儿童内脏非横纹肌肉瘤软组织肉瘤的治疗探讨

目的探讨儿童内脏非横纹肌肉瘤软组织肉瘤的诊治要点。方法2004年7月至2010年7月我们收治儿童内脏非横纹肌肉瘤软组织肉瘤10例,其中男性7例,女性3例,年龄最大12岁,最小2岁。结果10例患儿中,手术后病理类型包括滑膜肉瘤1例,婴儿型纤维肉瘤2例,促结缔组织增生性小圆细胞肿瘤（DSRCT）1例,淋巴管平滑肌肉瘤1例,腹膜后平滑肌肉瘤1例,大网膜脂肪肉瘤1例,原始神经外胚叶肿瘤3例。肿瘤直径均〉5cm,以手术、化疗或放疗综合治疗为原则,其中淋巴管平滑肌肉瘤1例,腹膜后平滑肌肉瘤1例,大网膜脂肪肉瘤1例手术完整切除,术后定期随访,未作放疗和化疗。其他7例手术后辅助化疗和放疗。所有病例最长随访时间7年,最短15个月,平均随访时间（41．3±23．19）个月。结论内脏非横纹肌肉瘤软组织肉瘤因部位及肿瘤大小而影响手术完整切除,提高手术切除的技能、寻找更加有效的化疗和放疗是内脏NRSTS今后治疗的方向。     

小儿软组织肉瘤预后因素探讨

本文通过对11例经病理证实的软组织肉瘤患儿进行临床分析。指出手术广泛切除仍是决定预后最重要因素。肿瘤大小、部位及组织学分期与局部复发关系不大,但>10cm及高分期肉瘤病人预后不良。对于高分期病人使用放疗可提高存活率,对于低分期的可以预防局部复发。本文还就临床分期、病理分级及治疗方案选择与预后的关系进行探讨。     

儿童横纹肌肉瘤23例临床分析

目的：探讨儿童横纹肌肉瘤的临床特点、治疗和转归。方法：对1998年1月至2008年10月收治的23例横纹肌肉瘤患儿临床资料进行回顾性分析。结果：23例患儿中,男15例,女8例,平均发病年龄5岁(7个月至12岁)。依据美国横纹肌肉瘤研究组(IRS)的分期标准,I期2例,Ⅱ期4例,Ⅲ期8例,Ⅳ期9例。原发于头颈部14例,四肢4例,膀胱2例,肾脏、腹膜后及胆道各1例。所有患儿均经病理活检及免疫组织化学染色确诊。临床表现无特异性,主要为肿瘤组织占位、压迫、浸润后引起。治疗严格依照患儿IRS分期进行。2002年前化疗方案以VDCA、VAC 和VadrC 为主,2002年后采用美国肿瘤学中心研究组（COG）横纹肌肉瘤化疗方案。其中19例接受手术、化疗和放疗综合治疗的患儿2年生存率为63%,4例接受单纯手术或手术结合单一化疗或放疗的患儿生存期均未超过2年。结论：儿童横纹肌肉瘤临床表现无特异性;联合手术、放疗、化疗是治疗横纹肌肉瘤的有效方法。     

儿童横纹肌肉瘤误诊一例

患儿男,6岁。因右腮部肿痛、右耳流脓、头痛2 0d ,右侧面瘫13d入院。2 0d前发现右腮腺区肿痛,继之右耳道流脓血性分泌物,时诉头痛。13d前又出现右侧面瘫、言语不清、喝水呛咳。体检:神志清,右眼闭合不全,口角左歪,伸舌偏左,右鼻唇沟变浅,鼓腮、吹口哨不能。右耳道有脓血性分泌物,右腮腺区明显肿胀,压痛( ) ,右耳听力减退。鼻通气畅,右颌下触及1 5cm×1 5cm肿大淋巴结,活动,有压痛。颈软,心肺腹部无异常。辅助检查:血、尿、粪常规,肝功能、电解质均正常。心电图、胸部X线透视,右下颌骨X线拍片均未见异常。诊断为腮腺炎、中耳炎、右侧面神经…     

