The economic burden of malignant and premalignant hematological diseases in Southern Nigeria.

BACKGROUND: Hematological malignancies are a significant cause of morbidity and mortality. They constitute an economic burden for the patients, their relatives, and the society because of the cost associated with their management, which is usually long term. We aimed to determine the total direct cost of managing patients with premalignant hematological disorders (PMHDs) and malignant hematological disorders (MHDs). MATERIALS AND METHODS: A hospital-based retrospective study was carried out between 1997 and 2015. Data were retrieved from the case notes of adult patients diagnosed with either PMHD or MHD. The total cost of medical care was calculated as the sum of in-patient and out-patient direct cost associated with their management. Data were analyzed using Statistical Package for Social Sciences. RESULTS: There was a total of 129 patients; 74 (57.4%) males and 55 (42.6%) females with mean age of 45.7 ± 16.3 years and the majority (n = 76, 58.9%) being employed. Males were more affected than the females except in chronic lymphocytic leukemia, myelodysplastic syndrome, and paroxysmal nocturnal hemoglobinuria. The commonest MHD was chronic myeloid leukemia with 37 (28.7%) patients. Full blood count was the commonest investigation done, whereas free light chains were the least (n = 2; 1.6%). The total cost of care for the 129 patients was N30,041,900.00 ($82,306.58) with an average total cost of care per patient of N232,882.95 ($638.04). Patients with non-Hodgkin lymphoma had the highest mean cost of care per patient (N373,196.30; $1,022.46). The average monthly expenditure per patient was about N70,000 ($190). CONCLUSION: In our setting, management of CHDs constitutes an economic burden.

Cord Blood Banking for Potential Future Transplantation.

This policy statement is intended to provide information to guide pediatricians, obstetricians, and other medical specialists and health care providers in responding to parents' questions about cord blood donation and banking as well as the types (public versus private) and quality of cord blood banks. Cord blood is an excellent source of stem cells for hematopoietic stem cell transplantation in children with some fatal diseases. Cord blood transplantation offers another method of definitive therapy for infants, children, and adults with certain hematologic malignancies, hemoglobinopathies, severe forms of T-lymphocyte and other immunodeficiencies, and metabolic diseases. The development of universal screening for severe immunodeficiency assay in a growing number of states is likely to increase the number of cord blood transplants. Both public and private cord blood banks worldwide hold hundreds of thousands of cord blood units designated for the treatment of fatal or debilitating illnesses. The procurement, characterization, and cryopreservation of cord blood is free for families who choose public banking. However, the family cost for private banking is significant and not covered by insurance, and the unit may never be used. Quality-assessment reviews by several national and international accrediting bodies show private cord blood banks to be underused for treatment, less regulated for quality control, and more expensive for the family than public cord blood banks. There is an unquestionable need to study the use of cord blood banking to make new and important alternative means of reconstituting the hematopoietic blood system in patients with malignancies and blood disorders and possibly regenerating tissue systems in the future. Recommendations regarding appropriate ethical and operational standards (including informed consent policies, financial disclosures, and conflict-of-interest policies) are provided for physicians, institutions, and organizations that operate or have a relationship with cord blood banking programs. The information on all aspects of cord blood banking gathered in this policy statement will facilitate parental choice for public or private cord blood banking.

Clinical decision support for hematology laboratory test utilization.

Clinical decision support (CDS) is the use of information and communication technologies to improve clinical decision making and patient care. CDS applications have been used in many aspects of health care, including medication ordering and diagnostic prediction algorithms. As economic and regulatory pressures place a strain on laboratory resources, the potential of CDS to improve utilization of laboratory testing has also begun to be realized. Hematology and coagulation laboratories stand to gain tremendously from the implementation of CDS interventions, given their mixture of high-volume, low-cost tests (eg CBC, PT, aPTT) and tests that carry a high potential of being misused or misinterpreted (eg lupus anticoagulant, erythrocyte sedimentation rate, heparin-induced thrombocytopenia testing). This brief review will define the key terms in the field of clinical decision support, provide instructive examples of CDS interventions to improve utilization of hematology and coagulation testing, introduce methods to implement these interventions effectively, and discuss metrics by which the success of these interventions can be evaluated.

Challenges around Access to and Cost of Life-Saving Medications after Allogeneic Hematopoietic Cell Transplantation for Medicare Patients.

