Liver transplantation in a 7-month-old girl with Caroli's disease

Caroli's disease (including Caroli's syndrome) is a rare autosomal recessive disorder of the liver characterized by diffuse cystic dilatation of the intrahepatic bile ducts. The disease may present at any age and is characterized by recurrent episodes of biliary obstruction, cholangitis, hepaticolithiasis, and liver abscesses. Caroli's syndrome is further associated with congenital hepatic fibrosis and portal hypertension. Patients with recurrent complications or cirrhosis may die because of recurrent infection, portal hypertension, liver failure, or cholangiocarcinoma. Liver transplantation is the treatment of choice for these complicated patients. Here we describe the youngest reported patient with Caroli's syndrome treated successfully using liver transplantation and review the recent literature.     

De novo malignancies following liver transplantation: a case-control study with long-term follow-up

BACKGROUND: Long-term survival data on de novo malignancy are limited following orthotopic liver transplantation (OLT) when compared with controls without malignancies. METHODS: Over a 12 yr period at our institution, 50 of 1043 patients (4.8%) who underwent OLT were identified to have 53 de novo malignancies. The clinical characteristics and survival of these patients were retrospectively reviewed and compared with a control cohort of 50 OLT recipients without malignancy matched with the incidence cases by age, year of OLT, sex, and type of liver disease. RESULTS: Chronic hepatitis C, alcohol and primary sclerosing cholangitis were the three leading causes of liver disease. Skin cancer was the most common malignancy (32%), followed by gastrointestinal (21%), including five small bowel tumors, and hematologic malignancies (17%). The cases and controls were not significantly different in the immunosuppressive regimen (p = 0.42) or the number of rejection episodes (p = 0.92). The five- and 10-year Kaplan-Meier survival rates for the cases were 77% and 34%, respectively, vs. 84% and 70%, respectively, for the controls (p = 0.02 by log-rank test). Patients with skin cancers had survival similar to the controls, but significantly better than non-skin cancers (p = 0.0001). The prognosis for patients with gastrointestinal tumors was poor, with a median survival of 8.5 months after the diagnosis. CONCLUSION: In this single institutional study, de novo malignancies after OLT were uncommon. Patients with non-skin cancer after OLT had diminished long-term survival compared with the controls. Our results differ from other reports in the high incidence of gastrointestinal malignancies with attendant poor prognosis.     

Long-term follow-up after liver transplantation in patients with hepatic iron overload.

Patients with hepatic iron overload who undergo orthotopic liver transplantation (OLT) have a worse 1-year survival than those who undergo transplantation for other indications; the long-term outcome in this population is unknown. The purpose of this study is to report long-term follow-up after OLT in a cohort of patients with hepatic iron overload. Five liver transplant centers in the United States reported follow-up data on 37 patients receiving a first liver transplant who had severe hepatic iron overload in their native livers. Kaplan-Meier 5-year survival among these patients was compared with survival data from all age-matched liver transplantations reported to the United Network for Organ Sharing (UNOS) over the same time period (1987 to 1993). The 5-year survival rate after OLT was 40% in the hepatic iron overload group compared with an overall survival rate of 62% for all patient groups from the UNOS registry (P =.0009). Although sepsis was the cause of 53% of all deaths occurring within the first year after OLT, cardiac complications accounted for 50% of the late mortality in patients with hepatic iron overload. In conclusion, long-term survival after OLT is significantly decreased in patients with hepatic iron overload. Infectious and cardiac complications are the most common causes of death in these patients. Further studies are needed to define the relationship between hepatic iron overload and mortality and to examine the effect of iron depletion on outcome after OLT in this patient population.     

Caroli's disease and outcomes after liver transplantation.

Caroli's disease is a rare autosomal recessive disorder characterized by intrahepatic cystic dilatation of the bile ducts that, when progressive, leads to intrahepatic stones, recurrent cholangitis, portal hypertension, cholangiocarcinoma, and liver failure. Liver transplantation is a promising curative option for advanced Caroli's disease. The aim of this study was to determine the outcomes of liver transplantation in unselected patients with Caroli's disease and recommend an evidence-based therapeutic algorithm for the management of Caroli's disease. Of the 78,124 patients transplanted in the United States between 1987 and 2006, 104 had Caroli's disease; 96 of these underwent liver alone, and 8 underwent combined liver/kidney transplantation. The patient survival and graft survival were analyzed by Kaplan-Meier survival analysis, and risk of death and risk of graft loss were analyzed by Cox proportional hazards regression. The overall 1-, 3-, and 5-year graft (79.9%, 72.4%, and 72.4%) and patient (86.3%, 78.4%, and 77%) survival rates were excellent for patients after liver transplantation. For combined liver/kidney transplantation (n = 8), the 1-year patient survival and graft survival were 100%. Proportional hazards analysis identified Asian ethnicity, elevated bilirubin, requirement of life support or hospitalization prior to transplantation, and a cold ischemia time greater than 12 hours as associated with increased risk of both graft loss and death. A history of prior transplant or prior abdominal surgery was also associated with increased risk of graft loss. In conclusion, liver transplantation is an excellent treatment option for patients with advanced Caroli's disease and should be considered in a timely fashion to prevent worsening complications including refractory cholangitis and cholangiocarcinoma.     

