Evidence map›Paper›PMID 38573482›Full record

ArticleAdvances in therapy2024

Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study.

Christopher J Patriquin, Andrija Bogdanovic, Morag Griffin, Richard J Kelly, Jaroslaw P Maciejewski, Brian Mulherin, Régis Peffault de Latour, Alexander Röth, Veena Selvaratnam, Jeffrey Szer and 5 more

Registry-linked trialOpen access · hybridAbstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03531255 (An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria), which is not on this map. Cited by 13 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 1 pooled it
3.8field-weighted citation impact, top 6% 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.

NCT03531255 phase3completednot on this map

An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

TypeinterventionalSponsorApellis Pharmaceuticals, Inc.Ran2018 to 2026Enrolled137ConditionsPNHArmsPegcetacoplan
3 · Its place in the literature

Who cites it

13 citing papers in PubMed, 1 synthesis or guideline pooled it, 16 citations in OpenAlex.

  1. Pooled it
  2. Trial
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  6. Review
  7. Article
  8. Observational
  9. Extravascular hemolysis in PNH.Blood advances · 2025
    Article
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  11. Article
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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

15 authors at 11 institutions in 9 countries.

Christopher J PatriquinHematology & Apheresis Medicine, University Health Network, Toronto General Hospital, Toronto, ON, Canada. Christopher.Patriquin@uhn.ca.
Andrija BogdanovicClinic of Hematology, Clinical Center of Serbia, Faculty of Medicine, University of Belgrade, Belgrade, Serbia.
Morag GriffinDepartment of Haematology, St. James's University Hospital, Leeds, UK.
Richard J KellyDepartment of Haematology, St. James's University Hospital, Leeds, UK.
Jaroslaw P MaciejewskiDepartment of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA.
Brian MulherinHematology Oncology of Indiana, Indianapolis, IN, USA.
Régis Peffault de LatourFrench Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France.
Alexander RöthDepartment of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, Essen, Germany.
Veena SelvaratnamDepartment of Hematology, Ampang Hospital, Ampang, Malaysia.
Jeffrey SzerDepartment of Clinical Haematology, The Royal Melbourne Hospital and Peter MacCallum Cancer Centre, Melbourne, VIC, Australia.
Mohammed Al-AdhamiApellis Pharmaceuticals, Waltham, MA, USA.
Regina HorneffSwedish Orphan Biovitrum AB, Stockholm, Sweden.
Lisa TanSwedish Orphan Biovitrum AB, Stockholm, Sweden.
Michael YehApellis Pharmaceuticals, Waltham, MA, USA.
Jens PanseCenter for Integrated Oncology, Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany.
Apellis Pharmaceuticals (United States) · USSt James's University Hospital · GBSwedish Orphan Biovitrum (Sweden) · SEAssistance Publique – Hôpitaux de Paris · FRCleveland Clinic · USKPJ Ampang Puteri Specialist Hospital · MYRWTH Aachen University · DEThe Royal Melbourne Hospital · AUToronto General Hospital · CAUniversity of Belgrade · RSUrology of Indiana · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionParoxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis and thrombosis. Pegcetacoplan, the first targeted complement component 3 (C3) PNH therapy, was safe and efficacious in treatment-naive and pre-treated patients with PNH in five clinical trials.

methodsThe 307 open-label extension (OLE) study (NCT03531255) is a non-randomized, multicenter extension study of long-term safety and efficacy of pegcetacoplan in adult patients with PNH who completed a pegcetacoplan parent study. All patients received pegcetacoplan. Outcomes at the 48-week data cutoff (week 48 of 307-OLE or August 27, 2021, whichever was earlier) are reported. Hemoglobin concentrations, Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores, and transfusion avoidance were measured. Hemoglobin > 12 g/dL and sex-specific hemoglobin normalization (i.e., male, ≥ 13.6 g/dL; female, ≥ 12 g/dL) were assessed as percentage of patients with data available and no transfusions 60 days before data cutoff. Treatment-emergent adverse events, including hemolysis, were reported.

resultsData from 137 patients with at least one pegcetacoplan dose at data cutoff were analyzed. Mean (standard deviation [SD]) hemoglobin increased from 8.9 (1.22) g/dL at parent study baseline to 11.6 (2.17) g/dL at 307-OLE entry and 11.6 (1.94) g/dL at data cutoff. At parent study baseline, mean (SD) FACIT-Fatigue score of 34.1 (11.08) was below the general population norm of 43.6; scores improved to 42.8 (8.79) at 307-OLE entry and 42.4 (9.84) at data cutoff. In evaluable patients, hemoglobin > 12 g/dL occurred in 40.2% (43 of 107) and sex-specific hemoglobin normalization occurred in 31.8% (34 of 107) at data cutoff. Transfusion was not required for 114 of 137 patients (83.2%). Hemolysis was reported in 23 patients (16.8%). No thrombotic events or meningococcal infections occurred.

conclusionPegcetacoplan sustained long-term improvements in hemoglobin concentrations, fatigue reduction, and transfusion burden. Long-term safety findings corroborate the favorable profile established for pegcetacoplan.

trial registrationClinicalTrials.gov identifier, NCT03531255.

Indexed as

Hemoglobinuria, ParoxysmalAdultAgedFemaleHemoglobinsHumansMaleMiddle AgedTreatment OutcomeHemoglobinsClinical trialComplement inhibitorEculizumabFACIT-FatigueHemoglobinOpen-label extensionParoxysmal nocturnal hemoglobinuria (PNH)PegcetacoplanThrombosisTransfusion

Identifiers

PMID38573482
PMCPMC11052769
OpenAlexW4393950389

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.