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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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CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS

GIMEMA-AIEOPAIDA 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 Aricò, Giuseppe Basso, Robert Foa, and Franco Mandelli

From the Dipartimento di Biotecnologie Cellulari ed Ematologia, Università "La Sapienza," Rome; the Dipartimento di Biopatologia e Diagnostica per Immagini, Università "Tor Vergata," Roma; Clinica Pediatrica, Ospedale Nuovo "San Gerardo," Monza; the Divisione Pediatria Ematologia, Ospedale Pausilipon, Napoli; Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia; the Dipartimento di Pediatria, Università di Bologna,; the Dipartimento Scienze Pediatriche Adolescenza, Università di Torino; Divisione di Ematologia Pediatrica, Ospedale Bambin Gesú, Roma; the Dipartimento di Ematologia-Oncologia, Istituto Giannina Gaslini, Genova; Oncoematologia Pediatrica, Ospedale dei Bambini, Palermo; and Clinica Pediatrica, Università di Padova; Italy.

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 Gruppo Italiano per le Malattie Ematologiche dell'Adulto (GIMEMA)–Italian Pediatric Hematology and Oncology Group (AIEOP) "AIDA" (ATRA and idarubicin) trial are presented. Of the 983 patients with APL enrolled in this protocol between January 1993 and June 2000, 124 (13%) had younger than 18 years. Treatment consisted of ATRA and idarubicin induction followed by 3 polychemotherapy consolidation courses. Molecular response by reverse transcriptase–polymerase chain reaction (RT-PCR) was assessed after consolidation and patients who were PCR were randomized for different maintenances. One hundred and seven children were eligible and evaluable for induction: 103 (96%) achieved a hematologically complete remission. Overt ATRA syndrome was observed in 2 patients and pseudotumor cerebri was observed in 10 patients. Ninety-four patients were evaluable for RT-PCR analysis at the end of consolidation: 91 (97%) proved PCR+ and 3 PCR. The overall survival and event-free survival (EFS) are 89% (95% confidence interval [c.i.]: 83%-95%) and 76% (c.i.: 65%-85%), respectively, at more than 10 years. A white blood cell (WBC) count at diagnosis of greater than 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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