. 2024 Oct 8;8(19):5112-5117.

doi: 10.1182/bloodadvances.2024012707.

What is in a name: defining pediatric refractory ITP

Taizo A Nakano¹, Amanda B Grimes², Robert J Klaassen³, Michele P Lambert⁴, Cindy Neunert⁵, Jennifer A Rothman⁶, Kristin A Shimano⁷, Christina Amend⁸, Megan Askew⁹, Sherif M Badawy¹⁰, Jillian M Baker¹¹, Vicky Breakey¹², Shelley Crary¹³, Monica Davini¹⁴, Stephanie Fritch Lilla¹⁵, Megan Gilbert¹⁶, Taru Hays¹, Kerry Hege¹⁷, Kirsty Hillier¹⁸, Amanda Jacobson-Kelly¹⁹, Shipra Kaicker²⁰, Taylor O Kim²¹, Manpreet Kochhar²², Thierry Leblanc²³, Marie Martinelli²⁴, Mara Nunez²⁵, Allison Remiker²⁶, Corrina Schultz⁹, Ruchika Sharma²⁷, Rachael F Grace²⁸

Affiliations

¹ Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
² Texas Children's Hematology Center, Baylor College of Medicine, Houston, TX.
³ Children's Hospital of Eastern Ontario, Ottawa, ON, Canada.
⁴ Division of Hematology, Children's Hospital of Philadelphia, Perelman School of Medicine at UPenn, Philadelphia, PA.
⁵ Columbia University Medical School, New York, NY.
⁶ Duke University Medical Center, Durham, NC.
⁷ UCSF Benioff Children's Hospital, San Francisco, CA.
⁸ University of Wisconsin School of Medicine and Public Health, Madison, WI.
⁹ Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders, Nemours Children's Hospital, Wilmington, DE.
¹⁰ Division of Hematology, Oncology and Stem cell Transplant, Lurie Children's Hospital of Chicago; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL.
¹¹ The Hospital for Sick Children and Unity Health Toronto (St. Michael's Hospital), Toronto, ON, Canada.
¹² Department of Pediatrics, McMaster University, Hamilton, ON, Canada.
¹³ University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR.
¹⁴ University of Arizona, Banner University Medical Center, Tucson, AZ.
¹⁵ Children's Minnesota, Minneapolis, MN.
¹⁶ Lucile Packard Children's Hospital, Stanford University School of Medicine, Palo Alto, CA.
¹⁷ Riley Children's Health, Indiana University School of Medicine, Indianapolis, IN.
¹⁸ Division of Pediatric Hematology/Oncology, Department of Pediatrics, Hassenfeld Children's Hospital at NYU Langone Health, NYU Grossman School of Medicine, New York, NY.
¹⁹ Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, OH.
²⁰ Weill Cornell Medicine, New York, NY.
²¹ Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA.
²² Hasbro Children's Hospital at Brown University, Providence, RI.
²³ Service d'hématologie et d'Immunologie, Hôpital Robert-Debré, L'Assistance publique Hôpitaux de Paris and Université Paris-Cité, Paris, France.
²⁴ Division of Hematology/Oncology, Oregon Health and Science University, Portland, OR.
²⁵ Division of Hematology, Instituto Nacional de Pediatría, Mexico City, Mexico.
²⁶ Children's Wisconsin, Medical College of Wisconsin, Milwaukee, WI.
²⁷ University of Texas Southwestern Medical Center, Dallas, TX.
²⁸ Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.

PMID: 39059013
PMCID: PMC11460440
DOI: 10.1182/bloodadvances.2024012707

What is in a name: defining pediatric refractory ITP

Taizo A Nakano et al. Blood Adv. 2024.

. 2024 Oct 8;8(19):5112-5117.

doi: 10.1182/bloodadvances.2024012707.

