Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2020 Mar 24;4(6):1159-1165.
doi: 10.1182/bloodadvances.2019001165.

Phase 2 trial of montelukast for prevention of pain in sickle cell disease

Joshua J Field  1   2 , Adetola Kassim  3 , Amanda Brandow  4 , Stephen H Embury  5 , Neil Matsui  5 , Karina Wilkerson  3 , Valencia Bryant  6 , Liyun Zhang  4 , Pippa Simpson  4 , Michael R DeBaun  3   6
Affiliations
Clinical Trial

Phase 2 trial of montelukast for prevention of pain in sickle cell disease

Joshua J Field et al. Blood Adv. .

Abstract

Cysteinyl leukotrienes (CysLTs) are lipid mediators of inflammation. In patients with sickle cell disease (SCD), levels of CysLTs are increased compared with controls and associated with a higher rate of hospitalization for pain. We tested the hypothesis that administration of the CysLT receptor antagonist montelukast would improve SCD-related comorbidities, including pain, in adolescents and adults with SCD. In a phase 2 randomized trial, we administered montelukast or placebo for 8 weeks. The primary outcome measure was a >30% reduction in soluble vascular cell adhesion molecule 1 (sVCAM), a marker of vascular injury. Secondary outcome measures were reduction in daily pain, improvement in pulmonary function, and improvement in microvascular blood flow, as measured by laser Doppler velocimetry. Forty-two participants with SCD were randomized to receive montelukast or placebo for 8 weeks. We found no difference between the montelukast and placebo groups with regard to the levels of sVCAM, reported pain, pulmonary function, or microvascular blood flow. Although montelukast is an effective treatment for asthma, we did not find benefit for SCD-related outcomes. This clinical trial was registered at www.clinicaltrials.gov as #NCT01960413.

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: J.J.F. conducts research studies with Cyclerion Therapeutics and Rigel Pharmaceuticals and has been a paid consultant for Ironwood Pharmaceuticals. S.H.E. and N.M. are employees of and stockholders in Vanguard Therapeutics, Inc. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
See this image and copyright information in PMC

References

    1. Zhang D, Xu C, Manwani D, Frenette PS. Neutrophils, platelets, and inflammatory pathways at the nexus of sickle cell disease pathophysiology. Blood. 2016;127(7):801-809. - PMC - PubMed
    1. Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical reappraisal. Blood. 2012;120(18):3647-3656. - PubMed
    1. Glassberg J, Minnitti C, Cromwell C, et al. Inhaled steroids reduce pain and sVCAM levels in individuals with sickle cell disease: a triple-blind, randomized trial. Am J Hematol. 2017;92(7):622-631. - PMC - PubMed
    1. Field JJ, DeBaun MR. Asthma and sickle cell disease: two distinct diseases or part of the same process? Hematology Am Soc Hematol Educ Program. 2009;2009;45-53. - PubMed
    1. Knight-Perry J, DeBaun MR, Strunk RC, Field JJ. Leukotriene pathway in sickle cell disease: a potential target for directed therapy. Expert Rev Hematol. 2009;2(1):57-68. - PubMed

Publication types

Associated data