RESULTS
Characteristics of included
trials
An initial search yielded 4687 articles, which were screened for
relevance, leading to the selection of 348 articles. Following a
rigorous selection process, 12 trials involving 392 HoFH patients were
included in the meta-analysis [22 – 33] (Figure
S1 ). Six RCTs used PCSK9i (n=152 patients) and in the other 6 RCTs the
treatment with ANGTPL3i was applied (n=240 patients). The
characteristics of included trials are presented in Table
1 . The mean age of the patients was 31.7 ± 13 and 48.4% were females.
55.2% of patients were with CVD disease when included in the trials.
The prevalence of White patients was significantly higher compared to
the Black, Asian or other races (75.4, 2.4, 8.49, 11%, p<0.01;
respectively).
When comparing the summary mean differences (SMD) of patients treated
with LLTs, patients age in the groups of PCSK9i and ANGPTL,3i trials
were similar (30.8±14.8 vs. 33.3±12.2, p=0.07). However, the prevalence
of female patients and the presence of CVD were significantly lower in
patients treated with PCSK9i compared to those treated with ANGPTL3i
(p<0.01 for both), (Table S3 ).
The mean baseline levels of TC [8.45 ± 3.2 mmol/L (326.7 ± 123.7
mg/dL) vs. 10.9 ± 3.9 mmol/L (421.5 ± 150.8 mg/dL); p <
0.001], LDL-C [7.17 ± 3.3 mmol/L (277.3 ± 127.6 mg/dL) vs. 9.64 ±
3.7 mmol/L (372.8 ± 143 mg/dL); p < 0.001], and Lp(a)
[34.9 ± 12.9 mg/dL vs. 60.1 ± 36 mg/dL; p < 0.001] were
significantly lower in the ANGPTL3i group compared to the PCSK9i group,
whereas HDL-C, TG, Apo-B, and Apo-A levels did not differ significantly
between the groups (p > 0.05 for all; Figure
S2 ).