Autologous hematopoietic stem cell transplantation in multiple sclerosis

Mancardi, Gianluigi; Sormani, Maria Pia; Gualandi, Francesca; Sáiz, Albert; Carreras, E; Merelli, E.; Donelli, Amedea; Lugaresi, Alessandra; Bartolomeo, Paolo Di; Rottoli, M. R.; Rambaldi, Alessandro; Amato, Maria Pia; Massacesi, Luca; Gioia, Massimo Di; Vuolo, Luisa; Currò, Daniela; Roccatagliata, Luca; Filippi, Massimo; Aguglia, Umberto; Iacopino, Pasquale; Farge, Dominique; Saccardi, Riccardo; Transplantation, Marrow; Ebmt, Marrow Transplantation

doi:10.1212/wnl.0000000000001329

Cited by 217 publications

(190 citation statements)

References 38 publications

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“…Early adverse events were considered as expected and occurred at least in 80% of treated cases. SAE occurred in the aHSCT arm only and resolved without sequelae [31]. Despite these encouraging results, and improvement of clinical expertise, this type of treatment need to be performed only in selected centers with known neurological and hematological expertise.…”

Section: Treatment Choices: Past and Present Experiencesmentioning

confidence: 99%

Induction treatment strategy in multiple sclerosis: a review of past experiences and future perspectives

Ruggieri

Pontecorvo

Tortorella

et al. 2018

Mult Scler Demyelinating Disord

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The scenario of multiple sclerosis (MS) treatment has changed profoundly in recent decades. In this setting, one of two strategies is usually used: escalation or induction. The first involves a pyramid of possible treatments of increasing efficacy (but also increasing safety risks) that are introduced progressively as needed. The induction strategy, on the other hand, immediately pursues higher efficacy, since drugs with a higher risk profile are used from the outset. Understanding which of these treatment strategies is the more suitable for a given patient is the first step in the therapeutic decision-making process. Prognostic factors evaluated on the basis of the clinical presentation and any disease activity on magnetic resonance imaging (MRI) should guide and help clinicians in making their choices. Even though the pathogenesis of MS is not yet completely understood, specific pathological changes are known to occur in the adaptive and innate immune system over the course of the disease. To date, treatment has been based mainly on two drugs, mitoxantrone and cyclophosphamide, autologous haematopoietic stem cell therapy (within clinical trial setting), but new compounds are now emerging. Among the new treatments, alemtuzumab and cladribine appear to be valid candidates as induction drugs. In this review we provide an overview of induction strategies based on literature evidence and our own past experiences, providing descriptions of clinical cases. We also outline the future perspectives in this field.

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Section: Treatment Choices: Past and Present Experiencesmentioning

confidence: 99%

Induction treatment strategy in multiple sclerosis: a review of past experiences and future perspectives

Ruggieri

Pontecorvo

Tortorella

et al. 2018

Mult Scler Demyelinating Disord

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“…It was also reported that patients with Gd-enhancing activity at baseline fared significantly better than patients without Gd-enhancing lesions 1,33 , pointing out that the clinical effect of aHSCT is mainly due to the capacity to suppress the inflammatory activity. Recently, a series of papers has unequivocally demonstrated the effect of aHSCT on MRI signs of activity and on the clinical outcome in severe forms of MS. A comparative randomized phase II trial evaluating aHSCT vs mitoxantrone -the ASTIMS trial-demonstrated that the number of new T2 lesions in a 4 year period was decreased of 79% in patients treated with aHSCT vs the cases that received mitoxantrone 7 . In the observational study reporting the Swedish experience 1 and in the prospective HALT study in USA 4,5 , patients with a RR form of the disease who experienced relapses and progression of the disease before treatment were followed for a median follow up of 5 years, demonstrating a sustained remission of disease without progression of disability in 77% and 80% of cases respectively.…”

Section: The Second Period From 2005 To 2017mentioning

confidence: 99%

Intense immunosuppression followed by autologous haematopoietic stem cell transplantation as a therapeutic strategy in aggressive forms of multiple sclerosis

et al. 2017

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Abstract:In the majority of relapsing multiple sclerosis patients the disease can be quite easily controlled by the already available and approved therapies. There are, however, some aggressive cases that continue to have clinical and MRI activity in spite of the treatment. These are the cases that may now receive benefit from intense immunosuppression followed by autologous haematopoietic stem cell transplantation (aHSCT). In this review we describe the method and the rationale of aHSCT, the more recently published studies that demonstrate its efficacy in selected multiple sclerosis cases, the problems related to the safety, and transplant related mortality risk of the procedure. Description of the ideal patient that can take advantage from aHSCT is outlined and, finally, the ongoing studies that are near to completition or are close to starting are briefly reported.

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“…One would probably not opt for fingolimod in that situation if the expected disease activity is very high despite very effective therapy. A few centers offer autologous stem cell transplantation in such a situation [97], but this approach is still in development and safety and efficacy still have to be assessed in larger cohorts. Breakthrough disease is, however, not the only reason for a need to switch therapies.…”

Section: Switching Between Second-line Therapiesmentioning

confidence: 99%

Advances in and Algorithms for the Treatment of Relapsing-Remitting Multiple Sclerosis

2016

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Treatment options in relapsing-remitting multiple sclerosis have increased considerably in recent years; currently, a dozen different preparations of diseasemodifying therapies are available and some more are expected to be marketed soon. For the treating neurologist this broad therapeutic repertoire not only greatly improves individualized management of the disease, but also makes choices more complex and difficult. A number of factors must be considered, including disease activity and severity, safety profile, and patient preference. We here discuss the currently existing options and suggest treatment algorithms for managing relapsing-remitting multiple sclerosis.

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Autologous hematopoietic stem cell transplantation in multiple sclerosis

Abstract: Intense immunosuppression followed by AHSCT is significantly superior to MTX in reducing MRI activity in severe cases of MS. These results strongly support further phase III studies with primary clinical endpoints. The study was registered as EUDRACT No. 2007-000064-24.

Cited by 217 publications

References 38 publications

Induction treatment strategy in multiple sclerosis: a review of past experiences and future perspectives

Induction treatment strategy in multiple sclerosis: a review of past experiences and future perspectives

Intense immunosuppression followed by autologous haematopoietic stem cell transplantation as a therapeutic strategy in aggressive forms of multiple sclerosis

Advances in and Algorithms for the Treatment of Relapsing-Remitting Multiple Sclerosis

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