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CLINICAL STUDY |
Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas M.D. Anderson Cancer Center, 1515 Holcombe Boulevard Unit 435, Houston, Texas 77030, USA1 Department of Endocrinology, University Hospital MAS, Malmö, Sweden2 Department of Endocrinology, University of Antwerp, Antwerp, Belgium3 Endocrinology, University of North Carolina School of Medicine, Chapel Hill, North Carolina 27599, USA4 Endocrinology, St Bartholomew's Hospital, London, UK5 Radiology, University of Tennessee College of Medicine, Chattanooga, Tennessee, USA6 Clinical R&D Statistics, Pfizer Inc., New York, New York 10017, USA7 Medicine, University of Virginia, Charlottesville, Virginia 22908, USA8 Endocrinology, Christie Hospital, Manchester, UK
(Correspondence should be addressed to R F Gagel; Email: rgagel{at}mdanderson.org)
Objective: We examined pituitary tumor volumes in patients treated with pegvisomant for 18 months or longer, and in whom the tumors were monitored for at least 3 years. We present details on 9 of 304 patients in clinical trials with pegvisomant who experienced tumor growth within the first year of treatment.
Method: Magnetic resonance images prior to start of pegvisomant and at last follow-up were examined in 43 patients (14% of participating patients). Twenty-nine had received prior radiation therapy (18% of irradiated patients) and all but five received somatostatin analogs between periods of pegvisomant treatment.
Results: At follow-up, the median tumor volume was 0.6 cc (range 0.0–19.7 cc), in comparison with 1.6 cc (0.0–19.7 cc) at baseline (P<0.001). Twenty-five patients, of which 23 received radiation therapy, had tumor volume reduction. Seventeen patients had no significant change. One patient, who had not received radiation therapy, had an increase in tumor volume from 1.61 to 1.93 cc. Of the nine patients with tumor growth, six had progressive growth before initiating pegvisomant. Two patients with stable tumors while on octreotide experienced enlargement after octreotide discontinuation but remained stable on long-term pegvisomant therapy.
Conclusion: The present data indicate that pegvisomant does not promote tumor growth and suggest that the nine observed cases of tumor progression, which occurred within 8 months after commencing pegvisomant, are likely rebound expansions after discontinuation of somatostatin analogs and/or the natural history of aggressively growing pituitary tumors. Continued long-term surveillance of tumor volume, particularly in non-irradiated patients, is recommended.
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