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ASCAT 2020 Abstract Book ABSTRACT BOOK October 26-31Virtual Global Conference Word of Welcome On behalf of the Annual Scientific Conference on Sickle Cell and Thalassaemia (ASCAT), the European Hematology Association (EHA), and the British Society for Haematology (BSH), we are pleased to present our 2020 Abstract Book. This year’s theme is ‘Haemoglobinopathies: Emerging Challenges & Future Therapies’. The Annual Scientific Conference on Sickle Cell and Thalassaemia is one of the must attend events of the year for consultants and specialist psychologists, nurses, scientists and all relevant experts. The event is an ideal opportunity to see the latest advances in diagnosis, treatment and emerging fields in haemoglobinopathies. It is an opportunity to interact on the latest advances in clinical care, transition services and emerging new therapies including updates for curative treatment options. Abstract and poster presentations will take place during the six days of this year’s scientific meeting covering key areas in Sickle Cell and Thalassaemia. The accepted abstracts are published in this official HemaSphere supplement. On behalf of the joint leadership of ASCAT, EHA, BSH, and the esteemed Steering Committee and abstract reviewers, we would like to welcome you to this year’s momentous congress, bringing to you a comprehensive programme on Sickle Cell Disease and Thalassemia; have a pleasant experience. Professor Baba Inusa Professor Maria Domenica Cappellini Katy Amberley ASCAT President EHA Chair BSH CEO TABLE OF CONTENTS Basic Science Abstracts .................................................................................................................. 1 Psychology, QOL and Patient Outcomes Abstracts ......................................................................... 13 Clinical and Community Implementation Research Abstracts ......................................................... 18 Public Health and Health Education Abstracts ................................................................................ 28 Aging and Haemoglobinopathies Abstracts ..................................................................................... 34 Other Aspects of Haemoglobinopathies Abstracts .......................................................................... 34 Supplement Information The Abstract Book of the 15th Annual Sickle Cell and Thalassaemia, 1st EHA European Sickle Cell Conference, and 60th Anniversary of BSH is published as a supplement of HemaSphere and is also available online at www.hemaspherejournal.com. HemaSphere is an open access journal powered by the European Hematology Association (EHA) and dedicated to supporting hematology patient care, research, and education worldwide. Copyright Information (Online) ISSN: 2572-9241 © 2020 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the Annual Scientific Conference on Sickle Cell and Thalassaemia (ASCAT), the British Society for Haematology (BSH), and the European Hematology Association (EHA). This is an open access Abstract Book distributed under the Attribution-NonCommercial-NoDerivs (CC BY-NC-ND) which allows third parties to download the articles and share them with others as long as they credit the author and the Abstract Book, but they cannot change the content in any way or use them commercially. Rights and Permissions Please refer to https://journals.lww.com/hemasphere/_layouts/15/1033/oaks.journals/rightsandpermissions.aspx for reprints and order of copies of this abstract book. Questions regarding permissions should be addressed to [email protected]. Abstract Book Citations: Authors, Abstract Title, HemaSphere, 2020;4:S3, 15th Annual Sickle Cell and Thalassaemia, 1st EHA European Sickle Cell Conference, and 60th Anniversary of BSH Abstract Book, DOI: http://dx.doi.org/10.1097/HS9.0000000000000498 Disclaimer Articles published in the journal HemaSphere exclusively reflect the opinions of the authors. The authors are responsible for all content in their abstracts including accuracy of the facts, statements, citing resources, etc. Abstract Book for the 15th Annual Sickle Cell and Thalassaemia, 1st EHA European Sickle Cell Conference, and 60th Anniversary of BSH profile provide a basis for developing guidelines on utilization of approved BASIC SCIENCE ABSTRACTS safe therapeutics such as Endari to reduce severe ARDS and to reduce ACS in SCD patients in regions with prevalent COVID-19 until effective vaccines are widely available and improve outcomes. L-GLUTAMINE ACTIONS IN SICKLE CELL DISEASE INDICATE BENEFIT VS. COVID-19 Table 1 – COVID-19 Pathology and Glutamine M.A.Z. Abdelaal1, D. Abdelrahman2, M. Cengiz3, H. Yavuzer4, COVID-19 Adverse Effects GLN Actions of Potential Benefit