Prospective evaluation of the first option, second-line Therapy in childhood chronic immune thrombocytopenia: splenectomy or immunomodulation
Langue
EN
Article de revue
Ce document a été publié dans
Journal of Pediatrics. 2021-04, vol. 231, p. 223-230
Résumé en anglais
OBJECTIVE: To describe four subgroups of pediatric patients treated with splenectomy, hydroxychloroquine, azathioprine or rituximab as the first-option, second-line treatment for chronic immune thrombocytopenia (cITP). ...Lire la suite >
OBJECTIVE: To describe four subgroups of pediatric patients treated with splenectomy, hydroxychloroquine, azathioprine or rituximab as the first-option, second-line treatment for chronic immune thrombocytopenia (cITP). STUDY DESIGN: Selection of patients with cITP 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 (EFS) was 62% (95%CI: 55; 71). It was 85% (95%CI: 77; 95) for the 56 splenectomized patients, 60% (95%: 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 EFS (p = 0.05). Female teenagers with ANA positivity benefited from hydroxychloroquine therapy. CONCLUSION: 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.< Réduire
Mots clés en anglais
Children
Second-line treatment
Rituximab
Hydroxychloroquine
Azathioprine
Unités de recherche