Hematopoietic cell transplantation (HCT) is an expensive, medically complicated, and potentially life-threatening therapy for multiple hematologic and nonhematologic disorders with a prolonged trajectory of recovery. Similar to financial issues in other cancer treatments, adverse financial consequences of HCT are emerging as an important issue and may be associated with poor quality of life and increased distress in HCT survivors. Prescription medicine coverage for HCT for Medicare and some Medicaid beneficiaries, especially in the long-term, remains suboptimal because of inadequate payer formularies or prohibitive copays. With an increasing number of older patients undergoing HCT and improvement in the overall survival after HCT, the problem of financial burden faced by Medicare beneficiaries with fixed incomes is going to worsen. In this article, we describe the typical financial burden borne by HCT recipients based on estimated copayment amounts attached to the categories of key medications as elucidated through 2 case studies. We also suggest some possible solutions for consideration to help these patients and families get through the HCT by minimizing the financial burden from essential medications needed during the post-HCT period.

Economic burden of non-malignant blood disorders across Europe: a population-based cost study.

BACKGROUND: Blood disorders comprise a wide range of diseases including anaemia, malignant blood disorders, and haemorrhagic disorders. Although they are a common cause of disease, no systematic cost-of-illness studies have been done to assess the economic effect of non-malignant blood disorders in Europe. We aimed to assess the economic burden of non-malignant blood disorders across the 28 countries of the European Union (EU), Iceland, Norway, and Switzerland. METHODS: Non-malignant blood disorder-related costs (WHO International Classification of Diseases, 10th revision [ICD] D50-89) were estimated for 28 EU countries, Iceland, Norway, and Switzerland for 2012. Country-specific costs were estimated with aggregate data on morbidity, mortality, and health-care resource use obtained from international and national sources. Health-care costs were estimated from expenditure on primary care, outpatient care, emergency care, hospital inpatient care, and drugs. Costs of informal care and productivity losses due to morbidity and early death were also included. To these costs we added those due to malignant blood disorders (ICD-10 C81-96 and D47) as estimated in a Burns and colleagues' companion Article to obtain the total costs of blood disorders. FINDINGS: Non-malignant disorders of the blood cost the 31 European countries €11 billion in 2012. Health-care costs accounted for €8 billion (75% of total costs), productivity losses for €2 billion (19%), and informal care for less than €1 billion (6%). Averaged across the European population studied, non-malignant disorders of the blood represented an annual health-care cost of €159 per ten citizens. Combining malignant and non-malignant blood disorders, the total cost of blood disorders was €23 billion in 2012. INTERPRETATION: Our study highlights the economic burden that non-malignant blood disorders place on European health-care systems and societies. Our study also shows that blood disorder costs were evenly distributed between malignant and non-malignant blood disorders. Our results should be of use to decision makers and research-funding authorities charged with allocating health-care resources and research funds. FUNDING: European Hematology Association.

The European Hematology Association Roadmap for European Hematology Research: a consensus document.

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at €23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap.The EHA Roadmap identifies nine 'sections' in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders.The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

[The ambivalence of progress - do we need a less-is-more strategy?**. **Extract from the programmtic opening speech delivered at the 59th Annual GTH Congress 2015]. / Die Ambivalenz des Fortschritts - ist weniger mehr?*. *Auszug aus der programmatischen Eröffnungsansprache zum 59. Jahreskongress der GTH 2015.

Economizing health care systems has led to industrialized hospitals. The consequences and side effects are substantial and rather promote costs explosion due to the dictated increase in cases and efficiency. Hence, position sensing and change in direction are required. Effective ways to find out of the current crisis are discussed.

[Cost-effectiveness and safety of telaprevir and boceprevir for chronic hepatitis C in real-world clinical practice]. / Coste-efectividad y seguridad de telaprevir y boceprevir para el tratamiento de la hepatitis C crónica en la práctica clínica.