Recurrence of primary sclerosing cholangitis in patients after liver transplantation

BACKGROUND: Primary sclerosing cholangitis (PSC) is a chronic cholestatic disease that progresses to end-stage liver disease. There are several specific problems related to the posttransplantation period in these patients. The aim of this study was to analyze a single center experience with 17 orthotopic liver transplantations (OLT) due to PSC. PATIENTS AND METHODS: Seventeen patients were included (10 men, 7 women). Actuarial patient and graft survival rates and the incidence of recurrent sclerosing cholangitis were determined at 1, 5, and 7 years. RESULTS: Fifteen patients received single grafts, whereas two patients required retransplants. Patients received either cyclosporine (n = 7) or tacrolimus (n = 10) based immunosuppression. The 1-, 5-, and 7-year patient survival rates were 80%, 60%, and 60%, respectively, whereas the graft survival rates were 88%, 65%, and 65%, respectively. Two patients had cholangiocarcinomas (CCA) diagnosed during OLT; both recurred within 6 months and had a fatal outcome. Two patients (12%) developed recurrent sclerosing cholangitis, as assessed by liver histology and imaging of biliary tree. CONCLUSIONS: Liver transplantation provides good patient and graft survival rates in cases affected with PSC. CCA is associated with poor recipient survival. Recurrent PSC occurs in approximately 12% of cases but does not significantly affect patient survival.     

Liver Transplantation Consequential to Caroli's Syndrome: A Case Report

Caroli's disease is a rare condition that includes fibrocystic malformations of the bile duct. It consists of multifocal congenital dilatations of the intrahepatic bile ducts, which may be diffuse or limited, presenting in sack form that produces cystic structures which communicate with the biliary tree. Herein we have presented the case of a 44-year-old woman with recurrent cholangitis consequential to Caroli's syndrome. The distinctive feature of this case was that it was the first and only liver transplantation performed to date for this cause at our center among 700 procedures that had been performed over 19 years. The hepatectomy sample from the liver transplantation showed large cystic dilatations at the level of segments VII and VIII. The pathological study reported congenital dilatation of the intrahepatic bile ducts, associated with congenital hepatic fibrosis (Caroli's syndrome). Caroli's syndrome is a complex association of conditions which usually presents together with polycystic kidney lesions. Orthotopic liver transplantation is still the only therapeutic option for diffuse, uncontrollable cases or those with significant portal hypertension, as well as being the final option in the other cases in the event of a lack of response to other therapeutic options or as an alternative to them.     

Orthotopic liver transplantation for primary sclerosing cholangitis. A 12-year single center experience.

