Comparison of reported efficacy of second-line treatments with regard to platelet count, bleeding, HRQoL, and fatigue
| . | Platelet count* . | Bleeding . | HRQoL . | Fatigue . |
|---|---|---|---|---|
| Splenectomy | Early response: 66-92%28 | No studies | No improvement in ITP-PAQ16 | No studies |
| Durable response: 60-70%29 | Lower ITP-PAQ scores as compared with those without splenectomy34 | |||
| Rituximab | Platelet counts >50 × 109/L 6 mo postinfusion: 40-60%38 | No difference (9.2% vs 5.2%) compared with non-rituximab treatment or placebo37 | No improvement as measured by change in ITP-PAQ or SF-3616,39 | Possible increase with 22% of patients receiving rituximab reporting fatigue (8% on placebo)39 |
| Durable response: 21-26% 5 y after infusion38 | ||||
| Romiplostim | ||||
| Adult trials | Platelet count ≥50 × 109/L during ≥6 of the last 8 wk of treatment: 38% romiplostim vs 0% placebo11 | No difference compared with non-romiplostim treatment or placebo11 | Improvement in symptoms, activity, psychological health, and overall HRQoL as measured with the ITP-PAQ and EQ-5D7 | No improvement meeting MID measured by ITP-PAQ16 |
| Platelet count ≥50 × 109/L: 71-92% romiplostim vs 0-51% placebo or non-romiplostim treatment46 | ||||
| Long-term response (≥5 y): maintained by all patients for a median of 92% of study visits on stable doses24 | ||||
| Pediatric trials | Platelet count >50 × 109/L for 2 consecutive weeks: 88% romiplostim vs 0% placebo47 | No difference compared with non-romiplostim treatment or placebo11 | Reduced parental burden17 | |
| Platelet counts >50 × 109/L were maintained for a median of 7 wk vs 0 wk for the placebo47 | ||||
| Platelet count >50 × 109/L for ≥6 of 8 wk: 52% romiplostim vs 10% placebo12 | ||||
| Long-term response (≥4 y): continued response on a tapering dose of romiplostim48 | ||||
| Eltrombopag | ||||
| Adult trials | Platelet count >50 × 109/L on day 43: 59% eltrombopag vs 16% placebo18 | Decreased bleeding from baseline shown across trials in adults and children19,20 | Improvement from baseline in physical, emotional, and mental function, as measured by SF-36v2 and FACT-thrombocytopenia18 | Improvement in vitality on SF-36v2 compared with placebo27 |
| Platelet count >50 × 109/L at least once; 79% eltrombopag vs 28% placebo49 | Fatigue was reported as an adverse effect in 15% of patients in the EXTEND trial27 | |||
| Long-term response (≥3 y): 62% platelet count >50 × 109/L in >50% of study assessments27 | ||||
| Pediatric trials | Platelet count >50 × 109/L: 62-75% eltrombopag vs 21-32% placebo19,21 | Decreased bleeding from baseline shown across trials in adults and children19,20 | No improvement in HRQoL by Kids’ ITP Tool scores21 | Improvement in vitality on SF-36v2 compared with placebo27 |
| Fatigue was reported as an adverse effect in 15% of patients in the EXTEND trial27 | ||||
| Dapsone | 40-62%45 | No studies | No studies | No studies |
| Mycophenolate mofetil | 52-69%41 | No studies | No studies | No studies |
| Sirolimus | 25-58%50,51 | No studies | No studies | No studies |
| . | Platelet count* . | Bleeding . | HRQoL . | Fatigue . |
|---|---|---|---|---|
| Splenectomy | Early response: 66-92%28 | No studies | No improvement in ITP-PAQ16 | No studies |
| Durable response: 60-70%29 | Lower ITP-PAQ scores as compared with those without splenectomy34 | |||
| Rituximab | Platelet counts >50 × 109/L 6 mo postinfusion: 40-60%38 | No difference (9.2% vs 5.2%) compared with non-rituximab treatment or placebo37 | No improvement as measured by change in ITP-PAQ or SF-3616,39 | Possible increase with 22% of patients receiving rituximab reporting fatigue (8% on placebo)39 |
| Durable response: 21-26% 5 y after infusion38 | ||||
| Romiplostim | ||||
| Adult trials | Platelet count ≥50 × 109/L during ≥6 of the last 8 wk of treatment: 38% romiplostim vs 0% placebo11 | No difference compared with non-romiplostim treatment or placebo11 | Improvement in symptoms, activity, psychological health, and overall HRQoL as measured with the ITP-PAQ and EQ-5D7 | No improvement meeting MID measured by ITP-PAQ16 |
| Platelet count ≥50 × 109/L: 71-92% romiplostim vs 0-51% placebo or non-romiplostim treatment46 | ||||
| Long-term response (≥5 y): maintained by all patients for a median of 92% of study visits on stable doses24 | ||||
| Pediatric trials | Platelet count >50 × 109/L for 2 consecutive weeks: 88% romiplostim vs 0% placebo47 | No difference compared with non-romiplostim treatment or placebo11 | Reduced parental burden17 | |
| Platelet counts >50 × 109/L were maintained for a median of 7 wk vs 0 wk for the placebo47 | ||||
| Platelet count >50 × 109/L for ≥6 of 8 wk: 52% romiplostim vs 10% placebo12 | ||||
| Long-term response (≥4 y): continued response on a tapering dose of romiplostim48 | ||||
| Eltrombopag | ||||
| Adult trials | Platelet count >50 × 109/L on day 43: 59% eltrombopag vs 16% placebo18 | Decreased bleeding from baseline shown across trials in adults and children19,20 | Improvement from baseline in physical, emotional, and mental function, as measured by SF-36v2 and FACT-thrombocytopenia18 | Improvement in vitality on SF-36v2 compared with placebo27 |
| Platelet count >50 × 109/L at least once; 79% eltrombopag vs 28% placebo49 | Fatigue was reported as an adverse effect in 15% of patients in the EXTEND trial27 | |||
| Long-term response (≥3 y): 62% platelet count >50 × 109/L in >50% of study assessments27 | ||||
| Pediatric trials | Platelet count >50 × 109/L: 62-75% eltrombopag vs 21-32% placebo19,21 | Decreased bleeding from baseline shown across trials in adults and children19,20 | No improvement in HRQoL by Kids’ ITP Tool scores21 | Improvement in vitality on SF-36v2 compared with placebo27 |
| Fatigue was reported as an adverse effect in 15% of patients in the EXTEND trial27 | ||||
| Dapsone | 40-62%45 | No studies | No studies | No studies |
| Mycophenolate mofetil | 52-69%41 | No studies | No studies | No studies |
| Sirolimus | 25-58%50,51 | No studies | No studies | No studies |
Measure of platelet count response by platelet count is not consistent or comparable among studies.