TY - JOUR
T1 - Magnetic resonance elastography identifies fibrosis in adults with alpha-1 antitrypsin deficiency liver disease
T2 - a prospective study
AU - Kim, R. G.
AU - Nguyen, P.
AU - Bettencourt, R.
AU - Dulai, P. S.
AU - Haufe, W.
AU - Hooker, J.
AU - Minocha, J.
AU - Valasek, M. A.
AU - Aryafar, H.
AU - Brenner, D. A.
AU - Sirlin, C. B.
AU - Loomba, R.
N1 - Funding Information:
Declaration of personal interests None. Declaration of funding interests: This study was supported in part by the Alpha-1 Foundation (A1F-SLU-7113 Alpha-1 Foundation; study chair: Jeffrey Teckman, MD). MR imaging and characterisation of normal and NAFLD groups was supported by the Intramural Research Program with co-funding from the NAFLD Research Center and Liver Imaging Group. The study was conducted at the Clinical and Translational Research Institute, University of California at San Diego. R.L. was supported in part by the American Gastroenterological Association Foundation-Sucampo-ASP Designated Research Award in Geriatric Gastroenterology (grant K23-DK090303) and by a T. Franklin Williams Scholarship Award. This study was also funded in part by Atlantic Philanthropies, the John A. Hartford Foundation, OM, the Association of Specialty Professors, and the American Gastroenterological Association.
Publisher Copyright:
© 2016 John Wiley & Sons Ltd
PY - 2016/8/1
Y1 - 2016/8/1
N2 - Background: Limited data exist on the clinical presentation and non-invasive detection of liver fibrosis in adults with homozygous Z genotype alpha-1 antitrypsin (AAT) deficiency. Aims: To compare demographic, biochemical, histological and imaging data of AAT deficient patients to normal-control and biopsy-proven non-alcoholic fatty liver disease (NAFLD) patients, and to assess the diagnostic accuracy of magnetic resonance elastography (MRE) in detecting fibrosis in AAT deficiency. Methods: Study includes 33 participants, 11 per group, who underwent clinical research evaluation, liver biopsy (AAT and NAFLD groups), and MRE. Histological fibrosis was quantified using a modified Ishak 6-point scale and liver stiffness by MRE. Diagnostic performance of MRE in detecting fibrosis was assessed by receiver operating characteristic (ROC) analysis. Results: Mean (±s.d.) of age and BMI of normal-control, AAT and NAFLD groups was 57 (±19), 57 (±18), and 57 (±13) years, and 22.7 (±2.5), 24.8 (±4.0) and 31.0 (±5.1) kg/m2 respectively. Serum ALT [mean ± s.d.] was similar within normal-control [16.4 ± 4.0] and AAT groups [23.5 ± 10.8], but was significantly lower in AAT than NAFLD even after adjustment for stage of fibrosis (P < 0.05, P = 0.0172). For fibrosis detection, MRE-estimated stiffness had an area under the ROC curve of 0.90 (P < 0.0001); an MRE threshold of ≥3.0 kPa provided 88.9% accuracy, with 80% sensitivity and 100% specificity to detect presence of any fibrosis (stage ≥1). Conclusions: This pilot prospective study suggests magnetic resonance elastography may be accurate for identifying fibrosis in patients with alpha-1 antitrypsin deficiency. Larger validation studies are warranted.
AB - Background: Limited data exist on the clinical presentation and non-invasive detection of liver fibrosis in adults with homozygous Z genotype alpha-1 antitrypsin (AAT) deficiency. Aims: To compare demographic, biochemical, histological and imaging data of AAT deficient patients to normal-control and biopsy-proven non-alcoholic fatty liver disease (NAFLD) patients, and to assess the diagnostic accuracy of magnetic resonance elastography (MRE) in detecting fibrosis in AAT deficiency. Methods: Study includes 33 participants, 11 per group, who underwent clinical research evaluation, liver biopsy (AAT and NAFLD groups), and MRE. Histological fibrosis was quantified using a modified Ishak 6-point scale and liver stiffness by MRE. Diagnostic performance of MRE in detecting fibrosis was assessed by receiver operating characteristic (ROC) analysis. Results: Mean (±s.d.) of age and BMI of normal-control, AAT and NAFLD groups was 57 (±19), 57 (±18), and 57 (±13) years, and 22.7 (±2.5), 24.8 (±4.0) and 31.0 (±5.1) kg/m2 respectively. Serum ALT [mean ± s.d.] was similar within normal-control [16.4 ± 4.0] and AAT groups [23.5 ± 10.8], but was significantly lower in AAT than NAFLD even after adjustment for stage of fibrosis (P < 0.05, P = 0.0172). For fibrosis detection, MRE-estimated stiffness had an area under the ROC curve of 0.90 (P < 0.0001); an MRE threshold of ≥3.0 kPa provided 88.9% accuracy, with 80% sensitivity and 100% specificity to detect presence of any fibrosis (stage ≥1). Conclusions: This pilot prospective study suggests magnetic resonance elastography may be accurate for identifying fibrosis in patients with alpha-1 antitrypsin deficiency. Larger validation studies are warranted.
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U2 - 10.1111/apt.13691
DO - 10.1111/apt.13691
M3 - Article
C2 - 27279429
AN - SCOPUS:84977576842
SN - 0269-2813
VL - 44
SP - 287
EP - 299
JO - Alimentary Pharmacology and Therapeutics
JF - Alimentary Pharmacology and Therapeutics
IS - 3
ER -