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1.
BMC Pediatr ; 21(1): 463, 2021 10 21.
Artigo em Inglês | MEDLINE | ID: mdl-34670517

RESUMO

BACKGROUND: The aim of this study was to summarize the clinical characteristics, therapeutic effects and prognosis of patients with rhabdomyosarcoma (RMS) and bone marrow metastasis, improve the understanding of this disease. METHOD: This was a single-institution retrospective study involving the children with RMS, who presented with bone marrow metastasis at initial presentation to our hospital between 1st, Jan, 2006 and 31st, Dec,2019. Follow-up concluded on 31st, Dec, 2020 and the clinical data were collected and analysed. RESULT: Between 1st Jan 2006 and 31st Dec 2019, 13 eligible patients presented to our hospital, including 10 males and 3 females, these eligible patients accounted for 4.5% of all RMS patients. The median age at onset was 5.6 years (range 1.7-14 years). The patients not only had unfavourable primary sites, but also had multiple metastases. The bone marrow aspirate samples of the patients comprised 8-95% blast-like cells. Nine of 13 patients were misdiagnosed with haematological malignancies or other solid tumours. With respect to histology, four of 13 children were classified as embryonal RMS and nine as alveolar RMS. Eleven patients underwent PAX-FOXO1 fusion testing; eight had the POX- FOXO1 fusion gene. Immunohistochemically(IHC) analysis revealed that the tumour cells were positive for Desmin, Vimentin, Myo-D1 and Myogenin. More importantly, the patients had extremely poor prognoses, the median EFS was 12.0 months (range 3-28.3 months) and the median OS was 27.0 months (range6-46.2 months). CONCLUSION: This study demonstrates that children with RMS and bone marrow metastasis usually exhibit atypical primary sites and multiple metastases, with presentation mimicking haematological malignancies or other solid tumors at initial presentation. Pathology and IHC analysis combined with POX-FOXO1 fusion gene detections can effectively confirm the diagnosis. These patients are more likely to relapse or progress during early treatment and are prone to intracranial metastasis. While multidisciplinary therapy combined with Temozolomide may prevent it, further prospective research is required to evaluate the therapeutic effects.


Assuntos
Neoplasias da Medula Óssea , Rabdomiossarcoma , Adolescente , Neoplasias da Medula Óssea/diagnóstico , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Recidiva Local de Neoplasia , Prognóstico , Estudos Retrospectivos , Rabdomiossarcoma/diagnóstico , Rabdomiossarcoma/terapia
2.
Turk J Pediatr ; 63(3): 427-433, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34254487

RESUMO

BACKGROUND: To analyze the relationship between tumor viability in specimens retrieved at second-look procedures (SLPs) and clinical outcomes in patients with bladder/prostate rhabdomyosarcoma (BP RMS). METHODS: We retrospectively analyzed patients` characteristics, times between diagnoses and SLPs, types of SLPs, the pathological findings, and clinical outcomes between January 2003 and May 2014. RESULTS: A total of 29 patients underwent at least one SLP before completing chemotherapy, including 24 boys and 5 girls. The mean age was 36 months. No patients with clinical/radiographic complete responses (CRs) had viable tumor cells and 7/18 patients (38.9%) without CR had no viable tumor cells. Seven patients experienced tumor relapse, progression, and metastasis, and three of these survived. Five-year event-free survival (EFS) rates were 88.5% in 18 patients without viable tumor at SLPs and 54.5% in 11 patients with viable tumor (Cox proportional hazards adjusted P=0.045). The respective five-year overall survival (OS) rates were 94.1% and 72.7% (Cox proportional hazards adjusted P=0.175). CONCLUSIONS: EFS was increased in patients with BP RMS having no viable tumor cells; however, OS was comparable in patients with and without viable tumor cells. Patients who achieved CR during the treatment generally had no viable tumor cells.


Assuntos
Rabdomiossarcoma , Bexiga Urinária , Protocolos de Quimioterapia Combinada Antineoplásica , Pré-Escolar , Terapia Combinada , Feminino , Humanos , Lactente , Masculino , Recidiva Local de Neoplasia , Próstata , Estudos Retrospectivos , Rabdomiossarcoma/terapia , Resultado do Tratamento
3.
Pediatr Radiol ; 51(10): 1940-1951, 2021 09.
Artigo em Inglês | MEDLINE | ID: mdl-34137936

RESUMO

Appropriate imaging is essential in the treatment of children and adolescents with rhabdomyosarcoma. For adequate stratification and optimal individualised local treatment utilising surgery and radiotherapy, high-quality imaging is crucial. The paediatric radiologist, therefore, is an essential member of the multi-disciplinary team providing clinical care and research. This manuscript presents the European rhabdomyosarcoma imaging guideline, based on the recently developed guideline of the European Paediatric Soft Tissue Sarcoma Study Group (EpSSG) Imaging Committee. This guideline was developed in collaboration between the EpSSG Imaging Committee, the Cooperative Weichteilsarkom Studiengruppe (CWS) Imaging Group, and the Oncology Task Force of the European Society of Paediatric Radiology (ESPR). MRI is recommended, at diagnosis and follow-up, for the evaluation of the primary tumour and its relationship to surrounding tissues, including assessment of neurovascular structures and loco-regional lymphadenopathy. Chest CT along with [F-18]2-fluoro-2-deoxyglucose (FDG) positron emission tomography (PET)/CT or PET/MRI are recommended for the detection and evaluation of loco-regional and distant metastatic disease. Guidance on the estimation of treatment response, optimal long-term follow-up, technical imaging settings and standardised reporting are described. This European imaging guideline outlines the recommendations for imaging in children and adolescents with rhabdomyosarcoma, with the aim to harmonise imaging and to advance patient care.


