Results 21 to 30 of about 22,402 (256)

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: 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

Clinicopathological Profile of Paroxysmal Nocturnal Hemoglobinuria among Omani Patients: A Case Series

Oman Medical Journal, 2022
We aimed to estimate the nature and prevalence of paroxysmal nocturnal hemoglobinuria (PNH) among Omani patients. We performed a retrospective review of all patients who were tested for PNH by flow cytometry at the Sultan Qaboos University Hospital ...
Arwa Z. Al-Riyami, Yahya Al-Kindi, Jamal Al-Qassabi, Sahimah Al-Mamari, Naglaa Fawaz, Murtadha Al-Khabori, Mohammed Al-Huneini, Salam Al Kindi   +7 more
doaj   +1 more source

Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study

Blood Advances, 2022
Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).
Jun-Ho Jang, Lily Wong, B. Ko, S. Yoon, Katie Li, Irina Baltcheva, P. Nidamarthy, Raghav Chawla, G. Junge, E. Yap   +9 more
semanticscholar   +1 more source

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

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

Post parturient hemoglobinuria in a sheep flock [PDF]

, 2018
ΔΕΝ ΥΠΑΡΧΕΙ ΠΕΡΙΛΗΨΗ ΣΤΑ ΕΛΛΗΝΙΚΑThe syndrome of intravascular hemolysis, hemoglobinuria, and anemia has been recognized in post parturient dairy cattle and buffaloes.
JALILZADEH-AMIN, G., ASRI REZAEI, S., NAJARNEZHAD, V., MOHSENI, M.   +3 more
core   +2 more sources

Herlyn Werner Wunderlich Syndrome Presenting with Ischemic Stroke due to Suspected Paroxysmal Nocturnal Hemoglobinuria: A Case Report

Journal of Nepal Medical Association, 2021
Paroxysmal nocturnal hemoglobinuria can rarely present as cerebral ischemia and stroke due to arterial thrombosis. However, it should be considered in a young patient with bone marrow failure features, systemic thromboses, and hemolysis.
Ayushma Acharya, Prajwala Yogi, Pramod Singh, Tulsi Ram Bhattarai   +3 more
doaj   +1 more source

Clinical and Molecular Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Journal of Clinical Oncology, 2022
PURPOSE Secondary myeloid neoplasms (sMNs) remain the most serious long-term complications in patients with aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH).
C. Gurnari, S. Pagliuca, P. Prata, J. Galimard, L. F. B. Catto, L. Larcher, M. Sébert, V. Allain, B. Patel, Arda Durmaz, A. Pinto, M. C. B. Inacio, L. Hernandez, N. Dhédin, S. Caillat-Zucman, E. Clappier, F. Sicre de Fontbrune, M. Voso, V. Visconte, Régis Peffault de Latour, J. Soulier, R. Calado, G. Socié, J. Maciejewski   +23 more
semanticscholar   +1 more source