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paroxysmal nocturnal hemoglobinuria (PNH)

David Jacobsohn, MD, ScM

Jacobsohn, David
Division Chief, Blood and Marrow Transplanation
Children's National

David Jacobsohn, MD, ScM is the Division Chief, Blood and Marrow Transplanation at Children's National Hospital in Washington, D.C.   Previously, he was at Children’s Memorial Hospital in Chicago, where he served as a member of the Stem Cell Transplant Program staff and as the Director of the Chronic Graft-versus-Host Disease Clinic. He has lectured widely on GvHD/BMT in Europe, Latin America, and the United States. Dr.

Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.

Author(s): 
Peipert JD, Kulasekararaj AG, Gaya A, Langemeijer SMC, Yount S, Gonzalez-Fernandez FA, Ojeda Gutierrez E, Martens C, Sparling A, Webster KA, Cella D, Tomazos I, Ogawa M, Piatek CI, Wells R, Sicre de Fontbrune F, Röth A, Mitchell L, Hill A, Kaiser K
Primary Author: 
Peipert JD
Journal Title: 
PloS One
Original Publication Date: 
Sep 2020

Background: 

A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. (COMMODORE 2)

This study will enroll participants aged 12 years or older with a body weight >= 40 kg diagnosed with PNH who have not been previously treated with complement inhibitor therapy. Approximately 200 participants will be randomized in a 2:1 ratio into the following regimens: [1] Crovalimab; [2]

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (

Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (APPLY-PNH)

The purpose of this Phase 3 randomized, multicenter, active-comparator controlled, open-label trial is to determine whether LNP023 is efficacious and safe for the treatment in PNH through demonstration of superiority of LNP023 compared to anti-C5 antibody treatment in adult PNH patients presenting with residual

Ramy M. Hanna, MD, FASN, FACP

Hanna, Ramy
Assistant Clinical Professor of Medicine and Nephrology
University of California Irvine

Dr. Ramy Hanna is an Assistant Clinical Professor of Medicine and Nephrology at the University of California Irvine UCI-Health.  He is a clinician-educator who works at the intersection of patient education and research, participating in ongoing research into diseases of the kidneys (nephrology) as it relates to complement disorders such as PNH.  Dr. Hanna is focused on working with underserved communities and the expanding field of Artificial Intelligence/Machine Learned related to improving the diagnostic process and treatment outcomes for patients. 

Patients with paroxysmal nocturnal hemoglobinuria demonstrate a prothrombotic clotting phenotype which is improved by complement inhibition with eculizumab

Author(s): 
Macrae FL, Peacock-Young B, Bowman P, Baker SR, Quested S, Linton E, Hillmen P, Griffin M, Munir T, Payne D, McKinley C, Clarke D, Newton DJ, Hill A, Ariëns RAS
Primary Author: 
Macrae FL
Journal Title: 
American Journal of Hematology
Original Publication Date: 
Aug 2020

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