Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Acc

Abstract:

:These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

journal_name

Bone Marrow Transplant

authors

Sharrack B,Saccardi R,Alexander T,Badoglio M,Burman J,Farge D,Greco R,Jessop H,Kazmi M,Kirgizov K,Labopin M,Mancardi G,Martin R,Moore J,Muraro PA,Rovira M,Sormani MP,Snowden JA,European Society for Blood and Marrow Tr

doi

10.1038/s41409-019-0684-0

subject

Has Abstract

pub_date

2020-02-01 00:00:00

pages

283-306

issue

2

eissn

0268-3369

issn

1476-5365

pii

10.1038/s41409-019-0684-0

journal_volume

55

pub_type

杂志文章
  • Unrelated donor stem cell transplantation in adult patients with thalassemia.

    abstract::Allogeneic SCT remains the only potential cure for patients with thalassemia. However, most BMT candidates lack a suitable family donor and require an unrelated donor (UD). We evaluated whether BMT using UDs in high-risk adult thalassemia patients can offer a probability of cure comparable to that reported employing a...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究

    doi:10.1038/sj.bmt.1705173

    authors: La Nasa G,Caocci G,Argiolu F,Giardini C,Locatelli F,Vacca A,Orofino MG,Piras E,Addari MC,Ledda A,Contu L

    更新日期:2005-12-01 00:00:00

  • Population pharmacokinetics of cyclophosphamide in patients with thalassemia major undergoing HSCT.

    abstract::CY in combination with BU is a widely used conditioning regimen for haematopoietic SCT (HSCT). The aim of this study was to evaluate the pharmacokinetics (PK) of CY and its major metabolite 4-hydroxyCY (HCY) in patients with thalassemia undergoing HSCT. A total of 55 patients received BU (16 mg/kg) followed by CY (160...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2011.254

    authors: Balasubramanian P,Desire S,Panetta JC,Lakshmi KM,Mathews V,George B,Viswabandya A,Chandy M,Krishnamoorthy R,Srivastava A

    更新日期:2012-09-01 00:00:00

  • Are backup BM harvests worthwhile in unrelated donor allogeneic transplants?

    abstract::The Cleveland Clinic blood and marrow transplant program has routinely performed 'backup' autologous harvests in unrelated recipients with hematological malignancies in remission, lymphoma without marrow involvement and CML in chronic phase. We reviewed all adult or cord unrelated donor (URD) transplants performed fro...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2009.95

    authors: Stotler C,Bolwell B,Sobecks R,Dean R,Serafino S,Rybicki L,Andresen S,Pohlman B,Kalaycio M,Copelan E

    更新日期:2010-01-01 00:00:00

  • Impact of IKZF1 deletions on long-term outcomes of allo-SCT following imatinib-based chemotherapy in adult Philadelphia chromosome-positive ALL.

    abstract::We investigated the prognostic relevance of IKZF1 deletions in 118 adult Ph-positive ALL patients who had minimal residual disease (MRD) data under a uniform treatment of allo-SCT following first-line imatinib-based chemotherapy. IKZF1 deletions were identified in 93 patients (78.8%). IKZF1-deleted patients had a lowe...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2014.281

    authors: Kim M,Park J,Kim DW,Kim YJ,Jeon YW,Yoon JH,Shin SH,Yahng SA,Lee SE,Cho BS,Eom KS,Kim HJ,Min CK,Cho SG,Kim Y,Lee JW,Han K,Min WS,Lee S

    更新日期:2015-03-01 00:00:00

  • Efficacy of prophylactic transfusions using single donor apheresis platelets versus pooled platelet concentrates in AML/MDS patients receiving allogeneic hematopoietic stem cell transplantation.

    abstract::Superiority of single-donor apheresis platelets (SDAP) over pooled platelet concentrates (PPC) transfusions is largely assumed, but unproven. We hypothesized that prophylactic SDAP and PPC transfusions are clinically equivalent after allogeneic hematopoietic stem cell transplants (HSCT). We studied all transfusions ad...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1705751

    authors: Gurkan E,Patah PA,Saliba RM,Ramos CA,Anderson BS,Champlin R,de Lima M,Lichtiger B

