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Treatment of Refractory ITP Patients

**Please note! The treatment approach described here is based on the author's experience and biases; other physicians who approach the treatment of refractory ITP differently may not be incorrect. The drug doses listed in some of the sections represent those commonly prescribed. However, drug doses in individual patients may differ from those listed here and depend on the patient's clinical situation and the doctor's preferences. Decisions on the treatment of individual patients with ITP are the sole responsibility of the treating physician.

The treatment of patients who fail to respond to corticosteroids, anti-D and splenectomy (refractory ITP) is often difficult since these patients tend to be resistant to many forms of treatment. The following approach describes three levels of treatment (first, second and third line) and also discusses some forms of therapy that are either of questionable value or are of value but expensive or inconvenient. The treatment levels are associated with progressively serious early or late side effects (i.e., level 1 has the least and level 3 the greatest). Click on the underlined headings for further detail.

First line therapy.
  • Corticosteroids
  • Danazol (Danocrine)
  • Colchicine
  • Dapsone
Second line therapy.
  • Cyclophosphamide (Cytoxan)
  • Azathioprine (Imuran)
  • Mycophenolate mofetil (Cellcept)
  • Cyclosporine
Third line therapy- aggressive chemotherapy.
  • High-dose cyclophosphamide
  • Combination chemotherapy
Fourt Line Therapy- treatments with various limitations. 
  • Ascorbic acid (vitamin C)
  • Gammaglobulin (long-term)
  • Vinblastine (long-term)

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