Journal of Pediatric Gastroenterology and Nutrition最新文献

Objectives: To describe the clinical and laboratory characteristics and outcomes of pediatric hepatopulmonary syndrome (HPS) secondary to portal hypertension (PH) in Brazil.

Methods: Fifty-four pediatric patients diagnosed with PH and HPS according to the European Respiratory Society criteria were included in this multicenter retrospective study. Clinical and laboratory data at the time of diagnosing the underlying disease, 12 months before diagnosing HPS, at the time of diagnosing HPS, and at the time of the last consultation were collected from the medical records.

Results: PH was cirrhotic in 87% of patients. Biliary atresia was the predominant etiology (35.2%). The median age at the time of diagnosis was 7.8 years (interquartile range [IQR]: 4.7-10.8). Partial arterial oxygen pressure (PaO₂) of asymptomatic patients (44.4%) was higher than that of symptomatic patients (p < 0.0001). Peripheral oxygen saturation (SpO₂) was ≥96% in eight patients, seven of whom had PaO₂ of <70 mmHg. The hemoglobin levels were elevated at the time of diagnosing HPS (p = 0.009), whereas the platelet count was decreased (p < 0.001). The survival rates of the 66.6% of patients who underwent liver transplantation (LT) at 2 months and 1 year post-LT were 90.3% and 84.6%, respectively. The severity of HPS did not affect the general post-LT survival (p = 0.787).

Conclusions: HPS remains asymptomatic during initial stages. SpO₂ may not be a reliable screening test in pediatric patients. Elevated hemoglobin levels in PH may be related to hypoxemia. The severity of hypoxemia at the time of diagnosis does not affect post-LT survival.

Objectives: Timely availability of a suitable allograft helps offset pediatric liver transplant (pLT) waitlist mortality. Candidate-donor blood type matching (CDBM) is an important attribute for allograft allocation. Current policy allows for blood type (ABO) compatible non-identical transplants for pLT candidates with high acuity. Additionally, centers may use ABO incompatible allografts for pLT candidates when required.

Methods: We conducted a retrospective analysis of the United Network for Organ Sharing (UNOS) database. We analyzed pediatric (<18 years) candidates listed for deceased donor liver-only transplant (DDLT) during the time-period 1/2008 to 12/2022. Specifically, we analyzed the impact of CDBM, including ABO non-identical LT on outcomes.

Results: 72.4% of the 8976 pLT candidates included in our analyses underwent DDLT. 19.9% of pLTs were ABO nonidentical, and 4.9% were ABO incompatible. Nonidentical pLT recipients had significantly higher listing urgency and lower waitlist times. Nonidentical pLTs led to a net loss of allografts for blood type O and net gain of allografts for all other blood types. Candidates with blood type O had significantly higher waitlist time (62.5 vs. 48 days) and lower proportion of candidates undergoing DDLT (70.2% vs. 74.7%), when compared to blood type A.

Conclusion: ABO nonidentical allografts result in timely transplant for pLT candidates with high acuity. Blood type O candidates are disadvantaged, while blood type A candidates are advantaged by ABO nonidentical allografts. The observations of our study, which differ from those of prior adult studies, could inform future policy changes pertaining to organ allocation based on CDBM.

Objectives: Approximately 9% of children with acute recurrent pancreatitis (ARP) or chronic pancreatitis (CP) have pancreatogenic diabetes during childhood; lifetime risk approaches 50%. To date, data are limited on pathophysiology and biomarkers identifying those at highest risk. This pilot study investigated glycemic physiology in children with ARP or CP.

Methods: Children (5-21 years) with an established diagnosis of CP or ARP, and participants of INSPPIRE-2 (INternational Study Group of Pediatric Pancreatitis: In search for a cuRE) were enrolled. Mixed meal tolerance testing (MMTT) measured glucose, insulin, C-peptide, glucagon, glucagon-like peptide-1 (GLP-1) and pancreatic polypeptide (PP) at -5, -1, 0, 30, 60, 90, 120 min before/after a Boost HP beverage. Other measures included hemoglobin A1c, continuous glucose monitoring (CGM, Dexcom Pro), HLA haplotype, and diabetes autoantibodies. Glycemic variability metrics were calculated using "cgmanalysis." Dysglycemia was defined by fasting glucose ≥100 mg/dL or HbA1c ≥ 5.7%.

