Male gender and unemployment are associated with lower levels of perceived social support in adults with cystic fibrosis.

OBJECTIVE: Adults with cystic fibrosis (CF) face unique challenges with regard to the attainment and maintenance of social support. Although social support has been shown to improve treatment outcomes in other patient-populations, research on social support in adults with CF is limited. In fact, no studies have examined factors associated with less perceived social support in this population. The current study aimed to fill this gap, thus providing CF care teams with empirical evidence about who may be most likely to lack support and inform future intervention. METHODS: Participants in this cross-sectional study included 233 adults with CF who were part of a larger, longitudinal study. Participants completed the Interpersonal Support Evaluation List, a measure of social support, and attended routine clinical visits where measures of disease severity were obtained. RESULTS: Being female and employed were associated with greater perceptions of social support in this sample. Age, income, education, marital status, and disease-severity were not related to perceptions of social support. CONCLUSION: The present study revealed that individuals with CF who are unemployed and those who are male perceived having lower social support, perhaps identifying subgroups for whom targeted interventions may be appropriate.

Social support is associated with fewer reported symptoms and decreased treatment burden in adults with cystic fibrosis.

BACKGROUND: Although social support has been linked to a variety of health outcomes in those with and without chronic illness, this construct has rarely been studied in adults with cystic fibrosis (CF), who may face a unique set of clinical and psychosocial barriers. The current study explored the relationships between social support, mental health, physical health, treatment activity, and disease-specific quality of life in a sample of adults with CF. METHODS: Participants in the study included 250 adults with CF who took part in a larger longitudinal study. Participants were administered a battery of measures including a social support evaluation (Interpersonal Support Evaluation List, ISEL), a health assessment (Memorial Symptom Assessment Scale, MSAS), a health-related quality of life measure (Cystic Fibrosis Questionnaire - Revised, CFQ-R), and treatment activity questionnaires (Tool for Adherence Behaviour Screening, TABS and other surveys). RESULTS: Linear regression analyses indicated that greater social support was associated with fewer self-reported mental and physical health symptoms, digestive symptoms, and eating disturbances over time. Social support also was associated with elevated emotional, social, and role functioning as well as vitality and improved body image. Those who reported more support perceived less treatment burden and better overall perceptions of their health. CONCLUSIONS: Social support has been shown to be associated with health outcomes in other chronic illnesses, and the same is true for adults with CF. This study fills gaps in the literature by examining outcomes of social support in this emerging adult population and providing a potential area for intervention.

Death after cessation of treatment by cystic fibrosis patients: An international survey of clinicians.

BACKGROUND: Little is known about cystic fibrosis patients, who are not considered to be terminally ill, and who die after voluntary cessation of treatment. AIM: This study was undertaken to provide an international snapshot of this issue. DESIGN: An online survey was distributed across three continents. SETTING: Distribution to the medical directors of the cystic fibrosis centres affiliated with the US Cystic Fibrosis Foundation, Cystic Fibrosis Australia (inclusion of New Zealand) and to every clinician member of the European Cystic Fibrosis Society. RESULTS: More than 200 cystic fibrosis patients not considered to be terminally ill and, who voluntarily ceased treatment, were reported by the clinicians surveyed. Detailed data were reported in 102 patients (4 children, 25 adolescents and 73 adults). Only one child, six adolescents and one adult were judged by clinicians not to be competent to make the decision to stop treatment. Time-consuming and low immediate-impact therapies, such as respiratory physiotherapy, were most frequently discontinued. Resignation was the main reported reason for discontinuing treatment, followed by reactive depression and lack of familial support. A total of 69% of the patients received palliative care and 72% died in the 6 months following cessation of treatment. CONCLUSION: Death of cystic fibrosis patients, not considered to be terminally ill, is reported in Europe, the United States and Australia due to voluntary cessation of treatment.

Motivating adherence among adolescents with cystic fibrosis: youth and parent perspectives.

