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      Growth Hormone Treatment of Idiopathic Short Stature

      Hormone Research in Paediatrics

      S. Karger AG

      Growth hormone, Short stature, Therapy, Trial

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          Abstract

          Considerable controversy exists about the use of growth hormone (GH) treatment in short children without classical GH deficiency (idiopathic short stature or ISS). ISS is a multifactorial disorder with many potential causes rather than a single diagnostic entity, and it is in essence a diagnosis of exclusion. A careful investigation of the components of the GH/insulin-like growth factor (IGF) axis and other potential causes of short stature must be carried out in patients with significant short stature before they are categorized as having the ISS syndrome. As a group, most patients with ISS can be expected to attain an adult stature within the normal range. The ISS patients who will not do so are generally those who have a poor initial predicted adult height (PAH) and present at a younger age. Those ISS patients with a shorter midparental height are also less likely to achieve a normal adult height. In addition, several studies have shown that, as a group, males with ISS do not attain their initial PAH. Most studies on the GH treatment of patients with ISS have shown a short-term increase in growth rate and relative height. However, the long-term outcome of GH treatment of ISS has been variously reported to be no significant change, a slight increase, or a significant increase. One of the largest and most optimistic experiences with GH treatment of ISS is the collaborative US study sponsored by Genentech. This group has treated 121 patients with ISS not due to classically defined GH deficiency for up to 9 years with GH (0.3 mg/kg per week). A total of 51 of these patients has now reached near final height, with bone age (BA) > 16 years for boys and ≥ 14 years for girls. The mean difference between the first available pretreatment PAH and the PAH at near-final height (ΔPAH) was 5.7 cm for the boys and 6.5 cm for the girls. The strongest predictors of response to treatment available before the start of GH treatment were bone age and pretreatment PAH. The increase in height seen with GH treatment of ISS was not enough to increase the mean expected final height to the midparental target height, or to completely correct a low pretreatment PAH. Whether this increase in expected final height in ISS patients with GH treatment is due to a correction of a subtle abnormality of GH secretion or action, or to the pharmacological effects of GH is unclear. The decision to use GH treatment in ISS is a complex issue which must involve the accuracy of the diagnosis of GH deficiency or abnormality in the GH/IGF axis, the PAH and BA of the patient, the potential benefits, and economic issues for the medical care system. In general, GH treatment should only be considered in young ISS patients with poor initial PAH and height prognosis, and GH treatment of older patients who have a normal PAH is not justified.

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          Author and article information

          Journal
          HRE
          Horm Res Paediatr
          10.1159/issn.1663-2818
          Hormone Research in Paediatrics
          S. Karger AG
          978-3-8055-6432-8
          978-3-318-00112-9
          1663-2818
          1663-2826
          1996
          1996
          09 December 2008
          : 46
          : 4-5
          : 208-214
          Affiliations
          Division of Pediatric Endocrinology, Department of Pediatrics, Stanford University, Stanford, Calif., USA
          Article
          185025 Horm Res 1996;46:208–214
          10.1159/000185025
          8950623
          © 1996 S. Karger AG, Basel

          Copyright: All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means, electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher. Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug. Disclaimer: The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publishers and the editor(s). The appearance of advertisements or/and product references in the publication is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.

          Page count
          Pages: 7
          Categories
          Session 3: Recent Advances in Therapy

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