A multicenter phase II trial of 4'-iodo-4'deoxydoxorubicin (IDOX) in primary amyloidosis (AL).
BACKGROUND
4'-Iodo-4'-deoxydoxorubicin (IDOX) has been reported to bind to and lead to the catabolism of amyloid deposits. A multicenter study 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 < or = 2.0 mg per dL, neutrophils > 1,500 per microL, and platelets > 100,000 per microL. IDOX was administered intravenously over 1 hour at a dose of 15 mg per m2 once a week for 4 consecutive weeks. This therapy was repeated every 3 months up 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 patient, 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


CONCLUSIONS
IDOX administered in this protocol was insufficiently active at the current dose.
Gertz
Morie A
MA
Division of Hematology and Internal Medicine, Mayo Clinic, Rochester, MN 55905, USA. gertm@mayo.edu
Lacy
Martha Q
MQ
Dispenzieri
Angela
A
Cheson
Bruce D
BD
Barlogie
Bart
B
Kyle
Robert A
RA
Palladini
Giovanni
G
Geyer
Susan M
SM
Merlini
Giampaolo
G
D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy*; D004317:Doxorubicin / Q000031:analogs & derivatives / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged
C053044:4'-deoxy-4'-iododoxorubicin; D004317:Doxorubicin