Researchers set out to determine whether existing patient-reported outcome (PRO) measures are sufficient to assess the impact on the quality of life of patients with cold agglutinin disease (CAD) and proposed their own set of PRO measures, as presented at the 63rd American Society for Hematology Annual Meeting & Exposition. The constellation of symptoms typically…
Increased risk of thrombotic events (TEs), especially thrombotic microangiopathies (TMAs), occurred with concomitant use of emicizumab, according to an updated safety analysis presented at the 63rd American Society for Hematology Annual Meeting & Exposition. Prothrombin complex concentrate (aPCC) was activated to prophylactically treat individuals with congenital hemophilia A with and without factor VIII inhibitors. No…
TALEN-based gene editing was able to correct mutations in the HBB gene of hematopoietic stem and progenitor cells (HSPCs) taken from patients with sickle cell disease (SCD), as presented at the 63rd American Society for Hematology Annual Meeting & Exposition. The researchers found that TALGlobin01, an autologous HSPC-based gene therapy, was able to achieve up…
The half-life of efanesoctocog alfa does not appear to be related to endogenous von Willebrand factor (VWF) antigen levels in the treatment of hemophilia A, according to a new post hoc analysis presented at the 63rd American Society for Hematology Annual Meeting & Exposition. Efanesoctocog alfa is an investigational recombinant factor VIII (rFVIII) treatment. The…
The C1q inhibitor ANX009 was found to be safe, tolerable, and have favorable pharmacology in a phase 1 clinical trial with healthy individuals, supporting its advancement into testing its efficacy in patients with complement-mediated autoimmune disorders such as cold agglutinin disease (CAD). These trial results were presented as a poster at the 63rd American Society…
Prophylactic treatment with rurioctocog alfa pegol, a recombinant factor VIII (FVIII), leads to dose-dependent normalization of joint health biomarkers in patients with severe hemophilia A, according to results from the PROPEL study. These findings were presented as a poster at the 63rd American Society for Hematology Annual Meeting and Exposition. “This novel biomarker data is…
Follow-up data from the phase 1/2 Alta study of giroctocogene fitelparvovec gene therapy continues to show positive results for patients with moderately severe to severe hemophilia A, as announced by Pfizer and Sangamo Therapeutics in a press release. “These latest results further suggest the potential of this investigational therapy to bring transformational benefit to eligible…
New interim data from the phase 3 HAVEN 6 trial of emicizumab (Hemlibra®) as prophylaxis has shown a safe and effective profile in patients with hemophilia A without factor VIII (FVIII) inhibitors. The results will be presented on December 12, 2021, as part of the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.…
Graphite Bio announced the enrollment of the first patient in their phase 1/2 clinical trial of GPH101 (NCT04819841) for the treatment of sickle cell disease (SCD). The company expects to treat this patient in the first half of 2022 and hopes to get initial proof-of-concept data by the end of 2022. GPH101 is an investigational…
The monoclonal antibody sutimlimab is well-tolerated and improved the quality of life in patients with cold agglutinin disease (CAD). The results from the placebo-controlled trial CADENZA (NCT03347422) and the single-arm CARDINAL study (NCT03347396) will be presented at the 63rd American Society of Hematology Annual Meeting & Exposition (ASH), being held December 11-14, 2021. Most (73%)…