As advances in the care of individuals with cystic fibrosis (CF) have resulted in improved survival, therapeutic regimens for treatment of CF have become increasingly complex. This high treatment burden poses challenges to chronic disease self-management, particularly amongst adolescents. The aim of this qualitative study was to understand the barriers and facilitators of adherence to chronic CF therapies as perceived by adolescents with CF and their parents. In a series of structured interviews with 18 youth and their parents, we explored issues related to daily routines, youth and parental roles regarding chronic therapy, and motivators for adherence. All interviews were audio-recorded and coded for themes and patterns. Reported barriers to adherence included time pressures, competing priorities, heightened awareness of disease trajectory, privacy concerns, and lack of perceived consequences from non-adherence. Identified facilitators for adherence included recognizing the importance of therapies, developing strong relationships with care teams, establishing structured routines, and focusing on shifting responsibilities from a parent to their adolescent child. The themes uncovered by these interviews identify areas for intervention and support by clinical programs seeking to improve adherence and self-management strategies for adolescents with CF.

Longitudinal trends in health-related quality of life in adults with cystic fibrosis.

BACKGROUND: Health-related quality-of-life (HRQOL) measures have been used as patient-reported outcomes in clinical trials in cystic fibrosis (CF), but there are limited data on HRQOL changes over time in adults with CF. METHODS: The Project on Adult Care in Cystic Fibrosis, a prospective, longitudinal panel study of 333 adults with CF at 10 CF centers in the United States, administered a disease-specific HRQOL measure, the Cystic Fibrosis Questionnaire-Revised (CFQ-R), seven times over 21 months. The CFQ-R assesses both physical and psychosocial domains of health. Growth curve regression models were developed for each CFQ-R domain, adjusting for demographic and clinical characteristics. RESULTS: Between 205 and 303 adults completed surveys (response rate, 70%-93%). Mean age at baseline was 33 years (range, 19-64 years); mean FEV1 % predicted was 59.8% (SD, 22%). Over the 21 months of follow-up, lung function, frequency of pulmonary exacerbations, and nutritional indices were associated with physical CFQ-R domain scores. There were no significant population trends over time in the physical domain scores; however, there were population time trends in three psychosocial domains: treatment burden (+8.9 points/y), emotional functioning (+3.2 points/y), and social functioning (-2.4 points/y). Individual variation in both physical and psychosocial subscales was seen over 21 months. CONCLUSIONS: In a longitudinal multicenter population of adults with CF, clinical variables such as FEV1, exacerbation frequency, and weight were correlated with related CFQ-R subscales. For the population as a whole, the physical domains of CFQ-R, such as respiratory symptoms, were stable. In contrast, population changes in several psychosocial domains of CFQ-R suggest that differentiating between the physical and the psychosocial trajectories in health among adults with CF is critical in evaluating patient-reported outcomes.

Watching Boston Med.

The author reflects on the ABC news documentary series Boston Med--both what it achieved, and what it could have achieved.

Palliation of dyspnea in pediatrics.

Dyspnea is a complex physiologic and psychosocial symptom that is difficult to characterize and measure, but essential to treat, as it has a significant effect on quality of life. Although palliation of dyspnea in the child with chronic illness is an under-researched area, children and their families cannot wait for the research to catch up with their current needs. This article addresses several aspects of dyspnea in pediatrics palliative care, with an eye toward practical suggestions for evaluation and management.

Associations between illness perceptions and health-related quality of life in adults with cystic fibrosis.

OBJECTIVE: The objective of this work was to examine the relationship between illness perception, health status, and health-related quality of life (HRQOL) in a cohort of adults with cystic fibrosis (CF). METHODS: In the Project on Adult Care in Cystic Fibrosis, we administered five subscales (Illness Consequences, Illness Coherence, Illness Timeline--Cyclical, Personal Control, and Treatment Control) of the Illness Perception Questionnaire--Revised (IPQ-R). Multivariable linear regression analyses explored the associations between illness perception, health status, symptom burden, and physical and psychosocial HRQOL, as measured by various domains of the Cystic Fibrosis Questionnaire--Revised (CFQ-R). RESULTS: Among the 199 respondents (63% female; mean age, 36.8 ± 10.2 years), IPQ-R scores did not differ on age, gender, or lung function. In multivariable regression models, neither clinical characteristics nor physical or psychological symptom burden scores were associated with CFQ-R physical domains. In contrast, higher scores on Illness Consequences were associated with lower psychosocial CFQ-R scores. Higher scores on the Illness Coherence and Personal Control scales were associated with higher psychosocial CFQ-R scores. CONCLUSION: Adults with CF report a high understanding of their disease, feel that CF has significant consequences, and endorse both personal and treatment control over their outcomes. Illness perceptions did not vary with increased age or worsening disease severity, suggesting that illness perceptions may develop during adolescence. Illness perceptions were associated with psychosocial, but not physical, aspects of HRQOL. Efforts to modify illness perceptions as part of routine clinical care and counseling may lead to improved quality of life for adults with CF.

