Autologous hematopoietic stem-cell transplantation in neurological disorders: current approach and future directions

被引:9
作者
Das, Joyutpal [1 ,2 ,3 ]
Sharrack, Basil [3 ]
Snowden, John A. [4 ]
机构
[1] Salford Royal NHS Fdn Trust, Manchester Acad Hlth Sci Ctr, Clin Neurosci, Salford M6 8HD, Lancs, England
[2] Univ Manchester, Cardiovasc Med, Manchester, Lancs, England
[3] Univ Sheffield, Sheffield Teaching Hosp NHS Fdn Trust, NIHR Translat Neurosci BRC, Dept Neurosci, Sheffield, S Yorkshire, England
[4] Sheffield Teaching Hosp NHS Fdn Trust, Dept Hematol, Sheffield, S Yorkshire, England
关键词
Autologous hematopoietic stem-cell transplantation; multiple sclerosis; neuromyelitis optica spectrum disorder; stiff-person spectrum disorder; chronic inflammatory demyelinating polyneuropathy; POEMS syndrome; myasthenia gravis; INFLAMMATORY DEMYELINATING POLYNEUROPATHY; AGGRESSIVE MULTIPLE-SCLEROSIS; REFRACTORY MYASTHENIA-GRAVIS; STIFF PERSON SYNDROME; DOSE IMMUNOSUPPRESSIVE THERAPY; RELAPSING-NEUROMYELITIS-OPTICA; SYSTEMIC-LUPUS-ERYTHEMATOSUS; DISEASE-MODIFYING THERAPIES; LONG-TERM OUTCOMES; 5-YEAR FOLLOW-UP;
D O I
10.1080/14737175.2020.1820325
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Introduction Autologous hematopoietic stem-cell transplantation (AHSCT) has become increasingly popular in recent years as an effective treatment of immune-mediated neurological diseases. Treatment-related mortality has significantly reduced primarily through better patient selection, optimization of transplant technique, and increased center experience. Area covered Multiple sclerosis is the main indication, but people with neuromyelitis optica spectrum disorder, stiff-person spectrum disorder, chronic inflammatory demyelinating polyneuropathy, myasthenia gravis, and other immune-mediated neurological disorders also have been treated. The review herein discusses the use of AHSCT in these neurological disorders, the importance of patient selection and transplant technique optimization and future directions. Expert opinion Phase II and III clinical trials have confirmed the safety and efficacy of AHSCT in multiple sclerosis and recent phase II clinical trials have also suggested its safety and efficacy in chronic inflammatory demyelinating polyneuropathy and neuromyelitis optica spectrum disorder, with the evidence in other neurological disorders limited to individual case reports, small case series, and registry data. Therefore, further randomized controlled clinical trials are required to assess its safety and efficacy in other neurological conditions. However, in rare neurological conditions, pragmatic treatment trials or registry-based studies may be more realistic options for gathering efficacy and safety data.
引用
收藏
页码:1299 / 1313
页数:15
相关论文
共 174 条
  • [1] Response to treatment of myasthenia gravis according to clinical subtype
    Akaishi, Tetsuya
    Suzuki, Yasushi
    Imai, Tomihiro
    Tsuda, Emiko
    Minami, Naoya
    Nagane, Yuriko
    Uzawa, Akiyuki
    Kawaguchi, Naoki
    Masuda, Masayuki
    Konno, Shingo
    Suzuki, Hidekazu
    Murai, Hiroyuki
    Aoki, Masashi
    Utsugisawa, Kimiaki
    [J]. BMC NEUROLOGY, 2016, 16
  • [2] Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus: data from the European Group for Blood and Marrow Transplantation registry
    Alchi, B.
    Jayne, D.
    Labopin, M.
    Kotova, O.
    Sergeevicheva, V.
    Alexander, T.
    Gualandi, F.
    Gruhn, B.
    Ouyang, J.
    Rzepecki, P.
    Held, G.
    Sampol, A.
    Voswinkel, J.
    Ljungman, P.
    Fassas, A.
    Badoglio, M.
    Saccardi, R.
    Farge, D.
    [J]. LUPUS, 2013, 22 (03) : 245 - 253
  • [3] CIDP diagnostic pitfalls and perception of treatment benefit
    Allen, Jeffrey A.
    Lewis, Richard A.
    [J]. NEUROLOGY, 2015, 85 (06) : 498 - 504
  • [4] Holder regularity for nondivergence nonlocal parabolic equations
    Allen, Mark
    [J]. CALCULUS OF VARIATIONS AND PARTIAL DIFFERENTIAL EQUATIONS, 2018, 57 (04)
  • [5] The TCR Repertoire Reconstitution in Multiple Sclerosis: Comparing One-Shot and Continuous Immunosuppressive Therapies
    Amoriello, Roberta
    Greiff, Victor
    Aldinucci, Alessandra
    Bonechi, Elena
    Carnasciali, Alberto
    Peruzzi, Benedetta
    Repice, Anna Maria
    Mariottini, Alice
    Saccardi, Riccardo
    Mazzanti, Benedetta
    Massacesi, Luca
    Ballerini, Clara
    [J]. FRONTIERS IN IMMUNOLOGY, 2020, 11
  • [6] [Anonymous], 2014, J NEUROL NEUROSUR PS, DOI DOI 10.1136/jnnp-2013-305450
  • [7] Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant
    Aouad, Patrick
    Li, Jamma
    Arthur, Chris
    Burt, Richard
    Fernando, Suran
    Parratt, John
    [J]. JOURNAL OF CLINICAL NEUROSCIENCE, 2015, 22 (07) : 1215 - 1217
  • [8] Homeostatic proliferation leads to telomere attrition and increased PD-1 expression after autologous hematopoietic SCT for systemic sclerosis
    Arruda, Lucas C. M.
    Lima-Junior, Joao R.
    Clave, Emmanuel
    Moraes, Daniela A.
    Douay, Corinne
    Fournier, Isabelle
    Moins-Teisserenc, Helene
    Covas, Dimas T.
    Simoes, Belinda P.
    Farge, Dominique
    Toubert, Antoine
    Malmegrim, Kelen C. R.
    Oliveira, Maria Carolina
    [J]. BONE MARROW TRANSPLANTATION, 2018, 53 (10) : 1319 - 1327
  • [9] A population-based study of neuromyelitis optica in Caucasians
    Asgari, N.
    Lillevang, S. T.
    Skejoe, H. P. B.
    Falah, M.
    Stenager, E.
    Kyvik, K. O.
    [J]. NEUROLOGY, 2011, 76 (18) : 1589 - 1595
  • [10] Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial
    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.
    [J]. LANCET, 2016, 388 (10044) : 576 - 585