Long‐term outcome following pediatric liver transplantation for metabolic disorders

Stevenson T, Millan MT, Wayman K, Berquist WE, Sarwal M, Johnston EE, Esquivel CO, Enns GM. Long‐term outcome following pediatric liver transplantation for metabolic disorders.
Pediatr Transplant 2010:14:268–275. © 2009 John Wiley & Sons, A/S. Abstract: In order to determine long‐term outcome, including survival, growth and development, following liver transplantation in children with metabolic disorders, we retrospectively reviewed charts of 54 children with metabolic disorders evaluated from 1989–2005 for presenting symptoms, transplantation timing and indications, survival, metabolic parameters, growth, and development. Thirty‐three patients underwent liver transplantation (12 received combined liver–kidney transplants) at a median age of 21 months. At a median follow‐up of 3.6 yr, patient survival was 100%, and liver and kidney allograft survival was 92%, and 100%, respectively. For the group as a whole, weight Z scores improved and body mass index at follow‐up was in the normal range. Two yr post‐transplantation, psychomotor development improved significantly (p < 0.01), but mental skills did not; however, both indices were in the low‐normal range of development. When compared to patients with biliary atresia, children with metabolic disorders showed significantly lower mental developmental scores at one and two yr post‐transplantation (p < 0.05), but psychomotor developmental scores were not significantly different. We conclude that, in patients with metabolic disorders meeting indications for transplantation, liver transplantation or combined liver–kidney transplantation (for those with accompanying renal failure) is associated with excellent long‐term survival, improved growth, and improved psychomotor development.     

Kidney and liver transplantation in children with fibrocystic liver–kidney disease: Data from the US Scientific Registry of Transplant Recipients: 1990–2010

The natural history and survival of children with fibrocystic liver–kidney disease undergoing solid organ transplantation have infrequently been described. We report outcomes in a cohort of US children with fibrocystic liver–kidney disease receiving solid organ transplants over 20 yr. Retrospective cohort study of pediatric transplant recipients with diagnoses of fibrocystic liver–kidney disease from 1/1990 to 3/2010, using data from the SRTR. Subjects were categorized by the first transplanted organ: LT, KT, or SLK. Primary outcomes were death, re‐transplant, transplant of the alternate organ, or initiation of dialysis. Seven hundred and sixteen subjects were transplanted in this period. Median age at first transplant was 9.7 yr. Of the LT, 14 (19%) required a second liver transplant at median of 0.2 yr, and five (7%) required kidney transplant or dialysis at a median of 9.0 yr. Of the KT, 188 (31%) required a second kidney transplant or dialysis at a median of 5.9 yr. Twenty‐nine (5%) subsequently received liver transplant at a median of 6.0 yr. Among patients in this registry, far more children underwent kidney than liver transplants. The risk of subsequently needing transplantation of an alternate organ was low.     

Pediatric hepatocellular carcinoma in a developing country: Is the etiology changing?

HCC is the second most common malignant liver tumor of childhood. It typically affects children with a median age of 10–14 yr on background hepatitis B‐related liver disease and is often metastatic or locally advanced at diagnosis. Children below the age of five yr typically constitute <10% of all children with HCC. In these children, it occurs on a background of congenital or metabolic liver disease. The records of all children with HCC who presented to our department over a six‐yr study period were reviewed. Twelve patients with a median age of 5.9 yr (range 1.6–15.4) were diagnosed to have HCC. All patients underwent liver transplantation, and none were resected. Eleven patients had background congenital or metabolic liver disease. All five of those with hereditary tyrosinemia type 1 who presented to us were found to have HCC. No patient had hepatitis B‐related liver (HBV) disease. Eight (66.7%) patients had incidentally discovered HCC on examination of the explant. Incidentally discovered HCC were smaller, well differentiated, and did not show microvascular invasion compared to those diagnosed preoperatively. There was no recurrence with a median follow‐up of five months. The patient demographic for pediatric HCC is changing probably as a consequence of successful immunization against HBV. Younger patients with congenital and metabolic liver disease in whom liver transplantation is the ideal treatment are likely to constitute an ever‐increasing proportion of patients with pediatric HCC as HBV disease is controlled or eradicated.     

