Therapy-resistant Idiopathic Thrombocytopenic Purpura

Idiopathic thrombocytopenic purpura (ITP) is a bleeding disorder in which the blood does not clot properly. The bleeding is due to a low number of platelets, which helps the blood clot and prevents bleeding. ITP is also accompanied by bruises that appear on the skin due to bleeding that has occurred in small blood vessels under the skin. Therapy-resistant idiopathic thrombocytopenic purpura does not respond to conventional treatment.

Commonly Prescribed (On-Label) Drugs: Cortisone, Immune Globulin, Methylprednisolone, Prednisolone, Prednisone, Triamcinolone

Off-Label Prescription Drugs Breakthrough Options: Azathioprine, Cyclophosphamide, Cyclosporine, Danazol, Dexamethasone, Mycophenolate Mofetil, Rituximab, Vincristine

Therapy-resistant Thrombotic Thrombocytopenic Purpura

Patients with Thrombotic Thrombocytopenic Purpura (TTP) have low numbers of platelets (thrombocytopenia), abnormal red cells, and a tendency to develop tiny clots in small blood vessels. TTP is caused by an immune reaction when your body makes an antibody that inactivates an important protein (ADAMTS13) whose function is to prevent platelets from sticking together and plugging blood vessels. You may have easy bruising, rashes with many little dots or large purple patches (purpura), fever, belly pain, small clots in the brain or kidneys, or bleeding due to few platelets.

Commonly Prescribed (On-Label) Drugs: None

Off-Label Prescription Drugs Breakthrough Options: Azathioprine, Cyclophosphamide, Rituximab, Vincristine

Thrombotic Thrombocytopenic Purpura

Patients with thrombotic thrombocytopenic purpura (TTP) have low numbers of platelets (thrombocytopenia), abnormal red cells and a tendency to develop tiny clots (thrombi) in small blood vessels. TTP is caused by an immune reaction when your body makes an antibody that inactivates an important protein (ADAMTS13) whose function is to prevent platelets from sticking together and plugging blood vessels. TTP may be accompanied by easy bruising, rashes with many little dots or large purple patches (purpura), fever, belly pain, thrombi in the brain or kidneys, or bleeding due to few platelets.

Commonly Prescribed (On-Label) Drugs: None

Off-Label Prescription Drugs Breakthrough Options: Prednisone, Vincristine

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