Treatment of desmoids and mesenteric fibromatosis in familial adenomatous polyposis with raloxifene.
BACKGROUND
Among the great variety of extracolonic manifestations of familial adenomatous polyposis, the most serious are desmoids and fibromatosis of the abdominal cavity. These may be a danger to the patient and a concern to the clinician. Pharmacological management of this relentless problem is favored by surgical intervention. At present, however, beneficial actions of medical therapy are not separable from undesirable side effects.


METHODS
We studied the effects of 120 mg daily of raloxifene, a non-steroidal benzothiophene, on progressive desmoid tumors and mesenteric fibromatosis by evaluation of lesion size and symptoms in 13 patients with familial adenomatous polyposis, selected on the basis of intra-abdominal localization of the lesion, on refractoriness to other medical treatments, and on estrogen receptor-alpha expression.


RESULTS
The patients had a significant response to raloxifene therapy, with complete remission in 8 cases and partial response in 5 cases, evaluated by regression of symptoms and tumor size. Serum biochemical parameters did not show any significant changes. Side effects were never observed.


CONCLUSIONS
Although the number of patients included in the study is limited and in spite of some limitations, the available results support that, in the evaluation of response, daily therapy with raloxifene decreases desmoid tumor and mesenteric fibromatosis size and symptoms and does not cause side effects. These findings offer a novel option in the pharmacological treatment of desmoids, leading to medical therapy of these neoplastic lesions in familial adenomatous polyposis patients.
Tonelli
Francesco
F
Department of Clinical Physiopathology, University of Florence, Italy.
Ficari
Ferdinando
F
Valanzano
Rosa
R
Brandi
Maria Luisa
ML
D011125:Adenomatous Polyposis Coli / Q000150:complications*; D000328:Adult; D018931:Antineoplastic Agents, Hormonal / Q000627:therapeutic use*; D005260:Female; D005350:Fibroma / Q000188:drug therapy* / Q000209:etiology; D018222:Desmoid Tumors / Q000188:drug therapy*; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008643:Mesentery / Q000473:pathology*; D008875:Middle Aged; D010532:Peritoneal Diseases / Q000188:drug therapy* / Q000209:etiology; D011446:Prospective Studies; D020849:Raloxifene Hydrochloride / Q000627:therapeutic use*; D020845:Selective Estrogen Receptor Modulators / Q000627:therapeutic use*; D016896:Treatment Outcome
D018931:Antineoplastic Agents, Hormonal; D020845:Selective Estrogen Receptor Modulators; D020849:Raloxifene Hydrochloride