Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysis

Burt, Richard K.; Oliveira, Maria Carolina; Shah, Sanjiv J.; Moraes, Daniela A.; Simões, Belinda Pinto; Gheorghiade, Mihai; Schroeder, James; Ruderman, Eric; Farge, Dominique; Chai, Zhong-Pei; Marjanovic, Zora; Jain, Sandeep; Morgan, Amy; Milanetti, Francesca; Han, Xiaoqiang; Jovanovic, Borko; Helenowski, Irene; Voltarelli, Júlio César

doi:10.1016/s0140-6736(12)62114-x

Cited by 136 publications

(122 citation statements)

References 30 publications

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“…The RI was higher in the presence of gastrointestinal involvement at SSc diagnosis (HR: 2.31; 95% CI 1.14-4.67; P = 0.02). These results are supported by previous studies where we 13,14 and others 15 had shown that heart involvement in SSc negatively affects patient outcome.…”

Section: Resultssupporting

confidence: 91%

Does ex vi vo CD34+ positive selection influence outcome after autologous hematopoietic stem cell transplantation in systemic sclerosis patients?

Soga

Labopin

Henes

et al. 2015

Bone Marrow Transplant

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This EBMT Autoimmune Disease Working Party study aimed to evaluate the influence of CD34+ positive graft selection (CD34+) on the outcome of systemic sclerosis (SSc) patients after autologous hematopoietic stem cell transplantation (AHSCT). Clinical and laboratory data from 138 SSc patients at diagnosis, before and after AHSCT were retrospectively analyzed. CD34+ selection was performed in 47.1% (n = 65) patients. By multivariate analysis adjusting for all factors differing between the two groups (without or with CD34+), there was no statistically significant difference in terms of overall survival (hazard ratio (HR): 0.98, 95% confidence interval (CI) 0.40-2.39, P = 0.96), PFS (HR: 1.55, 95% CI 0.83-2.88, P = 0.17) and incidence of relapse or progression (HR: 1.70, 95% CI 0.85-3.38, P = 0.13). We demonstrate that CD34+ does not add benefit to the outcome of SSc patient treated with AHSCT. These findings should be further confirmed by prospective randomized trials.

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Section: Resultssupporting

confidence: 91%

Does ex vi vo CD34+ positive selection influence outcome after autologous hematopoietic stem cell transplantation in systemic sclerosis patients?

Soga

Labopin

Henes

et al. 2015

Bone Marrow Transplant

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“…13 Recent studies suggest that a relevant lowering of mortality, as a direct consequence of AHSCT, could be achieved thanks to a more accurate selection of candidate patients and, namely, to a more careful pre-transplant evaluation of lung and heart conditions. 23 This more careful patient selection was suggested after the retrospective analysis of the AHSCT-treated patients that demonstrated that most of the patients that died after AHSCT had such a bad outcome because of poor cardiac conditions before transplant. 23 Thus, a more accurate heart assessment before directing a patient to AHSCT is certainly needed.…”

Section: Discussionmentioning

confidence: 99%

“…23 This more careful patient selection was suggested after the retrospective analysis of the AHSCT-treated patients that demonstrated that most of the patients that died after AHSCT had such a bad outcome because of poor cardiac conditions before transplant. 23 Thus, a more accurate heart assessment before directing a patient to AHSCT is certainly needed. Right catheterization with fluid challenge and heart magnetic resonance evaluation are the diagnostic tools that have been proposed for this purpose.…”

Section: Discussionmentioning

confidence: 99%

Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Papa

Onida

Zaccara

et al. 2016

Bone Marrow Transplant

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We retrospectively evaluated the efficacy of autologous hematopoietic stem cell transplantation (AHSCT) in 18 patients with rapidly progressive diffuse cutaneous systemic sclerosis (rp-dcSSc), and compared their disease outcomes with those of 36 demographically-and clinically-matched patients treated with conventional therapies. Cutaneous involvement, by performing modified Rodnan skin score (mRss), lung diffusion capacity, by measuring diffusing capacity of lung for carbon monoxide (DLCO), and disease activity, by applying the European Scleroderma Study Group (ESSG) scoring system, were the outcome variables measured at the baseline time and then every 12 months for the following 60 months in both the AHSCT-treated patients and the control group. In the AHSCT group, treatment-related mortality was 5.6%. In this group, both mRss and ESSG scores showed a significant reduction 1 year after AHSCT (P o 0.002); and these results were maintained until the end of follow-up. Conversely, DLCO values remained stable during the whole period of follow-up. Survival rate of AHSCT group was much higher than that observed in the whole control group (P = 0.0005). The probability that the ESSG score and mRss would remain at a high level, and DLCO could decrease, was significantly higher in the control group as a whole and in the subgroup of control patients treated with cyclophosphamide than in the AHSCT group. This study confirms that the AHSCT is effective in prolonging survival, as well as in inducing a rapid reduction of skin involvement and disease activity, and preserving lung function in patients with rp-dcSSc.

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“…Autologous transplant-related mortality is approximately 3-5% for patients with myeloma and lymphoma and approximately 5-10% for those with scleroderma (23, 27, 30, 31). Transplant is an intensive procedure with significant side effects including nausea, vomiting, diarrhea and oral mucositis which usually resolve with blood count recovery.…”

Section: The Process Of Autologous Hsctmentioning

confidence: 99%

Review: Hematopoietic Stem Cell Transplantation for Scleroderma: Effective Immunomodulatory Therapy for Patients With Pulmonary Involvement

Sullivan

Shah

Sarantopoulos

et al. 2016

Arthritis & Rheumatology

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Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysis

Cited by 136 publications

References 30 publications

Does ex vi vo CD34+ positive selection influence outcome after autologous hematopoietic stem cell transplantation in systemic sclerosis patients?

Does ex vi vo CD34+ positive selection influence outcome after autologous hematopoietic stem cell transplantation in systemic sclerosis patients?

Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Review: Hematopoietic Stem Cell Transplantation for Scleroderma: Effective Immunomodulatory Therapy for Patients With Pulmonary Involvement

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