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Background: LDL-cholesterol (LDL-C) treatment targets for patients with familial hypercholesterolemia (FH) is < 2.5 mmol/L, or < 1.8 mmol/L in high-risk-FH (presence of coronary heart disease, diabetes or late treatment start > 40 years of age). Conventional lipid-lowering therapy (LLT) includes statins, ezetemibe and resins, but even when these drugs are combined, treatment target might not be achieved. In the RUTHERFORD-2 study, by adding PCSK9 inhibitor to maximal tolerated dose of statins and ezetemibe, LDL-C was decreased incrementally 59 %, and 67 % of high-risk-FH patients reached a treatment target of LDL-C < 1.8 mmol/L.
Methods: Prospective registration of treatment data on 98 adult FH patients at a lipid-specialist clinic in Norway.
Results: All data is presented as mean (standard deviation) unless otherwise stated. Over all, on maximal tolerated dose of conventional LLT, pretreatment LDL-C was reduced 53 % from 7.0 (2.2) to 3.3 (1.1) mmol/L (P<0.001). Fifty-four of the patients were high-risk-FH. Only 34 % and 6 % of patients achieved the LDL-C treatment target of <2.5 mmol/L or <1.8 mmol/L in FH and high-risk-FH, respectively. Thirty-seven of the patients (number limited due to reimbursement policies) not achieving their individual treatment target, were initiated on PCSK9 inhibitor treatment. Except two patients reducing their dose of statin (one due to statin skepticism, and one due to misunderstanding), all patients also continued their maximal tolerated dose of conventional LLT. One patient stopped PCSK9 inhibitor due to an adverse event (myalgia without elevated creatinine kinase), but was included in the analyses as intention-to-treat principle. After addition of PCSK9 inhibitor, LDL-C was incrementally reduced 51 % from 3.5 (1.1) to 1.7 (1.3) mmol/L (P<0.001). Overall, LDL-C treatment target achievement was increased to 65 % (82 % in FH and 58 % high-risk-FH). Lp(a) was reduced from median 247 (range 25-3180) to median 184 (range 34-900) mg/L (P<0.002).
Conclusions: The incremental increase in treatment target achievement by addition of PCSK9 inhibitor shown in clinical studies was confirmed in a real-life setting.
Poster Hall, Hall C
Friday, March 17, 2017, 10:00 a.m.-10:45 a.m.
Session Title: Advances in Cholesterol Measurement and Management
Abstract Category: 32. Prevention: Clinical
Presentation Number: 1106-063
- 2017 American College of Cardiology Foundation