Safety and effectiveness of vedolizumab in paediatric patients with inflammatory bowel disease: an observational multicentre Spanish study.

Vedolizumab is a humanised monoclonal antibody that binds to integrin α4ß7 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-α) 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 CD 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 remission 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).

The role of endoscopy in caustic ingestion in the pediatric population: experience in a tertiary center.

INTRODUCTION: caustic ingestion in children is rare but has potentially serious consequences. AIM: to analyze the clinical and endoscopic features and the type of caustic ingested in our population. METHODS: the upper endoscopies performed in this setting, as well as the characteristics of patients and caustics, were analyzed from 2010 to 2018. RESULTS: fifty-one endoscopies were performed (48 cases of witnessed intake or high suspicion and three with a low suspicion) in patients with a mean age of 2.55 years. Alkali ingestion was more frequent (88.2 %) and 56.9 % of the endoscopies were normal, which was more frequent among those who ingested bleach (72 %). Alkali tended to produce more esophageal injuries (31.1 %) and acids tended to produce esophageal (20 %) and esophageal-gastric injuries (20 %). Four patients developed esophageal stenosis during follow-up. DISCUSSION: even though more than half of the studies were normal, endoscopy is important in the diagnosis and prognosis of these patients.

Efectividad y seguridad en nuestro entorno de adalimumab como tratamiento anti-TNF de primera linea en niños con enfermedad de Crohn / A real-world study focused on the effectiveness and safety of adalimumab as first-line anti-TNF treatment for pediatric Crohn's disease

Introducción y objetivos: Adalimumab (ADA), anticuerpo anti-TNF-α monoclonal recombinante de origen humano, generalmente se emplea como tratamiento de segunda línea en niños con enfermedad de Crohn (EC) que no han respondido o han perdido respuesta a infliximab (IFX). En las series publicadas más del 70% de los pacientes habían sido tratados inicialmente con IFX. Los datos sobre la eficacia a corto y a largo plazo de ADA en pacientes naïve a anti-TNF son muy limitados. El objetivo del presente estudio es describir nuestra experiencia con ADA como tratamiento anti-TNF de primera línea en niños con EC. Material y método: Estudio multicéntrico, retrospectivo que incluye pacientes con EC tratados con ADA como anti-TNF de primera línea. Resultados: Se incluyeron 62 pacientes (34 varones) con una edad media de 13,0 ± 2,4años, un tiempo de evolución de la enfermedad de 7,3 meses (RIQ 2,7-21) y un wPCDAI de 35 puntos (RIQ 24,3-47,5). En el momento de comenzar ADA, 58 pacientes (93,5%) estaban recibiendo tratamiento inmunomodulador. A las 12 semanas de tratamiento el 80,6% (50/62) habían alcanzado la remisión clínica, así como el 95% (57/60) a las 52 semanas. Ocho pacientes (13%) presentaron efectos adversos. Se constató un incremento significativo de los z-scores de talla, velocidad de crecimiento e índice de masa corporal (IMC) a las 52 semanas de tratamiento, en especial en aquellos con retraso de crecimiento. Conclusiones: El tratamiento con ADA favorece una remisión clínica prolongada en pacientes naïve a anti-TNF. El tratamiento con ADA mejora la velocidad de crecimiento en niños con EC y retraso de crecimiento al inicio del tratamiento (AU)

