Evidence map›Paper›PMID 27810992›Full record

ArticleHaematologica2017

Small-molecule factor D inhibitors selectively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

Xuan Yuan, Eleni Gavriilaki, Jane A Thanassi, Guangwei Yang, Andrea C Baines, Steven D Podos, Yongqing Huang, Mingjun Huang, Robert A Brodsky

Open access · goldAbstract read
In one paragraph

Article in Haematologica, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers.

0numbers the graph read from it
0cells of the map it votes in
59citing papers in PubMed
5.6field-weighted citation impact, top 4% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

59 citing papers in PubMed, 98 citations in OpenAlex.

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  8. Highlights on U.S. FDA-approved halogen-containing drugs in 2024.European journal of medicinal chemistry · 2025
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  20. Intertwined pathways of complement activation command the pathogenesis of lupus nephritis.Translational research : the journal of laboratory and clinical medicine · 2022
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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

9 authors at 3 institutions in 1 country.

Xuan YuanDivision of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Eleni GavriilakiDivision of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Jane A ThanassiAchillion Pharmaceuticals, New Haven, CT, USA.
Guangwei YangAchillion Pharmaceuticals, New Haven, CT, USA.
Andrea C BainesDivision of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Steven D PodosAchillion Pharmaceuticals, New Haven, CT, USA.
Yongqing HuangAchillion Pharmaceuticals, New Haven, CT, USA.
Mingjun HuangAchillion Pharmaceuticals, New Haven, CT, USA.
Robert A BrodskyDivision of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA rbrodsky@jhmi.edu.
Achillion Pharmaceuticals (United States) · USJohns Hopkins Medicine · USJohns Hopkins University · US

Funding

Complementopathies: Genotype and PhenotypeR01HL133113 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI BRODSKY, ROBERT A · 2016 to 2020
$2.0M
NHLBI NIH HHS R01 HL133113
6 · The paper itself

Abstract

Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome are diseases of excess activation of the alternative pathway of complement that are treated with eculizumab, a humanized monoclonal antibody against the terminal complement component C5. Eculizumab must be administered intravenously, and moreover some patients with paroxysmal nocturnal hemoglobinuria on eculizumab have symptomatic extravascular hemolysis, indicating an unmet need for additional therapeutic approaches. We report the activity of two novel small-molecule inhibitors of the alternative pathway component Factor D using

Indexed as

AdultAgedAnimalsAtypical Hemolytic Uremic SyndromeBiomarkersComplement C3Complement Factor DComplement Inactivating AgentsComplement Pathway, AlternativeCytotoxicity, ImmunologicDisease Models, AnimalErythrocytesFemaleHemoglobinuria, ParoxysmalHemolysisHumansBiomarkersComplement C3Complement Factor DComplement Inactivating Agents

Identifiers

PMID27810992
PMCPMC5394948
OpenAlexW2548090658

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

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.