A Descriptive 5-Year Analysis of the Demographics and Therapies for Patients With Immune Thrombotic Thrombocytopenic Purpura in the USA: A Multicenter Study of 390 Disease Episodes From 2017 to 2021

Jeremy W Jacobs; Brian D Adkins; Garrett S Booth; Caroline G Stanek; Elizabeth S Allen; Brenda J Grossman; Laura D Stephens; Elizabeth P Crowe; Laetitia Daou; Marisa B Marques; Rance C Siniard; Lisa R Wallace; Chisa Yamada; Miriam Andrea Duque; Yanyun Wu; Omar Aljuboori; Thomas J Harrington; Diana M Byrnes; Quentin Eichbaum; Cristina A Figueroa Villalba; Justin E Juskewitch; Ellen Klapper; Ingrid Perez-Alvarez; Monica S Klein; Fatima Aldarweesh; Rahaf Alkhateb; Meredith G Parsons; Annette J Schlueter; Christopher A Tormey; Allison P Wheeler; Amy A Powers; Christopher B Webb; Sean G Yates; Evan M Bloch; Jay S Raval

doi:10.1002/jca.70017

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A Descriptive 5-Year Analysis of the Demographics and Therapies for Patients With Immune Thrombotic Thrombocytopenic Purpura in the USA: A Multicenter Study of 390 Disease Episodes From 2017 to 2021

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A Descriptive 5-Year Analysis of the Demographics and Therapies for Patients With Immune Thrombotic Thrombocytopenic Purpura in the USA: A Multicenter Study of 390 Disease Episodes From 2017 to 2021

Jeremy W Jacobs, Brian D Adkins, Garrett S Booth, Caroline G Stanek, Elizabeth S Allen, Brenda J Grossman, Laura D Stephens, Elizabeth P Crowe, Laetitia Daou, Marisa B Marques, …

Journal of clinical apheresis, Vol.40(2), e70017

04/2025

DOI: 10.1002/jca.70017

PMCID: PMC11948952

PMID: 40145682

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Abstract

Immune thrombotic thrombocytopenic purpura (iTTP) is characterized by microangiopathic hemolytic anemia, thrombocytopenia, and microvascular occlusion secondary to acquired ADAMTS13 deficiency. Contemporary data regarding iTTP treatment practices in the US, including the use of caplacizumab, are lacking. We aimed to characterize the demographics and therapies, including medications and apheresis practices, in patients with iTTP in the US. We retrospectively analyzed iTTP cases at 15 sites in the US that provide comprehensive care for patients with iTTP. The time-period assessed was from January 1, 2017 to December 31, 2021. Our primary objective was to analyze data by iTTP episode, inclusive of initial episodes and relapses. A total of 390 iTTP episodes were reported for 280 unique individuals (187 females, 93 males). Thirty-day mortality was 3.7% (14/374), and 6-month mortality was 7.4% (27/367). TPE details were reported for 343 episodes, among which 261 underwent at least one procedure (median 6, IQR 3-11). Among the 261 episodes with at least one therapeutic plasma exchange (TPE) performed, 82.0% (214/261) used only plasma. Caplacizumab was used either alone or in combination with other agents in 43 (11.0%) episodes. Management strategies for iTTP remain varied across centers in the US, with a variety of combinations for TPE replacement fluids and therapeutic agents, as well as limited use of caplacizumab. Further research and standardization of treatment regimens may further reduce mortality in this condition.

ADAMTS13 Protein - deficiency

Adolescent

Adult

Aged

Blood Component Removal - methods

Female

Humans

Male

Middle Aged

Plasma Exchange - methods

Purpura, Thrombotic Thrombocytopenic - therapy

Retrospective Studies

Single-Domain Antibodies - therapeutic use

United States - epidemiology

Young Adult

Details

Title: Subtitle: A Descriptive 5-Year Analysis of the Demographics and Therapies for Patients With Immune Thrombotic Thrombocytopenic Purpura in the USA: A Multicenter Study of 390 Disease Episodes From 2017 to 2021
Creators: Jeremy W Jacobs - Vanderbilt University Medical Center
Brian D Adkins - The University of Texas Southwestern Medical Center
Garrett S Booth - Vanderbilt University Medical Center
Caroline G Stanek - University of Alabama at Birmingham
Elizabeth S Allen - University of California San Diego
Brenda J Grossman - Washington University in St. Louis
Laura D Stephens - University of California San Diego
Elizabeth P Crowe - Johns Hopkins Medicine
Laetitia Daou - Johns Hopkins Medicine
Marisa B Marques - University of Alabama at Birmingham
Rance C Siniard - University of Alabama at Birmingham
Lisa R Wallace - Washington University in St. Louis
Chisa Yamada - University of Michigan
Miriam Andrea Duque - University of Miami
Yanyun Wu - University of Miami
Omar Aljuboori - University of Miami
Thomas J Harrington - University of Miami
Diana M Byrnes - University of Miami
Quentin Eichbaum - Vanderbilt University Medical Center
Cristina A Figueroa Villalba - Yale School of Medicine
Justin E Juskewitch - Mayo Clinic
Ellen Klapper - Cedars-Sinai Medical Center
Ingrid Perez-Alvarez - Cedars-Sinai Medical Center
Monica S Klein - Mayo Clinic
Fatima Aldarweesh - University of Chicago
Rahaf Alkhateb - University of Chicago
Meredith G Parsons - University of Iowa
Annette J Schlueter - University of Iowa
Christopher A Tormey - Yale University
Allison P Wheeler - Washington Center
Amy A Powers - Queen's Medical Center
Christopher B Webb - The University of Texas Southwestern Medical Center
Sean G Yates - The University of Texas Southwestern Medical Center
Evan M Bloch - Johns Hopkins University
Jay S Raval - University of Vermont
Resource Type: Journal article
Publication Details: Journal of clinical apheresis, Vol.40(2), e70017
DOI: 10.1002/jca.70017
PMID: 40145682
PMCID: PMC11948952
NLM abbreviation: J Clin Apher
ISSN: 1098-1101
eISSN: 1098-1101
Publisher: WILEY
Grant note: 1K23HL15182 / National Heart Lung and Blood Institute NHLBI NIH HHS
Language: English
Date published: 04/2025
Academic Unit: Pathology
Record Identifier: 9984802504902771

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