PILOT-STUDY OF ANTITHYMOCYTE GLOBULIN IN SYSTEMIC-SCLEROSIS

Objective. Between June 1, 1992 and August 31, 1994 we conducted an op en pilot study of antithymocyte globulin (ATGAM; Upjohn, Kalamazoo, MI ) in 10 patients with early systemic sclerosis (SSc) to assess whether this agent might prevent the progression of cutaneous and pulmonary i nvolvement in this disease. Methods. Adult patients with early SSc (<3 Sears) and evidence of progressive skin and pulmonary disease were en rolled. All patients were hospitalized and received a single course of intravenous ATGAM, at a dosage of 10 mg/kg over 4 hours, on 5 consecu tive days, Patients were followed up at weeks 1, 2, 3, and 4, and mont hs 2, 3, 6, and 12. Patients were considered to be improved if the Rod nan skin score decreased greater than or equal to 25%, to be worse if the skin score increased greater than or equal to 25%, and to be not i mproved if the skin score was within 25% of baseline, For pulmonary in volvement, patients were considered to be improved if either the diffu sing capacity for carbon monoxide or the forced vital capacity was inc reased greater than or equal to 10%, worse if decreased by greater tha n or equal to 10%, and stable if within 10% of baseline. Results. Most patients tolerated the treatment well, although 1 patient developed a n allergic reaction necessitating discontinuation of treatment, 1 deve loped a serum sickness reaction after completion of therapy, and 1 dev eloped a central venous access-related axillary vein thrombosis, Two p atients died of SSc-related complications during the followup period, At 12 months, only 2 patients showed improvement in both skin and pulm onary function measures, whereas 5 patients were worse and 3 were stab le. Conclusion. At the dosage administered in this study, ATGAM appear s ineffective in improving the skin and pulmonary features of SSc.