Efficacy of sirolimus in the treatment of microcystic lymphatic malformation of the tongue

Ghariani, N.; Boussofara, L.; Gammoudi, R.; Belajouza, C.; Ghariani, Najet; Denguezli, M.

doi:10.1111/jdv.15628

Cited by 10 publications

(12 citation statements)

References 10 publications

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“…Occurrence of bacterial pneumonia during sirolimus therapy was only reported in one of all investigated cases [ 27 ], the causative bacterium was not reported. The sirolimus therapy was withheld in this case, and the girl was treated with a 2-week course of imipenem [ 27 ].…”

Section: Resultsmentioning

confidence: 99%

“…Zhang reported to use a reduced starting dose in neonates (0.5 mg/m 2 ). Other starting doses were 1–2 mg per day [ 31 ], 0.1 mg/kg per day [ 35 ], 0.05 mg/kg twice daily [ 16 ], 0.6 mg/m 2 per twice daily dose [ 33 ], 0.07 mg/kg twice daily [ 18 ], 0.4–0.8 mg/m 2 twice daily [ 20 ] or in two fractions [ 27 ], and 1.5 mg/kg/day administered in two divided daily doses [ 37 ]. In five publications, the initial dose of sirolimus was not reported [ 12 , 14 , 15 , 22 , 38 ].…”

Section: Resultsmentioning

confidence: 99%

“…The target blood level was 10–15 ng/mL in seven studies [ 14 , 17 , 20 , 23 , 24 , 26 , 39 ], 5–15 ng/mL in four studies [ 16 , 28 , 31 , 34 ] and 4–12 ng/mL in two studies [ 25 , 30 ]. In one study each, 3.5–6 ng/mL [ 30 ], 4–8 ng/mL [ 21 ], 4–10 ng/mL [ 38 ], 4–13 ng/mL [ 36 ], 5–12 ng/mL [ 33 ], 10–12 ng/mL [ 27 ], 10–13 ng/mL [ 19 ] and 12–20 ng/mL [ 35 ] were the target concentrations. Eight studies did not report the target blood level of sirolimus [ 12 , 13 , 15 , 18 , 22 , 29 , 32 , 37 ].…”

Section: Resultsmentioning

confidence: 99%

“…However, anti-infectious prophylactic measures were not standardized as data are lacking. Occurrence of bacterial pneumonia during sirolimus therapy was only reported in one of the presented patients [ 27 ], however, the causative bacterium was not reported. It is suspected that higher levels of sirolimus or concomitant medications such as corticosteroids may be a risk factor for Pneumocystis pneumonia [ 25 ] and therefore selected patients may benefit from Pneumocystis prophylaxis while on sirolimus.…”

Section: Discussionmentioning

confidence: 99%

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Efficacy of sirolimus in children with lymphatic malformations of the head and neck

Wiegand

Dietz

Wichmann

2022

Eur Arch Otorhinolaryngol

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Purpose Children with extensive lymphatic malformations of the head and neck often suffer from functional impairment and aesthetic deformity which significantly affect the quality of life and may be life-threatening. Treatment with sirolimus has the potential to improve symptoms and downsize lymphatic malformations. This systematic review summarizes the current information about sirolimus treatment of lymphatic malformations of the head and neck in children, its efficacy and side effects. Methods A systematic search of the literature regarding studies on sirolimus treatment of children with lymphatic malformations of the head and neck was performed in PubMed, Embase, and Google Scholar up to July 2021 with the search terms “lymphatic malformation”, “lymphangioma”, “cystic hygroma”, “low-flow malformation”, “sirolimus”, “rapamycin”, “mTOR inhibitor” and “children”. Results In all, 28 studies including 105 children from newborn to 17 years treated with sirolimus for lymphatic malformations of the head and neck were analyzed. The most frequent initial dose was 0.8 mg/m2 per dose, twice daily at 12-h interval. The target blood level differed between studies, 10–15 ng/mL and 5–15 ng/mL were most often used. More than 91% of the children responded to sirolimus treatment which lasts from 6 months to 4 years. Typical side effects were hyperlipidemia, neutropenia and infections. Methods Sirolimus could be an effective treatment for children with large complicated lymphatic malformations of the head and neck. As not all patients will benefit from treatment, the decision to treat sirolimus should be made by a multidisciplinary team.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Efficacy of sirolimus in children with lymphatic malformations of the head and neck

Wiegand

Dietz

Wichmann

2022

Eur Arch Otorhinolaryngol

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“…17,20 Most recently, oral sirolimus, which can inhibit lymphatic vessel regeneration, invasion, LAM growth, and vascular endothelial growth factor VEGF secretion, has been reported to be efficacious for patients with lymphangioma of the tongue. 17,21 As an immunosuppressant, sirolimus can cause neutropenia and may predispose the patient to an increased risk of infection. 22 F I G U R E 5 Appearance of the maxillary anterior region after extraction of the primary incisor at 3 months follow-up Ideally, all dental procedures should be completed before immunosuppressive therapy is initiated but when the dental needs cannot be treated before therapy, priorities should be infections, extractions, and periodontal care.…”

Section: Discussionmentioning

confidence: 99%

Oral manifestations and dental care management of a young patient with lymphangioma of the tongue: A case report

Chouchène

Masmoudi

Baaziz

et al. 2021

Clinical Case Reports

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Efficacy of Initial Sirolimus Therapy for 27 Patients with Intractable Lymphatic Malformations

et al. 2021

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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 < .05). Adverse drug reactions totaled 27 events in ten patients, the majority being mild with upper respiratory infections being most common.Conclusions: Sirolimus as initial therapy is effective in decreasing lesion volume in intractable LMs in head and neck region, especially in macrocystic subtypes. Although most cases cannot be completely cured, side effects are few and tolerable.

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Efficacy of sirolimus in the treatment of microcystic lymphatic malformation of the tongue

Cited by 10 publications

References 10 publications

Efficacy of sirolimus in children with lymphatic malformations of the head and neck

Efficacy of sirolimus in children with lymphatic malformations of the head and neck

Oral manifestations and dental care management of a young patient with lymphangioma of the tongue: A case report

Efficacy of Initial Sirolimus Therapy for 27 Patients with Intractable Lymphatic Malformations

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