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1.
Surg Oncol ; 29: 107-112, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-31196472

RESUMO

BACKGROUND: Despite being associated with a very poor prognosis, long-term survivors across all series of Desmoplastic Small Round Cell Tumor (DSRCT) have been reported. AIM OF THE STUDY: To analyze patients 'characteristics associated with a prolonged survival after DSRCT diagnosis. METHODS: All consecutive patients treated for DSRCT in nine French expert centers between 1991 and 2018 were retrospectively analyzed. Patients with a follow-up of less than 2 years were excluded and cure defined as being disease-free at least 5 years. RESULTS: 100 pts were identified (median age 25 years, 89% male). 27 had distant metastases at diagnosis and 80 pts underwent upfront chemotherapy (CT). 71 pts were operated, 20 pts without prior CT). Surgery was macroscopically complete (CC0/1) in 50 pts. Hyperthermic intraperitoneal Chemotherapy (HIPEC) was administered during surgery in 15 pts 54 pts had postoperative CT and 26 pts had postoperative whole abdomino-pelvic RT (WAP-RT). After a median follow-up of 103 months (range 23-311), the median overall survival (OS) was 25 months. The 1- year, 3-year and 5-year OS rates were 90%, 35% and 4% respectively. 5 patients were considered cured after a median disease-free interval of 100 months (range 22-139). Predictive factors of cure were female sex (HR = 0.49, p = 0.014), median PCI<12 (HR = 0.32, p = 0.0004), MD Anderson stage I (HR = 0.25, p < 0.0001), CC0/1 (HR = 0.34, p < 0.0001), and WAP-RT (HR = 0.36, p = 0.00013). HIPEC did not statistically improve survival. CONCLUSION: Cure in DSRCT is possible in 5% of patients and is best achieved combining systemic chemotherapy, complete cytoreductive surgery and WAP-RT. Despite aggressive treatment, recurrence is common and targeted therapies are urgently needed.


Assuntos
Quimioterapia do Câncer por Perfusão Regional/mortalidade , Procedimentos Cirúrgicos de Citorredução/mortalidade , Tumor Desmoplásico de Pequenas Células Redondas/mortalidade , Hipertermia Induzida/mortalidade , Neoplasias Peritoneais/mortalidade , Adolescente , Adulto , Criança , Pré-Escolar , Terapia Combinada , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Tumor Desmoplásico de Pequenas Células Redondas/terapia , Feminino , Seguimentos , Humanos , Masculino , Pessoa de Meia-Idade , Seleção de Pacientes , Neoplasias Peritoneais/patologia , Neoplasias Peritoneais/terapia , Prognóstico , Estudos Retrospectivos , Taxa de Sobrevida , Adulto Jovem
2.
Diagn Pathol ; 14(1): 43, 2019 May 18.
Artigo em Inglês | MEDLINE | ID: mdl-31103034

RESUMO

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is a rare soft tissue tumor that generally involves the retroperitoneum, pelvis, omentum and mesentery in younger patients. However, extra-abdominal DSRCT is very rare. CASE PRESENTATION: A 49-year-old Japanese man noticed a mass in the right parotid gland. Ultrasound examination revealed a solid tumor about 2 cm in diameter. Computed tomography (CT) of the whole body revealed no other tumors or lymph node swelling. Superficial parotidectomy was performed. Histologically, the tumor was composed of various-sized tumor cell nests in an abundant fibromyxoid and collagenous background. The tumor cells were small to medium-sized. Immunohistochemistry showed that the tumor cells were immunoreactive for epithelial markers and desmin. They also showed strong nuclear staining with a Wilms tumor 1 (WT1) antibody detecting the C-terminal region (C-WT1), but not the N-terminal region (N-WT1). We also performed 3'/5' expression imbalance assay based on reverse transcription polymerase chain reaction (RT-PCR) to determine whether aberrant WT1 gene expression was present. This tumor was found to lack 5'-regional expression of the WT1 gene, as well as immunoreactivity with the N-WT1 antibody. Finally, fluorescence in situ hybridization (FISH) and RT-PCR analyses revealed the presence of a gene showing fusion between exon 7 of EWSR1 and exon 8 of WT1. The tumor was diagnosed as a DSRCT of the right parotid gland. The patient has been followed for 3 years without recurrence or metastasis. CONCLUSIONS: Although DSRCT in the salivary gland is extremely rare, it should be included in the differential diagnosis of poorly differentiated salivary gland neoplasms, especially with a fibromyxoid background. Pathologists should bear in mind that DSRCT may occur in major salivary glands and should perform immunohistochemistry with appropriate antibodies, not only those against keratin and desmin, but also one detecting the C-terminal region of WT-1. Furthermore, molecular detection of EWSR1-WT1 fusion gene conclusively confirmed the diagnosis of DSRCT in this uncommon location.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/diagnóstico por imagem , Neoplasias Parotídeas/diagnóstico por imagem , Proteína EWS de Ligação a RNA/genética , Proteínas WT1/genética , Tumor Desmoplásico de Pequenas Células Redondas/genética , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Diagnóstico Diferencial , Humanos , Imuno-Histoquímica , Masculino , Pessoa de Meia-Idade , Proteínas de Fusão Oncogênica , Glândula Parótida/diagnóstico por imagem , Glândula Parótida/patologia , Neoplasias Parotídeas/genética , Neoplasias Parotídeas/patologia
3.
J Med Case Rep ; 13(1): 77, 2019 Mar 10.
Artigo em Inglês | MEDLINE | ID: mdl-30851735

