Abstract
Objective: While splenectomy is the gold standard treatment for refractory primary immune thrombocytopenia (ITP) in adult, its place remains debated in children. The French Rare Disease Plan provided us the opportunity to conduct a collaborative study of the efficiency and tolerance of this procedure in childhood ITP. Patients and methods: A retrospective study was conducted in France in order to identify children with ITP treated with splenectomy during a 9-year period. A total of 78 children were included. Data from the ongoing CEREVANCE national cohort of childhood auto-immune cytopenia in 30 units were reviewed and completed by a direct contact with the referent physicians. International terminology for response definition was used. Relapsefree survival was assessed by the Kaplan-Meier method. Results: The median ages at ITP diagnosis and splenectomy were 9.6 and 12.4 years respectively. The median duration of ITP before splenectomy was 24 months (1-162); 62 children had chronic ITP. The median number of treatment lines before splenectomy was 2 (1-7). Laparoscopy was used in 81% of cases. Four children underwent immediate surgical complications. With a median follow-up of 41 months, complete remission (CR) was maintained at the latest news in 84% of children. CR was obtained in 77% of cases with intra-splenic platelets destruction, and in no case with non-splenic destruction (p=0.11). Using a very strict definition for relapse, the 5-year relapse-free survival was 51% [IC95% 37-64]. No death or overwhelming sepsis was reported. Conclusions: In this national study with a long term follow up, the excellent benefit/risk ratio of splenectomy for refractory ITP confirms that in skilled and concerted teams, the procedure is still at the forefront of curative treatments. Isotopic evaluation is of value but other prognostic factors for CR are to be determined. Lifelong survey of potential infectious and thrombotic risk at adult age has to be coordinated by the referring physician. The place for other therapeutic options, in order to postpone as late as possible the splenectomy in childhood ITP is now to be determined.
Highlights
Immune thrombocytopenia (ITP) is one of the rare causes of childhood thrombocytopenia, defined as peripheral isolated thrombocytopenia under 100 G/L
Patients and methods: A retrospective study was conducted in France in order to identify children with ITP treated with splenectomy during a 9-year period
Isotopic evaluation is of value but other prognostic factors for complete remission (CR) are to be determined
Summary
Immune thrombocytopenia (ITP) is one of the rare causes of childhood thrombocytopenia, defined as peripheral isolated thrombocytopenia under 100 G/L. ITP is usually an acute self-limiting disease. There are no evidence-based guidelines available for the treatment of chronic ITP in children. As the spleen is the organ primarily responsible for the destruction of antibody-sensitized platelets, splenectomy remains the most reliable therapeutic option in adult ITP, allowing long-term remission in approximately 60-70% of cases [15]. The place of splenectomy in childhood ITP is still debated, because of the unpredictable occurrence of spontaneous recovery, the paucity of purely pediatric comparative data, and the risk of Overwhelming PostSplenectomy Infections (OPSI). At a time when many new therapeutic options are available with various potential side effects, it seems essential to share our standard pediatric practices, and to elaborate more precise guidelines highlighting the benefit/risk balance of each option
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
