Safety and effectiveness of vedolizumab in paediatric patients with inflammatory bowel disease: an observational multicentre Spanish study
Por:
García-Romero R, Martinez de Zabarte Fernandez JM, Pujol G, Donat-Aliaga E, Segarra-Cantón O, Irastorza-Terradillos I, Medina-Benitez E, Ruiz-Hernández CJ, Carrillo-Palau M, Ros-Arnal I, Rodriguez-Martínez A, Escartin-Madurga L, Gutiérrez-Junquera C, Vicente-Santamaría S, Velasco Rodriguez-Belvis M, Fernández-Fernández S, Alberto-Alonso JR, Montraveta M, Torres-Peral R, Navalon-Rubio M, Navas-López VM and Martín-de-Carpi J
Publicada:
1 sep 2021
Ahead of Print:
1 abr 2021
Resumen:
Vedolizumab is a humanised monoclonal antibody that binds to integrin alpha 4 beta 7 expressed in T-cells, inhibiting its binding to the mucosal addressin cell adhesion molecule-1 (MAdCAM-1), which is specifically expressed in the small intestine and colon, playing a fundamental role in T-cell migration to the gastrointestinal tract. Vedolizumab has been shown to be effective in treating adults with inflammatory bowel disease; however, efficacy data for paediatric use are scarce. The objective of the present study was to assess the effectiveness and safety of vedolizumab for inducing and maintaining clinical remission in children with inflammatory bowel disease. We conducted a retrospective multicentre study of patients younger than 18 years with inflammatory bowel disease refractory to anti-tumour necrosis factor alpha (anti-TNF-alpha) drugs, who underwent treatment with vedolizumab. Clinical remission was defined as a score < 10 points in the activity indices. We included 42 patients, 22 of whom were male (52.3%), with a median age of 13.1 years (IQR 10.2-14.2) at the start of treatment. Of the 42 patients, 14 (33.3%) had Crohn's disease (CD) and 28 (66.7%) had ulcerative colitis (UC). At the start of treatment with vedolizumab, the Paediatric Crohn's Disease Activity Index was 36 (IQR 24-40) and the Paediatric Ulcerative Colitis Activity Index was 47 (IQR 25-65). All of them had received prior treatment with anti-TNF and 3 patients ustekinumab. At week 14, 69% of the patients responded to the treatment (57.1% of those with UC and 75% of those with UC; p=0.238), and 52.4% achieved remission (35.7% with CD and 60.7% with UC; p=0.126). At 30 weeks, the response rate was 66.7% (46.2% and 78.3% for CD and UC, respectively; p=0.049), and 52.8% achieved remission (30.8% and 65.2% for CD and UC, respectively; p=0.047). Among the patients with remission at week 14, 80% of the patients with CD and 84.5% of those with UC maintained the remission at 52 weeks. Adverse effects were uncommon and mild. Three patients (7.1%) presented headaches, 1 presented alopecia, 1 presented anaemia and 1 presented dermatitis.
Conclusion: The results show that treatment with vedolizumab is a safe and effective option for achieving clinical remission in paediatric patients with inflammatory bowel disease with primary failure or loss of response to other treatments, especially in UC.
What is Known: Vedolizumab is effective in inducing and maintaining remission in adult patients with inflammatory bowel disease.
Most studies and clinical trials have been performed on adult populations, and there is currently no indication for paediatric populations.
What is New: Children with inflammatory bowel disease refractory to anti-TNF presented higher clinical emission rates than those published for adults.
There are few publications of this magnitude on paediatric populations treated with vedolizumab and with long-term follow-up (52 weeks).
Filiaciones:
García-Romero R:
Paediatric Gastroenterology, Hepatology and Nutrition, Paediatric University Hospital Miguel Servet, Zaragoza, Spain.
Martinez de Zabarte Fernandez JM:
Paediatric Gastroenterology and Nutrition, Hospital Obispo Polanco, Teruel, Spain
Pujol G:
Paediatric Gastroenterology, Hepatology and Nutrition, Hospital Sant Joan de Déu, Barcelona, Spain
Donat-Aliaga E:
Paediatric Gastroenterology, Hepatology and Nutrition, Polytechnic University Hospital La Fe, Valencia, Spain
Segarra-Cantón O:
Paediatric Gastroenterology, Hepatology and Nutrition, Mother-Child University Hospital, Vall Hebrón, Barcelona, Spain
Irastorza-Terradillos I:
Paediatric Gastroenterology, Hepatology and Nutrition, University Hospital of Cruces, Barakaldo, Spain
Medina-Benitez E:
Paediatric Gastroenterology, Hepatology and Nutrition Unit, University Hospital 12 de Octubre, Madrid, Spain
Ruiz-Hernández CJ:
Paediatric Gastroenterology and Nutrition, Department of Paediatrics, Hospital Parc Taulí, Sabadell, Spain
Carrillo-Palau M:
Department of Gastroenterology, University Hospital of the Canary Islands, La Laguna, Tenerife, Spain
Ros-Arnal I:
Paediatric Gastroenterology, Hepatology and Nutrition, Paediatric University Hospital Miguel Servet, Zaragoza, Spain
Rodriguez-Martínez A:
Paediatric Gastroenterology, Hepatology and Nutrition, University Hospital Virgen del Rocío, Sevilla, Spain
Escartin-Madurga L:
Paediatric Gastroenterology, Hepatology and Nutrition, University Clinic Hospital Lozano Blesa, Zaragoza, Spain
Gutiérrez-Junquera C:
Paediatric Gastroenterology, Hepatology and Nutrition, Hospital Universitario Puerta de Hierro-Majadahonda, Madrid, Spain
Vicente-Santamaría S:
Paediatric Gastroenterology, Hepatology and Nutrition, University Hospital Ramon y Cajal, Madrid, Spain
Velasco Rodriguez-Belvis M:
Paediatric Gastroenterology, Hepatology and Nutrition, Hospital Niño Jesús, Madrid, Spain
Fernández-Fernández S:
Paediatric Gastroenterology, Hepatology and Nutrition, University Hospital Severo Ochoa, Leganés, Madrid, Spain
Alberto-Alonso JR:
Paediatric Gastroenterology, Hepatology and Nutrition, University Hospital Ntra. Sra. de Candelaria, Tenerife, Spain
Montraveta M:
Paediatric Gastroenterology, Hepatology and Nutrition, Hospital Germans Trias i Pujol, Badalona, Spain
Torres-Peral R:
Paediatric Gastroenterology and Nutrition Unit, Department of Paediatrics, University Hospital Complex, Salamanca, Spain
Navalon-Rubio M:
Paediatric Gastroenterology, Hepatology and Nutrition, Hospital Virgen de la Arrixaca, Murcia, Spain
Navas-López VM:
Paediatric Gastroenterology and Nutrition Section, Regional University Hospital of Malaga, Málaga, Spain
Martín-de-Carpi J:
Paediatric Gastroenterology, Hepatology and Nutrition, Hospital Sant Joan de Déu, Barcelona, Spain
Open Access
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