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Consensus Statement on the Diagnosis and Treatment of Children with Idiopathic Short Stature: A Summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop

Department of Pediatric Endocrinology (P.C.), Mattel Children’s Hospital at University of California, Los Angeles, Los Angeles, California 90095; Department of Pediatrics (A.D.R.), University of Virginia, and ODR Consulting, Charlottesville, Virginia 22911; Endocrinology Service (C.L.D.), Sainte-Justine Hospital, Montreal, Quebec, Canada H3T 1C5; Department of Pediatrics (P.S.), Albert Einstein College of Medicine, Bronx, New York 10467; Baystate Children’s Hospital (E.O.R.), Tufts University School of Medicine, Springfield, Massachusetts 01199; Department of Pediatrics (J.L.R.), Thomas Jefferson University, Philadelphia, Pennsylvania 19107; Department of Pediatrics (S.D.C.), University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma 73104; Centre for Endocrinology (M.O.S.), the London School of Medicine and Dentistry, London E1 2AD, United Kingdom; and Department of Pediatrics (J.M.W.), Leiden University Medical Center, 2300 RC Leiden, The Netherlands

Address all correspondence and requests for reprints to: Pinchas Cohen, M.D., Professor and Chief of Endocrinology, Mattel Children’s Hospital at UCLA, David Geffen School of Medicine at UCLA, 10833 Le Conte Avenue MDCC 22-315, Los Angeles, California 90095-1752. E-mail: hassy{at}mednet.ucla.edu.

Objective: Our objective was to summarize important advances in the management of children with idiopathic short stature (ISS).

Participants: Participants were 32 invited leaders in the field.

Evidence: Evidence was obtained by extensive literature review and from clinical experience.

Consensus: Participants reviewed discussion summaries, voted, and reached a majority decision on each document section.

Conclusions: ISS is defined auxologically by a height below –2 SD score (SDS) without findings of disease as evident by a complete evaluation by a pediatric endocrinologist including stimulated GH levels. Magnetic resonance imaging is not necessary in patients with ISS. ISS may be a risk factor for psychosocial problems, but true psychopathology is rare. In the United States and seven other countries, the regulatory authorities approved GH treatment (at doses up to 53 µg/kg·d) for children shorter than –2.25 SDS, whereas in other countries, lower cutoffs are proposed. Aromatase inhibition increases predicted adult height in males with ISS, but adult-height data are not available. Psychological counseling is worthwhile to consider instead of or as an adjunct to hormone treatment. The predicted height may be inaccurate and is not an absolute criterion for GH treatment decisions. The shorter the child, the more consideration should be given to GH. Successful first-year response to GH treatment includes an increase in height SDS of more than 0.3–0.5. The mean increase in adult height in children with ISS attributable to GH therapy (average duration of 4–7 yr) is 3.5–7.5 cm. Responses are highly variable. IGF-I levels may be helpful in assessing compliance and GH sensitivity; levels that are consistently elevated (>2.5 SDS) should prompt consideration of GH dose reduction. GH therapy for children with ISS has a similar safety profile to other GH indications.

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