What is the role of immunosuppressive therapy in the treatment of chronic immune thrombocytopenia (ITP) in adults?

Updated: Nov 30, 2019
  • Author: Perumal Thiagarajan, MD; Chief Editor: Srikanth Nagalla, MBBS, MS, FACP  more...
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Limited benefit may be observed using immunosuppression with cytotoxic agents. Azathioprine (150 mg/d) or cyclophosphamide (50-100 mg/d) has been used with some success. These cytotoxic drugs can cause myelosuppression, alopecia, hemorrhagic cystitis (cyclophosphamide), sterility, and secondary malignancy. They are given for a minimum duration and are withdrawn as soon as remission is achieved. Blood counts must be monitored during therapy.

Vincristine infusion (0.02 mg/kg) with a maximum dose of 2 mg every week for 3 weeks has also been shown to induce remission. Refractory ITP has also been treated with the combination chemotherapy regimen used for low-grade non-Hodgkin lymphoma (6 cycles of cyclophosphamide, vincristine, and prednisone), with some success.

Rituximab, a monoclonal antibody directed against the lymphocyte antigen, has been reported to induce lasting remission in refractory ITP. A meta-analysis of adults suggests that surgery could be delayed and may be avoided altogether for patients who received rituximab earlier in the course of therapy. [30]

Several studies reported improved platelet counts in patients with Helicobacter pylori –positive ITP following standard H pylori eradication therapy, with cohorts from Japan and Italy reporting higher response rates. [31] Several explanations, such as molecular and immunomodulation by macrolides, have been provided. However, a recent small, multicenter, randomized controlled study that evaluated 55 patients aged 4-18 years with chronic ITP found no beneficial effect of H pylori eradication on platelet recovery. [32] Further investigation is needed.

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