S1, The 2nd Sickle Cell and Thalassaemia Virtual Conference Abstract Book 2022: HemaSphere: Vol 6, No S1

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HemaSphere: Volume 6, Issue S1

S1, The 2nd Sickle Cell and Thalassaemia Virtual Conference Abstract Book 2022

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Oral Presentations

Basic and Translational

S102: OBLIGATE N-TERMINAL BUT NOT C-TERMINAL MONOFERRIC TRANSFERRIN AMELIORATES ANEMIA IN β-THALASSEMIC MICE

Abstract

Abstract Book for the 2nd Sickle Cell & Thalassaemia Virtual Conference

Oral Presentations

Basic and Translational

S101: COMBINATION OF A LUSPATERCEPT-LIKE DRUG (RAP-GRL) AND TMPRSS6-ASO IS SUPERIOR TO EITHER DRUG ALONE FOR CORRECTING β-THALASSEMIA

S103: TRIAL IN PROGRESS: A PHASE 2, OPEN-LABEL STUDY EVALUATING THE SAFETY AND EFFICACY OF THE PKR ACTIVATOR ETAVOPIVAT (FT-4202) IN PATIENTS WITH THALASSEMIA OR SICKLE CELL DISEASE

Novel Therapies, Gene Therapies, Bone Marrow Transplant and Emerging Diagnostics

S104: A SEVERE MOUSE MODEL OF ALPHA-THALASSEMIA SHOWS ABNORMAL IRON METABOLISM, ERYTHROPOIESIS AND COAGULATION, AND CAN BE RESCUED BY A NOVEL GENE THERAPY APPROACH

S105: ADDING AZATHIOPRINE/HYDROXYUREA PRECONDITIONING TO ALEMTUZUMAB/TBI CONDITIONING IMPROVES DONOR CHIMERISM IN MATCHED SIBLING DONOR STEM CELL TRANSPLANTATION IN ADULT SICKLE CELL DISEASE PATIENTS

S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY

S107: P-SELECTIN INHIBITOR INCLACUMAB REDUCES CELL ADHESION IN AN IN-VITRO ASSAYS SHOWING POTENTIAL FOR PREVENTION OF VASO-OCCLUSION EVENTS IN SICKLE CELL DISEASE

Clinical and Epidemiological Studies

S108: A SYSTEMATIC POST-MARKETING (PM) REVIEW OF RARE INFUSION-RELATED REACTIONS (IRRS) PRESENTING AS PAIN EVENTS DURING OR AFTER CRIZANLIZUMAB INFUSION IN PATIENTS WITH SICKLE CELL DISEASE (SCD)

S109: ACTIVATION OF PYRUVATE KINASE-R WITH ETAVOPIVAT (FT-4202) IS WELL TOLERATED, IMPROVES ANEMIA, AND DECREASES INTRAVASCULAR HEMOLYSIS IN PATIENTS WITH SICKLE CELL DISEASE TREATED FOR UP TO 12 WEEKS

S110: ACUTE AND CHRONIC PAIN MANAGEMENT IN SICKLE CELL DISEASE: OUTCOMES OF AN ENGLISH NATIONAL AUDIT.

S111: BRAIN PERFUSION CHANGES IN BETA-THALASSEMIA

S112: COMPARATIVE SCORE OF HOWELL-JOLLY BODIES IN SICKLE CELL DISEASE PATIENTS ACCORDING TO GENOTYPE, AGE, SPLENECTOMY OR STEM CELL TRANSPLANTATION HISTORY

S113: COST OF ILLNESS, EPIDEMIOLOGY AND HEALTHCARE RESOURCE UTILIZATION FOR SICKLE CELL DISEASE IN GULF COUNTRIES (CESCGU)

S114: FEASIBILITY OF HAPLOIDENTICAL ALLOGENEIC STEM CELL TRANSPLANTATION WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE FOR PATIENTS WITH SEVERE SICKLE CELL DISEASE WITH END-STAGE RENAL DISEASE (ESRD) ON HEMODIALYSIS

S115: LONG-TERM EFFICACY AND SAFETY OF DEFERIPRONE FOR PATIENTS WITH SICKLE CELL DISEASE OR OTHER ANEMIAS

S116: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON—TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

S117: LONG-TERM FOLLOW UP OF DUTCH PATIENTS WITH SCD DIAGNOSED BY NEONATAL SCREENING -- EFFECT ON THE MORBIDITY AND MORTALITY IN THE NETHERLANDS.

