PMID- 38229269
OWN - NLM
STAT- MEDLINE
DCOM- 20240314
LR  - 20240314
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 30
IP  - 2
DP  - 2024 Mar
TI  - Safety and efficacy of damoctocog alfa pegol prophylaxis in patients with severe 
      haemophilia A: Results of an interventional, post-marketing study.
PG  - 388-394
LID - 10.1111/hae.14930 [doi]
AB  - INTRODUCTION: Damoctocog alfa pegol (BAY 94-9027, Jivi((R)) ) is an approved 
      extended half-life factor VIII (FVIII) for treatment of previously treated 
      patients with haemophilia A aged >/=12 years. We report the final results of an 
      interventional, post-marketing study of damoctocog alfa pegol prophylaxis in 
      patients with severe haemophilia A. METHODS: In this open-label, interventional, 
      post-marketing, phase 4 trial (NCT04085458), previously FVIII-treated patients 
      with severe haemophilia A aged >/=18 years received damoctocog alfa pegol for >/=100 
      exposure days (EDs). Patients initially received 45 IU/kg every 5 days 
      (recommended) or 40 IU/kg twice-weekly. At Visit 3, patients' doses could be 
      increased, or treatment frequency adapted. The primary endpoint was FVIII 
      inhibitor development (titre >/=.6 Bethesda units). Secondary endpoints included 
      anti-polyethylene glycol (PEG) antibody development, treatment-emergent adverse 
      events (AEs) and annualized bleeding rate (ABR). RESULTS: Overall, 36 patients 
      were enrolled; 32 patients received treatment, of whom, 27 completed the study. 
      No patients developed FVIII inhibitors; three tested transiently positive for 
      low-titre anti-PEG antibodies without clinical relevance. Three patients reported 
      study-drug-related AEs of mild or moderate intensity. Two patients discontinued 
      the study due to AEs. No deaths occurred. Most patients (70%) were treated with 
      E5D/E7D regimens. The median (Q1;Q3) total ABR (N = 30) was 3.0 (.0;9.0) 
      pre-study and 1.8 (.7;5.9) during the study. CONCLUSION: Damoctocog alfa pegol 
      individualized prophylaxis regimens were well-tolerated with no immunogenicity 
      concerns. ABRs improved following the switch from pre-study prophylaxis to 
      damoctocog alfa pegol prophylaxis. These results support the favourable safety 
      and efficacy profile of damoctocog alfa pegol prophylaxis.
CI  - (c) 2024 The Authors. Haemophilia published by John Wiley & Sons Ltd.
FAU - Holme, Pal Andre
AU  - Holme PA
AUID- ORCID: 0000-0002-3888-2905
AD  - Department of Haematology, Oslo University Hospital and Institute of Clinical 
      Medicine, University of Oslo, Oslo, Norway.
FAU - Poulsen, Lone Hvitfeldt
AU  - Poulsen LH
AD  - The Haemophilia Centre, Aarhus University Hospital, Aarhus, Denmark.
FAU - Tueckmantel, Claudia
AU  - Tueckmantel C
AD  - Bayer AG, Wuppertal, Germany.
FAU - Maas Enriquez, Monika
AU  - Maas Enriquez M
AD  - Bayer AG, Wuppertal, Germany.
FAU - Alvarez Roman, Maria Teresa
AU  - Alvarez Roman MT
AUID- ORCID: 0000-0003-3296-4288
AD  - Hospital Universitario La Paz, Madrid, Spain.
FAU - De Cristofaro, Raimondo
AU  - De Cristofaro R
AD  - Agostino Gemelli University Hospital Foundation IRCCS, Rome, Italy.
LA  - eng
PT  - Journal Article
DEP - 20240116
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 9001-27-8 (Factor VIII)
RN  - 0 (Hemostatics)
SB  - IM
MH  - Humans
MH  - Adolescent
MH  - Adult
MH  - Factor VIII/therapeutic use
MH  - *Hemophilia A/drug therapy
MH  - Treatment Outcome
MH  - Hemorrhage/prevention & control
MH  - *Hemostatics/therapeutic use
MH  - Marketing
OTO - NOTNLM
OT  - extended half-life
OT  - haemophilia A
OT  - prophylaxis
OT  - recombinant factor FVIII
EDAT- 2024/01/17 06:42
MHDA- 2024/03/14 06:47
CRDT- 2024/01/17 00:53
PHST- 2023/12/19 00:00 [revised]
PHST- 2023/06/27 00:00 [received]
PHST- 2023/12/19 00:00 [accepted]
PHST- 2024/03/14 06:47 [medline]
PHST- 2024/01/17 06:42 [pubmed]
PHST- 2024/01/17 00:53 [entrez]
AID - 10.1111/hae.14930 [doi]
PST - ppublish
SO  - Haemophilia. 2024 Mar;30(2):388-394. doi: 10.1111/hae.14930. Epub 2024 Jan 16.