Evidence map›Paper›PMID 37707673›Full record

ArticleAdvances in therapy2023

Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report.

Vivek Sharma, Jamie Koprivnikar, Kristen Drago, Jessica Savage, Allison Bachelor

2 registry-linked trialsOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2023. 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 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.5field-weighted citation impact, top 30% 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.

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
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

1 citing paper in PubMed, 3 citations in OpenAlex.

  1. Trial
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

5 authors at 3 institutions in 1 country.

Vivek SharmaUniversity of Louisville, Louisville, KY, USA. vivek.sharma@louisville.edu.ORCID http://orcid.org/0000-0003-4871-2114
Jamie KoprivnikarJohn Theurer Cancer Center at Hackensack University Medical Center, Hackensack, NJ, USA.
Kristen DragoApellis Pharmaceuticals, Inc, Waltham, MA, USA.
Jessica SavageApellis Pharmaceuticals, Inc, Waltham, MA, USA.
Allison BachelorApellis Pharmaceuticals, Inc, Waltham, MA, USA.
Apellis Pharmaceuticals (United States) · USHackensack University Medical Center · USUniversity of Louisville · US

Funding

Apellis PharmaceuticalsSwedish Orphan Biovitrum
6 · The paper itself

Abstract

introductionPegcetacoplan is a targeted complement component 3 (C3) therapy approved for adults with paroxysmal nocturnal hemoglobinuria (PNH; US) or PNH plus anemia despite C5-targeted therapy for ≥ 3 months (EU). Patients with PNH receiving pegcetacoplan in the phase 3 PEGASUS trial who experienced injection site reactions (ISRs) mostly experienced mild events. We evaluated ISR incidence and severity with longer-term treatment in the PEGASUS cohort of the Study 307 open-label extension (307 OLE).

methodsPatients from PEGASUS enrolled in the 307 OLE continued pegcetacoplan subcutaneous self-administration twice or three times weekly or every 3 days for an additional 48 weeks. ISRs were coded as adverse events (AEs) or treatment-emergent AEs (TEAEs) and summarized by MedDRA System Organ Class and Preferred Term.

resultsAs of August 27, 2021, 58/64 patients from PEGASUS completed an additional 48 weeks of treatment in the 307 OLE (median treatment duration 337.0 [range 55-344] days); 95.3% (61/64) of patients achieved compliance ≥ 80%. ISRs occurred in 9/64 (14.1%) patients in the 307 OLE, which was lower than observed at PEGASUS completion (20/77; 26.0%). Most patients with ISRs in the 307 OLE had events with a maximum severity of mild (7/9 patients; 77.8%). Injection site erythema and induration were the most common overall (4/64 patients each; 6.3%) and pegcetacoplan-related (3/64 patients each; 4.7%) ISRs. The exposure-adjusted rates of these events were each 6.5 per 100 patient-years. No ISRs were classified as severe or serious TEAEs or led to drug discontinuation.

conclusionThough ISRs were common, most were mild, and the percentage of patients reporting ISRs declined from PEGASUS through the 307 OLE. Patient compliance remained high, and no patients discontinued because of ISRs, suggesting that ISRs do not pose a barrier to long-term pegcetacoplan treatment.

trial registrationClinicalTrials.gov identifiers: NCT03500549 (PEGASUS) and NCT03531255 (307 OLE).

Indexed as

Hemoglobinuria, ParoxysmalAdultHumansInjection Site ReactionPeptides, CyclicpegcetacoplanPeptides, CyclicClinical trialInjection site reactionsManagementParoxysmal nocturnal hemoglobinuriaPegcetacoplan

Identifiers

PMID37707673
PMCPMC10567944
OpenAlexW4386726754

What Socratic 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 Socratic graph.