. Author manuscript; available in PMC: 2014 Jun 1.

Published in final edited form as: Hematol Oncol Clin North Am. 2013 Jun;27(3):495–520. doi: 10.1016/j.hoc.2013.03.001

Table 5.

Treatment options for refractory ITP^*

Medication	Approximate response rate	Major toxicities
Azathioprine 1-2 mg/kg (maximum 150 mg/day)	Up to two thirds, 40% in anecdotal reports	Infrequent and mild: weakness, sweating, transaminitis, severe neutropenia, pancreatitis

Cyclosporine A 5 mg/kg/day for 6 days then 2.5 -3.0 mg/kg/d (titrate to blood levels of 100-200 ng/ml)	Dose-dependent, up to 50-80% in small series	Moderate but transient in most patients: increased creatinine, hypertension, fatigue, paresthesias, myalgia, gingival hyperplasia, dyspepsia, hypertrichosis, tremor

Danazol 200 mg 2-4 times daily	67% CR or PR, 40% in anecdotal reports	Frequent: transaminitis, acne, hirsutism, increased cholesterol, amenorrhea

Cyclophosphamide 1-2 mg/kg orally daily for at least 16 wk, or IV 0.3-1 gm/m² for 1-3 doses every 2-4 wk	24-85%	Usually mild to moderate: neutropenia, DVT, nausea

Dapsone 75-100 mg	Up to 50%	Infrequent and reversible: anorexia, nausea, abdominal distention, methemoglobinuria, hemolytic anemia in G6PD deficiency
Dapsone 75-100 mg	Up to 50%	Severe: skin rash, requires discontinuation

Vinca alkaloids: Vincristine 1-2 mg weekly-total 6 mg	Highly variable, 10-75%	Neuropathy, particularly in elderly, Neutropenia, fever
Vinblastine: 10 mg weekly, total 30 mg	Highly variable, 10-75%	Thrombophlebitis at infusion site

adapted from Provan D, Stasi R, Newland AC et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 2010;115:168.