Article Text

Research paper
Fetal striatal grafting slows motor and cognitive decline of Huntington's disease
  1. Marco Paganini1,2,
  2. Annibale Biggeri3,4,
  3. Anna Maria Romoli1,
  4. Claudia Mechi1,
  5. Elena Ghelli1,
  6. Valentina Berti5,
  7. Silvia Pradella1,
  8. Sandra Bucciantini2,
  9. Dolores Catelan3,4,
  10. Riccardo Saccardi2,
  11. Letizia Lombardini2,6,
  12. Mario Mascalchi5,
  13. Luca Massacesi1,2,
  14. Berardino Porfirio2,5,
  15. Nicola Di Lorenzo7,
  16. Gabriella Barbara Vannelli8,
  17. Pasquale Gallina2,7
  1. 1Department of Neuroscience, University of Florence, Florence, Italy
  2. 2Careggi University Hospital, Florence, Italy
  3. 3Department of Statistics, Informatics and Applications “G. Parenti”, University of Florence, Florence, Italy
  4. 4Biostatistics Unit, ISPO Cancer Prevention and Research Institute, Florence, Italy
  5. 5Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy
  6. 6Italian National Health Institute, National Transplantation Center, Rome, Italy
  7. 7Department of Surgery and Translational Medicine, University of Florence, Florence, Italy
  8. 8Department of Clinical and Experimental Medicine, University of Florence, Florence, Italy
  1. Correspondence to Professor Berardino Porfirio, Department of Experimental and Clinical Biomedical Sciences, University of Florence, Viale G. Pieraccini 6, Florence 50139, Italy; nporfirio{at}unifi.it

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 [18F]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. [123I]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.

  • Huntington's
  • Stereotaxic Surgery

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