Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party.

This study confirms the efficacy of autologous stem cell transplantation in real-life practice for severe systemic sclerosis using non myeloablative conditioning. Careful cardio-pulmonary assessment to identify organ involvement at patient referral, reduced cyclophosphamide doses and CD34+ selection may improve outcomes. The study was registered at NCT02516124. PMID: 31949011 [PubMed - as supplied by publisher]
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research

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Purpose of review This review provides a risk-stratified and evidence-based management for subsets of systemic sclerosis (SSc) patients in the first five years from disease onset. Recent findings Cardiopulmonary disease remains the primary cause of mortality in SSc patients. Morbidity and mortality in SSc-associated pulmonary arterial hypertension have improved with combination treatment, in either an upfront or sequential treatment pattern. Traditional therapies for interstitial lung disease (SSc-ILD) have targeted those with clinically significant and progressive ILD with immunosuppression. New data suggest a possib...
Source: Current Opinion in Rheumatology - Category: Rheumatology Tags: CLINICAL THERAPEUTICS AND HEMATOLOGIC COMPLICATIONS: Edited by W. Joseph McCune Source Type: research
AbstractSystemic sclerosis (SSc) is an autoimmune rheumatic disease with heterogeneous clinical manifestations and variable disease course in which the severity of pathology dictates the disease prognosis and course. Among autoimmune rheumatic diseases, SSc carries the highest mortality, but there are exciting new therapeutic targets that appear to halt the progression of SSc manifestations such as skin or lung fibrosis. In selected patients, high ‐intensity regimens with autologous stem cell transplantation can favorably modify the disease course. From what was once thought to be an untreatable disease, targeted therapi...
Source: Arthritis and Rheumatology - Category: Rheumatology Authors: Tags: REVIEW ARTICLE Source Type: research
Resetting the immune system through autologous hematopoietic stem cell transplant (autoHSCT) is a highly effective treatment in patients with autoimmune diseases (AID). AutoHSCT achieved long-term remission in patients with relapsed refractory and secondary progressive multiple sclerosis (Muraro 2017), superior to their previous standard of care (Burt 2019). AutoHSCT in scleroderma patients achieved superior outcomes in two randomized studies (Tyndall 2014, Sullivan 2018). These impressive results are achieved by both eradication of autoreactive immune effector cells and re-establishment of a self-tolerant immune system, i...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 469 Source Type: research
CONCLUSION: Current drug studies may provide innovative therapeutic perspectives for SSc-ILD and could significantly improve the prognosis of affected patients in the future. PMID: 31754786 [PubMed - as supplied by publisher]
Source: Zeitschrift fur Rheumatologie - Category: Rheumatology Authors: Tags: Z Rheumatol Source Type: research
Purpose of review Autologous hematopoietic stem cell transplantation (HSCT) is a promising therapeutic modality for severe autoimmune diseases. In this review, we will outline the immunological mechanisms and the clinical evidence and experiences for therapeutic HSCT in autoimmune diseases, with particular focus on systemic sclerosis and multiple sclerosis. Recent findings Approximately 3000 patients with autoimmune diseases worldwide have been treated with HSCT. HSCT in systemic sclerosis has been shown in three randomized controlled trials to be associated with significant long-term event-free survival despite some ...
Source: Current Opinion in Hematology - Category: Hematology Tags: HEMATOPOIETIC STEM CELL TRANSPLANTATION: Edited by David Rizzieri Source Type: research
Abstract Systemic sclerosis is a complex autoimmune connective tissue disease which carries a significant burden of disease-related morbidity including potentially life-threatening complications. Systemic sclerosis can affect all the major organs and therefore, although the disease is uncommon, many hospital-based specialists are involved in patient care. Vascular disease (e.g. Raynaud's phenomenon) is an almost universal symptom in patients with systemic sclerosis and is often the earliest manifestation of the disease. Systemic sclerosis not uncommonly can overlap with other rheumatological conditions (e.g. rheum...
Source: British Journal of Hospital Medicine - Category: Internal Medicine Authors: Tags: Br J Hosp Med (Lond) Source Type: research
AbstractThree prospective controlled clinical trials and numerous small series and case reports have confirmed that durable, drug-free remission in systemic sclerosis is possible via an autologous hematopoietic stem cell transplantation. Similar results have been seen in other autoimmune diseases. The exact mechanism by which this immune “reset” was achieved in some but not all cases remains elusive, but includes major reduction of autoreactive immune competent cells, re-establishment of T- and B cell regulatory networks and normalization of tissue niche function, particularly vascular. Some aspects regarding m...
Source: BioDrugs - Category: Drugs & Pharmacology Source Type: research
Qiancheng Deng1, Yangyang Luo1,2, Christopher Chang3, Haijing Wu1, Yan Ding4* and Rong Xiao1* 1Hunan Key Laboratory of Medical Epigenetics, Department of Dermatology, The Second Xiangya Hospital, Central South University, Changsha, China 2Department of Dermatology, Hunan Children's Hospital, Changsha, China 3Division of Rheumatology, Allergy and Clinical Immunology, University of California, Davis, Davis, CA, United States 4Department of Dermatology, Hainan Provincial Dermatology Disease Hospital, Haikou, China Autoimmune diseases are usually complex and multifactorial, characterized by aberrant produc...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
This report presents a full-mouth implant-retained reconstruction in a 35-year-old male patient who had undergone alloHCT for treatment of acute myeloid leukemia and developed sclerotic cGVHD afterwards. Implant-retained prosthetics might be a practicable treatment for patients with these complications.
Source: Quintessence International - Category: Dentistry Source Type: research
ConclusionsThis first SFGM-TC retrospective report shows long-term benefit of AHSCT in AD patients with acceptable toxicity.
Source: Clinical Rheumatology - Category: Rheumatology Source Type: research
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