Thrombopoietin level predicts response to treatment with eltrombopag and romiplostim in immune thrombocytopenia.

Thrombopoietin receptor agonists (TPO-RAs) are used to treat immune thrombocytopenia (ITP), but predicting clinical response to TPO-RAs before initiation is not possible. To determine whether endogenous TPO levels predict treatment response to TPO-RAs we performed a retrospective analysis of ITP patients with known baseline TPO levels who received TPO-RAs. Data was collected for ITP patients with a baseline TPO level treated with eltrombopag or romiplostim. Multiple logistic regression was used to model the probability of 3 classes of treatment response (overall, moderate, and superior) based on TPO level; receiver operating characteristic (ROC) analysis was performed to identify optimal TPO thresholds for response; correlations between TPO level and various response characteristics were analyzed. A total of 67 patients (37 receiving eltrombopag and 46 receiving romiplostim) were included. Logistic regression models demonstrated a significant predictive relation between TPO level and probability of all classes of response; per 10 pg/mL TPO increase, odds ratio for overall response to eltrombopag was 0.524 (95% CI 0.327, 0.837) and romiplostim was 0.905 (95% CI, 0.844, 0.970). TPO level was inversely correlated with all classes of response; for overall response, r = -0.719 (P < .001) for eltrombopag and r = -0.584 (P < .001) for romiplostim. ROC analysis identified TPO thresholds of ≤136 pg/mL (eltrombopag) and ≤209 pg/mL (romiplostim) as optimally discriminating between responders and nonresponders. Most non-responders had high TPO levels but did respond after addition of low-dose prednisone. In conclusion, TPO levels predict response to eltrombopag and romiplostim in ITP patients, with lower levels predicting improved probability and magnitude of response.