Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis

Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR modulators, the first variant-specific therapy available, have transformed the management of CF.The latest standards of care from the European CF Society (2018) did not include guidance on variant-specific therapy, as CFTR modulators were becoming established as a novel therapy. We have produced interim standards to guide healthcare professionals in the provision of variant-specific therapy for people with CF.Here we provide evidence-based guidance covering the spectrum of care, established using evidence from systematic reviews and expert opinion. Statements were reviewed by key stakeholders using Delphi methodology, with agreement (≥80%) achieved for all statements after one round of consultation. Issues around accessibility are discussed and there is clear consensus that all eligible people with CF should have access to variant-specific therapy

Medication optimization in older adults with advanced cancer and a limited life expectancy:A prospective observational study

Introduction: Polypharmacy is common in older adults with cancer and is associated with drug related problems (DRPs) and potentially inappropriate medication (PIM). We introduced a medication optimization care pathway for older adults with advanced cancer and a limited life expectancy and studied the prevalence of DRPs and PIMs as well as the adherence to medication-related recommendations and the patient satisfaction. Materials and Methods: A medication review was performed in patients aged ≥65 years with polypharmacy and a life expectancy of &lt;24 months. Recommendations on adjustments of medication were discussed in a multidisciplinary team including a pharmacist, an oncologist, and a geriatrician. Implementation of the recommendations was left to the discretion of the oncologist. Four weeks after the implementation, the patient filled a questionnaire to assess satisfaction. Results: One hundred twenty patients were included. The mean age was 75 years and 39% were female. A mean of 12 medications was used. The median number of DRP was 6.0 per patient and median number of PIMs was 3.0 per patient. Overtreatment accounted for 26% of DRP and the most frequently involved drug classes were antihypertensive medication (22%), non-opioid analgesics (22%), and antilipemics (12%). The multidisciplinary team accepted 78% of the recommendations of the pharmacist and the oncologist implemented 54% of the recommendations. Overall, patients were satisfied or very satisfied with the intervention. Discussion: DRPs and PIMs are highly prevalent in this population and can be reduced by a multidisciplinary medication optimization intervention. Patients appreciate the medication optimization intervention and are satisfied with the intervention.</p

Use of potentially inappropriate medication in older patients with lung cancer at the end of life

Objectives: Medications at the end of life should be used for symptom control. Medications which potential adverse effects outweigh their expected benefits are called ‘potentially inappropriate medications’ (PIMs). PIMs are related with adverse drug events and reduced quality of life. In this study, we investigated to what extent PIMs are dispensed to older patients with lung cancer in the last month of life. Methods: We selected patients with lung cancer, aged 65+, diagnosed between 2009 and 2014, and who died before April 1st 2015 from the population-based Netherlands Cancer Registry (NCR). The NCR is linked to the PHARMO Database Network, that includes medications dispensed by community pharmacies in the Netherlands. The eight PIM groups were based on the OncPal Deprescribing Guideline: aspirin, dyslipidaemia medications, antihypertensives, osteoporosis medications, peptic ulcer prophylaxis, oral hypoglycaemics, vitamins and minerals. Results: Data of 7864 patients with lung cancer were analyzed. Median age was 74 year (IQR = 70–79) and 67% was male. 45% of all patients received at least one PIM in their last month of life. Taking into account all dispensed medications, patients receiving PIMs received more different medications compared to those receiving no PIMs, respectively 10 (SD = 5) vs. 3 (SD = 4) different medications (P < 0.001). Conclusion: Almost half of the older patients with lung cancer in the Netherlands received PIMs in their last month of life. Since PIM use is associated with reduced quality of life, it is important that health care professionals continue to critically assess which medication can be discontinued at the end of life

Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis

Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR modulators, the first variant-specific therapy available, have transformed the management of CF. The latest standards of care from the European CF Society (2018) did not include guidance on variant-specific therapy, as CFTR modulators were becoming established as a novel therapy. We have produced interim standards to guide healthcare professionals in the provision of variant-specific therapy for people with CF. Here we provide evidence-based guidance covering the spectrum of care, established using evidence from systematic reviews and expert opinion. Statements were reviewed by key stakeholders using Delphi methodology, with agreement (≥80%) achieved for all statements after one round of consultation. Issues around accessibility are discussed and there is clear consensus that all eligible people with CF should have access to variant-specific therapy

Standards for the care of people with cystic fibrosis (CF); recognising and addressing CF health issues.

This is the third in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF. This paper focuses on recognising and addressing CF health issues. The guidance was produced with wide stakeholder engagement, including people from the CF community, using an evidence-based framework. Authors contributed sections, and summary statements which were reviewed by a Delphi consultation. Monitoring and treating airway infection, inflammation and pulmonary exacerbations remains important, despite the widespread availability of CFTR modulators and their accompanying health improvements. Extrapulmonary CF-specific health issues persist, such as diabetes, liver disease, bone disease, stones and other renal issues, and intestinal obstruction. These health issues require multidisciplinary care with input from the relevant specialists. Cancer is more common in people with CF compared to the general population, and requires regular screening. The CF life journey requires mental and emotional adaptation to psychosocial and physical challenges, with support from the CF team and the CF psychologist. This is particularly important when life gets challenging, with disease progression requiring increased treatments, breathing support and potentially transplantation. Planning for end of life remains a necessary aspect of care and should be discussed openly, honestly, with sensitivity and compassion for the person with CF and their family. CF teams should proactively recognise and address CF-specific health issues, and support mental and emotional wellbeing while accompanying people with CF and their families on their life journey