Autologous transplantation of hematopoietic stem cells has recently been proposed as a possible treatment for autoimmune diseases that are associated with a very severe prognosis. A 12-year-old girl who, since 4 years of age, had systemic sclerosis with progressive pulmonary involvement underwent autologous peripheral blood-derived stem cell transplantation (aPBSCT) using CD34+ selection, cyclophosphamide, and the infusion of the monoclonal antibody CAMPATH-1G. Following transplantation, in the absence of any treatment other than symptomatic therapy, the patient's exertional dyspnea and alveolitis disappeared and she experienced a marked improvement in skin score, height velocity, and general well-being that has persisted 2 years after the transplantation procedure. Autologous PBSCT associated with the infusion of the monoclonal antibody CAMPATH-1G appears to be a useful therapy for otherwise intractable forms of progressive systemic sclerosis.
Marked and sustained improvement two years after autologous stem cell transplantation in a girl with systemic sclerosis / Martini, A.; Maccario, R.; Ravelli, A.; Montagna, D.; De Benedetti, F.; Bonetti, F.; Viola, S.; Zecca, M.; Perotti, C.; Locatelli, F.. - In: ARTHRITIS AND RHEUMATISM. - ISSN 0004-3591. - 42:4(1999), pp. 807-811. [10.1002/1529-0131(199904)42:4<807::AID-ANR26>3.0.CO;2-T]
Marked and sustained improvement two years after autologous stem cell transplantation in a girl with systemic sclerosis
Locatelli F.
1999
Abstract
Autologous transplantation of hematopoietic stem cells has recently been proposed as a possible treatment for autoimmune diseases that are associated with a very severe prognosis. A 12-year-old girl who, since 4 years of age, had systemic sclerosis with progressive pulmonary involvement underwent autologous peripheral blood-derived stem cell transplantation (aPBSCT) using CD34+ selection, cyclophosphamide, and the infusion of the monoclonal antibody CAMPATH-1G. Following transplantation, in the absence of any treatment other than symptomatic therapy, the patient's exertional dyspnea and alveolitis disappeared and she experienced a marked improvement in skin score, height velocity, and general well-being that has persisted 2 years after the transplantation procedure. Autologous PBSCT associated with the infusion of the monoclonal antibody CAMPATH-1G appears to be a useful therapy for otherwise intractable forms of progressive systemic sclerosis.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.