Treatment outcome in a population-based, ‘real-world’ cohort of patients with chronic myeloid leukemia

Abstract

Evaluations of the ‘real-world’ efficacy and safety of tyrosine kinase inhibitors in patients with chronic myeloid leukemia are scarce. A nationwide, population-based, chronic myeloid leukemia registry was analyzed to evaluate (deep) response rates to first and subsequent treatment lines and eligibility for a treatment cessation attempt in adults diagnosed between January 2008 and April 2013 in the Netherlands. The registry covered 457 patients; 434 in chronic phase (95%) and 15 (3%) in advanced disease phase. Seventy-five percent of the patients in chronic phase were treated with imatinib and 25% with a second-generation tyrosine kinase inhibitor. At 3 years 44% of patients had discontinued their first-line treatment, mainly due to intolerance (21%) or treatment failure (19%). At 18 months 73% of patients had achieved a complete cytogenetic response and 63% a major molecular response. Deep molecular responses (MR4.0 and MR4.5) were achieved in 69% and 56% of patients, respectively, at 48 months. All response milestones were achieved faster in patients treated upfront with a second-generation tyrosine kinase inhibitor, but ultimately patients initially treated with imatinib also reached similar levels of responses. The 6-year cumulative incidence of eligibility for a tyrosine kinase cessation attempt, according to EURO-SKI criteria, was 31%. Our findings show that in a ‘real-world’ setting the long-term outcome of patients treated with tyrosine kinase inhibitors is excellent and the conditions for an attempt to stop tyrosine kinase inhibitor therapy are met by a third of the patients.

<p>Validation of the EUTOS Long-Term Survival Score in Chinese Chronic Myeloid Leukemia Patients Treated with Imatinib: A Multicenter Real-World Study</p>

“…The whole data compared favorably with the results in prior reports. 11,14,[21][22][23][24][25] The cumulative 5-year CML-related death rate reported in our research was 11% in the ELTS high-risk group, 5% in the ELTS intermediate-risk group, and 2% in the ELTS lowrisk group. The data demonstrated there was higher incidence compared with the results reported in the confirmatory study by Geelen et al 18 Although the incidence was not exactly consistent, the ELTS score still distinguished the high-risk group with a significantly higher rate of disease-related death than the other risk groups.…”

The potential health gain and cost savings of improving adherence in chronic myeloid leukemia

“…Hence, we performed sensitivity analyses with results of the TIDEL-I study instead of the IRIS trial, resulting in a lower potential value of improving physician adherence and a potential underestimation. Additionally, at the time of developing the model preliminary results showed a rate of suboptimal monitoring 2% higher than the final published study [22], which could lead to an overestimation of the potential. Third, we assumed the possibility of discontinuing TKIs in patients in CMR.…”

“…The current assessment included patients in the Netherlands diagnosed in CML-CP. This comprises approximately 95% of the newly diagnosed patients with CML, resulting in around 160 new patients per year in the Netherlands [22].…”

Omitting cytogenetic assessment from routine treatment response monitoring in chronic myeloid leukemia is safe

“…Data were obtained from two complementary population‐based registries (PHAROS‐CML and HemoBase) for newly diagnosed CML patients in the Netherlands in the period 2008‐2013. Details on data sources and definitions have been previously published . We included all patients aged ≥18 years presenting with CML in chronic phase who received first‐line TKI treatment and analyzed matched data for simultaneous cytogenetic and molecular response assessment (not more than 2 weeks apart) at 3, 6, and 12 months after treatment initiation (with a margin of 6 weeks).…”

“…Details on data sources and definitions have been previously published. 11 We included all patients aged ≥18 years presenting with CML in chronic phase who received first-line TKI treatment and analyzed matched data for simultaneous cytogenetic and molecular response assessment (not more than 2 weeks apart) at 3, 6, and 12 months after treatment initiation (with a margin of 6 weeks). The cytogenetic assessment had to include at least twenty metaphase reviews.…”