Cost utility Analysis of Adult Patients with Severe Aplastic Anemia Based on Single-center Cohort Study

doi:10.21203/rs.3.rs-3111333/v1

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Research Article

Cost utility Analysis of Adult Patients with Severe Aplastic Anemia Based on Single-center Cohort Study

https://doi.org/10.21203/rs.3.rs-3111333/v1

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Objective

The purpose of this study is to objectively evaluate the efficacy of cyclosporine A + ALG and cyclosporine A + TPO-RA in the treatment of SAA patients, and to provide scientific data support for maximizing the therapeutic benefit of SAA patients.

Method

Cost-utility analysis was used to compare the quality of life adjusted years and total cost of the two schemes. The patient utility value was obtained from EQ-5D-3L and Japanese TTO conversion, and the total cost of treatment was obtained from electronic questionnaire survey and medical information data.

Results

A total of 26 adult patients were included in the study, including 17 with cyclosporine A + ALG and 9 with cyclosporine A + TPO-RA. In terms of QALY, patients with cyclosporine A + ALG regimen had higher intuitive feelings than those with cyclosporine A + TPO-RA regimen from admission to 6 months of follow-up, and the quality of life of patients with the former regimen was also significantly higher than that of the latter, with a difference of 0. 08 QALY (P < 0.01). In terms of total cost, the total cost of the former was twice that of the latter.Each QALY obtained by the two regimens was 687,000 yuan and 450,000 yuan, respectively.Compared with the cyclosporine A + TPO-RA regimen, the total cost of each QALY increased by the cyclosporine A + ALG regimen was 1.51 million yuan.

Conclusion

On the premise of ensuring the effectiveness of diagnosis and treatment, CsA + TPO-RA can be used as a good treatment option, which can reduce the economic pressure of SAA patients, reduce the burden of disease, reduce the pressure of medical insurance funds, and achieve multi-benefits.

severe aplastic anemia (SAA)

cyclosporine A (CsA)

antilymphocyte globulin (ALG)

cost-utility

quality of life adjusted years (QALY)

incremental cost-utility ratio (ICUR)

Severe aplastic anemia (SAA) is a rare hematological disorder characterized by pancytopenia and myelopenia, with high morbidity and mortality rate [1–4]. Patients often suffer from life-threatening severe infections and/or hemorrhage, and the severe cases are life-threatening [5]. The treatment of SAA is mostly seen in immunosuppressive therapy, and the European Blood and Marrow Transplantation (EBMT) guidelines recommend ATG/ALG + CsA as the first choice [6–10]. At present, the first-line standard treatment of SAA is still ATG/ALG + CsA, and the long-term survival rate of patients is close to 50% -70% [11–13]. In recent years, some scholars have used thrombopoietin receptor agonist combined with CsA as a new treatment for SAA patients, and the therapeutic effect is good [14–15]. The current research on SAA patients mainly focuses on the effect of different treatment schemes, but lacks the benefit research among different treatment schemes. Different research schemes have different disease burden and economic pressure on patients, and higher costs may affect patients' willingness to see a doctor and reduce their quality of life.

We found that while carrying out clinical research on many diseases in the world, we also carried out a series of studies on the quality of life and cost-effectiveness of patients to promote the level of diagnosis and treatment of patients, reduce the disease burden of patients, and fully enable patients to obtain maximum benefits [16–17]; Cost-utility analysis is the most common tool used by researchers in healthcare economics [18–19], where the result of the analysis is the cost per quality-adjusted life year. Cost utility analysis have been carried out in patients with bone marrow transplantation, lymphoma and leukemia in the field of hematological diseases, but there is a lack of relevant studies in patients with SAA.

We will analyze the cost input and quality of life (QALY) of adult newly diagnosed SAA patients under different diagnosis and treatment schemes from the perspective of patients,evaluate the benefits of patients under different diagnosis and treatment schemes,on the basis of ensuring the effect of diagnosis and treatment, we strive for the best benefit for patients,and provide important reference for reducing the burden of disease, reducing the loss of social economy and the decision-making of medical insurance by the government.

