Clinical outcomes and healthcare utilization in patients with sickle cell disease: a nationwide cohort study of Medicaid

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ORIGINAL ARTICLE

Clinical outcomes and healthcare utilization in patients with sickle cell disease: a nationwide cohort study of Medicaid beneficiaries Rishi J. Desai 1 & Mufaddal Mahesri 1 & Denise Globe 2 & Alex Mutebi 2 & Rhonda Bohn 3 & Maureen Achebe 4 & Raisa Levin 1 & Sebastian Schneeweiss 1 Received: 17 May 2020 / Accepted: 24 August 2020 # Springer-Verlag GmbH Germany, part of Springer Nature 2020

Abstract To add to the limited existing evidence on clinical outcomes and healthcare use in sickle cell disease (SCD) among beneficiaries of the US Medicaid program, we conducted a cohort study using nationwide Medicaid claims data (2000–2013). Patients were included based on HbSS SCD diagnosis and followed until Medicaid disenrollment, death, bone marrow transplant, or end of data availability to assess vasoocclusive crises (VOC), emergency room (ER) visits, hospitalizations, outpatient visits, and blood transfusions. Annualized event rates (with 95% confidence intervals [CI]) were reported. The impact of VOCs on the risk of mortality was analyzed using a multivariable Cox model with VOC modeled as time-varying and updated annually. In a total of 44,033 SCD patients included with a mean (SD) age of 15.7 (13.6) years, the VOC rate (95% CI) was 3.71 (3.70–3.72) per person-year, with highest rate among patients 19–35 years who had ≥ 5 VOCs at baseline (13.20 [13.15–13.26]). Event rates (95% CI) per person per year for other outcomes were 2.97 (2.97–2.98) ER visits, 2.39 (2.38–2.40) hospitalizations, 5.80 (5.79– 5.81) outpatient visits, and 0.91 (0.90–0.91) blood transfusions. A higher VOC burden in the preceding year was associated with an increased risk of mortality, with a hazard ratio (95% CI) of 1.26 (1.14–1.40) for 2–4 VOC vs. < 2 and 1.57 (1.41–1.74) for ≥ 5 VOC vs < 2. In conclusion, we documented a substantial burden of SCD in US Medicaid enrollees, especially during early adulthood and noted that ongoing burden of VOC is associated with mortality in these patients. Keywords Sickle cell disease . Epidemiology . Medicaid . Vasoocclusive crises

Introduction Sickle cell disease (SCD) is a monogenic disorder that is associated with a substantial burden of acute complications such as repeated episodes of vasoocclusive crises (VOC), acute chest syndrome, and infections as well as long-term organ damage. SCD affects nearly 100,000 individuals in the USA

* Rishi J. Desai [email protected] 1

Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital & Harvard Medical School, 1620 Tremont Street, Suite 3030-R, Boston, MA 02120, USA

2

Vertex Pharmaceuticals Incorporated, Boston, MA, USA

3

Bohn Epidemiology, Boston, MA, USA

4

Hematology Division, Department of Medicine, Brigham and Women’s Hospital & Harvard Medical School, Boston, MA, USA

[1], with markedly higher rates among African-Americans than any other race [1, 2]. About 90% of children born with SCD in the USA or EU are reported to survive into adulthood, but their lifespan is shortened substant