Second‐line treatment trends and long‐term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years

• Published in: British journal of haematology 2020-06, Vol.189 (5), p.931-942
• Main Authors: Ducassou, Stéphane, Gourdonneau, Anne, Fernandes, Helder, Leverger, Guy, Pasquet, Marlène, Fouyssac, Fanny, Bayart, Sophie, Bertrand, Yves, Michel, Gérard, Jeziorski, Eric, Thomas, Caroline, Abouchallah, Wadih, Viard, Florence, Guitton, Corinne, Cheikh, Nathalie, Pellier, Isabelle, Carausu, Liana, Droz, Cécile, Leblanc, Thierry, Aladjidi, Nathalie
• Format: Article
• Language: English
• Subjects: Adolescent; Adrenal Cortex Hormones - therapeutic use; Antinuclear antibodies; Azathioprine; Child; Child, Preschool; Children; Combined Modality Therapy; Diagnosis; Female; Follow-Up Studies; France - epidemiology; Hematology; Humans; Hydroxychloroquine; Hydroxychloroquine - therapeutic use; immune thrombocytopenic purpura; Immunoglobulins, Intravenous - therapeutic use; Immunosuppressive Agents - therapeutic use; immunosuppressive treatments; Male; Monoclonal antibodies; observational cohort; Observational studies; Observational Studies as Topic - statistics & numerical data; Prospective Studies; Purpura; Purpura, Thrombocytopenic, Idiopathic - drug therapy; Purpura, Thrombocytopenic, Idiopathic - epidemiology; Purpura, Thrombocytopenic, Idiopathic - surgery; Rare diseases; Remission; Remission Induction; Rituximab; Salvage Therapy; Sex Distribution; Splenectomy; Thrombocytopenic purpura; Treatment Outcome
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.16448

Summary Childhood chronic immune thrombocytopenic purpura (cITP) is a rare disease. In severe cases, there is no evidence for the optimal therapeutic strategy. Our aim was to describe the real‐life management of non‐selected children with cITP at diagnosis. Since 2004, patients less than 18 years old with cITP have been enrolled in the national prospective cohort, OBS’CEREVANCE. From 1990 to 2014, in 29 centres, 392 children were diagnosed with cITP. With a median follow‐up of six years (2·0–25), 45% did not need second‐line therapy, and 55% (n = 217) received one or more second lines, mainly splenectomy (n = 108), hydroxychloroquine (n = 61), rituximab (n = 61) or azathioprine (n = 40). The overall five‐year further second‐line treatment‐free survival was 56% [95% CI 49·5–64.1]. The use of splenectomy significantly decreased over time. Hydroxychloroquine was administered to children with positive antinuclear antibodies, more frequently older and girls, and reached 55% efficacy. None of the patients died. Ten years after the initial diagnosis, 55% of the 56 followed children had achieved complete remission. Children with cITP do not need second‐line treatments in 45% of cases. Basing the treatment decision on the pathophysiological pathways is challenging, as illustrated by ITP patients with positive antinuclear antibodies treated with hydroxychloroquine.