RESUMO
Treatment-free remission (TFR), in which patients discontinue pharmacotherapy and remain in molecular remission, is an emerging treatment goal for patients with chronic myeloid leukemia (CML). Attainment of TFR requires an increased frequency of molecular monitoring, to ensure that patients maintain a deep molecular response. The objective of this analysis was to assess the economic impact of stopping nilotinib among Japanese TFR-eligible patients. A Markov model evaluated the economic impact of TFR among the study population, TFR-eligible CML patients diagnosed since 2012. The model compared patients who had discontinued tyrosine kinase inhibitor (TKI) treatment (ie, attempted TFR) with patients that continued TKI treatment. A 3-y time horizon was modeled from a Japanese public payer perspective. Costs associated with drug treatment, hospital/physician visits, and molecular monitoring were considered. TFR-eligible patients were calculated from Japanese CML incidence rates and efficacy was derived from nilotinib trials. Japanese co-payment maximums were utilized to assess the patient perspective. An estimated 761 and 140 patients were eligible for first- and second-line nilotinib, respectively, in 2019. Assuming that 100% of eligible patients complied, TFR was associated with cost savings of ¥7 625 174 640 (US$66 567 775) over 3 y. In scenarios with reduced willingness to attempt TFR, cost savings persisted. Achievement of TFR was estimated to markedly reduce out-of-pocket expenses for CML patients, regardless of the timing of relapse. Stopping nilotinib for TFR-eligible patients in Japan may result in significant cost savings to both payers and patients. Monitoring costs contributed little to overall annual costs and decreased over time.
Assuntos
Orçamentos , Análise Custo-Benefício , Leucemia Mielogênica Crônica BCR-ABL Positiva/epidemiologia , Custos de Cuidados de Saúde , Humanos , Japão/epidemiologia , Leucemia Mielogênica Crônica BCR-ABL Positiva/diagnóstico , Leucemia Mielogênica Crônica BCR-ABL Positiva/tratamento farmacológico , Cadeias de Markov , Modelos Teóricos , Pirimidinas/administração & dosagem , Pirimidinas/efeitos adversos , Pirimidinas/uso terapêutico , Indução de Remissão , Resultado do TratamentoRESUMO
BACKGROUND: Treatment of patients with cat allergy with peptides derived from Fel d 1 (the major cat allergen) ameliorated symptoms of cat allergy in phase 2 clinical trials. OBJECTIVE: We sought to demonstrate that the tolerance induced by Fel d 1 peptide immunotherapy can be exploited to reduce allergic responses to a second allergen, ovalbumin (OVA), in mice sensitized dually to OVA and Fel d 1. METHODS: Induction of tolerance to OVA was achieved through simultaneous exposure to both allergens after peptide treatment. Functional tolerance to each allergen was assessed in a model of allergic airways disease in which treated mice were protected from eosinophilia, goblet cell hyperplasia, and TH2 cell infiltration. RESULTS: Suppression of allergic responses to cat allergen challenge was associated with significant increases in numbers of CD4+CD25+Foxp3+ T cells, IL-10+ cells, and CD19+IL-10+ B cells, whereas the response to OVA was associated with a marked reduction in numbers of TH2 cytokine-secreting T cells and less prominent changes in outcomes associated with immune regulation. CONCLUSIONS: These observations suggest that immune tolerance induced by peptide immunotherapy can be used experimentally to treat an allergic response to another allergen and that the molecular mechanisms underlying induction of tolerance to a treatment-specific allergen and a bystander allergen might be different.
