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Sirolimus Therapy for Patients With Lymphangioleiomyomatosis Leads to Loss of Chylous Ascites and Circulating LAM Cells

Sergio Harari, MD; Davide Elia, MD; Olga Torre, MD; Elisabetta Bulgheroni, MSc; Elena Provasi, PhD; Joel Moss, MD, PhD, FCCP
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aUnità di Pneumologia e Terapia Semi-Intensiva Respiratoria, Servizio di Fisiopatologia Respiratoria ed Emodinamica Polmonare, Ospedale San Giuseppe, MultiMedica, Istituto di Ricovero e Cura a Carattere Scientifico, Milan, Italy

bIstituto Nazionale di Genetica Molecolare “Romeo ed Enrica Invernizzi”, Milan, Italy

cCardiovascular and Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

CORRESPONDENCE TO: Davide Elia, MD, Ospedale San Giuseppe, MultiMedica, IRCCS, Unità di Pneumologia e Terapia Semi-Intensiva Respiratoria, Servizio di Fisiopatologia Respiratoria ed Emodinamica Polmonare, via S. Vittore 12, Milan 20142, Italy


Copyright 2016, American College of Chest Physicians. All Rights Reserved.


Chest. 2016;150(2):e29-e32. doi:10.1016/j.chest.2016.02.654
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A young woman received a diagnosis of abdominal, sporadic lymphangioleiomyomatosis (LAM) and multiple abdominal lymphangioleiomyomas and was referred for recurrent chylous ascites responding only to a fat-free diet. On admission, pulmonary function test (PFT) results showed a moderate reduction in the transfer factor for carbon monoxide with normal exercise performance. The serum vascular endothelial growth factor D (VEGF-D) level was 2,209 pg/mL. DNA sequences, amplified at loci kg8, D16S3395, D16S3024, D16S521, and D16S291 on chromosome 16p13.3, showed a loss of heterozygosity (LOH) only for kg8. Fat-free total parenteral nutrition in association with sirolimus (2 mg po daily) was initiated. Serum sirolimus levels were maintained at concentrations between 5 and 15 ng/mL. After 1 month, reintroduction of a low-fat oral feeding was achieved without recurrence of ascites. PFT results were stable. Interestingly, clinical improvement was associated with a reduction in the VEGF-D serum level (1,558 pg/mL). LOH at the kg8 biomarker in blood LAM cells was no longer detected.

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