Results 1 to 10 of about 10,942 (165)

Pegcetacoplan: A New Opportunity for Complement Inhibition in PNH

Journal of Blood Medicine, 2023
Ilene Ceil Weitz Jane Anne Nohl Division of Hematology, Keck-USC School of Medicine, Los Angeles, CA, USACorrespondence: Ilene Ceil Weitz, Jane Anne Nohl Division of Hematology, Keck-USC School of Medicine, Los Angeles, CA, USA, Tel +1-323-865-3950, Fax +
Ilene Weitz
exaly   +3 more sources

A novel patient-reported outcome instrument assessing the symptoms of paroxysmal nocturnal hemoglobinuria, the PNH-SQ

Journal of Patient-Reported Outcomes, 2021
Background Patient-reported outcome measures (PROs) used to measure symptoms of patients with paroxysmal nocturnal hemoglobinuria (PNH) in trials do not measure PNH symptoms comprehensively and do not assess daily fluctuations in symptoms.
Jessica Jacqueline Jalbert, Jamile M Shammo   +1 more
exaly   +2 more sources

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

Prognostic value of pre-treatment PNH clone among the patients with aplastic anemia: a meta-analysis

Hematology, 2023
Background Paroxysmal nocturnal hemoglobinuria (PNH) clone can be detected in some patients with aplastic anemia (AA) before treatment. But the prognostic value of the presence of pre-treatment PNH clone for intensive immunosuppressive therapy (IIST) is ...

exaly   +3 more sources

High Proportion of PNH Type II Neutrophils Is Associated With Thrombosis in Patients Displaying a PNH Clone ≥ 1. [PDF]

Am J Hematol
ABSTRACT The clinical significance of PNH Type II white blood cells (WBCs) remains unclear. We assessed the relative percentage (rel%) of Type II neutrophils in 355 patients with a PNH clone ≥ 1% on neutrophils enrolled by 33 flow cytometry laboratories in the 5‐year French nation‐wide multicenter prospective observational study.
Wagner-Ballon O, Lhoumeau AC, Raimbault A, Debord C, Vial JP, Soenen V, Pannetier M, Bonneville E, Bera E, Bret C, Chapuis N, Latger-Cannard V, Mayeur-Rousse C, Harrivel V, Vergez F, Loosveld M, Xuan JV, Mathis S, Socié G, de Fontbrune FS, de Latour RP, Drenou B, Debliquis A, Le Garff-Tavernier M, French PNH working group CytHem‐HPN.   +24 more
europepmc   +2 more sources

Prognostic significance of PNH clones in aplastic anemia treated with immunosuppression or allogeneic HSCT: a 20-year single-center experience [PDF]

Blood Research
Background Paroxysmal nocturnal hemoglobinuria (PNH) clones are detected in up to 60% of patients with aplastic anemia (AA); however, their prognostic impact remains incompletely defined, particularly in the context of frontline immunosuppressive therapy
Alfadil Haroon, Hazzaa Alzahrani, Mostafa F. Mohammed Saleh, Ali Aalahmari, Shaykhah Alotaibi, Tusneem Elhassan, Feras Alfraih, Fahad Alsharif, Syed O. Ahmed, Fahad Almohareb, Riad El Fakih, Mahmoud Aljurf   +11 more
doaj   +2 more sources

Paroxysmal Nocturnal Hemoglobinuria Presenting With Progressive Biventricular Thrombi [PDF]

Case Reports in Hematology
PNH is a clonal hematopoietic disorder causing complement-mediated hemolysis and thrombosis. While thrombosis typically affects atypical venous sites, intracardiac thrombi are rare. PNH was the patient’s only identified prothrombotic factor.
Jordan S. Nunnelee, Garrett A. Weber, Nicholas P. Bergeron, Katherine E. Talbott, Andrew J. Layman, David Dingli, Richard C. Godby   +6 more
doaj   +2 more sources

Clinical characteristics of 512 eculizumab-naive paroxysmal nocturnal hemoglobinuria patients in China: a single-center observational study

Hematology, 2022
Objectives With large patient population and complement inhibitors naïve background, the characteristics patients with paroxysmal nocturnal hemoglobinuria (PNH) in China have not been well studied, especially for different subtypes.
Yali Du, Yuan Yang, Chen Yang, Miao Chen, Bing Han   +4 more
doaj   +1 more source

Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience

Turkish Journal of Hematology, 2021
Objective: Although inhibition of the complement system at different steps is a promising therapy modality in patients with paroxysmal nocturnal hemoglobinuria (PNH), allogeneic hematopoietic stem cell transplantation (HCT) is still the only curative ...
Fergün Yılmaz, Nur Soyer, Güldane Cengiz Seval, Sinem Civriz Bozdağ, Pervin Topcuoğlu, Ali Ünal, Leylagül Kaynar, Gökhan Özgür, Gülsan Türköz Sucak, Hakan Göker, Mustafa Velet, Hakan Özdoğu, Mehmet Yılmaz, Emin Kaya, Ozan Salim, Burak Deveci, İhsan Karadoğan, Güray Saydam, Fahri Şahin, Filiz Vural   +19 more
doaj   +1 more source

Compound heterozygous variants in LAMC3 in association with posterior periventricular nodular heterotopia

BMC Medical Genomics, 2021
Background Periventricular nodular heterotopia (PNH) is a malformation of cortical development characterized by nodules of abnormally migrated neurons.
Carla De Angelis, Alicia B. Byrne, Rebecca Morrow, Jinghua Feng, Thuong Ha, Paul Wang, Andreas W. Schreiber, Milena Babic, Ajay Taranath, Nick Manton, Sarah L. King-Smith, Quenten Schwarz, Peer Arts, Hamish S. Scott, Christopher Barnett   +14 more
doaj   +1 more source