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  1. A retrospective study of paroxysmal nocturnal hemoglobinuria in pediatric and adolescent patients

    ... measures, four patients with classic PNH received eculizumab whereas four patients with PNH/BMD underwent hematopoietic stem ...

    Research Article last updated 05/02/2017 - 10:42am.

  2. Doctor Helps Determine Her Own Aplastic Anemia and PNH Diagnosis

    ... diagnosis. But after my diagnosis, I was placed on eculizumab (Soliris®) in a twice-monthly regimen. Although I remain ...

    Patient Chronicle last updated 04/26/2018 - 10:36am.

  3. Small-molecule Factor D inhibitors selectively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome

    ... alternative pathway of complement that are treated with eculizumab , a humanized monoclonal antibody against the terminal complement component C5. Eculizumab must be administered intravenously, and moreover some patients with ...

    Research Article last updated 11/28/2016 - 11:42am.

  4. Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician's guide

    ... . Patients with PNH prior to the availability of eculizumab had a median survival of between 10 and 22 years, with ...

    Research Article last updated 03/22/2017 - 2:43pm.

  5. Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK

    ... Kingdom were the first to treat patients with PNH with eculizumab , and were therefore able to report on long term results. This ... high rate of blood clotting ( thrombosis ) and that eculizumab reduces the chances of this complication by a factor of about 10. ...

    Research Review last updated 05/02/2016 - 9:24am.

  6. Genetic Polymorphism of the Complement Receptor-1 (CR1) Gene Correlates with the Clinical Response to Eculizumab of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

    ... factors that influence how patients will respond to eculizumab . As way of background, some patients with PNH who go on Eculizumab have an increase in their hemoglobin up to the normal range ...

    Research Review last updated 05/02/2016 - 9:25am.

  7. A Rare Genetic Polymorphism in C5 Confers Poor Response to the Anti-C5 Monoclonal Antibody Eculizumab by Nine Japanese Patients with PNH

    ... who seem to have absolutely no benefit at all from eculizumab . Eculizumab binds to and inactivates the C5 complement protein and protects the ...

    Research Review last updated 05/02/2016 - 9:25am.

  8. Improved Fatigue and Quality-of-Life in Patients with Paroxysmal Nocturnal Hemoglobinuria During Treatment with Eculizumab: Data from the Global PNH Registry

    ... PNH have a major impact on patients’ quality of life. Eculizumab (Soliris®) can prevent hemolysis in people with PNH. The ... age 41) in the PNH Registry who had been treated with eculizumab for 12 months. The patients completed questionnaires to assess their ...

    Research Review last updated 05/02/2016 - 9:21am.

  9. Allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: a retrospective single-center study

    ... available for PNH, such as supportive care , eculizumab , and hematopoietic stem cell transplantation (HSCT); only the ...

    Research Article last updated 11/02/2016 - 10:41am.

  10. Paroxysmal Nocturnal Hemoglobinuria: The Future Of Complement-Based Therapies

    ... in the clinic, the first complement inhibitor eculizumab has changed the paradigm of PNH treatment. However, a ten-year ... likely they parallel the safety and efficacy profile of eculizumab. However, there is a growing interest for agents which target early ...

    Research Article last updated 08/15/2016 - 9:11am.