Interventional arterial chemotherapy versus sorafenib for advanced hepatocellular carcinoma in China: a health economic evaluation of open-label, randomised, phase 3 study

Objectives
This post hoc study aimed to evaluate the cost-effectiveness of hepatic artery infusion chemotherapy (HAIC) with fluorouracil, leucovorin and oxaliplatin (HAIC-FO) compared with sorafenib in patients with advanced hepatocellular carcinoma (HCC). The analysis was conducted from the perspective of Chinese payers.

Design
A cost-effectiveness analysis was performed using a Markov model derived from data obtained in the FOHAIC-1 trial (phase 3 randomised controlled trial; conducted 2017–2020).

Setting
The study was conducted in tertiary care centres in China.

Participants
The study included advanced HCC patients enrolled in the FOHAIC-1 trial. Inclusion criteria followed the trial protocols, with patients stratified by disease severity (including the presence of Vp4 portal vein tumour thrombus (PVTT) and high tumour burden).

Interventions
HAIC-FO (fluorouracil, leucovorin and oxaliplatin) was compared with sorafenib for cost and health outcomes.

Primary outcome measure
The primary outcome was the incremental cost-effectiveness ratio (ICER), calculated as the additional cost per quality-adjusted life year (QALY) gained.

Results
Sorafenib yielded 0.66 QALYs at a cost of $15 011.73, whereas HAIC-FO yielded 1.00 QALY at a cost of $18 470.98. The ICER of HAIC-FO compared with sorafenib was $10 235.56 per QALY, which was below the willingness-to-pay (WTP) threshold of $30 492.00 per QALY. Sensitivity analyses confirmed that HAIC-FO remained cost-effective across variable assumptions, with probabilistic sensitivity analysis showing a 99.9% probability of cost-effectiveness at the WTP threshold. Subgroup analyses demonstrated more favourable ICERs for patients with Vp4 PVTT ($7003.33 per QALY) and those with high tumour burden ($7382.86 per QALY).

Conclusions
HAIC-FO is a more cost-effective treatment for advanced HCC than sorafenib from the Chinese payer’s perspective, particularly in patients with Vp4 PVTT and/or high tumour burden. Further research is needed to explore long-term economic implications and real-world effectiveness data.

Trial registration number
NCT03164382.

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Diagnostic value of BNLF2b antibody, dual-antibody testing and Epstein-Barr virus DNA in nasopharyngeal carcinoma: a prospective cohort study in Hunan Province, China

Objectives
This study evaluates the diagnostic value of the BNLF2b antibody, dual antibody testing and Epstein-Barr virus DNA (EBV-DNA) individually and in combination for nasopharyngeal carcinoma (NPC) detection.

Design
A prospective cohort study.

Setting
The study was conducted at Hunan Cancer Hospital, in a region in China with a high incidence of NPC, between January 2024 and June 2024.

Participants
A total of 350 patients with suspected NPC were enrolled based on clinical suspicion (eg, metastatic cervical lymph nodes or nasopharyngeal abnormalities with non-specific symptoms). The inclusion criteria included age ≥18 years, residency in Hunan Province, and provision of informed consent. The exclusion criteria included prior history of NPC or other head and neck malignancies, severe immunological/systemic diseases and inability to complete diagnostic evaluations.

Primary and secondary outcome measures
Demographic, clinical and biomarker data were collected, including BNLF2b antibody, EBV-DNA and dual antibody testing. Diagnostic performance metrics were calculated against histopathological confirmation as the gold standard. Follow-up assessments were conducted for non-NPC cases.

Results
Among 350 suspected NPC participants, 74 were diagnosed with NPC through biopsy. BNLF2b antibody exhibited the highest sensitivity (83.78%) and specificity (95.65%) among single biomarkers in NPC diagnosis, outperforming dual-antibody testing and EBV-DNA. Combining BNLF2b with dual-antibody testing improved specificity to 99.64%, although with reduced sensitivity (67.57%). NPC-diagnosed participants and those testing positive for BNLF2b or dual antibody biomarkers had a significantly higher prevalence of family history of NPC (p

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Mesenchymal intravenous stromal cell infusions in children with recessive dystrophic epidermolysis bullosa: MissionEB protocol for a randomised, double-blinded, placebo-controlled, two-centre, crossover trial with an internal phase I dose de-escalation phase and open-label extension

Introduction
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe genetic mucocutaneous fragility disorder characterised by chronic blistering, slow wound healing and increased risk of squamous cell carcinoma. Current management options are very limited.

