66th ASH Annual Meeting: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster I

Symposia: Disorders of Platelet Number or Function: Clinical and Epidemiological Oral and Poster Abstracts
Poster

Research, Bleeding and Clotting, Bleeding disorders, Autoimmune disorders, Combination therapy, Clinical trials, Adult, Clinical Practice (Health Services and Quality), Drug development, Translational Research, Epidemiology, Autoimmune hemolytic anemia, Assays, Genetic Disorders, Platelet disorders, Clinical Research, Health outcomes research, Pediatric, Supportive Care, Diseases, Immune Disorders, Thrombocytopenias, Immunodeficiency, Patient-reported outcomes, Neonatal, Treatment Considerations, Biological therapies, Real-world evidence, Pregnant, Adverse Events, Monoclonal Antibody Therapy, Technology and Procedures, Study Population, Human, Maternal Health, Serologic Tests

Halls G-H (San Diego Convention Center)

1174

The Use of ATP-Release Identifies Platelet Function Disorders in Patients with Bleeding Disorder of Unknown Cause (BDUC) and Normal LTA Results

Amaury Monard, MD¹^*, Floris Van Dellen²^*, Dave Hellenbrand³^*, Paul Verhezen⁴^*, Erik AM Beckers, MD, PhD⁵, Yvonne Henskens^6,7^* and Floor CJI Moenen, MD, PhD⁸^*

¹Maastricht University, CARIM - School for cardiovascular disease, Maastricht, New Zealand
²Internal Medicine - Hematology, Maastricht University Medical Center + (MUMC+), Maastricht, Netherlands
³Central Diagnostic Laboratory, Maastricht University Medical Center + (MUMC+), Maastricht, Netherlands
⁴Central Diagnostic Laboratory, Maastricht University Medical Center + (MUMC+), Maastricht, NLD
⁵Maastricht University Medical Center, Maastricht, NLD
⁶CARIM - School for cardiovascular disease, Maastricht, Netherlands
⁷Maastricht UMC+, Maastricht, NLD
⁸Department of Internal Medicine, Hematology, Maastricht University Medical Center+ and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands

1175

Correlation of Low Variant Allele Frequency STAT3 Gain-of-Function Mutations and T-Cell Clonality with Cyclic Thrombocytopenia

Haiyu Zhang, PhD¹^*, Henning Stehr, PhD¹^*, Robert D Bona, MD², Jean M. M Connors, MD³, William Shomali, MD⁴, Jason Gotlib, MD⁴^*, James B. Bussel, MD⁵ and James L. Zehnder, MD¹

¹Department of Pathology, Stanford University, Stanford, CA
²Section of Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT
³Harvard Medical School, Brigham and Women's Hospital, Boston, MA
⁴Division of Hematology, Stanford Cancer Institute/ Stanford University School of Medicine, Stanford, CA
⁵Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY

1176

Medical Problems of Mothers of Children with Fetal-Neonatal Alloimmune Thrombocytopenia (FNAIT): Autoimmunity and Psychological Symptoms

Eleanore A. McFarland, BS¹^*, Margaret H. McKelvy²^*, Katherine A. Knightly, BSc³^*, Stephanie V. Volpe⁴^*, Thea D. Palmer, BA⁴^*, Stacy Corke⁴^*, Emilie Vander Haar, MD³^* and James B. Bussel, MD⁵

¹Department of Obstetrics and Gynecology, Weill Cornell Medicine, New York City, NY
²Renaissance School of Medicine at Stony Brook University, New York, NY
³Department of Obstetrics and Gynecology, Weill Cornell Medicine, New York, NY
⁴NAITbabies, Penzance, United Kingdom
⁵Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY

1177

Association of Platelet Count and Mean Platelet Volume with Severity of Bleeding Events in Patients with Immune Thrombocytopenia

Sovijja Pou, MD¹^*, Charmi Trivedi, MD¹^*, Charles Milrod, MD², Christina Raker, ScD³^* and Andrew Hsu, MD⁴

