UNASSIGNED: We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis.
UNASSIGNED: We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence.
UNASSIGNED: The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal cancer but may be cost-effective for the remaining comparisons.
UNASSIGNED: We were unable to obtain individual participant data as planned. The limited number of randomised controlled trials for each comparison and the paucity of data on health-related quality of life mean that the recommendations may change as new evidence (from trials with a low risk of bias) emerges.
UNASSIGNED: In people with peritoneal metastases from colorectal cancer with limited peritoneal metastases and who are likely to withstand major surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should not be used in routine clinical practice (strong recommendation). There is considerable uncertainty as to whether hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy or cytoreductive surgery + systemic chemotherapy should be offered to patients with gastric cancer and peritoneal metastases (no recommendation). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered routinely to women with stage III or greater epithelial ovarian cancer and metastases confined to the abdomen requiring and likely to withstand interval cytoreductive surgery after chemotherapy (strong recommendation).
UNASSIGNED: More randomised controlled trials are necessary.
UNASSIGNED: This study is registered as PROSPERO CRD42019130504.
UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.
Cancers of the bowel, ovary or stomach can spread to the lining of the abdomen (‘peritoneal metastases’). Chemotherapy (the use of drugs that aim to kill cancer cells) given by injection or tablets (‘systemic chemotherapy’) is one of the main treatment options. There is uncertainty about whether adding cytoreductive surgery (cytoreductive surgery; an operation to remove the cancer) and ‘hyperthermic intraoperative peritoneal chemotherapy’ (warm chemotherapy delivered into the lining of the abdomen during cytoreductive surgery) are beneficial. We reviewed all the information from medical literature published until 14 April 2022, to answer the above uncertainty. We found the following from eight trials, including about 1000 participants. In people with peritoneal metastases from bowel cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably does not provide any benefits and increases harm compared to cytoreductive surgery + systemic chemotherapy, while cytoreductive surgery + systemic chemotherapy appears to increase survival compared to systemic chemotherapy alone. There is uncertainty about the best treatment for people with peritoneal metastases from stomach cancer. In women with peritoneal metastases from ovarian cancer who require systemic chemotherapy before cytoreductive surgery to shrink the cancer to allow surgery (‘advanced ovarian cancer’), hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably increases survival compared to cytoreductive surgery + systemic chemotherapy. In people who can withstand a major operation and in whom cancer can be removed, cytoreductive surgery + systemic chemotherapy should be offered to people with peritoneal metastases from bowel cancer, while hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered to women with peritoneal metastases from ‘advanced ovarian cancer’. Uncertainty in treatment continues for gastric cancer. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.