OBJECTIVE: The purpose of this study was to analyze a single center's 12-year experience with 127 orthotopic liver transplantations (OLT) for primary sclerosing cholangitis (PSC). SUMMARY BACKGROUND DATA: Primary sclerosing cholangitis is a chronic cholestatic liver disease of unknown origin that occurs most commonly in young men and is associated frequently (70-80%) with inflammatory bowel disease (IBD). Patients with PSC also are at risk for the development of cholangiocarcinoma (CCA) and those with IBD for colon carcinoma. Although the course of PSC is variable, it frequently is progressive, leading to cirrhosis and requirement for OLT. METHODS: The medical records of 127 consecutive patients undergoing OLT for PSC from July 1, 1984, to May 30, 1996, were reviewed. Actuarial patient and graft survival was determined at 1,2, and 5 years. The incidence and outcome of patients with CCA, recurrent sclerosing cholangitis, and post-transplant colon carcinoma was determined. Results were analyzed by way of stepwise Cox regression to determine the statistical strength of independent associations between pretransplant covariates and patient survival. The median follow-up period was 3.01 years. Incidental cholangiocarcinoma (ICCA) was defined as a tumor < 1 cm in size that was discovered at the time of pathologic sectioning of the explanted liver. RESULTS: Ninety-two patients (72%) had associated IBD. Seventy-nine (62%) had undergone previous biliary tract surgery. One hundred seven patients (84%) received a single graft, whereas 20 patients (16%) required 22 retransplants. Patients received either cyclosporine- (n = 76) or tacrolimus- (n = 51) based immunosuppression. The 1-, 2-, and 5-year actuarial patient survivals were 90%, 86%, and 85%, respectively, whereas graft survival was 82%, 77%, and 72%, respectively. The presence of previous biliary surgery had no effect on patient survival. Ten patients (8%) had ICCA and their survival was not significantly different from patients without ICCA (100%, 83%, and 83% at 1, 2, and 5 years, respectively). Four patients were known to have CCA at the time of OLT, all recurred within 6 months, and had a significantly worse outcome (p < 0.0001). Recurrent sclerosing cholangitis developed in 11 patients (8.6%). The patient and graft survival in this group was not different from those in whom recurrence did not develop (patient; 100%, 90%, and 90%; graft: 80%, 70%, and 52%). Thirty patients (23%) underwent colectomy after liver transplantation for dysplasia-carcinoma or symptomatic colitis. Of the nine covariates entered into the Cox multivariate regression analysis, only common bile duct frozen section biopsy specimen showing CCA was predictive of a survival disadvantage. CONCLUSIONS: Liver transplantation provides excellent patient and graft survival rates for patients affected with PSC independent of pretransplant biliary tract surgery. Incidental cholangiocarcinoma does not affect patient survival significantly. However, known CCA or common duct frozen section biopsy specimen or both showing CCA are associated with poor recipient survival, and OLT should be proscribed in these cases. Recurrent PSC occurs in approximately 9% of cases but does not affect patient survival. Post-transplant colectomy does not affect patient survival adversely.     

Liver transplantation in patients with Caroli's disease and recurrent cholangitis

Caroli's disease is an uncommon congenital disorder of the intrahepatic biliary tree. It is characterized by multiple and segmental dilatations of the bile ducts. The clinical course of Caroli's disease is often complicated by recurrent episodes of bacterial cholangitis that seriously impair the patient's quality of life. Despite wide spectrum antimicrobial agents, medical treatment of cholangitis is frequently unsuccessful in patients with Caroli's disease due to the persistence of bacteria in dilatated bile ducts. Other therapies, including internal or external biliary drainages and various surgical or endoscopic procedures, have been used in the treatment of Caroli's disease, with poor results. There are no previous reports in the literature of liver transplantation for recurrent cholangitis in patients with Caroli's disease. We present two such cases, in which cholangitis is resolved. Received: 16 July 1996 Received after revision: 4 February 1997 Accepted: 5 February 1997     

The role of surgery in Caroli's disease

BACKGROUND: Caroli's disease is a rare congenital disorder characterized by multifocal segmental dilation of the intrahepatic bile ducts. Whether conservative or surgical strategies should be preferred is still a matter of debate. The aim of this study was to evaluate the role of surgery in the management of Caroli's disease. STUDY DESIGN: From April 1998 until August 2005, 12 consecutive patients with Caroli's disease were treated in the Department of General, Visceral, and Transplantation Surgery, University Hospital Essen, Germany. All patients were intended to receive liver resections or liver transplantations. RESULTS: There were seven men and five women, with a median age of 39 years (range 7 months to 70 years). Eight patients had monolobar and four patients had bilobar liver involvement. All patients had a history of recurrent cholangitis, with up to 16 unsuccessful conservative treatment attempts. Nine patients (75%) underwent liver resection and two (17%) had liver transplantation. Intraoperatively, three patients (25%) were found to have cholangiocarcinoma, of which one was unresectable. There was no mortality and only low morbidity (16%) postoperatively. After a median followup of 31 months, 11 patients are well with no recurrent symptoms. CONCLUSIONS: Surgery can offer a definite therapy, with an acceptable morbidity and virtually no mortality in localized Caroli's disease. In diffuse disease, the use of extended resections or liver transplantation can provide good longterm results.     

