Excellent survival after liver transplantation for isolated polycystic liver disease: an European Liver Transplant Registry study.

Patients with end-stage isolated polycystic liver disease (PCLD) suffer from incapacitating symptoms because of very large liver volumes. Liver transplantation (LT) is the only curative option. This study assesses the feasibility of LT in PCLD. We used the European Liver Transplant Registry (ELTR) database to extract demographics and outcomes of 58 PCLD patients. We used Kaplan-Meier survival analysis for survival rates. Severe abdominal pain (75%) was the most prominent symptom, while portal hypertension (35%) was the most common complication in PCLD. The explantation of the polycystic liver was extremely difficult in 38% of patients, because of presence of adhesions from prior therapy (17%). Karnofsky score following LT was 90%. The 1- and 5-year graft survival rate was 94.3% and 87.5%, while patient survival rate was 94.8% and 92.3%, respectively. Survival rates after LT for PCLD are good.

Patients with isolated polycystic liver disease referred to liver centres: clinical characterization of 137 cases.

BACKGROUND AND AIM: Isolated polycystic liver disease (PCLD) is characterized by the presence of multiple cysts in the liver in the absence of polycystic kidneys. The clinical profile of PCLD is poorly defined and we set up a study for the clinical characteristics of PCLD. METHODS: We collected clinical data on 188 PCLD patients (defined as >10 liver cysts) from five tertiary referral centres, and 137 patients were selected for the purpose of this study. We performed molecular analysis of the PCLD associated genes PRKCSH and SEC63 in 91 patients. RESULTS: A total of 118 (86%) patients were female. The majority of patients (88%) had >20 cysts. The median age at diagnosis was 47 years (range 23-84). 37 (41%) patients carried a mutation. Clinical symptoms at presentation were present in 111 (84%) patients. γ-glutamyl transferase was elevated to 1.4 times upper limit of normal (interquartile range 1.0-2.7). The presence of a mutation and female gender predicted a more severe course: female patients were 9 years younger at the time of diagnosis (47 years; range 23-84) and 91% had symptoms (P<0.01); likewise, mutation carriers were younger at presentation (39 years; range 35-48) and 95% of this cohort had symptoms (P<0.01). During follow-up [median 8.2 years (range 0-35)], 10% of untreated and 51% of treated patients developed complications. Mortality in this cohort was 8%, but only 2% died of PCLD-related causes. 58% of patients were treated a median of 2 years (range 0-25) after diagnosis. CONCLUSION: Symptomatic PCLD patients are mainly females. Females and mutation carriers were younger at diagnosis and had a more severe course of disease.

Lanreotide reduces the volume of polycystic liver: a randomized, double-blind, placebo-controlled trial.

BACKGROUND & AIMS: Therapy for polycystic liver is invasive, expensive, and has disappointing long-term results. Treatment with somatostatin analogues slowed kidney growth in patients with polycystic kidney disease (PKD) and reduced liver and kidney volume in a PKD rodent model. We evaluated the effects of lanreotide, a somatostatin analogue, in patients with polycystic liver because of autosomal-dominant (AD) PKD or autosomal-dominant polycystic liver disease (PCLD). METHODS: We performed a randomized, double-blind, placebo-controlled trial in 2 tertiary referral centers. Patients with polycystic liver (n = 54) were randomly assigned to groups given lanreotide (120 mg) or placebo, administered every 28 days for 24 weeks. The primary end point was the difference in total liver volume, measured by computerized tomography at weeks 0 and 24. Analyses were performed on an intention-to-treat basis. RESULTS: Baseline characteristics were comparable for both groups, except that more patients with ADPKD were assigned to the placebo group (P = .03). The mean liver volume decreased 2.9%, from 4606 mL (95% confidence interval (CI): 547-8665) to 4471 mL (95% CI: 542-8401 mL), in patients given lanreotide. In the placebo group, the mean liver volume increased 1.6%, from 4689 mL (95% CI: 613-8765 mL) to 4895 mL (95% CI: 739-9053 mL) (P < .01). Post hoc stratification for patients with ADPKD or PCLD revealed similar changes in liver volume, with statistically significant differences in patients given lanreotide (P < .01 for both diseases). CONCLUSIONS: In patients with polycystic liver, 6 months of treatment with lanreotide reduces liver volume.

