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BACKGROUND: Administration of live vaccines following liver transplant (LT) has historically not been recommended due to concerns regarding risk of vaccine-attenuated disease. However, there is evidence suggesting that in select transplant recipients live vaccinations can be administered safely. Studies in other regions have indicated that despite this evidence many clinicians remain hesitant to administer live vaccinations. METHOD: A REDCap survey was distributed to gastroenterologists, pediatricians, and infectious diseases physicians at pediatric centers across Australia and New Zealand via email between September and November 2023. The survey included a series of questions regarding live vaccine and varicella postexposure prophylaxis (PEP) practices in pediatric LT recipients and barriers to live vaccine administration in this cohort. RESULTS: There was a total of 16 responses to the survey, from 10 different pediatric centers, including 10/11 pediatric gastroenterology centers and all four pediatric LT centers in the region. Only 31% (5/16) of respondents (from 3/10 different centers) offer live vaccines. The main barrier to live vaccine administration was clinician reluctance and the main reason for not offering live vaccines was insufficient safety data. Sixty-nine percent (11/16) of respondents take vaccination status and/or serology into account when deciding whether to offer varicella PEP to this cohort. Respondents universally offer varicella zoster immunoglobulin as PEP, though 31% (5/16) also offer antiviral medication. CONCLUSIONS: Many clinicians in our region remain hesitant to provide live vaccines to pediatric LT recipients, with concerns regarding insufficient safety data. Updated local guidelines may help to address this.
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Varicela , Transplante de Fígado , Profilaxia Pós-Exposição , Padrões de Prática Médica , Humanos , Austrália , Nova Zelândia , Varicela/prevenção & controle , Profilaxia Pós-Exposição/métodos , Criança , Padrões de Prática Médica/estatística & dados numéricos , Vacina contra Varicela/administração & dosagem , Vacina contra Varicela/uso terapêutico , Vacinas Atenuadas/administração & dosagem , Vacinas Atenuadas/uso terapêutico , Inquéritos e QuestionáriosRESUMO
AIMS: The New Zealand National Intestinal Failure and Rehabilitation Service (NZ-NIFRS) was established in October 2015 to gather longitudinal data on the aetiology, clinical course and outcomes of children with intestinal failure (IF). One main objective is to achieve health equity for patients with IF in NZ. METHODS: Clinical outcomes (enteral autonomy, parenteral nutrition (PN) dependence, death or intestinal transplantation) for IF patients diagnosed from October 2015 to 2018 were analysed; comparisons were made by ethnicity and socio-economic status (SES) using published 'prioritised-ethnicity' health data and the NZ index of deprivation, respectively. The Cox proportional-hazards model was used to assess time to enteral autonomy. RESULTS: Of the 208 patients (55.77% male, 43.75% preterm), 170 (81.73%) achieved enteral autonomy and 14 (6.73%) remained PN dependent. Pacific and Maori children accounted for 12.98% and 27.88% of the patient cohort, respectively, compared to 9.46% and 25.65% of the NZ paediatric population. More significantly, IF patients with a high NZ socio-economic deprivation score were overrepresented, with 35.92% in the highest deprivation quintile and 10.19% in the least deprived quintile, compared to 23.53% and 20.31%, respectively, of the NZ paediatric population. There were no significant differences in primary clinical outcomes for any patients based on ethnicity or SES. CONCLUSION: While disparities in ethnicity and social deprivation do exist in the incidence of IF in NZ children, clinical outcomes are similar for children regardless of ethnicity or SES. NZ-NIFRS has achieved one of its core objectives: to achieve health equity for all patients with IF nationwide.
