Results 21 to 30 of about 898 (115)
HematologyObjectives Paroxysmal nocturnal hemoglobinuria (PNH) may develop breakthrough hemolysis (BTH) despite C5 inhibition. Although iptacopan, an oral factor B inhibitor, has demonstrated efficacy in phase 3 trials, switching from crovalimab has not been ...
Daisuke Ogiya +10 more
doaj +2 more sources
Iptacopan treatment for recurrent dense deposit disease after kidney transplant: A case report [PDF]
SAGE Open Medical Case ReportsC3 glomerulopathies, including dense deposit disease, are rare kidney disorders caused by dysregulation of the alternative complement pathway. Recurrence after kidney transplantation is common and can threaten graft survival.
Lean AlKhatib +3 more
doaj +2 more sources
The complement factor B inhibitor iptacopan for relapsed and refractory immune thrombotic thrombocytopenic purpura [PDF]
Thrombosis JournalBackground Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy, classified as congenital TTP (cTTP) and immune TTP (iTTP).
Minran Zhou +7 more
doaj +2 more sources
Iptacopan for cold agglutinin disease: a case report with literature review [PDF]
Frontiers in ImmunologyThis study reports a case of cold agglutinin disease (CAD) secondary to lymphoplasmacytic lymphoma in a patient intolerant to rituximab plus bendamustine and with persistent uncontrolled hemolysis following zanubrutinib therapy.
Baozhi Fang +8 more
doaj +2 more sources
Frontiers in ImmunologyTransplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication of hematopoietic stem cell transplantation (HSCT), characterized by microangiopathic hemolytic anemia, thrombocytopenia, microthrombosis, and multi-organ dysfunction ...
Hong Qu, Shijie Bao
exaly +3 more sources
Iptacopan in C5 blockade refractory atypical hemolytic uremic syndrome with associated Castleman’s disease: case report [PDF]
BMC NephrologyBackground Atypical Hemolytic Uremic Syndrome (aHUS) is a life-threatening disease related to mutations in the complement system. We report the first known case of the use of factor B inhibition in a 21-year-old male with complement-mediated thrombotic ...
Matthew D. Nguyen +10 more
doaj +2 more sources
Disease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations [PDF]
Cost Effectiveness and Resource AllocationBackground Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, life-threatening hematologic disorder characterized by chronic hemolysis, bone marrow and organ failure, and thrombotic events.
Shaghayegh Moradi +4 more
doaj +2 more sources
Kidney MedicineC3 glomerulopathy is a rare disease caused by fluid phase dysregulation of the alternative complement pathway. Currently, treatment depends on clinical and histological severity and includes nephroprotection, unspecific immunosuppression, and terminal ...
Angela Gonzalez Rojas +1 more
exaly +3 more sources
Iptacopan for Immune Thrombocytopenia and Cold Agglutinin Disease: A Global Phase 2 Basket Clinical Trial [PDF]
American Journal of HematologyCristina Pascual +2 more
exaly +2 more sources
C]iptacopan in Healthy Male Volunteers and in In Vivo and In Vitro Studies
, 2023 Iptacopan (LNP023) is an oral, small-molecule, first-in-class, highly potent proximal complement inhibitor that specifically binds factor B and inhibits the alternative complement pathway.
David Pearson +13 more
core +1 more source