TY - JOUR
T1 - Prospective Evaluation of the First Option, Second-Line Therapy in Childhood Chronic Immune Thrombocytopenia
T2 - Splenectomy or Immunomodulation
AU - Ducassou, Stéphane
AU - Fernandes, Helder
AU - Savel, Hélène
AU - Bertrand, Yves
AU - Leblanc, Thierry
AU - Abou Chahla, Wadih
AU - Pasquet, Marlène
AU - Leverger, Guy
AU - Barlogis, Vincent
AU - Thomas, Caroline
AU - Bayart, Sophie
AU - Pellier, Isabelle
AU - Armari-Alla, Corinne
AU - Guitton, Corinne
AU - Cheikh, Nathalie
AU - Kherfellah, Djamel
AU - Vassal, Gilles
AU - Thiébaut, Rodolphe
AU - Laghouati, Salim
AU - Aladjidi, Nathalie
N1 - Publisher Copyright:
© 2020
PY - 2021/1/1
Y1 - 2021/1/1
N2 - 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.
AB - 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.
KW - azathioprine
KW - children
KW - hydroxychloroquine
KW - rituximab
KW - second-line treatment
UR - http://www.scopus.com/inward/record.url?scp=85099125573&partnerID=8YFLogxK
U2 - 10.1016/j.jpeds.2020.12.018
DO - 10.1016/j.jpeds.2020.12.018
M3 - Article
C2 - 33340549
AN - SCOPUS:85099125573
SN - 0022-3476
JO - Journal of Pediatrics
JF - Journal of Pediatrics
ER -