UNASSIGNED: Lynch syndrome is an inherited condition which leads to an increased risk of colorectal, endometrial and ovarian cancer. Risk-reducing surgery is generally recommended to manage the risk of gynaecological cancer once childbearing is completed. The value of gynaecological colonoscopic surveillance as an interim measure or instead of risk-reducing surgery is uncertain. We aimed to determine whether gynaecological surveillance was effective and cost-effective in Lynch syndrome.
UNASSIGNED: We conducted systematic reviews of the effectiveness and cost-effectiveness of gynaecological cancer surveillance in Lynch syndrome, as well as a systematic review of health utility values relating to cancer and gynaecological risk reduction. Study identification included bibliographic database searching and citation chasing (searches updated 3 August 2021). Screening and assessment of eligibility for inclusion were conducted by independent researchers. Outcomes were prespecified and were informed by clinical experts and patient involvement. Data extraction and quality appraisal were conducted and results were synthesised narratively. We also developed a whole-disease economic model for Lynch syndrome using discrete event simulation methodology, including natural history components for colorectal, endometrial and ovarian cancer, and we used this model to conduct a cost-utility analysis of gynaecological risk management strategies, including surveillance, risk-reducing surgery and doing nothing.
UNASSIGNED: We found 30 studies in the review of clinical effectiveness, of which 20 were non-comparative (single-arm) studies. There were no high-quality studies providing precise outcome estimates at low risk of bias. There is some evidence that mortality rate is higher for surveillance than for risk-reducing surgery but mortality is also higher for no surveillance than for surveillance. Some asymptomatic cancers were detected through surveillance but some cancers were also missed. There was a wide range of pain experiences, including some individuals feeling no pain and some feeling severe pain. The use of pain relief (e.g. ibuprofen) was common, and some women underwent general anaesthetic for surveillance. Existing economic evaluations clearly found that risk-reducing surgery leads to the best lifetime health (measured using quality-adjusted life-years) and is cost-effective, while surveillance is not cost-effective in comparison. Our economic evaluation found that a strategy of surveillance alone or offering surveillance and risk-reducing surgery was cost-effective, except for path_PMS2 Lynch syndrome. Offering only risk-reducing surgery was less effective than offering surveillance with or without surgery.
UNASSIGNED: Firm conclusions about clinical effectiveness could not be reached because of the lack of high-quality research. We did not assume that women would immediately take up risk-reducing surgery if offered, and it is possible that risk-reducing surgery would be more effective and cost-effective if it was taken up when offered.
UNASSIGNED: There is insufficient evidence to recommend for or against gynaecological cancer surveillance in Lynch syndrome on clinical grounds, but modelling suggests that surveillance could be cost-effective. Further research is needed but it must be rigorously designed and well reported to be of benefit.
UNASSIGNED: This study is registered as PROSPERO CRD42020171098.
UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: NIHR129713) and is published in full in Health Technology Assessment; Vol. 28, No. 41. See the NIHR Funding and Awards website for further award information.
Lynch syndrome is an inherited condition which puts people at a higher risk of getting bowel cancer, womb cancer and ovarian cancer. Although people with Lynch syndrome are more likely to get these cancers, they are more likely to survive cancer if they get it. People diagnosed with Lynch syndrome get regular testing (surveillance) using a camera to check for bowel cancer or polyps. For womb and ovarian cancer, surveillance may also be an option, but it is less well studied in these cancers. This means that many women are not offered surveillance. Women with Lynch syndrome are recommended to have risk-reducing surgery when their risk starts rising, if they do not want any more children. We wanted to find out whether surveillance for womb and ovarian cancer would work and would be good value for money. Doctors and patients have said that these are important research questions. We searched for published research on this subject and found a lot of studies, but these studies were often small or not well designed, so they could only tell us a limited amount. Studies did not always measure the things that patients want to know. There was some evidence that people having surveillance might live longer than people not having surveillance, but there was also some evidence that risk-reducing surgery is better than surveillance. Surveillance has detected some cancers which had no symptoms, but there are also cancers diagnosed soon after a surveillance visit where nothing was found. People often find surveillance painful, but experiences vary. Our work shows that surveillance and surgery could be good value for money for many women with Lynch syndrome. We need better research to help patients and doctors decide whether surveillance is right for them.

暂无摘要。

This study aimed to assess the cost-utility of romiplostim (ROMI) compared to eltrombopag (EPAG) as a second-line treatment for chronic primary immune thrombocytopenia (cITP) in Chinese adults. A decision tree-embedded Markov model with a lifetime horizon was used to estimate the quality-adjusted life years (QALYs) and costs for ROMI versus EPAG from the perspective of the Chinese health care system. The model was driven by platelet response with a 4-week cycle. Both QALYs and costs were discounted 5% per year. Clinical data comparing ROMI and EPAG were obtained by matching-adjusted indirect comparison (MAIC), utilizing individual patient data on ROMI and published Chinese Phase III trial data on EPAG. Costs were reported in 2022 US dollars and included drug acquisition costs, monitoring costs, bleeding-related costs, and costs associated with adverse events. Deterministic and probabilistic sensitivity analyses were performed. The CEA model indicated that treatment with ROMI resulted in an average of $4,344.4 higher costs for 0.004 QALYs. One-way sensitivity analysis (OSA) indicated that the model was most sensitive to the high bleeding rate in response (Markov stage) for EPAG and ROMI. Probabilistic sensitivity analysis (PSA) indicated that ROMI was likely to be cost effective in 0.16% cases at a willingness-to-pay threshold of $12039.1 (China per capita GDP in 2022) per QALY. If the price of ROMI is either lower than or equal to that of EPAG, ROMI could likely be considered cost-effective as a second-line treatment for Chinese adults with cITP.