INTRODUCTION: Triple therapy with telaprevir or boceprevir has proven to be effective in the treatment of chronic hepatitis C with response rates of up to 88%. However, the treatment may be associated with important adverse effects and a high economic impact. OBJECTIVE: To assess the cost-effectiveness and safety of triple therapy with telaprevir or boceprevir for the treatment of chronic hepatitis C. METHODS: Retrospective observational study. We included all patients who had started treatment with protease inhibitors before July 31(st), 2013. We evaluated sustained virological response, the cost per patient achieving sustained virological response, and the cost of the supportive treatment for adverse events associated with triple therapy. RESULTS: Fifty-nine patients were included; 35 had been treated with telaprevir (59.3%) and 24 with boceprevir (40.7%). Sustained virological response was achieved by 38 (64.4%) patients: 24 (68.6%) patients in the telaprevir treatment arm and 14 (58.3%) patients in the boceprevir treatment arm. The cost per patient with sustained virological response was 43,555 € (95% CI 35,389-51,722 €). There were no statistically significant differences between the overall costs of therapy with telaprevir, 43,494 € (95% CI 34,795 €-55,092 €) versus boceprevir, 42,005 € (95% CI 32,122-64,243€). The mean cost of supportive care per patient was 1,500 €, while the maximum cost was 11,374 €. Due to adverse events, 8 (13.6%) patients required hospital admission, 22 (37.3%) patients attended the accident and emergency department, and 26 (44.1%) patients needed additional medical consultations. CONCLUSIONS: The treatment of triple therapy with telaprevir or boceprevir resulted in high cost per patient with sustained virological response. Due to adverse events, a high number of patients required supportive care, whose costs should be added to those of triple therapy.

A dedicated protocol and environment for central venous catheter removal in pediatric patients affected by onco-hematological diseases.

PURPOSE: The removal of long-term central venous catheters (CVCs) is not performed according to evidence-based guidelines, thus conveying the message that it is a procedure of secondary importance. Our study aims at comparing the experience at Bambino Gesù Pediatric Hospital before and after the implementation of a dedicated protocol and the identification of a specific area to perform such a procedure under the so-called nonoperating room anesthesia (NORA). METHODS: Starting on January 1, 2010, an appropriate protocol regarding long-term CVC removal was applied. Then, data from all patients who underwent CVC removal under NORA regimen were compared with patients who have undergone the same procedure before the beginning of such protocol in terms of complication rate, duration of procedure, and costs. RESULTS: Between January 2010 and December 2012, 266 patients were evaluated for long-term CVC removal under a NORA regimen. Of these, 194 underwent the procedure. In the period from January 2007 to December 2009, 60 out of 82 patients scheduled for elective removal of long-term CVC in the operating theatre were eligible for this study. Median procedure time was 7 min for removal in NORA and 10 min for the operating theatre (p=0.016); no complications occurred. CONCLUSION: Long-term CVC removal is an often-neglected procedure, carrying a small, but definite rate of complications. Our study shows that CVC removal performed in NORA regimen is safe and feasible, also allowing multiple procedures in the same session with prompt management of possible complications and reduction of the anxiety and pain associated with the procedure.

Compensation for work-related hematologic, liver, and infectious diseases.

Occupational diseases may be defined only medically or scientifically, and even then, their definition is not simple. However, compensable occupational diseases involve the additional layer of legal systems and social welfare policies as well. Their multifaceted nature makes determining the work-relatedness of these diseases more complex. Korea has established standards for the recognition of occupational diseases in Schedule 5 of the Enforcement Decree of the Labor Standards Act, and specific criteria for the recognition of occupational diseases are listed in Schedule 3 of the Enforcement Decree of the Industrial Accident Compensation Insurance Act. The new list of compensable occupational diseases comprises 13 articles as an open-ended system. The newly added articles pertain to lymphohematopoietic (Article 5) and infectious diseases (Article 9), as well as diseases of other target organs. Furthermore, the article on liver diseases (Article 8) has been partially revised. The new act has been changed to clarify the meaning as it has been presented in recent research. It is necessary to achieve agreement among concerned parties, including experts from the legal, medical, and social domains to resolve the issues of work-relatedness, causation, notion of aggravation, and so on for preparing a list and a process that are more reasonable.

Transfusion in palliative cancer patients: a review of the literature.

