Balance Hope and Quality of Life for Phase I Clinical Trials
Leita, a 42-year-old patient with locally advanced pancreatic cancer, was treated with 5-fluorouracil, irinotecan, and oxaliplatin followed by capecitabine and radiation therapy. After she completed initial treatment, a computed tomography scan conducted in preparation for a possible surgical resection revealed metastatic liver lesions. Leita’s surgery was cancelled, and she began second-line therapy with gemcitabine and nab-paclitaxel.
The oncologist recommends that Leita considers a phase I clinical trial after surveillance imaging showed disease progression and she continues to experience side effects such as a 25-pound weight loss, grade 2 peripheral neuropathy, intermittent nausea, and right upper-quadrant pain. Leita tells Sam, her oncology nurse, that she is hopeful that the trial will work for her because she is a single mother of two children. “I have to live for my kids,” she says.
Sam is conflicted: He does not want to take away Leita’s hope, but because the evidence suggests that the clinical trial’s treatment might not be effective for her, he believes that she should not invest the time away from home or risk experiencing the debilitating side effects associated with the therapy.
What Would You Do?
Phase I clinical trials can advance oncology clinicians’ and providers’ knowledge and may lead to new cancer therapy approvals. Participants in phase I clinical trials receive escalating doses of investigational treatments to identify the highest tolerable dosage (https://www.ons.org/cjon/21/4/palliative-care-and-phase-1-trials-intervention-improve-quality-life-and-provide-education) without severe side effects. Efficacy is not the primary endpoint (https://ascopubs.org/doi/10.1200/JOP.2017.026070), and patient performance scores are part of the inclusion criteria. One study identified that participants accepted into a phase I clinical trial initially had higher performance scores (https://acsjournals.onlinelibrary.wiley.com/doi/10.1002/cncr.25389) but more long-term side effects than comparable patients not involved in a clinical trial.
Other researchers hypothesized that structured palliative care interventions during phase I clinical trials could help patients maintain a higher quality of life (QOL), reduce their symptom profiles, decrease their adverse events, and participate in the trial longer than those receiving usual care. Although the findings were not significant, the researchers said that their results suggested their findings had merit and warranted additional research (https://ascopubs.org/doi/10.1200/JCO.2020.38.15_suppl.3634).
In a study comparing simultaneous care (a structured plan of care prepared by an oncology nurse and social worker) with usual care, patients receiving simultaneous care transitioned to hospice sooner than those receiving usual care, leading the scientists to conclude that simultaneous care supported QOL. In the study’s follow-up, the researchers added an educational intervention to simultaneous care, with participants’ QOL as the primary endpoint. Although the outcomes suggested that QOL was not significantly affected, QOL for caregivers in the intervention group declined more slowly than those receiving usual care (https://www.ons.org/cjon/21/4/palliative-care-and-phase-1-trials-intervention-improve-quality-life-and-provide-education), quantified by psychological, social, and spiritual scores.
Sam feels better after learning that Leita and her children will be staying with family living within 25 miles of the clinical trial site. He balances hope with reality by sharing the palliative care research and encouraging Leita to advocate for a palliative care referral to help manage her symptoms and make a smooth transition to hospice as needed.