CN-03LONG-TERM FOLLOW-UP OF ENDOCRINE FUNCTION AMONG YOUNG CHILDREN WITH NEWLY-DIAGNOSED MALIGNANT CENTRAL NERVOUS SYSTEM (CNS) TUMORS TREATED WITH IRRADIATION-AVOIDING REGIMENS: THE CHILDRENS HOSPITAL LOS ANGELES (CHLA) EXPERIENCE

摘要

BACKGROUND: The adverse effects of radiation therapy on endocrine function among pediatric brain tumor patients have been well-documented. Brief (less than 5 months) intensive induction chemotherapy followed by marrow-ablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) and avoidance of CNS irradiation has demonstrated efficacy in the treatment of very young children (less than 6 years of age) with malignant CNS tumors. This study assessed the long-term growth and endocrine effects of young children following chemotherapy-only treatment regimens. METHODS: A retrospective chart review was performed on 99 patients less than 6 years of age with newly-diagnosed malignant brain tumors between May 1991 and October 2010 treated with irradiation-avoiding strategies at CHLA. Thirty patients survived relapse-free post-AuHCR without cranial irradiation for a mean of 8.1 years (range 3.0–22.25 years); one patient expired of a TP53-related adrenocortical carcinoma. The patient cohort consisted of 18 males and 12 females with a mean age at AuHCR of 2.5 years (range: 0.8 to 5.1 years). Tumor pathologies included 20 medulloblastomas, 6 choroid plexus carcinomas, 3 CNS PNET and 1 anaplastic ependymoma. RESULTS: All patients had documented normal age-related free T4, TSH, IGFBP3, prolactin, testosterone and estradiol levels. IGF1 age-related levels were abnormal in one child. FSH and LH levels among females, and cortisol levels, were normal in 93%, 97% and 97%, respectively. Growth charts demonstrated age-associated growth within 2 standard deviations of the mean in 67% patients. Of 10 patients (33%) with short stature, 6 had proportional diminutions in both height and weight. CONCLUSIONS: These findings demonstrate that the use of relatively brief, intensive chemotherapy regimens including marrow-ablative chemotherapy with AuHCR, results in less endocrine and growth abnormalities than treatment schemes utilizing CNS irradiation. However, the documented adverse impact upon physical growth in a proportion of survivors needs to be evaluated further.