ASH 2023
Heme Today editors highlight major news on classical hematology from the 65th American Society of Hematology Annual Meeting & Exposition, hosted 9-12, 2023, in San Diego, California.
Drs. Grant, Sutin, and Lin discuss their pilot study investigating cerebral hemodynamics in infants with sickle cell disease.
Dr. Pakbaz, from the University of California Irvine, shared data on the value of a dedicated classical hematology clinic.
Dr. Hobbs shared findings supporting the safety and feasibility of continuing ruxolitinib during and after HSCT for MF.
The panel shares their outlook for the future of MDS research and treatment.
Dr. Tanaka outlines a biomarker study that observed reduced inflammation in patients with MDS treated with luspatercept.
The panel discussed a study that assessed quality of life and PROs in patients receiving luspatercept for MDS.
The panel discusses whether starting treatment with luspatercept sooner in patients with MDS is beneficial.
Dr. Shammo shares her reaction to a study that assessed mutational burden and impact on primary outcomes in COMMANDS.
Dr. Safah outlines the findings from the full analysis of the COMMANDS trial.
The panel discusses the evolution of MDS therapy and how the MEDALIST trial set the stage for luspatercept.
Dr. Safah gave an overview of myelodysplastic syndromes.
The panel discusses forward-looking thoughts for the treatment of low-risk MDS.
The panel talks about the use of transplant for patients with low-risk myelodysplastic syndromes.
The panel discusses other treatments undergoing research for MDS, including imetelstat and KER-050.
The panel shared their thoughts on real-world data of luspatercept presented at ASH 2023.
The panel addresses continued MDS research needs.
The panel discusses appropriate treatment selection based on patient mutation status and comorbidities.
The panel shares their thoughts on the full analysis of the COMMANDS study.
Haydar Frangoul, MD, outlined the CLIMB-121 trial data on exagamglogene autotemcel in sickle cell disease.
The panel discussed the current slate of treatment options for patients with low-risk MDS.
Large deletions in the FVIII gene (F8) were shown to be predictors of ITI therapy failure.
Emicizumab plus ITI did not induce new safety signals in patients with hemophilia A.
A single infusion of AAV-HLP-hFVIII-V3 induced stable FVIII expression over five years.
Women with inherited bleeding disorders may need prophylaxis.
First patient treated with hemophilia A gene therapy demonstrates "promising" outcomes.
Dr. Lucia Masarova described data from SIMPLIFY-1 on medical costs and time burden related to outpatient transfusion in MF.
Adults with SCD experience HRQOL burden, such as the inability to work, following a vaso-occlusive crisis.
Pregnancy complications such as cesarean delivery and preeclampsia were more frequent in patients with SCD versus SCD trait.
Patients with severe SCD had clinically meaningful improvements in health-related quality of life after receiving exa-cel.
Children with sickle cell disease (SCD) had significantly higher heme-oxygenase-1 levels versus adults with SCD.
Patients with severe SCD treated with exa-cel achieved no vaso-occlusive crises or inpatient hospital visits.
Non-myeloablative versus myeloablative conditioning for HSCT varied in engraftment, hospitalization, and GVHD outcomes.
Allotransplantation had "equitable" cost-efficacy in pediatric patients with sickle cell disease in the United States.
CLIMB SCD-121 data showed exa-cel treatment induced meaningful improvements in health-related quality of life in SCD.
Recombinant ADAMTS13 treatment exhibited promising safety and efficacy profiles in patients with sickle cell disease.
Exagamglogene autotemcel met primary and key secondary endpoints in the phase III CLIMB-SCD-121 trial on sickle cell disease.
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