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Chronic kidney disease (CKD) is a common but often underdiagnosed condition associated with increased cardiovascular risk, premature mortality, and reduced quality of life. Early detection may prevent disease progression and complications. This rapid review summarises evidence from systematic reviews and clinical guidelines on the effectiveness and recommendations of CKD screening in different populations and places the findings in the context of the Austrian preventive medical check-up programme.

Publication: HTA Project Report No. 167: https://eprints.aihta.at/1571
Contact: Yui Hidaka

Lifileucel (AMTAGVI^®) is an autologous tumour-infiltrating lymphocyte therapy in which T-cells are isolated from the patient’s tissue, expanded ex vivo, and reinfused. The goal of this treatment is to trigger an antitumor response in patients with previously treated, unresectable or metastatic melanoma. In a single-arm Phase 2 multi-cohort study, the pooled analysis of two cohorts showed a complete response rate of 5%, a partial response rate of 26%, and a stable disease rate of 46%. Additional results include an objective response rate of 31.4%, a median overall survival of 13.9 months, and a progression-free survival of 4.1 months. Regarding safety, all patients experienced at least one treatment-emergent adverse event, with thrombocytopenia (76.9%), anaemia (50.0%), and febrile neutropenia (41.7%) being the most frequently observed. Six patients died within 30 days after lifileucel infusion. However, the available evidence is limited as the efficacy of lifileucel was not directly compared with other therapies.

After publication of the HTA report, the marketing authorization application was withdrawn from the EMA by the manufacturer in July 2025 – see https://www.ema.europa.eu/en/medicines/human/EPAR/amtagvi

Publication: Decision Support Document for the Austrian Appraisal Board No. 004: https://eprints.aihta.at/1570
Contact: Sarah Wolf

CAR-T cell therapy, a personalized form of immunotherapy for certain blood cancers, is gaining global relevance. This review analyzed real-world data from 26 studies (2,716 patients) on six EMA-approved CAR-T products. Results show that real-world outcomes are generally comparable to clinical trials, with better results than non-CAR-T treatments. However, due to study limitations and risk of bias, the effectiveness and safety of CAR-T therapy remain uncertain. Longer follow-ups and broader indications offer new insights but highlight the need for more robust evidence.

Teprotumumab (TEPEZZA®) is a human monoclonal antibody targeting the insulin-like growth factor 1 receptor (IGF-1R), blocking its activation and signalling. Thereby, it prevents symptoms of moderate-to-severe thyroid eye disease (TED, Graves´ orbitopathy), including lid retraction, soft tissue involvement, proptosis and diplopia. Currently, teprotumumab is under evaluation by the European Medicines Agency (EMA) for market authorisation and is expected to be approved by the European Commission (EC) in July 2025. Compared to placebo, teprotumumab significantly improved proptosis response, clinical activity score (CAS), and Graves’ ophthalmopathy-specific quality-of-life scores in three clinical studies and one observational study. The most frequent adverse events included muscle spasms, alopecia, nausea and fatigue. Additionally, 12-15% of teprotumumab patients reported (partially irreparable) hearing damage. TED reactivation rates varied from 26% to 29%, and 65.4% of patients experienced a regression. Notably, 33% of patients maintained a sustained response through the 24-month follow-up. The available evidence is limited, as efficacy has only been tested against a placebo, not the standard of care.