Abstract
ObjectiveTo assess whether quantitative assessment of symptom reduction is a better outcome parameter than cyst volume reduction for treatment success in patients treated by aspiration sclerotherapy.MethodsWe included patients with symptomatic, large (> 5 cm), hepatic cysts from a randomized controlled trial (NCT02048319). At baseline and 6 months after treatment, symptoms were assessed with the polycystic liver disease questionnaire (PLD-Q) and we measured cyst volume using ultrasonography. Patient-reported change in health was assessed on a 5-point Likert scale (much worse to much better) after 6 months. We tested whether PLD-Q scores and cyst volumes changed after aspiration sclerotherapy (responsiveness). Changes in PLD-Q scores and cyst volume were compared with change in health as a measure of treatment success (discriminative ability). As secondary analysis, we compared baseline characteristics between responders (improved) and non-responders (not improved).ResultsWe included 32 patients. Six months after treatment, 23 patients (72%) improved. Both PLD-Q score and cyst volume significantly decreased (median 38 to 18 points, p < 0.001, and 479 to 68 mL, p < 0.001). Larger improvement in PLD-Q score was associated with a positive change in health (p = 0.001), while larger proportional reduction in cyst volume was not significantly associated with health improvement after treatment (p = 0.136). Responders had larger baseline cyst volumes compared to non-responders (median 624 mL [IQR 343–1023] vs. 322 mL [IQR 157–423] p = 0.008).ConclusionCyst diameter reduction does not reflect treatment success in aspiration sclerotherapy from patients’ perspective, while symptoms measured with the PLD-Q can be used as a reliable outcome measure.Key Points• Cyst diameter reduction poorly reflects treatment success in aspiration sclerotherapy.• Symptoms measured by the polycystic liver disease questionnaire (PLD-Q) is a better outcome measure than cyst volume reduction for treatment success after aspiration sclerotherapy.• Particularly patients with larger cysts (≥ 529 mL) benefit from aspiration sclerotherapy.
Highlights
Hepatic cysts are fluid-filled cavities varying in volume from a few milliliters to liters [1]
Our study showed that in a population of patients with large hepatic cysts, the polycystic liver disease questionnaire (PLD-Q) and cyst volume were both highly responsive to change after aspiration sclerotherapy
We found that polycystic liver disease (PLD)-Q discriminated between patients who clinically benefited from this therapy or not, while cyst volume changes did not
Summary
Hepatic cysts are fluid-filled cavities varying in volume from a few milliliters to liters [1]. Most are found incidentally as isolated cysts but some patients present with inherited polycystic liver disease (PLD) [2]. Large space-occupying cysts may cause symptoms such as abdominal pain or discomfort [3]. A minimally invasive treatment option for symptomatic large cysts is. Most studies use cyst volume reduction as a rather technical endpoint to define the efficacy of aspiration sclerotherapy [5,6,7,8]. A limited number of studies investigated patient-reported outcome measures such as symptomatic relief, and none of these studies used this as primary endpoint [9]. From a patientcentered view, clinical response after the procedure is the most relevant outcome as it is the patient’s independent perception of symptoms, functioning, and well-being that counts most
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
