ReviewBritish journal of haematology2023
Novel therapeutics and future directions for refractory immune thrombocytopenia.
Review in British journal of haematology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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.
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.
Who cites it
19 citing papers in PubMed, 26 citations in OpenAlex.
- Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial.BMJ (Clinical research ed.) · 2025Trial
- Artificial intelligence prediction of therapy response in newly diagnosed adult severe primary immune thrombocytopenia.Research and practice in thrombosis and haemostasis · 2026Article
- Single low-dose daratumumab enhances rituximab efficacy for sustained remission in treatment-refractory autoimmune cytopenias.Annals of hematology · 2026Article
- Refractory Primary Immune Thrombocytopenia With Bleeding and Thrombosis: A Case Report.The American journal of case reports · 2026Article
- Case Report: Hematopoietic stem cell transplantation for refractory chronic immune thrombocytopenia in a child.Frontiers in immunology · 2026Review
- Emerging targeted therapies for primary immune thrombocytopenia: novel agents, combination strategies, and future directions.Frontiers in pharmacology · 2026Review
- Clinical characteristic of isolated thrombocytopenia in patients with bone marrow failure-related germline variants: a retrospective study from a single centre.Annals of medicine · 2025Article
- Primary versus Secondary Immune Thrombocytopenia (ITP): A Meeting Report from the 2023 McMaster ITP Summit.Thrombosis and haemostasis · 2025Article
- Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease.Lupus science & medicine · 2025Article
- Integrating QSAR modelling with reinforcement learning for Syk inhibitor discovery.Journal of cheminformatics · 2025Article
- Impact of Thrombopoietin Receptor Agonists on Pathophysiology of Pediatric Immune Thrombocytopenia.Current issues in molecular biology · 2025Review
- Combination Therapy with Romiplostim, Danazol, and a Thrombopoietin Receptor Agonist for Immune Restoration in Chronic Refractory Immune Thrombocytopenia: A Case Series.ImmunoTargets and therapy · 2025Article
- From C481 Resistance Evasion to Platelet Preservation: Rilzabrutinib Redefines ITP Targeted Therapy.Drug design, development and therapy · 2025Review
- Effective treatment with daratumumab in post-HSCT refractory immune-mediated cytopenias: a case report and literature review.Frontiers in immunology · 2025Review
- Current state and potential applications of neonatal Fc receptor (FcRn) inhibitors in hematologic conditions.American journal of hematology · 2024Review
- 2025 update on clinical trials in immune thrombocytopenia.American journal of hematology · 2024Review
- Treatment of Immune Thrombocytopenia: Contextualization from a Historical Perspective.Hematology reports · 2024Review
- Microbe-immune interactions: new perspectives on coagulation deficiencies, purpura, and other hemorrhagic conditions under the regulation of the gut microbiota.Frontiers in immunology · 2024Article
- Safety and Efficacy of Subcutaneous Daratumumab in Systemic AL Amyloidosis.Therapeutics and clinical risk management · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder affecting approximately 1 in 20 000 people. While most patients with ITP are successfully managed with the current set of standard and approved therapeutics, patients who cannot be adequately managed with these therapies, considered to have refractory ITP, are not uncommon. Therefore, there remains an ongoing need for novel therapeutics and drug development in ITP. Several agents exploiting novel targets and mechanisms in ITP are presently under clinical development, with trials primarily recruiting heavily pretreated patients and those with otherwise refractory disease. Such agents include the neonatal Fc receptor antagonist efgartigimod, the Bruton tyrosine kinase inhibitor rilzabrutinib, the complement inhibitors sutimlimab and iptacopan and anti-CD38 monoclonal antibodies such as daratumumab and mezagitamab, among others. Each of these agents exploits therapeutic targets or other aspects of ITP pathophysiology currently not targeted by the existing approved agents (thrombopoietin receptor agonists and fostamatinib). This manuscript offers an in-depth review of the current available data for novel therapeutics in ITP presently undergoing phase 2 or 3 studies in patients with heavily pretreated or refractory ITP. It additionally highlights the future directions for drug development in refractory ITP, including discussion of innovative clinical trial designs, health-related quality of life as an indispensable clinical trial end-point and balancing potential toxicities of drugs with their potential benefits in a bleeding disorder in which few patients suffer life-threatening bleeding.
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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.