The phase 3 ENESTPath study investigated treatment-free remission (TFR) rates in patients with chronic Philadelphia chromosome-positive (Ph+) and/or BCR::ABL1+ chronic myeloid leukemia who had not achieved deep molecular response (DMR) after >2 years of imatinib treatment and were switched to nilotinib 300 mg twice daily (BID). After 24 months of treatment, patients with a stable DMR were randomized to either enter the TFR phase (Arm 1) or continue nilotinib consolidation for an additional 12 months and then enter the TFR phase if in stable DMR (Arm 2). The primary endpoint was the proportion of patients who remained in TFR (≥MR4.0 [BCR::ABL1IS ≤ 0.01%]) without molecular relapse at the end of 12 months. Of the 620 patients enrolled, 239 (38.5%) achieved stable MR4.0 and were randomized to Arm 1 (n = 120) or Arm 2 (n = 119). In the TFR phase, MR4.0 rates at 12 months (Arm 1: 31.9%, Arm 2: 37.5%; p = 0.383) and 24 months (Arm 1: 29.4%, Arm 2: 30.8%) revealed no differences in TFR success between 2 and 3 years of nilotinib. Irrespective of the consolidation duration, switching to nilotinib 300 mg BID provided the opportunity to achieve TFR if patients were unable to reach stable DMR with first-line imatinib.

Departement d'Hematologie Institut Paoli Calmettes Marseille France

Department of Clinical and Biological Sciences University of Turin Turin Italy

Department of Clinical Hematology G Gennimatas Hospital Athens Greece

Department of Hematology and BMT Silesian Medical University Hospital SPSKM Katowice Poland

Department of Hematology and Transplantology Medical University of Gdansk Gdansk Poland

Department of Hematology Faculty of Medicine University of Debrecen Debrecen Hungary

Department of Internal Medicine Hematology and Oncology Masaryk University and University Hospital Brno Czechia

Department of Medicine 4 Klinikum Bayreuth GmbH Bayreuth and Comprehensive Center Erlangen EMN Erlangen Germany

Department of Medicine and Surgery Institute of Hematology and Center for Hemato Oncological Research University of Perugia Perugia Italy

France Intergroupe Des Leucémies Myéloïdes Chroniques FiLMC Paris France

Hematology Center Karolinska University Hospital and Karolinska Institutet Stockholm Sweden

Hospital de La Princesa Madrid Spain

Hospital Josep Trueta Institut Català d'Oncologia Girona Spain

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori IRCCS Dino Amadori Meldola Italy

Novartis Pharma AG Basel Switzerland

Service d'Hématologie Adulte Hôpital Saint Louis APHP Paris France

University of Leipzig Leipzig Germany

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