Assuntos
Alérgenos/imunologia , Dessensibilização Imunológica , Glicoproteínas/imunologia , Hipersensibilidade/terapia , Tolerância Imunológica , Ovalbumina/imunologia , Peptídeos/imunologia , Animais , Linfócitos B/imunologia , Efeito Espectador , Citocinas/imunologia , Feminino , Hipersensibilidade/imunologia , Pulmão/imunologia , Camundongos Endogâmicos BALB C , Linfócitos T/imunologiaRESUMO
INTRODUCTION: Approximately half of patients with non-small cell lung cancer (NSCLC) present with early-stage disease at diagnosis. Real-world outcomes data are limited for this population but are of interest given recent and impending results from trials evaluating epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) and immunotherapies in neoadjuvant, adjuvant, and perioperative settings. METHODS: A retrospective, longitudinal, population-level study was conducted in patients diagnosed with resected stage I-III non-squamous NSCLC in Ontario, Canada, between April 2010 and March 2019. Study outcomes included patient characteristics and median overall survival (mOS), with stratification by disease stage and treatment exposure. Patients receiving EGFR-TKIs (assumed EGFR mutation-positive by proxy) were a key population of interest. RESULTS: Among 8255 cases, 4881 had stage I, 2124 had stage II, and 1250 had stage III NSCLC at diagnosis. The mean patient age was 68 years; 53.5% were female. In the overall cohort, 19.6% received adjuvant chemotherapy. Receipt of adjuvant chemotherapy was associated with significantly longer mOS than not receiving such therapy: stage II (7.6 [95% confidence interval: 6.5-8.5] vs. 4.4 [4.0-4.9] years) or stage III (4.4 [3.6-5.1] vs. 2.7 [2.3-3.3] years), both p < 0.0001. Patients receiving treatment (EGFR-TKIs and chemotherapy) were assumed to have experienced disease recurrence/relapse; mOS was longer among those receiving an EGFR-TKI than among those receiving chemotherapy (2.3 [1.8-3.0] vs. 1.1 [1.0-1.3] years). CONCLUSION: In Ontario, between 2010 and 2019, uptake of adjuvant therapy was low among patients with resected NSCLC, despite such therapy being associated with improved survival. Patients assumed to have recurred/relapsed had markedly reduced mOS, regardless of subsequent therapy, compared with those who did not relapse/recur. Novel peri-adjuvant treatment options are needed to enhance outcomes after lung resection.
RESUMO
Improved understanding of the biological heterogeneity of breast cancer (BC) has facilitated the development of more effective and personalized approaches to treatment. This study describes real-world evidence on treatment patterns and outcomes for a population-based cohort of patients with human epidermal growth factor receptor (HER2) IHC0 and -low BC with de novo or recurrent disease from Alberta, Canada. Patients 18+ years old diagnosed with HER2 IHC0/-low, de novo/recurrent BC from 2010 to 2019 were identified using Alberta's cancer registry. Analyses of these patients' existing electronic medical records and administrative claims data were conducted to examine patient characteristics, treatment patterns, and survival outcomes. A total of 3413 patients were included in the study, of which 72.10% initiated first line hormonal and non-hormonal systemic therapy. The 1-year overall survival (OS) was 81.09% [95% CI, 79.52-82.69]. Recurrent patients had a higher OS compared to de novo patients: 54.30 months [95% CI, 47.80-61.90] vs. 31.5 months [95% CI, 28.40-35.90], respectively. Median OS was 43.4 months [95% CI, 40.70-47.10] and 35.80 months [95% CI, 29.00-41.70] among patients with HER2-low and HER2 IHC0 cancer, respectively. The study results provide real-world evidence regarding the clinical outcomes of HER2 IHC0/-low and de novo/recurrent disease.
RESUMO
PURPOSE: We sought to quantify mCRPC patient treatment patterns and survival across multiple lines of therapy after prior androgen-receptor-axis-targeted therapy (ARAT) failure. METHODS: Individuals diagnosed with prostate cancer between 2010 and 2018 were identified in the Ontario Cancer Registry (OCR). An algorithm was created to identify patients with mCRPC that was aligned to Prostate Cancer Clinical Trials Working Group 3 criteria (PCWG3) and validated with Canadian clinical experts. In the mCRPC setting, treatment patterns were assessed by line of therapy, and survival was calculated from treatment initiation until death or lost to follow-up. RESULTS: 64,484 men were diagnosed withprostate cancer in Ontario between 2010 and 2018with 5,588 men assessed to have mCRPC and 2,970 (53%) of those received first-line systemic treatment. Across the first-, second- and third-line of therapy, ARATs (abiraterone and enzalutamide) were the most used therapies. Survival for mCRPC patients treated with ARATs in first-, second- and third-line were 13.0 (95% CI, 11.6 - 14.5), 11.5 (95% CI, 10.1 - 13.4) and 8.9 (95% CI, 7.4 - 10.2) months, respectively. Survival for mCRPC patients treated with taxanes in first, second- and third-line were 16.7 (95% CI, 14.8 - 18.0), 11.3 (95% CI, 10.1 - 12.5) and 7.8 (95% CI, 6.5 - 10.6) months, respectively. No statistical difference in overall survival was found between taxanes and ARATs. CONCLUSION: In this analysis of a large retrospective cohort of Canadian men with mCRPC, we found that survival in patients treated with ARATs and taxanes was fairly similar across all lines of therapy. Importantly, this trend was maintained in ARAT-exposed patients, where sequential ARAT and taxanes offered similar survival. These data may help inform optimal sequencing of therapies in mCRPC.
