Evidence map›Paper›PMID 38238183›Full record

ArticleBMJ open2024

Efficacy and safety of avatrombopag in combination with immunosuppressive therapy in treatment-naïve and relapsed/refractory severe aplastic anaemia: protocol for the DIAAMOND-Ava-FIRST and DIAAMOND-Ava-NEXT Bayesian Optimal Phase II trials.

Zoe McQuilten, Stephane Heritier, Lucy Fox, Vanessa Fox, Lauren Young, Piers Blombery, Ilona Cunningham, Jennifer Curnow, Alisa Higgins, Devendra K Hiwase and 13 more

Open access · goldAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in BMJ open, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.7field-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.

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

8 citing papers in PubMed, 7 citations in OpenAlex.

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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

23 authors at 10 institutions in 4 countries.

Zoe McQuiltenDepartment of Haematology, Monash Health, Melbourne, Victoria, Australia Zoe.McQuilten@monash.edu.ORCID 0000-0001-9698-7185
Stephane HeritierSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Lucy FoxSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Vanessa FoxSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Lauren YoungSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Piers BlomberyDepartment of Clinical Haematology, Peter MacCallum Cancer Centre & The Royal Melbourne Hospital, Parkville, Victoria, Australia.
Ilona CunninghamConcord Repatriation General Hospital, Sydney, New South Wales, Australia.
Jennifer CurnowDepartment of Haematology, Westmead Hospital, Sydney, New South Wales, Australia.
Alisa HigginsSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Devendra K HiwaseDepartment of Haematology, Royal Adelaide Hospital, Adelaide, South Australia, Australia.
Robin FilshieHaematology Department, St Vincent's Hospital, Melbourne, Victoria, Australia.
Frank FirkinHaematology Department, St Vincent's Hospital, Melbourne, Victoria, Australia.
Paul LacazeSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Kylie MasonDepartment of Clinical Haematology, Peter MacCallum Cancer Centre & The Royal Melbourne Hospital, Parkville, Victoria, Australia.
Anthony K MillsUniversity of Queensland, Brisbane, Queensland, Australia.
Dominic PepperellDepartment of Haematology, Fiona Stanley Hospital, Murdoch, Perth, Australia.
Sushrut PatilDepartment of Haematology, Alfred Hospital, Melbourne, Victoria, Australia.
William StevensonDepartment of Haematology, Royal North Shore Hospital, St Leonards, Sydney, Australia.
Jeff SzerDepartment of Clinical Haematology, Peter MacCallum Cancer Centre & The Royal Melbourne Hospital, Parkville, Victoria, Australia.
Neil WatersSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.ORCID 0000-0002-8012-1827
Kate WilsonUniversity of Sydney, Sydney, New South Wales, Australia.
Stephen TingSchool of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, Australia.
Erica WoodDepartment of Haematology, Monash Health, Melbourne, Victoria, Australia.
Australian Regenerative Medicine Institute · AUThe Royal Melbourne Hospital · AUSt. Vincent's Birmingham · USThe University of Sydney · AUFiona Stanley Hospital · AURoyal Adelaide Hospital · AURoyal North Shore Hospital · AUThe Alfred Hospital · AUThe University of Queensland · AUWestmead Hospital · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionImmunosuppressive therapy (IST) with antithymocyte globulin (ATG) and ciclosporin is standard of care for patients with severe aplastic anaemia (sAA) not eligible or suitable for allogeneic stem cell transplant. While patients respond to IST, few achieve complete responses and a significant proportion are refractory or relapse. The addition of eltrombopag, a thrombopoietin-receptor agonist (TPO-A), to IST has been shown to improve haematological responses in sAA. Avatrombopag is a second-generation TPO-A with potential advantages over eltrombopag. However, to date avatrombopag has not been studied in sAA. METHODS AND ANALYSIS: Investigator-initiated, single-arm registry-based Bayesian Optimal Phase II trial of avatrombopag conducted in two cohorts, patients with untreated sAA (FIRST cohort) and in patients with sAA that has relapsed or is refractory to IST (NEXT cohort). In the FIRST cohort, participants receive IST (equine ATG and ciclosporin) plus avatrombopag from day 1 until day 180 at 60 mg oral daily, with dose adjusted according to platelet count. Participants in the NEXT cohort receive avatrombopag at 60 mg oral daily from day 1 until day 180, with or without additional IST at the discretion of the treating clinician.For each cohort, two primary endpoints (haematological response and acquired clonal evolution) are jointly monitored and the trial reviewed at each interim analysis where a 'go/no-go' decision is made by evaluating the posterior probability of the events of interests. ETHICS AND DISSEMINATION: The trial has received ethics approval (Monash Health RES-18-0000707A). The trial conduct will comply with ICH-GCP and all applicable regulatory requirements. The results of the trial will be submitted to a peer-review journal for publication. TRIAL REGISTRATION NUMBER: ACTRN12619001042134, ACTRN12619001043123.

Indexed as

Anemia, AplasticBenzoatesCyclosporineHydrazinesPyrazolesThiazolesThiophenesAnimalsAntilymphocyte SerumBayes TheoremClinical Trials, Phase II as TopicHorsesHumansImmunosuppression TherapyImmunosuppressive AgentsTreatment OutcomeAntilymphocyte SerumavatrombopagBenzoatesCyclosporineeltrombopagHydrazinesImmunosuppressive AgentsPyrazolesThiazolesThiophenesanaemiaclinical trialsafety

Identifiers

PMID38238183
PMCPMC10806710
OpenAlexW4391004756

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.