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. 2021 Jun 8;16(1):260.
doi: 10.1186/s13023-021-01893-3.

Sirolimus in the treatment of kaposiform lymphangiomatosis

Jiangyuan Zhou  1 , Kaiying Yang  1 , Siyuan Chen  2 , Yi Ji  3
Affiliations

Sirolimus in the treatment of kaposiform lymphangiomatosis

Jiangyuan Zhou et al. Orphanet J Rare Dis. .

Abstract

Background: Kaposiform lymphangiomatosis (KLA), which is a new subtype of generalized lymphatic anomaly, is a rare disease with a poor prognosis. Currently, there is no standard treatment due to the poor understanding of KLA. Sirolimus, which is an inhibitor of mammalian target of rapamycin, has been shown to have promising potential in the treatment of complicated vascular anomalies. The aim of this study was to introduce the use of sirolimus for the treatment of KLA and to highlight the challenges of managing this refractory disease.

Results: We reported seven patients with KLA who received sirolimus therapy in our center. Combined with previously reported cases, 58.3% achieved a partial response, 25.0% had stable disease, and 16.7% experienced disease progression. No severe sirolimus-related adverse events occurred during treatment.

Conclusions: This study suggests that sirolimus is currently an option for the treatment of KLA, and it is hoped that more specific therapies will be developed in the future. Rapid advances in basic science and clinical practice may facilitate the development of important new treatments for KLA.

Keywords: Kaposiform lymphangiomatosis; Lymphatic malformation; Mammalian target of rapamycin; Sirolimus; Vascular anomaly.

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Conflict of interest statement

The authors declare that they have no competing interests, either financial or nonfinancial, that could be perceived as prejudicing the impartiality of the research reported.

Figures

Fig. 1
Fig. 1
Patient 1 showed a grossly distended abdomen and perineal ecchymosis (A). Axial T2 fat-saturated image showed extensive mediastinal involvement (B). Coronal T2 fat-saturated image showed extensive abdominal cavity and pelvic cavity involvement (C). Axial T2 fat-saturated image showed bilateral inguinal regions, including testis involvement (D)
Fig. 2
Fig. 2
Patient 2 presented to our hospital because of dyspnea, shortness of breath, thrombocytopenia, and coagulopathy. T2-weighted MRI of the chest showed severe pleural effusion and thoracic lesions (A). After 5 months of treatment, thoracic MRI indicated a significant decrease in pleural effusion and a reduction in lesion size (B)
Fig. 3
Fig. 3
Patient 7 presented with dyspnea and cough. Imaging tests showed extensive lesions involving the mediastinum, lungs, neck and spleen
See this image and copyright information in PMC

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