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婴儿肌纤维瘤病伴皮肤、内脏和中枢神经系统受累:治疗的多模态方法。

Infantile Myofibromatosis With Cutaneous, Visceral, and CNS Involvement: A Multimodal Approach to Therapy.

机构信息

Departments of Pediatrics.

Neurosurgery.

出版信息

J Pediatr Hematol Oncol. 2023 Oct 1;45(7):e921-e923. doi: 10.1097/MPH.0000000000002717. Epub 2023 Jul 13.

Abstract

BACKGROUND

Infantile myofibromatosis (IM) is a rare benign tumor of infancy. Cases with solitary and multicentric disease usually spontaneously regress, but multicentric disease with visceral involvement carries a poor prognosis. Few cases of multicentric disease with central nervous system (CNS) involvement have been reported, and none report survival.

OBSERVATIONS

We present a newborn with multicentric IM with cutaneous, visceral, and CNS involvement. She was treated with vinblastine, methotrexate, and the novel addition of intrathecal methotrexate with treatment response after 1 year of therapy.

CONCLUSIONS

Multicentric IM with CNS involvement can be successfully treated with a multimodal approach of chemotherapy with the addition of intrathecal methotrexate and surgery.

摘要

背景

婴儿肌纤维瘤病(IM)是一种罕见的婴儿良性肿瘤。单发和多中心疾病的病例通常会自发消退,但伴有内脏受累的多中心疾病预后不良。有少数多中心疾病伴有中枢神经系统(CNS)受累的病例报告,且均无生存报告。

观察结果

我们报告了一例新生儿患有多中心 IM,伴皮肤、内脏和 CNS 受累。她接受了长春碱、甲氨蝶呤治疗,并新加入了鞘内注射甲氨蝶呤,在 1 年的治疗后有治疗反应。

结论

多中心伴有 CNS 受累的 IM 可以通过化疗联合鞘内注射甲氨蝶呤和手术的多模式方法成功治疗。

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