Correction to: Real-World Outcomes with Lomitapide Use in Paediatric Patients with Homozygous Familial Hypercholesterolaemia (Advances in Therapy, (2019), 36, 7, (1786-1811), 10.1007/s12325-019-00985-8)

In this article, the drug name in figure 2 is given as Atorvastatin 20 mg incorrectly. The correct Fig. 2 is given below. (Figure presented.) Evolution of LDL-C values in case 2 with lomitapide therapy. Upper panel shows mean interval LDL-C levels for patient 2. Middle panel shows lomitapide dose changes over time. Lower panel shows corresponding ALT (closed circles) and AST (open squares) levels over the same period. Dotted line on upper panel shows EAS targets for LDL-C levels in children with HoFH. Dotted line on lower panel indicates 3× upper limit of normal for LFTs; ALT alanine aminotransferase, AST aspartate aminotransferase, EAS European Atherosclerosis Society, HoFH homozygous familial hypercholesterolaemia, LDL-C low-density lipoprotein cholesterol, LFTs liver function tests, Q2W every 2 weeks, ULN upper limit of normal The Tables 1 and 2 consists few errors in published article. The correct Tables 1 and 2 are given below. (Table presented.) Individual data for the 11 patients Parameter Patient 1 2 3 4 5 6 7 8 9 10 11 Sex Female Male Male Male Female Male Female Male Male Female Male Age, years 13 12 16 7 11 16 4 14 15 11 9 Genetic variant LDLR c.313+5 G>A LDLR c.682G>T LDLR c.119_1207del LDLR c.666C>A, c.1646C>A c.-187-? 940+? Dup LDLR c.131G>A, c.2043C>A LDLR c.2043C>A LDLR c.1846-? 2311+?del, c.1895A>T LDLR c.313+1 G>A, del exon 1–6 LDLR c.1731G>T LDLR c.1731G>T LDL-C at diagnosis, mg/dL 799 672 981 1008 1009 901 739 474 982 1002 824 LLT prior to lomitapide Statins, ezetimibe, LA Statins, ezetimibe, LA Statins, ezetimibe, LA, EV Statins, ezetimibe, bile acid sequestrant Statins, ezetimibe Statins, ezetimibe, LA, EV Statins, ezetimibe Statins, ezetimibe Statins, ezetimibe, LA, EV Statins, ezetimibe, bile acid sequestrant Statins, ezetimibe, bile acid sequestrant Duration of therapy prior to lomitapide, years 11 2 14 3 8 6 < 1 6 11 8 8 LDL-C prior to lomitapide, mg/dL 299 326 187 833 443 274 649 223 81 630 705 LDL-C at nadir, mg/dL 56 98 73 360 231 23 236 75 62 441 460 Concomitant LLT Atv 40mg Ez 10mg LA Q2W Ro 20mg Ez 10mg LA Q15D Ro 20mg Ez 10mg Ev 420mg QW Co 3250mg LA Q1W Ro 20mg Ez 10mg Co 625mg Atv 10mg Ez 10mg Ro 20mg Ez 10mg LA Q2W Atv 10mg Ez 5mg Ro 30mg Ez 10mg Atv 40mg Ez 10mg LA 2xW Atv 40mg Ez 10mg Cholestyramine 4g Atv 40mg Ez 10mg Cholestyramine 4g Maximal reduction with lomitapide, % 81 70 61 57 48 92 64 66 24 27 34 Maximum dose of lomitapide, mg/day 20 40 60 30 20** 30 15 15*** 15 20 20 Length of lomitapide exposure, months 17 15 20 15 48 15 12 22 18 19 19 Change in concomitant LLT Ev stopped^§ LA stopped Ev stopped^§ LA reduced to Q4W Ev stopped^§ Ro stopped LA reduced to Q2W None Atv 40mg^† Atv 60mg^† Ev stopped^§ Ro 30mg Ro 40mg**** LA stopped None Ez stopped^§ LA stopped LA reduced 75% Ev stopped^§ None None Liver status Liver enzymes normal Liver enzymes normal Elevated liver enzymes resolved after Ro stopped Liver enzymes normal Liver enzymes normal Minimal ALT increase resolved without intervention Liver enzymes normal ALT