Search

Background Portal diversion by surgical shunt plays a major role in the treatment of medically refractory portal hypertension. We evaluate our center’s experience with surgical shunts for the treatment of pediatric portal hypertension. Methods All patients who underwent surgical shunt at a single institution from 2008 to 2017 were reviewed. The primary outcome was intervention-free shunt patency. Results In this study, 34 pediatric patients underwent portal shunt creation. The median age was 7.7 years (interquartile range 4.3–12.0). Twenty-nine patients (85%) had prehepatic portal hypertension and 5 patients (15%) had intrahepatic portal hypertension. The primary manifestations of portal hypertension were esophageal varices (97%) and gastrointestinal bleeding (77%). Eighteen patients (53%) underwent meso-Rex bypass, 10 patients (29%) underwent splenorenal shunt, and 6 patients (18%) underwent mesocaval shunt. Outcomes were notable for minimal wound complications (9%), rebleeding events (12%), and mortality (3%). In the postoperative setting, 10 patients (29%) experienced a shunt complication (occlusion or stenosis), 4 of which occurred in the early postoperative period and required urgent intervention. The 1-year and 5-year “primary patency” patency rates were 71% and 66%, respectively. Conclusion Children suffer significant morbidity from the sequelae of portal hypertension. Our experience reinforces the feasibility of surgical shunts as an effective treatment option associated with low rates of morbidity and mortality.

Objectives Megacystis–microcolon–hypoperistalsis syndrome (MMIHS) also called Berdon's Syndrome, is a smooth muscle myopathy that results in an enlarged bladder, microcolon, and small bowel hypoperistalsis. In our series of six patients with this disorder, all had disordered swallowing. Therefore, we prospectively characterized esophageal structure and function in all. Methods Diagnoses had been established by contrast radiography, small bowel manometry, and urodynamic studies. To investigate the esophagus, we endoscoped and biopsied the esophagus of each patient on multiple occasions. All patients also underwent water soluble contrast esophagography and esophageal manometry. Results Upon careful questioning, all patients had swallowing dysfunction, and the majority of their enteral intake was via gastrostomy or gastrojejunostomy. All took some oral alimentation, but eating was slow and none could aliment themselves completely by the oral route, receiving 50% or less of their calories by mouth. Four had megaesophagus whereas the esophagus of the two youngest was of normal caliber. All had eosinophilic esophagitis and/or esophageal Candidiasis from time to time, but successful treatment of these findings failed to improve their symptoms. Manometry revealed normal lower esophageal sphincter (LES) resting tone and normal LES relaxation, but for all, peristalsis was absent in the esophageal body. Conclusions This series expands the spectrum of findings in MMIHS, to include a primary motility disorder of the esophageal body. As patients age, the esophageal caliber appears to increase. Successful treatment of neither esophageal eosinophilia nor Candidiasis is effective in ameliorating the motility disorder. If our findings are confirmed in more patients with MMIHS, this disorder should be renamed, megacystis–microcolon–intestinal-and esophageal hypoperistalsis syndrome. Type of study Prognosis study, Level IV (case series).

Autosomal-recessive polycystic kidney disease (ARPKD; MIM 263200) is a severe, typically early-onset form of cystic disease that primarily involves the kidneys and biliary tract. Phenotypic expression and age at presentation can be quite variable. 1 The incidence of ARPKD is 1 in 20000livebirths, 2 anditspleotropicmanifestationsarepotentially life-threatening. Optimal care requires proper surveillance to limit morbidity and mortality, knowledgeable approaches to diagnosis and treatment, and informed strategies to optimize quality of life. Clinical management therefore isideallydirected by multidisciplinary care teams consisting of perinatologists, neonatologists, nephrologists, hepatologists, geneticists, and behavioral specialists to coordinate patient care from the perinatal period to adulthood. In May 2013, an international team of 25 multidisciplinary specialists from the United States, Canada, Germany, and the United Kingdom convened in Washington, DC, to review the literature published from 1990 to 2013 and to develop recommendations for diagnosis, surveillance, and clinical management. Identification of the gene PKHD1, and the significant advances in perinatal care, imaging, medical management, and behavioral therapies during the past decade provide the foundational elements to define diagnostic criteria and establish clinical management guidelines as the first steps towards standardizing the clinical care for patients with ARPKD. The key issues discussed included recommendations regarding perinatal interventions, diagnostic criteria, genetic testing, management of renal and biliary-associated morbidities, and behavioral assessment. The meeting was funded by the National Institutes of Health and an educational grant from the Polycystic Kidney Disease Foundation. Here we summarize the discussions and provide an updated set of diagnostic, surveillance, and management recommendations for optimizing the pediatric care of patients with ARPKD. Specialist care of ARPKD-related complications, including dialysis, transplantation, and management of severe portal hypertension (HTN), will be addressed in a subsequent report. Given the paucity of information regarding targeted therapies in ARPKD, this topic was not addressed in this conference.

