Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia Academic Article uri icon


MeSH Major

  • Antibodies, Monoclonal, Murine-Derived
  • Purpura, Thrombocytopenic, Idiopathic


  • Treatments for immune thrombocytopenic purpura (ITP) providing durable platelet responses without continued dosing are limited. Whereas complete responses (CRs) to B-cell depletion in ITP usually last for 1 year in adults, partial responses (PRs) are less durable. Comparable data do not exist for children and 5-year outcomes are unavailable. Patients with ITP treated with rituximab who achieved CRs and PRs (platelets > 150 × 10(9)/L or 50-150 × 10(9)/L, respectively) were selected to be assessed for duration of their response; 72 adults whose response lasted at least 1 year and 66 children with response of any duration were included. Patients had baseline platelet counts < 30 × 10(9)/L; 95% had ITP of > 6 months in duration. Adults and children each had initial overall response rates of 57% and similar 5-year estimates of persisting response (21% and 26%, respectively). Children did not relapse after 2 years from initial treatment whereas adults did. Initial CR and prolonged B-cell depletion predicted sustained responses whereas prior splenectomy, age, sex, and duration of ITP did not. No novel or substantial long-term clinical toxicity was observed. In summary, 21% to 26% of adults and children with chronic ITP treated with standard-dose rituximab maintained a treatment-free response for at least 5 years without major toxicity. These results can inform clinical decision-making.

publication date

  • June 21, 2012



  • Academic Article



  • eng

PubMed Central ID

  • PMC3383014

Digital Object Identifier (DOI)

  • 10.1182/blood-2011-11-393975

PubMed ID

  • 22566601

Additional Document Info

start page

  • 5989

end page

  • 95


  • 119


  • 25