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1.
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
2.
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
3.
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
4.
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 , Acesso 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
5.
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 , Acesso aos Serviços de Saúde/economia , Acesso 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
6.
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
7.
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
8.
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
9.
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
10.
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
11.
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
13.
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
14.
Blood ; 137(4): 556-568, 2021 01 28.
Artigo em Inglês | MEDLINE | ID: mdl-33104215

RESUMO

Social determinants of health, including poverty, contribute significantly to health outcomes in the United States; however, their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research database. We assembled 2 pediatric cohorts undergoing first allogeneic HCT from 2006 to 2015 at age ≤18 years, including 2053 children with malignant disease and 1696 children with nonmalignant disease. Neighborhood poverty exposure was defined a priori per the US Census definition as living in a high-poverty ZIP code (≥20% of persons below 100% federal poverty level) and used as the primary predictor in all analyses. Our primary outcome was overall survival (OS), defined as the time from HCT until death resulting from any cause. Secondary outcomes included relapse and transplantation-related mortality (TRM) in malignant disease, acute and chronic graft-versus-host disease, and infection in the first 100 days post-HCT. Among children undergoing transplantation for nonmalignant disease, neighborhood poverty was not associated with any HCT outcome. Among children undergoing transplantation for malignant disease, neighborhood poverty conferred an increased risk of TRM but was not associated with inferior OS or any other transplantation outcome. Among children with malignant disease, a key secondary finding was that children with Medicaid insurance experienced inferior OS and increased TRM compared with those with private insurance. These data suggest opportunities for future investigation of the effects of household-level poverty exposure on HCT outcomes in pediatric malignant disease to inform care delivery interventions.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Pobreza , Determinantes Sociais da Saúde , Adolescente , Causas de Morte , Criança , Pré-Escolar , Doença Crônica/mortalidade , Doença Crônica/terapia , Bases de Dados Factuais , Feminino , Seguimentos , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/mortalidade , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Humanos , Lactente , Infecções/epidemiologia , Cobertura do Seguro/estatística & dados numéricos , Masculino , Medicaid , Neoplasias/mortalidade , Neoplasias/terapia , Recidiva , Análise de Sobrevida , Transplante Homólogo , Resultado do Tratamento , Estados Unidos
15.
J Oncol Pharm Pract ; 27(4): 871-876, 2021 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-32686616

RESUMO

During autologous stem cell transplant, granulocyte colony-stimulating factors (G-CSF) serve the integral role of mobilizing hematopoietic cells into the peripheral blood for subsequent collection by leukapheresis. Filgrastim (Neupogen®) is a G-CSF and affects hematopoietic cells by stimulating growth and differentiation of neutrophils. Filgrastim-sndz (Zarxio®), a biosimilar of filgrastim, received landmark approval as the first biosimilar product approved by the FDA in the United States. As a result of the recent FDA approval, our medical center made the conversion in August 2016 from using filgrastim to filgrastim-sndz to provide patients the same benefits of the filgrastim injection at a reduced cost. This retrospective, observational cohort study evaluated the comparative efficacy of the filgrastim-sndz biosimilar in 147 patients who underwent mobilization prior to stem cell transplant with filgrastim between 1 August 2015 and 31 July 2016 or filgrastim-sndz between 1 September 2016 and 30 November 2017. The mean number of CD34 cells collected during apheresis was 7.38 × 106 in the filgrastim group and 8.86 × 106 in the filgrastim-sndz group. Filgrastim-sndz was significantly non-inferior, as the difference between filgrastim and filgrastim-sndz was -1.48 × 106 with an upper 95% confidence bound equal to -0.24 × 106 that did not include the non-inferiority margin of 1 × 106 (p = 0.0006). The median number of days of apheresis was 2 in both groups (p= 0.3273). In conclusion, the biosimilar product was non-inferior for mobilization and the conversion from filgrastim to filgrastim-sndz afforded patients similar efficacy for mobilization in stem cell transplant at a reduced cost.


