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Blood, 1 October 2004, Vol. 104, No. 7, pp. 1995-1999.
Prepublished online as a Blood First Edition Paper on June 8, 2004; DOI 10.1182/blood-2004-04-1550.
Previous Article | Next Article 
Submitted April 26, 2004
Accepted May 30, 2004
Gentuzumab ozogamicin (''mylotarg'') as a single agent for molecularly relapsed acute promyelocytic leukemia
Francesco Lo-Coco*, Giuseppe Cimino, Massimo Breccia, Nelida I Noguera, Daniela Diverio, Erica Finolezzi, Enrico M Pogliani, Eros Di Bona, Concetta Micalizzi, Mariagrazia Kropp, Adriano Venditti, Agostino Tafuri, and Franco Mandelli
Biopathology, University Tor Vergata, Roma, Italy
Cellular Biotechnology and Hematology, University La Sapienza, Roma, Italy
Medicine, Ospedale S. Gerardo, Milano-Monza, Italy
Terapia Cellulare ed Ematologia, Ospedale S. Bortolo, Vicenza, Italy
Ematologia ed Oncologia Pediatrica, Ospedale G. Gaslini, Genova, Italy
Ospedale Regionale, Catanzaro, Italy
* Corresponding author; email: francesco.lo.coco{at}uniroma2.it.
The anti-CD33 antibody-calicheamicin conjugate gentuzumab-ozogamicin (GO) was used as a single agent to treat 16 patients with acute promyelocytic leukemia relapsed at the molecular level. Of these, 8 patients were in first, 5 in second, 2 in third and 1 in fourth relapse. GO was administered at 6 mg/m2 for 2 doses and patients achieving a new molecular remission (MR, i.e. negativization of the RT-PCR test for PML/RAR ) received a final third dose. MR was obtained in 9/11 (91%) patients tested after 2 doses and in 13/13 (100%) patients tested after the third dose. Of the 3 remaining patients, 1 achieved MR after a single GO administration and received no further therapy due to hepatic toxicity, and 2 showed disease progression during treatment (morphological relapse). Quantitative RT-PCR studies showed in responding patients a dramatic decline (greater than 2 logs) of the hybrid PML/RAR transcript after the first GO dose. Of the 14 responders, 7 patients remained in sustained MR for a median time of 15 mos. (range 7-31 mos.) and received no further therapy, while 7 relapsed at 3-15 mos. GO was administered again in 2 relapsing patients and both obtained a new MR. These data indicate that: i) GO is highly effective as single treatment for patients with molecularly relapsed APL including those with very advanced disease; ii) Further treatment should be used in these patients after MR in order to obtain prolonged molecular response.

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