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PubMed1997

Venoocclusive disease of the liver after chemotherapy with vincristine, actinomycin D, and cyclophosphamide for the treatment of rhabdomyosarcoma. A report of the Intergroup Rhabdomyosarcoma Study Group. Childrens Cancer Group, the Pediatric Oncology Group, and the Pediatric Intergroup Statistical Center.

BACKGROUND: Venoocclusive disease (VOD) of the liver is a common complication after allogenic and autologous bone marrow transplantation for malignant disease. The authors report the unusual and unexpected complication of VOD of the liver occurring in children with rhabdomyosarcoma who were receiving vincristine, actinomycin D, and cyclophosphamide (VAC) according to the chemotherapy regimens of the Intergroup Rhabdomyosarcoma Study (IRS) IV. METHODS: The authors evaluated 821 patients with newly diagnosed rhabdomyosarcoma receiving treatment according to IRS IV who were considered at risk for VOD. RESULTS: Ten patients developed VOD of the liver for an overall incidence of 1.2%. VOD was found only after the administration of VAC chemotherapy. The highest incidence of VOD was observed among patients who had previously received vincristine and melphalan (Regimen 48). None of the patients receiving the chemotherapy regimen of vincristine and actinomycin D (Regimen 44) developed VOD. CONCLUSIONS: Patients receiving VAC-containing regimens on the IRS IV were found to be at risk for VOD. The VAC combination was used extensively in previous IRS studies (I, II, and III) and VOD was not reported during these studies, strongly suggesting that the escalation of the cyclophosphamide dose to 2.2 g/m2 (with the vincristine and actinomycin D doses and schedule remaining unchanged) triggered the development of VOD. The contributing role of previous therapy or events is unknown. At last follow-up, none of the nine surviving patients had developed recurrent VOD on continuation of chemotherapy.

Adolescent↗
PubMed1986

Late effects of therapy in orbital rhabdomyosarcoma in children. A report from the Intergroup Rhabdomyosarcoma Study.

Late effects of therapy were evaluated in 50 children surviving orbital rhabdomyosarcoma following treatment on Intergroup Rhabdomyosarcoma Study I. All patients had microscopic or gross tumor present following surgery and subsequently received radiotherapy and various combinations of chemotherapeutic agents. Problems in the eye included infections and functional and structural changes. Decreased vision in the treated eye was the most common functional problem and in most patients related to cataract formation, which occurred in 90% of eyes. Changes in the cornea and retina were also seen. Bony hypoplasia of the orbit and facial asymmetry were present in one half of the children. Gonadal development was normal. Statural growth was retarded in 61% of the patients. All children were in school, with five having learning or behavioral problems. One child developed acute myeloblastic leukemia. The excellent survival in patients with orbital rhabdomyosarcoma provides support for treatment programs that use multiple-agent chemotherapy and radiotherapy and do not include orbital exenteration initially.

Antineoplastic Combined Chemotherapy Protocols↗
PubMed2001

Rhabdomyosarcoma of the parotid region occurring in childhood and adolescence. A report from the Intergroup Rhabdomyosarcoma Study Group.

