Topic
Hemoglobinuria
About: Hemoglobinuria is a research topic. Over the lifetime, 1125 publications have been published within this topic receiving 28268 citations. The topic is also known as: presence of wbc.
Papers published on a yearly basis
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Papers
Leeds General Infirmary1, Saarland University2, Johns Hopkins University3, University of Paris4, Radboud University Nijmegen5, University of Duisburg-Essen6, Royal Melbourne Hospital7, St George's Hospital8, City of Hope National Medical Center9, Trinity College, Dublin10, University of Naples Federico II11, University of Ulm12, Cleveland Clinic13, Case Western Reserve University14, Alexion Pharmaceuticals15
TL;DR: Eculizumab is an effective therapy for PNH and Clinically significant improvements were found in the quality of life, as measured by scores on the Functional Assessment of Chronic Illness Therapy-Fatigue instrument and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire.
Abstract: BACKGROUND We tested the safety and efficacy of eculizumab, a humanized monoclonal antibody against terminal complement protein C5 that inhibits terminal complement activation, in patients with paroxysmal nocturnal hemoglobinuria (PNH). METHODS We conducted a double-blind, randomized, placebo-controlled, multicenter, phase 3 trial. Patients received either placebo or eculizumab intravenously; eculizumab was given at a dose of 600 mg weekly for 4 weeks, followed 1 week later by a 900-mg dose and then 900 mg every other week through week 26. The two primary end points were the stabilization of hemoglobin levels and the number of units of packed red cells transfused. Biochemical indicators of intravascular hemolysis and the patients' quality of life were also assessed. RESULTS Eighty-seven patients underwent randomization. Stabilization of hemoglobin levels in the absence of transfusions was achieved in 49% (21 of 43) of the patients assigned to eculizumab and none (0 of 44) of those assigned to placebo (P<0.001). During the study, a median of 0 units of packed red cells was administered in the eculizumab group, as compared with 10 units in the placebo group (P<0.001). Eculizumab reduced intravascular hemolysis, as shown by the 85.8% lower median area under the curve for lactate dehydrogenase plotted against time (in days) in the eculizumab group, as compared with the placebo group (58,587 vs. 411,822 U per liter; P<0.001). Clinically significant improvements were also found in the quality of life, as measured by scores on the Functional Assessment of Chronic Illness Therapy-Fatigue instrument (P<0.001) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire. Of the 87 patients, 4 in the eculizumab group and 9 in the placebo group had serious adverse events, none of which were considered to be treatment-related; all these patients recovered without sequelae. CONCLUSIONS Eculizumab is an effective therapy for PNH.
1,248 citations
TL;DR: PNH is a chronic disorder that curtails life, and a spontaneous long-term remission can occur, which must be taken into account when considering potentially dangerous treatments, such as bone marrow transplantation.
Abstract: BackgroundParoxysmal nocturnal hemoglobinuria (PNH), which is characterized by intravascular hemolysis and venous thrombosis, is an acquired clonal disorder associated with a somatic mutation in a totipotent hematopoietic stem cell. An understanding of the natural history of PNH is essential to improve therapy. MethodsWe have followed a group of 80 consecutive patients with PNH who were referred to Hammersmith Hospital, London, between 1940 and 1970. They were treated with supportive measures, such as oral anticoagulant therapy after established thromboses, and transfusions. ResultsThe median age of the patients at the time of diagnosis was 42 years (range, 16 to 75), and the median survival after diagnosis was 10 years, with 22 patients (28 percent) surviving for 25 years. Sixty patients have died; 28 of the 48 patients for whom the cause of death is known died from either venous thrombosis or hemorrhage. Thirty-one patients (39 percent) had one or more episodes of venous thrombosis during their illness....
869 citations
TL;DR: The primary clinical manifestations of paroxysmal nocturnal hemoglobinuria are hemolytic anemia, marrow failure, and thrombophilia.
782 citations
TL;DR: This antibody against terminal complement protein C5 reduces intravascular hemolysis, hemoglobinuria, and the need for transfusion, with an associated improvement in the quality of life in patients with PNH.
Abstract: background Paroxysmal nocturnal hemoglobinuria (PNH) arises from a somatic mutation of the PIG-A gene in a hematopoietic stem cell and the subsequent production of blood cells with a deficiency of surface proteins that protect the cells against attack by the complement system. We tested the clinical efficacy of eculizumab, a humanized antibody that inhibits the activation of terminal complement components, in patients with PNH. methods Eleven transfusion-dependent patients with PNH received infusions of eculizumab (600 mg) every week for four weeks, followed one week later by a 900-mg dose and then by 900 mg every other week through week 12. Clinical and biochemical indicators of hemolysis were measured throughout the trial. results Mean lactate dehydrogenase levels decreased from 3111 IU per liter before treatment to 594 IU per liter during treatment (P=0.002). The mean percentage of PNH type III erythrocytes increased from 36.7 percent of the total erythrocyte population to 59.2 percent (P = 0.005). The mean and median transfusion rates decreased from 2.1 and 1.8 units per patient per month to 0.6 and 0.0 units per patient per month, respectively (P=0.003 for the comparison of the median rates). Episodes of hemoglobinuria were reduced by 96 percent (P<0.001), and measurements of the quality of life improved significantly. conclusions Eculizumab is safe and well tolerated in patients with PNH. This antibody against terminal complement protein C5 reduces intravascular hemolysis, hemoglobinuria, and the need for transfusion, with an associated improvement in the quality of life in patients with PNH.
690 citations
TL;DR: Bone marrow transplantation remains the only cure for PNH but should be reserved for patients with suboptimal response to eculizumab, a first-in-class monoclonal antibody that inhibits terminal complement.
529 citations
Related Topics (5)
Performance Metrics
| Year | Papers |
|---|---|
| 2025 | 22 |
| 2024 | 51 |
| 2023 | 48 |
| 2022 | 87 |
| 2021 | 24 |
| 2020 | 30 |