Assuntos
Radiologia , Rabdomiossarcoma , Sarcoma , Adolescente , Criança , Humanos , Estadiamento de Neoplasias , Tomografia por Emissão de Pósitrons , Rabdomiossarcoma/diagnóstico por imagem , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia , Sarcoma/patologia
4.
Eur J Cancer ; 151: 84-93, 2021 07.
Artigo em Inglês | MEDLINE | ID: mdl-33971448

RESUMO

BACKGROUND/OBJECTIVES: The primary aim of this study was to analyse and evaluate the impact of different local treatments on the pattern of relapse in children with primary head and neck non-parameningeal (HNnPM) rhabdomyosarcoma (RMS), treated in the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS2005 study. The secondary aim was to assess whether current risk stratification is valid for this specific site. DESIGN/METHODS: This study includes all patients with localised HNnPM RMS enrolled in the RMS2005 study between 2005 and 2016. Treatment comprised chemotherapy adapted to risk group, with local surgery and/or radiation therapy. The main outcome measures were event-free survival (EFS) and overall survival (OS). RESULTS: A total of 165 patients were identified; the median age was 6.4 years (range, 0.1-25). The most common tumour sites were cheek/chin (22%) and nasal ala/nasolabial fold (20%). Histology was unfavourable for 40%, and regional nodal involvement present in 26%. Local therapy included surgery (58%) and/or radiotherapy (72%) to primary tumour and/or regional lymph nodes. After a median follow-up of 66 months (range, 6-158), 42 patients experienced an event, and 17 are still alive. Tumour events were frequent in oral primary (36%), parotid site (26%), cheek/chin (24%), and nasal ala/nasolabial fold (24%) and included locoregional failure in 84% of cases. The 5-year EFS and OS were 75% (95% confidence interval [CI]: 67.3-81.2) and 84.9% (95% CI: 77.5-89.7), respectively. Favourable histology was associated with a better EFS (82.3% versus 64.6%; p = 0.02) and nodal spread with a worse OS (88.6% versus 76.1%; p = 0.04). Different sublocations within the HNnPM primary did not have significant impact on outcome. CONCLUSION: Locoregional relapse/progression is the main tumour failure event in this site. Despite frequent unfavourable risk factors, HNnPM RMS remains a favourable location in the context of a risk-adapted strategy.


Assuntos
Neoplasias de Cabeça e Pescoço/terapia , Recidiva Local de Neoplasia , Rabdomiossarcoma/terapia , Adolescente , Adulto , Fatores Etários , Argentina , Brasil , Criança , Pré-Escolar , Progressão da Doença , Europa (Continente) , Feminino , Neoplasias de Cabeça e Pescoço/mortalidade , Neoplasias de Cabeça e Pescoço/patologia , Humanos , Lactente , Israel , Metástase Linfática , Masculino , Intervalo Livre de Progressão , Estudos Prospectivos , Rabdomiossarcoma/mortalidade , Rabdomiossarcoma/secundário , Medição de Risco , Fatores de Risco , Fatores de Tempo , Adulto Jovem
5.
Pediatr Blood Cancer ; 68 Suppl 2: e28254, 2021 05.
Artigo em Inglês | MEDLINE | ID: mdl-33818882

RESUMO

Rhabdomyosarcoma is a heterogeneous disease both in presentation and histology. Improvements in a multimodality therapy resulted in the improved overall survival for patients with a low-risk and intermediate-risk disease but not for patients with a metastatic disease. We reviewed and contrasted the North American and European practice patterns, though ultimately the principles of staging, surgery, radiation therapy, and chemotherapy are similar in both Children's Oncology Group and International Society of Paediatric Oncology treatment approaches. Efforts are underway to investigate improved local control rates in higher risk patients using radiation dose escalation strategies, and delayed primary excision in select cases. The prognostic significance of imaging-based chemotherapy response, proton therapy, novel biomarkers, and targeted drugs will be determined in upcoming clinical trials.


