Birth outcomes of patients with isolated anorectal malformations: A population‐based case‐control study

In most patients affected by isolated anorectal malformation (IARM) the etiology is largely unknown. Thus, the aim of our project was to analyze possible risk factors for IARM. In the first step, birth outcomes of cases with IARM were analyzed on the basis of maternal socio‐demographic variables, and these data are presented in this paper. Gestational age at delivery, birthweight, preterm birth, low birthweight and small for gestational age of cases with IARM were evaluated in the function of maternal age, birth/pregnancy order, marital and employment status of mothers in the population‐based large dataset of the Hungarian Case‐Control Surveillance of Congenital Abnormalities, 1980–1996. The study samples included 231 live‐born cases with IARM, 361 matched and 38 151 population controls without any defect. IARMs are more frequent in males, twins and newborn infants with low birthweight and small‐for‐gestational‐age, the latter being the consequence of intrauterine growth restriction. In addition, mothers of cases were younger but with higher birth order, and had lower socio‐economic status. These maternal variables are characteristic for the gypsy population in Hungary. The higher proportion of gypsy women among the mothers of cases with IARM was confirmed during the home visits of the study. Male sex and intrauterine growth restriction of cases, in addition to low socioeconomic status and gypsy origin of mothers may have a role in the risk of IARMs.     

Characterization of long‐term outcomes for pediatric patients with epithelioid hemangioma

Epithelioid hemangioma (EH) is a rare benign vascular tumor that occurs in soft tissues and bone and presents between the third and sixth decades of life. Little is known about the clinical course and outcomes of pediatric EH. We report 11 patients diagnosed with EH at a median age of 14.4 years. One patient treated with interferon and one with sirolimus exhibited partial response for >2 years. Although a benign neoplasm, EH is difficult to manage without standard protocols and portends considerable morbidity. Our findings suggest medical management, particularly sirolimus, may benefit these patients; however, long‐term follow‐up is needed.     

Effect of neonatal resuscitation courses on long‐term neurodevelopmental outcomes of newborn infants with perinatal asphyxia

Background: In previous studies, it has been demonstrated that Neonatal Resuscitation Program (NRP) courses improve the early outcomes of infants with perinatal asphyxia, but there has been no evidence to demonstrate the effect of NRP on long‐term outcomes of perinatal asphyxia. The goal of the present study was to determine the effect of NRP courses on the long‐term neurodevelopmental outcome of perinatal asphyxia. Methods: This prospective study included infants referred to the Neonatal Unit during the years 2003–2005. Those patients who were referred before NRP courses (pretraining period) were designated as group 1, those who were referred after the first NRP course (transition period) as group 2, and those who were referred after the second NRP course (post‐training period) as group 3. Neurodevelopmental outcomes were assessed and compared at 4–6 years of age. Results: The study involved 40 patients: 23 in group 1, nine in group 2 and eight in group 3. The number of patients who had been diagnosed with cerebral palsy was 13 in group 1, two in group 2, and one in group 3, which was a significant decrease. The number of patients with seizures and electroencephalography abnormality was 12 and 14 in group 1, three and two in group 2, and one and one in group 3, respectively, which was also a significant decrease. Conclusions: NRP courses have positive effects on short‐term as well as long‐term neurodevelopmental outcomes of infants with perinatal asphyxia. Further studies are required to determine the effects of NRP courses on minor deficits, such as cognitive and behavioral disturbances.     

Survival of a very low‐birthweight infant with Potter sequence on long‐term hemodialysis

Recent advances in neonatal cardiorespiratory management and neonatal renal replacement therapy have led to occasional reports of favorable short‐term and long‐term outcomes for Potter sequence, once thought to be fatal. The present patient was a girl born at a gestational age of 34 weeks 4 days with a birthweight of 1398 g. She was diagnosed with Potter sequence complicated by pulmonary hypoplasia due to left renal agenesis and small right kidney. Hemodialysis was started because anuria persisted even after persistent pulmonary hypertension receded and cardiorespiratory status improved. Peritoneal dialysis during the clinical course failed to achieve stability because of pericatheter leakage and frequent obstruction of the peritoneal dialysis catheter; in the meantime, hemodialysis was also performed. Once bodyweight passed 3.5 kg, peritoneal dialysis became more feasible with stability; the patient was discharged at 9 months and at the time of writing was on peritoneal dialysis at home, and renal transplantation was planned.     

Long‐term outcomes of liver transplantation for hepatoblastoma: A single‐center 14‐year experience

HB is the most common primary liver tumor in children. Complete tumor excision, either by partial resection or by total hepatectomy and liver transplantation, in combination with chemotherapy provides the best chance for cure. We performed a retrospective analysis of patients who underwent liver transplantation for HB and herein present our 14‐year single‐institution experience. Twenty‐five patients underwent liver transplantation for HB at a median age of 26 months (IQR: 15‐44). Graft survival was 96%, 87%, and 80% at 1, 3, and 5 years, respectively. There were four patient deaths, three of them due to disease recurrence within the first year post‐transplant. Ten‐year overall survival was 84%. Three recipients initially presented with pulmonary metastases and underwent resection of metastatic disease, of which two are alive at 3.9 years. Of three patients who underwent salvage transplants, two are alive at 1.5 years after transplant. Non‐survivors were associated with lower median alpha fetoprotein value at presentation compared to survivors (21 707 vs 343 214; P = .04). In conclusion, the overall long‐term outcome of primary liver transplantation for HB is excellent. Tumor recurrence was the highest contributor to mortality. Even patients with completely treated pulmonary metastases prior to transplant demonstrated a favorable survival.     

Association of post‐transplant donor‐specific HLA antibody with liver graft fibrosis during long‐term follow‐up after pediatric liver transplantation

The aim of this study was to evaluate the significance of post‐transplant DSA as a predictor of liver fibrosis during long‐term follow‐up after pediatric LT. We evaluated the histological findings in 18 LT recipients who underwent liver biopsy after DSA screening. Liver fibrosis was scored based on the METAVIR fibrosis staging. Patients were divided into 2 groups based on histological findings, and clinical characteristics among patients with liver fibrosis were assessed. Of 18 patients, 7 were included in the fibrosis group. No significant between‐group differences were found regarding peritransplant characteristics, including age, sex, primary disease, ABO incompatibility, and immunosuppressive regimen. Episodes of acute rejection and non‐adherence to immunosuppressive drugs were comparable between both groups. The MFI for anti‐DR DSA and positive rate were significantly higher in the fibrosis group (1655 vs 216; P = .019, 86% vs 27%; P = .012, respectively). MFI for anti‐DQ DSA was higher in the fibrosis group, but non‐significantly (2052 vs 384; P = .46). Post‐transplant anti‐DR DSA is associated with graft fibrosis during long‐term follow‐up. This finding seems useful for the implementation of valid histological examinations of liver grafts for patients with higher MFI, especially for anti‐DR DSA, after pediatric LT.