Evidence map›Paper›PMID 38371314›Full record

ArticleTherapeutic advances in hematology2024

The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study.

Margareth C Ozelo, Cedric Hermans, Manuel Carcao, Benoît Guillet, Joan Gu, Randy Guerra, Leilei Tang, Kate Khair

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Therapeutic advances in hematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02078427 (ADVATE/ ADYNOVI Hemophilia A Outcome Database), which is not on this map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
2.7field-weighted citation impact, top 10% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02078427 completednot on this map

ADVATE/ ADYNOVI Hemophilia A Outcome Database

TypeobservationalSponsorBaxalta now part of ShireRan2011 to 2024Enrolled951ConditionsHemophilia AArmsADVATE, ADYNOVI
3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. Article
  2. Contemporary approaches to treat people with hemophilia: what's new and what's not?Research and practice in thrombosis and haemostasis · 2025
    Review
  3. The imperative to prevent joint bleeding in everyone living with hemophilia.Research and practice in thrombosis and haemostasis · 2024
    Article
  4. Observational
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 6 institutions in 6 countries.

Margareth C OzeloHemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil.ORCID https://orcid.org/0000-0001-5938-0675
Cedric HermansSt-Luc University Hospital, Université catholique de Louvain, Louvain, Belgium.ORCID https://orcid.org/0000-0001-5429-8437
Manuel CarcaoDivision of Haematology/Oncology, Hospital for Sick Children, Toronto, ON, Canada.ORCID https://orcid.org/0000-0001-5350-1763
Benoît GuilletHaemophilia Treatment Center, Univ Rennes, CHU Rennes, Inserm, EHESP, Irset (Institut de recherche en santé, environnement et travail) - UMR_S 1085, F-35000 Rennes, France.ORCID https://orcid.org/0000-0003-2938-8013
Joan GuTakeda Development Center Americas, Inc., Cambridge, MA, USA.
Randy GuerraTakeda Development Center Americas, Inc., 500 Kendal Street, Cambridge, MA 02142, USA.ORCID https://orcid.org/0000-0002-7683-4429
Leilei TangTakeda Pharmaceuticals International AG, Zurich, Switzerland.
Kate KhairHaemnet, London, UK.
Takeda (United States) · USHospital for Sick Children · CAInserm · FRTakeda (Switzerland) · CHUCLouvain · BEUniversidade Estadual de Campinas (UNICAMP) · BR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Real-world data assessing treatment outcomes in patients with hemophilia A in routine clinical practice are limited. Objective: To evaluate the effectiveness and safety of octocog alfa in patients with moderate/severe hemophilia A receiving treatment in clinical practice. Design: The international Antihemophilic Factor Hemophilia A Outcome Database study is an observational, noninterventional, prospective, multicenter study. Methods: This planned interim data read-out was conducted following 7 years of observation of patients receiving octocog alfa (cut-off, 30 June 2020). The primary endpoint was joint health status, assessed by the Gilbert Score. Secondary endpoints included annualized bleeding rates (ABRs), Hemophilia Joint Health Score (HJHS), health-related quality of life, consumption, and safety. This Results: Of the 711 patients in this analysis, 582 (82%) were receiving prophylaxis with octocog alfa at enrollment, and 498 (70%) had severe disease. Median Gilbert Scores were higher with on-demand therapy Conclusion: These data demonstrate the long-term effectiveness and safety of octocog alfa in patients with moderate and severe hemophilia A, especially in those receiving prophylaxis. The high number of patients receiving on-demand treatment experiencing zero bleeds could be due to selection bias within the study, with patients with less severe disease more likely to be receiving on-demand treatment. Trial registration: ClinicalTrials.gov: NCT02078427.

Indexed as

bleedinghemarthrosishemophilia Aobservational studyrecombinant factor VIII

Identifiers

PMID38371314
PMCPMC10874143
OpenAlexW4391890091

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.