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Insuficiência Intestinal , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Etnicidade , Disparidades nos Níveis de Saúde , Disparidades em Assistência à Saúde/etnologia , Incidência , Nova Zelândia/epidemiologia , Classe Social , Fatores Socioeconômicos , População das Ilhas do Pacífico , Povo MaoriRESUMO
BACKGROUND: Achievement of enteral autonomy (EA) is the ultimate treatment goal in pediatric intestinal failure (IF). We aimed to assess predictors of EA in pediatric short bowel syndrome (SBS) and explore the impact of residual small bowel (SB) and large bowel (LB) length on EA. METHODS: A retrospective cohort study was performed on infants aged <12 months (n = 367, six centers) with SBS referred between 2010 and 2015. The cohort was stratified based on the achievement of EA. Statistical testing was completed using t-test, chi-square, Cox proportional hazards regression model, and Kaplan-Meier analysis. RESULTS: EA was achieved in 229 patients. In the multivariable analysis, the percentage of residual LB (hazard ratio [HR] = 1.02; 95% CI = 1.01-1.02) and SB (HR = 1.01; 95% CI = 1.01-1.02) length, presence of the ileocecal valve (HR = 2.02; 95% CI=1.41-2.88), and not coming from a high-volume transplantation center (HR = 2.42; 95% CI = 1.68-3.49) were positively associated with EA, whereas a negative association was seen with the presence of stoma at the time when shortest remnant was documented (HR = 0.72; 95% CI = 0.52-1.00). EA achievement was significantly different between the anatomical subgroups (log-rank test P < 0.001) with an EA rate of 80.4% in infants with ≥50% SB and LB (median time 209 days); 62.5% with ≥50% SB and <50% LB (397 days); 58.3% with <50% SB and ≥50% LB (1192 days), and 25.9% with <50% SB and LB. Necrotizing enterocolitis (NEC) was not associated with a better achievement of EA (NEC vs other etiologies: log-rank test P = 0.33). CONCLUSIONS: Overall, 62% of infants with IF secondary to SBS achieved EA over a mean time of follow-up of 2.3 years. A colon length of >50% can compensate for the loss of small bowel (<50%) and account for similar EA rates as those in children with residual SB > 50%.
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Insuficiência Intestinal , Síndrome do Intestino Curto , Lactente , Humanos , Recém-Nascido , Criança , Síndrome do Intestino Curto/terapia , Estudos Retrospectivos , Nutrição Parenteral , Intestino DelgadoRESUMO
BACKGROUND AND AIMS: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study. APPROACH AND RESULTS: Maralixibat trials comprise 84 patients with ALGS with up to 6 years of treatment. GALA contains retrospective data from 1438 participants. GALA was filtered to align with key maralixibat eligibility criteria, yielding 469 participants. Serum bile acids could not be included in the GALA filtering criteria as these are not routinely performed in clinical practice. Index time was determined through maximum likelihood estimation in an effort to align the disease severity between the two cohorts with the initiation of maralixibat. Event-free survival, defined as the time to first event of manifestations of portal hypertension (variceal bleeding, ascites requiring therapy), surgical biliary diversion, liver transplant, or death, was analyzed by Cox proportional hazards methods. Sensitivity analyses and adjustments for covariates were applied. Age, total bilirubin, gamma-glutamyl transferase, and alanine aminotransferase were balanced between groups with no statistical differences. Event-free survival in the maralixibat cohort was significantly better than the GALA cohort (HR, 0.305; 95% CI, 0.189-0.491; p <0.0001). Multiple sensitivity and subgroup analyses (including serum bile acid availability) showed similar findings. CONCLUSIONS: This study demonstrates a novel application of a robust statistical method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible, providing wide application for rare diseases. This comparison with real-world natural history data suggests that maralixibat improves event-free survival in patients with ALGS.
