Impact of ELN recommendations in the management of first‐line treated chronic myeloid leukaemia patients: a French cross‐sectional study

Abstract

The availability of tyrosine kinase inhibitors has extended therapeutic options for chronic myeloid leukaemia (CML) patients. Monitoring recommendations and clinical response goals have recently been updated. The objective of this study was to describe the profile of CML patients in chronic phase currently receiving first-line therapy, including treatment, monitoring and response kinetics. A multicentre, cross-sectional, epidemiological survey in unselected chronic phase CML patients in France attending consultations during a one-month period was performed. 438 of 697 (62·8%) reported patients were currently receiving first-line treatment and were analysed. Imatinib was the most frequently received treatment (72·4% of patients). Retrospective cytogenetic and molecular assessments at 3, 6, 12 or 18 months were available in 88·4% of patients. At the 12-month assessment, 32·2% were not in major molecular response (MMR). At last assessment, among 355 patients with duration of treatment ≥ 12 months, 91·5% had achieved MMR and 66·5% were in deep molecular response. This study, performed in everyday practice population of CML patients, suggests that monitoring of molecular responses in real-life practice is aligned with European LeukaemiaNet recommendations. The majority of patients still receiving first-line treatment are in optimal response, with a few being classified as in the warning area or responding to failure.

Nilotinib efficacy, safety, adherence and impact on quality of life in newly diagnosed patients with chronic myeloid leukaemia in chronic phase: a prospective observational study in daily clinical practice

“…Sokal risk scores observed in our study, as well as in the French epidemiological survey (Etienne et al , ), the GIMEMA trial (Castagnetti et al , ), the ENEST1st trial (Hochhaus et al , ) and a phase II Turkish trial (Saydam et al , ), are comparable, with a low Sokal score in 46·4%, 42·3%, 43·0%, 34·6% and 54·5% of patients respectively; an intermediate score in 34·4%, 39·7%, 36·0%, 37·5% and 39·3% respectively; and a high score in 14·2%, 18·1%, 21·0%, 18·1% and 6·3% respectively. However, in the ENESTnd study, patients with high Sokal risk were comparatively more represented (28·0%) (Saglio et al , ).…”

“…The median age of patients in this study was 54 years, which is consistent with a recent CML‐CP French cross‐sectional epidemiological survey in first‐line TKI treated patients in which median age was 61 years with a median time since diagnosis of 4 years. In this survey, imatinib was the most prescribed TKI in current practice (81·1%), and nilotinib the second one (12·4%) with a tendency to be preferentially used in patients aged under 65 years (Etienne et al , ). As anticipated in an observational study reflecting daily clinical practice, median age was higher than the reported 47 years in the ENEST‐Newly Diagnosed patients (ENESTnd) randomized clinical trial that led to the approval of nilotinib for first‐line use (Saglio et al , ).…”

“…Subsequently, cytogenetic assessment was less frequent at 6 and 12 months and concerned respectively 71/165 (43·0%) and 44/151 (29·1%) patients. The 2016 French epidemiological survey in unselected patients with CML‐CP described practices in a real‐life setting and showed that the cytogenetic response was less investigated than the molecular response (Etienne et al , ). The ELN guidelines indicated that molecular test alone may be sufficient to evaluate response to treatment after CCyR and MMR achievement (Baccarani et al , ).…”

“…Dasatinib and nilotinib were both approved in the United States and the European Union in 2011 for newly diagnosed patients with CML‐CP; however, nilotinib is the only second‐generation TKI reimbursed in this setting in France and imatinib remains the most frequently used TKI (Etienne et al , ). First‐line nilotinib therapy offers several advantages compared to imatinib, such as early reduction of molecular residual disease burden, significant reduction of progression rate, and a higher rate of deep molecular responses (DMRs) which represents a criterion for treatment discontinuation (Breccia et al , ; Rea et al , ).…”

First‐line treatment selection and early monitoring patterns in chronic phase‐chronic myeloid leukemia in routine clinical practice: SIMPLICITY

“…An observational study of 705 patients from 36 centers in France reported that MR monitoring in routine clinical practice is closely aligned with the updated recommendations. The authors of the study highlight the majority of patients were still receiving first‐line treatment and were in optimal response . There were no reported differences in monitoring patterns according to insurance status, which suggests that lower rates of monitoring were not the result of issues with insurance.…”