Literature DB >> 16061823

Pegvisomant therapy in pituitary gigantism: successful treatment in a 12-year-old girl.

M Rix1, P Laurberg, A S Hoejberg, B Brock-Jacobsen.   

Abstract

OBJECTIVE: The use of a growth hormone (GH) receptor antagonist, pegvisomant has shown great promise in adults with acromegaly, but experience in paediatric patients is lacking. We aimed to describe the results of pegvisomant therapy in a 12-year-old girl with an aggressive GH-secreting pituitary tumour.
DESIGN: To evaluate the ability of pegvisomant therapy to control the effects of peripheral GH excess in a case of pituitary gigantism.
METHODS: Pegvisomant was introduced at 10 mg/day, given subcutaneously, and gradually increased to 20 mg/day until serum IGF-I was normal for age.
RESULTS: A large pituitary adenoma with suprasellar extension was diagnosed in a 12-year-old girl with progressive tall stature (178 cm), GH hypersecretion without suppression during oral glucose loading (nadir serum GH, 90 mU/l), high serum IGF-I and serum prolactin levels. Surgical extirpation was not possible because tumour tissue was fibrous and adherent to the optical nerves. Histological examination showed a mixed GH- and prolactin-secreting adenoma with lymphocytic infiltration of B and T cells. Treatment with a dopamine agonist, cabergoline, normalized serum prolactin, but GH secretion was resistant to both somatostatin analogue, octreotide and cabergoline. Radiation followed by pegvisomant therapy titrated up in dose to 20 mg/day led to a marked reduction in GH secretion and normalization of IGF-I, and to growth arrest and improvement of well-being.
CONCLUSIONS: We suggest that treatment in pituitary gigantism with pegvisomant is safe and may normalize IGF-I levels and effectively stop growing.

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Year:  2005        PMID: 16061823     DOI: 10.1530/eje.1.01956

Source DB:  PubMed          Journal:  Eur J Endocrinol        ISSN: 0804-4643            Impact factor:   6.664


  10 in total

Review 1.  Pegvisomant: a growth hormone receptor antagonist used in the treatment of acromegaly.

Authors:  Nicholas A Tritos; Beverly M K Biller
Journal:  Pituitary       Date:  2017-02       Impact factor: 4.107

2.  Clinical and genetic characterization of pituitary gigantism: an international collaborative study in 208 patients.

Authors:  Liliya Rostomyan; Adrian F Daly; Patrick Petrossians; Emil Nachev; Anurag R Lila; Anne-Lise Lecoq; Beatriz Lecumberri; Giampaolo Trivellin; Roberto Salvatori; Andreas G Moraitis; Ian Holdaway; Dianne J Kranenburg-van Klaveren; Maria Chiara Zatelli; Nuria Palacios; Cecile Nozieres; Margaret Zacharin; Tapani Ebeling; Marja Ojaniemi; Liudmila Rozhinskaya; Elisa Verrua; Marie-Lise Jaffrain-Rea; Silvia Filipponi; Daria Gusakova; Vyacheslav Pronin; Jerome Bertherat; Zhanna Belaya; Irena Ilovayskaya; Mona Sahnoun-Fathallah; Caroline Sievers; Gunter K Stalla; Emilie Castermans; Jean-Hubert Caberg; Ekaterina Sorkina; Renata Simona Auriemma; Sachin Mittal; Maria Kareva; Philippe A Lysy; Philippe Emy; Ernesto De Menis; Catherine S Choong; Giovanna Mantovani; Vincent Bours; Wouter De Herder; Thierry Brue; Anne Barlier; Sebastian J C M M Neggers; Sabina Zacharieva; Philippe Chanson; Nalini Samir Shah; Constantine A Stratakis; Luciana A Naves; Albert Beckers
Journal:  Endocr Relat Cancer       Date:  2015-07-17       Impact factor: 5.678

