Hendricks Marc, Cois Annibale, Geel Jennifer, du Plessis Johan, Bassingthwaighte Mairi, Naidu Gita, Rowe Biance, Büchner Ané, Omar Fareed, Thomas Karla, Uys Ronelle, van Zyl Anel, van Heerden Jaques, Mahlachana Ngoakoana, Vermeulen Johani, Davidson Alan, Frazier A Lindsay, Donald Kirsty, Kruger Mariana
Haematology Oncology Service, Department of Paediatrics and Child Health, Red Cross War Memorial Children's Hospital, University of Cape Town, Cape Town, South Africa.
Division of Health Systems and Public Health, Department of Global Health, Stellenbosch University, Cape Town, South Africa.
Pediatr Blood Cancer. 2022 May;69(5):e29543. doi: 10.1002/pbc.29543. Epub 2021 Dec 31.
To determine the overall survival (OS) and prognostic factors influencing outcomes in children and adolescents with malignant extracranial germ cell tumours (MEGCTs) in preparation for the development of a harmonised national treatment protocol.
A retrospective folder review was undertaken at nine South African paediatric oncology units to document patient profiles, tumour and treatment-related data and outcomes for all children with biopsy-proven MEGCTs from birth up to and including 16 years of age.
Between 1 January 2000 and 31 December 2015, 218 patients were diagnosed with MEGCTs. Female sex (hazard ratio [HR] 0.284, p = .037) and higher socio-economic status (SES) (HR 0.071, p = .039) were associated with a significantly lower risk of death. Advanced clinical stage at diagnosis significantly affected 5-year OS: stage I: 96%; stage II: 94.3%; stage III: 75.5% (p = .017) and stage IV (60.1%; p < .001). There was a significant association between earlier stage at presentation and higher SES (p = .03). Patients with a serum alpha-fetoprotein (AFP) level of more than 33,000 ng/ml at diagnosis had significantly poorer outcomes (p = .002). The use of chemotherapy significantly improved survival, irrespective of the regimen used (p < .001).
The cohort demonstrated a 5-year OS of 80.3% with an event-free survival (EFS) of 75.3%. Stage, the use of chemotherapy and an elevated serum AFP level of more than 33,000 ng/ml were independently predictive of outcome. The relationship between SES and outcome is important as the implementation of the new national protocol hopes to standardise care across the socio-economic divide.
为制定统一的国家治疗方案做准备，确定影响儿童和青少年恶性颅外生殖细胞肿瘤（MEGCTs）预后的总生存期（OS）及预后因素。
对南非9个儿科肿瘤治疗单位进行回顾性病历审查，记录所有经活检证实为MEGCTs的儿童（从出生至16岁）的患者资料、肿瘤及治疗相关数据和预后情况。
2000年1月1日至2015年12月31日期间，218例患者被诊断为MEGCTs。女性（风险比[HR] 0.284，p = 0.037）和较高的社会经济地位（SES）（HR 0.071，p = 0.039）与显著较低的死亡风险相关。诊断时的临床分期较晚显著影响5年总生存期：I期：96%；II期：94.3%；III期：75.5%（p = 0.017），IV期（60.1%；p < 0.001）。就诊时分期较早与较高的SES之间存在显著关联（p = 0.03）。诊断时血清甲胎蛋白（AFP）水平超过33,000 ng/ml的患者预后明显较差（p = 0.002）。无论使用何种化疗方案，化疗的使用均显著提高了生存率（p < 0.001）。
该队列的5年总生存期为80.3%，无事件生存期（EFS）为75.3%。分期、化疗的使用以及血清AFP水平超过33,000 ng/ml可独立预测预后。SES与预后之间的关系很重要，因为新国家方案的实施希望跨越社会经济差距实现护理标准化。