RESUMO

INTRODUCTION: Desmoplastic small round cell tumor is an extremely rare and aggressive cancer that affects mainly adolescents and young adults. Despite multiple therapeutic strategies, most patients have resistant disease with very poor survival rates. CASE PRESENTATION: We present a case of a 10-year-old Caucasian boy with a desmoplastic small round cell tumor refractory to conventional treatment who exhibited a good response to alternative treatment. With use of irinotecan and vincristine in association with radiation therapy, a reduction of 96.9% of the dimensions of the target lesions compared with the initial image was observed. CONCLUSION: This chemotherapy regimen, in association with radiation therapy, demonstrated efficacy for refractory desmoplastic small round cell tumor in our patient, and it is cost-effective.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/tratamento farmacológico , Irinotecano/uso terapêutico , Neoplasias Gástricas/tratamento farmacológico , Vincristina/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica , Criança , Análise Custo-Benefício , Tumor Desmoplásico de Pequenas Células Redondas/diagnóstico por imagem , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Países em Desenvolvimento , Humanos , Masculino , Radiografia Abdominal , Neoplasias Gástricas/diagnóstico por imagem , Neoplasias Gástricas/patologia , Resultado do Tratamento
4.
J Surg Oncol ; 119(7): 880-886, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-30844086

RESUMO

BACKGROUND: Because of the rarity of desmoplastic small round cell tumors (DSRCT), there is a lack of data describing patterns of care and survival for these patients. Using a national tumor registry, the current study sought to describe patterns of care and clinical outcomes for patients with DSCRT. METHODS: Data from the National Cancer Database were used to identify 491 patients aged 18 years or older diagnosed with DSRCT between 2004 and 2014. Multivariable Cox proportional hazards regression analysis was used to identify factors associated with overall survival (OS). RESULTS: Among all patients, 41.2% (n = 200), underwent surgical resection of their primary tumor, chemotherapy was administered to 86.5% (n = 415) of patients, while radiation therapy was administered to 13.0% (n = 63) of patients. Over the study, 69.7% of patients died with a median OS of 25.9 months (interquartile range [IQR]: 22.7-27.5); 1-, 3-, and 5-year OS were 78.6%, 32.3%, and 18.4%, respectively. On multivariable analysis, stage IV disease (Hazard Ratio [HR] = 2.12, 95% CI: 1.41-3.18), receipt of surgery (HR = 0.68, 95% CI: 0.50-0.91), chemotherapy (HR = 0.52, 95% CI: 0.35-0.78), or radiation therapy (HR = 0.55, 95% CI: 0.33-0.92) were independently associated with OS. CONCLUSIONS: Although receipt of multimodality treatment may lead to improved survival, further research and clinical trials are required to establish best practices for the care of DSRCT.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/terapia , Oncologia/estatística & dados numéricos , Adulto , Terapia Combinada/estatística & dados numéricos , Tumor Desmoplásico de Pequenas Células Redondas/mortalidade , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Feminino , Humanos , Estimativa de Kaplan-Meier , Masculino , Oncologia/métodos , Estadiamento de Neoplasias , Estudos Retrospectivos , Estados Unidos/epidemiologia , Adulto Jovem
5.
AJR Am J Roentgenol ; 212(3): W45-W54, 2019 03.
Artigo em Inglês | MEDLINE | ID: mdl-30673334

