Racotumomab for treating lung cancer and pediatric refractory malignancies

Introduction: Racotumomab (originally known as 1E10 mAb) is an anti-idiotype murine IgG1 directed to membrane glycoconjugates expressed in aggressive solid tumors. It was developed as a mirror image of the idiotype of another antibody against N-glycolyl-containing molecules, such as the NeuGcGM3 ganglioside. After a successful phase II/III study, racotumomab formulated in alum was conditionally approved in Latin American countries as maintenance therapy for advanced non-small cell lung cancer. Areas covered: This review analyzes the biology of the target antigen, summarizes preclinical studies and discusses clinical trials in adults and the pediatric experience with racotumomab. Expert opinion: Proper patient selection and combination with chemotherapy, radiotherapy or checkpoint inhibitors appear to be critical issues to maximize the effects of racotumomab vaccination in lung cancer. In a recent phase I clinical trial in children with relapsed or resistant neuroectodermal malignancies, racotumomab was well tolerated and immunogenic, and its evaluation as immunotherapy for high-risk neuroblastoma is warranted.Fil: Gabri, Mariano Rolando. Universidad Nacional de Quilmes. Departamento de Ciencia y Tecnología. Laboratorio de Oncología Molecular; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; ArgentinaFil: Cacciavillano, Walter. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría ; ArgentinaFil: Chantada, Guillermo Luis. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría ; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; ArgentinaFil: Alonso, Daniel Fernando. Universidad Nacional de Quilmes. Departamento de Ciencia y Tecnología. Laboratorio de Oncología Molecular; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentin

Nefroma mesoblastico congénito: reporte de un caso

Congenital mesoblastic nephroma is a rare renal tumor, but common in the neonatal period. It has a benign behavior and excellent prognosis in its classic variety, but in the cellular subtype, it can present local recurrence, retroperitoneal invasion, and pulmonary and cerebral metastases. Treatment is radical nephrectomy. Prognostic factors are age less than three months and complete surgical resection. 17-day-old patient born at 35 weeks due to polyhydramnios, with prenatal diagnosis of abdominal mass. Physical examination showed right flank mass and arterial hypertension (121/79 mm Hg, TAM 77mm Hg). Abdominal ultrasound with enlarged right kidney and voluminous solid formation, 4.4 x 4.3 x 4.8 cm, heterogeneous and vascularized on Doppler in the lower pole. Abdominal tomography showed a heterogeneous nodular formation of 5.0 x 4.1 x 5.1 cm in the lower pole of the right kidney. Right radical nephrectomy was performed without complications. Evolution in the last 20 months was satisfactory and free of disease.El nefroma mesoblastico congénito es un tumor renal infrecuente, pero común en el periodo neonatal. De comportamiento benigno y pronóstico excelente en su variedad clásica pero en el subtipo celular puede presentar recurrencia local, invasión retroperitoneal y metástasis pulmonares y cerebrales. El tratamiento es la nefrectomía radical. Los factores pronósticos son la edad menor a tres meses y la resección quirúrgica completa. Paciente de 17 días nacida a las 35 semanas por polihidramnios, con diagnóstico prenatal de masa abdominal. Al examen físico masa en flanco derecho e hipertensión arterial (121/79 mm Hg, TAM 77mm Hg). Ecografía abdominal con riñón derecho aumentado de tamaño y voluminosa formación sólida, de 4,4 x 4,3 x 4,8 cm, heterogénea y vascularizada al Doppler en polo inferior. Tomografía de abdomen con formación nodular heterogénea de 5,0 x 4,1 x 5,1 cm en polo inferior del riñón derecho. Se realizó nefrectomía radical derecha sin complicaciones. Evolución en los últimos 20 meses satisfactoria y libre de enfermedad

Beliefs and Determinants of Use of Traditional Complementary/Alternative Medicine in Pediatric Patients Who Undergo Treatment for Cancer in South America

Purpose: The use of traditional complementary/alternative medicine (TCAM) among children with cancer has been well documented. South America has a rich history of traditional healers and medicinal resources; however, little is known about the use of TCAM among children with cancer. We sought to investigate patterns, beliefs, and determinants of TCAM use among South American children with cancer. Methods: A cross-sectional survey was administered to 199 children treated for cancer at institutions located in Buenos Aires, Argentina, and Montevideo, Uruguay. Participants were queried about the type of TCAM and strength of beliefs associated with its use. Logistic regression analysis was used to estimate the odds ratios with 95% CIs. Results: We found that the use of TCAM was common in both Argentina (47%) and Uruguay (76%). Variations in the forms of TCAM used were observed between the countries; however, both countries used TCAM primarily for supportive care. Mother’s education, wealth index, and TCAM belief system were significant predictors of TCAM. Conclusion: To our knowledge, this study is the first to report on the use of TCAM in pediatric oncology in South America. The study identifies several predictors of TCAM use, which may serve as target variables for educational and research initiatives. The finding that most families use TCAM for supportive care suggests that future efforts could evaluate the role of TCAM to enhance existing supportive care regimens, particularly in settings where access to conventional medications are limited

Beliefs and Determinants of Use of Traditional Complementary/Alternative Medicine in Pediatric Patients Who Undergo Treatment for Cancer in South America

