Elsevier

Journal of Clinical Epidemiology

Volume 59, Issue 12, December 2006, Pages 1257.e1-1257.e14
Journal of Clinical Epidemiology

Review Article
Methodological quality of patient-reported outcome research was low in complementary and alternative medicine in oncology

https://doi.org/10.1016/j.jclinepi.2006.03.006Get rights and content

Abstract

Objective

To evaluate the methodological robustness of patient-reported outcomes (PROs) evaluation in complementary and alternative medicine (CAM) randomized controlled trials (RCTs) in oncology.

Study Design and Setting

CAM RCTs with a PRO endpoint were retrieved from a number of electronic databases. CAM interventions were defined according to the five major categories of the National Center for Complementary and Alternative Medicine. The “Minimum Standard Checklist for Evaluating HRQOL Outcomes in Cancer Clinical Trials” was used to assess the quality of the PRO reporting in these trials.

Results

Forty-four RCTs enrolling 4,912 patients were identified: six studies involved alternative medical systems, 14 involved mind body interventions, 15 dealt with biologically-based therapies, seven involved manipulative and body-based methods, and two energy therapies. Eighty-nine percent of studies used a PRO as a primary endpoint and 59% documented PRO missing data. Although 84% of the studies used a validated PRO questionnaire, only 37% stated an a priori hypothesis and 20% addressed clinical significance of the outcomes. Overall, 64% of the studies analyzed exhibited a number of methodological drawbacks.

Conclusions

To facilitate the interpretation of results from such CAM RCTs, investigators are encouraged to pay greater attention to key methodological issues as identified in this study.

Introduction

Health-related quality of life (HRQOL) is now considered an important endpoint in cancer clinical trials and the number of trials incorporating HRQOL outcomes has increased significantly over the last decade [1]. Regulatory authorities such as the Food and Drug Administration as well as major professional organizations such as the American Society of Clinical Oncology consider HRQOL assessment in clinical trials to be a relevant endpoint in cancer clinical trials [2], [3].

At present there is no universally accepted definition of HRQOL. Nevertheless, there is a general consensus that it is multidimensional, encompassing physical, psychological, and social functioning, and that it is patient based [4], [5]. A large number of questionnaires are currently available for assessing the HRQOL of patients with cancer. One-dimensional instruments that only assess a single health domain are generally not considered to be HRQOL measures, as they are too limited in scope [6]. More recently, the term “patient-reported outcome” (PRO) has been introduced into the literature to describe a broader set of parameters that have in common their focus on assessing health outcomes from the patient's perspective [7]. PROs include a wide spectrum of measures, ranging from single-item instruments assessing a specific health domain (e.g., pain or fatigue) to multidimensional HRQOL measures. In this context HRQOL can be seen as a subset of PROs. This is important to keep in mind as this paper is concerned with any PRO, including HRQOL measures.

Although PROs have the potential to provide valuable insights into the treatment and care of patients, there is also evidence that a number of methodological issues still challenge investigators when assessing a PRO in cancer clinical trials [8]. Previous work has highlighted some of the methodological problems and challenges in HRQOL studies conducted as part of clinical trials of conventional treatments in breast, colorectal, prostate, and lung cancer [8], [9], [10], [11], [12]. To date, no such systematic methodological works are available on clinical trials of complementary and alternative medicine (CAM) interventions among cancer patients. There is no unique definition of what CAM is; however, throughout this work we adopted this commonly used acronym as a working compromise. It consists of two aspects: although “complementary” therapies allude to the additional use of these methods to mainstream cancer care, often intended to improve patients' well-being and/or control symptoms, “alternative” therapies are usually promoted for use instead of mainstream care [13], [14]. In general these practices are not considered standard medical approaches and can include a wide number of practices and interventions. Previous evidence has shown the popularity of CAM among cancer patients [15]. Key reasons to use CAM are to alleviate cancer- or therapy-related symptoms or enhance psychosocial well-being and HRQOL. Given this, the main purpose of this research is to evaluate the methodological robustness of PROs evaluation in CAM randomized controlled trials (RCTs) in oncology.

Section snippets

Systematic literature search

Studies included in this review were retrieved from systematic literature searches of CAM studies published between January 2000 and July 2005. MedLine and the Cochrane Central Register of Controlled Trials (CENTRAL) databases were searched for published manuscripts in English. Additional literature was identified via the reference lists of these articles and via contacts with experts in the field. Published conference abstracts were not included, as they typically do not provide sufficient

Results

Based on the eligibility criteria, 44 studies were selected enrolling overall 4,912 cancer patients. Six studies involved alternative medical systems [18], [19], [20], [21], [22], [23], 14 involved mind body interventions [24], [25], [26], [27], [28], [29], [30], [31], [32], [33], [34], [35], [36], [37], [38], 15 dealt with biologically based therapies [39], [40], [41], [42], [43], [44], [45], [46], [47], [48], [49], [50], [51], [52], [53], seven involved manipulative and body-based methods [54]

Discussion

Although earlier systematic reviews have highlighted a number of methodological limitations of the HRQOL components of RCTs of conventional treatments in oncology [10], [11], [12], no such work has been published in the area of CAM. Recent evidence has shown that the quality of reviews of CAM is at least as good as that of conventional medical interventions [66] and that this is particularly the case for the quality of reports of RCTs of CAM interventions [67]. Other studies in CAM have also

Acknowledgments

This research was supported by the CAM-CANCER project “Concerted Action for Complementary and Alternative Medicine Assessment in the Cancer Field” funded by the European Commission (5th Framework Program).

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