[HTML][HTML] Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT

AM Risitano, S Marotta, P Ricci, L Marano… - Frontiers in …, 2019 - frontiersin.org
The treatment of paroxysmal nocturnal hemoglobinuria has been revolutionized by the
introduction of the anti-C5 agent eculizumab; however, eculizumab is not the cure for …

[HTML][HTML] ESCMID Study Group for Infections in Compromised Hosts (ESGICH) Consensus Document on the safety of targeted and biological therapies: an infectious …

KL Winthrop, X Mariette, JT Silva, E Benamu… - Clinical Microbiology …, 2018 - Elsevier
Background The present review is part of the ESCMID Study Group for Infections in
Compromised Hosts (ESGICH) Consensus Document on the safety of targeted and …

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study

AG Kulasekararaj, A Hill, ST Rottinghaus… - Blood, The Journal …, 2019 - ashpublications.org
Ravulizumab, a new complement component C5 inhibitor administered every 8 weeks, was
noninferior to eculizumab administered every 2 weeks in complement-inhibitor–naive …

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study

JW Lee, F Sicre de Fontbrune… - Blood, The Journal …, 2019 - ashpublications.org
Abstract Ravulizumab (ALXN1210), a new complement C5 inhibitor, provides immediate,
complete, and sustained C5 inhibition. This phase 3, open-label study assessed the …

Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single …

AM Risitano, A Röth, J Soret, C Frieri… - The Lancet …, 2021 - thelancet.com
Background The haematological benefit of standard-of-care anti-C5 treatment for haemolytic
paroxysmal nocturnal haemoglobinuria is limited by residual intravascular haemolysis or …

The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria

A Röth, J Nishimura, Z Nagy… - Blood, The Journal …, 2020 - ashpublications.org
Complement C5 inhibition is the standard of care (SoC) for patients with paroxysmal
nocturnal hemoglobinuria (PNH) with significant clinical symptoms. Constant and complete …

Eculizumab reduces complement activation, inflammation, endothelial damage, thrombosis, and renal injury markers in aHUS

R Cofiell, A Kukreja, K Bedard, Y Yan… - Blood, The Journal …, 2015 - ashpublications.org
Atypical hemolytic uremic syndrome (aHUS) is a genetic, life-threatening disease
characterized by uncontrolled complement activation, systemic thrombotic microangiopathy …

The complement system and adverse pregnancy outcomes

JF Regal, JS Gilbert, RM Burwick - Molecular immunology, 2015 - Elsevier
Adverse pregnancy outcomes significantly contribute to morbidity and mortality for mother
and child, with lifelong health consequences for both. The innate and adaptive immune …

How we ('ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future

AM Risitano, R Peffault de Latour - British Journal of …, 2022 - Wiley Online Library
Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement‐mediated
intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients …

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab

AG Kulasekararaj, AM Risitano… - Blood, The Journal …, 2021 - ashpublications.org
Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by uncontrolled terminal
complement activation and subsequent intravascular hemolysis (IVH). C5 inhibitors prevent …