Economic burden and quality of life of caregivers of patients with sickle cell disease in the United Kingdom and France: a cross-sectional study.

BACKGROUND: Sickle cell disease (SCD), a genetic blood disorder that affects red blood cells and oxygen delivery to body tissues, is characterized by haemolytic anaemia, pain episodes, fatigue, and end-organ damage with acute and chronic dimensions. Caring for patients with SCD imposes a high burden on informal caregivers. This study aims to capture the impact on health-related quality of life (HRQoL) and economic burden of caregiving for patients with SCD. METHODS: Validated instruments of HRQoL (EQ-5D-5L, Carer Quality of Life-7 dimensions [CarerQol-7D]) and productivity (Work Productivity and Activity Impairment Questionnaire: Specific Health Problem [WPAI: SHP]) were administered via a cross-sectional online survey to caregivers in the United Kingdom (UK) and France. Demographics, HRQoL, and economic burden data were analyzed using descriptive statistics. Economic burden was determined using country-specific minimum and average wage values. Subgroup analysis examined caregivers with and without SCD. RESULTS: Sixty-nine caregivers were recruited (UK, 43; France, 26), 83% were female, and 22% had SCD themselves. The mean (SD) caregiver EQ-5D-5L score was 0.66 (0.28) (UK, 0.62; France, 0.73), and the mean CarerQol-7D score was 80.69 (24.40) (UK, 78.72 [25.79]; France, 83.97 [22.01]). Mental health problems were reported in 72% and 70% of caregivers measured using the EQ-5D-5L and CarerQol-7D, respectively. Financial problems were reported by 68% of caregivers, with mean annual minimum wage productivity losses of £4209 and €3485, increasing to £5391 and €9319 for average wages. Sensitivity analysis determined additional HRQoL decrements for caregivers with and without, SCD. CONCLUSION: Caring for patients with SCD impacts the HRQoL and economic burden of caregivers. Further research to support the complex needs of SCD caregivers is required.

Caregivers play an important role in the lives of the people with sickle cell disease that they care for; however, their mental and physical health and their finances can be affected, particularly if their ability to work is impacted. The extent to which caring for a person with sickle cell disease impacts caregivers is not fully understood. In this study, 69 caregivers of a family member, partner, or friend with sickle cell disease in the United Kingdom or France completed an online survey to share their experiences about how caring for someone with sickle cell disease can impact a caregiver's quality of life and financial well-being. Caregiving negatively affected the quality of life of caregivers compared with people in the general population and caused a large financial and social burden. Around 70% of caregivers reported having mental health problems, 68% reported financial problems, and lost work hours and lost income were not uncommon. More research is needed to understand the specific needs of caregivers of people with sickle cell disease and how best to support them.

Standardized Management of Sickle Cell Disease Patients and the Effects on Care Utilization and Costs.

An individualized management program for patients with sickle cell disease (SCD) was created to reduce health care utilization and cost. The program was implemented to standardize the management of patients with SCD. SCD encounters from January 2010 to December 2020 were reviewed for analysis. Preintervention utilization of inpatient, emergency room, and outpatient settings was compared to postintervention. There were 7114 encounters analyzed. Outpatient encounters increased from 36.5% to 70.9%; inpatient encounters decreased from 38.6% to 20.3%; and emergency department visits decreased from 20.3% to 8.8%. The number of high inpatient utilizers decreased 8.4% and the number of individuals who received any emergency care decreased 11.9%. When comparing average charges per time period, the median charge per encounter decreased by $1838 postintervention compared to preintervention. This newly implemented SCD program demonstrated success through shifting the care of the SCD patient to the outpatient setting rather than the emergency department or inpatient hospitalizations.

Budget Impact of Disease-Modifying Treatments and a CRISPR Gene-Edited Therapy for Sickle Cell Disease.

