ISTH hosted its first-ever virtual congress from July 12-14. ISTH 2020 presented the latest science and education in the fields of thrombosis and hemostasis. More than 15,000 scientists, researchers and physicians representing over 130 countries attended from their homes and workspaces globally. Four members of the HCD Economics haemophilia team attended, in support of data being presented and to participate in relevant sessions.
Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of conditions related to thrombosis and haemostasis. ISTH is an international professional membership organization with more than 5,000 clinicians, researchers and educators working together to improve the lives of patients in more than 100 countries around the world.
The ISTH Presentations covered basic, translational and clinical research, and discussed the diagnostic and therapeutic advances in bleeding and clotting disorders. ISTH 2020 received more than 2,000 scientific abstracts, which were presented as either online posters or oral communications. Poster presenters had the option to add a voice recording to their poster to help explain their research. HCD Economics has published HCD-Economics - RWE perspectives ISTH 2020 to share our learnings and publications from ISTH 2020 with clients and partners.
HCD Economics publications presented as posters included the following, all of which were drawn from research undertaken with partners and clients,
- Associations between Physical Activity Levels and Bleeding Frequency in People with Mild, Moderate, and Severe Hemophilia A (HA): A Preliminary Analysis of the CHESS II Study (PB0943).
- The Relationship between Societal Costs Associated with Haemophilia and Disease Severity: A Regression Analysis Using CHESS II Data (PB0848).
- Examining the Hemophilia Disability Paradox (PB081).
- Feasibility of Using Hospital Episode Statistics in England to Assess the Resource Use and Outcomes in Haemophilia (PB0840).
- Modelling the Cost of Equivalent trough Level across Gene Therapy and Factor IX Replacement Therapy in Haemophilia B (PB0849).
The following oral presentation was also accepted by ISTH 2020 for the Hemophilia and Rare Bleeding Disorders Oral Communication Session, ‘An Insight into Clinical Outcomes in Mild, Moderate, and Severe Hemophilia A (HA): A Preliminary Analysis of the CHESS II Study OC09.3.
The HCD Economics haemophilia team attended a number of sessions. A selection of on-site reports filed by our team is shared below. Reports from all sessions attended by HCD Economics researchers that are of interest to the Haemophilia community have been included in the dossier HCD Economics - RWE perspectives ISTH 2020.
Phase I/II trial of SPK-8011: stable and Durable FVIII expression for >2 years with significant ABR improvements in initial dose cohorts following AAV-mediated gene transfer for Haemophilia A – Lindsey A George. The HCD team report that the trial published encouraging results in gene therapy trials for haemophilia A, in which patients maintained stable levels of FVIII (although still in the haemophilia range), and a 95% reduction in annualised infusion rate and a 91% reduction in ABR.
Severity of acute events in patients with Hereditary Thrombotic Thrombocytopenic Purpura (hTTP) with or without regular plasma prophylaxis: results from the international Hereditary TTP registry – Erika Tarasco. The study reports that no difference was found between the severities of TTP episodes of patients under regular plasma prophylaxis and those who only receive it on demand. However, data suggests that the initiation of prophylaxis may be beneficial after the occurrence of severe events. Discontinuation of prophylaxis, on the other hand, was followed by severe TTP acute events. Plasma prophylaxis schedules that are currently used to prevent occurrences of TTP episodes are not sufficient for prevention in hTTP. hTTP has over 150 mutations that have been currently identified, with hugely different disease courses. While plasma prophylaxis seems somewhat effective in preventing events, they still do occur. Additionally, this therapy comes with its drawbacks (hugely time consuming, possible infections and risk of thrombosis, as well as risk of allergic reactions).
Longitudinal assessment of ADAMTS13-activity helps predict recurrence of immune thrombotic thrombocytopenic purpura (iTTP): results from the German iTTP registry – Charis von Auer-Wegener This presentation reported a Quarterly assessment of ADAMTS13 activity and preventive administration of rituximab is crucial in preventing relapse of iTTP. Declines in ADAMTS13 levels were strongly associated with the occurrence of relapses, suggesting that use of rituximab and caplacizumab maybe effective therapies in preventing relapses.
An International Multicenter Study of Bevacizumab for Bleeding in Hereditary Hemorrhagic Telangiectasia (The InHIBIT-Bleed Study) - Hanny Al-Samkari HCD Economics found this an Extremely interesting session outlining the unmet need for a disease that is the second most common bleeding disorder (rare disease that causes GI bleeds and epistaxis,with a conseuquence of severe anaemia and iron deficiency) but still without and FDA approved treatment. The study showed promising results in patients treated with Bevacizumab where maintenance treatment helped normalising HB levels as well as iron levels as a result of reduced bleeding. It is still unclear whether when maintenance is stopped patients revert to the initial level of bleeding right away or if a longer time is necessary.
Thrombosis, Bleeding, and the Effect of Anticoagulation on Survival in Critically Ill Patients with COVID-19 in the United States - Hanny Al-Samkari. This cross-sectional study was aimed at assessing whether administration of prophylactic anticoagulation improved survival. Results suggest that early initiation of anticoagulants after ICU admission does not affect survival.
Please contact Lesley Piltz at HCD Economics, to schedule a time when our team can present the HCD Economics – RWE Perspectives Dossier, Lesley can also provide links and introductions to the studies published by HCD Economics at ISTH 2020 – lesley.piltz@hcdeconomics.com
*HCD Economics - RWE perspectives are a series of ad hoc dossiers, created by the team at HCD Economics. The concept is to share our learnings from attending the major congresses that cover our research areas. The reports are focused on the research that is most pertinent to our research interests and the research interests of our clients and partners. The format is a powerpoint deck, presented by ZOOM or equivalent, to allow those interested in the reports to address questions and discuss matters arising from the report contents