Tumor Shrinkage With Lanreotide Autogel 120 mg as Primary Therapy in Acromegaly: Results of a Prospective Multicenter Clinical Trial

The Journal of Clinical Endocrinology and Metabolism (Impact Factor: 6.31). 01/2013; 99(4):jc20133318. DOI: 10.1210/jc.2013-3318
Source: PubMed

ABSTRACT Context Methodological shortcomings often compromise investigations into the effects of primary somatostatin-analog treatment on tumor size in acromegaly. There are also limited data for the long-acting lanreotide formulation. Objective To better characterize the effects of primary lanreotide Autogel treatment on tumor size in patients with growth hormone (GH)-secreting macroadenomas. Design PRIMARYS-48-week multicenter open-label single-arm (; EudraCT-2007-000155-34). Setting Specialist endocrine centers. Patients Treatment-naïve acromegalic patients with GH-secreting macroadenomas. Intervention Subcutaneous lanreotide Autogel 120 mg every 28 days (without dose titration). Outcome measures Primary endpoint: null hypothesis (H0) -proportion of patients with clinically significant (≥20%) tumor volume reduction (TVR) at week 48/last post-baseline value available (LVA) is ≤55%, using central assessments from three readers. Secondary endpoints included: TVR at other time points; GH and insulin-like growth factor-1 (IGF-1); acromegalic symptoms; quality of life (QoL); safety. Results 64/90 (71.1%) patients completed the study. Clinically significant TVR at 48 weeks/LVA achieved by 62.9% [95%CI: 52.0, 72.9] of 89 patients in the primary analysis (intention-to-treat population; H0 not rejected) and 71.9-75.3% in sensitivity (n = 89) and secondary analyses (n = 63) (H0 rejected). At 12 weeks, 54.1% had clinically significant TVR. Early and sustained improvements also occurred in GH and IGF-1, acromegalic symptoms, and QoL. No patients withdrew due to gastrointestinal intolerance. Conclusions Primary treatment with lanreotide Autogel, administered at 120 mg (highest available dose) without dose titration, in patients with GH-secreting macroadenomas provides early and sustained reductions in tumor volume, GH and IGF-1, and acromegalic symptoms, and improves QoL.