Marked and sustained improvement two years after autologous stem cell transplantation in a girl with systemic sclerosis.
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.
Martini
A
A
Department of Pediatrics, Universita' di Pavia, IRCCS Policlinico S. Matteo, Italy.
Maccario
R
R
Ravelli
A
A
Montagna
D
D
De Benedetti
F
F
Bonetti
F
F
Viola
S
S
Zecca
M
M
Perotti
C
C
Locatelli
F
F
D000293:Adolescent; D000911:Antibodies, Monoclonal; D018952:Antigens, CD34 / Q000032:analysis; D001827:Body Height; D005260:Female; D006128:Growth; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000737:chemistry / Q000276:immunology; D006801:Humans; D011379:Prognosis; D012595:Scleroderma, Systemic / Q000628:therapy*; D013601:T-Lymphocytes / Q000276:immunology; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D000911:Antibodies, Monoclonal; D018952:Antigens, CD34