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Síndrome de Alagille , Humanos , Síndrome de Alagille/complicações , Síndrome de Alagille/tratamento farmacológico , Feminino , Masculino , Estudos Retrospectivos , Criança , Lactente , Pré-Escolar , Intervalo Livre de Progressão , Adolescente , Proteínas de Transporte , Glicoproteínas de MembranaRESUMO
OBJECTIVES: High rates of inflammatory bowel disease (IBD) are reported in children of South Asian (SA) descent in some western countries. This population-based study describes the incidence and clinical course of IBD in SA children compared to non-South Asian (NSA) children in New Zealand (NZ). METHODS: Children (≤15 years) with new-onset IBD presenting to a centralized tertiary referral center in Auckland, NZ from 2010 to 2020 were identified. Disease phenotype, clinical characteristics, response to exclusive enteral nutrition, clinical remission rates at 3 and 12 months, biologic use, corticosteroid exposure, and disease complications were compared by ethnicity; IBD incidence was calculated. RESULTS: There were 127 (26 SA; 101 NSA) children with Crohn disease, 41 (10 SA; 31 NSA) with ulcerative colitis, and 10 (3 SA; 7 NSA) with IBD-unclassified. IBD incidence in SA and NSA children was 14.1 per 100,000 and 4.3 per 100,000 respectively ( P < 0.001). IBD incidence increased by 5.6% per year ( P = 0.022), due to a greater rise in incidence in SA (SA 16.8% per year, P = 0.015; NSA 4.5% per year, P = 0.317). At presentation, SA children had worse biochemical parameters, severe colitis, and vitamin D deficiency. SA children had lower rates of remission following exclusive enteral nutrition (28.5% vs 65.0%, P < 0.001) or biologic induction (35.7% vs 70.8%, P = 0.020), at 3-month (35.3% vs 69.8%, P < 0.001) and 12-month follow-up (29.4% vs 55.0%, P = 0.005). No significant differences were found in disease location or corticosteroid burden. CONCLUSIONS: Increasing incidence of IBD was disproportionately represented by SA children with more severe disease and lower remission rates following exclusive enteral nutrition or biologic therapy.
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Produtos Biológicos , Colite Ulcerativa , Doenças Inflamatórias Intestinais , Humanos , Estudos Retrospectivos , Incidência , Nova Zelândia/epidemiologia , Doenças Inflamatórias Intestinais/terapia , Doenças Inflamatórias Intestinais/tratamento farmacológico , Colite Ulcerativa/epidemiologia , Colite Ulcerativa/terapia , Colite Ulcerativa/complicações , Corticosteroides/uso terapêutico , Produtos Biológicos/uso terapêuticoRESUMO
AIM: There are increasing reports of atopy/allergy following solid organ transplantation, especially paediatric liver transplantation (LT) with minimal New Zealand (NZ) data. We describe the prevalence of transplant-acquired atopy and allergy (TAA) in NZ paediatric liver transplant recipients, compared to paediatric kidney and adult liver transplants. METHODS: TAA focussed health questionnaires were sent to patients selected from the NZ transplant registry (transplanted between January 2003 and December 2017). Demographic and clinical data were also obtained from electronic health records and follow-up phone calls. RESULTS: A total of 232 patients (62% male) participated (111 adult liver, 82 paediatric liver, 39 paediatric kidney transplant recipients). Tacrolimus was primary immunosuppression for all LT patients; with combined tacrolimus, mycophenolate and corticosteroids for kidney transplants. The number of patients who developed TAA was significantly higher (P < 0.001) in the paediatric LT group (36/82, 44%) compared to adult liver (12/111, 11%) and paediatric kidney transplants (4/39, 10%). Eczema was most common (73%), then IgE-mediated food allergy (FA, 33%), allergic rhinitis (19%) and asthma (17%). Six paediatric LT recipients developed eosinophilic oesophagitis (EoE). Egg was the most common allergen in the IgE-mediated FA group. TAAs were severe enough to warrant a switch from tacrolimus to another agent in seven paediatric LT patients. For paediatric LT patients, female gender and younger age at transplant were risk factors for developing TAA. CONCLUSIONS: TAA is common in paediatric liver transplant recipients, with female gender and younger age at transplant being risk factors identified. This highlights the need for detailed atopic and allergy history to be incorporated in all pre-transplant assessments.
