Surgical management of extremity rhabdomyosarcoma: A consensus opinion from the Children's Oncology Group,the European Pediatric Soft-Tissue Sarcoma Study Group,and the Cooperative Weichteilsarkom Studiengruppe

The treatment of extremity rhabdomyosarcoma remains a challenge due to several adverse prognostic factors frequently associated with this tumor site. The International Soft-Tissue Sarcoma Database Consortium (INSTRuCT) is a collaboration of the Children's Oncology Group Soft-Tissue Sarcoma Committee, the European Pediatric Soft-Tissue Sarcoma Study Group, and the Cooperative Weichteilsarkom Studiengruppe. The INSTRuCT surgical committee developed an internationally applicable consensus opinion document for the surgical treatment of extremity rhabdomyosarcoma. This document addresses surgical management, including biopsy, nodal staging, timing of therapy, resection and reexcision, reconstruction, and surgical approach at relapse.     

Rhabdomyosarcoma Arising in the Hand or Foot: A Clinicopathologic Analysis

Rhabdomyosarcomas (RMS) arising at different anatomic sites have distinct clinicopathologic features that affect prognosis and outcome. RMS arising in the extremities generally have a worse prognosis. Case reports and anecdotal experience suggests the possibility that RMS of the hand or foot (RMSHF) may behave even more aggressively than RMS elsewhere in the extremities. This study describes clinicopathologic features and outcome of RMSHF. Charts and pathologic material from RMSHF cases from institutional files over a 32-year interval were reviewed. Additional cases from published reports and series over a 42-year interval were identified by a MEDLINE search. Pertinent clinicopathologic features for each case were compiled. Survival analysis and other data from these patients were compared with previously reported statistics in the literature for RMS at other anatomic sites, including elsewhere in the extremities. Six patients from the institutional files and 46 from published reports were identified. A number of adverse prognostic indicators were observed in the majority of these patients including alveolar morphology (86%) and distant metastasis at presentation (63%). Despite the aggressive nature of these tumors, the majority were smaller than 5 cm (61%). A propensity was observed for metastases to unusual sites such as breast, ovary, testis, pancreas, and kidney. The median survival was 19 months, and the 5-year survival was 27%. While these may reflect a constellation of findings related to site, it is also possible that alveolar histology is the main influence. RMSHF can present as a very small primary tumor with clinically evident or occult metastatic disease. Whether this reflects the location, the predominantly alveolar histologic type, or a molecular genetic subtype will require further analysis. Patients presenting with RMSHF should be thoroughly evaluated for occult metastases at the time of diagnosis and followed closely throughout treatment for subsequent involvement of unusual sites.     

Treatment results for patients with localized,completely resected (Group I) alveolar rhabdomyosarcoma on Intergroup Rhabdomyosarcoma Study Group (IRSG) protocols III and IV, 1984–1997: A report from the Children's Oncology Group

Purpose

To assess local control, event‐free survival (EFS), and overall survival (OS) rates in 71 patients with localized, completely resected (Group I) alveolar rhabdomyosarcoma (ALV RMS) and their relation to radiation therapy (RT) on IRSG Protocols III and IV, 1984–1997.

Methods

Chart review and standard statistical procedures.

Patients and Tumors

Patients were 1–18 years at diagnosis (median, 6 years). Primary tumor sites were extremity/trunk (N = 54), head/neck (N = 9), genitourinary tract (N = 7), and perineum (N = 1). Thirty patients received VA ± C with RT; 41 received VA ± C alone. RT was assigned, not randomized.

Results

Fifty‐four patients had Stage 1 (favorable site, any size) or Stage 2 (unfavorable site, ≤5 cm) tumors. Eight‐year EFS was 90%, with 100% local control for 17 patients given RT. Eight‐year EFS was 88%, with 92% local control for 37 patients without RT; P = 0.52 for EFS comparisons, 0.3 for local control comparisons. In 17 Stage 3 patients (unfavorable site, tumors >5 cm, N0), 8‐year EFS was 84% with 100% local control in 13 patients given RT; 8‐year EFS was only 25% and local control 50% in 4 patients without RT. Local recurrence was the most common site of first failure in non‐irradiated patients.

Conclusion

Patients with Stage 1–2 ALV RMS had slightly but statistically insignificantly improved local control, EFS, and OS rates when local RT was given. The need for local RT in Stage 1–2 patients deserves evaluation in a randomized study. Local control, EFS, and OS rates were significantly improved in Stage 3 patients receiving local RT. Pediatr Blood Cancer. 2010;55:612–616. © 2010 Wiley‐Liss, Inc.     

