Cemiplimab in Advanced Cutaneous Squamous Cell Carcinoma: Real-World Experience in a Monographic Oncology Center / Cemiplimab en el carcinoma de células escamosas cutáneo avanzado: experiencia del mundo real en un centro oncológico monográfico

Management of advanced cSCC is challenging, and many available systemic medications have modest efficacy. Cemiplimab has demonstrated efficacy in the treatment of advanced cSCC in clinical trials, but real-world data are still limited. With the objective of evaluating the efficacy of cemiplimab in a real-world clinical setting, we conducted a prospective observational study of 13 patients with advanced cSCC. Six patients (46%) had locally advanced disease, while 7 (54%) had metastatic disease. A total of 8 patients (62%) responded to cemiplimab. Five (38%) showed a partial response, while 3 (23%) showed a complete response. Four patients with an initial partial response presented subsequent disease progression during follow-up. Six patients (46%) developed AEs, most of which were mild (G1). PFS was 5.9 months, with a median follow-up was 9 months. In conclusion, cemiplimab demonstrated its utility in the treatment of advanced cSCC, with acceptable response rates, a remarkable number of complete responses, and a very good safety profile (AU)

El manejo del carcinoma de células escamosas cutáneo (cSCC) avanzado es complicado, siendo modesta la eficacia de muchos de los fármacos sistémicos disponibles. Cemiplimab ha demostrado su eficacia en el tratamiento del cSCC avanzado en ensayos clínicos, pero los datos del mundo real siguen siendo limitados. Con el objetivo de evaluar la eficacia de cemiplimab en un entorno clínico del mundo real, realizamos un estudio observacional prospectivo de 13 pacientes con cSCC avanzado. Seis pacientes (46%) tenían enfermedad localmente avanzada, mientras que 7 (54%) tenían enfermedad metastásica. Un total de 8 pacientes (62%) respondieron a cemiplimab, 5 (38%) mostraron una respuesta parcial y 3 (23%) mostraron una respuesta completa. Cuatro pacientes con respuesta parcial inicial presentaron una progresión de la enfermedad subsiguiente durante el seguimiento. Seis pacientes (46%) desarrollaron efectos secundarios, siendo leve la mayoría de los mismos (G1). La supervivencia libre de progresión fue de 5,9 meses, con un seguimiento medio de 9 meses. En conclusión, cemiplimab demostró su utilidad en el tratamiento del cSCC avanzado, con unas tasas de respuesta aceptables, un número destacable de respuestas completas y un perfil de seguridad muy bueno (AU)

[Artículo traducido] Cemiplimab en el carcinoma de células escamosas cutáneo avanzado: experiencia del mundo real en un centro oncológico monográfico / Cemiplimab in Advanced Cutaneous Squamous Cell Carcinoma: Real-World Experience in a Monographic Oncology Center

El manejo del carcinoma de células escamosas cutáneo (cSCC) avanzado es complicado, siendo modesta la eficacia de muchos de los fármacos sistémicos disponibles. Cemiplimab ha demostrado su eficacia en el tratamiento del cSCC avanzado en ensayos clínicos, pero los datos del mundo real siguen siendo limitados. Con el objetivo de evaluar la eficacia de cemiplimab en un entorno clínico del mundo real, realizamos un estudio observacional prospectivo de 13 pacientes con cSCC avanzado. Seis pacientes (46%) tenían enfermedad localmente avanzada, mientras que 7 (54%) tenían enfermedad metastásica. Un total de 8 pacientes (62%) respondieron a cemiplimab, 5 (38%) mostraron una respuesta parcial y 3 (23%) mostraron una respuesta completa. Cuatro pacientes con respuesta parcial inicial presentaron una progresión de la enfermedad subsiguiente durante el seguimiento. Seis pacientes (46%) desarrollaron efectos secundarios, siendo leve la mayoría de los mismos (G1). La supervivencia libre de progresión fue de 5,9 meses, con un seguimiento medio de 9 meses. En conclusión, cemiplimab demostró su utilidad en el tratamiento del cSCC avanzado, con unas tasas de respuesta aceptables, un número destacable de respuestas completas y un perfil de seguridad muy bueno (AU)

