Long-term follow-up more than 10 years after HSCT: a monocentric experience

Frau, Jessica; Carai, Margherita; Coghe, Giancarlo; Fenu, Giuseppe; Lorefice, Lorena; Nasa, Giorgio La; Mamusa, E; Vacca, Adriana; Marrosu, Maria Giovanna; Cocco, Eleonora

doi:10.1007/s00415-017-8718-2

Cited by 11 publications

(4 citation statements)

References 29 publications

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“…In this analysis, with the exception of three cases of myelodysplastic syndromes typically associated with exposure to cytotoxic drugs, no organ-specific prevalence was detected [9]. Moreover, in the current literature, six malignancies occurred, where one of them was reported in a patient treated with DMT after AHSCT [32,33,71]. Notably, an increased risk of malignancies was reported in patients who received allogeneic HSCT (allo-HSCT) [75].…”

Section: Late Complicationsmentioning

confidence: 62%

“…Among viral reactivations, progressive multifocal leukoencephalopathy (PML), a severely disabling and potentially life-threatening disease caused by the reactivation of John Cunningham virus (JCV), mainly in immunosuppressed patients, has raised concerns in the last decade as a potential adverse event of several DMTs, particularly natalizumab [63][64][65]. According to the current literature, only one case of PML has been reported 11 years after an AHSCT in a patient who had received natalizumab treatment for about 3 years, and thus, it could not be considered as related to the AHSCT [71]. Therefore, to date, no cases of PML associated with AHSCT have been reported so far in MS patients.…”

Section: Late Complicationsmentioning

confidence: 99%

“…Thereafter, during the study, in order to reduce regimen-related toxicity, the dose and route of administration of busulfan were changed [27]. In most of the remaining studies, the mortality was 0% [25,26,[28][29][30][31][32][33][34]46,71]. According to recent meta-analyses, patients with specific clinical and demographic features, such as higher disability at baseline, older age and a non-RRMS course, were associated with higher mortality risk [39,40,79].…”

Section: Late Complicationsmentioning

confidence: 99%

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Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Patients: Monocentric Case Series and Systematic Review of the Literature

Patti

Chisari

Toscano

et al. 2022

JCM

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Multiple sclerosis (MS) is a chronic, inflammatory and immune-mediated disease of the central nervous system (CNS), commonly affecting young adults and potentially associated with life-long disability. About 14 disease-modifying treatments (DMTs) are currently approved for the treatment of MS. However, despite the use of highly effective therapies, some patients exhibit a highly active disease with an aggressive course from onset and a higher risk of long-term disability accrual. In the last few years, several retrospective studies, clinical trials, meta-analyses and systematic reviews have investigated autologous hematopoietic stem cell transplantation (AHSCT) as a possible therapeutic option in order to address this unmet clinical need. These studies demonstrated that AHSCT is a highly efficacious and relatively safe therapeutic option for the treatment of highly active MS. Particularly, over recent years, the amount of evidence has grown, with significant improvements in the development of patient selection criteria, choice of the most suitable transplant technique and clinical experience. In this paper, we present six patients who received AHSCT in our MS center and we systematically reviewed recent evidence about the long-term efficacy and safety of AHSCT and the placement of AHSCT in the rapidly evolving therapeutic armamentarium for MS.

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Section: Late Complicationsmentioning

confidence: 62%

Section: Late Complicationsmentioning

confidence: 99%

Section: Late Complicationsmentioning

confidence: 99%

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Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Patients: Monocentric Case Series and Systematic Review of the Literature

Patti

Chisari

Toscano

et al. 2022

JCM

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“…The calculated PFS one year after HSCT was 100% in the small study by Dayama et al [ 33 ]. Frau et al followed nine patients for a median of 11 years, and reported a notable reduction in the number of relapses two years after transplant [ 34 ]. The final EDSS improved in 22.22%, stabilized in 22.22%, and worsened in 55.6%.…”

Section: Reviewmentioning

confidence: 99%

Stem Cell Transplantation for Multiple Sclerosis: A 2023 Review of Published Studies

