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Prospective Evaluation of the First Option, Second-Line Therapy in Childhood Chronic Immune Thrombocytopenia: Splenectomy or Immunomodulation.
Ducassou, Stéphane; Fernandes, Helder; Savel, Hélène; Bertrand, Yves; Leblanc, Thierry; Abou Chahla, Wadih; Pasquet, Marlène; Leverger, Guy; Barlogis, Vincent; Thomas, Caroline; Bayart, Sophie; Pellier, Isabelle; Armari-Alla, Corinne; Guitton, Corinne; Cheikh, Nathalie; Kherfellah, Djamel; Vassal, Gilles; Thiébaut, Rodolphe; Laghouati, Salim; Aladjidi, Nathalie.
Afiliação
  • Ducassou S; Pediatric Hematology Unit, CIC1401, INSERM CICP, University Hospital of Bordeaux, Bordeaux, France; Centre de Référence National des Cytopénies Autoimmunes de l'Enfant (CEREVANCE), University Hospital of Bordeaux, Bordeaux, France.
  • Fernandes H; Centre de Référence National des Cytopénies Autoimmunes de l'Enfant (CEREVANCE), University Hospital of Bordeaux, Bordeaux, France.
  • Savel H; Unité de Soutien Méthodologique à la Recherche Clinique (USMR) University Hospital of Bordeaux, Bordeaux, France.
  • Bertrand Y; Pediatric Hematology Unit, Institute of Pediatric Hematology and Oncology, Claude Bernard University Lyon, Lyon, France.
  • Leblanc T; Department of Hematology, APHP - Hopital Robert Debré, Centre de Ré́férence National des Cytopénies Autoimmunes de l'Enfant (CEREVANCE), Paris, France.
  • Abou Chahla W; Department of pediatric Hematology, University Hospital of Lille, Lille, France.
  • Pasquet M; Department of pediatric Hematology, University Hospital of Toulouse, Toulouse, France.
  • Leverger G; Sorbonne Université, INSERM, Centre de Recherche Saint Antoine, AP-HP, Hôpital Armand Trousseau, Service d'Hématologie Oncologie Pédiatrique, Centre de Référence National des Cytopénies Autoimmunes de lEnfant (CEREVANCE), Paris, France.
  • Barlogis V; Department of Pediatric Hematology AP-HM Hôpital Timone Enfant, Marseille, France.
  • Thomas C; Department of Pediatric Hematology and Oncology, University Hospital of Nantes, Nantes, France.
  • Bayart S; Department of Pediatric Hematology, University Hospital of Rennes, Rennes, France.
  • Pellier I; Department of Pediatric Hematology and Oncology, University Hospital of Angers, Angers, France.
  • Armari-Alla C; Department of Pediatric Hematology, University Hospital of Grenoble, Grenoble, France.
  • Guitton C; Department of Pediatrics, University Hospital of Bicêtre, Le Kremlin-Bicêtre, France.
  • Cheikh N; Department of Pediatric Hematology and Oncology, University Hospital of Besançon, Besançon, France.
  • Kherfellah D; Centre de Référence National des Cytopénies Autoimmunes de l'Enfant (CEREVANCE), University Hospital of Bordeaux, Bordeaux, France.
  • Vassal G; Gustave Roussy Cancer Center, Clinical Research Direction, Paris-Saclay University, Villejuif, France.
  • Thiébaut R; Unité de Soutien Méthodologique à la Recherche Clinique (USMR) University Hospital of Bordeaux, Bordeaux, France.
  • Laghouati S; Gustave Roussy Cancer Center, Pharmacovigilance Unit, Paris-Saclay University, Villejuif, France.
  • Aladjidi N; Pediatric Hematology Unit, CIC1401, INSERM CICP, University Hospital of Bordeaux, Bordeaux, France; Centre de Référence National des Cytopénies Autoimmunes de l'Enfant (CEREVANCE), University Hospital of Bordeaux, Bordeaux, France. Electronic address: nathalie.aladjidi@chu-bordeaux.fr.
J Pediatr ; 2020 Dec 17.
Artigo em Inglês | MEDLINE | ID: mdl-33340549
ABSTRACT

OBJECTIVE:

To describe 4 subgroups of pediatric patients treated with splenectomy, hydroxychloroquine, azathioprine, or rituximab as the first-option, second-line treatment for chronic immune thrombocytopenia. STUDY

DESIGN:

Selection of patients with chronic immune thrombocytopenia from the French national prospective cohort of pediatric autoimmune cytopenia OBS'CEREVANCE and VIGICAIRE study, treated by splenectomy, hydroxychloroquine, azathioprine, or rituximab as a first second-line treatment.

RESULTS:

For 137 patients, treated between 1989 and 2016, the median follow-up after diagnosis and after treatment initiation was 8.5 (2.8-26.4) years and 4.7 (1.1-25.1) years, respectively. Median age at diagnosis and at initiation of treatment were 9 (0.7; 16) and 12 (2; 18.1) years, respectively without significant difference between subgroups. For the whole cohort, 24-month event-free survival was 62% (95% CI 55; 71). It was 85% (95% CI 77; 95) for the 56 patients treated with splenectomy, 60% (95% CI 44; 84) for the 23 patients treated with rituximab, 46% (95% CI 30; 71) for the 24 patients treated with azathioprine, and 37% (95% CI 24; 59) for the 34 patients treated with hydroxychloroquine (log-rank P < .0001). For the splenectomy subgroup, being older than 10 years at splenectomy tended to improve event-free survival (P = .05). Female teenagers with antinuclear antibody positivity benefited from hydroxychloroquine therapy.

CONCLUSIONS:

This national study, limiting pitfalls in the analysis of the effects of second-line therapies, showed that splenectomy remains the treatment associated with the better response at 24 months.
Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Tipo de estudo: Estudo observacional / Fatores de risco Idioma: Inglês Ano de publicação: 2020 Tipo de documento: Artigo País de afiliação: França

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Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Tipo de estudo: Estudo observacional / Fatores de risco Idioma: Inglês Ano de publicação: 2020 Tipo de documento: Artigo País de afiliação: França
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