STUDY: Sustained remission in MS after hematopoietic stem cell transplantation

[ActiveMSers]

After 10 years, a 50/50 shot at full remission sounds pretty darn impressive. An important caveat: They all had aggressive forms of relapsing remitting MS, as you can see from an earlier paper they published (immediately below). -D

During the last 15 years, high-dose chemotherapy with autologous hematopoietic stem cell transplantation (HSCT) has globally been performed for severe multiple sclerosis (MS). Most patients have been in progressive phase with long disease duration. As a rule, treatment effect has been minor or moderate. Since 2004, we have performed HSCT in nine young patients with "malignant" relapsing-remitting MS. Criteria for treatment were short duration of disease; very frequent, severe relapses; recent improvement periods indicating potential for recovery after strong immunosuppression. Median age at treatment was 27 (range 9-34) years, MS duration 26 (4-100) months, and annualized relapse rate 10 (4-12). Median Disability Status Scale (extended disability status scale, EDSS) at HSCT was 7.0 (3.5-8.0). Median follow-up time April 2008 is 29 (23-47) months. Median EDSS improvement is 3.5 (1.0-7.0), clearly surpassing most previous reports. One patient relapsed mildly with rapid recovery 7 months after HSCT. All patients are otherwise stable, median EDSS being 2.0 (0-6.0). Before HSCT, 61 relapses occurred in 82 patient months; during follow-up, one relapse in 289 patient months. This small series of patients with "malignant" relapsing-remitting MS suggests HSCT to be an effective treatment option for this relatively rare disease course. It further suggests that future criteria for HSCT in MS should be close to the present ones.

Sustained remission in multiple sclerosis after hematopoietic stem cell transplantation

Andreas Tolf Jan Fagius Kristina Carlson Torbjörn Åkerfeldt Tobias Granberg Elna‐Marie Larsson Joachim Burman

First published: 12 July 2019
https://doi.org/10.1111/ane.13147

Abstract

Objectives
To determine if treatment with autologous hematopoietic stem cell transplantation can induce sustained complete remission in patients with multiple sclerosis (MS).

Material and Methods
Case series of patients with relapsing‐remitting MS (n=10) treated at a single center between 2004 and 2007 and followed up for 10 years. The patients were treated with a BEAM/ATG conditioning regimen (n=9) or a cyclophosphamide/ATG conditioning regimen (n=1) followed by infusion of unmanipulated autologous hematopoietic stem cells. The primary endpoint was sustained complete remission. Sustained complete remission was defined as ‘no evidence of disease activity‐4', sustained for a period of at least 5 years without any ongoing disease‐modifying treatment. Furthermore, MS was considered as ‘resolved' if intrathecal IgG production and cerebrospinal fluid neurofilament light levels were normalized as well.

Results
Five out of 10 patients were in sustained complete remission at the end of the study. In 3 of them, MS was resolved.

Conclusions
Our data demonstrate that sustained complete remission after autologous hematopoietic stem cell transplantation for MS is possible.

[Suebee]

Thanks for posting this article Dave! I haven't been keeping tabs on follow up studies and I’m glad that it has shown effective for half. I initially had aggressive progression and it was scary to be in a position when drs had nothing more to offer.

[ActiveMSers]

Thanks Suebee. I think it is important for people to realize that even in the best-case scenarios, that holding this disease at bay for a decade is at best a coin toss. I cringe when people six months post transplant tell everyone "I'm cured!" Or point to 2-year studies with a high NEDA rate and assume those rates extend for years and years. Frustrating!