Assuntos
Rabdomiossarcoma/terapia , Criança , Terapia Combinada , Humanos , Prognóstico , Rabdomiossarcoma/patologia , Taxa de Sobrevida
6.
Strahlenther Onkol ; 197(8): 690-699, 2021 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-33914102

RESUMO

BACKGROUND: Treatment of extremity rhabdomyosarcomas (RMS) includes chemotherapy, surgery, and radiotherapy. Lymph node irradiation is recommended in the presence of regional node involvement at diagnosis. The aim of this study was to analyze the correlation between the pattern of relapse of non-metastatic extremity RMS and the initial therapies delivered. METHODS: All patients with localized extremity RMS prospectively treated in France in the MMT-95 and RMS-05 protocols were selected. Extent of disease and pattern of relapse were evaluated by clinical examination and imaging. RESULTS: We identified 59 patients with clinical characteristics corresponding to unfavorable prognostic factors. Twenty patients (34%) were considered to have lymph node involvement at diagnosis. Regional node biopsy was performed in 32 patients (54%) and modified the lymph node stage in 8 of the 59 patients (14%). Seventy-three percent of patients received radiotherapy. Fifty-two patients achieved first remission. Overall, 26 patients underwent complete tumor resection, 17 had R1 margins, and 5 were not operated due to early tumor progression. With a median follow-up of 82 months (range: 5-287), 18 relapses had occurred, at least locoregional in 12 cases. The 5­year local and nodal control rates were 73% (63-86%) and 86% (77-95%), respectively. Five-year progression-free and overall survival were 57% (95%CI [45-72%]) and 70% (95%CI [58-84%]), respectively. CONCLUSION: The main sites of extremity RMS relapse are locoregional. Nodal failures in non-irradiated fields are not uncommon. We recommend systematic biopsy of in-transit nodes, especially in alveolar RMS and/or RMS with regional positive nodes at diagnosis to ensure their negativity.


Assuntos
Extremidades/patologia , Recidiva Local de Neoplasia/patologia , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Linfonodos/patologia , Masculino , Estadiamento de Neoplasias , Estudos Retrospectivos , Rabdomiossarcoma/radioterapia , Rabdomiossarcoma/cirurgia
7.
J Pediatr Hematol Oncol ; 43(5): e625-e629, 2021 07 01.
Artigo em Inglês | MEDLINE | ID: mdl-33625089

RESUMO

The objectives of this study were to describe the clinicopathologic features and treatment outcomes of childhood rhabdomyosarcoma in a resource-constrained setting. All cases of childhood rhabdomyosarcoma seen over a 10-year period (July 2006 to June 2016) at the University College Hospital, Ibadan, Nigeria were reviewed. Data were extracted from the database of the pediatric Hematology/Oncology Unit of the hospital and analyzed. Ethical approval was obtained from the Institutional Ethics Committee. Fifty children were seen comprising 30 men and 20 women with bimodal ages of 4 and 5 years. Median duration of illness was 16 weeks and the most common primary tumor site was the head-and-neck region in 27 (54%) of cases. The histologic subtypes were embryonal in 30 (60%), alveolar in 9 (18%), and not specified in 11 (22%). The Intergroup Rhabdomyosarcoma Study group TNM Pretreatment stages were stage I in 15 (30%), stage III in 17 (34%), and stage IV in 18 (36%). Treatment included chemotherapy, surgery, and radiotherapy and abandoned in 20 (40%) cases. Median survival was 45 weeks (95% confidence interval: 16.4-73.6) and 5 (10%) patients were alive and disease free, 4 years or more after diagnosis. Outcome of childhood rhabdomyosarcoma is poor and early diagnosis and improved access to treatment are recommended.


Assuntos
Neoplasias de Cabeça e Pescoço/terapia , Rabdomiossarcoma/terapia , Pré-Escolar , Gerenciamento Clínico , Feminino , Neoplasias de Cabeça e Pescoço/epidemiologia , Neoplasias de Cabeça e Pescoço/patologia , Humanos , Masculino , Nigéria/epidemiologia , Estudos Retrospectivos , Rabdomiossarcoma/epidemiologia , Rabdomiossarcoma/patologia , Resultado do Tratamento
8.
Int J Oncol ; 58(2): 266-274, 2021 02.
Artigo em Inglês | MEDLINE | ID: mdl-33491749

RESUMO

Rhabdomyosarcoma (RMS), the most common pediatric soft tissue sarcoma, has an unfavorable outcome in advanced tumor stages with less than 30% failure­free survival. Curcumin (CUR) is a promising drug in complementary oncology with few side effects but proven efficacy in various adult oncological entities. The present study analyzed the effects of CUR on pediatric (RMS) cell lines in vitro. RMS cell lines (RD and RH30), and skeletal muscle cells (SKMC) were treated with different doses of CUR (1.5­30 µM) alone, with phototherapy (PDT, 488 nm) or in combination with vincristine (VCR) or dactinomycin (DAC). MTT assays were used for analysis of RMS tumor cell viability. Clonal cell growth was assessed via colony forming assays and migration of the cells was analyzed with scratch tests. Annexin V staining was used to determine apoptosis in flow cytometry. Possible RMS resistance towards CUR after long­term treatment was analyzed with MTT assays. CUR decreased cell viability in all assessed RMS cell lines in a concentration­dependent manner with IC50=14­20 µM. CUR enhanced the effects of the cytotoxic drugs VCR or DAC, and led to reduced migration and increased cell apoptosis. In combination with PDT, CUR decreased the cell viability in minute quantities with up to a 10­fold lower IC50 than without PDT. CUR effectively inhibited the malignant properties of pediatric RMS cells and should be focused on as a useful additional agent in standard chemotherapy of RMS in children.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/farmacologia , Curcumina/farmacologia , Fototerapia/métodos , Rabdomiossarcoma/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Apoptose/efeitos dos fármacos , Linhagem Celular Tumoral , Movimento Celular/efeitos dos fármacos , Proliferação de Células/efeitos dos fármacos , Sobrevivência Celular/efeitos dos fármacos , Criança , Terapia Combinada/métodos , Curcumina/uso terapêutico , Dactinomicina/farmacologia , Dactinomicina/uso terapêutico , Sinergismo Farmacológico , Humanos , Concentração Inibidora 50 , Rabdomiossarcoma/patologia , Transdução de Sinais/efeitos dos fármacos , Vincristina/farmacologia , Vincristina/uso terapêutico
9.
Pediatr Dev Pathol ; 24(2): 87-95, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-33439112