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Hipersensibilidade Alimentar , Hipersensibilidade Imediata , Transplante de Órgãos , Adulto , Criança , Humanos , Masculino , Feminino , Tacrolimo/efeitos adversos , Hipersensibilidade Imediata/epidemiologia , Hipersensibilidade Imediata/etiologia , Transplante de Órgãos/efeitos adversos , Hipersensibilidade Alimentar/epidemiologia , Imunoglobulina ERESUMO
BACKGROUND AND AIMS: Alagille syndrome (ALGS) is a multisystem disorder, characterized by cholestasis. Existing outcome data are largely derived from tertiary centers, and real-world data are lacking. This study aimed to elucidate the natural history of liver disease in a contemporary, international cohort of children with ALGS. APPROACH AND RESULTS: This was a multicenter retrospective study of children with a clinically and/or genetically confirmed ALGS diagnosis, born between January 1997 and August 2019. Native liver survival (NLS) and event-free survival rates were assessed. Cox models were constructed to identify early biochemical predictors of clinically evident portal hypertension (CEPH) and NLS. In total, 1433 children (57% male) from 67 centers in 29 countries were included. The 10 and 18-year NLS rates were 54.4% and 40.3%. By 10 and 18 years, 51.5% and 66.0% of children with ALGS experienced ≥1 adverse liver-related event (CEPH, transplant, or death). Children (>6 and ≤12 months) with median total bilirubin (TB) levels between ≥5.0 and <10.0 mg/dl had a 4.1-fold (95% confidence interval [CI], 1.6-10.8), and those ≥10.0 mg/dl had an 8.0-fold (95% CI, 3.4-18.4) increased risk of developing CEPH compared with those <5.0 mg/dl. Median TB levels between ≥5.0 and <10.0 mg/dl and >10.0 mg/dl were associated with a 4.8 (95% CI, 2.4-9.7) and 15.6 (95% CI, 8.7-28.2) increased risk of transplantation relative to <5.0 mg/dl. Median TB <5.0 mg/dl were associated with higher NLS rates relative to ≥5.0 mg/dl, with 79% reaching adulthood with native liver ( p < 0.001). CONCLUSIONS: In this large international cohort of ALGS, only 40.3% of children reach adulthood with their native liver. A TB <5.0 mg/dl between 6 and 12 months of age is associated with better hepatic outcomes. These thresholds provide clinicians with an objective tool to assist with clinical decision-making and in the evaluation of therapies.
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Síndrome de Alagille , Colestase , Hipertensão Portal , Humanos , Criança , Masculino , Feminino , Síndrome de Alagille/epidemiologia , Estudos Retrospectivos , Hipertensão Portal/etiologiaRESUMO
AIM: To explore the perceptions and practices of Australasian paediatric gastroenterologists in diagnosing coeliac disease (CD) before and during the COVID-19 pandemic. METHODS: Paediatric gastroenterologists in Australasia were invited via email to complete an anonymous online questionnaire over a 2-week period in 2021. RESULTS: The questionnaire was completed by 39 respondents: 33 from Australia and six from New Zealand (NZ) equating to a 66% response rate. Thirty-four (87%) of the 39 respondents reported they currently practised non-biopsy diagnosis of CD in eligible children, while the rest diagnosed CD using biopsy confirmation only. All NZ respondents practised non-biopsy CD diagnosis. A majority of responders (76%) who practised non-biopsy CD diagnosis followed the 2020 European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) guidelines. Twenty-two (56%) respondents reported that they started using a non-biopsy CD diagnosis protocol before the pandemic and did not change their practice during the pandemic, 10 (26%) started diagnosing non-biopsy CD during the pandemic, 5 (13%) stated their practices of CD were not impacted by the pandemic and 2 (5%) did not respond on whether the pandemic changed their practice. CONCLUSION: The majority of Australasian gastroenterologist respondents reported they routinely utilised the 2020 ESPGHAN diagnostic criteria in eligible children; half of them started prior to the pandemic and another quarter started this approach due to the pandemic. A minority of practitioners routinely rely only on biopsy confirmation to diagnose CD.
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COVID-19 , Doença Celíaca , Gastroenterologistas , Gastroenterologia , Criança , Humanos , Doença Celíaca/diagnóstico , Doença Celíaca/epidemiologia , Pandemias , COVID-19/diagnóstico , COVID-19/epidemiologiaRESUMO
Deciding which patients would benefit from intestinal transplantation (IT) remains an ethical/clinical dilemma. New criteria* were proposed in 2015: ≥2 intensive care unit (ICU) admissions, loss of ≥3 central venous catheter (CVC) sites, and persistently elevated conjugated bilirubin (CB ≥ 75 µmol/L) despite 6 weeks of lipid modification strategies. We performed a retrospective, international, multicenter validation study of 443 children (61% male, median gestational age 34 weeks [IQR 29-37]), diagnosed with IF between 2010 and 2015. Primary outcome measure was death or IT. Sensitivity, specificity, NPV, PPV, and probability of death/transplant (OR, 95% confidence intervals) were calculated for each criterion. Median age at IF diagnosis was 0.1 years (IQR 0.03-0.14) with median follow-up of 3.8 years (IQR 2.3-5.3). Forty of 443 (9%) patients died, 53 of 443 (12%) were transplanted; 11 died posttransplant. The validated criteria had a high predictive value of death/IT; ≥2 ICU admissions (p < .0001, OR 10.2, 95% CI 4.0-25.6), persistent CB ≥ 75 µmol/L (p < .0001, OR 8.2, 95% CI 4.8-13.9). and loss of ≥3 CVC sites (p = .0003, OR 5.7, 95% CI 2.2-14.7). This large, multicenter, international study in a contemporary cohort confirms the validity of the Toronto criteria. These validated criteria should guide listing decisions in pediatric IT.
