Gaucher disease status and treatment assessment: pilot study using magnetic resonance spectroscopy bone marrow fat fractions in pediatric patients

To assess magnetic resonance spectroscopy (MRS) bone marrow fat fractions' ability to discern between untreated Gaucher disease patients and healthy controls based on assessment of bone marrow infiltration and evaluate response to enzyme replacement therapy (ERT) on serial imaging. This retrosp...

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Bibliographic Details
Published in:Clinical imaging 2020-07, Vol.63, p.1-6
Main Authors: Degnan, Andrew J., Ho-Fung, Victor M., Wang, Dah-Jyuu, Ficicioglu, Can, Jaramillo, Diego
Format: Article
Language:English
Subjects:
Age
Bone imaging
Bone marrow
Bone marrow burden scoring
Bone marrow imaging
Control methods
Correlation analysis
Correlation coefficient
Correlation coefficients
Disease control
Enzyme replacement therapy
Enzymes
Fat fraction
Femur
Fractures
Gaucher disease
Gaucher's disease
Liver
Magnetic resonance imaging
Magnetic resonance spectroscopy
Medical imaging
Medical treatment
Patients
Pediatrics
Reliability
Resonance
Software
Spectroscopy
Spectrum analysis
Spine
Spine (lumbar)
Spleen
Studies
Work stations
Young adults
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Summary:To assess magnetic resonance spectroscopy (MRS) bone marrow fat fractions' ability to discern between untreated Gaucher disease patients and healthy controls based on assessment of bone marrow infiltration and evaluate response to enzyme replacement therapy (ERT) on serial imaging. This retrospective case-controlled study compared conventional MRI and bone marrow MRS findings in six pediatric and young adult Gaucher disease patients with age- and sex-matched controls, examining femoral neck and lumbar spine bone marrow fat fractions and bone marrow burden (BMB) scores. Separate analysis of six patients with serial imaging on ERT was performed with analysis of fat fractions, BMB scores, organ volumes, and serum chitotriosidase. Untreated patients had significantly lower femoral and lumbar spine fat fractions than controls (0.32 versus 0.67, p = 0.041 and 0.17 versus 0.34, p = 0.041, respectively). Total BMB scores were significantly higher in patients (8.0 versus 3.5, p = 0.015). In patients on ERT with average follow-up of 3.5 years, femoral neck fat fraction was the sole significant predictor of treatment duration (R square: 0.804, p 
ISSN:0899-7071
1873-4499
DOI:10.1016/j.clinimag.2020.02.009