BEAM/ATG or cyclophosphamide/ATG as conditioning regimen in autologous haemopoietic stem cell transplantation for multiple sclerosis: a retrospective analysis of the EBMT autoimmune diseases working party - Scorecard - MDSpire
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BEAM/ATG or cyclophosphamide/ATG as conditioning regimen in autologous haemopoietic stem cell transplantation for multiple sclerosis: a retrospective analysis of the EBMT autoimmune diseases working party
Clinical Scorecard: Comparison of BEAM/ATG and cyclophosphamide/ATG Conditioning Regimens in Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis: A Retrospective Study by the EBMT Autoimmune Diseases Working Party
At a Glance
Category
Detail
Condition
Multiple Sclerosis (MS), primarily relapsing-remitting type
Key Mechanisms
Autologous hematopoietic stem cell transplantation (HSCT) with intermediate intensity conditioning regimens (BEAM/ATG or cyclophosphamide/ATG) to reset immune system and halt inflammatory activity
Target Population
Adults (≥18 years) with highly active relapsing-remitting MS refractory to high-efficacy disease-modifying therapies
Care Setting
Specialized hematology and neurology centers reporting to the EBMT registry
Key Highlights
Autologous HSCT is a highly effective treatment for treatment-resistant inflammatory MS, achieving high rates of no evidence of disease activity (NEDA) ranging from 66% to 93%.
Two main intermediate intensity conditioning regimens are used: BEAM/ATG and cyclophosphamide (CYC)/ATG, both included in EBMT guidelines.
Retrospective data show comparable efficacy between BEAM/ATG and CYC/ATG, with BEAM/ATG associated with more severe adverse events and prolonged hospitalization.
Guideline-Based Recommendations
Diagnosis
Diagnosis of MS according to revised McDonald’s criteria.
Patient selection focusing on highly active relapsing-remitting MS failing high-efficacy DMTs.
Management
Use of autologous HSCT with intermediate intensity conditioning regimens (BEAM/ATG or CYC/ATG) as standard care for selected patients.
Avoid enrollment of patients in clinical trials like MIST when analyzing registry data.
Monitoring & Follow-up
Regular neurological assessments including Expanded Disability Status Scale (EDSS) and MRI scans pre- and post-transplant.
Monitoring for early infectious and non-infectious complications within 100 days post-HSCT.
Assessment of relapse rate and disability progression to evaluate NEDA status.
Risks
Transplant-related mortality (TRM) approximately 1.1% at 100 days and 1.5% at 3 years.
Higher incidence of severe adverse events and prolonged hospitalization with BEAM/ATG regimen.
Non-relapse mortality stable at about 1% from 2015 to 2020.
Patient & Prescribing Data
MS patients aged 18 or older undergoing first autologous HSCT with detailed clinical history and follow-up.
Both BEAM/ATG and CYC/ATG conditioning regimens demonstrate similar efficacy in maintaining NEDA; CYC/ATG may have a better safety and tolerability profile.
Clinical Best Practices
Careful patient selection focusing on early inflammatory phases of relapsing-remitting MS to optimize risk-benefit ratio.
Use of intermediate intensity conditioning regimens combining chemotherapy and ATG for effective disease control.
Close multidisciplinary collaboration between neurologists and hematologists for patient management and follow-up.
Regular monitoring of disease activity via clinical and MRI assessments to evaluate treatment success.
Vigilant management of early transplant-related complications and supportive care to minimize morbidity.
by Raffaella Greco, Riccardo Saccardi, Marta Ponzano, Manuela Badoglio, Grzegorz Helbig, Marek Smilowski, Alice Mariottini, Joachim Burman, Kristina Carlson, Majid Kazmi, Paolo A. Muraro, Ian Gabriel, Barbara Withers, Jennifer Massey, Riccardo Varaldo, Matilde Inglese, Jaime Sanz, Sara Gil-Perotin, Basil Sharrack, Elisa Roldan, Chiara Nozzoli, Alessio Signori, Maria Pia Sormani, Tobias Alexander, John A. Snowden
