Effect of a gonadotrophin-releasing hormone analogue on lung function in lymphangioleiomyomatosis.
BACKGROUND
Lymphangioleiomyomatosis (LAM), a multisystem disease occurring primarily in women, is characterized by cystic lung destruction, and kidney and lymphatic tumors, caused by the proliferation of abnormal-appearing cells (ie, LAM cells) with a smooth muscle cell phenotype that express melanoma antigens and are capable of metastasizing. Estrogen receptors are present in LAM cells, and this finding, along with reports of disease progression during pregnancy or following exogenous estrogen administration, suggest the involvement of estrogens in the pathogenesis of LAM. Consequently, antiestrogen therapies have been employed in treatment. The goal of this prospective study was to evaluate the efficacy of triptorelin, a gonadotrophin-releasing hormone analogue, in 11 premenopausal women with LAM.


METHODS
Patients were evaluated at baseline and every 3 to 6 months thereafter, for a total of 36 months. Hormonal assays, pulmonary function tests, 6-min walk tests, high-resolution CT scans of the chest, and bone mineral density studies were performed.


RESULTS
Gonadal suppression was achieved in all patients. Overall, a significant decline in lung function was observed; two patients underwent lung transplantation 1 year after study enrollment, and another patient was lost to follow-up. Treatment with triptorelin was associated with a decline in bone mineral density.


CONCLUSIONS
Triptorelin appears not to prevent a decline in lung function in patients with LAM. Its use, however, may be associated with the loss of bone mineral density.
Harari
Sergio
S
Unità di Pneumologia e Terapia Semi-Intensiva Respiratoria, Ospedale San Giuseppe AFAR, Milan, Italy.
Cassandro
Roberto
R
Chiodini
Iacopo
I
Chiodini
Jacopo
J
Taveira-DaSilva
Angelo M
AM
Moss
Joel
J
D000328:Adult; D018931:Antineoplastic Agents, Hormonal / Q000494:pharmacology*; D015519:Bone Density / Q000187:drug effects; D005260:Female; D005541:Forced Expiratory Volume / Q000187:drug effects; D005652:Functional Residual Capacity / Q000187:drug effects; D006801:Humans; D008168:Lung / Q000187:drug effects* / Q000503:physiopathology*; D018192:Lymphangioleiomyomatosis / Q000000981:diagnostic imaging / Q000503:physiopathology*; D008297:Male; D008875:Middle Aged; D017697:Premenopause; D014057:Tomography, X-Ray Computed; D017329:Triptorelin Pamoate / Q000494:pharmacology*; D014797:Vital Capacity / Q000187:drug effects
D018931:Antineoplastic Agents, Hormonal; D017329:Triptorelin Pamoate