1.1 Study Design

This study was a single-center cohort study designed with a decision tree model (see Fig. 1), and patients were selected according to the inclusion criteria adult patients with newly diagnosed SAA. The patients were treated with Cs A + thrombopoietin receptor agonist (group A) and Cs A + ALG (group B) according to their conditions, and the patients were followed up for 6 months after admission to evaluate the benefits of different treatment programs for patients. The study was approved by the patient and approved by the hospital ethics committee.

1.2 Research objects

The subjects of this study were patients who were hospitalized for the first time and discharged from a single center in China for the first time from November 1, 2020 to January 31, 2022. A total of 26 subjects were enrolled. Inclusion criteria: (1) the patient's primary diagnosis on admission was ICD-10 code: D61.905, (2) the patient was treated for the first time, and (3) the patient's age at admission was ≥ 18 years. Exclusion criteria: (1) patients with other hematological diseases in the main diagnosis of discharge, (2) patients during treatment to change the program, transfer, or transfer to another hospital, (3) repeated admission patients during the follow-up period, (4) patients with medical costs of 0 or ≥ 3 times the average; (5) patients hospitalized for more than 6 months; (6) patients did not complete the 6-month follow-up cycle.

1.3 Implementation process

The research is mainly divided into the following steps:

1) design a research scheme, and formulate a screening and exclusion standard, wherein all patients receive that same group of medical services, the patients receive the treatment scheme of group A or group B according to the disease condition, the research assistant number the patients, and the researcher exports information from an electronic questionnaire system at the end of the research;

2) that the research assistants evaluate the health efficacy of the patient at the time of admission, discharge and 6 months aft admission according to the number and follow-up time;

3) the research assistant investigates the information of the patient such as discharge disease diagnosis, hospitalization expense, hospitalization days and the like according to the derived data;

4) after the end of the study, all the original desensitization information was cross-checked by two other personnel. After confirming that the information was correct, it was handed over to two researchers again for data integration, conversion and analysis. The cost, QALY, CUA and ICUR of the two groups of SAA patients were compared and analyzed through SPSS19.0.

1.4 Treatment plan

All enrolled patients were treated with group A or group B. Group A: CsA + thrombopoietin receptor agonists, according to the patient's condition and disease situation, formulated a plan (CsA 50–100 mg/bid + itripopa 75–100 mg/qd or haitrippa 25–50 mg/qd). Group B: CsA + ALG treatment, according to the patient's situation and disease situation to develop a program (CsA 50-100mg/bid + ALG 4–6/d). All patients enjoy the same quality of medical care services during the treatment plan. After meeting the discharge guidelines, they will go through the discharge procedures without delay due to various reasons.

1.5 Assessment tools

1.5.1 Electronic Questionnaire

According to the research content, the electronic questionnaire of basic information was designed by ourselves, and the research assistant conducted the survey at the time of admission and discharge respectively. The following are the survey contents at different time points: 1) At the time of admission: basic information, such as name, gender, age, position, educational level, insurance status and past disease history; 2) At the time of discharge: disease diagnosis code, total cost of diagnosis and treatment, length of stay, proportion of insurance reimbursement, number of accompanying persons in this hospitalization, total additional expenses for this hospitalization (transportation, accommodation, diet), etc.

1.5.2 EQ-5D-3L

Individual health efficacy was followed up by the research assistant at the time of admission, at 3 months of follow-up (3 months after admission), and at 6 months of follow-up (6 months after admission), and the health efficacy measurement tool was the European Five-Dimensional Health Scale (EQ-5D-3L) [20–22]. It is a general measure of health-related quality of life and consists of two main parts: the first part is a descriptive system of five domains, namely, assessment of activity level, self-care, daily activities, pain-discomfort, and anxiety-depression. Each domain has three levels of description: "No problems" (Level 1), "Some problems" (Level 2), and "Extreme problems" (level 3). The second part uses a visual analogue scale (0 to 100) to capture the current health status of the subjects, and the respondents self-assess their current health status according to their actual situation at that time, with 0 being the worst health level and 100 being the best health level.

