65th ASH Annual Meeting & Exposition: 904. Outcomes Research – Non-Malignant Conditions: What to Know: Management Costs and Outcomes in Various Non-Malignant Disorders

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

Research, health outcomes research, Clinical Research

Pacific Ballroom Salons 15-17 (Marriott Marquis San Diego Marina)

Moderators:

Kleber Yotsumoto Fertrin, MD, PhD, University of Washington and Bart J. Biemond,

Disclosures:

Fertrin: Agios Pharmaceuticals: Consultancy, Research Funding, Speakers Bureau; Sanofi Genzyme: Consultancy; Pfizer: Consultancy; bluebird bio: Consultancy.

9:30 AM

487

Patient-Reported Improvements in Fatigue and Health-Related Quality of Life in the Phase 3 Studies Apply-PNH and Appoint-PNH Evaluating the Use of Iptacopan in C5 Inhibitor-Treated and Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

Antonio M Risitano, MD, PhD^1,2, Carlos M de Castro, MD³, Bing Han, MD, PhD⁴^*, Austin Kulasekararaj, MD, MBBS, FRCPath, MRCP^5,6,7, Jaroslaw P. Maciejewski, M.D., Ph.D., FACP⁸, Phillip Scheinberg, MD⁹, Yasutaka Ueda, MD, PhD¹⁰, Susan Vallow¹¹^*, Georgina Bermann¹²^*, Marion Dahlke, MD, MSc¹³^*, Rakesh Kumar¹⁴^* and Regis Peffault de Latour^15,16^*

¹AORN Moscati, Avellino, Italy
²University of Naples Federico II, Naples, Italy
³Duke University School of Medicine, Durham, NC
⁴Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, AL, China
⁵King’s College Hospital NHS, London, United Kingdom
⁶National Institute for Health and Care Research and Wellcome King’s Research Facility, London, United Kingdom
⁷King’s College London, London, United Kingdom
⁸Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
⁹Hospital A Beneficência Portuguesa, São Paulo, Brazil
¹⁰Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Suita, Japan
¹¹Novartis Pharmaceuticals Corporation, East Hanover, NJ
¹²Novartis Pharma AG, Basel, Switzerland
¹³Novartis, Basel, Switzerland
¹⁴Novartis Healthcare Private Limited, Hyderabad, India
¹⁵French Référence Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France
¹⁶Assistance Publique Hôpitaux de Paris, Université Paris Cité, Paris, France

9:45 AM

488

Cancer Risk in Patients with Gaucher Disease Using Real-World Data

Shoshana Revel-Vilk, MD, MSc^1,2,3, Ari Zimran, MD^1,2, Majdolen Istaiti¹^*, Orly Manor, PhD³^*, Varda Shalev, MD⁴^*, Litat Azani⁵^*, Gabriel Chodick^4,5^* and Ora Paltiel^2,3^*

¹Gaucher Unit, Shaare Zedek Medical Center, Jerusalem, Israel
²Faculty of Medicine, Hebrew University, Jerusalem, Israel
³Braun School of Public Health and Community Medicine, Hebrew University, Jerusalem, Israel
⁴Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
⁵MaccabiTech, Maccabi Healthcare Services, Tel Aviv, Israel

10:00 AM

489

Defining the Impacts of Iron Overload in Children Receiving Hematopoietic Stem Cell Transplantation for Non-Malignant Hematologic Conditions

Nora M Gibson, MD¹, Sandra Amaral, MD, MHS²^*, Jesse Y Hsu, PhD³^*, Timothy S Olson, MD, PhD⁴ and Janet L. Kwiatkowski, MD, MSCE⁵

¹Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Nephrology, Children's Hospital of Philadelphia, Philadelphia, PA
³Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
⁴Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
⁵Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA

10:15 AM

490

Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States

George Goshua, MD, MSc¹, Satoko Ito, MD PhD¹, Karthik Chetlapalli, MS, BS², Kunal C Potnis, MD³^*, Cecelia Calhoun, MD, MBA, MPH¹, Lakshmanan Krishnamurti, MD⁴, Harlan M. Krumholz, MD, SM^5,6^* and Ankur Pandya, PhD⁷^*

¹Section of Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT
²Yale School of Medicine, New Haven, CT
³Department of Internal Medicine, Yale School of Medicine, New Haven
⁴Section of Pediatric Hematology, Oncology and Bone Marrow Transplant, Yale School of Medicine, Atlanta, GA
⁵Center for Outcomes Research and Evaluation, Yale New Haven Hospital, New Haven, CT
⁶Section of Cardiovascular Medicine, Department of Internal Medicine, Yale School of Medicine, New Haven, CT
⁷Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, Boston, MA

10:30 AM

491

Real-World Analysis of the Underdiagnosis, Clinical Outcomes and Associated Burden of Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA) in the United States of America

Yan Wang¹^*, Andrew Rava, MPH²^*, Marlene Smuzynski³^*, Bonny Shah³^*, Anusorn Thanataveerat³^*, Imad Al Dakkak¹^*, Moh-Lim Ong⁴^*, Christopher C Dvorak, MD⁵ and Vincent Ho, MD⁶^*

¹Alexion, AstraZeneca Rare Disease, Boston
²Genesis Research, Hoboken, NJ
³Genesis Research, Hoboken
⁴Alexion, AstraZeneca Rare Disease, Boston, MA
⁵University of California, San Francisco, Millbrae, CA
⁶Dana-Farber Cancer Institute, Boston, MA

10:45 AM

492

Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States

Satoko Ito, MD, PhD¹, Kunal C Potnis, MD²^*, Cecily Allen, MD³, Rhys Richmond, BS⁴^*, Christina Waldron, BS⁴, Giri Viswanathan⁵^*, Robert D Bona, MD⁶ and George Goshua, MD, MSc¹

¹Section of Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT
²Department of Internal Medicine, Yale School of Medicine, New Haven
³Division of Hematology, Department of Medicine, Johns Hopkins Hospital, Baltimore, Md
⁴Yale School of Medicine, New Haven, CT
⁵Yale College, New Haven, CT
⁶Section of Hematology, Department of Internal Medicine, Yale University School of Medicine, New Haven, CT

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^*signifies non-member of ASH

904.O3.6 904. Outcomes Research – Non-Malignant Conditions: What to Know: Management Costs and Outcomes in Various Non-Malignant Disorders