Evidence map›Paper›PMID 28423296›Full record

Trial reportThe New England journal of medicine2017

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

Danielle M Townsley, Phillip Scheinberg, Thomas Winkler, Ronan Desmond, Bogdan Dumitriu, Olga Rios, Barbara Weinstein, Janet Valdez, Jennifer Lotter, Xingmin Feng and 8 more

5 registry-linked trialsOpen access · bronzeAbstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The New England journal of medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 5 registered trials, which are not on this map. Cited by 300 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
300citing papers in PubMed, 7 pooled it
37.7field-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.

NCT01623167 phase1 / phase2active not recruitingnot on this map

Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia

TypeinterventionalSponsorNational Heart, Lung, and Blood Institute (NHLBI)Ran2012 to 2029Enrolled207ConditionsSevere Aplastic AnemiaArmsCohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6, Extension Cohort
NCT03243656 phase4completednot on this map

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

TypeinterventionalSponsorAssiut UniversityRan2017 to 2020Enrolled20ConditionsEltrombopagArmsEltrombopag
NCT04485416 phase1suspendednot on this mapstarted 2021, after this paper: background citation

Open Label Single Arm Prospective Pilot Trial of Eltrombopag in Patients 1 Year to 18 Years of Age Undergoing Intensive Chemotherapy for Malignant Solid Tumors (CCPO011)

TypeinterventionalSponsorUniversity of California, DavisRan2021 to 2025Enrolled10ConditionsSolid Tumor, Childhood, Solid TumorArmsEltrombopag
NCT05433922 naunknown statusnot on this mapstarted 2022, after this paper: background citation

A Multicenter, Single-arm Clinical Study of the Efficacy and Safety of CSA in Combination With Avatrombopag for the Treatment of Primary Treatment of Severe Aplastic Anemia in the Elderly

TypeinterventionalSponsorInstitute of Hematology & Blood Diseases Hospital, ChinaRan2022 to 2024Enrolled80ConditionsSevere Aplastic AnemiaArmsAvatrombopag
NCT05518331 naunknown statusnot on this mapstarted 2022, after this paper: background citation

Avatrombopag in TPO-RA Refractory Aplastic Anemia Patients Safety and Efficacy Study --Single-arm, Multicenter, Open, Phase Π Clinical Study

TypeinterventionalSponsorInstitute of Hematology & Blood Diseases Hospital, ChinaRan2022 to 2026Enrolled39ConditionsRefractory Aplastic AnemiaArmsavatrombopag
3 · Its place in the literature

Who cites it

300 citing papers in PubMed, 7 syntheses or guidelines pooled it, 526 citations in OpenAlex.

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240 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

18 authors at 2 institutions in 2 countries.

Danielle M TownsleyFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Phillip ScheinbergFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Thomas WinklerFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Ronan DesmondFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Bogdan DumitriuFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Olga RiosFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Barbara WeinsteinFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Janet ValdezFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Jennifer LotterFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Xingmin FengFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Marie DesiertoFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Harshraj LeuvaFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Margaret BevansFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Colin WuFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Andre LarochelleFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Katherine R CalvoFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Cynthia E DunbarFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
Neal S YoungFrom the Hematology Branch (D.M.T., T.W., R.D., B.D., O.R., B.W., J.V., J.L., X.F., M.D., H.L., A.L., C.E.D., N.S.Y.) and the Office of Biostatistics Research (C.W.), National Heart, Lung, and Blood Institute, and the Nursing Research and Translational Science Section, Department of Nursing (M.B.), and the Hematology Section, Department of Laboratory Medicine (K.R.C.), Clinical Center - all at the National Institutes of Health, Bethesda, MD; and the Division of Clinical Hematology, Antônio Ermírio de Moraes Cancer Center, Hospital A Beneficência Portuguesa de São Paulo, São Paulo (P.S.).
National Heart Lung and Blood Institute · USBeneficência Portuguesa de São Paulo · BR

Funding

Pathogenesis And Treatment Of Aplastic AnemiaZIAHL002315 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI YOUNG, NEAL S · 2009 to 2025
$60.1M
Regenerative Therapies for Inherited Blood Disorders-Gene therapyZIAHL006172 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2013 to 2025
$13.0M
Regenerative Therapies for Inherited Blood Disorders-iPSC differentiationZIAHL006217 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2016 to 2025
$8.5M
Investigation of the mechanisms of action of eltrombopagZIAHL006205 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2015 to 2025
$6.8M
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
Regenerative Therapies for Inherited Blood Disorders-HSC expansionZIAHL006218 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2016 to 2022
$3.2M
The Role of microRNA in Hematologic MalignanciesZIACL090025 · CLC · CLINICAL CENTER · PI CALVO, KATHERINE · 2010 to 2025
$0k
Intramural NIH HHS Z99 HL999999
6 · The paper itself

Abstract

backgroundAcquired aplastic anemia results from immune-mediated destruction of bone marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.

methodsWe enrolled 92 consecutive patients in a prospective phase 1-2 study of immunosuppressive therapy plus eltrombopag. The three consecutively enrolled cohorts differed with regard to the timing of initiation and the duration of the eltrombopag regimen (cohort 1 received eltrombopag from day 14 to 6 months, cohort 2 from day 14 to 3 months, and cohort 3 from day 1 to 6 months). The cohorts were analyzed separately. The primary outcome was complete hematologic response at 6 months. Secondary end points included overall response, survival, relapse, and clonal evolution to myeloid cancer.

resultsThe rate of complete response at 6 months was 33% in cohort 1, 26% in cohort 2, and 58% in cohort 3. The overall response rates at 6 months were 80%, 87%, and 94%, respectively. The complete and overall response rates in the combined cohorts were higher than in our historical cohort, in which the rate of complete response was 10% and the overall response rate was 66%. At a median follow-up of 2 years, the survival rate was 97%; one patient died during the study from a nonhematologic cause. Marked increases in bone marrow cellularity, CD34+ cell number, and frequency of early hematopoietic progenitors were noted. Rates of relapse and clonal evolution were similar to our historical experience. Severe rashes occurred in two patients, resulting in the early discontinuation of eltrombopag.

conclusionsThe addition of eltrombopag to immunosuppressive therapy was associated with markedly higher rates of hematologic response among patients with severe aplastic anemia than in a historical cohort. (Funded by the National Heart, Lung, and Blood Institute; ClinicalTrials.gov number, NCT01623167 .).

Indexed as

AdolescentAdultAgedAnemia, AplasticAntigens, CD34Antilymphocyte SerumBenzoatesCell CountCyclosporineDrug Therapy, CombinationFemaleHematologic AgentsHumansHydrazinesImmunosuppression TherapyImmunosuppressive AgentsAntigens, CD34Antilymphocyte SerumBenzoatesCyclosporineeltrombopagHematologic AgentsHydrazinesImmunosuppressive AgentsPyrazolesReceptors, Thrombopoietin

Identifiers

PMID28423296
PMCPMC5548296
OpenAlexW2605781925

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.