RESUMO

Rhabdomyosarcoma, the most common soft tissue sarcoma in childhood, has challenged and intrigued soft tissue pathologists ever since the original descriptions. Once based on the identification of rhabdomyoblastic cells with elongate eosinophilic cytoplasm, the diagnosis has evolved to include tumors composed only of primitive mesenchymal cells but now relies heavily on immunohistochemical stains for desmin, myogenin, and MyoD. Rhabdomyosarcomas show a variety of histological patterns, giving rise to classifications that have included embryonal, alveolar, botryoid, pleomorphic, spindle cell, and sclerosing subtypes. These have been linked to prognosis and treatment assignment in the past, but that concept has been superseded by the identification of PAX3-FOXO1 or PAX7-FOXO1 fusions. Fusion testing results are more predictive of outcome and have become standard practice in clinical management. However, high risk tumors with alveolar histology or metastatic disease continue to resist oncologic treatment.


Assuntos
Rabdomiossarcoma/diagnóstico , Rabdomiossarcoma/terapia , Neoplasias de Tecidos Moles/diagnóstico , Neoplasias de Tecidos Moles/terapia , Biomarcadores Tumorais/genética , Criança , Diagnóstico Diferencial , Fusão Gênica , Humanos , Prognóstico , Rabdomiossarcoma/genética , Rabdomiossarcoma/patologia , Neoplasias de Tecidos Moles/genética , Neoplasias de Tecidos Moles/patologia
10.
Bull Cancer ; 108(2): 163-176, 2021 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-33455736

RESUMO

Sarcomas are a rare heterogeneous group of malignant neoplasms that can arise in almost any anatomic site and any age. Close collaboration among adult and pediatric cancer specialists in the management of these tumors is of foremost importance. In this review, we present the current multidisciplinary organization in care of patients with sarcoma in France and we review the main advances made in the last decades in systemic and radiotherapy treatment in the main sarcoma types diagnosed in children, adolescents and young adults (AYA), thanks to the international collaboration.


Assuntos
Neoplasias Ósseas/terapia , Institutos de Câncer , Equipe de Assistência ao Paciente/organização & administração , Sarcoma/terapia , Neoplasias de Tecidos Moles/terapia , Adolescente , Adulto , Fatores Etários , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Institutos de Câncer/organização & administração , Institutos de Câncer/provisão & distribuição , Criança , Redes Comunitárias/organização & administração , Redes Comunitárias/provisão & distribuição , Europa (Continente) , França , Humanos , Cooperação Internacional , Neoplasias Pulmonares/radioterapia , Neoplasias Pulmonares/secundário , Quimioterapia de Manutenção , Oncologia , Pessoa de Meia-Idade , Recidiva Local de Neoplasia/radioterapia , Neoplasias Embrionárias de Células Germinativas/terapia , Osteossarcoma/terapia , Dosagem Radioterapêutica , Ensaios Clínicos Controlados Aleatórios como Assunto , Rabdomiossarcoma/terapia , Sarcoma Alveolar de Partes Moles/terapia , Sarcoma de Ewing/terapia , Sociedades Médicas , Adulto Jovem
11.
Int J Pediatr Otorhinolaryngol ; 142: 110599, 2021 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-33422992

RESUMO

OBJECTIVES: Clinical trials have reported increases in the survival of pediatric rhabdomyosarcoma (RMS) from 25% in 1970 to 73% in 2001. The purpose of this study was to examine whether survival of pediatric patients with RMS of the head and neck improved at the US population level. METHODS: A population-based cohort of patients with rhabdomyosarcoma of the head and neck aged 0-19 years in the Surveillance, Epidemiology, and End Results (SEER) registry from 1973 to 2013 was queried. The cumulative incidence competing risks (CICR) method was used to estimate risk and survival trends. RESULTS: 718 cases were identified for analysis. Survival rates at 1-, 5-, and 10-years after diagnosis were 91.2%, 73.2%, and 69.4% respectively. Survival rates at 1 year after diagnosis increased from 82.6% to 93.1% during the study period. In the subdistributional hazard analysis, there was a significantly improved disease-specific risk of death in the first year after diagnosis. Overall risk of death did not improve significantly. Favorable prognostic factors included age <10 years at diagnosis, smaller tumor size, absence of distant metastasis, localized tumors, earlier stage at presentation, grossly complete surgical resection, and embryonal or botryoid histology. CONCLUSIONS: Disease-specific survival in the first year following diagnosis improved, but the change in overall survival at the population level was not statistically significant. These findings should be interpreted in light of the inclusion of patients with distant metastasis at diagnosis, who have poor prognoses, together with the limited statistical power afforded in studies of rare diseases.