S118: LONG-TERM SAFETY AND EFFICACY OF VOXELOTOR FOR PATIENTS WITH SICKLE CELL DISEASE: RESULTS FROM AN OPEN-LABEL EXTENSION OF THE PHASE 3 HOPE TRIAL

S119: PRELIMINARY RESULTS OF A PHASE 1 STUDY IN HEALTHY SUBJECTS ADMINISTERED INCLACUMAB, A FULLY HUMAN IGG4 ANTI-P-SELECTIN MONOCLONAL ANTIBODY IN DEVELOPMENT FOR TREATMENT OF SICKLE CELL DISEASE

S120: RANDOMIZED CONTROLLED TRIAL OF THE EFFICACY AND SAFETY OF DEFERIPRONE: SUBGROUP ANALYSIS OF PEDIATRIC PATIENTS IN IRON-OVERLOADED PATIENTS WITH SICKLE CELL DISEASE AND OTHER ANEMIAS

S121: REDUCED RATE OF VASO-OCCLUSIVE CRISES (VOCS) IN PATIENTS (PTS) WITH SICKLE CELL DISEASE (SCD) TREATED WITH CRIZANLIZUMAB FOR 12 MONTHS: RESULTS FROM A REAL-WORLD, MANAGED ACCESS PROGRAM (MAP)

S122: SAFETY AND EFFICACY OF CRIZANLIZUMAB IN ADOLESCENTS WITH SICKLE CELL DISEASE (SCD): INITIAL DATA FROM THE PHASE II, MULTICENTER, OPEN-LABEL SOLACE-KIDS TRIAL

S123: SAFETY AND EFFICACY OF EARLY-START DEFERIPRONE IN INFANTS AND YOUNG CHILDREN WITH BETA-THALASSEMIA (START STUDY)

Infection, Autoimmunity, Nutritional Deficiencies

S124: ADVERSE EVENTS FOLLOWING COVID-19 VACCINATION IN TRANSFUSION-DEPENDENT -THALASSEMIA PATIENTS

S125: THE ROLE OF THE MICROBIOME AS A SICKLE CELL DISEASE MODIFIER: AN EXPERT REVIEW

Health Services and Outcomes Research Including Psychology

S126: AN EU -- ARISE INITIATIVE GAP ANALYSIS APPROACH TO IMPROVING THE QUALITY OF LABORATORY SYSTEMS IN SUB-SAHARAN AFRICA.

S127: CONSORTIUM ON NEWBORN SCREENING IN AFRICA FOR SICKLE CELL DISEASE

S128: CONTINUOUS QUALITY IMPROVEMENT IN PAEDIATRIC SICKLE CELL DISEASE

S129: IMPLEMENTATION OF HYDROXYUREA THERAPY FOR SICKLE CELL DISEASE ON A LARGE SCALE IN GHANA

S130: PROCESSING SPEED DECLINES OVER TIME IN 4--25-YEAR-OLDS WITH SICKLE CELL DISEASE

S131: REGIONAL ASSESSMENT OF THE EXPERIENCES OF HEALTHCARE PROFESSIONALS (HCPS) TREATING PATIENTS WITH SICKLE CELL DISEASE (SCD): THE INTERNATIONAL SICKLE CELL WORLD ASSESSMENT SURVEY (SWAY)

Poster Presentations

Basic and Translational

P101: GUT MICROBIOTA IMPACT ON ANGOLAN CHILDREN WITH SICKLE CELL DISEASE

P102: PLASMA FREE HEMOGLOBIN AND REACTIVE OXYGEN SPECIES IN SICKLE CELL ANAEMIA PATIENTS UNDER HYDROXYUREA AND GLUTAMINE THERAPY

P103: THE ABNORMAL PRESENCE OF MITOCHONDRIA IN CIRCULATING SCD RED BLOOD CELLS ASSOCIATED WITH STRESS ERYTHROPOIESIS

Novel Therapies, Gene Therapies, Bone Marrow Transplant and Emerging Diagnostics

P104: BASE EDITING REPAIRS THE HBE MUTATION RESTORING THE PRODUCTION OF NORMAL GLOBIN CHAINS IN SEVERE HBE/β-THALASSEMIA PATIENT HEMATOPOIETIC STEM AND ERYTHROID CELLS

P105: BLOOD MICROVESICLES AS BIOMARKERS TO PREDICT VASO-OCCLUSIVE CRISIS IN SICKLE CELL ANEMIA

P106: EARLY PREVENTIVE DIAGNOSIS OF HEMOLYTIC ANEMIA IN SICKLE CELL PATIENTS BY DETECTING THE TRIGGERING OF ERYPTOSIS.

