Tailored combinations of tools are the way forward to assess quality of life in rare cancers

Assessing health-related quality of life (HRQoL) issues is considered a ‘must have’ in oncology clinical trials, as it strengthens the quality of research and ensures that patients’ voices are incorporated in their care. Despite this, data from sarcoma trials show that HRQoL measures are underused and under-reported (Crit Rev Oncol Hematol. 2024:197:104345). Also, most trials use generic questionnaires that are not disease-specific and do not cover all HRQoL aspects relevant to rare cancers (Crit Rev Oncol Hematol. 2024:197:104345). “Given the wide heterogeneity of rare cancer types, existing general HRQoL instruments and tools for frequent cancers do not fully capture the unique experiences of different patients and relatively few rare tumour-specific tools have been developed,” says Prof. Bernd Kasper from the Mannheim Cancer Center (MCC), Germany.

Concerns over the lack of relevance of current HRQoL tools were highlighted by research reported at the ESMO Sarcoma and Rare Cancers Congress 2026 (Lugano, 12–14 March). A study from the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group analysed the relevance of items from the EORTC Quality of Life Questionnaire–Core 30 (EORTC QLQ-C30) (Abstract 78MO). Using a subgroup of 31 patients with epithelioid haemangioendothelioma as an example, the items most frequently scored as relevant were related to pain. Qualitative analysis of interviews identified biopsychosocial themes, with predominant issues concerning lack of understanding by family, friends and the wider community, isolation and diagnostic uncertainty.

“The results suggest that the EORTC QLQ-C30 can be used as a basis for HRQoL measures but then should be supplemented with focused items,” notes Kasper. This approach has already been adopted in some trials, such as a phase III trial in desmoid tumours that assessed patient-reported outcomes via selected items from the EORTC-QLQ-30, the Brief Pain Inventory–Short Form and from one of the few validated rare tumour-specific tools, the Gounder–Desmoid Tumor Research Foundation Desmoid Symptom/Impact Scale (GODDESS) (N Engl J Med. 2023;388:898–912).

In a phase I trial in patients with advanced sarcoma, HRQoL domains from the EORTC QLQ-C15-PAL and EORTC QLQ-C30 questionnaires were rated for relevance (Abstract 189P). Overall, 51 of 386 issues were considered relevant and only 6/15 (40%) EORTC QLQ-C15-PAL items met the relevance criteria. Interestingly, the study revealed a distinct HRQoL profile for these patients with advanced-stage disease, primarily concerning hair changes and loss, frustration with illness and loss of work. “Customising measurement tools by combining items from generic, tumour-specific and domain-specific questionnaires may better describe rare cancer patients’ unique needs and experiences and we need to consider this in trial design with rare cancer treatments,” Kasper highlights.

In another study, HRQoL EORTC QLQ-C30 scores were found to be lowest at baseline but then recovered in 521 patients with newly diagnosed sarcoma (Abstract 123P). Clinically meaningful increases in social functioning were seen at 12 months, and by 24 months not only were improvements seen in role functioning, pain, fatigue and insomnia, but levels of physical, role and social functioning were comparable to those in the general population. Kasper notes that these patterns play an important role in informing clinical practice and shared-decision making, along with the timing of supportive care. Worse patient-reported outcomes were observed among patients with bone sarcomas and non-extremity locations, highlighting differences within rare tumour types.

According to Kasper, a major challenge in assessing HRQoL in rare cancers is the scarcity of long-term data. “Going forward, key considerations in combining general and tumour-specific instruments will be to balance the need for focused longitudinal data with the requirement to avoid overburdening patients with too many questions, and to ensure that all tools used are validated for the type of tumour in question,” he concludes.