Pediatric refractory chronic immune thrombocytopenia: Identification, patients' characteristics, and outcome.

Autor: Pincez T; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Division of Hematology-Oncology, Charles-Bruneau Cancer Center, Department of Pediatrics, Sainte-Justine University Hospital, Université de Montréal, Montréal, Québec, Canada., Fernandes H; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Haemato-Immunology, CIC1401, INSERM CICP, Bordeaux University Hospital, Bordeaux, France., Fahd M; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Hematology Unit, Robert-Debré University Hospital, AP-HP, Paris, France., Pasquet M; Pediatric Oncology Immunology Hematology Unit, Children's University Hospital, Toulouse, France., Chahla WA; Department of Pediatric Hematology, Jeanne de Flandre Hospital, Lille University Hospital, Lille, France., Granel J; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Haemato-Immunology, CIC1401, INSERM CICP, Bordeaux University Hospital, Bordeaux, France., Ducassou S; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Haemato-Immunology, CIC1401, INSERM CICP, Bordeaux University Hospital, Bordeaux, France., Thomas C; Pediatric Hematology Unit, Nantes University Hospital, Nantes, France., Garnier N; Institute of Pediatric Hematology and Oncology, Hospices Civils de Lyon, Lyon, France., Jeziorski E; Pediatric Oncology Hematology Unit, Arnaud de Villeneuve University Hospital, Montpellier, France., Bayart S; Pediatric Hematology Unit, Rennes University Hospital, Rennes, France., Chastagner P; Department of Pediatric Hematology and Oncology, Children's University Hospital, Nancy, France., Cheikh N; Department of Pediatric Hematology-Oncology, Besançon University Hospital, Besançon, France., Guitton C; Department of Pediatrics, Bicêtre University Hospital, AP-HP, Le Kremlin-Bicêtre, France., Paillard C; Department of Pediatric Hematology and Oncology, Hautepierre University Hospital, Strasbourg, France., Lejeune J; Department of Pediatric Hematology-Oncology, Clocheville Hospital, Tours University Hospital, Tours, France., Millot F; Department of Pediatric Hematology, Poitiers University Hospital, Poitiers, France., Li-Thiao Te V; Department of Pediatric Hematology/Oncology, Amiens University Hospital, Amiens, France., Mallebranche C; Pediatric Immuno-Hemato-Oncology, Angers University Hospital, CRCI2NA, UMR Inserm CNRS, Université d'Angers, Université de Nantes, Angers, France., Pellier I; Pediatric Immuno-Hemato-Oncology, Angers University Hospital, CRCI2NA, UMR Inserm CNRS, Université d'Angers, Université de Nantes, Angers, France., Castelle M; Pediatric Hematology-Immunology and Rheumatology Department, Necker-Enfants Malades Hospital, AP-HP, Paris, France., Armari-Alla C; Pediatric Hematology-Oncology Department, Grenoble University Hospital, Grenoble, France., Carausu L; Department of Pediatric Hematology, CHU de Brest, Brest, France., Piguet C; Pediatric Oncology Hematology Unit, Limoges University Hospital, Limoges, France., Benadiba J; Department of Hematology-Oncology Pediatrics, Nice University Hospital, Nice, France., Pluchart C; Pediatric Hematology-Oncology Unit, Institut Jean Godinot, Reims University Hospital, Reims, France., Stephan JL; Department of Pediatric Oncology, University Hospital of Saint Etienne, North Hospital, Saint Etienne, France., Deparis M; Pediatric Oncology-Hematology Unit Department, Caen University Hospital, Caen, France., Briandet C; Department of Pediatrics, Dijon University Hospital, Dijon, France., Doré E; Pediatric Unit, Clermont-Ferrand University Hospital, Clermont-Ferrand, France., Marie-Cardine A; Department of Pediatric Hematology and Oncology, Rouen University Hospital, Rouen, France., Barlogis V; Department of Pediatric Hematology, La Timone Hospital, Marseille University Hospital, Marseille, France., Leverger G; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Hematology Oncology Unit, Hôpital Armand Trousseau, AP-HP, Sorbonne Université Paris, Paris, France., Héritier S; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Hematology Oncology Unit, Hôpital Armand Trousseau, AP-HP, Sorbonne Université Paris, Paris, France., Aladjidi N; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Haemato-Immunology, CIC1401, INSERM CICP, Bordeaux University Hospital, Bordeaux, France., Leblanc T; Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux and Paris, France.; Pediatric Hematology Unit, Robert-Debré University Hospital, AP-HP, Paris, France.; Université Paris-Cité, Paris, France.
Jazyk: angličtina
Zdroj: American journal of hematology [Am J Hematol] 2024 Jul; Vol. 99 (7), pp. 1269-1280. Date of Electronic Publication: 2024 Apr 23.
DOI: 10.1002/ajh.27337
Abstrakt: 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 -4 ). In sum, children with cITP may be diagnosed with r-cITP at any time point of the follow-up and are at increased risk of IM and SLE. Second-line treatments seem to be effective for preventing grade 5 bleeding. Splenectomy may be considered to achieve CCR.
(© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.)
Databáze: MEDLINE
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