hrp0092rfc14.6 | Adrenals and HP Axis | ESPE2019

Growth Hormone Deficiency (GHD): Assessing Burden of Disease in Children and Adolescents: The Growth Hormone Deficiency – Child Impact Measure (GHD-CIM)

Brod Meryl , Højby Rasmussen Michael , Vad Knud , Alolga Suzanne , Bedoin Jacques

Background: Children with growth hormone deficiency (GHD) may have to deal with practical, emotional, and functional difficulties. Unfortunately, to date, there is no condition specific measure of the impact of GHD for these children. The Growth Hormone Deficiency – Child Impact Measure (GHD-CIM) was developed according to FDA/EMA guidances to address this gap. There are two GHD-CIM versions: child self-report (PRO) for ages 9 to <13 years and observe...

hrp0092p1-84 | Growth and Syndromes (to include Turner Syndrome) | ESPE2019

Development of a Parent Experience Measure for Parents of Children with Achondroplasia

Pfeiffer Kathyrn M. , Brod Meryl , Viuff Dorthe , Ota Sho , Gianettoni Jill , Leff Jonathan

Background: Limited research exists on how having a child with achondroplasia impacts parents' daily lives and well-being. The purpose of the study was to gather qualitative evidence to support the development of a parent experience measure that assess the impacts of having a child aged 2 to <12 years with achondroplasia.Methods: Concept elicitation interviews via individual telephone calls and an in-person focus...

hrp0092p1-235 | Growth and Syndromes (to include Turner Syndrome) (1) | ESPE2019

Development of a Measure for the Impacts of Achondroplasia on Children's Daily Functioning and Well-Being

Pfeiffer Kathryn M. , Brod Meryl , Viuff Dorthe , Ota Sho , Gianettoni Jill , Leff Jonathan

Background: Research on the impacts of achondroplasia on children's functioning and well-being is limited. The purpose of the study was to investigate the impacts of achondroplasia on children's daily lives to support the development of an impact measure of achondroplasia on children's functioning and well-being.Methods: Individual telephone interviews and one parent focus group were conducted in the United S...

hrp0092p1-371 | Growth and Syndromes (to include Turner Syndrome) (2) | ESPE2019

Growth Hormone Deficiency (GHD): Assessing Parent Burden for Child Growth Hormone Deficiency Treatment: The Growth Hormone Deficiency - Parent Treatment Burden Measure (GHD-PTB)

Brod Meryl , Rasmussen Michael Højby , Vad Knud , Alolga Suzanne , Bedoin Jacques

Background: Treatment for child GHD requires daily injections, which can be painful and disruptive. For most children, these injections are administered by an adult, usually their parent. Unfortunately, little is known about the burden that a child's treatment places on a parent. The GHD-PTB was developed according to FDA/EMA guidances to address this gap. Items were based on qualitative interviews of 31 parents of children with GHD, ages 4 to <13 year...

hrp0086p2-p671 | Growth P2 | ESPE2016

Assessing Disease and Treatment Burden for Young Children with Growth Hormone Deficiency (GHD)

Brod Meryl , Wilkinson Lars , Alolga Suzanne Lessard , Hojbjerre Lise , Beck Jane , Rasmussen Michael Hojby

Background: Children with GHD, in addition to short stature, may experience physiological symptoms as well as social and emotional problems. Assessing these impacts is critical for understanding the extent of GHD burden and assessing treatment benefit. Since many children initiating treatment are too young to self-report information, we must rely on adult reporters. However, according to FDA guidelines and established measure development principles, adult reporter information ...

hrp0084p1-84 | Growth Hormone | ESPE2015

Disease and Treatment Burden in Children and Adolescents with Growth Hormone Deficiency

Brod Meryl , Hojbjerre Lise , Alolga Suzanne , Nacson Alise , Nordholm Lars , Rassmussen Michael Hojby

Background: Children with growth hormone deficiency (GHD) may experience physiological symptoms as well as social and emotional problems.Objective and hypotheses: This qualitative study explored the burden of GHD and treatment for children and their parents.Method: 70 interviews were conducted with 39 children (age 8–12) and 31 parents of children with GHD (age 4–12) in Germany, UK and USA. Interviews were analysed using ...

hrp0095p1-108 | Growth and Syndromes | ESPE2022

Efficacy, Observer-Reported Outcomes, and Safety of Once-Weekly Somapacitan in Children with Growth Hormone Deficiency (GHD): 4-Year Results from the REAL 3 Trial

Sävendahl Lars , Battelino Tadej , Højby Rasmussen Michael , Brod Meryl , Wai Lee Kai , Saenger Paul , Horikawa Reiko

Children with GHD are currently treated with daily subcutaneous growth hormone (GH) injections, which can be burdensome. Somapacitan is a long-acting GH derivative in development for once-weekly use in children with GHD. REAL 3 (NCT02616562) is a phase 2, multinational, randomised, open label, controlled trial assessing efficacy and safety of somapacitan vs daily GH (Norditropin®). Prepubertal, GH-naïve children with GHD received 0.04 (n=16), 0.08 (n</e...