ACROSTUDY: the Italian experience

Abstract

ACROSTUDY is a world-wide non-interventional, post marketing surveillance study performed to monitor the safety and outcomes of pegvisomant (PEG) in clinical practice. We report data from acromegaly patients who have been included in the Italian ACROSTUDY registry. The data of 341 acromegaly patients (171 males) were available for analysis using data freeze (12/9/2012). Patients were enrolled in 25 Italian endocrine centres. Before and during PEG treatment IGF-I, liver enzymes, metabolic parameters, and pituitary MRI were assessed. Before PEG, 54.3% patients had been treated with medical therapy and surgery, 22.9% medical therapy only, and 15.8% medical plus radiation and surgical therapy. 199 adverse events were reported in 98 patients (28.7%). Serious adverse events were documented in 29 patients (8.5%). 71.1% of patients had no significant change in tumor volume. Central MRI reading was performed in 34 patients; in 7 patients, an increase in tumor volume was found. Hormonal efficacy progressively increased since the start of PEG. After 6 years, normal IGF-I levels were found in 70.9% of patients (mean daily dose 18.1 mg). 87.1% of patients were treated with daily PEG although in 8.8% of patients, it was administered 2-6 times per week and in 3.8% with weekly injections. 74.8% received a PEG dose 10-15 mg/daily. PEG is a drug with a favorable safety profile which is efficacious also considering that in Italy it is currently available as third-line therapy.

60 YEARS OF NEUROENDOCRINOLOGY: Acromegaly

“…In a worldwide surveillance study (Acrostudy) of patients treated only with PEG in daily s.c. injections, IGF1 was normalized in only 67.5% of cases after 3.8 years follow-up . The efficacy increases slightly with time (Schreiber et al 2007), reaching 70.9% after 6 years (Grottoli et al 2015). The response is dose-dependent: 89% of cases treated with 20 mg PEG daily reach a normal IGF1 in 12 weeks (Trainer et al 2000).…”

Growth hormone–secreting macroadenoma of the pituitary gland successfully treated with the radiolabeled somatostatin analog 90Y-DOTATATE: case report

“…Nonetheless, according to the ACROSTUDY, pegvisomant is currently available as a third-line therapy. 6 No data are yet available to indicate whether the size of a GH-producing tumor is modified by treatment with SSA coupled with 90 Y-DOTATATE. Somatostatin receptor expression can be evaluated using immunohistochemistry or receptor scintigraphy.…”

Optimal use of pegvisomant in acromegaly: are we getting there?

“…This aspect, which is somewhat expected since current indications for pegvisomant use are focused on acromegaly patients ''resistant to other treatments,'' has double implications: on one side, this means that use of the drug is still likely confined to difficult long standing (mean duration of acromegaly before pegvisomant start was 8 years) acromegaly patients who are seen in very selected institutions, and therefore, most endocrine centers have a very limited experience with the drug; on the other side, this implies that Acrostudy data are really the best available reflecting the practice of endocrinologists with the largest experience with this treatment. Interestingly, slightly less than one quarter of the patients in this Italian experience were only treated medically before starting pegvisomant [21]. In fact, the main safety issue with pegvisomant so far, that greatly limited its use in clinical practice, has been the concern of a potential tumor (re)growth.…”

“…Why? In this issue of Endocrine, Grottoli et al report data from acromegaly patients who have been included in the Italian Acrostudy registry [21] a world-wide non-interventional, post-marketing surveillance study initiated in 2004 not only to monitor mainly the safety but also outcomes of pegvisomant in clinical practice [22]. This single country experience (which adds to the long-term one obtained in Germany) [23] performed on a relevant number of patients ([300) with a long-term follow-up period ([4 years) offers some hints that may help to elaborate on this topic.…”