Treatment of Refractory ITP Patients
Fourth Line Treatment- Miscellaneous Treatments.
**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 treatments described here fall within two groups: (1) Treatments reported as beneficial in only a few patients where the results have not been confirmed (vitamin C). (2) Treatment with an expensive and/or inconvenient drug which results in only a temporary response (gammaglobulin, vinblastine).
Ascorbic acid (vitamin C). The first report described 9 responses in 11 ITP patients. Unfortunately, many patients have been treated since that time and few responses have occurred. Most physicians feel that this agent is rarely useful in ITP although it is a very safe drug with essentially no side effects. A trial of this agent is probably worthwhile since occasional patients are reported to respond.
Dose. 2 g/day orally.
Side effects. None.
Vinblastine (Velban). Responses to this drug are almost always temporary. However, patients who respond to this agent can be maintained with safe platelet counts for long intervals with periodic injections. This is inconvenient and should only be considered when no other approaches are successful.
Dose. The dose is 5-10 mg into the vein every 1-4 weeks as needed.
Side effects. Hair loss (variable amount), constipation, low white blood count and ulceration of the skin if the drug is not given cleanly into the vein.
Gammaglobulin. This agent is very effective in stimulating an increase in the platelet count (about two-thirds of patients will increase their count to more than 100,000 and about 80% to more than 50,000) but, unfortunately, responses are almost always temporary. The major use of this agent is to increase the platelet count under emergency circumstances (see emergency therapy) or prior to surgery. Occasionally, patients who are unresponsive to all forms of treatment can be maintained by periodic doses of gammaglobulin. This approach is extremely inconvenient for the patient, very expensive and is not recommended unless no other alternatives are available.
Side effects. Side effects are unusual and mild headaches are the most common. Rarely, severe complications can occur and you should discuss these with your physician.
Dose. The dose depends on the patient's weight and usually ranges between 50 to 100 grams given by vein over several hours. Treatment is usually needed at 1-4 week intervals.
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