Monica R. Gadelha, MD, Hospital Universitário Clementino Fraga Filho, Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil, presented a preliminary analysis of the phase 3 PAOLA study [NCT01137682] that showed that biochemical control continued through extension week 28. The intent within this extension phase was to assess pasireotide long-acting release (LAR) in patients with acromegaly over an extended period of time.

Dr Gadelha and colleagues examined data through extension week 28 of the extension phase of the 24-week, randomized, phase 3 PAOLA study, which enrolled patients with inadequately controlled acromegaly [Gadelha MR et al. Lancet Diabetes Endocrinol. 2014]. The 24-week, phase 3 study had found that biochemical control, defined as growth hormone < 2.5 mg/L and normalized insulinlike growth factor 1 (IGF-1), was achieved by significantly more patients who received pasireotide LAR 40 mg (15%; n = 10/65; P = .0006) or 60 mg (20%; n = 13/65; P < .0001) than those who received octreotide LAR or lanreotide Autogel (0%; n = 0/68). The most common adverse events in the phase 3 trial were hyperglycemia, diabetes, and diarrhea. Serious adverse events were reported in 6 patients receiving pasireotide 40 mg, 2 receiving pasireotide 60 mg, and 3 in the active control group.

The patients who were in the control group continued to receive the same doses in the extension phase from week 24 through a 4-week bridging phase. Next, patients from the control group who were not biochemically controlled received open-label pasireotide LAR 40 mg, and their dose was increased to 60 mg if their disease became uncontrolled. These were the crossover group.

The patients who were not in the control group remained on pasireotide LAR at 40 or 60 mg during the 4-week bridging phase. If they had not achieved biochemical control, they received open-label pasireotide LAR 60 mg beginning at extension week 4. These patients, the treatment groups, received a total of 52 weeks of treatment with pasireotide LAR for this analysis.

At extension week 28, biochemical control was achieved by 18% of the patients in the pasireotide LAR 40-mg group (n = 9/49; 95% CI, 9 to 32) and by 33% in the pasireotide LAR 60-mg group (n = 15/45; 95% CI, 20 to 49). Among the crossover group, 20% achieved biochemical control (n = 10/50; 95% CI, 10 to 34).

All 3 groups had a drop in IGF-1 levels during the extension time. At week 28, normal IGF-1 was obtained in 33% of the patients receiving pasireotide LAR 40 mg, in 38% receiving pasireotide LAR 60 mg, and in 24% of the crossover group. Growth hormone levels of < 2.5 mg/L were achieved by 39% of the patients receiving pasireotide LAR 40 mg, 47% of those receiving pasireotide LAR 60 mg, and 42% of the crossover group.

The safety profile was very similar to that of the core study. This longer-term treatment identified no new treatment-emergent safety signals.

Pasireotide may be a viable, long-term treatment option for patients with acromegaly that is inadequately controlled with first-generation somatostatin analogues.