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

被引:10
作者
Patti, Francesco [1 ]
Chisari, Clara Grazia [1 ]
Toscano, Simona [1 ]
Arena, Sebastiano [1 ]
Finocchiaro, Chiara [1 ]
Cimino, Vincenzo [2 ]
Milone, Giuseppe [3 ]
机构
[1] Univ Catania, Dept Med Surg Sci & Adv Technol GF Ingrassia, I-95125 Catania, Italy
[2] IRCCS Ctr Neurolesi Bonino Pulejo, I-98124 Messina, Italy
[3] Azienda Policlin Vittorio Emanuele, Hematol & Bone Marrow Transplant Unit, I-95124 Catania, Italy
关键词
multiple sclerosis; treatment; hematopoietic stem cell transplantation; DISEASE-MODIFYING THERAPIES; LONG-TERM OUTCOMES; BRAIN ATROPHY; AUTOIMMUNE-DISEASES; COST-EFFECTIVENESS; NATALIZUMAB; IMMUNOABLATION; PLACEBO; TRIAL; SCT;
D O I
10.3390/jcm11040942
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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.
引用
收藏
页数:26
相关论文
共 109 条
[1]   Non-myeloablative autologous haematopoietic stem cell transplantation expands regulatory cells and depletes IL-17 producing mucosal-associated invariant T cells in multiple sclerosis [J].
Abrahamsson, Sofia V. ;
Angelini, Daniela F. ;
Dubinsky, Amy N. ;
Morel, Esther ;
Oh, Unsong ;
Jones, Joanne L. ;
Carassiti, Daniele ;
Reynolds, Richard ;
Salvetti, Marco ;
Calabresi, Peter A. ;
Coles, Alasdair J. ;
Battistini, Luca ;
Martin, Roland ;
Burt, Richard K. ;
Muraro, Paolo A. .
BRAIN, 2013, 136 :2888-2903
[2]   Humoral immune response to COVID-19 mRNA vaccine in patients with multiple sclerosis treated with high-efficacy disease-modifying therapies [J].
Achiron, Anat ;
Mandel, Mathilda ;
Dreyer-Alster, Sapir ;
Harari, Gil ;
Magalashvili, David ;
Sonis, Polina ;
Dolev, Mark ;
Menascu, Shay ;
Flechter, Shlomo ;
Falb, Rina ;
Gurevich, Michael .
THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS, 2021, 14
[3]   Hematopoietic stem cell therapy for autoimmune diseases - Clinical experience and mechanisms [J].
Alexander, Tobias ;
Farge, Dominique ;
Badoglio, Manuela ;
Lindsay, James O. ;
Muraro, Paolo A. ;
Snowden, John A. .
JOURNAL OF AUTOIMMUNITY, 2018, 92 :35-46
[4]   COVID-19 in hematopoietic cell transplant recipients [J].
Altuntas, Fevzi ;
Ata, Naim ;
Yigenoglu, Tugce Nur ;
Basci, Semih ;
Dal, Mehmet Sinan ;
Korkmaz, Serdal ;
Namdaroglu, Sinem ;
Basturk, Abdulkadir ;
Hacibekiroglu, Tuba ;
Dogu, Mehmet Hilmi ;
Berber, Ilhami ;
Dal, Kursat ;
Erkurt, Mehmet Ali ;
Turgut, Burhan ;
Ulgu, Mustafa Mahir ;
Celik, Osman ;
Akunal, Abdullah ;
Birinci, Suayip .
BONE MARROW TRANSPLANTATION, 2021, 56 (04) :952-955
[5]   Autologous hematopoietic SCT normalizes miR-16,-155 and-142-3p expression in multiple sclerosis patients [J].
Arruda, L. C. M. ;
Lorenzi, J. C. C. ;
Sousa, A. P. A. ;
Zanette, D. L. ;
Palma, P. V. B. ;
Panepucci, R. A. ;
Brum, D. S. ;
Barreira, A. A. ;
Covas, D. T. ;
Simoes, B. P. ;
Silva, W. A., Jr. ;
Oliveira, M. C. ;
Malmegrim, K. C. R. .
BONE MARROW TRANSPLANTATION, 2015, 50 (03) :380-389
[6]   Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial [J].
Atkins, Harold L. ;
Bowman, Marjorie ;
Allan, David ;
Anstee, Grizel ;
Arnold, Douglas L. ;
Bar-Or, Amit ;
Bence-Bruckler, Isabelle ;
Birch, Paul ;
Bredeson, Christopher ;
Chen, Jacqueline ;
Fergusson, Dean ;
Halpenny, Mike ;
Hamelin, Linda ;
Huebsch, Lothar ;
Hutton, Brian ;
Laneuville, Pierre ;
Lapierre, Yves ;
Lee, Hyunwoo ;
Martin, Lisa ;
McDiarmid, Sheryl ;
O'Connor, Paul ;
Ramsay, Timothy ;
Sabloff, Mitchell ;
Walker, Lisa ;
Freedman, Mark S. .
LANCET, 2016, 388 (10044) :576-585
[7]   Graft-versus-tumor effects after allogeneic hematopoietic cell transplantation with nonmyeloablative conditioning [J].
Baron, F ;
Maris, MB ;
Sandmaier, BM ;
Storer, BE ;
Sorror, M ;
Diaconescu, R ;
Woolfrey, AE ;
Chauncey, TR ;
Flowers, MED ;
Mielcarck, M ;
Maloney, DG ;
Storb, R .
JOURNAL OF CLINICAL ONCOLOGY, 2005, 23 (09) :1993-2003
[8]   Cost of disease modifying therapies for multiple sclerosis: Is front-loading the answer? [J].
Batcheller, Lisa ;
Baker, David .
JOURNAL OF THE NEUROLOGICAL SCIENCES, 2019, 404 :19-28
[9]   Autologous Hematopoietic Stem Cell Transplantation (AHSCT): Standard of Care for Relapsing-Remitting Multiple Sclerosis Patients [J].
Bertolotto, Antonio ;
Martire, Serena ;
Mirabile, Luca ;
Capobianco, Marco ;
De Gobbi, Marco ;
Cilloni, Daniela .
NEUROLOGY AND THERAPY, 2020, 9 (02) :197-203
[10]   Aggressive multiple sclerosis: a single-centre, real-world treatment experience with autologous haematopoietic stem cell transplantation and alemtuzumab [J].
Boffa, G. ;
Lapucci, C. ;
Sbragia, E. ;
Varaldo, R. ;
Raiola, A. M. ;
Curro, D. ;
Roccatagliata, L. ;
Capello, E. ;
Laroni, A. ;
Mikulska, M. ;
Gualandi, F. ;
Uccelli, A. ;
Angelucci, E. ;
Mancardi, G. L. ;
Inglese, M. .
EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 (10) :2047-2055