BACKGROUND: Transfusion is not an exceptional circumstance in palliative cancer patients (PCPs). This makes it necessary to confront not only medical aspects but also those of infrastructure and ethical issues. On some occasions, literature needs to be consulted to work out the best approach in a patient's particular case. Our aim was to review the literature contained in PubMed and EMBASE so as to find out about the information available on transfusion in PCPs. METHODS: A search for literature was carried out in databases PubMed and EMBASE, using "transfusion," "cancer," "end-of-life care," "terminal care," and "palliative care" as key words. Publications were classified according to the main topic discussed (clinical, infrastructure, and ethics) and the information included in each article critically assessed. RESULTS: We found 334 articles but only 43 were considered valuable for the present study. Of these 43 articles, 21 deal with clinical topics while 12 deal with infrastructure and 10 with ethical issues. There is an absolute lack of randomized controlled trials or clinical guidelines. Trigger parameters for transfusion are not clearly established. Benefits of the procedure are shortly experienced and remain controversial. Home transfusions are encouraged, but this sole procedure has not been demonstrated to be cost effective. Different cultures, cases, and realities illustrate the diversity of the ethical management of transfusion in PCPs. DISCUSSION: Although transfusion is certainly a common practice in PCPs, there is a relative lack of literature on this topic. Publications are unconnected and hardly any prospective studies have been performed. A large part of the little literature available only concerns descriptive and very general aspects of the issue. As transfusional products and financial and human resources are finite, it would be desirable to establish clear research lines on the different aspects considered (clinical, infrastructure, and ethical) that can help clinicians, nurses, patients, and carers to make a decision.

Direct costs associated with febrile neutropenia in inpatients with hematological diseases in Singapore.

PURPOSE: This prospective cohort study aims to investigate the direct hospitalization costs incurred during febrile neutropenia (FN) in inpatients with underlying hematological conditions and also to elucidate the factors associated with a high cost of managing febrile neutropenia. METHODS: Patients with underlying hematological conditions and documented FN were recruited between October 2008 and February 2011. FN-related costs included all costs incurred from the first day of FN until the last day of antibiotics prescribed. Relevant clinical factors were analyzed using generalized estimating equation models to elucidate the factors that were associated with higher costs of FN. RESULTS: A total of 175 patients were recruited with 303 documented episodes of FN. In non-transplant patients, 75.6 % of the FN episodes occurred. The median and mean cost incurred for each FN episode was USD9,060 (interquartile range = USD5,047-16,631) and USD15,298 (standard deviation ± USD17,459), respectively, accounting for approximately 38 % of the median total hospitalization cost and 37 % of the mean total hospitalization cost. The ward charges (44.1 %) constituted the largest component of the cost, followed by the laboratory charges (27.3 %) and medications (18.7 %), of which antimicrobials constituted 9.6 % of the cost of FN. The factors associated with higher costs of FN include cytomegalovirus reactivation (p < 0.001), longer duration of antibiotics (p < 0.001), lower absolute neutrophil count nadir (p < 0.001), allogeneic stem cell transplantation (p < 0.01), and diagnosis of invasive fungal infection (p < 0.05). CONCLUSION: The economic cost of management of FN in hematology inpatients is considerable and in addition to the overall risk of mortality for this condition. Strategies to reduce FN or ameliorate its costs are essential for this group of patients.

Family-directed umbilical cord blood banking.

Umbilical cord blood transplantation from HLA-identical siblings provides good results in children. These results support targeted efforts to bank family cord blood units that can be used for a sibling diagnosed with a disease which can be cured by allogeneic hematopoietic stem cell transplantation or for research that investigates the use of allogeneic or autologous cord blood cells. Over 500 patients transplanted with related cord blood units have been reported to the Eurocord registry with a 4-year overall survival of 91% for patients with non-malignant diseases and 56% for patients with malignant diseases. Main hematologic indications in children are leukemia, hemoglobinopathies or inherited hematologic, immunological or metabolic disorders. However, family-directed cord blood banking is not widely promoted; many cord blood units used in sibling transplantation have been obtained from private banks that do not meet the necessary criteria required to store these units. Marketing by private banks who predominantly store autologous cord blood units has created public confusion. There are very few current validated indications for autologous storage but some new indications might appear in the future. Little effort is devoted to provide unbiased information and to educate the public as to the distinction between the different types of banking, economic models and standards involved in such programs. In order to provide a better service for families in need, directed-family cord blood banking activities should be encouraged and closely monitored with common standards, and better information on current and future indications should be made available.

The unit costs of inpatient hospital days, outpatient visits, and daycare treatments in the fields of oncology and hematology.

