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547 Critical Bleeding in Adults and Children with Immune Thrombocytopenia: A Multicentre Cohort Study

Program: Oral and Poster Abstracts
Type: Oral
Session: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Big Data and Basic Science
Hematology Disease Topics & Pathways:
Research, Autoimmune disorders, Combination therapy, Epidemiology, Clinical Research, Health outcomes research, Diseases, Immune Disorders, Therapy sequence, Treatment Considerations
Sunday, December 8, 2024: 12:00 PM

Saifur R. Chowdhury, PhD (c)1,2*, Emily Sirotich, PhD3,4, Dimpy Modi5*, Melanie St John5*, Kerolos Eisa5*, Adam Cuker, MD6, Carolyn E Beck7*, Rachael F Grace, MD8, Shuoyan Ning9*, Justin W Yan10*, Amanda Pfeiffer11*, Michele P Lambert, MD12,13*, Karen Panckeri14*, Allyson M Pishko, MD15, Kristine VanAarsen16*, Ahmed Slim17*, Kathryn M. Carrier18*, Rebecca Geer19*, Abinaya Arulselvan20*, Matthew Basara20*, Brenna Cannon20*, Ellis Bloom, DO21*, Sam J King20*, Daya Gill5*, Laura M. Venier5*, Emily Xu5*, Ishaq Siddiqui22*, Bonnie Liu5*, Taylah Buissereth, BS23*, Timothy Mercier24*, Emily M. Harris24*, Enass H. Raffa25,26*, Joel Livingston27*, Dongyoung Kim5*, Maryam Akbari-Moghaddam5,28*, Gordon Guyatt, MD29,30*, Dena Zeraatkar31,32*, Lehana Thabane31* and Donald Arnold, MD2,33

1Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
2Michael G. DeGroote Centre for Transfusion Research, McMaster University, Hamilton, ON, Canada
3Michael G. DeGroote Centre for Transfusion Research, Department of Medicine, Hamilton, ON, Canada
4Yale School of Medicine, New Haven
5Michael G. DeGroote Centre for Transfusion Research, Department of Medicine, McMaster University, Hamilton, ON, Canada
6Department of Medicine and Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
7Hospital for Sick Children, Toronto, Canada
8Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, MA
9McMaster University, Hamilton, ON, CAN
10London Health Sciences Centre, Western University, London, Canada
11Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
12Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
13Division of Hematology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
14Hospital of the University of Pennsylvania, University of Pennsylvania, Philadelphia
15Department of Medicine, Division of Hematology-Oncology, University of Pennsylvania, Philadelphia, PA
16London Health Sciences Centre, London, ON, Canada
17St. Joseph's Healthcare London, London, ON, Canada
18Harvard Medical School, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
19Department of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
20The Children's Hospital of Philadelphia, Division of Hematology, Philadelphia
21Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
22Faculty of Health Sciences, McMaster University, Hamilton, Canada
23Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
24Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston
25Department of Pediatrics, King Abdulaziz University, Jeddah, Saudi Arabia
26Department of Oncology, King Faisal Specialist Hospital and Research Center, Jeddah, Saudi Arabia
27Pediatric Hematology/KidClot, Department of Paediatrics, Stollery Children’s Hospital and University of Alberta, Edmonton, Canada
28Department of Computing and Software, McMaster University, Hamilton, ON, Canada
29Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, CAN
30Department of Medicine, McMaster University, Hamilton, Canada
31Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, Canada
32Department of Anesthesia, McMaster University, Hamilton, Canada
33Department of Medicine, McMaster University, Hamilton, ON, Canada

Introduction

Immune thrombocytopenia (ITP) is a rare autoimmune disease that causes low blood platelets and an increased risk of bleeding. Critical bleeds in patients with ITP such as intracranial hemorrhage (ICH) are life-threatening events that can cause death or life-long morbidity. Treatment of ITP bleeding emergencies requires a rapid, coordinated, multidisciplinary approach. The objective of this study was to describe critical ITP bleeds in adults and children, including the treatments they received and their outcomes. These data will be used to inform future guidelines on ITP bleeding emergencies.