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Unidades de Terapia Intensiva , Intestinos , Criança , Humanos , Masculino , Recém-Nascido , Lactente , Feminino , Estudos Retrospectivos , Resultado do Tratamento , Intestinos/transplante , Estudos de CoortesAssuntos
Doença de Crohn , Criança , Doença de Crohn/terapia , Nutrição Enteral , Humanos , Nova ZelândiaRESUMO
New Zealand (NZ) guidelines for the approach to diagnosis and management of inflammatory bowel disease (IBD) in children were developed in 2014. Objectives: This study aimed to assess the application of the guidelines in a group of children diagnosed with IBD in regards to baseline investigations. Methods: This retrospective observational study analyzed the application of recommended baseline investigations included in the NZ guidelines in a group of children aged <16 years diagnosed consecutively with IBD at the 2 NZ tertiary pediatric gastroenterology centers. Results: Fifty children were included from each center. Seventy-two were diagnosed with Crohn's disease (CD), 15 with ulcerative colitis (UC), and 13 were with IBD unclassified. The children with CD had a mean Pediatric Crohn's Disease Activity Index score of 31 and almost half had ileocolonic involvement (47%). The 15 children with UC had a mean PUCAI score of 42, and 13 had pancolonic involvement. All 100 children underwent upper and lower gastrointestinal endoscopy with biopsies, and 92% had magnetic resonance enterography at diagnosis. Iron studies, folate, and vitamin B12 were measured in >70 children. Serum zinc, magnesium, and phosphate were infrequently measured. Current anthropometry was recorded in all children but historical growth data were variably recorded. Vaccination status was also inconsistently recorded. Conclusion: Most of this group of children diagnosed with IBD in 2 NZ centers underwent key recommended investigations at diagnosis including gastrointestinal endoscopy and small bowel imaging. Other baseline assessments, including measurement of micronutrient levels, were completed variably. Measures to enhance consistent baseline assessments are required.
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Meckel's diverticulum is the most common congenital gastrointestinal abnormality. Clinical presentation is normally in childhood with either hemorrhage or an acute surgical abdomen. The much rarer giant Meckel's diverticulum is associated with a more varied clinical presentation. In this case report, we provide a unique example of presentation with isolated faltering growth related to a giant Meckel's diverticulum in a young boy. We discuss the diagnostic process, imaging modalities, and subsequent surgical procedure.
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We describe a female toddler with rectal bleeding from extensive colonic polyposis, and diagnosed with familial adenomatous polyposis. She has epilepsy from infancy attributed to focal cortical dysplasia. Hepatoblastoma was diagnosed at 13 months of age. Germline testing detected a pathogenic APC (adenomatous polyposis coli gene) variant. We discuss the anecdotal management of this case, including the clinical utility of genetic confirmation. We review the genotype-phenotype correlation of the APC mutational spectrum, and the existing evidence supporting the hypothesis that cortical dysplasia is part of the APC-related spectrum.
