P351. Long term treatment with autologous red blood cells loaded with dexamethasone 21-phosphate in pediatric patients affected by steroid-dependent Crohn's disease: In the era of biologics can steroids be reconsidered?
B. Papadatou1, D. Knafelz1, F. Bracci1, A. Diamanti1, M. Gambarara1, F. Ferretti1, F. Panetta1, G. Torre1
1Ospedale Bambino Gesù, Hepatogastroenterology, Roma, Italy
Background: Corticosteroids have an important role in the therapy of IBD to induct remission in patients with moderate to severe Crohn's Disease (CD). The use of the steroids for the maintenance of the remission facilitates the outbreak of adverse events and is not recommended, particularly in children. On the other hand intolerance to or toxicity from immunosuppressive agents (6-MP/AZA) used as steroid-sparing agents in the maintenance of the remission, occurs in 10–15% of patients.
Biologic therapy offers an important alternative treatment for induction and maintenance of the remission, but serious adverse events, opportunistic infections and malignancies have been reported. Overall a safe and effective long term strategy in the treatment of paediatric CD is still not available.
In a previous pilot study we demonstrated the efficacy and safety of the treatment with periodic infusions of autologous erythrocytes (RBCs) loaded with dexamethasone 21-phospate (Dex 21‑P) in steroid dependent paediatric patients. We continued this treatment in the patients who met the inclusion criteria and we report our 6 years experience of its use.
Methods: We treated 16 patients with corticodependent Crohn' disease (8 M, 8 F), mean age at beginning of treatment 15 years (8–22 years). The mean duration of the treatment was 6 years (9–84 months). Monthly the Paediatric Crohn Disease Activity Index (pCDAI) was calcolated. Bone mineral density (BMD) and body mass index were assessed before the start of treatment and every year; in all patients glycaemia and morning cortisol have been periodically controlled. Endoscopic and histological evaluations were also performed at 12 months and then every two years.
Results: Eight patients (50%) gained clinical remission (pCDAI values below 10); eight patients (50%), initially partial responders to treatment relapsed after a mean time of 23 months (range 9–48 months) and switched to biological therapy. Our data confirm the very long term (up to 6 years) efficacy and safety of periodic infusions of autologous RBCs loaded with Dex 21‑P in the maintenance of remission in the 50% of the 16 patients who continued the study with steroids dependent CD.
Conclusions: Even if there is no evidence that this treatment changes the natural history of the disease, our long term follow up confirmed that is safe, improves the quality of life preventing frequent relapses and hospitalizations and helps to delay more aggressive forms of therapy that sometimes could not be avoided.