increases managed with lomitapide dose reduction Liver enzymes and liver imaging normal Liver enzymes normal Liver enzymes normal Adverse events^¶ Nausea, vomiting, Diarrhoea, frequent bowel move-ments Diarrhoea, vomiting Flatulence, hypertransaminasaemia None Diarrhoea Gastrointestinal pain, Hypertransaminasaemia Diarrhoea Hypertransaminasaemia None None None AE adverse events, ALT alanine aminotransferase, At atorvastatin, Co colesevalem, Ev evolocumab (all Ev stopped prior to lomitapide), Ez ezetimibe, GI gastrointestinal, LA lipoprotein apheresis, LDL-C low-density lipoprotein cholesterol, LLT lipid-lowering therapies All oral drug doses are daily *Q2W **patient briefly received 30mg/day before back-titration to 20mg/day ***patient briefly received 20mg/day before back-titration to 15mg/day ****subsequent, post-hoc reduction to Ro 35mg ^†atorvastatin dose changes – Atv dose increased to 60mg near end of observation period ^§Patient had also received evolocumab (no response), which had been stopped before commencement on lomitapide ^¶MedDRA preferred term In the result section of abstract, text has been updated. The incorrect text is “In the 11 cases, mean baseline LDL-C was 419 ± 74.6 mg/dL and was markedly reduced by lomitapide to a nadir of 176.7 ± 46.3 mg/dL (58.4 ± 6.8% decrease). Six patients achieved recommended target levels for children below 135 mg/dL, five of whom had LA frequency reduced.” The correct text is “In the 11 cases, mean baseline LDL-C was 422 ± 245.4 mg/dL and was markedly reduced by lomitapide to a nadir of 192.2 ± 163.2 mg/dL (56.7 ± 21.7% decrease). Six patients achieved recommended target levels for children below 135 mg/dL, three of whom had LA frequency reduced and a further three stopped LA.” In the section of Summary of the Case Series, text has been updated. The incorrect text is “Table 2 provides summary descriptive statistics for all 11 patients. Baseline LDL-C was 419.9 ± 74.6 mg/dL. The mean at nadir was 176.7 ± 46.3 mg/dL, representing a 58.4 ± 6.8% reduction in LDL-C. Note that patients 9–11 had modest decreases in LDL-C levels (patient 9 was treated to reduce LA frequency, and patients 10 and 11 had compliance issues). These LDL-C reductions were achieved with a mean dose of lomitapide 24.5 ± 4.3 mg/day over a mean period of 20.0 ± 2.9 months.” (Table presented.) Summary data for the 11 patients Parameter Age Baseline LDL-C, mg/dL Nadir LDL-C, mg/dL Percentage reduction in LDL-C from baseline to nadir, % Lomitapide dose, mg/day Lomitapide exposure, months Mean 11.6 422.7 192.2 56.7 25.0 20.0 Median 12.0 325.5 98.0 61.2 20.0 18.2 SD 3.8 245.4 163.2 21.7 13.8 9.5 LDL-C low-density lipoprotein cholesterol, SD standard deviation The correct text is “Table 2 provides summary descriptive statistics for all 11 patients. Baseline LDL-C was 422.7 ± 245.4 mg/dL. The mean at nadir was 192.2 ± 163.2 mg/dL, representing a 56.7 ± 21.7% reduction in LDL-C. Note that patients 9–11 had modest decreases in LDL-C levels (patient 9 was treated to reduce LA frequency, and patients 10 and 11 had compliance issues). These LDL-C reductions were achieved with a mean dose of lomitapide 25.0 ± 13.8 mg/day over a mean period of 20.0 ± 2.9 months.”.