expertise in analyzing and understanding PK results poses a significant challenge for the implementation. According to International Society on Thrombosis and Haemostasis survey, only 9.7% of doctors conducted PK analysis when changing from SHL to EHL factor concentrates primarily because of restricted time, accessibility, and resources.…”

“…Despite the development of PK/PD models, they have yet to be validated or practiced with patients. 143,151…”