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<p>Sickle cell disease (SCD) care includes pharmacologic disease-modifying therapies (pDMTs) and chronic blood transfusion therapy (CBT). Using Optum Labs Data Warehouse (2014–2021), we identified individuals with SCD and classified yearly treatment as pDMT only, CBT only, combined pDMT + CBT, or no disease-modifying therapy. Among 4,100 patients, 26% used pDMT only, 3% received CBT only, 1% received both, and 70% received no DMT in a given year. pDMT uptake rose steadily while CBT remained low (3%–4%). In multinomial models (reference: no DMT), older age was associated with lower odds of pDMT and CBT, while prior vaso-occlusive crises and severe organ complications were associated with higher odds across categories. Men had higher odds of pDMT and combined therapy. These findings suggest treatment intensification in routine practice follows accumulating morbidity rather than preceding it, and that substantial implementation gaps persist, particularly among older adults and for CBT, which depends on specialized capacity. Expanding CBT capacity and improving pDMT uptake may narrow gaps and better align real-world care with guideline intent.</p>

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Real-world use of pharmacologic therapy and chronic blood transfusion in sickle cell disease, 2014–2021

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月2日

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  • Kangho Suh,
  • Terri V. Newman,
  • Jingye Yang,
  • Aaron Haubner,
  • Melissa Anderson,
  • Darrell J. Triulzi,
  • Enrico M. Novelli

摘要

Sickle cell disease (SCD) care includes pharmacologic disease-modifying therapies (pDMTs) and chronic blood transfusion therapy (CBT). Using Optum Labs Data Warehouse (2014–2021), we identified individuals with SCD and classified yearly treatment as pDMT only, CBT only, combined pDMT + CBT, or no disease-modifying therapy. Among 4,100 patients, 26% used pDMT only, 3% received CBT only, 1% received both, and 70% received no DMT in a given year. pDMT uptake rose steadily while CBT remained low (3%–4%). In multinomial models (reference: no DMT), older age was associated with lower odds of pDMT and CBT, while prior vaso-occlusive crises and severe organ complications were associated with higher odds across categories. Men had higher odds of pDMT and combined therapy. These findings suggest treatment intensification in routine practice follows accumulating morbidity rather than preceding it, and that substantial implementation gaps persist, particularly among older adults and for CBT, which depends on specialized capacity. Expanding CBT capacity and improving pDMT uptake may narrow gaps and better align real-world care with guideline intent.