1.5 Evaluation tools

1.5.1 Electronic questionnai

According to the research content, we designed the basic information electronic questionnaire by ourselves, and the research assistant investigated the patients at admission and discharge respectively. The following are the survey contents at different time nodes:

1) At admission: basic information, name, gender, age, position, education level, insurance participation, and previous disease history;

2) At discharge: disease diagnosis code, total cost of diagnosis and treatment, length of stay, proportion of insurance and reimbursement, number of attendants in this hospital, total additional expenditure (transportation, accommodation, food), etc.

1.5.2 EQ-5D-3L

The research assistant carried out personal health effectiveness follow-up at the time of admission, 3 months of follow-up (3 months after admission), and 6 months of follow-up (6 months after admission). The European Five dimensional Health Scale (EQ-5D-3L) was adopted as the measurement tool for health effectiveness [20–22].

It is a universal measurement of health-related quality of life, mainly including two parts: Part 1, a description system of five areas, namely, assessment of activity level, self-care, daily activities, pain discomfort and anxiety depression. Each domain has three levels of description: "no problem" (level 1), "some problems" (level 2), and "extreme problems" (level 3). In the second part, the current health status of the subjects was captured with a visual analog scale (0 to 100). The interviewees assessed their current health status according to their actual conditions at that time. 0 was the worst health level, and 100 was the best health level.

1.5.3 Efficiency value conversion table

According to the choices made by the patients on the three levels of the European Five-dimensional Health Scale in the questionnaire, the population in China is similar to that in Japan. The Japan TTO is used to calculate the EQ-5D questionnaire performance score, and the utility value of each patient is the mean of the EQ-5D questionnaire performance score and EQ-VAS score [20].

1.6 Cost

The total cost of this study is the sum of the patient's direct cost (yuan) and indirect cost (yuan), in which the direct cost mainly refers to the cost of diagnosis and treatment, while the indirect cost refers to the additional expenses during personal diagnosis and treatment, personal economic losses caused by illness, consumption of accompanying personnel during diagnosis and treatment and economic losses of accompanying personnel during diagnosis and treatment. In this study, the patients could not work during the treatment and follow-up period, and had no salary and other income; the economic loss and consumption expenditure of the accompanying staff were based on the patient's treatment cycle, and the rest of the time could work normally. The diagnosis and treatment cycle of the patients in this study is mostly in 2020 and 2021. Based on this, the average value of 2020 and 2021 is used as the discount rate adjustment in this study.

1) The calculation of economic loss of patients/caregivers is based on the average per capita income of China in 2020 and 2021.

Where:

$$Cp=\frac{（GDP2020 +GDP2021）\times Dp\times R}{2\times Y}$$

Cp means personal economic loss; Dp represents the number of days lost, Dp represents 180 days of follow-up when the patient is lost, and Dp represents the number of days in hospital when the attendant is lost; Y represents the total number of days in a year (365 days); R is the number of caregivers; The national per capita GDP will be 32189 yuan in 2020 and 35128 yuan in 2021 [23–24].

2) The patient's hospital is located in a city in China, and the consumption expenditure of caregivers is calculated based on the average per capita expenditure of the region in 2020 and 2021.

Where:

$$Ce=\frac{（P2020 +P2021）\times De\times R}{2\times Y}$$

Ce represents the consumption of caregivers; P represents consumption; De is the number of days in hospital; Y represents the total number of days in a year (365 days); R is the number of caregivers; in 2020, Tianjin's per capita expenditure will be 28461 yuan, and in 2021, 33188 yuan [25–26].

1.7 Utility

The utility value is the result of EQ-5D selection, the patient's health score is converted by Japan-Tto, and the economic evaluation is carried out by personal description of health status. QALYs were calculated from patient EQ-5D efficacy values [27]. QALYs are a common measure to assess the degree of benefit in terms of health-related quality of life (HRQoL) and quality of life for patients with different treatments. QALY emphasizes health status at different times. In one year, the utility value is 1 in the state of complete health, and less than 1 in the state of nonhealth.