NIH Consensus Development Conference statement: inhaled nitric-oxide therapy for premature infants.

Premature birth is a major public health problem in the United States and internationally. Infants born at or before 32 weeks' gestation (2% of all births in the United States in 2007) are at extremely high risk for death in the neonatal period or for pulmonary, visual, and neurodevelopmental morbidities with lifelong consequences including bronchopulmonary dysplasia, retinopathy of prematurity, and brain injury. Risks for adverse outcomes increase with decreasing gestational age. The economic costs to care for these infants are also substantial (estimated at $26 billion in 2005 in the United States). It is clear that the need for strategies to improve outcomes for this high-risk population is great, and this need has prompted testing of new therapies with the potential to decrease pulmonary and other complications of prematurity. Inhaled nitric oxide (iNO) emerged as one such therapy. To provide health care professionals, families, and the general public with a responsible assessment of currently available data regarding the benefits and risks of iNO in premature infants, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Heart, Lung, and Blood Institute, and the Office of Medical Applications of Research of the National Institutes of Health convened a consensus-development conference. Findings from a substantial body of experimental work in developing animals and other model systems suggest that nitric oxide may enhance lung growth and reduce lung inflammation independently of its effects on blood vessel resistance. Although this work demonstrates biological plausibility and the results of randomized controlled trials in term and near-term infants were positive, combined evidence from the 14 randomized controlled trials of iNO treatment in premature infants of ≤ 34 weeks' gestation shows equivocal effects on pulmonary outcomes, survival, and neurodevelopmental outcomes.

Research use of leftover newborn bloodspots: attitudes of Canadian geneticists regarding storage and informed consent requirements.

PURPOSE: Leftover newborn spots can provide a powerful research tool as a population-wide DNA bank. Some provinces/states store them for more than 20 years; however, parents are usually not informed of the retention of leftover newborn spots. To examine the opinions of Canadian geneticists regarding permission for leftover newborn spots storage for research purposes and the associated risks, a web-based survey was distributed to all members of the Canadian College of Medical Geneticists with a valid e-mail address (n = 209) and completed by 78 respondents (37%). RESULTS: The majority of respondents (73%) favored opt-out notification for retention of samples that would be held for longer than 2 years. For research on multifactorial conditions using leftover newborn spots originally banked without parental permission, geneticists favored different types of permission depending on the level of identifiable information attached to samples. Thirty-eight percent were concerned that information pamphlets that state that leftover newborn spots will be stored and may be "a source of DNA for research" would lead to a decreased participation in newborn screening. Twenty-eight percent believed that group stigma or family anxiety was likely to result from using nonidentified leftover newborn spots to study multifactorial conditions. CONCLUSION: The concerns of this knowledgeable cohort supports the critical importance of public engagement about both the potential risks and societal benefits associated with the use of leftover newborn spots in research as policy for leftover newborn spots is developed.

Lung transplantation for cystic fibrosis.

Lung transplantation is a complex, high-risk, potentially life-saving therapy for the end-stage lung disease of cystic fibrosis (CF). The decision to pursue transplantation involves comparing the likelihood of survival with and without transplantation as well as assessing the effect of wait-listing and transplantation on the patient's quality of life. Although recent population-based analyses of the US lung allocation system for the CF population have raised controversies about the survival benefits of transplantation, studies from the United Kingdom and Canada have suggested a definite survival advantage for those receiving transplants. In response to these and other controversies, leaders in transplantation and CF met together in Lansdowne, Virginia, to consider the state of the art in lung transplantation for CF in an international context, focusing on advances in surgical technique, measurement of outcomes, use of prognostic criteria, variations in local control over listing, and prioritization among the United States, Canada, the United Kingdom, and The Netherlands, patient adherence before and after transplantation and other issues in the broader context of lung transplantation. Finally, the conference members carefully considered how efforts to improve outcomes for lung transplantation for CF lung disease might best be studied. This Roundtable seeks to communicate the substance of our discussions.

