Relationship between paid leave, financial burden, and patient-reported outcomes among employed patients who have undergone bone marrow transplantation.

PURPOSE: The US does not have universal paid family and medical leave. We examine the direct effects of access to paid leave on patient-reported health, quality of life (QOL), and perceived stress of employed patients who underwent bone marrow transplantation (BMT) to treat advanced blood cancer as well as the indirect effects through reductions in the financial burden (FB) that patients face. METHODS: Our cross-sectional observational study took place at three US transplantation centers in 2014 and 2015. All English-speaking cancer patients 6-month post-BMT were mailed a 43-item survey assessing financial situation, employer benefits, and patient-reported health outcomes. The sample includes the 171 respondents who were employed at the time of BMT. RESULTS: Seemingly unrelated regression analysis confirms that patient access to paid leave was associated with reductions in all three measures of FB, and lower levels of financial hardship were related with improved health, QOL, and perceived stress outcomes. For self-reported health and perceived stress outcomes, all of the effects of patient paid leave operate indirectly through reductions in FB. For QOL outcomes, there is both a direct effect (over 80%) of paid leave and an indirect effect through reduction of FB. CONCLUSION: We found that paid leave affected health outcomes for BMT patients mostly through alleviating FB. These findings suggest universal paid leave policies in the US might alleviate financial hardship and have positive effects on the self-reported QOL of employed patients facing intensive medical treatments.

Patient Perspectives Regarding Allogeneic Bone Marrow Transplantation in Myelofibrosis.

Hematopoietic stem cell transplantation (HCT) is a curative treatment for patients with myelofibrosis (MF); however, many HCT-eligible patients decline this potentially life-saving procedure. The reasons behind this decision are not clear. We sought to survey patients with MF to understand their perspective on HCT. A 63-question survey was posted on myeloproliferative neoplasm patient advocacy websites. A total of 129 patients with MF responded to the survey. Among these patients, 49 (41%) were referred for HCT, and 41(32%) attended the transplantation consult. Of the patients who attended the transplantation consult, 24 (59%) did not plan on going on to HCT, and 16 (41%) intended to proceed with HCT. Reasons for the decision to not undergo transplantation included the desire to not be ill, desire to not spend time in the hospital, and concerns about overall quality of life. Specifically, concerns related to financial impact and the risk of graft-versus-host disease (GVHD) were expressed. Patients who decided to proceed with HCT felt that this would extend their survival and allow them to be around family for longer. This is the first survey to investigate patient perceptions regarding HCT for MF. Less than one-half of the patients were referred for HCT, and of those, less than one-half planned on proceeding with the transplantation, suggesting that many patients do not receive this life-saving procedure. Further exploration of the basis of patients' reluctance to proceed with HCT is warranted.

The Japan Marrow Donor Program, 25 years of experience in achieving 20,000 bone marrow transplantations: organization structure, activity, and financial basis.

The Japan Marrow Donor Program (JMDP), established in 1991, has continued to grow in its capacity to facilitate unrelated bone marrow (BMT) and peripheral blood stem cell transplantation (PBSCT) for the past 25 years in Japan. The current donor pool is 463,465 (as of 31 December 2016) and 20,237 transplants were performed with the help of the Japanese Red Cross, government, and supporters. As JMDP introduced PBSCT in 2010, the vast majority of transplants are BMT. All donors are fully typed for HLA-A, B, C, and DR. The peak age of registered donors is around 40 years. The 8/8 HLA-matched donors are found in our registry for 96% of the patients and 54% of the patients receive a transplant. The median time between the initiation of donor search and the transplantation is approximately 122 days. The median interval between the initiation of donor search and identification of the first potential donor is 40 days. The most common diseases for which unrelated BMT/PBSCT is indicated are acute myelogenous leukemia (AML), acute lymphocytic leukemia (ALL), myelodysplastic syndrome (MDS), and malignant lymphoma. In recent years we have seen a marked increase in elderly patients who received BMT.

Cost Analysis of Transplantation in Japan, Performed With the Use of the National Database.

