Malignant Melanoma in Children and Adolescents Treated in Pediatric Oncology Centers: An Australian and New Zealand Children's Oncology Group (ANZCHOG) Study.

OBJECTIVES: Unlike adults, malignant melanoma in children and adolescents is rare. In adult melanoma, significant progress in understanding tumor biology and new treatments, including targeted therapies and immunotherapy have markedly improved overall survival. In sharp contrast, there is a paucity of data on the biology and clinical behavior of pediatric melanoma. We report a national case series of all pediatric and adolescent malignant melanoma presenting to ANZCHOG Childhood Cancer Centers in Australia and New Zealand. METHODS: A retrospective, descriptive, multi-center study was undertaken to identify patients less than 18 years of age treated for cutaneous malignant melanoma over a twenty-year period (1994 to 2014). Data on clinical characteristics, histopathology, and extent of disease, treatment and follow-up are described. RESULTS: A total of 37 cases of malignant melanoma were identified from all of the Australasian tertiary Childhood Cancer Centers. The median age was 10 years (range 1 month - 17 years). Clinically, the most common type of lesion was pigmented, occurring in sixteen (57%) patients, whilst amelanotic was seen in 7 patients (25%). In 11 (27.9%) the Breslow thickness was greater than 4mm. A total of 11 (29.7%) patients relapsed and 90% of these died of disease. Five-year event free survival (EFS) and overall survival were 63.2 (95% CI: 40.6 - 79.1) and 67.7% (95% CI: 45.1 - 82.6) respectively. CONCLUSION: Our data confirms that melanoma is a rare presentation of cancer to tertiary Australasian Childhood Cancer Centers with only 37 cases identified over two decades. Notably, melanoma managed in Childhood Cancer Centers is frequently at an advanced stage, with a high percentage of patients relapsing and the majority of these patients who relapsed died of disease. This study confirms previous clinical and prognostic information to support the early multidisciplinary management in Childhood Cancer Centers, in conjunction with expert adult melanoma centers, of this rare and challenging patient group.

Stage at diagnosis for childhood solid cancers in Australia: A population-based study.

BACKGROUND: Stage of cancer at diagnosis is one of the strongest predictors of survival and is essential for population cancer surveillance, comparison of cancer outcomes and to guide national cancer control strategies. Our aim was to describe, for the first time, the distribution of cases by stage at diagnosis and differences in stage-specific survival on a population basis for a range of childhood solid cancers in Australia. METHODS: The study cohort was drawn from the population-based Australian Childhood Cancer Registry and comprised children (<15 years) diagnosed with one of 12 solid malignancies between 2006 and 2014. Stage at diagnosis was assigned according to the Toronto Paediatric Cancer Stage Guidelines. Observed (all cause) survival was calculated using the Kaplan-Meier method, with follow-up on mortality available to 31 December 2015. RESULTS: Almost three-quarters (1256 of 1760 cases, 71%) of children in the study had localised or regional disease at diagnosis, varying from 43% for neuroblastoma to 99% for retinoblastoma. Differences in 5-year observed survival by stage were greatest for osteosarcoma (localised 85% (95% CI = 72%-93%) versus metastatic 37% (15%-59%)), neuroblastoma (localised 98% (91%-99%) versus metastatic 60% (52%-67%)), rhabdomyosarcoma (localised 85% (71%-93%) versus metastatic 53% (34%-69%)), and medulloblastoma (localised 69% (61%-75%) versus metastases to spine 42% (27%-57%)). CONCLUSION: The stage-specific information presented here provides a basis for comparison with other international population cancer registries. Understanding variations in survival by stage at diagnosis will help with the targeted formation of initiatives to improve outcomes for children with cancer.

ANZCCSG BabyBrain99; intensified systemic chemotherapy, second look surgery and involved field radiation in young children with central nervous system malignancy.

BACKGROUND: ANZCCSG BabyBrain99 is a trial of intensive systemic chemotherapy with dual stem cell supported treatment, second look surgery and involved field radiation for children less than four years of age with malignant central nervous system tumours. PROCEDURE: Following primary resection, treatment included two courses of cisplatin and oral etoposide, a third course of mobilising chemotherapy (vincristine, etoposide, cyclophosphamide) with stem cell harvest, followed by intensive stem cell supported chemotherapy with high dose cyclophosphamide, etoposide and vincristine. Children were evaluated for second resection before proceeding to a second stem cell supported consolidation therapy consisting of melphalan and carboplatin. Patients then received involved field radiation therapy. RESULTS: Thirty three children with a range of diagnoses were enrolled. Nine percent of children had metastatic disease at diagnosis. Eighteen children completed treatment including irradiation. At the end of induction the event free survival was 70% (54-86). Forty eight percent of children had a complete response, 18% had stable disease and 3% had a partial response. Five year overall survival was 40% (22-56) and event free survival was 33% (17-50). Children in whom a complete resection were achieved had a significantly superior outcome compared to those children without a complete resection, 5 year EFS 60% (45-75), as compared to 22% (13-30), P-value <0.05. CONCLUSIONS: BabyBrain99 confirms that intensive stem cell supported chemotherapy can be safely administered to infants with CNS tumours however overall prognosis remains poor. Importantly, the study reinforces a complete surgical resection as an important favourable prognostic indicator. Pediatr Blood Cancer 2011;56:1055-1061. © 2011 Wiley-Liss, Inc.