Real-world evidence on efmoroctocog alfa in patients with
haemophilia A: A systematic literature review of treatment
experience in Europe

J 2023

Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe

BLATNÝ, Jan, Emma Munk NIELSEN, Signe Baattrup REITZEL, Annabell Cajus MCMILLAN, Anne DANO et. al.

Basic information

Original name

Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe

Authors

BLATNÝ, Jan (203 Czech Republic, belonging to the institution), Emma Munk NIELSEN, Signe Baattrup REITZEL, Annabell Cajus MCMILLAN, Anne DANO, Linda BYSTRICKA, Nana KRAGH and Robert KLAMROTH

Edition

Haemophilia, HOBOKEN, Wiley, 2023, 1351-8216

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.900 in 2022

RIV identification code

RIV/00216224:14110/23:00133675

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1111/hae.14797

UT WoS

000995616600001

Keywords in English

efmoroctocog alfa; Elocta; haemophilia A; prophylaxis; real-world evidence; rFVIIIFc; systematic review

Abstract

V originále

IntroductionThe real-world effectiveness of the efmoroctocog alfa (recombinant FVIII Fc fusion protein, a rFVIIIFc) has been investigated in numerous studies, however, currently, there exists no comprehensive collection of the existing real-world evidence (RWE) on the performance of prophylactic use of rFVIIIFc. AimThe aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe. MethodsWe searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality-of-life measures. Results46 eligible publications (eight full-text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half-life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105 IU/kg/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors. ConclusionrFVIIIFc prophylaxis treatment results in a low ABR across studies in patients with haemophilia A in a European real-world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.

Citovat

BLATNÝ, Jan, Emma Munk NIELSEN, Signe Baattrup REITZEL, Annabell Cajus MCMILLAN, Anne DANO, Linda BYSTRICKA, Nana KRAGH and Robert KLAMROTH. Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe. Haemophilia. HOBOKEN: Wiley, 2023, vol. 29, No 4, p. 963-974. ISSN 1351-8216. Available from: https://dx.doi.org/10.1111/hae.14797.

@article{2379581,
   author = {Blatný, Jan and Nielsen, Emma Munk and Reitzel, Signe Baattrup and McMillan, Annabell Cajus and Dano, Anne and Bystricka, Linda and Kragh, Nana and Klamroth, Robert},
   article_location = {HOBOKEN},
   article_number = {4},
   doi = {http://dx.doi.org/10.1111/hae.14797},
   keywords = {efmoroctocog alfa; Elocta; haemophilia A; prophylaxis; real-world evidence; rFVIIIFc; systematic review},
   language = {eng},
   issn = {1351-8216},
   journal = {Haemophilia},
   title = {Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe},
   url = {https://onlinelibrary.wiley.com/doi/10.1111/hae.14797},
   volume = {29},
   year = {2023}
}

TY  - JOUR
ID  - 2379581
AU  - Blatný, Jan - Nielsen, Emma Munk - Reitzel, Signe Baattrup - McMillan, Annabell Cajus - Dano, Anne - Bystricka, Linda - Kragh, Nana - Klamroth, Robert
PY  - 2023
TI  - Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe
JF  - Haemophilia
VL  - 29
IS  - 4
SP  - 963-974
EP  - 963-974
PB  - Wiley
SN  - 13518216
KW  - efmoroctocog alfa
KW  - Elocta
KW  - haemophilia A
KW  - prophylaxis
KW  - real-world evidence
KW  - rFVIIIFc
KW  - systematic review
UR  - https://onlinelibrary.wiley.com/doi/10.1111/hae.14797
N2  - IntroductionThe real-world effectiveness of the efmoroctocog alfa (recombinant FVIII Fc fusion protein, a rFVIIIFc) has been investigated in numerous studies, however, currently, there exists no comprehensive collection of the existing real-world evidence (RWE) on the performance of prophylactic use of rFVIIIFc. AimThe aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe. MethodsWe searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality-of-life measures. Results46 eligible publications (eight full-text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half-life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105 IU/kg/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors. ConclusionrFVIIIFc prophylaxis treatment results in a low ABR across studies in patients with haemophilia A in a European real-world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.
ER  -

BLATNÝ, Jan, Emma Munk NIELSEN, Signe Baattrup REITZEL, Annabell Cajus MCMILLAN, Anne DANO, Linda BYSTRICKA, Nana KRAGH and Robert KLAMROTH. Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe. \textit{Haemophilia}. HOBOKEN: Wiley, 2023, vol.~29, No~4, p.~963-974. ISSN~1351-8216. Available from: https://dx.doi.org/10.1111/hae.14797.

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