Quality of life and other outcomes in children treated for Cushing syndrome

Abstract

Context: Cushing syndrome (CS) in children is associated with residual impairment in measures of health-related quality of life, even after successful resolution of hypercortisolemia, highlighting the need for early identification of morbidities and improvements in long-term management of these patients.

Evidence acquisition and synthesis: A PubMed, Scopus, and Web of Science search of articles from 1900 onward identified available studies related to quality of life and complications of pediatric CS as well as important historical articles. This review summarizes studies through November 2012 and highlights recent developments.

Conclusions: A review of the literature identifies significant morbidities associated with CS of pediatric onset, which must not be treated in isolation. CS affects children and adolescents in many ways that are different than adults. Post-treatment challenges for the child or adolescent treated for CS include: optimize growth and pubertal development, normalize body composition, and promote psychological health and cognitive maturation. All these factors impact health-related quality of life, which is an important outcome measure to assess the burden of disease as well as the effect of treatment. Future research efforts are needed to improve management of the physical, psychological, and emotional aspects of this disease in order to diminish the residual impairments experienced by the pediatric CS patient population.

Figure 1.

Clinical findings and typical growth chart in CS before and after treatment. The most common presentation in children is lack of height gain concomitant with persistent weight gain.

Figure 2.

IQ scores were based on the Wechsler Intelligence Scale for healthy age- and sex-matched control subjects (white bars), and children with CS before treatment (black bars), and 1 year after surgery and correction of hypercortisolism (gray bars). The T-bars indicate standard error values (100).

Figure 3.

Before and 1 year after treatment CHQ (quality of life tool) individual domain z-scores in 40 children with CS treated with either TSS or adrenalectomy. Results are expressed as z-scores compared to age-matched normative data from the U.S. population. The CHQ domains represented are those that showed significant improvement from before to after treatment and include: physical summary score (PhS), physical function (PF), role-physical (RP), global health (GHP) (note: GHP was the only score that declined from before to after treatment), emotional impact on parent (PE), mental health (MH), bodily pain (BP), self-esteem (SE), time impact-parent (PT), and psychosocial summary score (PsS). P < .05 for differences between all domains and U.S. norms before treatment; P < .01 for differences between PhS, PF, RP, GHP, and PE post-treatment scores and U.S. norms (101).