This Article Full Text Full Text (PDF) Submit a related Letter to the Editor Alert me when this article is cited Alert me when eLetters are posted Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Request Copyright Permission Citing Articles Citing Articles via HighWire Citing Articles via Google Scholar Google Scholar Articles by Galland, F. Articles by Chanson, P. Search for Related Content PubMed PubMed Citation Articles by Galland, F. Articles by Chanson, P. Related Collections Neuroendocrinology and Pituitary

McCune-Albright Syndrome and Acromegaly: Effects of Hypothalamopituitary Radiotherapy and/or Pegvisomant in Somatostatin Analog-Resistant Patients

Assistance Publique-Hôpitaux de Paris, Centre de Référence des Maladies Endocriniennes Rares de la Croissance, Service d’Endocrinologie et des Maladies de la Reproduction (F.G., P.K., H.A., J.Y., P.C.), Hôpital de Bicêtre, 94275 Le Kremlin-Bicêtre, France; Service d’Endocrinologie (Y.R.), Centre Hospitalier Universitaire Côte de Nacre, 14033 Caen, France; Département de Radiothérapie (D.P.), Institut Curie, 75005 Paris, France; Institut National de la Santé et de la Recherche Médicale U693 (P.K., J.Y., P.C.), 94276 Le Kremlin-Bicêtre, France; Assistance Publique-Hôpitaux de Paris, Laboratoire d’Explorations Fonctionnelles Endocriniennes (Y.L.B.), Hôpital Trousseau, 75012 Paris, France; and Université Paris-Sud 11 (J.Y., P.C.), 94275 Le Kremlin-Bicêtre, France

Address all correspondence and requests for reprints to: Philippe Chanson, M.D., Service d’Endocrinologie et des Maladies de la Reproduction, Hôpital de Bicêtre, 78 rue du Général Leclercqq, 94275 Le Kremlin-Bicêtre, France. E-mail: philippe.chanson{at}bct.aphp.fr.

Background: Acromegaly, which may be present in patients with McCune-Albright syndrome (MCAS), in association with café-au-lait spots, precocious puberty, and fibrous dysplasia, is often difficult to treat surgically because skull base bone dysplasia prevents the removal of the pituitary adenoma. Somatostatin analogs (SAs) generally give only partial responses. The use of radiotherapy (RT) is controversial because of a possible risk of bone sarcomatous transformation.

Aim: This study was a retrospective analysis of the efficacy and adverse effects of different treatment modalities in six patients with both MCAS and acromegaly.

Patients and Methods: Because surgery was impossible and SA failed to normalize GH/IGF-I hypersecretion, five of the six patients received fractionated RT (45–55 Grays). Three patients (two with previous RT) were also prescribed pegvisomant. We analyzed the clinical features of acromegaly, GH, and IGF-I concentrations and bone radiological features.

Results: GH and IGF-I concentrations fell after RT (median follow-up, 5 yr; range, 0.5–9 yr). Symptoms of acromegaly improved in parallel. Bone sarcomatous transformation was only noted in one patient in a region (the mandible) outside the radiation field. RT alone and/or combined with SA failed to normalize GH/IGF-I levels in the five patients concerned. In contrast, IGF-I levels normalized very rapidly (5–9 months) in the three patients receiving pegvisomant (10–20 mg/d).

Conclusion: RT may be an option for the treatment of acromegaly in patients with MCAS when surgery is impossible and SA therapy is ineffective. However, although no bone sarcomatous transformation was observed within the radiation field in this series, this risk cannot be ruled out. As shown in this small series of severely affected patients, pegvisomant therapy may thus be useful to normalize IGF-I levels rapidly.

This article has been cited by other articles:

				S. A. Boikos and C. A. Stratakis Molecular genetics of the cAMP-dependent protein kinase pathway and of sporadic pituitary tumorigenesis Hum. Mol. Genet., April 15, 2007; 16(R1): R80 - R87. [Abstract] [Full Text] [PDF]