Young Women with Polycystic Liver Disease Respond Best to Somatostatin Analogues: a Pooled Analysis of Individual Patient Data
Gevers, Tom Jg × Inthout, Joanna Caroli, Anna Ruggenenti, Piero Hogan, Marie C Torres, Vicente E Nevens, Frederik Drenth, Joost Ph #
Gastroenterology vol:145 issue:2 pages:357-365.e2
BACKGROUND & AIMS: Clinical trials have shown that in patients with polycystic liver disease (PLD), short-term treatment with somatostatin analogues (SAs) reduces liver volumes by 4.5%-5.9%, compared with placebo. However, the effects of SA therapy vary among individuals. We collected data from individual patients with PLD to identify subgroups that benefit most from SA therapy. METHODS: We analyzed data from 107 patients with PLD from 3 randomized placebo controlled trials (67 received SAs, 52 received placebo). We used multiple linear regression analysis to determine the effects of SAs based on patients' age, sex, baseline liver volume, and diagnosis (autosomal dominant polycystic liver disease [PCLD] or kidney disease [ADPKD]). The primary outcome was change in liver volume after 6-12 months of treatment. RESULTS: The effects of SA therapy did not differ significantly among patients with different diagnoses or baseline liver volumes; the overall difference in liver volume between groups receiving SAs therapy vs placebo was 5.3% (P<.001). Among subjects given placebo, young women (≤48 y old) had the greatest increase in polycystic liver volume (4.8%; 95% confidence interval, 2.2%-7.4%), whereas mean liver volumes did not increase in older women and men. Women ≤48 y old had a greater response to therapy (a reduction in liver volume of 8.0%, compared with placebo; P<.001) than older women (a reduction in liver volume of 4.1%, compared with placebo; P=.022). CONCLUSIONS: Based on a pooled analysis of data from individual patients with PLD, treatment with somatostatin analogues is equally effective for patients with ADPKD or PCLD; efficacy does not depend on size of the polycystic liver. Young female patients appear to have the greatest benefit from 6-12 months of SA therapy, which might avert the progressive course of the disease in this specific group.