Contributions
Abstract: PB2118
Type: Publication Only
Background
Thrombopoietin-receptor agonists (TRA), romiplostim and eltrombopag, are part of the treatment of chronic immune thrombocytopenia (ITP), resistant to first line therapy (corticosteroids and/or immunoglobulins) and with a significant bleeding risk. Both are approved for adult patients, but only eltrombopag was approved for pediatric use. When used before splenectomy, these treatments may serve as a bridge for surgery or even postpone/avoid the procedure.
Aims
In this report, we aim to evaluate the response to TRA treatment in patients with ITP and associated side effects in our center.
Methods
Inclusion criteria: patients with ITP resistant to first line treatment. Patients characteristics, response to TRA, clinical evolution and adverse effects were evaluated by retrospective analysis.
Results
Thirty-eight patients with ITP were included: 31.4% (12) were male and the median age at diagnosis was 38 years. 44.7% (17) had relapsed/resistant disease after splenectomy and 13.2% (5) were treated with a TRA as a bridge for this procedure. Sixteen (42.1%) of ITP patients were treated with romiplostim: 12 patients (75%) had a response to treatment, and 4 (25%) were resistant. In 11 of these patients, romiplostim was replaced by eltrombopag, either because of resistant disease, or more convenient administration (oral therapy). Thirty-three (86.8%) patients were treated with eltrombopag (5 pediatric cases): 27 patients (81.8%) responded while 6 patients had resistant disease (3 of these were HIV positive). The response rate was higher in patients with previous splenectomy (91.7% with romiplostim and 92.9% with eltrombopag) compared to those with no previous splenectomy (25% with romiplostim and 73.7% with eltrombopag). Six patients maintained response after treatment suspension (5 treated with eltrombopag and 1 treated with romiplostim). Generally, both treatments were well tolerated, with only one case of eltrombopag suspension because of a thromboembolic event.
Conclusion
In the current study, both TRA were effective in the treatment of ITP resistant to several lines of treatment, with similar response rates. As described in the literature, the response rate was higher in patients with previous splenectomy, and some cases maintained response after treatment suspension.
Session topic: 32. Platelets disorders
Keyword(s): Immune thrombocytopenia (ITP)
Abstract: PB2118
Type: Publication Only
Background
Thrombopoietin-receptor agonists (TRA), romiplostim and eltrombopag, are part of the treatment of chronic immune thrombocytopenia (ITP), resistant to first line therapy (corticosteroids and/or immunoglobulins) and with a significant bleeding risk. Both are approved for adult patients, but only eltrombopag was approved for pediatric use. When used before splenectomy, these treatments may serve as a bridge for surgery or even postpone/avoid the procedure.
Aims
In this report, we aim to evaluate the response to TRA treatment in patients with ITP and associated side effects in our center.
Methods
Inclusion criteria: patients with ITP resistant to first line treatment. Patients characteristics, response to TRA, clinical evolution and adverse effects were evaluated by retrospective analysis.
Results
Thirty-eight patients with ITP were included: 31.4% (12) were male and the median age at diagnosis was 38 years. 44.7% (17) had relapsed/resistant disease after splenectomy and 13.2% (5) were treated with a TRA as a bridge for this procedure. Sixteen (42.1%) of ITP patients were treated with romiplostim: 12 patients (75%) had a response to treatment, and 4 (25%) were resistant. In 11 of these patients, romiplostim was replaced by eltrombopag, either because of resistant disease, or more convenient administration (oral therapy). Thirty-three (86.8%) patients were treated with eltrombopag (5 pediatric cases): 27 patients (81.8%) responded while 6 patients had resistant disease (3 of these were HIV positive). The response rate was higher in patients with previous splenectomy (91.7% with romiplostim and 92.9% with eltrombopag) compared to those with no previous splenectomy (25% with romiplostim and 73.7% with eltrombopag). Six patients maintained response after treatment suspension (5 treated with eltrombopag and 1 treated with romiplostim). Generally, both treatments were well tolerated, with only one case of eltrombopag suspension because of a thromboembolic event.
Conclusion
In the current study, both TRA were effective in the treatment of ITP resistant to several lines of treatment, with similar response rates. As described in the literature, the response rate was higher in patients with previous splenectomy, and some cases maintained response after treatment suspension.
Session topic: 32. Platelets disorders
Keyword(s): Immune thrombocytopenia (ITP)