Judith L Ross1, Harvey Kushner2, Karen Kowal3, Martha Bardsley3, Shanlee Davis4, Allan L Reiss5, Nicole Tartaglia4, David Roeltgen6. 1. Thomas Jefferson University, Department of Pediatrics, Philadelphia, PA; A.I. DuPont Hospital for Children, Wilmington, DE. Electronic address: judith.ross@jefferson.edu. 2. Biomedical Research Institute, Philadelphia, PA. 3. Thomas Jefferson University, Department of Pediatrics, Philadelphia, PA; A.I. DuPont Hospital for Children, Wilmington, DE. 4. Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO. 5. Department of Psychiatry, Stanford University, Palo Alto, CA. 6. Shore Physicians Group, Somers Point, NJ.
Abstract
OBJECTIVES: To examine the effects of early low-dose androgen on motor, cognitive, and behavioral function in prepubertal boys with Klinefelter syndrome (47,XXY). STUDY DESIGN: Double-blind trial of 84 boys, ages 4-12 years, randomized to oxandrolone (Ox; 0.06?mg/kg daily; n?=?43) or placebo (Pl; n?=?41) for 24 months. Standardized assessments were performed at baseline and every 12 months for 24 months evaluating motor, cognitive, and behavioral function. RESULTS: The 24-month outcomes were better in the Ox vs. Pl group on 1 of 5 primary endpoints (motor function/strength): Bruininks Visual-Motor scale (P?=?.005), without significant differences between the 2 groups for the other 4 components. Secondary analyses suggested improvement in the Ox vs. Pl group in the anxiety/depression (P?=?.03) and social problems (P?=?.01) scales on the Child Behavior Checklist, anxiety (P?=?.04) on the Piers Harris Self Concept Scale, and interpersonal problems (P?=?.02) on the Children's Depression Inventory, without significant differences in hyperactive or aggressive behaviors. CONCLUSIONS: This double-blind, randomized trial demonstrates that 24 months of childhood low-dose androgen treatment in boys with Klinefelter syndrome benefited 1 of 5 primary endpoints (visual-motor function). Secondary analyses demonstrated positive effects of androgen on aspects of psychosocial function (anxiety, depression, social problems), without significant effects on cognitive function, or hyperactive or aggressive behaviors. TRIAL REGISTRATION: ClinicalTrials.gov: NCT00348946.
RCT Entities:
OBJECTIVES: To examine the effects of early low-dose androgen on motor, cognitive, and behavioral function in prepubertal boys with Klinefelter syndrome (47,XXY). STUDY DESIGN: Double-blind trial of 84 boys, ages 4-12 years, randomized to oxandrolone (Ox; 0.06?mg/kg daily; n?=?43) or placebo (Pl; n?=?41) for 24 months. Standardized assessments were performed at baseline and every 12 months for 24 months evaluating motor, cognitive, and behavioral function. RESULTS: The 24-month outcomes were better in the Ox vs. Pl group on 1 of 5 primary endpoints (motor function/strength): BruininksVisual-Motor scale (P?=?.005), without significant differences between the 2 groups for the other 4 components. Secondary analyses suggested improvement in the Ox vs. Pl group in the anxiety/depression (P?=?.03) and social problems (P?=?.01) scales on the Child Behavior Checklist, anxiety (P?=?.04) on the Piers Harris Self Concept Scale, and interpersonal problems (P?=?.02) on the Children's Depression Inventory, without significant differences in hyperactive or aggressive behaviors. CONCLUSIONS: This double-blind, randomized trial demonstrates that 24 months of childhood low-dose androgen treatment in boys with Klinefelter syndrome benefited 1 of 5 primary endpoints (visual-motor function). Secondary analyses demonstrated positive effects of androgen on aspects of psychosocial function (anxiety, depression, social problems), without significant effects on cognitive function, or hyperactive or aggressive behaviors. TRIAL REGISTRATION: ClinicalTrials.gov: NCT00348946.
Authors: D W Hart; S E Wolf; P I Ramzy; D L Chinkes; R B Beauford; A A Ferrando; R R Wolfe; D N Herndon Journal: Ann Surg Date: 2001-04 Impact factor: 12.969
Authors: S Fox-Wheeler; L Heller; C M Salata; F Kaufman; M L Loro; V Gilsanz; M Haight; G C Umman; N Barton; J A Church Journal: Pediatrics Date: 1999-12 Impact factor: 7.124
Authors: Naama Barnea-Goraly; Vinod Menon; Mark Eckert; Leanne Tamm; Roland Bammer; Asya Karchemskiy; Christopher C Dant; Allan L Reiss Journal: Cereb Cortex Date: 2005-03-09 Impact factor: 5.357
Authors: Bronwyn A L Crawford; Peter Y Liu; Mary T Kean; Jane F Bleasel; David J Handelsman Journal: J Clin Endocrinol Metab Date: 2003-07 Impact factor: 5.958
Authors: Simon Chang; Anne Skakkebaek; Shanlee M Davis; Claus H Gravholt Journal: Am J Med Genet C Semin Med Genet Date: 2020-06-04 Impact factor: 3.908
Authors: Nicole Tartaglia; Susan Howell; Shanlee Davis; Karen Kowal; Tanea Tanda; Mariah Brown; Cristina Boada; Amanda Alston; Leah Crawford; Talia Thompson; Sophie van Rijn; Rebecca Wilson; Jennifer Janusz; Judith Ross Journal: Am J Med Genet C Semin Med Genet Date: 2020-06-07 Impact factor: 3.908
Authors: Shanlee M Davis; Najiba Lahlou; Matthew Cox-Martin; Karen Kowal; Philip S Zeitler; Judith L Ross Journal: J Clin Endocrinol Metab Date: 2018-09-01 Impact factor: 5.958
Authors: Carole A Samango-Sprouse; Debra R Counts; Selena L Tran; Patricia C Lasutschinkow; Grace F Porter; Andrea L Gropman Journal: Appl Clin Genet Date: 2019-10-23