Assuntos
Medicamentos Biossimilares , Filgrastim/uso terapêutico , Mobilização de Células-Tronco Hematopoéticas/métodos , Transplante de Células-Tronco Hematopoéticas/métodos , Adulto , Idoso , Idoso de 80 Anos ou mais , Antígenos CD34/imunologia , Remoção de Componentes Sanguíneos , Aprovação de Equipamentos , Feminino , Filgrastim/economia , Mobilização de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Resultado do Tratamento , Estados Unidos , United States Food and Drug Administration
16.
Placenta ; 103: 50-52, 2021 01 01.
Artigo em Inglês | MEDLINE | ID: mdl-33075720

RESUMO

Umbilical cord blood is an important graft source in the treatment of many genetic, hematologic, and immunologic disorders by hematopoietic stem cell transplantation. Millions of cord blood units have been collected and stored for clinical use since the inception of cord blood banking in 1989. However, the use of cord blood in biomedical research has been limited by access to viable samples. Here, we present a cost-effective, self-sustaining model for the procurement of fresh umbilical cord blood components for research purposes within hospital-affiliated academic institutions.


Assuntos
Pesquisa Biomédica/organização & administração , Bancos de Sangue/organização & administração , Sangue Fetal , Modelos Organizacionais , Academias e Institutos/economia , Academias e Institutos/organização & administração , Academias e Institutos/normas , Pesquisa Biomédica/economia , Pesquisa Biomédica/métodos , Pesquisa Biomédica/normas , Bancos de Sangue/economia , Bancos de Sangue/normas , Coleta de Amostras Sanguíneas/economia , Coleta de Amostras Sanguíneas/métodos , Coleta de Amostras Sanguíneas/normas , California , Análise Custo-Benefício , Feminino , Sangue Fetal/citologia , Sangue Fetal/transplante , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/métodos , Transplante de Células-Tronco Hematopoéticas/normas , Humanos , Recém-Nascido , Gravidez
18.
Eur J Haematol ; 105(5): 635-645, 2020 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-32705720

RESUMO

OBJECTIVE: Autologous stem cell transplantation (ASCT) has improved progression-free survival (PFS) and overall survival in eligible patients with newly diagnosed multiple myeloma (NDMM); however, relapse occurs. Maintenance therapy with lenalidomide (Len-Mt) extends survival and delays relapse and the subsequent initiation of costly second-line regimens. Here, we report the cost-effectiveness of Len-Mt following ASCT from a Dutch healthcare service perspective. METHODS: A partitioned survival model was developed to assess the lifetime costs and benefits for patients with NDMM. Efficacy was taken from a pooled meta-analysis of clinical trial data. Costs and subsequent therapy data were taken from sources appropriate for the Dutch market. RESULTS: Lenalidomide produced a quality-adjusted life year gain of 2.46 and a life year gain of 2.79 vs no maintenance treatment. The cost of lenalidomide was partially offset by savings of EUR 77 462 in subsequent treatment costs. The incremental cost-effectiveness ratio of Len-Mt vs no maintenance treatment was EUR 30 143. Key model drivers included subsequent therapies, dosing schedule, and time horizon. CONCLUSION: Lenalidomide is cost-effective after ASCT vs no maintenance therapy in the Netherlands. By extending PFS, lenalidomide delays the cost burdens associated with relapse and subsequent treatment lines.


Assuntos
Lenalidomida/uso terapêutico , Mieloma Múltiplo/epidemiologia , Mieloma Múltiplo/terapia , Cuidados Pós-Operatórios , Terapia Combinada/métodos , Análise Custo-Benefício , Custos de Cuidados de Saúde , Recursos em Saúde , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Lenalidomida/administração & dosagem , Lenalidomida/efeitos adversos , Quimioterapia de Manutenção , Mieloma Múltiplo/diagnóstico , Mieloma Múltiplo/mortalidade , Países Baixos/epidemiologia , Aceitação pelo Paciente de Cuidados de Saúde , Vigilância em Saúde Pública , Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , Transplante Autólogo , Resultado do Tratamento
19.
J Med Econ ; 23(9): 961-966, 2020 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-32568600