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Polipose Adenomatosa do Colo , Neoplasias Hepáticas , Malformações do Desenvolvimento Cortical , Polipose Adenomatosa do Colo/complicações , Polipose Adenomatosa do Colo/diagnóstico , Polipose Adenomatosa do Colo/genética , Proteína da Polipose Adenomatosa do Colo/genética , Feminino , Genes APC , Estudos de Associação Genética , Mutação em Linhagem Germinativa , Humanos , Neoplasias Hepáticas/genética , Malformações do Desenvolvimento Cortical/genéticaRESUMO
OBJECTIVES: To assess the natural history and outcomes of children with intestinal failure in a large, multicenter, geographically diverse contemporary cohort (2010-2015) from 6 pediatric intestinal failure programs. STUDY DESIGN: Retrospective analysis of a multicenter intestinal failure cohort (n = 443). Competing-risk analysis was used to obtain cumulative incidence rates for the primary outcome (enteral autonomy, transplantation, or death). The χ2 test and Cox proportional hazard regression were used for bivariate and multivariable analyses. RESULTS: The study cohort comprised 443 patients (61.2% male). Primary etiologies included short bowel syndrome (SBS), 84.9%; dysmotility disorder, 7.2%; and mucosal enteropathy, 7.9%. Cumulative incidences for enteral autonomy, transplantation, and death at 6 years of follow-up were 53.0%, 16.7%, and 10.5%, respectively. Enteral autonomy was associated with SBS, ≥50% of small bowel length, presence of an ileocecal valve (ICV), absence of portal hypertension, and follow-up in a non-high-volume transplantation center. The composite outcome of transplantation/death was associated with persistent advanced cholestasis and hypoalbuminemia; age <1 year at diagnosis, ICV, and intact colon were protective. CONCLUSIONS: The rates of death and transplantation in children with intestinal failure have decreased; however, the number of children achieving enteral autonomy has not changed significantly, and a larger proportion of patients remain parenteral nutrition dependent. New strategies to achieve enteral autonomy are needed to improve patient outcomes.
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Enteropatias/epidemiologia , Enteropatias/terapia , Adolescente , Criança , Pré-Escolar , Progressão da Doença , Feminino , Seguimentos , Humanos , Incidência , Lactente , Recém-Nascido , Enteropatias/etiologia , Intestinos/transplante , Masculino , Nova Zelândia/epidemiologia , América do Norte/epidemiologia , Nutrição Parenteral , Modelos de Riscos Proporcionais , Estudos Retrospectivos , Resultado do Tratamento , Reino Unido/epidemiologiaRESUMO
BACKGROUND: Endoscopically obtained mucosal biopsies are the gold standard for diagnosing acute graft-versus-host disease of the gastrointestinal tract (GI-GVHD). There is no consensus on the ideal endoscopic approach in children. We aimed to ascertain which gastrointestinal sites and endoscopic approaches were most helpful for diagnosing acute GVHD and whether clinical symptoms can guide the endoscopic approach. METHOD: A single-center retrospective review of all pediatric stem cell transplants (SCT) between January 1, 2007, and December 31, 2018. Of those with histologically diagnosed GI-GVHD, sensitivities of individual GI sites for making the diagnosis were calculated. Clinical symptoms were compared with GI site yielding diagnosis. RESULTS: 216 allogeneic SCTs were performed in 199 patients. 37 of 52 suspected GI-GHVD cases underwent endoscopy. There was marked variability in the endoscopic approaches chosen. 82% of these cases had lower gastrointestinal symptoms. 21 cases had histologically proven GI-GVHD. 19 (90%) of these had GVHD of non-gastrointestinal sites; 10 (48%) had concurrent infections. The most-sensitive GI sites were the rectosigmoid and duodenum (86% and 76%, respectively). Overall sensitivity of upper GI endoscopy (UGIE) and lower GI endoscopy (LGIE) was 86% and 90%, respectively. There was no statistically significant association between clinical symptoms and site at which histological diagnosis was obtained. CONCLUSION: We observed variability in the endoscopic approach used by clinicians. UGIE and sigmoidoscopy had high sensitivities for diagnosing GVHD, regardless of symptoms. LGIE had minimal additional diagnostic value. This would support a standardized approach with UGIE and sigmoidoscopy for all children with suspected GI-GVHD.