    更新日期:2007-09-01 00:00:00

  • Optimal timing for collection of PBPC after glycosylated G-CSF administration.

    abstract::Daily administration of granulocyte colony-stimulating Factor (G-CSF) results in progenitor cell mobilization with maximum blood levels achieved after 4-7 days. In this study the short-term effects of glycosylated G-CSF at a dose of 5 microg/kg s.c. were determined so as to allow optimization of the timing of progenit...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701099

    authors: Watts MJ,Addison I,Ings SJ,Long SG,Hartley S,Warrington S,Boyce M,Linch DC

    更新日期:1998-02-01 00:00:00

  • The role of cytokines following bone marrow transplantation: indications and controversies.

    abstract::The use of cytokines following bone marrow transplantation has become an area of increasing controversy with specific regard to indication, efficacy and cost. This review will attempt to summarize cytokine data in both adult and pediatric transplantation. Indications for cytokines following transplantation will be pre...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:

    authors: Weinthal JA

    更新日期:1996-12-01 00:00:00

  • Marked reduction in the incidence of hepatic veno-occlusive disease after allogeneic hematopoietic stem cell transplantation with CD34(+) positive selection.

    abstract::Veno-occlusive disease of the liver (VOD) is a common and severe complication of allogeneic hematopoietic stem cell transplantation (HSCT). To determine the incidence of, and the risk factors for the development of VOD, we performed a retrospective analysis of a series of 178 patients, who underwent allogeneic HSCT at...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1703025

    authors: Moscardó F,Sanz GF,de La Rubia J,Jiménez C,Saavedra S,Regadera A,Andreu R,García I,Plumé G,Martínez J,Martín G,Jarque I,Sanz MA

    更新日期:2001-05-01 00:00:00

  • Bone marrow steady-state CD34+/CD71- cell content is a predictive value of rG-CSF-mobilized CD34+ cells.

    abstract::Thirty-four patients diagnosed with breast cancer were included in a prospective study evaluating the bone marrow (BM) CD34+/CD71- cell content, as a predictive parameter of the CD34+ cell mobilization after rG-CSF administration. Analysis of the concentration of medullary CD34+/CD71- cells before priming schedules wa...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701227

    authors: Osma MM,Ortuño F,de Arriba F,Lozano ML,Heras I,Moraleda JM,Vicente V

    更新日期:1998-05-01 00:00:00

  • Incidence and severity of crucial late effects after allogeneic HSCT for malignancy under the age of 3 years: TBI is what really matters.

    abstract::Younger children are considered to be more vulnerable to late effects (LE), which prompted us to study LE in patients after haematopoietic stem cell transplantation (HSCT) for a haematological malignancy before the age of 3. In this multicentre EBMT study, cumulative incidence (CI) and severity of endocrine LE, centra...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/bmt.2016.139

    authors: Bresters D,Lawitschka A,Cugno C,Pötschger U,Dalissier A,Michel G,Vettenranta K,Sundin M,Al-Seraihy A,Faraci M,Sedlacek P,Versluys AB,Jenkins A,Lutz P,Gibson B,Leiper A,Diaz MA,Shaw PJ,Skinner R,O'Brien TA,Salooja

    更新日期:2016-11-01 00:00:00

  • Are new conditioning regimens for transplants in acute myelogenous leukemia better?

    abstract::Pretransplant conditioning regimens designed to decrease leukemia relapse and increase leukemia-free survival in allotransplants in acute myelogenous leukemia (AML) were developed recently. We review studies of new regimens containing drugs like busulfan, cytarabine, melphalan or etoposide as well as novel radiation s...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:

    authors: Aurer I,Gale RP

    更新日期:1991-04-01 00:00:00

  • Graft-versus-adult T-cell leukemia/lymphoma effect following allogeneic hematopoietic stem cell transplantation.

    abstract::Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has proven effective in adult T-cell leukemia/lymphoma (ATL) patients. To study the graft-versus-ATL (Gv-ATL) effects after allo-HSCT, we analyzed 21 ATL patients who had been treated at our hospital. Of these, 18 had acute-, 2 had lymphoma- and 1 had chro...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2008.39

    authors: Yonekura K,Utsunomiya A,Takatsuka Y,Takeuchi S,Tashiro Y,Kanzaki T,Kanekura T

