hrp0097p2-14 | Growth and Syndromes | ESPE2023

Unmet needs and challenges experienced by patients with growth disorders and their caregivers: A patient expert view.

Thomas Marc , Harvey Jamie , Kocabicak Sergül

Background: The diagnosis, treatment, and management of growth disorders can have a substantial burden on patients and caregivers. Research was conducted with patient experts to understand challenges experienced by patients with growth disorders and their caregivers, and identify opportunities to improve care.Methods: A mixed-methods approach was used to obtain insights from patient experts between October 2022-January 2...

hrp0098p2-156 | GH and IGFs | ESPE2024

Dosage of recombinant human growth hormone across geographic regions at enrolment into the Kabi/Pfizer International Growth Study (KIGS)

Geffner Mitchell , Thomas Marc , Cutfield Wayne , Gomez Roy , Tanaka Toshiaki , Carlsson Martin , Wajnrajch Michael

Objectives: The Kabi/Pfizer International Growth Study (KIGS) was the largest and longest running international database of pediatric patients receiving recombinant human growth hormone (rhGH). The objective of the current analysis is to describe the dosage of rhGH at enrolment into KIGS according to diagnosis and geographical region.Methods: Between 1987-2012, 83,803 pediatric patients were enrolled into KIGS if they re...

hrp0098p2-351 | Late Breaking | ESPE2024

Up to 5 years of once-weekly somatrogon treatment in pediatric patients with growth hormone deficiency: results from an open-label extension of a global Phase 3 study

Silverman Lawrence , Steelman Joel , Choe John , Stawerska Renata , Deal Cheri , Wajnrajch Michael , Thomas Marc , La Torre Daria , Phillip Moshe , Taylor Carrie , Wang Rong , Cara Jose

Objective: Somatrogon, a long-acting recombinant human growth hormone (rhGH) is approved as a once-weekly treatment for pediatric growth hormone deficiency (pGHD). This abstract describes the long-term efficacy and safety of somatrogon in patients with pGHD following up to 5 years of treatment in a global, Phase 3 study (NCT02968004).Methods: In the main study period, patients were randomized to receive either once-weekl...