Emerging treatments
Gene therapy
One of the challenges of gene therapy is achieving adequate transduction efficiency in haematopoietic stem cells carrying the corrected gene. Marrow conditioning, in which resident haematopoietic stem cells are eliminated to allow a selective survival advantage of infused gene-corrected stem cells, is considered necessary. A few patients treated in early trials experienced side effects arising from insertional mutagenesis. Recent trials have been more encouraging. After 12 months, six out of nine patients with X-linked CGD treated with lentiviral gene therapy demonstrated stable vector copy numbers, 16% to 46% normal neutrophils, no new CGD complications, and were able to discontinue antibiotic prophylaxis.[90]
Other novel therapies
Other described therapies include pioglitazone as an adjunct in refractory infection, which might improve mitochondrial reactive oxygen species production and efferocytosis; however, one trial was terminated early due to lack of efficacy.[91][92][93] Novel anti-inflammatory treatments have also been used successfully, including thalidomide and anakinra.[94] Vedolizumab, a monoclonal antibody that binds to integrin receptors, may be useful for the treatment of chronic granulomatous disease-associated fistulating colitis; it resulted in limited, short-lived improvement in one observational study.[95][96] None of these treatments have been formally investigated yet and it is too early to recommend them for routine clinical practice.
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