Dr Markman the Significance of PROs in Patients With Locally Advanced Cervical Cancer

"I believe this should be mandated. When a new drug is being approved by the FDA, the randomized trial needs to include patient-reported outcomes…it gets at the questions that are much more relevant. What do patients feel?"

Maurie Markman, MD, president of Medicine and Science at City of Hope, discussed the significance of patient-reported outcomes (PROs) from the phase 3 OUTBACK trial (ACTRN12610000732088) in patients with locally advanced cervical cancer, emphasizing the critical role of long-term quality-of-life assessments in evaluating standard treatment approaches.

The OUTBACK trial randomly assigned patients with locally advanced cervical cancer to receive standard cisplatin-based chemoradiation with or without additional adjuvant chemotherapy. Although the study demonstrated no survival benefit with the addition of adjuvant chemotherapy, it provided a robust dataset on patient experience, highlighting the persistent toxicity burden associated with treatment.

One year after therapy, patients in both arms reported moderate-to-severe impairments in multiple domains, including fatigue, sleep disturbance, sexual dysfunction, urinary and bowel symptoms, and mental health. These concerns remained prevalent at the 3-year mark, particularly among patients who received adjuvant chemotherapy. Participants in this group were more likely to report persistent numbness and tingling, suggesting additive neurotoxicity from extended systemic treatment.

Markman underscored the importance of these data, noting that although traditional end points such as progression-free and overall survival remain central, PROs are essential to understanding the lived experience of patients. He advocated for regulatory mandates requiring incorporation of PROs in both phase 3 and accelerated approval trial designs, stating that these outcomes offer critical context for treatment decision-making and drug approval.

According to Markman, the OUTBACK trial sets a precedent in cervical cancer by formally assessing the long-term impact of chemoradiation strategies, moving beyond efficacy to address survivorship. He noted that although patients may achieve disease control, the physical and emotional consequences of treatment often persist and require proactive management.