¹Department of Internal Medicine, Rhode Island Hospital, Providence, RI
²Department of Hematology/Oncology, Brown University, Providence, RI
³Biostatistics, Epidemiology, and Research Design, Rhode Island Hospital, Providence, RI
⁴Department of Hematology/Oncology, Rhode Island Hospital, Providence, RI

1178

Analysis of Screening for Autism Spectrum Disorders in Children Affected By Fnait with and without Intra-Cranial Hemorrhage

Katherine A. Knightly, BSc¹^*, Margaret H. McKelvy²^*, James B. Bussel, MD³, Emilie Vander Haar, MD⁴^*, Eleanore A. McFarland, BS⁵^*, Thea D. Palmer, BA⁶^*, Stephanie V. Volpe⁷^* and Stacy Corke⁷^*

¹Department of Obstetrics and Gynecology, Weill Cornell Medicine, New York, NY
²Renaissance School of Medicine at Stony Brook University, New York, NY
³Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY
⁴Weill Cornell Medicine, New York, NY
⁵Department of Obstetrics and Gynecology, Weill Cornell Medicine, New York City, NY
⁶NAIT Babies, Penzance, United Kingdom
⁷NAITbabies, Penzance, United Kingdom

1179

Fetal and Neonatal Alloimmune Thrombocytopenia: Risk of ICH in Second Pregnancy and Treatments to Maintain an Adequate Platelet Count

Madhavi Lakkaraja, MD, MPH^1,2, Emilie Vander Haar, MD³^*, Eleanore A. McFarland, BS⁴^*, Katherine A. Knightly, BSc³^* and James B. Bussel, MD^3,5

¹University of Washington School of Medicine, Fred Hutchinson Cancer Center, Seattle, WA
²Department of Pediatrics, Division of Hematology/Oncology, University of Washington School of Medicine, Seattle, WA
³Department of Obstetrics and Gynecology, Weill Cornell Medicine, New York, NY
⁴Department of Obstetrics and Gynecology, Weill Cornell Medicine, New York City, NY
⁵Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY

1180

Direct Glycoprotein-Specific Platelet Autoantibodies Detected in a Majority of Patients with Chemotherapy-Induced Thrombocytopenia

Andrew B Song, MD^1,2, Brian Curtis, PhD, D(ABMLI), MT (ASCP) SBB³ and Hanny Al-Samkari, MD^2,4

¹Division of Hematology, Massachusetts General Hospital, Boston, MA
²Harvard Medical School, Boston, MA
³Platelet and Neutrophil Immunology and Hematology Genetics Labs, Versiti, Milwaukee, WI
⁴Division of Hematology, Massachusetts General Hospital, Cambridge, MA

1181

Pediatric Extreme Thrombocytosis, Etiology, Management and Outcome

Assaf Barg¹^*, Adina Lashansky²^*, Amos Toren, MD³^*, Gili Kenet, MD⁴ and Hana Golan²^*

¹Faculty of Medicine, Tel Aviv University, Ramat Gan, Israel
²Tel Aviv University, Tel Aviv, Israel
³sheba medical center, Tel Aviv University, Tel Aviv, Israel
⁴Chaim Sheba Medical Center, Tel Hashomer, ISR

1182

Mean Platelet Volume in Patients with Liver Cirrhosis and Chronic Liver Disease: A Case-Control Study

Bianca Clerici^1,2^*, Mariangela Scavone¹^*, Carlo Urtis¹^*, Giuseppina Vismara²^*, Sara Vavassori²^*, Andrea Luigi Crosignani²^*, Sara De Matteis^1,2^*, Pier Maria Battezzati^1,2^*, Simone Birocchi²^*, Marco Natale Cattaneo³^* and Gian Marco Podda^1,2^*

¹University of Milan, Milan, Italy
²Ospedale San Paolo, ASST Santi Paolo e Carlo, Milan, Italy
³Fondazione Arianna Anticoagulazione, Bologna, Italy