Liver transplantation in pediatric patients: twenty years of experience at the University of Wisconsin

Developments in surgical technique, immunosuppression, organ procurement and preservation, and patient selection criteria have resulted in improved long-term patient and graft survival after pediatric liver transplantation. In this study, we examined the results of 196 liver transplants performed in 155 pediatric patients at University of Wisconsin Children's Hospital. Patients were divided into two groups according to age at the time of liver transplant. Infants under 12 months of age comprised Group 1 (n=74) and children from one to 18 yr comprised Group 2 (n=122). Outcomes for whole, reduced-size, and split liver transplantation were compared in infants and children. Biliary atresia was the most common indication in both groups. Patients underwent 128 whole size, 50 reduced size, and 18 split liver transplants. Forty-one retransplantations were performed in 14 infants (18.9%) and in 27 children (22.1%). One hundred eleven patients (56.6%) had one or more rejection episode [37 infants (50.0%) and 74 children (60.6%)]. Thirty-nine patients (19.8%) developed CMV infections, 42 (21.4%) developed EBV infections, and 14 developed PTLD (six infants and eight children). Thirty-six patients (18.3%) developed HAT. Seven patients (4.5%) developed malignancy (one infant and six children). Out of 155 patients, 33 (21.3%) died during the study period. The most common etiology of mortality included central nervous system pathology (n=7; 4.5%), sepsis (n=6; 3.8%), and cardiac causes (n=6; 3.8%). One-, five-, and 10-yr actuarial patient survival was 86, 79, and 74% in infants and 90, 83 and 80% in children. Graft survival at one, five, and 10 yr was 77, 73 and 71% in infants and 88, 81 and 78% in children, respectively. Despite its technical challenges, the outcomes of liver transplantation in pediatric patients with end-stage liver disease are excellent and result in significant long-term patient and graft survival.     

Liver transplantation in children with metabolic diseases: The studies of pediatric liver transplantation experience

Arnon R, Kerkar N, Davis MK, Anand R, Yin W, González‐Peralta RP for the SPLIT Research Group. Liver transplantation in children with metabolic diseases: The studies of pediatric liver transplantation experience.
Pediatr Transplantation 2010: 14:796–805. © 2010 John Wiley & Sons A/S. Abstract: Metabolic diseases are the second largest indication for LT in children after BA. There are limited data on the long‐term post‐transplant outcome in this unique group of patients. Therefore, our aim was to assess post‐liver transplant outcomes and to evaluate risk factors for mortality and graft loss in children with metabolic disorders in comparison to those with non‐metabolic diagnoses. We reviewed all patients enrolled in the SPLIT registry. Between 1995 and 2008, 446 of 2997 (14.9%) children enrolled in SPLIT underwent liver transplant for metabolic diseases. One‐yr and five‐yr patient survival for children with metabolic diseases was 94.6% and 88.9% and for those with other diseases 90.7% and 86.1% (log‐rank p = 0.05), respectively. One‐yr and five‐yr graft survival for children with metabolic disorders was 90.8% and 83.8%, and for those with other diseases 85.4% and 78.0% (log‐rank p = 0.005), respectively. Children with metabolic diseases were less likely to experience gastrointestinal complications (5.6% vs. 10.7%, p = 0.001), portal vein thrombosis (2.9% vs. 5.2%, p = 0.04), and reoperations within 30 days post‐transplant (33.4% vs. 37.8%, p = 0.05) than those with other indications. In conclusion, children who underwent liver transplant for metabolic disease had similarly excellent patient survival as, and better graft survival than, those who received a liver allograft for other indications.     

Cardiovascular risk factors and cardiac disorders in long‐term survivors of pediatric liver transplantation

The MetS and cardiovascular disease are leading causes of late morbidity in adult liver transplantation recipients; however, limited data are available in pediatric liver transplantation. A single‐center retrospective review was undertaken for patients who had a liver transplantation before 18 yr of age and were >5 yr post‐transplantation, to study the prevalence of MetS, its components, and cardiac disorders. Fifty‐eight patients were included in the study with a mean age at transplantation of 6.3 ± 6.1 yr and mean follow‐up of 14.1 ± 6.0 yr. Of the study group, 41.4% were overweight or obese, with ongoing prednisone use and increased duration of follow‐up being significant risk factors. Fifty‐three patients had sufficient data for determining MetS, which was present in 17% of the patients. Although the prevalence of MetS is low in pediatric liver transplant recipients, it is associated with CKD and prednisone therapy (p < 0.05). Echocardiography data were available for 23 patients, of whom 43.4% had LVH and 13% had evidence of PH. The spectrum of cardiac disorders in this population is much wider than in adults.     

