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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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| Published in: | Clinical imaging 2020-07, Vol.63, p.1-6 |
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| creator | Degnan, Andrew J. Ho-Fung, Victor M. Wang, Dah-Jyuu Ficicioglu, Can Jaramillo, Diego |
| description | 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 |
| doi_str_mv | 10.1016/j.clinimag.2020.02.009 |
| format | article |
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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 < 0.001) when adjusted for age. Femoral neck fat fraction also correlated with lumbar spine fat fraction, liver volume and chitotriosidase (p < 0.05). MRS test-retest reliability was excellent (Pearson correlations: 0.96, 0.99; p-values <0.001). BMB inter-rater reliability was good overall with an intra-class correlation coefficient of 0.79 for total score, although lumbar spine score reliability was poor at 0.45.
MRS-derived bone marrow fat fractions appear capable of detecting Gaucher disease severity and monitoring treatment-related changes as a predictor of ERT duration in pediatric and young adult patients.
•Pediatric Gaucher disease patients had significantly lower MRS femoral and lumbar spine fat fractions compared to controls.•Femoral fat fractions corresponded with duration of treatment and reflected liver volume and chitotriosidase.•MRS may be an alternative to chemical shift imaging for Gaucher disease response to therapy, pending further validation.</description><identifier>ISSN: 0899-7071</identifier><identifier>EISSN: 1873-4499</identifier><identifier>DOI: 10.1016/j.clinimag.2020.02.009</identifier><identifier>PMID: 32120306</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>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</subject><ispartof>Clinical imaging, 2020-07, Vol.63, p.1-6</ispartof><rights>2020 Elsevier Inc.</rights><rights>Copyright © 2020 Elsevier Inc. All rights reserved.</rights><rights>2020. Elsevier Inc.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c396t-1a30baff0377be37ac148a9589b238b91fe6ee769722491b8f1d13168790742c3</citedby><cites>FETCH-LOGICAL-c396t-1a30baff0377be37ac148a9589b238b91fe6ee769722491b8f1d13168790742c3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,777,781,27905,27906</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32120306$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Degnan, Andrew J.</creatorcontrib><creatorcontrib>Ho-Fung, Victor M.</creatorcontrib><creatorcontrib>Wang, Dah-Jyuu</creatorcontrib><creatorcontrib>Ficicioglu, Can</creatorcontrib><creatorcontrib>Jaramillo, Diego</creatorcontrib><title>Gaucher disease status and treatment assessment: pilot study using magnetic resonance spectroscopy bone marrow fat fractions in pediatric patients</title><title>Clinical imaging</title><addtitle>Clin Imaging</addtitle><description>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 < 0.001) when adjusted for age. Femoral neck fat fraction also correlated with lumbar spine fat fraction, liver volume and chitotriosidase (p < 0.05). MRS test-retest reliability was excellent (Pearson correlations: 0.96, 0.99; p-values <0.001). BMB inter-rater reliability was good overall with an intra-class correlation coefficient of 0.79 for total score, although lumbar spine score reliability was poor at 0.45.
MRS-derived bone marrow fat fractions appear capable of detecting Gaucher disease severity and monitoring treatment-related changes as a predictor of ERT duration in pediatric and young adult patients.
•Pediatric Gaucher disease patients had significantly lower MRS femoral and lumbar spine fat fractions compared to controls.•Femoral fat fractions corresponded with duration of treatment and reflected liver volume and chitotriosidase.•MRS may be an alternative to chemical shift imaging for Gaucher disease response to therapy, pending further validation.</description><subject>Age</subject><subject>Bone imaging</subject><subject>Bone marrow</subject><subject>Bone marrow burden scoring</subject><subject>Bone marrow imaging</subject><subject>Control methods</subject><subject>Correlation analysis</subject><subject>Correlation coefficient</subject><subject>Correlation coefficients</subject><subject>Disease control</subject><subject>Enzyme replacement therapy</subject><subject>Enzymes</subject><subject>Fat fraction</subject><subject>Femur</subject><subject>Fractures</subject><subject>Gaucher disease</subject><subject>Gaucher's disease</subject><subject>Liver</subject><subject>Magnetic resonance imaging</subject><subject>Magnetic resonance spectroscopy</subject><subject>Medical imaging</subject><subject>Medical treatment</subject><subject>Patients</subject><subject>Pediatrics</subject><subject>Reliability</subject><subject>Resonance</subject><subject>Software</subject><subject>Spectroscopy</subject><subject>Spectrum analysis</subject><subject>Spine</subject><subject>Spine (lumbar)</subject><subject>Spleen</subject><subject>Studies</subject><subject>Work stations</subject><subject>Young adults</subject><issn>0899-7071</issn><issn>1873-4499</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><recordid>eNqFkUFv1DAQhSMEokvhL1SWuHBJGNtJHHMCVVCQKnGBs-U4k-JV1g4eh2r_Br8Yr7blwIWTffjem5n3quqKQ8OB92_3jVt88Ad71wgQ0IBoAPSTascHJeu21fpptYNB61qB4hfVC6I9FKFu1fPqQgouQEK_q37f2M39wMQmT2gJGWWbN2I2TCwntPmAITNLhESn7zu2-iXmgm3TkW3kwx0rSwTM3rGEFIMNrris6HKK5OJ6ZGMMWKCU4j2bbWZzsi77GIj5wFacvM2pqFebfZlAL6tns10IXz28l9X3Tx-_XX-ub7_efLn-cFs7qftccythtPMMUqkRpbKOt4PV3aBHIYdR8xl7RNVrJUSr-TjMfOKS94PSoFrh5GX15uy7pvhzQ8rm4MnhstiAcSMjpIIOhr6Dgr7-B93HLYWynRGt6Pqhg24oVH-mXLmcEs5mTaWhdDQczKk1szePrZlTawaEKa0V4dWD_TYecPore6ypAO_PAJY8fnlMhlzJypXsUgnaTNH_b8YfDhSuuQ</recordid><startdate>202007</startdate><enddate>202007</enddate><creator>Degnan, Andrew J.