RESUMO

OBJECTIVE: The purpose of this study is to evaluate the clinical, pathologic, and multimodality cross-sectional imaging features of a cohort of 94 patients with desmoplastic small round cell tumor (DSRCT). MATERIALS AND METHODS: This retrospective study of 94 patients with pathologically verified DSRCT was conducted at a tertiary cancer center between 2001 and 2013. Epidemiologic, clinical, pathologic, and imaging findings were recorded. Tumor size, location, and shape and the distribution pattern of metastases at presentation were analyzed. RESULTS: DSRCT most often occurred in young patients (median age, 21.5 years; range, 5-53 years), showing a marked predominance in male patients (86 male patients vs eight female patients). Eighty nine-patients (95%) were white (defined in this study as white or Hispanic), four were African American, and one was of Asian descent. Most patients had symptoms, with abdominal pain noted as the most common symptom. At initial presentation, 85 patients (90%) had multifocal disease, nodular disease, diffuse omental and peritoneal disease, or a combination of these conditions. Thirty-eight patients (40%) had diaphragmatic involvement. Thirty-two patients (34%) had liver metastases, and 49 patients (52%) had retroperitoneal involvement in the form of implants, tumoral extension, or nodal involvement. With regard to thoracic findings, 33 patients (35%) had nodal disease, 17 (18%) had pleural effusions, and only two (2%) had lung metastases at presentation. Twelve patients (13%) had calcified lesions. CONCLUSION: DSRCT is a rare, multifocal peritoneal malignancy with frequently disseminated abdominal disease at presentation. In the abdomen, disease most commonly involves the omentum and peritoneum, followed by the retroperitoneum. The liver is the most common solid visceral metastatic site. A substantial number of patients have diaphragmatic involvement. In the thorax, nodal and pleural involvement is more common than lung involvement.


Assuntos
Neoplasias Abdominais/diagnóstico por imagem , Tumor Desmoplásico de Pequenas Células Redondas/diagnóstico por imagem , Neoplasias Abdominais/patologia , Neoplasias Abdominais/cirurgia , Adolescente , Adulto , Criança , Pré-Escolar , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Tumor Desmoplásico de Pequenas Células Redondas/cirurgia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Fatores Sexuais
6.
Ann Surg Oncol ; 26(1): 131-138, 2019 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-30353396

RESUMO

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive sarcoma. Cytoreductive surgery (CRS) and hyperthermic intraperitoneal chemotherapy (HIPEC) may improve survival. METHODS: A retrospective review of anesthetic management and postoperative pain control strategies after CRS/HIPEC for DSRCT from 2013 to 2017 was performed. RESULTS: The review analyzed 10 CRS/HIPEC procedures performed for nine DSRCT patients with a median age of 19 years (range 10-24 years). Six of these patients were Caucasian, and seven were men. The median operative duration was 551 min (range 510-725 min), and the median anesthesia duration was 621 min (range 480-820 min). Postoperative mechanical ventilation was necessary in 5 patients for a median duration of 1 day (range 0-2 days). The median intraoperative intravenous fluid administration was 13 ml/kg/h (range 6.3-24.4 ml/kg/h), and the colloid administration was 12 ml/kg (range 0.0-53.0 ml/kg). The median blood loss was 15 ml/kg (range 6.3-77.2 ml/kg). Nine patients received intraoperative transfusion with a median red blood cell transfusion volume of 14 ml/kg (range 10.1-58.5 ml/kg). The median intraoperative urine output was 2 ml/kg/h (range 0.09-8.40 ml/kg/h), and half of the patients received intraoperative diuretics. Cisplatin was used during HIPEC for eight surgeries. Acute kidney injury was observed in two patients, one of whom required short-term dialysis. Epidural infusions were used in eight cases for a median of 4 days (range 3-5 days). Postoperative intravenous opioid use (morphine equivalent) was 0.67 mg/kg/day (range 0.1-9.2 mg/kg/day) administered for a median of 11 days (range 2-35 days). CONCLUSION: Cytoreduction and HIPEC for DSRCT are associated with significant perioperative fluid requirements and potentially challenging pain management. Renal protective strategies should be considered for reduction of cisplatin-associated nephrotoxicity. Further investigation for a more effective, less systemically toxic HIPEC agent is warranted.


Assuntos
Anestésicos/uso terapêutico , Quimioterapia do Câncer por Perfusão Regional/efeitos adversos , Procedimentos Cirúrgicos de Citorredução/efeitos adversos , Tumor Desmoplásico de Pequenas Células Redondas/terapia , Hipertermia Induzida/efeitos adversos , Manejo da Dor , Dor/tratamento farmacológico , Adolescente , Adulto , Criança , Terapia Combinada , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Feminino , Seguimentos , Humanos , Masculino , Dor/etiologia , Neoplasias Peritoneais/patologia , Neoplasias Peritoneais/terapia , Prognóstico , Estudos Retrospectivos , Adulto Jovem
7.
Int J Surg Pathol ; 27(3): 236-243, 2019 May.
Artigo em Inglês | MEDLINE | ID: mdl-30522375