儿童部分性癫癎预后及相关因素分析

目的了解儿童部分性癫痫预后与临床、脑电图、药物等因素之间的关系。方法复习233例儿童部分性癫痫患者的临床、脑电图资料和药物治疗、控制情况。将患者按疗效分为控制、好转、无效。通过x^2检验和t检验分析以上各因素间癫痫控制结果的差异，并对目前常用的一线抗癫痫药物疗效进行比较；应用Logistic回归分析各相关因素的意义。结果癫痫得到控制167例，发作明显减少31例，无效35例。初次发作年龄〈3岁组与≥3岁组相比，控制预后差，差异有显著意义（t=-2．241 P=0．026）。癫痫发作频率〉3次／月患儿，发作频率较≤3次／月患儿控制效果差（x^2=8．976P=0）；简单部分性发作患儿预后最好，复杂部分性患儿其次，部分性发作继发全身性发作患儿预后最差，三者间差别有显著性意义（x^2=16．860 P=0）；常用一线抗癫疴药物卡马西平、丙戊酸、苯巴比妥三者间控制效果比较，差异无显著性意义（x^2=0．107P=0．948）。Logistic回归分析显示，癫痫治疗效果与初次发作年龄、发作频率、癫痫类型呈显著相关（P=0．053，0．007，0．054）。结论初次发作年龄越小，惊厥频率越高，癫痫控制预后越差；简单部分性发作预后最好，部分性发作继发全身性发作预后最差；治疗儿童部分性癫痫。丙戊酸、苯巴比妥和卡马西平同样有效。     

儿童原发性IgA肾病免疫、病理特点及预后因素分析

目的探讨儿童原发性IgA肾病(IgAN)的免疫、病理特点及其预后影响因素。方法选择1999年8月-2010年8月本院确诊的48例IgAN儿童,分析其临床资料并进行长期随访,根据随访终点时的临床表现分为A、B、C、D 4种结局作为预后评估指标。采用多因素分析评估影响预后的危险因素。结果 1.体液免疫:29.3%患儿血IgA升高,39.0%血IgG降低;细胞免疫:近50%的患儿细胞免疫指标不正常。2.病理结果:采用Lee氏分级,Ⅱ级25例,Ⅲ级18例,Ⅳ级5例,未发现Ⅰ和Ⅴ级。70.8%伴肾间质受损。免疫荧光以IgA+IgG+IgM沉积者占58.3%,单独IgA沉积者仅占6.3%。3.预后:A级29例(60.4%);B级15例(31.3%);C级4例(8.3%);未发现D级。4.影响预后因素:经Logistic回归分析,病理Ⅳ级与预后有关(P<0.05);高血压、大量蛋白尿和肾衰竭对预后影响无统计学意义。结论儿童IgAN存在免疫紊乱,但预后较好;肾脏病理分级是影响儿童IgAN预后的重要因素。     

重症病毒性脑炎患儿预后及其相关因素分析

目的 评估重症病毒性脑炎(SVE)患儿的预后,探讨影响其预后的因素.方法 回顾性分析2009年1月-2010年8月中国医科大学附属盛京医院儿科收治的56例SVE患儿的临床资料,并在患儿出院4个月后采用Liverpool预后评分量表评估其预后.根据预后将患儿分为治愈组和未愈组,采用SPSS 13.0软件进行数据处理,采用t检验和Binary Logistic回归检验分析2组临床资料与预后的相关性.结果 35％(19/54例)的SVE患儿死亡或留有严重神经系统后遗症,其中住院期间死亡7例(13％),随访中死亡3例(6％),留有严重后遗症9例(17％)、中度后遗症7例(13％)、轻微后遗症5例(9％).导致SVE患儿预后不良的有统计学意义的因素有脑电图中度异常、惊厥持续状态、发热病程长、头颅MRI累及病灶超过2处或累及幕下及合并应激性高血糖(Pa＜0.05),而脑脊液白细胞数、脑脊液蛋白量、偏瘫、意识障碍、合并多脏器损伤、低钾血症、低钠血症与SVE预后不良无关.结论 SVE严重威胁儿童的生存质量.伴有惊厥持续状态、应激性高血糖、发热时间长、脑电图中度异常及累及病灶超过2处或累及幕下等是影响儿童SVE预后的危险因素.     

Recurrence and mortality prognostic factors in childhood adrenocortical tumors: Analysis from the Brazilian National Institute of Cancer experience

Prognostic markers that can help identifying precocious risk of unfavorable outcomes in patients with childhood adrenocortical tumors (ACTs) are still unclear. This observational and retrospective study aimed to identify clinical and pathology prognostic factors of recurrence and death in a tertiary cancer center population. Clinical, pathology, demographic, staging, and therapy data from patients with childhood ACT (median age: 3.6 years) treated at the Brazilian National Institute of Cancer between 1997 and 2015 were assessed. Univariate and bivariate analyses were used to study the association of clinical and pathology characteristics with recurrence and mortality. Recurrence and disease-related mortality were the main outcomes. Twenty-seven patients were included. Complete tumor resection was performed in 21 cases. The median tumor size was 8.2 cm. Mitotane was the most common adjuvant/palliative therapy (n = 13). Recurrence occurred in 6 patients, after a median time of 7.2 months, and was more common among those with larger tumors (P =.008), higher Weiss score (P =.001), and microscopic tumoral necrosis (P =.002). Ten patients died from the disease. Older age (P =.04), larger tumor size (P =.002), metastatic disease (P =.003), previous recurrence (P =.003), incomplete resection (P =.002), intraoperative tumor spillage (P =.005), higher Weiss score (P =.03), microscopic necrosis (P =.005), and capsular invasion (P =.02) were all associated with increased death risk. Even though complete tumor resection was performed in most cases, a considerable number of cases of childhood ACT resulted in recurrence and death. Early identification of unfavorable outcomes is essential to determine ideal therapy and appropriate surveillance.     

Multiple listing for pediatric heart transplantation in the USA: Analysis of OPTN registry data from 1995 through 2009

Multiple listing is associated with shorter waitlist durations and increased likelihood of transplantation for renal candidates. Little is known about multiple listing in pediatric heart transplantation. We examined the prevalence and outcomes of multiple listing using OPTN data from 1995 through 2009. Characteristics and waitlist outcomes of propensity‐score‐matched single‐ and multiple‐listed patients were compared. Multiple listing occurred in 23 of 6290 listings (0.4%). Median days between listings was 35 (0–1015) and median duration of multiple listings was 32 days (3–363). Among multiple‐listed patients, there were trends toward less ECMO use (0% vs. 11%, p = 0.1) and more frequent requirement for a prospective cross‐match (17% vs. 8%, p = 0.08). Multiple‐listed patients more commonly had private insurance (78% vs. 56%; p = 0.03). Urgency status at listing was similar between groups (1/1A: 61% vs. 64%, 1B/2: 39 vs. 36%; p = 0.45) as were weight, age, diagnosis, ventilator/inotrope use, and median income (each p ≥ 0.17). There was a trend toward increased incidence of heart transplantation for multiple‐listed patients at three, six, and 24 months (50%, 65%, 80%) vs. single‐listed patients (40%, 54%, 64%; p = 0.11). Multiple listing for pediatric heart transplantation in the USA occurs infrequently and is more common in patients with private insurance.     

Exercise right heart catheterization for pulmonary hypertension identified on screening echocardiography in adult survivors of childhood cancer: A report from the St. Jude Lifetime Cohort

Pulmonary hypertension, determined noninvasively by tricuspid regurgitant jet velocity on Doppler echocardiography, was previously identified in 25% of long‐term survivors who received chest‐directed radiotherapy. To validate noninvasively defined pulmonary hypertension, survivors (mean age 48 years), exposed to chest radiotherapy, underwent right heart catheterization with planned cardiopulmonary exercise testing during catheterization. Eight participants had an elevated mean pulmonary artery pressure at rest (≥25 mm Hg) or with subsequent exercise (>30 mm Hg), evidence of hemodynamically confirmed pulmonary hypertension by right heart catheterization. Cardiopulmonary exercise testing further defined the magnitude and etiology of cardiopulmonary limitations in this life‐threatening late effect.     

Similar outcomes of allogeneic hematopoietic cell transplantation from unrelated donor and umbilical cord blood vs. sibling donor for pediatric acute myeloid leukemia: Multicenter experience in China

In a multicenter study, we have conducted a retrospective study on 73 pediatric AML patients who were primary refractory or in greater than CR1 and investigated MSD (or MMSD) (n = 20), URD (n = 23), and UCB (n = 30) HCT between January 1998 and October 2009. The median day to neutrophil engraftment was similar in all groups. The median day to platelet engraftment was longer in the UCB group. The number of HLA mismatch was higher in the UCB group (p = 0.034); however, the cumulative incidence of grade III–IV aGVHD was not different among all groups (p = 0.125); furthermore, cGVHD was lower in the UCB group (p = 0.078). The risk of relapse did not differ among all groups (RR = 1.28, p = 0.125), but the patients of MSD (or MMSD) grafts had a trend of higher risk recurrence. Sixty‐two patients survived with a median follow‐up of 58.2 months. Five‐yr LFS was 73.1%, 59.8%, and 59.6% for URD, UCB, and MSD (or MMDS), respectively (p = 0.426). Five‐yr LFS in CR1 was 68.9%, with a significantly better result compared to 41.7% in CR2 (p = 0.025). Our comparisons suggest that pediatric AML patients receiving UCB had a higher early TRM, a lower cGVHD rate, and a similar long‐term survival. The outcome of URD and UCB is comparable to that of a suitable sibling for pediatric AML.