Background and objectives: Adalimumab (ADA), a monoclonal humanised anti-TNF antibody, is usually prescribed as a second-line treatment in paediatric Crohn's disease (CD) patients who have become unresponsive or developed intolerance to infliximab (IFX). In the case series reported, more than 70% of patients had initially been treated with IFX. Data on short- and long-term effectiveness of ADA in anti-TNF naïve patients is limited. The aim of this study is to describe our experience with ADA as a first-line anti-TNF in paediatric CD patients. Material and methods: This is a multicentre retrospective study including anti-TNF naïve paediatric CD patients treated with ADA as first-line anti-TNF. Results: Sixty-two patients (34 males), with a mean age of 13.0 ± 2.4years and a disease duration of 7.3 (IQR 2.7-21) months were included. Median wPCDAI was 35 (IQR 24.3-47.5). Fifty-eight out of 62 (93.5%) were on combo therapy at baseline. Clinical remission at week 12 was achieved in 50 out of 62 (80.6%) and in 57 out of 60 (95.0%) at week 52. Eight patients (13%) reported adverse events. Mean height, growth rate and BMI z-scores improved significantly between baseline and week 52, especially in patients with growth failure. Conclusions: ADA treatment leads to lasting clinical remission in anti-TNF naïve paediatric patients with CD. ADA significantly improved growth rate in children with CD who had growth delay at baseline (AU)

[A real-world study focused on the effectiveness and safety of adalimumab as first-line anti-TNF treatment for pediatric Crohn's disease]. / Efectividad y seguridad en nuestro entorno de adalimumab como tratamiento anti-TNF de primera linea en niños con enfermedad de Crohn.

BACKGROUND AND OBJECTIVES: Adalimumab (ADA), a monoclonal humanised anti-TNF antibody, is usually prescribed as a second-line treatment in paediatric Crohn's disease (CD) patients who have become unresponsive or developed intolerance to infliximab (IFX). In the case series reported, more than 70% of patients had initially been treated with IFX. Data on short- and long-term effectiveness of ADA in anti-TNF naïve patients is limited. The aim of this study is to describe our experience with ADA as a first-line anti-TNF in paediatric CD patients. MATERIAL AND METHODS: This is a multicentre retrospective study including anti-TNF naïve paediatric CD patients treated with ADA as first-line anti-TNF. RESULTS: Sixty-two patients (34males), with a mean age of 13.0±2.4years and a disease duration of 7.3 (IQR 2.7-21) months were included. Median wPCDAI was 35 (IQR 24.3-47.5). Fifty-eight out of 62 (93.5%) were on combo therapy at baseline. Clinical remission at week12 was achieved in 50 out of 62 (80.6%) and in 57 out of 60 (95.0%) at week52. Eight patients (13%) reported adverse events. Mean height, growth rate and BMI z-scores improved significantly between baseline and week 52, especially in patients with growth failure. CONCLUSIONS: ADA treatment leads to lasting clinical remission in anti-TNF naïve paediatric patients with CD. ADA significantly improved growth rate in children with CD who had growth delay at baseline.

Manejo de las estenosis esofágicas refractarias en edad pediátrica / Management of refractory esophageal stenosis in the pediatric age

Introducción: las estenosis esofágicas refractarias (EER) a las dilataciones suponen un importante problema de salud en la pediatría. Se han descrito técnicas alternativas como la colocación de prótesis y el uso de mitomicina C (MC). Presentamos nuestra experiencia en una serie consecutiva con ambas técnicas, en nuestro caso con prótesis biodegradables (PB) y a veces ambas técnicas asociadas. Material y métodos: se incluyeron 6 pacientes, dos con fístula postquirúrgica en atresias esofágicas tipo 1 operadas, otro con atresia operada sin fístula y otros tres con estenosis caústicas. Se colocaron 5 PB en 4 niños, 3 en atresias operadas (en un paciente 2 prótesis) y 1 en estenosis. Dos de los niños con atresia tenían fístula asociada. Se utilizó MC en 5 casos, en dos de forma primaria y en tres tras recidiva al fracasar la prótesis. Resultados: las PB colocadas en las fístulas fueron recubiertas y en un caso solucionaron la fístula y en otro no. En las estenosis fueron efectivas en solo una (con la fístula no resuelta), recidivando en 2. La MC solucionó uno de estos casos, debiendo ponerse una segunda prótesis en el otro paciente. En los otros dos (una atresia y otra por caústicos), la MC fue efectiva, solucionando las estenosis tras 1 y 2 sesiones respectivamente. En el cómputo total hemos solucionado las estenosis en 5 de 6 estenosis refractarias (83,3%) y en 1 de 2 fístulas (50%). Conclusiones: la asociación de prótesis biodegradables y mitomicina ha sido efectiva en el rescate de algunos casos de estenosis esofágicas refractarias en niños, teniendo un papel secundario en la resolución de fístulas (AU)

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Management of refractory esophageal stenosis in the pediatric age.

