Table 2 –
Lipid-lowering therapy at the time of the lowest on-treatment LDL-C level recorded, overall and stratified by country income status
| Overall | High-income countries | Non-high-income countries | |
|---|---|---|---|
| N=534 | N=293 | N=241 | |
| Medication | |||
| Statins | 491 (91∙9%) | 262 (89∙4%) | 229 (95∙0%) |
| Ezetimibe | 342 (64∙0%) | 212 (72∙4%) | 130 (53∙9%) |
| PCSK9 inhibitors | 118 (22∙1%) | 76 (25∙9%) | 42 (17∙4%) |
| Lomitapide | 45 (8∙4%) | 40 (13∙7%) | 5 (2∙1%) |
| Evinacumab* | 13 (2∙4%) | 13 (4∙4%) | 0 |
| Mipomersen | 5 (0∙9%) | 0 | 5 (2∙1%) |
| Bile acid sequestrants | 33 (6∙2%) | 31 (10∙6%) | 2 (0∙8%) |
| Fibrates | 6 (1∙1%) | 2 (0∙7%) | 4 (1∙7%) |
| Other** | 17 (3∙2%) | 9 (3∙1%) | 8 (3∙3%) |
| Lipoprotein apheresis† | 243/621 (39∙1%) | 118/293 (39∙7%) | 125/328 (38∙1%) |
| Surgeries | |||
| Liver transplantation | 5 (0∙8%) | 4 (1∙3%) | 1 (0∙3%) |
| Age at liver transplantation (years) | 19∙4 (10∙5–30∙0) | 10, 16, 24, 36 | 11 |
| Ileal bypass surgery†† | 1 (0∙2%) | 1 (0∙3%) | 0 |
| Age at Ileal bypass surgery (years) | 21 | 21 | NA |
| Portacaval shunt surgery†† | 6 (1∙1%) | 0 | 6 (2∙9%) |
| Age at Portacaval shunt surgery (years) | 9∙7 (5∙7–14∙2) | NA | 5, 5, 7, 11, 12, 18 |
Data are shown as n (%) for categorical variables, as bootstrapped mean (95%CI) for quantitative variables. Classification of high- and non-high-income countries is shown in Table S1. NA, not applicable; PCSK9, proprotein convertase subtilisin/kexin type 9
Evinacumab is an investigational product that has been recently approved by FDA but is not yet approved by other regulatory agencies. It was given as compassionate use and/or open label extension as part of a clinical trial
Other therapies were red yeast rice, omega-3 fish oils and plant stanols
Apheresis includes all lipoprotein apheresis types including plasma exchange. For 87 patients from non-high-income countries it was only known that they were on lipoprotein apheresis but no additional information was available on other lipid-lowering therapies. Patients from non-high-income countries who are on apheresis were mainly from Turkey (n=87) and Lebanon (n=26).
Ileal bypass and portacaval shunt surgery are no longer considered treatments for HoFH, these entries reflect (abandoned) historic practice.