Photo by Rhoda Baer
Doctor consults with cancer patient and her father
A small study of 20 children aged 8 to 18 years with incurable or refractory cancers indicates children are reliable reporters of their symptoms and side effects.
The investigators collected reports from the children, who were enrolled on phase 1/2 clinical trials at 4 cancer centers and undergoing their first courses of chemotherapy.
The team assessed the feasibility and acceptability of collecting symptom, function, and quality of life (QOL) reports from the study participants.
The investigators also evaluated the measurement tool and interview questions at 2 time points.
They contend the youths’ self-reports potentially could be a new trial endpoint.
According to the investigators, only rarely do patient-reported outcomes (PROs) get incorporated into pediatric phase 1 or phase 2 trials.
And because these trials contribute to drug indications and labeling, the researchers decided to assess whether it was feasible to enroll young people and retain them in a PRO endeavor.
The researchers also assessed the reliability, validity, responsiveness, and range of the pediatric measures employed. They used the Patient-Reported Outcomes Measurement Information System (PROMIS) to capture statistically significant and clinically meaningful changes or minimally important differences (MIDs) in PROs.
Pamela S. Hinds, PhD, RN, of George Washington University in Washington, DC, reported the findings on behalf of the Children's National Health System researchers in Cancer, the journal of the American Cancer Society.
"When experimental cancer drugs are studied, researchers collect details about how these promising therapies affect children's organs, but rarely do they ask the children themselves about symptoms they feel or the side effects they experience," Dr Hinds said.
"Without this crucial information, the full impact of the experimental treatment on the pediatric patient is likely underreported and clinicians are hobbled in their ability to effectively manage side effects," she added.
The team recruited children and adolescents enrolled in phase 1 safety or phase 2 efficacy trials at Children's National, Seattle Children's Hospital, Children's Hospital of Philadelphia, and Boston Children's Hospital.
Findings
Sixty percent of the participants were male and 70% were white.
Median age of the participants was 13.6 years: 7 (35%) were age 8 to 12, and 13 (65%) were 13 to 17.
Thirteen participants (65%) had solid tumors, 5 (25%) had brain tumors, and 2 (10%) had lymphoma.
A total of 29 patients were eligible to participate in the trial during 20 months of screening. Five parents and 2 patients declined to participate.
The remaining 22 patients who agreed to participate accounted for a 75.9% enrollment rate. Twenty of them (90.9%) participated at the first data time point, which was at the time of enrollment, and 77.3% participated 3 weeks later at time point 2.
The authors noted that refusals to enroll were more likely to come from parents (17.2%) than the eligible patients (8.3%).
And refusals only occurred when the self-report measures were not embedded in the clinical trial.
Of the 10 protocols represented, 7 patients were enrolled on the same protocol in which the PRO measures were embedded.
The researchers administered the 6-item short-form measures for the scales of Mobility, Pain, Fatigue, Depressive Symptoms, Anxiety, and Peer Relationships.
They asked the 4 open-ended questions—concerning QOL while receiving therapy and acceptability of the patient reporting—at time point 2.
At time point 1, 3 patients did not complete 3 PROMIS measures, for a person-missing rate of 15% and a measure-missing rate of 3.3%.
At the second time point, 2 patients did not complete 1 measure each, for a person-missing rate of 11.8% and a measure-missing rate of 2%.
All but one measure at time point 1 met the reliability criterion and all measures did so at time point 2.
The research team believes their findings support the feasibility and acceptability of completing quantitative and qualitative measures regarding symptom, function, and QOL experiences among children and adolescents with incurable cancer.
The researchers note the small study size and the number of parent refusals are limitations of the trial.
Nevertheless, they recommend embedding PROs in future pediatric oncology phase 1/2 trials.