Assuntos
Neoplasias de Cabeça e Pescoço , Rabdomiossarcoma Embrionário , Rabdomiossarcoma , Criança , Neoplasias de Cabeça e Pescoço/terapia , Humanos , Prognóstico , Rabdomiossarcoma/epidemiologia , Rabdomiossarcoma/terapia , Programa de SEER , Taxa de Sobrevida
12.
Pediatr Blood Cancer ; 68(4): e28914, 2021 04.
Artigo em Inglês | MEDLINE | ID: mdl-33501771

RESUMO

BACKGROUND: Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The biliary tract is classified as a favorable primary site. Therefore, patients with localized biliary RMS were included in two consecutive low-risk studies, D9602 and ARST0331, by the Children's Oncology Group (COG). The outcome for these patients treated with low-risk therapy has not been reported. PROCEDURE: Patients with biliary RMS enrolled on COG low-risk trials D9602 or ARST0331 were analyzed. All patients received systemic chemotherapy and those with Group II (microscopic residual) or Group III (macroscopic residual) disease received 36-50.4 Gy adjuvant radiotherapy (RT). Delayed primary excision (DPE) was allowed on both studies. RESULTS: Seventeen patients with biliary RMS were treated on D9602 (n = 7) or ARST0331 (n = 10). Median age was 3.5 years (range 1.7-10.3). Ten (59%) patients had tumors >5 cm and 14 (82%) had Group III disease. Fifteen (88%) patients received RT. The 5-year event-free survival (EFS) and overall survival (OS) were 70.6% (95% confidence interval [CI]: 46.9-94.3%) and 76.5% (95% CI: 54.6-98.4%), respectively. The majority of patients (80%) who received RT did not have disease recurrence while both patients who did not receive RT had local relapse. Five (36%) of 14 patients with Group III disease underwent DPE; two experienced a local relapse. In the nine patients without DPE, two developed local relapse. CONCLUSIONS: Patients with localized biliary RMS treated on low-risk studies had suboptimal outcomes. These patients may benefit from therapy on intermediate-risk studies.


Assuntos
Neoplasias do Sistema Biliar/terapia , Rabdomiossarcoma/terapia , Neoplasias do Sistema Biliar/tratamento farmacológico , Neoplasias do Sistema Biliar/radioterapia , Neoplasias do Sistema Biliar/cirurgia , Criança , Pré-Escolar , Gerenciamento Clínico , Feminino , Humanos , Masculino , Neoplasia Residual/etiologia , Radioterapia Adjuvante , Rabdomiossarcoma/tratamento farmacológico , Rabdomiossarcoma/radioterapia , Rabdomiossarcoma/cirurgia , Resultado do Tratamento
13.
Pediatr Blood Cancer ; 68(4): e28889, 2021 04.
Artigo em Inglês | MEDLINE | ID: mdl-33438323

RESUMO

BACKGROUND: Rhabdomyosarcoma (RMS) of the female genitourinary tract (FGU-RMS) located at the vagina or uterus is one of the most favorable RMS sites. Little is known about treatment and outcome in infants and relapsed disease (RD). METHODS: Characteristics, treatment, and outcome of 71 children with FGU-RMS registered within five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry (1981-2019) were evaluated. RESULTS: FGU-RMS was diagnosed in 67 patients with localized disease (LD) at a median age of 2.89 years (0.09-18.08). Multimodal treatment consisted of chemotherapy (CHT) (n = 66), secondary surgery (n = 32), and radiotherapy (n = 11). Age at diagnosis ≤12 months was the only significant negative prognostic factor influencing the event-free survival (EFS). Ten-year EFS and overall survival (OS) for infants ≤12 months were 50% and 81%, respectively. In contrast, children with LD >1 year and ≤10 years had a 10-year EFS and OS of 78% and 94% (P = .038), and >10 years of 82% and 88%, respectively (P = .53). Metastatic disease was observed in four patients of which three are alive. RD occurred in five of 12 infants ≤1 year and 10/55 children at a median of 1.38 years (0.53-2.97) after initial diagnosis. Treatment of patients with RD consisted of multimodal treatment (n = 13) or resection only (n = 2). Nine patients (60%) were alive in clinical remission at a median of 7.02 years (1.23-16.72) after diagnosis of RD. CONCLUSION: Infants with FGU-RMS have a higher relapse rate than older children with FGU-RMS, but prognosis is fair.


