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Intermittent Administration Regimen of Sirolimus for Refractory Cervicofacial Lymphatic Malformation
Qingwen Gao1, Haini Chen, Buhao Sun
1Department of Burns and Plastic Surgery, Children's Hospital of Nanjing Medical University, Nanjing, China.
Background:
The cervicofacial lymphatic malformations (LMs) often have poor outcomes due to their microcystic component and diffuse infiltration. Mostly, traditional treatments are inadequate for these refractory cases. Recent researches have shown that sirolimus is effective in the treatment of complicated LMs, however, there is still no standard strategy.
Objective:
To evaluate the efficacy and safety of intermittent oral sirolimus in treating refractory cervicofacial LMs as a second-line treatment.
Methods:
Fifteen pediatric patients of refractory cervicofacial LMs were retrospectively analyzed in this study. All the cases had received traditional therapy before, but could not completely control the symptoms and eliminate lesions. As a remedy, sirolimus was then proceeded with an intermittent administration regimen, that is 3 continuous months as a course and started the next course after 1 month interval. The clinical characteristics, imaging data of patients, the changes in the signs and symptoms observed, and associated adverse effects were collected and analyzed.
Results:
The patients initiated sirolimus therapy at the average age of 2.3 years (range 28 days-8 years 9 months). At the end point of the study, 2 patients remained on sirolimus in continuous courses of treatment. Of 13 patients who withdrawn therapy, 4 had restarted due to recurrence of symptoms and re-expansion of LMs. All patients demonstrated reduction in residual LMs and complete disappearance of symptoms during treatment, and 2 patients with complete resolution on imaging. Toxicity was tolerant in this series. There was no patient develop opportunistic or systemic bacterial infection.
Conclusions:
Sirolimus is commended as a second-line treatment to treat intractable cervicofacial LMs after failure of traditional therapy. The intermittent administration regimen is efficacious to completely control symptoms and partially reduce residual lesions with good tolerance and limited side effects.
Insights
Intermittent oral sirolimus effectively manages refractory cervicofacial lymphatic malformations (LMs) in children. This treatment controls symptoms and reduces lesions with good tolerance, offering a viable second-line option.
Area of Science:
- Vascular Anomalies
- Pediatric Surgery
- Pharmacology
Background:
- Cervicofacial lymphatic malformations (LMs) present treatment challenges due to microcystic components and diffuse infiltration.
- Traditional therapies are often inadequate for refractory cases of cervicofacial LMs.
- Sirolimus shows promise for complicated LMs, but standardized treatment strategies are lacking.
Purpose of the Study:
- To assess the efficacy and safety of intermittent oral sirolimus as a second-line therapy for refractory cervicofacial LMs.
- To evaluate the outcomes of sirolimus treatment in pediatric patients with persistent LMs.
Main Methods:
- Retrospective analysis of 15 pediatric patients with refractory cervicofacial LMs previously treated with traditional therapies.
- Sirolimus administered via an intermittent regimen: 3 months on, 1 month off.
- Collection and analysis of clinical, imaging, symptomatic, and adverse effect data.
Main Results:
- All patients experienced symptom resolution and reduction in residual LMs during treatment; 2 showed complete imaging resolution.
- Sirolimus therapy was generally well-tolerated, with no opportunistic or systemic bacterial infections reported.
- Four patients restarted therapy due to recurrence after initial withdrawal, indicating potential for long-term symptom control.
Conclusions:
- Intermittent oral sirolimus is a recommended second-line treatment for intractable cervicofacial LMs following traditional therapy failure.
- The intermittent regimen effectively controls symptoms and partially reduces lesions with good tolerance and limited side effects.
- Sirolimus offers a promising therapeutic option for managing complex pediatric cervicofacial lymphatic malformations.
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