BACKGROUND AND OBJECTIVE: Treatment of sickle cell disease (SCD) has traditionally focused on symptomatic and preventative care. Recent advances in novel therapeutic developments, likely to be orphan-designated, are anticipated to carry a substantial price tag. This study assesses the potential budget impact of adopting disease-modifying treatments, crizanlizumab and voxelotor, and pioneering CRISPR gene-edited therapy, CTX001, in the Belgian healthcare system. METHODS: The perspective of the Belgian healthcare payer (RIZIV-INAMI including patient copayments), a 5-year horizon with a 2-10% uptake of disease-modifying interventions, and a 2% uptake of CTX001 were considered. Data, encompassing target population, current (chronic and acute management, curative hematopoietic stem cell transplantation) and new (crizanlizumab, voxelotor, and CTX001) interventions, clinical effectiveness, adverse events, healthcare resource utilization, and associated costs, were gathered through a comprehensive literature review (first phase) and two Delphi panels involving hematologists (second phase). The cost difference between a "world with and without crizanlizumab, voxelotor, and CTX001" was calculated to obtain the budget impact. Three scenario analyses were conducted: a 5-13% and 4% uptake analysis, a 10-18% and 8% uptake analysis, respectively for disease-modifying treatments (crizanlizumab and voxelotor) and CTX001, and a 0% crizanlizumab uptake and managed entry agreements analysis . A ± 20% univariate sensitivity analysis was performed to test the robustness of the analysis. RESULTS: The total five-year cumulative budget impact was estimated at €30,024,968, with 91% attributed to drug acquisition costs. The largest budget impact share was for CTX001 (€25,575,150), while crizanlizumab (€2,301,095) and voxelotor (€2,148,723) was relatively small. In scenarios one and three, a two-fold increase of the cumulative budget impact to €60,731,772 and a four-fold increase to €120,846,256 from the base case was observed. In scenario three, this budget impact decreased by 63% to €11,212,766. Patient population size, number of treated patients, and drug costs influenced the analysis the most, while discontinuation, acute crisis, and adverse event rates had comparatively minimal impact. CONCLUSIONS: Belgian decision-makers may consider alternative financing models, such as outcome-based risk-sharing agreements or annuities, to ensure sustainable coverage of these treatments. This study adheres to recommended practices for assessing budget impact of orphan drugs, distinguishing it from earlier studies with potentially weaker methodologies.

The Double-Edged Sword of Extremely High Prices for Gene Therapies in Sickle Cell Disease.

This Viewpoint discusses the high cost of new gene therapies for sickle cell disease, the challenges these costs pose for health care access, and new policy approaches to ensure fair reimbursement for payers and manufacturers without further increasing health care costs or barriers to access for underserved populations.

Optimizing the management of inherited blood disorders in a changing market: Findings from the AMCP Market Insights Program.

In this market insights program, AMCP brought together a panel of experts representing various stakeholders: national and regional health plans, integrated health care systems, employer benefits groups, clinical experts, the Centers for Disease Control and Prevention, and patient advocacy organizations. The objectives were to gain insights into the current and evolving treatments in hemophilia, sickle cell disease, and ß-thalassemia; measure the effects of recently approved therapies on clinicians, payers, and patients; recognize emerging trends within the stop-loss market; address potential issues and obstacles related to monitoring and reporting outcomes; and identify concerns associated with both existing and emerging contracting and reimbursement models. This article aims to summarize expert perspectives on health care system challenges and strategies concerning the management of inherited blood disorders and to advance managed care professionals' understanding of their role in supporting care for these patients. The experts emphasized that when shaping coverage policies, a patient-centered approach is crucial, focusing on preserving organ function to maintain eligibility for future gene therapies among individuals with inherited blood disorders. These strategies, including benefit design modifications, specialized provider networks, and centralized mechanisms like registries, are vital for evaluating effectiveness, facilitating decision-making, and managing costs and risks associated with new and emerging treatment options for inherited blood disorders.

Costs and impact of disease in adults with sickle cell disease: a pilot study.

ABSTRACT: We assessed the feasibility to estimate illness burden in adults with SCD, investigated factors associated with health-related quality of life (HRQoL), and estimated societal burden. We recruited 32 participants and collected data on fatigue, HRQoL, and work productivity and activity impairment via patient survey. Health care utilization was abstracted for the 12 months before enrollment using medical chart review. Mean age was 36.7 years; 84.4% of participants had hemoglobin SS or Sßthal0 disease, and 81.3% reported chronic pain (experiencing pain on ≥3 days per week in the past 6 months). Mean EQ-5D-3L visual analogue scale score was 63.4 and the index score was 0.79. The mean fatigue score was 57.9. Higher fatigue score was correlated with lower EQ-5D index score (correlation coefficient r = -0.35; P = .049) and Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) scores, including pain (r = -0.47; P = .006), sleep (r = -0.38; P = .03), and emotion scores (r = -0.79; P < .0001). The number of hospitalizations was negatively correlated with HRQoL (all P < .05). Patients who reported chronic pain had significantly lower mean ASCQ-Me sleep scores (48.3 vs 57.1; P = .04) and EQ-5D index scores (0.72 vs 0.89; P = .002) than those without chronic pain. Mean estimated annual per person costs were $51 779 (median, $36 366) for total costs, $7619 ($0) for indirect costs (estimated from lost earnings of participants), and $44 160 ($31 873) for medical costs. Fatigue, SCD complications, hospitalization, and chronic pain negatively affected HRQoL. This sample experienced a high economic burden, largely from outpatient doctor visits.

