Paroxysmal Nocturnal Hemoglobinuria (PNH)

Pegcetacoplan Bests Eculizumab for PNH Patients

Original Publication Date

December 28, 2020

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

December 9, 2020

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

Read more about 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

Read more about Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (pa

One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

Read more about One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

Background: Ravulizumab, the only long-acting complement C5 inhibitor for adults with paroxysmal nocturnal hemoglobinuria

PNH - Traveling with a Rare Disease - Update from Josh

Read more about PNH - Traveling with a Rare Disease - Update from Josh

Traveling with a rare disease

Crovalimab

Read more about 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

Read more about David Jacobsohn, MD, ScM

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.

Read more about Staci Arnold, MD, MBA, MPH

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

Read more about 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.

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NFL Player Teams Up with AAMDSIF

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Pegcetacoplan Bests Eculizumab for PNH Patients

Clinical Challenges: Paroxysmal Nocturnal Hemoglobinuria — Complement inhibitors a major advance but not a cure

Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease

Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

PNH - Traveling with a Rare Disease - Update from Josh

Crovalimab

David Jacobsohn, MD, ScM

Staci Arnold, MD, MBA, MPH

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

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