Management of advanced cSCC is challenging, and many available systemic medications have modest efficacy. Cemiplimab has demonstrated efficacy in the treatment of advanced cSCC in clinical trials, but real-world data are still limited. With the objective of evaluating the efficacy of cemiplimab in a real-world clinical setting, we conducted a prospective observational study of 13 patients with advanced cSCC. Six patients (46%) had locally advanced disease, while 7 (54%) had metastatic disease. A total of 8 patients (62%) responded to cemiplimab. Five (38%) showed a partial response, while 3 (23%) showed a complete response. Four patients with an initial partial response presented subsequent disease progression during follow-up. Six patients (46%) developed AEs, most of which were mild (G1). PFS was 5.9 months, with a median follow-up was 9 months. In conclusion, cemiplimab demonstrated its utility in the treatment of advanced cSCC, with acceptable response rates, a remarkable number of complete responses, and a very good safety profile (AU)

Cemiplimab in Advanced Cutaneous Squamous Cell Carcinoma: Real-World Experience in a Monographic Oncology Center. / [Artículo traducido] Cemiplimab en el carcinoma de células escamosas cutáneo avanzado: experiencia del mundo real en un centro oncológico monográfico.

Management of advanced cSCC is challenging, and many available systemic medications have modest efficacy. Cemiplimab has demonstrated efficacy in the treatment of advanced cSCC in clinical trials, but real-world data are still limited. With the objective of evaluating the efficacy of cemiplimab in a real-world clinical setting, we conducted a prospective observational study of 13 patients with advanced cSCC. Six patients (46%) had locally advanced disease, while 7 (54%) had metastatic disease. A total of 8 patients (62%) responded to cemiplimab. Five (38%) showed a partial response, while 3 (23%) showed a complete response. Four patients with an initial partial response presented subsequent disease progression during follow-up. Six patients (46%) developed AEs, most of which were mild (G1). PFS was 5.9 months, with a median follow-up was 9 months. In conclusion, cemiplimab demonstrated its utility in the treatment of advanced cSCC, with acceptable response rates, a remarkable number of complete responses, and a very good safety profile.

Cemiplimab in Advanced Cutaneous Squamous Cell Carcinoma: Real-World Experience in a Monographic Oncology Center.

Management of advanced cSCC is challenging, and many available systemic medications have modest efficacy. Cemiplimab has demonstrated efficacy in the treatment of advanced cSCC in clinical trials, but real-world data are still limited. With the objective of evaluating the efficacy of cemiplimab in a real-world clinical setting, we conducted a prospective observational study of 13 patients with advanced cSCC. Six patients (46%) had locally advanced disease, while 7 (54%) had metastatic disease. A total of 8 patients (62%) responded to cemiplimab. Five (38%) showed a partial response, while 3 (23%) showed a complete response. Four patients with an initial partial response presented subsequent disease progression during follow-up. Six patients (46%) developed AEs, most of which were mild (G1). PFS was 5.9 months, with a median follow-up was 9 months. In conclusion, cemiplimab demonstrated its utility in the treatment of advanced cSCC, with acceptable response rates, a remarkable number of complete responses, and a very good safety profile.

A randomized phase II study comparing the efficacy and safety of the glyco-optimized anti-EGFR antibody tomuzotuximab against cetuximab in patients with recurrent and/or metastatic squamous cell cancer of the head and neck - the RESGEX study.

BACKGROUND: The aim of the RESGEX study was to compare the efficacy and safety of the anti-epidermal growth factor receptor (anti-EGFR) antibody tomuzotuximab against cetuximab both in combination with chemotherapy in patients with recurrent and/or metastatic squamous cell cancer of the head and neck in the first-line treatment. PATIENTS AND METHODS: In this phase II trial 240 patients were equally randomized for six cycles to receive either tomuzotuximab (initial dose 990 mg then 720 mg) weekly and cisplatin 100 mg/m2 and fluorouracil (5-FU; 1000 mg/m2/day, days 1-4) every 3 weeks or cetuximab (400 mg/m2 subsequent 250 mg/m2) weekly with the same chemotherapeutic backbone followed by antibody maintenance treatment. The primary endpoint was progression-free survival. RESULTS: Median progression-free survival was 6.5 months [95% confidence interval (CI) 5.9-7.9 months] in the tomuzotuximab group and 6.2 months (95% CI 5.8-7.3 months) in the cetuximab group (P = 0.86). The median overall survival (OS) estimate was 11.6 months (95% CI 9.5-17.2 months) in the tomuzotuximab group and 13.8 months (95% CI 12.3-16.4 months) in the cetuximab group (P = 0.96). In an exploratory analysis a small subgroup of p16-positive patients had a significantly longer OS compared with p16-negative patients (hazard ratio 1.860, 95% CI 1.09-3.16, P = 0.02). CONCLUSIONS: The glyco-engineered antibody tomuzotuximab failed to demonstrate improved efficacy with a chemotherapeutic backbone in the first-line treatment of recurrent or metastatic head and neck squamous cell carcinoma. It remains a so far unanswered question whether such antibody would partner better with different drugs such as checkpoint inhibitors.

