Measuring Outcomes in Randomized Prospective Trials in Palliative Care

Northwestern University, Evanston, Illinois, United States
Journal of Pain and Symptom Management (Impact Factor: 2.8). 08/2007; 34(1 Suppl):S7-S19. DOI: 10.1016/j.jpainsymman.2007.04.004
Source: PubMed


Palliative care aims to improve the quality of life of patients and their families and reduce suffering from life-threatening illness. In assessing palliative care efficacy, researchers must consider a broad range of potential outcomes, including those experienced by the patient's family/caregivers, clinicians, and the health care system. The purpose of this article is to summarize the discussions and recommendations of an Outcomes Working Group convened to advance the palliative care research agenda, particularly in the context of randomized controlled trials. These recommendations address the conceptualization of palliative care outcomes, sources of outcomes data, application of outcome measures in clinical trials, and the methodological challenges to outcome measurement in palliative care populations. As other fields have developed and refined methodological approaches that address their particular research needs, palliative care researchers must do the same to answer important clinical questions in rigorous and credible ways.

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    • "Methodological challenges include identifying and recruiting a sample that reflects the heterogeneous seriously ill population that can be served by palliative care (McWhinney et al., 1994; Grande, 2000; Cook et al., 2002; McMillan & Weitzsner, 2003; Bakitas et al., 2006), finding tools that adequately measure targeted outcomes (Mularski et al., 2007), and finding analytical strategies that account for the attrition that can be expected when studying persons near the end of life (McWhinney et al., 1994; Jordhoy et al., 1999; Pickering , 2002). Few interventions are standardized and occur in the context of other medical interventions (Rinck et al., 1997; McMillan & Weitzsner, 2003). "
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    ABSTRACT: There is a paucity of randomized controlled trials (RCTs) to evaluate models of palliative care. Although interventions vary, all have faced a variety of methodological challenges including adequate recruitment, missing data, and contamination of the control group. We describe the ENABLE II intervention, methods, and sample baseline characteristics to increase intervention and methodological transparency, and to describe our solutions to selected methodological issues. Half of the participants recruited from our rural U.S. comprehensive cancer center and affiliated clinics were randomly assigned to a phone-based, nurse-led educational, care coordination palliative care intervention model. Intervention services were provided to half of the participants weekly for the first month and then monthly until death, including bereavement follow-up call to the caregiver. The other half of the participants were assigned to care as usual. Symptoms, quality of life, mood, and functional status were assessed every 3 months until death. Baseline data of 279 participants were similar to normative samples. Solutions to methodological challenges of recruitment, missing data, and "usual care" control group contamination are described. It is feasible to overcome many of the methodological challenges to conducting a rigorous palliative care RCT.
    Palliative and Supportive Care 04/2009; 7(1):75-86. DOI:10.1017/S1478951509000108 · 0.98 Impact Factor
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    • "Frequently encountered problems include recruitment and attrition, insufficient numbers of patients for any comparison, clinical heterogeneity between patients (condition palliated, comorbidity), heterogeneity in treatments (intervention, dose, duration), different outcomes reported, and use of non-standard scales. A palliative care Outcomes Working Group has recently made recommendations on outcomes they consider to be important in this context and how they might be sought in clinical trials [4]. "
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    ABSTRACT: In contemporary medical research, randomised controlled trials are seen as the gold standard for establishing treatment effects where it is ethical and practical to conduct them. In palliative care such trials are often impractical, unethical, or extremely difficult, with multiple methodological problems. We review the utility of Cochrane reviews in informing palliative care practice. Published reviews in palliative care registered with the Cochrane Pain, Palliative and Supportive Care Group as of December 2007 were obtained from the Cochrane Database of Systematic Reviews, issue 1, 2008. We reviewed the quality and quantity of primary studies available for each review, assessed the quality of the review process, and judged the strength of the evidence presented. There was no prior intention to perform any statistical analyses. 25 published systematic reviews were identified. Numbers of included trials ranged from none to 54. Within each review, included trials were heterogeneous with respect to patients, interventions, and outcomes, and the number of patients contributing to any single analysis was generally much lower than the total included in the review. A variety of tools were used to assess trial quality; seven reviews did not use this information to exclude low quality studies, weight analyses, or perform sensitivity analysis for effect of low quality. Authors indicated that there were frequently major problems with the primary studies, individually or in aggregate. Our judgment was that the reviewing process was generally good in these reviews, and that conclusions were limited by the number, size, quality and validity of the primary studies.We judged the evidence about 23 of the 25 interventions to be weak. Two reviews had stronger evidence, but with limitations due to methodological heterogeneity or definition of outcomes. No review provided strong evidence of no effect. Cochrane reviews in palliative care are well performed, but fail to provide good evidence for clinical practice because the primary studies are few in number, small, clinically heterogeneous, and of poor quality and external validity. They are useful in highlighting the weakness of the evidence base and problems in performing trials in palliative care.
    BMC Palliative Care 02/2008; 7(1):13. DOI:10.1186/1472-684X-7-13 · 1.78 Impact Factor

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