New Treatments for ITP

Eltrombopag

• Eltrombopag is one new drug showing promise in the treatment of chronic ITP. The drug, approved by the U.S. Food and Drug Administration, is classified as a thrombopoietin-receptor agonist, which means it is designed to boost production of blood platelets. Since ITP is a result of the destruction of platelets and the reduction of platelet production, researchers theorized that eltrombopag would be an effective measure against the disorder.
  
  According to clinical trial results published by the New England Journal of Medicine in 2007, the hypothesis was correct. Testing of 118 patients with blood platelet counts of less than 30,000 per cubic mm were given eltrombopag either in 30, 50 or 75 mg doses each day. The goal was to reach a platelet count of 50,000 or more within 43 days. The higher the dosage, the better the results. The goal was achieved by 28 percent receiving 30 mg, 70 percent receiving 50 mg and 81 percent receiving 75 mg. A placebo group reached the goal in 11 percent of patients, according to the study.
  
  

Stem Cell Transplant

• Stem cell transplants to treat extreme cases of ITP may be considered if all other attempted therapies have failed, and ITP is associated with nosebleeds or bleeding in the stomach or bowel, according to the Scripps Research Institute.
  
  Blood stem cells are removed from the patient, frozen and dosed with very high amounts of chemotherapy to remove the cells that are destroying the platelets. The cells are then thawed and returned to the patient. This allows easier recovery from the effects of chemotherapy on bone marrow. Mortality from complications associated with this stem cell transplant is less than 5 percent.
  
  Results based on the first 14 transplant recipients at the National Institutes of Health showed six patients had platelet counts of more than 100,000; these patients were able to cease medication. Two others showed significant improvements and were either able to discontinue medication or take a lower dose as a result. The other six patients showed only mild improvement.
  
  Because of the experimental nature of the treatment, many insurance policies will not cover the costs. According to Scripps Research Institute, the cost can be as high as $150,000. In some cases the treatment can be done at the National Institutes of Health for no cost to the patient.
  If you believe your case to be severe enough to qualify for for this experimental treatment and your doctor agrees that the treatment is a sensible alternative for you, have the doctor contact the National Institutes of Health to discuss the possibility of taking part in the trials.