Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry
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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
Leveraging Whole Genome Sequencing to Define the Mutational Landscape in Paroxysmal Nocturnal Hemoglobinuria
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DOI: 10.1182/blood-2020-139335
Abstract: Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a prototypic disorder of hematopoietic stem cells leading to recurrent complement-mediated intravascular hemolysis. The development of PNH has been linked to somatic mutations in the PIGA gene, coding for… read more here.
Keywords: clone size; pathogenesis; pnh; piga ... See more keywords
Impact of Clonal Size and High Disease Activity on Clinical Outcomes of Paroxysmal Nocturnal Hemoglobinuria: A Multicenter Registry
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DOI: 10.1182/blood-2024-203775
Abstract: Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder that can lead to outcomes such as thrombosis, cardiovascular diseases, renal disfunction (both acute kidney injury [AKI] and chronic kidney disease [CKD]), and death… read more here.
Keywords: disease; ci95; size; clinical outcomes ... See more keywords
Post-azacitidine clone size predicts outcome of patients with myelodysplastic syndromes and related myeloid neoplasms.
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DOI: 10.1182/bloodadvances.2022009564
Abstract: Azacitidine is a mainstay of therapy for MDS-related diseases. The purpose of our study is to elucidate the effect of gene mutations on hematological response and overall survival (OS), particularly focusing on their post-treatment clone… read more here.
Keywords: treatment; post treatment; clone size;