Evidence map›Paper›PMID 23238591›Full record

Trial reportThrombosis and haemostasis2013

Phase II prospective open-label trial of recombinant interleukin-11 in desmopressin-unresponsive von Willebrand disease and mild or moderate haemophilia A.

Margaret V Ragni, Enrico M Novelli, Anila Murshed, Elizabeth P Merricks, Mark T Kloos, Timothy C Nichols

Abstract readClinical Trial, Phase II
In one paragraph

Trial report in Thrombosis and haemostasis, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
1.0field-weighted citation impact, top 22% 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

11 citing papers in PubMed, 23 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. New therapies for von Willebrand disease.Hematology. American Society of Hematology. Education Program · 2019
    Review
  5. Review
  6. Evaluation and management of heavy menstrual bleeding in adolescents: the role of the hematologist.Hematology. American Society of Hematology. Education Program · 2018
    Review
  7. Review
  8. Review
  9. Review
  10. Lessons Learned from Animal Models of Inherited Bleeding Disorders.Hematology education. European Hematology Association. Congress. Education Program · 2014
    Article
  11. 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

6 authors at 3 institutions in 1 country.

Margaret V RagniDepartment of Medicine, Division of Hematology/Oncology and Vascular Medicine Institute, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA. ragni@dom.pitt.edu
Enrico M Novelli
Anila Murshed
Elizabeth P Merricks
Mark T Kloos
Timothy C Nichols
Hemophilia Center of Western Pennsylvania · USUniversity of Pittsburgh · USUniversity of Pittsburgh Medical Center · US

Funding

UNIVERSITY OF PITTSBURGH CLINICAL AND TRANSLATIONAL SCIENCE INSTITUTE: BPCAUL1RR024153 · NCRR · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2006 to 2011
$73.9M
University of Pittsburgh Clinical and Translational Science InstituteUL1TR000005 · NCATS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2012 to 2015
$50.8M
Maintenance of Models of Human Hemophilia and VWDR24HL063098 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI NICHOLS, TIMOTHY CHARLES · 1999 to 2019
$15.1M
NCATS NIH HHS UL1 TR000005NCRR NIH HHS UL-1 RR024153NCRR NIH HHS UL1 RR024153NHLBI NIH HHS R24 HL063098
6 · The paper itself

Abstract

Desmopressin (DDAVP) is the treatment of choice in those with mild von Willebrand disease (VWD), yet 20% are unresponsive to DDAVP, and among the 80% who respond, the response is transient, as endothelial stores are depleted after three days. We, therefore, conducted a single-center Phase II clinical trial to determine safety and biologic efficacy of recombinant interleukin-11 (rhIL-11, Neumega®) in patients with VWD unresponsive or allergic to DDAVP, or mild or moderate haemophilia A (HA). Increases in VWF:RCo wer e observed by 48 hours after rhIL-11, with a 1.54-fold increase by Day 4, 1.30-fold in VWD and 1.73-fold in HA. Similarly, by 48 hours, increases in VIII:C were observed, with a 1.65-fold increase by Day 4, 1.86-fold in VWD and 1.48-fold in HA. Platelet VWFmRNA expression by qPCR increased 0.81-fold but did not correlate with plasma VWF:Ag responses. rhIL-11 was well tolerated, with grade 1 or less fluid retention, flushing, conjunctival erythema, except for transient grade 3 hyponatraemia in one subject after excess fluid intake for diabetic hyperglycaemia, which resolved with fluid restriction. In summary, rhIL-11 increases VWF levels in two of four DDAVP-unresponsive or allergic VWD and F.VIII levels in four of five mild or moderate haemophilia A subjects, suggesting its potential use in treatment of these disorders.

Indexed as

Drug ResistanceAdultBiomarkersDeamino Arginine VasopressinFactor VIIIFemaleHemophilia AHemostaticsHumansInterleukin-11MaleMiddle AgedPennsylvaniaProspective StudiesProtein MultimerizationRecombinant ProteinsBiomarkersDeamino Arginine VasopressinF8 protein, humanFactor VIIIHemostaticsInterleukin-11oprelvekinRecombinant ProteinsRNA, Messengervon Willebrand Factor

Identifiers

PMID23238591
PMCPMC3689588
OpenAlexW2126618978

What OpenQuestion holds

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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.