Purpose Infants with multiple cutaneous hemangiomas often present with hepatic hemangiomas. They can follow a benign clinical course or require complex management. We reviewed our experience in the management of hepatic hemangiomas. Methods We performed a retrospective review of patients (1996-2007) with hepatic hemangiomas treated in our institution. Results Twenty-six patients were diagnosed with hepatic hemangiomas as follows: 8 focal, 12 multiple, and 6 diffuse lesions. Nineteen (73%) patients had associated cutaneous hemangiomas. Sixteen patients had multiple and 3 patients had single cutaneous hemangiomas. All patients with multiple or diffuse liver lesions were screened for heart failure and hypothyroidism. Congestive heart failure developed in 4 patients, 3/4 of these patients had diffuse lesions. Two patients required thyroid replacement because of elevated thyroid-stimulating hormone. Because of progression of disease, 9 patients required steroid treatment. Two patients were treated with vincristine and 3 patients received α -interferon because of poor response to steroid treatment. Two patients went on to surgical resection for failed response to medical management and worsening heart failure (left lobectomy, liver transplant). Both patients had uncomplicated postoperative courses. Five patients had a previously undescribed constellation of rapidly involuting cutaneous hemangiomas (gone by 3 months, glut-1–negative) with associated liver lesions also resolving at a faster pace (mean resolution of cutaneous hemangiomas, 1.9 vs 7.9 months; P = .001; liver, 5.8 vs 25.3 months; P = .004). All patients in our series survived. Conclusion Patients with multiple cutaneous hemangiomas should be screened for hepatic lesions. Patients with diffuse or multifocal liver hemangiomas should be screened for congestive heart failure and hypothyroidism. A subgroup of rapidly involuting cutaneous hemangiomas have a significantly shorter time for involution of hepatic lesions. The status of cutaneous lesions can be used as indicators for the liver hemangiomas.

Liver transplantation has become the accepted standard of care in the treatment of a child with a failing liver. Advances in the management of critical care and immunosuppression along with the development of innovative operative procedures have improved outcome such that 5-year survival rates of 80% to 90% are expected following liver transplantation. Organ allocation schemes have evolved in an effort to better stratify recipient risk thereby more appropriately distributing deceased donor grafts. A persistent shortage of appropriate donors continues to contribute to patient mortality. The consequences of long-term immunosuppression have become increasingly apparent such that health care providers need to be aware of the side effects of chronic immunosuppression. New strategies need to be defined to minimize the need of continuous immunosuppression. The continued success of pediatric liver transplantation will require multi-disciplinary health care teams comprised of general pediatricians, pediatric hepatologists, transplant surgeons, and transplant coordinators who focus on the complex needs of the transplant recipient.

During repair of an esophageal atresia with tracheoesophageal fistula (EA/TEF), a right-sided aortic arch (RAA) presents a technical challenge. We reviewed our experience with EA/TEF focusing on the impact of RAA. Methods A retrospective review of patients with EA/TEF at our institution from 1990 to 2004 was performed. Of 61 patients, 53 had a left aortic arch (LAA) and 8 had RAA. Results The mean gestational age and birth weight were similar between patients with LAA and RAA. There were more vascular rings in patients with RAA than with LAA (37.5% vs 3.8%, P = .009). A preoperative echocardiogram correctly identified the location of the arch in all with LAA but only 62.5% of those with RAA. Operative complications occurred in 25% of patients with RAA and 11% of patients with LAA. Conclusions Management of a child with RAA and EA/TEF is technically challenging. Preoperative identification of the great vessel anomalies may impact operative decision making. Therefore, careful diagnostic evaluation of a patient with an aortic arch that cannot be easily defined by ECHO should be considered before definitive repair.

