Quality-adjusted time without symptoms or toxicity analysis of haploidentical-related donor vs.identical sibling donor hematopoietic stem cell transplantation in acute myeloid leukemia

Objective:We aimed to compare the quality-adjusted time without symptoms or toxicity(Q-TWiST)in acute myeloid leukemia(AML)patients who received haploidentical-related donor(HID)and identical sibling donor(ISD)hematopoietic stem cell transplantation(HSCT).Methods:Five clinical health states were defined:toxicity(TOX),acute graft-versus-host disease(GVHD),chronic GVHD(cGVHD),time without symptoms and toxicity(TWiST)and relapse(REL).The equation used in this study was as follows:Q-TWiST=UTOX×TOX+UTWiST×TWiST+UREL×REL+UaGVHD×aGVHD+UcGVHD×cGVHD.Results:A total of 239 AML patients were enrolled.We established a mathematical model,i.e.,Q-TWiST HID HSCT>Q-TWiST ISD HSCT,to explore the range of utility coefficients satisfying the inequality.Based on the raw data,the utility coefficient is equivalent to the following inequality:10.57067UTOX-46.27733UREL+105.9374+3.388078UaGVHD-210.8198UcGVHD>0.The model showed that when UTOX,UREL,and UaGVHD were within the range of 0-1,as well as when UcGVHD was within the range of 0-0.569,the inequality Q-TWiST HID HSCT>Q-TWiST ISD HSCT was valid.According to the results of the ChiCTR1800016972 study,the median coefficients of TOX,acute GVHD(aGVHD),and cGVHD were 0.56(0.41-0.76),0.56(0.47-0.72),and 0.54(0.37-0.79),respectively.We selected a series of specific examples of the coefficients,i.e.,UTOX=0.5,UREL=0.05,UaGVHD-0.5,and UcGVHD-0.5.The Q-TWiST values of ISD and HID HSCT were 896 and 900 d,respectively(P=0.470).Conclusions:We first observed that Q-TWiST was comparable between AML patients receiving HID HSCT and those receiving ISD HSCT.

Quality-adjusted life years and surgical waiting list:Systematic review of the literature

BACKGROUND The quality-adjusted life year(QALY)is a metric that is increasingly used today in the field of health economics to evaluate the value of different medical treatments and procedures.Surgical waiting lists(SWLs)represent a pressing problem in public healthcare.The QALY measure has rarely been used in the context of surgery.It would be interesting to know how many QALYs are lost by patients on SWLs.AIM To investigate the relationship between QALYs and SWLs in a systematic review of the scientific literature.METHODS The study was conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Statement.An unlimited search was carried out in PubMed,updated on January 19,2024.Data on the following variables were investigated and analyzed:Specialty,country of study,procedure under study,scale used to measure QALYs,the use of a theoretical or real-life model,objectives of the study and items measured,the economic value assigned to the QALY in the country in question,and the results and conclusions published.RESULTS Forty-eight articles were selected for the study.No data were found regarding QALYs lost on SWLs.The specialties in which QALYs were studied the most in relation to the waiting list were urology and general surgery,with 15 articles each.The country in which the most studies of QALYs were carried out was the United States(n=21),followed by the United Kingdom(n=9)and Canada(n=7).The most studied procedure was organ transplantation(n=39),including 15 kidney,14 liver,5 heart,4 lung,and 1 intestinal.Arthroplasty(n=4),cataract surgery(n=2),bariatric surgery(n=1),mosaicplasty(n=1),and septoplasty(n=1)completed the surgical interventions included.Thirty-nine of the models used were theoretical(the most frequently applied being the Markov model,n=34),and nine were real-life.The survey used to measure quality of life in 11 articles was the European Quality of Life-5 dimensions,but in 32 articles the survey was not specified.The willingness-to-pay per QALY gained ranged from$100000 in the United States to€20000 in Spain.CONCLUSION The relationship between QALYs and SWLs has only rarely been studied in the literature.The rate of QALYs lost on SWLs has not been determined.Future research is warranted to address this issue.

Cost-utility of molecular adsorbent recirculating system treatment in acute liver failure

AIM:To determine the short-term cost-utility of mo-lecular adsorbent recirculating system(MARS) treatment in acute liver failure(ALF).METHODS:A controlled retrospective study was conducted with 90 ALF patients treated with MARS from 2001 to 2005.Comparisons were made with a historical control group of 17 ALF patients treated from 2000 to 2001 in the same intensive care unit(ICU) specializing in liver diseases.The 3-year outcomes and number of liver transplantations were recorded.All direct liver disease-related medical expenses from 6 mo before to 3 years after ICU treatment were determined for 31 MARS patients and 16 control patients.The health-related quality of life(HRQoL) before MARS treatment was estimated by a panel of ICU doctors and after MARS using a mailed 15D(15-dimensional generic healthrelated quality of life instrument) questionnaire.The HRQoL,cost,and survival data were combined and the incremental cost/quality-adjusted life years(QALYs) was calculated.RESULTS:In surviving ALF patients,the health-related quality of life after treatmeant was generally high and comparable to the age-and gender-matched general Finnish population.Compared to the controls,the average cost per QALY was considerably lower in the MARS group(64 732€ vs 133 858€) within a timeframe of 3.5 years.The incremental cost of standard medical treatment alone compared to MARS was 10 928€,and the incremental number of QALYs gained by MARS was 0.66.CONCLUSION:MARS treatment combined with standard medical treatment for ALF in an ICU setting is more cost-effective than standard medical treatment alone.

