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Quality of Life in Acromegalic Patients during Long-Term Somatostatin Analog Treatment with and without Pegvisomant

Department of Internal Medicine (S.J.C.M.M.N., M.O.v.A., W.W.d.H., R.A.F., J.A.M.J.L.J., A.J.v.d.L.), Erasmus University Medical Center Rotterdam, 3000 CA, Rotterdam, The Netherlands; Health Economics and Outcomes Research (X.B.), IMS Health, Centre for Biomedical Research on Rare Diseases (CIBERER Unit 747), 08034 Barcelona, Spain; and Department of Endocrinology (S.M.W.), Hospital Sant Pau, Centre for Biomedical Research on Rare Diseases (CIBERER Unit 747), Autonomous University of Barcelona, 08025 Barcelona, Spain

Address all correspondence and requests for reprints to: Sebastian J. C. M. M. Neggers, M.D., Erasmus University Medical Center Rotterdam, P.O. Box 2040, 3000 CA Rotterdam, The Netherlands. E-mail: s.neggers{at}erasmusmc.nl.

Objective: The objective of the study was to assess whether weekly administration of 40 mg pegvisomant (PEG-V) improves quality of life (QoL) and metabolic parameters in acromegalic patients with normal age-adjusted IGF-I concentrations during long-acting somatostatin analog (SSA) treatment.

Design: This was a prospective, investigator-initiated, double blind, placebo-controlled, crossover study. Twenty acromegalic subjects received either PEG-V or placebo for two consecutive treatment periods of 16 wk, separated by a washout period of 4 wk. Efficacy was assessed as change between baseline and end of each treatment period. QoL was assessed by the Acromegaly Quality of Life Questionnaire (AcroQoL) and the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ).

Results: The AcroQoL (P = 0.008) and AcroQoL physical (P = 0.002) improved significantly after PEG-V was added. The addition of PEG-V also significantly improved the PASQ (P = 0.038) and the single PASQ questions, perspiration (P = 0.024), soft tissue swelling (P = 0.036), and overall health status (P = 0.035). No significant change in Z-score of IGF-I (P = 0.34) was observed during addition of PEG-V. Transient liver enzyme elevations were observed in five subjects (25%).

Conclusion: Improvement in quality of life was observed without significant change in IGF-I after the addition of 40 mg pegvisomant weekly to monthly SSA therapy in acromegalic patients who had normalized IGF-I on SSA monotherapy. These data question the current recommendations in how to assess disease activity in acromegaly. Moreover, the findings question the validity of the current approach of medical treatment in which pegvisomant is used only when SSA therapy has failed to normalize IGF-I.