Molecular response and quality of life in chronic myeloid leukemia patients treated with intermittent TKIs: First interim analysis of OPTkIMA study

M. Malagola, A. Iurlo, E. Abruzzese, M. Bonifacio, F. Stagno, G. Binotto, M. D’Adda, M. Lunghi, M. Crugnola, M.L. Ferrari, F. Lunghi, F. Castagnetti, G. Rosti, R.M. Lemoli, R. Sancetta, M.R. Coppi, M.T. Corsetti, G. Rege Cambrin, A. Romano, A. De VivoA. Russo Rossi, S. Russo, L. Aprile, M. Bocchia, L. Gandolfi, M. Farina, S. Bernardi, N. Polverelli, A.M. Roccaro, M. Baccarani, D. Russo

Research output: Contribution to journalArticlepeer-review


Background: Intermittent treatment with TKIs is an option for the great majority (70%–80%) of CML patients who do not achieve a stable deep molecular response and are not eligible for treatment discontinuation. For these patients, the only alternative is to assume TKI continuously, lifelong. Methods: The Italian phase III multicentric randomized OPTkIMA study started in 2015, with the aim to evaluate if a progressive de-escalation of TKIs (imatinib, nilotinib, and dasatinib) is able to maintain the molecular response (MR3.0) and to improve Health Related Quality of Life (HRQoL). Results: Up to December 2018, 166/185 (90%) elderly CML patients in stable MR3.0/MR4.0 completed the first year of any TKI intermittent schedule 1 month ON and 1 month OFF. The first year probability of maintaining the MR3.0 was 81% and 23.5% of the patients who lost the molecular response regained the MR3.0 after resuming TKI continuously. Patients’ HRQoL at baseline was better than that of matched peers from healthy population. Women was the only factor independently associated with worse baseline HRQoL (p > 0.0001). Overall, global HRQoL worsened at 6 (p < 0.001) but returned to the baseline value at 12 months and it was statistically significantly worse in women (p = 0.001). Conclusions: De-escalation of any TKI by 1 month ON/OFF schedule maintains the MR3.0/MR4.0 in 81% of the patients during the first 12–24 months. No patients progressed to accelerated/blastic phase, all the patients (23.5%) losing MR3.0 regained the MR3.0 and none suffered from TKI withdrawn syndrome. The study firstly report on HRQoL in elderly CML patients moving from a continuous daily therapy to a de-escalated intermittent treatment.

Original languageEnglish
Pages (from-to)1726-1737
Number of pages12
JournalCancer Medicine
Issue number5
Publication statusPublished - 2021


  • chronic myeloid leukaemia
  • intermittent
  • quality of life
  • tyrosine kinase inhibitor
  • dasatinib
  • imatinib
  • nilotinib
  • 4-methyl-N-(3-(4-methylimidazol-1-yl)-5-(trifluoromethyl)phenyl)-3-((4-pyridin-3-ylpyrimidin-2-yl)amino)benzamide
  • protein kinase inhibitor
  • pyrimidine derivative
  • adult
  • aged
  • appendicitis
  • artery disease
  • arthritis
  • Article
  • ascites
  • atrial fibrillation
  • blast cell
  • bronchitis
  • cancer growth
  • cancer patient
  • chill
  • chronic myeloid leukemia
  • conjunctival hemorrhage
  • controlled study
  • data analysis
  • diarrhea
  • drowsiness
  • drug intermittent therapy
  • drug response
  • dyspnea
  • female
  • fever
  • gender
  • geriatric patient
  • hand pain
  • heart failure
  • hip fracture
  • human
  • hypertension
  • hypotension
  • influenza
  • inguinal hernia
  • interim analysis
  • Italy
  • leg edema
  • longitudinal study
  • maintenance therapy
  • major clinical study
  • male
  • molecular response
  • muscle cramp
  • patient monitoring
  • peer group
  • perianal abscess
  • periorbital edema
  • phase 3 clinical trial
  • pneumonia
  • postvitreous detachment
  • probability
  • pruritus
  • randomized controlled trial
  • reverse transcription polymerase chain reaction
  • side effect
  • clinical trial
  • disease exacerbation
  • drug administration
  • middle aged
  • preliminary data
  • sex factor
  • time factor
  • very elderly
  • Aged
  • Aged, 80 and over
  • Dasatinib
  • Disease Progression
  • Drug Administration Schedule
  • Female
  • Humans
  • Imatinib Mesylate
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Male
  • Middle Aged
  • Preliminary Data
  • Protein Kinase Inhibitors
  • Pyrimidines
  • Quality of Life
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sex Factors
  • Time Factors


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