Purpose The use of traditional complementary/alternative medicine (TCAM) among children with cancer has been well documented. South America has a rich history of traditional healers and medicinal resources; however, little is known about the use of TCAM among children with cancer. We sought to investigate patterns, beliefs, and determinants of TCAM use among South American children with cancer. Methods A cross-sectional survey was administered to 199 children treated for cancer at institutions located in Buenos Aires, Argentina, and Montevideo, Uruguay. Participants were queried about the type of TCAM and strength of beliefs associated with its use. Logistic regression analysis was used to estimate the odds ratios with 95% CIs. Results We found that the use of TCAM was common in both Argentina (47%) and Uruguay (76%). Variations in the forms of TCAM used were observed between the countries; however, both countries used TCAM primarily for supportive care. Mother's education, wealth index, and TCAM belief system were significant predictors of TCAM. Conclusion To our knowledge, this study is the first to report on the use of TCAM in pediatric oncology in South America. The study identifies several predictors of TCAM use, which may serve as target variables for educational and research initiatives. The finding that most families use TCAM for supportive care suggests that future efforts could evaluate the role of TCAM to enhance existing supportive care regimens, particularly in settings where access to conventional medications are limited.Fil: Rocha, Valeria. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Ladas, Elena J.. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Lin, Meiko. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Cacciavillano, Walter. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Ginn, Elizabeth. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Kelly, Kara M.. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Chantada, Guillermo Luis. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentina. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Castillo, Luis. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; Argentin

Tumores raros en pediatría: Primer reporte en a Argentina

Introduction: Collaborative clinical trials have enlarged the knowledge base about pediatric tumors; however, this is not the case for rare tumors (RT).Objective: To describe the prevalence, clinical characteristics, and course of RT in pediatric patients diagnosed at Hospital Garrahan.Material and methods: Descriptive, retrospective study of patients aged 0-18 years diagnosed with a RT and admitted between January 2007 and December 2017.Results: Out of 1657 patients diagnosed with solid tumors, 164 (9.9 %) were RT; 71.95 % (118) of patients were younger than 14 years and 81.7 % (130) were males. In order of frequency, RT were thyroid carcinoma (60), adrenal carcinoma (14), lung tumors (14), melanoma (13), salivary gland cancer (11), gastrointestinal tumors (8), non-germ cell gonadal tumors (7), pancreatic tumors (7), renal carcinomas (6), nasopharyngeal carcinomas (5), pheochromocytoma and paraganglioma (5), and thymic carcinoma in 1 patient. Treatment depended on tumor type and stage. The median follow-up was 34.9 months (range: 1-128.5 months); 133 patients (78.7 %) are alive and only 10 patients (6 %) were lost-to-follow-up.Conclusion: The prevalence of RT was 9.9 %. Twenty-seven percent occurred in adolescents. The most frequent tumors included thyroid carcinoma, adrenal carcinoma, and melanoma. Treatment and course varied based on tumor histology. Predisposing molecular alterations were found in 5.3 % of patients; 3.5 % had a history of cancer.Fil: Casanovas, Alejandra. Gobierno de la Ciudad de Buenos Aires. Hospital General de Niños Pedro Elizalde (ex Casa Cuna); Argentina. Ministerio de Salud. Instituto Nacional del Cáncer; ArgentinaFil: Viso, Marianela. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Felizzia, Guido. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Rose, Adriana. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Mattone, María Celeste. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentina. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Gazek, Natalia. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentina. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Cervini, Bettina. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Giuseppucci, Carlos. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Cacciavillano, Walter. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; Argentin

A phase I study of the anti-idiotype vaccine racotumomab in neuroblastoma and other pediatric refractory malignancies

BACKGROUND: Pediatric neuroectodermal malignancies express N-glycolylated gangliosides including N-glycolyl GM3 (NeuGcGM3) as targets for immunotherapy. PROCEDURE: We evaluated the toxicity and maximum tolerated dose and immunological response of racotumomab, an anti-idiotype vaccine targeting NeuGcGM3 through a Phase I study enrolling children with relapsed or resistant tumors expressing NeuGcGM3. MATERIALS AND METHODS: Drug dose was escalated to three levels (0.15-0.25-0.4 mg) of racotumomab administered intradermally. Each drug level included three patients receiving a total of three doses, every 14 days. A confirmation cohort was added to the highest dose level. Antibody response was assessed upon study entry and at 4-week intervals for at least three immunological determinations for each patient. RESULTS: Fourteen patients were enrolled (10 with neuroblastoma, one with retinoblastoma, one with Wilms' tumor, and two with brainstem glioma). Three patients completed the three drug levels and three were enrolled in the confirmation cohort. One patient died of tumor progression before completing the three applications. Racotumomab was well tolerated. The only side effect observed was grade 1-2 toxicity at the injection site. Racotumomab elicited an IgM and/or IgG antibody response directed against NGcGM3 in nine patients and IgM against racotumomab in 11 of 13 evaluable patients. The maximum tolerated dose was not reached and no dose-limiting toxicity was seen. CONCLUSIONS: Racotumomab vaccination has a favorable toxicity profile up to a dose of 0.4 mg, and most patients elicited an immune response. Its activity as immunotherapy for neuroectodermal malignancies will be tested in further clinical trials.Fil: Cacciavillano, Walter. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Sampor, Claudia. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Venier, Cecilia. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Inmunología, Genética y Metabolismo. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Inmunología, Genética y Metabolismo; ArgentinaFil: Gabri, Mariano Rolando. Universidad Nacional de Quilmes. Departamento de Ciencia y Tecnología. Laboratorio de Oncología Molecular; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; ArgentinaFil: Davila, María Teresa. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Galluzzo, María L.. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; ArgentinaFil: Guthmann, Marcelo D.. Laboratorio ELEA; ArgentinaFil: Fainboim, Leonardo. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Houssay. Instituto de Inmunología, Genética y Metabolismo. Universidad de Buenos Aires. Facultad de Medicina. Instituto de Inmunología, Genética y Metabolismo; ArgentinaFil: Alonso, Daniel Fernando. Universidad Nacional de Quilmes. Departamento de Ciencia y Tecnología. Laboratorio de Oncología Molecular; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; ArgentinaFil: Chantada, Guillermo Luis. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentin