Abstract
The aim of the present study was to evaluate the efficacy and safety of eltrombopag, an oral thrombopoietin receptor agonist, in patients with chronic immune thrombocytopenia (ITP). A total of 285 chronic ITP patients (187 women, 65.6%; 98 men, 34.4%) followed in 55 centers were enrolled in this retrospective cohort. Response to treatment was assessed according to platelet count (/mm3) and defined as complete (platelet count of >100,000/mm3), partial (30,000-100,000/mm3 or doubling of platelet count after treatment), or unresponsive (<30,000/mm3). Clinical findings, descriptive features, response to treatment, and side effects were recorded. Correlations between descriptive, clinical, and hematological parameters were analyzed. The median age at diagnosis was 43.9±20.6 (range: 3-95) years and the duration of follow-up was 18.0±6.4 (range: 6-28.2) months. Overall response rate was 86.7% (n=247). Complete and partial responses were observed in 182 (63.8%) and 65 (22.8%) patients, respectively. Thirty-eight patients (13.4%) did not respond to eltrombopag treatment. For patients above 60 years old (n=68), overall response rate was 89.7% (n=61), and for those above 80 years old (n=12), overall response rate was 83% (n=10). Considering thrombocyte count before treatment, eltrombopag significantly increased platelet count at the 1st, 2nd, 3rd, 4th, and 8th weeks of treatment. As the time required for partial or complete response increased, response to treatment was significantly reduced. The time to reach the maximum platelet levels after treatment was quite variable (1-202 weeks). Notably, the higher the maximum platelet count after eltrombopag treatment, the more likely that side effects would occur. The most common side effects were headache (21.6%), weakness (13.7%), hepatotoxicity (11.8%), and thrombosis (5.9%). Results of the current study imply that eltrombopag is an effective therapeutic option even in elderly patients with chronic ITP. However, patients must be closely monitored for response and side effects during treatment. Since both response and side effects may be variable throughout the follow-up period, patients should be evaluated dynamically, especially in terms of thrombotic risk factors.
Highlights
Results of the current study imply that eltrombopag is an effective therapeutic option even in elderly patients with chronic immune thrombocytopenia (ITP)
Immune thrombocytopenia (ITP) is an acquired disorder characterized by a transient or persistent decrease in platelets accompanied with an increased risk of bleeding [1,2,3]
Our results suggest that platelet counts obtained at different intervals in the course of eltrombopag treatment can serve as important predictors for treatment response and occurrence of side effects
Summary
Immune thrombocytopenia (ITP) is an acquired disorder characterized by a transient or persistent decrease in platelets accompanied with an increased risk of bleeding [1,2,3]. Immune thrombocytopenia has been linked to an increased rate of immune-mediated platelet destruction; the exact pathophysiological mechanism is still unclear [3]. In chronic ITP, antiplatelet antibodies facilitate platelet destruction and prevent the release of platelets from megakaryocytes, resulting in mild to serious thrombocytopenia. Therapeutic strategies for first- or second-line treatment such as corticosteroids, intravenous immunoglobulin, and splenectomy can reduce the destruction of antibody-coated platelets, but the efficacy is limited and serious adverse effects can be seen [7]. Use of immunosuppressive drugs has been restricted because of serious adverse events and splenectomy has been linked to important drawbacks such as infection and thrombosis. Monitoring patients for the effectiveness of the treatment and for side effects is an important issue in the improvement of therapeutic outcomes
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