Efficacy of hormonal manipulation in lymphangioleiomyomatosis. A 20-year-experience in 36 patients.
BACKGROUND
Lymphangioleiomyomatosis (LAM) is a rare disease of unknown aetiology which mainly affects women of reproductive age. A growing body of evidence suggests a relation between the disease and estrogenic hormones. In particular, pregnancy and use of estrogens favour the progression of the disease, which is in turn slowed down by menopause. These observations suggested already in the past a treatment strategy based on hormonal manipulation: discontinuation of estrogens containing medications, avoiding pregnancy, inhibition of estrogens activity and induction of menopause.


OBJECTIVE
In the present study, conducted on a relatively large population for a rare disease, we sought to show the hormonal manipulation's positive effects for the survival rate.


METHODS
36 women suffering from LAM were evaluated between January 1985 and March 2005. All our patients were treated with hormonal therapy, following different schemes. The response to the treatment was evaluated in all patients with arterial blood sampling, lung function tests (total body plethysmography, DLCO), measurement of the hormones in the blood, chest and abdomen CT scan.


RESULTS
The survival rate since the clinical onset was 97% at the 5 year timepoint, 90% at 10 years, and 71% at 25 years. Previous studies, conducted before the introduction of the hormonal treatment showed a survival rate of 20% at 10 years.


CONCLUSIONS
Our results highlight the role of the hormonal manipulation as a mainstay in the treatment of LAM, with the capability of reducing mortality and improving the quality of life as well.
Schiavina
Mario
M
Respiratory Pathophysiology Unit, S. Orsola-Malpighi Hospital, Via Massarenti 9 40138 Bologna, Italy. mschiavina@aosp.bo.it
Contini
Paola
P
Fabiani
Andrea
A
Cinelli
Franca
F
Di Scioscio
Valerio
V
Zompatori
Maurizio
M
Campidelli
Cristina
C
Pileri
Stefano A
SA
D000328:Adult; D004965:Estrogen Antagonists / Q000008:administration & dosage / Q000627:therapeutic use*; D004967:Estrogens / Q000378:metabolism*; D005260:Female; D006728:Hormones / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D016729:Leuprolide / Q000008:administration & dosage / Q000627:therapeutic use; D008186:Luteolytic Agents / Q000008:administration & dosage / Q000627:therapeutic use; D018192:Lymphangioleiomyomatosis / Q000188:drug therapy / Q000503:physiopathology* / Q000601:surgery / Q000628:therapy*; D017258:Medroxyprogesterone Acetate / Q000008:administration & dosage / Q000627:therapeutic use; D008593:Menopause; D008875:Middle Aged; D010052:Ovariectomy; D012129:Respiratory Function Tests; D017329:Triptorelin Pamoate / Q000008:administration & dosage / Q000627:therapeutic use
D004965:Estrogen Antagonists; D004967:Estrogens; D006728:Hormones; D008186:Luteolytic Agents; D017329:Triptorelin Pamoate; D017258:Medroxyprogesterone Acetate; D016729:Leuprolide