Cost-Effectiveness Analysis of Neoadjuvant Chemotherapy with Zoledronic Acid for HER2-Negative Breast Cancer in Japan: The JONIE1 Study

Objective: Zoledronic acid (ZOL) is a nitrogen-containing bisphosphonate that induces osteoclast apoptosis and inhibits bone resorption. Adding ZOL to neoadjuvant chemotherapy has been shown to have potential anticancer benefits in women with HER2-negative breast cancer. The objective of the present study was to investigate ZOL’s cost-effectiveness from the perspective of health care payers in Japan. Methods: A Markov model was developed to evaluate the costs and effectiveness associated with ZOL + chemotherapy (CTZ) and chemotherapy (CT) alone over a 10-year time horizon. Monthly transition probabilities were estimated according to JONIE1 (Japan Organization of Neoadjuvant Innovative Expert) Study data and an extrapolated Weibull model. Health outcomes were measured in quality-adjusted life years (QALYs). Costs were calculated using year-2018 Japanese yen (JPY) (1.00 US dollars (USD) = 110.4 JPY). Model robustness was addressed through one-way and probabilistic sensitivity analysis. The costs and QALYs were discounted at a rate of 2% per year. Results: In the base case, the use of CTZ was associated with a gain of 3.94 QALYs. The incremental cost per QALY of the CTZ gain was 681,056.1 JPY (6168.99 USD) per QALY. Conclusion: It is convincing that neoadjuvant CTZ for patients with breast cancer would be expected to have statistically significant clinical efficacy. Addition of ZOL to CT might be a cost-effective option compared with CT alone.

Health-related quality-of-life and health-utility reporting in critical care

Mortality is a well-established patient-important outcome in critical care studies.In contrast,morbidity is less uniformly reported(given the myriad of critical care illnesses and complications of each)but may have a common end-impact on a patient’s functional capacity and health-related quality-of-life(HRQoL).Survival with a poor quality-of-life may not be acceptable depending on individual patient values and preferences.Hence,as mortality decreases within critical care,it becomes increasingly important to measure intensive care unit(ICU)survivor HRQoL.HRQoL measurements with a preference-based scoring algorithm can be converted into health utilities on a scale anchored at 0(representing death)and 1(representing full health).They can be combined with survival to calculate quality-adjusted life-years(QALY),which are one of the most widely used methods of combining morbidity and mortality into a composite outcome.Although QALYs have been use for health-technology assessment decision-making,an emerging and novel role would be to inform clinical decision-making for patients,families and healthcare providers about what expected HRQoL may be during and after ICU care.Critical care randomized control trials(RCTs)have not routinely measured or reported HRQoL(until more recently),likely due to incapacity of some patients to participate in patient-reported outcome measures.Further differences in HRQoL measurement tools can lead to non-comparable values.To this end,we propose the validation of a gold-standard HRQoL tool in critical care,specifically the EQ-5D-5L.Both combined health-utility and mortality(disaggregated)and QALYs(aggregated)can be reported,with disaggregation allowing for determination of which components are the main drivers of the QALY outcome.Increased use of HRQoL,health-utility,and QALYs in critical care RCTs has the potential to:(1)Increase the likelihood of finding important effects if they exist;(2)improve research efficiency;and(3)help inform optimal management of critically ill patients allowing for decision-making about their HRQoL,in additional to traditional health-technology assessments.

First line nucleos(t)ide analog monotherapy is more cost-effective than combination strategies in hepatitis B e antigen-positive chronic hepatitis B patients in China

