F. Finlayson

End of life care in CF: Patients, families and staff experiences and unmet needs

UNLABELLED Palliative care is not well understood in CF. Unmet needs of patients with CF, their families and staff were explored. METHOD Focus groups and interviews with forty-two participants (12 patients, 10 family members and 20 staff) were conducted at a university teaching hospital. RESULTS Thematic analysis identified six themes. Knowledge: Patients and families felt their knowledge of palliative care was limited. Psychological frame: Hope and a positive psychological frame was essential to coping, however, this was a hindrance to the acquisition of information. Denial as a coping strategy resulted in a lack of preparation for declining health. Treating team: High expectations were placed on the treating team. Psychosocial support was valued. Communication: Timing, honest and clear discussions were important. Engagement with palliative care service: Increased palliative care. Unmet needs: The emotional burden of caring for dying patients/families and balancing hope against death was a challenge. CONCLUSIONS Opportunities exist to improve care.

End-of-life care in adults with cystic fibrosis.

BACKGROUND Cystic fibrosis (CF) is a life-limiting congenital disease, with most patients dying at a young age of progressive lung disease. Lung transplantation offers hope for many but may not occur. There is little to guide the provision of palliative care to this young population who maintain hope in the possibility of lung transplantation. METHODS To inform the development of an appropriate model of palliative care, a medical record review of CF patients dying within a 5-year period without lung transplantation was undertaken. The aspects of care that were preventative, therapeutic, or palliative were quantified. RESULTS Of the 20 records studied, all died in hospital. Only 15% of patients had a do-not-resuscitate order agreed to more than 1 week prior to death, increasing to 90% at the time of death (median of 2 days before death). Opioids were prescribed for 1 patient (5%) at 1 week prior to death, increasing to 85% of patients in the last 24 hours of life (median of 36 hours before death). During the last 24 hours of life, intravenous antibiotics continued in 85%, and assisted ventilation in 90% of subjects. CONCLUSION We conclude that the circumstances surrounding the death of patients with CF holds challenges for their effective palliative care. CF patients continue life prolonging and preventative treatments until the last hours of life. There is an urgent need to examine palliative care approaches that may usefully coexist with maintaining transplantation options in the end-of-life care of this population.

Distal intestinal obstruction syndrome in cystic fibrosis: presentation, outcome and management in a tertiary hospital (2007–2012)

Cystic fibrosis (CF) can result in distal intestinal obstruction syndrome (DIOS) due to inspissated mucus. This paper describes the clinicopathological characteristics of adult CF patients with DIOS and assesses risk factors for surgery.

Adverse events arising from a palliative care survey

Development of evidence-based practice requires investigation of the attitudes and needs of patients, families and healthcare professionals, particularly for sensitive subject areas. Cystic fibrosis (CF) is a recessively inherited life-limiting disorder resulting in early death. Patients with this condition generally expect that lung transplantation will be an available treatment option; however, this is uncertain. A dual approach to care that involves both preparing patients for transplant assessment, while simultaneously exploring acceptable palliative care options is needed. A survey amongst patients with CF, their families and health carers was conducted to understand their attitudes and needs in relation to end-of-life care. The survey encompassed five separate domains, with a total of 60 questions requiring approximately 20 min to complete. Of the 200 surveys sent to patients, 82 (41%) completed responses were received. The Institutional Ethics Committee received six complaints from families of seven patients (3.5% of those surveyed). This article explores the nature of the adverse responses to the survey. The majority of complaints were received from family members rather than from patients. Complaints described dissatisfaction with the topic, little warning about the study and felt it to be inappropriate for their family member’s level of health. Survey instruments used to determine attitudes and needs in relation to end-of-life patient care are likely to elicit adverse responses that should be reported in a similar way to other investigational studies. Also arising from adverse responses and the complaint process, is the impact of criticism on study researchers.

WS09.5 Effect of ivacaftor on wellness, quality of life and cognitive function in adults with cystic fibrosis and G551D mutation

The effects of the potentiating agent ivacaftor on well-being and cognitive function are little known in adults with CF and G551D mutation. Objectives To evaluate the effects of ivacaftor on wellness, quality of life (QOL) and cognitive function in adults with CF; to evaluate a new wellness score for adults with CF. Methods In a placebo controlled crossover trial with 28 days of treatment in 20 adult patients the following were measured at 5 timepoints: The Cystic Fibrosis Questionnaire-Revised (CFQ-R), The Alfred Wellness Score for CF (AweScoreCF) and the Montreal Objective Cognitive Function Assessment (MOCA). Results See the table. In the CFQ-R change in social, health, physical, role and overall QOL improved significantly as did energy, exercise participation and total wellness in the AweScoreCF. There was strong agreement between the CFQ-R and the AweScoreCF. Patients found the AweScoreCF (10 questions) quicker to complete compared to the CFQ-R (50 questions). There were more completed AweScoreCF questionnaires than CFQ-R. There were some improvements in the MOCA in some patients following treatment. EndpointPercentage change from baseline, median (IQR)PlaceboIvacaftorCFQ-R18.1 (−30.8, 67.0)90.6 (41.7, 139.5) * AweScoreCF−0.5 (−6.9, 5.9)10.2 (3.8, 16.6) * MOCA0.1 (−0.7, 0.9)0.7 (−0.2, 1.5)*p Conclusions Treatment with ivacaftor resulted in significant improvements in quality of life, increased wellness, energy and exercise participation. There was strong agreement between the CFQ-R and AweScoreCF. The AweScoreCF may be a useful wellness assessment tool in the CF clinic. Study supported by Vertex Inc.

