Liver transplantation for children with Wilson disease: comparison of outcomes between children and adults

Arnon R, Annunziato R, Schilsky M, Miloh T, Willis A, Sturdevant M, Sakworawich A, Suchy F, Kerkar N. Liver transplantation for children with Wilson disease: comparison of outcomes between children and adults.
Clin Transplant 2011: 25: E52–E60. © 2010 John Wiley & Sons A/S. Abstract: Liver transplantation (LT) is lifesaving for patients with Wilson disease (WD) presenting with fulminant hepatic failure (FHF) or chronic liver disease (CLD) unresponsive to treatment. Aim: To determine the outcome of LT in pediatric and adult patients with WD. Methods: United Network for Organ Sharing data on LT from 1987 to 2008 were analyzed. Outcomes were compared for patients requiring LT for FHF and CLD after 2002. Multivariate logistic regression was used to determine risk factors for death and graft loss. Results: Of 90 867 patients transplanted between 1987 and 2008, 170 children and 400 adults had WD. The one‐ and five‐yr patient survival of children was 90.1% and 89% compared to 88.3% and 86% for adults, p = 0.53, 0.34. After 2002, 103 (41 children) were transplanted for FHF and 67 (10 children) for CLD. One‐ and five‐yr patient survival was higher in children transplanted for CLD compared to FHF; 100%, 100% vs. 90%, 87.5% respectively, p = 0.30, 0.32. One‐ and five‐yr patient survival was higher in adults transplanted for CLD compared to FHF; 94.7%, 90.1% vs. 90.3%, 89.7%, respectively, p = 0.36, 0.88. Encephalopathy, partial graft, and ventilator use were risk factors for death by logistic regression. Conclusion: LT is an excellent treatment option for patients with WD. Patients transplanted for CLD had higher patient survival rates than patients with FHF.     

The changing face of post‐transplant lymphoproliferative disease in the era of molecular EBV monitoring

Kerkar N, Morotti RA, Madan RP, Shneider B, Herold BC, Dugan C, Miloh T, Karabicak I, Strauchen JA, Emre S. The changing face of post‐transplant lymphoproliferative disease in the era of molecular EBV monitoring.
Pediatr Transplantation 2010: 14:504–511. © 2010 John Wiley & Sons A/S. Abstract: Pediatric PTLD is often associated with primary EBV infection and immunosuppression. The aim was to retrospectively review the spectrum of histologically documented PTLD for two time intervals differentiated by changes in use of molecular EBV monitoring. Eleven of 146 patients (7.5%) in 2001–2005 (Era A) and 10 of 92 (10.9%) in 1993–1997 (Era B) were diagnosed with PTLD. The median age at liver transplantation (0.8 and 0.9 yr, respectively) and the median duration between liver transplant and diagnosis of PTLD (0.6 and 0.7 yr, respectively) were similar in both eras. However, patients in Era A presented with significantly less advanced histological disease compared to patients in Era B (p = 0.03). Specifically, nine patients (82%) in Era A had Pl hyperplasia/polymorphic PTLD, whereas in Era B, six had advanced histological disease (five monomorphic and one unclassified). Three transplant recipients in Era B died secondary to PTLD, whereas there were no PTLD‐related deaths in Era A (p = 0.03). Heightened awareness of risk for PTLD, alterations in baseline immunosuppression regimens, implementation of molecular EBV monitoring, pre‐emptive reduction in immunosuppression and improved therapeutic options may have all contributed to a milder PTLD phenotype and improved clinical outcomes.     

Liver transplantation in children weighing 5 kg or less: analysis of the UNOS database

Arnon R, Annunziato R, Miloh T, Sogawa H, Van Nostrand K, Florman S, Suchy F, Kerkar N. Liver transplantation in children weighing 5 kg or less: Analysis of the UNOS database.
Pediatr Transplantation 2011: 15: 650–658. © 2011 John Wiley & Sons A/S. Abstract: LT is a major medical and surgical challenge in very small patients. Aim of the study is to determine the outcomes after LT in infants ≤5 kg at transplant in a large cohort of patients. Methods: Infants ≤5 kg who had LT between 10/1987 and 5/2008 were identified from the UNOS database. Risk factors for death and graft loss were analyzed by multivariate logistic regression. Results: Of 11 467 children, 570 (5%) were ≤5 kg at LT. Mean age and weight at LT were 0.11 ± 0.48 yr, 4.32 ± 0.74 kg, respectively. One‐ and five‐yr patient and graft survival were 77.7%, 72.2% and 66.1%, 57.6%, respectively. The primary cause of death was infection (25.9%). Recipient age was a predictor of graft loss. Patient and graft survival have improved over time. Life support at transplant was identified as a risk factor for both death and graft loss (p < 0.02, p < 0.01, respectively). Conclusion: LT recipients ≤5 kg have high mortality and graft loss. Over time, graft survival has improved, although it is still inferior to the overall reported outcomes of pediatric LT. Being on life support at transplant is a significant risk factor for death and graft loss in very small recipients.     

