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Autologous stem cell transplantation for progressive multiple sclerosis: Update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database

BMT Unit Department of Hematology, Ospedale di Careggi, Florence, Italy

T Kozak

Department of Clinical Hematology, Third Medical School, Charles University, Prague 10, Czech Republic

C Bocelli-Tyndall

Department of Rheumatology, University Hospital, Basel, Switzerland

A Fassas

Hematology Department/BMT Unit, George Papanicolaou General Hospital, Thessaloniki, Greece

A Kazis

Department of Neurology, George Papanicolaou General Hospital, Thessaloniki, Greece

E Havrdova

Department of Neurology, First Medical School, Charles University, Prague 2, Czech Republic

E Carreras

Department of Hematology, Hospital Clinic, Institute of Hematology and Oncology, Barcelona, Spain

A Saiz

Laboratory of Experimental Neurology and Immunology, Hospital Clinic, Barcelona, Spain

B Löwenberg

Erasmus University Medical Center, Rotterdam, The Netherlands

P AW te Boekhorst

Erasmus University Medical Center, Rotterdam, The Netherlands

F Gualandi

Department of Hematology, San Martino Hospital, Genova, Italy

H Openshaw

City of Hope National Medical Center, Duarte, CA, USA

G Longo

BMT Unit Department of Hematology, Ospedale di Careggi, Florence, Italy

F Pagliai

BMT Unit Department of Hematology, Ospedale di Careggi, Florence, Italy

L Massacesi

Department of Neurology, Ospedale di Careggi, Florence, Italy

E Deconink

Service d’Hématologie, Hopital Jean Minjoz, Besançon, France

J Ouyang

Department of Hematology, Drum Tower Hospital, Nanjing, PR China

F JZ Nagore

Hospital Vall d’Hebron, Barcelona, Spain

J Besalduch

Hematology Service, Hospital Universitari Son Dureta, Palma de Mallorca, Spain

I A Lisukov

Institute of Clinical Immunology, Novosibirsk, Russia

A Bonini

Hematology Unit, Azienda Ospedaliera ASMN, Reggio Emilia, Italy

E Merelli

Clinica Neurologica, Azienda Ospedaliera Policlinico, Modena, Italy

S Slavin

Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel

A Gratwohl

Department of Hematology, University Hospital, Basel, Switzerland

J Passweg

Department of Hematology, University Hospital, Basel, Switzerland

A Tyndall

Department of Rheumatology, University Hospital, Basel, Switzerland

A J Steck

Department of Neurology, University Hospital, Basel, Switzerland

M Andolina

Istituto per l’Infanzia Burlo Garofolo, Trieste, Italy

M Capobianco

Ospedale di San Luigi Gonzaga, Orbassano, Torino, Italy

J LD Martin

Sección de Trasplante de Medula Osea, Hospital Gregorio Marañón, Madrid, Spain

A Lugaresi

Università Gabriele d’Annunzio, Chieti, Italy

G Meucci

Department of Neuroscience, University Hospital of Pisa, Neurology Clinic, Pisa, Italy

R A Sáez

Department of Hematology, Hospital de la Princesa, Madrid, Spain

R E Clark

Department of Haematology, Royal Liverpool University Hospital, Liverpool, UK

M N Fernandez

Clinica Puerta de Hierro, Servicio de Hematologia y Hemoterapia, Madrid, Spain

L Fouillard

Department of Hematology, Hopital Saint Antoine, Paris, France

B Herstenstein

Department of Hematology/Oncology, Hannover Medical University, Hannover, Germany

V Koza

Department of Hematology/Oncology, Charles University Hospital, Pilsen, Czech Republic

E Cocco

‘R Binaghi Hospital’, Centro Sclerosi Multipla, Cagliari, Italy

H Baurmann

Neurologie und Klinische Neurophysiologie, Wiesbaden, Germany

G L Mancardi

Department of Neuroscience, Ophthalmology and Genetics, San Martino Hospital, University of Genova, Genova, Italy

Autoimmune Diseases Working Party of EBMT

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.

Key Words: autoimmune diseases • immunosuppression • multiple sclerosis • stem cells transplantation • treatment safety

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