CML: From Tragedy to Triumph

Patient responsibilities and empowerment

Chronic myelogenous leukemia (CML), a rare blood disease affecting approximately 1 in 100,000, is usually diagnosed in middle age. It was once fatal, but now innovative and effective treatment discoveries, i.e. oral tyrosine kinase inhibitors (TKIs), have dramatically changed life outcomes. However, this has come with a challenge – CML patients must now actively participate in management of a complex chronic disease. Understanding CML, its treatment, therapy options, drug side-e ects, management, and comprehension of therapy milestones are essential to treatment adherence and potential functional cure. Well-designed educational tools help improve response to treatment. Equal, fair access to cost e ective treatments, irrespective of geography, is also key to helping patients achieve their best possible outcomes.

Research drivers

The majority of CML patients diagnosed today can expect a normal lifespan. However, most remain on treatment to prevent recurrence. In Canada, five Health Canada approved TKI therapies exist: Gleevec ™, Sprycel ™, Tasigna ™, Bosulif ™, and iClusig™ as well as two generic versions of Gleevec ™, Teva-imatinib, and Apo-imatinib. Despite improved survival, research must continue to identify new, e ective, better-tolerated drugs and combinations, increase the probability for treatment-free remission as well as target novel mechanisms of resistance arising in di cult-to-treat patients. Importantly, CML patient needs must be appropriately voiced and incorporated into clinical trial design to ensure high patient quality of life.

Stakeholder relations

Transparency (a metaphor for openness, truthful communication and accountability) and patient centricity (that is, information comes from the patient and guides actions) are examples of new concepts directing CML management. The intent underlying these phrases is noble but their application is di cult and requires significant ethical oversight, ensuring the best for the patient while protecting privacy. Balancing patients’ timely access to expensive CML therapy as well as adequate monitoring of response, while recognizing others’ opposing needs presents a challenge for all stakeholders, ultimately affecting CML patients.