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Tacrolimus as a Promising Drug for Epistaxis and Gastrointestinal Bleeding in HHT. | LitMetric

Tacrolimus as a Promising Drug for Epistaxis and Gastrointestinal Bleeding in HHT.

J Clin Med

CIBER Rare Diseases Unit 707, Centro de Investigaciones Biológicas Margarita Salas, Consejo Superior Investigaciones Científicas, CSIC, 28040 Madrid, Spain.

Published: November 2023

AI Article Synopsis

Article Abstract

Background: Hereditary Hemorrhagic Telangiectasia (HHT) is a vascular autosomically inherited rare disease. Epistaxis (nose bleeds) is the most common symptom in HHT, leading to anemia and affecting the patient's quality of life. In addition to epistaxis, gastrointestinal bleeding (GI), more often at older ages, may lead to severe anemia and the need for blood transfusions. Thus, finding drugs to control both types of bleeding is a primary necessity in HHT.

Methods: A cross-sectional observational study was conducted in a series of 11 HHT patients treated with low tacrolimus doses (0.5-2 mg/day) on an off-label prescription basis. Patients showed refractory bleeding to previous treatments. The epistaxis severity score (ESS) and hemoglobin levels were the parameters used to evaluate the impact of tacrolimus. The occurrence of side effects was also recorded.

Results: Tacrolimus was well tolerated in all of the patients except 2 (who stopped the treatment). The remaining patients tolerated the treatment, with a general improvement in their health condition. Epistaxis was significantly reduced when comparing the ESS before and after the treatment. Hemoglobin levels significantly increased, overcoming the anemia, during the course of the treatment.

Conclusion: Tacrolimus at low doses should be considered as a promising treatment for epistaxis and gastrointestinal bleeding in HHT.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10706934PMC
http://dx.doi.org/10.3390/jcm12237410DOI Listing

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