Clinical and Epidemiological Studies

P107: A PHASE 2/3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF MITAPIVAT IN PATIENTS WITH SICKLE CELL DISEASE

P108: ASSESSMENT OF HEALTHCARE WORKERS’ KNOWLEDGE AND RESOURCE AVAILABILITY FOR CARE OF SICKLE CELL DISEASE AT HEALTH FACILITIES IN DAR ES SALAAM, TANZANIA

P109: CLINICAL CATEGORIZATION OF CHILDREN WITH SICKLE CELL DISEASE USING SEVERITY SCORE AT A TERTIARY CARE CENTER

P110: CLINICAL CHARACTERISTICS OF COVID-19 IN SICKLE CELL DISEASE (SCD) PATIENTS

P111: COVID-19 IN PATIENTS WITH THALASSEMIA AND SICKLE CELL DISEASE: A SINGLE CENTER EXPERIENCE

P112: ENERGIZE AND ENERGIZE-T: TWO PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDIES OF MITAPIVAT IN ADULTS WITH NON--TRANSFUSION-DEPENDENT OR TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

P113: INCIDENCE OF HAEMOGLOBINOPATHY IN SLOVAKIA

P114: NEWBORN SCREENING FOR HAEMOGLOBINOPATHIES IN BIDA, NORTH CENTRAL NIGERIA.

P115: REAL-WORLD DATA ON THE EFFICACY OF PHARMACEUTICAL-GRADE L-GLUTAMINE IN PREVENTING SICKLE CELL DISEASE-RELATED ACUTE COMPLICATIONS AND HEMOLYSIS IN PEDIATRIC AND ADULT PATIENTS.

P116: THE IMPACT OF COVID19 PANDEMIC ON SICKLE CELL MANAGEMENT: EXPERIENCE OF A SINGLE PEDIATRIC CENTER

P117: THE INTERNATIONAL HAEMOGLOBINOPATHY RESEARCH NETWORK (INHERENT): AN INTERNATIONAL INITIATIVE TO STUDY THE ROLE OF GENETIC MODIFIERS IN HAEMOGLOBINOPATHIES

P118: THE ROLE OF HEMOGLOBIN AND HEMOLYSIS ON TRANSCRANIAL DOPPLER VELOCITIES IN CHILDREN WITH SICKLE CELL DISEASE: DATA FROM A NATURAL HISTORY COHORT

P119: TRIAL IN PROGRESS: THE RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 1B CROSSWALK-A TRIAL EVALUATING THE SAFETY OF CROVALIMAB FOR THE MANAGEMENT OF ACUTE UNCOMPLICATED VASO-OCCLUSIVE EPISODES (VOES) IN PATIENTS (PTS) WITH SICKLE CELL DISEASE (SCD)

P120: TRIAL IN PROGRESS: THE RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 2A CROSSWALK-C TRIAL EVALUATING THE EFFICACY OF CROVALIMAB AS ADJUNCT TREATMENT IN THE PREVENTION OF VASO-OCCLUSIVE EPISODES (VOES) IN PATIENTS (PTS) WITH SICKLE CELL DISEASE (SCD)

Infection, Autoimmunity, Nutritional Deficiencies

P121: PREVALENCE OF VERTEBRAL COMPRESSION FRACTURE AND HYPOVITAMINOSIS D IN CHILDREN WITH THALASSEMIA MAJOR

Ageing and End Organ Damage

P122: DEEP LEARNING-BASED ALGORITHM FOR AUTOMATIC SPLEEN LENGTH MEASUREMENT

Health Services and Outcomes Research Including Psychology

P123: A SYSTEMATIC REVIEW OF THE BARRIERS TO UPTAKE AND ADHERENCE TO HYDROXYCARBAMIDE FROM THE PERSPECTIVE OF CHILDREN AND ADULTS WITH SICKLE CELL DISEASE, PARENTS AND CLINICIANS.

P124: EFFICACY OF NON-PHARMACOLOGICAL INTERVENTIONS TO REDUCE PAIN IN CHILDREN WITH SICKLE CELL DISEASE: A SYSTEMATIC REVIEW

P125: EMPOWERING YOUNG ADOLESCENTS ON THE TRANSITION BY THE EDUCATION METHOD ORBIS SENSUALIUM PICTUS - THEIR OWN IMPROVING BLOOD CELL MORPHOLOGY AND MEDICAL RESULTS ON ACTIVE TREATMENT