Real-world results of ibrutinib in patients with relapsed or refractory chronic lymphocytic leukemia: Data from 95 consecutive patients treated in a compassionate use program. A study from the swedish chronic lymphocytic leukemia group

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Ibrutinib, a Bruton’s tyrosine kinase inhibitor is approved for relapsed/refractory and del(17p)/TP53 mutated chronic lymphocytic leukemia. Discrepancies between clinical trials and routine healthcare are commonly observed in oncology. Herein we report real-world results for 95 poor prognosis Swedish patients treated with ibrutinib in a compassionate use program. Ninety-five consecutive patients (93 chronic lymphocytic leukemia, 2 small lymphocytic leukemia) were included in the study between May 2014 and May 2015. The median age was 69 years. 63% had del(17p)/TP53 mutation, 65% had Rai stage III/IV, 28% had lymphadenopathy ≥10cm. Patients received ibrutinib 420 mg once daily until progression. At a median follow-up of 10. 2 months, the overall response rate was 84% (consistent among subgroups) and 77% remained progression-free. Progression-free survival and overall survival were significantly shorter in patients with del(17p)/TP53 mutation (P=0. 017 and P=0. 027, log-rank test); no other factor was significant in Cox proportional regression hazards model. Ibrutinib was well tolerated. Hematomas occurred in 46% of patients without any major bleeding. Seven patients had Richter's transformation. This real-world analysis on consecutive chronic lymphocytic leukemia patients from a well-defined geographical region shows the efficacy and safety of ibrutinib to be similar to that of pivotal trials. Yet, del(17p)/TP53 mutation remains a therapeutic challenge. Since not more than half of our patients would have qualified for the pivotal ibrutinib trial (RESONATE), our study emphasizes that real-world results should be carefully considered in future with regards to new agents and new indications in chronic lymphocytic leukemia.


  • Maria Winqvist
  • Anna Asklid
  • P. O. Andersson
  • Karin Karlsson
  • Claes Karlsson
  • Birgitta Lauri
  • Jeanette Lundin
  • Mattias Mattsson
  • Stefan Norin
  • Anna Sandstedt
  • Lotta Hansson
  • Anders Österborg
External organisations
  • Karolinska University Hospital
  • Södersjukhuset
  • Skåne University Hospital
  • Sunderby Hospital
  • Uppsala University Hospital
  • Linköping University Hospital
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology


  • refractory chronic lymphocytic leukemia
Original languageEnglish
Pages (from-to)1573-1580
Number of pages8
Issue number12
Publication statusPublished - 2016
Publication categoryResearch