Assuntos
Neoplasias de Próstata Resistentes à Castração , Masculino , Humanos , Neoplasias de Próstata Resistentes à Castração/tratamento farmacológico , Estudos Retrospectivos , Resultado do Tratamento , Ontário , Taxoides/uso terapêuticoRESUMO
BACKGROUND: For many patients with resected epidermal growth factor receptor mutation-positive (EGFRm) non-small cell lung cancer (NSCLC), current standard of care (SoC) is adjuvant chemotherapy; however, disease recurrence remains high. Based on positive results from ADAURA (NCT02511106), adjuvant osimertinib was approved for treatment of resected stage IBâIIIA EGFRm NSCLC. OBJECTIVE: The aim was to assess the cost-effectiveness of adjuvant osimertinib in patients with resected EGFRm NSCLC. METHODS: A five-health-state, state-transition model with time dependency was developed to estimate lifetime (38 years) costs and survival of resected EGFRm patients treated with adjuvant osimertinib or placebo (active surveillance), with/without prior adjuvant chemotherapy, using a Canadian Public Healthcare perspective. Transitions between health states were modeled using ADAURA and FLAURA (NCT02296125) data, Canadian life tables, and real-world data (CancerLinQ Discovery®). The model used a 'cure' assumption: patients remaining disease free for 5 years after treatment completion for resectable disease were deemed 'cured.' Health state utility values and healthcare resource usage estimates were derived from Canadian real-world evidence. RESULTS: In the reference case, adjuvant osimertinib treatment led to a mean 3.20 additional quality-adjusted life-years (QALYs; (11.77 vs 8.57) per patient, versus active surveillance. The modeled median percentage of patients alive at 10 years was 62.5% versus 39.3%, respectively. Osimertinib was associated with mean added costs of Canadian dollars (C$)114,513 per patient and a cost/QALY (incremental cost-effectiveness ratio) of C$35,811 versus active surveillance. Model robustness was demonstrated by scenario analyses. CONCLUSIONS: In this cost-effectiveness assessment, adjuvant osimertinib was cost-effective compared with active surveillance for patients with completely resected stage IBâIIIA EGFRm NSCLC after SoC.
RESUMO
Background: Extensive-stage small-cell lung cancer (ES-SCLC) is an incurable cancer with poor prognosis in which characteristics predictive of long-term survival are debated. The utility of agents such as immune checkpoint inhibitors highlights the importance of identifying key characteristics and treatment strategies that contribute to long-term survival and could help guide therapeutic decisions. Objective: This real-world analysis examines the characteristics, treatment patterns, and clinical outcomes of patients receiving chemotherapy without immunotherapy for ES-SCLC in Manitoba, Canada. Methods: A retrospective cohort study assessed patient characteristics, treatment, and survival duration (short: <6 months; medium: 6-24 months; long: >24 months) using the Manitoba Cancer Registry and CancerCare Manitoba records. Eligible patients were aged >18 years with cytologically confirmed ES-SCLC diagnosed between January 1, 2004, and December 31, 2018, and received cytotoxic chemotherapy (CT). The one-, two-, and five-year probabilities of overall survival (OS) were assessed relative to patient, disease, and treatment characteristics using Kaplan-Meier methods and Cox proportional hazards models. Results: This analysis included 537 patients. Cisplatin was used in 56.1% of patients, 45.6% received thoracic radiotherapy (RT), and few received prophylactic cranial irradiation (PCI). In the overall cohort, one-, two- and five-year OS rates were 26%, 8%, and 3%, respectively. For patients with Eastern Cooperative Oncology Group Performance Status (ECOG PS) 0, OS rates at one, two, and five years were 43%, 17%, and 10%, respectively, vs. 27%, 8%, and 2% for those with ECOG PS 1-2, and 16%, 3%, and 3% for those with ECOG PS 3-4. In long-term survivors, ECOG PS scores were lower and abnormal laboratory test results were less frequent. Overall, 74.4% of long-term survivors received thoracic RT and 53.5% received PCI. Known poor prognostic factors - including brain/liver metastases, high lactate dehydrogenase (LDH), abnormal sodium, and low hemoglobin levels - were less common but still seen in long-term survivors. Conclusion: Although rare, patients with ES-SCLC may experience long-term survival with CT ± thoracic RT ± PCI. Factors predicting long-term survival include traditional prognostic factors such as ECOG PS, LDH level, and receipt of thoracic RT or PCI. These findings support current treatment algorithms for ES-SCLC and provide baseline survival estimates to assess the real-world impact of adding immune checkpoint inhibitors in the future.
