Intermediate intensity regimen in autologous HSCT for severe multiple sclerosis: a large, single center experience
Author(s): ,
R Saccardi
Affiliations:
Hematology Department, University of Florence, Florence
,
A.M Repice
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
C Innocenti
Affiliations:
Hematology Department, University of Florence, Florence
,
A Mariottini
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
M.P Amato
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
S Guidi
Affiliations:
Hematology Department, University of Florence, Florence
,
A Fani
Affiliations:
Hematology Department, University of Florence, Florence
,
R Nistri
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
M Giannini
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
E Portaccio
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
A Barilaro
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
,
C Nozzoli
Affiliations:
Hematology Department, University of Florence, Florence
,
A Gozzini
Affiliations:
Hematology Department, University of Florence, Florence
L Massacesi
Affiliations:
Neurological Department, University of Florence, Firenze, Italy
ECTRIMS Online Library. Saccardi R. Sep 15, 2016; 146596; P756
Riccardo Saccardi
Riccardo Saccardi
Contributions
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Abstract

Abstract: P756

Type: Poster

Abstract Category: Therapy - disease modifying - Others

High dose chemotherapy with autologous hematopoietic stem cell transplantation (aHSCT) is a promising approach for treatment of aggressive multiple sclerosis (MS). Data reported in the literature are derived from either small, single center or larger registry analysis, with a wide variability in the transplant techniques and clinical conditions at baseline. We report a large retrospective, single-center analysis of 62 consecutive MS patients treated with an uniform transplant protocol.

Material and methods: we included 62 MS patients transplanted between 1999 and 2015. Female/male ratio was 0.76, median age was 36 ( 20-54). 37 patients (59.7%) were diagnosed with Relapsing-Remitting (RR) MS, 23 (37.1%) with Secondary Progressive (SP) MS, 2(3.2%) had Primary Progressive (PP) MS. Median EDSS score at transplant was 6.0 (range 1-7.5). PBSC were mobilized with cyclophosphamide 4 gr/sqm + G-CSF and conditioned with BEAM/ATG, except for 2 patients who could not accomplish the conditioning with BEAM due to either BCNU reaction or detection of Meropenem- resistant Klebsiella respectively.

Results: All patients mobilized adequately. Median duration of follow up is 4.2 years (range 0.1-14.9). Three out of 62 patients relapsed at 3.9, 4.9 and 5 years from transplant, respectively, without disability progression thereafter. Overall progression rate was 27.4% at a median time of 2.2 (0.47-4.39) years from HSCT: among progressed patients, 11 out of 23 had SPMS, 4 out of 37 RRMS and 2 out of 2 PPMS (RR vs SP, p=0.012). We had no transplant-associated mortality. Two severe adverse event occurred during the procedure: an anaphylactic shock to carmustine and a E. Coli-related sepsis. FUO (62.9%) and diarrhea (40.3%) were the most frequently recorded adverse events in the first 100 days. CMV and EBV reactivations occurred in 29% and 51% of patients. Adverse events occurred beyond 100 days were documented pneumonia (3), HVZ reactivation (4), transient monoclonal gammopathy (9) and autoimmune thyroiditis (3).

Disclosure:

Saccardi: nothing to disclose

Innocenti: nothing to disclose

Fani: nothing to disclose

Repice: nothing to disclose

Massacesi nothing to disclose

Barilaro nothing to disclose

Mariottini nothing to disclose

Guidi:nothing to disclose

Nozzoli: nothing to disclose

Nistri: nothing to disclose

Gozzini: nothing to disclose

Amato :nothing to disclose

Giannini: nothing to disclose

Portaccio: nothing to discolse

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