Cellular Therapies in Chronic Granulomatous Disease

Frontiers in Pediatrics
Tayfun Güngör, Robert Chiesa

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) has become the main curative treatment in patients with chronic granulomatous disease (CGD). CGD is caused by inherited defects of the phagolysomal NADPH-oxidase, leading to a lifelong propensity for invasive infections and granulomatous inflammation. After successful allogeneic HSCT, chronic infections and inflammation resolve and quality-of-life improves. Favorable long-term outcome after HSCT is dependent on the prevention of primary and secondary graft failure (GF), including falling myeloid donor chimerism (DC) below 10 %, and chronic graft-vs.-host-disease (cGVHD). The risk of GF and GvHD increases with the use of HLA-incompatible donors and this may outweigh the benefits of HSCT, mainly in patients with severe co-morbidities and in asymptomatic patients with residual NADPH-oxidase function. Seventeen scientific papers have reported on a total of 386 CGD-patients treated by HSCT with HLA-matched family/sibling (MFD/MSD), 9/10-/10/10-matched-unrelated volunteer (MUD) and cord blood donors. The median OS/EFS-rate of these 17 studies was 91 and 82%, respectively. The median rates of GF, cGVHD and de-novo autoimmune diseases were 14, 10, and 12%, respectively. Results ...Continue Reading

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Citations

Oct 23, 2020·Journal of Clinical Immunology·Maleewan KitcharoensakkulMary Dinauer
Feb 6, 2021·Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis·David G Justus, John P Manis
Jun 8, 2021·The Pediatric Infectious Disease Journal·Cinzia DedieuHorst von Bernuth
Jul 7, 2021·Bone Marrow Transplantation·A C LankesterUNKNOWN Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immune

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Methods Mentioned

BETA
biopsies
bronchoalveolar lavage
MDS

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