Life after Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis

Moraes, Daniela A.; Oliveira, Maria Carolina

doi:10.2147/jbm.s338077

Cited by 8 publications

(9 citation statements)

References 73 publications

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“…The first aHSCT was performed in 1997 in SSc [ 179 ]. Since then, around 500 aHSCTs have been reported [ 180 ] and more than 1000 SSc patients have been transplanted worldwide [ 181 ]. Furthermore, three randomized controlled trials have shown superiority of aHSCT versus intravenous cyclophosphamide therapy (ASSIST trial [ 182 ], ASTIS trial [ 183 ], SCOT trial [ 184 ]).…”

Section: Which Therapeutic Approaches Have Been Evaluated To Target A...mentioning

confidence: 99%

“…Currently, different regimens have been compared to outweigh therapeutic efficacy and adverse events. So far, specific recommendations regarding transplant procedures are missing [ 181 ]. Recently, the German Society for Rheumatology (DGRh) suggested criteria for patient selection [ 188 ].…”

Section: Which Therapeutic Approaches Have Been Evaluated To Target A...mentioning

confidence: 99%

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Autoantibodies as Biomarker and Therapeutic Target in Systemic Sclerosis

et al. 2022

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Systemic sclerosis (SSc) is a rare connective tissue disorder characterized by immune dysregulation evoking the pathophysiological triad of inflammation, fibrosis and vasculopathy. In SSc, several alterations in the B-cell compartment have been described, leading to polyclonal B-cell hyperreactivity, hypergammaglobulinemia and autoantibody production. Autoreactive B cells and autoantibodies promote and maintain pathologic mechanisms. In addition, autoantibodies in SSc are important biomarkers for predicting clinical phenotype and disease progression. Autoreactive B cells and autoantibodies represent potentially promising targets for therapeutic approaches including B-cell-targeting therapies, as well as strategies for unselective and selective removal of autoantibodies. In this review, we present mechanisms of the innate immune system leading to the generation of autoantibodies, alterations of the B-cell compartment in SSc, autoantibodies as biomarkers and autoantibody-mediated pathologies in SSc as well as potential therapeutic approaches to target these.

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Section: Which Therapeutic Approaches Have Been Evaluated To Target A...mentioning

confidence: 99%

Section: Which Therapeutic Approaches Have Been Evaluated To Target A...mentioning

confidence: 99%

Autoantibodies as Biomarker and Therapeutic Target in Systemic Sclerosis

et al. 2022

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“…In this review we summarized the results of studies of the last 5 years employing AHSCT for the treatment of SSc patients at high risk of organ failure. AHSCT ameliorates skin involvement and improves or stabilizes lung function, especially in patients with disease duration of less than five years [ 13 , 38 ]. The patient-related perspective confirms that this procedure is well-tolerated and improves quality of life, as shown by a recent systematic review [ 39 ].…”

Section: Discussionmentioning

confidence: 99%

“…As a form of intensive immunotherapy that targets the autoreactive adaptive immune system, HSCT deeply modifies the immune system, restoring the immunological balance that counteracts inflammation and fibrosis progression, enabling disease control and, eventually, tissue repair [ 13 ]. Briefly, HSCT consists of four steps: mobilization of hematopoietic stem cells using chemotherapy, such as CYC, and growth factors [granulocyte colony-stimulating factor (G-CSF)]; conditioning using myeloablative or non-myeloablative regimens to eradicate autoreactive immune cells; reinfusion of autologous stem cells and immune reconstitution [ 14 ].…”

Section: Introductionmentioning

confidence: 99%

Autologous Haematopoietic Stem Cell Transplantation and Systemic Sclerosis: Focus on Interstitial Lung Disease

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Rosa

et al. 2022

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Autologous hematopoietic stem cells transplantation (AHSCT) has been employed as treatment for severe systemic sclerosis (SSc) with high risk of organ failure. In the last 25 years overall survival and treatment-related mortality have improved, in accordance with a better patient selection and mobilization and conditioning protocols. This review analyzes the evidence from the last 5 years for AHSCT-treated SSc patients, considering in particular the outcomes related to interstitial lung disease. There are increasing data supporting the use of AHSCT in selected patients with rapidly progressive SSc. However, some unmet needs remain, such as an accurate patient selection, pre-transplantation analysis to identify subclinical conditions precluding the transplantation, and the alternatives for post-transplant ILD recurrence.

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“…HCT aims to ablate the adaptive immune system and restore normal immune regulation. Unfortunately, disease progression after HCT is seen in a subset of patients, with numbers ranging from 12% after two years up to 40% after 10 years ( 6 , 7 ). In these cases treatment options are often limited and treatment with immunosuppression including mycophenolate mofetil and cyclophosphamide is usually reintroduced.…”

Section: Introductionmentioning

confidence: 99%

Case report: A successful second autologous hematopoietic stem cell transplantation in refractory systemic sclerosis, with positive effect on skin involvement, pulmonary function and microcirculation

et al. 2022

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BackgroundSystemic sclerosis (SSc) is a complex autoimmune disease characterized by inflammation, vasculopathy and fibrosis of the skin and internal organs. Treatment with autologous hematopoietic cell transplantation (HCT) for progressive SSc has improved overall and event-free survival rates significantly, but unfortunately disease progression after HCT is seen in a subset of patients. Data on the efficacy and safety of second HCT is scarce.CaseWe present a patient with diffuse cutaneous SSc and associated interstitial lung disease (ILD) who successfully underwent a second HCT for progressive disease five years after a first HCT. We describe changes in skin involvement and pulmonary involvement as well as the changes observed in sequential nailfold microcapillaroscopy (NCM), performed from first presentation up to this moment.ConclusionThis case adds to the current limited literature on efficacy and safety of a second HCT in SSc refractory cases. Furthermore it outlines the potential of HCT on amelioration of microvasculopathy in SSc.

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Life after Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis

Cited by 8 publications

References 73 publications

Autoantibodies as Biomarker and Therapeutic Target in Systemic Sclerosis

Autoantibodies as Biomarker and Therapeutic Target in Systemic Sclerosis

Autologous Haematopoietic Stem Cell Transplantation and Systemic Sclerosis: Focus on Interstitial Lung Disease

Case report: A successful second autologous hematopoietic stem cell transplantation in refractory systemic sclerosis, with positive effect on skin involvement, pulmonary function and microcirculation

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