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Endoscopia Gastrointestinal/métodos , Gastroenteropatias/diagnóstico , Doença Enxerto-Hospedeiro/diagnóstico , Transplante de Células-Tronco Hematopoéticas , Biópsia , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Estudos Retrospectivos , Sensibilidade e EspecificidadeRESUMO
Children with inflammatory bowel disease (IBD) and their families benefit from improved knowledge of their disease and treatment. Knowledge levels of individual family members are infrequently studied but may identify where education is best directed. We aimed to assess disease-specific knowledge among children with IBD, parents, and siblings, using a validated assessment tool (IBD-KID2), and to establish generalizability of IBD-KID2. Methods: Children with IBD and family members were recruited from tertiary IBD clinics in New Zealand, Australia, and Canada. All participants completed IBD-KID2 online at baseline, and the children with IBD again after 2 weeks to assess reliability. Results: Participants included 130 children with IBD, 118 mothers, 55 fathers, and 37 siblings. Children with IBD had a mean score of 9.1 (SD 2.9) (maximum 15 points), significantly lower than parents (P < 0.005) and higher than siblings (P < 0.005). Scores of children with IBD were positively associated with current age (P < 0.005), age at diagnosis (P = 0.04) and fathers education level (P = 0.02). Significant score correlations were seen between children with IBD and their mother (P < 0.005) but not father. Sibling scores were not correlated with either parent. Test-retest reliability was high. The cohorts from each country were comparable, and no difference in group scores was seen between countries. Conclusion: IBD-KID2 is a generalizable and reliable tool for the assessment of disease and treatment knowledge for children with IBD and their families. Score correlations between parents and children with IBD suggest transfer of knowledge, but sibling knowledge is low and targeted education may be beneficial.
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AIM: Eosinophilic oesophagitis (EoE) is a rare, chronic, relapsing immune/antigen-mediated disease characterised by symptoms of oesophageal dysfunction, with a paucity of data among New Zealand (NZ) children. This 3-year prospective study aimed to characterise EoE diagnosed nationally and to describe initial treatment strategies adopted. METHODS: Information on new diagnoses of paediatric EoE was obtained via the New Zealand Paediatric Surveillance Unit, through monthly questionnaires. RESULTS: From February 2014 to January 2017, 73 new cases (74% male) of EoE were reported, including 74% NZ European, 10% Asian, 7% Maori, 5% Middle-Eastern and 3% Pacific peoples. Median age of symptom onset was 4 years; dysphagia (48%) was the most common, followed by vomiting/regurgitation (40%), food impaction (19%) and epigastric pain (16%). A co-morbid history of other allergic conditions was present in 62% of patients, and 41% had a first degree relative with atopy. Seventy-nine percent of patients had abnormal endoscopic findings, most commonly linear furrows and white plaques; none had strictures. Median eosinophil count per high-powered field was 40 and 50 in the mid and distal oesophagus, respectively. Fifty-four percent of patients were initially managed with dietary manipulation alone (four required elemental feeds, five nasogastric tubes). Fifty-four percent of patients were treated with swallowed corticosteroids and 7% with prednisone. One patient was also treated with a leukotriene receptor antagonist. CONCLUSION: This first prospective study on paediatric patients with EoE in NZ finds similar demographics and disease characteristics as in other populations despite our unique ethnic population. Long-term prospective observational data should significantly improve our knowledge of this rare condition.
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Transtornos de Deglutição , Esofagite Eosinofílica , Criança , Pré-Escolar , Transtornos de Deglutição/epidemiologia , Transtornos de Deglutição/etiologia , Esofagite Eosinofílica/diagnóstico , Esofagite Eosinofílica/tratamento farmacológico , Esofagite Eosinofílica/epidemiologia , Feminino , Humanos , Masculino , Nova Zelândia/epidemiologia , Estudos ProspectivosRESUMO
Intestinal failure is a complex and debilitating condition characterised by inadequate small intestinal function requiring parenteral or intravenous nutrition to maintain health and, for children, to enable growth and development. Although parenteral nutrition can be prescribed in many hospitals, children with chronic intestinal failure have improved outcomes when managed at a paediatric centre by a multidisciplinary team with specialised expertise in the comprehensive management of intestinal failure. Recent advances in the medical, surgical and nutritional approach have been effective at optimising intestinal rehabilitation and achieving enteral autonomy while limiting complications of intestinal failure. The role of intestinal transplantation in the management of the child with intestinal failure continues to evolve as an option for children with life-threatening complications of intestinal failure. The aim of this review is to highlight key advances in the care of children with intestinal failure.