Involvement of bile acid in diarrhoea and therapeutic effect of colestimide in hereditary ATTR amyloidosis
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View abstract on PubMed
Summary
This summary is machine-generated.Diarrhoea in hereditary transthyretin amyloidosis (ATTRv) is linked to bile acid levels. Bile acid sequestrants like colestimide may effectively reduce diarrhoea frequency in affected patients.
Area Of Science
- Gastroenterology
- Rare Diseases
- Metabolic Disorders
Background
- Diarrhoea is a significant complication in hereditary transthyretin amyloidosis (ATTRv), with an unknown pathomechanism.
- This study investigates the role of bile acids in ATTRv-associated diarrhoea.
- The therapeutic potential of colestimide, a bile acid sequestrant, was explored.
Purpose Of The Study
- To determine the association between bile acid levels and diarrhoea in ATTRv amyloidosis patients.
- To evaluate the efficacy of colestimide in managing diarrhoea in ATTRv amyloidosis.
Main Methods
- Prospective enrollment of 19 ATTRv patients (9 with diarrhoea, 10 without) and 20 healthy controls.
- Measurement of serum 7a-hydroxy-4-cholesten-3-one (C4) levels.
- Treatment of ATTRv patients with diarrhoea using oral colestimide (1.5g twice daily for 28 days) and evaluation of diarrhoea frequency and C4 levels pre- and post-treatment.
Main Results
- ATTRv patients with diarrhoea exhibited significantly higher mean serum C4 levels compared to those without diarrhoea (62.3 ng/mL vs. 24.0 ng/mL, p=0.03).
- Colestimide treatment led to a significant reduction in diarrhoea frequency (9.1 to 6.6 times/week, p=0.04).
- Colestimide administration significantly increased mean serum C4 levels (66.2 ng/mL pre-treatment to 187.1 ng/mL post-treatment, p=0.02).
Conclusions
- Bile acid status is significantly associated with diarrhoea in ATTRv amyloidosis.
- Colestimide and similar bile acid sequestrants show promise in reducing diarrhoea frequency in ATTRv patients.
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