Background:Nucleos(t)ide analog(NA)in combination with peginterferon(PegIFN)therapy in patients with hepatitis B e antigen(HBeAg)-positive chronic hepatitis B(CHB)shows better effectiveness than NA monotherapy in hepatitis B surface antigen loss,termed"functional cure,"based on previous published studies.However,it is not known which strategy is more cost-effective on functional cure.The aim of this study was to analyze the cost-effectiveness of first-line monotherapies and combination strategies in HBeAg-positive CHB patients in China from a social perspective.Methods:A Markov model was developed with functional cure and other five states including CHB,compensated cirrhosis,decompensated cirrhosis,hepatocellular carcinoma,and death to assess the cost-effectiveness of seven representative treatment strategies.Entecavir(ETV)monotherapy and tenofovir disoproxil fumarate(TDF)monotherapy served as comparators,respectively.Results:In the two base-case analysis,compared with ETV,ETV generated the highest costs with$44,210 and the highest quality-adjusted life-years(QALYs)with 16.78 years.Compared with TDF,treating CHB patients with ETV and NA-PegIFN strategies increased costs by$7639 and$6129,respectively,gaining incremental QALYs by 2.20 years and 1.66 years,respectively.The incremental cost-effectiveness ratios were$3472/QALY and$3692/QALY,respectively,which were less than one-time gross domestic product per capita.One-way sensitivity analysis and probabilistic sensitivity analyses showed the robustness of the results.Conclusion:Among seven treatment strategies,first-line NA monotherapy may be more cost-effective than combination strategies in HBeAg-positive CHB patients in China.

Burden of epilepsy in China and its provinces,1990 to 2019:findings from the Global Burden of Disease Study 2019

Background: Epilepsy accounts for a significant portion of the global disease burden. However, little is known about the disease burden of epilepsy in China and its provinces. Methods: We assessed the burden of epilepsy in China and its provinces, municipalities, and autonomous regions from 1990 to 2019. Burden was measured as incidence, prevalence, deaths, years lived with disability, years of life lost, and disability-adjusted life years (DALYs), by age, sex, year, and province. We used the Socio-Demographic Index (SDI) to determine the association between the provincial development level and age-standardized DALY rates of epilepsy from 1990 to 2019. Results: In 2019, epilepsy caused 1367.51 thousand (95% uncertainty interval [UI]: 979.92–1837.61 thousand) DALYs, and the age-standardized DALY rate was 99.77 (95% UI: 71.33–133.52)/100,000. The age-standardized incidence and prevalence rates for epilepsy in China were 24.65/100,000 and 219.69/100,000, increased by 45.00% (95% UI: 8.03–98.74%) and 35.72% (95% UI: 0.47–86.19%) compared with that in 1990, respectively. From 1990 to 2019, the proportion of DALY caused by epilepsy in the age group under 25 years steadily decreased. The proportion of DALYs caused by epilepsy in people aged 50 years and over increased from 9.45% and 10.22% in 1990 to 29.01% and 32.72% for male and female individuals in 2019, respectively. The highest age-standardized mortality rates were seen in Tibet (4.26 [95% UI: 1.43–5.66]/100,000), Qinghai (1.80 [95% UI: 1.15–2.36]/100,000), and Yunnan (1.30 [95% UI: 0.88–1.62]/100,000), and the lowest mortality rates were in Guangdong (0.48 [95% UI: 0.39–0.64]/100,000), Zhejiang (0.56 [95% UI: 0.44–0.70]/100,000), and Shanghai (0.57 [95% UI: 0.41–0.73]/100,000). The age-standardized DALY rates across the country and in provinces, municipalities, and autonomous regions generally decreased as their SDI increased. Conclusions: The disease burden of epilepsy is still heavy in China, especially in the western provinces. The incidence and prevalence of epilepsy increased between 1990 and 2019, and the burden of epilepsy in the elderly increases gradually. This study provides evidence on epilepsy prevention and care of different regions in China.

Burden of multiple myeloma in China:an analysis of the Global Burden of Disease,Injuries,and Risk Factors Study 2019

Background:There is limited data to comprehensively evaluate the epidemiological characteristics of multiple myeloma(MM)in China;therefore,this study determined the characteristics of the disease burden of MM at national and provincial levels in China.Methods:The burden of MM,including incidence,mortality,prevalence,and disability-adjusted life years(DALYs),with a 95%uncertainty interval(UI),was determined in China following the general analytical strategy used in the Global Burden of Disease,Injuries,and Risk Factors Study 2019.The trends in the burden of MM from 1990 to 2019 were also evaluated.Results:There were an estimated 347.45 thousand DALYs with an age-standardized DALY rate of 17.05(95%UI,12.31-20.77)per 100,000 in 2019.The estimated number of incident case and deaths of MM were 18,793 and 13,421,with age-standardized incidence and mortality rates of 0.93(95%UI,0.67-1.15)and 0.67(95%UI,0.50-0.82)per 100,000,respectively.The age-specific DALY rates per 100,000 increased to more than 10.00 in the 40 to 44 years age group reaching a peak(93.82)in the 70 to 74 years age group.Males had a higher burden than females,with approximately 1.5-to 2.0-fold sex difference in age-specific DALY rates in all age groups.From 1990 to 2019,the DALYs of MM increased 134%,from 148,479 in 1990 to 347,453 in 2019.Conclusion:The burden of MM has doubled over the last three decades,which highlights the need to establish effective disease prevention and control strategies at both the national and provincial levels.