Predictive factors of response to eltrombopag and romiplostim in Egyptian immune thrombocytopenia patients: a single center experience

Background Thrombopoietin receptor agonists (TPO-RA) are a well-established treatment in patients with immune thrombocytopenia (ITP). Predictors of response to some lines of treatment in ITP have been reported; yet, to date there are no predictors of response to TPO-RA were identified. We aimed to identify predictive factors of response to TPO-RA in adult ITP patients to avoid unwanted adverse effects and to individualize the treatment. Patients and methods We investigated demographic features, clinical-laboratory data as well as previous lines of treatment in 48 adult ITP patients who received TPO-RA for at least 3 months duration to detect reliable predictive factors of response to TPO-RA, in addition, health-related quality of life and fatigue burden was assessed in all studied ITP patients using 2 questionnaires which are 36-item short-form health survey and functional assessment of chronic illness therapy. Results The percentage of platelet change from days 0 to 28 of initiation of romiplostim can be a predictive factor of response to treatment with romiplostim (P=0.008) but none of the other studied factors has influenced response to TPO-RA. Both 36-item short-form health survey domains, as well as functional assessment of chronic illness therapy questionnaires had no statistically significant difference between the romiplostim and eltrombopag groups. Conclusion Slow or minimal change of platelet count during the first month of therapy with romiplostim could be used as a predictive factor of no response to romiplostim in ITP patients; In addition, none of the demographic features, initial clinical-laboratory features, previous lines of treatment with splenectomy or rituximab, or even the number of previous lines of therapy have influenced response to TPO-RA. (c) 2023 The Egyptian Journal of Haematology