ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Nov 26th, 2021 - BACKGROUND Paroxysmal cold hemoglobinuria (PCH) is an autoimmune hemolytic disease caused by the Donath-Landsteiner (DL) antibody. Paroxysmal nocturnal hemoglobinuria (PNH) is a non-autoimmune hemolytic disease that is caused by a dysfunction in t...

Nov 24th, 2021 - The study consisted of a 4-week screening period and a 26-week randomized treatment period (Primary Evaluation Period). After completion of the 26-week Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, ...

Nov 23rd, 2021 - Flow cytometry is increasingly applied in cytopenic patients suspected for myelodysplastic syndromes (MDS). Analysis includes evaluation of antigen expression patterns in granulocytes of which, for example, partial lack of CD16 may indicate dyspla...

Nov 23rd, 2021 - The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or...

Nov 19th, 2021 - Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor. Recently, the use of eltrombopag (ELT) has shown blood count improve...

Jun 10th, 2010 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem cell disorder characterized by a somatic mutation in the PIGA gene, leading to a deficiency of proteins linked to the cell membrane via glycophosphatidylinositol (GPI) anchors....

Nov 24th, 2021 - The study consisted of a 4-week screening period and a 26-week randomized treatment period (Primary Evaluation Period). After completion of the 26-week Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, ...

Nov 23rd, 2021 - The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or...

Nov 19th, 2021 - Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor. Recently, the use of eltrombopag (ELT) has shown blood count improve...

Nov 18th, 2021 - This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis. This observational study consists of two parts, one part is retrospective study which aims to collect medical ...

Oct 28th, 2021 - LNP023 is a novel oral small molecular weight compound, that inhibits alternative complement pathway (AP). Blockade of the AP with oral LNP023 has the potential to prevent both intra - and extravascular hemolysis. This three-period study includes:...

Sep 3rd, 2021 - The European Commission has granted an expanded approval to ravulizumab-cwvz (Ultomiris) to include children with a body weight of at least 10 kg, as well as adolescents, with paroxysmal nocturnal hemoglobinuria (PNH).1 The approval is based on...

Aug 20th, 2021 - A global phase 3 trial of ravulizumab (Ultomiris) for adults with amyotrophic lateral sclerosis (ALS) is being discontinued following a data review, its manufacturer has announced. The CHAMPION-ALS placebo-controlled trial had enrolled 382 adults ...

Jun 22nd, 2021 - The FDA has approved the expanded use of ravulizumab-cwvz (Ultomiris) to include children aged 1 month and older, as well as adolescents, with paroxysmal nocturnal hemoglobinuria. The decision was based on interim findings of a phase 3 trial, whi...

May 19th, 2021 - A rare, life-threatening anemia now has a new treatment option. The Food and Drug Administration announced the approval of pegcetacoplan (Empaveli) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan is the firs...

Apr 22nd, 2021 - When I was a medical student, I always found it gratifying when there was a unifying mechanism that explained the symptoms of a disease. Part of the reason I chose dermatology as a specialty was how frequently we are able to "see" these mechanisms...