    更新日期:2008-06-01 00:00:00

  • Ethical problems in bone marrow transplantation in children.

    abstract::The medical staff caring for children with hematological or oncological diseases is often faced with delicate ethical and, at times, legal problems. Although many of these are common to other branches of pediatrics where patients are children lacking decision-making capacity, others, such as bone marrow transplantatio...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:

    authors: Massimo L

    更新日期:1996-11-01 00:00:00

  • SCT for severe autoimmune diseases: consensus guidelines of the European Society for Blood and Marrow Transplantation for immune monitoring and biobanking.

    abstract::Over the past 15 years, SCT has emerged as a promising treatment option for patients with severe autoimmune diseases (ADs). Mechanistic studies recently provided the proof-of-concept that restoration of immunological tolerance can be achieved by haematopoietic SCT in chronic autoimmunity through eradication of the pat...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,评审

    doi:10.1038/bmt.2014.251

    authors: Alexander T,Bondanza A,Muraro PA,Greco R,Saccardi R,Daikeler T,Kazmi M,Hawkey C,Simoes BP,Leblanc K,Fibbe WE,Moore J,Snarski E,Martin T,Hiepe F,Velardi A,Toubert A,Snowden JA,Farge D

    更新日期:2015-02-01 00:00:00

  • Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation.

    abstract::We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and poten...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/s41409-018-0155-z

    authors: Duncan CN,Brazauskas R,Huang J,Shaw BE,Majhail NS,Savani BN,Flowers MED,Battiwalla M,Beebe K,Dietz AC,Dvorak CC,Giller R,Jacobsohn DA,Kletzel M,Martin PL,Nemecek ER,Nuechterlein B,Talano JA,Pulsipher MA,Baker KS

    更新日期:2018-10-01 00:00:00

  • Long-term outcome of HLA-haploidentical hematopoietic stem cell transplantation without in vitro T-cell depletion based on an FBCA conditioning regimen for hematologic malignancies.

    abstract::Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is an alternative for patients who need a transplant without having conventional donors. One hundred and five consecutive patients with hematologic malignancies who underwent G-CSF-primed peripheral blood haplo-HSCT without in vitro T-cell depletion i...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/bmt.2015.108

    authors: Lin X,Lu ZG,Song CY,Huang YX,Guo KY,Deng L,Tu SF,He YZ,Xu JH,Long H,Wu BY

    更新日期:2015-08-01 00:00:00

  • Results of single and double autografts for high-risk neuroblastoma patients.

    abstract::Although intensive therapy with autologous bone marrow transplantation (ABMT) has improved the outcome of advanced neuroblastoma, nearly half the patients with this disease still relapse after a single ABMT. In our previous study, 10 of 22 patients relapsed within 16 months post-transplantation. Predictive risk-factor...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Kawa-Ha K,Yumura-Yagi K,Inoue M,Park DY,Okamura T,Yasui M,Oota H,Sakata N,Yoneda M,Imura K

    更新日期:1996-06-01 00:00:00

  • The role of granulocyte colony-stimulating factor (G-CSF) in the post-transplant period.

    abstract::The administration of G-CSF post transplant has been shown to accelerate the time to neutrophil engraftment. However, this does not necessarily translate into a meaningful clinical benefit to the patient. This randomized study was designed to determine the role of G-CSF following transplantation in patients with breas...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章,多中心研究,随机对照试验

    doi:10.1038/sj.bmt.1703539

    authors: Hornedo J,Solá C,Solano C,López JJ,Alonso S,Lluch A,Ojeda B,Garcia-Conde J,Cortés-Funes H,SOLTI Group.

