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1.
Clin Transplant ; 38(6): e15375, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-39031785

RESUMO

BACKGROUND: Cytomegalovirus infection (CMV) is a common complication after allogeneic hematopoietic stem cell transplantation (AHSCT). CMV infection increases transplantation costs; however, the extent of the financial burden may vary in different countries. This study aims to determine the clinical and economic impact of CMV infection in patients undergoing AHSCT in a middle-income country. METHODS: A total of 150 adult and pediatric patients post-AHSCT were included for analysis. In addition to incidence of CMV infections, data on graft versus host disease (GVHD) were also collected. Standard hospital charges for AHSCT and any additional transplantation-related expenditure within 12 months were also retrieved in 104 patients. RESULTS: CMV infection, acute GVHD and chronic GVHD occurred in 38.7%, 60.7%, and 22.0% of patients, respectively. Patients with CMV infections had higher readmission rates compared to those who did not (67.2% vs. 47.8%; p = 0.020). Additional expenditure was seen in HLA-haploidentical AHSCT and CMV infection (MYR11 712.25/USD2 504.49; p < 0.0001 and MYR5 807.24/USD1 241.79; p = 0.036), respectively. CONCLUSION: This single-center study demonstrated that patients who underwent HLA-haploidentical AHSCT and subsequently developed CMV infection had higher transplantation expenditures compared to those who had matched-related transplantation. Further studies should be conducted to evaluate if primary prophylaxis against CMV is cost-effective, especially in patients who undergo HLA-haploidentical AHSCT.


Assuntos
Infecções por Citomegalovirus , Citomegalovirus , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Transplante Homólogo , Humanos , Infecções por Citomegalovirus/economia , Infecções por Citomegalovirus/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Masculino , Feminino , Adulto , Seguimentos , Citomegalovirus/isolamento & purificação , Criança , Doença Enxerto-Hospedeiro/economia , Doença Enxerto-Hospedeiro/etiologia , Adolescente , Pessoa de Meia-Idade , Adulto Jovem , Prognóstico , Fatores de Risco , Pré-Escolar , Estudos Retrospectivos , Incidência , Condicionamento Pré-Transplante/efeitos adversos
2.
Ann Hematol ; 103(7): 2455-2462, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38809456

RESUMO

PURPOSE: To evaluate whether BeEAM is an alternative to BEAM for autologous stem cell transplantation (ASCT) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). METHODS: Data of 60 patients with relapsed or refractory DLBCL who underwent ASCT from January 2018 to June 2023 in our center, including 30 patients in the BeEAM group and 30 patients in the BEAM group, were retrospectively analyzed. The time to hematopoietic reconstitution, treatment-related adverse events, number of hospitalization days, hospitalization cost, and survival benefit were compared between the two groups. RESULTS: The clinical characteristics of the patients did not significantly differ between the two groups. The median number of reinfused CD34 + cells was 5.06 × 106/kg and 5.17 × 106/kg in the BeEAM and BEAM groups, respectively, which did not significantly different (p = 0.8829). In the BeEAM and BEAM groups, the median time to neutrophil implantation was 10.2 and 10.27 days, respectively (p = 0.8253), and the median time to platelet implantation was 13.23 and 12.87 days, respectively (p = 0.7671). In the BeEAM and BEAM groups, the median hospitalization duration was 30.37 and 30.57 days, respectively (p = 0.9060), and the median hospitalization cost was RMB 83,425 and RMB 96,235, respectively (p = 0.0560). The hospitalization cost was lower in the BeEAM group. The most common hematologic adverse events were grade ≥ 3 neutropenia and thrombocytopenia, whose incidences were similar in the two groups. The most common non-hematologic adverse events were ≤ grade 2 and the incidences of these events did not significantly differ between the two groups. Median overall survival was not reached in either group, with predicted 5-year overall survival of 72.5% and 60% in the BeEAM and BEAM groups, respectively (p = 0.5872). Five-year progression-free survival was 25% and 20% in the BeEAM and BEAM groups, respectively (p = 0.6804). CONCLUSION: As a conditioning regimen for relapsed or refractory DLBCL, BeEAM has a desirable safety profile and is well tolerated, and its hematopoietic reconstitution time, number of hospitalization days, and survival benefit are not inferior to those of BEAM. BeEAM has a lower hospitalization cost and is an alternative to BEAM.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica , Transplante de Células-Tronco Hematopoéticas , Linfoma Difuso de Grandes Células B , Condicionamento Pré-Transplante , Transplante Autólogo , Humanos , Masculino , Feminino , Pessoa de Meia-Idade , Adulto , Linfoma Difuso de Grandes Células B/terapia , Estudos Retrospectivos , Condicionamento Pré-Transplante/métodos , Transplante de Células-Tronco Hematopoéticas/economia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Citarabina/administração & dosagem , Citarabina/uso terapêutico , Carmustina/administração & dosagem , Melfalan/administração & dosagem , Melfalan/uso terapêutico , Idoso , Etoposídeo/administração & dosagem , Etoposídeo/uso terapêutico , Ciclofosfamida/administração & dosagem , Ciclofosfamida/uso terapêutico , Taxa de Sobrevida
3.
Pharmacoeconomics ; 42(6): 693-714, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38684631

