Chronic Myeloid Leukaemia

Gender Neutral
Type Module
Testing Phase IV - in development
Principal investigator(s)
Fabio Efficace
Rome, Italy
Study coordinator(s)
Michela Servadio
Rome, Italy

Project summary

Five-year relative survival rates of chronic myeloid leukemia (CML) patients have increased dramatically over the last fifteen years. This reflects the major advances made in the treatment of this disease due to the advent of tyrosine kinase inhibitors (TKI). The number of people living with this disease has doubled since 2001 and this trend is expected to continue. Health-related quality of life (HRQoL) has thus become an important aspect to be considered in this disease. Implementing HRQoL, as an outcome measure in a clinical study of patients with CML, can greatly contribute to provide invaluable information to make more informed treatment decisions.

Some ten years ago, the treatment of CML was relatively straightforward as all patients received imatinib as first-line treatment and for those who failed imatinib, the only available proven alternative was allogeneic stem cell transplantation. In recent years, also dasatinib and nilotinib (i.e., second generation TKIs) have been approved for first-line use in CML making the selection of which TKIs to use front line a challenge for many physicians. Also, several other drugs are available for second line therapies and data on HRQOL are thus essential to understand the effects of these drugs from the patients’ perspective.

In an effort to provide the CML community with a valid HRQoL measurement tool, an international program to eventually develop such tool has started.


The provisional EORTC QLQ-CML24, has been cross-culturally developed in 10 countries including the USA and countries in Europe and Asia. This development process involved overall 655 CML patients treated with targeted therapies. The CML Advocates Network was also involved in the project further strengthening the quality of the final version of the questionnaire.

The EORTC QLQ-CML24 module consists of 24 items assessing the following aspects: Symptom Burden, Impact on Daily Life, Impact on Worry/Mood, Body Image Problems, Satisfaction with Care and Satisfaction with Social Life.

An international module field testing, involving 12 countries, is now ongoing in order to provide additional prospective validation data (Phase IV validation).

Future plans

Closing of the study is expected by the end of March 2019 Final report and publication are planned for Spring/Summer 2019.

For patients

Nowadays, chronic myeloid leukemia (CML) patients receive prolonged, even lifelong, treatment. Thus, consideration on the effects of the available therapies on patients’ quality of life (QoL) is critical to robustly inform and improve their care. The main objective of this project is to provide the CML community with an internationally validated QoL questionnaire to be used in clinical research and clinical practice.