Evidence map›Paper›PMID 39201278›Full record

Trial reportInternational journal of molecular sciences2024

Moving toward Individual Treatment Goals with Pegcetacoplan in Patients with PNH and Impaired Bone Marrow Function.

Jeff Szer, Jens Panse, Austin Kulasekararaj, Monika Oliver, Bruno Fattizzo, Jun-Ichi Nishimura, Regina Horneff, Johan Szamosi, Régis Peffault de Latour

2 registry-linked trialsAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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.

NCT03500549 phase3completednot on this map

A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

TypeinterventionalSponsorApellis Pharmaceuticals, Inc.Ran2018 to 2020Enrolled80ConditionsParoxysmal Nocturnal HemoglobinuriaArmsPegcetacoplan, Soliris
NCT04085601 phase3completednot on this map

A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

TypeinterventionalSponsorApellis Pharmaceuticals, Inc.Ran2019 to 2021Enrolled53ConditionsParoxysmal Nocturnal HemoglobinuriaArmsAPL-2
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Review
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

9 authors.

Jeff SzerDepartment of Clinical Haematology, Peter MacCallum Cancer Centre, Royal Melbourne Hospital, Melbourne, VIC 3052, Australia.ORCID 0000-0001-6783-2301
Jens PanseDepartment of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, 52074 Aachen, Germany.
Austin KulasekararajDepartment of Haematological Medicine, King's College Hospital, National Institute of Health Research/Wellcome King's Clinical Research Facility, London SE5 9RS, UK.
Monika OliverDivision of Hemtatology, Department of Apheresis Medicine, University of Alberta Hospital, University of Alberta, Edmonton, AB T6G 2B7, Canada.ORCID 0000-0002-0826-1649
Bruno FattizzoHematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.ORCID 0000-0003-0857-8379
Jun-Ichi NishimuraDepartment of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka 565-0871, Japan.
Regina HorneffSwedish Orphan Biovitrum AB, 171 65 Stockholm, Sweden.
Johan SzamosiSwedish Orphan Biovitrum AB, 171 65 Stockholm, Sweden.
Régis Peffault de LatourFrench Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, 75010 Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, potentially life-threatening haematological disease characterised by chronic complement-mediated haemolysis with multiple clinical consequences that impair quality of life. This post hoc analysis assessed haematological and clinical responses to the first targeted complement C3 inhibitor pegcetacoplan in patients with PNH and impaired bone marrow function in the PEGASUS (NCT03500549) and PRINCE (NCT04085601) studies. For patients with impaired bone marrow function, defined herein as haemoglobin <10 g/dL and absolute neutrophil count <1.5 × 10

Indexed as

Bone MarrowHemoglobinuria, ParoxysmalAdultAgedComplement C3FemaleHemoglobinsHumansL-Lactate DehydrogenaseMaleMiddle AgedQuality of LifeTreatment OutcomeComplement C3HemoglobinsL-Lactate Dehydrogenasebone marrow dysfunctionclinical responsehaematological responsepegcetacoplanPNH

Identifiers

PMID39201278
PMCPMC11354612

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.