Department of Radiation Oncology, Brigham and Women's Hospital, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts.
Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts.
Pediatr Blood Cancer. 2020 Oct;67(10):e28559. doi: 10.1002/pbc.28559. Epub 2020 Jul 19.
Desmoplastic small round cell tumor (DSRCT) is a rare aggressive sarcoma that affects children and young adults, and portends poor outcomes despite intensive multimodal treatment approaches. We report toxicity, response, and outcomes of patients with DSRCT treated with the addition of vincristine, irinotecan, and temozolomide (VIT) to interval-compressed chemotherapy as per Children's Oncology Group ARST08P1.
All newly diagnosed pediatric patients with DSRCT treated at Dana-Farber Cancer Institute and Boston Children's Hospital between 2014 and 2019 as per ARST08P1, Arm P2 with replacement of VAC cycles with VIT, were identified. Medical records were reviewed for clinical and disease characteristics, and treatment response and outcomes.
Six patients were treated as per the above regimen. Median age at diagnosis was 15.1 years (range 3.2-16.4) and five patients were male. Five patients had abdominal primary tumors, of which one had exclusively intraabdominal and four had extraabdominal metastases. Two initial cycles of VIT were well tolerated with nausea, vomiting, diarrhea, and constipation as the most common adverse events. Overall response rate defined as partial or complete response after two initial cycles of VIT was 50%. For local control, all patients had surgical resection followed by radiotherapy, and two patients received hyperthermic intraperitoneal chemotherapy at the time of surgery. Of the four patients who have completed therapy to date, three remain disease-free with median follow-up time of 46.7 months.
The addition of VIT to interval-compressed chemotherapy is tolerable and active in DSRCT, with activity warranting additional investigation.
促结缔组织增生性小圆细胞肿瘤（DSRCT）是一种罕见的侵袭性肉瘤，影响儿童和青少年，尽管采用强化多模式治疗方法，预后仍不佳。我们报告了根据儿童肿瘤学组 ARST08P1 方案，在间隔压缩化疗中添加长春新碱、伊立替康和替莫唑胺（VIT）治疗 DSRCT 患者的毒性、反应和结果。
在 2014 年至 2019 年期间，根据 ARST08P1 方案，在达纳-法伯癌症研究所和波士顿儿童医院接受治疗的所有新诊断的 DSRCT 儿科患者，以及 ARST08P1 方案，臂 P2 用 VIT 替代 VAC 周期的患者，均被确定为符合条件的患者。对病历进行了回顾，以评估临床和疾病特征、治疗反应和结果。
按照上述方案治疗了 6 例患者。诊断时的中位年龄为 15.1 岁（范围 3.2-16.4 岁），男性 5 例。5 例患者有腹部原发性肿瘤，其中 1 例仅为腹腔内，4 例为腹腔外转移。2 个初始周期的 VIT 耐受性良好，最常见的不良反应为恶心、呕吐、腹泻和便秘。2 个初始周期 VIT 后定义为部分或完全缓解的总缓解率为 50%。为了局部控制，所有患者均接受了手术切除，随后进行了放疗，其中 2 例患者在手术时接受了腹腔内热化疗。截至目前，4 例患者已完成治疗，3 例患者无疾病，中位随访时间为 46.7 个月。
在间隔压缩化疗中添加 VIT 是可以耐受的，并且在 DSRCT 中具有活性，其活性值得进一步研究。
