Auto-HSCT induces sustained responses in severe systemic sclerosis patients failing pulse cyclophosphamide

Moore, John J.; Englert, H.; Furlong, Timothy J.; Poon, Teresa; Milliken, Sam; Ma, Dávid

doi:10.1038/bmt.2012.67

Cited by 8 publications

(5 citation statements)

References 10 publications

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“…The exact variation of the mRSS was not specified and was not obtained from the authors after request. The mean improvement of the mRSS was 12 points at 1 year after HSCT and 14 points at 2 years for studies with available qualitative data . The mRSS improvement was sustained for up to 8 years after HSCT according to the available data (high rate of loss to follow‐up and low number of patients in each study).…”

Section: Resultsmentioning

confidence: 91%

“…The characteristics of the included studies are summarized in Table and Table S3 (see Supporting Information). The study designs included four studies comparing autologous HSCT with CYC (two randomized studies, 89 patients), two nonrandomized controlled studies (20 patients) and 34 case series (247 patients) . There were three cohort studies derived from the EBMT registry .…”

Section: Resultsmentioning

confidence: 99%

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Efficacy and safety of autologous haematopoietic stem cell transplantation in systemic sclerosis: a systematic review of the literature

et al. 2018

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We aimed to assess the efficacy of autologous haematopoietic stem cell transplantation (HSCT) for skin sclerosis (SSc) and lung function in SSc. We performed a systematic literature review in the PubMed and Scopus databases from the earliest records to March 2016. We assessed study quality using the Cochrane tool for randomized studies, the Newcastle-Ottawa Scale for controlled cohort studies and an 18-item quality-appraisal checklist for case series. The primary outcome was the improvement of skin thickening using the modified Rodnan Skin Score (mRSS). The secondary outcome was efficacy on lung function, using diffusing capacity of the lungs for carbon monoxide and forced vital capacity (FVC). The safety of the procedure was evaluated. The literature search identified 431 citations. There were 38 studies involving a total of 344 patients who fulfilled our inclusion criteria. No meta-analysis was performed due to a high heterogeneity. There was a significant improvement in mRSS in the majority of the reports (P < 0·05), and the results were sustained for up to 8 years after autologous HSCT. The randomized studies and the four cohort studies each showed a slight but statistically significant improvement in FVC at 1 or 2 years. The treatment-related mortality calculated by pooling patients of 35 studies (336 patients with a follow-up up to 146 months) was 8·3% after autologous HSCT and 1% in cyclophosphamide-treated groups. Despite heterogeneity among the studies, we determined that autologous HSCT significantly improved cutaneous fibrosis and slightly improved FVC. Safety of autologous HSCT is acceptable given the severity of the disease. This systematic review was registered on PROSPERO, number CRD42016027951.

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Section: Resultsmentioning

confidence: 91%

Section: Resultsmentioning

confidence: 99%

Efficacy and safety of autologous haematopoietic stem cell transplantation in systemic sclerosis: a systematic review of the literature

et al. 2018

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“…Another larger retrospective study showed that 48% patients who had an autologous HSCT had significant improvement of skin score of more than 25% at 5‐year follow up . Data from St Vincents Hospital demonstrated that HSCT could be performed in heavily pre‐treated patients who had failed up to a median 3.6 g/m of cyclophosphamide . suggesting that the mechanism of HSCT is almost certainly different to chemotherapy alone as the same immunosuppressant was used again in the HSCT.…”

Section: Introductionmentioning

confidence: 92%

“…52 Data from St Vincents Hospital demonstrated that HSCT could be performed in heavily pre-treated patients who had failed up to a median 3.6 g/m 2 of cyclophosphamide. 54 suggesting that the mechanism of HSCT is almost certainly different to chemotherapy alone as the same immunosuppressant was used again in the HSCT. More recently, two randomised trials have confirmed the superiority of HSCT over intravenous cyclophosphamide (a commonly used standard of care for severe SSc).…”

Section: Systemic Sclerosismentioning

confidence: 99%

The role of autologous haemopoietic stem cell transplantation in the treatment of autoimmune disorders

Rebeiro

Moore

2016

Internal Medicine Journal

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Autologous haemopoietic stem cell transplantation (HSCT) has been used for over 30 years for malignant haematological diseases, such as myeloma and lymphoma, with considerable success. More recently this procedure has been adopted as a form of high dose immunosuppression in selected patients with autoimmune diseases that are resistant to conventional therapies. Animal models have previously outlined the rationale and validity of HSCT in patients with these non-malignant, but in many cases, life-threatening conditions. Recent data have that deletion of putative autoreactive immune clones with reconstitution of a thymic driven, tolerant immune system occurs in HSCT for auto-immune patients. Two randomised control trials have confirmed that HSCT is superior to monthly cyclophosphamide in systemic sclerosis with a highly significant disease free and overall survival benefit demonstrated in the Autologous Stem cell Transplantation International Scleroderma trial. Over 2000 patients worldwide with autoimmune conditions have been treated with HSCT - the commonest indications being multiple sclerosis (MS) and systemic sclerosis. Encouraging relapse free survival of 70-80% at 4 years, in heavily pre-treated MS patients, has been demonstrated in Phase II trials. A Phase III trial in MS patients who have failed interferon is currently accruing patients. Future challenges include improvements in safety of HSCT, particularly in cardiac assessment of systemic sclerosis patients, cost-benefit analyses of HSCT compared to standard therapy and establishment of centres of excellence to continue to enhance the safety and benefit of this exciting new therapy.

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“…The role of bone marrow transplantation in the treatment of systemic sclerosis As a treatment often still considered as last resort, autologous Human Stem Cell Transplantation (aHSCT) has been studied in both adult and pediatric patients with refractory rheumatologic diseases including systemic sclerosis [72][73][74][75][76][77][78][79][80] [81,82]. The proposed hypothesis for the therapeutic benefits of HSCT in systemic sclerosis in particular is based on the elimination of the effector mechanisms that produce the inflammatory response and the subsequent increased collagen deposition.…”

Section: New Insights Into the Pathogenesis And Management Of Jssc Reviewmentioning

confidence: 99%

New insights into the pathogenesis and management of juvenile systemic sclerosis

Reiff

2015

International Journal of Clinical Rheumatology

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Auto-HSCT induces sustained responses in severe systemic sclerosis patients failing pulse cyclophosphamide

Cited by 8 publications

References 10 publications

Efficacy and safety of autologous haematopoietic stem cell transplantation in systemic sclerosis: a systematic review of the literature

Efficacy and safety of autologous haematopoietic stem cell transplantation in systemic sclerosis: a systematic review of the literature

The role of autologous haemopoietic stem cell transplantation in the treatment of autoimmune disorders

New insights into the pathogenesis and management of juvenile systemic sclerosis

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