RESUMO

OBJECTIVES: Desmoplastic small round cell tumor (DSRCT) is an aggressive round cell sarcoma that arises in the abdominal cavity/pelvis of young males. We sought to expand its clinicopathologic spectrum. METHODS: Cases of DSRCT presenting in patients >30 years of age or tumors arising outside of the abdominal cavity/pelvis were retrieved. RESULTS: Thirty-four cases were identified. Sixteen tumors arose at atypical sites (head/neck, intracranial, thigh, axilla/shoulder, inguinal/paratesticular, intraosseous, and uterine corpus). The remaining 18 patients were older than 30 years, and their tumors involved the abdomen or pelvis. The majority of cases showed areas with classic histology, while 6 cases exhibited solid growth and 5 showed macronodular architecture. Cytologic appearance included round cell, rhabdoid, epithelioid, and small cell. CONCLUSION: DSRCT may arise at nonabdominal locations in both pediatric and adult populations, as well as intra-abdominally in older adults, and these tumors exhibit high rates of metastasis and morbidity.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/patologia , Adolescente , Adulto , Criança , Pré-Escolar , Estudos de Coortes , Tumor Desmoplásico de Pequenas Células Redondas/genética , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Proteínas de Fusão Oncogênica/genética , Proteína EWS de Ligação a RNA/genética , Fatores Sexuais , Proteínas WT1/genética , Adulto Jovem
10.
Cancer Commun (Lond) ; 38(1): 70, 2018 11 28.
Artigo em Inglês | MEDLINE | ID: mdl-30486883

RESUMO

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive, and poorly investigated simple sarcoma with a low frequency of genetic deregulation other than an Ewing sarcoma RNA binding protein 1 (EWSR1)-Wilm's tumor suppressor (WT1) translocation. We used whole-exome sequencing to interrogate six consecutive pre-treated DSRCTs whose gene expression was previously investigated. METHODS: DNA libraries were prepared from formalin-fixed, paraffin-embedded archival tissue specimens following the Agilent SureSelectXT2 target enrichment protocol and sequenced on Illumina NextSeq 500. Raw sequence data were aligned to the reference genome with Burrows-Wheeler Aligner algorithm. Somatic mutations and copy number alterations (CNAs) were identified using MuTect2 and EXCAVATOR2, respectively. Biological functions associated with altered genes were investigated through Ingenuity Pathway Analysis (IPA) software. RESULTS: A total of 137 unique somatic mutations were identified: 133 mutated genes were case-specific, and 2 were mutated in two cases but in different positions. Among the 135 mutated genes, 27% were related to two biological categories: DNA damage-response (DDR) network that was also identified through IPA and mesenchymal-epithelial reverse transition (MErT)/epithelial-mesenchymal transition (EMT) already demonstrated to be relevant in DSRCT. The mutated genes in the DDR network were involved in various steps of transcription and particularly affected pre-mRNA. Half of these genes encoded RNA-binding proteins or DNA/RNA-binding proteins, which were recently recognized as a new class of DDR players. CNAs in genes/gene families, involved in MErT/EMT and DDR, were recurrent across patients and mostly segregated in the MErT/EMT category. In addition, recurrent gains of regions in chromosome 1 involving many MErT/EMT gene families and loss of one arm or the entire chromosome 6 affecting relevant immune-regulatory genes were recorded. CONCLUSIONS: The emerging picture is an extreme inter-tumor heterogeneity, characterized by the concurrent deregulation of the DDR and MErT/EMT dynamic and plastic programs that could favour genomic instability and explain the refractory DSRCT profile.


Assuntos
Dano ao DNA/genética , Tumor Desmoplásico de Pequenas Células Redondas/genética , Transição Epitelial-Mesenquimal/genética , Genômica/métodos , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Feminino , Humanos , Masculino
11.
J Pediatr Endocrinol Metab ; 31(8): 947-950, 2018 Aug 28.
Artigo em Inglês | MEDLINE | ID: mdl-30044763