RESUMO

BACKGROUND AND OBJECTIVE: Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months. METHODS: We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase 1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses. RESULTS: Patients treated with lovo-cel were predicted to survive 23.84 years longer on average (standard deviation [SD], 12.80) versus common care (life expectancy, 62.24 versus 38.40 years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10.20 (SD, 4.10) and direct costs avoided of $1,329,201 (SD, $1,346,446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1.19 (SD, 1.38) and indirect costs avoided of $540,416 (SD, $262,353) per patient. Including lovo-cel costs ($3,282,009 [SD, $29,690] per patient) resulted in incremental cost-effectiveness ratios of $191,519 and $124,051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs. CONCLUSIONS: Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden.


Assuntos
Anemia Falciforme , Análise Custo-Benefício , Terapia Genética , Anos de Vida Ajustados por Qualidade de Vida , Humanos , Anemia Falciforme/terapia , Terapia Genética/economia , Estados Unidos , Adulto , Feminino , Masculino , Adolescente , Transplante de Células-Tronco Hematopoéticas/economia , Criança , Qualidade de Vida , Adulto Jovem , Modelos Econômicos , Pessoa de Meia-Idade , Recidiva
4.
Leuk Lymphoma ; 65(7): 922-931, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38567630

RESUMO

The efficacy of chimeric antigen receptor (CAR) T-cell therapy for large B-cell lymphoma (LBCL) is well-established. This study, using the Premier PINC AI Healthcare Database, assessed hospital costs and healthcare resource utilization (HRU) between CAR T-cell therapy and autologous hematopoietic cell transplant (AHCT) for 733 LBCL patients from 01/01/2017-04/30/2021 (166 CAR T and 567 AHCT from 37 US hospital systems. CAR T-cell therapy had higher index costs but lower non-pharmacy costs, shorter hospital stays, lower ICU utilization than AHCT. The CAR T-cell cohort also presented fewer preparatory costs and HRU. At a 180-day follow-up, AHCT had lower hospitalization rates and costs. Overall, despite higher index costs, CAR T-cell therapy has lower non-pharmacy costs and HRU during the index procedure and requires less preparation time with lower preparation HRUs and costs than AHCT. This has important implications for resource management and informed decision-making for stakeholders.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Imunoterapia Adotiva , Linfoma Difuso de Grandes Células B , Transplante Autólogo , Humanos , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/métodos , Imunoterapia Adotiva/economia , Imunoterapia Adotiva/métodos , Masculino , Feminino , Pessoa de Meia-Idade , Estados Unidos , Linfoma Difuso de Grandes Células B/terapia , Linfoma Difuso de Grandes Células B/economia , Idoso , Receptores de Antígenos Quiméricos , Adulto , Custos de Cuidados de Saúde/estatística & dados numéricos , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Recursos em Saúde/economia , Recursos em Saúde/estatística & dados numéricos , Custos Hospitalares/estatística & dados numéricos , Terapia Combinada/economia
5.
Eur J Oncol Nurs ; 70: 102584, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38631123

RESUMO

PURPOSE: Financial toxicity (FT) refers to the subjective perception of financial distress resulting from objective economic strain due to illness, exerting a detrimental influence on health outcomes. This study aimed to describe FT among allogeneic haematopoietic stem cell transplant (allo-HSCT) recipients within a public health framework, employing a social determinants of health approach. METHODS: A multi-centre cross-sectional study involving adult allo-HSCT patients was conducted across three public hospitals in Madrid. FT was assessed using a validated COST scale (range 0-44; lower scores indicating higher FT). Patient-administered paper/online questionnaires were utilized to collect data on sociodemographic, socioeconomic, clinical, and healthcare access variables. Descriptive, non-parametric univariate statistical analysis and multiple linear regression models were performed. RESULTS: Sixty-six patients, with a mean age: 52.5 years (SD: 11.5), 50% women, 28.7% displaced to Madrid for HSCT, and 71.4% lacking financial support were included. The median FT score was 20 points (IQR 12-27.25). Independent factors associated with higher FT included being females (Coef = -3.26; p = 0.079), perceived income loss after HSCT (Coef = -6.81; p < 0.001) and a monthly household income of ≤1000 € compared to 1001-2500€ (Coef = 8.29; p = 0.005) or >2500 € (Coef = 15.75; p < 0.001). CONCLUSIONS: Despite the limited sample size, our findings underscore the presence of financial toxicity among allo-HSCT patients, shaped by social determinants of health. Recognizing and addressing FT within the HSCT process is essential to mitigate social inequalities in health.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Determinantes Sociais da Saúde , Humanos , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Feminino , Masculino , Pessoa de Meia-Idade , Estudos Transversais , Adulto , Espanha , Inquéritos e Questionários , Transplante Homólogo , Idoso , Estresse Financeiro , Fatores Socioeconômicos , Efeitos Psicossociais da Doença
6.
Appl Health Econ Health Policy ; 22(4): 569-582, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38448720

