T. I. Ionova, D. A. Fedorenko, T. P. Nikitina, K. A. Kurbatova, E. I. Usacheva

Validation of Comprehensive Symptom Profile Questionnaire for patients with Chronic Myeloid Leukemia – CSP Leuk-CML

At present patient benefits is an important outcome of treatment of hematological disorders, in particular, of chronic myeloid leukemia (CML). Symptom profile and severity is one of the important treatment outcomes in patients with chronic myeloid leukemia (CML). Comprehensive symptom assessment before treatment as well as monitoring during treatment is helpful to enhance control of adverse events and side effects of CML therapy.

Recently a new tool, Comprehensive Symptom Profile in Chronic Myeloid Leukemia (CSP Leuk-CML), has been developed to assess 47 disease and treatment related symptoms in CML patients. We aimed to test practicability, validity, reliability and sensitivity of CSP- Leuk-CML in CML patients. 90 patients with chronic phase CML were enrolled in the study: mean age – 54.9 years old, SD 15.4; male/female – 44/46. The patients filled out CSP Leuk-CML and generic quality of life (QoL) questionnaire SF-36 before and at 3 and 6 months after treatment with TKI start.

Hematologists participating in the study were interviewed regarding the use of PROs in their decision-making; patients were interviewed regarding the format and value of CSP Leuk-CML to reveal different problems specific to CML and its' treatment. In order to assess the construct validity of CSP Leuk-CML the factor analysis was used as well as the method of "known-groups" and correlation analysis.

Chronbach alpha coefficient was calculated for reliability assessment. Analysis of symptom severity in different patient groups was performed using t-test for sensitivity and responsiveness assessment. Practicability of CSP Leuk-CML was shown: patients needed about 15 min to answer it; the proportion of missing values was 0.56% for all questions; all items were easy for the patients to read and understand.

Hematologists considered information about changes in symptom profile practical and useful for their decision-making. Of special importance for them was information about patient-reported treatment side effects. Usefulness of the tool to distinguish patients in terms of severity and number of disease- and treatment specific symptoms was demonstrated. The construct validity of CSP Leuk-CML was proven by factor analysis and "known-group" comparison. Factor analysis found five underlining constructs (domains) of the tool (explained 66% of the total variance) with