Paroxysmal nocturnal hemoglobinuria: Where we stand
Sign Up to like & getrecommendations! Published in 2023 at "American Journal of Hematology"
DOI: 10.1002/ajh.26832
Abstract: For the last 20 years, therapy of paroxysmal nocturnal hemoglobinuria (PNH) relied—up until recently—on antibody based terminal complement inhibitionon. PNH pathophysiology—a mutational defect leading to partial or complete absence of complement‐regulatory proteins on blood cells—leads to… read more here.
Keywords: hemoglobinuria stand; complement; nocturnal hemoglobinuria; paroxysmal nocturnal ... See more keywords
Paroxysmal nocturnal hemoglobinuria: Where are we going
Sign Up to like & getrecommendations! Published in 2023 at "American Journal of Hematology"
DOI: 10.1002/ajh.26882
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare nonmalignant clonal hematological disorder that is characterized by a deficiency of the GPI‐linked complement regulators on the membrane of hematopoietic cells, which renders them susceptible to complement‐mediated damage.… read more here.
Keywords: hemoglobinuria going; complement; nocturnal hemoglobinuria; paroxysmal nocturnal ... See more keywords
Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry
Sign Up to like & getrecommendations! Published in 2020 at "Annals of Hematology"
DOI: 10.1007/s00277-020-04052-z
Abstract: The International Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry (NCT01374360) was initiated to optimize patient management by collecting data regarding disease burden, progression, and clinical outcomes. Herein, we report updated baseline demographics, clinical characteristics, disease burden data,… read more here.
Keywords: pnh; disease burden; clone size; clone ... See more keywords
Screening for paroxysmal nocturnal hemoglobinuria (PNH) in patients presenting with cerebral sinovenous thrombosis (CSVT): Results of a FLAER based flowcytometry study in Indian patients
Sign Up to like & getrecommendations! Published in 2019 at "Journal of Thrombosis and Thrombolysis"
DOI: 10.1007/s11239-019-01982-y
Abstract: Patients with paroxysmal nocturnal hemoglobinuria (PNH) may present with thrombosis at unusual sites, of which cerebral sinovenous thrombosis (CSVT) is one and screening for PNH is recommended in this condition. Though many patients diagnosed with… read more here.
Keywords: flowcytometry; pnh; paroxysmal nocturnal; nocturnal hemoglobinuria ... See more keywords
A Set of Active Promoters with Different Activity Profiles for Superexpressing Rhodococcus Strain.
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DOI: 10.1021/acssynbio.0c00508
Abstract: Rhodococcus bacteria are a promising platform for biodegradation, biocatalysis, and biosynthesis, but the use of rhodococci is hampered by the insufficient number of both platform strains for expression and promoters that are functional and thoroughly… read more here.
Keywords: pnh; activity profiles; strain; growth ... See more keywords
Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia
Sign Up to like & getrecommendations! Published in 2021 at "Leukemia"
DOI: 10.1038/s41375-021-01190-9
Abstract: In this large single-centre study, we report high prevalence (25%) of, small ( read more here.
Keywords: aplastic anemia; pnh; paroxysmal nocturnal; small paroxysmal ... See more keywords
Clinical characteristics of 512 eculizumab-naive paroxysmal nocturnal hemoglobinuria patients in China: a single-center observational study
Sign Up to like & getrecommendations! Published in 2022 at "Hematology"
DOI: 10.1080/16078454.2021.2022849
Abstract: ABSTRACT Objectives With large patient population and complement inhibitors naïve background, the characteristics patients with paroxysmal nocturnal hemoglobinuria (PNH) in China have not been well studied, especially for different subtypes. Methods We retrospectively reviewed patients… read more here.
Keywords: classic pnh; nocturnal hemoglobinuria; bmf pnh; paroxysmal nocturnal ... See more keywords
P1845HISTORY AND FUTURE PERSPECTIVES OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
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DOI: 10.1093/ndt/gfaa142.p1845
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disorder that affects about 1-1,5 cases per million individuals, characterized by hemolysis, peripheral blood cytopenia, bone marrow dysfunction, thrombosis, renal impairment and arterial and pulmonary hypertension. The… read more here.
Keywords: pnh; future perspectives; paroxysmal nocturnal; nocturnal hemoglobinuria ... See more keywords
Autoimmune neuromyotonia
Sign Up to like & getrecommendations! Published in 2022 at "Current Opinion in Neurology"
DOI: 10.1097/wco.0000000000001104
Abstract: Purpose of review Autoimmune neuromyotonia encompasses a group of rare immune-mediated neurological disorders frequently associated with anti-contactin-associated protein-like 2 (CASPR2) antibodies and featuring clinical and electrical signs of peripheral nerve hyperexcitability (PNH). We aim to… read more here.
Keywords: anti caspr2; autoimmune neuromyotonia; caspr2 antibodies; autoimmune ... See more keywords
Concomitant Immunosuppressive Therapy and Eculizumab Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Analysis
Sign Up to like & getrecommendations! Published in 2022 at "Acta Haematologica"
DOI: 10.1159/000526979
Abstract: Introduction: Complement C5 inhibitor eculizumab is the first approved treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder caused by uncontrolled terminal complement activation. Approximately 50% of patients with aplastic anemia (AA) have PNH… read more here.
Keywords: ist ecu; registry; ecu ist; paroxysmal nocturnal ... See more keywords
Iron Overload in Patients with Paroxysmal Nocturnal Hemoglobinuria
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DOI: 10.1182/blood-2018-99-114654
Abstract: Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal bone marrow disorder caused by a somatic mutation in PIGA that leads to a marked deficiency or absence of the complement regulatory proteins CD55 and CD59.… read more here.
Keywords: iron metabolism; pnh; pnh clone; iron overload ... See more keywords