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).
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.
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.
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.