Localized infant neuroblastomas often show spontaneous regression: results of the prospective trials NB95-S and NB97.
OBJECTIVE
The excellent prognosis of localized neuroblastoma in infants, the overdiagnosis observed in neuroblastoma screening studies, and several case reports of regression of localized neuroblastoma prompted us to initiate a prospective cooperative trial on observation of localized neuroblastoma without cytotoxic treatment.


METHODS
For infants with localized neuroblastoma without MYCN amplification, chemotherapy was scheduled only in cases with threatening symptoms; otherwise, the tumor was either resected or observed by ultrasound and magnetic resonance imaging (MRI).


RESULTS
Of 340 eligible participants, 190 underwent resection, 57 were treated with chemotherapy, and 93 were observed with gross residual tumor. Of those 93 patients with unresected tumors, spontaneous regression was seen in 44, local progression in 28, progression to stage 4S in seven, and progression to stage 4 in four. Time to regression was quite variable, with first signs of regression noted 1 to 18 months after diagnosis and in 15 of 44 patients even after the first year of life. So far, complete regression was observed in 17 of 44 patients 4 to 20 months after diagnosis. Known clinical risk factors were not able to differentiate between patients with regression and regional or metastatic progression. Overall survival (OS; 3-year OS, 0.99 +/- 0.01) and metastases-free survival (rate at 3 years, 0.94 +/- 0.03) for patients with unresected tumors was excellent and was not different from patients treated with surgery or chemotherapy.


CONCLUSIONS
Spontaneous regression is regularly seen in infants with localized neuroblastoma and is not limited to the first year of life. A wait-and-see strategy is justified in those patients.
Hero
Barbara
B
Children's Hospital, Department of Pediatric Oncology and Hematology, University of Cologne, Kerpener Str. 62, 50924 Cologne, Germany. barbara.hero@uk-koeln.de
Simon
Thorsten
T
Spitz
Ruediger
R
Ernestus
Karen
K
Gnekow
Astrid K
AK
Scheel-Walter
Hans-Guenther
HG
Schwabe
Dirk
D
Schilling
Freimut H
FH
Benz-Bohm
Gabriele
G
Berthold
Frank
F
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009365:Neoplasm Regression, Spontaneous; D009367:Neoplasm Staging; D009447:Neuroblastoma / Q000175:diagnosis* / Q000000981:diagnostic imaging / Q000188:drug therapy / Q000473:pathology / Q000601:surgery; D011379:Prognosis; D011446:Prospective Studies; D016019:Survival Analysis; D014463:Ultrasonography