To investigate the effects of sera on the growth of single CD34(+) cells from patients with paroxysmal nocturnal hematoglobinuria (PNH), sera from both PNH patients and normal individuals were added separately to the single cell culture system and semi-solid colony formation system. The growth of the normal CD34(+) and PNH CD34(+) CD59(+) and CD34(+) CD59(-) cells was evaluated. No growth difference was found for growth of the normal CD34(+) and PNH CD34(+) CD59(+) cells when PNH or normal sera were added to the culture media in either single cell culture or colony formation culture. While no difference was detected for single PNH CD34(+) CD59(-) cells growth when PNH or normal sera were added, more colonies were observed in semi-solid culture with PNH sera. A conclusion was reached that compared with those from normal controls, the sera from PNH patients had no significant influence on single hematopoietic stem/progenitor cells derived from normal subjects and from PNH patients, but the PNH sera might promote the colony formation of the CD34(+) CD59(+) cells in semi-solid culture
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Drugs R D
December 2024
Swedish Orphan Biovitrum AB, Stockholm, Sweden.
Int J Mol Sci
November 2024
Cancer Center, Division of Hematology and Oncology, Department of Medicine, Taipei Municipal Wanfang Hospital, Taipei Medical University, No. 111, Section 3, Xinglong Rd, Wenshan District, Taipei City 11696, Taiwan.
Paroxysmal nocturnal hemoglobinuria (PNH) clones can be identified in a significant proportion of patients with aplastic anemia (AA). Screening for PNH clones at the time of an AA diagnosis is recommended by national and international guidelines. In this report, an expert panel of physicians discusses current best practices and provides recommendations for managing PNH in patients with AA in the Asia-Pacific region.
View Article and Find Full Text PDFBull Cancer
October 2024
Service d'hématologie clinique et d'oncologie pédiatrique, faculté de médecine et de pharmacie de Casablanca, centre hospitalier universitaire 20 Août de Casablanca, Casablanca, Maroc.
Bone marrow aplasia is a rare and serious hematologic disorder. Although benign, it is a hematologic disorder whose prognosis can be poor and whose spontaneous development can be fatal. Treatment is long, difficult and costly.
View Article and Find Full Text PDFBlood Adv
June 2024
Department of Haematology, King's College Hospital, National Institute of Health Research/Wellcome King's Clinical Research Facility, King's College London, London, United Kingdom.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease of uncontrolled terminal complement activation leading to intravascular hemolysis, thrombotic events and increased morbidity and mortality. This phase 3, open-label, single-arm, multicenter study evaluated ravulizumab treatment in eculizumab-naive or -experienced pediatric patients (aged <18 years) with PNH over a 26-week primary evaluation period (PEP) and 4-year extension period (EP). Patients included in the study received weight-based intravenous ravulizumab dosing.
View Article and Find Full Text PDFFront Immunol
January 2024
Department of Hematology, Kanazawa University, Kanazawa, Japan.
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disease characterized by intravascular hemolysis, thrombosis, and bone marrow (BM) failure. Although PNH is caused by excessive proliferation of hematopoietic stem cell (HSC) clones with loss of function mutations in phosphatidylinositol N-acetylglucosaminyltransferase subunit A () genes, what drives PNH clones to expand remains elusive.
Case Description: We present a case of a 26-year-old female who presented with hemolytic anemia, thrombocytopenia, and leukopenia.
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