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Cystic fibrosis (CF) is a progressive, genetic disease affecting multiple organ systems. Treatments are complex and take 2–4 h per day. Adherence is 50% or less for pulmonary medications, airway clearance, and enzymes. Prior research has identified demographic and psychological variables associated with better adherence; however, no study has extensively identified facilitators of treatment adherence (e.g., adaptive behaviors and cognitions) in a sample of parents and adolescents. Forty-three participants were recruited from four CF centers as part of a larger measurement study. Participants included 29 parents (72% mothers; 72% Caucasian) and 14 adolescents (ages 11–20, 64% female, 71% Caucasian). Participants completed semi-structured interviews to elicit barriers to adherence. However, facilitators of adherence naturally emerged, therefore indicating need for further exploration. Interviews were audiotaped, transcribed and content-analyzed in NVivo to identify those behaviors and beliefs that facilitated adherence, using a phenomenological analysis. Frequencies of these themes were tabulated. Nine themes emerged, with individual codes subsumed under each. Themes included social support, community support, organizational strategies, “intrinsic characteristics,” combining treatments with pleasurable activity, flexibility, easier or faster treatment, prioritizing treatments, and negative effects of non-adherence. Results demonstrated the importance of identifying strategies that positively affect adherence. Interventions that are strength-focused, build on prior success, and utilize positive models generated by those who have successfully integrated CF treatments into their lives are more likely to be efficacious.

Patient-reported outcomes (PROs) are instruments that evaluate daily functioning and health outcomes from the patient's perspective. If developed using standardized procedures (FDA Guidance, 2009), they can be used as primary or secondary outcomes in clinical trials evaluating new medications and treatments. This chapter reviews the definition, development, and utilization of PROs for both research and clinical purposes, including developmental considerations for administration of PROs with children. Health-related quality of life measures (HRQoL) are one type of PRO, and several condition-specific PROs have been developed for a variety of pediatric respiratory diseases, including vocal cord dysfunction, asthma, cystic fibrosis, sleep-related breathing disorders, and primary ciliary dyskinesia. A substantial body of literature has demonstrated that condition-specific, rather than generic measures, are more sensitive to change and better reflect the patient's symptoms and functioning. This chapter reviews the currently available PROs for pediatric respiratory conditions, including a description of the instrument, the domains of functioning it measures, the appropriate developmental age for administration, and the psychometric properties of the instrument, including its reliability and validity. Use of PROs is becoming standard practice for both randomized clinical trials and clinical care. The current shift in medicine toward patient-centered care is consistent with development and use of PROs. These measures provide unique information about patient symptoms, level of daily functioning, and systematic response to treatment. These measures have also been shown to facilitate patient-provider communication and shared decision-making. Integration of PROs into clinical care is a critical step in promoting patient-centered, quality health care practice.

Purpose: ASCO recommends early involvement of palliative care for patients with advanced cancers on the basis of evidence from 18 randomized trials. We examined racial and ethnic minority representation in these trials and the role of race and ethnicity in the statistical analyses. The goal was to identify specific gaps in the palliative care evidence base for these individuals and potential strategies to address them. Methods: We reviewed the 18 trials cited in the 2012 and 2017 ASCO clinical statements on integrating palliative care into oncology. We extracted data on the reporting and categorization of race and ethnicity, on the enrollment of specific racial and ethnic minority groups, and on how race and ethnicity were addressed in the analyses. Results: One third of patient trials reported representation of specific racial and ethnic minority groups, one third reported rates of “white” versus “other,” and one third did not report race or ethnicity data. Among the patient trials with race and ethnicity data, 9.9% of participants were Asian, 8.8% Hispanic/Latino, and 5.7% African American. Analyses that used race and ethnicity were primarily baseline comparisons among randomized groups. Conclusion: Race and ethnicity were inconsistently reported in the trials. Among those that provided race and ethnicity data, representation of specific racial and ethnic minority groups was low. In addition to more research in centers with large minority populations, consistent reporting of race and ethnicity and supplementary data collection from minority patients who participate in trials may be strategies for improvement.

Cystic fibrosis (CF) is a life-shortening and recessive genetic disorder that affects approximately 1 in 2500 live births worldwide. The disorder affects the functions of multiple organs including the lungs, pancreas, liver, and reproductive system and requires adherence to a time-consuming and burdensome treatment regimen. This chapter reviews literature regarding adherence to this treatment regimen, the role of the family in treatment adherence, the impact of CF on family functioning and psychological health, the effect of infection control guidelines on CF patient social support and functioning, and the processes associated with the transition from pediatric to adult care. Empirically evaluated interventions targeting each of these challenges are discussed. Reviewed literature points to the importance of: 1) considering the changing needs of individuals with CF in light of developments in medical practices and policy, 2) the need for supportive interventions for families and individuals with CF to improve family functioning, 3) treatment adherence and mental health, and 4) the necessity of programs that facilitate the transition of treatment responsibility from parents to their children with CF.

