Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Recurrent Neuroblastoma
The current standard of care is based on the experience from the COG Intermediate-Risk treatment plan (COG-A3961). Local regional recurrence of neuroblastoma with favorable biology that occurs more than 3 months after completion of 12 weeks of chemotherapy may be treated surgically. If resection is less than near total, then 12 additional weeks of chemotherapy may be given. Chemotherapy consists of moderate doses of carboplatin, cyclophosphamide, doxorubicin, and etoposide. The cumulative dose of each agent is kept low to minimize permanent injury from the chemotherapy regimen, as used in a prior COG trial (COG-A3961).
If the recurrence is metastatic and/or occurs while on chemotherapy or within 3 months of completing chemotherapy and/or has unfavorable biologic properties, the prognosis is poor and the patient should be treated with an aggressive regimen of combination chemotherapy consisting of very high doses of the drugs listed above, and often also including ifosfamide and high-dose cisplatin. Both myeloablative therapy and postchemotherapy retinoic acid may improve outcome of newly diagnosed patients with a poor prognosis. These modalities are commonly employed in the treatment of patients with a recurrence that augurs a poor prognosis.
Recurrent or Refractory Neuroblastoma in Patients Initially Classified as High Risk
(Risk categories are defined in Table 1 in the Stage Information section of this summary.)
Any recurrence in patients initially classified as high risk signifies a very poor prognosis. Data from three consecutive German high-risk neuroblastoma trials described 253 children relapsing after intensive chemotherapy with autologous stem cell transplantation (SCT) who had a 5-year OS rate of less than 10%. Only 23 of the 253 patients eventually proceeded to a second autologous SCT following retrieval chemotherapy. Among these patients, the 3-year OS rate was 43%, but the 5-year OS rate was less than 20%. This shows that intensive second-line therapy is feasible, although even with intensive therapy and second autologous SCT, only a small minority of relapsed high-risk neuroblastoma patients may benefit.[Level of evidence: 3iiiA] Whether this intense therapeutic approach is better than other salvage therapy approaches is unknown. Topotecan alone and in combination with cyclophosphamide or etoposide has been used in patients with recurrent disease who did not receive topotecan initially.[14,15]; [Level of evidence: 1A] High-dose carboplatin-irinotecan-temozolomide has been used in patients resistant or refractory to regimens containing topotecan. The combination of irinotecan and temozolomide had a 15% response rate in one study.[Level of evidence: 2A]
For children with recurrent or refractory neuroblastoma, 131 I-MIBG is an effective palliative agent and should be considered.; [Level of evidence: 3iiiA]
Additionally, phase I or II clinical trials are appropriate and should be considered.