A proposal for new definition (s) and management approach to paediatric refractory ITP: Reflections from the Intercontinental ITP Study Group

Abstract

SummaryImmune thrombocytopenia (ITP) in children is a relatively mild and self‐limited disorder with the majority of children demonstrating normalization of platelet count by 12 months from diagnosis. Because of this, many children with ITP can be observed without the need for treatment. When needed, treatment with either intravenous immunoglobulin (IVIG) or corticosteroids is highly effective (>80% IVIG and >95% corticosteroids). For those children who require second‐line therapies, response rates of >60% are seen with both the thrombopoietin‐receptor agonists and rituximab. Despite this, some children will have ‘refractory’ ITP (rITP) with poor or transient responses to platelet‐raising therapies. Here, we review the clinical features of rITP in children, outline proposed classifications and explore potential predictors for children with rITP.

Multidrug rescue for pediatric refractory ITP complicated by intracranial bleeding

“…Although we cannot identify which specific treatment component had the most significant impact on the patient's remission, this case report supports the opinion that prompt initiation of second-line treatments in refractory ITP cases is justified [2,4,5]. It is highly probable that a combination of medications, which exhibit a cumulative effect, played a crucial role.…”

“…As the previous name suggests, an ITP diagnosis follows the exclusion of other identifiable diseases and confirmation via successful therapy [1]. It is estimated that c.95% of children with ITP experience spontaneous remission or a positive response to initial treatments, while the majority of the remaining non-responders eventually achieve platelet normalization with second-line therapies [2,3]. Newly diagnosed refractory ITP is rare among pediatric patients.…”

“…Although there is no consensus definition for this condition in childhood, it is generally considered after a failure to achieve a response to two standard first-line treatments, i.e. intravenous immunoglobulin (IVIG) and steroids [2,4]. It is becoming accepted that newly diagnosed refractory ITP requires intensive diagnostics and a rapid transition to second-line treatments [2,5].…”

“…intravenous immunoglobulin (IVIG) and steroids [2,4]. It is becoming accepted that newly diagnosed refractory ITP requires intensive diagnostics and a rapid transition to second-line treatments [2,5].…”