Cost analysis for initiating an integrated package of essential non-communicable disease interventions (PEN-Plus) in Kondoa District Hospital, Tanzania: a time-driven activity-based costing (TDABC) study protocol.

INTRODUCTION: Non-communicable diseases (NCDs) constitute approximately 74% of global mortality, with 77% of these deaths occurring in low-income and middle-income countries. Tanzania exemplifies this situation, as the percentage of total disability-adjusted life years attributed to NCDs has doubled over the past 30 years, from 18% to 36%. To mitigate the escalating burden of severe NCDs, the Tanzanian government, in collaboration with local and international partners, seeks to extend the integrated package of essential interventions for severe NCDs (PEN-Plus) to district-level facilities, thereby improving accessibility. This study aims to estimate the cost of initiating PEN-Plus for rheumatic heart disease, sickle cell disease and type 1 diabetes at Kondoa district hospital in Tanzania. METHODS AND ANALYSIS: We will employ time-driven activity-based costing (TDABC) to quantify the capacity cost rates (CCR), and capital and recurrent costs associated with the implementation of PEN-Plus. Data on resource consumption will be collected through direct observations and interviews with nurses, the medical officer in charge and the heads of laboratory and pharmacy units/departments. Data on contact times for targeted NCDs will be collected by observing a sample of patients as they move through the care delivery pathway. Data cleaning and analysis will be done using Microsoft Excel. ETHICS AND DISSEMINATION: Ethical approval to conduct the study has been waived by the Norwegian Regional Ethics Committee and was granted by the Tanzanian National Health Research Ethics Committee NIMR/HQ/R.8a/Vol.IX/4475. A written informed consent will be provided to the study participants. This protocol has been disseminated in the Bergen Centre for Ethics and Priority Setting International Symposium, Norway and the 11th Muhimbili University of Health and Allied Sciences Scientific Conference, Tanzania in 2023. The findings will be published in peer-reviewed journals for use by the academic community, researchers and health practitioners.

Cost-effectiveness of newborn screening for sickle cell disease: a systematic review protocol.

OBJECTIVE: The purpose of this systematic review is to assess the cost-effectiveness of targeted/selective newborn screening compared with universal screening for sickle cell disease across various countries and settings. INTRODUCTION: The incidence of sickle cell disease is a widespread and potentially fatal hematologic disorder that affects thousands of newborns worldwide. The cost of newborn screening creates a burden on households and the economy. INCLUSION CRITERIA: Studies will be eligible for inclusion in the review if they focus on the cost-effectiveness of newborn screening for sickle cell disease, comparing targeted/selective screening with universal screening. METHODS: A preliminary search of MEDLINE (PubMed) was undertaken using MeSH terms, such as sickle cell disease, newborn , and economic evaluations . Two reviewers will screen the titles, abstracts, and full text independently against the inclusion criteria. Disagreements will be resolved by discussion or with a third reviewer. To assess methodological quality, the JBI checklist for economic evaluation will be used. Data will be extracted by 2 reviewers using a modified JBI data extraction form. The JBI dominance ranking matrix for economic evaluations will be used to summarize and compare the results. Cost-effectiveness will be measured on the basis of cost per test/case detected, quality-adjusted life years gained, or disability-adjusted life years averted. The Grading of Recommendations, Assessment, Development and Evaluation (GRADE) assessment will be conducted to evaluate the certainty of economic evidence, such as use of resources and expenditures, and to incorporate the results into the decision-making process. REVIEW REGISTRATION: PROSPERO CRD42017057963.

Health State Utilities for Sickle Cell Disease: A Catalog Prepared From a Systematic Review.