Where:

$$Ui=\frac{（E1+E2）\times Di}{2\times Y}+\frac{（E2+E3）\times （180-Di）}{2\times Y}$$

Ui means i patient quality of life adjustment year; E1 is the time limit for admission of patient i; E2 is the utility value of patient i within 3 months after admission; E3 is the utility value of patient i 6 months after admission; Di is the hospitalization days of patient i; Y represents the total number of days in a year (365 days).

1.8 Cost-utility

ICUR is expressed as the ratio between incremental cost and incremental utility (QALY), i.e., the additional cost added by the Group B regimen to achieve one QALY compared with patients treated in Group A. By calculating the incremental cost-utility ratio (ICUR), we evaluate the cost utility of different schemes for patients, which can reduce the cost of diagnosis and treatment and reduce the burden of disease while ensuring that patients enjoy a high level of diagnosis and treatment services.

1.9 Statistical analysis

The currency of this study is Chinese RMB (yuan), and the data are analyzed by SPSS19.0. The quantitative data are expressed by (X ± s), and the independent sample t-test is used; the qualitative data are tested by λ test, and P < 0.01 is statistically significant.

2.1 Basic profile

In general, 126 patients were diagnosed with severe aplastic anemia. Through decision tree model screening, 26 patients met the inclusion criteria, including 17 patients who received CsA+ALG and 9 patients who received CsA+TPO-RA. 26 subjects completed the 6-month follow-up evaluation. See Figure 1 for the specific path of economic utility evaluation decision analysis. The initial sociodemographic characteristics of the two treatment groups are shown in Table 1. There were no statistically significant differences in gender, age, education, position and participation in medical insurance. In addition, all patients had no family history.

Table 1 Overview of basic information of 26 subjects

Category	Total number of cases	Group A（n=17）	Group B（n=9）
Gender
Male	16	12	4
Female	10	5	5
Age
18-25	8	7	1
26-30	5	5
31-40	5	2	3
41-50	5	3	2
51-60	3		3
Position
Civil servants ☆	10	7	3
Farmers	1		1
Freelance	11	6	5
Student	4	4
degree of education
Under junior college□	16	8	8
Undergraduate	7	6	1
Postgraduate	3	3
Insurance participation♣
Urban residents	1	1
Urban employees	10	8	2
New rural cooperative medical system	15	7	8

☆civil servants, including public institutions;

□ Junior college or below, including senior high school, junior high school, primary school, etc;

♣ It is indicated here that there is only one kind of national medical service insurance, excluding commercial insurance.

2.2 Cost of the two groups of treatment options

Table 2 shows the estimated total cost of direct costs (medical expenses) and indirect costs during the patient's visit. The mean direct and indirect costs of Cs A + ALG were higher than those of Cs A + TPO-RA, and the hospitalization period of the former was longer than that of the latter, and the hospitalization period was positively correlated with the indirect costs. Based on the inconsistency of patients' medical insurance reimbursement benchmark, the direct cost (medical expenses) was adjusted by different reimbursement benchmark levels. The higher the reimbursement benchmark, the smaller the gap between the direct cost and the total cost of the two groups.

Table 2 Cost of different treatment options

Project	Group A M（SD）	Group B M（SD）
Indirect cost (yuan)	42736.87（6063.93）	26474.25（10067.60）
Patient income loss (follow-up period)	16598.71	16598.71
Income loss of caregivers	8055.25（2946.90）	1413.97（1387.06）
Accompanying expenses	7377.01（2698.78）	1294.91（1270.27）
Other additional expenses of patients	10705.88（2543.73）	7166.67（7745.96）
Direct cost (yuan)
The reimbursement rate of medical insurance is 0	204567.21（15977.08）	99796.52（82811.24）
The reimbursement rate of medical insurance is 50%	102283.60（7988.54）	49898.26（41405.62）
The reimbursement rate of medical insurance is 70%	61370.16（4793.12）	29938.96（24843.37）
Total cost (yuan)
The reimbursement rate of medical insurance is 0	247304.07（14941.30）	126270.78（92370.28）
The reimbursement rate of medical insurance is 50%	145020.47（8135.73）	76372.52（51013.60）
The reimbursement rate of medical insurance is 70%	104107.09（6253.52）	56413.21（34503.81）
Hospitalization days (days)	77.94（18.33）	16.93（18.92）

Direct costs can be divided into three levels according to the reimbursement of patients without medical insurance. The reimbursement rates are 0, 50% and 70%.

