64th ASH Annual Meeting and Exposition: 904. Outcomes Research—Non-Malignant Conditions: Sickle Cell Disease Therapies & Outcomes

Symposia: Outcomes Research—Non-Malignant Conditions Oral and Poster Abstracts
Oral

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

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

577

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

578

Trends of Disease Modifying Treatment Use in Sickle Cell Disease: Retrospective Analysis of Claims Data, 2014-2020

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

579

Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study

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

580

Quality of Life and Personal Life Goals of Adult Sickle Cell Disease Patients after Curative Allogeneic Stem Cell Transplantation: A Mixed Methods Study

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

581

Gene Therapy Equity in Sickle Cell Disease: Distributional Cost-Effectiveness Analysis (DCEA) of Gene Therapy Vs. Standard-of-Care in Patients with Sickle Cell Disease in the United States

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

582

Preliminary Results of Promis Sexual Function and Satisfaction and Perceived Risk for Infertility after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Stellar Study

Lillian R Meacham, MD^1,2,3^*, Anirudh Veludhandi, BS²^*, Yijin Xiang, MPH⁴^*, Elizabeth Stenger, MD, MSc², Staci D. Arnold, MD, MBA, MPH², Akanksha Mehta, MD⁵^*, Lydia H. Pecker, MD^6,7, Michael Hsieh, MD, PhD⁸^*, Jacqueline Y Maher, MD^9,10^* 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

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^*signifies non-member of ASH

904.O2.6 904. Outcomes Research—Non-Malignant Conditions: Sickle Cell Disease Therapies & Outcomes