Economic and Clinical Burden of Managing Sickle Cell Disease with Recurrent Vaso-Occlusive Crises in the United States

Abstract

Introduction: The aim of this study was to describe the clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in the US.

Methods: Merative MarketScan Databases were used to identify patients with SCD with recurrent VOCs from March 1, 2010, to March 1, 2019. Inclusion criteria were ≥ 1 inpatient or ≥ 2 outpatient claims for SCD and ≥ 2 VOCs per year in any 2 consecutive years after the first qualifying SCD diagnosis. Individuals without SCD in these databases were used as matched controls. Patients were followed for ≥ 12 months, from their second VOC in the 2nd year (index date) to the earliest of inpatient death, end of continuous enrollment in medical/pharmacy benefits, or March 1, 2020. Outcomes were assessed during follow-up.

Results: In total, 3420 patients with SCD with recurrent VOCs and 16,722 matched controls were identified. Patients with SCD with recurrent VOCs had a mean of 5.0 VOCs (standard deviation [SD] = 6.0), 2.7 inpatient admissions (SD 2.9), and 5.0 emergency department visits (SD 8.0) per patient per year during follow-up. Compared to matched controls, patients with SCD with recurrent VOCs incurred higher annual ($67,282 vs. $4134) and lifetime ($3.8 million vs. $229,000 over 50 years) healthcare costs.

Conclusion: Patients with SCD with recurrent VOCs experience substantial clinical and economic burden driven by inpatient costs and frequent VOCs. There is a major unmet need for treatments that alleviate or eliminate clinical complications, including VOCs, and reduce healthcare costs in this patient population.

Keywords: Clinical decision-making; Costs and cost analysis; Healthcare costs; Healthcare economics and organizations; Medicaid; Sickle cell disease; Vaso-occlusive crisis.

Fig. 1

Patient attrition. ^aClaims must have occurred within 365 days of each other. Only non-diagnostic claims were included; non-diagnostic claims are medical claims in which diagnosis codes reflect confirmed diagnoses rather than suspected diagnoses reported to justify diagnostic tests or procedures. ^bVOC claims were considered discrete events if they occurred ≥ 3 days apart from each other. BMT bone marrow transplant; HSCT hematopoietic stem cell transplant; SCD sickle cell disease; VOC vaso-occlusive crisis

Fig. 2

a Annual healthcare costs for all patients with SCD with recurrent VOCs, the subgroup of patients who continued to experience ≥ 2 VOCs per year during the variable-length follow-up, and matched controls, as well as b payer-specific annual healthcare costs for all patients with SCD with recurrent VOCs and the subgroup of patients who continued to experience ≥ 2 VOCs per year during the variable-length follow-up. ^aOutpatient visits/encounters included emergency department, physician office, laboratory, and other outpatient visits/encounters. PPPY per patient per year; SCD sickle cell disease; VOC vaso-occlusive crisis

Fig. 3

Total annual healthcare costs by number of VOCs per year for patients with SCD with recurrent VOCs. SCD sickle cell disease; VOC vaso-occlusive crisis

Fig. 4

Lifetime healthcare costs for all patients with SCD with recurrent VOCs^a, the subgroup of patients who continued to experience ≥ 2 VOCs per year during the variable-length follow-up^b, and matched controls^c. ^aAnnual costs for patients with SCD with recurrent VOCs per age group were $24,697 (age 0–5 years [n = 503]), $33,499 (age 6–10 years [n = 473]), $54,715 (age 11–15 years [n = 504]), $81,875 (age 16–20 years [n = 657]), $95,082 (age 21–25 years [n = 436]), $83,612 (age 26–30 years [n = 247]), $98,964 (age 31–35 years [n = 208)], $82,573 (age 36–40 years [n = 143)], $105,580 (age 41–50 years [n = 152]), and $102,262 (age ≥ 51 years [n = 97]). ^bAnnual costs for patients in the ≥ 2 VOCs PPPY subgroup per age group were $34,191 (age 0–5 years [n = 234]), $48,581 (age 6–10 years [n = 252]), $68,295 (age 11–15 years [n = 341]), $99,588 (age 16 to 20 years [n = 506]), $104,578 (age 21 to 25 years [n = 367]), $101,813 (age 26–30 years [n = 194]), $121,490 (age 31–35 years [n = 152]), $98,856 (age 36–40 years [n = 102]), $135,580 (age 41–50 years [n = 103]), and $129,340 (age ≥ 51 years [n = 59]). ^cAnnual costs for matched controls per age group were $1423 (age 0–5 years [n = 2725]), $2334 (age 6–10 years [n = 2384]), $2947 (age 11–15 years [n = 2860]), $5517 (age 16–20 years [n = 2257]), $6040 (age 21–25 years [n = 2261]), $5523 [age 26–30 years [n = 1259]), $4351 (age 31–35 years [n = 1016]), $3984 (age 36–40 years [n = 405]), $7432 (age 41–50 years [n = 828]), and $8253 (age ≥ 51 years [n = 727]). K thousand; M million; SCD sickle cell disease; VOC vaso-occlusive crisis