Cost Of Stem Cell Transplantation Research Articles

What Does the Economic Burden of Acute Myeloid Leukemia Treatment Look Like for the Next Decade? An Analysis of Key Findings, Challenges and Recommendations.

Acute myeloid leukemia (AML) is conventionally treated with chemotherapy in eligible patients. Potentially curative regimens are associated with significant toxicity, and the major cost drivers in AML historically have been hospitalization and hematopoietic stem cell transplantation. The past several years have seen a dramatic increase in the number of treatment options, including oral therapies and drugs targeted to biological pathways implicated in AML. Major current and future drivers of cost in AML include hospitalization and medical costs, stem cell transplantation for eligible patients, and medication costs. It is likely that hospitalization and medical costs will decline as more AML treatment moves to the outpatient setting. Stem cell transplantation costs may increase, if more patients are eligible for improved procedures, although the overall cost of transplantation could decrease if new procedures reduce the need for hospitalization. Medication costs are likely to increase, with various branded drugs available and in development. From a broader perspective, another driver of cost is the proportion of patients with AML who can undergo treatment. Patients who may previously have been unable to tolerate chemotherapy are more likely to be treated with the range of less intensive, more tolerable options now available. The effectiveness of newer AML treatment options also suggests that, overall, there may be more patients staying alive and on treatment longer than in the past. While certain advances, such as increased use of oral and outpatient therapies, could potentially reduce costs, the overall economic impact of AML is likely to increase as more patients are eligible for novel therapies across several phases from induction to maintenance to relapsed/refractory disease. While these novel therapies have the potential to deliver value in the form of improved efficacy, safety, and convenience, payers will need to determine how to cover a longer, more complex AML treatment pathway.

PCN49 COST-EFFECTIVENESS MODEL COMPARING EXPANDED UMBILICAL CORD BLOOD TRANSPLANTS WITH OTHER HSCT STRATEGIES FOR PATIENTS WITH HAEMATOLOGICAL MALIGNANCIES

A cost-effectiveness model was developed to compare UM171-expanded UCB (E-UCB) to unexpanded UCB (UCB) and unrelated donor transplant (URD) from a Canadian payer perspective. A lifetime-horizon Markov model was developed to evaluate various stem cell sources in patients with haematological malignancies requiring stem cell transplantation (SCT). Health states included: Diagnosis, Pre-progression (SCT recovery), Post-SCT (without GVHD), Post-SCT (with GVHD), and Graft failure/Relapse. UCB and URD estimation of patients transitioning to each health state was derived from published literature (pooled Kaplan-Meier data) and extrapolated using exponential functional forms. E-UCB efficacy data were based on the Kaplan-Meier estimator function derived from the patient level data. Model utilities varied by health state and were based on SCT-specific literature and included complication disutility. Resource utilization and cost calculations associated with each SCT source included costs of SCT, routine medical care, complications (acute and chronic GVHD, infection, and veno-occlusive disease), indirect and mortality (end of life) costs. The primary economic endpoints assessed were life years (LYs) and quality-adjusted life years (QALYs). When published data were applied in the economic model, E-UCB showed economic dominance over both UCB and URD, with 1.50 and 1.46 LYs gained, respectively, and 1.23 and 1.19 QALYs gained, respectively. The total cost of treating one patient with E-UCB was estimated at $232,842, compared with $291,501 for UCB and $237,355 for URD over a lifetime horizon. While treatment cost was the highest for E-UCB, these costs were offset by lower relapse and GVHD costs in the E-UCB arm resulting in lower overall costs. Deterministic and probabilistic sensitivity analyses generally showed consistency with base case findings. E-UCB appeared to be cost-saving and associated with a QALYs gain when compared to UCB and URD. Sensitivity analyses supported the findings and contributed to the robustness of the economic case.

Overall Management, Outcomes and Costs on a 4-Year Period of 21,757 Acute Leukemia (AL) Patients in France. Data Report from the National Health Registry

