Target joint resolution in patients with haemophilia A receiving long‐term prophylaxis with BAY 94‐9027

BAY 94‐9027 (damoctocog alfa pegol; Jivi®; Bayer AG, Germany) is an extended half‐life B‐domain–deleted, site‐specifically PEGylated recombinant FVIII. Due to its prolonged half‐life, BAY 94‐9027 has the potential to maintain FVIII at a higher haemostatic level for longer periods versus standard‐acting agents.This longer half‐life also allows less frequent dosing, which in turn could help to improve adherence when used for prophylaxis.

We performed a post hoc analysis on the PROTECT VIII study data to explore the impact of long‐term BAY 94‐9027 prophylaxis on target joints.

The numbers of historic target joints (as judged by the investigators) were recorded at study entry. In addition, the number of new target joints that developed on‐study was evaluated, using the International Society on Thrombosis and Haemostasis (ISTH) definition of a target joint (three or more spontaneous bleeds within 6 months).We also assessed the number of target joints that had resolved by data cut‐off (28 August 2019), using the ISTH definition of joint resolution (a recorded target joint with two or fewer spontaneous bleeds during the last 12 months).These variables were analysed in patients known to have received prophylaxis before study entry and who continued on prophylaxis into the main study and its ongoing extension.

Of a total of 122 historic or new target joints, 111 (91%) had resolved at the data cut‐off. Resolution occurred in all prophylactic regimens. When we examined data for the most common types of target joints at baseline, we found that resolution occurred in 94% of ankle, 93% of knee and 89% of elbow joints. In addition, all shoulder target joints resolved, and 67% and 50% of wrist and hip target joints, respectively, resolved by the data cut‐off point (Figure).

In summary, our post hoc analysis of the PROTECT VIII study found a high rate of resolution of target joints with long‐term prophylaxis with BAY 94‐9027 among patients with severe haemophilia A. At least one target joint resolved in over 90% of patients, and over 80% of patients achieved resolution of all of their target joints. This effect was seen across all prophylactic regimens, including treatment of up to every 7 days.


Site: https://www.ncbi.nlm.nih.gov/pubmed / Data: Luglio 2020

Title: Target joint resolution in patients with haemophilia A receiving long‐term prophylaxis with BAY 94‐9027

Authors: Mark T. Reding, Ingrid Pabinger, Shadan Lalezari, Elena Santagostino,Maria Elisa Mancuso

Magazine: Haemophilia 2020;26:e201-e204