RESUMO

BACKGROUND: Survival outcomes for multiple myeloma have improved dramatically since the introduction of novel therapeutic agents. While these drugs are highly effective in improving survival outcomes and quality of life in patients with multiple myeloma, they come at a significant cost. We assessed the cost-effectiveness of bortezomib-based triplet or quadruplet drug regimens in isolation and followed by autologous hematopoietic stem cell transplantation (AHSCT) for the treatment of newly diagnosed multiple myeloma (NDMM) in the Indian context. METHODS: A Markov model was developed to assess the health and economic outcomes of novel drug regimens with and without AHSCT for the treatment of NDMM in India. We estimated the lifetime quality-adjusted life-years (QALYs) and costs in each scenario. The incremental cost-effectiveness ratios (ICERs) were computed and compared against the current willingness-to-pay threshold of a one-time per capita gross domestic product of ₹146,890 (US$1,927.70) for India. Parameter uncertainty was assessed through Monte Carlo probabilistic sensitivity analysis. RESULTS: Among seven treatment sequences, the VCd (bortezomib, cyclophosphamide, dexamethasone) alone arm has the lowest cost and health benefits as compared to four treatment sequences, namely VTd (bortezomib, thalidomide, dexamethasone) alone, VRd (bortezomib, lenalidomide, dexamethasone) alone, VRd plus AHSCT and DVRd (Daratumumab, bortezomib, lenalidomide, dexamethasone) plus AHSCT. It was found that VTd plus AHSCT and VCd plus AHSCT arms were extendedly dominated (ED) by combination of two alternative treatments. Among the five non-dominated strategies, VRd has a lowest incremental cost of ₹ 2,20,093 (US$2,888) per QALY gained compared to VTd alone followed by VRd plus AHSCT [₹3,14,530 (US$4,128) per QALY gained] in comparison to VRd alone. None of the novel treatment sequences were found to be cost-effective at the current WTP threshold of ₹1,46,890 (US$1,927.7). CONCLUSION: At the current WTP threshold of one-time per capita GDP (₹ 146,890) of India, VRd alone and VRd plus AHSCT has 38.1% and 6.9% probability to be cost-effective, respectively. Reduction in current reimbursement rates of novel drugs, namely VRd, lenalidomide, and pomalidomide plus dexamethasone under national insurance program and societal cost of transplant by 50%, would make VRd plus AHSCT and VTd plus AHSCT cost-effective at an incremental cost of ₹40,671 (US$34) and ₹97,639 (US$1,281) per QALY gained, respectively.


Assuntos
Bortezomib , Análise Custo-Benefício , Transplante de Células-Tronco Hematopoéticas , Cadeias de Markov , Mieloma Múltiplo , Anos de Vida Ajustados por Qualidade de Vida , Mieloma Múltiplo/tratamento farmacológico , Humanos , Índia , Transplante de Células-Tronco Hematopoéticas/economia , Bortezomib/uso terapêutico , Bortezomib/economia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Dexametasona/uso terapêutico , Dexametasona/economia , Dexametasona/administração & dosagem , Masculino , Feminino , Lenalidomida/uso terapêutico , Pessoa de Meia-Idade , Ciclofosfamida/uso terapêutico , Ciclofosfamida/economia , Talidomida/economia , Talidomida/uso terapêutico , Talidomida/análogos & derivados , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Anticorpos Monoclonais
7.
Transplant Cell Ther ; 30(5): 542.e1-542.e29, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38331192

RESUMO

This scoping review summarizes the evidence regarding healthcare resource utilization (HRU) and costs associated with allogeneic hematopoietic stem cell transplantation (allo-HSCT). This study was conducted in accordance with the Joanne Briggs Institute methodology for scoping reviews. The PubMed, Embase, and Health Business Elite Electronic databases were searched, in addition to grey literature. The databases were searched from inception up to November 2022. Studies that reported HRU and/or costs associated with adult (≥18 years) allo-HSCT were eligible for inclusion. Two reviewers independently screened 20% of the sample at each of the 2 stages of screening (abstract and full text). Details of the HRU and costs extracted from the study data were summarized, based on the elements and timeframes reported. HRU measures and costs were combined across studies reporting results defined in a comparable manner. Monetary values were standardized to 2022 US Dollars (USD). We identified 43 studies that reported HRU, costs, or both for allo-HSCT. Of these studies, 93.0% reported on costs, 81.4% reported on HRU, and 74.4% reported on both. HRU measures and cost calculations, including the timeframe for which they were reported, were heterogeneous across the studies. Length of hospital stay was the most frequently reported HRU measure (76.7% of studies) and ranged from a median initial hospitalization of 10 days (reduced-intensity conditioning [RIC]) to 73 days (myeloablative conditioning). The total cost of an allo-HSCT ranged from $63,096 (RIC) to $782,190 (double umbilical cord blood transplantation) at 100 days and from $69,218 (RIC) to $637,193 at 1 year (not stratified). There is heterogeneity in the reporting of HRU and costs associated with allo-HSCT in the literature, making it difficult for clinicians, policymakers, and governments to draw definitive conclusions regarding the resources required for the delivery of these services. Nevertheless, to ensure that access to healthcare meets the necessary high cost and resource demands of allo-HSCT, it is imperative for clinicians, policymakers, and government officials to be aware of both the short- and long-term health resource requirements for this patient population. Further research is needed to understand the key determinants of HRU and costs associated with allo-HSCT to better inform the design and delivery of health care for HSCT recipients and ensure the quality, safety, and efficiency of care.


