ArticleBone marrow transplantation2024
Safety and efficacy of narsoplimab in pediatric and adult patients with transplant-associated thrombotic microangiopathy: a real-world experience.
Article in Bone marrow transplantation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
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Who cites it
15 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Targeting the Roots of Kidney Disease: Systematic Review of the Therapies Targeting the Complement System.Medicina (Kaunas, Lithuania) · 2025Pooled it
- Causes and outcomes of pediatric thrombotic microangiopathy.Research and practice in thrombosis and haemostasis · 2026Article
- Structure and function of therapeutic antibodies approved by the US FDA in 2025.Antibody therapeutics · 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 KLF4-CD46 axis: a novel therapeutic target in transplant-associated thrombotic microangiopathy and beyond.Haematologica · 2026Article
- KLF4 overexpression protects against complementmediated endothelial injury in transplant-associated thrombotic microangiopathy.Haematologica · 2026Article
- 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
- Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA-TMA).American journal of hematology · 2025Article
- Narsoplimab for refractory transplantation-associated thrombotic microangiopathy (TA-TMA) in adult patients receiving allogeneic hematopoietic stem cell transplantation (AHSCT).Bone marrow transplantation · 2025Article
- Translating biomarker insights into practice: a path forward in TA-TMA management.Frontiers in medicine · 2025Article
- Complement-targeted therapeutics: Are we there yet, or just getting started?European journal of immunology · 2024Review
Corrections and comments
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Authors and funding
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Transplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication following hematopoietic stem cell transplantation (HSCT). No approved treatments are currently available. This study presents real-world data obtained with narsoplimab, a human immunoglobulin G4 monoclonal antibody that inhibits MASP-2, the effector enzyme of the lectin pathway of the complement system. Between January 2018 and August 2023, 20 (13 adult and 7 pediatric) patients diagnosed with TA-TMA received narsoplimab under an ongoing compassionate use program. The diagnosis was based on internationally defined criteria for pediatric and adult patients. Fifteen patients fulfilled the criteria recently established by an international consensus on TA-TMA. Nineteen patients exhibited high-risk characteristics. Thirteen patients (65%) responded to narsoplimab, achieving transfusion independence and significant clinical improvement. The one-hundred-day Overall Survival (OS) post-TA-TMA diagnosis was 70%, and 100% for responders. Narsoplimab proved to be effective and safe in the treatment of high-risk TA-TMA, with no increased infectious complications or other safety signals of concern across all age groups. The high response rates and the encouraging survival outcomes underscore the potential of narsoplimab as a valuable therapeutic option, particularly for high-risk cases.
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