The aim of this study is to evaluate the management outcomes and hospital costs of acute leukemia (AL) patients over a 4-year period in France using the national Program for a Medicalised Information System (PMSI) database, which records all hospitalisations in the country.We identified patients with AL or another underlying hematologic malignancy prone to transform into AL (e.g. myelodysplastic syndromes) at diagnosis and hospitalized between 2006 and 2012 and retained only incident patients. We used a retrospective cohort approach, with a 5-year follow-up time from index date (i.e. 2008 to 2012), with an additional focus on the autologous and allogeneic stem cell transplanted (SCT) patient populations during the year 2012. Hospital costs calculations were based on the national scale of costs.A total of 21,757 patients were included with 5,655 acute lymphoblastic leukemias (ALL) (26%) and 16,102 acute myelogenous leukemias (AML) at an incidence rate of 4,300 newly diagnosed AL/year. 54.7% were males (57.6% for ALL and 53.7 for AML) with a median age of 18 years (range 0-100, peak between 0-10 years) for ALL and 71 years (range 0-104, peak between 71-80 years) for AML. In 96.7% of the cases patients were hospitalized in a public healthcare hospital. The median duration of the first hospitalization was 7 days (quartiles: 1-29), with an overall death rate during this first hospitalization of 10.5% (4.4% for ALL and 12.6% for AML). Fifty-nine percent of the patients received chemotherapy, (77% for ALL, and 52% for AML); 3,079 (14%) of patients received an autologous or allogeneic stem cell transplantation (SCT) [883 in ALL patients, 2,196 in AML patients]. Among SCT patients, 2,732 (89%) had an allogeneic SCT [774 in ALL patients and 1,958 in AML patients]. Over the 2,732 allogeneic SCT group, 1,243 (45%) suffered from an acute GVHD (55% of ALL patients and 42% of AML patients), and 21% from chronic GVHD (25% of ALL patients and 20% of AML patients).Post SCT transplant survival rates for AML and ALL were 83% and 86% at one year and 55% and 58% at 4 years. Without SCT transplant, survival rates were 40% and 77% at one year and 24% and 70% at 4 years for AML and ALL respectively. The overall survival rates were significantly improved with transplant for AML patients but not for ALL patients at 4 years (see figure 1). [Display omitted] Cost analyses showed an overall median hospitalization cost the first year after diagnosis of 44,453 € for AL (72,360 € for ALL, and 40,477 € for AML), the second year after diagnosis the median overall hospitalization cost for AL was reduced to 2,185 € (6,212 € for ALL and 904 € for AML). The median hospitalization costs of the stay for SCT were 42 068€ for ALL and also 42 068€ for ALM patients, while the cost of allogenic vs. autologous patients was nearly three times higher.In conclusion, this study in an extremely large national cohort illustrates the current characteristics of the care of acute leukemia patients in our country and provides significant information on the economic impact of the hospital care as well as the additional cost of stem cell transplant. DisclosuresNicolini:Novartis: Consultancy. Laurendeau:CEMKA: Employment. Bureau:CEMKA: Employment. Ricci:Wellmera: Employment. Gerbeau:Novartis: Employment. Bouee:CEMKA: Employment.

Dasatinib, nilotinib and standard-dose imatinib for the first-line treatment of chronic myeloid leukaemia: systematic reviews and economic analyses.

Nilotinib and dasatinib are now being considered as alternative treatments to imatinib as a first-line treatment of chronic myeloid leukaemia (CML). This technology assessment reviews the available evidence for the clinical effectiveness and cost-effectiveness of dasatinib, nilotinib and standard-dose imatinib for the first-line treatment of Philadelphia chromosome-positive CML. Databases [including MEDLINE (Ovid), EMBASE, Current Controlled Trials, ClinicalTrials.gov, the US Food and Drug Administration website and the European Medicines Agency website] were searched from search end date of the last technology appraisal report on this topic in October 2002 to September 2011. A systematic review of clinical effectiveness and cost-effectiveness studies; a review of surrogate relationships with survival; a review and critique of manufacturer submissions; and a model-based economic analysis. Two clinical trials (dasatinib vs imatinib and nilotinib vs imatinib) were included in the effectiveness review. Survival was not significantly different for dasatinib or nilotinib compared with imatinib with the 24-month follow-up data available. The rates of complete cytogenetic response (CCyR) and major molecular response (MMR) were higher for patients receiving dasatinib than for those with imatinib for 12 months' follow-up (CCyR 83% vs 72%, p < 0.001; MMR 46% vs 28%, p < 0.0001). The rates of CCyR and MMR were higher for patients receiving nilotinib than for those receiving imatinib for 12 months' follow-up (CCyR 80% vs 65%, p < 0.001; MMR 44% vs 22%, p < 0.0001). An indirect comparison analysis showed no difference between dasatinib and nilotinib for CCyR or MMR rates for 12 months' follow-up (CCyR, odds ratio 1.09, 95% CI 0.61 to 1.92; MMR, odds ratio 1.28, 95% CI 0.77 to 2.16). There is observational association evidence from imatinib studies supporting the use of CCyR and MMR at 12 months as surrogates for overall all-cause survival and progression-free survival in patients with CML in chronic phase. In the cost-effectiveness modelling scenario, analyses were provided to reflect the extensive structural uncertainty and different approaches to estimating OS. First-line dasatinib is predicted to provide very poor value for money compared with first-line imatinib, with deterministic incremental cost-effectiveness ratios (ICERs) of between £256,000 and £450,000 per quality-adjusted life-year (QALY). Conversely, first-line nilotinib provided favourable ICERs at the willingness-to-pay threshold of £20,000-30,000 per QALY. Immaturity of empirical trial data relative to life expectancy, forcing either reliance on surrogate relationships or cumulative survival/treatment duration assumptions. From the two trials available, dasatinib and nilotinib have a statistically significant advantage compared with imatinib as measured by MMR or CCyR. Taking into account the treatment pathways for patients with CML, i.e. assuming the use of second-line nilotinib, first-line nilotinib appears to be more cost-effective than first-line imatinib. Dasatinib was not cost-effective if decision thresholds of £20,000 per QALY or £30,000 per QALY were used, compared with imatinib and nilotinib. Uncertainty in the cost-effectiveness analysis would be substantially reduced with better and more UK-specific data on the incidence and cost of stem cell transplantation in patients with chronic CML. The Health Technology Assessment Programme of the National Institute for Health Research.