Purpose of review Cystic fibrosis (CF) is the most common genetic, life-shortening illness among white populations. Management of the disease requires a complex, time-consuming treatment regimen. The purpose of this review is to highlight current research examining the psychological burden of CF, including psychological distress, social challenges, treatment burden, and adherence to daily treatments. Recent findings Individuals with CF and their parent caregivers report elevated symptoms of depression and anxiety. Recent international guidelines (Cystic Fibrosis Foundation and European Cystic Fibrosis Society) recommend annual screening of these symptoms using the Patient Health Questionnaire-9 (PHQ-9) and Generalized Anxiety Disorder-7 (GAD-7) scales. Symptoms of depression have been associated with decreased adherence, lower quality of life, and higher healthcare costs. Adherence to pulmonary medications has been found to be 50% or less and decreases with age. Poor adherence has been associated with higher healthcare costs, more frequent hospitalizations, and worse quality of life. Summary Individuals with CF face unique challenges that can lead to psychological burden. Screening for these symptoms and developing effective interventions to improve adherence are the key targets for the next 5 years of research.

Background: Patient-reported outcomes (PROs) offer key information about living with a chronic illness. NTM can co-occur with bronchiectasis, causing symptoms that impair health-related quality of life (HRQoL). NTM is hard to treat and few good outcome measures exist. Thus, PROs could measure symptom improvement when testing new treatments or when using existing therapies. This study developed a HRQoL module specific to NTM symptoms to accompany the QoL–Bronchiectasis (QoL-B). Methods: Following FDA Guidance on PRO development, NTM literature was reviewed, focus groups were led by a psychologist, and items were created and tested. Focus groups were conducted with adults with NTM and non-CF bronchiectasis at 2 sites (N=31,M age=67.4,94% female,100% white). Patients were asked how NTM affects their daily lives, completed the QoL-B, and proposed items specific to NTM. Nine physicians specializing in pulmonology and NTM participated in a panel to suggest relevant items. Open-ended interviews were conducted (N=9,M age=65.6,89% female,89% white) to assess clarity of instructions, items, and rating scales. A draft measure was created and tested in cognitive interviews with 46 adults (M age=68.5,94% female,98% white); feedback was elicited. Results: Focus groups identified eating issues, fever, and chills; physicians also identified body image as a concern. The NTM Module consisted of: NTM Symptoms, Body Image, Eating Problems, and Digestive Symptoms. Strong reliability was obtained (α=.72-.87). Conclusions: Cognitive testing indicated that the draft items were relevant and easy to understand. Next steps include psychometric validation and identification of the minimal important difference score (MID).

104 Background: While ASCO guidelines recommend early palliative care (PC) for patients with metastatic cancer, few African Americans (AA) have participated in PC trials and little data exist on the acceptability and effect of early PC in this population. Using the ADAPT-ITT framework for culturally tailoring evidence-based interventions, we assessed perceptions of PC and its early introduction in oncology in AAs. Methods: We conducted four focus groups with AAs from community centers in Miami. We enrolled adults without a history of cancer to approximate the knowledge and beliefs of someone with newly diagnosed cancer. Focus groups explored knowledge of PC, value of different components of early PC, and perceptions about introducing PC early after cancer diagnosis. We recorded and transcribed the focus groups, performing thematic analysis with NVivo. Results: Thirty-seven AAs participated. Over half were female (24/37), and median age was 54 years old (range 35-71). Participants had little to no knowledge of PC. Lack of familiarity was attributed to AAs receiving sub-standard care and the belief that PC has been kept from AAs. Rather than being concerned that it might result in less cancer treatment, they reported that PC was actually part of doing everything possible for cancer, and it is the “best care.” PC was viewed as a solution to a major theme of doctors not providing adequate information for patients and families to prepare for negative outcomes such as death. However, some objected to discussing specific survival estimates for fear of speaking them into existence. Distinctions were drawn between what doctors manage (treatments) and what God manages (life/death). Participants could not understand why PC would be reserved for later in the course of illness instead of immediately after cancer diagnosis (“Don’t wait until I’m dying; give it to me now!”). PC was described as “supervising your needs and making sure they are addressed.” Conclusions: AAs were enthusiastic about early PC in oncology, but did not support providing specific estimates of survival. End-of-life planning was considered by some to be spiritual and not medical.