Assuntos
Custos de Cuidados de Saúde , Transplante de Células-Tronco Hematopoéticas , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Custos de Cuidados de Saúde/estatística & dados numéricos , Transplante Homólogo/economia , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Recursos em Saúde/economia , Recursos em Saúde/estatística & dados numéricos
8.
Neurol Sci ; 45(7): 3379-3387, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38277051

RESUMO

BACKGROUND: Autologous haematopoietic stem cell transplantation (AHSCT) is a highly effective one-off treatment for relapsing-remitting multiple sclerosis (RR-MS), potentially representing an optimal front-loading strategy for costs. OBJECTIVE: Exploring cost/effectiveness of AHSCT and high-efficacy disease-modifying treatments (HE-DMTs) in RR-MS, estimating costs at our centre in Italy, where National Health Service (NHS) provides universal health coverage. METHODS: Costs (including drugs, inpatient/outpatient management) for treatment with AHSCT and HE-DMTs were calculated as NHS expenditures over 2- and 5-year periods. Cost-effectiveness for each treatment was estimated as "cost needed to treat" (CNT), i.e. expense to prevent relapses, progression, or disease activity (NEDA) in one patient over n-years, retrieving outcomes from published studies. RESULTS: Costs of AHSCT and HE-DMTs were similar over 2 years, whereas AHSCT was cheaper than most HE-DMTs over 5 years (€46 600 vs €93 800, respectively). When estimating cost-effectiveness of treatments, over 2 years, mean CNT of HE-DMTs for NEDA was twofold that of AHSCT, whereas it was similar for relapses and disability. Differences in CNT were remarkable over 5 years, especially for NEDA, being mean CNT of HE-DMTs €382 800 vs €74 900 for AHSCT. CONCLUSIONS: AHSCT may be highly cost-effective in selected aggressive RR-MS. Besides priceless benefits for treated individuals, cost-savings generated by AHSCT may contribute to improving healthcare assistance at a population level.


Assuntos
Análise Custo-Benefício , Transplante de Células-Tronco Hematopoéticas , Esclerose Múltipla Recidivante-Remitente , Transplante Autólogo , Humanos , Esclerose Múltipla Recidivante-Remitente/economia , Esclerose Múltipla Recidivante-Remitente/terapia , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/métodos , Transplante Autólogo/economia , Masculino , Feminino , Adulto , Itália , Resultado do Tratamento , Pessoa de Meia-Idade
9.
PLoS One ; 17(1): e0261871, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-35061709

RESUMO

BACKGROUNDS: An increasing incidence of Acute Myeloid Leukaemia (AML) has been reported in several Western countries. However, the epidemiology of AML in Asia is very limited. According to the National Comprehensive Cancer Network (NCCN) guideline of AML, a range of conventional therapy options is available to AML patients. Nevertheless, different treatment strategies may result in diverse healthcare utilization and costs. Understanding the treatment patterns, healthcare utilization and costs of AML would thus be essential for clinicians and policymakers to optimize the treatment strategies of AML. OBJECTIVES: The objective of this study was to investigate the incidence, treatment patterns, healthcare utilization and costs of AML in Taiwan using a nationwide population database. METHODS: We retrospectively identified AML patients diagnosed from 2006 to 2015 from the Taiwan Cancer Registry Database (TCRD) and estimated the epidemiology of AML in Taiwan. The TCRD was linked to National Health Insurance Research Database (NHIRD) to collect the treatment patterns and health care utilization. Patients diagnosed with AML from 2011 to 2015 were further identified to analyze treatment patterns, healthcare utilization and costs. RESULTS: The crude annual incidence of AML increased from 2.78 to 3.21 cases per 100,000 individuals from 2006 to 2015. However, the age-standardized rate (ASRs) of AML slightly declined from 2.47 to 2.41 cases per 100,000 individuals in the same period. Among 2,179 AML patients who received induction therapy (median age: 56 years), most of them (n = 1744; 80.04%) received standard-dose cytarabine (SDAC) regimen. The remaining 162 patients received high dose cytarabine (HDAC) and 273 patients received non-standard dose cytarabine (N-SDAC) regimen as the induction therapy. The median medical costs in our study for patients treated with chemotherapy alone was $42,271 for HDAC, $36,199 for SDAC and $36,250 for N-SDAC. For those who received hematopoietic stem cell transplantation (HSCT) after induction therapy, their median medical costs were $78,876 for HDAC, $78,593 for SDAC and $79,776 for N-SDAC. CONCLUSIONS: This study is the first population-based study conducted in Asia to provide updated and comprehensive information on epidemiology, treatment patterns and healthcare resource utilization and costs of AML.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica , Atenção à Saúde/economia , Transplante de Células-Tronco Hematopoéticas/economia , Leucemia Mieloide Aguda , Sistema de Registros , Adulto , Idoso , Aloenxertos , Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Custos e Análise de Custo , Feminino , Humanos , Incidência , Leucemia Mieloide Aguda/economia , Leucemia Mieloide Aguda/epidemiologia , Leucemia Mieloide Aguda/terapia , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Taiwan/epidemiologia
10.
Ann Hematol ; 101(3): 667-679, 2022 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-35091794