RESUMO

Background Tumor-induced hypoglycemia is a rare and serious complication that is usually a consequence of either excessive insulin secretion (insulinoma) or because of non-islet cell tumor hypoglycemia (NICTH). NICTH is a rare phenomenon seen most often in adult patients. It is associated with different tumor types. Here, we report the first case to the best of our knowledge in the literature of a pediatric patient with NICTH associated with desmoplastic small round cell tumor (DSRT). Case presentation This is a 15-year-old girl who presented with symptomatic hypoglycemia and abdominal mass. She required an intravenous glucose infusion rate as high as 9 mg/kg/min in addition to glucose containing oral supplements in order to maintain her blood glucose above 60 mg/dL. Computed tomography (CT) scan of the chest, abdomen and pelvis showed multiple hepatic lesions with an intraperitoneal soft tissue mass which subsequently was diagnosed as DSRT. When the blood glucose was 45 mg/dL, the insulin, growth hormone (GH) and insulin-like growth factor-1 (IGF-1) levels were suppressed with an appropriate elevation of cortisol. Subsequently, an insulin-like growth factor-2 (IGF-2) level was sent and the IGF-2:IGF-1 ratio was found to be elevated >10 consistent with NICTH. After the first dose of chemotherapy, hypoglycemia improved, and she was weaned off glucose containing fluids. Conclusions NICTH should be considered in all cancer patients regardless of their age with refractory hypoglycemia.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/patologia , Resistência a Medicamentos , Hipoglicemia/patologia , Adolescente , Tumor Desmoplásico de Pequenas Células Redondas/complicações , Tumor Desmoplásico de Pequenas Células Redondas/tratamento farmacológico , Feminino , Humanos , Hipoglicemia/complicações , Hipoglicemia/tratamento farmacológico , Prognóstico
12.
Pediatr Blood Cancer ; 65(11): e27313, 2018 11.
Artigo em Inglês | MEDLINE | ID: mdl-30015384

RESUMO

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) and synovial sarcoma are rare tumors with dismal outcomes requiring new therapeutic strategies. Immunotherapies have shown promise in several cancer types, but have not been evaluated in DSRCT and synovial sarcoma. Because the immune microenvironment can provide indications of the inflammatory nature of tumors, immunohistochemical staining is able to assess the tumor immune infiltrates in both tumor types. PROCEDURE: Using tissue microarrays of DSRCT and synovial sarcoma tumor samples, we detected tumoral HLA-A/B/C, beta-2-microglobulin(B2M), and PD-L1 expression, and quantified tumor-infiltrating lymphocytes expressing CD4, CD8, CD56, CD45RO, or FOXP3 by immunohistochemistry. We used staining intensity on a scale of 0-3 and percentage of tumor stained to determine HLA, B2M, and PD-L1 scores. We calculated the cytotoxic T lymphocyte (CTL) target score as HLA score × B2M score/100. RESULTS: In diagnostic samples, we found high HLA and CTL target scores and low PD-L1 expression with decreased scores in recurrence for both tumor types. We found an increase in CD56+ natural killer cells in DSRCT samples from diagnosis to recurrence. CONCLUSIONS: We found similar immunostimulatory profiles in DSRCT and synovial sarcoma. Our findings suggest that DSRCT and synovial sarcoma may be amenable to immunotherapies, albeit there was significant heterogeneity. Interestingly, HLA and CTL target scores decreased at recurrence, possibly reflecting immunoevasion. Our findings suggest both tumor types may be amendable to CTL-based therapies at diagnosis but less so at relapse. Our results support further investigation into the prognostic and predictive value of these findings in a larger dataset.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/imunologia , Recidiva Local de Neoplasia/imunologia , Sarcoma Sinovial/imunologia , Neoplasias de Tecidos Moles/imunologia , Biomarcadores Tumorais/imunologia , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Antígenos HLA/imunologia , Humanos , Imunofenotipagem , Imunoterapia , Linfócitos do Interstício Tumoral/imunologia , Recidiva Local de Neoplasia/patologia , Sarcoma Sinovial/patologia , Neoplasias de Tecidos Moles/patologia , Linfócitos T Citotóxicos/imunologia , Microambiente Tumoral/imunologia
13.
Clin Cancer Res ; 24(19): 4865-4873, 2018 10 01.
Artigo em Inglês | MEDLINE | ID: mdl-29871905

