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Continuous subcutaneous IGF-1 therapy via insulin pump in a patient with Donohue syndrome

  • David R. Weber , Diana E. Stanescu , Robert Semple , Cheryl Holland and Sheela N. Magge EMAIL logo
Published/Copyright: August 5, 2014

Abstract

Donohue syndrome (DS) is a severe form of congenital insulin resistance due to mutation(s) in the insulin receptor (INSR) gene. Given the similarities between insulin and insulin-like growth factor 1 (IGF-1) receptors, recombinant human IGF-1 (rhIGF-1) has been used to treat severe insulin resistance due to INSR mutation(s). Traditional subcutaneous therapy may be limited by the shortened IGF-1 half-life in these patients. We report the case of a female with molecularly confirmed DS treated with continuous rhIGF-1 therapy via an insulin pump. With treatment, the patient’s hemoglobin A1c decreased from 9.8% to 8.8%, and her weight increased by 0.8 kg. Development of an ovarian tumor complicated her course, but it was unclear whether this was related to rhIGF-1 therapy. Limited treatment options exist for patients with DS. The use of continuous rhIGF-1 via an insulin pump may be a viable option, although further experience is needed to establish safety and efficacy.


Corresponding author: Sheela N. Magge, M.D., M.S.C.E., Children’s National Health System, CTS Suite, M7659, CRI-6, 6th Floor Main, 111 Michigan Ave, N.W., Washington, DC, 20010, USA, Phone: +202-476-7031, Fax: +202-476-3425, E-mail:
aDrs. Weber and Stanescu contributed equally.bResearched data, contributed to discussion, wrote manuscript, reviewed/edited manuscript.cResearched data.dResearched data, contributed discussion, reviewed/edited manuscript.

Acknowledgments

The authors would like to express our sincere gratitude to our patient’s parents for their tremendous strength and genuine desire to help other children. We would also like to thank Dr. David Dunger for his expert advice in treating this child.

Conflict of interest statement

Authors’ conflict of interest disclosure: None of the authors has a conflict of interest to disclose.

Funding source: Dr. Weber was supported by NIH grant K12 DK094723.

Financial disclosure: Dr. Magge served on an advisory board for Ipsen Group in December, 2010 on treatment guidelines for severe insulin resistance, after care for this child was completed. The authors have no financial relationships relevant to this article to disclose.

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Supplemental Material

The online version of this article (DOI: 10.1515/jpem-2013-0402) offers supplementary material, available to authorized users.


Received: 2013-10-11
Accepted: 2014-6-21
Published Online: 2014-8-5
Published in Print: 2014-11-1

©2014 by De Gruyter

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