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Journal Article Research Support, Non-U.S. Gov't Systematic Review Epublish 35 citations

PubMed: 34727959 DOI: 10.1186/s13023-021-02096-6

A systematic literature review of frequency of vaso-occlusive crises in sickle cell disease.

Orphanet J Rare Dis 16(1):460

Authors: Ahmar U Zaidi, Alexander K Glaros, Soyon Lee, Taiji Wang, Rhea Bhojwani, Eric Morris, Breanne Donohue, Jincy Paulose, Şerban R Iorga, Dave Nellesen

Affiliations

  • Children's Hospital of Michigan, Detroit, MI, USA.
  • Central Michigan University, Mount Pleasant, MI, USA.
  • Novartis Pharmaceutical Corporation, East Hanover, NJ, USA.
  • Analysis Group, Inc. 1010 El Camino Real, Suite 310, Menlo Park, CA, 94025, USA.
  • Analysis Group, Inc. 1010 El Camino Real, Suite 310, Menlo Park, CA, 94025, USA.

Abstract

BACKGROUND AND PURPOSE

Sickle cell disease (SCD) is a collection of rare inherited blood disorders affecting approximately 100,000 people in the U.S. and 20-25 million people globally. Individuals with SCD experience recurrent episodes of severe and unpredictable pain that are caused by vaso-occlusive crises (VOCs), a hallmark of the disease. VOCs are the primary cause of hospitalization in SCD, result in missed workdays and school days, and decrease quality of life (QoL). Although VOCs cause significant burden in the lives of individuals with SCD, there is no synthesis on the frequency of VOCs in the real world. This systematic literature review sought to identify literature describing the frequency of VOCs experienced by individuals with SCD in real-world settings.

METHODS

MEDLINE and 6 congresses were searched (date range: January 1, 2000 to June 30, 2020). Studies were reviewed independently by two researchers. Studies assessing frequency or prevalence of VOCs or VOC-related outcomes were included.

RESULTS

Of 1438 studies identified in the search, 52 met pre-specified inclusion and exclusion criteria. Reported frequency of VOCs varied widely ranging from a mean or median of 0 VOCs/year to 18.2 VOCs/year. The proportion of patients experiencing ≥ 3 VOCs/year ranged from 4 to 67% and the proportion of patients experiencing ≥ 5 VOCs/year ranged from 18 to 59%. Measures of VOC severity were limited, with 13 studies considering frequency of complicated VOCs and only 1 study reporting duration of VOC episodes.

CONCLUSIONS

This is the first study to systematically assess published evidence pertaining to VOCs in real-world settings. Reported VOC frequency in real-world settings varied widely, with a majority of studies only considering VOCs managed in an inpatient or outpatient setting. Studies that considered VOCs managed at home reported a higher frequency of VOCs, suggesting that many studies may underestimate the frequency of VOCs. This systematic literature review (SLR) highlights the need for consistent reporting of (1) self-reported VOCs, including those managed at home, (2) definitions of VOCs, (3) complicated VOCs, and (4) duration of VOC episodes in literature.

MeSH

Cells, Anemia, Sickle Cell, Rare Diseases, Vaso-Occlusive Crises

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