Experiencia con vismodegib en carcinoma basocelular avanzado en un centro oncológico / Experience With Vismodegib in the Treatment of Advanced Basal Cell Carcinoma at a Cancer Center

INTRODUCCIÓN Y OBJETIVOS: Vismodegib es el primer inhibidor selectivo de la vía de la señalización Hedgehog aprobado para el tratamiento del carcinoma basocelular (CBC) localmente avanzado y metastásico. Describimos nuestra experiencia en un centro oncológico con el vismodegib en el tratamiento de pacientes con CBC avanzados y/o múltiples durante un periodo de 5 años. MATERIAL Y MÉTODOS: Analizamos variables como la edad y el sexo del paciente, la localización, el tamaño, el tipo y las características del tumor, el tiempo de evolución, si son tumores primarios o recidivas, la duración del tratamiento, la respuesta a este (completa, parcial, estabilización o ausencia de respuesta), los efectos secundarios observados y las recidivas. RESULTADOS: Un total de 22 pacientes fueron tratados, 20 con CBC localmente avanzados y 2 con CBC metastásicos con afectación ganglionar. El tratamiento fue administrado durante 11,8 meses de media. El 41% (9) de los pacientes obtuvieron una respuesta completa al tratamiento, un 45% (10) respuesta parcial y en el 14% (3) de los pacientes el tratamiento consiguió estabilizar la enfermedad. Tras una mediana de 21 meses, 2 casos recidivaron. Los principales efectos secundarios observados fueron disgeusia, alopecia y calambres musculares, todos ellos de carácter leve. Ningún paciente desarrolló un carcinoma epidermoide sobre el área tratada con vismodegib, aunque sí cambios metatípicos tras el tratamiento. CONCLUSIONES: El vismodegib es un fármaco seguro y eficaz para el tratamiento del CBC localmente avanzado, con un porcentaje de respuesta del 86%. Los efectos adversos deben tenerse en cuenta por su alta frecuencia, aunque estos suelen ser de carácter leve

INTRODUCTION AND OBJECTIVES: Vismodegib is the first selective Hedgehog inhibitor approved for the treatment of locally advanced and metastatic basal cell carcinoma (BCC). In this article, we describe our experience with the use of this drug to treat advanced and/or multiple BCCs at a cancer center over 5 years. MATERIAL AND METHODS: We analyzed the following variables: patient age and sex; tumor location, size, type, and characteristics; time since onset; primary or recurrent status; duration of treatment; response to treatment (complete, partial, stabilization, or absence of response); adverse effects; and recurrences. RESULTS: We treated 22 patients, of whom 20 had locally advanced BCCs and 2 had metastatic BCCs with lymph node involvement. The treatment was administered over a mean of 11.8 months. Nine patients (41%) achieved complete response and 10 (45%) partial response. The disease was stabilized in 3 (14%). Two patients relapsed after a median of 21 months. The main adverse effects were dysgeusia, alopecia, and muscle cramps, all of which were mild. None of the patients developed squamous cell carcinoma in an area treated with vismodegib, although metatypical changes were observed after treatment. CONCLUSIONS: With a response rate of 96%, vismodegib is a safe and effective treatment for locally advanced BCC. Adverse effects are generally mild but they need to be taken into account owing to their high frequency

Experience With Vismodegib in the Treatment of Advanced Basal Cell Carcinoma at a Cancer Center. / Experiencia con vismodegib en carcinoma basocelular avanzado en un centro oncológico.

