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Background: We have previously reported that the safety and efficacy of ipilimumab in real-world patients with metastatic melanoma were comparable to clinical trials. Few studies have explored health-related quality of life (HRQL) in real-world populations receiving checkpoint inhibitors. This study reports HRQL in real-world patients receiving ipilimumab and assesses the prognostic value of patient-reported outcome measures., Patients and Methods: Ipi4 (NCT02068196) was a prospective, multicentre, interventional phase IV trial. Real-world patients (N = 151) with metastatic melanoma were treated with ipilimumab 3 mg/kg intravenously as labelled. HRQL was assessed by the European Organisation of Research and Treatment of Cancer Quality of Life Questionnaire at baseline and after 10-12 weeks., Results: The European Organisation of Research and Treatment of Cancer Quality of Life Questionnaire was completed by 93% (141/151 patients) at baseline, and by 82% at 10-12 weeks. Poor performance status and elevated C-reactive protein (CRP) were associated with worse baseline HRQL. Clinically relevant and statistically significant deteriorations in HRQL from baseline to weeks 10-12 were reported (P <0.05). Baseline physical functioning [hazard ratio (HR) 1.96, P = 0.016], role functioning (HR 2.15, P <0.001), fatigue (HR 1.60, P = 0.030), and appetite loss (HR 1.76, P = 0.012) were associated with poorer overall survival independent of performance status, lactate dehydrogenase (LDH), and CRP. We further developed a prognostic model, combining HRQL outcomes with performance status, LDH, and CRP. This model identified three groups with large and statistically significant differences in survival., Conclusions: Systemic inflammation is associated with impaired HRQL. During treatment with ipilimumab, HRQL deteriorated significantly. Combining HRQL outcomes with objective risk factors provided additional prognostic information that may aid clinical decision making., Competing Interests: Disclosure EA, KDJ, KTH, KH, SK, SA, and TKG have received clinical research support from the Norwegian Ministry of Health and Care Services. MN has received personal fees and honoraria for lectures and expert meetings from Bristol-Myers Squibb, Merck Sharpe and Dohme, Novartis, and Pierre Fabre. JAK has received honoraria for lectures and clinical research support from the Norwegian Ministry of Health and Care Services, Bristol-Myers Squibb, and Roche. OH reports honoraria for lectures from Bristol-Myers Squibb and Merck Sharpe and Dohme and research funding from Novartis. CK reports former employment with Roche. The remaining authors have declared no conflicts of interests. Data sharing The datasets generated during and analysed during the current trial are available from the corresponding author on reasonable request. Ethics approval and consent to participate The Ipi4 trial (https://clinicaltrials.gov/ct2/show/NCT02068196) was approved by the Regional Committee for Medical and Health Research Ethics South East (REC ID 2013/1518) and conducted in accordance with the ethical principles of the Declaration of Helsinki (1964). All patients provided written informed consent to participate in the trial., (Copyright © 2022 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Ipilimumab was the first treatment that improved survival in advanced melanoma. Efficacy and toxicity in a real-world setting may differ from clinical trials, due to more liberal eligibility criteria and less intensive monitoring. Moreover, high costs and lack of biomarkers have raised cost-benefit concerns about ipilimumab in national healthcare systems and limited its use. Here, we report the prospective, interventional study, Ipi4 (NCT02068196), which aimed to investigate the toxicity and efficacy of ipilimumab in a real-world population with advanced melanoma. This national, multicentre, phase IV trial included 151 patients. Patients received ipilimumab 3 mg/kg intravenously and were followed for at least 5 years or until death. Treatment interruption or cessation occurred in 38%, most frequently due to disease progression (19%). Treatment-associated grade 3 to 4 toxicity was observed in 28% of patients, and immune-related toxicity in 56%. The overall response rate was 9%. Median overall survival was 12.1 months (95% CI: 8.3-15.9); and progression-free survival 2.7 months (95% CI: 2.6-2.8). After 5 years, 20% of patients were alive. In a landmark analysis from 6 months, improved survival was associated with objective response (HR 0.16, P = .001) and stable disease (HR 0.49, P = .005) compared to progressive disease. Poor performance status, elevated lactate dehydrogenase and C-reactive protein were identified as biomarkers. This prospective trial represents the longest reported follow-up of a real-world melanoma population treated with ipilimumab. Results indicate safety and efficacy comparable to phase III trials and suggest that the use of ipilimumab can be based on current cost-benefit estimates., (© 2021 The Authors. International Journal of Cancer published by John Wiley & Sons Ltd on behalf of UICC.)

Background: The introduction of high-dose treatment with autologous stem cell support (HMAS) in Norwegian regional hospitals in the early 1990s was controversial. Concerns that low numbers of patients would lead to unacceptably low quality were expressed., Material and Methods: We present treatment results in the health region of Middle Norway, based on nearly 10 years of experience and 100 treated patients. Myeloma results are compared to the results from other Norwegian regional hospitals., Results and Interpretation: Overall survival for multiple myeloma after HMAS (median 6.8 years) was not significantly different in middle Norway compared to the rest of the country, and comparable with published results. Treatment-related mortality was low (1.2%). Results and complications in malignant lymphoma, breast cancer or germ cell tumours are described. HMAS can be satisfactorily given in a regional hospital with relatively few patients.