Childhood Central Nervous System Embryonal Tumors Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Recurrent Childhood CNS Embryonal Tumors
Recurrence of all forms of central nervous system embryonal tumors is not uncommon, usually occurring within 18 months of treatment; however, recurrent tumors may develop many years after initial treatment.[1,2] Disease may recur at the primary site or may be disseminated at the time of relapse. Sites of noncontiguous relapse may include the spinal leptomeninges, intracranial sites, and cerebrospinal fluid, in isolation or in any combination, and is variably associated with primary tumor relapse.[1,2,3] One series has found that, independent of the dose of radiation therapy employed or the type of chemotherapy utilized, approximately one-third of patients will relapse at the primary tumor site alone, one-third will relapse at the primary tumor site plus distant sites, and one-third will relapse at distant sites without relapse at the primary site.[1,2,3] At the time of relapse, a complete evaluation for extent of recurrence is indicated for all embryonal tumors. Biopsy or surgical resection may be necessary for confirmation of relapse because other entities such as secondary tumors and treatment-related brain necrosis may be clinically indistinguishable from tumor recurrence. The need for surgical intervention must be individualized on the basis of the initial tumor type, the length of time between initial treatment and the reappearance of the lesion, and clinical symptomatology. Extraneural disease relapse may occur but is rare and is seen primarily in patients treated with radiation therapy alone.[Level of evidence: 3iiiA]
Patients with recurrent embryonal tumors who have already received radiation therapy and chemotherapy may be candidates for salvage chemotherapy and/or stereotactic radiation therapy. These tumors can be responsive to chemotherapeutic agents used singularly or in combination, including cyclophosphamide, cisplatin, carboplatin, lomustine, etoposide, and topotecan.[6,7,8,9,10,11,12,13,14]; [Level of evidence: 2A] Approximately 30% to 50% of these patients will have objective responses to conventional chemotherapy, but long-term disease control is rare. For select patients with recurrent medulloblastoma, primarily infants and young children who were treated at the time of diagnosis with chemotherapy alone and developed local recurrence, long-term disease control may be obtained after further treatment with chemotherapy plus local radiation therapy; this potential may be greatest in patients who are able to undergo complete resection of the recurrent disease.[Level of evidence: 2A]; [Level of evidence: 3iiiA]
Treatment Options for Stages I and II
Complete surgical resection of primary tumor followed by chemotherapy.In a randomized trial, seven of eight patients with stage I hepatocellular carcinoma survived disease free after adjuvant cisplatin-based chemotherapy. In a survey of childhood liver tumors treated prior to the consistent use of chemotherapy, only 12 of 33 patients with hepatocellular carcinoma who had complete excision of the tumor survived. This suggests that adjuvant chemotherapy...
For patients who have previously received radiation therapy, higher-dose chemotherapeutic regimens, supported with autologous bone marrow rescue or peripheral stem cell support, have been used with variable results.[18,19,20,21,22,23][Level of evidence: 2A]; [Level of evidence: 3iiB]; [25,26][Level of evidence: 3iiiA] With such regimens, objective response is frequent, occurring in 50% to 75% of patients; however, long-term disease control is obtained in fewer than 30% of patients and is primarily seen in patients in first relapse and in those with only localized disease at the time of relapse.; [Level of evidence: 2A]; [Level of evidence: 3iiB] Additionally, results from national trials for relapsed medulloblastoma that specified intent to transplant as part of their treatment plan showed that only approximately 5% of patients initiating retrieval therapy achieve long-term disease-free survival with this strategy.[23,27] Thus, studies that report from the time of transplant overestimate the benefit of transplant-based approaches for the total population of relapsing patients. Long-term disease control for patients with disseminated disease is infrequent.[Level of evidence: 3iA]