Table 1.
Overview of the studies on thrombopoietic growth factors
| Thrombopoietic growth factor/phase study | Study population | Efficacy | Serious adverse events |
|---|---|---|---|
| ELT, TPO non-peptide mimetic | |||
| II [63] | 74 pts, HCV-induced cirrhosis, platelet count 20–70 × 109/L | 71–91 % started PegIFN-based treatment; 36–65 % finished 12 weeks of treatment | Ascites, retinal exudates, myositis |
| III (ELEVATE [64]) | 292 pts, chronic liver disease, platelet count <50 × 109/L, before elective invasive procedures | Platelet transfusion was avoided in 72 % (ELT) vs. 19 % (PL) | Early termination of the study due to six thrombotic events in ELT group vs. one event in the PL group |
| III (ENABLE-1 [65]) | 715 pts, HCV-induced cirrhosis, platelet count <75 × 109/L | 95 % initiated PegIFN-based treatment; SVR 23 % (ELT) vs. 14 % (PL) | 10 % (ELT group) developed hepatic decompensation vs. 5 % (PL group); thromboembolic events 3 % (ELT) vs. 1 % (PL) |
| III (ENABLE-2 [65]) | 805 pts, HCV-induced cirrhosis, platelet count <75 × 109/L | 94 % initiated PegIFN-based treatment; SVR 19 % (ELT) vs. 13 % (PL) | |
| Awaited: single-arm pilot (SQUELCH-C) | Pts with HCV-induced cirrhosis, platelet count <75 × 10 9 /L | ELT in combination with PegIFN, RBV, and boceprevir | |
| Avatrombopag, TPO non-peptide mimetic | |||
| II [68] | 130 pts, chronic liver disease, platelet count 10–58 × 109/L, before elective invasive procedures | Cohort A: 49 vs. 6 % had at least a platelet count >50 × 109/L and increase of 20 × 109/L from baseline; cohort B 48 vs. 10 % | Only one pt had a portal vein thrombosis |
| Awaited: two phase III trials | Pts with chronic liver disease before elective invasive procedures | ||
| Awaited: phase II trial | Initiating therapy in HCV-induced cirrhosis | ||
| Romiplostim, TPO peptide mimetic | |||
| Single-arm open-label [69] | 35 Egyptian pts with liver cirrhosis (CTP-C) and platelet count ≤50 × 109/L before elective procedures | 94 % reached a platelet count ≥70 × 109/L within 4 weeks | None reported |
| Awaited: phase II trial | Initiating therapy in HCV-induced cirrhosis | Suspended participant recruitment | |
Studies presented in italics are still ongoing or data are awaited
CTP Child-Turcotte-Pugh, ELT eltrombopag, HCV hepatitis C virus, PegIFN pegylated interferon alpha, PL placebo, pt(s) patient(s), SVR sustained virological response, TPO thrombopoietin