3.  X-linked acrogigantism syndrome: clinical profile and therapeutic responses.

Authors:  Albert Beckers; Maya Beth Lodish; Giampaolo Trivellin; Liliya Rostomyan; Misu Lee; Fabio R Faucz; Bo Yuan; Catherine S Choong; Jean-Hubert Caberg; Elisa Verrua; Luciana Ansaneli Naves; Tim D Cheetham; Jacques Young; Philippe A Lysy; Patrick Petrossians; Andrew Cotterill; Nalini Samir Shah; Daniel Metzger; Emilie Castermans; Maria Rosaria Ambrosio; Chiara Villa; Natalia Strebkova; Nadia Mazerkina; Stéphan Gaillard; Gustavo Barcelos Barra; Luis Augusto Casulari; Sebastian J Neggers; Roberto Salvatori; Marie-Lise Jaffrain-Rea; Margaret Zacharin; Beatriz Lecumberri Santamaria; Sabina Zacharieva; Ee Mun Lim; Giovanna Mantovani; Maria Chaira Zatelli; Michael T Collins; Jean-François Bonneville; Martha Quezado; Prashant Chittiboina; Edward H Oldfield; Vincent Bours; Pengfei Liu; Wouter W de Herder; Natalia Pellegata; James R Lupski; Adrian F Daly; Constantine A Stratakis
Journal:  Endocr Relat Cancer       Date:  2015-02-24       Impact factor: 5.678

Review 4.  Guidelines for the treatment of growth hormone excess and growth hormone deficiency in adults.

Authors:  A Giustina; A Barkan; P Chanson; A Grossman; A Hoffman; E Ghigo; F Casanueva; A Colao; S Lamberts; M Sheppard; S Melmed
Journal:  J Endocrinol Invest       Date:  2008-09       Impact factor: 4.256

5.  Advances in the Diagnosis, Treatment, and Molecular Genetics of Pituitary Adenomas in Childhood.

Authors:  Margaret F Keil; Constantine A Stratakis
Journal:  US Endocrinol       Date:  2009-02-01

6.  Treatment of pituitary gigantism with the growth hormone receptor antagonist pegvisomant.

Authors:  Naila Goldenberg; Michael S Racine; Pamela Thomas; Bernard Degnan; William Chandler; Ariel Barkan
Journal:  J Clin Endocrinol Metab       Date:  2008-05-20       Impact factor: 5.958

Review 7.  Pituitary tumors in childhood: update of diagnosis, treatment and molecular genetics.

Authors:  Margaret F Keil; Constantine A Stratakis
Journal:  Expert Rev Neurother       Date:  2008-04       Impact factor: 4.618

8.  Aggressive tumor growth and clinical evolution in a patient with X-linked acro-gigantism syndrome.

Authors:  Luciana A Naves; Adrian F Daly; Luiz Augusto Dias; Bo Yuan; Juliano Coelho Oliveira Zakir; Gustavo Barcellos Barra; Leonor Palmeira; Chiara Villa; Giampaolo Trivellin; Armindo Jreige Júnior; Florêncio Figueiredo Cavalcante Neto; Pengfei Liu; Natalia S Pellegata; Constantine A Stratakis; James R Lupski; Albert Beckers
Journal:  Endocrine       Date:  2015-11-25       Impact factor: 3.633

9.  Combined treatment with octreotide LAR and pegvisomant in patients with pituitary gigantism: clinical evaluation and genetic screening.

Authors:  Ruth Mangupli; Liliya Rostomyan; Emilie Castermans; Jean-Hubert Caberg; Paul Camperos; Jaime Krivoy; Elvia Cuauro; Vincent Bours; Adrian F Daly; Albert Beckers
Journal:  Pituitary       Date:  2016-10       Impact factor: 4.107

10.  Gigantism caused by growth hormone secreting pituitary adenoma.

Authors:  Noorisaem Rhee; Kumi Jeong; Eun Mi Yang; Chan Jong Kim
Journal:  Ann Pediatr Endocrinol Metab       Date:  2014-06-30
  10 in total

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