A Descriptive 5‐Year Analysis of the Demographics and Therapies for Patients With Immune Thrombotic Thrombocytopenic Purpura in the <scp>USA</scp>: A Multicenter Study of 390 Disease Episodes From 2017 to 2021
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.
Authors
- Chisa Yamada (ORCID: https://orcid.org/0000-0002-1968-2385)
- Jay S. Raval (ORCID: https://orcid.org/0000-0001-9835-957X)
- Marisa B. Marques (ORCID: https://orcid.org/0000-0002-6689-7856)
- Christopher B. Webb
- Jeremy W. Jacobs (ORCID: https://orcid.org/0000-0002-5719-9685)
- Caroline G. Stanek (ORCID: https://orcid.org/0009-0006-4274-2354)
- Sean G. Yates (ORCID: https://orcid.org/0000-0001-9703-5047)
- Christopher A. Tormey (ORCID: https://orcid.org/0000-0003-1785-2245)
- Evan M. Bloch (ORCID: https://orcid.org/0000-0001-8181-9517)
- Quentin Eichbaum (ORCID: https://orcid.org/0000-0003-3480-7892)
- Cristina A. Figueroa Villalba (ORCID: https://orcid.org/0009-0004-1870-4708)
- Brenda J. Grossman (ORCID: https://orcid.org/0000-0002-1500-8211)
- Diana M. Byrnes (ORCID: https://orcid.org/0000-0003-0781-7577)
- Thomas J. Harrington
- Justin E. Juskewitch
- Annette J. Schlueter (ORCID: https://orcid.org/0000-0003-2442-7079)
- Becky Adkins
- Meredith G Parsons (ORCID: https://orcid.org/0000-0001-9979-1938)
- Garrett S. Booth (ORCID: https://orcid.org/0000-0002-8422-9480)
- Elizabeth P. Crowe (ORCID: https://orcid.org/0000-0003-1910-2800)
- Fatima Aldarweesh (ORCID: https://orcid.org/0000-0001-8295-1405)
- Lisa R. Wallace
- Rance C. Siniard (ORCID: https://orcid.org/0000-0002-7889-3887)
- Allison P. Wheeler (ORCID: https://orcid.org/0000-0003-3967-4873)
- Ingrid Pérez-Álvarez (ORCID: https://orcid.org/0000-0001-8600-7454)
- Rahaf Alkhateb
- Laetitia Daou (ORCID: https://orcid.org/0000-0001-9375-2612)
- Yanyun Wu (ORCID: https://orcid.org/0000-0001-8410-4349)
- Elizabeth S. Allen (ORCID: https://orcid.org/0000-0002-1004-3851)
- Ellen Klapper
- Laura D. Stephens (ORCID: https://orcid.org/0000-0002-9101-9326)
- Omar Aljuboori
- Amy Powers
- Miriam Andrea Duque
- Monica S. Klein
Institutions
- University of Vermont (US)
- University of Hawaiʻi at Mānoa (US)
- University of Iowa (US)
- Cedars-Sinai Medical Center (US)
- Mayo Clinic (US)
- Johns Hopkins University (US)
- University of Miami (US)
- University of New Mexico (US)
- University of Washington (US)
- Washington University in St. Louis (US)
- Johns Hopkins Medicine (US)
- University of Alabama at Birmingham (US)
- Yale University (US)
- University of California San Diego (US)
- University of Chicago (US)
- Queen's Medical Center (US)
- Michigan Medicine (US)
- Washington Center (US)
- The University of Texas Southwestern Medical Center (US)
- Vanderbilt University Medical Center (US)
Publication Details
- Journal
- Journal of Clinical Apheresis
- Published
- 2025-03-27
- DOI
- https://doi.org/10.1002/jca.70017
- Citations
- 2
- Primary Topic
- Complement system in diseases
- Type
- article
- Field-Weighted Citation Impact
- 1.59
Funders
- National Heart, Lung, and Blood Institute