Development of the measure of ovarian symptoms and treatment concerns: aiming for optimal measurement of patient-reported symptom benefit with chemotherapy for symptomatic ovarian cancer.

OBJECTIVE: The aim of this study was to determine the optimal patient-reported outcome measure (PROM) for assessing symptom benefit in trials of palliative chemotherapy for women with symptomatic ovarian cancer. METHODS: Candidate PROMs were EORTC QLQ-C30 plus ovarian-specific QLQ-OV28, Functional Assessment of Cancer Therapy-Ovarian (FACT-O), FACT Ovarian Symptom Index (FOSI), and gynecologic cancer-specific Symptom Representation Questionnaire. Predefined optimality criteria were inclusion of all symptoms necessary for the specified purpose, recall period covering typical length of palliative chemotherapy, numerical item rating scales, and all necessary symptoms included in a single symptom index. Qualitative and quantitative methods were applied to data from stage 1 of the Gynecologic Cancer Intergroup Symptom Benefit Study to determine the set of necessary symptoms and to objectively assess candidate PROMs against the optimality criteria. RESULTS: Ten necessary symptoms were identified: pain, fatigue, abdominal bloating/discomfort, sleep disturbance, bowel disturbance, nausea and vomiting, shortness of breath, poor appetite, urinary symptoms, and weight changes. Although QLQ-C30 and QLQ-OV28 together cover all these symptoms, they split them into numerous scales, dissipating potential symptom-benefit signal. Conversely, FACT-O does not cover all necessary symptoms and contains many other HRQoL-related items and treatment side effects, diluting potential symptom-benefit signal when summed into scales. Item response scales and composite scoring of all candidate PROMs were suboptimal to our specific purpose. We therefore developed a new PROM, the Measure of Ovarian Symptoms and Treatment (MOST) concerns, to provide optimal measurement for the specified purpose. CONCLUSIONS: This article documents the development of the MOST, a new PROM designed to assess patient-reported benefits and burden as end points in clinical trials of palliative chemotherapy for women with symptomatic ovarian cancer. The validity, reliability, and statistical efficiency of the MOST, relative to the best candidate scales of existing PROMs, will be assessed in the stage 2 of Gynecologic Cancer Intergroup Symptom Benefit Study.

Parents' attitudes to children's participation in randomized controlled trials.

OBJECTIVE: To explore parents' attitudes to children's participation in randomized controlled trials. STUDY DESIGN: Qualitative analysis of focus group discussions involving 33 parents from a pediatric teaching hospital and local school in Australia. Parents varied in age, gender, ethnicity, level of education, research experience, and child's health status. The transcribed discussions were analyzed by theme linkage using the constant comparative method. RESULTS: Parents balance risks and benefits when deciding about trial participation for their child. Perceived benefits include the offer of hope, better care of their child, the opportunity to access new treatments, healthcare professionals and health information, meeting others in similar circumstances, and helping others. Perceived risks include potential side effects, being randomized to ineffective treatments, and the inconvenience of participation. The decision for trial participation is also influenced by parental factors (parents' knowledge, beliefs, and emotional responses), child factors (the child's health status and preference about participation), trial factors (the use of placebos and uncertainties of participation), and doctor factors (doctor's recommendations and communication of trial information). CONCLUSIONS: Educating parents about trials, improving communication between trialists, pediatricians, and parents, increasing incentives while decreasing inconveniences, and providing decision aids for parents may increase parents' willingness to participate in trials.

Pediatricians' attitudes toward randomized controlled trials involving children.

OBJECTIVE: To examine pediatricians' attitudes toward children's participation in randomized controlled trials (RCTs) and identify possible barriers to participation. STUDY DESIGN: Qualitative analysis of focus group discussions involving 16 pediatricians and 5 trainees from a pediatric teaching hospital in Australia. Doctors varied in occupation, experience, research activity, age, sex, ethnicity, and parenthood experience. A professional facilitator conducted the semistructured group discussions. The transcribed audiotapes were analyzed by theme linkage by using the constant comparative method. RESULTS: Pediatricians believed parents balanced perceived gains and risks when deciding about trial participation. They thought the child's condition, parents' health beliefs and personal attributes, and the doctors' beliefs and relationship with the investigators influenced parents' attitudes. Perceived gains included professional benefits for pediatricians, improved patient care, convenience for the families and themselves, and scientific advancement. Perceived risks included inconvenience, inadequate resources, and potential harms to the patient and doctor-patient relationship. Pediatricians with previous research experience were most knowledgeable about RCTs and perceived greatest gains from trial participation. Pediatricians' personal treatment preferences hindered trial support. CONCLUSIONS: This study suggests that children's participation in trials will be enhanced by increasing pediatricians' awareness of RCTs through education and involvement in trials and by improving the gains-risk balance.