Often the best choice is a hematologist a doctor who specializes in blood diseases working with a team of specialists. Common illnesses, like the flu, can quickly become dangerous for a child with sickle cell disease. The best defense is to take simple steps to help prevent infections.
Transition Toolkit external icon. People with sickle cell disease should drink 8 to 10 glasses of water every day and eat healthy food. Try not to get too hot, too cold, or too tired. Children can, and should, participate in physical activity to help stay healthy. New clinical research studies external icon are happening all the time to find better treatments and, hopefully, a cure for sickle cell disease.
One study limitation is that the higher estimates of life expectancy yielded conservative estimates of lost life-years and income. The analysis only considered the value of lost personal income owing to premature mortality and did not consider direct medical costs or other societal costs associated with excess morbidity eg, lost workdays for disability, time spent in the hospital. The model was most sensitive to changes in income levels and mortality rates.
Conclusions and Relevance In this simulated cohort modeling study, SCD had societal consequences beyond medical costs in terms of reduced life expectancy, quality-adjusted life expectancy, and lifetime earnings. These results underscore the need for disease-modifying therapies to improve the underlying morbidity and mortality associated with SCD. Owing to the pathophysiologic characteristics of SCD, most patients experience significant lifelong morbidities as a result of the underlying hemolysis and vascular damage that results in acute eg, vaso-occlusive crises and acute chest syndrome and chronic injury to multiple end organs, including brain eg, stroke and silent infarcts , kidney eg, renal failure , and the cardiopulmonary system eg, pulmonary hypertension.
The life expectancy of individuals with SCD is decades shorter compared with those without SCD, primarily owing to early mortality during adulthood. The total burden of SCD includes not only direct medical costs, but also income and productivity loss 18 - 20 ; however, there are limited data available on the association between SCD mortality and morbidity and lifetime income.
In this study, we estimated the average life expectancy of patients with SCD, compared this with a matched, non-SCD cohort, and calculated loss of lifetime income. Together, these 2 outcomes provide a clearer picture of both the disease burden, from the perspective of individuals with SCD and the health economic burden from the perspective of society.
Each population was followed up over a complete lifetime horizon and all results were calculated separately for each cohort and by sex. Each step is described in detail below. This modeling study did not require institutional review board approval or informed consent per the University of Alabama at Birmingham because it relied solely on publications and deidentified publicly available data.
To evaluate the association between SCD and life expectancy and lifetime income, we developed a cohort simulation model. Figure 1 outlines the conceptual model and Table 1 and Figure 2 summarize model inputs and data sources, which are detailed below. These differences resulted in a decreased life expectancy as well as lost income for the individual with SCD. The model then calculated the difference in life-years and income for these 2 populations.
For comparison, we also calculated life expectancy and income for the US general population. Data for the model were collected from November 29, , to March 21, , and the analysis was performed from April 28 to December 3, All subtypes were combined to yield an overall SCD birth rate of 0. These codes have been used and validated in other studies, 11 , 17 including the source of the death rates for the model.
Therefore, the number of deaths from US mortality statistics, corrected for underreporting, 12 was combined with age-specific death probabilities to calculate the expected SCD prevalence. To quantify the morbidity outcome of SCD, patient-reported assessment of the effect of pain was linked to health utility values derived from the Euroqual-5 Dimensions EQ-5D EuroQol Research Foundation from 3 SCD studies that reported on pain using visual analog scales.
We calculated the lost income associated with premature mortality. This approach does not account for decreased earnings by patients with SCD to the extent that they are replaced through government disability payments see Discussion. Undiscounted income was used with income inflation being cancelled out by the need to discount future income. The expected lifetime income was estimated by cumulating the results for each year of age through age using methods similar to those used in estimating life expectancy.
The model was constructed based on published data and publicly available, deidentified vital statistics data. Because the cohorts were simulated, no demographic or clinical characteristics could be reported or summarized. Mortality rates by age group for SCD, as reported for California and Georgia for , 12 were smoothed by fitting a Poisson regression model to estimate mortality rates by single year of age eFigure 1 in the Supplement compares model-predicted mortality rates with the rates from Paulukonis et al Paulukonis et al identified underreporting in the MCOD data after reviewing death statistics and accounted for this underreporting.
Analyses of mortality rates and prevalence incorporated Monte Carlo sampling of the Poisson model to characterize uncertainty. Small differences in mortality rates at the youngest ages lead to large differences in prevalence estimates by year of age because of the inherent uncertainty in the published data; therefore, prevalence was further smoothed by using a Loess curve.
To understand the key factors associated with of income losses in the model, we conducted 1-way sensitivity analyses. All analyses, including the model, were performed using the R statistical programming language, version 3. Mortality rates in the SCD and the matched non-SCD cohorts were similar up to age 25 years and then were notably higher for the SCD cohort between the ages of approximately 25 and 80 years.
Life expectancy for the SCD cohort was estimated at 54 years, which was 22 years lower than the life expectancy for the matched non-SCD cohort, and 25 years lower than the US general population life expectancies of 76 and 79 years, respectively.
When the quality-of-life decrement was considered, the SCD cohort had a quality-adjusted life expectancy of 33 years, compared with 67 years for the matched non-SCD cohort and 69 years for the US general population Figure 2. The quality-adjusted life expectancy was the same 33 years for men and women in the SCD group eFigure 6 in the Supplement. The expected lifetime incomes by age and sex for the SCD cohort, the matched non-SCD cohort, and the general population eFigure 7 in the Supplement were in line with the data for the overall population Figure 3.