RESUMO
Background: Although therapy for limited-stage small-cell lung cancer (LS-SCLC) is administered with curative intent, most patients relapse and eventually die of recurrent disease. Chemotherapy (CT) with concurrent radiotherapy (RT) remains the standard of care for LS-SCLC; however, this could evolve in the near future. Therefore, understanding the current prognostic factors associated with survival is essential. Objective: This real-world analysis examines factors associated with long-term survival in patients with LS-SCLC treated with CT in Manitoba, Canada. Methods: A retrospective cohort study was conducted using Manitoba Cancer Registry and CancerCare Manitoba records. Eligible patients were aged >18 years and had cytologically confirmed LS-SCLC diagnosed between January 1, 2004, and December 31, 2018, for which they received CT ± RT. Baseline patient, disease, and treatment characteristics and survival duration, characterized as short (<6 months), medium (6-24 months), and long term (>24 months), were extracted. Overall survival (OS) was estimated at one, two, and five years and assessed using Kaplan-Meier methods and Cox proportional hazards models. Results: Over the 15-year study period, 304 patients met the eligibility criteria. Long-term survivors comprised 39.1% of the cohort; at diagnosis, this subgroup was younger, more likely to have Eastern Cooperative Oncology Group Performance Status (ECOG PS) 0, and have normal lactate dehydrogenase, sodium, and hemoglobin levels. OS estimates for the entire cohort at one, two, and five years were 66%, 38%, and 18%, respectively. In the ECOG PS 0 subgroup, OS estimates at one, two, and five years were 85%, 52%, and 24%, respectively; OS estimates were 60%, 35%, and 17%, respectively, for ECOG PS 1-2 and were 47%, 23%, and 10%, respectively, for ECOG PS 3-4. OS was significantly higher among patients with normal serum sodium and hemoglobin levels than those with abnormal levels. Univariable hazard regression models found that ECOG PS, age at diagnosis, receipt of prophylactic cranial irradiation (PCI), and thoracic RT were associated with survival. On multivariable hazard regression, ECOG PS and receipt of PCI were associated with survival. Conclusion: Survival for greater than two years in patients with LS-SCLC treated with CT ± RT was associated with ECOG PS and receipt of PCI.
