Evidence map›Paper›PMID 37668788›Full record

ArticleAnnals of hematology2023

Risk factors for thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH): a nested case-control study in the International PNH Registry.

Britta Höchsmann, Regis Peffault de Latour, Anita Hill, Alexander Röth, Timothy Devos, Christopher J Patriquin, Wen-Chien Chou, Deepak Jain, Ke Zu, Chuntao Wu and 1 more

Registry-linked trialOpen access · hybridAbstract readMulticenter Study
In one paragraph

Article in Annals of hematology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01374360 (Paroxysmal Nocturnal Hemoglobinuria), which is not on this map. Cited by 12 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed, 1 pooled it
2.5field-weighted citation impact, top 10% 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.

NCT01374360 completednot on this map

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

TypeobservationalSponsorAlexion Pharmaceuticals, Inc.Ran2004 to 2025Enrolled6,061ConditionsParoxysmal Nocturnal Hemoglobinuria
3 · Its place in the literature

Who cites it

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

  1. Guideline
  2. Trial
  3. Observational
  4. Article
  5. Article
  6. Article
  7. Review
  8. Article
  9. Review
  10. Paroxysmal Nocturnal Hemoglobinuria, Pathophysiology, Diagnostics, and Treatment.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2024
    Review
  11. Review
  12. When does a PNH clone have clinical significance?Hematology. American Society of Hematology. Education Program · 2021
    Review
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

11 authors at 8 institutions in 10 countries.

Britta HöchsmannInstitute of Transfusion Medicine, University of Ulm, and Institute for Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm, Helmholtzstraße 10, 89081, Ulm, Germany. b.hoechsmann@blutspende.de.ORCID http://orcid.org/0000-0003-2257-6601
Regis Peffault de LatourBone Marrow Transplantation Unit, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.ORCID http://orcid.org/0000-0001-6222-4753
Anita HillDepartment of Hematology, Leeds Teaching Hospitals, Leeds, England, UK.ORCID http://orcid.org/0000-0001-9131-0896
Alexander RöthDepartment of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, Essen, Germany.ORCID http://orcid.org/0000-0003-4414-7699
Timothy DevosDepartment of Hematology, University Hospitals Leuven and Department of Microbiology and Immunology, Laboratory of Molecular Immunology (Rega Institute), KU Leuven, Leuven, Belgium.ORCID http://orcid.org/0000-0002-6881-417X
Christopher J PatriquinDivision of Medical Oncology & Hematology, University of Toronto, Toronto, ON, Canada.ORCID http://orcid.org/0000-0002-2089-8650
Wen-Chien ChouDepartment of Laboratory Medicine, National Taiwan University Hospital, Taipei, Taiwan.ORCID http://orcid.org/0000-0003-2967-698X
Deepak JainAlexion, AstraZeneca Rare Disease, Boston, MA, USA.
Ke ZuAlexion, AstraZeneca Rare Disease, Boston, MA, USA.ORCID http://orcid.org/0000-0002-8399-7779
Chuntao WuAlexion, AstraZeneca Rare Disease, Boston, MA, USA.ORCID http://orcid.org/0000-0002-8860-8402
Jong Wook LeeDepartment of Hematology, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea.ORCID http://orcid.org/0000-0003-2949-4166
Alexion Pharmaceuticals (United States) · USLeeds Teaching Hospitals NHS Trust · GBNational Taiwan University Hospital · TWRega Institute for Medical Research · BEThe Catholic University of Korea Seoul St. Mary's Hospital · KRUniversität Ulm · DEUniversité Paris Cité · FRUniversity of Toronto · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The objective of this analysis was to identify risk factors for thromboembolic events (TE) in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were not treated with C5 inhibitors. Patients with PNH and a history of ≥ 1 TE at enrollment in the International PNH Registry (NCT01374360; registration date, January 2011) were each matched with up to 5 patients without TE. Multivariable analysis was performed with the following variables: percentage glycosylphosphatidylinositol (GPI)-negative cells, high disease activity (HDA), non-TE major adverse vascular event history, and recent anticoagulation. Of 2541 eligible patients, 57 with TE and 189 matched controls were analyzed. Multivariable analysis (odds ratio [95% CI]) identified the following factors as being associated with increased thrombotic risk: patients with no history of TE (with recent anticoagulation, 9.30 [1.20-72.27]), patients with history of TE (with recent anticoagulation, 8.91 [0.86-92.62]; without recent anticoagulation, 5.33 [0.26-109.57]), patients with ≥ 30% GPI-negative granulocytes (≥ 30% to < 50%, 4.94 [0.54-45.32]; ≥ 50%, 1.97 [0.45-8.55]), or patients with lactate dehydrogenase (LDH) ratio ≥ 1.5 × upper limit of normal (ULN) plus ≥ 2 HDA criteria (2-3 criteria, 3.18 [0.44-23.20]; ≥ 4 criteria, 3.60 [0.38-33.95]). History of TE, ≥ 30% GPI-negative granulocytes, and LDH ratio ≥ 1.5 × ULN with ≥ 2 HDA criteria are TE risk factors for patients with PNH. These findings will aid physicians by providing important clinical and laboratory risk factors that can be used to identify and manage patients with PNH who are at risk of developing TE.

Indexed as

Hemoglobinuria, ParoxysmalRegistriesThromboembolismAdultAgedAnticoagulantsCase-Control StudiesFemaleHumansMaleMiddle AgedRisk FactorsAnticoagulantsCohort studyMultivariable analysisParoxysmal nocturnal hemoglobinuriaRisk factorsThromboembolism

Identifiers

PMID37668788
PMCPMC10567964
OpenAlexW4386438360

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.