ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2014
Eculizumab therapy in children with severe hematopoietic stem cell transplantation-associated thrombotic microangiopathy.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 99 papers, 3 of them syntheses that pooled it.
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Who cites it
99 citing papers in PubMed, 3 syntheses or guidelines pooled it, 236 citations in OpenAlex.
- Guidelines on the Use of Therapeutic Apheresis in Clinical Practice-Evidence-Based Approach From the Writing Committee of the American Society for Apheresis: The Tenth Special Issue.Journal of clinical apheresis · 2026Guideline
- Systematic Review of Signs and Symptoms Associated with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy.Transplantation and cellular therapy · 2023Pooled it
- Efficacy and Safety of Eculizumab in the Treatment of Transplant-Associated Thrombotic Microangiopathy: A Systematic Review and Meta-Analysis.Frontiers in immunology · 2020Pooled it
- Incidence, risk factors, and outcomes of transplant-associated thrombotic microangiopathy in pediatric patients after allogeneic hematopoietic cell transplantation: a single-institution prospective study.Bone marrow transplantation · 2025Trial
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- High-dose Carboplatin/Etoposide/Melphalan increases risk of thrombotic microangiopathy and organ injury after autologous stem cell transplantation in patients with neuroblastoma.Bone marrow transplantation · 2018Trial
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- KLF4 overexpression protects against complementmediated endothelial injury in transplant-associated thrombotic microangiopathy.Haematologica · 2026Article
- BK virus kinetics and associated cystitis/urethritis symptoms after PTCy-based allogeneic hematopoietic cell transplant.Blood advances · 2026Article
- Complement activation in secondary thrombotic microangiopathies.Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2025Review
- An ASTCT, CIBMTR, EBMT, and APBMT Consensus Statement Defining Response Criteria for Hematopoietic Cell Transplantation Associated Thrombotic Microangiopathy (TA-TMA) Directed Therapy.Transplantation and cellular therapy · 2025Article
- Navigating Hyperhemolysis in Sickle Cell Disease: Insights from Literature.Diagnostics (Basel, Switzerland) · 2025Review
- [Retrospective clinical analysis of eculizumab treatment for hematopoietic stem cell transplantation-associated thrombotic microangiopathy: a report of 11 cases].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Time course of organ and hematological response to complement blockage in transplant-associated thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation.Frontiers in medicine · 2025Article
- Pediatric transplant-associated thrombotic microangiopathy health care utilization and implications of eculizumab therapy.Blood advances · 2024Article
- Compassionate Use Narsoplimab for Severe Refractory Transplantation-Associated Thrombotic Microangiopathy in Children.Transplantation and cellular therapy · 2024Article
- Transplant-associated thrombotic microangiopathy in pediatrics: incidence, risk factors, therapeutic options, and outcome based on data from a single center.Frontiers in oncology · 2024Article
- Prevention and management of acute toxicities from conditioning regimens during hematopoietic stem cell transplantation.Clinical hematology international · 2024Review
- Effects ofBiomedicines · 2023Article
39 more citing papers are in PubMed but not listed here.
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Authors and funding
12 authors at 3 institutions in 2 countries.
Funding
Abstract
We recently observed that dysregulation of the complement system may be involved in the pathogenesis of hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA). These findings suggest that the complement inhibitor eculizumab could be a therapeutic option for this severe HSCT complication with high mortality. However, the efficacy of eculizumab in children with HSCT-TMA and its dosing requirements are not known. We treated 6 children with severe HSCT-TMA using eculizumab and adjusted the dose to achieve a therapeutic level >99 μg/mL. HSCT-TMA resolved over time in 4 of 6 children after achieving therapeutic eculizumab levels and complete complement blockade, as measured by low total hemolytic complement activity (CH50). To achieve therapeutic drug levels and a clinical response, children with HSCT-TMA required higher doses or more frequent eculizumab infusions than currently recommended for children with atypical hemolytic uremic syndrome. Two critically ill patients failed to reach therapeutic eculizumab levels, even after dose escalation, and subsequently died. Our data indicate that eculizumab may be a therapeutic option for HSCT-TMA, but HSCT patients appear to require higher medication dosing than recommended for other conditions. We also observed that a CH50 level ≤ 4 complement activity enzyme units correlated with therapeutic eculizumab levels and clinical response, and therefore CH50 may be useful to guide eculizumab dosing in HSCT patients as drug level monitoring is not readily available.
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