Assuntos
Rabdomiossarcoma/terapia , Neoplasias Uterinas/terapia , Neoplasias Vaginais/terapia , Criança , Pré-Escolar , Gerenciamento Clínico , Feminino , Humanos , Lactente , Recidiva Local de Neoplasia/diagnóstico , Recidiva Local de Neoplasia/epidemiologia , Recidiva Local de Neoplasia/terapia , Prognóstico , Rabdomiossarcoma/diagnóstico , Rabdomiossarcoma/epidemiologia , Resultado do Tratamento , Neoplasias Uterinas/diagnóstico , Neoplasias Uterinas/epidemiologia , Neoplasias Vaginais/diagnóstico , Neoplasias Vaginais/epidemiologia
14.
Pediatr Hematol Oncol ; 38(4): 397-402, 2021 May.
Artigo em Inglês | MEDLINE | ID: mdl-33095072

RESUMO

We present the case of a 13 year old boy, with sudden onset painful unilateral visual loss, prior to commencing chemotherapy for alveolar rhabdomyosarcoma. Cases of para-neoplastic optic neuritis have been reported in adult cancer patients, however there are no published reports of this phenomenon occurring in children. Our patient had full recovery of his vision, following 6 weeks treatment with steroids, immunoglobulins and standard chemotherapy as per high risk arm of European pediatric soft tissue sarcoma group (EpSSG) Rhabdomyosarcoma (RMS) 2005 guidelines. Our case highlights that para-neoplastic optic neuritis can occur in children. In pediatric patients presenting with optic neuritis and normal auto-antibody screen, an occult or underlying tumor should be considered.


Assuntos
Cegueira/etiologia , Neurite Óptica/complicações , Rabdomiossarcoma/complicações , Neoplasias Testiculares/complicações , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Cegueira/patologia , Cegueira/terapia , Ciclofosfamida/uso terapêutico , Doxorrubicina/uso terapêutico , Etoposídeo/uso terapêutico , Humanos , Ifosfamida/uso terapêutico , Imunoglobulinas/uso terapêutico , Masculino , Neurite Óptica/patologia , Neurite Óptica/terapia , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia , Esteroides/uso terapêutico , Neoplasias Testiculares/patologia , Neoplasias Testiculares/terapia , Vincristina/uso terapêutico
15.
Laryngoscope ; 131(3): E984-E992, 2021 03.
Artigo em Inglês | MEDLINE | ID: mdl-33107076

RESUMO

OBJECTIVES: Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children. The goal of this research is to analyze the role of surgery in the management of pediatric parameningeal (PM) and non-PM head and neck RMS (HNRMS). STUDY DESIGN: Retrospective review. METHODS: Retrospective chart review of patients <20 years of age treated for HNRMS between 1970 and 2015. Clinical presentation, tumor characteristics, treatment, recurrence, follow-up, and outcome data were collected. RESULTS: Of 97 patients with HNRMS, 56% were male. Overall median (IQR: interquartile range) age at diagnosis was 5.8 (3.3-9.8) years. Sixty-five patients (67%) had PM tumors. Of 75 patients with histologic subtype identified, 51 (53%) had embryonal and 20 (21%) alveolar RMS. Almost all patients received chemotherapy (99%) and radiotherapy (95%). Forty-four patients (45%) underwent surgery. Surgery was more likely to be conducted in patients with lesions of a non-PM site. Median follow-up time was 3.4 years (IQR: 1.1-10.8). In 5 years of follow-up, 20% (17 of 85) died and 29% (20 of 70) had recurrence. The estimated 5-year survival rate was 72% (95% CI, 57.8, 81.5%). Surgery was associated with a reduced risk of mortality after accounting for TNM stage 4 and tumor site (adjusted HR 0.24; 95% CI, 0.07, 0.79; P = .02). The association between surgery and risk of mortality was similar in PM and non-PM tumors. CONCLUSION: A multimodal protocol for treatment including chemotherapy, surgery, and radiotherapy is the mainstay for management of children with HNRMS. While surgery is more commonly used to treat non-PM HNRMS, patients who are able to undergo surgery have significantly higher 5-year survival. LEVEL OF EVIDENCE: 4 Laryngoscope, 131:E984-E992, 2021.


Assuntos
Quimiorradioterapia Adjuvante/estatística & dados numéricos , Neoplasias de Cabeça e Pescoço/terapia , Recidiva Local de Neoplasia/epidemiologia , Procedimentos Cirúrgicos Otorrinolaringológicos/estatística & dados numéricos , Rabdomiossarcoma/terapia , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Seguimentos , Neoplasias de Cabeça e Pescoço/diagnóstico , Neoplasias de Cabeça e Pescoço/mortalidade , Humanos , Masculino , Recidiva Local de Neoplasia/prevenção & controle , Estudos Retrospectivos , Rabdomiossarcoma/diagnóstico , Rabdomiossarcoma/mortalidade
16.
World Neurosurg ; 145: 134-141, 2021 01.
Artigo em Inglês | MEDLINE | ID: mdl-32891846

