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A cluster-randomised, controlled proof-of-concept study to explore the feasibility and effect of a patient-directed intervention on quality of life in patients with advanced soft tissue sarcoma.

ABSTRACT: INTRODUCTION:Even with evolving and expanding therapeutical options for the treatment of advanced sarcomas over recent years, the balance between efficacy and toxicity still remains a major concern. Moreover, the symptom burden in patients with sarcoma remains high compared with other malignant diseases. It is, therefore, crucial to assess treatment effectiveness not only in terms of disease-related outcomes (eg, overall survival) but also from an individual and patient-centred perspective using the assessment of patient-reported outcomes (PROs). By focusing on PROs as a primary study endpoint, we aim to address key issues for patients with advanced soft tissue sarcoma (STS) undergoing palliative treatment. METHODS AND ANALYSIS:The protocol of the YonLife study describes a multicentre, cluster-randomised, controlled, open-label proof-of-concept study conducted in patients with advanced or metastatic STS treated with trabectedin in seven German hospitals. The primary objective of the study is to exploratively compare overall quality of life between the patients receiving a multidimensional intervention based on individual PROs and those receiving usual supportive treatment. This complex intervention consists of the (1) electronic assessment of PRO, (2) creation of a case vignette based on PRO and clinical data and (3) treatment suggestions based on the discussion of these vignettes in a regularly meeting expert panel. Additionally, the YonLife trial assesses the applicability of a tablet-based assessment of PROs. Patients' and physicians' acceptance and challenges concerning the implementation process will be evaluated. ETHICS AND DISSEMINATION:The YonLife trial has been approved by the Ethics Committee of the University Hospital Dresden as well as by the relevant institutions of each participating centre before patient enrolment. The findings will be reported via relevant peer-reviewed journals as well as through presentation at international conferences. TRIAL REGISTRATION NUMBER:NCT02204111, pre-results.

SUBMITTER: Schuler M

PROVIDER: S-EPMC5734294 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature

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A cluster-randomised, controlled proof-of-concept study to explore the feasibility and effect of a patient-directed intervention on quality of life in patients with advanced soft tissue sarcoma.

Schuler Markus M Richter Stephan S Ehninger Gerhard G Bornhäuser Martin M Hentschel Leopold L

BMJ open 20170630 6

<h4>Introduction</h4>Even with evolving and expanding therapeutical options for the treatment of advanced sarcomas over recent years, the balance between efficacy and toxicity still remains a major concern. Moreover, the symptom burden in patients with sarcoma remains high compared with other malignant diseases. It is, therefore, crucial to assess treatment effectiveness not only in terms of disease-related outcomes (eg, overall survival) but also from an individual and patient-centred perspecti ...[more]

PMID: 28667207

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Project description:BACKGROUND:Alveolar soft part sarcoma (ASPS) is an exceedingly rare and orphan disease, without active drugs approved in the front line. Pazopanib and trabectedin are licensed for sarcoma treatment from second-line, but very little and contradictory data are available on their activity in ASPS. Lacking ongoing and/or planned clinical trials, we conducted a multi-institutional study involving the reference sites for sarcoma in Europe, U.S., and Japan, within the World Sarcoma Network, to investigate the efficacy of pazopanib and trabectedin. MATERIALS AND METHODS:From May 2007, 14 of the 27 centers that were asked to retrospectively review their databases had identified 44 advanced ASPS patients treated with pazopanib and/or trabectedin. Response was evaluated by Response Evaluation Criteria in Solid Tumors 1.1. Progression-free survival (PFS) and overall survival (OS) were computed by Kaplan-Meier method. RESULTS:Among 30 patients who received pazopanib, 18 were pretreated (13 with other antiangiogenics). Response was evaluable in 29/30 patients. Best responses were 1 complete response, 7 partial response (PR), 17 stable disease (SD), and 4 progressions. At a 19-month median follow-up, median PFS was 13.6 months (range: 1.6-32.2+), with 59% of patients progression-free at 1 year. Median OS was not reached.Among 23 patients treated with trabectedin, all were pretreated and evaluable for response. Best responses were 1 PR, 13 SD, and 9 progressions. At a 27-month median follow-up, median PFS was 3.7 months (range: 0.7-109), with 13% of patients progression-free at 1 year. Median OS was 9.1 months. CONCLUSION:The value of pazopanib in advanced ASPS is confirmed, with durable responses, whereas the value of trabectedin appears limited. These results are relevant to defining the best approach to advanced ASPS. IMPLICATIONS FOR PRACTICE:This retrospective study, conducted among the world reference centers for treatment of sarcoma, confirms the value of pazopanib in patients with advanced alveolar soft part sarcoma (ASPS), with dimensional and durable responses, whereas trabectedin shows a limited activity. Alveolar soft part sarcoma is resistant to conventional cytotoxic chemotherapy. Pazopanib and trabectedin are licensed for treatment of sarcoma from second line; in the lack of prospective clinical trials, these results are relevant to defining ASPS best management and strongly support initiatives aimed at obtaining the approval of pazopanib in the front line of the disease.

Project description:IntroductionNon-communicable diseases (NCDs) are the leading cause of death globally. Even though NCD disproportionally affects low-to-middle income countries, these countries including South Africa, often have limited capacity for the prevention and control of NCDs. The standard evidence-based care for the long-term management of NCDs includes rehabilitation. However, evidence for the effectiveness of rehabilitation for NCDs originates predominantly from high-income countries. Despite the disproportionate disease burden in low-resourced settings, and due to the complex context and constraints in these settings, the delivery and study of evidence-based rehabilitation treatment in a low-resource setting is poorly understood. This study aims to test the design, methodology and feasibility of a minimalistic, patient-centred, rehabilitation programme for patients with NCD specifically designed for and conducted in a low-resource setting.Methods and analysisStable patients with cancer, cardiovascular disease, chronic respiratory disease and/or diabetes mellitus will be recruited over the course of 1?year from a provincial day hospital located in an urban, low-resourced setting (Bishop Lavis, Cape Town, South Africa). A postponed information model will be adopted to allocate patients to a 6-week, group-based, individualised, patient-centred rehabilitation programme consisting of multimodal exercise, exercise education and health education; or usual care (ie, no care). Outcomes include feasibility measures, treatment fidelity, functional capacity (eg, 6?min walking test), physical activity level, health-related quality of life and a patient-perspective economic evaluation. Outcomes are assessed by a blinded assessor at baseline, postintervention and 8-week follow-up. Mixed-method analyses will be conducted to inform future research.Ethics and disseminationThis study has been approved by the Health Research and Ethics Council, Stellenbosch University (M17/09/031). Information gathered in this research will be published in peer-reviewed journals, presented at national and international conferences, as well as local stakeholders.Trial registration numberPACTR201807847711940; Pre-results.

Dataset Information

A cluster-randomised, controlled proof-of-concept study to explore the feasibility and effect of a patient-directed intervention on quality of life in patients with advanced soft tissue sarcoma.

Publications

A cluster-randomised, controlled proof-of-concept study to explore the feasibility and effect of a patient-directed intervention on quality of life in patients with advanced soft tissue sarcoma.

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