Luiza D. Campos, Brazil

Presenter of 1 Presentation

Poster Display Innate Immunity

LONG-TERM OUTCOMES FOR ADULTS WITH CHRONIC GRANULOMATOUS DISEASE (CGD) IN THE UNITED KINGDOM (UK)

Lecture Time
10:29 - 10:30
Room
Poster Area
Date
19.09.2019, Thursday
Session Time
10:00 - 17:00
Board Number
130
Presentation Topic
Innate Immunity

Abstract

Background and Aims

Life expectancy of CGD patients has improved but few data on its clinical course in adulthood are available. We studied long-term outcomes of uncorrected CGD adults in the UK.

Methods

Retrospective analysis of clinical records of adult CGD patients without prior corrective stem cell therapy from 8 centers across the UK.

Results

A total of 891 years of observation were collected (mean=17 years/patient) for 53 patients (mean age at symptom onset=6.2 years; 41 males: 12 females; 32:21 X-linked vs AR inheritance). 177 infectious events and 117 hospitalizations were recorded, of which skin and liver abscesses, and pulmonary infections were the most frequent. Active gastrointestinal inflammatory disease was present in 43% of adult patients, over a third of whom required major surgical intervention. CT chest scans were abnormal in 95%. Poor gas transfer was found in 62% of patients despite normal spirometry and positively correlated to bronchiectasis and inflammation on chest CT. Malignancy affected 4 patients, including 3 cases of bowel cancer. The mortality rate was 10%, predominantly related to chronic respiratory failure.

Conclusions

Survival to adulthood is frequently associated with the development of severe CGD complications and end-organ damage. Gastrointestinal and pulmonary complications were the major causes of serious morbidity and mortality in this study. These data inform counselling for pediatric and adult patients and families considering curative stem cell treatments for CGD.

Hide