IMPE2023 Poster Presentations GH and IGFs (14 abstracts)
Experts’ Priorities for Treatment of Pediatric Growth Hormone Deficiency – Results of an Expert Panel of Physicians from Latin America
Cecilia Camacho-Hubner 1 , Joshua Coulter 2 , Brett Hauber 2 , Berenice Aguirre 3 , Shokery Awadalla 4 , Roberto Bogarin 5 , Margaret Boguszewski 6 , Paola Duran 7 , Ana Keselman 8 , Veronica Mericq 9 , Mirta Miras 10 , Analia Morin 11 , Lucero Ruiz 12 & Paulo Solberg 13
1Rare Disease, Pfizer, New York, USA. 2Global Medical Impact Assessment, Pfizer, New York, USA. 3Endocrinología Pediátrica, Hospital Ángeles Clínica Londres, Ciudad de Mexico, Mexico. 4Endocrinología Pediátrica, Hospital San José, Bogotá, Colombia. 5Endocrinología Pediátrica. Hospital Nacional de Niños, San José, Costa Rica. 6Department of Pediatric, Federal University of Parana, Curitiba, Brazil. 7Fundación Cardioinfantil-Instituto de Cardiología, Bogotá, Colombia. 8División de Endocrinología, Hospital de Niños, R Gutierrez, Buenos Aires, Argentina. 9Instituto de Investigaciones Maternoinfantiles de la Facultad de Medicina de la Universidad de Chile, Santiago, Chile. 10Endocrinología, Hospital de Niños de Córdoba, Córdoba, Argentina. 11Hospital Sor Maria Ludovica, La Plata, Argentina. 12Hospital Nacional de Pediatría, Ciudad de Mexico, Mexico. 13Endocrinologia Faculdade de Ciências Médicas da Universidad Estado de Rio de Janeiro, Rio de Janeiro, Brazil
Background and Objective: Daily injections of growth hormone (GH) are the standard treatment for pediatric GH deficiency (pGHD). Long-acting GH have been developed for pGHD and may soon be available in Latin American (LATAM) markets. The objective of this preference exercise was to elicit treatment priorities and practices in pediatric GH deficiency among experts in Latin America.
Methods: A panel of experts in pediatric endocrinology from Central and South America was convened to assess priorities in treating children with GH deficiency. Swing weighting was used to evaluate 23 attributes of pediatric GH treatments. The attributes were divided into 5 blocks of related attributes: efficacy, safety, injection mode, treatment burden, and “other”. In each block, participants ranked the attributes, and the highest-ranked attribute was assigned a score of 100. Each of the other attributes in the block was weighted relative to the highest-ranked attribute. A final ranking and weighting of the blocks provided the information needed to weigh all attributes relative to each other. Additional questions were included to facilitate discussions of patient and physician behavior.
Results: The panel included 11 pediatric endocrinologist experts. The most important attributes were safety (change from having only short-term safety data to having both short- and long-term safety data) and efficacy followed by injection frequency (change in injection frequency from once daily to once weekly). Other highly rated attributes were height velocity (change in growth velocity from <6 cm to ≥12 cm at 12 months) and IGF-I levels (change in IGF-I from > 2 standard deviations (SD) to within ±2 SD or normal range for age and sex. In addition, the need to change injection site (change from needing to use new injection site each time to being able to use the same injection site) was highlighted. There was significant variation in weighing among the physician experts. However, nearly all (9/11) thought that patients who stop taking treatment do so because they get tired of taking treatments and 8/11 said that they would consider prescribing a weekly injection rather than a daily injection if the patient had difficulty adhering to daily treatment and 6/11 said they would consider prescribing a weekly injection if the patient/parents asked to switch.
Conclusions: In this exercise, LATAM pediatric endocrinologists rated safety and efficacy as the most important attributes in GH treatment followed by injection frequency. Future research in this area should also elicit patient and caregiver preferences.
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