622 - Growth hormone therapy and quality of life in children with chronic kidney disease and short stature

Publication Number: 622.5301

Kanza Baqai, Weill Cornell Medicine, New York City, NY, United States; Akeem Noziere, Weill Cornell Medicine, Brooklyn, NY, United States; Pamela Singer, Cohen Children's Medical Center, New Hyde Park, NY, United States; John D. Mahan, John D Mahan, Professor of Pediatrics, Nationwide Children's/The Ohio State University, Columbus, OH, United States; Amy J. Kogon, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States; Oleh Akchurin, Weill Cornell Medical College, New York, NY, United States

Background: Short stature is a common complication of chronic kidney disease (CKD) in children. Recombinant human growth hormone (GH) therapy effectively improves growth velocity in most children with CKD. Interestingly, studies that assessed whether GH improves quality of life (QoL) in children with idiopathic short stature have been inconclusive. Whether GH improves QoL in children with CKD-induced short stature, remains unknown.

Objective: Determine the change of QoL in children with CKD and short stature after GH therapy from baseline and compare with QoL of short children with CKD not receiving GH therapy.

Design/Methods: GH treatment group was enrolled in 4 participating tertiary medical centers as part of a PNRC study. Comparison group was abstracted from the CKiD study using NIDDK Central Repository. QoL was assessed by Pediatric Quality of Life Inventory (PedsQL, parental version) in 4 domains: physical, emotional, social, and school, at GH therapy initiation (baseline) and 6 months later in the treatment group. In the comparison group, we used first two CKiD study visits with available PedsQL data.

Results: We enrolled 24 patients (22 boys, age 8.1±4.9 years, 58% White and 25% African American, baseline eGFR 49.6±40.6 mL/min/1.73 m2) into the treatment group; comparison group comprised of 70 patients. Height z-score was -2.3±1.1 at enrollment and -1.8±1.3 at 6-month follow-up (p=0.017) in the treatment group. At follow up, QoL improved in the physical, social, and school domains by 8-10 points (p < 0.05) in the treatment group compared to baseline. No major changes in PedsQL were observed in the comparison group between the visits. QoL changes in the treatment group exceeded those seen in the comparison group for physical, emotional, and school domains (p < 0.05).
The GH satisfaction survey was filled out by 21 parents; among them, 85.7% indicated that they would recommend the use of GH to other short CKD children and 80.9% reported that they would use GH therapy again. Pain and discomfort from GH injections was not considered a problem by 47.6% parents while 33.3 % parents considered it to be a minor problem, and none considered it a major problem. When asked about side effects, 71.4% parents reported no side effects associated with GH treatment.

Conclusion(s): This study is the first, to our knowledge, to demonstrate that children with CKD and short stature experience a significant improvement in QoL as a result of growth hormone therapy, compared to short CKD children not receiving growth hormone. Families of children from the treatment group reported high levels of satisfaction with GH use.

Quality of life changes in short children with Chronic Kidney Disease (CKD) treated vs. not treated with growth hormone
Health related quality of life (QoL) was assessed by the Pediatric Quality of Life Inventory (PedsQL, parental version) in 4 domains: physical, emotional, social, and school, at growth hormone (GH) therapy initiation (baseline) and at 6 months in the treatment group. In the comparison (Ctr) group from CKiD study, we used first two visits with the available PedsQL data. QoL improved after GH therapy in the physical, emotional and school domains in the treatment group compared to the control group. * p<0.05.