INTRODUCTION: Refractory esophageal stenosis (RES) is a major health problem in the pediatric population. Several techniques such as stent placement or C-mitomycin (CM) have been described as alternative treatments. We present our experience with both techniques, in our case with biodegradable stents (BS) and sometimes the association with stents and CM. MATERIAL AND METHODS: Six patients have been included: 2 post-operative fistulas in patients with type I esophageal atresia; 1 operated atresia without fistula; and 3 caustic strictures. 5 BS were placed in 4 children: 3 of them in cases of atresia (2 prosthesis in one case) and the other one in a case of stricture. CM was used in 5 cases: in 2 of them from the beginning, and in the other 3 cases after failure of the stent. RESULTS: When placed in fistulas, BS were fully covered. One of them successfully treated the fistula, but the other one was not effective. One stenosis was successfully treated with SB (in the case of persistent fistula), but recurrence was observed in the other 2 cases. One of these was solved with CM, and the other one needed a second stent. In the remaining 2 cases (one atresia and one caustic stricture) CM was effective after 1 and 2 sessions respectively. Overall, 5 out of 6 stenosis have been successfully treated (83.3%), and 1 out of 2 fistulas (50%). CONCLUSIONS: Association of BS and CM has been effective in the management of RES in children.

Direct percutaneous endoscopic jejunostomy in pediatric age with a pexies triangulation system: report of a case and literature review.

Direct percutaneous endoscopic jejunostomy (DPEJ) is an infrequent procedure as it is not always easy to obtain transillumination, being this the main reason for failure of this technique. In patients with previous surgery, this procedure is more complex and there are only 6 reported cases in pediatric population. In our case, we provide the use of an endoscopic triangulation system with "T" pexies not used before in these cases. With this technique, we guarantee not to replace the introduction system afterwards, obtaining the placement of a balloon fixation system initially. We also provide several improvements that helped us developing the procedure: Placement of the jejunostomy through a gastrostomy; use of water column to avoid penetration in hollow viscus; use of a guide wire, and a triangulation pexy system.

Yeyunostomía endoscópica directa en edad pediátrica mediante sistema de triangulación (PEXIA): aportación de un caso y revisión de la literatura / Direct percutaneous endoscopic jejunostomy in pediatric age with a pexies triangulation system: Report of a case and literature review

La yeyunostomía endoscópica directa es una técnica infrecuente pues no siempre es fácil conseguir la transiluminación, motivo por el que se fracasa en su colocación con mayor frecuencia. En pacientes con cirugía previa aún es más compleja, y en la edad pediátrica sólo hay 6 casos publicados en la literatura. Además se aporta la utilización de un sistema de triangulación con pexias en T que no se ha utilizado hasta ahora en esta indicación. De esta forma nos aseguramos el no tener que retirar posteriormente el sistema de introducción, dejando desde el principio un sistema de fijación con balón. Aportamos varias mejoras que nos ayudaron en la técnica: realización a través de la gastrostomía, uso de columna de agua para evitar paso por vísceras huecas, uso de hilo guía y sistema de pexia-triangulación (AU)

Direct percutaneous endoscopic jejunostomy (DPEJ) is an infrequent procedure as it is not always easy to obtain transillumination, being this the main reason for failure of this technique. In patients with previous surgery, this procedure is more complex and there are only 6 reported cases in pediatric population. In our case, we provide the use of an endoscopic triangulation system with "T" pexies not used before in these cases. With this technique, we guarantee not to replace the introduction system afterwards, obtaining the placement of a balloon fixation system initially. We also provide several improvements that helped us developing the procedure: Placement of the jejunostomy through a gastrostomy; use of water column to avoid penetration in hollow viscus; use of a guide wire, and a triangulation pexy system