Results: Twenty participants were enrolled (mean age 16.3 years; 65% female, 60% non-Hispanic white, 2 with pre-existing diabetes). Mean HbA1c was 5.7% (range 5.0-8.9); 7/20 had dysglycemia, 1 with previously unrecognized diabetes. Those with dysglycemia differed from normoglycemic participants by having greater insulin resistance, lower GLP-1, and trend toward lower insulin and C-peptide but higher PP on MMTT.

Conclusions: In this small study, 35% of children with pancreatitis had dysglycemia, which may be mechanistically related to insulin resistance. Other trends associated with dysglycemia included impaired insulin secretion, reduced GLP-1, and unexpectedly elevated PP.

Objectives: Celiac disease (CeD) is an autoimmune disease induced by ingestion of gluten. Patients may present with stereotypical gastrointestinal symptoms, including bloating and diarrhea, but can also present with nongastrointestinal symptoms such as migraines or rashes. The clinical presentation is well described in older pediatric and adult populations; however, the clinical picture is less clear for younger, preschool-aged pediatric patients. This retrospective chart review aims to describe the clinical presentation and serological values seen in patients diagnosed with CeD whose symptoms began before 5 years of age.

Methods: A retrospective review of a single pediatric CeD institution was conducted, and 82 children diagnosed with CeD with symptom onset before 5 years of age were identified.

Results: Children with symptom onset on or before 24 months of age were significantly more likely to have higher symptom burden (5 vs. 4, p = 0.0120). Most notable are vomiting (52.5% vs. 19%, p = 0.0024), mood changes (40% vs. 7.1%, p = 0.0005), poor weight gain (67.5% vs. 41.5%, p = 0.0164), and poor linear growth (35% vs. 11.9%, p = 0.0182). This subgroup was more likely to be hospitalized within 3 months of diagnosis (12.5% vs. 0, p = 0.0241). Children who had symptom onset after 24 months were more likely to have abdominal pain (30% vs. 59.5%, p = 0.0086) and sparser extraintestinal symptoms (p = 0.0224). No matter the age of symptom onset, there was no difference in the time interval to diagnosis.

Conclusions: This study adds to the literature evidence of the extraintestinal presentation of CeD that is seen in toddler-aged patients, as well as increased incidence of hospitalization at the time of diagnosis in this age group.

Objectives: Chronic intestinal failure (CIF) is a rare and complex disease, requiring home parenteral nutrition (HPN) to sustain growth and development. The impact on parents taking care for children with HPN remains underexplored. This qualitative study aimed to elucidate the experiences and challenges faced by parents in caring for HPN-dependent children.

Methods: Parents of children aged 0-18 years, diagnosed with CIF receiving HPN in the Emma Children's Hospital-Amsterdam UMC, were eligible. One-to-one semistructured interviews were conducted with parents at their home with open-ended questions regarding experiences around diagnosis, experiences with healthcare (both home care and at the hospital), mental health, holidays, social functioning and leisure time, work, relationship with the partner and family, fear for the future, and overall impact.

Results: Parents of 24 children were invited. Thirteen parents (four fathers and nine mothers) of 10 children (four females, median age 9 years) with HPN agreed to participate and were interviewed. Most important recurring themes among all parents, emerging from open-ended questions, were as follows: control in caregiving and reluctance to trust others to provide care over their child, social isolation due to limited time and flexibility, and importance of maintaining their own identity by self-fulfillment activities such as work, and physical or social activities.

Conclusions: This qualitative study underlines the profound impact of managing a child with HPN on daily life, relationships and well-being. There is need for tailored support and interventions to help families face the burden of having a child with HPN.

Objectives: Primary intestinal lymphangiectasia (PIL) is a very rare disease responsible for protein-losing enteropathy. There is little published data about treatments efficacy and outcomes. Our main objective was to describe the clinical profile, response to therapy, and outcomes of children with PIL.

