Abstract
Treatment of children with refractory immune thrombocytopenic purpura (ITP) is challenging and poorly established. We retrospectively reviewed the clinical data of 87 patients under the age of 16 years who were diagnosed with ITP from April 1998 to March 2017 in our institution. Refractory ITP was defined as a platelet count of < 50 × 109/L at 14 days after receiving intravenous immunoglobulin (IVIG) and prednisolone. We presumed that there was a pathophysiological overlap between refractory ITP and refractory thrombocytopenia (RT): a subtype of refractory cytopenia of childhood (RCC). Immunosuppressive therapies including anti-thymocyte globulin and cyclosporine (CsA) have been adopted for children with RCC in Japan. Thus, from 2009 onwards, we changed the diagnosis from refractory ITP to RT and introduced CsA for refractory ITP/RT. Nine of 42 patients developed refractory ITP in the 1998–2008 group, who received conventional treatments such as IVIG and steroid therapy. Eight of 45 patients developed refractory ITP in the 2009–2017 group, who received CsA with or without IVIG therapy. The response rate at three years after diagnosis was significantly higher in the 2009–2017 group (98%) than in the 1998–2008 group (83%) (p = 0.019). In conclusion, our strategy of introducing CsA for refractory ITP/RT contributed to better outcomes.
Highlights
Treatment of children with refractory immune thrombocytopenic purpura (ITP) is challenging and poorly established
We presumed that there was a pathophysiological overlap between refractory ITP and refractory thrombocytopenia (RT), which is a subtype of refractory cytopenia of childhood (RCC)
A total of 87 children (50 boys and 37 girls) with ITP were included in this study, of which 42 children were in the 1998–2008 group and 45 children were in the 2009–2017 group
Summary
Treatment of children with refractory immune thrombocytopenic purpura (ITP) is challenging and poorly established. Nine of 42 patients developed refractory ITP in the 1998–2008 group, who received conventional treatments such as IVIG and steroid therapy. Eight of 45 patients developed refractory ITP in the 2009–2017 group, who received CsA with or without IVIG therapy. The incidence of complete remission in children with chronic ITP who received IVIG, glucocorticoid therapy, or splenectomies was 30–52% at five y ears[6,7]. From 2009 onwards, we changed the diagnosis from refractory ITP to RT and introduced CsA treatment for such patients instead of glucocorticoid therapy and splenectomy. The long-term outcomes of 87 children with ITP in our institution and aim to evaluate whether the strategy of introducing CsA for refractory ITP/RT was appropriate
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