RESUMO

BACKGROUND: Neuroblastoma (NB) is notorious in childhood cancer because of its high incidence and poor prognosis. The Children's Oncology Group reported that the 3-year OS in the high-risk (HR) group is 50%, and the HR-NB with bone marrow metastasis in our center is 43.1%. Thousands of families in China suffer from the cost of NB, but the true costs of therapy are unknown; to date, no study has ever performed a detailed therapy costs analysis for NB. The objective of this study was to assess the economic burden of NB treatment in children to the family, to ultimately reduce related expenses for patients and promote the establishment of NB management policy. MATERIALS AND METHODS: Data in this cross-sectional study were collected via questionnaires completed by parents at the outpatient clinic and was verified via a computer system. Therapy costs of children with NB of differing risks were analyzed through descriptive statistics (1 CNY ≈ 0.1412 USD). RESULTS: Median direct medical costs of low risk (LR), middle risk (MR), and HR NB during treatment were 180.0 (120.0, 300.0), 200.0 (166.0, 300.0), and 650.0 (415.5, 850.0) thousand Chinese yuan (CNY), respectively. Direct non-medical costs including transportation, food, and accommodation were 60.0 (37.0, 100.0), 80.0 (60.0, 120.0), and 100.0 (80.0, 157.5) thousand CNY in the LR, MR, and HR groups, respectively. Additionally, parents accrued work absences to attend treatment, and lost a total of 100.0 (50.0, 150.0) thousand CNY in indirect costs. LIMITATIONS: Families whose children had relapsed or died were excluded from this analysis and therefore limited the conclusions drawn. Parents were asked to recall costs since initial diagnosis (1-6 years in the past), but this extended time period may have introduced recall bias. CONCLUSIONS: Direct non-medical and indirect costs play an important role in the total treatment costs of NB. Children with NB treated in local hospitals and followed up in hospital specialized in childhood oncology may save many unnecessary expenses. China's healthcare system should establish mechanisms and provide financial support for children with NB.


Assuntos
Efeitos Psicossociais da Doença , Gastos em Saúde/estatística & dados numéricos , Neuroblastoma/economia , Neuroblastoma/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Pré-Escolar , China , Estudos Transversais , Feminino , Alimentos/economia , Transplante de Células-Tronco Hematopoéticas/economia , Humanos , Lactente , Masculino , Modelos Econométricos , Radioterapia/economia , Fatores de Risco , Procedimentos Cirúrgicos Operatórios/economia , Meios de Transporte/economia , Viagem/economia
20.
Ann Hematol ; 99(6): 1331-1339, 2020 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-32382775

RESUMO

Autologous stem cell transplantation (ASCT) is the only curable therapy for multiple myeloma (MM), while its success primarily relies on mobilization to obtain sufficient hematopoietic stem/progenitor cells (HPC). Although the role of Pegfilgrastim (PEG), a novel PEGylated form of the recombinant G-CSF filgrastim (FIL), in mobilization has been demonstrated, it remains unclear whether this approach is cost-effective in MM treatment. Here, we performed a real-world analysis to evaluate the efficacy and cost of PEG for mobilization in a cohort of MM patients, of which 53% carried high-risk cytogenetic abnormalities. A total of 91 patients who received either a single dose of PEG (6 or 12 mg, n = 42) or multiple dosing of 10 µg/kg/day FIL (n = 49) after chemotherapy for HPC mobilization were included. The yield of MNCs and CD34+ cells per milliliter of blood collected via apheresis was significantly greater in the PEG group than that in the FIL group (P = 0.014 and P = 0.038). Mobilization with PEG yielded significantly higher median number of collected CD34+ cells than FIL (5.56 vs. 4.82 × 106/kg; P = 0.038). Moreover, the average time-to-recovery of leukocytes and platelets after transplantation was markedly shorter in the PEG group than that in the FIL group (leukocyte, 11.59 ± 1.98 vs 12.93 ± 2.83 days, P = 0.019; platelet, 12.86 ± 2.62 vs 14.80 ± 5.47, P = 0.085). However, the total cost of mobilization and apheresis using PEG or FIL was comparable (P = 0.486). Of note, mobilization with 12 mg PEG further shortened time-to-recovery of leukocytes (10.64 ± 0.51 vs. 12.04 ± 2.26 days, P = 0.05) and platelets (10.60 ± 2.89 vs. 13.33 ± 2.35 days, P = 0.031) compared with 6 mg PEG. Our results support a notion that PEG (especially 12 mg) combined with chemotherapy is a cost-effective and convenient regimen of mobilization, which might improve the outcome of ASCT in MM.


Assuntos
Filgrastim/uso terapêutico , Mobilização de Células-Tronco Hematopoéticas/métodos , Transplante de Células-Tronco Hematopoéticas/métodos , Mieloma Múltiplo/sangue , Mieloma Múltiplo/terapia , Polietilenoglicóis/uso terapêutico , Adulto , Idoso , Estudos de Coortes , Análise Custo-Benefício , Feminino , Filgrastim/economia , Mobilização de Células-Tronco Hematopoéticas/economia , Mobilização de Células-Tronco Hematopoéticas/tendências , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/tendências , Humanos , Masculino , Pessoa de Meia-Idade , Mieloma Múltiplo/economia , Polietilenoglicóis/economia , Transplante Autólogo/economia , Transplante Autólogo/métodos , Transplante Autólogo/tendências , Resultado do Tratamento
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