An assessment of Patient Reported Outcome (PRO) clinical trial data reporting, specific to Cholangiocarcinoma (CCA)

Objectives

Patient reported outcome (PRO) data from clinical trials are often underreported or reported inconsistently (Calvert et al, 2013). We investigate the extent to which data from PRO instruments used to measure quality of life (QoL) are reported from clinical trials, focusing on trials of patients with cholangiocarcinoma (CCA).

Methods

Using the ClinicalTrials.gov database, a search for PROs in CCA was conducted, exploring the number of clinical trials with published PRO data. Our search strategy comprised of two filters: “conditions: cholangiocarcinoma”, and “other terms: quality of life”, targeting interventional studies in CCA, and including conditions stated as CCA/bile duct cancer. Full publications were identified for completed studies, either directly from the trials registry or via PubMed or Google Scholar search tools using identified clinical trial numbers.

Results

The search identified 41 interventional trials of CCA, of which 12 (29%) were reported as ‘complete’ (conducted from 2009 to 2016), 16 (39%) ‘ongoing’ and the remaining 13 (32%) ‘on-hold’, ‘terminated’, ‘withdrawn’ or of ‘unknown’ status. Over half (n=24, 59%) listed the PROs included, describing EORTC QLQ-C30 (n=16), EORTC QLQ-BIL21 (n=5), EQ-5D (n=5), FACT-HEP (n=2) and PGI-C/PGI-S (n=1). The majority of trials (29, 71%) positioned PROs as secondary endpoints, and 7 (17%) as the primary endpoint. For the 12 completed trials with published data to-date, less than half (5, 42%) reported QoL findings, and only one trial positioned PRO as the primary endpoint.

Conclusions

In the CCA clinical trials completed and reported to-date, PRO findings are scarce; this is problematic, especially in rare diseases, where there is paucity of published QoL data. Whilst it will be important to continue to follow publications from the completed trials, the current lack of PRO data limits the information available to healthcare professionals to inform clinical practice, and to guide researchers on the design of future protocols.