ePS03.4 Patient reported adherence to ivacaftor

Introduction The improvements in lung function, symptom burden and quality of life with the use of the gene potentiator ivacaftor in patients with G551D CF mutations have been established. Previous studies have suggested that adherence to medication in chronic illness is poor. Objectives To compare patient perception of adherence to ivacaftor to pharmacy dispensing data. Methods Twenty adult patients with at least one copy of the G551D mutation were recruited to a placebo-controlled trial. This consisted of 2 active phases during which participants were randomised to ivacaftor vs placebo. On completion all participants entered an open-label extension of 6 months. Medication returns within blister packs were examined following Active Phase 1 and Active Phase 2. During the Open Label extension pharmacy dispensing was recorded. Patients self-reported a percentage adherence during Active Phases 1 and 2 and also during the Open Label Extension. Results See the table. Adherence (%)Patient reportedActualActive Phase 196.797.6 a Active Phase 296.996.3Open Label Extension94.494.5aA 1% change in reported adherence is approximately equivalent to a 10 tablet difference for this study. Conclusion Patient-reported adherence was similar to medication return and dispensing data. Adherence when receiving open label medication declined slightly but remain higher than average rates in chronic illness. Supported by a grant from Vertex Inc.

ePS05.3 Ivacaftor and its effects on body composition in adults with G551D related cystic fibrosis

Background Ivacaftor, a potentiating agent has been shown to improve weight and BMI in patients with at least one copy of CFTR-G551D mutation. Effects on body composition are less well reported. Objectives To evaluate changes in body composition following ivacaftor therapy. Methods Twenty patients were included in a placebo-controlled crossover study of ivacaftor (28day treatment period), with 3 month open label extension. Bioelectrical impedance analysis (BIA) was conducted to measure weight, fat mass (FM), fat free mass (FFM), skeletal muscle mass (SMM) and total body water (TBW). Results Weight significantly increased above baseline at 1 month and post 3 months of ivacaftor treatment. FFM, SMM and TBW significantly increased following 1 month. FM did not significantly increase with 1 month of ivacaftor treatment but increased significantly above baseline post 3 months. Endpoint1 month (Crossover Study)+ 3 month Follow-Up (Open Label)% D Weight, kg1.6 (0.7, 2.5) *** 3.9 (2.4, 5.3) *** % D FM, kg0.9 (−2.4, 4.2)13.3 (5.0, 21.6) ** % D FFM, kg1.9 (0.9, 2.8) *** 0.6 (−1.9, 3.2)% D SMM, kg2.4 (0.6, 4.2) ** 1.6 (0.04, 3.2) * % D TBW, L2.0 (0.8, 3.2) *** 1.7 (0.3, 3.1) * Values are mean (95% CI).*P Conclusion The significant increase in weight is comparable to other seminal papers. The increase in FFM and SMM in the first 28days reflects an increase in TBW. Increase in FM did not occur until after 1month of treatment. These changes impact on nutrition practice. To our knowledge, this study is the first to investigate body composition with ivacaftor treatment using the BIA method. Validation with DEXA is underway. This study was supported by Vertex Inc.

WS18.4 Does the presence of diabetes affect lung function outcomes in a pregnant individual with cystic fibrosis

More women with CF now become pregnant. Clinicians and patients have many questions about the evolution and prognosis of these pregnancies. Aim: To describe evolution of pregnancies and clinical status of pregnant women in the French CF Registry. Methods: Data collected between 1992 and 2011 from women aged 15−49 registered in the Registry were used. For some incomplete data, only follow-up data collected between 2002 and 2011 were analyzed. Results: During those 20 years, 322 women became pregnant, for a total of 394 pregnancies (average number of pregnancies per woman: 1.2). The annual number of cases has increased (8 in 1992 to 48 in 2011). Age at first pregnancy remains steady (mean±SD: 26.8±5.4 years). Mean VEMS was 51.6% in 1992 and 71.1% in 2011 and is concordant with evolution of VEMS in CF adult women. BMI collected the year preceding pregnancy is constant over the period (mean±SD: 20.4±3). Between 2002 and 2011, the early pregnancy rate was 24.7‰; 72.5% of the women were PI and 22% had diabetes. In total, 35 deaths were registered, occurring 5±3.8 years after pregnancy on average. Mean age at death (31.4±8.3 years) exceeds that of the CF population as a whole (29 years in 2010). Interestingly, 24 women became pregnant after transplant (mean time to pregnancy after transplantation: 3.9±2.3 years). Conclusion: The annual number of pregnancies has increased dramatically over the last 20 years. The clinical status of pregnant women evolves in a similar manner to that of other adult women in the Registry. This work highlights the increasing number of pregnancies in transplanted patients and it will be interesting to study this specific population.