Hematopoietic stem cell transplantation for autoimmune diseases in the time of COVID-19: EBMT guidelines and recommendations DOI Creative Commons
Raffaella Greco, Tobias Alexander, Joachim Burman

et al.

Bone Marrow Transplantation, Journal Year: 2021, Volume and Issue: 56(7), P. 1493 - 1508

Published: May 24, 2021

Coronavirus disease-19 (COVID-19), caused by Severe Acute Respiratory Syndrome 2 (SARS-CoV-2), represents one of the biggest challenges 21st century, threatening public health around globe. Increasing age and presence co-morbidities are reported risk factors for severe disease mortality, along with autoimmune diseases (ADs) immunosuppressive treatments such as haematopoietic stem cell transplantation (HSCT), which also associated adverse outcomes. We review impact pandemic on specific groups patients neurological, rheumatological, gastroenterological indications, delivering HSCT in adult pediatric populations. Moving forward, we developed consensus-based guidelines recommendations best practice quality patient care order to support clinicians, scientists, their multidisciplinary teams, well carers. These aim national international organizations related local clinical teams HSCT. Areas unmet need future research questions highlighted. The waves COVID-19 predicted be followed an "endemic" phase therefore ongoing within a "new normality". reflect currently available evidence, coupled expert opinion, will revised according necessary modifications practice.

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
High-Efficacy Therapies for Treatment-Naïve Individuals with Relapsing–Remitting Multiple Sclerosis DOI Creative Commons
Léorah Freeman, Erin E. Longbrake, Patricia K. Coyle

et al.

CNS Drugs, Journal Year: 2022, Volume and Issue: 36(12), P. 1285 - 1299

Published: Nov. 9, 2022

There are > 18 distinct disease-modifying therapy (DMT) options covering 10 mechanisms of action currently approved by the US Food and Drug Administration for treatment relapsing-remitting multiple sclerosis (RRMS). Given multitude available options, recent international consensus guidelines offering differing recommendations, there is broad heterogeneity in how DMTs used clinical practice. Choosing a DMT newly diagnosed patients with MS topic significant debate care. Historically, an escalation approach to was RRMS. However, evidence benefits early high-efficacy therapies (HETs) this population emerging. In review, we provide overview strategies, discuss HETs (including ofatumumab, ocrelizumab, natalizumab, alemtuzumab, cladribine) stages MS, along safety concerns associated these DMTs. By minimizing accumulation neurological damage disease course, may enhance long-term outcomes over lifetime patient.

Language: Английский

Citations

76
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 Applications of Machine Learning in the Management of Patients Undergoing Stem Cell Transplantation: Are We Ready? DOI Open Access

Luca Garuffo,

Alessandro Leoni,

Roberto Gatta

et al.

Cancers, Journal Year: 2025, Volume and Issue: 17(3), P. 395 - 395

Published: Jan. 25, 2025

Hematopoietic stem cell transplantation (HSCT) is a life-saving therapy for hematologic malignancies, such as leukemia and lymphoma other severe conditions but associated with significant risks, including graft versus host disease (GVHD), relapse, treatment-related mortality. The increasing complexity of clinical, genomic, biomarker data has spurred interest in machine learning (ML), which emerged transformative tool to enhance decision-making optimize outcomes HSCT. This review examines the applications ML HSCT, focusing on donor selection, conditioning regimen, prediction post-transplant outcomes. Machine approaches, decision trees, random forests, neural networks, have demonstrated potential improving compatibility algorithms, mortality relapse prediction, GVHD risk stratification. Integrating “omics” models enabled identification novel biomarkers development highly accurate predictive tools, supporting personalized treatment strategies. Despite promising advancements, challenges persist, standardization, algorithm interpretability, ethical considerations regarding patient privacy. While holds promise revolutionizing HSCT management, addressing these barriers through multicenter collaborations regulatory frameworks remains essential broader clinical adoption. In addition, can cope some harmonization, patients’ protection, availability adequate infrastructure. Future research should prioritize larger datasets, multimodal integration, robust validation methods fully realize ML’s

Language: Английский

Citations

3
Hematopoietic Stem Cell Transplantation for Autoimmune Disease DOI
Tobias Alexander, Raffaella Greco, John A. Snowden

et al.

Annual Review of Medicine, Journal Year: 2020, Volume and Issue: 72(1), P. 215 - 228

Published: Oct. 27, 2020

The introduction of targeted biologic therapies has changed the treatment landscape for autoimmune diseases (ADs) substantially, but although these provide more specificity, they require continuous administration, rarely restore organ function or reverse disability, and are not curative. Over last 25 years, hematopoietic stem cell transplantation (HSCT) been increasingly used to treat patients in whom risk:benefit ratio HSCT is acceptable. In contrast chronic suppression immune function, this intensive one-off procedure aims treatment-free remissions by reinduction self-tolerance. European Society Blood Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) central development approach, with over 3,300 registrations ADs. Recent data have improved evidence base support autologous multiple sclerosis, systemic Crohn's disease, along a wide range rarer disease indications, become an integral part algorithms various

Language: Английский

Citations

77
Telerehabilitation in response to constrained physical distance: an opportunity to rethink neurorehabilitative routines DOI Open Access
Arturo Nuara, Maddalena Fabbri‐Destro, Emilia Scalona

et al.

Journal of Neurology, Journal Year: 2021, Volume and Issue: 269(2), P. 627 - 638

Published: Jan. 15, 2021

Language: Английский

Citations

67
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

57
Stiff person syndrome spectrum disorders; more than meets the eye DOI Creative Commons
Scott D. Newsome, Tory P. Johnson

Journal of Neuroimmunology, Journal Year: 2022, Volume and Issue: 369, P. 577915 - 577915

Published: June 12, 2022

Stiff person syndrome spectrum disorders (SPSD) are a group of rare neuroimmunological that often include painful spasms and rigidity. However, patients have highly heterogeneous signs symptoms which may reflect different mechanistic disease processes. Understanding subsets based on clinical phenotype be important for prognosis guiding treatment. The goal this review is to provide updates SPSD its expanding spectrum, prognostic markers, treatment considerations. Further, we describe the current understanding in immunopathogenesis highlight gaps our knowledge appropriate future research directions. Examples revised diagnostic criteria also presented.

Language: Английский

Citations

56
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
Vaccination and immunotherapies in neuroimmunological diseases DOI Open Access

Alexander Winkelmann,

Micha Loebermann, Michael Barnett

et al.

Nature Reviews Neurology, Journal Year: 2022, Volume and Issue: 18(5), P. 289 - 306

Published: April 6, 2022

Language: Английский

Citations

46