横纹肌肉瘤分子病理学研究进展

横纹肌肉瘤(Rhabdomyosarcoma,RMS)是儿童期最常见的软组织肉瘤。RMS的光镜诊断基于识别横纹肌母细胞,但其表现可呈从原始间充质细胞到胚胎型肌小管的不同分化状态,低分化类型非常多见,约占20％～60％,不仅难以与其它小圆细胞瘤相鉴别,而且也难以进行分类及估计预后。     

儿童横纹肌肉瘤的诊断及治疗

横纹肌肉瘤(RMS)有早期就诊率低、恶性程度高、进展快及手术难以完全切除且易致术后畸形等特点。影像学检查、局部细针穿刺病理诊断是早期诊断RMS的主要方法。化疗、外放疗、放射性粒子植入术、自体外周血造血干细胞移植及手术治疗是目前治疗RMS的主要方法。[临床儿科杂志,2012,30(5):404-407]     

高危头颈部横纹肌肉瘤临床特征及预后分析

目的研究高危头颈部横纹肌肉瘤(RMS)的高危因素与诊疗及预后的相关性。方法根据美国儿童肿瘤组织(COG)、中国抗癌协会小儿血液肿瘤学组儿童横纹肌肉瘤协作组(CCCG-RMS)制定的危险度分组,筛选原发于头颈部的高危儿童横纹肌肉瘤为研究对象。采用穿刺病理/手术+化疗+放疗等综合治疗,分析原发部位、年龄、病理分型、转移等因素与诊疗策略、预后的相关性。结果入组患儿29例。其中男17例(58.6%),女12例(41.4%);原发部位:眼眶区占位7例(24.1%),脑膜旁占位22例(75.9%);临床分期:Ⅲ期2例(6.9%),Ⅳ期27例(93.1%)。随访至2018年12月31日,死亡14例,存活15例,无病生存10例,总生存率为48.3%(15/29例),无病生存率为34.5%(10/29例)。Kaplan-Merier曲线分析提示总生存时间(76.0±12.0)个月,95%可信区间(CI):53.5~93.2个月;无事件生存时间(62.5±10.6)个月,95%CI:47.0~83.9个月。结论高危组头颈部RMS患儿预后相对较差,中枢神经侵犯是重要的致死因素,密切随访及防治中枢神经系统侵犯是提高RMS预后的主要任务。     

横纹肌肉瘤的治疗

目的：讨横纹肌肉瘤的有效治疗措施。方法：顾分析上海儿童医学中心1998年10月～2006年6月诊断治疗的29例横纹肌肉瘤。肿瘤部位包括颈背部5例、胸壁1例、腹壁2例、后腹膜盆腔12例、肢体9例。病理分类胚胎型19例（65．5％），腺泡型8例（27．6％），多型性2例（6．9％）。临床分期Ⅰ期2例，Ⅱ期6例，Ⅲ期9例，Ⅳ期12例。26例（89．4％）肿瘤5～25cm，3例（10、3％）肿瘤2～5cm。结果：29例随访中2例1个疗程后自行终止治疗并失访。19例获得完全缓解（其中4例停药3个月到3年后复发或死亡，1例自行停药3个月复发），4例部分缓解（其中3例发病6个月后复发），4例治疗无反应（其中3例均复发或进展，1例病情反复处于带瘤生存）。14例持续缓解时间为5～77个月，平均22．9个月。停药平均16．9个月。结论：横纹肌肉瘤手术的完整切除、术前、术后的化疗起重要作用。     

