"Il faut continuer à poser des questions" patient reported outcome measures in cystic fibrosis: An anthropological perspective.

BACKGROUND: People with cystic fibrosis (pwCF) are central in the development of patient-led assessment tools. Qualitative analysis of a frequently used CF-specific patient-reported outcome measure (PROM) sought patient recommendations for development of a new quality of life (QoL) tool. METHODS: We performed an inventory of PROMs, symptom-report and QoL tools used in clinical trials within the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) and in routine clinical practice among Cystic Fibrosis Europe and ECFS members. A qualitative study using cognitive interviews with pwCF and their caregivers reviewed the Cystic Fibrosis Questionnaire (CFQ), the French initial form of the Cystic Fibrosis Questionnaire-Revised (CFQ-R). RESULTS: Survey results from 33 countries revealed over 70 tools used in routine clinical practice, utilized by clinical specialists (n=124), pwCF/parents/carers (n=49) and other allied health professionals (n=60). The CFQ-R was the main PROM used in clinical trials. The qualitative study enrolled 99 pwCF, 6 to 11 years (n=31); 12 to 18 years (n=38); >18 years (n=30) and 26 parents. Inductive thematic analysis based on the CFQ, revealed 19 key themes. Themes common across all cohorts included burden of treatment, impact of disease on day-to-day life, relationships/family, stress/mood, and nutrition. Themes unique to individual groups included, treatment when not symptomatic for the paediatric group; education/studies and planning for the future for adolescents, impact of anxiety and depression on day-to-day life for adults, and for parents, questions addressing anxiety and their role as carers. CONCLUSIONS: Patient-centeredness is paramount in development of an up-to-date PROM in the era of novel therapies.

[Family support upon immediate cystic fibrosis announcement]. / L'accompagnement des familles à l'annonce de la mucoviscidose.

Cystic fibrosis, a rare, hereditary and chronic disease, affects the psychic functioning of children and their families. Since it is incurable, constant adjustments are essential to make sense of the inevitable evolution of the disease. The psychologists support the families and the patients so that all live as well as possible with cystic fibrosis.

ECFS best practice guidelines: the 2018 revision.

Developments in managing CF continue to drive dramatic improvements in survival. As newborn screening rolls-out across Europe, CF centres are increasingly caring for cohorts of patients who have minimal lung disease on diagnosis. With the introduction of mutation-specific therapies and the prospect of truly personalised medicine, patients have the potential to enjoy good quality of life in adulthood with ever-increasing life expectancy. The landmark Standards of Care published in 2005 set out what high quality CF care is and how it can be delivered throughout Europe. This underwent a fundamental re-write in 2014, resulting in three documents; center framework, quality management and best practice guidelines. This document is a revision of the latter, updating standards for best practice in key aspects of CF care, in the context of a fast-moving and dynamic field. In continuing to give a broad overview of the standards expected for newborn screening, diagnosis, preventative treatment of lung disease, nutrition, complications, transplant/end of life care and psychological support, this consensus on best practice is expected to prove useful to clinical teams both in countries where CF care is developing and those with established CF centres. The document is an ECFS product and endorsed by the CF Network in ERN LUNG and CF Europe.

Pain is an underestimated symptom in cystic fibrosis.

PURPOSE OF REVIEW: Life expectancy is increasing in cystic fibrosis and new aspects of the disease have to be taken into account in cystic fibrosis care. RECENT FINDINGS: Pain is encountered among 70% of adult and pediatric patients with cystic fibrosis. This symptom is underestimated by the multidisciplinary team. It has been reported as impacting quality of life and adherence to treatments. The location of pain is inconstant among the different studies but the major symptoms are headaches, gastrointestinal, and chest pain. Pain is different for each patient and requires careful evaluation using questionnaires some of which specifically developed for patients with cystic fibrosis. Medical and nonmedical treatment such as ostheopathy or sophrology may relieve pain symptoms but have to be adjusted in the frame of a global personalized care. SUMMARY: Pain maybe an underestimated symptom among patients with cystic fibrosis and impacts negatively on quality of life. VIDEO ABSTRACT.

[Physical activity and exercise training for patients with cystic fibrosis]. / Activité physique et réentraînement à l'effort du patient atteint de mucoviscidose.

In France patients with cystic fibrosis benefit from a multidisciplinary follow-up in Cystic Fibrosis Centres. In this follow-up, despite the numerous therapeutic benefits of exercise in this disease, little emphasis is placed on the promotion of physical activity. The aim of this article is to improve this aspect of management, giving advice from a working group of experts, based on the medical literature and clinical experience. These proposals include quantification of physical activity, evaluation of exercise, training and rehabilitation programs and finally, modification of behaviour to include physical activity in the overall cystic fibrosis treatment strategy. It is intended to set up multicentre studies to evaluate the impact of these proposals.

Pain in children and adults with cystic fibrosis: a comparative study.

Pain is a potential complication of cystic fibrosis (CF), but its consequences in daily life and other issues of pain management are not yet clearly understood. We undertook a comparative study of children and adults with CF to assess the prevalence of pain symptoms, their characteristics and treatment, their impact on daily quality of life, and the occurrence of procedural pain. The study included 73 children (1-18 years) and 110 adults (18-52 years); 59% of the children and 89% of the adults reported at least one episode of pain during the previous month. Pain was significantly more intense and lasted significantly longer among adults, but its rate and recurrence did not differ significantly between the two populations and were not related to the severity of CF. The most prevalent locations were the abdomen for children, and the back, head, and chest for adults. Although pain significantly limited physical activity, only 15% of patients reported that it caused absenteeism, and 27% reported that it negatively affected their family life. The mean pain intensity rates on a visual analog scale for the episode that had caused the greatest pain during the past month were 4.9 (2) (mean [SD]) for children and 6 (2) for adults; however, only 40% and 50%, respectively, of those with pain reported the use of analgesic treatment, mainly paracetamol (acetaminophen). At least one episode of procedural pain during the previous month was reported by 85% of children and 78% of adults. Our study demonstrates the high incidence of undertreated pain in CF patients throughout their lives.