ArticleFrontiers in medicine2020
Sirolimus as Rescue Therapy for Refractory/Relapsed Immune Thrombocytopenia: Results of a Single-Center, Prospective, Single-Arm Study.
Article in Frontiers in medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.
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Who cites it
24 citing papers in PubMed, 1 synthesis or guideline pooled it, 39 citations in OpenAlex.
- The Addition of Sirolimus to GVHD Prophylaxis After Allogeneic Hematopoietic Stem Cell Transplantation: A Meta-Analysis of Efficacy and Safety.Frontiers in oncology · 2021Pooled it
- Platelet Autophagy as a Druggable Intracellular Pathway: Therapeutic Opportunities in Thromboinflammatory Diseases.Pharmaceutics · 2026Review
- Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study.European journal of medical research · 2025Article
- Targeting the CD74 signaling axis suppresses inflammation and rescues defective hematopoiesis inScience translational medicine · 2025Article
- Effect of sirolimus on connective tissue disease related refractory thrombocytopenia: clinical efficacy and immunomodulatory mechanism.Postepy dermatologii i alergologii · 2025Article
- Dynamic changes in platelet counts and psychological state in ITP patients after COVID-19 infection.Frontiers in medicine · 2025Article
- [Chinese expert consensus on the diagnosis and treatment of chronic graft-versus-host disease (2024)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2024Article
- Association of thrombocytopenia with immune checkpoint inhibitors: a large-scale pharmacovigilance analysis based on the data from FDA adverse event reporting system database.Frontiers in pharmacology · 2024Article
- The role of T cells and myeloid-derived suppressor cells in refractory immune thrombocytopenia.British journal of haematology · 2023Review
- The mTORC1 pathway participate in hyper-function of B cells in immune thrombocytopenia.Annals of hematology · 2023Article
- Sirolimus is effective for primary refractory/relapsed warm autoimmune haemolytic anaemia/Evans syndrome: a retrospective single-center study.Annals of medicine · 2023Article
- Older Adults and Immune Thrombocytopenia: Considerations for the Clinician.Clinical interventions in aging · 2023Review
- Delayed spontaneous remission of acquired factor V inhibitor refractory to immunosuppressive therapy with pregnancy-associated improvement.Pathology oncology research : POR · 2023Article
- Rituximab resistance in ITP and beyond.Frontiers in immunology · 2023Review
- Clinical characteristics in immune thrombocytopenia patients after COVID-19 vaccination.Human vaccines & immunotherapeutics · 2022Article
- Immune thrombocytopenia in a small cell lung cancer patient treated with atezolizumab: a case report.Translational lung cancer research · 2022Article
- Article
- Refractory immune thrombocytopenia: Lessons from immune dysregulation disorders.Frontiers in medicine · 2022Article
- Current therapeutic strategies and perspectives in refractory ITP: What have we learned recently?Frontiers in immunology · 2022Review
- Advances in Immunosuppressive Agents Based on Signal Pathway.Frontiers in pharmacology · 2022Review
Corrections and comments
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Authors and funding
20 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Immune thrombocytopenia (ITP) is an autoimmune disease which arises due to self-destruction of circulating platelets. Failure to respond or maintain a response to first-line treatment can lead to refractory/relapsed (R/R) ITP. The mechanism remains complicated and lacks a standard clinical treatment. Sirolimus (SRL) is a mammalian target of rapamycin (mTOR) inhibitor that has been demonstrated to inhibit lymphocyte activity, indicating potential for SRL in treatment of ITP. Activation of the mTOR pathway in autoimmune diseases suggests that SRL might be a useful agent for treating ITP. Accordingly, we initiated an open-label, prospective clinical trial using SRL for patients with R/R ITP (ChiCTR-ONC-17012126). The trial enrolled 86 patients, each dosed with 2-4 mg/day of SRL. By the third month, 40% of patients (34 of 86) achieved complete remission (CR) and 45% of patients (39 of 86) achieved partial remission (PR), whereby establishing an overall response rate (ORR) of 85%. By 6 months of treatment, 41% of patients (32 of 78) achieved CR and 29% of patients (23 of 78) achieved PR, establishing an ORR of 70% without serious side effects. After 12 months follow-up, the ORR remained at 65%. We also found that SRL treatment exhibited higher efficacy in achieving CR in ITP patients who were younger than 40 years old or steroid dependent by univariate analysis. Importantly, in patients who responded, SRL treatment was associated with a reduction in the percentage of Th2, Th17 cells, and increase in the percentage of M-MDSCs and Tregs, indicating that SRL may reestablish peripheral tolerance. Taken together, Sirolimus demonstrated efficacy as a second-line agent for R/R ITP.
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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.