Mean annual personal income and the predicted SCD mortality rate by single year of age were the 2 most important drivers of lost lifetime income in a univariate sensitivity analysis eFigure 8 in the Supplement. Sensitivity analysis of utility values had no association with income or mortality as indicated in Figure 2.
Results for frequency of admissions and length of stay and for SCD hospitalizations are presented in Table 2. These data were derived from the NIS for length of stay and the NRD accounting for repeat hospitalizations to allow for reporting of number of admissions. When averaging data reported in Table 2 over all ages, hospitalized individuals with SCD spend an average of 5 to 6 days per admission with an average of 2 to 3 admissions each year.
The results from this analysis suggest that individuals with SCD experience reduced life expectancy, diminished quality-adjusted life expectancy, and lower lifetime income than individuals without SCD. We estimated that individuals with SCD live 22 fewer years 54 vs 76 years and have a quality-adjusted life expectancy of about one-half that of individuals without SCD 33 vs 67 years. To our knowledge, our study is the first to provide information on lost income owing to SCD, providing information on the association between SCD and productivity from a lifetime societal perspective.
Our analysis showed substantial reductions in lifetime income as a result of premature mortality. These losses do not include other societal costs, such as lost workdays for disability, lost educational potential, lost workdays for caregivers caring for their affected children, and patient time spent in the hospital or the emergency department. Our analysis did not capture the economic burden to society from the replacement of earnings with government-funded disability support.
Similarly, there are scant contemporary data available to quantify underemployment in this population. Moreover, caregiver loss of productivity and productivity losses associated with lost school, work, and other activities associated with SCD were not captured in our analysis, further underestimating income losses related to SCD; however, estimating these losses requires a prospective study that was out of the scope of this analysis.
The overall societal perspective is that investing in interventions that prevent the morbidity and mortality associated with SCD can lead to large gains in income that are underestimated by this study; nonetheless, this study describes the potential magnitude of these income losses.
Because we had MCOD data sets, we calculated observed age at death over several time periods and noticed a trend toward increasing survival eTable 4 in the Supplement , consistent with the literature. In addition to reduced mortality, SCD is associated with significant morbidity resulting in a year quality-adjusted life expectancy difference between the SCD and non-SCD cohorts.
It is well established that health-related quality of life is diminished in individuals with SCD. Individuals with SCD report health-related quality of life similar to that of individuals undergoing hemodialysis. This analysis also did not include any direct medical costs associated with SCD, which are substantial. We observed that hospitalized patients with SCD aged 15 to 64 years spend an average of 5 to 7 days in the hospital and younger individuals in our analysis had an average length of stay of about 4 days, consistent with other studies.
To our knowledge, recent data for direct health care costs of SCD in the United States are not available. There are a number of study limitations to consider. Our SCD prevalence estimates depended on correcting the CDC MCOD counts for underreporting; in age groups where the death rate was 0 because of the small sample size in the study of Paulukonis et al 12 , we used the adjacent lower age group as a proxy.
Our utility estimates did not account for fatigue and other important SCD symptoms, and therefore may not be reflective of the full range of quality-of-life outcomes. We also estimated future deaths for a contemporary birth population of SCD based on current death rates.
Potential clinical advances or other factors may alter those death rates. Our higher estimates of life expectancy yielded more conservative estimates of lost life-years approximately 20 years than for life expectancy in the mids. The analysis only considered the value of lost personal income owing to premature mortality. These losses do not include any direct medical costs or other societal costs, such as lost educational potential, lost workdays owing to caregivers caring for their affected children, or patient time spent in the hospital or the emergency department, nor do they account for additional challenges in finding and maintaining active employment because of SCD.
In a 1-way sensitivity analysis, personal income and mortality rates stood out as an important factor associated with the total lost lifetime income in this model.
However, we were limited to a data set that only includes mortality data from 2 states California and Georgia in this study. The lack of contemporaneous countrywide mortality data underscores the need for national registries that track the natural history of SCD and provide robust data on mortality, resource use, quality of life, employment, underemployment, and other metrics that can help quantify the true societal costs of SCD.
This information would facilitate the appropriate deployment of resources and encourage innovation in a disease that disproportionately affects an underserved population.
A contemporary simulated population of individuals born with SCD was projected to live approximately 22 years less than a matched population of individuals without SCD. These losses do not include any direct medical costs or other societal costs. Nonetheless, our results suggest that, even measured solely as lost productivity and income, SCD has serious societal consequences beyond the resources required to provide medical care for individuals with SCD, information that can be used to anticipate public health care service needs and develop policy for this condition.
Therefore, there is a need to develop disease-modifying therapies that can improve the underlying morbidity and mortality of individuals with SCD. Published: November 15, Author Contributions: Drs Lubeck and Danese had full access to all of the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. No other disclosures were reported.
Outcomes Insights Inc was responsible for data collection and development and for data analysis. All authors had authority over manuscript preparation and revision and the decision to submit the manuscript for publication.
No other compensation was received. In comparing my results with this study, there is a difference of approximately six years, from 48 to 54, a difference of From this perspective, it may be the case that the period life expectancy estimates I generated are biased downward because they represent older cohorts that did not have the same levels of screening, treatment, and education as those who were younger. However, this may have been picked up to some extent in the cohort simulation approach, as it estimates that the SCD population has six more years of life expectancy at birth than the estimate I generated.
In any case, both estimates are consistent with an observation that the average projected lifespan of affected adults those in the older cohorts has not improved beyond the fifth decade. Hopefully, additional funding will continue under the Biden Administration and beyond. Search Options. Skip to main content Press Enter. Sign in. Skip auxiliary navigation Press Enter.
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