Long-term outcomes for adults with chronic granulomatous disease in the United Kingdom

To the Editor: Chronic granulomatous disease (CGD) is an inherited primary immunodeficiency caused by genetic defects that impact the structural subunits or function of the nicotinamide adenine dinucleotide phosphate oxidase complex. Consequent reduction in respiratory burst impairs phagocyte function, causing granulomatous inflammation and recurrent life-threatening bacterial and fungal infections.1-3 Although clinically variable with respect to presentation and disease severity,1,4 improvement in life expectancy now allows most patients to reach adulthood even without corrective therapy. In the few large multicenter studies published, data for adults have mainly been combined with pediatric data1-6 and the only targeted study of adult CGD outcomes reported high levels of morbidity and early mortality.7 Because improved survival following corrective hematopoietic stem cell transplantation (HSCT) has expanded this option for treating both asymptomatic children and symptomatic adults with CGD, accurate data for outcomes and quality of life for conservatively managed CGD in adulthood are urgently required to improve counseling for patients and families considering curative treatment. Seventy percent of patients had at least 1 hospital admission during the follow-up period (see Fig E1 in this article’s Online Repository at www.jacionline.org), with no correlation between the number of hospital admissions and the duration of follow-up or genetic type of CGD (P > .05).