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Effect of early versus late azathioprine treatment in pediatric ulcerative colitis: Data from the Italian registry for pediatric IBD
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Abstracts / Digestive and Liver Disease 47S (2015) e237–e276
studies are limited and are not fully clarified the peculiarities of this association. We conducted a retrospective survey (2009–2015), enrolling patients with IBD PSC-associated (Group 1), in order to evaluate clinical, serological, and instrumental characteristics, therapeutic options, and outcome. Data were compared with those with IBD without hepatobiliary involvement (Group 2). Sixteen (13.9%) out of 115 pts (71 UC, 44 CD) showed hepatobiliary disease (11 PSC, 5 Overlap Syndrome). There were no significant differences for gender, age at diagnosis of IBD, family history of IBD, duration of follow-up, presence of other EIM, time between symptoms onset and diagnosis of IBD. GI symptoms were similar, even if in 2 pts with PSC, diagnosis of IBD was performed for screening. Eight pts were asymptomatic at diagnosis of PSC. Ulcerative colitis (UC) (p 0.004) with pancolic extension (p < 0.001) was predominant in Group 1; there were no differences for back-wash ileitis or rectal sparing. The presence of p-ANCA has been found as independent factor associated with PSC (p < 0.000001). No patient in Group 1 underwent surgery; treatment with immunosuppressors has been used predominantly in this group. The PSC is a heterogeneous disease with a prevalence >10% in our population. Pancolitis and p-ANCA positivity were the main IBD features significantly associated with PSC. Close monitoring is necessary even in asymptomatic patients and the control of hepatobiliary disease seems to result in a better outcome. http://dx.doi.org/10.1016/j.dld.2015.07.099 P052 MCCUNE ALBRIGHT SYNDROME AND NEONATAL CHOLESTASIS: A NEW ASSOCIATION D. Liccardo 1,∗ , A. Pietrobattista 1 , M. Candusso 1 , S. Cardile 1 , M.S. Basso 1 , R. De Vito 2 , M. Iascone 3 , M. Riminucci 4 , G. Torre 1 1
Unit of Hepatology, Gastronterology and Nutrition, Bambino Gesù Children’s Hospital, IRCCS, Rome, Italy 2 Pathology Unit, “Bambino Gesù” Children’s Hospital, IRCCS, Rome, Italy 3 Molecular Genetics Laboratory, UO Anatomia Patologica, Ospedali Riuniti di Bergamo, Bergamo, Italy 4 Department of Molecular Medicine, Sapienza University of Rome, Rome, Italy Background: McCune Albright syndrome is characterized by a classic triade: polyostotic fibrous dysplasia, cafe-au-lait spots, and sexual precocity. Hepatobiliary dysfunction is considered a nonendocrine manifestation. The activating mutation has been identified in the gene for the alpha subunit of Gs protein. This molecular defect may play a role in the pathogenesis of cholestasis, possibly by interfering with normal biliary excretion by the liver cell. Patients: A male infant with neonatal cholestasis (total bilirubin 15.33 mg/dl; conjugated bilirubin 11.75 mg/dl; AST 124 U/L, ALT 94 U/L) with high gGt refers to our Unit. He presented hepatomegaly with normal size of spleen. He showed no peculiar phenotype, and the most common causes of cholestasis were ruled out. Liver biopsy showed idiopathic neonatal cholestasis with mild portal inflammatory infiltrate. During the follow up jaundice subsided spontaneously. From the 14 months of life he presented welldefined cafe-au-lait spots on dorsal regions and arms, pruritis and poor growth. Successively he showed lameness in full health. A complete X-ray film and after bone biopsy showed lesions of polyostotic fibrous dysplasia most marked in many body segments. The McCune-Albright syndrome was suspected and mutations in the gene for the alpha subunit of Gs protein were identified in both lymphocytes and liver tissue (R201H). The child is now 2 years
