ActiveMSers Forums  

Go Back   ActiveMSers Forums > Forums > Dave's SCT Journey

Thread Tools Display Modes
Old 12-16-2018, 06:45 PM
ActiveMSers's Avatar
ActiveMSers ActiveMSers is offline
Dave @ ActiveMSers
Join Date: Jun 2008
Location: Albuquerque, NM
Posts: 3,989
Default Prospective phase II clinical trial of autologous HSCT for treatment refractory MS

Prospective phase II clinical trial of autologous haematopoietic stem cell transplant for treatment refractory multiple sclerosis

John J Moore1, Jennifer C Massey2, Carole D Ford3, Melissa L Khoo3, John J Zaunders4, Kevin Hendrawan3, Yael Barnett5, Michael H Barnett6, Kain A Kyle6, Robert Zivadinov7, Kris C Ma, Sam T Milliken1, Ian J Sutton2, David D F Ma1


Autologous haematopoietic stem cell transplantation (AHSCT) has been explored as a therapeutic intervention in multiple sclerosis (MS) over the last two decades; however, prospective clinical trials of the most common myeloablative conditioning regimen, BEAM, are limited. Furthermore, patient selection, optimal chemotherapeutic regimen and immunological changes associated with disease response require ongoing exploration. We present the outcomes, safety and immune reconstitution (IR) of patients with active, treatment refractory MS.

This study was a single-centre, phase II clinical trial of AHSCT for patients with active relapsing remitting (RRMS) and secondary progressive MS (SPMS). Patients underwent AHSCT using BEAM (carmustine, etoposide, cytarabine, melphalan)+antithymocyte globulin chemotherapeutic regimen.

The primary outcome was event-free survival (EFS); defined as no clinical or radiological relapses and no disability progression. Multiparameter flow cytometry was performed for evaluation of post-transplant IR in both MS and lymphoma patients receiving the same chemotherapy regimen.

Thirty-five patients (20 RRMS, 15 SPMS) completed AHSCT, with a median follow-up of 36 months (range 1266). The median Expanded Disability Status Scores (EDSS) was 6 (27) and patients had failed a median of 4 (27) disease modifying therapies. 66% failed treatment with natalizumab. EFS at 3 years was 60%, (70% RRMS). Sustained improvement in EDSS was seen in 15 (44%) of patients. There was no treatment-related mortality. A sustained rise in CD39+ T regulatory cells, immunosuppressive CD56hi natural killer cells and ablation of proinflammatory mucosal-associated invariant T cells was seen for 12 months following AHSCT in patients with MS. These changes did not occur in patients with lymphoma receiving the same chemotherapy for AHSCT.

The EFS in our MS cohort is significantly greater than other high-efficacy immunosuppressive therapies and similar to other AHSCT studies despite a more heavily pretreated cohort.

Trial registration number ACTRN12613000339752.
Dave Bexfield
Reply With Quote

Thread Tools
Display Modes

Posting Rules
You may not post new threads
You may not post replies
You may not post attachments
You may not edit your posts

BB code is On
Smilies are On
[IMG] code is On
HTML code is Off
Forum Jump

All times are GMT -4. The time now is 08:04 AM.

Powered by vBulletin® Version 3.7.0
Copyright ©2000 - 2020, Jelsoft Enterprises Ltd.