RESUMO

This study aims to evaluate the efficacy, safety, and long-term cost-effectiveness of fixed-dose busulfan (Bu) administration and pharmacokinetically (PK) guided adjustment of Bu dose for patients who underwent hematopoietic stem cell transplantation. The efficacy and safety of both dosing strategies were compared using a systematic review and meta-analysis. A Markov model was used in estimating relevant cost and health outcomes from the perspective of the health system. The primary outcomes of interest were lifetime cost, quality adjusted life-years (QALYs) gained, and incremental cost-effectiveness ratio (ICER) in dollar per QALY gained. Results showed that progression-free survival and overall survival in the PK-guided group were higher than that in the fixed-dose group, and the PK-guided group was associated with low non-relapse mortality and relapse rate. In contrast to safety, the incidence of acute graft-versus-host disease (GVHD) was the same in the two groups (P > 0.05). Cost-effectiveness analysis showed that the QALY of the PK-guided group (12.8135 QALYs and $582,475.07) increased by 2.0609 relative to that in the fixed-dose group (10.7526 QALYs and $562,833.20), and the ICER was $9530.72/QALY. One-way and probability sensitivity analyses confirmed the reliability of the results. In conclusion, the PK-guided approach has higher efficacy and is safer.


Assuntos
Bussulfano/uso terapêutico , Imunossupressores/uso terapêutico , Bussulfano/administração & dosagem , Bussulfano/economia , Bussulfano/farmacocinética , Análise Custo-Benefício , Doença Enxerto-Hospedeiro/economia , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/prevenção & controle , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Imunossupressores/administração & dosagem , Imunossupressores/economia , Imunossupressores/farmacocinética , Anos de Vida Ajustados por Qualidade de Vida
11.
JAMA Pediatr ; 176(2): 176-184, 2022 02 01.
Artigo em Inglês | MEDLINE | ID: mdl-34779842

RESUMO

Importance: Lifelong immunoglobulin replacement therapy (IRT) is standard-of-care treatment for congenital agammaglobulinemia but accrues high annual costs ($30 000-$90 000 per year) and decrements to quality of life over patients' life spans. Hematopoietic stem cell transplant (HSCT) offers an alternative 1-time therapy, but has high morbidity and mortality. Objective: To evaluate the cost utility of IRT vs matched sibling donor (MSD) and matched unrelated donor (MUD) HSCT to treat patients with agammaglobulinemia in the US. Design, Setting, and Participants: This economic evaluation used Markov analysis to model the base-case scenario of a patient aged 12 months with congenital agammaglobulinemia receiving lifelong IRT vs MSD or MUD HSCT. Costs, probabilities, and quality-of-life measures were derived from the literature. Microsimulations estimated premature deaths for each strategy in a virtual cohort. One-way sensitivity and probabilistic sensitivity analyses evaluated uncertainty around parameter estimates performed from a societal perspective over a 100-year time horizon. The threshold for cost-effective care was set at $100 000 per quality-adjusted life-year (QALY). This study was conducted from 2020 across a 100-year time horizon. Exposures: Immunoglobulin replacement therapy vs MSD or MUD HSCT for treatment of congenital agammaglobulinemia. Main Outcomes and Measures: The primary outcomes were incremental cost-effectiveness ratio (ICER) expressed in 2020 US dollars per QALY gained and premature deaths associated with each strategy. Results: In this economic evaluation of patients with congenital agammaglobulinemia, lifelong IRT cost more than HSCT ($1 512 946 compared with $563 776 [MSD] and $637 036 [MUD]) and generated similar QALYs (20.61 vs 17.25 [MSD] and 17.18 [MUD]). Choosing IRT over MSD or MUD HSCT yielded ICERs of $282 166 per QALY gained over MSD and $255 633 per QALY gained over MUD HSCT, exceeding the US willingness-to-pay threshold of $100 000/QALY. However, IRT prevented at least 2488 premature deaths per 10 000 microsimulations compared with HSCT. When annual IRT price was reduced from $60 145 to below $29 469, IRT became the cost-effective strategy. Findings remained robust in sensitivity and probabilistic sensitivity analyses. Conclusions and Relevance: In the US, IRT is more expensive than HSCT for agammaglobulinemia treatment. The findings of this study suggest that IRT prevents more premature deaths but does not substantially increase quality of life relative to HSCT. Reducing US IRT cost by 51% to a value similar to IRT prices in countries implementing value-based pricing may render it the more cost-effective strategy.


