Articolo del 20 febbraio 2014 preso da www.ccsviitalia.org
di Matteo Scibilia
Uno studio osservazionale pubblicato sul Journal of Neurology, Neurosurgery & Psichiatry illustra i risultati a 5 anni su un gruppo di 48 malati di Sclerosi Multipla (SM), sottoposti a trapianto di Cellule Staminali Ematopoietiche (HSCT).
I pazienti erano tutti con una forma aggressiva della SM (RR 83%)
I risultati a 5 anni sono molto incoraggianti:
senza alcuna ricaduta 87%
senza riscontri RM 85%
senza peggioramento EDSS 77%
senza alcuna attività della malattia 68%
Abstract
BACKGROUND:
Autologous haematopoietic stem cell transplantation (HSCT) is a viable option for treatment of aggressive multiple sclerosis (MS). No randomised controlled trial has been performed, and thus, experiences from systematic and sustained follow-up of treated patients constitute important information about safety and efficacy. In this observational study, we describe the characteristics and outcome of the Swedish patients treated with HSCT for MS.
METHODS:
Neurologists from the major hospitals in Sweden filled out a follow-up form with prospectively collected data. Fifty-two patients were identified in total; 48 were included in the study and evaluated for safety and side effects; 41 patients had at least 1 year of follow-up and were further analysed for clinical and radiological outcome. In this cohort, 34 patients (83%) had relapsing-remitting MS, and mean follow-up time was 47 months.
RESULTS:
At 5 years, relapse-free survival was 87%; MRI event-free survival 85%; expanded disability status scale (EDSS) score progression-free survival 77%; and disease-free survival (no relapses, no new MRI lesions and no EDSS progression) 68%. Presence of gadolinium-enhancing lesions prior to HSCT was associated with a favourable outcome (disease-free survival 79% vs 46%, p=0.028). There was no mortality. The most common long-term side effects were herpes zoster reactivation (15%) and thyroid disease (8.4%).
CONCLUSIONS:
HSCT is a very effective treatment of inflammatory active MS and can be performed with a high degree of safety at experienced centres.
di Matteo Scibilia
Uno studio osservazionale pubblicato sul Journal of Neurology, Neurosurgery & Psichiatry illustra i risultati a 5 anni su un gruppo di 48 malati di Sclerosi Multipla (SM), sottoposti a trapianto di Cellule Staminali Ematopoietiche (HSCT).
I pazienti erano tutti con una forma aggressiva della SM (RR 83%)
I risultati a 5 anni sono molto incoraggianti:
senza alcuna ricaduta 87%
senza riscontri RM 85%
senza peggioramento EDSS 77%
senza alcuna attività della malattia 68%
Effetti collaterali importanti :
riattivazione di herpes zoster 15%
problemi alla tiroide 8,4%
riattivazione di herpes zoster 15%
problemi alla tiroide 8,4%
Le conclusioni degli Autori sono che "HSCT costituisce un trattamento molto efficace per il trattamento dell'attività infiammatoria della SM, e può essere applicato con sufficiente sicurezza in Centri Specializzati".
Abstract
BACKGROUND:
Autologous haematopoietic stem cell transplantation (HSCT) is a viable option for treatment of aggressive multiple sclerosis (MS). No randomised controlled trial has been performed, and thus, experiences from systematic and sustained follow-up of treated patients constitute important information about safety and efficacy. In this observational study, we describe the characteristics and outcome of the Swedish patients treated with HSCT for MS.
METHODS:
Neurologists from the major hospitals in Sweden filled out a follow-up form with prospectively collected data. Fifty-two patients were identified in total; 48 were included in the study and evaluated for safety and side effects; 41 patients had at least 1 year of follow-up and were further analysed for clinical and radiological outcome. In this cohort, 34 patients (83%) had relapsing-remitting MS, and mean follow-up time was 47 months.
RESULTS:
At 5 years, relapse-free survival was 87%; MRI event-free survival 85%; expanded disability status scale (EDSS) score progression-free survival 77%; and disease-free survival (no relapses, no new MRI lesions and no EDSS progression) 68%. Presence of gadolinium-enhancing lesions prior to HSCT was associated with a favourable outcome (disease-free survival 79% vs 46%, p=0.028). There was no mortality. The most common long-term side effects were herpes zoster reactivation (15%) and thyroid disease (8.4%).
CONCLUSIONS:
HSCT is a very effective treatment of inflammatory active MS and can be performed with a high degree of safety at experienced centres.
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