Results 11 to 20 of about 15,382 (226)

Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations

Journal of Blood Medicine, 2023
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, acquired clonal abnormality, which renders hematopoietic cells exquisitely sensitive to complement-mediated destruction.
Monika Oliver, Christopher J. Patriquin
semanticscholar   +1 more source

Paroxysmal nocturnal hemoglobinuria: Where are we going

American journal of hematology/oncology, 2023
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 ...
A. Kulasekararaj, I. Lazana
semanticscholar   +1 more source

Biomarkers and laboratory assessments for monitoring the treatment of patients with paroxysmal nocturnal hemoglobinuria: Differences between terminal and proximal complement inhibition.

Blood reviews, 2023
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening, acquired disease in which blood cells lacking complement regulatory proteins are destroyed because of uncontrolled complement activity. Since 2007, terminal complement inhibitors have
A. Kulasekararaj, D. Kuter, M. Griffin, I. Weitz, A. Röth   +4 more
semanticscholar   +1 more source

Paroxysmal nocturnal hemoglobinuria [PDF]

Vnitřní lékařství, 2018
Paroxysmal nocturnal hemoglobinuria (PNH) results from a deficiency in inhibitors of activated complement. This lack leads to complement mediated intravascular hemolysis, to activation of coagulation system with increased risk of thrombotic complications and to various degree of bone marrow failure.
openaire   +3 more sources

Paroxysmal nocturnal hemoglobinuria: Review of the patient experience and treatment landscape.

Blood reviews, 2023
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by complement-mediated hemolysis and thrombosis through the alternative pathway. The most common symptom of PNH is fatigue due to chronic anemia, which can negatively impact quality of ...
A. Waheed, J. Shammo, D. Dingli
semanticscholar   +1 more source

Paroxysmal nocturnal hemoglobinuria: Where we stand

American journal of hematology/oncology, 2023
For the last 20 years, therapy of paroxysmal nocturnal hemoglobinuria (PNH) relied—up until recently—on antibody based terminal complement inhibitionon.
J. Panse
semanticscholar   +1 more source

Danazol for paroxysmal nocturnal hemoglobinuria [PDF]

American Journal of Hematology, 1997
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal stem-cell disorder in which blood cells lack complement inhibiting membrane proteins, and become susceptible to complement-mediated injury, leading to chronic intravascular hemolysis and pancytopenia. Glucocorticoids have been a mainstay of therapy.
W J, Harrington, L, Kolodny, L L, Horstman, W, Jy, Y S, Ahn   +4 more
openaire   +2 more sources

Results of a phase 1/2 study of cemdisiran in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria

eJHaem, 2023
Complement dysregulation underpins the physiopathology of paroxysmal nocturnal hemoglobinuria (PNH). Cemdisiran, an RNA interference investigational treatment, silences complement component 5 (C5) expression in the liver.
A. Gaya, T. Munir, Á. Urbano-Ispizua, M. Griffin, J. Taubel, J. Bush, I. Bhan, A. Borodovsky, Yue Wang, P. Badri, P. Garg   +10 more
semanticscholar   +1 more source

Pegcetacoplan controls hemolysis in complement inhibitor–naive patients with paroxysmal nocturnal hemoglobinuria

Blood Advances, 2023
Key Points • Complement inhibitor–naive patients with PNH had greater hemoglobin stabilization and LDH reduction with pegcetacoplan vs control.• Pegcetacoplan’s comprehensive control of hemolysis and favorable safety profile in these patients may help ...
R. Wong, J. R. Navarro-Cabrera, N. S. Comia, Y. Goh, Henry Idrobo, D. Kongkabpan, D. Gómez-Almaguer, M. Al-Adhami, T. Ajayi, P. Alvarenga, J. Savage, Pascal Deschatelets, C. Francois, F. Grossi, T. Dumagay   +14 more
semanticscholar   +1 more source

Paroxysmal Nocturnal Hemoglobinuria [PDF]

Blood, 2014
AbstractParoxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder that manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias. The absence of two glycosylphosphatidylinositol (GPI)-anchored proteins, CD55 and CD59, leads to uncontrolled complement activation that accounts for hemolysis and other PNH ...
openaire   +2 more sources