Rheumatology 1999; 38: 773
© 1999 British Society for Rheumatology
Paediatric Rheumatology: Autologous Stem Cell Transplantation in Rheumatic Diseases of Childhood |
Marked and sustained improvement 2 years after autologous stem cell transplantation in a girl with systemic sclerosis
Series Editor: P. Woo
Dipartimento di Scienze Pediatriche, Università di Pavia,
1 Laboratori Sperimentali and
2 Servizio di Immunoematologia e Trasfusione, IRCCS Policlinico S. Matteo, Pavia, Italy
Correspondence to:
A. Martini, Clinica Pediatrica, Universitá di Pavia, IRCCS Policlinico San Matteo, P. le Golgi 2, 27100 Pavia, Italy.
Recently, autologous peripheral blood stem cell (PBSC) transplantation has been proposed as a possible treatment for autoimmune diseases associated with a very severe prognosis.
IP, a girl affected by a severe form of systemic sclerosis, had the first symptoms of her disease at the age of 4 yr. The disease course was characterized by progressive cutaneous, articular and pulmonary involvement, and stunted growth. Despite aggressive immunosuppressive treatment including steroids, cyclophosphamide (CY), methotrexate and D-penicillamine, her lung function continued to deteriorate, leading to significant functional disability. Since pulmonary fibrosis, once established, is progressive, at the age of 11 yr she discontinued all medications and received an autologous PBSC transplantation.
After obtaining local ethical committee approval and parents' informed consent, the child was given CY (4 g/m2 ) followed by granulocyte colony-stimulating factor (10 mg/kg/day) in order to allow the collection of PBSC. The pre-transplant conditioning regimen consisted of CY at a dose of 50 mg/kg from day -5 to day -2 and the monoclonal antibody Campath-1G at a dose of 10 mg/day for 2 days. The post-transplant period was uneventful.
Following transplantation, in the absence of any treatment other than symptomatic, the girl had an impressive improvement that persists 2 yr after the transplant procedure. Exertional dyspnoea and alveolitis disappeared, and a marked general skin softening was observed with an improvement in skin score from 21 to 10. She had an impressive improvement in her growth rate (+16 cm); parent global assessment of general well-being by means of a visual analogue scale (range 0–3) improved from 2.1 to 0.8 and the CHAQ disability index from 2.87 to 2.4 transplantation.
Autologous PBSC transplantation associated with infusion of the monoclonal antibody Campath-1G appears to be a promising therapy for otherwise intractable forms of systemic progressive sclerosis.
Accepted 15 March 1999
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