Frequency and self-management of pain, dyspnea, and cough in cystic fibrosis.

Cystic fibrosis (CF) has been transformed from a fatal diagnosis in infancy to a chronic disease of children and young adults. Symptom patterns and disease burden in CF may be shifting to reflect the relatively healthier, older population with the disease. Self-management of symptoms is a hallmark of chronic illness, and yet we do not have a good understanding of how CF patients monitor or manage their symptoms. Children and adults were recruited through clinics in three Canadian provinces. Questionnaires with open-ended and close-ended questions in English and French, designed to assess the frequency, severity, and self-management of pain, breathlessness, and cough, were mailed to all the eligible participants. One hundred twenty-three respondents completed the survey, for a response rate of 64%. Eighty-four percent (103 of 123) of participants reported having pain. They reported an average of 2.1 locations of pain, with headache and abdominal pain most frequently described. Sixty-four percent (76 of 123) of participants reported having breathlessness, and 83% (99 of 123) of participants reported experiencing cough. Sixty-three percent (62 of 99) of participants with cough reported that cough always or sometimes interfered with their sleep. A variety of pharmacological and nonpharmacological treatments were used to manage symptoms. Pain and dyspnea are more common than suspected and a wide variety of pharmacological and nonpharmacological measures are used to treat symptoms. Cough is difficult to assess, but disturbed sleep may be an indicator of cough severity and an important symptom to consider when evaluating the overall burden of illness in those with CF.

Palliative and end-of-life care in cystic fibrosis: what we know and what we need to know.

PURPOSE OF REVIEW: This review will examine what is now known about palliative and end-of-life care for cystic fibrosis (CF) patients, including the changing demographics and context of such care, and then outline a brief research agenda to guide further work in this area. RECENT FINDINGS: We have now entered an era of more invasive end-of-life care in CF, due in part to changes in the demographics of death in CF and in part to the wider pursuit of lung transplantation. Recent single-center studies suggest that lung transplant listing changes the location and intensity of end-of-life care and complicates end-of-life care decision-making for the patient, family, and clinician. Further studies have demonstrated a high burden of daily symptoms in CF, especially pain and depression. In addition, there is evidence of an increasing burden related to the expanding daily treatment regimen for CF. SUMMARY: The time has come for national, multicenter studies of palliative and end-of-life care practices in CF, followed by systematic evaluation of the efficacy of existing interventions. Studies of the symptoms and treatment burdens in CF should continue with an emphasis on interventions to improve health-related quality of life throughout the lifespan of children and adults with CF.

High treatment burden in adults with cystic fibrosis: challenges to disease self-management.

BACKGROUND: More aggressive management of cystic fibrosis (CF), along with the use of new therapies, has led to increasing survival. Thus, the recommended daily treatment regimens for most CF adults are complex and time consuming. METHODS: In the Project on Adult Care in CF (PAC-CF), an ongoing longitudinal study of CF adults, we assessed self-reported daily treatment activities and perceived treatment burden as measured by the CF Questionnaire-Revised (CFQ-R), a disease-specific quality of life measure. RESULTS: Among the 204 respondents, the median number of daily therapies reported was 7 (IQR 5-9) and the mean reported time spent on treatment activities was 108 minutes per day (SD 58 min). Respondents reported a median of 3 inhaled and 3 oral therapies on the day prior to the survey. Only 49% reported performing airway clearance (ACT) on that day. There were no differences in the number of medications or the time to complete therapies based on gender, age or FEV1. The mean CFQ-R treatment burden domain score was 52.3 (SD 22.1), with no significant differences in the treatment burden based on age or FEV1. In a multivariable model controlling for age, gender, and FEV1, using 2 or more nebulized medications and performing ACT for >or=30 min were significantly associated with increased treatment burden. CONCLUSION: The level of daily treatment activity is high for CF adults regardless of age or disease severity. Increasing number of nebulized therapies and increased ACT time, but not gender, age, or pulmonary function, are associated with higher perceived treatment burden. Efforts to assess the effects of high treatment burden on outcomes such as quality of life are warranted.

