Contributions
Abstract: 150
Type: Oral
Currently, 14 medications have regulatory approval to treat relapsing multiple sclerosis (MS) with several additional medications under regulatory review or in late-stage testing. As a result, clinicians and patients with MS have a broad range of options for disease therapy with differing potency, tolerability, safety, and ease of use. Accumulating evidence suggests that immunoablation followed by hematopoietic stem cell transplantation (I/HSCT) is a potential option for patients considered at high-risk for disability and with an inadequate response to other available therapies. Potential advantages include potent and durable efficacy, the potential not only for effective control of ongoing inflammatory disease activity but also possible improvement in disability, front-loaded safety and tolerability concerns, and competitive total costs compared to approved medications. However, the available evidence supporting the utility I/HSCT has a number of important shortcomings. Many of the initial studies were small and carried out at single centers. Several larger studies have been published more recently, including several multicenter efforts, but these studies either were uncontrolled or did not include a currently relevant comparator drug. In addition, although recent adaptations in the conditioning protocol have led to reduced toxicity, significant safety issues still exist. Thus, the principal issue now is not whether I/HSCT is an option for active relapsing MS. Rather, the issue is whether I/HSCT is a reasonable alternative to the highly effective therapies already available or soon to be approved or it should still be considered a “last resort.”. Until an appropriately designed head-to-head trial of I/HSCT vs. contemporary highly-effective therapy is conducted, we don"t know. There is great need for such a study. In its absence, general use of I/HSCT to treat relapsing MS is not justified.
Disclosure: Dr. Cohen reports personal compensation for consulting for Genentech, Genzyme, Novartis, and Receptos; as a speaker for Teva; and for serving as Co-Editor of Multiple Sclerosis Journal - Experimental, Translational and Clinical.
Abstract: 150
Type: Oral
Currently, 14 medications have regulatory approval to treat relapsing multiple sclerosis (MS) with several additional medications under regulatory review or in late-stage testing. As a result, clinicians and patients with MS have a broad range of options for disease therapy with differing potency, tolerability, safety, and ease of use. Accumulating evidence suggests that immunoablation followed by hematopoietic stem cell transplantation (I/HSCT) is a potential option for patients considered at high-risk for disability and with an inadequate response to other available therapies. Potential advantages include potent and durable efficacy, the potential not only for effective control of ongoing inflammatory disease activity but also possible improvement in disability, front-loaded safety and tolerability concerns, and competitive total costs compared to approved medications. However, the available evidence supporting the utility I/HSCT has a number of important shortcomings. Many of the initial studies were small and carried out at single centers. Several larger studies have been published more recently, including several multicenter efforts, but these studies either were uncontrolled or did not include a currently relevant comparator drug. In addition, although recent adaptations in the conditioning protocol have led to reduced toxicity, significant safety issues still exist. Thus, the principal issue now is not whether I/HSCT is an option for active relapsing MS. Rather, the issue is whether I/HSCT is a reasonable alternative to the highly effective therapies already available or soon to be approved or it should still be considered a “last resort.”. Until an appropriately designed head-to-head trial of I/HSCT vs. contemporary highly-effective therapy is conducted, we don"t know. There is great need for such a study. In its absence, general use of I/HSCT to treat relapsing MS is not justified.
Disclosure: Dr. Cohen reports personal compensation for consulting for Genentech, Genzyme, Novartis, and Receptos; as a speaker for Teva; and for serving as Co-Editor of Multiple Sclerosis Journal - Experimental, Translational and Clinical.