2.3 EQ-5D Performance Scores and QALY of the Two Treatment Regimens at Different Stages

The utility values of EQ-5D were different between the two groups at different stages, and there was no significant difference at admission (P > 0.01), but there was a significant difference at follow-up (P < 0.01). We further analyzed that the EQ-5D utility values of CsA + ALG and CsA + TPO-RA increased gradually, and the former was higher than the latter; The QALY of the two groups were 0.36 QALY and 0.28 QALY respectively, and the CsA + ALG group was 0.08 QALY more than the CsA+ TPO-RA group, with significant difference (t = 6.699, P < 0.001). See Table 3 for details.

Table 3 EQ-5D utility values and QALY of the two treatment regimens

Stage	Group A M（SD）	Group B M（SD）
On admission（n=26）
EQ-5D score	0.42（0.14）	0.29（0.17）
EQ-VAS score	0.47（0.11）	0.35（0.08）
EQ-5D utility value	0.45（0.12）	0.32（0.11）▲
At 3-month follow-up（n=26）
EQ-5D score	0.74（0.05）	0.65（0.11）
EQ-VAS score	0.80（0.09）	0.61（0.12）
EQ-5D utility value	0.77（0.07）	0.63（0.12）■
At 6-month follow-up（n=26）
EQ-5D score	0.87（0.12）	0.73（0.06）
EQ-VAS score	0.96（0.04）	0.69（0.09）
EQ-5D utility value	0.91（0.08）	0.71（0.07）■
QALY (based on EQ-5D utility value)	0.36（0.02）	0.28（0.03）■

EQ-5D=EuroQoL 5-Dimension;VAS=Visual Analogue Scale;QALY is the quality adjusted life year

▲independent-samples T Test, P＞ 0.01;

■ independent-samples T Test, P＜ 0.01.

2.4 CUR and ICUR of patients in the two treatment groups

CUR analysis showed that the total cost per quality-adjusted life year of patients in the CsA + ALG group was higher than that in the CsA + TPO-RA group at all levels of medical insurance reimbursement, and the gap between the two groups gradually narrowed with the increase of medical insurance reimbursement. According to our further ICUR analysis, for each additional QALY of complete health, the CsA + ALG group and the CsA + TPO-RA group need more cost input; when the medical insurance reimbursement ratio reaches 70%, an additional cost input of 596,000yuan is needed. See Table 4 for details.

Table 4 CUR and ICUR of different treatment regimens

Classification	COST（yuan）	QALY（year）	CUR（OST/QALY）	ICUR
The reimbursement rate of medical insurance is 0
Group A	247304.07	0.36	686955.75	1512916.12
Group B	126270.78	0.28	450967.07
The reimbursement rate of medical insurance is 50%
Group A	145020.47	0.36	402834.64	858099.37
Group B	76372.52	0.28	272759.00
The reimbursement rate of medical insurance is 70%
Group A	104107.09	0.36	289186.36	596173.50
Group B	56413.21	0.28	201475.75

ALG is anti-lymphocyte globulin; TPO-RA is a thrombopoietin receptor agonist; Group A: CsA+ALG regimen; Group B was treated with CsA+TPO-RA regimen, indicating that the patients were treated with CsA in combination with Hertrapolpa or one of Hertrapolpa; QALY is the quality adjusted life year; CUR is the cost utility ratio; ICUR is the incremental cost utility ratio.