OBJECTIVES: Sickle cell disease (SCD) is a complex, chronic condition that impairs health-related quality of life of affected individuals and their caregivers. As curative therapies emerge, comprehensive cost-effectiveness models will inform their value. These models will require descriptions of health states and their corresponding utility values that accurately reflect health-related quality of life over the disease trajectory. The objectives of this systematic review were to develop a catalog of health state utility (HSU) values for SCD, identify research gaps, and provide future directions for preference elicitation. METHODS: Records were identified through searches of PubMed and Embase, Tufts Medical Center Cost-Effectiveness Analysis Registry, reference lists of relevant articles, and consultation with SCD experts (2008-2020). We removed duplicate records and excluded ineligible studies. For included studies, we summarized the study characteristics, methods used for eliciting HSUs, and HSU values. RESULTS: Five studies empirically elicited utilities using indirect methods (EQ-5D) (n = 3) and Short Form-6 Dimension (n = 2); these represent health states associated with general SCD (n = 1), SCD complications (n = 2), and SCD treatments (n = 3). Additionally, we extracted HSUs from 7 quality-adjusted life-years-based outcome research studies. The HSU among patients with general SCD without specifying complications ranged from 0.64 to 0.887. Only 36% of the HSUs used in the quality-adjusted life-year-based outcomes research studies were derived from individuals with SCD. No study estimated HSUs in caregivers. CONCLUSIONS: There is a dearth of literature of HSUs for use in SCD models. Future empirical studies should elicit a comprehensive set of HSUs from individuals with SCD and their caregivers.

Profile of Medicaid enrollees with sickle cell disease: A high need, high cost population.

Sickle cell disease is a progressively debilitating genetic condition that affects red blood cells and can result in a variety of serious medical complications, reduced life expectancy, and diminished quality of life. Medicaid nationwide covered 66 percent of sickle cell disease hospitalizations in 2004 and 58 percent of emergency department visits for the disease between 1999 and 2007. Using Medicaid data from four states with large populations that account for more than one-third of Medicaid program enrollment, we examined the characteristics of those with sickle cell disease. We found instances of mortality rates more than nine times the age-adjusted population average (in Texas, a mortality rate for Medicaid enrollees with SCD of 1.11 percent compared to 0.12 percent overall); rates of disability-related eligibility-which is associated with long-term Medicaid enrollment-of up to 69 percent; and half or more of affected enrollees having (all-cause) hospital stays, emergency department visits, and opioid prescription fills. With gene therapies on the horizon that will spur discussions of treatment coverage, costs, and outcomes for people with sickle cell disease, it is important for relevant stakeholders to understand the affected populations.

Total Annual Economic Burden of Patients with Sickle Cell Disease in Steady State in Greece.

Sickle cell disease includes a group of congenital hemolytic anemias, all characterized by the predominance of Hb S (HBB: c.20A>T). The population movement due to economic migration or escape from conflict zones will further affect the health systems of countries by either increasing the number of patients or forcing countries to create care units for sickle cell disease patients. This will probably also increase the incidence of the disease in areas where their incidence and prevalence were previously low. In the present study, an attempt has been made to estimate the total annual cost of the treatment of sickle cell disease in Greece. This was the first attempt to calculate the total annual cost of treating sickle cell disease patients in a steady state. The annual cost of sickle cell disease was estimated to be €21,152,340.00 (US$25,219,300.41), without calculating the cost of hospitalization for severe complications. Since 2013, in Greece, a pharmaceutical expenditure limit (decreasing with the years) has been budgeted at €1,945,000,000.00 (US$2,318,965,150.00), annually. It is therefore calculated that approximately 1.0% of the budget allocated to pharmaceutical spending is used to treat patients with sickle cell disease.

Indirect Economic Burden of Sickle Cell Disease.

