Treatment-free remission (TFR) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP) treated with frontline nilotinib: Results from the ENESTFreedom study.

Hochhaus, Andreas; Masszi, Tamás; Giles, Francis J.; Radich, Jerald P.; Ross, David M.; Casares, María Teresa Gómez; Hellmann, Andrzej; Stentoft, Jesper; Conneally, Eibhlin; Gutiérrez, Valentín García; Gattermann, Norbert; Wiktor-Jedrzejczak, W; Coutre, Philipp D. le; Martino, Bruno; Saußele, Susanne; Menssen, Hans D.; Deng, Weiping; Krunic, Nancy; Bédoucha, Véronique; Saglio, Giuseppe

doi:10.1200/jco.2016.34.15_suppl.7001

Cited by 8 publications

(10 citation statements)

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“…22 The ENESTFreedom is from the name of the trial "Nilotinib Treatment-free Remission Study in CML Patients (ENESTFreedom)" also showed that 52% of patients in deep remission remained treatment-free at 48 weeks after the discontinuation of nilotinib. 23 These results suggest that the expected outcome is similar, regardless of the TKI used before discontinuation, although the likelihood of achieving these responses may be higher with a second-generation TKI versus imatinib. We thus evaluated the best fit average and minimum acceptable molecular levels within 1 year of treatment to make predictions for the achievement of a sustained MR 4.5 at any point on the basis of the BCR-ABL1 levels achieved within the first year of TKI therapy.…”

Section: Discussionmentioning

confidence: 99%

Prediction for sustained deep molecular response of BCR‐ABL1 levels in patients with chronic myeloid leukemia in chronic phase

Sasaki

Kantarjian

O’Brien

et al. 2017

Cancer

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Section: Discussionmentioning

confidence: 99%

Prediction for sustained deep molecular response of BCR‐ABL1 levels in patients with chronic myeloid leukemia in chronic phase

Sasaki

Kantarjian

O’Brien

et al. 2017

Cancer

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“…Numerous studies showed the efficacy of nilotinib in the first-line treatment of Philadelphia chromosome-positive chronic myeloid leukemia adult patients [6,19,20]. Besides these numerous advantages, obvious economic benefits can be identified, from the point of view of the National Health Service.…”

Section: Discussionmentioning

confidence: 99%

“…In recent clinical practice it has been shown that, after an adequate period of treatment and in the presence of a very deep and stable molecular response, TKI treatment can be safely discontinued, with good chance of success [7,19]. Numerous studies showed the efficacy of nilotinib in the first-line treatment of Philadelphia chromosome-positive chronic myeloid leukemia adult patients [6,19,20].…”

Section: Discussionmentioning

confidence: 99%

Budget Impact analysis of the first-line treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) adult patients

Mennini¹,

Marcellusi²,

Viti³

et al. 2017

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Background: Tyrosine kinase inhibitors (TKI) have dramatically improved survival in chronic myeloid leukemia in chronic phase (CML‐CP), with a high percentage of patients reaching a major molecular response (MMR). Recently, several clinical trials demonstrated that some patients with CML-CP who achieve a sustained MMR on tyrosine kinase inhibitor (TKI) therapy can safely discontinue their therapy and attempt treatment-free remission (TFR).Objective: The aim of the study was to evaluate the clinical and economic impact of TFR in naïve patients with CML-CP who start treatment with nilotinib, imatinib or dasatinib as first-line therapy, from the perspective of the Italian National Health Service (NHS).Methods: An Excel-based budget impact model was developed, in order to estimate the costs of the patients in first-line pharmacological treatment with CML. A specific Markov model was built, to simulate seven years of treatment with different TKIs. A systematic literature review was carried out, to identify the epidemiological and economic data, which were subsequently used to inform the model. The model considers two scenarios: 1) a Standard of Care (SoC) scenario, with the current estimated distribution of patients over the various TKI treatment, versus 2) an innovative scenario, characterized by an increase in the use of nilotinib (+28%) and generic imatinib (+35%) and a decrease in the use of dasatinib (-17%). A one-way deterministic sensitivity analysis was performed, in order to consider the variability of the results as a function of the main parameters considered in the model.Results: The model estimated that 775 patients with CML-CP could be treated with a TKI as first-line drug. The innovative scenario could increase TFR patients by approximately 60% and reduce the costs by more than € 30 million over 7 years. The increase in the use of nilotinib and the generic imatinib would generate a significant expenditure reduction.Conclusions: This study demonstrates the economic effects of discontinuing TKIs in CML-CP patients. The increase in the use of nilotinib and the generic imatinib could generate an increase in the number of patients who achieve TFR, as well as an actual cost reduction.

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“…Molecular relapse was defined as the loss of MMR on a single occasion. The follow-up is still short and the probability to remain in TFR at 48 weeks was 51.6% (95% CI 44.2-58.9) [31]. Patient follow-up is planned for up to 4 years.…”

Section: Treatment-free Remission Studies: Dasatinib and Nilotinibmentioning

confidence: 99%

Treatment-free remission in patients with chronic myeloid leukemia

Réa

Cayuela

2017

Int J Hematol

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Clinical trials have formally demonstrated that in chronic myeloid leukemia (CML), patients treated with tyrosine kinase inhibitors (TKI) who achieved and maintained deep molecular responses could discontinue their treatment after several years without facing overt signs of disease relapse in approximately 50% of the cases. In patients with a molecular relapse, prompt re-introduction of TKI therapy was able to rapidly restore deep molecular responses. The concept of a lifelong therapy with TKI has thus been challenged and treatment-free remission (TFR) strategies will soon integrate clinical practice, providing that safe recommendations will be established. In this article, we give an update on TKI discontinuation studies in CML and we also provide an overview of upcoming TFR clinical and biological challenges.

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Treatment-free remission (TFR) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP) treated with frontline nilotinib: Results from the ENESTFreedom study.

Cited by 8 publications

References 0 publications

Prediction for sustained deep molecular response of BCR‐ABL1 levels in patients with chronic myeloid leukemia in chronic phase

Prediction for sustained deep molecular response of BCR‐ABL1 levels in patients with chronic myeloid leukemia in chronic phase

Budget Impact analysis of the first-line treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) adult patients

Treatment-free remission in patients with chronic myeloid leukemia

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