BACKGROUND: Rhabdomyosarcoma (RMS) of the parotid region is rare and to the authors' knowledge little information is available regarding the site of tumor origin, clinical presentation, and outcome in these patients. Therefore, the authors reviewed the files of all patients with RMS of the parotid region who were registered on the Intergroup Rhabdomyosarcoma Studies (IRS) I-IV. METHODS: Patient charts and the Intergroup Rhabdomyosarcoma Study Group (IRSG) database were reviewed. RESULTS: Sixty-two patients presenting with a mass in the parotid region were identified. None of the tumors was localized exclusively to the parotid gland, so the primary site was referred to as the "parotid region." The tumor invaded a parameningeal site in 30 patients. These cases have been designated as parameningeal-parotid tumors to distinguish them from 32 cases that did not invade a parameningeal site and were designated as nonparameningeal-parotid tumors. The majority of patients had Group III tumors in both the nonparameningeal-parotid and parameningeal-parotid subgroups. However, although there were 16 patients with Group I or II tumors in the nonparameningeal-parotid subgroup, no patients with Group I or II tumors were found in the parameningeal-parotid subgroup (P = 0.001). Fifty-six of 62 patients (90%) received radiotherapy. The parameningeal primary site designation resulted in intensification of both chemotherapy and radiotherapy for patients with parameningeal-parotid RMS. The 5-year failure-free survival rate was 81% and the 5-year survival rate was 84%. There were no deaths reported among patients with Group I or II tumors. The 5-year failure-free survival did not appear to differ when comparing patients with parameningeal-parotid tumors with patients with nonparameningeal-parotid tumors (P = 0.21). CONCLUSIONS: Treatment as defined by the IRS protocols has been reported to be highly effective for patients with RMS of the parotid region. Outcome for the more aggressively treated patients with parameningeal-parotid RMS appears similar to that for patients with nonparameningeal-parotid RMS.

Adolescent↗
PubMed1995

Conservative management of uterine pediatric rhabdomyosarcoma: a report from the Intergroup Rhabdomyosarcoma Study III and IV pilot.

Previous studies have suggested that women with uterine rhabdomyosarcomas (RMS) represent a distinct group of patients who present at an older age, are less responsive to treatment, and have a poorer prognosis than patients with vaginal RMS. During the Intergroup Rhabdomyosarcoma Study (IRS) III and the IRS IV pilot study, 14 patients were registered with uterine primary RMS. Three patients presented with cervical tumors that were completely removed (group 1). Eight patients had initial biopsies with gross residual disease (group 3), and 3 had metastatic disease at presentation (group 4). Of the 5 patients treated with primary chemotherapy or chemotherapy and radiation, 2 had delayed hysterectomy and vaginectomy, 1 had no further surgery, and 2 had exploratory laparotomy with no evidence of disease. There were no relapses or deaths in this group. One patient underwent initial resection of a broad ligament mass, experienced an early (3-week) recurrence of the mass while on chemotherapy, and progressed to developing distant metastases and death. Four patients died of chemotherapy toxicity or sepsis, one after achieving a complete response from chemotherapy and hysterectomy. This primary chemotherapy or chemotherapy and radiotherapy regimen resulted in 8 of 9 (89%) patients (not including those who died of chemotoxicity) surviving between 1.5 and 6 years without evidence of disease. Of the surviving patients, 2 had hysterectomy and vaginectomy, but pathological specimens showed only localized microscopic residual tumor. This report suggests that less vigorous operative resection may be possible in combination with primary chemotherapy when treating uterine rhabdomyosarcomas.(ABSTRACT TRUNCATED AT 250 WORDS)

Adolescent↗
PubMed2002

Sclerosing rhabdomyosarcoma in adults: report of four cases of a hyalinizing, matrix-rich variant of rhabdomyosarcoma that may be confused with osteosarcoma, chondrosarcoma, or angiosarcoma.

Rhabdomyosarcomas (RMSs) are classified into embryonal (ERMS), alveolar (ARMS), and pleomorphic (PRMS) subtypes. ERMS, including botryoid variants, typically occurs in young children, ARMS typically occurs in older children and young adults, and PRMS occurs in older adults. Although ARMSs show thin fibrous bands separating nests of cells, abundant extracellular matrix production is rare in RMS. In the course of reviewing hyalinizing sarcomas we discovered a distinctive RMS in adults that closely mimicked osteosarcoma or chondrosarcoma because of the extensive matrix production. Four RMSs with hyalinized matrix were retrieved from our files. These cases were evaluated with respect to patient age and sex, tumor site and size, growth pattern, nuclear grade, cellularity, mitotic figures/20 high power fields, vascular invasion, necrosis, the presence of rhabdomyoblasts, multinucleated cells, and alveolar growth pattern. Immunohistochemistry for desmin, myogenin, MyoD1, actin, cytokeratin, S-100 protein, collagen II, and CD99 was performed. Reverse transcriptase polymerase chain reaction for the ARMS-associated PAX3/FKHR and PAX7/PKHF was also performed on three cases. The cases involved the forearm, hand, orbit, and nasopharynx of a 40-year-old woman, a 50-year-old man, an 18-year-old man, and a 21-year-old man, respectively. The tumors ranged from 3.7 to 8 cm and consisted of lobules and infiltrating cords of small round malignant cells embedded in a densely hyalinized matrix having both a chondroid and osteoid-like appearance. No definite lacunae or matrix calcification was present. An alveolar pattern was only present focally, and tumor giant cells were not present. One case had a single focus of rhabdomyoblastic differentiation with strap cells. Mitotic activity was >20 mitotic figures/20 high power fields in three of four cases. Immunohistochemically, one case strongly expressed desmin, whereas three cases expressed it focally, with a dot-like pattern. Myogenin was only focally positive, but MyoD1 was present in nearly every cell of each case. Two cases expressed actin and one expressed CD99. No case expressed cytokeratin, S-100 protein, or collagen II. Only one case contained adequate RNA for reverse transcriptase polymerase chain reaction, and this case was negative for the ARMS-associated gene fusions. Follow-up showed one patient to be dead of metastatic disease at 60 months despite intensive therapy, another patient to be disease free at 26 months, and the third patient to be disease free at 5 months. The fourth case is recent. These cases are a distinctive-appearing rhabdomyosarcoma easily mistaken for variants of chondrosarcoma, osteosarcoma, or even sclerosing epithelioid fibrosarcoma because of their hyalinizing appearance compounded by their typically focal and dot-like desmin expression. These four cases are essentially identical to the three unusual RMSs recently reported by Mentzel and Katenkamp as "sclerosing, pseudovascular rhabdomyosarcoma in adults." Although the focal alveolar architecture and the primitive cytologic appearance of these hyalinizing RMS suggest a relationship with ARMS, the presence of abundant strap cells in one case, the predominant expression of MyoD1 rather than myogenin, and the absence of ARMS-associated fusions genes point more strongly toward a variant of ERMS. However, the late adult age in two cases is unusual for both EMRS and ARMS, suggesting that sclerosing RMS may prove to be a distinct subtype of RMS. Study of additional cases will be necessary to more fully elucidate its place among RMS and its prognostic significance.

Adolescent↗
PubMed2001

Ifosfamide and etoposide are superior to vincristine and melphalan for pediatric metastatic rhabdomyosarcoma when administered with irradiation and combination chemotherapy: a report from the Intergroup Rhabdomyosarcoma Study Group.

PURPOSE: This study was designed to estimate the partial and complete response rates (CR and PR) of two novel drug pairs (vincristine and melphalan vs. ifosfamide and etoposide) and to improve overall survival of previously untreated patients with metastatic rhabdomyosarcoma. PATIENTS AND METHODS: One hundred twenty-eight patients were randomly assigned to phase II window therapy consisting of vincristine and melphalan (VM-containing regimen) or ifosfamide and etoposide (IE-containing regimen). Brief window therapy (12 wks) was immediately followed-up by vincristine, dactinomycin, and cyclophosphamide (VAC), chemotherapy, surgery, and irradiation, with continuation of either VM or IE in patients with initial response. Major endpoints were initial CR and PR rates after the phase II window phase of therapy, failure-free survival (FFS), and survival. RESULTS: Patients who received the VM-containing regimen experienced significantly more anemia, neutropenia, thrombocytopenia, and had more cyclophosphamide dose reductions. The initial PR and CR rates were not significantly different for patients treated with either regimen (VM, 74%; IE, 79%; P = 0.428). However, FFS and overall survival (OS) at 3 years were significantly better with the IE-containing regimen (FFS: 33% vs. 19%; P = 0.043; OS: 55% vs. 27%; P = 0.012). CONCLUSIONS: Although the VM-containing regimen produced a high response rate, inclusion of melphalan appeared to limit the cyclophosphamide dose that could be administered, and ultimately, this regimen was associated with a significantly worse outcome than was the IE-containing regimen. Also, the IE-containing regimen was associated with a gratifyingly high survival rate at 3 years (55%), which is significantly higher than has been observed on any previous Intergroup Rhabdomyosarcoma Study Group regimen for similar patients. We believe that this promising outcome indicates that this drug pair merits further randomized testing in metastatic rhabdomyosarcoma.

Adolescent↗
PubMed2003

Treatment of localized nonorbital, nonparameningeal head and neck rhabdomyosarcoma: lessons learned from intergroup rhabdomyosarcoma studies III and IV.

PURPOSE: The characteristics and clinical outcomes of children and adolescents with localized nonorbital, nonparameningeal head and neck rhabdomyosarcoma (RMS) treated on national protocols from the Intergroup Rhabdomyosarcoma Group are reported. PATIENTS AND METHODS: We conducted a retrospective review of 164 children and adolescents enrolled in the third and fourth Intergroup Rhabdomyosarcoma Studies. Variables analyzed included age, sex, primary tumor site, histologic subtype, clinical group, therapy, site and rate of treatment failure, and time to initial recurrence. RESULTS: Localized nonorbital, nonparameningeal RMS accounted for 9% of all cases of RMS. The median age at diagnosis was 5 years; the median follow-up was 6.6 years. Estimated 5-year failure-free survival (FFS) and survival (S) rates were 76% (95% CI, 69% to 83%) and 83% (95% CI, 77% to 89%), respectively. In multivariate analysis, patients with clinically involved regional nodes (N1) had worse FFS (P =.02). For patients with embryonal tumors, FFS was significantly improved, especially among patients with Group I/II without nodal disease clinical Group I, II N0. For patients with alveolar/undifferentiated histology, FFS was significantly worse in children under the age of 1 year. Actuarial estimates of recurrences at 15 years were local (19%), regional (5%), and distant (9%). CONCLUSION: More than 80% of patients with RMS of the head and neck are cured of their disease using surgery and vincristine, dactinomycin +/- cyclophosphamide with or without radiotherapy. Our results indicate that early, limited exposure to cyclophosphamide might reduce recurrence in low-risk embryonal patients and that reduced dosages might achieve comparable results with improved toxicity profiles. These hypotheses will be tested in the next generation of trials from the Soft Tissue Committee of the Children's Oncology Group.

Adolescent↗
PubMed1997

Primary and metastatic rhabdomyosarcoma in the breast: neoplasms of adolescent females, a report from the Intergroup Rhabdomyosarcoma Study.

UNLABELLED: The occurrence of rhabdomyosarcoma (RMS) primary in or metastatic to breast has been regarded as an uncommon event, associated with an unfavorable outcome. Records of 26 patients with diagnoses of breast RMS, either primary or secondary, entered in the Intergroup Rhabdomyosarcoma Study (IRS) (1972-1992) were reviewed and compared with data regarding 47 similar patients in published reports. Of the 26 IRS cases, the histologic subtype was alveolar in 24, embryonal in 1, and not determined in 1. All were female with ages ranging from 11.5 to 20.2 years (median, 15.2 years; mode, 14-16 years). This compact age distribution of both primary (n = 7) and metastatic (n = 19) breast RMS was seen in previously reported series. Among the 19 cases of RMS with initial dissemination to breast, primary tumor sites, were extremity (n = 8), nasopharynx/paranasal sinuses (n = 7), and trunk (n = 4). IRS treatment was risk-based according to site and extent of disease. Four of 7 patients with primary RMS remain disease free 2.9 to 7 years post diagnosis. Among 19 patients with RMS initially metastatic to breast, including 7 in IRS clinical group IV at original diagnosis, three are disease free at 7.6, 15.7 and 17.0 years. CONCLUSIONS: primary or metastatic RMS in breast is almost confined to adolescent females having tumors with alveolar histology. Approximately one-half of the patients with primary breast disease and 15% of those with metastatic breast disease as an initial recurrence are long-term survivors.

Adolescent↗
PubMed2001

Routine brain imaging is unwarranted in asymptomatic patients with rhabdomyosarcoma arising outside of the head and neck region that is metastatic at diagnosis: a report from the Intergroup Rhabdomyosarcoma Study Group.

BACKGROUND: To the authors' knowledge, the incidence of brain metastases at the time of diagnosis in children with metastatic rhabdomyosarcoma (RMS) arising outside the head and neck region is unknown, and routine imaging to identify metastatic brain involvement is costly. METHODS: The authors retrospectively reviewed the results of computed tomography (CT) or magnetic resonance imaging (MRI) scans of the head, which was mandated by protocol, in patients with metastatic RMS arising outside the head and neck region who were enrolled on the fourth Intergroup Rhabdomyosarcoma Study (IRS-IV; 1991--1997). RESULTS: Of 100 eligible patients with metastatic RMS arising outside the head and neck region, 56 (56%) underwent head CT (n = 51) and/or MRI (n = 11) scans. Seven of these 56 patients (12.5%) had abnormal scans. Three patients with physical findings suggesting head or neck pathology underwent imaging that confirmed the presence of metastases in bone (one patient), orbit (one patient), or lymph nodes (one patient). One patient who presented with seizures had imaging findings consistent with cerebral embolic infarctions. Of three asymptomatic patients, one had bone metastases that also were identified on skeletal survey and one had bone metastases in the base of the skull that were not identified on bone scan. The remaining asymptomatic patient had a retroperitoneal paraspinal tumor with spinal canal extension and subsequently developed leptomeningeal disease dissemination. CONCLUSIONS: Brain metastases are uncommon at the time of initial diagnosis of metastatic RMS arising outside the head and neck region, and the majority of abnormalities detected on head CT or MRI scans are evident clinically or on other imaging studies. Patients with clinical findings suggesting intracranial pathology and those with paraspinal tumors may benefit from brain imaging, but cost savings may be realized by foregoing imaging in patients without these features.

Brain Neoplasms↗
PubMed2003

Prognostic factors and surgical treatment guidelines for children with rhabdomyosarcoma of the perineum or anus: a report of Intergroup Rhabdomyosarcoma Studies I through IV, 1972 through 1997.

BACKGROUND/PURPOSE: Rhabdomyosarcoma (RMS) of the perineum or anus is a rare sarcoma of childhood with a poor prognosis. This study reviews the Intergroup Rhabdomyosarcoma Study Group (IRSG) studies I through IV to identify determinants of patient outcome and to refine surgical treatment guidelines. METHODS: From 1972 through 1997, 71 eligible patients were treated and studied. The median patient age was 6 years. The majority (64%) were at an advanced stage (clinical group III and IV) at initial presentation and 50% had positive regional lymph node (LN) involvement. RESULTS: The 5-year failure-free survival rate (FFS) for all patients was 45% and the overall survival rate (OS) was 49%. Characteristics that were associated with significantly improved survival rate were primary tumor size less than 5 cm, lower (less advanced) clinical group and stage, negative regional lymph node status, and age less than 10 years. When the extent of disease was controlled for in multivariate analysis, only age less than 10 predicted an improved outcome. The 5-year overall survival rate for patients less than 10 years of age was 71% versus 20% in older patients (P <.001). Histology (alveolar versus embryonal) and primary site (perineal versus anal) did not significantly affect outcome. CONCLUSIONS: Because of the high incidence of regional LN involvement in these patients, a strategy of routine surgical evaluation of ilioinguinal lymph nodes in all patients with perineal or anal RMS is recommended.

Adolescent↗
PubMed2001

Molecular cytogenetic analysis consistently identifies translocations involving chromosomes 1, 2 and 15 in five embryonal rhabdomyosarcoma cell lines and a PAX-FOXO1A fusion gene negative alveolar rhabdomyosarcoma cell line.

Rhabdomyosarcoma in children is a "small round blue cell tumour" that displays skeletal muscle differentiation. Two main histological variants are recognised, alveolar (ARMS) and embryonal (ERMS) rhabdomyosarcoma. Whereas consistent chromosome translocations characteristic of ARMS have been reported, no such cytogenetic abnormality has yet been described in ERMS. We have used multiple colour chromosome painting to obtain composite karyotypes for five ERMS cell lines and one PAX-FOXO1A fusion gene negative ARMS. The cell lines were assessed by spectral karyotyping (SKY), tailored multi-fluorophore fluorescence in situ hybridisation (M-FISH) using series of seven colour paint sets generated to examine specific abnormalities, and comparative genomic hybridisation (CGH). This approach enabled us to obtain karyotypes of the cell lines in greater detail than previously possible. Several recurring cytogenetic abnormalities were demonstrated, including translocations involving chromosomes 1 and 15 and chromosomes 2 and 15, in 4/6 and 2/6 cell lines respectively. All six cell lines demonstrated abnormalities of chromosome 15. Translocations between chromosomes 1 and 15 have previously been recorded in two primary cases of ERMS by conventional cytogenetics. Analysis of the translocation breakpoints may suggest mechanisms of ERMS tumourigenesis and may enable the development of novel approaches to the clinical management of this tumour.

Child↗
PubMed1987

Pediatric orofacial and laryngopharyngeal rhabdomyosarcoma. An Intergroup Rhabdomyosarcoma Study report.

Eighty-nine children with localized rhabdomyosarcoma of orofacial and laryngopharyngeal sites were treated in accordance with the first and second Intergroup Rhabdomyosarcoma Study (IRS) protocols (IRS-I and IRS-II) between 1972 and 1984. Treatment included surgery (or biopsy) and chemotherapy for all patients and radiotherapy in the majority. The actuarial estimate of the three-year survival rate for all patients was 83% and did not differ significantly by primary site, histologic findings, or presence of adenopathy. A trend for a worse survival rate was seen in clinical group III patients and in those less than 5 years of age at diagnosis. Factors associated with an increased risk of local/regional relapse included omission of radiotherapy and a radiation dose of less than 40 Gy (4000 rad). We conclude that treatment of these patients as recommended in the IRS-I and IRS-II protocols results in very good local and regional tumor control and survival rates. Salvage therapy for local/regional recurrence may yield long-term remission and possibly cure.

Adolescent↗
PubMed2000

Late effects of therapy in 94 patients with localized rhabdomyosarcoma of the orbit: Report from the Intergroup Rhabdomyosarcoma Study (IRS)-III, 1984-1991.

BACKGROUND: We reviewed the late complications of therapy in 94 patients with localized, primary rhabdomyosarcoma of the orbit treated on the Intergroup Rhabdomyosarcoma Study (IRS)-III protocol (1984-1991). PROCEDURE: A questionnaire was sent to the institutions that had registered 106 patients with orbital RMS on the IRS-III protocol, seeking information about vision, periocular structures, and growth and development of the 102 survivors. RESULTS: Ninety-four questionnaires were returned. The median follow-up interval was 7.6 years. The affected eye was removed from 13 patients because of local recurrence (N = 10) or other causes (N = 3). Seventy-nine of the eighty-one remaining patients had received radiation therapy. Sixty-five of these seventy-nine patients (82%) developed a cataract, and 43 of them (66%) underwent cataract surgery. Fifty-five patients (70%) had decreased visual acuity. Twenty-four patients had a dry eye, and 22 had chronic keratitis, conjunctivitis, or corneal changes. Strabismus, diplopia, retinopathy, and uveitis were uncommon. The orbit was hypoplastic in 48 of 82 patients assessed (59%). Ptosis and enophthalmos were reported in 22 patients. Decreased statural growth was noted in 13 of the 53 irradiated patients aged 3-14 years at diagnosis with sufficient data (24%). CONCLUSIONS: The overall survival rate was 96% (102/106). The eye was preserved in 86% of the patients, but vision was impaired in 70% of them. Other frequent complications were cataract, orbital hypoplasia, keratoconjunctivitis, and ptosis/enophthalmos. The current IRS-V study recommends decreasing the dose of irradiation and using conformal techniques in an attempt to minimize these complications.

Adolescent↗
PubMed1982

Three-year relapse-free survival rates in childhood rhabdomyosarcoma of the head and neck: report from the Intergroup Rhabdomyosarcoma Study.

In 202 patients with rhabdomyosarcoma of the head and neck who registered in the first Intergroup Rhabdomyosarcoma Study, the primary lesions arose about the eye and orbit in 26%, in parameningeal sites in 46%, and in other head and neck areas in 28%. Histopathologically, 78% were embryonal-botryoid, 9% alveolar, 10% undifferentiated, and 3% extraosseous Ewing's types. Actual three-year relapse-free survival rates were calculated from data on 103 of these patients who were free of distant metastases at diagnosis and in whom follow-up had been completed for a three-year period. The actual relapse-free survival rates were 91% (21/23) for those with eye/orbit primaries, 46% (20/44) for those with parameningeal primaries, and 75% (27/36) for those with other head and neck sites affected. Among those with no clinical evidence of tumor activity at two years, 8% (6/75) had subsequent relapses.

Child↗
PubMed1985

Rhabdomyosarcoma of the biliary tree in childhood. A report from the Intergroup Rhabdomyosarcoma Study.

Ten cases of rhabdomyosarcoma of the biliary tree (RMS/BT) in children are reported in this review of the Intergroup Rhabdomyosarcoma Study (IRS) I and II. RMS/BT constitutes 0.8% of evaluable tumors on IRS I and II. Intermittent obstructive jaundice with or without abdominal distention, fever, and loss of appetite is the typical presentation. Attribution of these symptoms to hepatitis commonly delayed definitive treatment. Demonstration of a mass in the porta hepatitis by ultrasound led quickly to surgical consultation and an exploratory laparotomy. Intraoperative cholangiography was a valuable technique in establishing the level of biliary tree obstruction and verifying a functioning drainage procedure. Resection of the mass with only microscopic or minimal gross residual disease was possible at the initial surgery in six of the ten patients. Included in this group are all four of the surviving patients. The continuity of bile flow was usually maintained by variations of a Roux-en-Y jejunostomy. Histology was embryonal RMS with some botryoid elements. Consultation with a surgical pathologist during the course of the surgery was of invaluable assistance. Multi-drug chemotherapy and radiotherapy consistent with the IRS protocols were given postoperatively. A second-look surgery was useful in evaluating residual or recurrent disease. Four patients are surviving free of disease at 6 months, 3, 6 1/4, and 6 1/2 years from diagnosis, respectively. This review demonstrates that a multidisciplinary approach in RMS/BT can lead to long-term survival.

Bile Duct Neoplasms↗
PubMed1987

Lymphatic metastases with childhood rhabdomyosarcoma. A report from the Intergroup Rhabdomyosarcoma Study.

A review of 1415 patients without distant metastasis from the Intergroup Rhabdomyosarcoma Study (IRS) I and II revealed an overall 10% incidence of identified lymphatic spread at diagnosis, whereas 81 of 592 children with localized rhabdomyosarcoma who had grossly complete resection (and therefore with more complete pathologic data) had histologically proven lymphatic spread (14%). The percentage of patients in this latter group with nodal metastases was highest for the prostate (41%), paratesticular sites (26%), and genitourinary sites overall (24%). Sites with a small percentage of proven lymphatic involvement were the orbit (0%), nonorbital head and neck sites (7%), and truncal sites (3%), whereas the percentage of patients with nodal metastases from extremity lesions was 12%. The primary tumor mean diameter was significantly larger in the group with nodal metastases, but there was no evidence of a relationship between lymphatic spread and age, sex, or histologic subtype. Patients with lymph node metastases who had resection had a poorer survival rate (logrank P value = 0.001), with a 3-year survival estimate of 54%, compared with 78% for patients without lymphatic metastases. Patients with extremity lesions and positive lymph nodes also did poorly when compared with patients with normal nodes (P = 0.006), and a similar observation was made for patients with paratesticular sarcoma (P = 0.06).

Actuarial Analysis↗
PubMed1991

Prognostic factors in 951 nonmetastatic rhabdomyosarcoma in children: a report from the International Rhabdomyosarcoma Workshop.

A total of 951 newly diagnosed patients with nonmetastatic rhabdomyosarcoma (RMS) under 21 years of age from four different cooperative study groups were analyzed to identify the most important pretreatment tumor characteristics in predicting survival. The patient characteristics considered were tumor invasiveness (T), tumor size, status of locoregional lymph nodes, and primary site. The cooperative groups and studies analyzed were: SIOP [RMS-75 (1975-1984)], the Intergroup Rhabdomyosarcoma Study [IRS-II (1978-1982)], Federal Republic of Germany [CWS-81 (1981-1986)] and Italy [RMS-79 (1979-1986), each study involving a different therapeutic protocol. Patient characteristics identified as having a significant relationship to survival by univariate analysis were (favorable features given in brackets): tumor invasiveness [T1], tumor size [less than or equal to 5 cm], status of locoregional lymph nodes [clinically negative], and primary site [orbit and genitourinary non-bladder prostate (GU-non-BP) sites]. T2 tumors tended to have large size (greater than 5 cm), to be associated with positive nodes, and to have a different distribution by primary site than T1 tumors. A multivariate Cox regression analysis of the pooled data identified tumor invasiveness (T), primary site, and the interaction between T and primary site as significant predictive factors for survival. The prognosis of orbital tumors was consistently favorable regardless of T status, whereas the prognosis for "other sites" was consistently unfavorable. International cooperation in this study has permitted the identification of good and poor risk patient subgroups, permitting the focusing of therapeutic protocols to particular risk groups and setting the stage for common future therapeutic protocols for rare subgroups of patients.

Child↗
PubMed1995

Conservative surgical management of vaginal and vulvar pediatric rhabdomyosarcoma: a report from the Intergroup Rhabdomyosarcoma Study III.

The current management of pediatric vaginal and vulvar rhabdomyosarcoma (RMS) uses cytoreductive chemotherapy before surgical intervention. During the Intergroup Rhabdomyosarcoma Study (IRS)-III (1984 to 1988), 27 evaluable patients were entered on a preoperative chemotherapy protocol. Among 24 patients with vaginal primaries, 20 had an initial biopsy with gross residual (group III), 3 had resection with positive margins (group IIA) and 1 had metastatic disease (group IV). At subsequent surgery, 7 patients underwent partial or complete vaginectomy and 6 of them had no viable tumor identified in the specimen. Only 1 of these 7 patients underwent a cystectomy, whereas 5 underwent hysterectomy. Seventeen patients in this group have no evidence of disease 66 to 108 months after diagnosis; 2 died of chemotoxicity and 1 of unknown causes after achieving a complete response. Ten of these 17 patients were treated with biopsy and chemotherapy only. Four of these 10 had radiotherapy as well. All group IIA patients have no evidence of disease. The 1 group IV patient had rapidly progressive disease with early death. This primary chemotherapy protocol resulted in less need for operative intervention or irradiation compared with previous experience. There was no local recurrence, and 20 of 24 patients remain continuously relapse-free with no evidence of disease. All 3 patients with vulvar primaries were treated by wide local excision and chemotherapy and have no evidence of disease. Conservative surgical intervention for vaginal RMS with primary chemotherapy and adjunctive radiation when necessary appears to result in excellent disease-free survival.

Adolescent↗