old and has no endocrinological dysfunction. Liver function tests remain abnormal. Conclusion: Neonatal cholestasis rarely may be a first manifestation of McCune Albright syndrome and it can be considered in differential diagnosis of chronic cholestasis of infant and children. http://dx.doi.org/10.1016/j.dld.2015.07.100 P053 EFFECT OF EARLY VERSUS LATE AZATHIOPRINE TREATMENT IN PEDIATRIC ULCERATIVE COLITIS: DATA FROM THE ITALIAN REGISTRY FOR PEDIATRIC IBD G. D’Arcangelo 1,∗ , M. Bramuzzo 2 , M. Gasparetto 3 , M. Martinelli 4 , P. Alvisi 5 , M.T. Illiceto 6 , S. Valenti 7 , S. Pellegrino 8 , C. Catassi 9 , S. Arrigo 10 , S. Martelossi 2 , S. Cucchiara 1 , M. Aloi 1 1 Pediatric Gastroenterology and Liver Unit, Sapienza University of Rome, Rome, Italy 2 Pediatric Gastroenterology, Institute for Maternal and Child Health IRCCS “Burlo Garofalo”, Trieste, Italy 3 Pediatric Gastroenterology, University of Padua, Padua, Italy 4 Pediatric Gastroenterology, University of Naples “Federico II”, Naples, Italy 5 Pediatric Department, Maggiore Hospital, Bologna, Italy 6 Pediatric Gastroenterology and Endoscopy Unit, Spirito Santo Hospital, Pescara 7 Pediatric Gastroenterology and Endoscopy, University of Messina, Messina, Italy 8 Pediatric Gastroenterology and Cystic Fibrosis, University of Messina, Messina, Italy 9 Department of Pediatrics, Università Politecnica delle Marche, Ancona, Italy 10 Paediatric Gastroenterology Unit, Institute “Giannina Gaslini”, Genoa, Italy
Aims: To describe the efficacy of AZA in newly diagnosed pediatric UC, comparing the outcomes of “early” (0–6 months) versus “late” (6–12 months) therapy. Methods: Data were collected from the SIGENP registry. Corticosteroid (CS) free remission at 12 months was the primary outcome. Secondary outcomes were mucosal healing (MH), need for treatment escalation, therapy-related adverse events (AE) and need for surgery at 12 and 24-month follow-up. Results: Of 401 children with UC, 95 were included for efficacy analyses (mean age 10.7 ± 3.8 years, 59% females). Fifty-four patients (57%) were in the early and 41 (43%) in the late group. Twenty-seven (51%) of early patients were in CS-free remission at 1 year, compared to 24 (61.5%) of late ones (p = 0.39). Mucosal healing was achieved by 32% of early versus 45% of late group (p = 0.45). Serious AE occurred in 3 patients (2 fungal pneumonia, 1 pancreatitis), 2 in the early and 1 in the late group. Overall, mild side effects were recorded in 16 patients (17%; 5 leucopenia, 11 pancreatic enzyme elevation): 10 in the early and 6 in the late group (p = 0.78), 3 requiring AZA discontinuation. No difference was found for need of treatment escalation, use of infliximab and rate of surgery over time. Conclusion: Introduction of AZA within 6 months of diagnosis is not more effective than later treatment. Mucosal healing is not related to the timing of AZA initiation. Serious AE are uncommon. http://dx.doi.org/10.1016/j.dld.2015.07.101
Abstracts / Digestive and Liver Disease 47S (2015) e237–e276
studies are limited and are not fully clarified the peculiarities of this association. We conducted a retrospective survey (2009–2015), enrolling patients with IBD PSC-associated (Group 1), in order to evaluate clinical, serological, and instrumental characteristics, therapeutic options, and outcome. Data were compared with those with IBD without hepatobiliary involvement (Group 2). Sixteen (13.9%) out of 115 pts (71 UC, 44 CD) showed hepatobiliary disease (11 PSC, 5 Overlap Syndrome). There were no significant differences for gender, age at diagnosis of IBD, family history of IBD, duration of follow-up, presence of other EIM, time between symptoms onset and diagnosis of IBD. GI symptoms were similar, even if in 2 pts with PSC, diagnosis of IBD was performed for screening. Eight pts were asymptomatic at diagnosis of PSC. Ulcerative colitis (UC) (p 0.004) with pancolic extension (p < 0.001) was predominant in Group 1; there were no differences for back-wash ileitis or rectal sparing. The presence of p-ANCA has been found as independent factor associated with PSC (p < 0.000001). No patient in Group 1 underwent surgery; treatment with immunosuppressors has been used predominantly in this group. The PSC is a heterogeneous disease with a prevalence >10% in our population. Pancolitis and p-ANCA positivity were the main IBD features significantly associated with PSC. Close monitoring is necessary even in asymptomatic patients and the control of hepatobiliary disease seems to result in a better outcome. http://dx.doi.org/10.1016/j.dld.2015.07.099 P052 MCCUNE ALBRIGHT SYNDROME AND NEONATAL CHOLESTASIS: A NEW ASSOCIATION D. Liccardo 1,∗ , A. Pietrobattista 1 , M. Candusso 1 , S. Cardile 1 , M.S. Basso 1 , R. De Vito 2 , M. Iascone 3 , M. Riminucci 4 , G. Torre 1 1
