Paroxysmal Nocturnal Hemoglobinuria (PNH)
Original Publication Date
Article Source
External Web Content
Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were still anemic despite at least three months of eculizumab therapy…
Clinical Challenges: Paroxysmal Nocturnal Hemoglobinuria — Complement inhibitors a major advance but not a cure
Original Publication Date
Article Source
External Web Content
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare, acquired, life-threatening disease of the blood. The name comes from one of the clinical consequences of the disease -- the breakdown of red blood cells and consequent release of hemoglobin into the urine. It often…
Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease
In the UK, early work on paroxysmal nocturnal haemoglobinuria (PNH) was conducted by John Dacie who, at the Hammersmith Hospital, first hypothesised that the PNH abnormality might arise through a somatic mutation mutation:
Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (pa
PNH - Traveling with a Rare Disease - Update from Josh
Traveling with a rare disease
Crovalimab
An experimental complement inhibitor C5 monoclonal antibody monoclonal antibody: A type of protein called an antibody that is engineered to look for a speci
David Jacobsohn, MD, ScM
Institution
Children's National
Physician Status
accepting new patients
Primary Disease Area of Focus
Acute Myeloid Leukemia (AML)
Aplastic Anemia
Chronic Lymphocytic Leukemia (CLL)
Chronic Myelomonocytic Leukaemia (CMML)
Graft Versus Host Disease (GVHD)
Myelodysplastic Syndromes (MDS)
Myeloproliferative Neoplasms (MPN)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pure Red Cell Aplasia (PRCA)
Pediatric
About
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. Jacobsohn obtained a Bachelor of Science from Yale University, his medical degree from Tufts University, and a Masters in Clinical Investigation from Johns Hopkins School of
Staci Arnold, MD, MBA, MPH
Institution
Children's Healthcare of Atlanta
Physician Status
accepting new patients
Primary Disease Area of Focus
Aplastic Anemia
Myelodysplastic Syndromes (MDS)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pediatric
About
Dr. Arnold is an Assistant Professor of Pediatrics, Emory University School of Medicine. She specializes in pediatric hematology and has particular expertise in the care of children undergoing stem cell transplantation for noncancerous disorders, such as sickle cell anemia, aplastic anemia and thalassemia. In addition to caring for patients, Dr. Arnold conducts research. She has an MBA and does outcome research to demonstrate the cost-effectiveness of the therapies she uses.
Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.
Background: Eculizumab Eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center.