Developing a Disease-Specific Health-Related Quality of Life Instrument for Patients with Soft Tissue Sarcoma: A Proof-of-Concept Study

Sarcomas represent a rare, heterogeneous group of malignancies arising from bone and soft tissue. Advances in treatment has resulted in increased survival rates for these patients, and thus, patients with low grade soft tissue sarcoma (STS) are often treated with curative intent. With survival comes consideration of other patient outcomes, as treatment for STS can have a significant impact on a patient’s health-related quality of life (HRQoL). HRQoL is measured using patient-reported HRQoL instruments. HRQoL instruments can be generic or specific to a particular disease. The latter are either standalone, or are diseasespecific modules used in conjunction with a core, generic instrument. HRQoL research in patients with STS has been sparse in part due to the rarity and complexity of the disease, and in part due to the lack of a disease-specific HRQoL instrument for use with patients with STS. To address this gap, the aims of this dissertation were to: 1. Review the literature reporting on HRQoL issues in patients with sarcoma, 2. Elicit and analyse patient and clinician perspectives on how being diagnosed with, and treated for STS affects patients’ HRQoL, 3. Create a conceptual framework depicting HRQoL in patients with STS, 4. Develop a HRQoL instrument for patients with STS based on the conceptual framework, and 5. Conduct and report on a proof-of-concept study for a potential STS-specific HRQoL instrument. This dissertation reports on the following research activities to address the above aims: 1. A systematic review of HRQoL, psychosocial issues, and unmet health needs research in patients with sarcoma, summarising the findings of 31 peer-reviewed journal articles within a 10-year period. 2. Qualitative interviews with 18 patients with STS eliciting and thematically analysing their perspectives on the impact of their disease on their HRQoL during diagnosis, treatment, and follow-up. 3. A survey of 46 healthcare workers with experience in treating and/or managing patients with STS regarding their perceptions of relevant HRQoL issues for this cohort. 4. The development of a conceptual framework depicting the HRQoL experience for patients throughout diagnosis, treatment, and follow-up with STS by combining the results of the literature review, patient interviews, and healthcare worker surveys. 5. The development of an STS-specific HRQoL instrument based on the conceptual framework for use with patients with STS treated with curative intent as part of their follow-up schedule. 6. Preliminary psychometric validation of the developed STS-specific HRQoL instrument with 97 patients. The systematic review identified 23 different instruments used within the literature to measure HRQoL in patients with sarcoma, with only one instrument being disease-specific. This instrument (the Toronto Extremity Salvage Score) is limited to measuring physical functioning in patients treated for a sarcoma located in a limb with limb salvage surgery. Indepth qualitative research with patients discussing their HRQoL issues was limited to one cohort of patients with bone sarcoma. During interviews, patients discussed how their experience with the healthcare system affected their HRQoL, where issues of misdiagnosis, a delayed diagnosis, and a lack of disease-specific services resulted in feelings of frustration and isolation. Physical immobility, difficulty returning to work, and having to constantly wait for outcomes caused patients to feel they did not have control over their situation, which had negative effects on their HRQoL. Surveys of clinicians identified fear of cancer recurrence, restriction to physical mobility, pain, and difficulty returning to work as the most relevant HRQoL issues for patients with STS. Feeling embarrassed by their disease, difficulty with memory, and shortness of breath were rated among the least relevant issues. These findings were consistent with patients’ views of which HRQoL issues were relevant to them. In the developed conceptual framework, issues of psychosocial distress were present at each disease phase (diagnosis, treatment, and follow-up). If left unmanaged, these issues may compound over the course of a patient’s disease trajectory, and reduce a patient’s HRQoL. While patients with STS experience similar HRQoL issues as patients with cancer more broadly, there were issues that were relevant and unique to this cohort: physical dysfunction, isolation, motivation, ability to participate in work, leisure, and socialising, as well as information needs and medical mismanagement. As such, currently available and validated HRQoL instruments may not adequately capture the HRQoL experience of patients with STS treated with curative intent. A new modular approach to HRQoL measurement (where a disease-specific module is added to an existing generic HRQoL instrument) was trialled and showed good internal consistency. In addition, patients found the questions easy to understand and relevant to their disease experience, and most found reflecting on their HRQoL before their follow-up appointment to be useful. This dissertation discusses new evidence about HRQoL experiences of patient with STS treated with curative intent. Data from the dissertation studies has contributed to recent initiatives to facilitate international collaboration between researchers in this area, thus allowing large scale research projects to investigate treatment options to improve survival for this heterogeneous group of rare malignancies. This evolving collaboration focusing on HRQoL can help us better understand, and therefore improve, HRQoL for patients with STS and sarcoma more broadly.

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