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Prediction of therapy response in acromegalic patients under pegvisomant therapy within the German ACROSTUDY cohort

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Abstract

Purpose

This study aimed at investigating predicting factors for therapy response under growth hormone receptor antagonist therapy with a focus on subjective and patient-oriented measures.

Methods

Observational, multicenter nested-cohort study including 271 selected patients with the diagnosis of acromegaly and a minimum of one-year follow-up period within the German ACROSTUDY cohort (total cohort: n = 514). Outcome measures were the change of the biomarker IGF-1 (IGF-1 change and IGF-1 normalisation) between baseline and after 1 year of pegvisomant therapy (12 ± 6 months). Main predictors were patient-assessed subjective measures according to the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ) in conjugation with age, gender, BMI, max. dosage of pegvisomant at follow-up and IGF-1 before the start of pegvisomant therapy.

Results

The mean age of the study population was 51.2 (13.9) years and the mean BMI was 29.5 (5.1) kg/m2. In adjusted analyses, none of the individual perceived health (PASQ) scores, but age, BMI and IGF-1 at baseline were predictive for an IGF-1 decrease after 1 year of pegvisomant therapy and BMI and IGF-1, but equally none of the PASQ items, were predicting IGF-1 normalisation.

Conclusions

Age, BMI and baseline IGF-1 but not subjective perceived health measures predict therapy response under second line medical therapy with pegvisomant.

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Acknowledgments

We gratefully acknowledge the excellent support and critical discussion of the ACROSTUDY board members. Moreover, the authors thank all the investigators and study nurses of the individual centres for contributing data to the ACROSTUDY. ACROSTUDY is sponsored by Pfizer Inc.

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Author notes

    Authors and Affiliations

    1. Clinical Neuroendocrinology, Clinical Research Department, Max Planck Institute of Psychiatry, Kraepelinstr. 2-10, 80804, Munich, Germany

      Caroline Sievers & Günter K. Stalla

    2. II Medizinische Klinik und Poliklinik, Klinikum rechts der Isar, Ismaningerstrasse 22, 81675, Munich, Germany

      Dorothee M. Baur

    3. Endocrine Care, Pfizer Pharma GmbH, Berlin, Germany

      Anja Schwanke

    4. Department of Neurosurgery, University of Erlangen-Nuremberg, Erlangen, Germany

      Michael Buchfelder

    5. Endokrinologische Praxis, Oldenburg, Germany

      Michael Droste

    6. Department of Endocrinology and Metabolism, University of Duisburg-Essen and Endokrinologiezentrum, Alter Hof, Munich, Germany

      Klaus Mann

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    1. Caroline Sievers
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    Corresponding author

    Correspondence to Caroline Sievers.

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    Conflict of interset

    M. D., M. B., K. M., and G. K. S. are members of the Scientific ACROSTUDY German Board, which is sponsored by Pfizer GmbH, Germany. C. S. has received consultant fees as a member of ACROSTUDY Steering Committee. A. S. is currently employed by Pfizer GmbH Germany. K. M. is a member of the HNR board, which is funded by the Heinz Nixdorf Stiftung, Essen. C. S., M. B., K. M. and G. K. S. received German Pfizer Grants for other projects as well as lecture fees from Pfizer. D. M. B. has nothing to declare.

    Additional information

    On behalf of the German Pegvisomant Investigators.

    Caroline Sievers and Dorothee M. Baur have contributed equally to this work.

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    Sievers, C., Baur, D.M., Schwanke, A. et al. Prediction of therapy response in acromegalic patients under pegvisomant therapy within the German ACROSTUDY cohort. Pituitary 18, 916–923 (2015). https://doi.org/10.1007/s11102-015-0673-2

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