BACKGROUND: When assessing the cost of transplants in Japan, earlier studies have been limited to case series that investigated inpatient cost alone. Few studies have evaluated total cost, which includes inpatient, outpatient, and pharmaceutical costs, or compared costs before and after transplantation. Using the National Database of Health Insurance Claims and Specific Health Checkups of Japan (NDB), we investigated the total cost of major transplantation and contributing factors. METHODS: We analyzed the cost and complications of patients who underwent a cadaveric renal transplantation (CRT), living renal transplantation (LRT), living-donor liver transplantation (LDLT), allogeneic bone marrow transplantation, autologous bone marrow transplantation, allogeneic peripheral blood stem cell transplantation, or autologous peripheral blood stem cell transplantation (auto-PBSCT) from April 2009 to March 2010. RESULTS: The highest total cost of the month of transplantation was 4.95 million yen (JPY) for LDLT. Among renal transplantations, the cost of CRT was higher than LRT (3.69 vs 3.55 million JPY). Recipients of auto-PBSCT complicated by graft-versus-host disease, urinary tract infection, sepsis, or pneumonia had a significantly higher average total cost during the month of transplantation and the 2 following months than patients without it, as well as statistically longer total treatment days. CONCLUSIONS: In Japan, almost all medical services are covered by national health insurance, and the Japan government has begun to allow the use of the NDB for research activities. This is the first study to use the NDB to analyze the cost of transplantation, with technical and institutional limitations.

Healthcare costs and outcomes of managing ß-thalassemia major over 50 years in the United Kingdom.

BACKGROUND: The objective was to estimate the incidence-based costs of treating ß-thalassemia major (BTM) to the United Kingdom's National Health Service (NHS) over the first 50 years of a patient's life in terms of healthcare resource use and corresponding costs and the associated health outcomes. STUDY DESIGN AND METHODS: This was a modeling study based on information obtained from a systematic review of published literature and clinicians involved in managing BTM in the United Kingdom. A state transition model was constructed depicting the management of BTM over a period of 50 years. The model was used to estimate the incidence-based health economic impact that BTM imposes on the NHS and patients' health status in terms of the number of quality-adjusted life-years (QALYs) over 50 years. RESULTS: The expected probability of survival at 50 years is 0.63. Of patients who survive, 33% are expected to be without any complication and the other 67% are expected to experience at least one complication. Patients' health status over this period was estimated to be a mean of 11.5 discounted QALYs per patient. Total healthcare expenditure attributable to managing BTM was estimated to be £483,454 ($720,201) at 2013/14 prices over 50 years. The cost of managing BTM could be potentially reduced by up to 37% if one in two patients had a bone marrow transplant, with an ensuing improvement in health-related quality of life. CONCLUSION: This analysis provides the best estimate available of NHS resource use and costs with which to inform policy and budgetary decisions pertaining to this rare disease.

ReCAP: Pharmacists' Impact in Hematopoietic Stem-Cell Transplantation: Economic and Humanistic Outcomes.

PURPOSE: This study seeks to evaluate the impact of pharmacists' involvement in the care of patients undergoing bone marrow transplantation (BMT). METHODS: This was a three-phase study. In phase 1, inpatient and outpatient pharmacist encounters were totaled and services provided were translated to revenue generated from prescription revenue and billing charges. In phase 2, pharmacists' activities and interventions were associated with time savings estimated by providers. In phase 3, patients and providers were surveyed to assess their expectations, experiences, and value perceptions of pharmacists.A positive response rate of 80%for each survey item was set as the threshold for high expectations and successful service delivery. RESULTS: In phase 1, after 6 months of data collection, clinical services were provided to 170 inpatients and 290 outpatients. For inpatients, there was an average discharge prescription revenue of $990 per patient through the outpatient pharmacy. In the outpatient clinic, pharmacist visits generated an additional $23,000 in charges (approximately $80 per patient) and an annual prescription revenue of approximately $840,000 through the outpatient pharmacy. In phase 2, pharmacists' activities led to a total time savings of 122 hours. In phase 3, patients and providers met the predetermined 80% positive response rate for most survey items. The item for which patient and provider responses consistently did not meet this threshold related to pharmacists educating patients about their BMT. CONCLUSION: Pharmacists are valuable resources in the care of patients undergoing BMT, as their care translates to increased revenue, provider time savings, and positive perceptions from patients and providers.

The effect of concentrated bone marrow aspirate in operative treatment of fifth metatarsal stress fractures; a double-blind randomized controlled trial.

