Crizanlizumab and comparators for adults with sickle cell disease: a systematic review and network meta-analysis.

Journal Article (Journal Article;Systematic Review)

OBJECTIVES: Treatment options for preventing vaso-occlusive crises (VOC) among patients with sickle cell disease (SCD) are limited, especially if hydroxyurea treatment has failed or is contraindicated. A systematic literature review (SLR) and network meta-analysis (NMA) were conducted to evaluate the efficacy and safety of crizanlizumab for older adolescent and adult (≥16 years old) SCD patients. METHODS: The SLR included randomised controlled trials (RCTs) and uncontrolled studies. Bayesian NMA of VOC, all-cause hospitalisation days and adverse events were conducted. RESULTS: The SLR identified 51 studies and 9 RCTs on 14 treatments that met the NMA inclusion criteria. The NMA found that crizanlizumab 5.0 mg/kg was associated with a reduction in VOC (HR 0.55, 95% credible interval (0.43, 0.69); Bayesian probability of superiority >0.99), all-cause hospitalisation days (0.58 (0.50, 0.68); >0.99) and no evidence of difference on adverse events (0.91 (0.59, 1.43) 0.66) or serious adverse events (0.93 (0.47, 1.87); 0.59) compared with placebo. The HR for reduction in VOC for crizanlizumab relative to L-glutamine was (0.67 (0.50, 0.88); >0.99). These results were sensitive to assumptions regarding whether patient age is an effect modifier. CONCLUSIONS: This NMA provides preliminary evidence comparing the efficacy of crizanlizumab with other treatments for VOC prevention.

Full Text

Duke Authors

Cited Authors

  • Thom, H; Jansen, J; Shafrin, J; Zhao, L; Joseph, G; Cheng, H-Y; Gupta, S; Shah, N

Published Date

  • September 17, 2020

Published In

Volume / Issue

  • 10 / 9

Start / End Page

  • e034147 -

PubMed ID

  • 32948541

Pubmed Central ID

  • PMC7500297

Electronic International Standard Serial Number (EISSN)

  • 2044-6055

Digital Object Identifier (DOI)

  • 10.1136/bmjopen-2019-034147


  • eng

Conference Location

  • England