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在一名全身状况极差的婴儿髓母细胞瘤患者中成功使用剂量密集新辅助化疗和丙戊酸钠,且毒性极小。

Successful Use of Dose Dense Neoadjuvant Chemotherapy and Sodium Valproate with Minimal Toxicity in an Infant with Medulloblastoma in Extremely Poor General Condition.

作者信息

Gupta Ajay, Kumar Amit, Abrari Andaleeb, Patir Rana, Vaishya Sandeep

机构信息

Asian Hospital, Faridabad, India; Medical Oncology, Max Hospital, Saket, New Delhi, India.

Department of Radiology, Max Hospital, Saket, New Delhi, India.

出版信息

World Neurosurg. 2016 Sep;93:485.e1-5. doi: 10.1016/j.wneu.2016.07.044. Epub 2016 Jul 20.

Abstract

BACKGROUND

Medulloblastoma is the most common malignant brain tumor in children. Infants are in the high-risk category. Complete surgical resection is the single most important determinant of prognosis and survival in nonmetastatic disease. Infants with large primaries after incomplete resection/biopsy and poor general condition have bad prognosis. They are considered poor candidates for intensive chemotherapy involving high dose methotrexate/autologous stem cell transplantation as they are often unable to tolerate these aggressive regimens.

CASE DESCRIPTION

The patient, withinfantile medulloblastoma, was supposed to have complete resection but only a biopsy could be attempted because of increased tumor vascularity. He was in very poor general condition after surgery and his parents declined aggressive chemotherapy and shunt surgery. He was given dose dense neo-adjuvant chemotherapy along with the histone deactylase inhibitor valproate for 5 cycles, with minimal toxicity, after which the tumor was resected. The examination of the resected specimen revealed a complete pathologic response. He then received a total of 18 cycles of chemotherapy and valproate to complete 1 year of systemic treatment. The child is now 6.5 years of age, disease-free, without evidence of any neurocognitive or developmental abnormalities.

CONCLUSIONS

We suggest that the role of neoadjuvant chemotherapy should be explored in patients with infantile medulloblastoma in whom upfront complete resection is not possible, considering the gratifying results obtained in our case.

摘要

背景

髓母细胞瘤是儿童最常见的恶性脑肿瘤。婴儿属于高危类别。完整的手术切除是影响非转移性疾病预后和生存的最重要单一决定因素。不完全切除/活检后有大的原发性肿瘤且一般状况较差的婴儿预后不良。由于他们通常无法耐受这些激进的治疗方案,所以被认为不适合接受包含高剂量甲氨蝶呤/自体干细胞移植的强化化疗。

病例描述

该患有婴儿型髓母细胞瘤的患者本应进行完整切除,但由于肿瘤血管增多,仅尝试了活检。术后他的一般状况非常差,其父母拒绝了激进的化疗和分流手术。在给予剂量密集的新辅助化疗以及组蛋白去乙酰化酶抑制剂丙戊酸进行了5个周期的治疗,毒性极小,之后切除了肿瘤。对切除标本的检查显示有完全的病理反应。然后他总共接受了18个周期的化疗和丙戊酸治疗以完成1年的全身治疗。该患儿现在6.5岁,无病生存,没有任何神经认知或发育异常的迹象。

结论

考虑到我们病例中获得的令人满意的结果,我们建议对于无法进行 upfront 完整切除的婴儿型髓母细胞瘤患者,应探索新辅助化疗的作用。 (注:upfront 直译为“预先”,这里可理解为一开始就进行完整切除)

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