Long-term Outcomes and Health Perceptions in Pediatric-onset Portal Hypertension Complicated by Varices.

OBJECTIVES: Outcomes of pediatric-onset portal hypertension are poorly defined. We aimed to assess population-based long-term outcomes of pediatric-onset portal hypertension complicated by varices. METHODS: All children with esophageal varices (n = 126) were identified from 14,144 single nationwide referral center endoscopy reports during 1987 to 2013, and followed up through national health care and death registers. A questionnaire was sent to survivors (n = 94) of whom 65 (69%) responded. RESULTS: Nineteen underlying disorders included biliary atresia (35%), extrahepatic portal vein obstruction (35%), autosomal recessive polycystic kidney disease (7%), and other disorders (23%). During median follow-up of 15.2 (range 0.5-43.1) years patients underwent median 9 (1-74) upper gastrointestinal endoscopies. Esophageal varices were first observed at a median age of 4.0 (0.3-18.2) years, 112 (89%) patients underwent median 6 (1-56) sclerotherapy/banding sessions, and 61 (48%) experienced median 2 (range 1-20) variceal bleeding episodes. Forty-eight surgical shunt procedures were performed to 41 (36%) patients and 38% underwent liver transplantation. Portal hypertensive biliopathy was diagnosed in 4 patients. Hepatopulmonary syndrome necessitated liver transplantation in 2 patients, hepatic encephalopathy in 2, and hepatorenal syndrome in 1. No patient died of variceal bleeding. Patient-reported perception of health on a scale of 1 to 10 was 9 (range 4-10), and 86% reported no current symptoms attributable to esophageal varices. CONCLUSIONS: Pediatric-onset portal hypertension is a heterogeneous disease with significant long-term morbidity, requiring multimodal approach with considerable resources and continuation of follow-up in adulthood. Although mortality to variceal bleeding was avoided, bleeding episodes recurred also in adulthood, while patient-reported health of long-term survivors was encouraging.

Long-term outcomes after pediatric splenectomy.

BACKGROUND: Splenectomy is performed frequently for various and primarily hematologic indications in children and adolescents. We analyzed the long-term outcome after splenectomy (median, 8.7 years) focusing on sepsis, portal vein thrombosis (PVT), and retained accessory spleen. METHODS: In total, 141 consecutive children after open (n = 89; 63%) or laparoscopic (n = 52; 37%) splenectomy from 1991 to 2010 were followed up through nationwide registries for septic infections, PVT, and causes of death. Sixty-six patients (58% of survivors) answered a structured questionnaire on infections, abdominal symptoms, and general health, and 64 (laparoscopic n = 26, open n = 38) consented to ultrasonography of the portal venous system. RESULTS: Median operation age was 8.8 years (range, 1.0-22). Reoperations were required for bleeding after open procedures (n = 1) and retained accessory spleen after laparoscopic procedures (n = 3). Postsplenectomy sepsis occurred after a median of 1.7 years (range, 0.2-5.9) in 11 patients (8%), of whom 10 had an underlying immunodeficiency. No cases of PVT were observed, although the median portal vein flow was 1,130 mL/min (range, 440-2200) and diameter was 9.9 mm (range, 7-15) at a median follow-up of 9.5 years (range, 2.0-22) after splenectomy. Twenty-seven patients (19%) died after 8.7 years (0.03-23.00). The most common cause of death was the underlying malignancy (n = 15), with sepsis being an additional cause of death in 5 patients. CONCLUSION: Postsplenectomy sepsis was associated almost exclusively with an underlying immunodeficiency with a high mortality rate. No PVT was observed. The overall risk of retained accessory spleen was around 7%, and was slightly greater after laparoscopic operation.

Liver disease in autosomal recessive polycystic kidney disease: clinical characteristics and management in relation to renal failure.

OBJECTIVES: We correlated liver and kidney manifestations in a national cohort of patients with autosomal recessive polycystic kidney disease (ARPKD). METHODS: A total of 27 consecutive patients with ARPKD were included. Hepatobiliary disorders were comparatively evaluated in 2 groups: children in group 1 (n = 10) displayed renal failure as infants and those in group 2 (n = 17) had normal kidney function through the first year of life. RESULTS: Median follow-up time was 10.6 (range, 0.4-40) years. Portal hypertension was diagnosed in 13 patients (48%) at the median age 5.0 (1.5-27.9) years. Esophageal varices developed in 8 patients (30%) at age 8.0 (2.1-11.9) years; 4 patients (15%) had variceal bleeding, and hypersplenism/splenomegaly occurred in 52%, similarly in both groups. Biliary tract dilatation was detected at 2.8 years in group 1 and at 7.9 years in group 2, significantly more frequently in group 1 (60% vs 18%, P = 0.039), causing cholangitis in 2 (20%) versus none in group 2 (P = 0.055). A total of 10 patients (37%) underwent cadaveric liver transplantation (LT) at a median age of 6.6 (1.0-20.0) years. In 1 patient LT was performed because of hepatoblastoma. Nine of these were combined liver-kidney transplantations (CLKT). Patients in group 1 required LT earlier (4.1 years vs 18.2 years, P = 0.017) and more frequently (70% vs 18%, P = 0.01). Overall survival beyond neonatal period was 85%. Two patients died because of infectious complications after CLKT, and 1 patient because of recurrent hepatoblastoma. CONCLUSIONS: Although correlation of renal and liver manifestations was variable, biliary dilatation was associated with early renal failure. CLKT may be a treatment for patients with ARPKD with marked hepatobiliary complications.