Surgery and observation (in infants)

A French study classified infants as stage 4 because of a primary tumor infiltrating across the midline (INSS stage 3 primary with metastases limited to 4S category) or positive bone scintigraphy not associated with changes in the cortical bone documented on plain radiographs and/or CT.[16]

A French study classified infants as stage 4 because of a primary tumor infiltrating across the midline (INSS stage 3 primary with metastases limited to 4S category) or positive bone scintigraphy not associated with changes in the cortical bone documented on plain radiographs and/or CT.[16]

Infants with this classification were reported to have a better outcome with less aggressive chemotherapy than were other stage 4 infants (EFS rate, 90% vs. 27%).

A French study classified infants as stage 4 because of a primary tumor infiltrating across the midline (INSS stage 3 primary with metastases limited to 4S category) or positive bone scintigraphy not associated with changes in the cortical bone documented on plain radiographs and/or CT.[16]

However, a much higher proportion of those with radiologically demonstrated cortical bone lesions also had tumors with MYCN amplification.

Building on the French study, SIOPEN conducted a prospective trial of 125 infants (n = 41 with INSS 3 primary tumors or positive scintigraphy) with disseminated neuroblastoma without MYCN amplification to determine whether these patients could be observed in the absence of symptoms. However, treating physicians did not always follow the wait-and-see strategy.[13]

Building on the French study, SIOPEN conducted a prospective trial of 125 infants (n = 41 with INSS 3 primary tumors or positive scintigraphy) with disseminated neuroblastoma without MYCN amplification to determine whether these patients could be observed in the absence of symptoms. However, treating physicians did not always follow the wait-and-see strategy.[13]

There was no significant difference in 2-year OS rates between patients with unresectable primary tumors and patients with resectable primary tumors (97% vs. 100%) and between patients with negative and positive skeletal scintigraphy without radiological abnormalities (100% vs. 97%).

A German prospective clinical trial enrolled 340 infants aged 1 year or younger whose tumors were stage 1, 2, or 3; verified histologically; and lacked MYCN amplification. Of the 190 infants who underwent resection, 8 had stage 3 disease. A total of 93 infants whose tumors were not resectable without high-risk surgery, because of age or organ involvement, were observed without chemotherapy, which included 21 patients with stage 3 disease. Fifty-seven infants, including 41 with stage 3 disease, were treated with chemotherapy to control threatening symptoms.[5]

A German prospective clinical trial enrolled 340 infants aged 1 year or younger whose tumors were stage 1, 2, or 3; verified histologically; and lacked MYCN amplification. Of the 190 infants who underwent resection, 8 had stage 3 disease. A total of 93 infants whose tumors were not resectable without high-risk surgery, because of age or organ involvement, were observed without chemotherapy, which included 21 patients with stage 3 disease. Fifty-seven infants, including 41 with stage 3 disease, were treated with chemotherapy to control threatening symptoms.[5]

The 3-year OS rate was excellent for the entire group of infants with unresected tumors (99%), infants who received chemotherapy (95%), and infants with resected tumors (98%) (P = .45).