Abstract
Rationale: Lymphatic malformations (LMs) are rare and benign anomalies resulting from the defective embryological development of the primordial lymphatic structures. Due to their permeative growth throughout all tissue layers, treatment is often challenging. Small asymptomatic lesions can be conservatively managed, while symptomatic lesions require active management. Surgery has been historically considered the treatment of choice, but today less invasive therapeutic options are preferred (sclerotherapy, laser therapy, oral medications). However, there are not uniform therapeutic protocols. Sirolimus is an oral medication that has been reported to be effective in the recent literature. Here we present the case of 4 newborns with giant multicystic lymphangioma treated with oral sirolimus after surgical resection had failed. Patient concerns: At birth the LMs were clinically appreciated as giant masses involving different organs and structures. Diagnoses: All patients had a prenatal diagnosis of giant multicystic lymphangioma confirmed at histological and cytological analysis. Interventions: Patients were treated with oral sirolimus after unsuccessful surgical resection. Outcomes: In all patients, sirolimus determined an overall reduction of the mass and a global involution from the macro-to the microcystic composition. Sirolimus was safe and poor disadvantages had been observed. The main and isolated adverse effect at laboratory analysis was progressive dyslipidemia, with increasing levels of total cholesterol and triglycerides. Lessons: To date, our experience with sirolimus in the management of LMs is favorable. We recommend the use of sirolimus after unsuccessful surgical excision have been tried or when the surgical approach is not feasible. A multidisciplinary follow-up is needed to monitor disease evolution.
| Original language | English |
|---|---|
| Article number | e8871 |
| Journal | Medicine (United States) |
| Volume | 96 |
| Issue number | 51 |
| DOIs | |
| Publication status | Published - Dec 1 2017 |
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Keywords
- cystic lymphangioma
- lymphangiomatosis
- lymphatic abnormalities
- neonate
- sirolimus
ASJC Scopus subject areas
- Medicine(all)
Cite this
The use of sirolimus in the treatment of giant cystic lymphangioma : Four case reports and update of medical therapy. / Amodeo, Ilaria; Colnaghi, Mariarosa; Raffaeli, Genny; Cavallaro, Giacomo; Ciralli, Fabrizio; Gangi, Silvana; Leva, Ernesto; Pignataro, Lorenzo; Borzani, Irene; Pugni, Lorenza; Mosca, Fabio.
In: Medicine (United States), Vol. 96, No. 51, e8871, 01.12.2017.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - The use of sirolimus in the treatment of giant cystic lymphangioma
T2 - Four case reports and update of medical therapy
AU - Amodeo, Ilaria
AU - Colnaghi, Mariarosa
AU - Raffaeli, Genny
AU - Cavallaro, Giacomo
AU - Ciralli, Fabrizio
AU - Gangi, Silvana
AU - Leva, Ernesto
AU - Pignataro, Lorenzo
AU - Borzani, Irene
AU - Pugni, Lorenza
AU - Mosca, Fabio
PY - 2017/12/1
Y1 - 2017/12/1
N2 - Rationale: Lymphatic malformations (LMs) are rare and benign anomalies resulting from the defective embryological development of the primordial lymphatic structures. Due to their permeative growth throughout all tissue layers, treatment is often challenging. Small asymptomatic lesions can be conservatively managed, while symptomatic lesions require active management. Surgery has been historically considered the treatment of choice, but today less invasive therapeutic options are preferred (sclerotherapy, laser therapy, oral medications). However, there are not uniform therapeutic protocols. Sirolimus is an oral medication that has been reported to be effective in the recent literature. Here we present the case of 4 newborns with giant multicystic lymphangioma treated with oral sirolimus after surgical resection had failed. Patient concerns: At birth the LMs were clinically appreciated as giant masses involving different organs and structures. Diagnoses: All patients had a prenatal diagnosis of giant multicystic lymphangioma confirmed at histological and cytological analysis. Interventions: Patients were treated with oral sirolimus after unsuccessful surgical resection. Outcomes: In all patients, sirolimus determined an overall reduction of the mass and a global involution from the macro-to the microcystic composition. Sirolimus was safe and poor disadvantages had been observed. The main and isolated adverse effect at laboratory analysis was progressive dyslipidemia, with increasing levels of total cholesterol and triglycerides. Lessons: To date, our experience with sirolimus in the management of LMs is favorable. We recommend the use of sirolimus after unsuccessful surgical excision have been tried or when the surgical approach is not feasible. A multidisciplinary follow-up is needed to monitor disease evolution.
AB - Rationale: Lymphatic malformations (LMs) are rare and benign anomalies resulting from the defective embryological development of the primordial lymphatic structures. Due to their permeative growth throughout all tissue layers, treatment is often challenging. Small asymptomatic lesions can be conservatively managed, while symptomatic lesions require active management. Surgery has been historically considered the treatment of choice, but today less invasive therapeutic options are preferred (sclerotherapy, laser therapy, oral medications). However, there are not uniform therapeutic protocols. Sirolimus is an oral medication that has been reported to be effective in the recent literature. Here we present the case of 4 newborns with giant multicystic lymphangioma treated with oral sirolimus after surgical resection had failed. Patient concerns: At birth the LMs were clinically appreciated as giant masses involving different organs and structures. Diagnoses: All patients had a prenatal diagnosis of giant multicystic lymphangioma confirmed at histological and cytological analysis. Interventions: Patients were treated with oral sirolimus after unsuccessful surgical resection. Outcomes: In all patients, sirolimus determined an overall reduction of the mass and a global involution from the macro-to the microcystic composition. Sirolimus was safe and poor disadvantages had been observed. The main and isolated adverse effect at laboratory analysis was progressive dyslipidemia, with increasing levels of total cholesterol and triglycerides. Lessons: To date, our experience with sirolimus in the management of LMs is favorable. We recommend the use of sirolimus after unsuccessful surgical excision have been tried or when the surgical approach is not feasible. A multidisciplinary follow-up is needed to monitor disease evolution.
KW - cystic lymphangioma
KW - lymphangiomatosis
KW - lymphatic abnormalities
KW - neonate
KW - sirolimus
UR - http://www.scopus.com/inward/record.url?scp=85039695349&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85039695349&partnerID=8YFLogxK
U2 - 10.1097/MD.0000000000008871
DO - 10.1097/MD.0000000000008871
M3 - Article
VL - 96
JO - Medicine; analytical reviews of general medicine, neurology, psychiatry, dermatology, and pediatries
JF - Medicine; analytical reviews of general medicine, neurology, psychiatry, dermatology, and pediatries
SN - 0025-7974
IS - 51
M1 - e8871
ER -