ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Mar 28th, 2023 - Describe the real-world clinical profile of eculizumab-treated patients by characterizing their short- and long-term clinical and laboratory outcomes. This retrospective study used preexisting medical records of eculizumab-treated patients with pa...

Mar 23rd, 2023 - Inhibitors of complement and coagulation are present in the saliva of a variety of blood feeding arthropods that transmit parasitic and viral pathogens. Here we describe the structure and mechanism of action of the sand fly salivary protein lufaxi...

Mar 17th, 2023 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

Mar 15th, 2023 - Approach Considerations Therapy for aplastic anemia may consist of supportive care only, immunosuppressive therapy, or hematopoietic cell transplantation (HCT). Severe and very severe aplastic anemia (SAA and VSAA, respectively; see Workup/Staging...

Mar 13th, 2023 - Background The history of stem cell transplant goes back as early as 1939, when the first documented clinical transplant was performed. In 1968, the first successful allogenic stem cell transplant was made possible followed by series of achievemen...

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....

Mar 17th, 2023 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

Mar 3rd, 2023 - The primary objective of the study is: To evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 26-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with activ...

Mar 2nd, 2023 - This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be evaluated in healthy...

Feb 27th, 2023 - The primary objective of the study is to describe the long-term safety, tolerability, and efficacy of pozelimab and cemdisiran combination therapy in patients with PNH The secondary objectives of the study are to describe the long-term effect of t...

Feb 24th, 2023 - A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.

Nov 2nd, 2022 - The exploration of combination therapies with an azacitidine (Vidaza) backbone could alter the treatment landscape for patients with myelodysplastic syndromes (MDS), according Mikkael A. Sekeres, MD, MS. Data are expected soon from the phase 3...

Jul 12th, 2022 - Venous thromboembolism (VTE) is a common diagnosis indicating a blood clot located in a vein.1 Most hematology/oncology fellows will care for patients with this diagnosis throughout their training and long after their graduation from fellowship. D...

Apr 9th, 2022 - Pegcetacoplan (Empaveli) treatment was associated with hemoglobin stabilization and normalization, as well as a superior change from baseline in hemoglobin levels, which showed a meaningful correction of anemia, in patients with complement inhibit...

Oct 6th, 2021 - According to Mohamed Kharfan-Dabaja, MD, there is ever-increasing hope for patients suffering from paroxysmal nocturnal hemoglobinuria (PNH), a rare disease characterized by the premature breakdown of blood cells that is caused by a somatic mutati...

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...