Evidence map›Paper›PMID 35869170›Full record

ArticleAnnals of hematology2022

Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria.

Raymond S M Wong, Humphrey W H Pullon, Ismail Amine, Andrija Bogdanovic, Pascal Deschatelets, Cedric G Francois, Kalina Ignatova, Surapol Issaragrisil, Pimjai Niparuck, Tontanai Numbenjapon and 7 more

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

Article in Annals of hematology, 2022. 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 24 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 2 pooled it
3.1field-weighted citation impact, top 7% 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.

NCT02588833 phase1completednot on this map

A Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

TypeinterventionalSponsorApellis Pharmaceuticals, Inc.Ran2015 to 2019Enrolled23ConditionsParoxysmal Nocturnal HemoglobinuriaArmsPegcetacoplan
NCT03593200 phase2completednot on this map

Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).

TypeinterventionalSponsorApellis Pharmaceuticals, Inc.Ran2018 to 2019Enrolled4ConditionsPNHArmsPegcetacoplan
3 · Its place in the literature

Who cites it

24 citing papers in PubMed, 2 syntheses or guidelines pooled it, 38 citations in OpenAlex.

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  9. [Advances in complement inhibition therapy for paroxysmal nocturnal hemoglobinuria].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025
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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

17 authors at 11 institutions in 8 countries.

Raymond S M WongSir Y.K. Pao Centre for Cancer and Department of Medicine and Therapeutics, Prince of Wales Hospital, The Chinese University of Hong Kong, Shatin, Hong Kong, China.
Humphrey W H PullonWaikato Hospital, Hamilton, New Zealand.
Ismail AmineTokuda Hospital, Sofia, Bulgaria.
Andrija BogdanovicClinic of Hematology, Clinical Center of Serbia, Faculty of Medicine, University of Belgrade, Belgrade, Serbia.
Pascal DeschateletsApellis Pharmaceuticals, 100 5th Avenue, Waltham, MA, 02451, USA.
Cedric G FrancoisApellis Pharmaceuticals, 100 5th Avenue, Waltham, MA, 02451, USA.
Kalina IgnatovaNational Specialized Hospital for Active Treatment of Hematologic Diseases, Sofia, Bulgaria.
Surapol IssaragrisilFaculty of Medicine Siriraj Hospital, Bangkok, Thailand.
Pimjai NiparuckRamathibodi Hospital, Bangkok, Thailand.
Tontanai NumbenjaponPhramongkutklao Hospital and Phramongkutklao College of Medicine, Bangkok, Thailand.
Eloy RomanLakes Research, Miami Lakes, FL, USA.
Jameela SatharDepartment of Hematology, Ampang Hospital, Selangor, Malaysia.
Raymond XuApellis Pharmaceuticals, 100 5th Avenue, Waltham, MA, 02451, USA.
Mohammed Al-AdhamiApellis Pharmaceuticals, 100 5th Avenue, Waltham, MA, 02451, USA.
Lisa TanLisa Tan Pharma Consulting Ltd, Cambridge, UK.
Eric TseDepartment of Medicine, The University of Hong Kong, Pokfulam, Hong Kong, China.
Federico V GrossiApellis Pharmaceuticals, 100 5th Avenue, Waltham, MA, 02451, USA. federico@apellis.com.
Apellis Pharmaceuticals (United States) · USChinese University of Hong Kong · HKKPJ Ampang Puteri Specialist Hospital · MYNational Specialized Hospital for Active Treatment of Hematologic Diseases · BGPhramongkutklao College of MedicineRamathibodi Hospital · THSiriraj Hospital · THTokuda Hospital · BGUniversity of Belgrade · RSUniversity of Hong Kong · HKWaikato Hospital · NZ

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic disorder characterized by complement-mediated hemolysis. C5 inhibitors (eculizumab/ravulizumab) control intravascular hemolysis but do not prevent residual extravascular hemolysis. The newly approved complement inhibitor, pegcetacoplan, inhibits C3, upstream of C5, and has the potential to improve control of complement-mediated hemolysis. The PADDOCK and PALOMINO clinical trials assessed the safety and efficacy of pegcetacoplan in complement inhibitor-naïve adults (≥ 18 years) diagnosed with PNH. Patients in PADDOCK (phase 1b open-label, pilot trial) received daily subcutaneous pegcetacoplan (cohort 1: 180 mg up to day 28 [n = 3]; cohort 2: 270-360 mg up to day 365 [n = 20]). PALOMINO (phase 2a, open-label trial) used the same dosing protocol as PADDOCK cohort 2 (n = 4). Primary endpoints in both trials were mean change from baseline in hemoglobin, lactate dehydrogenase, haptoglobin, and the number and severity of treatment-emergent adverse events. Mean baseline hemoglobin levels were below the lower limit of normal in both trials (PADDOCK: 8.38 g/dL; PALOMINO: 7.73 g/dL; normal range: 11.90-18.00 g/dL), increased to within normal range by day 85, and were sustained through day 365 (PADDOCK: 12.14 g/dL; PALOMINO: 13.00 g/dL). In PADDOCK, 3 serious adverse events (SAE) led to study drug discontinuation, 1 of which was deemed likely related to pegcetacoplan and 1 SAE, not deemed related to study drug, led to death. No SAE led to discontinuation/death in PALOMINO. Pegcetacoplan was generally well tolerated and improved hematological parameters by controlling hemolysis, while also improving other clinical PNH indicators in both trials. These trials were registered at www.clinicaltrials.gov (NCT02588833 and NCT03593200).

Indexed as

Complement Inactivating AgentsHemoglobinuria, ParoxysmalPeptides, CyclicAdultBiomarkersClinical Trials, Phase I as TopicClinical Trials, Phase II as TopicHemoglobinsHemolysisHumansBiomarkersComplement Inactivating AgentsHemoglobinspegcetacoplanPeptides, CyclicHemoglobinLDHParoxysmal nocturnal hemoglobinuria (PNH)Phase I/II trialsQuality of lifeSafety

Identifiers

PMID35869170
PMCPMC9375762
OpenAlexW4286587544

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.