Table 1. Therapies with adequate RCT... | American Society of Hematology

Table 1.

Therapies with adequate RCT evidence

Therapy	Route of administration	Mechanism of action	Dosage	Response rate	Adverse effects
Romiplostim	Subcutaneous injections	TPO-RA	1-10 µg/kg once weekly	Overall response of 75%; durable response at 6 mo is 65%; 10% to 30% may achieve treatment remission	Headache, muscle aches, venous and arterial thromboembolism, possible increase in bone marrow reticulin, and collagen fibrosis
Eltrombopag	Oral (restricted diet)	TPO-RA	25-75 mg once daily		Headache, venous and arterial thromboembolism, elevated liver enzymes, possible increase in bone marrow reticulin, and collagen fibrosis
Avatrombopag	Oral	TPO-RA	20-40 mg once daily	65% at day 8*	Headaches, arthralgia, and venous and arterial thromboembolism
Rituximab	Intravenous administration	Immunosuppressive; anti-CD20	Infusions of 375 mg/m² each week for 4 weeks or 1000 mg every other week, for 2 weeks†	Initial response rate 60%; durable response rate at 6-12 mo is ∼40% and at 5 y is ∼20% to 30%	Infusion-related side effects (chills, upper respiratory discomfort, and bronchospasm), neutropenia, hypogammaglobulinemia, serum sickness, increased risks of infection and progressive multifocal leukoencephalopathy (very rare).
Fostamatinib	Oral	Immunosuppressive; splenic tyrosine kinase inhibitor	100-150 mg twice daily	Overall response 43%‡; stable response 18%§	Hypertension, diarrhea, nausea, and transaminitis

Therapy	Route of administration	Mechanism of action	Dosage	Response rate	Adverse effects
Romiplostim	Subcutaneous injections	TPO-RA	1-10 µg/kg once weekly	Overall response of 75%; durable response at 6 mo is 65%; 10% to 30% may achieve treatment remission	Headache, muscle aches, venous and arterial thromboembolism, possible increase in bone marrow reticulin, and collagen fibrosis
Eltrombopag	Oral (restricted diet)	TPO-RA	25-75 mg once daily		Headache, venous and arterial thromboembolism, elevated liver enzymes, possible increase in bone marrow reticulin, and collagen fibrosis
Avatrombopag	Oral	TPO-RA	20-40 mg once daily	65% at day 8*	Headaches, arthralgia, and venous and arterial thromboembolism
Rituximab	Intravenous administration	Immunosuppressive; anti-CD20	Infusions of 375 mg/m² each week for 4 weeks or 1000 mg every other week, for 2 weeks†	Initial response rate 60%; durable response rate at 6-12 mo is ∼40% and at 5 y is ∼20% to 30%	Infusion-related side effects (chills, upper respiratory discomfort, and bronchospasm), neutropenia, hypogammaglobulinemia, serum sickness, increased risks of infection and progressive multifocal leukoencephalopathy (very rare).
Fostamatinib	Oral	Immunosuppressive; splenic tyrosine kinase inhibitor	100-150 mg twice daily	Overall response 43%‡; stable response 18%§	Hypertension, diarrhea, nausea, and transaminitis

Platelet counts >50 × 10³//μL.

Lower doses down to 100 mg have been used.

One or more platelet counts ≥50 × 10³/μL during weeks 0 to 12 of the study.

Platelet counts ≥50 × 10³/μL at ≥4 of 6 biweekly clinic visits during weeks 14 to 24 of the study.