Abstract #5421

Quantitative 1H Single Voxel Spectroscopy In Metachromatic Leukodystrophy Patients Treated With Haematopoietic Stem Cell Gene Therapy: Preliminary Results

Paola Scifo¹, Pasquale Anthony Della Rosa², Matteo Canini², Francesca Fumagalli^3,4,5, Valeria Calbi^3,4, Andrea Falini⁶, Alessandro Aiuti^3,4,7, and Cristina Baldoli⁶

¹Nuclear Medicine Dept., IRCCS San Raffaele Scientific Institute, Milan, Italy, ²Neuroradiology Unit and CERMAC, IRCCS San Raffaele Scientific Institute, Milan, Italy, ³San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy, ⁴Unit of Pediatric Immunohematology and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy, ⁵Neurology Dept., IRCCS San Raffaele Scientific Institute, Milan, Italy, ⁶Neuroradiology Unit and CERMAC, Vita Salute San Raffaele University and IRCCS San Raffaele Scientific Institute, Milan, Italy, ⁷Vita Salute San Raffaele University, Milan, Italy

The efficacy of Haematopoietic Stem Cell-Gene Therapy for Metachromatic Leukodystrophy is under evaluation. CNS damage is currently rated by means of visual MR severity scores. The aim of this study was to investigate whether quantitative spectroscopy measurements correlate with MR severity scores. This preliminary analysis shows that both NAA/Cr and NAA/Cho ratios are associated with progression in total MR Severity Score, Atrophy and Demyelination Load. This suggests that MR spectroscopy could be useful to longitudinally and quantitatively evaluate the disease burden in MLD patients.

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