PMID- 38420884
OWN - NLM
STAT- MEDLINE
DCOM- 20240328
LR  - 20240328
IS  - 1744-8298 (Electronic)
IS  - 1479-6678 (Linking)
VI  - 20
IP  - 2
DP  - 2024 Feb
TI  - Real-world safety and efficacy of lomitapide in homozygous familial 
      hypercholesterolemia: interim report of special-use survey in Japan.
PG  - 67-80
LID - 10.2217/fca-2023-0136 [doi]
AB  - Aim: To evaluate the safety and efficacy of lomitapide in real-world clinical 
      practice in Japan. Patients & methods: Interim analysis of 39 patients with 
      homozygous familial hypercholesterolemia from an all-case surveillance study. 
      Results: Median lomitapide dose (across 42 months) was 9.8 mg/day. 74 
      drug-related adverse events (AEs) were reported in 24 (61.5%) patients, including 
      14 (35.9%) with liver-related AEs, 19 (48.7%) with gastrointestinal disorders and 
      1 (2.6%) bleeding disorder. Lomitapide dose was reduced for 39.2% of drug-related 
      AEs, withdrawn temporarily for 12.2%, and discontinued for 1 event (1.4%). 
      Mean +/- SD blood LDL-C level decreased from 225.9 +/- 172.0 mg/dl (5.8 +/- 4.5 mmol/l) 
      predose to 159.4 +/- 93.0 mg/dl (4.1 +/- 2.4 mmol/l) at 12 months (p = 0.0245). 
      Conclusion: This interim analysis suggests lomitapide is safe and effective in 
      real-world clinical practice in Japan.
FAU - Harada-Shiba, Mariko
AU  - Harada-Shiba M
AUID- ORCID: 0000-0002-7600-2059
AD  - Cardiovascular Center, Osaka Medical & Pharmaceutical University, Osaka, 
      569-8686, Japan.
FAU - Haruna, Shigenori
AU  - Haruna S
AD  - Recordati Rare Diseases Japan K.K., Tokyo, 102-0082, Japan.
FAU - Kogawa, Noriaki
AU  - Kogawa N
AD  - Recordati Rare Diseases Japan K.K., Tokyo, 102-0082, Japan.
LA  - eng
GR  - Recordati Rare Diseases Japan K.K./
PT  - Journal Article
DEP - 20240229
PL  - England
TA  - Future Cardiol
JT  - Future cardiology
JID - 101239345
RN  - 0 (BMS201038)
RN  - 0 (Anticholesteremic Agents)
RN  - 0 (Benzimidazoles)
SB  - IM
MH  - Humans
MH  - *Hyperlipoproteinemia Type II
MH  - *Homozygous Familial Hypercholesterolemia
MH  - *Anticholesteremic Agents
MH  - Japan
MH  - *Benzimidazoles
OAB - What is this article about? Lomitapide is a drug used to treat homozygous 
      familial hypercholesterolemia (HoFH), a rare inherited disorder that causes very 
      high cholesterol levels. Because HoFH is rare, only a limited number of patients 
      were enrolled into the clinical trials that showed it was safe and effective, 
      before its approval. Therefore, a study is now underway to evaluate the efficacy 
      and safety of lomitapide when it is used in daily clinical practice in Japan. We 
      have analyzed data for 39 patients who have been enrolled in this study so far. 
      What are the results? We found that although most patients experienced some side 
      effects, only one patient had to discontinue lomitapide. Most side effects could 
      be managed without having to alter lomitapide treatment, or in some cases by 
      reducing the dose or stopping the drug temporarily. We also found that lomitapide 
      reduced cholesterol levels. What do the results mean? The results suggest that 
      lomitapide is generally safe and effective in patients with HoFH being treated in 
      routine clinical practice. The study is ongoing and additional analyses will be 
      performed when a greater number of patients have been treated.
OABL- eng
OTO - NOTNLM
OT  - Japan
OT  - efficacy
OT  - homozygous familial hypercholesterolemia
OT  - lomitapide
OT  - real-world
OT  - safety
EDAT- 2024/02/29 06:42
MHDA- 2024/03/28 06:45
CRDT- 2024/02/29 05:52
PHST- 2024/03/28 06:45 [medline]
PHST- 2024/02/29 06:42 [pubmed]
PHST- 2024/02/29 05:52 [entrez]
AID - 10.2217/fca-2023-0136 [doi]
PST - ppublish
SO  - Future Cardiol. 2024 Feb;20(2):67-80. doi: 10.2217/fca-2023-0136. Epub 2024 Feb 
      29.