Assuntos
Agamaglobulinemia/terapia , Análise Custo-Benefício , Doenças Genéticas Ligadas ao Cromossomo X/terapia , Transplante de Células-Tronco Hematopoéticas/economia , Imunoglobulinas Intravenosas/administração & dosagem , Imunoglobulinas Intravenosas/economia , Humanos , Cadeias de Markov , Estados Unidos
12.
Hematology ; 26(1): 950-955, 2021 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-34904529

RESUMO

OBJECTIVES: To compare the efficacies and costs between pegfilgrastim and filgrastim prophylaxis for FN post-ASCT for lymphoma and multiple myeloma patients. METHODS: 43 patients who received pegfilgrastim (6 mg) were compared to a retrospective cohort of 129 patients that had received filgrastim post-ASCT. Hematopoietic recovery time, FN incidence and treatment costs were assessed and compared. RESULTS: The mean time to absolute neutrophil count engraftment was 8.72 ± 2.38 days for the prospective pegfilgrastim group and 9.87 ± 3.13 days for the retrospective filgrastim group (P = 0.027). The incidence of FN was 18.60% and 50.39% in prospective pegfilgrastim and retrospective filgrastim groups, respectively (P = 0.000). The mean cost of filgrastim was $617.22 ± 37.87, compared with $525.78 for pegfilgrastim (P = 0.032). DISCUSSION: Convenience, effectiveness, and safety of prophylaxis for FN in the prospective pegfilgrastim group were significantly improved compared to the retrospective filgrastim group in ASCT patients. CONCLUSION: Pegfilgrastim prophylaxis was more effective and convenient than filgrastim for FN prophylaxis in patients post-ASCT, especially for MM patients.


Assuntos
Neutropenia Febril/prevenção & controle , Filgrastim/uso terapêutico , Fármacos Hematológicos/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Linfoma/terapia , Mieloma Múltiplo/terapia , Polietilenoglicóis/uso terapêutico , Adolescente , Adulto , Idoso , Análise Custo-Benefício , Neutropenia Febril/economia , Feminino , Filgrastim/efeitos adversos , Filgrastim/economia , Fármacos Hematológicos/efeitos adversos , Fármacos Hematológicos/economia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Linfoma/economia , Masculino , Pessoa de Meia-Idade , Mieloma Múltiplo/economia , Polietilenoglicóis/efeitos adversos , Polietilenoglicóis/economia , Estudos Prospectivos , Estudos Retrospectivos , Transplante Autólogo/efeitos adversos , Transplante Autólogo/economia , Resultado do Tratamento , Adulto Jovem
13.
Hematology Am Soc Hematol Educ Program ; 2021(1): 275-280, 2021 12 10.
Artigo em Inglês | MEDLINE | ID: mdl-34889386

RESUMO

Allogeneic hematopoietic cell transplantation (HCT) is particularly susceptible to racial, socioeconomic, and geographic disparities in access and outcomes given its specialized nature and its availability in select centers in the United States. Nearly all patients who need HCT have a potential donor in the current era, but racial minority populations are less likely to have an optimal donor and often rely on alternative donor sources. Furthermore, prevalent health care disparity factors are further accentuated and can be barriers to access and referral to a transplant center. Research has primarily focused on defining and quantifying a variety of social determinants of health and their association with access to allogeneic HCT, with a focus on race/ethnicity and socioeconomic status. However, research on interventions is lacking and is an urgent unmet need. We discuss the role of racial, socioeconomic, and geographic disparities in access to allogeneic HCT, along with policy changes to address and mitigate them and opportunities for future research.


Assuntos
Disparidades em Assistência à Saúde , Transplante de Células-Tronco Hematopoéticas , Minorias Étnicas e Raciais , Acessibilidade aos Serviços de Saúde/economia , Disparidades em Assistência à Saúde/economia , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Fatores Socioeconômicos , Transplante Homólogo/economia , Estados Unidos
14.
Hematology Am Soc Hematol Educ Program ; 2021(1): 264-274, 2021 12 10.
Artigo em Inglês | MEDLINE | ID: mdl-34889391