An institutional experience of pre‐emptive liver transplantation for pediatric primary hyperoxaluria type 1

Primary hyperoxaluria type 1 (PH1) is an inherited metabolic disease that culminates in ESRF. Pre‐emptive liver transplantation (pLTx) treats the metabolic defect and avoids the need for kidney transplantation (KTx). An institutional experience of pediatric PH1 LTx is reported and compared to the literature. Between 2004 and 2015, eight children underwent pLTx for PH1. Three underwent pLTx with a median GFR of 40 (30–46) mL/min/1.73 m² and five underwent sequential combined liver‐kidney transplantation (cLKTx); all were on RRT at the time of cLKTx. In one case of pLTx, KTx was required eight and a half yr later. pLTx was performed in older (median 8 vs. 2 yr) and larger children (median 27 vs. 7.75 kg) that had a milder PH1 phenotype. In pediatric PH1, pLTx, ideally, should be performed before renal and extrarenal systemic oxalosis complications have occurred, and pLTx can be used “early” or “late.” Early is when renal function is preserved with the aim to avoid renal replacement. However, in late (GFR < 30 mL/min/1.73 m²), the aim is to stabilize renal function and delay the need for KTx. Ultimately, transplant strategy depends on PH1 phenotype, disease stage, child size, and organ availability.     

Living donor liver transplantation for inborn errors of metabolism – An underutilized resource in the United States

Inborn metabolic diseases of the liver can be life‐threatening disorders that cause debilitating and permanent neurological damage. Symptoms may manifest as early as the neonatal period. Liver transplant replaces the enzymatically deficient liver, allowing for metabolism of toxic metabolites. LDLT for metabolic disorders is rarely performed in the United States as compared to countries such as Japan, where they report >2000 cases performed within the past two decades. Patient and graft survival is comparable to that of the United States, where most of the studies are based on deceased donors. No living donor complications were observed, suggesting that LDLT is as safe and effective as deceased donor transplants performed in the USA. Increased utilization of living donors in the USA will allow for early transplantation to prevent permanent neurological damage in those with severe disease. Pediatric transplant centers should consider utilizing living donors when feasible for children with metabolic disorders of the liver.     

Prevalence of hepatitis E virus infection in pediatric solid organ transplant recipients - A single-center experience

Hoerning A, Hegen B, Wingen A‐M, Cetiner M, Lainka E, Kathemann S, Fiedler M, Timm J, Wenzel JJ, Hoyer PF, Gerner P. Prevalence of hepatitis E virus infection in pediatric solid organ transplant recipients – A single‐center experience. Abstract: HEV infection appears to be an emerging disease in industrialized countries. The aim of this study was to evaluate the prevalence of HEV infection in pediatric solid organ transplant recipients. One hundred and twenty‐four pediatric recipients of liver (n = 41) or kidney (n = 83) transplants aged between one and 18 yr were screened for anti‐HEV IgG antibodies. Patients were tested for fecal HEV RNA excretion if they showed anti‐HEV seropositivity. As a control group, 108 immunocompetent pediatric patients without liver disease aged between three and 18 yr were screened for anti‐HEV IgG. HEV seroprevalence was 2.4% in renal Tx (2/83), 4.9% in liver Tx patients (2/41), and 3.2% overall (4/124). Three of these four patients were HEV RNA‐negative. In one renal transplant patient, HEV genotype 3 RNA excretion persisted and liver enzymes were elevated, indicating chronic hepatitis. In the control group, eight patients (7.4%) were HEV IgG‐positive without biochemical evidence of hepatitis. The prevalence of HEV infection in pediatric renal or liver transplant recipients is not higher compared with immunocompetent children. Chronic HEV infection with long‐term carriage of the virus may develop in pediatric transplant recipients. Autochthonous HEV infection needs to be considered in uncertain cases of hepatitis in immunosuppressed as well as immunocompetent children.     

Pediatric en bloc kidney transplantation into pediatric recipients: the French experience

Chronic shortage of available donor organs has led to re-evaluation of the use of en bloc kidney transplants. Although excellent results have been reported in adult patients, experience in pediatric patients remains limited because of potential early complications and poor long-term graft outcome. We report 14 pediatric en bloc renal transplantations into 14 pediatric recipients, performed between 1990 and 2007 in France. We retrospectively analyzed demographic data, postoperative complications, and graft function with a median follow-up of five yr. Donor age ranged from four to 54 months. Complications were vascular graft thrombosis in four patients, leading to graft loss in two cases, and to excellent long-term graft function in the two others. Two hemorrhagic complications resulted in death in one case and in graft loss in the other. Six acute rejection episodes occurred in four patients. Median glomerular filtration rate at three months, one, five, and 10 yr was 90.8, 106, 87.8, and 66.1 mL/1.73 m(2) /min. We believe that en bloc transplantation may be an option for children with end-stage kidney disease.     

Living donor liver transplantation for pediatric patients with metabolic disorders: The Japanese multicenter registry

LDLT is indicated for a variety of metabolic disorders, primarily in Asian countries due to the absolute scarcity of deceased donor LT. We analyzed data for all pediatric LDLTs performed between November 1989 and December 2010, during which 2224 pediatric patients underwent LDLT in Japan. Of these patients, 194 (8.7%) underwent LDLT for metabolic disorders. Wilson's disease (n = 59; 30.4%) was the most common indication in the patients with metabolic disorders, followed by OTCD (n = 40; 20.6%), MMA (n = 20; 10.3%), and GSD (n = 15; 7.7%). The one‐, five‐, 10‐, and 15‐yr patient and graft survival rates were 91.2%, 87.9%, 87.0%, and 79.3%, and 91.2%, 87.9%, 86.1%, and 74.4%, respectively. Wilson's disease and urea cycle deficiency were associated with better patient survival. The use of heterozygous donors demonstrated no negative impact on either the donors or recipients. With regard to X‐linked OTCD, symptomatic heterozygote maternal donors should not be considered potential donor candidates. Improving the understanding of the long‐term suitability of this treatment modality will require the registration and ongoing evaluation of all patients with inherited metabolic disease considered for LT.     

Transmission of clear cell tumor in a graft liver from cadaveric donor: Case report

Backes AN, Tannuri ACA, de Mello ES, Gibelli NEM, de Castro Andrade W, Tannuri U. Transmission of clear cell tumor in a graft liver from cadaveric donor: Case report. Abstract: Neoplasms in children after organ transplantation are related to the type and intensity of immunosuppression and the donor–recipient serostatus, especially in relation to the Epstein–Barr virus. The patient was a two‐yr‐old female child with biliary atresia who underwent a liver transplantation from a female cadaver donor. Two adults received kidney transplants from the same donor. Nine months after transplantation, one of the adult recipients developed an urothelial tumor in the kidney graft. Imaging tests were repeated monthly in the liver‐transplanted child and revealed no abnormalities. However, one yr and two months after the transplantation, the patient developed episodes of fever. At that time, imaging and liver biopsy showed a clear cell tumor of urothelial origin in the graft and the disease was limited to the liver. The patient underwent liver retransplantation, and she is currently free of tumor recurrence. Although rare, the occurrence of tumors in the post‐transplant period from cadaver donors, without previously diagnosed tumors, is one of the many problems encountered in the complex world of organ transplantation.     

Current role of liver transplantation for methylmalonic acidemia: a review of the literature

Abstract:  To evaluate the current role of liver transplantation (LT) for methylmalonic acidemia (MMA), we reviewed the literature on outcomes of this treatment, and describe three of our own cases of living-donor liver transplantation (LDLT). The total number of LT cases identified was 18. Transplantation mode was deceased donor LT in 12, including five combined liver-kidney transplantations (CLKT) from deceased donors, and LDLT in six. Three hospital mortalities were noted, because of metabolic decompensation, sepsis and aspergillosis. Although mean postoperative serum MMA level decreased to 13.8% ± 9.2% (range 1.25–26.1%) of preoperative levels, four patients (22.2%) had renal insufficiency after isolated LT and three (16.7%) had postoperative neurological disability. Continuing metabolic damage to the kidney and brain may occur even after successful LT. Further evaluation is required to determine the long-term suitability of this treatment modality.     

The current status of pediatric liver transplantation in Mainland China

Zhou J, Shen Z, He Y, Zheng S, Fan J. The current status of pediatric liver transplantation in Mainland China.
Pediatr Transplantation 2010: 14:575–582. © 2010 John Wiley & Sons A/S. Abstract: The aim of this article is to study the current status of pediatric liver transplantation in Mainland China. A total of 337 cases of pediatric liver transplantation enrolled in CLTR between 1993 and May 2009 were analyzed retrospectively. The median transplant age was 8.7 yr (64 day–17.8 yr), and Wilson’s disease was the most common indication (35.4%). Liver transplantation for biliary atresia accounted for 49.3% and 54.2% in 2008 and 2009 and had become the most common indication nowadays. One‐ and three‐yr survival rates of children transplanted at age<1 yr were 69.2% and 59.3%, respectively, and were significantly worse than those transplanted at age≥1 yr (83.9% and 76.6%, p < 0.05).In 63.8% (208/326) of the patients, LDLT was used with an overall one‐ and three‐yr survival rates of 87.5% and 84.4%, respectively. The one‐ and three‐yr survival for DDLT was significantly lower (66.7% and 52.2%, p < 0.05). The one‐ and three‐yr survival rates for those transplanted in era 1993–2000 were 63.6% and 36.4%, respectively, and the one‐ and two‐yr survival rates in the latest era (2007‐2009) were markedly improved (81.9% and 79.0%, p < 0.05). Cox’s analysis identified DDLT (HR = 2.90, CI 95% 1.5–5.6), being transplanted in era 1993–2000(HR = 3.4, CI 95% 1.1–10.2), fulminant liver failure (HR = 6.0, CI 95% 2.0–17.5), and malignancy (HR = 3.8, CI 95% 1.4–10.3) as predictors of increased mortality, and children transplanted at age 8–17 yr have an better survival (HR = 0.2, CI 95% 0.1–0.6). We concluded that pediatric liver transplantation is gradually developing and would probably be a promising therapy for pediatric end‐stage liver diseases in Mainland China.     

Pediatric liver transplantation for acute liver failure at a single center: A 10‐yr experience

Heffron TG, Pillen T, Smallwood G, Rodriguez J, Sekar S, Henry S, Vos M, Casper K, Gupta NA, Fasola CG, Romero R. Pediatric liver transplantation for acute liver failure at a single center: A 10‐yr experience.
Pediatr Transplantation 2010:14:228–232. © 2009 John Wiley & Sons A/S. Abstract: Children transplanted for ALF urgently require an optimal graft and have lower post‐transplant survival compared with children transplanted for chronic liver disease. Over 10 yr, 33 consecutive children transplanted for ALF were followed. Demographics, encephalopathy, intubation, dialysis, laboratory values, graft type ABOI, XL (GRWR > 5%), DDSLT, LDLT and WLT were evaluated. Complications and survival were determined. ALF accounted for 33/201 (16.4%) of transplants during this period. Twelve of 33 received ABOI, five XL grafts, 18 DDSLT, and three LDLT. Waiting time pretransplant was 2.1 days. One‐ and three‐yr patient survival in the ALF group was 93.4% and 88.9%, and graft survivals were 86.4% and 77.7%. Median follow‐up was 1452 days. ABOI one‐ and three yr patient and graft survival in the ALF was 91.6% and 78.6%. No difference in graft or patient survival was noted in the ALF and chronic liver disease group or the ABOI and the ABO compatible group. A combination of ABO incompatible donor livers, XL grafts, DDSLT, LDLT and WLT led to a short wait time and subsequent graft and patient survival comparable to patients with non‐acute disease.     

Pediatric renal transplantation: single center experience

Although renal transplantation (RTx) is actually the first choice of treatment for children with end-stage renal disease, the number of transplanted children remains low in comparison with adults. The experience of the individual pediatric transplant center is very important in the outcome of pediatric transplant recipients. In this study, our pediatric renal transplantation experience is presented. We retrospectively analyzed the results of 72 pediatric renal transplants performed at Ege University Pediatric Nephrology Transplantation Center between June 1989 and May 2003. They were 40 girls, 32 boys and their mean RTx age was 13.27+/-3.73 yr (range 3-20 yr). Thirty-eight (52.8%) of the transplanted kidneys came from a living related donor, and 34 (47.2%) from a cadaveric donor. Preemptive RTx was performed in one patient and a second RTx was performed in one patient after two-period hemodialysis. Hypertension (31.9%), acute rejection (27.8%) and chronic rejection (13.9%) were the most common complications. Cytomegalovirus (CMV) infection occurred in 15 children (20.8%), none of whom died or lost their graft as a result of the infection. Pretransplant acquired hepatitis C virus (HCV) infection was detected in 12 patients (16.7%). Urinary tract infections (UTIs) were seen in 31 (43.1%) recipients. The 1, 5 and 10 yr graft survival rates were 91, 84 and 77%, respectively, and corresponding patient survival rates were 97, 84 and 77%, respectively by Kaplan-Meier method. The graft and overall survival was not correlated with sex, donor type, treatment modality, acute rejection episodes, hypertension, UTIs, CMV and HCV infection.     

Pediatric post‐transplant metabolic syndrome: New clouds on the horizon

Liver transplantation (LT) is a standard treatment for children with end‐stage liver disease, standing at more than 90% survival rate after one yr, and at over a 70% survival rate after five yr. The majority of transplanted children enjoy an excellent quality of life but complications can occur in the long term, and can develop subclinically in otherwise well children; there are various underestimated nutritional and metabolic aspects, including the so‐called post‐transplant metabolic syndrome (PTMS). During the post‐transplant period, the use of immunosuppressants, corticosteroids, calcineurin inhibitors, and the presence of risk factors, including non‐alcoholic fatty liver disease (NAFLD), and kidney and bone complications have been largely implicated in PTMS development. Strategies to reduce the progression of PMTS should include careful screening of patients for diabetes, dyslipidemia, and obesity, and to support weight reduction with a carefully constructed program, particularly based on diet modification and exercise. With early identification and appropriate and aggressive management, excellent long‐term health outcomes and acceptable graft survival can be achieved.     

Heart transplantation in children and infants: Short-term outcome and long-term follow-up

Since 1988, 82 heart transplants have been performed in 80 infants and children. Diagnoses pretransplant were: hypoplastic left heart syndrome (HLHS) (n = 43); cardiomyopathy (n = 19); endocardial fibroelastosis (n = 6); and other complex congenital heart diseases (n = 12). Age at transplantation was < 1 yr in 61 patients. Overall survival rate was 79% at 1 yr and 73% at 5 and 10 yr. To date, 20 patients have died after transplantation. Causes of death were: rejection (eight patients); right ventricular failure (four patients); transplant coronary artery disease (TCAD) (two patients); and other causes (six patients). In the majority of patients somatic growth is not impaired, and renal function is reduced (but stable) in all patients. Two patients developed post-transplant lymphoproliferative disease, which was treated successfully. Major long-term morbidity is neurologic deficit - severe in three patients and minor in six. TCAD was present or suspected in six surviving patients. We conclude that heart transplantation in infants and children can be performed with good early and late results. Quality of life is excellent in most patients. TCAD, however, will become an increasing problem in the long term.     

A single center experience of donation after cardiac death liver transplantation in pediatric recipients

Bartlett A, Vara R, Muiesan P, Mariott P, Dhawan A, Mieli‐Vergani G, Rela M, Heaton N. A single center experience of donation after cardiac death liver transplantation in pediatric recipients.
Pediatr Transplantation 2010:14: 388–392. © 2009 John Wiley & Sons A/S. Abstract: Many centers are now performing DCD adult LT. There has been a reluctance to transplant pediatric recipients with DCD livers due to concern over the medium to long‐term outcome. We describe the outcome of 14 children (median age seven yr, 8 months–16 yr) that underwent LT with DCD grafts from July 2001 to December 2007. Donors had a median age of 23 yr (10–64), intensive care stay of five d (2–14) and bilirubin of 9 mmol/L (6–60). Median warm and cold ischemic time was 16 min (11–29) and seven h (5.5–8.4). Livers were transplanted as a whole organ (4), reduced graft (8), formal split (1) or auxiliary transplant (1). Compared to DBD recipients AST was significantly higher on the first three post‐operative days and there was no difference in the INR, bilirubin or GGT out to 12 months. There were no biliary or vascular complications and patient and graft survival is 100% at a median follow‐up of 41.8 months (1.7–74 months). LT with DCD grafts in pediatric recipients can be performed with low morbidity and excellent short‐to‐medium term patient and graft outcome.     

Glycogen storage disease type I: indications for liver and/or kidney transplantation

Even though significant progress has been achieved in the management of patients with glycogen storage disease type I, hepatic (mainly adenomas) and renal (proteinuria, renal failure) complications may still develop. Orthotopic liver transplantation has been reported in less than 20 patients, and, in most cases, its indications were multiple hepatic adenomas, sometimes combined with poor metabolic control and/or growth retardation. Even though short-term outcome seems to be favourable, long-term complications have been reported in several cases. Thus it appears that improved metabolic control has to be attempted before performing liver transplantation in such patients. As for renal transplantation, it has been performed in patients with terminal renal failure. It is hoped that improving longterm metabolic control will prevent renal involvement from evolving to terminal renal failure. Finally, combined liver and kidney transplantation may be indicated in a few patients.Conclusion: organ (liver/kidney) transplantation in glycogen storage disease type I may be advantageous when long-term metabolic control has been attempted. Nevertheless, post-transplantat longterm complications may still develop. Published online: 19 July 2002