Hematopoietic stem cell transplantation in autoimmune diseases: update from EBMT Autoimmune Diseases Working Party with special reference to Poland DOI Creative Commons
John A. Snowden, Jan Styczyński, Emilian Snarski

et al.

Acta Haematologica Polonica, Journal Year: 2021, Volume and Issue: 52(4), P. 217 - 224

Published: Aug. 31, 2021

Hematopoietic stem cell transplantation (HSCT) is now evolving into a standard treatment in some autoimmune diseases (AD) alongside other modern therapy. The main indications are multiple sclerosis and systemic for which HSCT has become an integral standard-of-care part of algorithms. From 1994 to the beginning 2021, data from EBMT Registry indicates that 3,442 patients (60% females, 40% males; 91% adults, 9% pediatric) received 3,514 transplant procedures diseases, with over 90% receiving autologous transplant. Autoimmune currently fastest growing indication European Society Blood Marrow Transplantation (EBMT), whilst allogeneic ADs mainly restricted pediatrics, especially genetic component. Patient selection plays key role providing best risk/benefit ratio procedure. Intensity conditioning regimen center experience also important. Ultimately, future depends on care therapy, influences uptake within national/international disease specialist communities. Further studies necessary order establish relative benefit current/future each disease, define mechanisms, develop clinical biomarkers help select monitor patients, health economic benefits public delivery. We present current perspective summarizing activity across EBMT, including centers Poland.

Language: Английский

Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022 DOI Creative Commons
John A. Snowden, Isabel Sánchez‐Ortega, Selim Corbacioglu

et al.

Bone Marrow Transplantation, Journal Year: 2022, Volume and Issue: 57(8), P. 1217 - 1239

Published: May 19, 2022

For over two decades, the EBMT has updated recommendations on indications for haematopoietic cell transplantation (HCT) practice based clinical and scientific developments in field. This is eighth special report HCT haematological diseases, solid tumours immune disorders. Our aim to provide general guidance according prevailing countries centres. In order inform patient decisions, these must be considered conjunction with risk of disease, procedure non-transplant strategies, including evolving cellular therapies. techniques are constantly we make no specific recommendations, but encourage harmonisation practice, where possible, ensure experience across can meaningfully aggregated via registry outputs. We also recommend working JACIE accreditation standards maintain quality laboratory components benchmarking survival outcomes. Since last edition, COVID-19 pandemic affected decision making activity indications. Although full impact yet determined, that delivered ongoing reference national guidance, accordance current local conditions.

Language: Английский

Citations

263

SARS-CoV-2 in immunocompromised individuals DOI Creative Commons
Susan DeWolf, Justin Laracy, Miguel‐Angel Perales

et al.

Immunity, Journal Year: 2022, Volume and Issue: 55(10), P. 1779 - 1798

Published: Sept. 13, 2022

Language: Английский

Citations

92

Hematopoietic cell transplantation and cellular therapies in Europe 2021. The second year of the SARS-CoV-2 pandemic. A Report from the EBMT Activity Survey DOI Creative Commons
Jakob Passweg, Helen Baldomero, Fabio Ciceri

et al.

Bone Marrow Transplantation, Journal Year: 2023, Volume and Issue: 58(6), P. 647 - 658

Published: March 6, 2023

Abstract In 2021, 47,412 HCT (19,806 (42%) allogeneic and 27,606 (58%) autologous) in 43,109 patients were reported by 694 European centers. 3494 received advanced cellular therapies, 2524 of which CAR-T treatments, an additional 3245 DLI. Changes compared to the previous year treatment (+35%), +5.4%, autologous +3.9%, more pronounced non-malignant disorders. Main indications for myeloid malignancies 10,745 (58%), lymphoid 5127 (28%) disorders 2501 (13%). 22,129 (90%) solid tumors 1635 (7%). HCT, use haploidentical donors decreased −0.9% while unrelated sibling increased +4.3% +9%. Cord blood −5.8%. Pediatric overall +5.6% (+6.9% +1.6% autologous). Increase was mainly restricted high-income countries. The drop activity 2020 partially recovered second SARS-CoV-2 pandemic. transplant community confronted with pandemic challenge, continued providing access treatment. This annual EBMT report reflects current activities useful health care resource planning.

Language: Английский

Citations

58

Hematopoietic cell transplantation and cellular therapies in Europe 2022. CAR-T activity continues to grow; transplant activity has slowed: a report from the EBMT DOI Creative Commons
Jakob Passweg, Helen Baldomero, Fabio Ciceri

et al.

