Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report
Background Pediatric vascular anomalies comprise a heterogeneous group of clinical disorders of varying severity. These anomalies are divided into vascular tumors or vascular malformations. Different therapeutic strategies have been used depending on the pathology in question. In recent years, mamma...
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| Format: | Article |
| Language: | English |
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Mashhad University of Medical Sciences
2018-01-01
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| Series: | International Journal of Pediatrics |
| Subjects: | |
| Online Access: | http://ijp.mums.ac.ir/article_9994_d4160839760b332a5d27ba96611d9d8c.pdf |
| _version_ | 1798041807168208896 |
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| author | Maria Jesus Garcia Verde Cristina Martinez Roca Pedro Yanez Gomez Maria Isabel Martin Herranz |
| author_facet | Maria Jesus Garcia Verde Cristina Martinez Roca Pedro Yanez Gomez Maria Isabel Martin Herranz |
| author_sort | Maria Jesus Garcia Verde |
| collection | DOAJ |
| description | Background Pediatric vascular anomalies comprise a heterogeneous group of clinical disorders of varying severity. These anomalies are divided into vascular tumors or vascular malformations. Different therapeutic strategies have been used depending on the pathology in question. In recent years, mammalian target of rapamycin (mTOR) inhibitors have been explored as a therapeutic option in patients with complex vascular anomalies that fail to respond to conventional therapies. Case Report Two patients affected by complicated vascular anomalies were treated with sirolimus. Case 1: fourteen-month-old male patient with primary congenital lymphedema in the right arm. Case 2: boy aged two years and eight months, diagnosed with a non-respectable cervical aposiform hemangioendothelioma (KHE). Both patients received sirolimus at doses of 0.8 mg/m2/12 h during 12 and 13 months respectively. Monitoring plasma sirolimus concentrations made it possible to safely deal with problems associated with the medication. Both patients experiment an important improvement of their pathology, maintained after suspending treatment. No adverse effects related to treatment with sirolimus were observed. Conclusion Oral sirolimus proved to be an effective strategy in the treatment of complicated vascular anomalies in our pediatric population. |
| first_indexed | 2024-04-11T22:26:46Z |
| format | Article |
| id | doaj.art-b2a99fe45a9e45218bde574c3f5ca238 |
| institution | Directory Open Access Journal |
| issn | 2345-5047 2345-5055 |
| language | English |
| last_indexed | 2024-04-11T22:26:46Z |
| publishDate | 2018-01-01 |
| publisher | Mashhad University of Medical Sciences |
| record_format | Article |
| series | International Journal of Pediatrics |
| spelling | doaj.art-b2a99fe45a9e45218bde574c3f5ca2382022-12-22T03:59:38ZengMashhad University of Medical SciencesInternational Journal of Pediatrics2345-50472345-50552018-01-01616873687710.22038/ijp.2017.26943.23219994Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case ReportMaria Jesus Garcia Verde0Cristina Martinez Roca1Pedro Yanez Gomez2Maria Isabel Martin Herranz3Hospital Pharmacy Specialist, Hospital Pharmacy Department, Complexo Hospitalario Universitario, A Coruña, Spain.Hospital Pharmacy Specialist, Hospital Pharmacy Department, Complexo Hospitalario Universitario, A Coruña, Spain.Hospital Pharmacy Specialist, Hospital Pharmacy Department, Complexo Hospitalario Universitario, A Coruña, Spain.Hospital Pharmacy Specialist, Hospital Pharmacy Department, Complexo Hospitalario Universitario, A Coruña, Spain.Background Pediatric vascular anomalies comprise a heterogeneous group of clinical disorders of varying severity. These anomalies are divided into vascular tumors or vascular malformations. Different therapeutic strategies have been used depending on the pathology in question. In recent years, mammalian target of rapamycin (mTOR) inhibitors have been explored as a therapeutic option in patients with complex vascular anomalies that fail to respond to conventional therapies. Case Report Two patients affected by complicated vascular anomalies were treated with sirolimus. Case 1: fourteen-month-old male patient with primary congenital lymphedema in the right arm. Case 2: boy aged two years and eight months, diagnosed with a non-respectable cervical aposiform hemangioendothelioma (KHE). Both patients received sirolimus at doses of 0.8 mg/m2/12 h during 12 and 13 months respectively. Monitoring plasma sirolimus concentrations made it possible to safely deal with problems associated with the medication. Both patients experiment an important improvement of their pathology, maintained after suspending treatment. No adverse effects related to treatment with sirolimus were observed. Conclusion Oral sirolimus proved to be an effective strategy in the treatment of complicated vascular anomalies in our pediatric population.http://ijp.mums.ac.ir/article_9994_d4160839760b332a5d27ba96611d9d8c.pdfPediatric PatientsSirolimusVascular Anomalies |
| spellingShingle | Maria Jesus Garcia Verde Cristina Martinez Roca Pedro Yanez Gomez Maria Isabel Martin Herranz Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report International Journal of Pediatrics Pediatric Patients Sirolimus Vascular Anomalies |
| title | Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report |
| title_full | Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report |
| title_fullStr | Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report |
| title_full_unstemmed | Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report |
| title_short | Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report |
| title_sort | use of oral sirolimus in paediatric patients with serious vascular anomalies case report |
| topic | Pediatric Patients Sirolimus Vascular Anomalies |
| url | http://ijp.mums.ac.ir/article_9994_d4160839760b332a5d27ba96611d9d8c.pdf |
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