Methods
This is a randomised (1:1), placebo-controlled, double-blinded crossover (A/B) trial with an internal phase I dose de-escalation (4+5 design) in the first 3 months and a 12-month continued treatment follow-on open-label study if 3-month outcome data from the crossover trial indicate safe and beneficial effects. RDEB is a rare condition, so we expect to recruit a maximum of 36 participants based on feasibility and not formal power considerations. Participants aged >6 months and

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Impact of comprehensive genomic profiling and molecular tumour board on costs and access to tailored therapies: real-world observational study

Objective
There is limited evidence on the economic implications of assessing patients’ access to personalised treatments through Comprehensive Genomic Profiling (CGP) and Molecular Tumour Board (MTB), prompting the need to analyse their impact on the cost of the cancer diagnostic journey (from hospital admission to MTB evaluation) and accessibility to personalised therapies.

Design
Retrospective observational cohort.

Setting
Patients discussed from April 2020 to September 2021 by the institutional MTB operating at Fondazione IRCCS Istituto Nazionale Tumori of Milan, an Italian centre of excellence in oncology pertaining to the national health system.

Participants
676 patients focused on: non-small cell lung cancer (NSCLC), cholangiocarcinoma (CCA), pancreatic carcinoma (PC) and gastro-oesophageal carcinoma (GEC). We defined two different scenarios: (1) patients tested with small Next-Generation Sequencing (NGS) panels (≤60 biomarkers) vs (2) patients tested with comprehensive panels ( >60 biomarkers).

Main outcomes and measures
We measured (1) patients’ eligibility to personalised therapies based on genomic data obtained using targeted somatic NGS panels, (2) MTB cost and the overall diagnostic journey cost and (3) the cost to find a patient eligible to access personalised treatments.

Results
Tumour profiling with comprehensive NGS panels improved patients’ eligibility to personalised therapies compared with small panels (NSCLC: 39% comprehensive panel vs 37% small panel; CCA: 43% vs 17%; PC: 35% vs 3%; GEC: 40% vs 0%). The overall diagnostic journey cost per patient was between 3.2K and 7.4K (NSCLC: 7.4K comprehensive panel vs 6.4K small panel; CCA: 4.9K vs 3.7K; PC: 5.8K vs 4.5K; GEC: 4.2K vs 3.2K). MTB discussion accounted for only 2–3% of the diagnostic journey cost per patient (around 113/patient). The cost to find patient eligible for personalised treatments varied significantly according to panel size and tumour setting (NSCLC: 5K comprehensive panel vs 2.8K small panel; CCA: 4.4K vs 4.4K; PC: 5.5K vs 27K; GEC: 5.2K vs not measurable since none of the patients analysed with small NGS panels were eligible).

Conclusions and relevance
MTB discussion of genomic data obtained with NGS comprehensive panels significantly increases patient eligibility to targeted therapies and optimise the cost to find a patient eligible to personalised treatments, mainly for CCA, PC and GEC patients.

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Health economics evaluation of diagnostic strategies for gastro-oesophageal reflux disease with reflux symptoms in China: a modelling study

Objectives
American College of Gastroenterology (ACG) and Chinese expert consensus recommended different algorithmic approaches for the diagnosis of gastro-oesophageal reflux disease (GERD) are not yet defined. We compared the two recommended diagnostic processes using a Chinese population-based health economics analysis.

Methods
Our analysis considered a hypothetical cohort of patients with typical reflux symptoms. We constructed a decision tree model to compare the two recommended diagnostic processes described in ACG clinical guidelines (stratified endoscopy strategy) and Chinese expert consensus (endoscopy-first strategy). The first strategy begins with hazard stratification based on alarm symptoms. Patients with alarm symptoms directly undergo endoscopic examination, while patients without alarm symptoms receive proton pump inhibitors as diagnostic treatment. In the second strategy, all patients with reflux symptoms complete an endoscopic examination. Sensitivity analysis was performed to evaluate a range of cost and probability estimates on costs and health outcomes over a 1-year time horizon from the healthcare system perspective.

Results
The total expected costs were US$122.51 for the stratified endoscopy strategy and US$150.12 for the endoscopy-first strategy. The incremental cost-effectiveness ratio (ICER) comparing the endoscopy-first strategy with the stratified endoscopy strategy was US$440.39 per additional correct case of GERD. The rates of detecting upper gastrointestinal carcinoma of the two strategies were 0.0088 and 0.0120, and the ICER was US$8561.34.

Conclusions
The use of endoscopy for all patients with reflux symptoms was more effective but with an increased cost compared with the strategy recommended in international guidelines.

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