RESUMO
OBJECTIVES: The ADAURA trial demonstrated the benefit of adjuvant osimertinib among patients with resected, early-stage, epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC). To understand the potential population impact, it is critical to deduce the prevalence, management, and outcomes of this patient population in the real-world setting before use of adjuvant osimertinib. MATERIALS AND METHODS: Using PALEOS (Pan-cAnadian Lung cancEr Observational Study) data (2012-2019), a retrospective, multi-center, observational cohort study was conducted among patients with early-stage (IB-IIIA) resected NSCLC who had not received neoadjuvant therapy. Study outcomes included EGFRm prevalence, treatment patterns, recurrence outcomes, and overall and disease-free survival (OS/DFS). RESULTS: Among patients undergoing reflexive EGFRm testing by a pathologist at time of diagnosis irrespective of disease stage (N = 535), 23 % were EGFRm-positive; 15.9 % had common mutations and 5.6 % had uncommon mutations. Within the EGFRm-positive cohort (N = 156), mean age at diagnosis was 68 years, 65 % of patients were female, and 35 % were of Asian descent. At diagnosis, 48 %, 31 %, and 21 % had stage IB, II, or IIIA disease, respectively; 46 % received adjuvant therapy after resection. Half of patients experienced disease recurrence, typically involving distant sites; central nervous system metastasis varied from 12 % to 15.0 % across disease stages. EGFR tyrosine kinase inhibitors were the most commonly received therapy after first metastatic recurrence. Median OS (DFS) was not reached, 71.2 (22.8) months, and 50.1 (18.0) months among stage IB, II, and IIIA patients. Patients with uncommon EGFRm had a lower probability of survival than those with common EGFRm (2 years: 87 % vs 91 %-94 %; 4 years: 56 % vs 73 %-82 %). CONCLUSION: Approximately-one-quarter of patients with resected, early-stage NSCLC were EGFRm-positive in this study. These patients had high recurrence rates and suboptimal long-term survival after treatment with current therapies. New adjuvant treatments are warranted.
Assuntos
Carcinoma Pulmonar de Células não Pequenas , Neoplasias Pulmonares , Carcinoma de Pequenas Células do Pulmão , Humanos , Feminino , Masculino , Carcinoma Pulmonar de Células não Pequenas/epidemiologia , Carcinoma Pulmonar de Células não Pequenas/genética , Carcinoma Pulmonar de Células não Pequenas/cirurgia , Neoplasias Pulmonares/epidemiologia , Neoplasias Pulmonares/genética , Neoplasias Pulmonares/cirurgia , Estudos Retrospectivos , Prevalência , Estadiamento de Neoplasias , Quimioterapia Adjuvante , Recidiva Local de Neoplasia/genética , Recidiva Local de Neoplasia/patologia , Canadá/epidemiologia , Receptores ErbB/genética , Mutação , Inibidores de Proteínas Quinases/efeitos adversos , Carcinoma de Pequenas Células do Pulmão/patologiaRESUMO
The prognosis for extensive-stage small cell lung cancer (ES-SCLC) is poor. Real-world evidence can highlight the unmet clinical need within this population. We conducted a population-based cohort study of ES-SCLC patients diagnosed in a large Canadian province (2010-2018) using electronic medical records and administrative claims data. In all, 1941 ES-SCLC patients were included, of which 476 (25%) were recurrent cases. Median age at diagnosis was 70 years (range: 39-94) and 50.2% were men. Of the 1941 ES-SCLC patients, 29.5% received chemotherapy and radiotherapy, 17.0% chemotherapy alone, 8.7% radiotherapy alone, and 44.8% received best supportive care. Chemotherapy was initiated by 46.5%, 8.5%, and 1.4% of first-, second-, and third-line patients, with lower uptake for recurrent cases. Median survival from first-, second-, and third-line chemotherapy was 7.82 months (95% CI: 7.50-8.22), 5.72 months (95% CI: 4.90-6.87), and 3.83 months (95% CI: 2.99-4.60). Among patients who received first-line therapy, the 2-year and 5-year survival was 7.3% (95% CI: 5.7-9.2) and 2.9% (95% CI: 1.8-4.5). In conclusion, initiation of first-line treatment in ES-SCLC was low with significant attrition in subsequent lines. These results underscore the need for effective front-line treatments and highlight the potential for novel therapies to improve patient outcomes.
