Multiple Sclerosis, 1 October 2003, vol. 9, no. 5, pp. 446-450(5)
Portaccio E.; Zipoli V.; Siracusa G.; Piacentini S.; Sorbi S.; Amato M.P.
 Department of Neurology, University of Florence, Florence, Italy
To assess the safety and tolerability of cyclophosphamide (CTX) 'pulse' therapy in progressive or very active multiple sclerosis (MS), we reviewed our experience in a cohort of MS patients who have been treated and prospectively followed-up in our Department since 1997.
One hundred and twelve patients received intravenous CTX in monthly 'pulses' for 12 months at the dosage of 700 mg/m2 of body surface, then bimonthly for another 12 months.
We evaluated the frequency and the severity of side-effects, as well as overall tolerability by the patient perspective using a visual analogue scale (VAS).
Side-effects resulted in the discontinuation of therapy in 20 (18%) cases.
Serious side-effects were observed in 24 patients (21.4%), most commonly definitive amenorrhea (33.3% of fertile women), hypogammaglobulinemia (5.4%), and hemorrhagic cystitis (4.5%).
Malignancies were diagnosed in four (3.6%) subjects, three of whom were previously treated with azathioprine (AZA).
Finally, 81.8% of the patients judged the treatment regimen as very or relatively acceptable and tolerable.
Our data point to a reasonable safety and tolerability of CTX 'pulse' therapy.
Further trials are needed to definitively assess the efficacy of CTX as an alternative therapeutic option for progressive or very active MS.