Anna Gayà
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View article: Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dl on anti-C5 therapy: Subgroup analysis of the appulse-pnh Phase 3b trial
Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dl on anti-C5 therapy: Subgroup analysis of the appulse-pnh Phase 3b trial Open
Introduction: Despite achieving a ‘good response’ with anti-C5 therapy (defined as hemoglobin [Hb] levels 10 to <12 g/dL), some patients with paroxysmal nocturnal hemoglobinuria (PNH) remain anemic and continue to experience symptoms due t…
The 2-year efficacy and safety of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria with a history of aplastic anemia on concomitant immunosuppressive therapy who entered the roll-over extension program Open
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis, thrombosis, and potential concurrent bone marrow failure. A strong clinical relationship exists bet…
Unmet medical needs in paroxysmal nocturnal hemoglobinuria treatment: Insights from the SATURNO Study on C5 inhibitor therapy in Spain Open
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder. Benefits of current C5 inhibitor (C5i) treatments are indisputable, but some symptoms and impairment of daily quality of life persist.Objective: To assess satisfac…
Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH Open
Complement C5 inhibitor treatment with ravulizumab or eculizumab for paroxysmal nocturnal hemoglobinuria (PNH) improves outcomes and survival. Some patients remain anemic due to clinically significant extravascular hemolysis (cs-EVH; hemog…
Danicopan As Add-on Therapy to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Patient-Reported Outcomes from the Phase 3 ALPHA Trial Open
Background In patients with paroxysmal nocturnal hemoglobinuria (PNH), the complement C5 inhibitors (C5i) ravulizumab and eculizumab have proven efficacy and safety; ravulizumab provides immediate, complete and sustained inhibition of term…
Phase 3 randomized <span>COMMODORE</span> 2 trial: Crovalimab versus eculizumab in patients with <span>paroxysmal nocturnal hemoglobinuria</span> naive to complement inhibition Open
Crovalimab is a novel C5 complement inhibitor that enables rapid and sustained C5 inhibition with subcutaneous, low‐volume self‐administration every 4 weeks. COMMODORE 2 (NCT04434092) is a global, randomized, open‐label, multicenter, phase…
View article: Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria
Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria Open
Iptacopan treatment improved hematologic and clinical outcomes in anti-C5-treated patients with persistent anemia - in whom iptacopan showed superiority to anti-C5 therapy - and in patients who had not received complement inhibitors. (Fund…
Paradoxical Findings in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors: Suboptimal Disease Control Perceived As Satisfactory Open
Introduction: The introduction of C5 inhibitors (C5i) for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) has resulted in lower morbidity and mortality rates. However up to 86% of patients still experience anemia a…
PB2064: THE COMPLETE STUDY: A SINGLE ARM, MULTICENTER OBSERVATIONAL STUDY TO EVALUATE EFFECTIVENESS OF PEGCETACOPLAN UNDER REAL WORLD CONDITIONS IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) Open
Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: Paroxysmal nocturnal hemoglobinuria (PNH) is caused by complement-mediated hemolysis with resultant thrombosis risk and a substantial patient symptom burden. Complem…
S182: ORAL IPTACOPAN MONOTHERAPY INCREASES PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) RED BLOOD CELL CLONE SIZE VIA CONTROL OF INTRA- AND EXTRAVASCULAR HEMOLYSIS IN ANTI-C5-TREATED PNH PATIENTS WITH ANEMIA Open
Background: Anemia caused by C3-mediated extravascular hemolysis (EVH) commonly occurs in patients (pts) with PNH receiving standard-of-care (SoC) anti-C5 therapies. Iptacopan (IPTA) is a first-in-class, oral, selective complement factor B…
View article: S181: THE PHASE III, RANDOMIZED COMMODORE 2 TRIAL: RESULTS FROM A MULTICENTER STUDY OF CROVALIMAB VS ECULIZUMAB IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) PATIENTS NAIVE TO COMPLEMENT INHIBITORS
S181: THE PHASE III, RANDOMIZED COMMODORE 2 TRIAL: RESULTS FROM A MULTICENTER STUDY OF CROVALIMAB VS ECULIZUMAB IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) PATIENTS NAIVE TO COMPLEMENT INHIBITORS Open
Background: COMMODORE 2 (NCT04434092) is a global, randomized, open-label, multicenter Phase III trial evaluating the non-inferiority of the C5 complement inhibitor (C5i) crovalimab (crova) vs eculizumab (ecu) in C5i-naive patients (pts) w…
View article: Results of a phase 1/2 study of cemdisiran in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria
Results of a phase 1/2 study of cemdisiran in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria Open
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. Previously report…
View article: IMMUNE PROFILE IN THE PERIPHERAL BLOOD (PB) OF PATIENTS WITH FOLLICULAR LYMPHOMA (FL) AT DIAGNOSIS AND UPON RELAPSE
IMMUNE PROFILE IN THE PERIPHERAL BLOOD (PB) OF PATIENTS WITH FOLLICULAR LYMPHOMA (FL) AT DIAGNOSIS AND UPON RELAPSE Open
Introduction: FL is characterized by a heterogeneous clinical course. The biologic importance of the tumour microenvironment in FL pathogenesis is well established. However, detailed information on the immune profile in PB has not been pre…
PB2088: IMMUNE PROFILE OF PATIENTS WITH FOLLICULAR LYMPHOMA ASSESSED BY FLOW CYTOMETRY IN PERIPHERAL BLOOD: CHARACTERISTICS AT DIAGNOSIS AND AT RELAPSE OF THE DISEASE Open
Background: Follicular lymphoma (FL) is the indolent lymphoma more frequently diagnosed in Western countries and is characterized by a heterogeneous clinical course. The biologic importance of the tumour microenvironment in FL pathogenesis…
Prolonged viral replication in patients with hematologic malignancies hospitalized with COVID-19 Open
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One‐year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab Open
Ravulizumab every 8 weeks showed non‐inferiority to eculizumab every 2 weeks in a 26‐week, phase 3, randomized controlled trial in adults with paroxysmal nocturnal hemoglobinuria (PNH) who were clinically stable on eculizumab (NCT03056040)…
View article: Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria
Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria Open
This study shows that a substantial proportion of patients preferred ravulizumab over eculizumab and provides an important patient perspective on PNH treatment when there is more than one treatment option.
Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study Open
Ravulizumab, a new complement component C5 inhibitor administered every 8 weeks, was noninferior to eculizumab administered every 2 weeks in complement-inhibitor–naive patients with paroxysmal nocturnal hemoglobinuria (PNH). This study ass…
PiwiRNA-651 as marker of treatment response and survival in classical Hodgkin lymphoma Open
PiwiRNAs, small non-coding RNAs processed by Piwi proteins, are involved in maintaining genome stability in germline cells. Recently, piwiRNA expression has been identified in some tumors. We have examined the potential reactivation of the…