吉维诺司他的用药指南

Givinostat is the first histone deacetylase inhibitor approved for the treatment of Duchenne muscular dystrophy (DMD). Its standardized use is crucial for therapeutic efficacy and safety. This article will systematically explain the key points of clinical application of this drug from three aspects: medication guide, side effect management, and medication for special groups.

Givenostat Medication Guide

Standard use requires strict pre-medication assessment and individualized dose adjustment.

Pre-medication assessment and testing

Platelet count and triacylglycerol level testing must be completed before treatment. Patients with platelet counts below 150 × 10⁹/L are prohibited from initiating treatment. Patients with underlying cardiac diseases or who are taking drugs that prolong the QT interval need to monitor their electrocardiogram when starting treatment, during combined use, and when clinically necessary.

Recommended dosage regimen

The dosage needs to be accurately adjusted according to the patient's weight and taken orally twice a day with meals: 22.2mg (2.5mL suspension) each time for patients from 10kg to <20kg; 20k g to <40kg: 31mg each time (3.5mL suspension); patients 40kg to <60kg: 44.3mg each time (5mL suspension); patients ≥60kg: 53.2mg each time (6mL suspension).

Method of administration

The drug should be taken with food to increase absorption rate. The suspension must be shaken thoroughly before use and measured accurately using a special dosing device. If you miss a dose, you can take it again if it is more than 6 hours before the next dose, otherwise skip the dose.

Strictly following the medication guidelines can maximize the efficacy of the medication while reducing the risk of adverse reactions.

Side effects of givenostat

The adverse reactions of givenostat are dose-dependent, and timely recognition and treatment are crucial to maintaining treatment continuity.

Abnormalities of the blood system

Thrombocytopenia often occurs in the early stages of treatment. When platelets are <100×10⁹/L, administration should be suspended until recovery to ≥150×10⁹/L.

Metabolic disorders

The incidence of hypertriglyceridemia is high. In severe cases (>500 mg/dL), administration must be suspended and lipid-lowering therapy initiated. Some patients may experience elevated cholesterol levels.

Digestive system reactions

Mainly include diarrhea, vomiting and abdominal pain. These symptoms usually appear during the dose adjustment period and can be gradually tolerated after 1-2 weeks.

Establishing a systematic side effect monitoring system will help early detection and intervention of adverse drug reactions.

Givenostat’s use in special populations

The use of givenostat in different populations requires consideration of their physiological characteristics and potential risks, and the implementation of individualized dosing plans.

Pediatric use

Currently only approved for use in patients 6 years of age and older. Safety and effectiveness in children under 6 years of age have not been established. Patients during the growth and development period need to regularly evaluate changes in height and weight.

Hepatic and renal insufficiency

No dose adjustment is required in patients with mild hepatic impairment (Child-Pugh class A). Use with caution in patients with moderate to severe hepatic insufficiency. Patients with renal insufficiency need to adjust the dose according to the creatinine clearance rate: reduce the dose by 25% for patients with 30-80mL/min, and disable it for patients with <30mL/min.

Elderly patients

Data on patients over 65 years old are limited. Due to decreased physiological function, it is recommended to reduce the starting dose and strengthen adverse reaction monitoring.

Developing individualized medication plans for special groups is an important guarantee for improving treatment safety.