BACKGROUND: Fifth metatarsal (MT-V) stress fractures often exhibit delayed union and are high-risk fractures for non-union. Surgical treatment, currently considered as the gold standard, does not give optimal results, with a mean time to fracture union of 12-18 weeks. In recent studies, the use of bone marrow cells has been introduced to accelerate healing of fractures with union problems. The aim of this randomized trial is to determine if operative treatment of MT-V stress fractures with use of concentrated blood and bone marrow aspirate (cB + cBMA) is more effective than surgery alone. We hypothesize that using cB + cBMA in the operative treatment of MT-V stress fractures will lead to an earlier fracture union. METHODS/DESIGN: A prospective, double-blind, randomized controlled trial (RCT) will be conducted in an academic medical center in the Netherlands. Ethics approval is received. 50 patients will be randomized to either operative treatment with cB + cBMA, harvested from the iliac crest, or operative treatment without cB + cBMA but with a sham-treatment of the iliac crest. The fracture fixation is the same in both groups, as is the post-operative care.. Follow up will be one year. The primary outcome measure is time to union in weeks on X-ray. Secondary outcome measures are time to resumption of work and sports, functional outcomes (SF-36, FAOS, FAAM), complication rate, composition of osteoprogenitors in cB + cBMA and cost-effectiveness. Furthermore, a bone biopsy is taken from every stress fracture and analysed histologically to determine the stage of the stress fracture. The difference in primary endpoint between the two groups is analysed using student's t-test or equivalent. DISCUSSION: This trial will likely provide level-I evidence on the effectiveness of cB + cBMA in the operative treatment of MT-V stress fractures. TRIAL REGISTRATION: Netherlands Trial Register (reg.nr NTR4377 ).

Análise de custos do transplante alogênico de células-tronco hematopoiéticas: uma revisão integrativa / Análisis de costo del trasplante alogénico de células madre hematopoyéticas: una revisión integradora / Cost of hematopoietic stem cell’s allogeneic transplantation: an integrative review

Objective: Identifying what has been produced on cost analysis of allogeneic transplantation of hematopoietic stem cell. Method: It consists of an integrative review, where was done a search of studies on cost analysis in allogeneic transplantation. Results: There were found 265 articles, which, after application of inclusion and exclusion criteria, 13 articles, with twelve in English and two in Portuguese were selected. Eleven of these articles have made partial cost analysis, a study done systematic review of cost-effectiveness; one made economic evaluation of cost-effectiveness and cost evaluation study made about coverage for curative catheter in transplantation. Conclusion: There is a gap in the area of economic evaluation studies and the nurse should occupy this space, not only as a care manager, but also of cost.

Objetivo: Identificar o que se tem sido produzido sobre análise de custos do transplante alogênico de células tronco hematopoiéticas. Método: Consta de uma revisão integrativa, em que se fez a busca de estudos sobre análise de custos em transplante alogênico. Resultados: Foram encontrados 265 artigos dos quais após aplicação dos fatores de inclusão e exclusão foram selecionados 13 artigos, sendo 12 de língua inglesa e dois de língua portuguesa. Onze desses artigos fizeram análise parcial de custos, um estudo fez revisão sistemática sobre custo-efetividade, um fez avaliação econômica de custo-efetividade e um estudo fez avaliação de custo sobre cobertura para curativo de cateter no transplante. Conclusão: Existe uma lacuna na área de estudos de avaliação econômica e o enfermeiro deveria ocupar este espaço como gerenciador não só do cuidado, mas também de custos.

Objetivo: Identificar lo que se ha producido en el análisis de costos de trasplante de las células madre hematopoyéticas alogénicas. Método: Se trata de una revisión integradora, donde hizo la búsqueda de los estudios sobre el análisis de costos en el trasplante alogénico. Resultados: Se encontraron 265 artículos que después de la aplicación de criterios de inclusión y exclusión 13 artículos, con doce en Inglés y dos en Portugués fueron seleccionados. Once de estos artículos han hecho análisis parcial de los costos, un estudio realizado una revisión sistemática de la rentabilidad, una evaluación económica hecha de costo-efectividad y el estudio de evaluación de costos sobre la cobertura de catéter curativa en el trasplante. Conclusión: Existe un vacío en el área de estudios de evaluación económica y la enfermera debe ocupar este espacio, no sólo como gestora de la atención, sino también de costo.

Design of a cost-effectiveness analysis alongside a randomized trial of transplantation using umbilical cord blood versus HLA-haploidentical related bone marrow in advanced hematologic cancer.

BACKGROUND: BMT CTN 1101 is a Phase III randomized controlled trial evaluating the comparative effectiveness of double unrelated umbilical cord blood (dUCB) versus HLA-haploidentical related donor bone marrow (haplo-BM) donor cell sources for blood or bone marrow transplantation (BMT) in patients with hematologic malignancies. Herein, we present the rationale, design and methods of the first cost-effectiveness analysis to be conducted alongside a BMT trial. METHODS: Consenting patients will provide health insurance information to allow calculation of direct medical costs from reimbursement records, and will provide out-of-pocket costs, time costs and health-related quality of life measures through an online survey. These outcomes will inform a cost-effectiveness analysis comparing dUCB and haplo-BM donor cell sources from patient, payer and societal perspectives. CONCLUSION: Novel approaches may significantly change the cost, outcomes or availability of BMT. The results of this analysis will be the first to provide a comprehensive evaluation of the comparative effectiveness of these approaches from multiple perspectives.