</creator><creator>Ho-Fung, Victor M.</creator><creator>Wang, Dah-Jyuu</creator><creator>Ficicioglu, Can</creator><creator>Jaramillo, Diego</creator><general>Elsevier Inc</general><general>Elsevier Limited</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>8FD</scope><scope>FR3</scope><scope>K9.</scope><scope>M7Z</scope><scope>P64</scope><scope>7X8</scope></search><sort><creationdate>202007</creationdate><title>Gaucher disease status and treatment assessment: pilot study using magnetic resonance spectroscopy bone marrow fat fractions in pediatric patients</title><author>Degnan, Andrew J. ; Ho-Fung, Victor M. ; Wang, Dah-Jyuu ; Ficicioglu, Can ; Jaramillo, Diego</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c396t-1a30baff0377be37ac148a9589b238b91fe6ee769722491b8f1d13168790742c3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>Age</topic><topic>Bone imaging</topic><topic>Bone marrow</topic><topic>Bone marrow burden scoring</topic><topic>Bone marrow imaging</topic><topic>Control methods</topic><topic>Correlation analysis</topic><topic>Correlation coefficient</topic><topic>Correlation coefficients</topic><topic>Disease control</topic><topic>Enzyme replacement therapy</topic><topic>Enzymes</topic><topic>Fat fraction</topic><topic>Femur</topic><topic>Fractures</topic><topic>Gaucher disease</topic><topic>Gaucher's disease</topic><topic>Liver</topic><topic>Magnetic resonance imaging</topic><topic>Magnetic resonance spectroscopy</topic><topic>Medical imaging</topic><topic>Medical treatment</topic><topic>Patients</topic><topic>Pediatrics</topic><topic>Reliability</topic><topic>Resonance</topic><topic>Software</topic><topic>Spectroscopy</topic><topic>Spectrum analysis</topic><topic>Spine</topic><topic>Spine (lumbar)</topic><topic>Spleen</topic><topic>Studies</topic><topic>Work stations</topic><topic>Young adults</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Degnan, Andrew J.</creatorcontrib><creatorcontrib>Ho-Fung, Victor M.</creatorcontrib><creatorcontrib>Wang, Dah-Jyuu</creatorcontrib><creatorcontrib>Ficicioglu, Can</creatorcontrib><creatorcontrib>Jaramillo, Diego</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Biochemistry Abstracts 1</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Clinical imaging</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Degnan, Andrew J.</au><au>Ho-Fung, Victor M.</au><au>Wang, Dah-Jyuu</au><au>Ficicioglu, Can</au><au>Jaramillo, Diego</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Gaucher disease status and treatment assessment: pilot study using magnetic resonance spectroscopy bone marrow fat fractions in pediatric patients</atitle><jtitle>Clinical imaging</jtitle><addtitle>Clin Imaging</addtitle><date>2020-07</date><risdate>2020</risdate><volume>63</volume><spage>1</spage><epage>6</epage><pages>1-6</pages><issn>0899-7071</issn><eissn>1873-4499</eissn><abstract>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 < 0.001) when adjusted for age. Femoral neck fat fraction also correlated with lumbar spine fat fraction, liver volume and chitotriosidase (p < 0.05). MRS test-retest reliability was excellent (Pearson correlations: 0.96, 0.99; p-values <0.001). BMB inter-rater reliability was good overall with an intra-class correlation coefficient of 0.79 for total score, although lumbar spine score reliability was poor at 0.45.
MRS-derived bone marrow fat fractions appear capable of detecting Gaucher disease severity and monitoring treatment-related changes as a predictor of ERT duration in pediatric and young adult patients.
•Pediatric Gaucher disease patients had significantly lower MRS femoral and lumbar spine fat fractions compared to controls.•Femoral fat fractions corresponded with duration of treatment and reflected liver volume and chitotriosidase.•MRS may be an alternative to chemical shift imaging for Gaucher disease response to therapy, pending further validation.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>32120306</pmid><doi>10.1016/j.clinimag.2020.02.009</doi><tpages>6</tpages></addata></record> |
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| 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 |
| title | Gaucher disease status and treatment assessment: pilot study using magnetic resonance spectroscopy bone marrow fat fractions in pediatric patients |
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