双侧肾母细胞瘤患儿的首个前瞻性多机构治疗研究（AREN0534）结果：来自儿童肿瘤协作组的报告
Results of the First Prospective Multi-institutional Treatment Study in Children With Bilateral Wilms Tumor (AREN0534): A Report From the Children's Oncology Group.
作者信息
Ehrlich Peter, Chi Yuen Y, Chintagumpala Murali M, Hoffer Fred A, Perlman Elizabeth J, Kalapurakal John A, Warwick Ann, Shamberger Robert C, Khanna Geetika, Hamilton Tom E, Gow Ken W, Paulino Arnold C, Gratias Eric J, Mullen Elizabeth A, Geller James I, Grundy Paul E, Fernandez Conrad V, Ritchey Michael L, Dome James S
机构信息
*Section of Pediatric Surgery CS Mott Children's Hospital, University of Michigan, Ann Arbor MI †COG Data Center, University of Florida, Gainesville, FL ‡Texas Children's Cancer Center at Baylor College of Medicine, Houston, TX §Fred Hutchison Cancer Center, University of Washington, Seattle, WA ¶Ann and Robert H Lurie Children's Hospital, Chicago, IL ||Northwestern University, Chicago, IL **Walter Reed National Military Medical Center, Washington DC ††Boston Children's Hospital and Dana Farber Cancer Center, Boston, MA ‡‡Washington University of St Louis, St Louis, MO §§University of Washington, Seattle, WA ¶¶MD Anderson Cancer Center, Houston, TX ||||Children's Oncology Group, Philadelphia, PA ***Cincinnati Children's Hospital, Cincinnati, OH †††University of Alberta Children's Hospital, Edmonton, Alberta, Canada ‡‡‡IWK Children's Hospital, Halifax, Nova Scotia, Canada §§§Phoenix Children's Hospital, Phoenix, AZ ¶¶¶Children National Medical Center, Washington, DC.
出版信息
Ann Surg. 2017 Sep;266(3):470-478. doi: 10.1097/SLA.0000000000002356.
OBJECTIVE
The Children's Oncology Group study AREN0534 aimed to improve event-free survival (EFS) and overall survival (OS) while preserving renal tissue by intensifying preoperative chemotherapy, completing definitive surgery by 12 weeks from diagnosis, and modifying postoperative chemotherapy based on histologic response.
BACKGROUND
No prospective therapeutic clinic trials in children with bilateral Wilms tumors (BWT) exist. Historical outcomes for this group were poor and often involved prolonged chemotherapy; on NWTS-5, 4-year EFS for all children with BWT was 56%.
METHODS
Patients were enrolled and imaging studies were centrally reviewed to assess for bilateral renal lesions. They were treated with 3-drug induction chemotherapy (vincristine, dactinomycin, and doxorubicin) for 6 or 12 weeks based on radiographic response followed by surgery and further chemotherapy determined by histology. Radiation therapy was provided for postchemotherapy stage III and IV disease.
RESULTS
One hundred eighty-nine of 208 patients were evaluable. Four-year EFS and OS were 82.1% (95% CI: 73.5%-90.8%) and 94.9% (95% CI: 90.1%-99.7%. Twenty-three patients relapsed and 7 had disease progression. After induction chemotherapy 163 of 189 (84.0%) underwent definitive surgical treatment in at least 1 kidney by 12 weeks and 39% retained parts of both kidneys. Surgical approaches included: unilateral total nephrectomy with contralateral partial nephrectomy (48%), bilateral partial nephrectomy (35%), unilateral total nephrectomy (10.5%), unilateral partial nephrectomy (4%), and bilateral total nephrectomies (2.5%).
CONCLUSION
This treatment approach including standardized 3-drug preoperative chemotherapy, surgical resection within 12 weeks of diagnosis and response and histology-based postoperative therapy improved EFS and OS and preservation of renal parenchyma compared with historical outcomes for children with BWT.
目的
儿童肿瘤研究组的AREN0534研究旨在通过强化术前化疗、在诊断后12周内完成根治性手术以及根据组织学反应调整术后化疗，在保留肾组织的同时提高无事件生存率（EFS）和总生存率（OS）。
背景
目前尚无针对双侧肾母细胞瘤（BWT）患儿的前瞻性治疗临床试验。该组患儿的历史预后较差，且常需接受长时间化疗；在国家肾母细胞瘤研究组（NWTS）-5研究中，所有BWT患儿的4年EFS为56%。
方法
招募患者并对影像学检查进行集中审查以评估双侧肾脏病变。根据影像学反应，患者接受为期6周或12周的三联诱导化疗（长春新碱、放线菌素D和阿霉素），随后进行手术，并根据组织学结果确定进一步化疗方案。对化疗后处于Ⅲ期和Ⅳ期的疾病进行放射治疗。
结果
208例患者中有189例可评估。4年EFS和OS分别为82.1%（95%CI：73.5%-90.8%）和94.9%（95%CI：90.1%-99.7%）。23例患者复发，7例疾病进展。诱导化疗后，189例患者中有163例（84.0%）在12周内至少对1个肾脏进行了根治性手术，39%的患者保留了双侧肾脏的部分组织。手术方式包括：单侧肾全切加对侧部分肾切除（48%）、双侧部分肾切除（35%）、单侧肾全切（10.5%）、单侧部分肾切除（4%）以及双侧肾全切（2.5%）。
结论
与BWT患儿的历史预后相比，这种治疗方法包括标准化的三联术前化疗、诊断后12周内进行手术切除以及根据反应和组织学进行术后治疗，提高了EFS和OS，并保留了肾实质。
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