Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia

Rgia A. Othman, Semone B. Myrie, David Mymin, Louise S. Merkens, Jean-Baptiste Roullet, Robert D. Steiner, Peter J H Jones

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Objective: To assess if ezetimibe (EZE), a sterol-absorption inhibitor, improves platelet (PLT) count and size relative to its effect on plasma plant sterol (PS) in patients with sitosterolemia (STSL). Study design: Patients with STSL (5 males, 3 females, 16-56 years of age) receiving EZE intervention as part of their routine care participated in this study. EZE was discontinued for 14 weeks (off) and then resumed for another 14 weeks (on). Hematology variables along with plasma and red blood cells (RBC) PS and total cholesterol (TC) levels were measured at the end of each phase. Results: EZE increased PLT count (23% ± 9%) and decreased mean PLT volume (MPV; 10% ± 3%, all P <.05). In patients off EZE, PLT counts inversely correlated (r = -0.96 and r = -0.91, all P <.01) with plasma and RBC PS to TC ratio (PS/TC), and MPV positively correlated (r = 0.91, P = .03 and r = 0.93, P = .02) with plasma and RBC PS/TC. EZE reduced plasma and RBC sitosterol (-35% ± 4% and -28% ± 3%), total PS (-37% ± 4% and -28% ± 3%, all P <.0001) levels, and PS/TC (-27% ± 4% and -28% ± 4%, P <.01). Conclusions: EZE reduces plasma and RBC PS levels, while increasing PLT count and decreasing MPV, and thereby may reduce the risk for bleeding in STSL. Plasma PS levels and ABCG5/ABCG8 genes should be analyzed in patients with unexplained hematologic abnormalities.

Original languageEnglish (US)
Pages (from-to)125-131
Number of pages7
JournalJournal of Pediatrics
Volume166
Issue number1
DOIs
StatePublished - Jan 1 2015

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Phytosterols
Platelet Count
Erythrocytes
Cholesterol
Mean Platelet Volume
Sitosterolemia
Ezetimibe
Sterols
Hematology
Hemorrhage

Keywords

  • ABC
  • Adenosine triphosphate-binding cassette
  • EZE
  • Ezetimibe
  • Hb
  • Hemoglobin
  • LDL
  • Low-density lipoprotein
  • MCHC
  • Mean corpuscular hemoglobin concentration
  • Mean PLT volume
  • MPV
  • Plant sterol
  • Platelet
  • PLT
  • PS
  • PS to TC ratio
  • PS/TC
  • RBC
  • RDW
  • Red blood cells
  • Red cell distribution width
  • Sitosterolemia
  • STSL
  • TC
  • Total cholesterol

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Othman, R. A., Myrie, S. B., Mymin, D., Merkens, L. S., Roullet, J-B., Steiner, R. D., & Jones, P. J. H. (2015). Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia. Journal of Pediatrics, 166(1), 125-131. https://doi.org/10.1016/j.jpeds.2014.08.069

Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia. / Othman, Rgia A.; Myrie, Semone B.; Mymin, David; Merkens, Louise S.; Roullet, Jean-Baptiste; Steiner, Robert D.; Jones, Peter J H.

In: Journal of Pediatrics, Vol. 166, No. 1, 01.01.2015, p. 125-131.

Research output: Contribution to journalArticle

Othman, RA, Myrie, SB, Mymin, D, Merkens, LS, Roullet, J-B, Steiner, RD & Jones, PJH 2015, 'Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia', Journal of Pediatrics, vol. 166, no. 1, pp. 125-131. https://doi.org/10.1016/j.jpeds.2014.08.069
Othman, Rgia A. ; Myrie, Semone B. ; Mymin, David ; Merkens, Louise S. ; Roullet, Jean-Baptiste ; Steiner, Robert D. ; Jones, Peter J H. / Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia. In: Journal of Pediatrics. 2015 ; Vol. 166, No. 1. pp. 125-131.
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abstract = "Objective: To assess if ezetimibe (EZE), a sterol-absorption inhibitor, improves platelet (PLT) count and size relative to its effect on plasma plant sterol (PS) in patients with sitosterolemia (STSL). Study design: Patients with STSL (5 males, 3 females, 16-56 years of age) receiving EZE intervention as part of their routine care participated in this study. EZE was discontinued for 14 weeks (off) and then resumed for another 14 weeks (on). Hematology variables along with plasma and red blood cells (RBC) PS and total cholesterol (TC) levels were measured at the end of each phase. Results: EZE increased PLT count (23{\%} ± 9{\%}) and decreased mean PLT volume (MPV; 10{\%} ± 3{\%}, all P <.05). In patients off EZE, PLT counts inversely correlated (r = -0.96 and r = -0.91, all P <.01) with plasma and RBC PS to TC ratio (PS/TC), and MPV positively correlated (r = 0.91, P = .03 and r = 0.93, P = .02) with plasma and RBC PS/TC. EZE reduced plasma and RBC sitosterol (-35{\%} ± 4{\%} and -28{\%} ± 3{\%}), total PS (-37{\%} ± 4{\%} and -28{\%} ± 3{\%}, all P <.0001) levels, and PS/TC (-27{\%} ± 4{\%} and -28{\%} ± 4{\%}, P <.01). Conclusions: EZE reduces plasma and RBC PS levels, while increasing PLT count and decreasing MPV, and thereby may reduce the risk for bleeding in STSL. Plasma PS levels and ABCG5/ABCG8 genes should be analyzed in patients with unexplained hematologic abnormalities.",
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AU - Othman, Rgia A.

