title: Fetal striatal grafting slows motor and cognitive decline of Huntington's disease.
abstract: OBJECTIVE
  To assess the clinical effect of caudate-putaminal transplantation of fetal striatal tissue in Huntington's disease (HD).
  
  
  METHODS
  We carried out a follow-up study on 10 HD transplanted patients and 16 HD not-transplanted patients. All patients were evaluated with the Unified HD Rating Scale (UHDRS) whose change in motor, cognitive, behavioural and functional capacity total scores were considered as outcome measures. Grafted patients also received morphological and molecular neuroimaging.
  
  
  RESULTS
  Patients were followed-up from disease onset for a total of 309.3 person-years (minimum 5.3, median 11.2 years, maximum 21.6 years). UHDRS scores have been available since 2004 (median time of 5.7 years since onset, minimum zero, maximum 17.2 years). Median post-transplantation follow-up was 4.3 years, minimum 2.8, maximum 5.1 years. Adjusted post-transplantation motor score deterioration rate was reduced compared to the pretransplantation period, and to that of not-transplanted patients by 0.9 unit/years (95% CI 0.2 to 1.6). Cognitive score deterioration was reduced of 2.7 unit/years (95% CI 0.1 to 5.3). For grafted patients the 2-year post-transplantation [(18)F]fluorodeoxyglucose positron emission tomography (PET) showed striatal/cortical metabolic increase compared to the presurgical evaluation; 4-year post-transplantation PET values were slightly decreased, but remained higher than preoperatively. [(123)I]iodobenzamide single photon emission CT demonstrated an increase in striatal D2-receptor density during postgrafting follow-up.
  
  
  CONCLUSIONS
  Grafted patients experienced a milder clinical course with less pronounced motor/cognitive decline and associated brain metabolism improvement. Life-time follow-up may ultimately clarify whether transplantation permanently modifies the natural course of the disease, allowing longer sojourn time at less severe clinical stage, and improvement of overall survival.
authors.affiliation: Department of Neuroscience, University of Florence, Florence, Italy Careggi University Hospital, Florence, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Paganini
authors.affiliation: Department of Statistics, Informatics and Applications "G. Parenti", University of Florence, Florence, Italy Biostatistics Unit, ISPO Cancer Prevention and Research Institute, Florence, Italy.
authors.forename: Annibale
authors.initials: A
authors.lastname: Biggeri
authors.affiliation: Department of Neuroscience, University of Florence, Florence, Italy.
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Romoli
authors.affiliation: Department of Neuroscience, University of Florence, Florence, Italy.
authors.forename: Claudia
authors.initials: C
authors.lastname: Mechi
authors.affiliation: Department of Neuroscience, University of Florence, Florence, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Ghelli
authors.affiliation: Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy.
authors.forename: Valentina
authors.initials: V
authors.lastname: Berti
authors.affiliation: Department of Neuroscience, University of Florence, Florence, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Pradella
authors.affiliation: Careggi University Hospital, Florence, Italy.
authors.forename: Sandra
authors.initials: S
authors.lastname: Bucciantini
authors.affiliation: Department of Statistics, Informatics and Applications "G. Parenti", University of Florence, Florence, Italy Biostatistics Unit, ISPO Cancer Prevention and Research Institute, Florence, Italy.
authors.forename: Dolores
authors.initials: D
authors.lastname: Catelan
authors.affiliation: Careggi University Hospital, Florence, Italy.
authors.forename: Riccardo
authors.initials: R
authors.lastname: Saccardi
authors.affiliation: Careggi University Hospital, Florence, Italy Italian National Health Institute, National Transplantation Center, Rome, Italy.
authors.forename: Letizia
authors.initials: L
authors.lastname: Lombardini
authors.affiliation: Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy.
authors.forename: Mario
authors.initials: M
authors.lastname: Mascalchi
authors.affiliation: Department of Neuroscience, University of Florence, Florence, Italy Careggi University Hospital, Florence, Italy.
authors.forename: Luca
authors.initials: L
authors.lastname: Massacesi
authors.affiliation: Careggi University Hospital, Florence, Italy Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy.
authors.forename: Berardino
authors.identifier: http://orcid.org/0000-0003-4646-2502
authors.initials: B
authors.lastname: Porfirio
authors.affiliation: Department of Surgery and Translational Medicine, University of Florence, Florence, Italy.
authors.forename: Nicola
authors.initials: N
authors.lastname: Di Lorenzo
authors.affiliation: Department of Clinical and Experimental Medicine, University of Florence, Florence, Italy.
authors.forename: Gabriella Barbara
authors.initials: GB
authors.lastname: Vannelli
authors.affiliation: Careggi University Hospital, Florence, Italy Department of Surgery and Translational Medicine, University of Florence, Florence, Italy.
authors.forename: Pasquale
authors.initials: P
authors.lastname: Gallina
mesh_terms: D000328:Adult; D016380:Brain Tissue Transplantation; D002540:Cerebral Cortex / Q000378:metabolism; D003342:Corpus Striatum / Q000378:metabolism / Q000601:surgery*; D005260:Female; D016332:Fetal Tissue Transplantation; D019788:Fluorodeoxyglucose F18; D005500:Follow-Up Studies; D059907:Functional Neuroimaging; D006801:Humans; D006816:Huntington Disease / Q000378:metabolism / Q000503:physiopathology* / Q000523:psychology / Q000628:therapy*; D007462:Iodobenzenes; D008297:Male; D008875:Middle Aged; D009483:Neuropsychological Tests; D049268:Positron-Emission Tomography; D017448:Receptors, Dopamine D2 / Q000378:metabolism; D016896:Treatment Outcome
chemical_list: C581293:DRD2 protein, human; D007462:Iodobenzenes; D017448:Receptors, Dopamine D2; C426805:idobenzamide; D019788:Fluorodeoxyglucose F18
keywords: Huntington's; Stereotaxic Surgery