RESUMO

Allogeneic hematopoietic cell transplantation (allo-HCT) is a highly complex, costly procedure for patients with oncologic, hematologic, genetic, and immunologic diseases. Demographics and socioeconomic status as well as donor availability and type of health care system are important factors that influence access to and outcomes following allo-HCT. The last decade has seen an increase in the numbers of allo-HCTs and teams all over the world, with no signs of saturation. More than 80 000 procedures are being performed annually, with 1 million allo-HCTs estimated to take place by the end of 2024. Many factors have contributed to this, including increased numbers of eligible patients (older adults with or without comorbidities) and available donors (unrelated and haploidentical), improved supportive care, and decreased early and late post-HCT mortalities. This increase is also directly linked to macro- and microeconomic indicators that affect health care both regionally and globally. Despite this global increase in the number of allo-HCTs and transplant centers, there is an enormous need for increased access to and improved outcomes following allo-HCT in resource-constrained countries. The reduction of poverty, global economic changes, greater access to information, exchange of technologies, and use of artificial intelligence, mobile health, and telehealth are certainly creating unprecedented opportunities to establish collaborations and share experiences and thus increase patient access to allo-HCT. A specific research agenda to address issues of allo-HCT in resource-constrained settings is urgently warranted.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Seleção do Doador , Acessibilidade aos Serviços de Saúde/economia , Acessibilidade aos Serviços de Saúde/estatística & dados numéricos , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Humanos , Internacionalidade , Fatores Socioeconômicos , Transplante Homólogo/economia , Transplante Homólogo/estatística & dados numéricos
15.
Hematology Am Soc Hematol Educ Program ; 2021(1): 570-577, 2021 12 10.
Artigo em Inglês | MEDLINE | ID: mdl-34889432

RESUMO

With improvement in survival after hematopoietic cell transplantation (HCT), it has become important to focus on survivors' psychosocial issues in order to provide patient-centered care across the transplant continuum. The goals of this article are to describe updates in the literature on certain psychosocial domains (emotional/mental health and social/financial) in HCT survivors, offer a brief overview of the status of the screening and management of these complications, and identify opportunities for future practice and research. An evidence-based approach to psychosocial care can be broken down as primary (promoting health, raising awareness, and addressing risk factors), secondary (screening and directing early pharmacological and nonpharmacological interventions), and tertiary (rehabilitating, limiting disability, and improving quality of life) prevention. Implementing such an approach requires close coordination between multiple stakeholders, including transplant center staff, referring hematologist/oncologists, and other subspecialists in areas such as palliative medicine or psychiatry. Innovative models of care that leverage technology can bring these stakeholders together to fulfill unmet needs in this area by addressing barriers in the delivery of psychosocial care.


Assuntos
Transplante de Células-Tronco Hematopoéticas/psicologia , Sobreviventes/psicologia , Adolescente , Emoções , Feminino , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Masculino , Saúde Mental , Pessoa de Meia-Idade , Qualidade de Vida
16.
PLoS One ; 16(8): e0254135, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34383780

RESUMO

The number of Hematopoietic Stem Cell Transplantations has risen in the past 20 years. The practice of outpatient Hematopoietic Stem Cell Transplantation programs is increasing in an attempt to improve the quality of patient care and reduce the demand for hospital admission. A systematic review of 29 comparative studies between in-hospital and outpatient treatment of Hematopoietic Stem Cell Transplantation, with no restriction by outpatient regime was conducted. This study aims to analyse the current evidence on the effects of the outpatient model on patient-centred outcomes, comparing both in-hospital and outpatient models for autologous and allogeneic HSCT using the Triple Aim framework: health outcomes, costs and experience of care. We found evidence on improved health outcomes and quality of life, on enhanced safety and effectiveness and on reduced overall costs and hospital stays, with similar results on overall survival rates comparing both models for autologous and allogeneic patients. We also found that the outpatient Hematopoietic Stem Cell Transplantation is a safe practice as well as less costly, it requires fewer days of hospital stay both for autologous and allogeneic transplantations. Under a situation of an increasing number of transplants, rising healthcare costs and shortages of hospital capacity, incorporating outpatient models could improve the quality of care for people requiring Hematopoietic Stem Cell Transplantation programs.


Assuntos
Transplante de Células-Tronco Hematopoéticas/economia , Tempo de Internação/economia , Modelos Econômicos , Qualidade de Vida , Custos e Análise de Custo , Intervalo Livre de Doença , Transplante de Células-Tronco Hematopoéticas/mortalidade , Humanos , Taxa de Sobrevida , Transplante Autólogo , Transplante Homólogo
17.
Bull Cancer ; 108(12S): S10-S19, 2021 Dec.
Artigo em Francês | MEDLINE | ID: mdl-34247762

RESUMO

Hematopoietic cell transplantation (HCT) is the curative treatment for many malignant and non-malignant blood disorders and some solid cancers. However, transplant procedures are considered tertiary level care requiring a high degree of technicality and expertise and generating very high costs for hospital structures in developing countries as well as for patients without health insurance. During the 11th annual harmonization workshops of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to elaborate unified guidelines, for developing the transplant activity in emerging countries. Access to infrastructure must comply with international standards and therefore requires a hospital system already in place, capable of accommodating and supporting the HCT activity. In addition, the commitment of the state and the establishment for the financing of the project seems essential.