Msheik,

Assi,

Hamed

et al. 2023

Cureus

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This comprehensive literature review underscores the potential of stem cell transplantation (SCT) as a therapeutic intervention for multiple sclerosis (MS). By amalgamating evidence from various sources, including randomized controlled trials (RCTs), observational, retrospective, and comparative studies, this review offers a holistic understanding of SCT's effectiveness, safety, and feasibility in diverse contexts of MS management. SCT has shown promise in mitigating disease activity and progression, particularly in relapsing-remitting MS (RRMS). RCTs like the high dose immunoablation and autologous hematopoietic stem cell transplantation in MS (ASTIMS) versus mitoxantrone therapy in severe multiple sclerosis and multiple sclerosis international stem cell transplant (MIST) trials reveal SCT's capacity to reduce new lesion occurrences and inflammatory activity. However, variability exists in disability score improvements among these studies. Observational and retrospective investigations further affirm SCT's potential, highlighting decreased relapse rates, enhanced expanded disability status scale (EDSS) scores, and a noteworthy proportion of patients achieving no evidence of disease activity (NEDA). The initial literature search using all of the search items produced a total of 3,636 articles. After title, abstract, and article type screening and article retrieving, 147 articles were assessed for eligibility using the inclusion criteria. At the end of the literature search, 37 articles met the eligibility criteria. They were included in our review according to preferred reporting items for systematic reviews and meta-analyses (PRISMA) guidelines. Patients treated with hematopoietic stem cell transplantation (HSCT) present lower progression and relapse rates, suppression of inflammatory activity, and a greater reduction in T2 lesions on MRI than those treated with disease-modifying therapies (DMTs). In summary, while SCT presents promise as a therapeutic option for MS, its deployment should be tailored to individual patient characteristics, disease stages, and responses.

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The current standing of autologous haematopoietic stem cell transplantation for the treatment of multiple sclerosis

et al. 2022

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Autologous haematopoietic stem cell transplantation (aHSCT) is gaining traction as a valuable treatment option for patients affected by severe multiple sclerosis (MS), particularly the relapsing–remitting form. We describe the current literature in terms of clinical trials, observational and retrospective studies, as well as immune reconstitution following transplantation, with a focus on the conditioning regimens used for transplantation. The evidence base predominantly consists of non-randomised, uncontrolled clinical trials or data from retrospective or observational cohorts, i.e. very few randomised or controlled trials. Most often, intermediate-intensity conditioning regimens are used, with promising results from both myeloablative and lymphoablative strategies, as well as from regimens that are low and high intensity. Efficacy of transplantation, which is likely secondary to immune reconstitution and restored immune tolerance, is, therefore, not clearly dependent on the intensity of the conditioning regimen. However, the conditioning regimen may well influence the immune response to transplantation. Heterogeneity of conditioning regimens among studies hinders synthesis of the articles assessing post-aHSCT immune system changes. Factors associated with better outcomes were lower Kurtzke Expanded Disability Status Scale, relapsing–remitting MS, younger age, and shorter disease duration at baseline, which supports the guidance for patient selection proposed by the European Society for Blood and Marrow Transplantation. Interestingly, promising outcomes were described for patients with secondary progressive MS by some studies, which may be worth taking into account when considering treatment options for patients with active, progressive disease. Of note, a significant proportion of patients develop autoimmune disease following transplantation, with alemtuzumab-containing regimens associated with the highest incidence.

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Long-term follow-up more than 10 years after HSCT: a monocentric experience

Abstract: Our results confirm in a long follow-up the efficacy of HSCT in reducing relapses and disability progression. The risk/benefit profile is better for intermediate intensity regimens.

Cited by 11 publications

References 29 publications

Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Patients: Monocentric Case Series and Systematic Review of the Literature

Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Patients: Monocentric Case Series and Systematic Review of the Literature

Stem Cell Transplantation for Multiple Sclerosis: A 2023 Review of Published Studies

The current standing of autologous haematopoietic stem cell transplantation for the treatment of multiple sclerosis

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