title: GIMEMA-AIEOPAIDA protocol for the treatment of newly diagnosed acute promyelocytic leukemia (APL) in children.
abstract: 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 10(9)/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.
authors.affiliation: Dipartimento di Biotecnologie Cellulari ed Ematologia, Università La Sapienza, Via Benevento 6, 00161 Rome, Italy. testi@bce.uniroma1.it
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Testi
authors.forename: Andrea
authors.initials: A
authors.lastname: Biondi
authors.forename: Francesco
authors.initials: F
authors.lastname: Lo Coco
authors.forename: Maria Luisa
authors.initials: ML
authors.lastname: Moleti
authors.forename: Fiorina
authors.initials: F
authors.lastname: Giona
authors.forename: Marco
authors.initials: M
authors.lastname: Vignetti
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Menna
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Elena
authors.initials: E
authors.lastname: Barisone
authors.forename: Giulio
authors.initials: G
authors.lastname: De Rossi
authors.forename: Daniela
authors.initials: D
authors.lastname: Diverio
authors.forename: Concetta
authors.initials: C
authors.lastname: Micalizzi
authors.forename: Maurizio
authors.initials: M
authors.lastname: Aricò
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Basso
authors.forename: Robert
authors.initials: R
authors.lastname: Foa
authors.forename: Franco
authors.initials: F
authors.lastname: Mandelli
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D002985:Clinical Protocols; D005260:Female; D006801:Humans; D015255:Idarubicin / Q000008:administration & dosage* / Q000009:adverse effects; D007223:Infant; D007558:Italy; D015473:Leukemia, Promyelocytic, Acute / Q000188:drug therapy* / Q000235:genetics; D008297:Male; D009363:Neoplasm Proteins / Q000235:genetics; D015514:Oncogene Proteins, Fusion / Q000235:genetics; D012333:RNA, Messenger / Q000235:genetics; D012334:RNA, Neoplasm / Q000235:genetics; D014212:Tretinoin / Q000008:administration & dosage* / Q000009:adverse effects
chemical_list: D009363:Neoplasm Proteins; D015514:Oncogene Proteins, Fusion; D012333:RNA, Messenger; D012334:RNA, Neoplasm; C093476:promyelocytic leukemia-retinoic acid receptor alpha fusion oncoprotein; D014212:Tretinoin; D015255:Idarubicin