Efficacy of Initial Sirolimus Therapy for 27 Patients with Intractable Lymphatic Malformations
| Autor: | Yongli Guo, Jun Tai, Zhiyong Liu, Xiaoling Cheng, Nian Sun, Yanzhen Li, Xin Ni, Jie Zhang, Xiaoling Wang, Xiaodan Li, Shengcai Wang, Yuwei Liu, Yuanhu Liu, Qiaoyin Liu, Jiangnan Du, Xuexi Zhang |
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| Rok vydání: | 2021 |
| Předmět: |
Male
medicine.medical_specialty Adolescent Administration Oral Lesion volume 03 medical and health sciences 0302 clinical medicine 030225 pediatrics Internal medicine medicine Humans Prospective Studies Drug reaction Lymphatic malformations Child Adverse effect Head and neck Sirolimus Lymphatic Abnormalities business.industry Sirolimus therapy Infant Newborn Infant Treatment Outcome Otorhinolaryngology Child Preschool 030220 oncology & carcinogenesis Female Primary treatment Drug Monitoring business Head Neck medicine.drug |
| Zdroj: | The Laryngoscope. 131:1902-1908 |
| ISSN: | 1531-4995 0023-852X |
| DOI: | 10.1002/lary.29419 |
| Popis: | OBJECTIVE/HYPOTHESIS To evaluate the efficacy of initial sirolimus therapy in the treatment of intractable head and neck lymphatic malformations (LMs) in children. STUDY DESIGN Prospective open-label study. METHODS In this study, Twenty-seven children diagnosed with LMs were given oral sirolimus as primary treatment over a minimum 6-month trial. The major parameter to evaluate therapeutic outcome was percentage of lesion volume change compared with baseline. Average serum sirolimus concentrations, and adverse side effects, were monitored throughout the study period. RESULTS Fifteen girls and twelve boys, average age 27 months (16 days-171 months), constitute the study group. Treatment was deemed effective for twenty-three participants, judged as fair in seven, good in nine, and excellent in seven. Two patients had minimal improvement, and two had increased volume to some degree. Effectiveness differed among LMs subtypes with responsiveness of macrocystic LMs exceeding that of microcystic LMs (P |
| Databáze: | OpenAIRE |
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