What is in a name: defining pediatric refractory ITP.

Autor: Nakano TA; Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO., Grimes AB; Texas Children's Hematology Center, Baylor College of Medicine, Houston, TX., Klaassen RJ; Children's Hospital of Eastern Ontario, Ottawa, ON, Canada., Lambert MP; Division of Hematology, Children's Hospital of Philadelphia, Perelman School of Medicine at UPenn, Philadelphia, PA., Neunert C; Columbia University Medical School, New York, NY., Rothman JA; Duke University Medical Center, Durham, NC., Shimano KA; UCSF Benioff Children's Hospital, San Francisco, CA., Amend C; University of Wisconsin School of Medicine and Public Health, Madison, WI., Askew M; Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders, Nemours Children's Hospital, Wilmington, DE., Badawy SM; 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., Baker JM; The Hospital for Sick Children and Unity Health Toronto (St. Michael's Hospital), Toronto, ON, Canada., Breakey V; Department of Pediatrics, McMaster University, Hamilton, ON, Canada., Crary S; University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR., Davini M; University of Arizona, Banner University Medical Center, Tucson, AZ., Fritch Lilla S; Children's Minnesota, Minneapolis, MN., Gilbert M; Lucile Packard Children's Hospital, Stanford University School of Medicine, Palo Alto, CA., Hays T; Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO., Hege K; Riley Children's Health, Indiana University School of Medicine, Indianapolis, IN., Hillier K; Division of Pediatric Hematology/Oncology, Department of Pediatrics, Hassenfeld Children's Hospital at NYU Langone Health, NYU Grossman School of Medicine, New York, NY., Jacobson-Kelly A; Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, OH., Kaicker S; Weill Cornell Medicine, New York, NY., Kim TO; Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA., Kochhar M; Hasbro Children's Hospital at Brown University, Providence, RI., Leblanc T; Service d'hématologie et d'Immunologie, Hôpital Robert-Debré, L'Assistance publique Hôpitaux de Paris and Université Paris-Cité, Paris, France., Martinelli M; Division of Hematology/Oncology, Oregon Health and Science University, Portland, OR., Nunez M; Division of Hematology, Instituto Nacional de Pediatría, Mexico City, Mexico., Remiker A; Children's Wisconsin, Medical College of Wisconsin, Milwaukee, WI., Schultz C; Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders, Nemours Children's Hospital, Wilmington, DE., Sharma R; University of Texas Southwestern Medical Center, Dallas, TX., Grace RF; Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.
Jazyk: angličtina
Zdroj: Blood advances [Blood Adv] 2024 Oct 08; Vol. 8 (19), pp. 5112-5117.
DOI: 10.1182/bloodadvances.2024012707
Abstrakt: 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.)
Databáze: MEDLINE