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

Rix, Mariane; Laurberg, Peter; Hoejberg, AS; Brock-Jacobsen, Bendt

doi:10.1530/eje.1.01956

Cited by 39 publications

(23 citation statements)

References 36 publications

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“…It further confirms the limited pediatric experience (6 patients reported in the literature) in which pegvisomant has shown encouraging results [14,15,16,20]. …”

Section: Discussionsupporting

confidence: 82%

“…Medical therapy in acromegalic patients has highly progressed with the availability of dopamine agonists and somatostatin analogues (such as octreotide and lanreotide). In gigantism, treatment with somatostatin analogues has been found to be effective in some cases [8,10,11,12], but not in others, even in combination with cabergoline [13,14,15,16]. Since 2004, pegvisomant, a pegylated GH receptor antagonist, represents a new therapeutic option for the management of patients with acromegaly.…”

Section: Introductionmentioning

confidence: 99%

“…Normal doses are between 10 and 40 mg s.c. per day. Nevertheless, current experience in pediatric patients is still limited, being reported in only 6 case reports [14,15,16,20,21]. …”

Section: Introductionmentioning

confidence: 99%

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Eight-Year Follow-Up of a Child with a GH/Prolactin-Secreting Adenoma: Efficacy of Pegvisomant Therapy

et al. 2010

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A 3.4-year-old girl was admitted to the Pediatric Department because of tall stature (116.0 cm, +5.1 SDS) and increased height velocity (16.3 cm/year, +6.1 SDS). Basal hormonal evaluation revealed elevated insulin-like growth factor I (IGF-I) levels (938 ng/ml, nv 40–190), prolactin (PRL) (98.0 ng/ml, nv 1.7–24.0) and mean growth hormone (GH) nocturnal concentration (147 ng/ml). Basal adrenal, gonadal and thyroid functions were normal. Hand-wrist bone age was 3.6 years. Magnetic resonance imaging revealed a macroadenoma with moderate suprasellar invasion. The adenoma was surgically removed and histological characterization confirmed the diagnosis of GH/PRL-secreting adenoma. The patient was admitted to our Endocrine Unit when 7.9 years old, because of the persistence of elevated GH, IGF-I and PRL levels, although there was a slight height velocity reduction and absence of tumor recurrence. Treatment with cabergoline was initiated, but only PRL levels normalized. Afterwards, octreotide long-acting release (LAR) was added without reaching the normalization of GH and IGF-I levels. Thus, treatment with octreotide LAR was discontinued and pegvisomant was added to cabergoline, leading to the normalization of IGF-I levels and height velocity without side effects. Other anterior pituitary functions were always normal. To conclude, treatment of pituitary gigantism with pegvisomant was effective and well tolerated in a young giant unresponsive to combined cabergoline and octreotide treatment.

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“…It further confirms the limited pediatric experience (6 patients reported in the literature) in which pegvisomant has shown encouraging results [14,15,16,20]. …”

Section: Discussionsupporting

confidence: 82%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Eight-Year Follow-Up of a Child with a GH/Prolactin-Secreting Adenoma: Efficacy of Pegvisomant Therapy

et al. 2010

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“…Daily injection of 10 mg pegvisomant, however, induced growth factors below the normal range, which may have adverse long-term effects on growth. In a recent report of a 12-year-old patient with a GH-secreting pituitary adenoma, pegvisomant treatment with 20 mg daily resulted in a prompt decline of growth factors, effectively stopping further growth [21]. In adult patients with acromegaly alternate-day administration of pegvisomant was shown to allow maintenance of normal IGF-I levels [22].…”

Section: Discussionmentioning

confidence: 99%

Pegvisomant Treatment in a 4-Year-Old Girl with Neurofibromatosis Type 1

Main

Sehested²,

Feldt‐Rasmussen

2006

Horm Res Paediatr

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Background/Aims: Growth hormone (GH) excess in childhood is a rare disorder. Current treatment options such as somatostatin analogues, pituitary surgery or irradiation can have serious side effects. Recently, a GH receptor antagonist, pegvisomant, was introduced for the treatment of adults with acromegaly. We wanted to investigate whether pegvisomant was effective in a child with octreotide-resistant GH excess. Case: A 4-year-old girl with neurofibromatosis type 1 and GH excess associated with optic glioma received pegvisomant injections (10 mg subcutaneously) with increasing intervals from daily to every 4th day. Results: IGF-I and IGFBP-3 decreased from +6.9 and 4.6 standard deviation scores (SDS), respectively, to within normal range. Height velocity dropped from 12.4 SDS to mean –0.7 SDS (range: –5.0 to 5.0) and height SDS decreased from +1.3 to +0.6 (target height: +0.2). Random non-fasting serum GH values were mean 5.0 mlU/l (range: 1.6–9.5). There was no change in fasting blood glucose (4.6–4.7 mmol/l) or glycosylated haemoglobin (5.5%) and no subjective or biochemical side effects. Repeated tests of thyroid, adrenal and gonadal function showed no alterations during the treatment period. Intracranial tumours remained unchanged in size and visual impairment did not deteriorate. Conclusion: Pegvisomant normalized IGF-I and IGFBP-3 levels. Growth velocity was normalized after initial catch-down growth, and it remains to be seen whether this result can be maintained during long-term treatment.

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“…There is limited data on pegvisomant treatment in children, mostly case studies, which report successful outcomes [80,81].…”

Section: Pituitary Adenomasmentioning

confidence: 99%

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

Keil

Stratakis

2008

Expert Review of Neurotherapeutics

126

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Pituitary tumors are rare in childhood and adolescence, with a reported prevalence of up to 1 per million children. Only 2 -6% of surgically treated pituitary tumors occur in children. Although pituitary tumors in children are almost never malignant and hormonal secretion is rare, these tumors may result in significant morbidity. Tumors within the pituitary fossa are of two types mainly, craniopharyngiomas and adenomas; craniopharyngiomas cause symptoms by compressing normal pituitary, causing hormonal deficiencies and producing mass effects on surrounding tissues and the brain; adenomas produce a variety of hormonal conditions such as hyperprolactinemia, Cushing disease and acromegaly or gigantism. Little is known about the genetic causes of sporadic lesions, which comprise the majority of pituitary tumors, but in children, more frequently than in adults, pituitary tumors may be a manifestation of genetic conditions such as multiple endocrine neoplasia type 1 (MEN 1), Carney complex, familial isolated pituitary adenoma (FIPA), and McCune-Albright syndrome. The study of pituitary tumorigenesis in the context of these genetic syndromes has advanced our knowledge of the molecular basis of pituitary tumors and may lead to new therapeutic developments.

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

Cited by 39 publications

References 36 publications

Eight-Year Follow-Up of a Child with a GH/Prolactin-Secreting Adenoma: Efficacy of Pegvisomant Therapy

Eight-Year Follow-Up of a Child with a GH/Prolactin-Secreting Adenoma: Efficacy of Pegvisomant Therapy

Pegvisomant Treatment in a 4-Year-Old Girl with Neurofibromatosis Type 1

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

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