Evidence map›Paper›PMID 30784097›Full record

Trial reportAmerican journal of hematology2019

Long-term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program.

James B Bussel, Donald M Arnold, Michael A Boxer, Nichola Cooper, Jiri Mayer, Hany Zayed, Sandra Tong, Anne-Marie Duliege

Abstract readClinical Trial, Phase IIIMulticenter StudyRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in American journal of hematology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 64 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
64citing papers in PubMed, 2 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.

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

64 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Guideline
  3. Trial
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  8. Article
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  18. Platelet Disorders and Medication Strategies.Thrombosis and haemostasis · 2026
    Review
  19. Review
  20. Targeting the Neonatal Fc Receptor in Autoimmune Diseases: Pipeline and Progress.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    Review

4 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

8 authors.

James B BusselDivision of Pediatric Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, New York.
Donald M ArnoldDepartment of Medicine, Michael G. DeGroote School of Medicine, McMaster University, and McMaster Centre for Transfusion Research, Hamilton Health Sciences, Hamilton, Ontario, Canada.
Michael A BoxerArizona Oncology Associates, Tucson, Arizona.
Nichola CooperImperial College Healthcare NHS Trust, Hammersmith Hospital, London, United Kingdom.
Jiri MayerFakultni nemocnice Brno, Brno, Czech Republic.
Hany ZayedRigel Pharmaceuticals Inc., South San Francisco, California.
Sandra TongRigel Pharmaceuticals Inc., South San Francisco, California.
Anne-Marie DuliegeRigel Pharmaceuticals Inc., South San Francisco, California.

Funding

Rigel Pharmaceuticals, Inc
6 · The paper itself

Abstract

Two randomized, double-blind, placebo-controlled studies demonstrated responses (≥50 000/μL) to fostamatinib in adults with long-standing immune thrombocytopenia (ITP). The long-term safety and efficacy of fostamatinib were evaluated in a follow-on, open-label extension (OLE) study. Patients received double-blind fostamatinib in the randomized trials, and responders continued the same dose, 100 to 150 mg BID, in the OLE study. Nonresponders received 100 mg BID for 4 weeks and could escalate to 150 mg BID at week 4. Endpoints included stable response, platelet count ≥50 000/μL at 4/6 biweekly (randomized trials) or 2/3 monthly visits (OLE), and overall response, ≥1 platelet count ≥50 000/μL during weeks 1 to 12. A total of 146 patients received fostamatinib including 123 in the OLE study. Median treatment duration was 6.7 months. Baseline median ITP duration was 8 years and median platelet count was 16 000/μL; prior treatments included thrombopoietic (TPO) agents (47%), splenectomy (35%), and rituximab (32%). Twenty-seven (18%) patients achieved a stable response with median duration of >28 months and a median platelet count of 89 000/μL. Sixty-four (44%) patients achieved an overall response (including stable responders) with a median platelet count of 63 000/μL and a median response duration of >28 months. Twenty-four of 71 (34%) patients who had failed TPO agents achieved overall responses to fostamatinib. The most common adverse events (AEs) were diarrhea, hypertension, nausea, epistaxis, and abnormal liver function tests. Most AEs were mild/moderate and resolved or were managed with dose reduction, dose interruption, and/or secondary medication. Almost half of the patients achieved an overall response, and most of these maintained their responses for >2 years. No new or increased frequency of AEs was seen at up to 31 months of treatment.

Indexed as

OxazinesPyridinesAdultAgedAged, 80 and overAminopyridinesDouble-Blind MethodFemaleHumansMaleMiddle AgedMorpholinesPlatelet CountPurpura, Thrombocytopenic, IdiopathicPyrimidinesTime FactorsAminopyridinesfostamatinibMorpholinesOxazinesPyridinesPyrimidines

Identifiers

PMID30784097
PMCPMC6594140

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.