Objective: To evaluate the effectiveness of low oral doses of methotre
xate as a steroid-sparing agent in children with sarcoidosis. Study de
sign: An open-label, noncontrolled trial. Methotrexate was administere
d orally at a single weekly dose of 10 to 15 mg/m(2). Duration of ther
apy was open ended, but patients received treatment for a minimum of 6
months to be considered as having completed the study. Results: Seven
children with biopsy-proven sarcoidosis completed the study. The mean
dose of prednisone was successfully tapered from 49 mg/day (1.3 mg/kg
) to 18 mg/day (0.5 mg/kg) after 3 months of methotrexate therapy and
to 9.9 (0.2 mg/kg) and 7.3 mg/day (0.1 mg/kg) after 6 months and at th
e end of the follow-up period, respectively. Other clinical and labora
tory parameters improved significantly after methotrexate therapy was
started. There was significant clinical improvement, as confirmed by t
he reduction of the clinical severity score from 8 +/- 1.1 to 0.8 +/-
0.5 point after 3 months of methotrexate therapy, and to 0.7 and 0.5 /- 0.3 point after 6 months and at the end of the follow-up, respectiv
ely. Laboratory measurements revealed marked improvement, as reflected
by a significant reduction in the erythrocyte sedimentation rate and
an increase of hemoglobin values. The mean serum angiotensin-convertin
g enzyme activity dropped significantly. No adverse side effects were
noted with methotrexate therapy. Conclusion: Our study demonstrated th
at low-dose oral methotrexate therapy was effective and safe and had s
teroid-sparing properties in seven children with sarcoidosis.