UNASSIGNED: Influenza infection induces cardiovascular events in heart failure (HF) patients, with potential risk reduction through vaccination. This study aims to evaluate the cost-effectiveness of influenza vaccination for HF patients in China.
UNASSIGNED: We developed a Markov model with a 3-month cycle to simulate the cost-effectiveness of administering the influenza vaccine to patients with HF over a 3-year period. Patients in the model received either the influenza vaccine or a placebo, in addition to standard HF treatment. Cost data, sourced from the China Healthcare Statistic Yearbook and other public records, and effectiveness data from the IVVE (Influenza Vaccine to Prevent Adverse Vascular Events in HF) trial, were incorporated. Specifically, the cost of the influenza vaccine was 75 Chinese Yuan (CNY) (11 USD), the cost of hospitalization for heart failure (HHF) was 9,326 CNY (1,386 USD), and the cost of treatment for pneumonia was 5,984 CNY (889 USD). The study\'s primary outcome, the incremental cost-effectiveness ratio (ICER), quantifies the incremental cost (CNY and USD) per incremental quality-adjusted life year (QALY). Additional outcomes included total cost, total effectiveness, incremental cost, and incremental effectiveness. We conducted one-way and probabilistic sensitivity analyses (PSA) to assess certainty and uncertainty, respectively. Scenario analysis, considering various situations, was performed to evaluate the robustness of the results.
UNASSIGNED: In the base case analysis, influenza vaccine, compared to placebo, among Chinese HF patients, resulted in a cost increase from 21,004 CNY (3,121 USD) to 21,062 CNY (3,130 USD) and in QALYs from 1.89 to 1.92 (2.55 life years vs. 2.57 life years) per patient. The resulting ICER was 2,331 CNY (346 USD) per QALY [2,080 CNY (309 USD) per life year], falling below the willingness-to-pay threshold based on per capita GDP. One-way sensitivity analysis revealed that disparities in HHF and cardiovascular death rates between groups had the most significant impact on the ICER, while the cost of vaccines had a marginal impact. PSA and scenario analysis collectively affirmed the robustness of our findings.
UNASSIGNED: This study suggests that adding the influenza vaccine to standard treatment regimens for Chinese patients with HF may represent a highly cost-effective option. Further real-world data studies are essential to validate these findings.

OBJECTIVE: Evidence on the cost effectiveness of decision aids to guide management decisions for men with prostate cancer is limited. We examined the cost utility of the Navigate online decision aid for men with prostate cancer in comparison to usual care (no decision aid).
METHODS: A Markov model with a 10-yr time horizon was constructed from a government health care perspective. Data from the Navigate trial (n = 302) and relevant published studies were used for model inputs. Incremental costs and quality-adjusted life-years (QALYs) were calculated for the two strategies. One-way and probabilistic sensitivity analyses were undertaken to address model uncertainty.
UNASSIGNED: On average, the Navigate strategy was estimated to cost AU$8899 (95% uncertainty interval [UI] AU$7509-AU$10438) and produce 7.08 QALYs (95% UI 6.73-7.36) in comparison to AU$9559 (95% UI AU$8177-AU$11017) and 7.03 QALYs (95% UI 6.67-7.31) or usual care. The Navigate strategy dominated usual care as it produced cost-savings and higher QALYs, although differences for both outcomes were small over 10 yr. The likelihood of Navigate being cost effective at a conventionally acceptable threshold of AU$50000 per QALY gained was 99.7%. This study is limited by the availability, quality, and choice of the data used in the model.
CONCLUSIONS: Use of an online decision aid for men with prostate cancer appears to be cost effective relative to usual care in Australia, driven by the higher acceptance and uptake of active surveillance. Wider implementation of decision aids may better inform men diagnosed with prostate cancer about their management options.
RESULTS: We looked at the cost effectiveness of an online decision aid for guiding Australian men with prostate cancer in choosing a management option. We found that this decision aid was cost effective, mainly because more men chose active surveillance. Decision aids that inform patients about their management options should be more widely used in health care.

UNASSIGNED: Utility values offer a quantitative means to evaluate the impact of novel cancer treatments on patients\' quality of life (QoL). However, the multiple methods available for valuing QoL present challenges in selecting the most appropriate method across different contexts.
UNASSIGNED: This review provides cancer clinicians and researchers with an overview of methods to value QoL for economic evaluations, including standalone and derived preference-based measures (PBMs) and direct preference elicitation methods. Recent developments are described, including the comparative performance of cancer-specific PBMs versus generic PBMs, measurement of outcomes beyond health-related QoL, and increased use of discrete choice experiments to elicit preferences. Recommendations and considerations are provided to guide the choice of method for cancer research.
UNASSIGNED: We foresee continued adoption of the QLU-C10D and FACT-8D in cancer clinical trials given the extensive use of the EORTC QLQ-C30 and FACT-G in cancer research. While these cancer-specific PBMs offer the convenience of eliciting utility values without needing a standalone PBM, researchers should consider potential limitations if they intend to substitute them for generic PBMs. As the field advances, there is a greater need for consensus on the approach to selection and integration of various methods in cancer clinical trials.

