Short Stature in Childhood — Challenges and Choices

Allen, David B.; Cuttler, Leona

doi:10.1056/nejmcp1213178

Cited by 76 publications

(54 citation statements)

References 49 publications

(58 reference statements)

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“…Short stature (SS) in childhood is a frequent reason for referral to pediatric endocrinologists [2,3]. Normal height is determined according to age, sex and ethnic group as well as the family context.…”

Section: Introductionmentioning

confidence: 99%

Challenges in the Management of Short Stature

Argente¹

2015

Horm Res Paediatr

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Human growth, from fetal life to adolescence, is dynamic and a good marker of health. Growth is a complex process influenced by genetic, hormonal, nutritional and environmental factors, both pre- and postnatally. To date, no international agreement regarding normal height has been established. Auxological parameters are fundamental to investigate potential short stature (SS), either with a known diagnosis, e.g. disproportionate or proportionate, prenatal and/or postnatal onset, or an unknown diagnosis, i.e. idiopathic SS. The incidence/prevalence of SS is difficult to establish. The measurement of choice in children aged <2 years is length, while in those >2 years of age it is height. A number of monogenic diseases that lead to proportionate SS due to either isolated growth hormone deficiency, multiple pituitary hormone deficiency, growth hormone insensitivity, primary acid-labile subunit deficiency, primary IGF-1 deficiency, IGF-1 resistance, primary IGF-2 deficiency or primary protease deficiency have been discovered in the last 30 years. In addition, the Nosology and Classification of Genetic Skeletal Disorders revised in 2015 includes 436 conditions, with a number of genes of 364. A practical algorithm for the evaluation of SS as well as therapeutic options are discussed.

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Section: Introductionmentioning

confidence: 99%

Challenges in the Management of Short Stature

Argente¹

2015

Horm Res Paediatr

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“…Ethical and evidence-based counseling for parents at decision point A would therefore include the following: while concern about your child’s short stature is understandable, (1) your child’s height is most likely not the primary factor affecting his/her psychological well-being [18]; (2) hGH treatment will improve the growth rate and may modestly increase height attainment, but has not been shown to predictably improve psychosocial well-being[28]; and (3) it is therefore uncertain to what degree the benefits of treatment outweigh the risks, however small, for your otherwise healthy child [29]. For children who are old enough to provide informed assent (e.g., 10–12 years of age, see below) at decision point A, inclusion in the hGH initiation assenting process is appropriate, and given the discretionary nature of the intervention, serious consideration should be given to nontreatment if the child dissents.…”

Section: Decision Point A: Initiating Hgh-for-height Treatment – a Prmentioning

confidence: 99%

“…Determining a responsible endpoint for hGH-for-height challenges us to distinguish between what patients want and what they truly need. Regardless of diagnosis, continuing treatment until the child reaches maximum height substantially increases costs [29], with the last 1–3% of potential height gain increasing total expenditure by ∼20% [38]. Thus, for the adolescent treated for short stature due to any cause who has achieved a height in the low-normal adult range, the mere fact that he or she can claim potential to be taller with continued treatment does not justify entitlement to highly expensive therapy.…”

Section: Decision Point C: Therapeutic Objective – a Duty To Deliver mentioning

confidence: 99%

“…Thus, for the adolescent treated for short stature due to any cause who has achieved a height in the low-normal adult range, the mere fact that he or she can claim potential to be taller with continued treatment does not justify entitlement to highly expensive therapy. Further, without evidence that increased height proportionately improves patient well-being, treatment endpoints also cannot be differentiated according to quality-of-life outcomes [29]. It is difficult to defend that children with any short-stature-associated diagnosis are entitled to subsidized hGH-for-height (or any other growth-promoting) treatment beyond a height within the statistically normal adult range.…”

Section: Decision Point C: Therapeutic Objective – a Duty To Deliver mentioning

confidence: 99%

“…However, to counter criticisms of “cosmetic endocrinology” with some ethical clarity, future guidelines for responsible growth promotion treatment could assist by (1) recommending nontreatment to the vast majority of short children that do not have a problem solved by growth promotion and restricting treatment to severe and (likely) disabling short stature, (2) promoting strategies that minimize hGH treatment duration and dosage, and incorporate less expensive and less invasive alternative approaches (e.g., counseling, oxandrolone[29], possibly aromatase inhibitors [41]), and (3) strongly discouraging the enhancement of normal adult stature by any means. Finally, informative and honest counseling of potential hGH-for-height recipients should include discussion of realistic, modest, and variable height gain, limited evidence for psychosocial benefit, and ongoing studies for potential post-treatment adverse effects.…”

Section: The Big Picture: Cosmetic Endocrinology – Do We Have a Defenmentioning

confidence: 99%

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Growth Promotion Ethics and the Challenge to Resist Cosmetic Endocrinology

Allen¹

2017

Horm Res Paediatr

410

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The advancement of “human growth hormone (hGH)-for-height” – increasing height attainment in children short for reasons other than GH deficiency – arose from intuitive, deep-seated assumptions about the disability of short stature, its improvement with hGH-mediated height gain, and the safety of escalating dosages of hGH in healthy children. Evidence challenging these assumptions now strengthens criticism of hGH-for-height as cosmetic endocrinology. To counter this characterization, collective acceptance of guidelines is needed that advise nontreatment of the vast majority of short children, support strategies that minimize treatment duration and dosage, and restrain enhancement of normal adult stature. Through a clinical case analysis, ethical issues underlying these recommendations are explored. These include duties to provide informed assent and re-assent, protect children from unnecessary treatment, consider fairness to nontreated children, and allocate healthcare resources responsibly. Informed assent for hGH-for-height should ensure awareness of modest, variable height gain expectations, limited evidence for psychosocial benefit, ongoing studies for potential posttreatment adverse effects, and options for less expensive/invasive approaches, including nontreatment and counseling. Approaching growth promotion in this way fosters therapeutic restraint, resists the al lure of enhancement therapy, and minimizes contributions to society’s perception that to be taller is to be better.

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Disorders of Growth

Grissom¹,

Ungar²,

Iroku‐Malize³

2020

The Encyclopedia of Child and Adolescent Development

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Children who are significantly (two or more standard deviations) below or above average height can be considered to be of short or tall stature, although it is more common for children and their parents to be concerned about short stature. Evaluation for a suspected growth disorder should involve standardized assessment to identify both normal (e.g., familial short or tall stature) and abnormal variants (e.g., a genetic disorder) of growth in order to determine etiology and guide intervention, including the decision not to intervene medically. Treatment should be based on the determined etiology of the tall or short stature and, for many children, medical treatment may not be necessary. Providing information to patients and their parents regarding the potential risks and benefits of treatment is essential in order to provide the best care, to reassure parents and families, and to manage expectations appropriately.

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Short Stature in Childhood — Challenges and Choices

Cited by 76 publications

References 49 publications

Challenges in the Management of Short Stature

Challenges in the Management of Short Stature

Growth Promotion Ethics and the Challenge to Resist Cosmetic Endocrinology

Disorders of Growth

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