OBJECTIVES: Many economic evaluations are conducted in the fields of oncology and hematology, partially owing to the introduction of new expensive drugs in this field. Even though inpatient days, outpatient visits, and daycare treatments are frequently the main drivers of total treatment costs, their unit costs often lack generalizability. Therefore, we aimed to determine the unit costs of inpatient hospital days, outpatient visits, and daycare treatments specifically for oncological and hematological diseases in The Netherlands from the hospital's perspective. METHODS: Unit costs were collected from 30 oncological and hematological departments of 6 university and 24 general hospitals. Costs included direct labor and indirect labor, hotel and nutrition, overheads and capital. Ordinary least squares regression models were constructed to examine the degree of association between unit costs and hospital and hospital department characteristics. All costs were based on Euro 2007 cost data. RESULTS: At university hospitals, the unit costs per inpatient day were determined at €633 in oncological and €680 in hematological departments. At general hospitals, the mean costs per inpatient day were €400. Unit costs for inpatient hospital days, outpatient visits. and daycare treatments equalled the relative ratio 100:21:44. Direct labor costs were the major cost driver and the type of hospital (university, yes/no) was a strong predictor of unit costs. CONCLUSIONS: The present study provided unit costs for inpatient hospital days, outpatient visits, and daycare treatments in the fields of oncology and hematology. The results may be used as Dutch reference unit prices in economic evaluations assessing oncological and hematological diseases.

Costs of managing adverse events in the treatment of first-line metastatic renal cell carcinoma: bevacizumab in combination with interferon-alpha2a compared with sunitinib.

BACKGROUND: Bevacizumab plus interferon-alpha2a (IFN) prolongs progression-free survival to >10 months, which is comparable with sunitinib as first-line treatment of metastatic renal cell carcinoma (RCC). The two regimens have different tolerability profiles; therefore, costs for managing adverse events may be an important factor in selecting therapy. METHODS: Costs of managing adverse events affecting patients with metastatic RCC eligible for treatment with bevacizumab plus IFN or sunitinib were evaluated using a linear decision analytical model. Management costs were calculated from the published incidence of adverse events and health-care costs for treating adverse events in the United Kingdom, Germany, France and Italy. RESULTS: Adverse event management costs were higher for sunitinib than for bevacizumab plus IFN. The average cost per patient for the management of grade 3-4 adverse events was markedly lower with bevacizumab plus IFN compared with sunitinib in the United Kingdom (euro1475 vs euro804), Germany (euro1785 vs euro1367), France (euro2590 vs euro1618) and Italy (euro891 vs euro402). The main cost drivers were lymphopaenia, neutropaenia, thrombocytopaenia, leucopaenia and fatigue/asthaenia for sunitinib; and proteinuria, fatigue/asthaenia, bleeding, anaemia and gastrointestinal perforation for bevacizumab plus IFN. CONCLUSION: The costs of managing adverse events are lower for bevacizumab plus IFN than for sunitinib. The potential for cost savings should be considered when selecting treatments for RCC.

Stem cell transplantation in hematological disorders. A developing country experience-impact of cost considerations.

OBJECTIVE: To describe the experience in setting up a bone marrow transplant program at Ain Shams University, Cairo, Egypt. METHODS: Sixteen patients were transplanted at Ain Shams University Bone Marrow Transplantation unit from March 2005 to January 2008. RESULTS: Sixteen patients were transplanted with a median age of 25 years. Indications for transplantation were chronic myeloid leukemia, acute myeloid leukemia, aplastic anemia, acute lymphoblastic leukemia, and aggressive lymphoma. Seven donors and 6 patients were positive for cytomegalovirus immunoglobulin G (IgG) antibody (Ab) pretransplant. Only one patient was positive for toxoplasma IgG Ab and another had a high titre for toxoplasma IgM Ab pretransplant. Two donors and 2 recipients were positive for hepatitis B antibody markers; however, none were positive for hepatitis B virus DNA by polymerase chain reaction (PCR). None of the patients or donors were positive for hepatitis C virus via PCR pre-transplant. Acute graft versus host disease (GVHD) was seen in 3 patients, while chronic GVHD was seen in 5 patients. Primary cause of death was recurrence in 2 patients and graft failure in one patient. Thirteen are alive and disease free with a median follow-up of 20 months. CONCLUSION: Although our unit is a relatively new unit, these results are comparable to those achieved in the Western world and cost a mean of US$250,000.