Evidence map›Paper›PMID 37113811›Full record

ArticleTherapeutic advances in hematology2023

Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities:

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

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Therapeutic advances in hematology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01580293 (A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A), which is not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.9field-weighted citation impact, top 24% 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.

NCT01580293 phase2 / phase3completednot on this map

A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A

TypeinterventionalSponsorBayerRan2012 to 2019Enrolled145ConditionsHemophilia AArmsBAY94-9027
3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 3 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
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

10 authors at 10 institutions in 7 countries.

Mark T RedingCenter for Bleeding and Clotting Disorders, University of Minnesota Medical Center, 420 Delaware Street SE, MMC 480, Minneapolis, MN 55455, USA.ORCID https://orcid.org/0000-0003-3319-2806
Ingrid PabingerUniversity Clinic for Internal Medicine I, Medical University of Vienna, Vienna, Austria.
Pål Andrè HolmeDepartment of Haematology, Oslo University Hospital and Institute of Clinical Medicine, University of Oslo, Oslo, Norway.
Monika Maas EnriquezBayer AG, Wuppertal, Germany.
Maria Elisa MancusoCenter for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy; Humanitas University, Milan, Italy.
Shadan LalezariNational Haemophilia Centre, Chaim Sheba Medical Centre, Tel Aviv University, Tel Aviv, Israel.
Wolfgang MiesbachUniversity Hospital Frankfurt, Frankfurt am Main, Germany.
Giovanni Di MinnoDepartment of Clinical and Experimental Medicine, University of Naples Federico II, Naples, Italy.
Robert KlamrothVivantes Hospital Friedrichshain, Berlin, Germany.
Cedric HermansDivision of Haematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain (UCLouvain), Louvain, Belgium.
Bayer (Germany) · DECliniques Universitaires Saint-Luc · BEGoethe University Frankfurt · DEHumanitas University · ITKlinikum im Friedrichshain · DEMedical University of Vienna · ATOslo University Hospital · NOTel Aviv University · ILUniversity of Minnesota Medical Center · USUniversity of Naples Federico II · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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 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 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 ( 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.

Indexed as

comorbiditiesextended half-lifehaemophilia APEGylatedprophylaxis

Identifiers

PMID37113811
PMCPMC10126693
OpenAlexW4366768545

What OpenQuestion holds

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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.