20例横纹肌肉瘤患儿的临床特点及预后分析

目的 分析儿童横纹肌肉瘤(rhabdomyosarcoma,RMS)的临床特点及影响预后的因素。方法 纳入2014年2月至2020年9月就诊于中南大学湘雅医院儿科血液肿瘤专科的20例RMS患儿为研究对象,回顾性分析患儿的临床资料和随访资料。结果 20例RMS患儿初次就诊时临床症状依次为无痛性肿块(13/20,65%)、眼球突出(4/20,20%)、腹痛(3/20,15%)。依据国际横纹肌肉瘤研究组(Intergroup Rhabdomyosarcoma Study Group,IRSG)的分期标准,Ⅰ期1例(5%)、Ⅱ期4例(20%)、Ⅲ期9例(45%)、Ⅳ期6例(30%)。20例患儿中位随访时间为19 (3～93)个月,2年总体生存(overall survival,OS)率为79.5%(95%CI:20.1～24.3),2年无事件生存(event-free survival,EFS)率为72.0%(95%CI:19.5～23.9)。病理类型为多形性可降低RMS的2年OS率(P<0.05),有远处转移、IRSG分期为Ⅳ期、危险度为高危组可降低RMS的2年EFS率(P<0....     

儿童胆道横纹肌肉瘤的诊断与治疗

目的分析儿童胆道横纹肌肉瘤(rhabdomyosarcoma of the biliary tree,RMS/BT)的临床特点、治疗策略及预后情况。方法回顾性研究2013年1月至2020年3月首都医科大学附属北京儿童医院诊治的6例RMS/BT患儿临床资料,并对患儿预后情况及相关并发症进行随访。结果 6例RMS/BT患儿中男5例、女1例,中位发病年龄28.5个月(7～50个月),中位随访时间21个月(3～43个月)。原发于肝内胆道2例,原发于肝外胆道3例,原发于肝门部胆总管近端和左右肝管1例。肝内型主要以腹痛或局部包块为原发症状就诊,肝外型和肝门部肿瘤主要以胆道梗阻表现为首发症状就诊。根据横纹肌肉瘤治疗前TNM(tumor-node-metastasis)临床分期标准,T2bN0M0期2例,T2bNxM0期3例,T1bN0M0期1例;根据美国横纹肌肉瘤研究组术后-病理分期系统,5例为Ⅲ期,1例为Ⅰ期。RMS/BT患儿采取以手术、化疗及放疗为主的综合性治疗模式。2例行一期肿瘤切除术,4例行肿瘤活检化疗后再手术。术后6例均行化疗,3例进行了原发部位的放疗,未结束化疗的1例拟接受放射治疗。在治疗过程中1例发生肿瘤破裂、胆瘘、胆汁性腹膜炎。本组6例RMS/BT患儿中,5例存活,2例复发,1例死亡。存活的5例中,2例结束治疗无瘤生存,2例仍在治疗中,1例肿瘤复发继续治疗中。结论RMS/BT对化疗敏感,遵循肿瘤活检、化疗、延迟手术及放疗的诊疗流程,大多能达到肿瘤根治性切除、有效减少局部复发的目的。     

The value of postoperative radiotherapy in childhood nonrhabdomyosarcoma soft tissue sarcoma

OBJECTIVE: To determine the value of postoperative radiotherapy (RT) in the management of nonrhabdomyosarcoma soft tissue sarcoma (NRSTS) of childhood. PROCEDURE: From 1964 to 2000, 62 children with a median age of 14 years were seen at the University of Iowa and underwent a wide local excision for non-metastatic NRSTS. Tumors were high grade in 36 (58%) and >5 cm in 24 (39%). Margins of resection were negative (Group I) in 37 (60%) and positive (Group II) in 25 (40%). Postoperative RT was delivered to 20 patients (32%); eight of 37 (22%) Group I and 12 of 25 (48%) Group II children received postoperative RT. Chemotherapy was employed in 19 patients (31%). Median follow-up was 9.6 years. RESULTS: The 5- and 10-year overall survival rates for Group I were 69 and 63% and for Group II were 66 and 60%. The 5- and 10-year local control rate was 66%. On multivariate analysis, size of tumor (P < 0.001) and postoperative RT (P = 0.017) were prognostic factors for local control. All 13 Group I children with low grade, 相似文献   

