Cinzia Dedieu, Germany
Charité Universitätsklinikum Berlin Pediatric Immunology and pneumologyPresenter of 1 Presentation
CONVENTIONAL TREATMENT VERSUS STEM CELL TRANSPLANTATION – OUTCOME IN 104 PATIENTS WITH CHRONIC GRANULOMATOUS DISEASE
Abstract
Background and Aims
Recurrent infections and non-infectious inflammation still limit quality of life and life expectancy in Chronic Granulomatous Disease (CGD). Continuous anti-infectious prophylaxis and immunosuppression (Conventional treatment (CT)) improve the prognosis but only stem cell transplantation (SCT) can cure the disease. However, it remains debated whether all patients with CGD benefit from SCT, whether active infections and non-infectious inflammation are still risk factors and at what age SCT should be performed.
Methods
We describe 104 patients with CGD, recruited from 6 participating centers and in collaboration with the ESID registry. 50 of whom underwent SCT and 54 remained on CT. We compared incidence and frequency of infections, non-infectious inflammation, hospitalizations, operations, and immune modulating therapy.
Results
With CT 84,6% of patients suffered from at least one infection, 75% from non-infectious inflammation. Patients on CT developed infection/inflammation or needed hospitalization/surgery at a median of 1,87 per year (IQR [0,94-4,24]), versus 7 (IQR [5-10,9]) in the first year after SCT but none of the patients undergoing SCT developed severe complications after the initial year following SCT (IQR [0-0,53]). Complications decreased (p <.05) and growth improved significantly after SCT (weight (p.017), height (p.029)). Nevertheless, complications post-SCT remain frequent: 8% had extended chronic GvHD, 16% initial graft failure, 12% died. Age > 8 years at SCT and active complications at SCT coincided with an increased risk for complications.
Conclusions
Infections, non-infectious inflammation and failure to thrive occur in all patients with CGD on CT. SCT can cure the disease, but age and disease activity must be considered in an individualized risk/benefit assessment.