-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
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904.O2.6 904. Outcomes Research—Non-Malignant Conditions: Sickle Cell Disease Therapies & Outcomes

Symposia: Outcomes Research—Non-Malignant Conditions Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Research, Sickle Cell Disease, Biological therapies, adult, epidemiology, Sickle Cell Trait, Clinical Practice (Health Services and Quality), health outcomes research, Clinical Research, Genetic Disorders, health disparities research, Diversity, Equity, and Inclusion (DEI) , Hemoglobinopathies, pediatric, patient-reported outcomes, Diseases, Gene Therapy, Therapies, real-world evidence, registries, Adverse Events, young adult , survivorship, Study Population, Human
Sunday, December 11, 2022: 12:00 PM-1:30 PM
R06-R09 (Ernest N. Morial Convention Center)
Moderators:
Olubusola Oluwole, MD, MS, University of Pittsburgh School of Medicine and Sherif M. Badawy, MD, Ann %26 Robert Lurie Children's Hospital
Disclosures:
No relevant conflicts of interest to declare.
This session highlights current treatment outcomes and upcoming therapies in sickle cell disease
12:00 PM

Jennifer Drahos1*, Adriana Boateng-Kuffour1*, Melanie Calvert2*, Ashley Valentine3*, Anthony Mason4*, Zahra Pakbaz, MD5, Fikret Kaan Oran6*, Kamran Iqbal6*, Farrukh Shah7*, Nanxin Li1 and Antony P. Martin8*

1Vertex Pharmaceuticals Incorporated, Boston, MA
2Institute of Applied Health Research, University of Birmingham, Birmingham, United Kingdom
3Sick Cells, Washington, DC
4Sickle Cell Care Manchester, Manchester, United Kingdom
5Division of Hematology/Oncology, The University of California Irvine School of Medicine, Orange, CA
6Vertex Pharmaceuticals (Europe) Ltd, London, United Kingdom
7NHS Blood and Transplant, London, United Kingdom
8QC Medica, Liverpool, United Kingdom

12:15 PM

Terri Newman, MS, PharmD1, Jingye Yang, MS1*, Kangho Suh, PharmD, PhD1*, Charles Jonassaint, PhD1, Sandra Kane-Gill, MSc, PharmD1* and Enrico M Novelli, M.D., M.S.2

1University of Pittsburgh, Pittsburgh, PA
2Heart, Lung and Blood Vascular Medicine Institute, University of Pittsburgh, Pittsburgh, PA

12:30 PM

Sobenna A George, MD1*, Anirudh Veludhandi, BS2*, Yijin Xiang, MPH3*, Elizabeth Stenger, MD, MSc4, Staci D. Arnold, MD, MBA, MPH4, Akanksha Mehta, MD5*, David A Schirmer, MD6*, Jessica B Spencer, MD, MSc6*, Gregory M Guilcher, MD, BSc, FRCPC7*, Monica Bhatia, MD8*, Allistair Abraham, MD9, Veronica Gomez-Lobo, MD10*, Lakshmanan Krishnamurti, MD11 and Lillian R Meacham, MD4*

1Division of Endocrinology, Department of Pediatrics, Children's Healthcare of Atlanta/Emory University, Atlanta, GA
2Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA
3Pediatric Biostatistics Core, Children's Healthcare of Atlanta/Emory University, Atlanta, GA
4Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta/Emory University, Atlanta, GA
5Department of Urology, Emory University School of Medicine, Atlanta, GA
6Division of Reproductive Endocrinology & Infertility, Department of Gynecology and Obstetrics, Emory University School of Medicine, Atlanta, GA
7Section of Oncology/Cellular Therapy, Department of Oncology and Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada
8Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Columbia University Medical Center, New York, NY
9Children's National Hospital, Washington, DC
10Pediatric and Adolescent Gynecology, National Institute of Child Health and Human Development, Bethesda, MD
11Section of Pediatric Hematology/Oncology/BMT, Yale School of Medicine, New Haven, CT

12:45 PM

Elisabeth Dovern, MD1*, Sterre J.A.M. Nijland1*, Maud M. Van Muilekom, PhD2*, Liesbeth E.M. Suijk1*, Hilda Mekelenkamp3*, Bart J Biemond1, Lotte Haverman, PhD2* and Erfan Nur, MD, PhD1

1Department of Hematology, Amsterdam UMC location University of Amsterdam, Amsterdam, Netherlands
2Child and Adolescent Psychiatry & Psychosocial Care, Amsterdam UMC location University of Amsterdam, Emma Children's Hospital, Amsterdam, Netherlands
3Department of pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, Netherlands

1:00 PM

George Goshua, MD, MSc1, Cecelia Calhoun, MD, MBA, MPH1, Vivien Cheng, MPH2*, Lyndon James, MD MPH2*, Andrea Luviano, MD MPH2*, Lakshmanan Krishnamurti, MD3 and Ankur Pandya, PhD2*

1Section of Hematology, Yale University School of Medicine, New Haven, CT
2Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, Boston
3Section of Pediatric Hematology/Oncology/BMT, Yale School of Medicine, New Haven, CT

1:15 PM

Lillian R Meacham, MD1,2,3*, Anirudh Veludhandi, BS2*, Yijin Xiang, MPH4*, Elizabeth Stenger, MD, MSc2, Staci D. Arnold, MD, MBA, MPH2, Akanksha Mehta, MD5*, Lydia H. Pecker, MD6,7, Michael Hsieh, MD, PhD8*, Jacqueline Y Maher, MD9,10* and Lakshmanan Krishnamurti, MD11

1Pediatric Endocrinology, Emory University, Atlanta, GA
2Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta/Emory University, Atlanta, GA
3Pediatric Endocrinology, Children's Healthcare of Atlanta/Emory University, Atlanta, GA
4Pediatric Biostatistics Core, Children's Healthcare of Atlanta/Emory University, Atlanta, GA
5Department of Urology, Emory University School of Medicine, Atlanta, GA
6Division of Hematology, Department of Medicine, Johns Hopkins Medicine, Baltimore, MD
7Gynecology & Obstetrics, Johns Hopkins University School of Medicine, Baltimore, MD
8Urology, Children's National Hospital/The George Washington University, Washington, DC
9Pediatric and Adolescent Gynecology, Children's National Hospital, Washington, DC
10National Institutes of Health, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda
11Section of Pediatric Hematology/Oncology/BMT, Yale School of Medicine, New Haven, CT

*signifies non-member of ASH