Table 1.
Romiplostim in the treatment of children with chronic immune thrombocytopenia
| Study | Design | N | Median age (years) | Duration of ITP prior to romiplostim (median years) | Treatments prior to romiplostim |
Dose (median µg/kg) | Duration treatment/time to response | Outcome | Treatment-related SAE |
|---|---|---|---|---|---|---|---|---|---|
| Tarantino et al42 | Randomized | 42 | 10 | 1.9 | 38% received ≥3 treatments |
3.9 | 24 weeks/4.5 weeks | 22/42 (52%) with platelets ≥50 k/µL for 6 weeks with no rescue | 1 SAE (headache and |
| Bussel et al40 | Randomized | 17 | 9 | 2.4 | 88% received ≥3 treatments |
5 | 12 weeks/7 weeks | 15/17 (88%) with platelets >50 k/µL for 2 weeks and increase 20 k/µL above baseline for 2 weeks | None |
| Elalfy et al39 | Randomized | 12 | 8.5 | 2.3 | 44% received ≥3 treatments |
5 | 12 weeks/NR | 10/12 (83%) with platelets >50 k/µL | None |
| Bussel et al41 | 2nd extension | 12 | 10 | 2.4 | NR | 5.4 | 167 weeks/NR | After 1st 12 weeks – median platelet counts were >50 k/µL | None |
| Pasquet et a35 | Retrospective, observational | 10 | NR | 1.9 | 90% received ≥3 treatments |
4–10 (at the time of clinical/lab response) | 9 months/NR | 1/10 (10%) with platelets >100 k/µL, 4/10 (40%) with platelet 30–100 k/µL and 2× baseline without bleeding | None |
| Mokhtar et al33 | Retrospective, observational | 7 | 5.5 | 2.4 | 100% received ≥3 treatments |
5.7 | Median 12 weeks/7 weeks | 1/7 (14%) with platelets >50 k/µL for 6 weeks, 4/7 (57%) with 2× baseline platelet count | None |
| Marquínez-Alonso et al32 | Retrospective, observational | 4 | 10.3 | 2.7 | 75% received ≥3 treatments |
4 | Median 50 weeks/NR | 3/4 (75%) with platelets >50 k/µL for 6 or more of last 8 weeks | None |
| Escudero Vilaplana et al31 | Retrospective, observational | 3 | 9 | 2.8 | 100% received ≥3 treatments |
3.3 | Mean 33 weeks/NR | 1/3 (33%) with platelets >100 k/µL | None |
| Ramaswamy et al36 | Retrospective, observational | 21 | 11.4 | NR | Mean number of 3.6 treatments | Duration: 6–44 months – 11/18 ongoing | 18/21 (85%) with platelets >50 k/µL and >20 k/µL above baseline for 2 weeks | None | |
| Seidel et al34 | Single-center cohort | 7 | 11 | NR | 100% received ≥3 treatments |
5–10 | Median 14 months/NR | 6/7 (85%) with platelets >50 k/µL | None |
| Neunert et al38 | Retrospective, observational | 43 | Mean 10.9 | Mean 1.7 | 42% received steroids at start of therapy | 5 | 6.4 weeks (response time) | 71% with platelets ≥20 k/µL above baseline ×2 consecutive weeks, 84% with platelets ≥50 k/µL without rescue therapy in 7 days | 1 neutralizing |
Abbreviations: ITP, immune thrombocytopenia; NR, not recorded; SAE, serious adverse event.