Use of Bisphosphonates in GLA/GSD/KLA

Presented by Yaser A. Diab, M.B.B.S., Children’s National Health System, Washington, DC

Complex lymphatic anomalies including Generalized Lymphatic Anomaly (GLA), Gorham–Stout disease (GSD), and Kaposiform Lymphangiomatosis (KLA) can involve the bones leading to bone destruction which can negatively impact the quality of life for patients with these lymphatic anomalies. At present, no standard treatment for these diseases exist, and due to the rarity of these diseases, no clinical trials are available. Sirolimus has been shown to be effective in the treatment of various types of lymphatic anomalies. Bisphosphonates are medications that can slow down normal bone destruction and are commonly used to treat bone diseases, such as thin or fragile bones. Administration of bisphosphonates may help in treating the bone disease in patients with GLA, GSD or KLA. In addition, there is some evidence that suggests that bisphosphonates may enhance the effect of sirolimus. New treatment regimens that include sirolimus in combination with a bisphosphonate have been recently introduced and used for treatment of refractory or high risk GLA, GSD and KLA.

About the Presenter

Dr. Diab is an Attending Hematologist, Assistant Professor of Pediatrics, and the Director of the Thrombosis Program Center for Cancer and Blood Disorders at Children’s National Health System in Washington, DC. Children’s National serves patients in Washington, DC; Maryland; and Virginia. Among Dr. Diab’s interests are rare vascular anomalies.

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