Real-Life Rates of Disease Monitoring in Clinical Practice in Europe: The “Unmet Needs in CML and Ph+ALL” (UNIC) Study.
Authors: Morra Enrica , Michallet Mauricette , Steegmann Juan , Costa David Marin , Ossenkoppele Gert , Verhoef Gregor , Kühr Thomas , Björeman Mats , Paga Cosimo , Cohen Carine , Villanueva Isidro , Halkin Véronique , Intorcia Michele , Cerri Karin 
Published: April 2020

Current recommendations for patients with CML (Baccarani M, et al. Blood2006;108:1809–20) advocate regular treatment response monitoring, including evaluation of: cytogenetic response (CyR) at least every 6 months until complete CyR is achieved, then every 12 months; molecular response (MolR) every 3 months; mutational analysis in cases of failure/suboptimal response. Yet, few data exist on monitoring in current clinical practice. A survey of CML physicians in the US/Europe suggests practices in some CML management areas are not in line with recommendations (Kantarjian H, et al. Cancer2007;109:1365–75). UNIC is a cross-sectional study, with retrospective chart review of currently treated CML or Ph+ALL patients in Austria, Belgium, France, Italy, Netherlands, Spain, Sweden and UK. Patients were recruited September 2006-March 2007. The study aimed to estimate the proportion of patients ever treated with imatinib and those who experienced imatinib resistance and/or intolerance (primary objectives). Here, we focus on data collected on patterns of disease monitoring (a secondary objective) in CML/Ph+ALL patients. A patient was defined as imatinib resistant if reported as such by the physician in the medical chart. Case Report Forms (CRFs) were completed for 1716 patients. CRFs were analyzable for 1551 CML and 48 Ph+ALL patients. Of the CML patients, 98% were in chronic phase; 2% were in advanced phases. Of the 1493 (96%) CML and 46 (96%) Ph+ALL patients who received imatinib, 241 (16%) CML and 6 (13%) Ph+ALL patients were reported as resistant. Patterns of cytogenetic, molecular and mutational testing are shown in the tables. In the last 12 months, 31% of CML patients had not had a cytogenetic analysis and 10% had not had a PCR analysis to assess MolR. Of imatinib-resistant patients, 57% CML and 50% Ph+ALL patients had not been assessed for mutations.

Examination CML Ph+ALL Number of cytogenetic analyses per patient in the last 12 months, n (%) N=1427 N=42 0 447 (31.3) 13 (31.0) 1 559 (39.2) 15 (35.7) 2 271 (19.0) 2 (4.8) ≥3 150 (10.5) 12 (28.6) ≥1 fluorescent in situ hybridization since diagnosis, n (%) N=1497 N=45 No 852 (56.9) 18 (40.0) Yes 645 (43.1) 27 (60.0) ≥1 PCR in the last 12 months, n (%) N=1504 N=48 No 149 (9.9) 2 (4.2) Yes 1355 (90.1) 46 (95.8) Patients with 4 PCRs in the last 12 months, n (%) N=1422 N=40 200 (14.1) 4 (10.0) Number of mutational analyses since diagnosis in imatinib-resistant patients, n (%) Imatinib-resistant patients CML (N=209) Ph+ALL (N=6) 0 120 (57.4) 3 (50.0) 1 75 (35.8) 3 (50.0) 2 12 (5.7) 0 ≥3 2 (1.0) 0

This large European observational study suggests different methods of disease monitoring are used less often in real life than according to recommendations. Further research into the consequences of suboptimal monitoring of patients’ disease status is warranted.