What are the risks of treating chronic, refractory immune thrombocytopenic purpura (ITP)?

Updated: Jun 28, 2019
  • Author: Craig M Kessler, MD, MACP; Chief Editor: Srikanth Nagalla, MBBS, MS, FACP  more...
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Answer

The treatment of chronic, refractory ITP may introduce risks of toxicity from medications that are comparable in severity to the risks of untreated thrombocytopenia. These treatments also may impact adversely on the patient's quality of life. [88]

Fostamatinib was approved by the FDA in April 2018 for thrombocytopenia in adults with chronic ITP who have had an insufficient response to a previous treatment. It is the first spleen tyrosine kinase (SYK) inhibitor approved in the U.S. Approval was based on the FIT clinical program (n=163), which included 2 randomized placebo-controlled Phase 3 trials and an open-label extension trial. [51]

For patients with chronic refractory ITP who have access to investigational programs, the authors encourage them to participate in controlled clinical trials to support the development of effective treatments for this category.


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