Residual burden of liver disease after HCV clearance in hemophilia: a word of caution in the era of gene therapy

Author:

La Mura Vincenzo12ORCID,Bitto Niccolò1ORCID,Capelli Cecilia1ORCID,Caputo Camilla1ORCID,Siboni Simona1,Arcudi Sara1ORCID,Ciavarella Alessandro1,Gualtierotti Roberta12ORCID,Fracanzani Anna Ludovica23ORCID,Sangiovanni Angelo4ORCID,Peyvandi Flora12ORCID

Affiliation:

1. 1Foundation Istituto di Ricovero e Cura a Carattere Scientifico Ca’ Granda, Ospedale Maggiore Policlinico-Angelo Bianchi Bonomi and Thrombosis Center, Milan, Italy

2. 2Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

3. 3Unit of Internal Medicine and Metabolic Diseases, Foundation Istituto di Ricovero e Cura a Carattere Scientifico Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy

4. 4Gastroenterology and Hepatology, Foundation Istituto di Ricovero e Cura a Carattere Scientifico Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy

Abstract

Abstract Ruling out advanced fibrosis/cirrhosis is mandatory for persons with hemophilia (PWH) who are candidates for gene therapy. However, clinical evaluation and noninvasive tests (NITs) may be inaccurate after hepatitis C virus (HCV) clearance. We conducted a prospective hepatological screening to detect advanced fibrosis/cirrhosis in PWH after HCV clearance. Any risk factor of chronic liver damage was registered by using biochemical data, liver stiffness measurement (LSM), and ultrasound (US). A pre/post-HCV clearance analysis was conducted prospectively in a subgroup of patients who underwent LSM, US, and NITs for fibrosis. We evaluated 119 patients (median age, 53 years; range, 36-87 years) with a previous HCV infection (hemophilia A, n = 108; hemophilia B, n = 11). Ninety-six (81%) presented at least 1 potential risk factor of chronic liver damage. Metabolic risk factors were the most prevalent, with 51 patients (44%) having US steatosis. In 21 patients (18%), clinical, biochemical, liver morphology, and/or LSM were suggestive of advanced fibrosis/cirrhosis. Furthermore, 10 patients (8%) had esophageal varices and 3 (3%) had hepatocellular carcinoma. In 57 patients included in the prospective analysis, LSM and NITs were reduced after HCV clearance (P < .05), but US signs specific of cirrhosis remained unchanged. Overall, 23 of 80 patients (29%) with LSM <10 KPa had at least 1 US sign suggestive of advanced fibrosis/cirrhosis. A similar proportion (18%) was observed for LSM <8 KPa. Overall, risk factors of chronic liver damage are frequent after HCV clearance, but changes in LSM and NITs after clearance may be inaccurate to rule out advanced fibrosis/cirrhosis. A specific diagnostic workup is warranted to evaluate liver health in PWH in the era of gene therapy.

Publisher

American Society of Hematology

Subject

Hematology

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