    更新日期:2002-05-01 00:00:00

  • Cytomegalovirus-specific immune recovery following allogeneic HLA-identical sibling transplantation with reduced-intensity preparative regimen.

    abstract::Cytomegalovirus (CMV) represents a major cause of morbidity after allogeneic stem cell transplantation (allo-SCT). Using interferon-gamma-enzyme-linked immunospot (ELISPOT) assay and HLA-peptide tetramers, we analysed 54 patients who received a reduced-intensity conditioning regimen, including fludarabine, busulphan a...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704442

    authors: Mohty M,Mohty AM,Blaise D,Faucher C,Bilger K,Isnardon D,Sainty D,Gastaut JA,Viens P,Olive D,Gaugler B

    更新日期:2004-04-01 00:00:00

  • Low incidence of molecular evidence for tumour in PBPC harvests from patients with high risk Ewing tumours.

    abstract::Reverse transcriptase polymerase chain reaction (RT-PCR) was applied to evaluate the frequency of tumour cells in PBPC products from 15 high risk Ewing tumour (ET) patients who were treated according to EICESS 92 with high-dose chemotherapy (HDC) and stem cell rescue. Initial tumour cell contamination of the bone marr...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1701924

    authors: Fischmeister G,Zoubek A,Jugovic D,Witt V,Ladenstein R,Fritsch G,Höcker P,Gadner H,Kovar H

    更新日期:1999-08-01 00:00:00

  • Bone marrow transplantation for patients with chronic myeloid leukaemia: T-cell depletion with Campath-1 reduces the incidence of graft-versus-host disease but may increase the risk of leukaemic relapse.

    abstract::Between December 1983 and November 1985 we treated 39 patients with chronic myeloid leukaemia by chemoradiotherapy and transplantation from HLA-identical sibling donors using bone marrow that had been depleted of T cells ex vivo with the rat monoclonal antibody Campath-1. Twenty-eight of the patients were in the chron...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Apperley JF,Jones L,Hale G,Waldmann H,Hows J,Rombos Y,Tsatalas C,Marcus RE,Goolden AW,Gordon-Smith EC

    更新日期:1986-05-01 00:00:00

  • Salvage haploidentical transplantation for graft failure using reduced-intensity conditioning.

    abstract::Graft failure is a major concern after cord blood transplantation (CBT) or HLA-haploidentical transplantation (haplo-SCT). As patients who undergo CBT or haplo-SCT almost always lack both matched-related and -unrelated donors, salvage transplantation would also be limited to either CBT or haplo-SCT. In this study, we ...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2011.84

    authors: Yoshihara S,Ikegame K,Taniguchi K,Kaida K,Kim EH,Nakata J,Kato R,Inoue T,Fujioka T,Tamaki H,Okada M,Soma T,Ogawa H

    更新日期:2012-03-01 00:00:00

  • Tandem autologous non-myeloablative allogeneic transplantation in patients with multiple myeloma relapsing after a first high dose therapy.

    abstract::We retrospectively studied a series of 23 patients (median age 50 years, range 29-59 years) with multiple myeloma (MM), treated in first relapse by a sequential autologous-allogeneic tandem approach. Tandem transplantation (TT) consisted in high dose melphalan (HDT) and auto-SCT followed by an (allo-SCT) preceded by t...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/bmt.2010.90

    authors: Karlin L,Arnulf B,Chevret S,Ades L,Robin M,De Latour RP,Malphettes M,Kabbara N,Asli B,Rocha V,Fermand JP,Socie G

    更新日期:2011-02-01 00:00:00

  • Early measles vaccination in bone marrow transplant recipients.

    abstract::Measles vaccination has been recommended after the second year following bone marrow transplant (BMT) in patients not receiving immunosuppressive drugs. During a measles outbreak, we vaccinated all patients after the first year of transplant, and conducted a prospective trial to evaluate safety, effectiveness and sust...

    journal_title:Bone marrow transplantation

    pub_type: 临床试验,杂志文章

    doi:10.1038/sj.bmt.1704878

    authors: Machado CM,de Souza VA,Sumita LM,da Rocha IF,Dulley FL,Pannuti CS

    更新日期:2005-04-01 00:00:00

  • The effect of hematopoietic growth factors on the risk of graft-vs-host disease after allogeneic hematopoietic stem cell transplantation: a meta-analysis.

