Pediatric refractory chronic immune thrombocytopenia: Identification, patients' characteristics, and outcome.
Journal
American journal of hematology
ISSN: 1096-8652
Titre abrégé: Am J Hematol
Pays: United States
ID NLM: 7610369
Informations de publication
Date de publication:
23 Apr 2024
23 Apr 2024
Historique:
received:
29
03
2024
accepted:
06
04
2024
medline:
23
4
2024
pubmed:
23
4
2024
entrez:
23
4
2024
Statut:
aheadofprint
Résumé
Refractory chronic immune thrombocytopenia (r-cITP) is one of the most challenging situations in chronic immune thrombocytopenia (cITP). Pediatric r-cITP is inconsistently defined in literature, contributing to the scarcity of data. Moreover, no evidence is available to guide the choice of treatment. We compared seven definitions of r-cITP including five pediatric definitions in 886 patients with cITP (median [min-max] follow-up 5.3 [1.0-29.3] years). The pediatric definitions identified overlapping groups of various sizes (4%-20%) but with similar characteristics (higher proportion of immunopathological manifestations [IM] and systemic lupus erythematosus [SLE]), suggesting that they adequately captured the population of interest. Based on the 79 patients with r-cITP (median follow-up 3.1 [0-18.2] years) according to the CEREVANCE definition (≥3 second-line treatments), we showed that r-cITP occurred at a rate of 1.15% new patients per year and did not plateau over time. In multivariate analysis, older age was associated with r-cITP. One patient (1%) experienced two grade five bleeding events after meeting r-cITP criteria and while not receiving second-line treatment. The cumulative incidence of continuous complete remission (CCR) at 2 years after r-cITP diagnosis was 9%. In this analysis, splenectomy was associated with a higher cumulative incidence of CCR (hazard ratio: 5.43, 95% confidence interval: 1.48-19.84, p = 7.8 × 10
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : French Ministry of Health (Programme Hospitalier de Recherche Clinique [PHRC] and filière de Santé Maladies Rares Immuno-Hématologiques [MARIH])
Organisme : Association Bordelaise pour l'Avancement des Sciences Pédiatriques (ABASP)
Organisme : Association pour la Recherche et les Maladies Hématologiques de l'Enfant (RMHE)
Organisme : Association Française du Syndrome d'Evans (AFSE)
Organisme : O-CYTO Patients' Association
Organisme : GlaxoSmithKline
Organisme : AMGEN
Organisme : Novartis
Informations de copyright
© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.
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