Current management of pediatric soft tissue sarcomas

Pediatric soft tissue sarcomas are a group of malignant neoplasms arising within embryonic mesenchymal tissues during the process of differentiation into muscle, fascia and fat. The tumors have a biphasic peak for age of incidence. Rhabdomyosarcoma(RMS) is diagnosed more frequently in younger children, whereas adult-type non-RMS soft tissue sarcoma is predominately observed in adolescents. The latter group comprises a variety of rare tumors for which diagnosis can be difficult and typically requires special studies, including immunohistochemistry and molecular genetic analysis. Current management for the majority of pediatric sarcomas is based on the data from large multi-institutional trials, which has led to great improvements in outcomes over recent decades. Although surgery remains the mainstay of treatment, the curative aim cannot be achieved without adjuvant treatment. Pre-treatment staging and risk classification are of prime importance in selecting an effective treatment protocol. Tumor resectability, the response to induction chemotherapy, and radiation generally determine the risk-group, and these factors are functions of tumor site, size and biology. Surgery provides the best choice of local control of small resectable tumors in a favorable site. Radiation therapy is added when surgery leaves residual disease or there is evidence of regional spread. Chemotherapy aims to reduce the risk of relapse and improve overall survival. In addition, upfront chemotherapy reduces the aggressiveness of the required surgery and helps preserve organ function in a number of cases. Long-term survival in low-risk sarcomas is feasible, and the intensity of treatment can be reduced. In high-risk sarcoma, current research is allowing more effective disease control.     

The challenge of the management of adolescents and young adults with soft tissue sarcomas

Soft tissue sarcomas are relatively frequent in adolescents and young adults and their clinical management may be complex, partly due to tumor associated factors, but also because different approaches have been adopted by pediatric and adult medical oncologists dealing with the same disease. However, times are changing and in the last few years, management has tended to converge towards a common strategy. Continued and increased international collaboration between pediatric and adult sarcoma groups is of critical importance to improve the quality of treatment as well as research programs dedicated to young patients with soft tissue sarcomas.     

Adjuvant chemotherapy for the treatment of advanced pediatric nonrhabdomyosarcoma soft tissue sarcoma: the National Cancer Institute experience

BACKGROUND: The survival of children and adolescents with advanced (unresectable or metastatic) nonrhabdomyosarcoma soft tissue sarcoma (NRSTS) is poor. In order to clarify the role of combining chemotherapy with aggressive local control using surgery and/or radiation, we reviewed our institutional experience with the treatment of advanced pediatric NRSTS. PROCEDURE: We reviewed the charts of all patients less than 21 years treated for an advanced NRSTS at the National Cancer Institute (NCI) between 1983 and 2003. Tumor pathology was confirmed and demographic, disease, and treatment data were abstracted. Survival was calculated using standard methods. RESULTS: Of the 25 patients who were treated over the study period, 15 had metastatic disease and 10 had unresectable or incompletely resected disease at presentation. Twenty-one patients received chemotherapy consisting of the combination of vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide, and the remaining 4 received regimens that included doxorubicin. Twenty patients (80%) had a complete (5/25) or partial (15/25) response after chemotherapy alone. After the combination of chemotherapy and local control, 14 patients (56%) had a complete response (CR). The estimated 5-year overall and event-free survival (EFS) for all patients was 0.50 (standard error = 0.11) and 0.34 (standard error = 0.10), respectively. CONCLUSIONS: The combination of chemotherapy with aggressive local control in this cohort of pediatric patients with advanced NRSTS yielded results comparable to those observed in patients with advanced sarcomas that are chemotherapy responsive. Prospective randomized trials are needed to quantify the contribution of chemotherapy and to determine the ideal regimen.     

Interstitial brachytherapy for childhood soft tissue sarcoma

BACKGROUND: To evaluate the efficacy of interstitial brachytherapy (BRT) in children undergoing combined modality treatment for soft tissue sarcomas (STS). PROCEDURE: From September 1984 to December 2003, 50 children (median age 13 years, range 1 to 18) with STS who received BRT as part of loco-regional treatment were included. There were 30 males and 20 females, the majority (68%) had primary lesions, synovial sarcoma (32%) was the most common histological type, and 26% had high-grade lesions. Treatment included wide local excision and BRT with or without external beam radiotherapy (EBRT). Thirty children (60%) received BRT alone. RESULTS: After a median follow-up of 51 months, the local control (LC), disease-free survival, and overall survival were 82%, 68%, and 71%, respectively. LC was superior in patients with tumor size 5 cm (96% vs. 67%, P = 0.04), symptom duration <2 months versus >2 months (100% vs. 73%, P = 0.05), and Grade I versus Grade II versus Grade III tumors (100% vs. 93% vs. 57%, P = 0.03). Children receiving a combination of BRT and EBRT had comparable LC to those receiving BRT alone (78% vs. 84%, P = 0.89). There was no significant difference in LC for patients receiving LDR versus HDR BRT (77% vs. 92%, P = 0.32, for BRT alone; and 67% vs. 100%, P = 0.17, for BRT + EBRT). CONCLUSION: Interstitial BRT with or without EBRT appears to result in satisfactory outcome in children with STS. Radical BRT alone, when used judiciously in select groups of children, results in excellent local control and functional outcome with reduced treatment-related morbidity.     

Children's Oncology Group's 2013 blueprint for research: Soft tissue sarcomas

In the US, approximately 850–900 children are diagnosed each year with soft tissue sarcomas (STS). Key findings from recent Children's Oncology Group (COG) clinical trials include safe reduction in therapy for low risk rhabdomyosarcoma (RMS), validation of FOXO1 fusion as a prognostic factor, a modest improvement in outcome for high‐risk RMS, and a biologically designed non‐cytotoxic therapy for pediatric desmoid tumor. Planned Phase 2 trials include targeted agents for VEGF/PDGF, mTOR, and IGF‐1R for children with RMS and VEGF for children with non‐RMS STS (NRSTS). For RMS, COG Phase 3 trials potentially will explore VEGF/mTOR inhibition or chemotherapy interval compression. For NRSTS, a COG Phase 3 trial will explore VEGF inhibition. Pediatr Blood Cancer 2013; 60: 1001–1008. © 2012 Wiley Periodicals, Inc.     

Sentinel node biopsy in pediatric soft tissue sarcomas of extremities

Background

Sentinel Node Biopsy is an established staging technique in many adult malignancies. However, only few reports describe this procedure for the evaluation of regional lymph nodes in childhood and adolescents. Our experience with sentinel node biopsy in soft tissue sarcomas of extremities in children is reported.

Methods

Seventeen children were evaluated with sentinel node biopsy between 2002 and 2007: 11 at initial surgery, 5 at primary re‐excision, 1 at local relapse. The diagnosis was rhabdomyosarcoma in 5 and other soft tissue sarcomas in 12: Ewing/PNET sarcoma 6, epithelioid sarcoma 1, malignant peripheral‐nerve‐sheath tumor 1, undifferentiated sarcoma 1, myxoid liposarcoma 2, adult‐type fibrosarcoma 1. Primary sites included lower limbs (8), upper limbs (9). Mapping of nodes was performed with lymphoscintigraphy plus intra‐operative injections with blue‐dye in 14 cases, with lymphoscintigraphy and intra‐operative injections alone in 2 and 1, respectively.

Results

Of the 17 lymphatic regions (9 axilla, 8 inguinal), 16 were identified with lymphoscintigraphy, 15 by intra‐operative injections. Thirty‐five lymph nodes were removed. Nodes were positive for metastasis in two patients with alveolar rhabdomyosarcoma and undifferentiated sarcoma. There were no complications. No further lymph node metastases were recognized either at diagnosis or during the follow‐up (6–78 months).

Conclusions

Sentinel node biopsy was technically feasible, reliable, and free of complications. It could be an alternative to aggressive or random biopsies for extremity rhabdomyosarcoma and it can contribute to define those non‐rhabdomyosarcoma soft tissue sarcomas that spread to regional nodes. Pediatr Blood Cancer 2009;52:51–54. © 2008 Wiley‐Liss, Inc.