    abstract::The effect of hematopoietic growth factors on neutrophil recovery after allogeneic transplantation is well-recognized. Recent laboratory studies demonstrated that these cytokines may also modify T-cell and dendritic cell function, but whether the effect is strong enough to alter the risk of GVHD is unclear. We perform...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,meta分析

    doi:10.1038/sj.bmt.1704228

    authors: Ho VT,Mirza NQ,Junco Dd Dd,Okamura T,Przepiorka D

    更新日期:2003-10-01 00:00:00

  • Prophylactic donor lymphocyte infusions after moderately ablative chemotherapy and stem cell transplantation for hematological malignancies: high remission rate among poor prognosis patients at the expense of graft-versus-host disease.

    abstract::We investigated the use of 'prophylactic' donor lymphocyte infusions (DLI) containing 1 x 107 CD3+ cells, given at 30, 60 and 90 days post-allogeneic blood and marrow transplantation (BMT), following conditioning with fludarabine 30 mg/m(2)/4 days and melphalan 70 mg/m(2)/2 days. GVHD prophylaxis consisted of cyclospo...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1702726

    authors: de Lima M,Bonamino M,Vasconcelos Z,Colares M,Diamond H,Zalcberg I,Tavares R,Lerner D,Byington R,Bouzas L,da Matta J,Andrade C,Carvalho L,Pires V,Barone B,Maciel C,Tabak D

    更新日期:2001-01-01 00:00:00

  • Effects of incubation temperature and time after thawing on viability assessment of peripheral hematopoietic progenitor cells cryopreserved for transplantation.

    abstract::Three widely used viability assessments were compared: (1) membrane integrity of nucleated cells using trypan blue (TB) exclusion and a fluorometric membrane integrity assay (SYTO 13 and propidium iodide), (2) enumeration of viable CD34+ cells, and (3) clonogenic assay (granulocyte-macrophage colony-forming units, CFU...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:10.1038/sj.bmt.1704247

    authors: Yang H,Acker JP,Cabuhat M,McGann LE

    更新日期:2003-11-01 00:00:00

  • Prospective phase II study of prophylactic low-dose azacitidine and donor lymphocyte infusions following allogeneic hematopoietic stem cell transplantation for high-risk acute myeloid leukemia and myelodysplastic syndrome.

    abstract::Thirty patients, with high-risk acute myeloid leukemia (AML, n = 20) or myelodysplastic syndrome (MDS, n = 10), were enrolled in a phase II trial entailing prophylactic post-transplant azacitidine (AZA) plus escalated doses of donor lymphocyte infusion (DLI). The median number of AZA cycles was 5 (1-12) with 10 patien...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章,多中心研究

    doi:10.1038/s41409-019-0536-y

    authors: Guillaume T,Malard F,Magro L,Labopin M,Tabrizi R,Borel C,Chevallier P,Vigouroux S,Peterlin P,Garnier A,Rubio MT,Huynh A,Milpied N,Moreau P,Gaugler B,Yakoub-Agha I,Mohty M

    更新日期:2019-11-01 00:00:00

  • In vitro and in vivo depletion of T cells.

    abstract::Transplant related mortality and relapse after bmt have a negative influence on the outcome of patients transplanted for acute leukaemia in first remission. Transplant related mortality includes graft-versus-host disease, infections and graft failure. To prevent gvhd and associated infections without increased graft r...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Arnold R,Bunjes D,Wiesneth M,Hertenstein B,Theobald M,Heimpel H,Hale G,Waldmann H

    更新日期:1993-01-01 00:00:00

  • Successful pregnancy in a 28-year-old patient autografted for acute lymphoblastic leukemia following myeloablative treatment including total body irradiation.

    abstract::We report a successful pregnancy in a woman who at the age of 28 years received total body irradiation (TBI; 7.5 Gy) and high-dose chemotherapy prior to autografting of purged bone marrow for acute lymphoblastic leukemia. Four years after transplantation she delivered a healthy girl. Only five previous cases of succes...

    journal_title:Bone marrow transplantation

    pub_type: 杂志文章

    doi:

    authors: Samuelsson A,Fuchs T,Simonsson B,Björkholm M

    更新日期:1993-12-01 00:00:00