Methods: We conducted a national retrospective study including children with PIL followed in French university hospitals between 2010 and 2022. Response to treatment was defined as clinical remission and no need for albumin infusion. Response was considered complete if albuminemia was normal (>35 g/l) during follow-up and partial if less than 35 g/l.

Results: Thirty-four children (22 males) were included; median age at diagnosis was 7 (3-29,5) months. The median follow-up in our cohort was 4.5 years. Edema (79%), chronic diarrhea (50%), and ascites (35%) were the main symptoms. Thirty-one patients received a low long-chain triglycerides dietary therapy and 25 (81%) responded: 15 had a partial response and 10 a complete response. Four patients (12%) required a second-line drug treatment. The presence of lymphedema or an identified genetic variant were associated with a partial response to diet (p  < 0.05). A normal diet could be reintroduced in 14 patients (45%) without relapse during the follow-up. Among them, nine children (26%) were considered cured with a complete and prolonged remission under a normal diet.

Conclusions: Most children with PIL responded to diet therapy and about a quarter of the cohort had a good prognosis with complete remission even after discontinuation of the diet. Presence of lymphedema or a genetic variant was associated with a chronic condition.

Objectives: Chronic nonbacterial osteomyelitis (CNO) is a rare, sterile autoinflammatory bone disorder that can develop in patients with inflammatory bowel disease (IBD). We aimed to identify the clinical features and natural history of patients with a dual diagnosis of CNO and IBD.

Methods: Medical records of patients with a dual diagnosis of IBD and CNO were reviewed in centers from the Paediatric IBD Porto Group and IBD Interest Group of European Society for Pediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN). Collected data included demographic characteristics, disease features, laboratory studies, bone imaging findings, and clinical outcomes.

Results: Forty-five patients (24 [53%] males), were included. Median age at the time of dual diagnosis was 10 (interquartile range [IQR]: 12-13) years. Thiry-two (71%) patients were diagnosed with Crohn's disease, and 14 (44%) of them exhibited perianal disease. CNO presented in 15 patients (33%) within 3 months of IBD diagnosis, and in additional 20 (44%) patients after IBD diagnosis. In most patients, CNO manifested while IBD was clinically active, but not necessarily. However, in 10 (22%) patients CNO preceded the diagnosis of IBD with a median time 46 (25-248) weeks. Upon diagnosis of CNO, most patients were treated with anti-tumor necrosis factor. CNO remission was achieved in all patients at some point during follow-up; However, complications occurred in six patients and included vertebral collapse, bone fracture, and bone deformity.

Conclusions: CNO can be associated with active or quiescent intestinal inflammation, manifesting before, during, or after the diagnosis of IBD. While CNO remission was achieved in all patients, some developed significant bone complications.

Objective: Celiac disease (CeD) is a chronic autoimmune condition triggered by gluten consumption. CeD is screened for using total serum immunoglobulin A (IgA) and tissue transglutaminase immunoglobulin A antibodies (tTG IgA Ab). This study aimed to evaluate IgA patterns over time in patients diagnosed with CeD during childhood.

Methods: A retrospective chart review was performed at Lucile Packard Children's Hospital (January 2012-December 2023). Patients diagnosed before 18 years of age with biopsy-confirmed CeD or those who met European Society of Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) nonbiopsy diagnostic criteria were included. Total serum IgA and tTG IgA Ab levels were required at diagnosis and two subsequent time points. Patients with missing data were excluded.

Results: Of 1861 identified patients, 365 met study criteria. The average age at diagnosis was 9 years old, with an average follow-up time of 5 years. Sixteen patients (4.4%) were IgA insufficient at diagnosis; 11 (69%) remained insufficient while five (31%) became sufficient over the observation period. Nineteen patients (5.2%) initially identified as IgA sufficient developed IgA insufficiency.

Conclusions: IgA levels can fluctuate over time and should be monitored routinely to assess IgA sufficiency and ensure reliable tTG IgA Ab levels. Patients who regain IgA sufficiency can transition to tTG IgA Ab testing, allowing for more reliable assessments of mucosal healing and reducing the need for invasive procedures.