RESUMO

BACKGROUND: Malignant neuroectodermal tumor of infancy is a rare neural crest cell-derived neoplasm of infants. Histologically, melanotic neuroectodermal tumor of infancy usually consists of 2 types of cells: neuroblast-like and melanocyte-like cells. Here we present a rare case of melanotic neuroectodermal tumor of infancy containing a third type of cell population, that is, rhabdomyoblasts in addition to the above two. CASE DESCRIPTION: We report a case of a 10-month-old female child who was brought to us with complaints of swelling over the right forehead for the last 9 months, which started increasing in size rapidly 3 months before presenting to us. Noncontrast computed tomography scan showed a large well-defined extra-axial lesion in the right frontotemporal region. The child underwent an open biopsy under general anesthesia. Histopathological sections showed a malignant small round cell tumor consisting of hyperchromatic cells lying in sheets and lobules separated by fibrous septae. The patient underwent 7 cycles of neoadjuvant chemotherapy over a period of 2 months. The patient underwent right frontotemporal craniotomy and gross total excision of the lesion as a definitive surgery. Postoperatively, the patient was stable, and there was no new deficit. Histopathology revealed neuroblast-like and melanocyte-like cells with rhabdomyosarcomatous differentiation. The patient received chemotherapy in the postoperative period. The patient had recurrence of the tumor and died 8 months after the surgery. CONCLUSIONS: Calvarial malignant neuroectodermal tumor of infancy with rhabdomyosarcomatous differentiation is a rare entity with no cases being reported before. Neoadjuvant chemotherapy with surgical excision can be a promising modality of treatment.


Assuntos
Neoplasias Encefálicas/patologia , Neoplasias Encefálicas/terapia , Tumor Neuroectodérmico Melanótico/patologia , Tumor Neuroectodérmico Melanótico/terapia , Procedimentos Neurocirúrgicos/métodos , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia , Biópsia , Neoplasias Encefálicas/cirurgia , Terapia Combinada , Craniotomia , Evolução Fatal , Feminino , Humanos , Lactente , Terapia Neoadjuvante , Recidiva Local de Neoplasia , Crista Neural/patologia , Crista Neural/cirurgia , Tumor Neuroectodérmico Melanótico/cirurgia , Rabdomiossarcoma/cirurgia , Tomografia Computadorizada por Raios X
17.
J Pediatr Hematol Oncol ; 43(6): e854-e858, 2021 08 01.
Artigo em Inglês | MEDLINE | ID: mdl-32769567

RESUMO

Malignant ectomesenchymoma (MEM) is a rare multiphenotypic tumor comprised of mesenchymal and neuroectodermal components. MEM is typically diagnosed in infants and younger children and outcomes are variable. The current approach for treating MEM includes targeting the more aggressive mesenchymal component of the tumor, which is often rhabdomyosarcoma. Here, we describe a case of an orbital tumor initially diagnosed and treated as low-risk rhabdomyosarcoma. Local failure prompting a second biopsy revealed neuronal differentiation consistent with a diagnosis of MEM. Intensifying therapy and local radiotherapy led to a long-term cure. This case offers a cautionary tale that while outcomes for MEM were similar to matched rhabdomyosarcoma cohorts when treated on conventional Intergroup Rhabdomyosarcoma Study Group (IRSG) III/IV protocols, treating MEM using a decreased intensity low-risk rhabdomyosarcoma regimen may not be sufficient.


Assuntos
Neoplasias Orbitárias/diagnóstico , Rabdomiossarcoma Embrionário/diagnóstico , Rabdomiossarcoma/diagnóstico , Gerenciamento Clínico , Humanos , Lactente , Masculino , Neoplasias Orbitárias/patologia , Neoplasias Orbitárias/terapia , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia , Rabdomiossarcoma Embrionário/patologia , Rabdomiossarcoma Embrionário/terapia
18.
Cancer ; 127(6): 946-956, 2021 03 15.
Artigo em Inglês | MEDLINE | ID: mdl-33216382

RESUMO

BACKGROUND: The objective of this analysis was to evaluate the clinical factors influencing survival outcomes in patients with localized (clinical group I-III), FOXO1 fusion-positive rhabdomyosarcoma (RMS). METHODS: Patients with confirmed FOXO1 fusion-positive RMS who were enrolled on 3 completed clinical trials for localized RMS were included in the analytic cohort. Outcomes were analyzed using the Kaplan-Meier method to estimate event-free survival (EFS) and overall survival (OS), and the curves were compared using the log-rank test. A Cox proportional hazards regression model was used to perform multivariate analysis of prognostic factors that were significant in the univariate analysis. RESULTS: The estimated 4-year EFS and OS of 269 patients with localized, FOXO1 fusion-positive RMS was 53% (95% CI, 47%-59%) and 69% (95% CI, 63%-74%), respectively. Univariate analysis revealed that several known favorable clinical characteristics, including age at diagnosis between 1 and 9 years, complete surgical resection, tumor size ≤5 cm, favorable tumor site, absence of lymph node involvement, confinement to the anatomic site of origin, and PAX7-FOXO1 fusion, were associated with improved outcomes. Multivariate analysis identified older age (≥10 years) and large tumor size (>5 cm) as independent, adverse prognostic factors for EFS within this population, and patients who had both adverse features experienced substantially inferior outcomes. CONCLUSIONS: Patients with localized, FOXO1 fusion-positive RMS can be further risk stratified based on clinical features at diagnosis, and older patients with large primary tumors have the poorest prognosis.


