Haematopoietic Stem Cell Transplant for Norovirus-Induced Intestinal Failure in X-linked Agammaglobulinemia.

Since the first clinical description in 1952, immunoglobulin replacement therapy remains the mainstay of treatment of patients with X-linked agammaglobulinemia (XLA). However, this therapy only replaces IgG isotype and does not compensate for the loss of Bruton tyrosine kinase in non-B-lymphocytes. Patients may still therefore develop complications despite current standard of care.

Use of Infliximab Biosimilar Versus Originator in a Pediatric United Kingdom Inflammatory Bowel Disease Induction Cohort.

Objectives: The aim of the study was to summarize short-term effectiveness, safety, and cost of using infliximab biosimilar (IFX-B) drugs, (Inflectra [Hospira] and Remsima [NAAP]) compared to originator infliximab (IFX-O) (Remicade [MSD]) in biologic naive pediatric inflammatory bowel disease in the United Kingdom.

Methods: Prospective audit of patients starting anti-tumour necrosis factor (TNF) therapy. Disease severity, response to treatment, and remission rate was measured by Pediatric Crohn's Disease Activity Index (PCDAI) and/or Physician Global Assessment.

Segmental dilatation of small bowel presenting with severe anaemia: a diagnostic puzzle.

A 7-year-old boy presented to Paediatric outpatient with worsening lethargy and tiredness. On examination he had extreme pallor. Blood investigations confirmed severe iron deficiency anaemia.

Real-life Anti-tumor Necrosis Factor Experience in More Than 500 Patients: High Co-immunosuppression Rates But Low Rates of Quantifying Treatment Response.

Objective: The aim of this study was to measure the effectiveness, safety, and use of anti-tumor necrosis Factor (TNF) therapy in pediatric inflammatory bowel disease in the United Kingdom (UK).

Methods: Prospective UK audit of patients newly starting anti-TNF therapy. Disease severity was assessed using Physician Global Assessment +/or the Paediatric Crohn Disease Activity Index.

Contemporary outcomes for ulcerative colitis inpatients admitted to pediatric hospitals in the United Kingdom.

Background: Pediatric ulcerative colitis (UC) care is variable with a lack of appropriate guidelines to guide practice until recently.

Methods: UC inpatients <17 years old admitted to 23 U.K.

Ramos-Arroyo syndrome: confirmation of an entity.

In 1987, Ramos-Arroyo et al. described a family with a previously unreported combination of features, which included corneal anesthesia, short stature, sensorineural deafness, learning difficulties, and a characteristic facial appearance. The family was reviewed in 2008 and additional features were noted.

Coeliac disease presenting as chronic cough in an 8-year-old child.

Patients with the 'classical' gastrointestinal symptoms of coeliac disease (CD) are usually readily diagnosed and treated with a gluten exclusion diet. However, the advent of sensitive serological investigations has revealed that over half of childhood CD remains asymptomatic or presents with more subtle non-gastrointestinal symptoms, with a significant risk to the health of undiagnosed children. In view of its changing presentation there is an increasing need for clinicians to have a low threshold to investigate patients for this disease.