Registry for patients with chronic myeloid leukemia (CML) who have received at least two different medications and are switching to a different medication

Chronic myeloid leukemia (CML) is a form of blood cancer in which a subtype of white blood cells multiplies uncontrollably. In almost all cases, it is caused by a specific genetic mutation. Two chromosomes exchange segments, resulting in the so-called Philadelphia chromosome, which carries the mutated BCR-ABL gene. This gene causes the cells to divide continuously, leading to an accumulation of too many immature cells in the blood and bone marrow. This form of the disease is referred to as Philadelphia-positive (Ph+) or BCR-ABL-positive CML.

The standard treatment for CML consists of medications from the group of tyrosine kinase inhibitors (TKIs). They specifically block the enzyme that, as a result of the genetic mutation, promotes uncontrolled cell growth. However, in most cases, the disease cannot be cured. The goal of treatment is to suppress the disease to such an extent that it has little impact on patients’ daily lives. This also means that the medications must be administered over a very long period of time. Over time, however, resistance or intolerance to the TKIs used may develop. For this reason, various TKIs have since been developed, some of which target different sites on the enzyme. One new TKI, for example, is the drug asciminib, which is already approved for patients who have previously received at least two drugs from the TKI class.

The goal of this registry study is to collect various data from patients with CML who have already been treated with at least two TKIs and are also scheduled to receive a TKI as third-line therapy. The study does not test new therapies nor does it specifically analyze the data. The sole purpose is to collect data that will then be analyzed as part of future projects. The study collects information on the reasons for the switch, the dosage, the duration of treatment, the efficacy, and the tolerability of the therapies. In addition, questionnaires are used to assess how the disease and treatment affect patients’ daily lives and quality of life.​

Women and men with CML who are at least 18 years old at the time of switching to third-line or subsequent-line TKI therapy are eligible to participate in the study. The CML must be Philadelphia chromosome-positive or BCR-ABL1-positive. Patients must have previously been treated with TKIs in at least two prior lines of therapy. The disease must not have a T315I mutation and must be in the chronic phase.