Costs and Cost-Effectiveness of Allogeneic Stem Cell Transplantation in Children Are Predictable

The overall costs of pediatric stem cell transplantation (SCT), including donor search and costs during the first year post-SCT, were calculated in a cohort of 141 consecutive children undergoing SCT in a single institution. Costs were correlated with patient and transplantation characteristics and with a risk score for transplantation-related mortality. Cost-effectiveness was calculated based on the overall cost per surviving patient. Life-years gained were extrapolated from overall survival, and the costs per expected life-year gained were calculated. The overall median cost was €136,382 (175,815$), with a wide range, of €26,897 (34,679$) to €601,348 (775,343$). Increased costs were significantly associated with age, use of donors other than matched siblings, and advanced disease. There was a strong correlation of costs with a simple transplantation-related mortality risk score; median total costs were €89,550 (115,463$) for a score of 0, €127,349 (164,179$) for a score of 1, €156,578 (201,861$) for a score of 2, and €274,915 (354,499$) for a score of 3 (P < .001). Cost-effectiveness decreased with increasing transplantation-related mortality risk score; costs per survivor increased from €93,209 (120,200$) for a score of 0 to a maximum of €1,216,348 (1,568,579$) for a score of 3. Costs associated with pediatric SCT vary substantially; however, the combination of variables such as age, disease, and donor type is predictive of costs and cost-effectiveness. Costs per life-year gained are within the broadly accepted range in life-threatening hemato-oncologic diseases, even in the most cost-intensive patient cohort.

The importance of lowering the costs of stem cell transplantation in developing countries.

The effectiveness and efficiency of health care systems can be assessed by economic evaluation, comparing the costs and outcomes of alternative interventions. Direct costs include accommodation, nursing, physicians' fees, diagnostic tests and treatment. Indirect costs derive from the loss of a persons ability to use life in a productive way e.g. employment. In cost-effectiveness analyses, cost is the numerator and effectiveness (related to health outcomes) is the denominator. The unit of measurement of effectiveness is usually years of life saved. Cost utility analyses require a preference-based measurement of health-related quality of life (HRQL) to allow the calculation of utility scores for health states and so the adjustment of effectiveness for quality. Typically, this allows the calculation of quality-adjusted life years (QALYs). A review of published reports, to be presented in detail, yields the following summation. The greater cost of stem cell harvest from peripheral blood is more than offset by the reduced costs associated with a shorter hospital stay. Transplants early in first remission cost less than those undertaken at later points in disease evolution/treatment experience. Changing the primary locus of care from inpatient to outpatient may result in notable cost savings but can produce cost-shifting (from inpatient to outpatient). Nevertheless, in selected patient, the use of non-myeloablative transplants may offer a cost-effective option, especially in the developing country context.

Economic analyses of bone marrow and blood stem cell transplantation for leukemias and lymphoma: what do we know?

The use of blood and/or bone marrow stem cell transplantation (SCT) grew extensively in the last decade as technological advances led to improved outcomes and wider availability. The first study of SCT costs, however, was not published until 1989. This paper summarizes current knowledge about costs and cost-effectiveness of allogeneic and autologous SCT for leukemias and lymphoma. Methodological issues in cost studies such as types of costs, methods of data collection, and time horizons are discussed, and studies are evaluated with regard to these issues. Considerations specific to economic analyses of SCT are considered, including the potential impact of technological changes, learning curve effects, and inter-institutional differences.