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Polycystic livers occur in the setting of two inherited conditions: (1) autosomal dominant polycystic kidney disease (ADPKD), also characterised by progressive development of renal cysts resulting in loss of renal function, and (2) polycystic liver disease (PCLD), with a polycystic liver as the sole manifestation. Symptoms, such as abdominal distension, result from hepatomegaly. Ascites is a rare complication and might be due to portal hypertension or caused by a ruptured cyst.
Treatment strategies for polycystic livers are aimed at reducing liver volume. So far, only surgical options are available, but these are associated by considerable morbidity and mortality.1 Alternatively, ascites in the setting of polycystic livers might respond to endovascular stent placement in the narrowed caval vein,2 but medical options are conspicuously lacking so far. We wish to report two cases with severe polycystic liver disease that were successfully treated with somatostatin analogues.
A 53-year-old woman with ADPKD complicated by a polycystic liver presented with abdominal pain and increased abdominal distension. Her renal function was normal. On physical examination she had hepatomegaly. An abdominal CT scan demonstrated ascites, a polycystic liver and a ruptured liver cyst. She was treated with diuretics (spironolactone 25–50 mg/day and furosemide 40 mg/day), but ascites progressed with concomitant increase of abdominal distension and onset of orthopnoea. Drainage of ascites (7 litres, serum ascites albumin gradient of 23.8 g/l) yielded only temporary relief. As no other therapeutic options were available, octreotide (50 μg/h intravenously) was started 9 months after the onset of ascites. Within days, the patient improved and we switched to octreotide 100 μg subcutaneously (three times a day). After 1 month, her abdominal distension had disappeared and diuretics could be stopped. Three months after start of octreotide treatment, the patient was free of abdominal pain. 3D-volumetry of the polycystic liver was performed using Pinnacle3 (version 8.0d, Philips, Eindhoven, The Netherlands) according to previously published methods.3 A CT scan 112 days after the start of octreotide demonstrated a decrease of liver volume from 4609 ml to 2843 ml (38.3%) and of kidney volume (left, from 246 ml to 240 ml (2.4%); right, from 554 ml to 455 ml (17.9%)) (fig 1).
The second patient, a 43-year-old woman with PCLD, developed ascites and oedema due to inferior caval vein syndrome, 11 days after laparoscopic fenestration of liver cysts. Her weight had increased from 79 to 94 kg. In order to relieve the caval pressure, three cysts were aspirated and 14.5 litres of ascites was drained, without much benefit. One month after surgery, we started her on octreotide (100 μg three times a day subcutaneously) and 12 days later we switched her to lanreotide (120 mg once a month subcutaneously). 3D-volumetry of the polycystic liver 230 days after start of treatment demonstrated a volume decrease of 14.9% (from 8232 ml to 7004 ml). Renal function remained unchanged over this time period (creatinine previously 42 μmol/l, and after 46 μmol/l).
Both patients developed intractable ascites secondary to polycystic liver disease. While liver transplantation was considered, we decided to start somatostatin analogues first. In both patients, this resulted in a dramatic clinical improvement, disappearance of ascites and a decrease of liver volume.
Cyst growth is modulated by cAMP, which stimulates cholangiocyte proliferation and cyst fluid secretion, a process which is inhibited by somatostatin.4 In an animal model for polycystic liver disease, octreotide reduced hepatic cyst volume in a dose-dependent manner.5 In ADPKD, octreotide inhibited renal cyst growth by ∼55%, but hepatic cyst volume was not evaluated in this trial.6
How does this effect compare with other therapeutic modalities for polycystic livers? We evaluated aspiration sclerotherapy and laparoscopic fenestration for this indication and found that these achieve a volume reduction of 19.2% and 12.5%, respectively.3 7
Although somatostatin reduces the hepatic venous pressure gradient, this alone would be insufficient to ameliorate the intractable ascites.8 Therefore, we surmise that the therapeutic effect of octreotide results from direct reduction of hepatic cyst volume. In addition, this report implies that cystic fluid accumulation is a dynamic process which can be reversed by somatostatin analogues.
Competing interests: None.
Patient consent: Obtained.
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