A translational and systemic approach to transferring liver transplant recipients from pediatric to adult‐oriented care settings

Annunziato RA, Hogan B, Barton C, Miloh T, Arnon R, Iyer K, Kerkar N. A translational and systemic approach to transferring liver transplant recipients from pediatric to adult‐oriented care settings.
Pediatr Transplantation 2010: 14:823–829. © 2010 John Wiley & Sons A/S. Abstract: The purpose of this review is to (i) describe systemic changes made in our clinical practice to facilitate transfer of transplant recipients from the pediatric to the adult service and (ii) provide the rationale for instituting these organizational changes. To determine specific areas patients struggle to master, a survey assessing behaviors indicative of health care management was administered in pediatrics. Based on the results of the survey, all liver transplant recipients who transfer out of pediatrics are given a comprehensive clinical protocol, which includes a transfer checklist containing prerequisite items derived from our prior work. Patients are furthermore invited to enroll in a research study prospectively tracking both medical and psychosocial outcomes at six‐month intervals. Data are discussed by the pediatric and adult teams, and additional adjustments to the transfer process are implemented in response. A summary of our clinical interventions and the resources required for implementation are presented. In conclusion, preliminary studies have demonstrated that transplant recipients are vulnerable to disruptions in health care management when they transfer out of pediatrics. This study describes one site’s translational efforts to improve the transfer process for both the recipients and the clinicians, thus improving outcomes.     

Improved outcomes in pediatric liver transplantation for acute liver failure

Miloh T, Kerkar N, Parkar S, Emre S, Annunziato R, Mendez C, Arnon R, Suchy F, Rodriguez‐Laiz G, Del Rio Martin J, Sturdevant M, Iyer K. Improved outcomes in pediatric liver transplantation for acute liver failure.
Pediatr Transplantation 2010: 14:863–869. © 2010 John Wiley & Sons A/S. Abstract: OLT is a life‐saving option for ALF. Aim: To evaluate our outcomes in pediatric OLT for ALF. Methods: Retrospective review of our data between 1992 and 2007. Results: Of 142 children with ALF, 126 were listed, of which 40 spontaneously improved, nine died, and 77 underwent OLT (median waiting time four days). Fifty‐three children received deceased donor grafts (34 whole and 19 split grafts), and there were 24 living donor grafts. The one‐ and five‐yr patient survival was 87% and 80%, and graft survival 83% and 79%, respectively. Thirteen patients died after OLT, and there were nine retransplants in seven patients. Patient weight, length of stay, creatinine, and infection were significantly associated with death; increased weight and black ethnicity were associated with graft loss on univariate analysis, but not on multivariate analysis. There were no significant differences in patient survival (one and five yr), graft loss, or other complications between the groups. Conclusion: We report the largest single‐center study of OLT in pediatric ALF, demonstrating no difference in outcomes between different graft types. Our liberal use of segmental grafts may allow earlier OLT in this high‐risk cohort and contribute to our excellent outcomes.     

Transdiaphragmatic intercostal hernia due to chronic cough.

Intercostal transdiaphragmatic hernia is a rarely reported lesion. Trauma is the commonest cause. We report a 75-year-old man who presented with transdiaphragmatic intercostal hernia due to chronic cough. He recovered after surgical repair.     

Partial internal biliary diversion for Alagille syndrome: case report and review of the literature

This is a case report of the first patient with Alagille syndrome (AGS) to undergo a partial internal biliary diversion (PIBD) for the treatment of symptoms refractory to medical therapy. Alagille syndrome is a hereditary disease resulting in chronic cholestasis and hypercholesterolemia that can lead to severe and intractable pruritus and disfiguring and debilitating xanthomas. PIBD has proven to be an effective treatment option for other causes of cholestatic liver disease. This report reviews the immediate and 2-year follow-up of a patient after this surgical procedure. The results suggest that PIBD has potential to provide relief of intractable symptoms and improve the quality of life in patients with AGS while avoiding an external stoma. It does not, however, appear to prevent the progression of liver disease. Long-term follow-up is still needed.