Trial reportJournal of clinical oncology : official journal of the American Society of Clinical Oncology2022
Narsoplimab, a Mannan-Binding Lectin-Associated Serine Protease-2 Inhibitor, for the Treatment of Adult Hematopoietic Stem-Cell Transplantation-Associated Thrombotic Microangiopathy.
Trial report in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02222545 (A Phase 2, Uncontrolled, Three-Stage, Dose-Escalation Cohort Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Clinical Activity of OMS721 in Adults With Thrombotic Microangiopathies), which is not on this map. Cited by 60 papers, 2 of them syntheses that pooled it.
What it found
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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.
A Phase 2, Uncontrolled, Three-Stage, Dose-Escalation Cohort Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Clinical Activity of OMS721 in Adults With Thrombotic Microangiopathies
Who cites it
60 citing papers in PubMed, 2 syntheses or guidelines pooled it, 90 citations in OpenAlex.
- Targeting the Roots of Kidney Disease: Systematic Review of the Therapies Targeting the Complement System.Medicina (Kaunas, Lithuania) · 2025Pooled it
- A systematic review of diagnostic, prognostic, and risk blood and urine biomarkers of transplant-associated thrombotic microangiopathy.Frontiers in immunology · 2022Pooled it
- Randomized phase I trial outcomes show safe and sustainable inhibition of classical and lectin complement pathways by empasiprubart.Nature communications · 2025Trial
- Narsoplimab in critically ill patients with high-risk transplant-associated thrombotic microangiopathy requiring intensive care.Bone marrow transplantation · 2026Article
- Complement Inhibition in the Clinic: Are We Doing Enough to Protect Patients From Infection?European journal of immunology · 2026Review
- Structure and function of therapeutic antibodies approved by the US FDA in 2025.Antibody therapeutics · 2026Review
- Narsoplimab: First Approval.Drugs · 2026Review
- Shifting From Systemic to Precision-Targeted Complement Therapies: Opportunities and Hurdles.European journal of immunology · 2026Review
- Complement and transplant-associated thrombotic microangiopathy: Current and future approaches.HemaSphere · 2026Article
- Outcomes of Terminal Complement Blockade in Adults with High-Risk Transplant-Associated Thrombotic Microangiopathy: A Comparative Analysis.Transplantation and cellular therapy · 2026Article
- The resistance to Toxoplasma gondii in Microtus fortis is associated with the activation of the complement lectin pathway.PLoS neglected tropical diseases · 2026Article
- Advances in the lectin pathway in systemic lupus erythematosus: from clinical correlations and mechanisms to targeted interventions.Inflammation research : official journal of the European Histamine Research Society ... [et al.] · 2026Review
- Use of narsoplimab for eculizumab-refractory adult transplant-associated thrombotic microangiopathy (TA-TMA).Annals of hematology · 2026Article
- Targeting the lectin pathway in TA-TMA.Blood advances · 2026Article
- Survival in adults with high-risk TA-TMA: a comparative analysis of narsoplimab vs supportive care.Blood advances · 2026Article
- Efficacy analysis of iptacopan in a patient with thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: a case report.Frontiers in immunology · 2026Article
- Current standards and novel concepts in thrombotic thrombocytopenic purpura.Frontiers in medicine · 2026Review
- New Anticomplement Drugs in Nephrology: Mechanism and Indication.Kidney & blood pressure research · 2026Review
- Complement-targeted therapies for transplant-associated thrombotic microangiopathy: recent advances.Frontiers in pharmacology · 2026Review
- Update in the diagnosis of complement-mediated thrombotic microangiopathy/atypical hemolytic uremic syndrome.Hematology. American Society of Hematology. Education Program · 2025Review
Corrections and comments
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Authors and funding
13 authors at 9 institutions in 7 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeHematopoietic stem-cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) is a serious complication with significant mortality and no approved therapy. HSCT-TMA results from endothelial injury, which activates the lectin pathway of complement. Narsoplimab (OMS721), an inhibitor of mannan-binding lectin-associated serine protease-2 (MASP-2), was evaluated for safety and efficacy in adults with HSCT-TMA.
methodsIn this single-arm open-label pivotal trial (NCT02222545), patients received intravenous narsoplimab once weekly for 4-8 weeks. The primary end point (response rate) required clinical improvement in two categories: (1) laboratory TMA markers (both platelet count and lactate dehydrogenase) and (2) organ function or freedom from transfusion. Patients receiving at least one dose (full analysis set [FAS]; N = 28) were analyzed.
resultsThe response rate was 61% in the FAS population. Similar responses were observed across all patient subgroups defined by baseline features, HSCT characteristics, and HSCT complications. Improvement in organ function occurred in 74% of patients in the FAS population. One-hundred-day survival after HSCT-TMA diagnosis was 68% and 94% in FAS population and responders, respectively, whereas median overall survival was 274 days in the FAS population. Narsoplimab was well tolerated, and adverse events were typical of this population, with no apparent safety signal of concern.
conclusionIn this study, narsoplimab treatment was safe, significantly improved laboratory TMA markers, and resulted in clinical response and favorable overall survival.
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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.