Reframing Success in CLL With Real-World and Patient-Reported Data

This interview explores how patient-reported outcomes are enhancing CLL treatment strategies and helping clinicians understand the full impact of care.

Erin Mulvey, MD, is an assistant professor of medicine in the Division of Hematology and Medical Oncology at Weill Cornell Medicine. She competed her undergraduate education at Boston University, where she earned a degree in biochemistry and molecular biology. She received her medical degree from New York Medical College. Dr Mulvey completed both her residency in internal medicine and her fellowship in hematology/oncology at NewYork-Presbyterian Hospital and Weill Cornell Medical College.

How do you approach treatment selection for patients with chronic lymphocytic leukemia (CLL), especially those with comorbidities or treatment-resistant disease?

Erin Mulvey, MD: When I meet a new patient, either with treatment-naive or relapsed disease, I like to spend time getting to understand the patient, their background, and how their diagnosis of CLL is impacting their life. I always review the biology of their CLL, taking into account factors like their particular cytogenetic and molecular abnormalities. I also discuss their other comorbidities and any side effects they may have experienced with treatments. Together, we review these factors and discuss treatment options, including potential clinical trials, and develop an individualized plan.

How do patient-reported outcomes (PROSs) inform your clinical practice and ongoing research in CLL?

Dr Mulvey: I am very eager to see more CLL studies collecting PROs. Given the nature of CLL, the ultimate goal of treatment is to improve the quality of life for our patients, and as someone who treats CLL, I am constantly advising patients on issues of symptoms and side effects. PROs are a way to systemically evaluate the burden of treatments beyond the traditionally collected toxicity data, which typically focuses on physical side effects as estimated by a clinician.

Are there specific PROs that you believe are currently underutilized but could provide valuable insights into patient quality of life or treatment efficacy?

Dr Mulvey: There are a lot of excellent PRO tools that can help to augment traditional safety data. Some of these tools help to better quantify subjective side effects like fatigue or nausea. The PRO-CTCAE is a terrific tool for this purpose. These data can substantially help in guiding treatment selection. Also very important are tools that evaluate other quality-of-life domains like psychologic and social functioning, which are vastly important for patients with CLL. These domains are often impacted by receiving a diagnosis of CLL before treatment is even considered, and it's crucial to understand how treatment affects these facets of a person's life in addition to physical side effects like nausea or bruising. Several tools have been developed, including the EORTC QLQ-CLL17.

How is the evolving understanding of immune system modulation influencing the development of novel therapies in CLL?

Dr Mulvey: Patients with CLL have abnormal lymphocyte function, even prior to starting therapy. As the use of novel therapies such as CAR-T and bispecific antibodies evolves in CLL, a better understanding of microenvironment dysregulation can shape how we use these treatments. For example, CAR-T is a promising treatment option for patients with CLL, with durable remissions achieved by subsets of patients. For other patients, however, responses are much more limited, and this is likely in part due to CLL-mediated T-cell dysfunction. Understanding the role of the CLL microenvironment may lead to mitigation strategies to allow more patients with CLL to benefit from CAR-T therapy.

How can insights from real-world evidence complement clinical trial data in guiding treatment decisions for CLL?

Dr Mulvey: Real-world evidence can be a helpful supplement to clinical trial data, providing access to larger, more diverse, and often sicker populations. Real-world evidence also can give meaningful insight into side effects and management strategies such as dose reductions that a clinical trial may not capture.