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NCCN Trends™ Highlights: Monitoring and Managing Chronic Myelogenous Leukemia

By Sherry L. Ulrich, MBA, Market Insights Specialist

With new targeted cancer therapies available, such as Tyrosine kinase inhibitors (TKIs) and other agents, Chronic Myelogenous Leukemia (CML) has been transformed into a manageable, chronic condition for many patients.[i]  An essential key to this transformation is the monitoring of patients’ therapy response and drug adherence, as well as early detection of therapy failure.[ii]  Frequent monitoring is required for measurement of hematologic, cytogentic, and molecular responses in order to evaluate therapy response and disease progression from chronic phase to accelerated or blast phase.[iii]

In March 2013, the National Comprehensive Cancer Network® (NCCN®) conducted an NCCN Trends™ survey about CML.  When survey respondents were asked what their next step would be if compliance or drug-drug interactions were not factors for patients who did not achieve a ≤10% BCR-ABL response by qRT-PCR at three months, the majority—41%—said they would continue on the originally prescribed TKI with regular qRT-PCR testing for 12 months before changing therapy, assuming a downward trend of transcript levels from baseline.

Assuming compliance or drug-drug interactions are not factors, what would be your next step if your patients did not achieve a ≤10% BCR-ABL response by qRT-PCR at 3 months? (Select all that apply) (n=464)

According to Assouline and Lipton, research has identified causes of resistance, including, but not limited to, mutations in the BCR-ABL gene.[iv]  In order to help guide the decision about which TKI (or other agent) to select when patients are unresponsive to therapy, it is suggested that patients’ CML cells are tested for BCR-ABL kinase domain mutations.[v]

Clinicians were asked when mutational analyses are performed on their patients with CML. The top two responses were “When there is a loss of response to therapy (e.g., a loss of CCyR, MMR, etc.),” and “when there is disease progression,” with response rates of 53% and 34%, respectively.

Additionally, the survey revealed that the majority of respondents would switch or modify therapy for their patients with CML anytime an NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) milestone is not reached, and/or if a BCR-ABL T3151 mutation is detected, following a mutational analysis test.

Following a mutational analysis test on your patients with CML, when do you decide to switch or modify therapy? (Select all that apply) (n=382)

In summary, new therapies have transformed CML into a manageable disease for most patients.  Essential treatment includes frequent monitoring of patients to manage CML in its chronic stage, as well as performing mutational analysis in order to discern the best-suited TKI or other agent for a patient unable to achieve response to therapy. Overall, frequent monitoring and timely therapy changes, as well as patient compliance, provide the potential for a better long-term outcome. 

Additional Resources:

October 16 -17, 2015, NCCN will be hosting its 10th Annual Congress: Hematologic Malignancies™ in San Francisco, California. This congress will focus on the new approaches that have been incorporated into patient management, including the use of drugs, biologics, and diagnostics. For additional information, please visit: http://www.nccn.org/professionals/meetings/hematological/default.aspx

The NCCN 9th Annual Congress: Hematologic Malignancies™ took place September 19 – 20, 2014, in New York, NY. Accredited materials from this congress are available through the NCCN Continuing Education Portal. Please visit: http://education.nccn.org/hem2014

Recorded presentations featuring Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Multiple Myeloma, and Non-Hodgkin’s Lymphomas from the NCCN 19th Annual Conference: Advancing the Standard of Cancer Care™ held March 13 – 15, 2014, in Hollywood, FL, are also available through the NCCN Continuing Education Portal. Please visit: http://education.nccn.org/ac2014


NCCN Trends Surveys & Data

NCCN Trends is a survey-based analytics tool from NCCN that focuses on how clinicians in the United States and abroad deliver cancer care. NCCN Trends Surveys pose questions regarding topics including, but not limited to, patterns of care and awareness and utilization of various treatment modalities, as well as key topics impacting oncology stakeholders, such as how changes in the health care environment impact them and their patients.

Data is gathered through brief electronic surveys to more than 154,000 health care providers who access NCCN.org on a frequent basis and express interest in responding to NCCN Trends Surveys. These clinicians consist of practicing physicians in diverse practice settings, including academic/research cancer centers, community hospitals, and private practices. Survey participants also represent pharmacists, nurses, and other oncology stakeholders. In 2014, NCCN conducted 12 NCCN Trends Surveys, averaging more than 900 clinician respondents per survey.

NCCN Trends™ Surveys and Data are independent of any NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) or other NCCN clinical recommendations.


[i] Zhen C, Wang YL. Molecular Monitoring of Chronic Myeloid Leukemia International Standardization of BCR-ABL1 QuantitationThe Journal of Molecular Diagnostics. 2013; 15(5):556-564.

[ii] Cortes J, Goldman JM, Hughes T. Current Issues in Chronic Myeloid Leukemia: Monitoring, Resistance, and Functional Cure. JNCCN. 2012; 10(3):S1-S13.

[iii] National Comprehensive Cancer Network®, NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®); Chronic Myelogenous Leukemia, Version 1.2015. http://www.nccn.org/professionals/physician_gls/pdf/cml.pdf

[iv] Assouline S., Lipton JH. Monitoring Response and Resistance to Treatment in Chronic Myeloid Leukemia. Current Oncology. 2011; 18(2):e71-e83.

[v] Jabbour E, Kantarjian H. Chronic Myeloid Leukemia: 2014 Update on Diagnosis, Monitoring, and Management. American Journal of Hematology. 2014;89(5): 547-556.