INTRODUCTION AND OBJECTIVES: Vismodegib is the first selective Hedgehog inhibitor approved for the treatment of locally advanced and metastatic basal cell carcinoma (BCC). In this article, we describe our experience with the use of this drug to treat advanced and/or multiple BCCs at a cancer center over 5 years. MATERIAL AND METHODS: We analyzed the following variables: patient age and sex; tumor location, size, type, and characteristics; time since onset; primary or recurrent status; duration of treatment; response to treatment (complete, partial, stabilization, or absence of response); adverse effects; and recurrences. RESULTS: We treated 22 patients, of whom 20 had locally advanced BCCs and 2 had metastatic BCCs with lymph node involvement. The treatment was administered over a mean of 11.8 months. Nine patients (41%) achieved complete response and 10 (45%) partial response. The disease was stabilized in 3 (14%). Two patients relapsed after a median of 21 months. The main adverse effects were dysgeusia, alopecia, and muscle cramps, all of which were mild. None of the patients developed squamous cell carcinoma in an area treated with vismodegib, although metatypical changes were observed after treatment. CONCLUSIONS: With a response rate of 96%, vismodegib is a safe and effective treatment for locally advanced BCC. Adverse effects are generally mild but they need to be taken into account owing to their high frequency.

Angiosarcoma cutáneo: estudio clínico-patológico de 16 casos / Cutaneous Angiosarcoma: Clinical and Pathology Study of 16 Cases

Introducción y objetivos: El angiosarcoma primario cutáneo es uno de los tumores más agresivos y de peor pronóstico de la piel. Su clínica inicialmente indolente justifica frecuentes diagnósticos tardíos, lo que sumado a su carácter muchas veces multifocal y a su mala delimitación suele dificultar la cirugía. Debido a su baja frecuencia existen pocas series largas de casos tratados en un mismo centro. Revisamos las características clínico-patológicas de los angiosarcomas cutáneos tratados en nuestro centro en búsqueda de factores pronósticos, así como de posibles rasgos que faciliten un diagnóstico precoz. Material y métodos: Se realizó un estudio observacional retrospectivo de todos los pacientes diagnosticados de angiosarcoma cutáneo atendidos en nuestro hospital entre enero de 2000 y diciembre de 2015. Se recogieron 16 parámetros clínicos incluidos —entre otros— edad, sexo, tipo de angiosarcoma, localización, tamaño, tiempo de evolución y además 8 parámetros histopatológicos. Resultados: Se recogieron 16 pacientes con angiosarcoma cutáneo —11 mujeres y 5 varones—, la media de edad fue de 67 años y la mediana de 71 años. La localización más frecuente fue el tronco con 10 casos, seguida de la cabeza y el cuello con 5 casos. La media del tamaño tumoral fue de 10cm y la mediana de 6,5cm. Se realizó escisión quirúrgica del tumor a 14 pacientes. Tras una media de seguimiento de 42,5 meses, 6 de los 16 pacientes seguían vivos al finalizar el estudio. Conclusiones: La supervivencia de los pacientes con angiosarcoma cutáneo viene determinada principalmente por el tamaño tumoral y la edad. Otros rasgos asociados a peor pronóstico en nuestros pacientes fueron la infiltración a planos más profundos (músculo), un patrón histológico predominantemente sólido y un mayor número de mitosis (AU)

Introduction and objectives: Primary cutaneous angiosarcoma is one of the most aggressive skin tumors and carries a very poor prognosis. Its initially indolent clinical presentation explains the frequently late diagnosis that, together with its typically multifocal pattern and poor delimitation, often makes surgery difficult. The low incidence of primary cutaneous angiosarcoma means that few large single-center series have been published. We review the clinical and pathologic characteristics of cutaneous angiosarcomas treated in our hospital, looking for prognostic factors and for possible diagnostic traits that could facilitate early diagnosis. Material and methods: This was a retrospective observational study including all patients diagnosed with cutaneous angiosarcoma in Instituto Valenciano de Oncología in Valencia, Spain between January 2000 and December 2015. We recorded 16 clinical parameters, including age, sex, type of angiosarcoma, site, size, and time since diagnosis, and 8 histopathologic parameters. Results: We identified 16 patients (11 women and 5 men) with cutaneous angiosarcoma. Their mean age was 67 years (median, 71 years). The most common site was the trunk (10 cases), followed by the head and neck (5 cases). The mean size of the tumor was 10cm (median, 6.5cm). Fourteen patients underwent surgical excision. Six of the 16 patients were alive at the end of the study, after a mean follow-up period of 42.5 months. Conclusions: The major determinants of survival among patients with cutaneous angiosarcoma are tumor size and patient age. Other characteristics associated with a poor prognosis were infiltration of deep planes (muscle), a predominantly solid histologic pattern, and a larger number of mitoses (AU)

Cutaneous Angiosarcoma: Clinical and Pathology Study of 16 Cases. / Angiosarcoma cutáneo: estudio clínico-patológico de 16 casos.

