H. Jean Khoury Cure CML Consortium

Roswell Park Cancer Institute
James Thompson, MD

Duke University Cancer Institute
Lindsay Rein, MD

Memorial Sloan Kettering Cancer Center
Michael Mauro, MD

Medical College of Wisconsin
Ehab Atallah, MD
Mary Horowitz, MD
Kathryn Flynn, PhD
Michael Deininger, MD, PhD 

Oregon Health & Science University
Brian Druker, MD
Michael Heinrich, MD

H. Lee Moffitt Cancer Center
Kendra Sweet, MD
Javier Pinilla-Ibarz, MD, PhD
Onyee Chan, MD
Zoey Xie, MD, MS

University Health Network- Princess Margaret
Jeffrey Lipton, MD, PhD

Augusta University Research Institute
Jorge Cortes, MD
Vamsi Kota, MD

Barbara Ann Karmanos Cancer Hospital
Jay Yang, MD
Charles Schiffer, MD

The Johns Hopkins University
B. Douglas Smith, MD

Weil Medical College of Cornell University & NY Presbyterian Hospital
Ellen Ritche, MD
Richard Silver, MD

Huntsman Cancer Institute - University of Utah
Srinivas Tantravahi, MD

Fred Hutchinson Cancer Research Center 
Jerald Radich, MD
Vivian Oehler, MD

University of California - San Francisco
Neil Shah, MD, PhD
Kelly Schoenbeck, MD

Mayo Clinic - Minnesota & Jacksonville
Talha Badar, MD

Winship Cancer Institute of Emory University
Anthony Hunter, MD

Dana-Farber Cancer Institute, Dana-Farber/Partners Cancer Care, Inc., Beth Israel Deaconess Medical Center, Inc.
Martha Wadleigh, MD

City of Hope
Haris Ali, MD
Paul Koller, MD

Cleveland Clinic Cancer Center
Sudipto Mukherjee, MD
Akriti Jain, MD

University of Chicago Comprehensive Cancer Center
Richard Larson, MD

University of Alabama
Ravi Bhatia, MD
Omer Jamy, MD

The results of this study, reveal what patients think about stopping TKI treatment for CML. Researchers conducted in-person interviews with 22 patients, half of whom wanted to try stopping their TKI. Participants included men and women across the typical age range for patients with CML.

There were multiple factors relevant to their decision about stopping TKIs:

• Thoughts about risk of relapse
• Reducing side effects
• Maintaining a treatment that’s working
• Financial considerations
• Thoughts about one’s age or social roles (e.g., being a caretaker)
• Wanting to take fewer medications
• Being reminded of having CML
• Need for increased monitoring 

Keep in Mind
Patients in this study were recruited from academic cancer centers only. A larger study that includes patients from both academic and community sites would help confirm the findings.

Learn More about HJKC3-0001

Using the results of the Life After Stopping TKIs (LAST) study, researchers investigated the possibility of discontinuing TKI therapy among 172 patients who demonstrated a strong and sustained response to treatment. The study explains why this approach was successful for some but not others and details the impacts on patient quality of life.

CML is caused when pieces of two different chromosomes break off and switch places, creating an abnormal gene called BCR-ABL1. This gene then tells the body to create a protein called BCR-ABL. Doctors test levels of BCR-ABL throughout CML treatment to determine how their patients are responding to drug therapy.

This study found that the presence of the BCR-ABL protein at the time of treatment discontinuation may be predictive of molecular recurrence (MRec). Over a three-year period:

• Patients with NO detectable levels of BCR-ABL at the time of treatment discontinuation had a 10% chance of MRec
• Patients with detectable levels of BCR-ABL at the time of treatment discontinuation had a 50%-64% chance of MRec

These findings suggest that patients who do not have detectable levels of BCR-ABL at the time of treatment discontinuation may be able to safely scale back the number of monitoring appointments that they attend during treatment-free remission. Doing so may help reduce the stress and anxiety often felt by patients leading up to these check-ups.

During treatment-free remission, there were significant improvements in patient-reported symptoms:

• 88% of patients had improvement in diarrhea
• 80% of patients had improvement in fatigue
• 35% of patients had improvement in depression
• 21% of patients had improvement in sleep disturbance
• 5% of patients had improvement in pain interference

The results of this study determined that TKI discontinuation was safe, and 61% of patients remained in treatment-free remission after 3 years.

Learn More about assessment of outcomes

This report explains the results of the Life After Stopping TKIs (LAST) study regarding patient changes in function after stopping and restarting TKI treatment.

Patient-reported outcome measurements (PROMs) were collected from 172 chronic myeloid leukemia (CML) patients who discontinued drug therapy over a 36-month period. Of those who remained in treatment-free remission (TFR) at 12 months:

• 92% reported improvement in social function
• 71% reported improvements in social isolation
• 10% reported improvements in satisfaction with sex life
• 4% reported improvements in physical function

These results can aid patients and physicians in deciding to pursue treatment discontinuation. For those who remain in TFR, functional outcomes improved overall. However, functional outcomes began to worsen for those who restarted treatment.

Learn More about functional outcomes

This study describes patient-reported pain in people with chronic myeloid leukemia (CML) after discontinuing tyrosine kinase inhibitor (TKI) treatment.

For CML patients who respond well to TKI, discontinuation of the treatment may allow for achievement of treatment-free remission with improvement of treatment-related symptoms. However, discontinuing TKIs has been associated with physician-reported pain increases in some patients.

Within three months of discontinuation of treatment:

• 20% of patients had a physician report pain on their behalf
• 13% of patients self-reported a large increase in pain
• 11% of patients began taking a new pain medication 

Each of these events was classified as an increase in pain level. There was limited overlap in these measures (that is, few patients had more than one indicator of increased pain).

In total, 60/172 (34.9%) of patients had an increase in pain in the first three months following treatment discontinuation. At six months, pain had returned to a baseline level and continued to decrease beyond that.

Three patients restarted TKI therapy to treat their pain. However, the results of this study show that the pain level trajectory was similar in patients who did and did not restart TKI treatment. This information suggests that restarting TKI treatment may not be more effective at reducing pain than remaining off the treatment.

To remain in TFR, patients should consider the following to manage any discontinuation-associated pain they may experience:

• Increased physical activity
• Use of an analgesic or and/or anti-inflammatory pain medication
• Behavioral/cognitive approaches to pain management

Learn more about reported pain

A survey of patients with chronic myeloid leukemia (CML) provides a patient definition of cure in CML treatment and outlines patients’ priorities for future CML research.

Researchers surveyed 458 people with CML across the United States. The overwhelming majority of patients (90%) considered the definition of cure for CML as “permanently stopping any CML treatment with no evidence of disease.”

Only 3% considered cure to be “taking a pill daily.”

To reach a treatment-free remission, many patients reported a willingness to add a new therapy to their TKI.

Patients universally supported more research in CML, specifically in:

• Stopping CML treatment
• Side effects of CML treatment
• CML not responding to treatment
• Long term complications from treatment