Therapeutic options for children with portal hypertension now include a broad range of pharmacologic, endoscopic, and surgical procedures. Thoughtful application of all of these options can improve quality of life by decreasing the complications of portal hypertension and can decrease mortality by preventing the consequences of variceal hemorrhage. The development of portal hypertensive gastropathy following palliative procedures such as endoscopic sclerotherapy and band ligation may limit their long-term success in children. The excellent results now obtained with selective portosystemic shunts and liver transplantation assure that definitive surgical treatments will continue to be important components in the treatment of children with portal hypertensive complications or progressive liver disease. Evolving procedures, such as TIPS, represent excellent short-term life-preserving techniques to stabilize critically ill patients while awaiting liver transplantation. Their role in the future, long-term management of children is yet to be defined.

At the beginning of the twenty-first century, hepatocyte transplantation (HTX) continues to evolve as a potential therapeutic adjunct to orthotopic liver transplantation. Hepatocyte transplantation can correct a metabolic defect by serving as the essential vehicle for ex vivo gene therapy or by providing the vital enzyme that is deficient in patients with a liver-based inborn error of metabolism. Hepatocyte transplantation can also function as a life-saving bridge by providing a cellular mass temporarily sufficient carry out metabolic function for patients with liver failure awaiting liver transplantation. Significant technical advances with reduced-size, split-liver and living-related donors and improved public awareness of the feasibility and need for organ donation have improved the liver donor pool. A disequilibrium remains, however, with the ever-increasing recipient demand outnumbering the limited donor supply. Therefore, the quest for suitable alternatives to help relieve the burden of organ shortages remains paramount. One such alternative is HTX. This article discusses key advances in the evolution of HTX, from its use as an experimental tool for the study of liver cell biology to a potential treatment modality for children and adults with liver disease.

Undifferentiated embryonal sarcoma of the liver is a rare hepatic malignancy of childhood with a historically poor prognosis. Recent improvements in outcomes have been reported in small numbers of cases with the use of combination therapy involving aggressive surgical resection and chemotherapy. Complete surgical resection is frequently difficult to achieve when the location of the tumor is along the margins of the major hepatic vessels (portal vein, hepatic vein, and hepatic artery). Here we report a case of undifferentiated embryonal sarcoma of the liver that recurred along surgical hepatic vein margins in a 9-year-old boy who subsequently underwent orthotopic liver transplantation from a cadaveric donor. The patient has been in continuous clinical remission for the last 5 years.

Background: Liver transplantation (LT) remains a high-risk operation, especially during the first months after LT when technical complications and preexisting illness exert their influence on survival. However, there are late deaths. The authors have reviewed their experience to identify factors impacting on long-term survival. Methods: A total of 150 patients who had undergone liver transplantation over an 11-year period were reviewed. Thirty-three patients died after LT (22%). Of these, 18 of 33 (55%) died in the first 3 postoperative months. One hundred thirty-two patients survived beyond 3 months, and 15 patients (11%) suffered late deaths. This review concentrates on the latter group. Results: The primary cause of death was sepsis in 11 of 15 (73%). In two, sepsis complicated retransplantation in chronically debilitated patients. Two additional patients had late-presenting postoperative complications (bile leak or abscess, intestinal obstruction with perforation). In two cases, pneumocystis carinii pneumonia occurred; noncompliance or unplanned discontinuation of prophylaxis was directly responsible. Multiple organ system failure from presumed immunoincompetence developed in four patients; one had undergone bone marrow transplantation for aplastic anemia (AA) after fulminant hepatic failure (FHF). Lymphoproliferative disease (LPD) was the cause of death in 3 of 15 cases (20%). In only three cases was the cause of death related to the patient's primary disease (chronic hepatitis, Alper's syndrome or seizures, and AA with FHF). Pretransplant diagnosis, and UNOS status at the time of LT did not influence the long-term survival. Conclusions: Long-term survival in patients who have undergone LT was compromised by immunosuppressive complications and sepsis. Early mortality factors, such as UNOS status, age at LT, primary diagnosis, and technical complications do not predict late deaths. In children who adhere to their medical regimen and have good initial allograft function, late postoperative infection, especially with Ebstein-Barr virus, accounts for most of the late mortality. Improved and decreased immunosuppression may further improve these long-term results.