Authors

Affiliations

¹ Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
² Texas Children's Hematology Center, Baylor College of Medicine, Houston, TX.
³ Children's Hospital of Eastern Ontario, Ottawa, ON, Canada.
⁴ Division of Hematology, Children's Hospital of Philadelphia, Perelman School of Medicine at UPenn, Philadelphia, PA.
⁵ Columbia University Medical School, New York, NY.
⁶ Duke University Medical Center, Durham, NC.
⁷ UCSF Benioff Children's Hospital, San Francisco, CA.
⁸ University of Wisconsin School of Medicine and Public Health, Madison, WI.
⁹ Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders, Nemours Children's Hospital, Wilmington, DE.
¹⁰ Division of Hematology, Oncology and Stem cell Transplant, Lurie Children's Hospital of Chicago; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL.
¹¹ The Hospital for Sick Children and Unity Health Toronto (St. Michael's Hospital), Toronto, ON, Canada.
¹² Department of Pediatrics, McMaster University, Hamilton, ON, Canada.
¹³ University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR.
¹⁴ University of Arizona, Banner University Medical Center, Tucson, AZ.
¹⁵ Children's Minnesota, Minneapolis, MN.
¹⁶ Lucile Packard Children's Hospital, Stanford University School of Medicine, Palo Alto, CA.
¹⁷ Riley Children's Health, Indiana University School of Medicine, Indianapolis, IN.
¹⁸ Division of Pediatric Hematology/Oncology, Department of Pediatrics, Hassenfeld Children's Hospital at NYU Langone Health, NYU Grossman School of Medicine, New York, NY.
¹⁹ Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, OH.
²⁰ Weill Cornell Medicine, New York, NY.
²¹ Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA.
²² Hasbro Children's Hospital at Brown University, Providence, RI.
²³ Service d'hématologie et d'Immunologie, Hôpital Robert-Debré, L'Assistance publique Hôpitaux de Paris and Université Paris-Cité, Paris, France.
²⁴ Division of Hematology/Oncology, Oregon Health and Science University, Portland, OR.
²⁵ Division of Hematology, Instituto Nacional de Pediatría, Mexico City, Mexico.
²⁶ Children's Wisconsin, Medical College of Wisconsin, Milwaukee, WI.
²⁷ University of Texas Southwestern Medical Center, Dallas, TX.
²⁸ Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.

PMID: 39059013
PMCID: PMC11460440
DOI: 10.1182/bloodadvances.2024012707

Abstract

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.

© 2024 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: A.B.G. receives research support from Novartis. R.J.K. receives consultancy fees from Agios, Amgen, Bayer Canada, F. Hoffman-La Roche, Novo Nordisk, Octapharma AG, Sanofi, and Takeda. M.P.L. serves on an advisory board for Octapharma, Dova, Principia, Rigel, Argenx, Platelet Disorder Support Association, 22q Society, and CdLS Foundation; receives consultancy fees from Agios, Novartis, Dova, Principia, Argenx, Rigel, Sobi, Sanofi, and Janssen; and receives research support from the Foundation for Women and Girls with Blood Disorders, the Platelet Disorder Support Association, the National Institutes of Health, Sysmex, Novartis, Principia, Argenx, Dova, Octapharma, and Sanofi. C.N. receives research support and consultancy fees from Novartis, and consultancy fees from Argenx, Sanofi, and Sobi. K.A.S. receives research support from Novartis, Sobi, and Sanofi. S.C. serves on an advisory board for Sanofi, and receives consultancy fees from Novartis, Pfizer, and Medexus. S.F.L. receives research support from Sobi and Octapharma. A.R. receives consultancy fees from bluebird bio, Horizon Therapeutics, and Novartis. R.F.G. receives research support from Novartis, Sobi, and Agios, and consultancy fees from Agios and Sanofi. The remaining authors declare no competing financial interests.

Cited by

Working toward international consensus defining pediatric "refractory ITP".
O'Farrell C, Bell Grimes A, Nakano TA, Klaassen RJ, Lambert MP, Neunert C, Rothman JA, Shimano KA, Grace RF. O'Farrell C, et al. Blood Adv. 2025 Mar 11;9(5):1244-1245. doi: 10.1182/bloodadvances.2024015219. Blood Adv. 2025. PMID: 39787601 Free PMC article. No abstract available.
Pediatric refractory ITP, a definition to be defined.
Parodi E, Russo G. Parodi E, et al. Blood Adv. 2025 Mar 11;9(5):1242-1243. doi: 10.1182/bloodadvances.2024015195. Blood Adv. 2025. PMID: 39536294 Free PMC article. No abstract available.
The intersection of epigenetics and immune thrombocytopenia: new insights into disease mechanisms and treatments.
Zhou X, Shan NN. Zhou X, et al. Mol Biol Rep. 2025 Feb 21;52(1):257. doi: 10.1007/s11033-025-10363-z. Mol Biol Rep. 2025. PMID: 39982580 Review.

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What is in a name: defining pediatric refractory ITP

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What is in a name: defining pediatric refractory ITP

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