SURVIVAL FROM LOCALLY INVASIVE OR WIDESPREAD NEUROBLASTOMA WITHOUT CYTOTOXIC THERAPY

Purpose: To test the hypothesis that cytotoxic therapy is not needed a t diagnosis to assure the survival of most patients with non-stage 4 n euroblastoma. Methods: Patients with non-stage 4 disease received no c ytotoxic therapy in the absence of N-myc amplification. The Internatio nal Neuroblastoma Staging System (INSS) was used. Results: Of 84 conse cutive patients with previously untreated, newly diagnosed neuroblasto ma, 31 (37%) had non-stage 4 disease. All 31 patients initially receiv ed no cytotoxic therapy because none of them had N-myc amplification. Nine stage 1 patients are relapse-free. This report focuses on the 22 patients with locally invasive or distant disease: two stage SA with g ross residual tumor postsurgery, 11 stage 2B with ipsilateral or midli ne lymph node involvement, four stage 3, and five stage 4S. Eight of t he 22 patients were older than 1 year. Postsurgery, 13 patients had vi sible residual disease, and two others had markedly increased urinary catecholamine levels for more than 1 year, Recurrent or enlarging tumo rs regressed spontaneously (n = 2) or were excised 5 to 39 months afte r diagnosis (n = 4). One of the latter had chromosome 1p deletions (co mmon in poor-risk neuroblastoma) that were not detected in the patient 's original tumor resected 23 months earlier-findings consistent with clonal evolution or multifocal disease. The patient received chemother apy. All 22 patients are alive 24 to 98 months (median, 64) from diagn osis. Conclusion: Our results suggest that non-stage 4 patient without N-myc amplification can be spared cytotoxic therapy because (1) resid ual postsurgical or recurrent biologically favorable neuroblastoma rar ely evolves into lethal stage 4 disease; and (2) neuroblastoma in lymp h nodes has no prognostic significance. These findings are remarkable because no other cancer includes subtypes that are curable without the rapy to ablate residual disease. (C) 1996 by American Society of Clini cal Oncology.