Longitudinal low density lipoprotein cholesterol goal achievement and cardiovascular outcomes among adult patients with familial hypercholesterolemia: The CASCADE FH registry

P. Barton Duell, Samuel S. Gidding, Rolf L. Andersen, Thomas Knickelbine, Lars Anderson, Eugenia Gianos, Peter Shrader, Iris Kindt, Emily C. O'Brien, Dervilla McCann, Linda C. Hemphill, Catherine D. Ahmed, Seth S. Martin, John A. Larry, Zahid S. Ahmad, Iftikhar J. Kullo, James A. Underberg, John Guyton, Paul Thompson, Katherine WilemonMatthew T. Roe, Daniel J. Rader, Marina Cuchel, MacRae F. Linton, Michael D. Shapiro, Patrick M. Moriarty, Joshua W. Knowles

Research output: Contribution to journalArticle

Abstract

Background and aims: There are limited data from the US on outcomes of patients in specialty care for familial hypercholesterolemia (FH). Methods: CASCADE FH Registry data were analyzed to assess longitudinal changes in medication usage, in low density lipoprotein cholesterol (LDL-C) levels, and the rate of major adverse cardiovascular events (MACE (myocardial infarction, coronary revascularization, stroke or transient ischemic attack) in adults with FH followed in US specialty clinics. Results: The cohort consisted of 1900 individuals (61% women, 87% Caucasian), with mean age of 56 ± 15 years, 37% prevalence of ASCVD at enrollment, mean pretreatment LDL-C 249 ± 68 mg/dl, mean enrollment LDL-C 145 mg/dl and 93% taking lipid lowering therapy. Over follow up of 20 ± 11 months, lipid lowering therapy use increased (mean decrease in LDL-C of 32 mg/dl (p < 0.001)). Only 48% of participants achieved LDL-C < 100 mg/dl and 22% achieved LDL-C < 70 mg/dl; ASCVD at enrollment was associated with greater likelihood of goal achievement. MACE event rates were almost 6 times higher among patients with prior ASCVD compared to those without (4.6 vs 0.8/100 patient years). Also associated with incident MACE were markers of FH severity and conventional ASCVD risk factors. Conclusions: With care in FH specialized clinics, LDL-C decreased, but LDL-C persisted >100 mg/dl in 52% of patients. High ASCVD event rates suggest that adults with FH warrant designation as having an ASCVD risk equivalent. Earlier and more aggressive therapy of FH is needed to prevent ASCVD events.

Original languageEnglish (US)
Pages (from-to)85-93
Number of pages9
JournalAtherosclerosis
Volume289
DOIs
StatePublished - Oct 2019

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Hyperlipoproteinemia Type II
LDL Cholesterol
Registries
Lipids
Transient Ischemic Attack
Therapeutics
Stroke
Myocardial Infarction

Keywords

  • Cardiovascular disease
  • Familial hypercholesterolemia
  • LDL cholesterol goal achievement
  • Low density lipoprotein (LDL) cholesterol
  • PCSK9 inhibitor

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Longitudinal low density lipoprotein cholesterol goal achievement and cardiovascular outcomes among adult patients with familial hypercholesterolemia : The CASCADE FH registry. / Duell, P. Barton; Gidding, Samuel S.; Andersen, Rolf L.; Knickelbine, Thomas; Anderson, Lars; Gianos, Eugenia; Shrader, Peter; Kindt, Iris; O'Brien, Emily C.; McCann, Dervilla; Hemphill, Linda C.; Ahmed, Catherine D.; Martin, Seth S.; Larry, John A.; Ahmad, Zahid S.; Kullo, Iftikhar J.; Underberg, James A.; Guyton, John; Thompson, Paul; Wilemon, Katherine; Roe, Matthew T.; Rader, Daniel J.; Cuchel, Marina; Linton, MacRae F.; Shapiro, Michael D.; Moriarty, Patrick M.; Knowles, Joshua W.

In: Atherosclerosis, Vol. 289, 10.2019, p. 85-93.