Bone Marrow Transplantation, Journal Year: 2024, Volume and Issue: 59(6), P. 803 - 812

Published: March 4, 2024

Abstract In 2022, 46,143 HCT (19,011 (41.2%) allogeneic and 27,132 (58.8%) autologous) in 41,854 patients were reported by 689 European centers. 4329 received advanced cellular therapies, 3205 of which CAR-T. An additional 2854 DLI. Changes compared to the previous year an increase CAR-T treatments (+27%) decrease (−4.0%) autologous (−1.7%). Main indications for myeloid malignancies (10,433; 58.4%), lymphoid (4,674; 26.2%) non-malignant disorders (2572; 14.4%). lymphomas (7897; 32.9%), PCD (13,694; 57.1%) solid tumors (1593; 6.6%). HCT, use sibling donors decreased −7.7%, haploidentical −6.3% unrelated −0.9%. Overall cord blood −16.0%. Use allogeneic, a lesser degree likely reflecting availability new treatment modalities, including small molecules, bispecific antibodies, cells. Pediatric activity remains stable (+0.3%) with differences between HCT. continues reached cumulative total 9039 treated wide across countries. After many years continuous growth, application seems have slowed down.

Language: Английский

Citations

37

Innovative cellular therapies for autoimmune diseases: expert-based position statement and clinical practice recommendations from the EBMT practice harmonization and guidelines committee DOI Creative Commons
Raffaella Greco, Tobias Alexander, Nicoletta Del Papa

et al.

EClinicalMedicine, Journal Year: 2024, Volume and Issue: 69, P. 102476 - 102476

Published: Feb. 10, 2024

Autoimmune diseases (ADs) are characterized by loss of immune tolerance, high chronicity, with substantial morbidity and mortality, despite conventional immunosuppression (IS) or targeted disease modifying therapies (DMTs), which usually require repeated administration. Recently, novel cellular (CT), including mesenchymal stromal cells (MSC), Chimeric Antigen Receptors T (CART) regulatory (Tregs), have been successfully adopted in ADs. An international expert panel the European Society for Blood Marrow Transplantation International Cell Gene Therapy, reviewed all available evidence, based on current literature practices, use MSC, CART Tregs, AD patients rheumatological, neurological, gastroenterological indications. Expert-based consensus recommendations best practice quality patient care were developed to support clinicians, scientists, their multidisciplinary teams, as well providers will be regularly updated.

Language: Английский

Citations

31

The 2023 EBMT report on hematopoietic cell transplantation and cellular therapies. Increased use of allogeneic HCT for myeloid malignancies and of CAR-T at the expense of autologous HCT DOI Creative Commons
Jakob Passweg, Helen Baldomero,

Marina Atlija

et al.

Bone Marrow Transplantation, Journal Year: 2025, Volume and Issue: unknown

Published: Feb. 12, 2025

Language: Английский

Citations

2

Impact of the SARS-CoV-2 pandemic on hematopoietic cell transplantation and cellular therapies in Europe 2020: a report from the EBMT activity survey DOI Creative Commons
Jakob Passweg, Helen Baldomero, Christian Chabannon

et al.

Bone Marrow Transplantation, Journal Year: 2022, Volume and Issue: 57(5), P. 742 - 752

Published: Feb. 22, 2022

Abstract In 2020, 45,364 HCT in 41,016 patients, 18,796 (41%) allogeneic and 26,568 (59%) autologous 690 centers were reported. Changes observed as follows: total number of −6.5%, −5.1%, −7.5%, more pronounced non-malignant disorders for autoimmune disease HCT. Main indications myeloid malignancies 10,441 (25%), lymphoid 26,120 (64%) 2532 (6%). A continued growth CAR-T cellular therapies to 1874 (+65%) patients 2020 was observed. HCT, the use haploidentical donors increased while unrelated sibling decreased. Cord blood by 11.7% first time since 2012. There a significant increase non-myeloablative but drop myeloablative conditioning marrow stem cell source. We interpreted these changes being due SARS-CoV-2 pandemic starting early Europe provided additional data reflecting varying impact across selected countries larger cities. The transplant community confronted with challenge, providing access treatment. This annual report EBMT reflects current activities useful health care planning.

Language: Английский

Citations

66

Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT) DOI Creative Commons
Tobias Alexander, Raffaella Greco

Bone Marrow Transplantation, Journal Year: 2022, Volume and Issue: 57(7), P. 1055 - 1062

Published: May 16, 2022

Abstract Autoimmune diseases (ADs) represent a heterogenous group of complex with increasing incidence in Western countries and are major cause morbidity. Hematopoietic stem cell transplantation (HSCT) has evolved over the last 25 years as specific treatment for patients severe ADs, through eradication pathogenic immunologic memory profound immune renewal. HSCT ADs is recently facing unique developmental phase across transplant centers. This review provides comprehensive overview recent evidence developments area, including fundamentals preclinical research, clinical studies neurologic, rheumatologic gastroenterologic diseases, which indications at present, along rarer indications. Moreover, we describe interwoven challenges delivering more advanced cellular therapies, exploiting mesenchymal cells, regulatory T cells potentially CAR-T affected by ADs. Overall, discuss past current indications, efficacy, associated risks benefits, future directions therapies severe/refractory integrating available literature European Society Blood Marrow Transplantation (EBMT) registry data.

