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Blood, 15 July 2005, Vol. 106, No. 2, pp. 447-453.
Prepublished online as a Blood First Edition Paper on January 27, 2005; DOI 10.1182/blood-2004-05-1971.
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Submitted June 7, 2004
Accepted December 20, 2004
GIMEMA-AIEOP AIDA protocol for the treatment of newly diagnosed acute promyelocytic leukemia (APL) in children
Anna Maria Testi*, Andrea Biondi, Francesco Lo Coco, Maria Luisa Moleti, Fiorina Giona, Marco Vignetti, Giuseppe Menna, Franco Locatelli, Andrea Pession, Elena Barisone, Giulio De Rossi, Daniela Diverio, Concetta Micalizzi, Maurizio Arico, Giuseppe Basso, Robert Foa, and Franco Mandelli
Dipartimento di Biotecnologie Cellulari ed Ematologia, Universita 'La Sapienza', Roma, Italy
Clinica Pediatrica, Ospedale Nuovo 'San Gerardo', Monza, Italy
Dipartimento di Biopatologia e Diagnostica per Immagini, Universita 'Tor Vergata', Roma, Italy
Divisione Pediatria Ematologia, Ospedale Pausilipon, Napoli, Italy
Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy
Dipartimento di Pediatria, Universita di Bologna, Bologna, Italy
Dipartimento Scienze Pediatriche Adolescenza, Universita di Torino, Torino, Italy
Divisione Ematologia Pediatrica, Ospedale Bambin Gesu, Roma, Italy
Dipartimento di Ematologia-Oncologia, Istituto Giannina Gaslini, Genova, Italy
Oncoematologia Pediatrica, Ospedale dei Bambini, Palermo, Italy
Clinica Pediatrica, Universita di Padova, Padova, Italy
* Corresponding author; email: testi{at}bce.uniroma1.it.
The role of all-trans retinoic acid (ATRA) in pediatric acute promyelocytic leukemia (APL) is the topic of several ongoing studies. The results of the Italian pediatric experience with the multicentric GIMEMA-AIEOP "AIDA" trial are presented. Of the 983 patients with APL enrolled in this protocol between January 1993 and June 2000, 124 (13%) had less than 18 years. Treatment consisted of ATRA and Idarubicin induction followed by 3 polychemotherapy consolidation courses. Molecular response by RT-PCR was assessed after consolidation and patients who were PCR-negative were randomized for different maintenances. One hundred and seven children were eligible and evaluable for induction: 103 (96%) achieved an hematological complete remission. Overt ATRA syndrome was observed in 2 patients and pseudotumor cerebri in 10. Ninety four patients were evaluable for RT-PCR analysis at the end of consolidation: 91 (97%) proved PCR-negative and 3 PCR-positive. The overall survival and event-free-survival (EFS) are 89% (95% c.i: 83-95%) and 76% (c.i: 65-85%), respectively, at more than 10 years. The WBC count at diagnosis > 10 x 109/l had a significant impact on EFS (59% vs 83%, at 10 years). These results highlight the efficacy and feasibility of the AIDA protocol in the pediatric APL population.

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