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The purpose of this study is to assess the effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) as currently performed by different study protocols used across Europe in various EBMT centres through the careful recording and analysis of routinely collected clinical and biological data.
Different protocols are used in the different centres, it is not yet clear which approach will be the most efficient and the safest. Every centre will follow its own local protocol for AHSCT which usually refers to the recent update of the EBMT Guidelines for HSCT in autoimmune disease. Patient selection for AHSCT treatment technique with regard to the risk/benefit balance has to be carefully addressed by standard patient pretransplant evaluation, whereas treatment local regimen, follow-ups evaluation, supportive medication and prophylaxis will be recorded and analysed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NISSC | Autologous HSCT |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous HSCT | Procedure | 1st AHSCT |
|
| Measure | Description | Time Frame |
|---|---|---|
| Progression free survival | Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc. | 2 year post transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix) | Incidence of Adverse Events (AE) and Serious Adverse Events (SE) Neutrophil and platelet engraftment, defined as first day after transplantation with absolute neutrophil count > 500 cells/μL and >20.000 platelets/μL without platelet transfusion, respectively |
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Inclusion Criteria:
Exclusion Criteria:
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Patients treated with autologous HSCT for severe systemic sclerosis
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| Name | Affiliation | Role |
|---|---|---|
| Dominique Farge, PhD | EBMT ADWP | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Badoglio Manuela- EBMT Paris Office | Paris | 75010 | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31949011 | Derived | Henes J, Oliveira MC, Labopin M, Badoglio M, Scherer HU, Del Papa N, Daikeler T, Schmalzing M, Schroers R, Martin T, Pugnet G, Simoes B, Michonneau D, Marijt EWA, Lioure B, Olivier Bay J, Snowden JA, Rovira M, Huynh A, Onida F, Kanz L, Marjanovic Z, Farge D. 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. Haematologica. 2021 Feb 1;106(2):375-383. doi: 10.3324/haematol.2019.230128. |
| Label | URL |
|---|---|
| Organisation website | View source |
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| ID | Term |
|---|---|
| D001327 | Autoimmune Diseases |
| D012595 | Scleroderma, Systemic |
| ID | Term |
|---|---|
| D007154 | Immune System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
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| 2 year post transplant |
| Overall Survival | Overall Survival | 2 year post transplant |
| Response to treatment | Response to treatment within 1 year following autologous HSCT, defined as
| at 1 year post transplant |
| Improvement in Quality of life | Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution | 2 year post transplant |
| Relapse incidence | Defined as any of the following changes after prior response to treatment on quarterly follow up as defined below:
| 2 year post transplant |
| 100-day Treatment related mortality | any death during 100 day following transplant that cannot be attributed to progression or relapse of the disease | 100 days post transplant |