多数SEGA患儿可用雷帕霉素类似物治疗而不必手术

圣迭戈——辛辛那提大学和加州大学洛杉矶分校的研究人员发现，结节性硬化症(TSC)和室管膜下巨细胞星形细胞瘤(SEGA)儿童患者采用西罗莫司或依维莫司药物治疗比手术治疗更为有效。

主要研究者、辛辛那提大学的Susanne Yoon在美国神经病学会(AAN)2013年会上报告称，虽然对这类良性肿瘤通常采取手术治疗，但近年来已发现雷帕霉素类似物对该类肿瘤同样有效，现在的问题是究竟哪种方法更佳。已知相对于手术治疗而言，药物治疗更为温和但不能根治。

研究者比较了23例接受手术治疗的SEGA患者、81例接受西罗莫司或依维莫司治疗的患者以及9例接受两种方法治疗的患者的结局。手术患者确诊年龄约10岁，中位随访8.9年；药物治疗患者确诊年龄约7岁，中位随访2.8年。两组患者中多数为男孩。

结果显示，接受雷帕霉素类似物治疗的患者无1例需要手术治疗，61%(45例)的患者肿瘤缩小超过一半。部分患者出现药物导致的感染、体重变化或高脂血症，但仅13%(11例)的患者因药物副作用而需要停药或住院治疗。

而手术治愈患者仅为39%(9例)，部分患者接受了2或3次手术治疗。61%(14例)患者因术后并发症延长住院时间或再次住院治疗，包括颅内出血8例、脑积水/分流故障6例以及神经损伤和癫痫。

犹他大学儿科学教授David H. Viskochil博士评论指出：“药物治疗不仅更为有效，而且安全性也更佳。雷帕霉素类似物单独或联合治疗越来越成为神经皮肤肿瘤治疗的基石。”

他也承认，在紧急情况下仍必须采取手术治疗，不可能等待3个月以观察药物是否有效。但如果患者只是刚刚出现某些症状且没有恶化，可采取药物治疗并观察病情发展情况。但对于SEGA早期确诊患者，手术治愈的可能性是否更大？这一问题尚有待研究。

Yoon女士和Viskochil博士均声称无利益冲突。

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By: M. ALEXANDER OTTO, Oncology Practice

SAN DIEGO – Medical management with sirolimus or everolimus for pediatric patients with tuberous sclerosis complex and subependymal giant cell astrocytomas is more effective and safer than surgery, researchers from the University of Cincinnati and University of California, Los Angeles, have found.

Although the benign tumors have traditionally been left to surgeons, it’s become clear in recent years that rapamycin analogs are effective, too. The question has been "which [approach] is best? Medical management "is known to be pretty mild compared to the surgery," but it’s not curative, explained lead investigator Susanne Yoon, the University of Cincinnati medical student who presented the results at the annual meeting of the American Academy of Neurology.

The team compared outcomes for 23 SEGA (subependymal giant cell astrocytoma) patients who underwent surgery, 81 who took sirolimus or everolimus, and 9 who got both. The surgery patients were diagnosed when they were about 10 years old and were followed for a median of 8.9 years; the medical patients were about 7 years old when diagnosed, and were followed for a median of 2.8 years. Boys made up the majority of both groups.

None of the children who took a rapamycin analog needed surgery; tumors shrank by more than half in 61% (45). The drugs caused infections, weight change, or hyperlipidemia in some, but only 13% (11) needed to stop the drug or go to the hospital because of side effects.

Meanwhile, surgery cured just 39% (9) of the children who got it, sometimes after two or three operations; 61% (14) of those patients had prolonged hospitalizations or were hospitalized due to postoperative complications that included intracranial hemorrhage in 8, hydrocephalus/shunt malfunction in 6, neurologic impairment, and seizures.

"Not only does medical management win in efficacy, but it also wins in the safety issues. Rapalog [rapamycin] therapy, alone or in combination, is becoming a cornerstone of tumor management" in neurocutaneous disorders, said Dr. David H. Viskochil, professor of pediatrics at the University of Utah, Salt Lake City, commenting on the study.

"Of course, there are emergent situations where you’ve just got to go in and get the tumor out; you can’t wait 3 months to see" if drugs work. "But if a child is just starting to show some symptoms and not deteriorating, then you can start with medicine first and see what happens," he said.

"The question is if you got [SEGAs] really early, would surgical cure be much more likely? The studies aren’t quite there yet," he said in an interview.

Ms. Yoon and Dr. Viskochil said they have no disclosures.