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Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report

BACKGROUND: Besides major clinical/biochemical features, neutropenia and inflammatory bowel disease (IBD) constitute common complications of Glycogen storage disease type Ib (GSD Ib). However, their management is still challenging. Although previous reports have shown benefit of empagliflozin admini...

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Autores principales: Rossi, Alessandro, Miele, Erasmo, Fecarotta, Simona, Veiga-da-Cunha, Maria, Martinelli, Massimo, Mollica, Carmine, D’Armiento, Maria, Mozzillo, Enza, Strisciuglio, Pietro, Derks, Terry G. J., Staiano, Annamaria, Parenti, Giancarlo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8254289/
https://www.ncbi.nlm.nih.gov/pubmed/34215305
http://dx.doi.org/10.1186/s13052-021-01100-w
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author Rossi, Alessandro
Miele, Erasmo
Fecarotta, Simona
Veiga-da-Cunha, Maria
Martinelli, Massimo
Mollica, Carmine
D’Armiento, Maria
Mozzillo, Enza
Strisciuglio, Pietro
Derks, Terry G. J.
Staiano, Annamaria
Parenti, Giancarlo
author_facet Rossi, Alessandro
Miele, Erasmo
Fecarotta, Simona
Veiga-da-Cunha, Maria
Martinelli, Massimo
Mollica, Carmine
D’Armiento, Maria
Mozzillo, Enza
Strisciuglio, Pietro
Derks, Terry G. J.
Staiano, Annamaria
Parenti, Giancarlo
author_sort Rossi, Alessandro
collection PubMed
description BACKGROUND: Besides major clinical/biochemical features, neutropenia and inflammatory bowel disease (IBD) constitute common complications of Glycogen storage disease type Ib (GSD Ib). However, their management is still challenging. Although previous reports have shown benefit of empagliflozin administration on neutropenia, no follow-up data on bowel (macro/microscopic) morphology are available. We herein present for the first time longitudinal assessment of bowel morphology in a GSD Ib child suffering from Crohn disease-like enterocolitis treated with empagliflozin. CASE PRESENTATION: A 14-year-old boy with GSD Ib and severe IBD was (off-label) treated with empagliflozin (20 mg/day) after informed oral and written consent was obtained from the patient’s parents. No adverse events were noted. Clinical symptoms and stool frequency improved within the first week of treatment. Pediatric Crohn disease activity index (PCDAI) normalised within the first month of treatment. Abdomen magnetic resonance imaging (MRI) performed 3 months after treatment initiation showed dramatic decrease in disease activity and length. Similar findings were reported on histology at 5.5 months. At 7.5 months hemoglobin levels normalised and fecal calprotectin almost normalised. Improved neutrophil count, metabolic control and quality of life were also noted. G-CSF dose was decreased by 33% and the patient was partly weaned from tube feeding. CONCLUSIONS: This is the first report presenting extensive gastrointestinal morphology follow-up in a GSD Ib patient receiving empagliflozin. The present case suggests that empagliflozin can be safe and effective in inducing IBD remission in GSD Ib patients and can even postpone surgery. Future studies are required to confirm its effect over time and assess its benefit in various disease stages. The development of an international collaborating networks for systematic data collection is worthy. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13052-021-01100-w.
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spelling pubmed-82542892021-07-06 Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report Rossi, Alessandro Miele, Erasmo Fecarotta, Simona Veiga-da-Cunha, Maria Martinelli, Massimo Mollica, Carmine D’Armiento, Maria Mozzillo, Enza Strisciuglio, Pietro Derks, Terry G. J. Staiano, Annamaria Parenti, Giancarlo Ital J Pediatr Case Report BACKGROUND: Besides major clinical/biochemical features, neutropenia and inflammatory bowel disease (IBD) constitute common complications of Glycogen storage disease type Ib (GSD Ib). However, their management is still challenging. Although previous reports have shown benefit of empagliflozin administration on neutropenia, no follow-up data on bowel (macro/microscopic) morphology are available. We herein present for the first time longitudinal assessment of bowel morphology in a GSD Ib child suffering from Crohn disease-like enterocolitis treated with empagliflozin. CASE PRESENTATION: A 14-year-old boy with GSD Ib and severe IBD was (off-label) treated with empagliflozin (20 mg/day) after informed oral and written consent was obtained from the patient’s parents. No adverse events were noted. Clinical symptoms and stool frequency improved within the first week of treatment. Pediatric Crohn disease activity index (PCDAI) normalised within the first month of treatment. Abdomen magnetic resonance imaging (MRI) performed 3 months after treatment initiation showed dramatic decrease in disease activity and length. Similar findings were reported on histology at 5.5 months. At 7.5 months hemoglobin levels normalised and fecal calprotectin almost normalised. Improved neutrophil count, metabolic control and quality of life were also noted. G-CSF dose was decreased by 33% and the patient was partly weaned from tube feeding. CONCLUSIONS: This is the first report presenting extensive gastrointestinal morphology follow-up in a GSD Ib patient receiving empagliflozin. The present case suggests that empagliflozin can be safe and effective in inducing IBD remission in GSD Ib patients and can even postpone surgery. Future studies are required to confirm its effect over time and assess its benefit in various disease stages. The development of an international collaborating networks for systematic data collection is worthy. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13052-021-01100-w. BioMed Central 2021-07-02 /pmc/articles/PMC8254289/ /pubmed/34215305 http://dx.doi.org/10.1186/s13052-021-01100-w Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Case Report
Rossi, Alessandro
Miele, Erasmo
Fecarotta, Simona
Veiga-da-Cunha, Maria
Martinelli, Massimo
Mollica, Carmine
D’Armiento, Maria
Mozzillo, Enza
Strisciuglio, Pietro
Derks, Terry G. J.
Staiano, Annamaria
Parenti, Giancarlo
Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
title Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
title_full Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
title_fullStr Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
title_full_unstemmed Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
title_short Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
title_sort crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type ib: a case report
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8254289/
https://www.ncbi.nlm.nih.gov/pubmed/34215305
http://dx.doi.org/10.1186/s13052-021-01100-w
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