Packer Roger J, Vezina Gilbert
Division of Neurology, Children's National Medical Center, Washington, DC 20010, USA.
Arch Neurol. 2008 Nov;65(11):1419-24. doi: 10.1001/archneur.65.11.1419.
Medulloblastoma is the most common malignant childhood brain tumor and, although relatively uncommon in older patients, poses a therapeutic challenge in adults. With current means of therapy, children with nondisseminated medulloblastoma have a high likelihood of long-term survival; 80% or more will be alive 5 years after diagnosis and treatment, with many free of the disease. Even in children with disseminated disease, intensified therapy has been associated with improved survival rates, although some of this improvement may be more apparent than real. The quality of life in long-term survivors is a major issue, and most children who survive have substantial neurologic and cognitive sequelae. The outcome in infants and younger children with medulloblastoma is suboptimal, although there is some evidence to suggest that intensification of therapy has improved the likelihood of disease control. A better understanding of the biological characteristics of medulloblastoma including the cell or cells of origin and the aberrant cellular signaling pathways involved has the promise of dramatically changing tumor stratification and treatment in the near future. However, these biological advances have yet to be integrated into the treatment of medulloblastoma in children or adults.
髓母细胞瘤是儿童最常见的恶性脑肿瘤，虽然在老年患者中相对少见，但在成人中却是一个治疗难题。采用目前的治疗方法，非播散性髓母细胞瘤患儿有很高的长期生存可能性；80%或更多的患儿在诊断和治疗后5年仍存活，其中许多已无疾病。即使是患有播散性疾病的儿童，强化治疗也与生存率提高相关，尽管这种提高有些可能只是表面上的而非实际的。长期存活者的生活质量是一个主要问题，大多数存活的儿童有严重的神经和认知后遗症。婴儿和年幼儿童髓母细胞瘤的治疗效果并不理想，尽管有一些证据表明强化治疗提高了疾病控制的可能性。更好地了解髓母细胞瘤的生物学特性，包括起源细胞或细胞以及所涉及的异常细胞信号通路，有望在不久的将来显著改变肿瘤分层和治疗。然而，这些生物学进展尚未整合到儿童或成人髓母细胞瘤的治疗中。
