Globe Salvage With Intra-Arterial Topotecan-Melphalan Chemotherapy in Children With a Single Eye.

INTRODUCTION: Intra-arterial chemotherapy is a novel therapeutic modality for retinoblastoma patients. Intra-arterial chemotherapy involves the administration of a super-selective drug through the ophthalmic artery, resulting in better ocular penetration and low systemic toxicity. OBJECTIVE: The aim of this report was to evaluate the feasibility of intra-arterial chemotherapy in a large referral center in Mexico City. METHODS: We included patients with bilateral retinoblastoma, one enucleation, and active disease in the other eye after at least two courses of systemic chemotherapy combined with topical treatments. All patients were treated with three courses of a combination of melphalan 4 mg and topotecan 1 mg. Patients were examined under general anesthesia three weeks after each chemotherapy cycle. RESULTS: From 14 eligible patients, three could not be treated due to inaccessibility of the ophthalmic artery. A complete response was observed in 5/11 patients, three in Stage C according to the International Classification for Intraocular Retinoblastoma, one in Stage D, and one in Stage B. The eyes of three patients were enucleated as a result of active/progressive disease, one in Stage B and two in Stage D. Eye preservation was 55% after a mean follow-up of 171 days (range 21-336). CONCLUSIONS: Super-selective intra-arterial chemotherapy is safe and effective for preventing the enucleation of 55% of affected eyes in this group of patients.

Vascular malformations in pediatric patients: 10-year experience of a vascular anomalies clinic.

BACKGROUND: Vascular malformations (VaMs) are caused by errors in vascular morphogenesis. Diagnosis and treatment can be complex. Few specialized centers care for these patients, and limited literature exists regarding their characteristics and clinical course. The vascular anomalies clinic (VAC) at the Instituto Nacional de Pediatría (National Institute for Pediatrics) is a multidisciplinary team and has been a reference center for patients with VaMs since 2012. We sought to describe the characteristics of patients cared for at the VAC, types of VaMs, treatments used, and clinical course. METHODS: This was a descriptive, observational, retrospective, and cross-sectional study conducted from 2012 to 2022. RESULTS: We included 435 patients with VaMs; the median age of presentation was 1 month. The most frequent signs and symptoms were increased volume (97.2%), superficial color change (65.5%), and pain (43.3%). The most common VaMs were lymphatic (36.7%) and venolymphatic (18.3%). Sclerotherapy was the most frequent treatment (73.4%), followed by medical treatment with sirolimus (18.5%); response to both was excellent/good in > 85% of cases. CONCLUSION: In this retrospective study of children with VaMs, we found that low-flow malformations were the most common, and sclerotherapy and sirolimus were the most frequently used treatments. The therapeutic response was excellent/good in most cases.

INTRODUCCIÓN: Las malformaciones vasculares (MaV) son secundarias a errores en la morfogénesis vascular. El diagnóstico y tratamiento puede ser complejo. Existen pocos centros especializados en su atención y escasa literatura respecto a características y evolución clínica. La Clínica de Anomalías Vasculares (CAV) del Instituto Nacional de Pediatría es un equipo multidisciplinario y centro de referencia para estos pacientes desde 2012. Buscamos describir las características de los pacientes atendidos en la CAV, tipo de MaV, tratamiento y evolución clínica. MÉTODOS: Estudio descriptivo, observacional, retrospectivo y transversal del periodo 2012 al 2022. RESULTADOS: Se incluyeron 435 pacientes con MaV, con edad mediana de presentación de 1 mes de vida. Los síntomas y signos más reportados fueron aumento de volumen (97.2%), cambio en coloración de la piel (65.5%) y dolor (43.3%). Las MaV más comunes fueron linfáticas (36.7%), siguiéndoles las venolinfáticas (18.3%). La escleroterapia fue el tratamiento más frecuente (73.4%) y el tratamiento médico más utilizado fue sirolimus (18.5%), ambos con excelente/buena respuesta en > 85% de los pacientes. CONCLUSIONES: En este estudio retrospectivo de niños con MaV encontramos que las más frecuentes son de bajo flujo y el tratamiento más usado escleroterapia y sirolimus. La respuesta terapéutica de la mayoría fue excelente/buena.

Experiencia en el manejo de malformaciones linfáticas macroquísticas con alcohol y microquísticas con bleomicina / Experience In the Management of Macrocystic Lymphatic Malformations with Alcohol and Microcystic Malformations with Bleomycin

Las malformaciones linfáticas son anomalías raras de flujo lento y bajo que se presentan en 1 de 6.000 a 1 en 16.000 recién nacidos vivos. Las malformaciones quísticas se clasifican según su tamaño en macroquísticas, microquísticas o mixtas. Esta clasificación tiene impacto sobre el tratamiento y el pronóstico. Las malformaciones linfáticas macroquísticas tienen mejor respuesta al tratamiento, mientras que las microquísticas son difíciles de tratar y con frecuencia recidivan. El objetivo de este trabajo es describir los resultados obtenidos en pacientes con malformaciones linfáticas macro y microquísticas intervenidos con escleroterapia utilizando alcohol y bleomicina, respectivamente. Se realizó un estudio descriptivo de pacientes con malformaciones linfáticas tratadas en el Servicio de Radiología. Se incluyeron 38 pacientes, de los cuales 24 tenían lesiones macroquísticas, 10, microquísticas y 4, mixtas. El 68 % fueron tratados con alcohol y el 32 % con bleomicina. El tratamiento mostró una resolución excelente en 5 pacientes, 25 tuvieron reducción del tamaño de la lesión entre 50 y 90 %, 13 % mostró una respuesta pobre, y solo en un 7 % hubo crecimiento, a pesar del tratamiento esclerosante. De lo anterior se concluye que el tratamiento percutáneo es seguro, reduce el tamaño de las lesiones y hay pocas complicaciones informadas. Se podría utilizar como tratamiento inicial, antes de considerar una cirugía.

Lymphatic malformations are rare slow and low flow abnormalities that occur in 1 of 6,000 to 1 in 16,000 live newborns. Cystic malformations are classified according to their size as macrocystic, microcystic, or mixed. This classification has an impact on treatment and prognosis. Macrocystic lymphatic malformations have a better response to treatment, while microcystic malformations are difficult to treat and frequently recur. The objective of this study is to describe the results obtained in patients with macro and microcystic lymphatic malformations who underwent sclerotherapy using alcohol and Bleomycin, respectively. A descriptive study of patients with lymphatic malformations treated in the Radiology Service of the National Institute of Pediatrics was carried out during the period from 2014 to 2016. Thirty-eight patients were included, 24 with macrocystic, 10 microcystic and 4 mixed lesions. 68% were treated with alcohol and 32% of the patients were treated with Bleomycin. The treatment showed excellent resolution in 5 patients, 25 patients had a lesion size reduction between 50 and 90%, 13% had a poor response, and only 7% had growth despite sclerosing treatment. From the above we conclude that percutaneous treatment is safe, reduces the size of the lesions and there are few reported complications. It could be used as initial treatment before considering surgery.