Topotecán en el tratamiento de niños con tumores sólidos refractarios o recidivantes

Pérez Martínez, A.; Contra, T.; Scaglione, C.; Díaz Pérez, M.A.; Madero López, L.

doi:10.1016/S1695-4033(03)78738-4

Article information

Antecedentes

Topotecán es un quimioterápico alcaloide derivado de la planta Camptotheca acuminata (originaria de China) con capacidad de inhibir la enzima topoisomerasa I y de reciente uso en el tratamiento del cáncer pediátrico.

Objetivos

Evaluar nuestra experiencia preliminar con el topotecán en el tratamiento de segunda línea de tumores sólidos refractarios en la edad pediátrica.

Pacientes y métodos

Estudio retrospectivo de 10 pacientes con tumores sólidos refractarios o recidivantes a la primera línea de tratamiento que reciben topotecán en monoterapia o en asociación con otros agentes quimioterápicos.

Resultados

Se incluyeron 10 pacientes con tumores sólidos refractarios o recidivantes al tratamiento convencional (dos neuroblastomas, tres rabdomiosarcomas, dos tumores neuroectodérmicos primitivos (PNET)/Ewing, un astrocitoma anaplásico, un tumor desmoplásico y un sarcoma sinovial). Obtuvieron respuesta favorable 5 pacientes (dos remisiones completas, dos remisiones parciales y un estabilización de la enfermedad). En 5 pacientes no se consiguió ninguna respuesta. Todos los pacientes presentaron toxicidad hematológica de grado III-IV.

Conclusiones

En nuestra experiencia, topotecán sería beneficioso en determinados tumores sólidos refractarios o recidivantes, sobre todo neuroblastomas y sarcomas de partes blandas, con una aceptable tolerancia a la toxicidad hematopoyética con el uso de factores estimulantes de colonias granulocíticas. Los pacientes en remisión completa de su enfermedad tras topotecán podrían beneficiarse de una intensificación de quimioterapia con rescate autólogo de progenitores hematopoyéticos.

Palabras clave:

Glioma de tronco

Neuroblastoma

PNET

Rabdomiosarcoma

Sarcoma sinovial

Topotecán

Tumor desmoplásico

Tumores sólidos refractarios

Background

Topotecan is a cytotoxic drug isolated from the Camptotheca acuminata tree (from China). It is able to block the enzyme DNA topoisomerase I and has recently been used in the treatment of pediatric cancer.

Objectives

To evaluate our preliminary experience with topotecan in the second line treatment of refractory solid tumors in the pediatric age group.

Patients and methods

We performed a retrospective study of 10 patients with various recurrent solid tumors resistant to first line treatment who were treated with topotecan alone or in association with other chemotherapeutic agents.

Results

Ten patients with recurrent solid tumors or tumors that were refractory to conventional treatment (two neuroblastomas, three rhabdomyosarcoma, two PNET/Ewing's sarcoma, one anaplastic astrocytoma, one soft tissue sarcoma and one synovial sarcoma) were included. Five patients showed favorable responses (two had complete responses, two had partial responses and one had stable disease). Five patients showed no response. All patients showed grade II-IV hematological toxicity.

Conclusions

In our experience, topotecan is beneficial in some refractory or recurrent solid tumors, especially neuroblas-tomas and soft tissue sarcomas. Myelosuppression was tolerable with the use of granulocyte colony-stimulating factors. Patients with a complete response to topotecan could benefit from high-dose chemotherapy and autologous stem cell rescue therapy.

Key words:

Brainstem glioma

Neuroblastoma

PNET/Ewing's sarcoma

Rhabdomyosarcoma

Soft tissue sarcoma

Synovial sarcoma

Topotecan

Recurrent and refractory pediatric solid tumors

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