Improved Surveillance, Registry Data Could Improve Outcomes in Sickle Cell Disease

Population-based data is a crucial aspect to evaluating current care quality and improving outcomes for patients with sickle cell disease.

A review published in JAMA Health Forum emphasized the importance of national surveillance programs to gather longitudinal data and evaluate care access and quality for patients with sickle cell disease (SCD).

SCD, while rare, is the most prevalent and clinically significant inherited blood disorder in the United States. The disease most often occurs in Black individuals and is characterized by misshapen red blood cells with a “sickle” appearance that cause a range of complications, including chronic pain, acute pain crises, acute chest syndrome, stroke, organ damage, an increased risk of severe infections, and reduced longevity.

Treatment with evidence-backed therapies can improve longevity as well as patient quality of life. Still, there is very little longitudinal data evaluating access to care or treatment quality among patients with SCD. The review highlights a lack of population-based data and emphasizesthe importance of a longitudinal clinical registry and a national surveillance programto optimize care for this patient population.

The complications of SCD significantly impact patient quality of life, and while child mortality has been reduced in high-resource countries, the majority of adults with SCD die before reaching 50 years of age. The clinical manifestations and severity of SCD also vary from patient to patient, even among those with the same sickle genotype. Both the acute and chronic complications of SCD lead to high health care utilization and costs.


“Despite the substantial personal and societal burden of SCD, compared with other genetic disorders SCD has received relatively little attention and minimal resources since it was first described in 1910,” the authors wrote.“Investments in discoveries and clinical care infrastructure have been limited and have not kept pace with other equally devastating but less common inherited conditions.”

They noted that individuals with SCD are often part of a racial or ethnic minority group,are economically disadvantaged, and have limited access to health care—all factors that perpetuate a lack of awareness, attention, and investment into SCD. Due to limited financial resources, there is a lack of clinicians who are well-trained to handle SCD, especially in adults. Symptoms progress with age, and proper treatment should involve multiple subspecialists, although many adult patients do not receive adequate treatment.

Population-based data is a crucial aspect to evaluating current care quality and improving care for patients with SCD. This would ideally include both a longitudinal clinical registry and a national surveillance program, which could provide complementary data to paint a picture of the state of SCD care overall.

“If well developed, a SCD surveillance system would incorporate all individuals with SCD regardless of payer, including those not seen in established SCD care centers, and would determine the true SCD prevalence and geographic distribution of individuals with SCD,” the authors wrote.“A longitudinal clinical registry, on the other hand, would include a subset of the individuals in the surveillance system with more in-depth information, including laboratory and radiographic findings, as well as patient-reported outcomes.”

Linking these distinct surveillance data would not just expand our knowledge of the disease, but also provide a deeper understanding of the effects of current treatment strategies. From a financial standpoint, these data could help estimate resource allocations to mitigate costs associated with acute care utilization and the risk of premature death due to insufficient preventative therapy. Improved surveillance would also provide insight into the type and quality of care patients are receiving, disparities, and outcomes over time.

Several efforts to implement surveillance programs have been initiated with varied success rates, but no comprehensive national programs are in place. Regarding a clinical registry, the authors note that newborn children in the US undergo screening for SCD, providing a clear opportunity for inclusion in a registry that could help interpret and validate surveillance findings among other benefits. Surveillance and registry data from other rare diseases, including cystic fibrosis and hemophilia, have also shown the potential value in such systems for SCD.

Overall, the review highlights a need to close knowledge gaps in SCD via surveillance data in tandem with clinical registry data, both on a national scale.

“A national SCD surveillance program that makes its findings available to all stakeholders (patients, health care clinicians, researchers, legislators, advocates) would make it possible for the US to finally begin to address the health disparities imperiling individuals living with SCD,” the authors concluded.


Kanter J, Meier ER, Hankins JS, Paulukonis ST, Snyder AB. Improving outcomes for patients with sickle cell disease in the United States: Making the case for more resources, surveillance, and longitudinal data. JAMA Health Forum. Published online October 29, 2021. doi:10.1001/jamahealthforum.2021.3467

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