Charalampia Kyriakou

The European Organisation for the Research and Treatment of Cancer Myeloma module (EORTC QLQ-MY20) assesses health-related quality of life in patients with multiple myeloma (MM). Treatment advances and increased survival rates prompted the conduct of the assessment of the content validity of the QLQ-MY20 and inform modification. Two literature reviews were conducted to generate a list of MM issues and review measurement properties. Patients (n = 93) and healthcare professionals (n = 20), largely across Europe, were interviewed to assess the relevance/importance and identify any missing issues from the list, (inclusive of original MY20 issues). Pre-specified criteria were used to retain important/relevant issues specific to MM and/or MM treatment. A conceptual framework was developed based on the elicited issues. The resulting conceptual framework for 24 provisional items included domains of pain, neuropathy, infection, edema, tiredness, weight loss, ocular, oral, restlessness/agitation, and hair loss. Eleven items assessed issues captured by the original QLQ-MY20. New items aim to capture the current MM disease experience including side effects of novel MM therapies. Preliminary evaluation of psychometric properties and international validation field testing of the proposed 24 item module is planned following debriefing interviews with MM patients to identify/rectify potential problems in administration and to identify missing/redundant concepts.

Background: Treatment options after Bruton’s tyrosine kinase inhibitors (BTKi) failure in Waldenstrom macroglobulinaemia are limited. Methods: We retrospectively analysed the use of bortezomib after BTKi failure in 17 patients who were heavily pre-treated and chemotherapy-exposed at our centre between 2018 and 2025. Results: Reasons for BTKi failure were disease progression (59%) and intolerance (41%). At bortezomib initiation, the median age was 73 years and two patients experienced grade 1–2 neuropathy. The best overall response rate (ORR) was 88%. At a median follow up of 39 months (interquartile range 35–78), median treatment-free survival and overall survival were 18 (95% confidence interval [CI] 13–22) and 22 (95% CI 17–45) months, respectively. Conclusion: Bortezomib may be efficacious in patients who experience BTKi failure.

Waldenström Macroglobulinaemia is an indolent disease which is recognised as contributing to a diverse range of disabling symptoms which can impact quality of life. Little data are available in evaluating patient and healthcare professional views on quality-of-life questionnaires (QLQ) used to evaluate quality of life in Waldenström Macroglobulinaemia. We aimed to assess patient perception of their disease and the relevance of the currently employed QLQs in practice by patients and healthcare professionals. One-hundred-and-twenty patients (22% treatment-naïve) and thirty-four healthcare professionals completed an international survey to understand their perception of the relevance of the currently employed QLQ. Out of 415 questions, 257 questions were classified by patients as relevant. Five themes were discerned as relevant: fatigue, mobility, dyspnoea, paraesthesia, pain in the hands and feet, sleep (physical health limitations); contentment (psychological impact); social and family support. The following were considered irrelevant: lumps or swelling, dry mouth, trouble hearing, sexual dysfunction (physical symptoms); post-traumatic stress (psychological impact); education and training and financial support. Healthcare professionals identified EORTC QLQ-C30 as the most commonly used instruments for Waldenström Macroglobulinaemia trials.

Background: Radiotherapy (RT) is a well‐established treatment modality in multiple myeloma (MM), particularly for skeletal‐related events. However, its role is likely evolving with the introduction of modern systemic treatments. Methods: We conducted a retrospective analysis of a large, single‐centre MM cohort treated with contemporary protocols to describe current patterns of RT use. Results: RT continues to offer clinical benefit, with shifting patterns of use observed compared to historical cohorts. Conclusion: RT remains relevant in the modern MM landscape and may hold increasing value in later treatment stages. We postulate a future role as bridging therapy prior to CAR‐T/bispecific antibody treatment. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission

As multiple myeloma (MM) patients live longer, maintaining quality of life (QOL) has become an important treatment goal. However, the commonly used general quality of life questionnaires (QOLQs) were developed over 20 years ago. In this survey, 224 MM patients and 48 healthcare professionals (HCPs) were asked to grade the relevance and importance of QOL items from 9 validated, frequently used MM QOLQs. The results from this survey highlighted significant discrepancy between MM patients’ and HCPs’ perception of important and relevant QOL issues. Whilst MM HCPs found all QOL items relevant, the patients reported a proportion of these items being relevant. These were mainly related to physical functioning, social/family wellbeing, pain and fatigue. This real-world survey stressed the need for the development of an updated QOLQ that is relevant to patients and current MM therapies.

Introduction: Belantamab mafodotin (belamaf) was the first BCMA‐targeting immunotherapy licensed in myeloma and was available as monotherapy for a fifth or greater line of treatment. Outcomes for patients in the United Kingdom and the Republic of Ireland potentially differ from those of other regions and may illuminate factors predicting response to therapy.Methods and Results: We performed a retrospective study of patients treated with belamaf monotherapy in the United Kingdom and the Republic of Ireland. In our cohort of 88 patients, we saw an overall response rate (ORR) of 60%, a median progression‐free survival (PFS) of 8.7 months and a median duration of response (DoR) of 15.8 months. The spectrum of adverse events was as expected, with 84% (71/85) of patients experiencing toxicity. Eye‐related adverse events were the most common, affecting 66% (56/85), leading to dose reduction or delay in 41% (35/85) and discontinuation in 6% (5/85). We specifically assessed physician decision‐making in the context of ocular side effects and found a relatively high frequency of the drug being administered despite moderate levels of toxicity.Conclusion: Our cohort's ORR is significantly different from those of the DREAMM‐2 and ‐3 trials and other real‐world studies, though a long‐duration response has been reported in other cohorts. Comparative analysis with other real‐world studies did not reveal any significant factors predictive of ORR. The frequent administration of belamaf to patients with eye disease may well reflect a more pragmatic approach than was originally prescribed in the landmark trials.Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.