ArticleInternational journal of hematology2011
Chronic treatment of paroxysmal nocturnal hemoglobinuria patients with eculizumab: safety, efficacy, and unexpected laboratory phenomena.
Article in International journal of hematology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 48 citations in OpenAlex.
- A Retrospective Claims Analysis of the Rate of Complications in Patients Undergoing Treatment for Paroxysmal Nocturnal Hemoglobinuria.Advances in therapy · 2025Article
- Effect of Eculizumab on Iron Metabolism in Transfusion-independent Patients With Paroxysmal Nocturnal Hemoglobinuria.HemaSphere · 2023Article
- No evidence for hypogammaglobulinemia in patients with paroxysmal nocturnal hemoglobinuria (PNH) chronically treated with ravulizumab.PloS one · 2020Article
- Pharmacology, Pharmacokinetics and Pharmacodynamics of Eculizumab, and Possibilities for an Individualized Approach to Eculizumab.Clinical pharmacokinetics · 2019Review
- Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT.Frontiers in immunology · 2019Review
- Low-dose prednisolone in patients with paroxysmal nocturnal hemoglobinuria and inadequate response to eculizumab.Blood research · 2017Article
- Eculizumab treatment: stochastic occurrence of C3 binding to individual PNH erythrocytes.Journal of hematology & oncology · 2017Article
- Interim analysis of post-marketing surveillance of eculizumab for paroxysmal nocturnal hemoglobinuria in Japan.International journal of hematology · 2016Article
- Complement-mediated haemolysis and the role of blood transfusion in paroxysmal nocturnal haemoglobinuria.Blood transfusion = Trasfusione del sangue · 2015Review
- Platelets in inflammation and atherogenesis.Frontiers in immunology · 2015Review
- Resolution of ocular involvement with systemic eculizumab therapy in atypical hemolytic-uremic syndrome.Eye (London, England) · 2013Article
- Complement in immune and inflammatory disorders: therapeutic interventions.Journal of immunology (Baltimore, Md. : 1950) · 2013Review
- Successful anticoagulant therapy for two pregnant PNH patients, and prospects for the eculizumab era.International journal of hematology · 2013Review
- Progress and Trends in Complement Therapeutics.Advances in experimental medicine and biology · 2013Article
- Eculizumab in atypical haemolytic-uraemic syndrome allows cessation of plasma exchange and dialysis.Clinical kidney journal · 2012Article
- Review
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The terminal complement inhibitor eculizumab has become the standard of treatment in patients with symptomatic paroxysmal nocturnal hemoglobinuria (PNH). In this retrospective study, 19 PNH patients received chronic therapy with eculizumab with a median duration of 16 months (range 6-46 months). Parameters of hemolysis, transfusion requirements, and serum iron parameters were analyzed. Lactate dehydrogenase levels were significantly decreased by 85% from a median of 1897 U/l (range 293-3360) to 283 U/l (range 143-667), with an 86% reduction of transfusion requirements, whereas other parameters of hemolysis remained abnormal. Six patients (31.6%) became completely transfusion independent. A significant increase in ferritin levels from a median of 104 μg/l before to a median of 528 μg/l (p = 0.011) during treatment with eculizumab was observed. This was more pronounced in patients with low reticulocyte production index and/or requiring blood transfusions. Monospecific direct Coombs test was positive in most PNH patients, indicating a shift to extravascular hemolysis. Positive immunofixation for IgG kappa was observed, due to the presence of eculizumab in the serum. Eculizumab was safe and well tolerated long term in our study population. Iron should not be routinely supplemented in PNH patients treated with eculizumab without close monitoring of iron parameters, and iron depletion therapy should be considered in the case of overload.
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