RESUMO

Purpose: Desmoplastic small round cell tumor (DSRCT), which harbors EWSR1-WT1 t(11;22)(p13:q12) chromosomal translocation, is an aggressive malignancy that typically presents as intra-abdominal sarcomatosis in young males. Given its rarity, optimal treatment has not been defined.Experimental Design: We conducted a retrospective study of 187 patients with DSRCT treated at MD Anderson Cancer Center over 2 decades. Univariate and multivariate regression analyses were performed. We determined whether chemotherapy, complete cytoreductive surgery (CCS), hyperthermic intraperitoneal cisplatin (HIPEC), and/or whole abdominal radiation (WART) improve overall survival (OS) in patients with DSRCT. Critically, because our institutional practice limits HIPEC and WART to patients with less extensive, potentially resectable disease that had benefited from neoadjuvant chemotherapy, a time-variant analysis was performed to evaluate those adjunct treatment modalities.Results: The pre-2003 5-year OS rate of 5% has substantially improved to 25% with the advent of newer chemotherapies and better surgical and radiotherapy techniques (HR, 0.47; 95% CI, 0.29-0.75). Chemotherapy response (log rank P = 0.004) and CCS (log rank P < 0.0001) were associated with improved survival. Although WART and HIPEC lacked statistical significance, our study was not powered to detect their potential impact upon OS.Conclusions: Improved 3- and 5-year OS were observed following multidisciplinary treatment that includes Ewing sarcoma (ES)-based chemotherapy and complete tumor cytoreductive surgery, but few if any patients are cured. Prospective randomized studies will be required to prove whether HIPEC or WART are important. In the meantime, chemotherapy and CCS remain the cornerstone of treatment and provide a solid foundation to evaluate new biologically targeted therapies. Clin Cancer Res; 24(19); 4865-73. ©2018 AACR.


Assuntos
Procedimentos Cirúrgicos de Citorredução , Tumor Desmoplásico de Pequenas Células Redondas/tratamento farmacológico , Sarcoma de Ewing/tratamento farmacológico , Adulto , Terapia Combinada , Tumor Desmoplásico de Pequenas Células Redondas/genética , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Tumor Desmoplásico de Pequenas Células Redondas/cirurgia , Intervalo Livre de Doença , Feminino , Humanos , Masculino , Proteína EWS de Ligação a RNA/genética , Sarcoma de Ewing/genética , Sarcoma de Ewing/patologia , Sarcoma de Ewing/cirurgia , Adulto Jovem
14.
J Surg Oncol ; 117(8): 1759-1767, 2018 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-29878371

RESUMO

BACKGROUND AND OBJECTIVES: Desmoplastic small round cell tumor (DSRCT) is a rare peritoneal surface malignancy. Current research is limited by the scarcity of this disease. METHODS: Patients with DSRCT were identified in the 2004-2014 NCDB. Factors affecting overall survival (OS) were assessed. Additionally, trends were examined based on the volume of cases treated at individual facilities. RESULTS: A total of 125 patients were identified with a median age of 21 (IQR 15-27). Six had extra-abdominal disease and 15 (12%) had liver involvement. Median OS was 28 months. Systemic chemotherapy (HR 0.4, P = 0.015) and surgery (HR 0.6, P = 0.047) were associated with reduced mortality. For the 74 patients undergoing surgery, absence of liver involvement and receipt of postoperative chemotherapy were associated with improved OS on univariate analysis. On multivariable analysis, two factors approached significance: adjuvant chemotherapy was associated with a reduced risk of mortality (HR 0.3, P = 0.073) and residual macroscopic disease after resection correlated with increased risk of mortality (HR 5.3, P = 0.071). High-volume facilities (≥5 cases) experienced improved OS (median 59.1 vs 28.8 months), albeit not significantly (P = 0.135), compared to low-volume centers. CONCLUSION: Despite multimodal treatment, DSRCT is associated with dismal outcomes. Facilities familiar with treating this uncommon disease may experience superior outcomes.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/mortalidade , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Neoplasias Peritoneais/mortalidade , Neoplasias Peritoneais/patologia , Adulto , Neoplasias Ósseas/secundário , Quimioterapia Adjuvante , Bases de Dados Factuais , Tumor Desmoplásico de Pequenas Células Redondas/terapia , Feminino , Humanos , Neoplasias Hepáticas/mortalidade , Neoplasias Hepáticas/patologia , Neoplasias Hepáticas/terapia , Neoplasias Pulmonares/secundário , Masculino , Neoplasia Residual , Neoplasias Peritoneais/terapia , Radioterapia Adjuvante , Doenças Raras , Estados Unidos/epidemiologia , Adulto Jovem
15.
Medicine (Baltimore) ; 97(17): e0494, 2018 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-29703011

