63rd ASH Annual Meeting & Exposition

doctor and patient

New Patient-Reported Measures Can Better Assess Quality of Life in CAD

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…

Study Shows Risk-Benefit of Emicizumab Unchanged in Hemophilia A

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…

DNA

TALEN-Based Process Leads to Gene Correction in SCD Cells

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…

clinical trial

Efanesoctocog Alfa Shows VWF-Independent Results in Hemophilia A

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…

Phase 1 Study Results Support Advancement of ANX009 for CAD

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…

doctor and patient

FVIII Prophylaxis Leads to Normalization of Joint Remodeling in Hemophilia A

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…

Phase 1/2 Study Results Show Sustained Bleeding Control in Hemophilia A

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…

Sutimlimab Posts Positive Results in Phase 3 Trials for Cold Agglutinin Disease

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%)…

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