Research output: Contribution to journalArticle

Duell, PB, Gidding, SS, Andersen, RL, Knickelbine, T, Anderson, L, Gianos, E, Shrader, P, Kindt, I, O'Brien, EC, McCann, D, Hemphill, LC, Ahmed, CD, Martin, SS, Larry, JA, Ahmad, ZS, Kullo, IJ, Underberg, JA, Guyton, J, Thompson, P, Wilemon, K, Roe, MT, Rader, DJ, Cuchel, M, Linton, MF, Shapiro, MD, Moriarty, PM & Knowles, JW 2019, 'Longitudinal low density lipoprotein cholesterol goal achievement and cardiovascular outcomes among adult patients with familial hypercholesterolemia: The CASCADE FH registry', Atherosclerosis, vol. 289, pp. 85-93. https://doi.org/10.1016/j.atherosclerosis.2019.08.007
Duell, P. Barton ; Gidding, Samuel S. ; Andersen, Rolf L. ; Knickelbine, Thomas ; Anderson, Lars ; Gianos, Eugenia ; Shrader, Peter ; Kindt, Iris ; O'Brien, Emily C. ; McCann, Dervilla ; Hemphill, Linda C. ; Ahmed, Catherine D. ; Martin, Seth S. ; Larry, John A. ; Ahmad, Zahid S. ; Kullo, Iftikhar J. ; Underberg, James A. ; Guyton, John ; Thompson, Paul ; Wilemon, Katherine ; Roe, Matthew T. ; Rader, Daniel J. ; Cuchel, Marina ; Linton, MacRae F. ; Shapiro, Michael D. ; Moriarty, Patrick M. ; Knowles, Joshua W. / Longitudinal low density lipoprotein cholesterol goal achievement and cardiovascular outcomes among adult patients with familial hypercholesterolemia : The CASCADE FH registry. In: Atherosclerosis. 2019 ; Vol. 289. pp. 85-93.
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abstract = "Background and aims: There are limited data from the US on outcomes of patients in specialty care for familial hypercholesterolemia (FH). Methods: CASCADE FH Registry data were analyzed to assess longitudinal changes in medication usage, in low density lipoprotein cholesterol (LDL-C) levels, and the rate of major adverse cardiovascular events (MACE (myocardial infarction, coronary revascularization, stroke or transient ischemic attack) in adults with FH followed in US specialty clinics. Results: The cohort consisted of 1900 individuals (61{\%} women, 87{\%} Caucasian), with mean age of 56 ± 15 years, 37{\%} prevalence of ASCVD at enrollment, mean pretreatment LDL-C 249 ± 68 mg/dl, mean enrollment LDL-C 145 mg/dl and 93{\%} taking lipid lowering therapy. Over follow up of 20 ± 11 months, lipid lowering therapy use increased (mean decrease in LDL-C of 32 mg/dl (p < 0.001)). Only 48{\%} of participants achieved LDL-C < 100 mg/dl and 22{\%} achieved LDL-C < 70 mg/dl; ASCVD at enrollment was associated with greater likelihood of goal achievement. MACE event rates were almost 6 times higher among patients with prior ASCVD compared to those without (4.6 vs 0.8/100 patient years). Also associated with incident MACE were markers of FH severity and conventional ASCVD risk factors. Conclusions: With care in FH specialized clinics, LDL-C decreased, but LDL-C persisted >100 mg/dl in 52{\%} of patients. High ASCVD event rates suggest that adults with FH warrant designation as having an ASCVD risk equivalent. Earlier and more aggressive therapy of FH is needed to prevent ASCVD events.",
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T1 - Longitudinal low density lipoprotein cholesterol goal achievement and cardiovascular outcomes among adult patients with familial hypercholesterolemia

T2 - The CASCADE FH registry

AU - Duell, P. Barton

AU - Gidding, Samuel S.

AU - Andersen, Rolf L.

AU - Knickelbine, Thomas

AU - Anderson, Lars

AU - Gianos, Eugenia

AU - Shrader, Peter

AU - Kindt, Iris

AU - O'Brien, Emily C.

