ActiveMSers Forums  

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

Reply
 
Thread Tools Display Modes
  #1  
Old 12-05-2019, 12:59 PM
ActiveMSers's Avatar
ActiveMSers ActiveMSers is online now
Dave @ ActiveMSers
 
Join Date: Jun 2008
Location: Albuquerque, NM
Posts: 3,863
Default BEAM Vs Cyclophosphamide-Based Conditioning Regimen in Aggressive Multiple Sclerosis

731.CLINICAL AUTOLOGOUS TRANSPLANTATION: RESULTS| NOVEMBER 13, 2019

BEAM Vs Cyclophosphamide-Based Conditioning Regimen in Aggressive Multiple Sclerosis: A Retrospective Analysis of European Blood and Marrow Transplantation Society

Riccardo Saccardi, MD , Manuela Badoglio , Joachim Burman , Grzegorz Helbig , Majid A. Kazmi, FRCP , Giovanni Mancardi , Stephan Mielke, MD , John Moore, MDFRACP,FRCPA , Paolo Muraro , Jaime Sanz, MD PhD , Basil Sharrack , Marek Smilowski , Maria Pia Sormani , John A Snowden, MD PhD

Blood (2019) 134 (Supplement_1): 3313.
https://doi.org/10.1182/blood-2019-125233


Background

Multiple Sclerosis (MS) is a chronic, immuno-mediated disease of Central Nervous System (CNS), mostly affecting young adults and frequently resulting in a progressive, irreversible disability despite the administration of approved Disease Modifying Treatments (DMTs). Autologous HSCT was shown to induce a high rate of sustained, treatment-free remissions in cases of aggressive MS, seldom associated to a partial reversal of disability. Toxicity of Conditioning Regimen is still a major concern. We retrospectively analyzed the outcome of 926 MS patients reported to the EBMT Registry who underwent autologous HSCT following the two most frequent CRs for this indication in the last 20 years.

Patients and Methods

Patient data were extracted from both the EBMT database and a disease-specific database developed by the EBMT Autoimmune Diseases Working Party (ADWP). Patients were selected for having received either BEAM + ATG (BEAM) or HD-Cyclophosphamide + ATG (CYC) as conditioning regimen. Hematological toxicity was assessed through Neutrophil (PMN) engraftment and 100-days (early) mortality (eTRM). MS forms at HSCT were reported as Relapsing-Remitting (RR), Secondary Progressive (SP), Primary Progressive (PP) and Progressive-Relapsing (PR). The impact of variables related to both patients (age, gender, year of HSCT, EDSS at HSCT) and disease characteristics (MS form, interval diagnosis-HSCT) at HSCT in the two groups were also evaluated.

Results

The utilization of conditioning regimens along the observed time period (1998-2018) was variable, with an increase of the HSCT activity in general after 2010 (230 vs 697 procedures) and a prevalence of BEAM before 2010 (205 BEAM vs 25 CYC) and of CYC thereafter (205 BEAM vs 492 CYC, p=0.001). Also, RR forms of MS prevailed over Progressive forms after 2010 (p=0.001) which is reflected in the different distribution across the two regimens, with RR significantly more frequently treated with CYC-based regimen (p<0.001). Gender distribution and age at HSCT was similar in the two groups (p=ns), whilst the interval between diagnosis and HSCT was longer in BEAM group than CYC (8.47 years 5.9 vs 7.57 5.5, mean SD, p=0.012). PMN engraftment in BEAM/ATG- and CYC/ATG-treated patients occurred at +11.0 (8-42) and +10.9 (8-95) days, respectively (median and range, p=ns). Overall eTRM was low (1.4%), but slightly higher in BEAM over CYC (8/402, 2% vs 5/517, 1%, p=ns).

Discussion

Although autologous HSCT is increasingly used as a treatment in highly active MS, toxicity remains a principal concern in the neurological community despite a marked decrease of TRM over time. The intensity of conditioning regimens has varied in the literature, but the best toxicity/efficacy ratio remains unclear. The non-myeloablative regimen CYC-ATG has become the most common conditioning regimen despite a lack of comparative data with more intense regimens. In our large retrospective analysis of the two most frequent conditioning regimens in the EBMT Registry, there was no significant difference in major toxicity indicators despite differences in chronological period and patients characteristics in the two groups. Comparative analysis of neurological efficacy is currently ongoing and will inform the toxicity/efficacy ratio and clinical choice of conditioning regimen in autologous HSCT in MS.
__________________
Dave Bexfield
ActiveMSers
Reply With Quote
Reply

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 11:21 AM.


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