Paroxysmal nocturnal haemoglobinuria
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DOI: 10.1038/nrdp.2017.29
Abstract: The progeny of PIGA‐mutated HSCs lack GPI‐anchored proteins, including complement‐ regulating factors that protect ‘self’ cells from the activated complement. Paroxysmal nocturnal haemoglobinuria (PNH) is a rare haematological disorder named after its characteristic episodes of… read more here.
Keywords: paroxysmal nocturnal; nocturnal haemoglobinuria;
The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors: clinical outcomes and medical encounters from the patient perspective
Sign Up to like & getrecommendations! Published in 2022 at "Hematology"
DOI: 10.1080/16078454.2022.2127630
Abstract: ABSTRACT Objectives: To assess the clinical and healthcare resource burden among C5 inhibitor (C5i)-treated patients with paroxysmal nocturnal haemoglobinuria (PNH), using patient-reported data. Methods: This web-based, cross-sectional survey (01FEB2021–31MAR2021) of adults with PNH treated with… read more here.
Keywords: patients paroxysmal; nocturnal haemoglobinuria; paroxysmal nocturnal; medical encounters ... See more keywords
Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome: 10‐year pharmacovigilance analysis
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DOI: 10.1111/bjh.15790
Abstract: Eculizumab is the first and only medication approved for paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) treatment. However, eculizumab safety based on long‐term pharmacovigilance is unknown. This analysis summarises safety data collected… read more here.
Keywords: nocturnal haemoglobinuria; uraemic syndrome; infection; atypical haemolytic ... See more keywords
How we('ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future.
Sign Up to like & getrecommendations! Published in 2021 at "British journal of haematology"
DOI: 10.1111/bjh.17753
Abstract: Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement-mediated intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients with bone marrow failure the treatment follows that of immune-mediated aplastic anaemia, that of classic, haemolytic… read more here.
Keywords: haemoglobinuria diving; nocturnal haemoglobinuria; paroxysmal nocturnal; diving future ... See more keywords
Fatigue and health-related quality of life in paroxysmal nocturnal haemoglobinuria: a post-hoc analysis of the pegcetacoplan PEGASUS trial data.
Sign Up to like & getrecommendations! Published in 2023 at "European journal of haematology"
DOI: 10.1111/ejh.13969
Abstract: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, non‐malignant haematological disorder associated with disabling fatigue and reduced health‐related quality of life. Post hoc analysis of PEGASUS phase 3 trial (NCT03500549) characterised improvements in patient‐reported fatigue measured… read more here.
Keywords: nocturnal haemoglobinuria; quality life; paroxysmal nocturnal; related quality ... See more keywords
Validation of a single tube 3‐colour immature red blood cell screening assay for the detection and enumeration of small, medium and large paroxysmal nocturnal haemoglobinuria clones by flow cytometry
Sign Up to like & getrecommendations! Published in 2022 at "International Journal of Laboratory Hematology"
DOI: 10.1111/ijlh.13877
Abstract: The reliable diagnosis of paroxysmal nocturnal haemoglobinuria (PNH) by flow cytometry is based on mandatory analysis of the erythroid, neutrophilic and monocytic lineages. In this study, we have evaluated the performance characteristics of a recently… read more here.
Keywords: nocturnal haemoglobinuria; immature red; flow cytometry; red blood ... See more keywords
Pregnancy outcomes in women receiving eculizumab for the management of paroxysmal nocturnal haemoglobinuria
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DOI: 10.1177/1753495x211019899
Abstract: Aims To report pregnancy outcomes and complications in women receiving eculizumab for the management of paroxysmal nocturnal haemoglobinuria. Methods A service evaluation of routinely collected medical records across 49 pregnancies in 21 women. Results Eculizumab… read more here.
Keywords: nocturnal haemoglobinuria; paroxysmal nocturnal; women receiving; pregnancy outcomes ... See more keywords