INTRODUCTION AND OBJECTIVES: Primary cutaneous angiosarcoma is one of the most aggressive skin tumors and carries a very poor prognosis. Its initially indolent clinical presentation explains the frequently late diagnosis that, together with its typically multifocal pattern and poor delimitation, often makes surgery difficult. The low incidence of primary cutaneous angiosarcoma means that few large single-center series have been published. We review the clinical and pathologic characteristics of cutaneous angiosarcomas treated in our hospital, looking for prognostic factors and for possible diagnostic traits that could facilitate early diagnosis. MATERIAL AND METHODS: This was a retrospective observational study including all patients diagnosed with cutaneous angiosarcoma in Instituto Valenciano de Oncología in Valencia, Spain between January 2000 and December 2015. We recorded 16 clinical parameters, including age, sex, type of angiosarcoma, site, size, and time since diagnosis, and 8 histopathologic parameters. RESULTS: We identified 16 patients (11 women and 5 men) with cutaneous angiosarcoma. Their mean age was 67 years (median, 71 years). The most common site was the trunk (10 cases), followed by the head and neck (5 cases). The mean size of the tumor was 10cm (median, 6.5cm). Fourteen patients underwent surgical excision. Six of the 16 patients were alive at the end of the study, after a mean follow-up period of 42.5 months. CONCLUSIONS: The major determinants of survival among patients with cutaneous angiosarcoma are tumor size and patient age. Other characteristics associated with a poor prognosis were infiltration of deep planes (muscle), a predominantly solid histologic pattern, and a larger number of mitoses.

Cost-effectiveness analysis of pazopanib in second-line treatment of advanced soft tissue sarcoma in Spain

Purpose. To assess the efficiency of pazopanib compared with trabectedin in the treatment of adult patients with selective subtypes of advanced soft-tissue sarcoma (STS) after chemotherapy failure. Methods. The progression of STS was modeled using a partitioned survival analysis model. Survival curves for pazopanib and trabectedin were modeled using data from PALETTE phase III clinical trial and based on unadjusted indirect comparison. Effectiveness was measured in quality-adjusted life years (QALY). The Spanish National Health System perspective was considered over a 10-year time horizon, including direct health care costs (Euros, 2014). A discount rate of 3 % was applied to both costs and outcomes. The robustness of the results was evaluated using univariate and probabilistic sensitivity analyses (PSA). Results. Pazopanib was associated with better health outcomes than trabectedin (0.705 versus 0.686 QALY). Pazopanib also showed lower direct health care costs (€21,861 versus €45,338), mainly due to lower cost of pharmacological treatment (€13,762 versus €33,392), administration (€57 versus €2,955) and AE management (€658 versus €1,695) costs. PSA confirmed that pazopanib was a dominant option in 71 % of the simulations performed. Conclusions. In this analysis, and from a health economics perspective, pazopanib was the option of choice versus trabectedin in the treatment of adult patients with advanced soft-tissue sarcoma after chemotherapy failure (AU)

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Cost-effectiveness analysis of pazopanib in second-line treatment of advanced soft tissue sarcoma in Spain.

PURPOSE: To assess the efficiency of pazopanib compared with trabectedin in the treatment of adult patients with selective subtypes of advanced soft-tissue sarcoma (STS) after chemotherapy failure. METHODS: The progression of STS was modeled using a partitioned survival analysis model. Survival curves for pazopanib and trabectedin were modeled using data from PALETTE phase III clinical trial and based on unadjusted indirect comparison. Effectiveness was measured in quality-adjusted life years (QALY). The Spanish National Health System perspective was considered over a 10-year time horizon, including direct health care costs (, 2014). A discount rate of 3% was applied to both costs and outcomes. The robustness of the results was evaluated using univariate and probabilistic sensitivity analyses (PSA). RESULTS: Pazopanib was associated with better health outcomes than trabectedin (0.705 versus 0.686 QALY). Pazopanib also showed lower direct health care costs (21,861 versus 45,338), mainly due to lower cost of pharmacological treatment (13,762 versus 33,392), administration (57 versus 2,955) and AE management (658 versus 1,695) costs. PSA confirmed that pazopanib was a dominant option in 71% of the simulations performed. CONCLUSIONS: In this analysis, and from a health economics perspective, pazopanib was the option of choice versus trabectedin in the treatment of adult patients with advanced soft-tissue sarcoma after chemotherapy failure.