Carbohydrate antigen 19-9 is extremely elevated in polycystic liver disease.

BACKGROUND/AIMS: Carbohydrate antigen 19-9 (CA19-9) is used as a biomarker to differentiate benign from malignant gastrointestinal disorders. We examined the value of CA19-9 measurement in polycystic livers after observing high CA19-9 cyst fluid levels in a benign polycystic liver case. METHODS: We determined CA19-9 levels in serum (n=120) and hepatic cyst fluid (n=81), from patients with polycystic livers (n=109) and simple hepatic cysts (n=24). Further, we analysed CA19-9 expression in normal and polycystic liver tissue (n=17). RESULTS: Cyst fluid CA19-9 levels from both polycystic livers and simple hepatic cysts were extremely high (median 91 000 U/ml, range 14-15 870 000 U/ml; median 85 000 U/ml, range 332-1 744 000 U/ml respectively). Serum CA19-9 levels were significantly higher in polycystic liver patients (median 30 U/ml, range 0-1200 U/ml) compared with patients with simple hepatic cysts (median 10 U/ml, range 3-200 U/ml, P=0.0011). Serum CA19-9 levels correlated with those in cyst fluid (r=0.3979, P=0.0399), polycystic liver volume (r=0.3870, P=0.0025) and the size of the largest cyst (simple cysts group; r=0.5319, P=0.0280). Cyst epithelia showed strong CA19-9 expression. Evacuation of cyst fluid in four patients resulted in a dramatic decrease in the serum CA19-9 levels (60-95%). CONCLUSIONS: CA19-9 levels are high in the cyst fluid and serum of polycystic liver disease patients due to production and secretion by cyst epithelia. It does not reflect malignancy in these patients and may be of value as a biomarker for intervention efficiency assessment.

Aspiration-sclerotherapy results in effective control of liver volume in patients with liver cysts.

PURPOSE: To study the extent to which aspiration-sclerotherapy reduces liver volume and whether this therapy results in relief of symptoms. RESULTS: Four patients, group I, with isolated large liver cysts, and 11 patients, group II, with polycystic livers, underwent aspiration-sclerotherapy. Average volume of aspirated cyst fluid was 1,044 ml (range 225-2,000 ml) in group I and 1,326 ml (range 40-4,200 ml) in group II. Mean liver volume before the procedure was 2,157 ml (range 1,706-2,841 ml) in group I and 4,086 ml (range 1,553-7,085 ml) in group II. This decreased after the procedure to 1,757 ml (range 1,479-2,187 ml) in group I. In group II there was a statistically significant decrease to 3,347 ml (range 1,249-6,930 ml, P = 0.008). Volume reduction was 17.1% (range -34.7% to -4.1%) and 19.2% (range -53.9% to +2.4%) in groups I and II, respectively. Clinical severity of all symptoms decreased, except for involuntary weight loss and pain in group II. CONCLUSION: Aspiration-sclerotherapy is an effective means of achieving liver volume reduction and relief of symptoms.

Laparoscopic fenestration of liver cysts in polycystic liver disease results in a median volume reduction of 12.5%.

INTRODUCTION: Patients with polycystic liver disease (PCLD) may develop symptoms due to increased liver volume. Laparoscopic fenestration is one of the options to reduce liver volume and to relieve symptoms. This study was performed to evaluate the safety and efficacy of laparoscopic liver cyst fenestration. PATIENTS AND METHODS: Twelve patients (all female, median age 45 years, range 35-58) with symptomatic PCLD were included between August 2005 and April 2007. Surgical data were recorded, liver volumes were measured on pre- and postoperative computed tomography (CT) scans, and patients completed a validated symptom-based questionnaire pre- and postoperatively. RESULTS: Median preoperative liver volume was 4,854 ml (range 1,606-8,201) and decreased to 4,153 ml postoperatively (range 1,556-8,232) resulting in median liver volume reduction of 12.5% (range +9.5 to -24.7%). Median procedural time was 123.5 min (range 50-318), and median hospitalization period was 3.5 days (range 1-8). Postoperative complications occurred in three patients including biliary leakage, obstruction of inferior vena cava and sepsis, all recovering with conservative management. Patients reported decreased symptoms of postprandial fullness and abdominal distension. CONCLUSION: Laparoscopic fenestration in PCLD patients results in volume reduction of 12.5% and decrease of symptoms.