Parameters to Assess Response to Intra-Venous Antibiotic Treatment for Pulmonary Exacerbations in Cystic Fibrosis

Belfast Health and Social Care Trust31 enrolled

Overview

Cystic fibrosis (CF) is a multisystem disease characterized by viscid secretions in multiple organ systems. Lung infection and damage account for most of the disease burden. Acute changes in respiratory signs and symptoms termed Pulmonary Exacerbations require treatment with intravenous antibiotics and hospital admission. These episodes cause substantial disruption to people's lives and impact on lung function, quality of life and lifespan. Current treatment regimes require improvement but further study is needed to identify who might benefit from a different approach. This observational study aims to assess if multi-dimensional measurements taken during treatment correspond with later treatment response. This may allow us to personalise treatment more effectively in the future and to better understand how individuals respond to treatment.

Pulmonary Exacerbations in CF require treatment with intravenous (IV) antibiotics and hospital admission for between 10-21 days. These episodes cause substantial disruption to people's lives and impact on lung function, quality of life and lifespan.Robust evidence to support current treatment approaches is lacking with awareness that current regimes could be optimised. As yet there is no model for predicting how patients with CF will respond to IV antibiotic treatment- other than clinical judgement and lung function response.This is due to lack of robust measures to identify clinical response at the time of treatment and safely predict later clinical outcomes. The heterogeneity of the 21st Century CF population means a multi-dimensional composite measure is needed. This study has therefore been designed to provide an overall picture of people's response including clinical, biochemical and patient related outcome measures. Using multi-dimensional assessment we hope the measures assessed in this study will give a better picture of how people feel and how they respond to treatment.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Cystic Fibrosis

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients attending the adult service with a confirmed diagnosis of CF by sweat test or genotype 2. Able to provide written informed consent 3. Able to understand and comply with protocol requirements and instructions 4. Confirmed Pulmonary Exacerbation requiring treatment with intravenous antibiotics using defined criteria (Physician and patient agreed need for admission for intravenous antibiotic treatment) Exclusion Criteria: 1. Previous recipient of a lung or other solid organ transplant 2. Inability to complete questionnaires 3. Current enrolment in other investigational medicinal product randomised trial 4. Admission for reasons other than pulmonary exacerbation of CF

Outcomes

Primary Outcomes

Forced Expiratory Volume in 1 second (FEV1)

Lung function Measure

Time frame: Change in lung function between baseline and day 0, Day 5 and Day 14 of treatment

Secondary Outcomes

Treatment Failure defined by need for further IV antibiotic treatment within 30 days

The number of participants requiring a further course of IV antibiotics within 30 days

Time frame: Need for a further course of IV antibiotics within 30 days of treatment completion

Change in Respiratory Symptoms

Change in Respiratory Symptoms assessed by CF-Respiratory Symptom Diary