Reasons for participation in pain research: can they indicate a lack of informed consent?

OBJECTIVE: To ascertain the self-reported reasons for participation in the clinical research of chronic low back pain and to evaluate those reasons in the context of informed consent and the concept of therapeutic misconception. This is the belief that research participation is equivalent to clinical care. DESIGN: Qualitative descriptive study with semistructured interviews. SETTING: Phone interviews with subjects with chronic low back pain after they completed a double-blind controlled trial. PARTICIPANTS: Fifty-two of 60 (86%) randomized controlled trial completers. RESULTS: Seventy-seven percent had more than one reason for study participation, including the following: to contribute to research; to seek relief of pain (both short- and long-term); to try a different drug; monetary remuneration; and to have their pain taken seriously. An initial altruistic reason for participation was often followed later in the interview by reasons of personal benefit. In most cases, the single question, "why did you participate?" was insufficient to reveal these multiple reasons. "Personal benefit" had many individual meanings, framed in the context of an illness narrative of coping with chronic pain. Despite reasons of personal benefit, subjects were still able to make the distinction between research and clinical treatment. CONCLUSIONS: Assessing the adequacy of informed consent requires a thorough understanding of how subjects viewed a study and their reasons for participation. Quantitative-based surveys may not capture the complexities of reasons for study participation. Reasons of personal benefit, seemingly contradictory reasons for participation, or overriding desire for relief may all affect the quality of informed consent. Yet, these issues may not automatically signal the presence of TM.

Advance care planning in adults with cystic fibrosis.

BACKGROUND: Because many patients with cystic fibrosis (CF) continue to survive into adulthood, discussion of end-of-life care decisions between clinicians and patients becomes a crucial part of CF adult care. Advance care planning (ACP) promotes alignment of patient care at the end of life with an individual's goals, however minimal research exists on ACP in CF. METHODS: We surveyed adults enrolled in the Project on Adult Care in Cystic Fibrosis (PAC-CF). We assessed experiences with ACP processes and communication and sought to identify factors associated with completion of an advance directive. RESULTS: The mean age of respondents (n = 234) was 34 years and the mean forced expiratory volume in 1 second (FEV(1)) was 64% predicted. Seventy-four percent reported that they had spoken to someone, generally a family member, about the care they would want if they became too ill to make decisions for themselves. However, only 30% reported completing an advance directive. Although 79% reported feeling comfortable talking to their clinician about ACP, only 28% said that their CF clinicians have asked about ACP. Having specific wishes about treatment decisions (odds ratio [OR] 7.8, 95% confidence interval [CI] 1.9-32.1) and reporting that a clinician had discussed ACP (OR 4.4, 95% CI 1.5-12.6) were significantly associated with reporting the completion of an advance directive. DISCUSSION: Though the majority of adults with CF report thinking about and communicating with family about advance care wishes, only a minority report completing an advance directive. Few adults with CF report being asked about ACP by their clinicians. Formulating specific wishes and discussing ACP with a clinician are strongly associated with completing an advance directive. Efforts to improve clinician communication with CF adults around ACP are needed to ensure that discussion of advance directives becomes an integral component of adult CF care.

Self-reported physical and psychological symptom burden in adults with cystic fibrosis.

Symptom burden is a key component of health-related quality of life in patients with cystic fibrosis (CF). To examine symptom prevalence and characteristics of adults with CF, we administered the Memorial Symptom Assessment Scale (MSAS), a previously validated measure of symptom burden, to CF patients enrolled in the Project on Adult Care in CF. The mean age of the 303 respondents (response rate 91%) was 32.8 years (range, 19-64); 58% were female, and their mean baseline pulmonary function (FEV(1) % predicted) was 69% (SD 28%). The median number of symptoms reported was 10, and there was no difference in the number of symptoms reported based on age, gender, or FEV(1). The most prevalent symptoms were cough (94%), shortness of breath (77%), and lack of energy (77%). Lack of energy and irritability caused the highest level of distress. MSAS symptom subscales were only moderately correlated with symptom status domains from existing CF health-related quality of life measures. Factor analysis led to the development of three distinct MSAS CF-symptom subscales, each with high internal validity. These findings show that adults with CF have a high symptom burden, particularly with respiratory and psychological symptoms, and that the new MSAS CF-specific subscales are a reliable measure of symptom distress in the CF population.