68例Caroli病的外科治疗

目的 分析Caroli病的诊断及外科治疗效果。方法 回顾分析1996年至2002年期间我院肝胆外科手术治疗的68例Caroli病患者的临床资料。结果 患者男女比例为1：1．35,平均年龄46岁。主要症状为反复发作性胆管炎,26例曾诊断为“胆道结石、胆管炎”而接受过胆道手术。病变局限性于左叶44例,右叶9例,弥漫型15例,合并胆总管囊状扩张20例,癌变率为8．8％。56例（82．4％）实施肝叶切除,术后并发症发生率为15．0％,住院死亡率为0。随访3—10年,肝切除组症状缓解率为90．2％（46／51）,显著高于非肝切除组的33．3％（P〈0．01）。结论 肝切除是Caroli病治愈性的治疗措施,对于无法切除的病例,应考虑肝移植术。     

Post-liver transplant acute renal failure: factors predicting development of end-stage renal disease

BACKGROUND: Acute renal failure (ARF) occurs in 5-50% of patients undergoing orthotopic liver transplantation (OLT). The aim of this study was to determine factors that might predict the development of end stage renal disease (ESRD) in patients who had ARF after OLT. METHODS: We studied all OLT recipients between 9/1/1988 through 12/31/2000. RESULTS: A total of 1602 patients underwent OLT during the study period. About 350 patients (22%) developed ARF requiring dialysis post-operatively. One hundred and twenty-three (39.8%) died within a year after OLT. Median follow up was 5.8 yr (range 1-12 yr). Forty-three patients (23%) developed ESRD over median of 3.79 yr (range 1-8 yr). Multivariate logistic regression analysis revealed creatinine levels > 1.7 mg/dL at 1 yr (p < 0.001), cyclosporine as immunosuppression (p = 0.026), and the presence of diabetes pre-OLT (p < 0.001) to be associated with the development of ESRD. The development of ESRD did not decrease patient survival (p = 0.111). ESRD patients who received subsequent kidney transplantation had significantly improved survival rates (p = 0.005). CONCLUSIONS: Serum creatinine levels at 1 yr, cyclosporine as immunosuppression, and the presence of diabetes pre-OLT are independent predictive factors for the development of ESRD. ESRD patients who received kidney transplantation had higher 10-yr survival rates when compared with patients maintained on dialysis.     

Liver transplantation for Wilson's disease: The burden of neurological and psychiatric disorders.

A retrospective data analysis on liver transplantation for Wilson's disease (WD) was performed among Italian Liver Transplant Centers. Thirty-seven cases were identified. The main indication for liver transplantation was chronic advanced liver disease in 78% of patients. Mixed hepatic and neuropsychiatric symptoms were recorded in 32.3%. Eight patients presented with fulminant liver failure; 44.8% were on medical treatment. Patient and graft survival at 3 months, 12 months, 3 years, 5 years, and 10 years after transplantation were, respectively, 91.8%, 89.1%, 82.9%, 75.6%, and 58.8%, and 85.3%, 83.0%, 77.1%, 70.3%, and 47.2%. Neurological symptoms significantly improved after orthotopic liver transplantation (OLT), but the survival of patients with mixed hepatic and neuropsychiatric involvement was significantly lower than in patients with liver disease alone (P = 0.04). WD characterized by hepatic involvement alone is a rare but good indication for liver transplantation when specific medical therapy fails. Patients with neuropsychiatric signs have a significantly shorter survival even though liver transplantation has a positive impact on neurological symptoms. In conclusion, a combination of hepatic and neuropsychiatric conditions deserves careful neurological evaluation, which should contraindicate OLT in case of severe neurological impairment.     

Surgical Treatment of Hepatocellular Carcinoma beyond Milan Criteria. Results of Liver Resection, Salvage Transplantation, and Primary Liver Transplantation

Background There is no clear consensus regarding the best treatment strategy for patients with advanced hepatocellular carcinoma (HCC). Methods Patients with cirrhosis and HCC beyond Milan who had undergone liver resection (LR) or primary orthotopic liver transplantation (OLT) between November 1995 and December 2005 were included in this study. Pathological tumor staging was based on the American Liver Tumor Study Group modified Tumor-Node-Metastasis classification. Results A total of 23 HCC patients were primarily treated by means of LR, 5 of whom eventually underwent salvage OLT. An additional 32 patients underwent primary OLT. The overall actuarial survival rates at 3 and 5 years were 35% after LR, and 69% and 60%, respectively, after primary OLT. Recurrence-free survival at 5 years was significantly higher after OLT (65%) than after LR (26%). Of the patients who underwent LR, 11 (48%) experienced HCC recurrence only in the liver; 6 of these 11 presented with advanced HCC recurrence, poor medical status, or short disease-free intervals and were not considered for transplantation. Salvage OLT was performed in 5 patients with early stage recurrence (45% of patients with hepatic recurrence after LR and 22% of all patients who underwent LR). At a median of 18 months after salvage OLT, all 5 patients are alive, 4 are free of disease, and 1 developed HCC recurrence 16 months after salvage OLT. Conclusion For patients with HCC beyond Milan criteria, multimodality treatment—including LR, salvage OLT, and primary OLT—results in long-term survival in half of the patients. When indicated, LR can optimize the use of scarce donor organs by leaving OLT as a reserve option for early stage HCC recurrence.     

