A multicenter phase II trial of 4′-iodo-4′deoxydoxorubicin (IDOX) in primary amyloidosis (AL)

Introduction. 4'-Iodo-4'-demydoxorubicin (IDOX) has been reported to bind to and lead to the catabolism of amyloid deposits. A multicenter stuck, attempted to develop a dosing schedule to confirm those results. Methods. Patients with biopsy-proven amyloidosis were required to have a cardiac ejection fraction > 50%, ventricular septal thickness < 20 mm, serum creatinine < 2.5 mg per dL, bilirubin < 2. 0 mg per dL, neutrophils > 1, 500 per muL, andplatelets > 100, 000 per uL. IDOX was administered intravenously over 1 hour at a dose of 15 mg per m, once a week for 4 consecutive weeks. This therapy was repeated every 3 months tip to 4 times. Results. Twenty-five previously treated and 15 untreated patients with primary, amyloidosis (AL) received therapy. Fifteen patients had > 3 g of protein per day in the urine. Eleven patients had an ejection fraction < 60%. One, 2, 3, 4, and 5 organ systems were involved in 22, 10, 4, 3, and 1 patients respectively. The median time between diagnosis and initiation of IDOX was 17.4 months. There were 6 responses (15%). Twelve of the patients have died. Conclusion. IDOX administered in this protocol was insufficiently active at the current dose.