Assuntos
Neoplasias Pulmonares , Carcinoma de Pequenas Células do Pulmão , Canadá/epidemiologia , Estudos de Coortes , Atenção à Saúde , Humanos , Neoplasias Pulmonares/epidemiologia , Neoplasias Pulmonares/terapia , Masculino , Carcinoma de Pequenas Células do Pulmão/tratamento farmacológico , Carcinoma de Pequenas Células do Pulmão/epidemiologiaRESUMO
OBJECTIVES: Treatment of advanced NSCLC (aNSCLC) is rapidly evolving, as new targeted and immuno-oncology (I-O) treatments become available. The iTEN model was developed to predict the cost and survival benefits of changing aNSCLC treatment patterns from a Canadian healthcare system perspective. This report describes iTEN model development and validation. MATERIALS & METHODS: A discrete event patient simulation of aNSCLC was developed. A modified Delphi process using Canadian clinical experts informed the development of treatment sequences that included commonly used, Health Canada approved treatments of aNSCLC. Treatment efficacy and the timing of progression and death were estimated from published Kaplan-Meier progression free and overall survival data. Costs (2018 CDN$) included were: drug acquisition and administration, imaging, monitoring, adverse events, physician visits, best supportive care, and end-of-life. RESULTS AND CONCLUSION: Clinical validity of the iTEN model was assessed by comparing model survival predictions to published real-world evidence (RWE). Four RWE studies that reported the overall survival of patients treated with a broad sampling of common aNSCLC treatment patterns were used for validation. The validation coefficient of determination was R2â¯=â¯0.95, with the model generally producing estimates that were neither optimistic nor conservative. The model estimated that current Canadian practice patterns yield a median survival of almost 13 months, a five-year survival rate of 3% and a life-time per-treated-patient cost of $110,806. Cost and survival estimates are presented and were found to vary by aNSCLC subtype. In conclusion, the iTEN model is a reliable tool for forecasting the impact on cost and survival of new treatments for aNSCLC.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/economia , Biomarcadores Tumorais/metabolismo , Carcinoma Pulmonar de Células não Pequenas/economia , Análise Custo-Benefício , Neoplasias Pulmonares/economia , Modelos Estatísticos , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Carcinoma Pulmonar de Células não Pequenas/metabolismo , Carcinoma Pulmonar de Células não Pequenas/mortalidade , Seguimentos , Custos de Cuidados de Saúde , Humanos , Neoplasias Pulmonares/tratamento farmacológico , Neoplasias Pulmonares/metabolismo , Neoplasias Pulmonares/mortalidade , Prognóstico , Estudos Retrospectivos , Taxa de SobrevidaRESUMO
DISCLOSURES: No additional funding was received for the writing of this letter. The published study referred to in this letter was funded by Janssen Scientific Affairs, which employs Maiese and funded Cornerstone Research Group, a health economic consulting group, to conduct the study. Grima is a founding partner of Cornerstone Research Group, which employs Hollmann, Goyert, and Moldaver.
Assuntos
Mieloma Múltiplo/tratamento farmacológico , Mieloma Múltiplo/economia , Humanos , Intervalo Livre de Progressão , Qualidade de Vida , Estados Unidos , United States Food and Drug AdministrationRESUMO
BACKGROUND: In recent years, the FDA has approved several 3-agent (i.e., triplet) combinations for previously treated multiple myeloma (MM), and the National Comprehensive Cancer Network (NCCN) now recommends triplet regimens over doublets. Little is known about the real-world cost of triplet combinations because of the limited time that they have been on the market since FDA approval. Furthermore, traditional cost analyses developed to support market entrance rely on utilization assumptions that are difficult to validate when numerous comparators simultaneously enter the market. OBJECTIVE: To perform a 1-year cost analysis of novel triplets used for the treatment of patients with previously treated MM controlling for differences in utilization. METHODS: FDA-approved, NCCN-recommended (preferred and category 1 for previously treated MM) treatments included in the analysis were daratumumab plus lenalidomide plus dexamethasone (DARA/LEN/DEX), daratumumab plus bortezomib plus dexamethasone (DARA/BOR/DEX), elotuzumab plus lenalidomide plus dexamethasone (ELO/LEN/DEX), carfilzomib plus lenalidomide plus dexamethasone (CAR/LEN/DEX), and ixazomib plus lenalidomide plus dexamethasone (IXA/LEN/DEX). To control for market uptake, the model was designed to estimate the cost of treating an average patient over a 1-year time horizon. Drug administration and dosing, required comedications, postprogression therapy, monitoring requirements, and adverse event (AE) rates were based on FDA prescribing information or clinical trials. AEs ≥ grade 3 that occurred in ≥ 5% of patients were included. RED BOOK wholesale acquisition costs were used for drug acquisition costs. Costs of drug administration, AE management, and patient monitoring were based on the 2018 Center for Medicare & Medicaid Services payment rates or from published literature (inflated to 2018 U.S. dollars). The treatment duration for each regimen was estimated from modeled progression-free survival data; the 12-month progression-free survival rate was assumed to be equivalent to the probability that an average patient remained on therapy for at least 1 year after treatment initiation, which was used to estimate time-depended treatment-related costs. The probability of progression within 1 year of treatment initiation was used to inform the average postprogression therapy costs for each regimen. RESULTS: The estimated cost per patient for each triplet regimen was $13,890 (DARA/BOR/DEX), $22,231 (IXA/LEN/DEX), $24,322 (ELO/LEN/DEX), $26,410 (DARA/LEN/DEX), and $27,432 (CAR/LEN/DEX). Drug acquisition costs and treatment duration were the largest drivers of cost. Scenario analyses with plausible alternative input parameters found the maximum per month cost of therapy to be $30,657 (CAR/LEN/DEX) and the minimum per month cost of therapy to be $13,784 (DARA/BOR/DEX). CONCLUSIONS: This analysis controlled for differential utilization rates for 5 FDA-approved, NCCN-recommended triplet therapies for the treatment of previously treated MM. Of the examined regimens, treatment with DARA/BOR/DEX was estimated to have the lowest average monthly cost per patient, while CAR/LEN/DEX was the most expensive. As is common with modeling, some assumptions were necessary, and results may not be generalizable. DISCLOSURES: This study was funded by Janssen Scientific Affairs, which employs Maiese and funded Cornerstone Research Group, a health economic consulting group, to complete the cost analysis, interpret data, and develop the manuscript. Janssen was involved in the design of the analysis, interpretation of results, and manuscript development and approval. Grima is a founding partner of Cornerstone Research Group, which employs Hollmann, Goyert, and Moldaver. Hollmann, Goyert, and Moldaver were responsible for creation of the economic model. This work was peer-reviewed and presented as an abstract at the Lymphoma and Myeloma 2017 International Congress; October 26-28, 2017; New York, NY.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Modelos Econômicos , Mieloma Múltiplo/tratamento farmacológico , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Progressão da Doença , Intervalo Livre de Doença , Custos de Medicamentos , Humanos , Mieloma Múltiplo/economia , Estados UnidosRESUMO
BACKGROUND: Canadian and international guidelines recommend specialized, multidisciplinary teams for the treatment of patients with idiopathic pulmonary fibrosis (IPF). The objective of this cross-sectional clinical study was to investigate the effect of a care coordinator on IPF patient satisfaction and quality of life. METHODS: Forty IPF patients were enrolled from the practices of two physicians (n=20/physician), with either low (LCU) or high-coordinator use (HCU). Patient satisfaction was measured with modified FAMCARE and IPF Care UK Patient Support Program (UK-CARE) surveys. Health related quality of life (HRQoL) was assessed with the living with IPF impacts (L-IPFi) survey. An economic model assessed the impact of the coordinator; staff surveys informed patient management requirements, and costs were derived from published literature. RESULTS: Patient satisfaction was similar between the clinics; a trend (P=0.1) towards increased satisfaction among HCU patients was observed. Patients in the HCU clinic reported increased satisfaction (P<0.05) with their current care compared with care prior to joining the tertiary-care clinic, while LCU patients did not. IPF patient HRQoL did not differ between clinics. The coordinator was estimated to alleviate approximately 30% of a physician's IPF-related work load, and to facilitate the care of more patients per physician. Modelled estimates suggest the coordinator lead to annual cost-savings of $137,212. CONCLUSIONS: Reliance upon a coordinator during routine management of IPF patients may improve patient satisfaction, spare physician time and lead to annual cost-savings. Future studies should examine the impact of a coordinator on healthcare resource utilization.
RESUMO
Inflammation is a hallmark of many airway diseases. Improved understanding of the cellular and molecular mechanisms of airway disease will facilitate the transition in our understanding from phenotypes to endotypes, thereby improving our ability to target treatments based on pathophysiologic characteristics. For example, allergic asthma has long been considered to be driven by an allergen-specific T helper 2 response. However, clinical and mechanistic studies have begun to shed light on the role of other cell subsets in the pathogenesis and regulation of lung inflammation. In this review, we discuss the importance of different lymphocyte subsets to asthma and other airway diseases, while highlighting the growing evidence that asthma is a syndrome that incorporates many immune phenotypes.