Self-reported involvement of family members in the care of adults with CF.

BACKGROUND: Few empirical studies have examined the role of family caregivers in the lives of adults with CF. METHODS: As part of the Project on Adult Care in CF (PAC-CF), an on-going prospective, longitudinal panel study of adults with CF, 119 family members and friends of adults with CF completed a mail survey in which they reported the frequency of help they provide for their family member with CF during routine care, hospitalization, and home IV treatment. RESULTS: The 119 caregivers were mainly spouses or unmarried partners (56%) and parents (29%). Fifteen percent of caregivers were children, friends, siblings or roommates. Family caregivers for adults with CF report assisting mainly with communication and social support during routine treatment, although one third provide some clinical care on a regular basis. Family caregivers report an increase in assistance during periods of acute illness, such as during a hospitalization and home IV treatment, especially with clinical care tasks. CONCLUSIONS: The depth of commitment required of families of children with CF has been well documented for decades. Our results suggest that the responsibilities of family members diminish only moderately as those with CF reach adulthood.

Chronic ventilator need in the community: a 2005 pediatric census of Massachusetts.

OBJECTIVES: The purpose of this study was to describe the population of children with chronic mechanical ventilation in Massachusetts and their patterns of medical care. PATIENTS AND METHODS: Investigators surveyed all of the Massachusetts home ventilator clinics, pediatric pulmonary services, hospital-based pediatric services for special health care needs, insurers, home care vendors, nursing agencies, the Massachusetts Department of Public Health, selected individual providers, and rehabilitation and long-term care facilities providing services to children with chronic respiratory support needs. Support was defined as daily use of noninvasive, negative-pressure, or invasive/transtracheal ventilators. Subsequent matching of demographic data, including date of birth, zip code, and gender supported maximal census yield without duplications. Geographic information systems were used to create distribution maps and estimate distances between children with chronic mechanical ventilator needs and key resources. RESULTS: A total of 197 children were identified as requiring chronic mechanical respiratory support in Massachusetts in 2005, which was a nearly threefold increase in this population in the 15-year interval since the last census. Congenital or perinatal-acquired neurologic or neuromuscular disorders constituted the majority of primary diagnoses (n = 107 [54%]). Chronic lung disease attributed to prematurity represented only 7% of the sample. CONCLUSIONS: Children receiving chronic mechanical respiratory support are a growing population. The shift in underlying diagnoses from pulmonary disease to neurogenic respiratory insufficiency has implications for hospital and community-based providers from all disciplines in extending services to the home setting. Barriers encountered when performing this study, however, reflect an overall lack of coordination among the many individuals and agencies involved in their care. Coordinated and centralized care efforts require a clear and managed flow of information; census reports such as this one are only the beginning. Direct needs assessments and quality-of-life surveys from families are needed to design and implement programmatic changes and advocacy efforts.

Integrating palliative care into chronic care for children with severe neurodevelopmental disabilities.

BACKGROUND: Children with severe neurodevelopmental disabilities and complex medical conditions are a growing and unique segment of the pediatric population. The increasing use of life sustaining technologies has provided the chance at an extended life and increasing inclusion within the broader community. Families work to overcome personal and professional biases, clinical uncertainties, and pragmatic obstacles to improve quality of life. Little attention, however, has been paid to the unique challenges of caring for a dying child affected with neurodevelopmental delay. DISCUSSION: In this paper we outline several specific barriers to the provision of excellent end-of-life care for these children and their families. We also outline our approach for overcoming these barriers. SUMMARY: The benefit of comprehensive palliative care in select pediatric populations has been demonstrated. Extending and tailoring those service to meet the unique needs of children with severe disabilities is the next logical step in that continuum. Ultimately, acknowledging that the lives of children with neurodevelopmental disabilities and their families have a unique quality will permit the human face of medical care to keep pace with technologic advances. Appreciation of the value of a perhaps incomparable quality of life will allow for a better quality of dying for children with severe neurodevelopmental disabilities.