Evidence map›Paper›PMID 28516949›Full record

ReviewNature reviews. Disease primers2017

Paroxysmal nocturnal haemoglobinuria.

Anita Hill, Amy E DeZern, Taroh Kinoshita, Robert A Brodsky

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Nature reviews. Disease primers, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 249 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
249citing papers in PubMed, 3 pooled it
14.5field-weighted citation impact, top 1% 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

249 citing papers in PubMed, 3 syntheses or guidelines pooled it, 425 citations in OpenAlex.

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189 more citing papers are in PubMed but not listed here.

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

4 authors at 4 institutions in 3 countries.

Anita HillDepartment of Haematology, St. James' University Hospital, Leeds, UK.
Amy E DeZernDivision of Hematology, Johns Hopkins Department of Medicine, Johns Hopkins University, Ross Research Building, Room 1025, 720 Rutland Avenue, Baltimore, Maryland 21205, USA.
Taroh KinoshitaLaboratory of Immunoglycobiology, Immunology Frontier Research Center, Osaka University, Osaka, Japan.
Robert A BrodskyDivision of Hematology, Johns Hopkins Department of Medicine, Johns Hopkins University, Ross Research Building, Room 1025, 720 Rutland Avenue, Baltimore, Maryland 21205, USA.
Johns Hopkins University · USOsaka University · JPSidney Kimmel Comprehensive Cancer Center · USSt James's University Hospital · GB

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 haemoglobinuria (PNH) is a clonal haematopoietic stem cell (HSC) disease that presents with haemolytic anaemia, thrombosis and smooth muscle dystonias, as well as bone marrow failure in some cases. PNH is caused by somatic mutations in PIGA (which encodes phosphatidylinositol N-acetylglucosaminyltransferase subunit A) in one or more HSC clones. The gene product of PIGA is required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIGA mutations lead to a deficiency of GPI-anchored proteins, such as complement decay-accelerating factor (also known as CD55) and CD59 glycoprotein (CD59), which are both complement inhibitors. Clinical manifestations of PNH occur when a HSC clone carrying somatic PIGA mutations acquires a growth advantage and differentiates, generating mature blood cells that are deficient of GPI-anchored proteins. The loss of CD55 and CD59 renders PNH erythrocytes susceptible to intravascular haemolysis, which can lead to thrombosis and to much of the morbidity and mortality of PNH. The accumulation of anaphylatoxins (such as C5a) from complement activation might also have a role. The natural history of PNH is highly variable, ranging from quiescent to life-threatening. Therapeutic strategies include terminal complement blockade and bone marrow transplantation. Eculizumab, a monoclonal antibody complement inhibitor, is highly effective and the only licensed therapy for PNH.

Indexed as

Antibodies, Monoclonal, HumanizedBone Marrow TransplantationCD55 AntigensCD59 AntigensComplement Inactivator ProteinsHemoglobinuria, ParoxysmalHumansMembrane ProteinsMutationTreatment OutcomeAntibodies, Monoclonal, HumanizedCD55 AntigensCD59 AntigensCD59 protein, humanComplement Inactivator ProteinseculizumabMembrane Proteinsphosphatidylinositol glycan-class A protein

Identifiers

PMID28516949
PMCPMC7879566
OpenAlexW2616618502

What OpenQuestion holds

Textmetadata
LicenceTDM
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.