Over the last decade, hematopoietic stem cells transplantation (HSCT) has been increasingly used in the treatment of severe progressive autoimmune diseases. We report a retrospective survey of 183 multiple sclerosis (MS) patients, recorded in the database of the European Blood and Marrow Transplantation Group (EBMT). Transplant data were available from 178 patients who received an autologous graft. Overall, transplant related mortality (TRM) was 5.3% and was restricted to the period 1995-2000, with no further TRM reported since then. Busulphan-based regimens were significantly associated with TRM. Clinical status at the time of transplant and transplant techniques showed some correlations with toxicity. No toxic deaths were reported among the 53 patients treated with the BEAM (carmustine, etoposide, cytosine-arabinoside, melphalan)/antithymocyte globulin (ATG) regimen without graft manipulation, irrespective of their clinical condition at the time of the transplant. Improvement or stabilization of neurological conditions occurred in 63% of patients at a median follow-up of 41.7 months, and was not associated with the intensity of the conditioning regimen. In this large series, HSCT was shown as a promising procedure to slow down progression in a subset of patients affected by severe, progressive MS; the safety and feasibility of the procedure can be significantly improved by appropriate patient selection and choice of transplant regimen.

Autologous stem cell transplantation for progressive multiple sclerosis: update of the European Group for Blood and Marrow Transplantation auto immune diseases working party database / SACCARDI R, KOZAK T, BOCELLI-TYNDALL C, FASSAS A, KAZIS A, HAVRDOVA E, CARRERAS E, SAIZ A, LOWENBERG B, TE BOEKHORST P; GUALANDI F, OPENSHAW H, LONGO G, PAGLIAI F, MASSACESI L, DECONINK E, OUYANG J, ZUAZU NAGORE F, BESALDUCH J, LISUKOV IA, BONINI A, MERELLI E, SLAVIN S, GRATWOHL A, PASSWEG J, TYNDALL A, STECK AJ, ANDOLINA M, CAPOBIANCO M, DIEZ MARTIN J, LUGARESI A, MEUCCI G, SAEZ RA, CLARK RE, FERNANDEZ MN, FOUILLARD L, HERSTENSTEIN B, KOZA V, COCCO E, BAURMANN H, MANCARDI GL, ON BEHALF OF THE AUTOIMMUNE DISEASES WORKING PARTY OF EBMT. - In: MULTIPLE SCLEROSIS. - ISSN 1352-4585. - ELETTRONICO. - 12(6):(2006), pp. 814-823. [10.1177/1352458506071301]

Autologous stem cell transplantation for progressive multiple sclerosis: update of the European Group for Blood and Marrow Transplantation auto immune diseases working party database.

MASSACESI, LUCA;
2006

Abstract

Over the last decade, hematopoietic stem cells transplantation (HSCT) has been increasingly used in the treatment of severe progressive autoimmune diseases. We report a retrospective survey of 183 multiple sclerosis (MS) patients, recorded in the database of the European Blood and Marrow Transplantation Group (EBMT). Transplant data were available from 178 patients who received an autologous graft. Overall, transplant related mortality (TRM) was 5.3% and was restricted to the period 1995-2000, with no further TRM reported since then. Busulphan-based regimens were significantly associated with TRM. Clinical status at the time of transplant and transplant techniques showed some correlations with toxicity. No toxic deaths were reported among the 53 patients treated with the BEAM (carmustine, etoposide, cytosine-arabinoside, melphalan)/antithymocyte globulin (ATG) regimen without graft manipulation, irrespective of their clinical condition at the time of the transplant. Improvement or stabilization of neurological conditions occurred in 63% of patients at a median follow-up of 41.7 months, and was not associated with the intensity of the conditioning regimen. In this large series, HSCT was shown as a promising procedure to slow down progression in a subset of patients affected by severe, progressive MS; the safety and feasibility of the procedure can be significantly improved by appropriate patient selection and choice of transplant regimen.
2006
12(6)
814
823
SACCARDI R, KOZAK T, BOCELLI-TYNDALL C, FASSAS A, KAZIS A, HAVRDOVA E, CARRERAS E, SAIZ A, LOWENBERG B, TE BOEKHORST P; GUALANDI F, OPENSHAW H, LONGO G, PAGLIAI F, MASSACESI L, DECONINK E, OUYANG J, ZUAZU NAGORE F, BESALDUCH J, LISUKOV IA, BONINI A, MERELLI E, SLAVIN S, GRATWOHL A, PASSWEG J, TYNDALL A, STECK AJ, ANDOLINA M, CAPOBIANCO M, DIEZ MARTIN J, LUGARESI A, MEUCCI G, SAEZ RA, CLARK RE, FERNANDEZ MN, FOUILLARD L, HERSTENSTEIN B, KOZA V, COCCO E, BAURMANN H, MANCARDI GL, ON BEHALF OF THE AUTOIMMUNE DISEASES WORKING PARTY OF EBMT
File in questo prodotto:
File Dimensione Formato  
2006 Autologous stem cell transplantation for progressive....pdf

Accesso chiuso

Tipologia: Versione finale referata (Postprint, Accepted manuscript)
Licenza: Tutti i diritti riservati
Dimensione 153.35 kB
Formato Adobe PDF
153.35 kB Adobe PDF   Richiedi una copia

I documenti in FLORE sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/216867
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 201
  • ???jsp.display-item.citation.isi??? 167
social impact