Prospective study.
SETTING: Tertiary referral center.
PATIENTS: In 10 patients, response to
therapy was determined by pretreatment and posttreatment clinical (
dyspnea), radiographic (chest radiograph), and physiologic (pulmonary function, including exercise saturation) scores (CRP). Response was defined as a > 10-point drop in CRP; stable as +/- 10-point change in CRP; and nonresponders as > 10-point rise in CRP. In nine patients, physiologic criteria were used to assess response; significant changes in pulmonary function were defined as follows: total lung capacity, +/- 10% of baseline value; FVC, +/- 10% of baseline value, diffusion capacity of the lung for
carbon monoxide, +/- 20% of baseline value; and resting pulse oximetry, +/- 4% of baseline value. Patients who died while receiving or shortly after discontinuing
cyclophosphamide were classified as nonresponders (n = 2). Among 19 patients treated with
cyclophosphamide, only 1 patient demonstrated sustained response; 7 patients remained stable and 11 deteriorated while receiving the
drug. Toxicity associated with
cyclophosphamide was substantial; more than two thirds of the patients developed
drug-related adverse effects, and almost half discontinued the
drug prematurely due to side effects. In the remaining patients,
cyclophosphamide therapy was discontinued due to lack of improvement or progressive deterioration.
CONCLUSIONS: