CML is a myeloproliferative neoplasm characterized by the presence of the Philadelphia chromosome.1
Most patients with CML are diagnosed in chronic phase disease, where the cancer cells multiply slowly. During accelerated phase and blastic crisis, the leukemic cells multiply more rapidly, producing a greater number of undifferentiated blastic cells that crowd out the healthy cells to a greater extent.1
With the breakthrough of targeted tyrosine kinase inhibitors (TKIs), most patients with CML in chronic phase (CML-CP) have the chance of living a normal life expectancy. Some patients may not respond optimally to TKI therapy, or may develop mutations that cause resistance to treatment. For these patients, and patients in accelerated phase and blastic crisis, there is a further need to advance treatment options.1
CML Facts and Figures
- CML accounts for approximately 15% of newly diagnosed cases of leukemia in adults1
- Approximately 9000 new cases of CML were diagnosed in the United States in 20192
- The median age at diagnosis is 65 years2
Diagnosis of CML
CML-CP can be diagnosed from peripheral blood findings combined with the detection of the BCR-ABL1 fusion gene t(9;22)(q34.1;q11.2).3
A bone marrow biopsy is essential to ensure a complete karyotype, and for morphologic evaluation to confirm the phase of disease.3
Molecular monitoring of response to treatment is critical. qPCR to detect BCR-ABL1 transcript is the most reliable tool for monitoring responses and can identify patients who achieve deep molecular responses and detect emergence of primary or secondary resistance.3,4
Unmet Needs in CML
Most patients with CML-CP have near normal life spans; however, helping more patients achieve deep molecular responses, which is associated with improved long-term outcomes and the possibility of treatment discontinuation, is an emerging treatment goal.5
BCR-ABL1 = breakpoint cluster region-Abelson murine leukemia 1 gene; CML = chronic myelogenous leukemia; qPCR = quantitative polymerase chain reaction.
References: 1. Jabbour E, Kantarjian H. Am J Hematol. 2018;93:442-459. 2. National Cancer Institute. Cancer Stat Facts: Leukemia – Chronic Myeloid Leukemia (CML). 2019. https://seer.cancer.gov/statfacts/html/cmyl.html. Accessed November 4, 2019. 3. Arber DA et al. Blood. 2016;127:2391-2405. 4. Druker BJ. Blood. 2008;112:4808-4817. 5. Radich JP et al. J Natl Compr Canc Netw. 2018;16:1108-1132.
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.