Methods

We conducted a retrospective cohort study of ITP patients with critical bleeds across 7 centers in the United States and Canada from 2010 – 2020. Eligible patients were adults and children with an established diagnosis of ITP and a platelet count <20 x109/L at presentation to the emergency room. A critical ITP bleed was defined using published criteria: (i) a bleed in a critical anatomical site including ICH, intraspinal, intraocular, retroperitoneal, pericardial, or intramuscular with compartment syndrome; or (ii) an ongoing bleed that results in hemodynamic instability or respiratory compromise (Sirotich et al., 2021). We summarized the data using descriptive statistics. None of the critical bleeds identified as intraocular were associated with vision loss, thus these were excluded post-hoc.

Results

We identified 1226 patients (n=296 adults; and n= 930 children) with ITP from 7 centers in North America who had a platelet count <20 x109/L at presentation to the emergency room for evaluation. Of those, 1192 (97.2%) had any type of bleeding, and 28 (2.2%) had critical bleeding (n=15 adults; and n=13 children). Among the 28 patients with critical bleeding, 16 (57.1%) were female. Median (IQR) age for adults was 68 years (45-74); and median age for children was 11 years (5-16). Median lowest platelet count at the time of the critical bleed was 8 x109/L (IQR 3-15) for adults and 3 x109/L (IQR 1-6) for children.

Most critical bleeds were ICH (n=18; 64.3%), followed by epistaxis (n=4; 14.3%), gynecological (n=3; 10.7%), gastrointestinal (n=2; 7.1%), and pulmonary hemorrhage (n=1; 3.6%). Of the 28 ITP patients with a critical bleed, 14 (50%) were newly diagnosed (<3 months). Ten patients were admitted to the intensive care unit (n=4 adults; n=6 children).

Interventions used to treat critical bleeds were platelet transfusion (n=19), intravenous immune globulin (IVIG; n=18), methylprednisolone (n=13), prednisone (n=11), plasma transfusion (n=6), dexamethasone (n=5), and romiplostim (n=4). One patient (20 years old) underwent urgent splenectomy. Time to administration of first ITP treatment was 4.7 hours (median; IQR 3.2-9.5) for 21 evaluable patients; 6.9 hours (median; IQR 4.3-9.6) for adults (n=11) and 3.5 hours (median; IQR 2.7-7.8) for children (n=10). The most common treatment combination was corticosteroids, IVIG and platelet transfusion.

Overall, 10 patients died (35.7%), including 7 (46.7%) adults and 3 (23.1%) children. Causes of death were bleeding (ICH, n=7; gastrointestinal bleeding, n=1; pulmonary hemorrhage, n=1) and septic shock (n=1). Four patients developed neurological disability (14.3%, n=2 adults; and n=2 children).

Conclusion

Critical bleeds in ITP patients were rare, representing 2.2% of ITP patients in the emergency room with severe thrombocytopenia; yet, they were associated with a high risk of death (35.7%). ICH was the most common critical bleed. Common interventions included corticosteroids, IVIG and platelet transfusion; however, time to first treatment was approximately 5 hours, and longer for adults than children. A standardized approach to the management of ITP critical bleeds is needed.

Disclosures: Cuker: MingSight: Consultancy; UpToDate: Patents & Royalties: Authorship royalties; Synergy: Consultancy; Pfizer: Consultancy; Sanofi: Consultancy. Grace: Novartis: Research Funding; Sobi: Consultancy, Research Funding; Sanofi: Consultancy; Agios: Consultancy, Research Funding. Pishko: Biomarin: Consultancy. Arnold: Amgen: Consultancy; Argenx: Consultancy; Medison: Consultancy; Principia: Consultancy; Rigel: Consultancy; Sanofi: Consultancy; Sobi: Consultancy; Novartis: Research Funding; Paradigm: Research Funding.

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