From the Guidelines
For the treatment of multiple sclerosis (MS), intermediate-intensity conditioning protocols, such as BEAM–ATG or cyclophosphamide–ATG, are recommended to achieve the best balance of efficacy and risk in most settings. This approach is based on the latest guidelines from the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and the European Society for Blood and Marrow Transplantation (EBMT) 1. The use of autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsing forms of MS that are refractory to disease-modifying therapy (DMT) 1.
When considering AHSCT for MS treatment, it is essential to weigh the potential benefits against the risks. The recommended intermediate-intensity conditioning protocols aim to minimize toxicity while maximizing efficacy. In contrast, low-intensity regimens are not recommended outside clinical trials due to poor evidence of efficacy, and high-intensity, myeloablative conditioning protocols are not recommended outside clinical trials owing to a higher risk of toxicity 1.
Key considerations for AHSCT in MS treatment include:
- Patient selection: AHSCT is generally considered for patients with aggressive forms of relapsing-remitting MS who have failed high-efficacy DMT 1.
- Conditioning protocols: Intermediate-intensity protocols, such as BEAM–ATG or cyclophosphamide–ATG, are recommended 1.
- Monitoring and supportive care: Close monitoring and supportive care are crucial before and after AHSCT to optimize outcomes and minimize risks 1.
In summary, AHSCT with intermediate-intensity conditioning protocols is a viable treatment option for selected patients with MS, particularly those with aggressive disease who have failed other therapies. This approach should be considered in the context of individual patient needs and risk factors, and in accordance with the latest guidelines and recommendations from ECTRIMS and EBMT 1.
From the FDA Drug Label
1 INDICATIONS AND USAGE REBIF is indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults.
1 INDICATIONS AND USAGE 1. 1 Multiple Sclerosis (MS) TYSABRI is indicated as monotherapy for the treatment of relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults.
Medical Treatment Options for Multiple Sclerosis:
- Interferon beta-1a (SQ) is indicated for the treatment of relapsing forms of multiple sclerosis 2.
- Natalizumab (IV) is indicated as monotherapy for the treatment of relapsing forms of multiple sclerosis 3. Key Considerations:
- Natalizumab increases the risk of progressive multifocal leukoencephalopathy (PML), an opportunistic viral infection of the brain 3.
- The expected benefit of natalizumab should be weighed against the risk of PML when initiating and continuing treatment 3.
From the Research
Medical Treatment for Multiple Sclerosis
- Multiple sclerosis (MS) is a chronic inflammatory condition of the central nervous system, and its treatment has evolved over the years to focus on preventive therapeutic options that lessen the risk for exacerbations, changes on magnetic resonance imaging, and disability 4.
- The first disease-modifying therapy for MS was approved in 1993, and since then, several other therapies have been approved, including beta-interferons, fingolimod, glatiramer acetate, mitoxantrone, and natalizumab 5.
- Interferon beta (IFNβ) was the first disease-modifying therapy available to treat MS, and it has been shown to reduce relapse rates and delays in the onset of disability 6.
- There are four IFNβ drugs currently approved to treat relapsing forms of MS: subcutaneous (SC) IFNβ-1b, SC IFNβ-1a, intramuscular IFNβ-1a, and SC peginterferon beta-1a 6.
- Peginterferon beta-1a has an extended half-life and requires less frequent administration than other available treatments, which may improve adherence and reduce discomfort 6.
- The efficacy and long-term safety profile of interferons make them an important first-line option for treatment, although other disease-modifying therapies may be considered based on individual patient characteristics and disease stage 6, 5, 4.
Comparison of Interferons and Glatiramer Acetate
- Interferons-beta (IFNs-beta) and glatiramer acetate (GA) are two disease-modifying therapies that have been compared in head-to-head trials 7.
- The comparative effectiveness of IFNs-beta and GA on disease course has been assessed through the analysis of head-to-head trials, which showed similar clinical efficacy at 24 months, but higher relapse rates in the IFNs group at 36 months 7.
- Secondary magnetic resonance imaging (MRI) outcomes analysis showed that effects on new or enlarging T2- or new contrast-enhancing T1 lesions at 24 months were similar, but the reduction in T2- and T1-weighted lesion volume was significantly greater in the groups given IFNs than in the GA groups 7.
- The number of participants who dropped out of the study because of adverse events was similar in the two groups, and the quality of evidence for primary outcomes was judged as moderate for clinical end points, but low for safety and some MRI outcomes 7.
Treatment Strategies
- The decision to start, switch, or stop disease-modifying therapies for MS should be individualized based on patient characteristics, disease stage, and response to treatment 4.
- Patient education and mitigation strategies are key to managing adverse events and supporting therapy adherence 6.
- The potential advantage of a given treatment is condensed in a relatively limited window of opportunity for each patient, depending on individual characteristics and disease stage 4.
- A sizable proportion of patients with MS may have a very mild clinical course not requiring a disease-modifying therapy, and individual response to existing therapies for MS varies significantly across subjects 4.