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A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis.

Publication ,  Journal Article
Dampier, CD; Telen, MJ; Wun, T; Brown, RC; Desai, P; El Rassi, F; Fuh, B; Kanter, J; Pastore, Y; Rothman, J; Taylor, JG; Readett, D ...
Published in: Blood
January 12, 2023

The efficacy and safety of rivipansel, a predominantly E-selectin antagonist, were studied in a phase 3, randomized, controlled trial for vaso-occlusive crisis (VOC) requiring hospitalization (RESET). A total of 345 subjects (204 adults and 141 children) were randomized and 320 were treated (162 with rivipansel, 158 with placebo) with an IV loading dose, followed by up to 14 additional 12-hourly maintenance doses of rivipansel or placebo, in addition to standard care. Rivipansel was similarly administered during subsequent VOCs in the Open-label Extension (OLE) study. In the full analysis population, the median time to readiness for discharge (TTRFD), the primary end point, was not different between rivipansel and placebo (-5.7 hours, P = .79; hazard ratio, 0.97), nor were differences seen in secondary end points of time to discharge (TTD), time to discontinuation of IV opioids (TTDIVO), and cumulative IV opioid use. Mean soluble E-selectin decreased 61% from baseline after the loading dose in the rivipansel group, while remaining unchanged in the placebo group. In a post hoc analysis, early rivipansel treatment within 26.4 hours of VOC pain onset (earliest quartile of time from VOC onset to treatment) reduced median TTRFD by 56.3 hours, reduced median TTD by 41.5 hours, and reduced median TTDIVO by 50.5 hours, compared with placebo (all P < .05). A similar subgroup analysis comparing OLE early-treatment with early-treatment RESET placebo showed a reduction in TTD of 23.1 hours (P = .062) and in TTDIVO of 30.1 hours (P = .087). Timing of rivipansel administration after pain onset may be critical to achieving accelerated resolution of acute VOC. Trial Registration: Clinicaltrials.gov, NCT02187003 (RESET), NCT02433158 (OLE).

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Published In

Blood

DOI

EISSN

1528-0020

Publication Date

January 12, 2023

Volume

141

Issue

2

Start / End Page

168 / 179

Location

United States

Related Subject Headings

  • Volatile Organic Compounds
  • Pain
  • Immunology
  • Humans
  • Hemoglobinopathies
  • E-Selectin
  • Double-Blind Method
  • Child
  • Anemia, Sickle Cell
  • Analgesics, Opioid
 

Citation

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Dampier, C. D., Telen, M. J., Wun, T., Brown, R. C., Desai, P., El Rassi, F., … RESET Investigators, . (2023). A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood, 141(2), 168–179. https://doi.org/10.1182/blood.2022015797
Dampier, Carlton D., Marilyn Jo Telen, Ted Wun, R Clark Brown, Payal Desai, Fuad El Rassi, Beng Fuh, et al. “A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis.Blood 141, no. 2 (January 12, 2023): 168–79. https://doi.org/10.1182/blood.2022015797.
Dampier CD, Telen MJ, Wun T, Brown RC, Desai P, El Rassi F, et al. A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood. 2023 Jan 12;141(2):168–79.
Dampier, Carlton D., et al. “A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis.Blood, vol. 141, no. 2, Jan. 2023, pp. 168–79. Pubmed, doi:10.1182/blood.2022015797.
Dampier CD, Telen MJ, Wun T, Brown RC, Desai P, El Rassi F, Fuh B, Kanter J, Pastore Y, Rothman J, Taylor JG, Readett D, Sivamurthy KM, Tammara B, Tseng L-J, Lozier JN, Thackray H, Magnani JL, Hassell KL, RESET Investigators. A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood. 2023 Jan 12;141(2):168–179.

Published In

Blood

DOI

EISSN

1528-0020

Publication Date

January 12, 2023

Volume

141

Issue

2

Start / End Page

168 / 179

Location

United States

Related Subject Headings

  • Volatile Organic Compounds
  • Pain
  • Immunology
  • Humans
  • Hemoglobinopathies
  • E-Selectin
  • Double-Blind Method
  • Child
  • Anemia, Sickle Cell
  • Analgesics, Opioid