Ruxolitinib exposure in patients with acute and chronic graft versus host disease in routine clinical practice-a prospective single-center trial

Cancer Chemother Pharmacol. 2021 Dec;88(6):973-983. doi: 10.1007/s00280-021-04351-w. Epub 2021 Sep 10.

Abstract

Purpose: Knowledge on Ruxolitinib exposure in patients with graft versus host disease (GvHD) is scarce. The purpose of this prospective study was to analyze Ruxolitinib concentrations of GvHD patients and to investigate effects of CYP3A4 and CYP2C9 inhibitors and other covariates as well as concentration-dependent effects.

Methods: 262 blood samples of 29 patients with acute or chronic GvHD who were administered Ruxolitinib during clinical routine were analyzed. A population pharmacokinetic model obtained from myelofibrosis patients was adapted to our population and was used to identify relevant pharmacokinetic properties and covariates on drug exposure. Relationships between Ruxolitinib exposure and adverse events were assessed.

Results: Median of individual mean trough serum concentrations was 39.9 ng/mL at 10 mg twice daily (IQR 27.1 ng/mL, range 5.6-99.8 ng/mL). Applying a population pharmacokinetic model revealed that concentrations in our cohort were significantly higher compared to myelofibrosis patients receiving the same daily dose (p < 0.001). Increased Ruxolitinib exposure was caused by a significant reduction in Ruxolitinib clearance by approximately 50%. Additional comedication with at least one strong CYP3A4 or CYP2C9 inhibitor led to a further reduction by 15% (p < 0.05). No other covariate affected pharmacokinetics significantly. Mean trough concentrations of patients requiring dose reduction related to adverse events were significantly elevated (p < 0.05).

Conclusion: Ruxolitinib exposure is increased in GvHD patients in comparison to myelofibrosis patients due to reduced clearance and comedication with CYP3A4 or CYP2C9 inhibitors. Elevated Ruxolitinib trough concentrations might be a surrogate for toxicity.

Keywords: CYP2C9; CYP3A4; Graft versus host disease; Ruxolitinib; Therapeutic drug monitoring; Toxicity.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Disease
  • Adult
  • Aged
  • Chronic Disease
  • Cytochrome P-450 CYP2C9 / chemistry
  • Cytochrome P-450 CYP2C9 Inhibitors / pharmacology*
  • Cytochrome P-450 CYP3A / chemistry
  • Cytochrome P-450 CYP3A Inhibitors / pharmacokinetics*
  • Drug Interactions*
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease / drug therapy*
  • Graft vs Host Disease / metabolism
  • Graft vs Host Disease / pathology
  • Humans
  • Male
  • Metabolic Clearance Rate
  • Middle Aged
  • Nitriles / administration & dosage
  • Nitriles / blood
  • Nitriles / pharmacokinetics*
  • Practice Patterns, Physicians' / statistics & numerical data*
  • Primary Myelofibrosis / drug therapy*
  • Primary Myelofibrosis / metabolism
  • Primary Myelofibrosis / pathology
  • Prognosis
  • Prospective Studies
  • Pyrazoles / administration & dosage
  • Pyrazoles / blood
  • Pyrazoles / pharmacokinetics*
  • Pyrimidines / administration & dosage
  • Pyrimidines / blood
  • Pyrimidines / pharmacokinetics*
  • Tissue Distribution
  • Young Adult

Substances

  • Cytochrome P-450 CYP2C9 Inhibitors
  • Cytochrome P-450 CYP3A Inhibitors
  • Nitriles
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib
  • CYP2C9 protein, human
  • Cytochrome P-450 CYP2C9
  • Cytochrome P-450 CYP3A
  • CYP3A4 protein, human