AU - Myrie, Semone B.

AU - Mymin, David

AU - Merkens, Louise S.

AU - Roullet, Jean-Baptiste

AU - Steiner, Robert D.

AU - Jones, Peter J H

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N2 - Objective: To assess if ezetimibe (EZE), a sterol-absorption inhibitor, improves platelet (PLT) count and size relative to its effect on plasma plant sterol (PS) in patients with sitosterolemia (STSL). Study design: Patients with STSL (5 males, 3 females, 16-56 years of age) receiving EZE intervention as part of their routine care participated in this study. EZE was discontinued for 14 weeks (off) and then resumed for another 14 weeks (on). Hematology variables along with plasma and red blood cells (RBC) PS and total cholesterol (TC) levels were measured at the end of each phase. Results: EZE increased PLT count (23% ± 9%) and decreased mean PLT volume (MPV; 10% ± 3%, all P <.05). In patients off EZE, PLT counts inversely correlated (r = -0.96 and r = -0.91, all P <.01) with plasma and RBC PS to TC ratio (PS/TC), and MPV positively correlated (r = 0.91, P = .03 and r = 0.93, P = .02) with plasma and RBC PS/TC. EZE reduced plasma and RBC sitosterol (-35% ± 4% and -28% ± 3%), total PS (-37% ± 4% and -28% ± 3%, all P <.0001) levels, and PS/TC (-27% ± 4% and -28% ± 4%, P <.01). Conclusions: EZE reduces plasma and RBC PS levels, while increasing PLT count and decreasing MPV, and thereby may reduce the risk for bleeding in STSL. Plasma PS levels and ABCG5/ABCG8 genes should be analyzed in patients with unexplained hematologic abnormalities.

AB - Objective: To assess if ezetimibe (EZE), a sterol-absorption inhibitor, improves platelet (PLT) count and size relative to its effect on plasma plant sterol (PS) in patients with sitosterolemia (STSL). Study design: Patients with STSL (5 males, 3 females, 16-56 years of age) receiving EZE intervention as part of their routine care participated in this study. EZE was discontinued for 14 weeks (off) and then resumed for another 14 weeks (on). Hematology variables along with plasma and red blood cells (RBC) PS and total cholesterol (TC) levels were measured at the end of each phase. Results: EZE increased PLT count (23% ± 9%) and decreased mean PLT volume (MPV; 10% ± 3%, all P <.05). In patients off EZE, PLT counts inversely correlated (r = -0.96 and r = -0.91, all P <.01) with plasma and RBC PS to TC ratio (PS/TC), and MPV positively correlated (r = 0.91, P = .03 and r = 0.93, P = .02) with plasma and RBC PS/TC. EZE reduced plasma and RBC sitosterol (-35% ± 4% and -28% ± 3%), total PS (-37% ± 4% and -28% ± 3%, all P <.0001) levels, and PS/TC (-27% ± 4% and -28% ± 4%, P <.01). Conclusions: EZE reduces plasma and RBC PS levels, while increasing PLT count and decreasing MPV, and thereby may reduce the risk for bleeding in STSL. Plasma PS levels and ABCG5/ABCG8 genes should be analyzed in patients with unexplained hematologic abnormalities.

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KW - MPV

KW - Plant sterol

KW - Platelet

KW - PLT

KW - PS

KW - PS to TC ratio

KW - PS/TC

KW - RBC

KW - RDW

KW - Red blood cells

KW - Red cell distribution width

KW - Sitosterolemia

KW - STSL

KW - TC

KW - Total cholesterol

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