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.
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