1183

Use of Steroids in Subjects with ITP in Spain: Duration and Toxicity

Maria Eva Mingot-Castellano, MD, PhD¹^*, Beatriz Chiclana Rodriguez¹^*, Blanca Sánchez-González^2,3^*, Daniel Martinez Carballeira⁴^*, Mariana Canaro Hinryk, MD⁵^*, Isabel Caparros Miranda⁶^*, Denis Zafra⁷^*, Aída Calo Pérez⁸^*, Maria Cristina Pascual Izquierdo, MD, PhD⁹^*, Gonzalo Caballero Navarro¹⁰^*, Marta Canet Maldonado¹¹^*, Maria Carmen Gómez del Castillo Solano¹²^*, Amalia Cuesta Garcia¹³^*, Patricia Alcalde Mellado, MD¹^*, Marta García Culebra¹^*, Francesc Garcia-Pallarols, BSc¹⁴^*, Lola Piquer Monsonis¹⁵^*, Paula Sanchez Llorca¹⁶^*, Diana Casares¹⁷^*, Rocio Perez Montes¹³^*, David Valcarcel, MD, PhD¹⁸ and Begoña Pedrote Amador, MD¹^*

¹Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla. Universidad de Sevilla, Sevilla, Spain
²Hospital del Mar Research Institute, Barcelona, Spain
³Hematology Department, Hospital del Mar, Barcelona, Spain
⁴Hospital Universitario Central de Asturias, Oviedo, Asturias, Spain, Oviedo, Spain
⁵Hematology Department., Hospital Universitario Son Espases, IdISBa., Palma, Spain
⁶Hospital Universitario Virgen de la Victoria, Málaga., Malaga, Spain
⁷Hospital Universitario 12 De Octubre, Madrid, Spain
⁸Hospital de Henares, Coslada, Spain
⁹Servicio de Hematología, Hospital Universitario Gregorio Marañón, Instituto de Investigación Gregorio Marañón, Madrid, Madrid, Spain
¹⁰Hematology Department. Hospital Miguel Servet, Zaragoza, Spain., Zaragoza, ESP
¹¹Hospital Universitari Mútua Terrassa, Terrassa, Barcelona., Barcelona, Spain
¹²Complexo Hospitalario Universitario A Coruña, La Coruña, Spain
¹³Hospital de Sierrallana, Torrelavega, Spain
¹⁴Hospital del Mar, Barcelona, Spain
¹⁵Hospital Universitario Son Espases, Mallorca, Spain
¹⁶Hospital 12 de octubre, Madrid, Spain
¹⁷Hospital Universitario Gregorio Marañon, Madrid, Spain
¹⁸Hematology Department, Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain

1184

Platelet Transfusion-Sparing Regimens for Severe Thrombocytopenia in Patients Undergoing Autologous Stem Cell Transplantation - a Comparative Retrospective Analysis

Sailaja Vinnakota¹, Ronak H. Mistry, DO², Rebecca Pesce-Noga, MS, CSSBB³^*, Maria Rybacka, MBBS⁴^*, Stefan Radevic, MBBS⁵^*, Carrie Marvill⁶^* and Patricia A. Locantore-Ford, MD⁷

¹Department of Medicine, Pennsylvania Hospital, Philadelphia, PA
²Division of Hematology/Oncology, Department of Medicine, Hospital of University of Pennsylvania, Philadelphia, PA
³Department of Hematology/Oncology, Pennsylvania Hospital, Philadelphia, PA
⁴Royal College of Surgeons in Ireland, Dublin, AL, IRL
⁵Royal College of Surgeons in Ireland, Dublin, Ireland
⁶Abramson Cancer Center- Pennsylvania Hospital, Philadelphia
⁷Division of Hematology/Oncology, Department of Medicine, Pennsylvania Hospital, Philadelphia, PA

1185

Prophylactic Administration of HPA-1a–Specific Antibody RLYB212 Safely Prevents Fetal/Neonatal Alloimmune Thrombocytopenia in Pregnant Mice

Huiying Zhi, MD & PhD¹, Douglas Sheridan, PhD²^*, Peter J. Newman, PhD³ and Debra K. Newman, PhD³

¹Blood Research Institute, Versiti, Milwaukee, WI
²Rallybio,, New Haven, CT
³Blood Research Institute, Versiti BloodCenter of Wisconsin, Milwaukee, WI

