Managing Inflammatory Manifestations in Patients with Chronic Granulomatous Disease
- 14 June 2016
- journal article
- therapy in-practice
- Published by Springer Science and Business Media LLC in Pediatric Drugs
- Vol. 18 (5), 335-345
- https://doi.org/10.1007/s40272-016-0182-4
Abstract
Chronic granulomatous disease (CGD) is a primary immunodeficiency caused by lack of phagocyte nicotinamide adenine dinucleotide phosphate (NADPH) oxidase, which results in inflammatory dysregulation and increased susceptibility to infections. Patients with CGD may develop severe obstructive disorders of the digestive tract as a result of their dysregulated inflammatory response. Despite a growing focus on inflammatory manifestations in CGD, the literature data on obstructive complications are far less extensive than those on infectious complications. Diagnosis and management of patients with concomitant predispositions to infections and hyperinflammation are particularly challenging. Although the inflammatory and granulomatous manifestations of CGD usually respond rapidly to steroid treatment, second-line therapies (immunosuppressants and biologics) may be required in refractory cases. Indeed, immunosuppressants (such as anti-tumor necrosis factor agents, thalidomide, and anakinra) have shown some efficacy, but the value of this approach is controversial, given the questionable risk-to-benefit ratio and the small numbers of patients treated to date. Significant progress in allogeneic hematopoietic stem cell transplantation (the only curative treatment for CGD) has been made through better supportive care and implementation of improved, reduced-intensity conditioning regimens. Gene therapy may eventually be an option for patients lacking a suitable donor; clinical trials with new, safer vectors are ongoing at a few centers.Keywords
This publication has 100 references indexed in Scilit:
- Corticosteroid Therapy for Liver Abscess in Chronic Granulomatous DiseaseClinical Infectious Diseases, 2011
- Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II TrialMolecular Therapy, 2011
- Chronic granulomatous disease: Overview and hematopoietic stem cell transplantationJournal of Allergy and Clinical Immunology, 2011
- Gene Therapy of Chronic Granulomatous Disease: The Engraftment DilemmaMolecular Therapy, 2011
- Residual NADPH Oxidase and Survival in Chronic Granulomatous DiseaseThe New England Journal of Medicine, 2010
- Complications of Tumor Necrosis Factor–α Blockade in Chronic Granulomatous Disease–Related ColitisClinical Infectious Diseases, 2010
- Reactive oxygen species–independent activation of the IL-1β inflammasome in cells from patients with chronic granulomatous diseaseProceedings of the National Academy of Sciences of the United States of America, 2010
- Twenty-Year Follow-Up of Esophageal Involvement in Chronic Granulomatous DiseaseThe American Journal of Gastroenterology, 2009
- Chronic granulomatous disease as a risk factor for autoimmune diseaseJournal of Allergy and Clinical Immunology, 2008
- Inflammatory Manifestations in Chronic Granulomatous Disease (CGD)Journal of Clinical Immunology, 2008