Unit of Hepatology, Gastronterology and Nutrition, Bambino Gesù Children’s Hospital, IRCCS, Rome, Italy 2 Pathology Unit, “Bambino Gesù” Children’s Hospital, IRCCS, Rome, Italy 3 Molecular Genetics Laboratory, UO Anatomia Patologica, Ospedali Riuniti di Bergamo, Bergamo, Italy 4 Department of Molecular Medicine, Sapienza University of Rome, Rome, Italy Background: McCune Albright syndrome is characterized by a classic triade: polyostotic fibrous dysplasia, cafe-au-lait spots, and sexual precocity. Hepatobiliary dysfunction is considered a nonendocrine manifestation. The activating mutation has been identified in the gene for the alpha subunit of Gs protein. This molecular defect may play a role in the pathogenesis of cholestasis, possibly by interfering with normal biliary excretion by the liver cell. Patients: A male infant with neonatal cholestasis (total bilirubin 15.33 mg/dl; conjugated bilirubin 11.75 mg/dl; AST 124 U/L, ALT 94 U/L) with high gGt refers to our Unit. He presented hepatomegaly with normal size of spleen. He showed no peculiar phenotype, and the most common causes of cholestasis were ruled out. Liver biopsy showed idiopathic neonatal cholestasis with mild portal inflammatory infiltrate. During the follow up jaundice subsided spontaneously. From the 14 months of life he presented welldefined cafe-au-lait spots on dorsal regions and arms, pruritis and poor growth. Successively he showed lameness in full health. A complete X-ray film and after bone biopsy showed lesions of polyostotic fibrous dysplasia most marked in many body segments. The McCune-Albright syndrome was suspected and mutations in the gene for the alpha subunit of Gs protein were identified in both lymphocytes and liver tissue (R201H). The child is now 2 years
old and has no endocrinological dysfunction. Liver function tests remain abnormal. Conclusion: Neonatal cholestasis rarely may be a first manifestation of McCune Albright syndrome and it can be considered in differential diagnosis of chronic cholestasis of infant and children. http://dx.doi.org/10.1016/j.dld.2015.07.100 P053 EFFECT OF EARLY VERSUS LATE AZATHIOPRINE TREATMENT IN PEDIATRIC ULCERATIVE COLITIS: DATA FROM THE ITALIAN REGISTRY FOR PEDIATRIC IBD G. D’Arcangelo 1,∗ , M. Bramuzzo 2 , M. Gasparetto 3 , M. Martinelli 4 , P. Alvisi 5 , M.T. Illiceto 6 , S. Valenti 7 , S. Pellegrino 8 , C. Catassi 9 , S. Arrigo 10 , S. Martelossi 2 , S. Cucchiara 1 , M. Aloi 1 1 Pediatric Gastroenterology and Liver Unit, Sapienza University of Rome, Rome, Italy 2 Pediatric Gastroenterology, Institute for Maternal and Child Health IRCCS “Burlo Garofalo”, Trieste, Italy 3 Pediatric Gastroenterology, University of Padua, Padua, Italy 4 Pediatric Gastroenterology, University of Naples “Federico II”, Naples, Italy 5 Pediatric Department, Maggiore Hospital, Bologna, Italy 6 Pediatric Gastroenterology and Endoscopy Unit, Spirito Santo Hospital, Pescara 7 Pediatric Gastroenterology and Endoscopy, University of Messina, Messina, Italy 8 Pediatric Gastroenterology and Cystic Fibrosis, University of Messina, Messina, Italy 9 Department of Pediatrics, Università Politecnica delle Marche, Ancona, Italy 10 Paediatric Gastroenterology Unit, Institute “Giannina Gaslini”, Genoa, Italy
Aims: To describe the efficacy of AZA in newly diagnosed pediatric UC, comparing the outcomes of “early” (0–6 months) versus “late” (6–12 months) therapy. Methods: Data were collected from the SIGENP registry. Corticosteroid (CS) free remission at 12 months was the primary outcome. Secondary outcomes were mucosal healing (MH), need for treatment escalation, therapy-related adverse events (AE) and need for surgery at 12 and 24-month follow-up. Results: Of 401 children with UC, 95 were included for efficacy analyses (mean age 10.7 ± 3.8 years, 59% females). Fifty-four patients (57%) were in the early and 41 (43%) in the late group. Twenty-seven (51%) of early patients were in CS-free remission at 1 year, compared to 24 (61.5%) of late ones (p = 0.39). Mucosal healing was achieved by 32% of early versus 45% of late group (p = 0.45). Serious AE occurred in 3 patients (2 fungal pneumonia, 1 pancreatitis), 2 in the early and 1 in the late group. Overall, mild side effects were recorded in 16 patients (17%; 5 leucopenia, 11 pancreatic enzyme elevation): 10 in the early and 6 in the late group (p = 0.78), 3 requiring AZA discontinuation. No difference was found for need of treatment escalation, use of infliximab and rate of surgery over time. Conclusion: Introduction of AZA within 6 months of diagnosis is not more effective than later treatment. Mucosal healing is not related to the timing of AZA initiation. Serious AE are uncommon. http://dx.doi.org/10.1016/j.dld.2015.07.101