1186

Variation in Diagnostic Evaluation Patterns for Pediatric Evans Syndrome: Survey Results from the Pediatric ITP Consortium of North America (ICON)

Taylor Olmsted Kim, MD^1,2, Kirsty Hillier, MD³, Elizabeth Gunn, MD⁴, Sherif M. Badawy, MD, MS, MBA^5,6, Amanda B. Grimes, MD^7,8, Megan Gilbert, MD, MS⁹^*, Allison Remiker, MD¹⁰^*, Stephanie A. Fritch Lilla, MD¹¹, Shipra Kaicker, MD¹², Michele P. Lambert, MD^13,14, Rachael F. Grace, MD, MMSc^15,16 and Deirdra R Terrell, PhD¹⁷

¹University of Southern California, Keck School of Medicine, Los Angeles, CA
²University of Southern California Keck School of Medicine, Children's Hospital Los Angeles, Pasadena, CA
³Hassenfeld Children's Hospital at NYU Langone Health, New York, NY
⁴Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta, Emory University, Atlanta, GA
⁵Division of Hematology, Oncology, Neuro-Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL
⁶Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
⁷Texas Children’s Cancer and Hematology Center, Houston, TX
⁸Baylor College of Medicine, Houston, TX
⁹Lucile Packard Children's Hospital Stanford, Palo Alto, CA
¹⁰Medical College of Wisconsin, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Pewaukee, WI
¹¹Children's Minnesota, Minneapolis, MN
¹²Department of Pediatric Hematology Oncology, Weill Cornell Medicine/New York Presbyterian-Weill Cornell Medical College, New York, NY
¹³Children's Hospital of Philadelphia, Philadelphia, PA
¹⁴Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
¹⁵Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
¹⁶Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA
¹⁷University of Oklahoma Health Sciences Ctr, Oklahoma City, OK

1187

Sirolimus Combined Dexamethasone As the First-Line Therapy for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Multicenter, Open-Label, Prospective, Randomized Controlled Clinical Trial

Yimei Feng¹^*, Hanqing Zeng²^*, Yi Liao³^*, Yi Yang⁴^*, Wenzheng Yu⁵^*, Changyong Yuan⁶^*, Guojun Li⁷^*, Zhanshu Liu, MD⁸^*, Huae Shu⁹^*, Guoxiang Zhang¹⁰^*, Yaqing Feng¹¹^*, Chunfang Peng¹²^*, Xiaoli Chen¹³^*, Jia Liu¹⁴^*, Han Yao^13,15^*, Ming Hou¹⁶^* and Xi Zhang, PhD¹⁷

¹Army medical University affiliated Xinqiao Hospital, Chongqing, China
²Department of Hematology, The Second Affiliated Hospital of Chongqing Medical University, Chongqing, CHN
³Department of Hematology, Chongqing Emergency Medical Center, Chongqing, China
⁴Department of Hematology, Congqing University Three Gorges Hospital, Chongqing, China
⁵Department of Hematology, Binzhou Medical University Hospital, Binzhou, China
⁶Dezhou People's Hospital (Shandong University Qilu Hospital Dezhou Hospital), Dezhou City, China
⁷Department of Hematology, Chongqing Hechuan District People's Hospital, Chongqing, China
⁸Department of Hematology, Yongchuan Hospital of Chongqing Medical University, Chongqing, China
⁹Department of Hematology and Rheumatology, Chongqing Kaizhou District People's Hospital, Chongqing, CHN
¹⁰Heping Hospital Affiliated to Changzhi Medical College, Changzhi, China
¹¹Department of Hematology, The Third People's Hospital of Datong,, Datong, China
¹²Department of oncology, Chongqing University Jiangjin Hospital, Chongqing, China
¹³Xinqiao Hospital, Army Medical University, Chongqing, China
¹⁴The Second Affiliated Hospital of Army Military Medical University, Chongqing, China
¹⁵Xinqiao Hospital, Chongqing, China
¹⁶Qilu Hospital of Shandong University, Jinan, China
¹⁷Medical Center of Hematology, Xinqiao Hospital of Army Medical University, State Key Laboratory of Trauma and Chemical Poisoning, Chongqing Key Laboratory of Hematology and Microenvironment, Chongqing, China

