Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: post hoc analysis from the PROTECT VIII study

Therapeutic Advances in Hematology · 10 authors, 10 centres

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TL;DR

The treatment was effective in reducing bleeding and was well-tolerated with high adherence over a median of 3.9 years.

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The study evaluated the efficacy and safety of prophylactic treatment with damoctocog alfa pegol over a median follow-up of 3.9 years. The main efficacy outcome, annualised bleeding rate, was low, with median total ABRs of 2.1 during the main study and 2.2 during the extension.

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other

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hematology

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ORIGINAL PAPER

PMC10126693

Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: post hoc analysis from the PROTECT VIII study

Therapeutic Advances in Hematology

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ABSTRACT

Abstract Background: Advances in treatment have enabled patients with haemophilia A to live longer and therefore may be subjected to comorbidities associated with ageing, in addition to disease-associated morbidities. There have been few reports to date on efficacy and safety of treatment specifically in patients with severe haemophilia A and comorbidities. Objective: To explore the efficacy and safety of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A aged ⩾40 years with comorbidities of interest. Design: A post hoc analysis of data from the phase 2/3 PROTECT VIII study and its extension. Methods: Bleeding and safety outcomes were analysed in a subgroup of patients aged ⩾40 years with ⩾1 comorbidity receiving damoctocog alfa pegol (BAY 94-9027; Jivi ® ) prophylaxis. Results: Thirty-four patients with severe haemophilia A were included in this analysis, with a mean age of 49.4 years at time of enrolment. The most prevalent comorbidities were hepatitis C ( n = 33; chronic, n = 23), hepatitis B ( n = 8) and hypertension ( n = 11). Four patients had human immunodeficiency virus. All received damoctocog alfa pegol prophylaxis for the entire study [median (range) time in study = 3.9 (1.0–6.9) years]. During the main study and extension, median total annualised bleeding rates (ABRs) (Q1; Q3) were 2.1 (0.0; 5.8) and 2.2 (0.6; 6.0), respectively; median joint ABRs were 1.9 (0.0; 4.4) and 1.6 (0.0; 4.0), respectively. Mean adherence with prophylaxis schedule was greater than 95% throughout the study. No deaths or thrombotic events were reported. Conclusion: Efficacy, safety and adherence of damoctocog alfa pegol were confirmed in patients aged ⩾40 years with haemophilia A and one or more comorbidities, with data for up to 7 years supporting its use as a long-term treatment option in this group. Plain language summary Advances in treatment mean that people with haemophilia A are now living longer and, as a result, may have additional medical conditions that occur with ageing. We aimed to investigate the efficacy and safety of the long-acting replacement factor VIII damoctocog alfa pegol in people with severe haemophilia A who had additional medical conditions. To do this, we investigated the recorded information about patients aged 40 years of age or older who had been treated with damoctocog alfa pegol in a previously completed clinical trial. We found that the treatment was well-tolerated; no deaths or thrombotic events (undesirable clotting events) were reported. Treatment was efficacious in reducing bleeding in this group of patients. The findings support the use of damoctocog alfa pegol as a long-term treatment for older patients with haemophilia A and coexisting conditions. Keywords: comorbidities, extended half-life, haemophilia A, PEGylated, prophylaxis

CITATION EXPORT

Mark T Reding, Ingrid Pabinger, Pål Andrè Holme et al.. Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: post hoc analysis from the PROTECT VIII study. Therapeutic Advances in Hematology. (2023). https://doi.org/10.1177/20406207231166779

AUTHORS

10 · 10 CENTRES

Mark T Reding, Ingrid Pabinger, Pål Andrè Holme, Monika Maas Enriquez, Maria Elisa Mancuso, Shadan Lalezari, Wolfgang Miesbach, Giovanni Di Minno, Robert Klamroth, Cedric Hermans

1Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center, 420 Delaware Street SE, MMC 480, Minneapolis, MN 55455, USA
2University Clinic for Internal Medicine I, Medical University of Vienna, Vienna, Austria
3Department of Haematology, Oslo University Hospital and Institute of Clinical Medicine, University of Oslo, Oslo, Norway
4Bayer AG, Wuppertal, Germany
5Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy; Humanitas University, Milan, Italy
6National Haemophilia Centre, Chaim Sheba Medical Centre, Tel Aviv University, Tel Aviv, Israel
7University Hospital Frankfurt, Frankfurt am Main, Germany
8Department of Clinical and Experimental Medicine, University of Naples Federico II, Naples, Italy
9Vivantes Hospital Friedrichshain, Berlin, Germany
10Division of Haematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain (UCLouvain), Louvain, Belgium