Autologous HSCT for severe progressive Multiple Sclerosis in a multicenter
trial: impact on disease activity and quality of life
From the Bone Marrow Transplantation Unit, Careggi Hospital, University of Florence, Italy; the
Department of Neurological Sciences, Ophtalmology and Genetics, Centre of Excellence for
Biomedical Research, University of Genoa, Italy; the Istituto Nazionale Neurologico “Carlo
Besta”, Milan, Italy; the Unit of Clinical Epidemiology and Trials, National Cancer Institute,
Genova, Italy; the Bone Marrow Transplantation Unit, S. Spirito Hospital, Pescara, Italy; the
Department of Oncology and Hematology, Policlinico of Modena, Italy; the Neuroimaging
Research Unit, Department of Neuroscience, Scientific Institute, S. Raffaele Hospital, Milan,
Italy; the Internal Medicine and Hematology Department, S. Luigi Hospital, Orbassano, Italy; the
Bone Marrow Transplantation Unit, S. Martino Hospital, Genova, Italy; the Bone Marrow
Transplantation Unit, Binaghi Hospital, Cagliari, Italy; the Department of Haematology, S.
Chiara Hospital, Pisa, Italy.
Результаты:
Patients’ characteristics are summarized in Table 1. There were 12 females and 7 males, with a
median age of 36 (range 26-52) years. Disease course was SPMS in 15 cases, and RRMS in 4. In
9
SPMS, relapses were superimposed in 5 cases; in 3 out of 4 RRMS patients, relapses were not
followed by improvement, with an ensuing rapid worsening of disability. The median age at the
onset of disease was 24 (range 15-46) years, the median disease duration was 12 (range 4-19)
years. The median duration of the progressive phase was 5 (range 1-11) years. Twelve patients
had been previously treated with interferon- 1a or 1b, and 7 only with azathioprine and/or
steroids, all without clinical response. Two cases were treated after interferon beta with
cyclophosphamide at the dosage of 1 gr intravenously monthly for 4 months and for 6 months,
respectively. Another case, following failure with interferon beta 1b, was treated with
mitoxantrone (3 cycles at the dosage of 20 mg intravenously each), without any benefit on the
relapse rate and progression.
At the screening visit, the median EDSS was 6.5 (range 5.0-6.5). At baseline, immediately before
stem cell mobilization, the neurological condition had deteriorated in 11 cases (median EDSS
6.5, range 5.5-8).
вот обозор по теме (свежак)
Autologous haematopoietic-stem-cell transplantation for
multiple sclerosis
Lancet Neurol 2005; 4: 54–63