title: Prolonged response to oral gefitinib, cyclophosphamide, and topotecan in heavily pretreated relapsed stage 4 neuroblastoma: a case report.
abstract: A girl with metastatic neuroblastoma diagnosed at 4 years of age experienced an early disseminated relapse after high-dose chemotherapy. After reinduction, compassionate treatment with gefitinib (250 mg/d fixed dose) with oral topotecan (0.8 mg/m(2)/d) and cyclophosphamide (50 mg/m(2)/d) for 14 consecutive days, each course repeated every 28 days, was administered. Complete marrow clearance and metaiodobenzylguanidine response were achieved after 4 courses. After the first course, topotecan and cyclophosphamide were reduced by 25% for grade 4 hematologic toxicity. Subsequent courses were well tolerated. The girl remained progression free for 27 months. This combination may represent a viable therapeutic option and deserves further evaluation in resistant neuroblastoma.
authors.affiliation: Division of Pediatric Oncology, Ospedale Pediatrico Bambino Gesù IRCCS, Rome, Italy. donfrancesco@opbg.net
authors.forename: Alberto
authors.initials: A
authors.lastname: Donfrancesco
authors.forename: Alessandro
authors.initials: A
authors.lastname: Jenkner
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: De Ioris
authors.forename: Ilaria
authors.initials: I
authors.lastname: Ilari
authors.forename: Aurora
authors.initials: A
authors.lastname: Castellano
authors.forename: Clementina
authors.initials: C
authors.lastname: De Laurentis
authors.forename: Maria Carmen
authors.initials: MC
authors.lastname: Garganese
authors.forename: Giuseppe Maria
authors.initials: GM
authors.lastname: Milano
authors.forename: Carlo
authors.initials: C
authors.lastname: Dominici
mesh_terms: D000284:Administration, Oral; D000310:Adrenal Gland Neoplasms / Q000000981:diagnostic imaging / Q000188:drug therapy*; D000971:Antineoplastic Combined Chemotherapy Protocols; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D005260:Female; D000077156:Gefitinib; D006801:Humans; D009447:Neuroblastoma / Q000000981:diagnostic imaging / Q000188:drug therapy*; D011799:Quinazolines / Q000008:administration & dosage*; D011877:Radionuclide Imaging; D012008:Recurrence; D019772:Topotecan / Q000008:administration & dosage*; D016896:Treatment Outcome
chemical_list: D011799:Quinazolines; D019772:Topotecan; D003520:Cyclophosphamide; D000077156:Gefitinib