RESUMO

RATIONALE: Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive and malignant tumor. This report describes a case involving DSRCT of the middle ear which no case has been reported in the literature till date. PATIENT CONCERN: A 59-year-old Chinese man with a 40-year history of repeated suppuration of his right ear and 1-year history of drooping of the angle of mouth. The CT of the middle ear and brain scan and enhanced MRI showed space occupying lesion in the right middle ear. DIAGNOSES: Desmoplastic small round cell tumor of the middle ear. INTERVENTIONS: After relevant examinations, radical mastoidectomy and subtotal temporal bone resection were performed on the right ear under general anesthesia. The patient underwent postoperative adjuvant chemoradiation therapy. OUTCOMES: The patient was counterchecked regularly,there was norecurrence of DSRCT of the middle ear. Four years after surgery, the CT and MRI of the middle ear mastoid showed right middle ear soft tissue shadow,but postoperative pathological results showed proliferative fibrous and vascular tissues with chronic inflammatory cell infiltration and necrosis. LESSONS: DSRCT is a relatively aggressive, malignant mesenchymal tumor, with a very poor prognosis.The diagnosis of DSRCT relies on immunohistological data. Early diagnosis, radical surgery, chemotherapy, and radiotherapy are considered a reasonable way to prolong survival.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/diagnóstico , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Tumor Desmoplásico de Pequenas Células Redondas/terapia , Neoplasias da Orelha , Orelha Média , Humanos , Masculino , Pessoa de Meia-Idade , Prognóstico
16.
Bull Cancer ; 105(5): 523-536, 2018 May.
Artigo em Francês | MEDLINE | ID: mdl-29576221

RESUMO

Desmoplastic small round cell tumor (DSRCT) is a rare sarcoma that typically affects pediatric and young adult patients with a median age in the general and in the pediatric population of 24.6 years (range 4-58 years) and 15.0 years (range 0-21 years) respectively, with a strong male predominance. This tumor is characterized by a specific t(11;22)(p13;q12) that results in fusion of EWS and WT1 genes which can be demonstrated by RT-PCR or by FISH. DSRCT most frequently presents as an intra-abdominal primary mass associated with peritoneal seeding and a highly aggressive pattern of spread. Generally, all tumors showed the typical histologic findings of variably sized clusters of poly-phenotypic small, round, or spindled cells lying in a desmoplastic stroma. Treatment of this malignancy remains a challenge. The combination of polychemotherapy regimens and aggressive surgery followed by whole abdomen radiation therapy represents nowadays a classical protocol for DSRCT. The survival rate of DSRCT patients is still disappointing around 20 %. However, the survival of patients who had complete resection of the tumor appears better. Hopes are turning to targeted therapeutics against this simple genomic sarcoma. Authors summarize medical knowledge of this rare tumor.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas , Doenças Raras , Adolescente , Criança , Pré-Escolar , Cromossomos Humanos Par 11 , Cromossomos Humanos Par 22 , Tumor Desmoplásico de Pequenas Células Redondas/genética , Tumor Desmoplásico de Pequenas Células Redondas/mortalidade , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Tumor Desmoplásico de Pequenas Células Redondas/terapia , Feminino , Humanos , Masculino , Proteínas de Fusão Oncogênica/genética , Prognóstico , Doenças Raras/genética , Doenças Raras/mortalidade , Doenças Raras/patologia , Doenças Raras/terapia , Translocação Genética , Adulto Jovem
17.
Ann Surg Oncol ; 25(4): 872-877, 2018 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-29383611

RESUMO

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is a rare sarcoma that primarily affects adolescents and young adults. Patients can present with many peritoneal implants. We conducted a phase 2 clinical trial utilizing cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (CRS-HIPEC) with cisplatin for DSRCT and pediatric-type abdominal sarcomas. PATIENTS AND METHODS: A prospective cohort study was performed on 20 patients, who underwent CRS-HIPEC procedures, with cisplatin from 2012 to 2013. All patients were enrolled in the phase 2 clinical trial. Patients with extraabdominal disease and in whom complete cytoreduction (CCR0-1) could not be achieved were excluded. All outcomes were recorded. RESULTS: Fourteen patients had DSRCT, while five patients had other sarcomas. One patient had repeat HIPEC. Patients with DSRCT had significantly longer median overall survival after surgery than patients with other tumors (44.3 vs. 12.5 months, p = 0.0013). The 3-year overall survival from time of diagnosis for DSRCT patients was 79 %. Estimated median recurrence-free survival (RFS) was 14.0 months. However, RFS for patients with DSRCT was significantly longer than for non-DSRCT patients (14.9 vs. 4.5 months, p = 0.0012). Among DSRCT patients, those without hepatic or portal metastases had longer median RFS than those with tumors at these sites (37.9 vs. 14.3 months, p = 0.02). In 100 % of patients without hepatic or portal metastasis, there was no peritoneal disease recurrence after CRS-HIPEC. CONCLUSIONS: Complete CRS-HIPEC with cisplatin is effective in select DSRCT patients. DSRCT patients with hepatic or portal metastasis have poorer outcomes.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Quimioterapia do Câncer por Perfusão Regional , Procedimentos Cirúrgicos de Citorredução , Tumor Desmoplásico de Pequenas Células Redondas/terapia , Hipertermia Induzida , Neoplasias Peritoneais/terapia , Adolescente , Adulto , Quimioterapia Adjuvante , Criança , Pré-Escolar , Terapia Combinada , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Feminino , Seguimentos , Humanos , Lactente , Masculino , Pessoa de Meia-Idade , Neoplasias Peritoneais/patologia , Prognóstico , Estudos Prospectivos , Taxa de Sobrevida , Adulto Jovem
18.
Oncologist ; 23(3): 360-366, 2018 03.
Artigo em Inglês | MEDLINE | ID: mdl-29212731

