title: Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis.
abstract: 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<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.
authors.affiliation: U.O.C. Day Hospital Reumatologia, Centro Specialistico Ortopedico, Traumatologico G. Pini-CTO, Milano, Italy.
authors.forename: N
authors.initials: N
authors.lastname: Del Papa
authors.affiliation: U.O.C. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Onida
authors.affiliation: U.O.C. Day Hospital Reumatologia, Centro Specialistico Ortopedico, Traumatologico G. Pini-CTO, Milano, Italy.
authors.forename: E
authors.initials: E
authors.lastname: Zaccara
authors.affiliation: U.O.C. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: G
authors.initials: G
authors.lastname: Saporiti
authors.affiliation: U.O.C. Day Hospital Reumatologia, Centro Specialistico Ortopedico, Traumatologico G. Pini-CTO, Milano, Italy.
authors.forename: W
authors.initials: W
authors.lastname: Maglione
authors.affiliation: U.O.C. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: E
authors.initials: E
authors.lastname: Tagliaferri
authors.affiliation: U.O.C. Day Hospital Reumatologia, Centro Specialistico Ortopedico, Traumatologico G. Pini-CTO, Milano, Italy.
authors.forename: R
authors.initials: R
authors.lastname: Andracco
authors.affiliation: U.O.C. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: D
authors.initials: D
authors.lastname: Vincenti
authors.affiliation: Cell Factory and Cell Therapy Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: T
authors.initials: T
authors.lastname: Montemurro
authors.affiliation: U.O.C. Malattie Cardiovascolari, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: L
authors.initials: L
authors.lastname: Mircoli
authors.affiliation: Sezione di Reumatologia, Istituto San Giuseppe, Anzano del Parco, Como, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Vitali
authors.affiliation: U.O.C. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
authors.forename: A
authors.initials: A
authors.lastname: Cortelezzi
mesh_terms: D000328:Adult; D064592:Autografts; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D045743:Scleroderma, Diffuse / Q000401:mortality* / Q000628:therapy*; D015996:Survival Rate