Language: Английский

Citations

59

Immune Reconstitution Following Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis: A Review on Behalf of the EBMT Autoimmune Diseases Working Party DOI Creative Commons
Maria Teresa Cencioni, Angela Genchi, Gavin Brittain

et al.

Frontiers in Immunology, Journal Year: 2022, Volume and Issue: 12

Published: Feb. 1, 2022

Multiple sclerosis (MS) is a central nervous system (CNS) disorder, which mediated by an abnormal immune response coordinated T and B cells resulting in areas of inflammation, demyelination, axonal loss. Disease-modifying treatments (DMTs) are available to dampen the inflammatory aggression but ineffective many patients. Autologous hematopoietic stem cell transplantation (HSCT) has been used as treatment patients with highly active disease, achieving long-term clinical remission most. The rationale intervention eradicate autoreactive lympho-ablative regimens restore tolerance. Immunological studies have demonstrated that autologous HSCT induces renewal TCR repertoires, resurgence regulatory cells, depletion proinflammatory subsets, suggesting “resetting” immunological memory. Although our understanding effects progressed, further work required characterize mechanisms underlie efficacy. Considering memory disease-promoting stem-like multipotent progenitors involved self-regeneration effector investigating reconstitution compartment subsets following could elucidate those mechanisms. Since all subjects need be optimally protected from vaccine-preventable diseases (including COVID-19), there ensure vaccination undergoing effective safe. Additionally, study HSCT-treated means evaluating responses distinguish broad immunosuppression resetting.

Language: Английский

Citations

48

French protocol for the diagnosis and management of hematopoietic stem cell transplantation in autoimmune diseases DOI Creative Commons
Dominique Farge, G. Pugnet, Matthieu Allez

et al.

La Revue de Médecine Interne, Journal Year: 2024, Volume and Issue: 45(2), P. 79 - 99

Published: Jan. 13, 2024

Hematopoietic stem cell transplantation (HSCT) for severe ADs was developed over the past 25 years and is now validated by national international medical societies early systemic sclerosis (SSc) relapsing-remitting multiple (MS) available as part of routine care in accredited center. HSCT also recommended, with varying levels evidence, an alternative treatment several ADs, when refractory to conventional therapy, including specific cases connective tissue diseases or vasculitis, inflammatory neurological diseases, more rarely Crohn's disease. The aim this document provide guidelines current indications, procedures follow-up ADs. Patient safety considerations are central guidance on patient selection conditioning, always at MATHEC multidisciplinary team meeting (MDTM) based recent (less than 3 months) thorough evaluation. procedural aspects then carried out within appropriately experienced Joint Accreditation Committee International Society Cellular Therapy SFGM-TC centres close collaboration specialist. These French recommendations were performed according HAS/FAI2R standard operating coordinated Île-de-France Reference Centre Rare Systemic Autoimmune Diseases (CRMR MATHEC) Filière FAI2R association MaRIH. task force consisted patients 64 clinical experts from various specialties centres. data-derived consensus-derived will help clinicians propose their evidence-based way. give directions future research area. be updated newly emerging data. Of note, other therapies that have not yet been approved practice subject ongoing addressed document. La greffe de cellules souches hématopoïétiques (GCSH) pour les maladies auto-immunes (MAI) sévères s'est développée au cours des dernières années et est maintenant validée par sociétés savantes nationales internationales la sclérodermie systémique sévère précoce sclérose en plaques récurrente-rémittente disponible dans le cadre soins courants accrédités. GCSH également recommandée, avec niveaux preuve variables, comme traitement alternatif plusieurs MAI, lorsqu'elles sont réfractaires aux thérapies conventionnelles, comprenant cas spécifiques connectivites ou vascularites ; neuro-inflammatoires plus rarement Crohn sévères. L'objectif ce fournir recommandations sur indications actuelles, procédures suivi MAI. sécurité reste l'élément majeur guidant sélection conditionnement patients, toujours validées lors réunion concertation pluridisciplinaire nationale base d'une évaluation exhaustive récente (moins mois) du patient. ensuite effectués expérimentés accrédités SFGM-TC, étroite spécialiste Ces françaises ont été réalisées conformément opérationnelles coordonnées référence systémiques rares d'Île-de-France sein Le groupe travail était composé cliniciens issus différentes spécialités différents français. issues données publiées consensus d'experts aideront à proposer leurs atteints MAI sévère. ouvrent perspectives recherche clinique. seront régulièrement mises jour fonction nouvelles publiées. Il convient noter que autres cellulaires qui n'ont pas encore approuvées autorités santé font l'objet d'essais cliniques ne abordées

Citations

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