There are no agreed upon terminology to define "refractory" pediatric Immune Thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.
Review Article|
July 26, 2024
What's in a name: defining pediatric refractory ITP
Taizo A Nakano,
Taizo A Nakano
Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, United States
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Amanda B Grimes,
Texas Children's Hospital, Baylor College of Medicine, United States
* Corresponding Author; email: abgrimes@texaschildrens.org
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Robert J Klaassen,
Robert J Klaassen
Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada
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Michele P Lambert,
Michele P Lambert
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
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Cindy E. Neunert,
Cindy E. Neunert
Columbia University Medical School, United States
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Jennifer A Rothman,
Jennifer A Rothman
Duke University Medical Center, Durham, North Carolina, United States
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Kristin A Shimano,
Kristin A Shimano
UCSF Benioff Children's Hospital, United States
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Christina Amend,
Christina Amend
University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, United States
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Megan Askew,
Megan Askew
Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders, Nemours Children's Hospital, Wilmington, Delaware, United States
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Sherif M Badawy,
Sherif M Badawy
Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, Illinois, United States
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Jillian M Baker,
Jillian M Baker
The Hospital for Sick Children & Unity Health Toronto, Canada
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Vicky Breakey,
Vicky Breakey
McMaster University, OAKVILLE, Canada
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Shelley E Crary,
Shelley E Crary
Arkansas Children's Hospital, United States
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Monica Davini,
Monica Davini
University of Arizona, Banner University Medical Center, Tucson, Arizona, United States
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Stephanie Fritch Lilla,
Stephanie Fritch Lilla
Children's Minnesota, Minneapolis, Minnesota, United States
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Megan Gilbert,
Megan Gilbert
Lucile Packard Children's Hospital, Stanford University School of Medicine, Palo Alto, California, United States
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Taru Hays,
Taru Hays
Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, Colorado, United States
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Kerry Hege,
Kerry Hege
Riley Children's Health, Indiana University School of Medicine, United States
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Kirsty Hillier,
Kirsty Hillier
Department of Pediatrics, Division of Pediatric Hematology/Oncology, Hassenfeld Children's Hospital at NYU Langone Health, NYU Grossman School of Medicine, United States
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Amanda Jacobson-Kelly,
Amanda Jacobson-Kelly
Nationwide Children's Hospital, The Ohio State University College of Medicine, United States
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Shipra Kaicker,
Shipra Kaicker
Weill Cornell Medical College/New York Presbyterian Hospital, New York, New York, United States
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Taylor Olmsted Kim,
Taylor Olmsted Kim
Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California, United States
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Manpreet Kochhar,
Manpreet Kochhar
Hasbro Children's Hospital at Brown University, Providence, Rhode Island, United States
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Thierry M Leblanc,
Thierry M Leblanc
Hôpital Robert-Debré - APHP and Université Paris-Cité, Paris Cedex 19, France
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Marie Martinelli,
Marie Martinelli
Oregon Health & Science University, Portland, Oregon, United States
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Mara Nunez,
Mara Nunez
Instituto Nacional de Pediatría, Mexico City, Mexico
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Allison Sarah Remiker,
Allison Sarah Remiker
Children's Wisconsin, Medical College of Wisconsin, Milwaukee, Wisconsin, United States
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Corinna L. Schultz,
Corinna L. Schultz
Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders, Nemours Children's Hospital, United States
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Ruchika Sharma,
Ruchika Sharma
The University of Texas Southwestern Medical Center, Dallas, Texas, United States
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Rachael F. Grace
Rachael F. Grace
Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, United States
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Blood Adv bloodadvances.2024012707.
Article history
Submitted:
April 10, 2024
Revision Received:
June 24, 2024
Accepted:
July 6, 2024
Citation
Taizo A Nakano, Amanda B Grimes, Robert J Klaassen, Michele P Lambert, Cindy E. Neunert, Jennifer A Rothman, Kristin A Shimano, Christina Amend, Megan Askew, Sherif M Badawy, Jillian M Baker, Vicky Breakey, Shelley E Crary, Monica Davini, Stephanie Fritch Lilla, Megan Gilbert, Taru Hays, Kerry Hege, Kirsty Hillier, Amanda Jacobson-Kelly, Shipra Kaicker, Taylor Olmsted Kim, Manpreet Kochhar, Thierry M Leblanc, Marie Martinelli, Mara Nunez, Allison Sarah Remiker, Corinna L. Schultz, Ruchika Sharma, Rachael F. Grace; What's in a name: defining pediatric refractory ITP. Blood Adv 2024; bloodadvances.2024012707. doi: https://doi.org/10.1182/bloodadvances.2024012707
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