1188

Efficacy of Combined Herombopag and Recombinant Human Thrombopoietin Therapy Compared to Monotherapy on Platelet Engraftment after Autologous Hematopoietic Stem Cell Transplantation in Patients

Jing Wu¹^*, Jieping Li²^*, Chaoyu Wang²^*, Dehong Huang²^*, Bingling Guo²^* and Yao Liu¹

¹Chongqing University Cancer Hospital, Chongqing, China
²Department of Hematology-Oncology, Chongqing University Cancer Hospital, Chongqing, China

1189

Real World Romiplostim Dosing and Response in Pediatric Immune Thrombocytopenia

Emily M Harris, MD^1,2 and Rachael F. Grace, MD, MMSc^1,2

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA
²Harvard Medical School, Boston, MA

1190

Efficacy and Safety of Pegylated Peptide Agonist of Thrombopoietin Receptor (PN20) in Secondary Prevention of Chemotherapy-Induced Thrombocytopenias: A Multicenter, Single Arm, Phase Ib Trial

Kai Fan, Ph.D, MD^*, Qing Chen, M.Sc^*, Shehui Hu^*, Xin Zeng^*, Xiaolan Qin^* and Jing Sun, M.Sc^*

Chongqing Peg-Bio BioPharm.Co.,Ltd., Chongqing, China

1191

Analysis of Durability of Response to Avatrombopag (AVA) from a Phase 3b Multicenter, Randomized, Double-Blind, Placebo (PBO)-Controlled, Parallel-Group Trial to Evaluate the Efficacy and Safety of AVA for the Treatment of Pediatric Patients with Immune Thrombocytopenia

Rachael F. Grace, MD, MMSc¹, Goksel Leblebisatan, MD²^*, Yesim Aydinok, MD³^*, Sule Unal⁴^*, John D. Grainger, MD, MRCP, FRPATH⁵^*, Jessica Zhang, MS⁶^*, Brian Jamieson⁶ and Scott Kolodny, MD⁶^*

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA
²Department of Pediatric Hematology, Çukurova University Medical Faculty, Adana, Turkey
³Department of Pediatric Hematology and Oncology, Ege University School of Medicine, Izmir, Turkey
⁴Department of Pediatric Hematology, Hacettepe University Faculty of Medicine, Ankara, Turkey
⁵Royal Manchester Children's Hospital, Pendlebury, GBR
⁶Sobi Inc., Waltham, MA

1192

Efficacy and Safety of Mesenchymal Stem Cells in Improving Thrombocytopenia after Chemotherapy in Malignant Tumors

Bingling Guo¹^*, Jieping Li¹^* and Yao Liu²

¹Department of Hematology-Oncology, Chongqing University Cancer Hospital, Chongqing, China
²Chongqing University Cancer Hospital, Chongqing, China

1193

Herombopag for Preventing Chemotherapy-Induced Thrombocytopenia in Lymphoma: A Single Center, Randomized Controlled, Phase II Study

Chunyan Xiao¹^*, Yao Ding¹^*, Chensi Zeng¹^*, Lingqian Zhang¹^*, Jing Wu²^*, Yingyu Nan¹^* and Yao Liu²

¹Department of Hematology-Oncology, Chongqing University Cancer Hospital, Chongqing, China
²Chongqing University Cancer Hospital, Chongqing, China

1193.1

Design of a Phase 3, Multicenter, Randomized, Open-Label Study of Nipocalimab or IVIG in Pregnancies at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia (FREESIA-3)

James B. Bussel, MD^1,2, Pamela Baker, PharmD³^*, Edwin Lam³^*, Paige Meizlik³^*, Hein Fennema³^*, Rebecca Zaha³^*, Hillary Van Valkenburgh³^* and Kattayoun Kordy³^*

¹Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY
²New York Presbyterian Hospital, New York
³Janssen Research & Development, LLC, a Johnson & Johnson Company, Spring House

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^*signifies non-member of ASH

311.P1.62 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster I