Malignant peripheral nerve sheath tumors in children: a single-institution twenty-year experience

A retrospective series of pediatric patients with localized malignant peripheral nerve sheath tumors (MPNST) treated during a 20-year period at one institution is reported. Between 1976 and 1996, 24 consecutive children were treated by a multimodality approach. Conservative surgery was the treatment of choice: primary radical surgery was performed in 10. Postoperative radiotherapy was administered in 12 and adjuvant chemotherapy in 19. Eight patients were alive without evidence of disease, six in first complete remission and two in second complete remission, after a median follow-up of 230 months. The 10-year event-free survival (EFS) and survival were 29% and 41%, respectively. Survival was 80% for the patients who underwent radical surgery, and 14% for the others; 71% for patients with tumors smaller than 5 cm, and 29% for those with tumors 5 cm or larger. Local recurrence was the major cause for treatment failure (13 of 17; 76%); the rate of local relapse was 33% v 75% in patients who either received or did not receive radiotherapy. Complete surgical excision remains the most effective treatment for MPNST and represents the main prognostic factor along with tumor size. Radiotherapy seems to play a role in achieving local control, whereas the role of chemotherapy is uncertain.     

放疗及二次根治手术对儿童横纹肌肉瘤的疗效影响分析

目的探讨儿童横纹肌肉瘤(RMS)的治疗手段与疗效、预后的关系。方法回顾性分析中山大学孙逸仙纪念医院儿科2013年1月—2017年12月收治的24例儿童RMS的临床资料,总结儿童RMS的治疗策略、疗效,分析放疗及二次根治手术对儿童RMS预后的影响。结果初治儿童RMS共24例,男18例,女6例,发病中位年龄3.6岁(11个月~11岁),其中原发于颌面部14例,颈部1例,腹盆腔2例,肢体4例,生殖系统2例,肝脏1例;病理类型包括胚胎型22例,腺泡型2例;低危组3例,中危组18例,高危组3例。所有患儿均按横纹肌肉瘤全国协作组重庆方案(RMS-CQ-2009)进行化疗,并依据实际病情及疗效反应选择性采取根治手术及放疗。截止2020年6月1日共9例患儿死亡,3年总体生存率(OS)为65.7%。化疗+放疗组的3年OS(80.0%)高于化疗+二次根治术组(50.0%)(χ²=3.896,P=0.048)。对于颌面部RMS,行二次根治术的患儿3年0S(25.0%)低于未行二次根治术的患儿(80.0%)(χ²=6.521,P=0.011);行放疗的患儿3年OS(80.0%)高于未行放疗的患儿(25.0%)(χ²=4.168,P=0.041)。对于非颌面部RMS,行二次根治术和未行二次根治术的患儿3年OS分别是68.6%和66.7%(χ²=0.035,P=0.852),行放疗和未行放疗的患儿3年OS分别为53.3%和80.0%(χ²=0.076,P=0.783)。结论放疗有助于提高儿童RMS生存率,二次根治术并不能显著改善颌面部RMS的预后。     

Pelvic rhabdomyosarcoma in children

From 1961 to 1986, 29 patients with pelvic rhabdomyosarcoma (including pelvic retroperitoneal rhabdomyosarcoma) were admitted to the paediatric surgical service at the Red Cross Children's Hospital, Cape Town. During the period 1961–1970, the mainstay of treatment was radical surgery with or without subsquent chemotheraphy and radiotherapy. Of the 13 patients, only 2 have survived long-term (15%). From 1971 a new management policy of pre-treatment with chemotherapy and in some instances radiotherapy followed by conservative limited surgery was commenced. Twelve of the 16 patients have survived (75%). One patient with stage III prostatic tumour had treatment with only chemotherapy and radiotherapy and is surviving 8 years after diagnosis; another with a vaginal tumour, also stage III, whose parents refused surgery is alive tumour-free 4 years after similar treatment. Chemotherapy has not only significantly improved survival in pelvic rhabdomyosarcoma but has permitted limited surgery and in some instances has resulted in survival without surgical excision. Offprint requests to: S. Cywes     

Outcome after relapse in an unselected cohort of children and adolescents with Ewing sarcoma

BACKGROUND: Survival after relapse in patients with Ewing sarcoma is very poor and this retrospective study attempts to identify of prognostic factors predicting survival after relapse. PROCEDURE: A total of 191 patients with localised Ewing sarcoma were registered in the ET-2 trial of the United Kingdom Children's Cancer Study Group (UKCCSG). All patients received standardised primary treatment with chemotherapy and surgery and or radiotherapy as local modality treatment. Sixty-four patients who relapsed are included in this report. Treatment at relapse was variable and included chemotherapy, surgery, radiotherapy and high dose therapy (HDT) or megatherapy with peripheral stem cell transplantation (PBSCT) or autologous bone marrow transplantation (ABMT) in various combinations. A subgroup of patients had only non-specific symptomatic treatment at relapse. Both univariate and multivariate methods were used to investigate variables affecting survival after relapse. RESULTS: The overall actuarial median survival from relapse for all patients was 14 months (95% CI 11-16 months). Univariate analysis showed that males had a longer survival (median, 16 months vs. 11 months); patients who relapsed while on treatment did worse (median, 3 months vs. 16 months) and patients who had a longer disease-free interval (DFI) prior to relapse had a better outcome (DFI <1 year, median survival = 3 months; DFI 1-2 years, survival = 8 months; DFI > 2 years, median survival = 24 months, P < 0.001). Multivariate analysis confirmed that duration of first remission was the only factor associated with longer survival after relapse. CONCLUSIONS: These data suggest that although aggressive therapy may delay disease progression after relapse for some children, the course of the disease after relapse is usually fatal. International co-operative studies are needed to evaluate new strategies.     

Curability of recurrent disseminated disease after surgery alone for local-regional neuroblastoma using intensive chemotherapy and anti-G(D2) immunotherapy

PURPOSE: A reluctance to treat local-regional neuroblastoma by surgery alone derives partly from concern that if widespread neuroblastoma develops, the chance for cure is small, and partly from hope that mild chemotherapy will prevent relapse. The authors report on a series of patients who had distant recurrences after surgery alone for local-regional neuroblastoma. METHODS: Seven patients treated with surgery alone for local-regional neuroblastoma had widespread relapses 2.5 to 25 (median 7) months later and were treated at Memorial Sloan-Kettering Cancer Center (MSKCC). During the period of this study (1995-1999), MSKCC patients with high-risk neuroblastoma received the N7 protocol (dose-intensive chemotherapy, immunotherapy with the anti-G(D2) 3F8 antibody, targeted radiotherapy using 131I-3F8, local radiotherapy) if they had assessable disease, or 3F8 plus granulocyte-macrophage colony-stimulating factor (GM-CSF) followed by 13-cis-retinoic acid if they were in remission after treatment elsewhere. RESULTS: Five patients were in complete remission 3 years 11 months to 7 years 4 months from the start of retrieval therapy, including three who received all of their N7 treatment of relapsed neuroblastoma at MSKCC, one who received two cycles of chemotherapy elsewhere before starting N7, and one who was referred for 3F8/GM-CSF because of neuroblastoma cells in pretransplantation bone marrow. CONCLUSIONS: The encouraging survival results of our cohort are consistent with the concept that surgery alone for local-regional neuroblastoma might be beneficial to the overall neuroblastoma population because many patients will never need chemotherapy (and will therefore be spared its potential toxicities), and most of those who do have widespread relapses are likely to be cured with contemporary treatments.     

Preoperative chemotherapy of nephroblastoma. Preliminary results of the SIOP-9/GPO therapy study]

The therapeutic strategy of the SIOP-9 study includes pre-operative chemotherapy for all patients with nephroblastoma diagnosed by imaging methods aged between 0.5 and 16 years. By pre-operative chemotherapy the rate of radical resectable tumors should be increased and thereby the intensity of postoperative therapy, in particular of radiotherapy, diminished. Patients with nephroblastoma stage I-III were in case of tumorresponse randomised in either a 4 weeks or 8 weeks arm of pre-operative treatment with ACT D and VCR. The question was, if a prolongation of pre-operative chemotherapy could increase the relative part of stage I. Between 1/1/89 and 6/30/91 from 49 oncologic centres of former western Germany 188 patients were registered in the SIOP-9/GPO. From the stage I-III patients between 0.5 and 16 years 80.1% were pretreated with cytostatic agents. Only 53.9% of the patients with tumorresponse were randomised. The relative frequency of intraoperative ruptures was with 3% lower in the group of pretreated patients than in the primary operated (15.3%). The stage distribution for all Wilms' tumor patients showed a prevalence of stage I with 43.3% (after pre-operative treatment 59%; after primary operation 28%). Abdominal radiotherapy was performed in 22.4%. The event-free survival rate of all nephroblastoma lay at 85% 3 years after diagnosis (stage I standard 96%; unfavorable histology all stages 45%). 7.3% of the patients developed a hepatopathy under treatment and 7.8% even a VOD according to the criteria of McDonald.(ABSTRACT TRUNCATED AT 250 WORDS)     

Intensive cisplatin and cyclophosphamide-based chemotherapy without radiotherapy for intracranial germinomas: failure of a primary chemotherapy approach

PURPOSE: High rates of overall and event-free survival have been reported in patients with intracranial germinomas treated with craniospinal radiotherapy. More recently, similar results have been reported with chemotherapy combined with radiotherapy to more localized treatment volumes. Our interest in exploring chemotherapy without radiotherapy in patients with CNS germinomas was based on concerns about the late sequelae of radiotherapy to the brain or neuraxis and also the well documented success of chemotherapy alone in patients with disseminated extracranial germinomas. The primary objective of this study was to determine whether intensive cisplatin and cyclophosphamide-based combination chemotherapy, without radiotherapy, was effective in patients with CNS germinomas. PATIENTS AND METHODS: Nineteen patients were enrolled, ranging in age from 1 to 24 years (median, 14 years). Thirteen were male. Nine had diabetes insipidus. Therapy comprised two courses of Regimen 'A' (cisplatin, etoposide, cyclophosphamide, and bleomycin) followed by MRI evaluation. Patients achieving a complete remission (CR) completed all planned therapy with two courses of regimen 'B' (carboplatin, etoposide, and bleomycin). Patients achieving less than a CR received two courses of Regimen 'B' followed by another evaluation. Those in CR after four courses of treatment received one additional course of Regimen 'A' and Regimen 'B', while those not in CR after four treatment courses underwent second look surgery and/or radiation therapy. RESULTS: Eleven of 11 patients with residual postoperative disease assessable for response achieved a CR. With a median follow-up of 6.5 years, eight out of 19 (0.42) patients remain in CR 1 without radiotherapy and another three patients are in stable second or subsequent remissions. Three patients died from treatment-related toxicity and another died in CR 1 from an uncharacterized leukoencephalopathy. The 5-year event-free survival (EFS) was 0.47 +/- 0.23 and 5-year overall survival (OS) was 0.68 +/- 0.22. CONCLUSIONS: Intensive cisplatin and cyclophosphamide-based chemotherapy was effective in achieving remissions, however, the long-term outcome using this treatment program was unsatisfactory and associated with unacceptable morbidity and mortality, particularly in patients with diabetes insipidus.     

hCG-secreting pineal teratoma causing precocious puberty: report of two patients and review of the literature

Two boys are described with precocious puberty (PP) due to pineal immature teratoma associated with choriocarcinoma. Patient 1 was a 7 year-old boy with a 2-year history of PP. He had elevated CSF and plasma beta-hCG levels. Magnetic resonance imaging (MRI) showed a 3.0 cm pineal mass. He was initially submitted to a trial with radiotherapy, followed by radical surgical resection, stereotactic radiotherapy and chemotherapy. Long-term follow up included the appearance of acute hydrocephalus requiring CSF shunting, local hemorrhage and extensive radionecrosis. Death occurred 1.5 years after diagnosis. Patient 2 was a 7 year-old boy with an 8-month history of PP. He had elevated CSF and plasma beta-hCG and alpha-fetoprotein levels. MRI showed a 1.0 cm pineal mass. He was submitted to radical surgical resection (which caused normalization of levels of markers) and prophylactic chemotherapy. The boy is doing well 1.5 years after diagnosis. An extensive review of the literature corroborates the idea that this last treatment paradigm (surgery and chemotherapy) probably represents the best treatment regimen for these patients.     

Granulosa cell tumors of the ovary in children and adolescents. Multicenter retrospective study in 40 patients aged 7 months to 22 years]

BACKGROUND. Juvenile granulosa cell tumors (JGCT) of the ovary are rare in children. The over-all outcome after surgery is relatively good, but the indication and type of complementary treatment for severe forms are still unclear. POPULATION. A retrospective survey of the majority of patients with JGCT of the ovary admitted between 1965 and 1990 to 11 French oncologic pediatric centers was carried out. Medical records including surgical and histological data, were analyzed and each tumor was retrospectively classified by the same pathologist according to the Wollner classification. RESULTS. There were 40 patients aged 7 months to 22 years (mean: 6 years); 28 were less than 10 year old at diagnosis. Three had enchondromatosis (Ollier's disease). At diagnosis, all patients presented with an abdominal tumor, 23 had developed manifestations of precocious pseudopuberty, 2 had signs of virilization after a normal puberty and 2 had secondary amenorrhea. Surgery was always the primary treatment: unilateral ovariectomy in 35 cases, bilateral in 4 and biopsy alone in 1 case. There were 21 stage I, 1 stage II, 16 stage III and 2 stage IV cases. 13 patients received combined chemotherapy and 2 abdominal radiotherapy. 34 patients were alive and disease-free 10 months to 26 years after surgery and 6 died. All 23 patients with precocious pseudopuberty had a favorable outcome. CONCLUSIONS. This study confirms earlier reports. Unilateral ovariectomy is the first-choice therapy. There is no evidence that tumors complicated by rupture and hemoperitoneum require chemotherapy. Combined chemotherapy does not appear to improve the prognosis for the rare malignant forms. The factors of good prognosis are age less than 10 years and the presence of precocious pseudopuberty.     

Therapy and outcome of primitive neuroectodermal tumor of the jaw

Data pertaining to outcomes in jaw primitive neuroectodermal tumor (PNET) are lacking. Eleven cases of jaw PNET (five maxillary and six mandibular) were treated at our cancer center with the same chemotherapeutic agents from June 2003 to January 2009. Four patients underwent surgery after neoadjuvant chemotherapy and all received local radiotherapy. At median follow-up of 56 months (range: 19-77 months), 7/11 patients are in sustained remission. There was no difference in outcome with respect to site of tumor, and whether surgery was performed or not. These results support the use of chemoradiation as initial modality of treatment rather than going for extensive and mutilating surgery.     

Carboplatin and VP 16 in medulloblastoma: A phase II study of the French Society of Pediatric Oncology (sfop)

The purpose of this study is to evaluate the antitumor activity of combination carboplatin and etoposide in measurable medulloblastoma. From January '89 to January '92, 26 patients with medulloblastoma were included in a multicentric phase II study of 2 courses of carboplatin 160 mg/m²/d day 1 to day 5 and VP16 100 mg/m²/d day 1 to day 5. Median age was 10 years (19 months-14.5 years). First treatment was surgery alone in 1 patient, surgery + radiotherapy in 4 patients, surgery + chemotherapy in 2 patients less than 3 years old, surgery + radiotherapy + chemotherapy in 19 patients (“8 drugs in 1 day” based:17, SIOP I:1, SIOP II:1). Previous treatment included cisplatin (20 cases), carboplatin (1 case), and VP16 (7 cases). Measurable disease was evaluated by CT scan, MRI or myelogram and CSF. Response rate (RR) was 72 ± 10%:8 complete responses (CR), 10 partial responses (PR), 1 objective effect (OE), 6 progressive disease (PD), 1 non-evaluable. Thirty-six courses were evaluated for toxicity. Median duration of WHO grade 4 neutropenia was 8 days (0–23). One patient died at day 18 after the first course because of diffuse haemorrhage during septic aplasia. Five other non-life-threatening septicemias were recorded. Median number of platelet transfusions was 1 (0–4). One child who had achieved a PR after two courses died from CNS bleeding after the third course. This drug combination achieves a high response rate in childhood medulloblastoma. Severe toxicity has been mainly encountered in previously heavily treated patients. Tolerance may be acceptable in newly diagnosed children, but careful hematological follow-up and platelet transfusional support are definitely mandatory. © 1994 Wiley-Liss, Inc.     

Hungarian experience with Langerhans cell histiocytosis in childhood

The Langerhans cell histiocytosis (LCH) in children is relatively rare and the long-term analysis of therapy results has not been done yet in Hungary. The aim of this study was to investigate the incidence, clinical features, prognostic risk factors, and treatment results of children's LCH in Hungary in a 20-year period. Children less than 18 years of age with newly diagnosed LCH in Hungary were entered in this study. Clinical data of all children with LCH were reported to the National Childhood Cancer Registry in Hungary from 1981 to 2000. The clinical files were collected and abstracted for information regarding age at diagnosis, gender, disease characteristics, treatment, and outcome of treatment. Median follow-up duration of surviving patients is 10.98 years. Between January 1981 and December 2000, 111 children under 18 years of age were newly diagnosed with LCH in Hungary. The annual incidence of LCH in children younger than 18 years of age was 2.24/million children. The male-female ratio was 1.36:1; the mean age was 4 years 11 months. Thirty-eight children had localized disease and in 73 cases systemic dissemination was found already at the time of diagnosis. Twenty-two patients were treated only by local surgery, 7 by surgery with local irradiation, and 5 children got only local irradiation. In 2 cases remission was achieved with local steroid administration. Seventy-five patients received chemotherapy. In the 20 years of the study 14 children died, 9 due to the progression of the disease. Sixteen patients had relapse with a mean of 2.16 ± 1.29 years after the first diagnosis. Three patients with relapse got chemotherapy generally used in lymphoma and remission was achieved. The overall survival of all patients (n = 111) was 88.3 ± 3.1% at 5 years and 87.3 ± 3.2% at 10 and 20 years. Childhood LCH is a well-treatable disease and the survival rate is high. Even disseminated diseases have a quite good prognosis in childhood.     

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。     

MALE GONADAL FUNCTION IN SURVIVORS OF CHILDHOOD HODGKIN AND NON-HODGKIN LYMPHOMA

The aim of this study was to investigate the impact of therapy on long-term gonadal function of young people cured of childhood lymphomas and to assess whether a prepubertal state during the treatment protects the gonads from chemotherapy and/or radiotherapy late effects. Clinical evaluation, semen analysis, and endocrine status were studied in 20 survivors of childhood lymphomas. Five patients received Inverted Y radiotherapy, 2320 cGy (1550-4000); all 20 received chemotherapy as follows: MOPP/ABVD protocol, 9 patients; COMP protocol, 5 patients; MOPP protocol, 3 patients; other protocols, 3 patients. Semen analysis results were as follows: normal values, 4/20 patients; oligospermia, 8/20 patients; azoospermia, 8/20 patients; FSH above normal level, 10/20 patients; 4/5 who received Inverted Y irradiation were azoospermic and 1 was severely oligospermic. Treatment damage to the testis involves tubular germinal elements. Radiotherapy and chemotherapy combinations that included nitrogen mustard or cyclophosphamide were associated with high rates of oligospermia and azoospermia. MOPP/ABVD combination did not have a significant better outcome of sperm counts compared to MOPP alone. Age at chemotherapy did not correlate with the sperm count; hence a prepubertal state did not protect the gonad from the late effects of treatment.     

Male gonadal function in survivors of childhood Hodgkin and non-Hodgkin lymphoma

The aim of this study was to investigate the impact of therapy on long-term gonadal function of young people cured of childhood lymphomas and to assess whether a prepubertal state during the treatment protects the gonads from chemotherapy and/or radiotherapy late effects. Clinical evaluation, semen analysis, and endocrine status were studied in 20 survivors of childhood lymphomas. Five patients received Inverted Y radiotherapy, 2320 cGy (1550-4000); all 20 received chemotherapy as follows: MOPP/ABVD protocol, 9 patients; COMP protocol, 5 patients; MOPP protocol, 3 patients; other protocols, 3 patients. Semen analysis results were as follows: normal values, 4/20 patients; oligospermia, 8/20 patients; azoospermia, 8/20 patients; FSH above normal level, 10/20 patients; 4/5 who received Inverted Y irradiation were azoospermic and 1 was severely oligospermic. Treatment damage to the testis involves tubular germinal elements. Radiotherapy and chemotherapy combinations that included nitrogen mustard or cyclophosphamide were associated with high rates of oligospermia and azoospermia. MOPP/ABVD combination did not have a significant better outcome of sperm counts compared to MOPP alone. Age at chemotherapy did not correlate with the sperm count; hence a prepubertal state did not protect the gonad from the late effects of treatment.     

Multimodal therapy for the treatment of nonmetastatic Ewing sarcoma of pelvis

The purpose of this study was to evaluate the outcome of patients with Ewing sarcoma (ES) of the pelvis, attempting to identify prognostic factors to select patients for more aggressive treatment. Seventy-seven patients with nonmetastatic ES of the pelvis were treated at Rizzoli Institute between 1979 and 1996. Four different protocols of chemotherapy were used successively. Two protocols consisted of VACAc, and two of VACAc plus ifosfamide and etoposide. Local treatment consisted of surgery in 5 patients, radiotherapy in 60, and surgery followed by radiotherapy in 12. Mean follow-up was 11 years (range 5-25 years). Thirty-three patients remained continuously free of disease; 43 relapsed (24 due to metastases and 19 to local recurrence and metastases); 1 died of treatment-related complications. The 5- and 10-year event-free survival rates were 45% and 44%, respectively, and the 5- and 10-year overall survival rates were 48 and 44. These results are significantly worse than the ones achieved in 329 contemporary patients with extrapelvic lesions treated with the same protocols of chemotherapy (5- and 10-year event-free survival = 46% vs. 64% and 44% vs. 69%). Thus, despite associated chemotherapy, the outcome of ES localized in the pelvis remains poor, and new innovative methods for the treatment of this tumor are needed.     

Does chemotherapy have a role in low-grade astrocytoma management? a report of 13 cases

Surgery is the treatment of choice for low-grade astrocytoma while radiotherapy is carried out only when total resection is not possible. This study assessed the effectiveness of chemotherapy in nonresectable cases. Thirteen children with nonresectable astrocytoma were treated with carboplatin and etoposide and after four cycles the response to treatment was evaluated according to radiologic criteria. The results were: one with complete response (CR), three with minor response (MR), six with stable disease (SD), and three with progressive disease (PD). Moreover, in 77% there was an improvement in the neurologic picture. In particular, two cases with hypothalamic astrocytoma showed a regression of the diencephalic syndrome following chemotherapy. In six cases chemotherapy was carried out, at reduced dosage, after the first four cycles either because there was clinical improvement or because it was necessary to postpone radiotherapy in very young patients. After a follow-up period ranging between 11 and 63 months (average: 30 months), nine of the 13 patients are alive (69%) while four died of disease progression. Further studies would be useful to evaluate the role of chemotherapy in the management of low-grade astrocytoma. © 1995 Wiley-Liss, Inc.     

Chemotherapy in fibromatoses of childhood and adolescence: results from the Cooperative soft tissue sarcoma study (CWS) and the Italian Cooperative study group (ICG-AIEOP)]

BACKGROUND: Fibromatoses are a group of semimalignant tumors which grow infiltratively without metastases. If radical surgery is not at all possible or only by mutilation the question of neoadjuvant concepts arises. PATIENTS: We report on therapy and outcome of 36 patients registered to the German CWS study group and the Italian ICG study group for soft tissue sarcoma who were between 0 and 23 years of age. 15 had a histology of infantile (myo-) fibromatosis (9 unifocal, 6 multifocal). 15 patients had desmoid-like, aggressive fibromatosis. METHODS: We performed a retrospective analysis of the patients registered to the CWS and ICG study group. For literature review a medline search was performed. RESULTS: 23 patients received chemotherapy with anthracyclines (VAIA) or without (VAC) or monotherapy with alkylating agents. In 19 cases response to chemotherapy was measurable. In 9 patients tumors responded to chemotherapy. In some cases response was evident only after 20 weeks of treatment. In the group of non responders treatment was stopped early (after 4 weeks) in some cases. Relapses occurred mainly in the group of desmoid-like, aggressive fibromatosis (7 out of 15). In the group of infantile (myo-) fibromatosis only one patient suffered a relapse (1 out of 9). The literature review showed that mainly ADR/DTIC, VAC and MTX/VBL was used for inoperable fibromatoses. Concerning the response rates there where no major differences. Concerning toxicity the regimen with methotrexate and vinblastine seems superior to the others. CONCLUSIONS: As a chemotherapy regimen of first choice vinblastine with low dose methotrexate can be recommended. The therapy should not be stopped before the 20th week of treatment. The histological subgroup can provide good information on the relapse rate and aggressivity to be expected.     

Diffuse intraabdominal desmoplastic small round cell tumor: a ten-year experience.

BACKGROUND: Intraabdominal desmoplastic small round cell tumors (IDSRCT) are rare in children and predominantly affect male adolescents and young adults. We present our experience in the management of five children with diffuse IDSRCT, managed with aggressive chemotherapy, surgery, radiotherapy and peripheral blood stem cell transplantation. MATERIAL AND METHODS: During the last decade five patients, four males and one female (mean age 9.6 years), with diffuse IDSRCT were managed in our department. The main symptoms were abdominal distention, vague abdominal pain, and vomiting. Three patients with inoperable tumor on admission were submitted initially to open biopsy followed by aggressive chemotherapy. Regression of the tumor was followed by a second laparotomy and radical excision of any macroscopically distinguishable masses, followed by chemotherapy. In the remaining two patients a debulking procedure was done initially, followed by chemotherapy. The accurate diagnosis of the disease was established by immunohistochemistry, additionally confirmed in the last two patients by molecular analysis. RESULTS: Three patients who had radical excision of the tumor and adjuvant chemotherapy had recurrence after two to six months. In the remaining two patients, recurrence was evident after two and eighteen months, respectively, following debulking. In addition, one patient with recurrence received radiotherapy and two others underwent peripheral blood stem cell transplantation. All but one patient died within three years from diagnosis. The last patient, who was submitted to a debulking procedure, is still alive eight months after the operation. CONCLUSIONS: Intrabdominal desmoplastic small round cell tumor is a highly aggressive malignancy with a very poor prognosis. Multiagent chemotherapy usually leads initially to a temporary regression of the tumor, but recurrence is the rule. Radical surgical excision, radiotherapy and peripheral blood stem cell transplantation does not seem to improve prognosis significantly. Despite all therapeutic modalities the outcome is dismal and surgical efforts can be considered only as palliative.     

Malignant sacrococcygeal teratoma: improved survival with chemotherapy

Between 1954 and 1985, 15 cases of malignant sacrococcygeal teratoma were treated in our institution. Median age at presentation was 20 months (range 9–46 months); there were 14 females and 1 male. Six cases presenting prior to 1970 were treated without chemotherapy with a median survival of 2 months (range 1–7 months). Five patients treated from 1971 to 1979 had tumour excision plus vincristine, actinomycin D, and cyclophosphamide (VAC). Three received radiotherapy in addition and median survival was 11 months (range 0–31 months). Four patients presenting since 1980 had chemotherapy alone after tumour excision. 1 receiving VAC and adriamycin and the remaining patients cisplatinum, bleomycin, and VP16. All these are alive and disease free (median follow-up 27 months; range 15–96 months). It is concluded that initial surgery aimed at complete excision followed by aggressive chemotherapy gives good results. Offprint requests to: D. C. S. Gough