OBJECTIVE: This study aimed to quantify the indirect costs of sickle cell disease in the United States. METHODS: Adult patients from a sickle cell disease clinic at an urban academic healthcare system completed an adapted Institute for Medical Technology Assessment Productivity Cost Questionnaire related to the impact of their disorder on absenteeism, presenteeism, ability to contribute through unpaid work outside of employment, and other aspects of life. Additional data were collected from patient records about each participant's genotype, total hemoglobin level, and pain level. RESULTS: Of the 192 individuals, 187 who completed the survey reported experiencing vaso-occlusive crisis pain events during the last year that negatively affected their productivity at work and in daily roles. Three-fourths of respondents reported impairment in their ability to complete everyday tasks, such as caring for children, running errands, doing housework, shopping for groceries, and volunteer (unpaid) work. Only 30% of respondents reported being employed or self-employed. Of those employed, estimated costs of absenteeism and presenteeism attributable to pain events averaged $15 103 per person annually. Estimated total annual losses in unpaid work productivity averaged $3 145 862 for the study respondents and another $2 870 652 for their caregivers. CONCLUSIONS: Sickle cell disease affected the work productivity, nonwork productivity, and the daily lives of adults seen with the disorder in an academic medical center.

Epidemiology and disease burden of sickle cell disease in France: A descriptive study based on a French nationwide claim database.

CONTEXT: Sickle cell disease (SCD) is a severe hematological disorder. The most common acute complication of SCD is vaso-occlusive crisis (VOC), but SCD is a systemic disease potentially involving all organs. SCD prevalence estimates rely mostly on extrapolations from incidence-based newborn screening programs, although recent improvements in survival may have led to an increase in prevalence, and immigration could account for a substantial number of prevalent patients in Europe. The primary objective of this study was to estimate SCD prevalence in France. METHODS: A cross-sectional observational study was conducted using a representative sample of national health insurance data. SCD patients followed up in France between 2006 and 2011 were captured through hydroxyurea reimbursement and with the International Classification of Diseases (ICD-10) SCD specific code D570.1.2, excluding code D573 (which corresponds to sickle cell trait (SCT)). Nevertheless, we assumed that ICD-10 diagnosis coding for inpatient stays could be imperfect, with the possibility of SCT being miscoded as SCD. Therefore, prevalence was analyzed in two groups of patients [with at least one (G1) or two (G2) inpatient stay] based on the number of SCD-related inpatient stays in the six-year study period, assuming that SCT patients are rarely rehospitalized compared to SCD. The prevalence of SCD in the sample, which was considered to be representative of the French population, was then extrapolated to the general population. The rate of vaso-occlusive crisis (VOC) events was estimated based on hospitalizations, emergencies, opioid reimbursements, transfusions, and sick leave. RESULTS: Based on the number of patients identified for G1 and G2, the 2016 French prevalence was estimated to be between 48.6 per 100,000 (G1) or 32,400 patients and 29.7 per 100,000 (G2) or 19,800 patients. An average of 1.51 VOC events per year were identified, with an increase frequency of 15 to 24 years of age. The average annual number of hospitalizations was between 0.70 (G1) and 1.11 (G2) per patient. Intensive care was observed in 7.6% of VOC-related hospitalizations. Fewer than 34% of SCD patients in our sample received hydroxyurea at any point in their follow-up. The annual average cost of SCD care is €5,528.70 (G1) to €6,643.80 (G2), with most costs arising from hospitalization and lab testing. CONCLUSION: Our study estimates SCD prevalence in France at between 19,800 and 32,400 patients in 2016, higher than previously published. This study highlights the significant disease burden associated with vaso-occlusive events.

[Sickle cell disease in the Democratic Republic of the Congo: what are the barriers to treatment using hydroxyurea?] / Drépanocytose en République Démocratique du Congo: quels sont les obstacles à un traitement par hydroxyurée?

INTRODUCTION: hydroxyurea is the unique medication that has been proven to prevent complications in patients with sickle cell disease and is approved by the Food and Drug Administration. This medication requires a prescription to be dispensed, it must be available and at an affordable price. The purpose of this study was to determine the availability and market price of hydroxyurea in the Democratic Republic of the Congo and to make a comparison between these two aspects in a small city, such as Mbujimayi, and in a big city, such as Lubumbashi. METHODS: we conducted a cross-sectional study in the context of a face-to-face survey involving 188 Congolese pharmacies from 1st April to 1st September 2017. RESULTS: hydroxyurea was available at 41/188 (22%) participating pharmacies, but more frequently at those of a big city than at those of a small city (34/96 versus 7/92). Most patients got a prescription (36/41; 88%). The average price of hydroxyurea was $15 (from $10 to $35 a blister packs of 25 capsules), which was higher than the purchasing power of the majority of sickle cell patients. Hydroxyurea is still an imported product from Europe, the United States or Asia. CONCLUSIONS: hydroxyurea is one of the main treatments to slow down disease progression in sickle cell patients. Nevertheless, in the Democratic Republic of the Congo, its availability could be improved, in particular in small cities, and its price is still too high.

