Pediatric refractory immune thrombocytopenia: A systematic review

Vanderbilt University School of Medicine, Nashville, Tennessee, USA. University of Ottawa, Faculty of Medicine, Ottawa, Ontario, Canada. Division of Hematology/Oncology, Department of Pediatrics, Children's Hospital of Eastern Ontario Research Institute, Ottawa, Ontario, Canada. Department of Pediatrics, Baylor College of Medicine, Texas Children's Hematology Center, Houston, Texas, USA. Division of Pediatric Hematology and Oncology, New York Presbyterian Hospital/Weill Cornell Medical College, New York, New York, USA. Cancer and Blood Disorders Program, Children's Minnesota, Minneapolis, Minnesota, USA. Division of Pediatric Hematology/Oncology, McMaster University, Hamilton, Ontario, Canada. Pediatric Hematology/Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA. University of Alabama at Birmingham, Birmingham, Alabama, USA. Department of Pediatrics, Perelman School of Medicine at University of Pennsylvania, Philadelphia, Pennsylvania, USA. Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Pediatric Blood & Cancer. 2023;70(3):e30173
Abstract
Pediatric immune thrombocytopenia (ITP) is an acquired disorder associated with autoimmune destruction and impairment of platelet production in children. Some children exhibit poor or transient response to ITP-directed treatments and are referred to as having refractory ITP (rITP). There is currently no consensus on the definition of rITP, nor evidence-based treatment guidelines for patients with rITP. After a survey of pediatric ITP experts demonstrated lack of consensus on pediatric rITP, we pursued a systematic review to examine the reported clinical phenotypes and treatment outcomes in pediatric rITP. The search identified 253 relevant manuscripts; following review, 11 studies proposed a definition for pediatric rITP with no consensus amongst them. Most definitions included suboptimal response to medical management, while some outlined specific platelet thresholds to define this suboptimal response. Common attributes identified in this study should be used to propose a comprehensive definition, which will facilitate outcome comparisons of future rITP studies.
Study details
Study Design : Systematic Review
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine