Pediatric ITP Consortium Publications
Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia
A total of 591 patients with ITP were genotyped and 6 variants met genome-wide significance in comparing children with ITP to a cohort of health children. These genes have been linked to the canonical Wnt signaling pathway.
Kim TO, Geris JM, Flanagan JM, et al. Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia. Blood Adv. 2024 Nov 12;8(21):5529-5538.
What is in a name: defining pediatric refractory ITP
This manuscript describes the work of the consortium to come up with a consensus-based definitions of pediatric ITP that is refractory to emergent therapy and multiple classes of disease-modifying therapy.
Nakano TA, Grimes AB, Klaassen RJ, et al. What is in a name: defining pediatric refractory ITP. Blood Adv. 2024 Oct 8; 8(19): 5112-5117.
Pediatric refractory immune thrombocytopenia: A systematic review.
This manuscript reports the findings of a survey of pediatric hematologists on the definition of refractory ITP and the results of a systematic review which identified the clinical phenotypes and treatment outcomes of refractory ITP.
Ibrahim L, Dong SX, O'Hearn K, et al Pediatric refractory immune thrombocytopenia: A systemic review. Pediatr Blood Cancer. 2023 Mar;70(3):e30173.
Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol.
This trial design manuscript describes the protocol for an ongoing randomized multicenter clinical trial for children with newly diagnosed ITP.
Shimano KA, Grace RF, Despotovic JM, et al. BMJ Open. 2021 Aug 27;11(8):e044885.
Quality of life is an important indication for second-line treatment in children with immune thrombocytopenia.
This manuscript reports the reasons hematologists started children with ITP on second-line therapies; quality of life was the most frequently cited reason for starting a second line agent.
Shimano KA, Neunert C, Bussel JB, et al. Pediatr Blood Cancer. 2021 Jun;68(6):e29023.
Fatigue in children and adolescents with immune thrombocytopenia.
A prospective, multi-center, observational study of 120 children starting second-line treatments for ITP evaluated ITP-associated fatigue in this population and the correlation with bleeding and platelet count and the effect of treatment.
Grace RF, Klaassen RJ, Shimano KA, et al. Br J Haematol. 2020 Oct;191(1):98-106.
Second-line treatments in children with ITP: Effect on Platelet count and patient-centered outcomes
A prospective, multi-center, observational study of 120 children starting second-line treatments for ITP compared treatment outcomes including platelet count, bleeding, and HRQoL utilizing the Kids ITP Tool (KIT)
Grace RF, Shimano KA, Bhat R, et al. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 Jul;94(7):741-750.
Association of a positive direct antiglobulin test with chronic immune thrombocytopenia and use of second line therapies in children: a multi-institutional review
A multi-institutional retrospective review of 311 pediatric patients with ITP identified clinical characteristics associated with disease course.
Kim TO, Grimes AB, Kirk S, et al. Association of a positive direct antiglobulin test with chronic immune thrombocytopenia and use of second line therapies in children: A multi-institutional review. Am J Hematol. 2019 Apr;94(4):461-466.
Physician decision making in selection of second-line treatments in immune thrombocytopenia in children
This prospective, observational, longitudinal cohort study of 120 children with ITP from 21 ICON centers examined physician treatment decisions in selecting specific second line agents in ITP.
Grace RF, Despotovic JM, Bennett CM, et al. Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. Am J Hematol 2018 Jul; 93(7):882-888.
Thrombopoietin Receptor Agonist (TPO-RA) Use in Pediatric Patients: Data from the Pediatric ITP Consortium of North America
The purpose of this multicenter retrospective study was to understand the off-label use of the thrombopoietin-receptor agonists in pediatric patients with ITP.
Neunert C, Despotovic J, Haley K, et al. Thrombopoietin Receptor Agonist Use in Children: Data from the Pediatric ITP Consortium of North America ICON2 Study. Pediatr Blood Cancer 2016 Aug; 63(8): 1407-13.
Sports Participation in Children and Adolescents with Immune Thrombocytopenia
Pediatric hematologists from North America (n=278) were surveyed about their counseling to families about sports participation in children with ITP.
Kumar M, Lambert MP, Breakey V, et al. Sports Participation in Children and Adolescents with Immune Thrombocytopenia (ITP). Pediatr Blood Cancer 2015 Dec; 62(12): 2223-5.
