Using healthcare claims data to analyze the prevalence of BCR‐ABL‐positive chronic myeloid leukemia in France: A nationwide population‐based study

Abstract

Background Data on Chronic Myeloid Leukemia (CML) prevalence are scarce. Here we provide an estimation of the prevalence of CML in France for the year 2014 using French national health insurance data. Methods We selected patients claiming reimbursement for tyrosine kinase inhibitors (TKI) or with hospital discharge diagnoses for CML, BCR/ABL‐positive or with full reimbursement of health care expenses for myeloid leukemia. We built an algorithm which we validated on a random sample of 100 potential CML patients by comparing the results obtained using the algorithm and the opinion of two hematologists who reviewed the patient demographics and sequence of care abstracted from claims data (internal validity). For external validity, we compared the number of incident CML patients identified using the algorithm with those recorded in French population‐based cancer registries in departments covered by such a registry. Results We identified 10 789 prevalent CML patients in 2014, corresponding to a crude prevalence rate of 16.3 per 100 000 inhabitants [95% confidence interval (CI) 16.0‐16.6]: 18.5 in men [18.0‐19.0] and 14.2 in women [13.8‐14.6]. The crude CML prevalence was less than 1.6 per 100 000 [1.2‐2.0] under age 20, increasing to a maximum of 48.2 [45.4‐51.2) at ages 75‐79. It varied from 10.2 to 23.8 per 100 000 across French departments. The algorithm showed high internal and external validity. Concordance rate between the algorithm and the hematologists was 96%, and the numbers of incident CML patients identified using the algorithm and the registries were 162 and 150, respectively. Conclusion We built and validated an algorithm to identify CML patients in administrative healthcare databases. In addition to prevalence estimation, the algorithm could be used for future economic evaluations or pharmaco‐epidemiological studies in this population.

Patterns of Tyrosine Kinase Inhibitor Utilization in Newly Treated Patients With Chronic Myeloid Leukemia: An Exhaustive Population-Based Study in France

“…Median age at occurrence of CML was 60 years, and 52.2% of patients were 60 years or older with a M/F ratio of 1.2. Age and gender characteristics (aging population with increasing male preponderance) corroborate and update findings from previous studies on the incidence of CML in France (from a few cancer registries) and the prevalence at a regional and national level (8,10). As reported in other studies, the overall survival rates in this population was similar to that of the general population (8,30).…”

“…or differences in study periods. However, the difference between different geographical areas and/or ethnical sub-groups cannot be excluded to explain these incidence variations ( 7 , 10 , 31 ).…”

“…The algorithm could not identify patients who had always received TKI in the setting of a clinical trial, in which case the TKI was provided by a sponsor and therefore not reimbursed by the National Health Insurance. In the EU Clinical Trials Register, there were 25 clinical trials on CML between 2004 and 2014 in France ( 48 ), which corresponds to an upper range of 628 prevalent patients that could have been missed by the algorithm ( 10 ). Among those patients, several discontinued the trials and were thus identifiable by the algorithm if treated by a TKI outside the trial.…”

“…A more recent study based on a health insurance database estimated the prevalence of CML in France at 16.3 (95% Confidence Interval (95%CI): 16.0-16.6) per 100,000 inhabitants, with a median age of 63 years (Inter Quartile Range (IQR) 51-73) ( 10 ).…”

Philadelphia Chromosome-Positive Leukemia in the Lymphoid Lineage—Similarities and Differences with the Myeloid Lineage and Specific Vulnerabilities

“…It constitutes 15% of all diagnosed adult leukemia cases in the US (1.95 new cases annually per 100,000 people) [ 19 ]. In Europe it affects one in 100,000 people each year, with the average age at diagnosis 64 years [ 20 ]. CML is usually diagnosed in a manageable, chronic phase (CP), but it can progress into accelerated phase (AP) or a fatal blast phase (BP).…”