SEARCH

SEARCH BY CITATION

Keywords:

  • clinical trial;
  • intravenous immunoglobulins;
  • platelets;
  • primary immune thrombocytopenia

Summary

Primary immune thrombocytopenia (ITP) is a common autoimmune disease characterized by autoantibody-induced platelet destruction, suboptimal megakaryocytic production of platelets and consequent bleeding. Numerous controlled trials have proved that the administration of high-dose intravenous immunoglobulins (IVIG) results in rapid increases in the platelet numbers. Therefore, they are indicated in emergency settings or in patients needing surgical procedures. Here, the efficacy and safety of Flebogammadif®, a new high-purity IVIG, was assessed by an open, multi-centre, non-controlled, prospective study in 20 adult patients diagnosed with ITP for at least 6 months before recruitment and with a platelet count ≤ 20 000/µl before treatment. Patients received 0·4 g/kg/body weight of Flebogammadif® for 5 consecutive days and were followed-up for 3 months. There were three efficacy end-points: proportion of patients who reached a platelet count ≥ 50 000/µl; time for the platelet count to reach that level; and duration of response. Safety parameters [adverse events (AE), laboratory determinations and vital signs] and viral markers were monitored regularly. A total of 14 patients achieved a platelet count of ≥ 50 000/µl. The median time to platelet response was ≤ 2·5 days and the median number of days in which the platelet count remained ≥ 50 000/µl was ≥ 7·0 days. A regression of haemorrhages was reported for 17 patients on day 14. Eight patients presented 20 AEs (mainly mild) related potentially to the study drug. Neither abnormalities in laboratory values nor in viral markers were registered during the follow-up period. In conclusion, Flebogammadif® was well tolerated and succeeded in providing a haemostatic platelet count in ITP patients.