Table 2.
Published studies of eltrombopag in paediatric patients with ITP
| Paper* | Type of study | Patient population | Results |
| Giordano et al38 | Retrospective multicentre study | 386 children with chronic ITP enrolled retrospectively at 17 sites | Prevalence of eltrombopag use was 19% in patients with chronic ITP |
| Koca Yozgat et al9 | Retrospective multicentre study | 105 children with chronic or acute refractory ITP treated with epag | Overall response rate was 74%. 27.6% developed iron deficiency or iron deficiency anaemia. |
| Cheng et al39 | Single-centre observational study | 20 patients with severe chronic ITP treated with epag | The durable response rate was 70% (14/20) |
| Grace et al40 | Multicentre prospective observational study | 120 children with ITP starting second-line therapies. 20 patients treated with epag. | Increased platelet counts and HRQoL. Decrease in skin, but not non-skin, bleeding symptoms in patients on epag. |
| Suntsova et al41 | Single-centre retrospective analysis | 23 patients with chronic ITP who failed first TPO-RA. 10 patients switched to epag. | Response rates after switching TPO-RAs were 80% (romi→ epag) and 62% (epag → romi) |
| Tumaini Massaro et al42 | Meta-analysis | Five randomised controlled trials with total of 261 paediatric patients. 159 treated with epag. | TPO-RAs superior to placebo |
| Grainger et al43 | Multicentre RCT | 82 patients with ITP >6 months who had received at least one prior treatment | Epag did not impact HRQoL as assessed by KIT |
| Grace et al44 | Multicentre prospective observational study | 120 children with ITP starting second-line therapies. 20 patients treated with eltrombopag. | Oral agents, including eltrombopag, were chosen for ease of administration and expected adherence (p<0.001) |
| Leblebisatan et al45 | Single-arm study | 19 patients with chronic ITP | 58% of patients responded with either increased platelet counts or decreased bleeding |
| Zhang et al46 | Indirect-comparison meta-analysis | Five randomised controlled trials with total of 261 paediatric patients. 159 treated with epag. | Epag and romi similar in efficacy and safety, but decreased bleeding w/ epag. |
| Guo et al47 | Meta-analysis | Seven randomised controlled trials with total of 345 paediatric patients. 159 treated with epag. | TPO-RAs superior to placebo |
| Zhang et al48 | Systematic review | Five randomised controlled trials with total of 261 paediatric patients. 159 treated with epag. | Overall response and durable platelet response increased in TPO-RAs versus placebo |
| Lambert et al27 | Retrospective chart review | 12 patients with ITP treated with eltrombopag | 8/11 patients developed iron deficiency during treatment with epag |
| Neunert et al10 | Multicentre retrospective study | 79 patients with ITP treated with TPO-RAs | 89% achieved platelet count >50×1 (no difference between epag or romi); 40% achieved stable response |
| Grainger et al7 | Multicentre RCT | 92 patients with chronic ITP and platelets <30 k | 40% (vs 3% placebo) achieved platelet count >50 for 6/8 weeks |
| Bussel et al6 | Multicentre RCT | 82 patients with ITP >6 months who had received at least one prior treatment | 62% (vs 32% placebo) achieved platelet count >50×1 |
| Ramaswamy et al49 | Multicentre retrospective study | 33 paediatric patients with ITP who had received at least one prior treatment; 12 received eltrombopag | 75% achieved platelet counts ≥50 k and ≥20 k above baseline for two consecutive weeks |
*Search was performed in PubMed using terms ‘eltrombopag’ and either ‘paediatric’ or ‘children’. Clinical trials and meta-analyses were included. Papers were excluded if the patient population was anything other than paediatric patients with ITP, or if they included fewer than 10 patients treated with eltrombopag.
HRQoL, health-related quality of life; ITP, immune thrombocytopaenia; KIT, Kids ITP Tool; RCT, randomised controlled trial; TPO-RA, thrombopoietin receptor agonist.