Prospective Evaluation of the First Option, Second-Line Therapy in Childhood Chronic Immune Thrombocytopenia: Splenectomy or Immunomodulation

• Published in: The Journal of pediatrics Vol. 231; pp. 223 - 230
• Main Authors: 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
• Format: Journal Article
• Language: English
• Published: Elsevier 01.12.2020
• Subjects: Life Sciences; second-line treatment; hydroxychloroquine; children; azathioprine; rituximab
• ISSN: 0022-3476; 1097-6833
• DOI: 10.1016/j.jpeds.2020.12.018

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.