RESUMO

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is an aggressive, often fatal soft tissue sarcoma that lacks an optimal salvage regimen. We retrospectively reviewed data from 29 pretreated DSRCT patients who received pazopanib at MD Anderson Cancer Center after failure of standard chemotherapies. SUBJECTS, MATERIALS, AND METHODS: Medical records of patients treated from January 2012 to December 2016 were reviewed and regression analyses were performed. Median progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan-Meier method and differences in survival were assessed by a log-rank test. A landmark statistical analysis was used to assess OS at a predefined 12-week time point following pazopanib initiation. RESULTS: The mean age at pazopanib treatment was 27.5 years (range, 6.3-50.1 years). According to RECIST 1.1 criteria, 16 patients (55%) had stable disease, 1 patient (3%) had partial response, 1 patient (3%) had complete response, and 11 patients (38%) had progressive disease. Estimated median PFS was 5.63 months (95% confidence interval [CI]: 3.23-7.47). Median OS was 15.7 months (95% CI: 10.3-32.4). As of December 2016, 11 patients (38%) were still alive, with a median follow-up time of 16.8 (range 3.8-30.1) months. Doses between 400 and 800 mg were included. Pazopanib was well tolerated and 23 (79%) of the patients continued it until progression or death, 4 discontinued because of side effects, and 2 were still on pazopanib at the time of data analysis. CONCLUSION: In the largest study conducted to date in DSRCT, pazopanib was well tolerated and clinically active in heavily pretreated patients who otherwise lack good treatment options. IMPLICATIONS FOR PRACTICE: Desmoplastic small round cell tumor (DSRCT) is a rare, extremely aggressive soft tissue sarcoma subtype that most commonly occurs in adolescent and young adult males. No DSRCT-specific therapies exist, and for lack of a better treatment approach, current therapies have relied upon U.S. Food and Drug Administration-approved drugs like pazopanib that exhibit clinical activity in other sarcoma subtypes. This article describes the largest experience to date using pazopanib as salvage treatment in heavily pretreated DSRCT patients. Pazopanib was well tolerated and clinically active, surpassing predefined metrics proposed by the European Organization for Research and Treatment of Cancer indicative of "active" sarcoma drugs (5.63 months progression-free survival [PSF], with 62% of the study population achieving progression-free survival at 12 weeks).


Assuntos
Antineoplásicos/uso terapêutico , Tumor Desmoplásico de Pequenas Células Redondas/tratamento farmacológico , Pirimidinas/uso terapêutico , Sulfonamidas/uso terapêutico , Adolescente , Adulto , Antineoplásicos/efeitos adversos , Criança , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Pirimidinas/efeitos adversos , Estudos Retrospectivos , Terapia de Salvação , Sulfonamidas/efeitos adversos , Análise de Sobrevida , Resultado do Tratamento , Adulto Jovem
20.
Anticancer Drugs ; 28(9): 1053-1055, 2017 10.
Artigo em Inglês | MEDLINE | ID: mdl-28863036

RESUMO

Desmoplastic small round-cell tumor is a rare but highly aggressive tumor occurring mainly in adolescents and young adults. Prolonged progression-free survival has been documented in patients who have undergone aggressive multimodality therapy - that is, multiagent intensive chemotherapy, debulking surgery, and radiation therapy. Eribulin is a microtubule-dynamics inhibitor, and it has recently been shown to be active in liposarcomas. In preclinical models, eribulin activities have also been shown to occur in Ewing's sarcoma cell lines, rhabdomyosarcomas and osteosarcomas. In this study, we report three cases of male patients suffering from desmoplastic small round-cell tumor and the clinical response to eribulin in two of them.


Assuntos
Tumor Desmoplásico de Pequenas Células Redondas/tratamento farmacológico , Furanos/uso terapêutico , Cetonas/uso terapêutico , Neoplasias Pancreáticas/tratamento farmacológico , Neoplasias Peritoneais/tratamento farmacológico , Adulto , Tumor Desmoplásico de Pequenas Células Redondas/patologia , Intervalo Livre de Doença , Humanos , Masculino , Neoplasias Pancreáticas/patologia , Neoplasias Peritoneais/patologia
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