Table 2 Efficacy and safety of different treatments for ITP
From: How we treat primary immune thrombocytopenia in adults
Medications | Dosage | Time to response | Overall response | Response durability | Side effects |
|---|---|---|---|---|---|
Initial treatment | |||||
Prednisone | 1 mg/kg/day for 1–2 weeks, then gradually tapper and stop by 6–8 weeks, rapid tapering in nonresponders | 1–2 weeks | 60–80% | 30–50% of patients maintain the response after discontinuation | Weight gain, Cushingoid appearance, mood disorders, gastrointestinal toxicities, hyperglycemia, insomnia, hypertension, increased risks of infection, neuropsychiatric symptoms |
HD-DXM | 40 mg/day × 4 days, up to 2–4 cycles | 1–9 days | 60–80% | 30–50% of patients maintain the response after discontinuation | Weight gain, gastrointestinal toxicities, hyperglycaemia, insomnia, hypertension, increased risk of infection, neuropsychiatric symptoms |
IVIg | 0.4 g/kg/day × 5 days or 1 g/kg/day × 1–2 days | 1–4 days | Up to 80% | Transient | Headache, renal insufficiency, aseptic meningitis, anaphylactic reactions in IgA deficient patient |
Anti-RhD | 50–75 μg/kg | 4–5 days | Up to 80% | Transient | Hemolysis, fever, chills, renal failure |
Subsequent treatment with robust evidence | |||||
Eltrombopag | 25–75 mg/day | 2–4 weeks | 60–80% | 40–60% with maintenance therapy, 10–30% keep the response after drug discontinuation | Headache, upper respiratory tract infection, diarrhea, hepatotoxicity, thromboembolic events, bone marrow fibrosis |
Avatrombopag | 5–40 mg/day | 1–2 weeks | 65% by day 8, 85% by day 28 | 70–80% with maintenance therapy | Headache, arthralgia, increased risk of thrombosis and bone marrow fibrosis |
Hetrombopag | 2.5–7.5 mg/day | 15–25 days | 58.9–64.3% | 40–50% with maintenance therapy | Headache, upper respiratory tract infection, diarrhea, hepatotoxicity, increased risk of thrombosis and bone marrow fibrosis |
Romiplostim | 1–10 μg/kg once weekly | 1–2 weeks | 74–88% | 40–60% with maintenance therapy | Headache, muscle aches, thrombosis, bone marrow fibrosis or increased reticulin |
rhTPO | 300 U/kg/day | 1–2 weeks | 60–75% | – | Drowsiness, dizziness, hypertension, fatigue, rash, urticaria, diarrhea |
Rituximab | 375 mg/m2 weekly × 4 weeks, 100 mg weekly × 4 weeks, 1000 mg on day 1 and day 15 | 1–8 weeks | 60–80% | 40–50% | Infusion-related reactions, hypogammaglobulinemia, increased risk of infections and HBV reactivation, progressive multifocal leukoencephalopathy |
Fostamatinib | 50–150 mg twice daily | 1–2 weeks | 40–50% | 18–43% with maintenance therapy | Diarrhea, hypertension, nausea, dizziness, transaminitis |
Subsequent treatment with less robust evidence | |||||
Azathioprine | 1–2 mg/kg/day | 6–12 weeks | 30–60% | Up to a quarter of patients off therapy keep the response after long treatment duration | Fatigue, transaminitis, neutropenia, increased risk of malignancy |
Cyclosporin A | 2.5–3 mg/kg/day, titration to blood levels of 100–200 ng/ml | 3–4 weeks | 50–80% | 50% keep the response on low-dose maintenance | Hypertension, renal dysfunction, hypertrichosis, gingival hyperplasia, tremor |
Cyclophosphamide | 1–2 mg/kg/day orally for at least 16 weeks or IV 0.3–1 g/m2 1–3 doses every 2–4 weeks | 1–16 weeks | 24–85% | Approximate 50% | Nausea, vomiting, hematuria, neutropenia |
Danazol | 200 mg, 2–4 times daily | 3–6 months | 30–60% | – | Acne, hirsutism, hypercholesterolemia, amenorrhea, transaminitis |
Dapsone | 75–100 mg/day | 3–4 weeks | 30–60% | – | Nausea, rash, dyspepsia methemoglobinemia, hemolytic anemia in glucose-6-phosphate dehydrogenase |
MMF | 1.5–2 g/day | 4–8 weeks | 30–60% | 40% | Headache, gastrointestinal symptoms, increased risks of infection and cancer, |
Vinca alkaloids | Vincristine: 1–2 mg weekly for 2–3 weeks Vinblastine: 10 mg weekly for 2–3 weeks | 5–7 days | Transient. response in 10–75% of patients | – | Neuropathy, neutropenia, constipation, hepatotoxicity |
Novel agents under investigation | |||||
Rilzabrutinib | 400 mg twice daily | 1–10 weeks (median 11.5 days) | 40% | – | Diarrhea, nausea, fatigue, infection |
Rozanolixizumab | Starting at 4 mg/kg weekly, can be uptitrated to a maximum of 20 mg/kg weekly | 1–2 weeks | 35–45% | – | Headache, diarrhea, vomiting, pyrexia, infection |
Efgartigimod | 5 mg/kg or 10 mg/kg weekly × 4 weeks | 8–43 days | 38.5% | – | Rash, hypertension, vomiting, cystitis |
Decitabine | 3.5 mg/m2/day × 3 days for 3 cycles with a 4-week interval between cycles | 2–10 weeks | 51% | SR rate at 6 months was 44% | Nausea, fever, diarrhea, constipation, transaminitis |
Surgical treatment | |||||
Splenectomy | Open or laparoscopic surgery | immediately | 75–90% | 50–70% | Bleeding, infection, thrombosis, increased risk of cancer |