Systematic assessment of decision-analytic models for chronic myeloid leukemia.

BACKGROUND: Several tyrosine kinase inhibitors (TKIs) are approved for the treatment of chronic myeloid leukemia (CML). Decision-analytic modeling can help to extrapolate data from short-term clinical trials and also consider quality of life when evaluating different treatment strategies. OBJECTIVE: Our goal was to describe and analyze the structural and methodological approaches of published decision-analytic models for various treatment strategies in CML and to derive recommendations for the development of future CML models. DATA SOURCES: We performed a systematic literature search in electronic databases (MEDLINE/PreMEDLINE, EconLit, EMBASE, NHS EED, and Tuft's CEA Registry) to identify published studies evaluating CML treatment strategies using mathematical models. The search was updated in August 2013. STUDY SELECTION: The models were required to compare different treatment strategies in relation to relevant clinical and patient-relevant health outcomes [e.g., life-years gained, quality-adjusted life-years] over a defined time horizon and population. STUDY APPRAISAL AND SYNTHESIS METHODS: We used standardized forms for data extraction, description of study design, methodological framework, and data sources for each model. RESULTS: We identified 18 different decision-analytic modeling studies. Of these, 17 included economic evaluations. Modeling approaches included decision trees, Markov cohort models, state-transition models with individual (Monte Carlo) simulations, and mathematical equations. Analytic time horizons ranged from 2 years to a lifetime. Treatment strategies compared included bone marrow or stem cell transplantation, conventional chemotherapy, interferon-α, and TKIs. Only one model evaluated a second-generation TKI. Most models did not report a model validation. All models conducted deterministic sensitivity analyses and four reported a probabilistic sensitivity analysis. LIMITATIONS: Articles that were not published in English or German were not included in this review. Our literature search was restricted to published full-text articles in certain databases. Therefore, publications that met our inclusion criteria but were published in different databases, different languages, or as abstracts only may have been missed. CONCLUSIONS: While several well-designed models of CML treatment strategies exist, there remains a need for the assessment of the long-term efficacy and cost effectiveness of novel treatment options such as second-generation TKIs. Additionally, these models should be validated using independent data.

Removing financial barriers to organ and bone marrow donation: the effect of leave and tax legislation in the U.S.

Many U.S. states have passed legislation providing leave to organ and bone marrow donors and/or tax benefits for live and deceased organ and bone marrow donations and to employers of donors. We exploit cross-state variation in the timing of such legislation to analyze its impact on organ donations by living and deceased persons, on measures of the quality of the transplants, and on the number of bone marrow donations. We find that these provisions do not have a significant impact on the quantity of organs donated. The leave laws, however, do have a positive impact on bone marrow donations, and the effect increases with the size of the population of beneficiaries and with the generosity of the legislative provisions. Our results suggest that this legislation works for moderately invasive procedures such as bone marrow donation, but these incentives may be too low for organ donation, which is riskier and more burdensome.

Treatment trade-offs in myeloma: A survey of consecutive patients about contemporary maintenance strategies.

BACKGROUND: Two randomized trials have demonstrated improved progression-free survival (PFS) with lenalidomide maintenance after autologous transplantation for multiple myeloma (MM). Overall survival (OS) results are conflicting, and quality-of-life (QOL) data are lacking. The authors conducted a systematic survey of patients with MM regarding what constitutes a meaningful benefit that would make burdens of maintenance treatments (toxicity and cost) acceptable. METHODS: A self-administered survey was mailed to 1159 consecutive, living patients who were evaluated at Mayo Clinic. The survey provided background information on the standard of care for MM and data on maintenance. Patients were asked to estimate the magnitude of OS benefit that would be acceptable for various degrees of toxicity and cost. RESULTS: Of 1159 surveys sent, 886 patients (83.2%) responded, and 736 patients returned a completed survey (66% raw response rate). The most worrisome potential toxicity was identified as peripheral neuropathy by 27% of patients, cytopenias by 24%, deep vein thrombosis by 20%, fatigue by 15%, nausea by 8%, and diarrhea/constipation by 7%. If treatment was free, had no toxicity, and the OS benefit was ≤1 year, then 49% of patients indicated that they would choose maintenance; with moderate toxicity, this proportion decreased to 42%. Adding a treatment cost of $25 per month decreased the proportion that would choose maintenance to 39% of patients. CONCLUSIONS: The current results indicated that willingness to receive maintenance treatment declined when actual benefits were provided in concrete numeric terms compared with a general statement of PFS benefit. The authors also observed that the magnitude of benefit required to consider maintenance was affected by cost and toxicity.