Assuntos
Proteína Forkhead Box O1/genética , Fusão Gênica , Rabdomiossarcoma/mortalidade , Adolescente , Criança , Pré-Escolar , Ensaios Clínicos como Assunto , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Proteínas de Fusão Oncogênica/genética , Fatores de Transcrição Box Pareados/genética , Rabdomiossarcoma/genética , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia
19.
Cancer Med ; 10(3): 857-866, 2021 02.
Artigo em Inglês | MEDLINE | ID: mdl-33340280

RESUMO

BACKGROUND: Strategies to optimize management in rhabdomyosarcoma (RMS) include risk stratification to assign therapy aiming to minimize treatment morbidity yet improve outcomes. This analysis evaluated the relationship between complete metabolic response (CMR) as assessed by 18 F-fluorodeoxyglucose positron emission tomography-computed tomography (FDG-PET) imaging and event-free survival (EFS) in intermediate-risk (IR) and high-risk (HR) RMS patients. METHODS: FDG-PET imaging characteristics, including assessment of CMR and maximum standard uptake values (SUVmax) of the primary tumor, were evaluated by central review. Institutional reports of SUVmax were used when SUVmax values could not be determined by central review. One hundred and thirty IR and 105 HR patients had FDG-PET scans submitted for central review or had SUVmax data available from institutional report at any time point. A Cox proportional hazards regression model was used to evaluate the relationship between these parameters and EFS. RESULTS: SUVmax at study entry did not correlate with EFS for IR (p = 0.32) or HR (p = 0.86) patients. Compared to patients who did not achieve a CMR, EFS was not superior for IR patients who achieved a CMR at weeks 4 (p = 0.66) or 15 (p = 0.46), nor for HR patients who achieved CMR at week 6 (p = 0.75) or 19 (p = 0.28). Change in SUVmax at week 4 (p = 0.21) or 15 (p = 0.91) for IR patients or at week 6 (p = 0.75) or 19 (p = 0.61) for HR patients did not correlate with EFS. CONCLUSION: Based on these data, FDG-PET does not appear to predict EFS in IR or HR-RMS. It remains to be determined whether FDG-PET has a role in predicting survival outcomes in other RMS subpopulations.


Assuntos
Biomarcadores Tumorais/metabolismo , Fluordesoxiglucose F18/metabolismo , Tomografia por Emissão de Pósitrons combinada à Tomografia Computadorizada/métodos , Rabdomiossarcoma/metabolismo , Rabdomiossarcoma/mortalidade , Criança , Pré-Escolar , Terapia Combinada , Feminino , Seguimentos , Humanos , Lactente , Recém-Nascido , Masculino , Prognóstico , Compostos Radiofarmacêuticos/metabolismo , Rabdomiossarcoma/patologia , Rabdomiossarcoma/terapia , Taxa de Sobrevida
20.
Int J Clin Pract ; 75(4): e13951, 2021 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-33342027

RESUMO

AIM: To describe the pattern of paediatric Rhabdomyosarcomas (RMS) and Non-Rhabdomyosarcomas (NRMS) with emphasis on the indices that affect survival outcomes. METHODS: We reviewed all patients with histologically confirmed RMS and NRMS in the Departments of Pathology and Paediatrics, University College Hospital (UCH), Ibadan, Nigeria; in children aged 0-14 years. The study period was January 1991 to December 2016. Information obtained included age, gender, morphology and site of the tumours. The tumour grade and pathologic/clinical staging of all patients were also obtained and verified by the clinical records. Tumour grading was carried out using the Fédération Nationale des Centres de Lutte Contre le Cancer (FNCLCC) Sarcoma group grading system and staging was done using TNM. Follow up, survival information and final outcome were retrieved. RESULTS: The 104 patients included in the study had almost equal male-to-female ratio, age ranged between 5 months and 14 years (median 8.2 years). Rhabdomyosarcoma had mean age of 5.6 (±3.8) years while that of NRMS was 9.2(±4.1) years. Overall, the modal age group was 5-9 years. Rhabdomyosarcoma was the commonest histological type (76%), undifferentiated sarcomas (6.7%), fibrosarcoma (3.8%) and 2.9% each for synovial sarcoma and dermatofibrosarcoma protuberans. The common primary sites were the head and neck (including the orbit) 49 (47.1%), and the abdominopelvic 26 (25%) regions. Majority (89%) had histologic grade 3 at presentation. Seventy per cent and 64% of patients with RMS and NRMS, respectively, had high stage tumour at presentation. Median survival for all patients with Rhabdomyosarcoma was 45 weeks with a 1-year survival of 43% and 2-year survival of 25%. Non-RMS (Dermatofibrosarcoma protuberans and Solitary fibrous tumours) had survival of over 4 year's duration. CONCLUSION: Majority of our patients presented at a late stage with histologic high grade which confers poor prognosis and reduced chances for good overall survival outcome.


Assuntos
Rabdomiossarcoma , Sarcoma , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Nigéria , Prognóstico , Estudos Retrospectivos , Rabdomiossarcoma/terapia
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