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Pilot study of antithymocyte globulin in systemic sclerosis
E L Matteson1, M I Shbeeb, T G McCarthy
1Mayo Clinic and Foundation, Rochester, MN 55905, USA.
Arthritis and Rheumatism
|July 1, 1996
Summary
Antithymocyte globulin (ATGAM) did not improve skin or lung disease in early systemic sclerosis (SSc) patients. The pilot study found ATGAM ineffective for treating SSc progression.
Area of Science:
- Immunology
- Rheumatology
- Pulmonology
Background:
- Systemic sclerosis (SSc) is a chronic autoimmune disease characterized by skin thickening and internal organ involvement.
- Early diagnosis and intervention are crucial for managing SSc progression and improving patient outcomes.
Purpose of the Study:
- To evaluate the efficacy of antithymocyte globulin (ATGAM) in preventing the progression of cutaneous and pulmonary manifestations in early systemic sclerosis.
- To assess the safety and tolerability of ATGAM in patients with early SSc.
Main Methods:
- An open pilot study involving 10 adult patients with early SSc (< 3 years) and progressive skin and pulmonary disease.
- Patients received a single course of intravenous ATGAM (10 mg/kg/day for 5 days).
- Clinical assessments included Rodnan skin score and pulmonary function tests (diffusing capacity for carbon monoxide, forced vital capacity) over 12 months.
Main Results:
- ATGAM was generally tolerated, with adverse events including allergic reaction, serum sickness, and deep vein thrombosis.
- Two patients died from SSc-related complications during the follow-up period.
- At 12 months, only 2 out of 10 patients showed improvement in both skin and pulmonary measures; 5 worsened, and 3 remained stable.
Conclusions:
- Antithymocyte globulin (ATGAM), at the dosage and regimen used, appears ineffective in improving the skin and pulmonary features of early systemic sclerosis.
- Further research is needed to identify effective treatments for SSc progression.