AU - McCann, Dervilla

AU - Hemphill, Linda C.

AU - Ahmed, Catherine D.

AU - Martin, Seth S.

AU - Larry, John A.

AU - Ahmad, Zahid S.

AU - Kullo, Iftikhar J.

AU - Underberg, James A.

AU - Guyton, John

AU - Thompson, Paul

AU - Wilemon, Katherine

AU - Roe, Matthew T.

AU - Rader, Daniel J.

AU - Cuchel, Marina

AU - Linton, MacRae F.

AU - Shapiro, Michael D.

AU - Moriarty, Patrick M.

AU - Knowles, Joshua W.

PY - 2019/10

Y1 - 2019/10

N2 - Background and aims: There are limited data from the US on outcomes of patients in specialty care for familial hypercholesterolemia (FH). Methods: CASCADE FH Registry data were analyzed to assess longitudinal changes in medication usage, in low density lipoprotein cholesterol (LDL-C) levels, and the rate of major adverse cardiovascular events (MACE (myocardial infarction, coronary revascularization, stroke or transient ischemic attack) in adults with FH followed in US specialty clinics. Results: The cohort consisted of 1900 individuals (61% women, 87% Caucasian), with mean age of 56 ± 15 years, 37% prevalence of ASCVD at enrollment, mean pretreatment LDL-C 249 ± 68 mg/dl, mean enrollment LDL-C 145 mg/dl and 93% taking lipid lowering therapy. Over follow up of 20 ± 11 months, lipid lowering therapy use increased (mean decrease in LDL-C of 32 mg/dl (p < 0.001)). Only 48% of participants achieved LDL-C < 100 mg/dl and 22% achieved LDL-C < 70 mg/dl; ASCVD at enrollment was associated with greater likelihood of goal achievement. MACE event rates were almost 6 times higher among patients with prior ASCVD compared to those without (4.6 vs 0.8/100 patient years). Also associated with incident MACE were markers of FH severity and conventional ASCVD risk factors. Conclusions: With care in FH specialized clinics, LDL-C decreased, but LDL-C persisted >100 mg/dl in 52% of patients. High ASCVD event rates suggest that adults with FH warrant designation as having an ASCVD risk equivalent. Earlier and more aggressive therapy of FH is needed to prevent ASCVD events.

AB - Background and aims: There are limited data from the US on outcomes of patients in specialty care for familial hypercholesterolemia (FH). Methods: CASCADE FH Registry data were analyzed to assess longitudinal changes in medication usage, in low density lipoprotein cholesterol (LDL-C) levels, and the rate of major adverse cardiovascular events (MACE (myocardial infarction, coronary revascularization, stroke or transient ischemic attack) in adults with FH followed in US specialty clinics. Results: The cohort consisted of 1900 individuals (61% women, 87% Caucasian), with mean age of 56 ± 15 years, 37% prevalence of ASCVD at enrollment, mean pretreatment LDL-C 249 ± 68 mg/dl, mean enrollment LDL-C 145 mg/dl and 93% taking lipid lowering therapy. Over follow up of 20 ± 11 months, lipid lowering therapy use increased (mean decrease in LDL-C of 32 mg/dl (p < 0.001)). Only 48% of participants achieved LDL-C < 100 mg/dl and 22% achieved LDL-C < 70 mg/dl; ASCVD at enrollment was associated with greater likelihood of goal achievement. MACE event rates were almost 6 times higher among patients with prior ASCVD compared to those without (4.6 vs 0.8/100 patient years). Also associated with incident MACE were markers of FH severity and conventional ASCVD risk factors. Conclusions: With care in FH specialized clinics, LDL-C decreased, but LDL-C persisted >100 mg/dl in 52% of patients. High ASCVD event rates suggest that adults with FH warrant designation as having an ASCVD risk equivalent. Earlier and more aggressive therapy of FH is needed to prevent ASCVD events.

KW - Cardiovascular disease

KW - Familial hypercholesterolemia

KW - LDL cholesterol goal achievement

KW - Low density lipoprotein (LDL) cholesterol

KW - PCSK9 inhibitor

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