3.1 Study Summary

We found a large difference in total cost between the two groups, but the QALY for CsA + ALG was 0.08 QALY higher than that for CsA + TPO-RA. When the proportion of medical insurance reimbursement is 0, the former spends 121000 yuan more than the latter. With the increase of the proportion of medical insurance reimbursement, the cost gap between the two groups narrows. At present, China's per capita GDP in 2021 is 35,000 yuan[24]. According to the international three times per capita GDP as the threshold of patient cost payment, it is about 105,000yuan. In reality, the total cost of the CsA + ALG group can meet the standard only when the proportion of medical insurance reimbursement reaches 70%. In the CsA + TPO-RA group, the medical insurance reimbursement rate was 15%, which reached the standard. Therefore, the CsA + TPO-RA regimen may be more cost-effective for patients and the healthcare system.

3.2 Cost-utility analysis shows that CsA + TPO-RA can be used as one of the therapeutic regimens for SAA.

At present, CsA + ALG/ATG immunosuppressive therapy is mainly used to treat SAA at home and abroad, and the diagnosis and treatment effect is consistent with that in the world [5, 6, 8, 11, 12]. Some SAA are intolerant or ineffective to immunosuppressive therapy, and usually use hematopoietic stem cell transplantation as an alternative treatment. However, patients with high complications after transplantation will increase the total disease burden of patients and society, which may seriously affect the quality of life and survival expectations of patients [28–29]. However, the high response rates exhibited with the introduction of thrombopoietin receptor agonists in the treatment of SAA have led to considerable interest in their use in combination with immunosuppressive therapy (IST). Based on this, from the perspective of patients, we explored the efficacy of CsA + ALG and CsA + TPO-RA in the treatment of adult patients with newly diagnosed SAA from the perspective of cost utility. The results of our study showed that patients with CsA + ALG regimen had higher visual perception than those with CsA + TPO-RA regimen from admission to 6 months of follow-up, and the quality of life of patients with the former regimen was also significantly higher than that of the latter, with a difference of 0.08 QALY (P < 0.01). In terms of total cost, the total cost of patients in the former scheme is twice as much as that in the latter scheme, and the period of patients in the former scheme is 61 days longer, which may be an important reason for the increase of additional cost. Further analysis shows that when the medical insurance reimbursement ratio is 0, the QALY obtained by patients with CsA + ALG scheme is about 687,000 yuan, while that obtained by patients with CsA + TPO-RA scheme is 450,000 yuan. Even if the medical insurance reimbursement ratio reaches 70%, the cost of each QALY obtained by the two schemes is more than 200,000 yuan, which is equal to that in the world [30]; compared with the CsA + TPO-RA scheme, the total cost of using the CsA + ALG scheme is 1.51 million yuan using each additional QALY, but this is far more than the cost utility threshold for each QALY obtained in China [24]. On the premise of ensuring the effectiveness of diagnosis and treatment, CsA + TPO-RA can be used as a cost-effective option for the treatment of SAA, which can maximize the benefits of patients, further reduce the economic pressure of patients, reduce the burden of disease, reduce the pressure of medical insurance funds, and achieve multi-benefits.

3.3 Advantages of the study design

In this study, patients with classical and atypical SAA treatment regimens were selected as the subjects to carry out a cohort study, which is the first time in China to analyze the cost-effectiveness of different treatment regimens for patients with hematological malignancies from an economic point of view. The study included the loss of income due to illness, the loss of income during the treatment of accompanying staff and consumption expenditure at the cost of patients, which more objectively reflected the economic burden of patients. The mature EQ-5D scale was used to obtain the utility values of patients at different stages, to ensure the stability of the research results, and to test the benefits of different schemes for patients with scientific data, so as to maximize the benefits for patients on the basis of ensuring the effectiveness of diagnosis and treatment, and to provide more possibilities and choices for patients.

