Results 11 to 20 of about 2,525 (118)

A real-world, multi-center, prospective, observational study for paroxysmal nocturnal haemoglobinuria (PNH) in China: Baseline characteristics, disease burden and treatment patterns [PDF]

Therapeutic Advances in Hematology
Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare life-threatening hematologic disorder with high thromboembolic mortality. In the context of large patient population and limited use of complement inhibitors, the clinical characteristics and
Bing Han, Hui Liu, Guangsheng He, Jinsong Jia, Mei Hong, Jianyu Weng, Shunqing Wang, Fengkui Zhang, Ting Niu, Tianjun Ma, Jinghan Xu, Rong Fu   +11 more
doaj   +2 more sources

Higher doses of eculizumab may be beneficial in paroxysmal nocturnal haemoglobinuria in pregnancy [PDF]

European Journal of Case Reports in Internal Medicine
Introduction: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare bone marrow failure disorder that results in complement-mediated haemolysis (mostly intravascular), thrombosis and bone marrow suppression.
Harshank Vishnubhai Patel, Steve Stone, Madison C. Laird, Vanita Motiani, Ayema Haque, Hemasri Tokala, Akshay Amaraneni   +6 more
doaj   +2 more sources

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria [PDF]

eJHaem
Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive ...
Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour   +8 more
doaj   +2 more sources

PB2043: CLINICAL EFFECTIVENESS, TREATMENT USE, SATISFACTION AND IMPACT ON QUALITY-OF-LIFE OF PEGCETACOPLAN TREATMENT FOR PATIENTS WITH PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA (PNH). [PDF]

HemaSphere, 2023
Koo Wilson, Carly Rich, Zalmai Hakimi, Jesse Fishman, Jennifer Mellor, Lucy Earl, Yasmin Taylor, Alice Simons, Joe Conyers, Alexander Röth   +9 more
doaj   +2 more sources

Clinical Outcomes and Disease Management for Patients with Paroxysmal Nocturnal Haemoglobinuria in China: Results from a Real-World Study [PDF]

Drugs - Real World Outcomes
Background Paroxysmal nocturnal haemoglobinuria (PNH) is a chronic haematological disorder caused by uncontrolled complement activation, leading to intravascular haemolysis, indicated by increased lactate dehydrogenase levels (LDH).
Rong Fu, Hui Liu, Pin-Tzu Su, Xuefei Liu, Karl-Johan Myrén, Francesca Gatta, Lucy Earl, Yasmin Taylor, Alice Simons, Elmira Lechat   +9 more
doaj   +2 more sources

Severe renal haemosiderosis in a patient with untreated paroxysmal nocturnal haemoglobinuria: a case report [PDF]

BMC Nephrology
Background Paroxysmal nocturnal haemoglobinuria (PNH) is a life-threatening disease in which intravascular haemolysis of the red blood cells frequently manifests with chronic haemolysis, anaemia and thrombosis.
Zhong Zhen Goh, Kenny Tang, Katrina Chau, Seethalakshmi Viswanathan   +3 more
doaj   +2 more sources

MUC4 mutations promote a thrombotic phenotype in patients with paroxysmal nocturnal haemoglobinuria by increasing the deposition of terminal complement [PDF]

Clinical and Translational Medicine
Background Thrombosis is a common complication in paroxysmal nocturnal haemoglobinuria (PNH) patients, but primary prevention remains controversial. Identifying high‐risk individuals could enable risk‐stratified prophylactic anticoagulation strategies ...
Yingying Chen, Mengting Che, Chaomeng Wang, Qiaoyi Bronte Zhang, Weixin Chen, Hui Liu, Chunyan Liu, Guang Sheng Ling, Rong Fu   +8 more
doaj   +2 more sources

Pancytopenia in a Case of Aplastic Anaemia/Paroxysmal Nocturnal Haemoglobinuria Unmasked by SARS-CoV-2 Infection: A Case Report

Medicina, 2022
During an acute SARS-CoV-2 infection, a diagnosis of Aplastic Anaemia associated with Paroxysmal Nocturnal Haemoglobinuria (AA/PNH) was made in a 78-year-old woman who had presented to the emergency department with severe pancytopenia.
Arcangelo Iannuzzi, Antonio Parrella, Francesca De Ritis, Anna Cammarota, Lucia Berloco, Francesca Paudice, Giovanni D’Angelo, Emilio Aliberti, Gabriella Iannuzzo   +8 more
doaj   +1 more source

Cholecystectomy in a patient with paroxysmal nocturnal haemoglobinuria undergoing ravulizumab maintenance treatment

eJHaem, 2022
A 47‐year‐old male with paroxysmal nocturnal haemoglobinuria (PNH) controlled with routine ravulizumab administration suffered a massive haemolytic crisis due to choledocholithiasis.
Mitsuru Moriyama, Yasuo Aota, Masahiro Okabe, Yoshiaki Osaka, Seiichiro Katagiri, Daigo Akahane, Akihiko Gotoh   +6 more
doaj   +1 more source

Non-capsulated Neisseria meningitidis sepsis in a paroxysmal nocturnal hemoglobinuria patient treated with ravulizumab: case report and review of the literature

Frontiers in Immunology, 2023
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare acquired haematopoietic stem cell disease characterized by complement-mediated intravascular hemolysis, thrombosis, and bone marrow failure. Eculizumab and ravulizumab are anti-C5 monoclonal antibodies
Nicole Galli, Nicole Galli, Loredana Pettine, Mauro Panigada, Laura Daprai, Laura Daprai, Grazia Suriano, Anna Grancini, Anna Grancini, Wilma Barcellini, Bruno Fattizzo, Bruno Fattizzo   +11 more
doaj   +1 more source