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

Morie A. Gertz, Martha Q. Lacy, Angela Dispenzieri, Bruce D. Cheson, Bart Barlogie, Robert A. Kyle, Giovanni Palladini, Susan M. Geyer, Giampaolo Merlini

Research output: Contribution to journalArticlepeer-review


Introduction. 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 ≤ 2.0 mg per dL, neutrophils <1,500 per μL, and platelets > 100,000 per μL. 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. Conclusion. IDOX administered in this protocol was insufficiently active at the current dose.

Original languageEnglish
Pages (from-to)24-30
Number of pages7
Issue number1
Publication statusPublished - 2002


  • Amyloid
  • Amyloidosis
  • Anthracycline
  • Cardiomyopathy
  • Chemotherapy
  • Nephrotic syndrome

ASJC Scopus subject areas

  • Pathology and Forensic Medicine
  • Medicine(all)

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