Curative partial hepatectomy in adult Caroli's disease

Caroli's disease consists of a congenital malformation of the intrahepatic bile ducts characterized by saccular, segmental, cystic dilatations giving rise to recurrent cholangitis. The inheritance pattern is unclear and the disease may be associated with other hereditary diseases such as adult polycystic kidney disease. The most effective therapeutic option in symptomatic unilobar Caroli's disease is resection of the affected lobe. In bilobar involvement, liver transplantation should be considered. Due to their rarity, we report three cases of Caroli's disease successfully resolved through lobectomy of the affected lobe: two patients with recurrent cholangitis and one patient with associated adult polycystic kidney disease.     

肝移植治疗乙肝相关疾病

目的 总结原位肝移植（OLT）治疗乙肝相关疾病的疗效,并评价了拉米夫定对肝移植术后乙肝复发的防治作用。方法 自1993年4月-2000年12月,中山医科大学器官移植中心为54例乙肝相关疾病患者实施了肝移植,其中乙肝坏死后肝硬化17例,为第1组,25例同时合并肝癌者为第2组、其余12例暴发性肝功能衰竭患者为3组。回顾性地分析了3组患者术后存活率、早期死亡原因以及拉米夫定对术后乙肝复发的防治情况。结果 乙肝相关疾病患者肝移植术后早期存活率为75.9%,暴发性肝衰组患者术后早期并发症发生率明显高于其它2组;OLT对小肝癌患者的疗效明显优于大肝癌患者;拉米夫定防治乙肝复发辣效好且未发现副作用。结论 结合拉米夫定,OLT是治疗暴发性乙肝、乙肝肝硬化及小肝癌甚或某些选择性大肝癌患者的有效手段。     

肝移植治疗Caroli病七例报道

目的 探讨肝移植治疗Caroli病的临床效果.方法 回顾性分析1999年9月至2008年2月期间7例弥漫型Caroli病患者行肝移植治疗后的临床资料.对其临床特征、手术方式、并发症类型以及随访情况进行分析.结果 7例患者术前均表现为复发性胆管炎症状并最终确诊为弥散型Caroli病.其中男性3例,女性4例;年龄10～31岁,平均16岁.6例患者肝移植术前接受保守治疗,1例曾行胆囊切除、T型管引流术.4例患者行劈离式肝移植,2例行全肝移植,1例行亲属活体供肝肝移植.其中2例患者术中采用静脉-静脉转流术.手术平均耗时9.1 h.术后并发症包括:肺部感染3例、急性排斥反应2例、胸腔积液2例、肝断面胆汁漏1例.经抗感染、调整免疫抑制方案及用量、胸腔穿刺抽吸及经皮穿刺引流等对症保守治疗后症状得到控制.1例肝移植受者术后19 d死于急性肾功能衰竭合并多器官功能衰竭,其余6例受者均存活.随访发现,存活的6例受者健康状况良好,其中最长存活已达7年.结论 应用肝移植治疗弥散型Caroli病具有良好的临床效果.     

Successful treatment of Caroli''s disease by hepatic resection. Report of six patients.

Caroli's disease is a congenital disease of cystic or saccular dilatation of the intrahepatic bile ducts. There are two disease entities: a simple type and a periportal fibrosis type. Frequent complications with the simple type are recurrent cholangitis, liver abscess, intraductal lithiasis, abdominal pain, and fever that often lead to fatal sepsis. Development of portal hypertension and esophageal varices is usually a final feature of the periportal fibrosis type. Malignancies are also possible complications with Caroli's disease. During the recent 13 years, the author had experiences with eight patients with Caroli's disease of the simple type; six of these eight underwent hepatic resection: right lobectomy in two, left lobectomy in three, and left lateral segmentectomy in one. Other two patients died of sepsis and cholangiocellular carcinoma, respectively. All six patients with hepatic resections were relieved from the disabling symptoms after surgery and have had no recurrent hepatobiliary problems for 3 months to 13 years. Hepatic resection may be indicated for more patients than previously assumed in the treatment of Caroli's disease of the simple type.     

70例原位肝脏移植

目的总结肝脏移植治疗不同终末期肝病的经验,探讨肝移植在治疗肝细胞癌(HCC)和重症乙型肝炎(乙肝)的疗效,以及评价拉米夫定对预防乙肝复发的价值.方法回顾性分析了自1993年4月～2000年12月实施的70例肝移植患者的临床资料.肝移植的主要指征是原发性肝癌(26例)、肝硬化(21例)、重症乙型肝炎(12例)、硬化性胆管炎(4例)以及其它终末期肝病(7例).对12例重症乙型肝炎患者应用拉米夫定治疗.采用多元回归分析确定影响肝移植预后的危险因素.结果54例患者存活1个月以上,16例患者在移植术后30d内死亡,院内存活率为77.1%,肝功能属ChildA级和B级的患者院内存活率明显高于ChildC级患者(P＜0.05),小肝癌患者的疗效优于大肝癌患者.移植前APACEⅢ评分,腹水量以及血肌酐水平与肝移植预后有密切关系.在重症乙肝患者中,9例仍存活、存活时间为2～24个月,拉米夫定可有效地预防肝移植术后乙肝复发,且无明显的副作用.结论本研究结果表明原位肝移植可使部分HCC患者获得治愈,部分病例可获得良好的姑息疗效,病例选择对肝癌肝移植的预后极其重要;结果还提示肝移植是治疗各种终末期肝病的有效手段.同时认为拉米夫定是一种疗效肯定,副作用小的预防肝移植术后乙肝病毒复发的药物.     

Choice of transplantation techniques and indications for liver transplantation in polycystic liver disease in patients with no signs of end-stage liver disease

OBJECTIVE: Since the initiation of the Liver Transplant Program, 500 liver procedures have been performed. Polycystic liver disease (PLD) and polycystic kidney-liver disease (PKLD) have been rare indications for orthotopic liver transplantation (OLT). Only 7 patients (1.4%) underwent transplantation due to PLD and PKLD. MATERIALS AND METHODS: The group consisted of 4 patients who underwent OLT (0.8%) and 3 patients who received simultaneous liver kidney transplantation (LKT; 0.6%). Our objective was to analyze the indications for either OLT or combined LKT as well as indications for surgical techniques during OLT among patients with PLD or PKLD. RESULTS: The main indication for OLT was massive hepatomegaly causing severe physical handicaps, fatigue, and clinically advanced malnutrition. All 3 patients with indications for combined LKT were dialysis-dependent. None of the patients had symptoms of end-stage liver disease and/or hepatic failure. In 4 cases, a portal bypass was applied, and the piggy-back method used in the other 3 cases. The hepatectomy caused no uncommon difficulty. In cases of simultaneous transplantations, the kidney was implanted separately after OLT. All patients are alive following the transplantation; major surgical complications have occurred. CONCLUSIONS: Patients with PLD can undergo OLT safely with good results. They benefit from the relief of abdominal distension and anorexia. Patients with PKLD who are dialysis-dependent should undergo simultaneous LKT. The surgical technique was solely dependent on the intraoperative conditions determined during the dissection phase.     

Primary sclerosing cholangitis: surgical options, prognostic variables and outcome

The natural history of primary sclerosing cholangitis (PSC) is poorly defined and its management remains controversial. Forty-eight symptomatic patients (median age 39 years, range 8-67 years; 30 male) with PSC were reviewed retrospectively. Thirty patients had inflammatory bowel disease. Four patients (8 per cent) developed or had an associated malignancy. Twenty-one (44 per cent) died; overall 5 year actuarial survival was 30 per cent. Twenty-three patients had 27 non-transplant related biliary operations (16 patients specifically for PSC) of whom 12 died. Serum bilirubin was the only parameter to improve after biliary surgery. Seventeen patients (35 per cent) underwent orthotopic liver transplantation (OLT) of whom nine are currently alive (1 year projected survival of 55 per cent). Previous biliary surgery correlated with a poor outcome (P less than 0.0001) after OLT. Being male, presence of cirrhosis, duration of symptomatic disease (greater than 3 years) and a serum bilirubin level greater than 100 mumol/l at presentation, were independently associated with a poor outcome (P less than 0.05). These data provide evidence that PSC is a progressive disease and conventional surgical options have little influence on the outcome. Previous biliary surgery adversely affects outcome following OLT. For progressive liver disease, liver transplantation should be considered the treatment of choice.