The study has certain limitations with small sample size per group, lack of detail in other expenditures, and short follow-up time and we interpret the results with caution. Referring to previous studies, follow-up studies for at least one year or even longer are generally needed to evaluate the effectiveness of different treatment options on China's health care system or patients more objectively and impartially. Generally speaking, longer studies for at least six months to one year are needed. In a word, further research is needed to evaluate and analyze whether the different treatment options for SAA patients are really practical and have the influence of promoting the cost-utility reform of China's health care system. It is also necessary to make careful scientific design, use a variety of economic evaluation tools to analyze other different treatment methods and means of SAA patients, make a comprehensive and systematic assessment, and clarify which scheme is more beneficial to patients, but also to the health care system and the whole society.

McKee N. Diagnosing and treating severe aplastic anemia. JAAPA. 2015 Sep;28(9):36-8. doi: 10.1097/01.JAA.0000470975.32388.bc.PMID: 26302322.
Li JP,Peng GX,Ye L, Li Y,Yang WR, Li Y,Fan HH, Zhao X, Zhou K, Jing LP, Zhang L,Zhang FK. Retreatment with immunosuppression for 23 patients with refractory or relapsed severe aplastic anemia.Zhonghua Xue Ye Xue Za Zhi.2020 Aug 14;41(8):661-665.Chinese.doi: 10.3760/cma.j.issn.0253-2727.2020.08.008.PMID:32942820; PMCID:PMC7525170.
Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017 Mar 16;129(11):1428-1436. doi: 10.1182/blood-2016-08-693481.Epub 2017 Jan 17.PMID:28096088.
Patel BA,Townsley DM, Scheinberg P.Immunosuppressive therapy in severe aplastic anemia.Semin Hematol.2022 Jan;59(1):21-29.doi:10.1053/j.seminhematol.2022.01.002. Epub 2022 Jan 19.PMID: 35491055.
Zhang FK. How I treat refractory sever aplastic anemia. Zhonghua Xue Ye Xue Za Zhi. 2020 Sep 14;41(9):705-709. Chinese. doi: 10.3760/cma.j.issn.0253-2727.2020.09.001. PMID: 33113600; PMCID: PMC7595865.
Bacigalupo A,Sica S.Alternative donor transplants for severe aplastic anemia:current experience.Semin Hematol.2016 Apr;53(2):115-9.doi:10.1053/j.seminhematol. 2016.01.002.Epub 2016 Jan 15.PMID: 27000736.
Ades L, Mary J-Y, Robin M et al. Long-term outcome after bone marrow transplantation for severe aplastic anemia.Blood 2004;103(7):2490–7.doi:10.1182/blood-2003-07-2546.
Locasciulli A, Oneto R, Bacigalupo A et al.Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation(EBMT).Haematologica 2007;92(1):11–8.
Kennedy-Nasser AA, Leung KS, Mahajan A et al. Comparable outcomes of matched-related and alternative donor stem cell transplantation for pediatric severe aplastic anemia.Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant 2006;12(12):1277–84.doi:10.1016/j.bbmt.2006.07.011.
Gupta V, Eapen M, Brazauskas R et al.Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors. Haematologica 2010;95(12):2119–25. doi:10.3324/haematol.2010.026682.
Afable MG 2nd, Shaik M, Sugimoto Y, et al.Efficacy of rabbit anti-thymocyte globulin in severe aplastic anemia. Haematologica.2011;96(9):1269-1275.
Vallejo C, Montesinos P, Polo M, et al; Bone Marrow Failure Spanish Study Group (PethemaGETH).Rabbit antithymocyte globulin versus horse antithymocyte globulin for treatment of acquired aplastic anemia: a retrospective analysis.Ann Hematol.2015;94(6):947-954.
