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This is the first case of PML that I've noticed with HSCT. Still powerful results. - D
J Neurol. 2017 Dec 21. doi: 10.1007/s00415-017-8718-2. [Epub ahead of print]
Long-term follow-up more than 10 years after HSCT: a monocentric experience.
Frau J1, Carai M2, Coghe G2, Fenu G2, Lorefice L2, La Nasa G3, Mamusa E2, Vacca A3, Marrosu MG2, Cocco E2.
Abstract
BACKGROUND:
Autologous hematopoietic stem cell transplantation (aHSCT) is used in aggressive relapsing and progressive multiple sclerosis (MS). The multicentre studies and case series reported have relatively short follow-up.
AIM:
To evaluate long-term effect and safety of HSCT in MS.
MATERIALS AND METHODS:
Patients referred to the MS centre of Cagliari and undergoing HSCT were included. Variations in relapses and EDSS before and after HSCT were evaluated by Wilcoxon test. A descriptive analysis was made for other clinical data.
RESULTS:
Nine patients (female 6, males 3; 5 relapsing-remitting, 2 secondary progressive, 1 primary progressive, and 1 progressive relapsing) performed HSCT (1999-2006). The median follow-up was 11 years (11-18). Eight patients underwent aHSCT, seven using a low intensity conditioning regimen, and one an intermediate intensity. The primary progressive underwent allogeneic HSCT, due to onco hematological disease. The relapses number decreased in the 2 years following the procedure compared to the two preceding years (p = 0.041). New relapses or disease progressions were observed after a range of 7 (low intensity regimen)-118 (intermediate intensity) months. At last follow-up, the EDSS was stable in two patients, improved in two, and worse in five (maximum 2 EDSS in one patient). Six patients showed new lesions, and seven gadolinium-enhancing on brain MRI after a mean of 23.3 and 19.8 months, respectively. Two serious adverse events were reported: melanoma, and progressive multifocal leukoencephalopathy.
CONCLUSIONS AND DISCUSSION:
Our results confirm in a long follow-up the efficacy of HSCT in reducing relapses and disability progression. The risk/benefit profile is better for intermediate intensity regimens.
J Neurol. 2017 Dec 21. doi: 10.1007/s00415-017-8718-2. [Epub ahead of print]
Long-term follow-up more than 10 years after HSCT: a monocentric experience.
Frau J1, Carai M2, Coghe G2, Fenu G2, Lorefice L2, La Nasa G3, Mamusa E2, Vacca A3, Marrosu MG2, Cocco E2.
Abstract
BACKGROUND:
Autologous hematopoietic stem cell transplantation (aHSCT) is used in aggressive relapsing and progressive multiple sclerosis (MS). The multicentre studies and case series reported have relatively short follow-up.
AIM:
To evaluate long-term effect and safety of HSCT in MS.
MATERIALS AND METHODS:
Patients referred to the MS centre of Cagliari and undergoing HSCT were included. Variations in relapses and EDSS before and after HSCT were evaluated by Wilcoxon test. A descriptive analysis was made for other clinical data.
RESULTS:
Nine patients (female 6, males 3; 5 relapsing-remitting, 2 secondary progressive, 1 primary progressive, and 1 progressive relapsing) performed HSCT (1999-2006). The median follow-up was 11 years (11-18). Eight patients underwent aHSCT, seven using a low intensity conditioning regimen, and one an intermediate intensity. The primary progressive underwent allogeneic HSCT, due to onco hematological disease. The relapses number decreased in the 2 years following the procedure compared to the two preceding years (p = 0.041). New relapses or disease progressions were observed after a range of 7 (low intensity regimen)-118 (intermediate intensity) months. At last follow-up, the EDSS was stable in two patients, improved in two, and worse in five (maximum 2 EDSS in one patient). Six patients showed new lesions, and seven gadolinium-enhancing on brain MRI after a mean of 23.3 and 19.8 months, respectively. Two serious adverse events were reported: melanoma, and progressive multifocal leukoencephalopathy.
CONCLUSIONS AND DISCUSSION:
Our results confirm in a long follow-up the efficacy of HSCT in reducing relapses and disability progression. The risk/benefit profile is better for intermediate intensity regimens.