Some Lives Matter: The Dirty Little Secret of the U.S. Health Care System.

Our health care system in the United States reflects the inequities that are part of the larger society, which is why our system for financing access to needed and effective health care is so complicated and unfair.

Vaso-occlusive crises and costs of sickle cell disease in patients with commercial, Medicaid, and Medicare insurance - the perspective of private and public payers.

AIM: To characterize vaso-occlusive crises (VOCs) and describe healthcare costs among commercially-insured, Medicaid-insured, and Medicare-insured patients with sickle cell disease (SCD). MATERIALS AND METHODS: The IBM Truven Health MarketScan Commercial (2000-2018), Medicaid Analytic eXtract (2008-2014), and Medicare Research Identifiable Files (2012-2016) databases were used to identify patients with ≥2 SCD diagnoses. Study measures were evaluated during a 12-month follow-up period, stratified by annual number of VOCs (i.e. 0, 1, and ≥2). RESULTS: Among 16,092 commercially-insured patients (mean age = 36.7 years), 35.3% had 1+ VOCs. Mean annual total all-cause healthcare costs were $15,747, $27,194, and $64,555 for patients with 0, 1, and 2+ VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 31.0%, 1 VOC = 53.1%, 2+ VOCs = 65.4%) and SCD-related costs (0 VOC = 56.4%, 1 VOC = 78.4%, 2+ VOCs = 93.9%). Among 18,287 Medicaid-insured patients (mean age = 28.5 years, fee-for-service = 50.2%), 63.9% had 1+ VOCs. Mean annual total all-cause healthcare costs were $16,750, $29,880, and $64,566 for patients with 0, 1, and 2+ VOCs, respectively. Inpatient costs (0 VOC = 37.2%, 1 VOC = 64.3%, 2+ VOCs = 72.9%) and SCD-related costs (0 VOC = 60.9%, 1 VOC = 73.8%, 2+ VOCs = 92.2%) accounted for a significant proportion of total all-cause healthcare costs. Among 15,431 Medicare-insured patients (mean age = 48.2 years), 55.1% had 1+ VOCs. Mean annual total all-cause healthcare costs were $21,877, $29,250, and $58,308 for patients with 0, 1, and ≥2 VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 47.9%, 1 VOC = 54.9%, 2+ VOCs = 67.5%) and SCD-related costs (0 VOC = 74.9%, 1 VOC = 84.4%, 2+ VOCs = 95.3%). LIMITATIONS: VOCs managed at home were not captured. Analyses were descriptive in an observational setting; thus, no causal relationships can be inferred. CONCLUSIONS: A high proportion of patients experienced VOCs across payers. Furthermore, inpatient and SCD-related costs accounted for a significant proportion of total all-cause healthcare costs, which increased with VOC frequency.

Cost of health care for paediatric patients with sickle cell disease: An analysis of resource use and costs in a European country.

BACKGROUND: While multiple studies have examined the cost of health care for one aspect of sickle cell disease care, few have focussed on the overall cost of comprehensive care for sickle cell disease. METHODS: We conducted a retrospective cohort study of children with sickle cell disease treated in a comprehensive care centre from 1 January 2015 to 31 December 2016. Health care utilisation of included patients was based upon data from two main sources. The clinical practice guideline was used to determine the expected resource use of routine comprehensive care (planned elective care), and the financial claims database was used to estimate real-world resource use associated with acute and inpatient care (additional care). RESULTS: A total of 125 children with sickle cell disease were analysed. Expenditures for these patients averaged €5049 [standard deviation (SD) €1634] per child per year. Total yearly costs per patient varied considerably, ranging from €669 to €84 010, and less than 15% of patients were responsible for 50% of the health care costs. The majority (37%) of costs was associated with inpatient hospital care, which increased by age group, 27% with diagnostics, 19% with treatment, 11% with outpatients' visits and 6% with emergency care. CONCLUSION: We have described real-world resource use and expenditures for children with sickle cell disease in a European comprehensive care centre. It seems that costs of a comprehensive approach with effective management in the outpatient setting is favourable when compared to episodic health care.