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Open Access 01-12-2021 | Crohn's Disease | Case report

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

Authors: Alessandro Rossi, Erasmo Miele, Simona Fecarotta, Maria Veiga-da-Cunha, Massimo Martinelli, Carmine Mollica, Maria D’Armiento, Enza Mozzillo, Pietro Strisciuglio, Terry G. J. Derks, Annamaria Staiano, Giancarlo Parenti

Published in: Italian Journal of Pediatrics | Issue 1/2021

Abstract

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.

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Title: Crohn disease-like enterocolitis remission after empagliflozin treatment in a child with glycogen storage disease type Ib: a case report
Authors: Alessandro Rossi
Erasmo Miele
Simona Fecarotta
Maria Veiga-da-Cunha
Massimo Martinelli
Carmine Mollica
Maria D’Armiento
Enza Mozzillo
Pietro Strisciuglio
Terry G. J. Derks
Annamaria Staiano
Giancarlo Parenti
Publication date: 01-12-2021
Publisher: BioMed Central
Keywords: Crohn's Disease
Chronic Inflammatory Bowel Disease
Filgrastim
Neutropenia
Empagliflozin
Filgrastim
Published in: Italian Journal of Pediatrics / Issue 1/2021
Electronic ISSN: 1824-7288
DOI: https://doi.org/10.1186/s13052-021-01100-w

At a glance: The STEP trials

Springer Medicine

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

Abstract

Background

Case presentation

Conclusions

At a glance: The STEP trials

Springer Medicine

Abstract

Background

Case presentation

Conclusions

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