Disclosures: Miller reports nonfinancial support from Sanofi/Genzyme, research support from Mallinckrodt and MedDay, personal fees and research support from Novartis, and personal fees from AbbVie, Alexion, Biogen, Bristol-Myers Squibb, Corrona, EMD Serono, Mapi Pharma, Medpage, Medscape, Roche/Genentech and Verana Health outside the submitted work. Please see the study for all other authors’ relevant financial disclosures.
November 03, 2020
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National MS Society issues guidance on autologous hematopoietic stem cell transplant

Disclosures: Miller reports nonfinancial support from Sanofi/Genzyme, research support from Mallinckrodt and MedDay, personal fees and research support from Novartis, and personal fees from AbbVie, Alexion, Biogen, Bristol-Myers Squibb, Corrona, EMD Serono, Mapi Pharma, Medpage, Medscape, Roche/Genentech and Verana Health outside the submitted work. Please see the study for all other authors’ relevant financial disclosures.
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Autologous hematopoietic stem cell transplant represents “a useful treatment option” for patients with relapsing MS who experience breakthrough disease activity despite disease-modifying therapy, according to the National MS Society.

The National MS Society stated that autologous hematopoietic stem cell transplant (AHSCT) is also efficacious for patients with relapsing MS and breakthrough disease activity who have contraindications to “high-efficacy” disease-modifying therapy (DMT). Researchers published the recommendations on AHSCT for relapsing MS in JAMA Neurology, where they outlined specific criteria that make patients with relapsing MS “the best candidates” for this treatment.

Aaron Miller

“Prior research suggests a long-lasting benefit of AHSCT in relapsing MS, but studies are mostly uncontrolled and small,” Aaron Miller, MD, medical director of the Corrine Goldsmith Dickinson Center for MS and professor of neurology at the Icahn School of Medicine at Mount Sinai Hospital, told Healio Neurology. “There have been several studies published in the last few years and patients increasingly inquire about stem cell therapies. However, they are often confused about the difference between stem cell therapies. AHSCT primarily works through the process of immunoablation.”

To evaluate the use of AHSCT in MS, Miller and other members of the National Medical Advisory Committee of the National Multiple Sclerosis Society examined recent literature on the topic to make recommendations about its use. They explored which patients with MS make the best candidates for AHSCT, the optimal treatment locations for this procedure and the protocols for AHSCT in patients with MS, as well as the treatment course and follow-up for the procedure.

Studies examining the use of AHSCT in relapsing MS “have repeatedly demonstrated high efficacy and a durable outcome,” the researchers wrote, and a substantial improvement in the safety of the procedure, including a decrease in mortality rates. While agreement about the characteristics of the best candidates for the procedure is evolving, questions persist about the ideal protocol, especially the best conditioning regimen used for killing the immune cells. Miller and colleagues wrote that larger randomized clinical trials are necessary to determine whether AHSCT is advantageous compared with the most efficacious DMT currently available to patients with relapsing MS; one such trial — Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis [BEAT-MS) — is in progress.

Available evidence and recommendations from the International Conference on Cell-Based Therapies for Multiple Sclerosis and the Society of Blood and Bone Marrow indicated AHSCT “may be a useful treatment for people with MS who demonstrate substantial breakthrough disease activity” in spite of treatment with highly efficacious DMT, according to the researchers. They defined substantial breakthrough disease activity as new inflammatory central nervous system lesions and/or clinical relapses. The procedure also may be effective for patients with contraindications to high-efficacy DMTs who are younger than 50 years and have a disease duration of less than 10 years, according to the researchers.

Additional recommendations include:

  • AHSCT for people with MS should only occur at centers with experience and expertise in both MS care and stem cell transplant;
  • People with MS treated with AHSCT should be entered into a single database for long-term follow-up;
  • Research is needed to establish standards for cell mobilization and immune-conditioning regimens; and
  • Continuing research on comparative effectiveness of AHSCT and high-efficacy DMT is needed.

“This treatment would likely be potentially employed in patients with relapsing MS who are failing other DMT. MS disease activity continues to be uncontrolled with disease-modifying medications in some patients,” Miller said. “Also, if AHSCT can be performed with low morbidity and mortality, it could offer a less expensive and more durably effective treatment option.”