Eculizumab for treating patients with paroxysmal nocturnal hemoglobinuria

Martí‐Carvajal, Arturo J; Anand, Vidhu; Cardona, Andrés Felipe; Solà, Iván

doi:10.1002/14651858.cd010340

Cited by 9 publications

(7 citation statements)

References 113 publications

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“…[8] It has been shown to also reduce the risk of further thrombosis in open-label studies [10] and in case reports, but not in a controlled trial. [19] Eculizumab blocks terminal complement pathway, which appears to be 1 of the key factors of thrombosis in PNH. Consequently, eculizumab is recommended in PNH patients not only in the presence of symptomatic hemolysis but also in cases with thrombosis, even if there are no prospective data concerning this complication.…”

Section: Discussionmentioning

confidence: 99%

Atypical presentation of paroxysmal nocturnal hemoglobinuria treated by eculizumab

et al. 2017

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Rationale:Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant acquired hematopoietic stem cell disease, which can be revealed by hemolytic anemia, thromboembolism, or bonemarrow failure. Thrombosis can occur at any site, but coronary thrombosis is extremely rare. Controlled trials have demonstrated that eculizimab, an inhibitor of the terminal complement cascade, was able to reduce both hemolysis and thrombosis, but its efficacy in cases of PNH with coronary thrombosis is unknown.Patient concerns and diagnoses:We report herein the unusual case of a 73-year-old patient presenting with recurrent coronary syndromes without associated stenosis, fever, marked inflammatory syndrome, and anemia, leading to a delayed diagnosis of PNH.Intervention and outcomes:Eculizumab allowed the resolution of fever and inflammation, and prevented further thromboembolism.Lessons:This case emphasizes the importance of performing aflow cytometry test for PNH in front of unusual or unexplained recurrent thromboses. Thromboses, as observed in our case, may be associated with fever and marked inflammation. This case also provides useful information on eculizumab ability to prevent further thromboembolism in PNH patients with a medical history of arterial thrombosis.

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Section: Discussionmentioning

confidence: 99%

Atypical presentation of paroxysmal nocturnal hemoglobinuria treated by eculizumab

et al. 2017

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“…The first studies on its use have shown encouraging results with decreased need for blood transfusions, reduced rates of thrombotic events and marked improvement on quality of life 12 13. Nevertheless, prospective studies are lacking and a recent Cochrane systematic review concluded that its prescription can neither be supported nor rejected 14. Furthermore, it requires indefinite administration for a sustained response, making it a very costly treatment, besides not being readily available in most centres.…”

Section: Discussionmentioning

confidence: 99%

Recurrent cerebral ischaemic events in the setting of paroxysmal nocturnal haemoglobinuria

et al. 2016

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The authors present a case of a 28-year-old man with a known history of paroxysmal nocturnal haemoglobinuria (PNH), under platelet antiaggregation, admitted following recurrent transitory arterial ischaemic attacks. Concomitant thrombosis of the superior mesenteric artery and bilateral renal infarction was found. Cardioembolism, namely patent foramen ovale, was excluded and anticoagulation added, with no further events on 10-month follow-up. PNH is a rare acquired disorder of haematopoietic stem cells, characterised by haemolytic anaemia, pancytopenia and thrombotic events classically involving the venous system. Reports of cerebral artery stroke and accompanying intra-abdominal arterial thrombosis are especially rare. Complementary investigation and treatment options in these patients are discussed.

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“…The reviews covered all types of research questions: three articles reviewed diagnostic methods for investigating fatigue by doing a validation or appraisal of outcome measures for a specific rare disease (lysosomal storage diseases, myotonic dystrophy type 1, and pyruvate kinase deficiency) [45][46][47]. Two articles reviewed intervention effects on fatigue in disease-specific medication interventions, (in paroxysmal nocturnal hemoglobinuria and peripheral neuropathy, including the rare disease Charcot Marie-Tooth) [12,48]. Two articles reviewing literature on the burden of living with Pompe disease gave some data on prevalence of fatigue and associated factors [49,50].…”

Section: Secondary Research Articles Characteristics and Investigated...mentioning

confidence: 99%

Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research

Bathen

Johansen

Strømme

et al. 2022

Orphanet J Rare Dis

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Background Experienced fatigue is an under-recognized and under-researched feature in persons with many different rare diseases. A better overview of the characteristics of existing research on experienced fatigue in children and adults with rare diseases is needed. The purpose of this review was to map and describe characteristics of existing research on experienced fatigue in a selection of rare diseases in rare developmental defects or anomalies during embryogenesis and rare genetic diseases. Furthermore, to identify research gaps and point to research agendas. Methods We applied a scoping review methodology, and performed a systematic search in March 2020 in bibliographic databases. References were sorted and evaluated for inclusion using EndNote and Rayyan. Data were extracted on the main research questions concerning characteristics of research on experienced fatigue (definition and focus on fatigue, study populations, research questions investigated and methods used). Results This review included 215 articles on ten different rare developmental defects/anomalies during embryogenesis and 35 rare genetic diseases. Of the 215 articles, 82 had investigation of experienced fatigue as primary aim or outcome. Included were 9 secondary research articles (reviews) and 206 primary research articles. A minority of articles included children. There were large differences in the number of studies in different diseases. Only 29 of 215 articles gave a description of how they defined the concept of experienced fatigue. The most common research-question reported on was prevalence and/ -or associations to fatigue. The least common was diagnostics (development or validation of fatigue assessment methods for a specific patient group). A large variety of methods were used to investigate experienced fatigue, impeding comparisons both within and across diagnoses. Conclusion This scoping review on the characteristics of fatigue research in rare diseases found a large variety of research on experienced fatigue. However, the minority of studies had investigation of experienced fatigue as a primary aim. There was large variation in how experienced fatigue was defined and also in how it was measured, both within and across diagnoses. More research on experienced fatigue is needed, both in children and adults with rare diseases. This review offers a basis for further research.

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Eculizumab for treating patients with paroxysmal nocturnal hemoglobinuria

Cited by 9 publications

References 113 publications

Atypical presentation of paroxysmal nocturnal hemoglobinuria treated by eculizumab

Atypical presentation of paroxysmal nocturnal hemoglobinuria treated by eculizumab

Recurrent cerebral ischaemic events in the setting of paroxysmal nocturnal haemoglobinuria

Experienced fatigue in people with rare disorders: a scoping review on characteristics of existing research

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