ASH 2022

Clinical trials

Session Highlights Trials That Lead to Eculizumab Approval for PNH

NEW ORLEANS, La.—Researchers highlight the many trials that contributed to the development of therapies and treatment for paroxysmal nocturnal hemoglobinuria, according to a recent session held at the 64th ASH Annual Meeting and Exposition. PNH is the archetypal complement disorder. Patients with PNH often experience intravascular hemolysis, thrombosis, bone marrow failure, and anemia. Intravascular hemolysis…

clinical trial

Trial: Iptacopan Superior to Standard-of-Care Therapy in PNH

NEW ORLEANS, La.—Oral iptacopan treatment may be superior to standard-of-care treatment among patients with paroxysmal nocturnal hemoglobinuria (PNH), according to a phase 3 trial. Study data showed that, from baseline, none of the patients receiving standard-of-care who had evaluable/complete data and 85% of the patients treated with iptacopan had hemoglobin levels rise by 2 g/dL…

intravenous medication

Crovalimab May Be Safe and Effective in Patients with PNH

NEW ORLEANS, La.—Crovalimab may be safe and effective in patients with paroxysmal nocturnal hemoglobinuria (PNH), according to a single-arm, multicenter trial. The study results demonstrated that the average number of patients reaching hemolysis control was about 79%. By week 3, average lactate dehydrogenase 1.5 times the upper limit of normal was achieved and maintained through week…

Eculizumab and Ravulizumab Safe and Efficient in PNH Treatment, Data Show

Eculizumab and ravulizumab are safe and efficient in the long term for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), according to 20-year-long data from the UK presented at the 64th ASH Annual Meeting and Exposition. The study authors also reported that the overall survival of the treated patients remained high and complications such as thromboses,…

pregnant woman

Maternal Health in Hematology Warrants Advocacy

NEW ORLEANS, La.—Mortality and maternal morbidity are closely related within the field of hematology, suggesting that this topic warrants advocacy among clinicians, according to a recent research presentation held at the 64th ASH Annual Meeting and Exposition. The Michigan Society of Hematology and Oncology (MSHO) is an affiliate partner of ASH and a state affiliate…

bone marrow

Bone Marrow Failure Syndromes Require a Multidisciplinary Approach

NEW ORLEANS, La.—Managing patients with bone marrow failure (BMF) syndromes requires a multidisciplinary approach, according to a recent research presentation held at the 64th ASH Annual Meeting and Exposition. “Bone marrow failure syndromes are a diverse collection of disorders characterized by bone marrow abnormalities and a variety of extra hematopoietic phenotypes. The causes of bone…

Pills and bottle

Optimal Dose of Iptacopan for Maximal Disease Control in PNH Identified

Iptacopan at a dose of 200 mg twice a day is optimal to provide maximal disease control in paroxysmal nocturnal hemoglobinuria (PNH), according to a new dose-exposure-response analysis presented at the 64th ASH Annual Meeting and Exposition. The dose-exposure-response relationship was comparable among patients regardless of whether they had a partial response to C5 inhibition or…

Health care provider with IV set up

Intensive PEG Treatment Can Safely Manage Acute Hemolysis in PNH

Acute hemolysis can be managed effectively with intensive subcutaneous or intravascular pegcetacoplan (PEG) treatment in patients with paroxysmal nocturnal hemoglobinuria (PNH), according to results presented at the 64th ASH Annual Meeting and Exposition. These results from a subset of patients enrolled in the ongoing phase 3 PEG 307 open-label extension study also demonstrate that this…

Syprine Wilson disease

Vemircopan Monotherapy Appears Safe and Effective in Patients With PNH

NEW ORLEANS, La.—Vemircopan appears to prevent clinically significant extravascular hemolysis (EVH) and control intravascular hemolysis (IVH) in patients with paroxysmal nocturnal hemoglobinuria, according to an ongoing, phase 2, open-label study. Absolute reticulocyte count declined from 212.4 x 103/uL to 120.0 x 103/uL, average hemoglobin went up from 7.9 g/dL to 11.9 g/dL, lactate dehydrogenase rapidly…

COVID-19

Humoral Response Reported in Select Patients After Allogeneic SCT and COVID-19 Vaccination

NEW ORLEANS, La.—New findings on patients who have undergone allogeneic stem cell transplant (SCT) suggest a humoral response may be reached following the regularly approved SARS-CoV-2 vaccination schedule, particularly for patients who were free from immunosuppressive medications and immune reconstitution. The results of a retrospective study showed that only 3% of patients had a reactivation…

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