S. Yavuzer3, I. Bien5, P. Bhuva5, J.V. Pham5, R. Siu5, M. Tang6, A. Ward7, J. Goodrow7, P. Ludlum7, C.W. Stark5,7, S.P. Perrine8,9 Acute respiratory distress syn- GLN deficiency in inflammatory drome (ARDS) in COVID-19 is states in ICU patients is associat- 1 2 Benha University Faculty of Medicine, Benha, Arab Republic of Egypt; Medical a primary cause of severe disease, ed with higher mortality, which 3 University of Vienna, Vienna, Austria; Biruni University Medical Faculty, Istanbul, ICU admissions, prolonged hospi- GLN reduces. For experimentally 4 5 Turkey; Istanbul University-Cerrahpasa, Istanbul, Turkey; University of Southern talization, and mortality. induced ARDS caused by sepsis, 6 California, School of Pharmacy, Los Angeles, CA, USA; Kaiser Permanente, Los aspiration, and endotoxin, GLN 7 8 Oxidative damage to the lung and Angeles, CA, USA; Emmaus Medical, Inc., Torrance, CA, USA; Boston University has beneficial effects: School of Medicine, Boston, MA, USA; 9Phoenicia BioScience, Weston, MA, USA cytokine storm, hyperactive im- mune responses to COVID-19, are 1. Decreases pulmonary consol- Background: Acute respiratory distress syndrome (ARDS) is a leading reported as primary contributors idation, edema, and neutrophil cause of mortality from SARS-coronavirus2 (SARSCoV2). Severe coro- to ARDS. infiltration navirus disease 2019 (COVID-19) disproportionally affects African Amer- Deficient plasma GLN (< 420 2. Increases lung compliance, ican and Hispanic populations, the racial groups most affected by sickle cell µmol/L) is a defined risk for higher heat shock protein activation, disease (SCD) in the US, which the Centers for Disease Control designates mortality in ICU and COVID-19 oxygen saturation, at increased risk for severe COVID-19. Global fatalities are reported to the patients. SCD COVID-19 registry in all age ranges, from asymptomatic to severely ill 3. Increases survival by 2.5-fold subjects. Repurposing of available safe therapeutics could benefit high-risk (Huang, Lancet, 2020; Santos, over saline controls. populations until effective vaccines are widely available. Amino Acids, 2019; Shen, Cell, (Perng WC, Clin Exp Pharmacol Multiple actions of glutamine (GLN) ameliorate elements of the clinical pa- 2020). Physiol, 2010; Singleton, Crit thology of SCD which also contribute to ARDS in COVID-19. Pharmaceuti- (Polonikov, ACS Infect Dis, Care Med, 2005; Shen, Cell, cal-grade L-glutamine (PGLG) (Endari, Emmaus Medical) is FDA-approved 2020). 2020). for reducing sickle cell crises in patients ≥ 5 years of age. A Ph3 trial had 63% fewer occurrences of acute chest syndrome (ACS). Annualized median rates Increased proinflammatory cy- GLN modulates inflammatory re- of sickle cell crises for the trial were 4.30 in controls vs 2.37 in GLN-treated tokines and elevated interleukin 6 sponses by suppressing C-reactive subjects (45% fewer occurrences, p=0.02), median hospital days were 13.17 (IL-6) levels contribute to severity protein, IL6, and TNF-a release. in controls vs 8.45 in GLN-treated (p=0.04). PGLG decreases red cell-en- of COVID-19 (Chuang, BMC Pulm Med, 2014; dothelial adhesion and oxidative stress by increasing the ratio of reduced nic- (Yuki, Clin Immunol, 2020). 37% decrease in IL-6, p < 0.05). otinamide adenine dinucleotide (NAD) to total NAD, which may increase reduced glutathione. Multiple complications of COVID-19 are now attributed Myocardial injury in COVID-19 GLN protects against similar to oxidative damage and elevated cytokines such as IL-6, which GLN modu- occurs by viral entry through myocardial damage in related lates. These actions may have important relevance in the pandemic. ACE-2 receptors, microvascular conditions. Recent computational modeling screens of approved therapeutics, directed to damage, endothelial shedding, and viral protein and chemistry binding targets, and to gene expression changes inflammation-mediated damage. (Shao, Pak J Med Sci, 2015). induced by SARSCoV2 in human lung cells, predict GLN and glutathione (Shi, Eur Heart J, 2020; Shi, should confer benefit in COVID-19 (Kim, J Translat Med, 2020). JAMA Cardiol, 2020). Methods: To investigate actions of GLN relevant to COVID-19, we reviewed reports of multi-system effects of GLN in experimental ARDS models and ICU and COVID19 patients. We also conducted a clinical trial in hospitalized Table 2 – Controlled Clinical Trial of Oral L-Glutamine in COVID-19 COVID19 patients of standard care +/- GLN. Patients Results: Multiple studies demonstrate that SARS-CoV-2 causes oxidative Patients were admitted with COVID-19 confirmed by RT-PCR and lung damage and severe respiratory disease, which are associated with GLN positive CT scans. Balanced groups received ESPEN recommended and glutathione deficiency. Table 1 summarizes beneficial actions of GLN nutrition for COVID
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