Immune thrombocytopenia – a single centre experience

Česká verzia

Authors: V. Fiamoli ¹; J. Blatný ¹; P. Ovesná ²
Authors place of work: Oddělení dětské hematologie a biochemie, Fakultní nemocnice Brno ¹; Institut biostatistiky a analýz s. r. o., Brno ²
Published in the journal: Čes-slov Pediat 2021; 76 (8): 431-439.
Category: Original Papers

Summary

Objective: Current treatment options and their efficacy in patients with immune thrombocytopenia – a single centre experience.

Patients and methods: Between years 2009 and 2019, 153 children with immune thrombocytopenia aged 4 months to 17 years (79 boys and 74 girls) were treated at our department. Early diagnosis of immune thrombocytopenia was made in 101 (66%) children, whereas 21 (14%) patients developed a persistent form and 31 (20%) chronic form.

Results: In 52 (34%) patients, „watch and wait“ strategy was sufficient. At the time of diagnosis, 72 (47%) patients were treated with intravenous immunoglobulins, 20 (13%) with steroids only, and 9 (6%) required both intravenous immunoglobulins and steroids. A complete response was achieved in 128 (84%) patients, 12 (8%) patients achieved partial response, and 13 (8%) had no response at all. There were no significant differences in the best achieved response between boys and girls or children of different ages. When evaluating the efficacy of steroid versus intravenous immunoglobulins treatment, there was no significant difference in the best response between the two groups (p=0.793). At least one relapse of immune thrombocytopenia occurred in 38 (25%) patients.

Patients with persistent or chronic immune thrombocytopenia were commenced on thrombopoietin analogues – 14 (9%), rituximab – 4 (3%), mycophenolate mofetil – 2 (1%), and sirolimus – 2 (1%). Splenectomy had to be performed only in three (2%) patients with chronic immune thrombocytopenia who failed all other previous treatment attempts.

Conclusion: Immune thrombocytopenia is relatively frequent haematological diagnosis in children. Most patients have achieved at least partial remission after the first-line treatment. For children with chronic immune thrombocytopenia, there is currently relatively wide range of treatment options available – thrombopoietin analogues, rituximab, sirolimus, mycophenolate mofetil and other immunosuppressives. Splenectomy, on the other hand, is considered as a last option, mainly for its potential risk of severe infections.

Keywords:

treatment options – children – immune thrombocytopenia

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