Evidence map›Paper›PMID 31691462›Full record

Trial reportJournal of thrombosis and haemostasis : JTH2020

Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study.

Paul Knoebl, Spero Cataland, Flora Peyvandi, Paul Coppo, Marie Scully, Johanna A Kremer Hovinga, Ara Metjian, Javier de la Rubia, Katerina Pavenski, Jessica Minkue Mi Edou and 2 more

Registry-linked trialAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Journal of thrombosis and haemostasis : JTH, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02553317 (A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura), which is not on this map. Cited by 32 papers.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed
–field-weighted citation impact
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.

NCT02553317 phase3completednot on this map

A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura

TypeinterventionalSponsorAblynx, a Sanofi companyRan2015 to 2017Enrolled145ConditionsAcquired Thrombotic Thrombocytopenic PurpuraArmsCaplacizumab, Placebo
3 · Its place in the literature

Who cites it

32 citing papers in PubMed.

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  13. Large-Scale Production of Anti-RNase A VHH Expressed inCurrent issues in molecular biology · 2023
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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

12 authors.

Paul KnoeblDivision of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Wien, Austria.
Spero CatalandDivision of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, OH, USA.ORCID 0000-0001-8977-6910
Flora PeyvandiAngelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.ORCID 0000-0001-7423-9864
Paul CoppoDepartment of Hematology, Reference Center for Thrombotic Microangiopathies (CNR-MAT), Saint-Antoine University Hospital, AP-HP, Paris, France.
Marie ScullyCardiometabolic Programme-NIHR UCLH/UCL BRC, Department of Haematology, University College London Hospital, London, UK.
Johanna A Kremer HovingaDepartment of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.ORCID 0000-0002-1300-7135
Ara MetjianDepartment of Medicine, Duke University School of Medicine, Durham, NC, USA.
Javier de la RubiaHematology Department, Internal Medicine, School of Medicine and Dentistry, Catholic University of Valencia, Valencia, Spain.
Katerina PavenskiDepartments of Medicine and Laboratory Medicine, St. Michael's Hospital, University of Toronto, Toronto, ON, Canada.
Jessica Minkue Mi EdouClinical Development, Ablynx, a Sanofi Company, Ghent, Belgium.
Hilde De WinterFormerly Clinical Development, Ablynx, a Sanofi Company, Ghent, Belgium.
Filip CallewaertMedical Affairs, Sanofi, Diegem, Belgium.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundAcquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life-threatening autoimmune thrombotic microangiopathy. Caplacizumab, an anti-von Willebrand Factor Nanobody OBJECTIVES AND

methodsIn the phase 3 HERCULES trial (NCT02553317), patients with aTTP received double-blind caplacizumab or placebo during daily therapeutic plasma exchange (TPE) and for ≥30 days thereafter. Patients who experienced an exacerbation while on blinded study drug treatment switched to receive open-label caplacizumab plus re-initiation of daily TPE. Exacerbations were defined as recurrence of disease occurring within 30 days after cessation of daily TPE.

resultsThirty-one patients (placebo, n = 28; caplacizumab, n = 3) had an exacerbation during double-blind treatment. Twenty-eight patients switched to open-label caplacizumab (placebo, n = 26; caplacizumab, n = 2); the three others discontinued upon exacerbation. Median time to platelet count response (≥150 × 10

conclusionsThese results suggest that caplacizumab was efficacious and well tolerated in patients with aTTP who experienced a disease exacerbation during double-blind treatment in HERCULES.

Indexed as

Purpura, Thrombotic ThrombocytopenicSingle-Domain AntibodiesADAMTS13 ProteinFibrinolytic AgentsHumansPlasma ExchangeADAMTS13 ProteincaplacizumabFibrinolytic AgentsSingle-Domain AntibodiesADAMTS13 proteincaplacizumabplasma exchangepurpurathrombotic thrombocytopenicvon Willebrand factor

Identifiers

PMID31691462
PMCPMC7027866

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.