Evidence map›Paper›PMID 24345753›Full record

Trial reportBlood2014

Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug.

Ronan Desmond, Danielle M Townsley, Bogdan Dumitriu, Matthew J Olnes, Phillip Scheinberg, Margaret Bevans, Ankur R Parikh, Kinneret Broder, Katherine R Calvo, Colin O Wu and 2 more

3 registry-linked trialsAbstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Blood, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 201 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
201citing papers in PubMed, 6 pooled it
–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.

NCT00922883 phase2completednot on this map

A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Aplastic Anemia Patients With Immunosuppressive-Therapy Refractory Thrombocytopenia

TypeinterventionalSponsorNational Heart, Lung, and Blood Institute (NHLBI)Ran2009 to 2018Enrolled43ConditionsAnemia, Aplastic, Anemia, Hypoplastic, ThrombocytopeniaArmsEltrombopag
NCT03243656 phase4completednot on this mapstarted 2017, after this paper: background citation

Randomized Clinical Trial of the Use of & Cyclosporine in Children With Sever Idiopathic Aplastic Anemia

TypeinterventionalSponsorAssiut UniversityRan2017 to 2020Enrolled20ConditionsEltrombopagArmsEltrombopag
NCT03896971 phase4completednot on this mapstarted 2019, after this paper: background citation

Treatment With Thrombopoietin Mimetic Plus Immunosuppressive Therapy in Egyptian Patients With Aplastic Anaemia

TypeinterventionalSponsorSafaa AA KhaledRan2019 to 2021Enrolled66ConditionsAplastic AnemiaArmsCombination of thrombopoietin mimetic and cyclosporin A
3 · Its place in the literature

Who cites it

201 citing papers in PubMed, 6 syntheses or guidelines pooled it.

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

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors.

Ronan DesmondHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD;
Danielle M Townsley
Bogdan Dumitriu
Matthew J Olnes
Phillip Scheinberg
Margaret Bevans
Ankur R Parikh
Kinneret Broder
Katherine R Calvo
Colin O Wu
Neal S Young
Cynthia E Dunbar

Funding

Pathogenesis And Treatment Of Aplastic AnemiaZIAHL002315 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI YOUNG, NEAL S · 2009 to 2025
$60.1M
Novel therapies for bone marrow failure and Diamond-Blackfan AnemiaZIAHL006186 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI DUNBAR, CYNTHIA E · 2014 to 2025
$5.7M
The Role of microRNA in Hematologic MalignanciesZIACL090025 · CLC · CLINICAL CENTER · PI CALVO, KATHERINE · 2010 to 2025
$0k
Intramural NIH HHS
6 · The paper itself

Abstract

About a quarter of patients with severe aplastic anemia remain pancytopenic despite immunosuppressive therapy. We have previously demonstrated that eltrombopag has efficacy in this setting with 44% (11/25) of patients having clinically significant hematologic responses. We now report safety and efficacy data on a further 18 patients and long-term follow-up on the entire cohort of 43 patients. The overall response rate was 17 of 43 patients (40%) at 3 to 4 months, including tri- and bilineage responses. The majority of patients who remained on eltrombopag in an extension study (14/17) continued to show improvement, and 7 eventually had significant increases in neutrophil, red cell, and platelet lineages. Five patients with robust near-normalization of blood counts had drug discontinued at a median of 28.5 months after entry (range, 9-37 months), and all maintained stable counts a median of 13 months (range, 1-15 months) off eltrombopag. Eight patients, including 6 nonresponders and 2 responders, developed new cytogenetic abnormalities on eltrombopag, including 5 with chromosome 7 loss or partial deletion. None evolved to acute myeloid leukemia to date. Eltrombopag is efficacious in a subset of patients with aplastic anemia refractory to immunosuppressive therapy, with frequent multilineage responses and maintenance of normalized hematopoiesis off treatment. This study is registered at www.clinicaltrials.gov as #NCT00922883.

Indexed as

AdolescentAdultAgedAnemia, AplasticBenzoatesBone MarrowClonal EvolutionFemaleHematologic AgentsHematopoiesisHematopoietic Stem CellsHumansHydrazinesImmunosuppressive AgentsMaleMiddle AgedBenzoateseltrombopagHematologic AgentsHydrazinesImmunosuppressive AgentsMPL protein, humanPyrazolesReceptors, Thrombopoietin

Identifiers

PMID24345753
PMCPMC3962161

What OpenQuestion holds

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Read underepoch 390

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.