Deyell RJ, Shereck EB, Milner RA, Schultz KR.Immunosuppressive therapy without hematopoietic growth factor exposure in pediatric acquired aplastic anemia.Pediatr Hematol Oncol.2011;28(6):469-478.
Scheinberg P. Novel therapeutic choices in immune aplastic anemia. F1000Res.2020 Sep 10;9:F1000 Faculty Rev-1118.doi: 10.12688/f1000research.22214.1.PMID:32953089;PMCID: PMC7484718.
Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program.2018 Nov 30;2018(1):450-456.doi:10.1182/asheducation-2018.1.450. PMID:30504345;PMCID:PMC6245975.
Abraham P, Sarkar R, Brandel MG, Wali AR, Rennert RC, Lopez Ramos C,Padwal J,Steinberg JA,Santiago-Dieppa DR,Cheung V, Pannell JS, Murphy JD, Khalessi AA.Cost-effectiveness of Intraoperative MRI for Treatment of High-Grade Gliomas. Radiology.2019 Jun;291(3):689-697.doi: 10.1148/radiol.2019182095.Epub 2019 Mar 26.PMID:30912721; PMCID:PMC6543900.
D'Amico F,Feliu-Soler A,Montero-Marín J, Peñarrubía-María MT,Navarro-Gil M,Van Gordon W, García-Campayo J,Luciano JV.Cost-Utility of Attachment-Based Compassion Therapy (ABCT)for Fibromyalgia Compared to Relaxation: A Pilot Randomized Controlled Trial.J Clin Med. 2020 Mar 7;9(3):726. doi:10.3390/jcm9030726.PMID:32156065; PMCID:PMC7141201.
Higgins, A.M.;Harris, A.H.Health economic methods: Cost-minimization,cost-effectiveness,cost-utility,and cost-benefit evaluations.Crit.Care Clin.2012,28,11–24.
Konnopka, A.;Schaefert, R.;Heinrich, S.;Kaufmann, C.; Luppa, M.;Herzog, W.;König, H.H.Economics of medically unexplained symptoms: A systematic review of the literature. Psychother.Psychosom.2012,81,265–275.
Badia, X.; Roset, M.; Montserrat, S.; Herdman, M.; Segura, A. The Spanish version of EuroQol: A descriptionand its applications: European Quality of Life scale. Med. Clin. 1999, 112, 79–85.
Chang TJ，Tarn YH，Hsieh CL，et al.Taiwanese Version of the EQ-5D: Validation in a representative sample of the Taiwanese population[J]. J Formos Med Assoc，2007，106(12):1023-1031.
Luo N，Chew LH，Fong KY，et al. Do English and Chinese EQ-5D versions demonstrate measurement equivalence? An exploratory study[J].Health and Quality of Life Outcomes，2003，1:7.
http://www.stats.gov.cn/tjsj/ndsj/2020/indexch.htm
http://www.stats.gov.cn/tjsj/ndsj/2021/indexch.htm
http://tjzd.stats.gov.cn/dcfw/system/2021/01/21/010004408.shtml
http://tjzd.stats.gov.cn/dcfw/system/2022/01/21/010004736.shtml
Badia, X.; Roset, M.; Herdman, M.; Kind, P. A comparison of United Kingdom and Spanish general population
time trade-off values for EQ-5D health states. Med. Decis. Making 2001, 21, 7–16. [CrossRef] [PubMed]
Vo P, Onstad L, Flowers ME, Storb R. Cancers after HLA-matched related bone marrow transplantation for aplastic anemia. Bone Marrow Transplant. 2022 Jan;57(1):83-88. doi: 10.1038/s41409-021-01498-1. Epub 2021 Oct 16. PMID: 34657145; PMCID: PMC8738111.
Marsh JCW, Risitano AM, Mufti GJ. The Case for Upfront HLA-Matched Unrelated Donor Hematopoietic Stem Cell Transplantation as a Curative Option for Adult Acquired Severe Aplastic Anemia. Biol Blood Marrow Transplant. 2019 Sep;25(9):e277-e284. doi: 10.1016/j.bbmt.2019.05.012. Epub 2019 May 24. PMID: 31129354.
Heublein S, Wehner F, Höchsmann B, Hochhaus A, Hartmann M, La Rosée P. Modelling cost effectiveness of horse antithymocyte globulin for treating severe aplastic anaemia in Germany. Ann Hematol. 2013 Jun;92(6):825-30. doi: 10.1007/s00277-013-1688-2. Epub 2013 Feb 13. PMID: 23404582.

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Cost utility Analysis of Adult Patients with Severe Aplastic Anemia Based on Single-center Cohort Study

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