Assuntos
Países em Desenvolvimento , Transplante de Células-Tronco Hematopoéticas , Desenvolvimento de Programas , Fatores Etários , Aloenxertos , Autoenxertos , Características Culturais , Países em Desenvolvimento/economia , Apoio Financeiro , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/normas , Hospitais Especializados/organização & administração , Hospitais Especializados/normas , Humanos , Pessoas sem Cobertura de Seguro de Saúde , Equipe de Assistência ao Paciente/organização & administração , Equipe de Assistência ao Paciente/normas , Qualidade da Assistência à Saúde , Sociedades Médicas , Fatores Socioeconômicos , Atenção Terciária à Saúde/economia , Condicionamento Pré-Transplante/métodos , Condicionamento Pré-Transplante/normas
18.
Bull Cancer ; 108(10): 893-894, 2021 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-34229855

RESUMO

A successful Hematopoietic stem cell transplantation (HSCT) is often the only hope of survival for children suffering from a range of potentially life-threatening hematological malignancies. The financial, ethical, and emotional problems faced by the matched sibling donor and their recipient siblings during the HSCT are extremely complex and challenging. Herein, the authors have attempted to pen down these in the configuration of a poem.


Assuntos
Neoplasias Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas/ética , Poesia como Assunto , Temas Bioéticos , Criança , Comissão de Ética , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Índia , Consentimento Informado por Menores , Irmãos , Doadores de Tecidos/psicologia
19.
Oral Oncol ; 118: 105296, 2021 07.
Artigo em Inglês | MEDLINE | ID: mdl-33933777

RESUMO

Mucositis is one of the more frequent and costly adverse events following cancer treatment. To evaluate and report the direct economic outcomes associated with the management of mucositis across several cancer treatments we conducted a systematic review according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement. Scopus, MEDLINE/PubMed, and Embase were searched electronically and a total of 37 relevant studies were included. The costs attributable to mucositis in the hematopoietic stem cell transplantation setting ranged from 1124,47 US dollars (USD) to 299 214,14 USD per patient. The radiotherapy/chemoradiotherapy/radiotherapy plus molecular targeted therapy accounted for mucositis costs that ranged from 51,23 USD to 33 560,58 USD per patient. Costs for mucositis in the chemotherapy setting ranged from 4,18 USD to 31 963,64 USD per patient. When the cancer treatment was not specified, costs of mucositis ranged from 565,85 USD to as high as 20 279, 12 USD per patient. Mucositis costs from multimodal therapy ranged from 12,42 USD to 5670,46 USD per patient. The molecular targeted therapy setting included only one study and depending on the healthcare providers' perspective of each country evaluated, mucositis' costs ranged from 45,78 USD to 3484,91 USD per patient. Mucositis is associated with increased resource use, consultations, hospitalizations and extended hospitalizations, leading to a substantial incremental cost that exacerbates the economic burden on the patient, health plan and health system across several cancer treatments and diagnosis. More studies with a prospective evaluation of the economic costs associated with mucositis management are needed.


Assuntos
Custos de Cuidados de Saúde , Transplante de Células-Tronco Hematopoéticas , Mucosite , Neoplasias , Terapia Combinada , Transplante de Células-Tronco Hematopoéticas/economia , Hospitalização , Humanos , Mucosite/economia , Mucosite/terapia , Neoplasias/economia , Neoplasias/terapia
20.
J Clin Apher ; 36(4): 553-562, 2021 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-33710672

RESUMO

Administration of plerixafor with granulocyte-colony stimulating factor (G-CSF) mobilizes CD34+ cells much more effectively than G-CSF alone, but cost generally limits plerixafor use to patients at high risk of insufficient CD34+ cell collection based on low peripheral blood (PB) CD34+ counts following 4 days of G-CSF. We analyzed costs associated with administering plerixafor to patients with higher day 4 CD34+ cell counts to decrease apheresis days and explored the use of a fixed split dose of plerixafor instead of weight-based dosing. We analyzed 235 patients with plasma cell disorders or non-Hodgkin's lymphoma who underwent progenitor cell mobilization and autologous hematopoietic cell transplantation (AHCT) between March 2014 and December 2017. Two hundred ten (89%) received G-CSF plus Plerixafor and 25 (11%) received G-CSF alone. Overall, 180 patients (77%) collected in 1 day, 53 (22%) in 2 days and 2 (1%) in 3 days. Based on our data, we present a probabilistic algorithm to identify patients likely to require more than one day of collection using G-CSF alone. CD34+ cell yield, ANC and platelet recovery were not significantly different between fixed and standard dose plerixafor. Plerixafor enabled collection in 1 day and with estimated savings of $5000, compared to patients who did not receive plerixafor and required collection for three days. While collection and processing costs and patient populations vary among institutions, our results suggest re-evaluation of current algorithms.


Assuntos
Mobilização de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/métodos , Células-Tronco/química , Adulto , Idoso , Algoritmos , Redução de Custos , Feminino , Filgrastim/farmacologia , Fator Estimulador de Colônias de Granulócitos , Custos de Cuidados de Saúde , Humanos , Linfoma não Hodgkin/economia , Transtornos Linfoproliferativos/economia , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Risco , Células-Tronco/citologia , Transplante Autólogo , Adulto Jovem
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