OBJECTIVE: The current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared to maintenance treatment over 24 months from a mental health services, health and social care, and societal perspectives.
METHODS: Nineteen mental health trusts recruited patients to the RADAR randomised controlled trial. Quality adjusted life years (QALYs) were calculated from patient-reported EQ-5D-5L, with years of full capability (YFCs) calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources.
RESULTS: 253 participants were randomised: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in QALYs (-0.035; 95% CI: -0.123 to 0.052), whereas YFCs were significantly lower in the reduction arm compared to the maintenance arm (baseline-adjusted difference: -0.103; 95% CI: -0.192 to -0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective.
CONCLUSIONS: It is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over two-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.

UNASSIGNED: To determine the appropriate treatment for patients with advanced/recurrent nonsquamous non‒small-cell lung cancer (NSCLC), a companion diagnostic was conducted to detect driver mutations through genetic testing. In Japan, Oncomine Dx Target Test (DxTT) using next-generation sequencing (NGS) that can comprehensively detect gene mutations or single-gene tests are conducted as companion diagnostics. Furthermore, cost-effectiveness analysis was conducted to compare the cost-effectiveness of Oncomine DxTT using NGS with that of single-gene test in Japan.
UNASSIGNED: The target population included patients with advanced/recurrent nonsquamous NSCLC. A model structure was constructed for the Oncomine DxTT strategy and three single-gene tests (i.e., epidermal growth factor receptor (EGFR) mutations and anaplastic lymphoma kinase (ALK)/c-ros oncogene 1 (ROS1) rearrangements) with reference to previous studies and the Clinical Practice Guidelines of Lung Cancer 2022 in Japan. The model structure assumed that genetic testing would be conducted and first-line treatment used the drug most recommended in the 2022 Japanese Lung Cancer Clinical Practice Guidelines, depending on the driver mutation,. Model inputs were obtained from the literature and price list in Japan, and cost-utility analysis was conducted.
UNASSIGNED: For the Oncomine DxTT strategy, the expected incremental costs and effectiveness were estimated to be approximately JPY 172,361 (JPY 12,285,228 vs. JPY 12,112,867 for strategies A and B, respectively) and -0.51 quality-adjusted life-year (QALY) per patient (21.93 QALY vs. 22.44 QALY for strategies A and B). As a result, the costs increased but the effectiveness decreased. Therefore, the Oncomine DxTT strategy was dominated by the three single-gene tests. Sensitivity and scenario analyses revealed that the test success rate of Oncomine DxTT affected the results.
UNASSIGNED: The genetic test using Oncomine DxTT before the first-line treatment is not cost-effective compared with the three single-gene tests (EGFR/ALK/ROS1) for patients with advanced/recurrent nonsquamous NSCLC.

Chemotherapy has helped prolong survival in patients with malignant lymphoma, enhancing their quality of life (QOL). Despite the eventual decline in the QOL of patients, the impact of initial chemotherapy remains poorly understood. A prospective patient-reported QOL survey among patients with malignant lymphoma receiving initial chemotherapy was conducted, targeting those treated at Gifu Municipal Hospital (Gifu, Japan) between January 2021 and December 2022. Surveys were conducted pre- and post-chemotherapy based on the EuroQol 5 dimensions. Drug costs were calculated using official prices and analyzed from the cost payer\'s perspective via cost-utility analysis. Among the 60 patients included in the present study, 28 had diffuse large B-cell lymphoma. Cyclophosphamide, doxorubicin, vincristine, prednisolone ± rituximab therapy was the most common treatment (38 patients) and demonstrated superior cost-effectiveness due to its lowest cost and change in utility value. Initial chemotherapy for patients with malignant lymphoma generally improved the QOL. Clinical trial registration: UMIN000042868 (registered on December 28, 2020).