Joeri Vliegenthart¹, Marten Poley², Paul de Laat³, Edmond Rings⁴, Erica van den Akker¹, Danielle van der Kaay¹, on behalf of the SEENEZ Growth Hormone Consortium Other members of the SEENEZ Growth Hormone Consortium include B. Bakker, A. M. Boot, C. de Bruin, M. Finken, J. C. van der Heyden, A. C. S. Hokken-Koelega, H. J. van der Kamp, E. G. van Mil, T. C. J. Sas, D. A. Schott, P. van Setten, S. Straetemans, V. van Tellingen, R. N. H. Touwslager, A. S. P. van Trotsenburg, P. G. Voorhoeve, J.M. Wit⁵

(1) Pediatric endocrinology, Erasmus University Medical Center, Sophia Children’s Hospital, Rotterdam, Netherlands.

(2) Erasmus School of Health Policy and Management and institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, Netherlands.

(3) Pediatrics, Franciscus Gasthuis en Vlietland, Rotterdam, Netherlands.

(4) Pediatrics, Erasmus University Medical Center, Sophia Children’s Hospital, Rotterdam, Netherlands.

(5) .

Background

Idiopathic isolated growth hormone deficiency (IIGHD) is treated with recombinant human growth hormone (rhGH) to achieve normal adult height (AH). RhGH treatment is expensive and imposes a significant treatment burden. Current protocols recommend continuation of rhGH until near AH (NAH). Recent studies suggest that many adolescents may not need prolonged rhGH treatment, as 70-80% of patients with IIGHD show a sufficient GH peak when retested. This raises the question whether earlier discontinuation could be cost-effective.

Aims

We aimed to analyze the cost savings of withdrawing rhGH treatment at mid-puberty in GH-sufficient adolescents with IIGHD.MethodsWe conducted a cost analysis alongside a multi-center patient preference trial in mid-pubertal adolescents treated with rhGH for >=3 years. GH-sufficient adolescents (peak >6.7 ug/L) chose to stop or continue rhGH until NAH; continuers received standard care, stoppers had outpatient visits twice yearly. Costs per patient over 3 years, including healthcare and non-healthcare costs, were analyzed.Results127 patients (75% male) participated. 44 patients (35%) continued rhGH treatment until NAH (GHcont), 83 patients (65%) stopped treatment 2-3 years earlier (GHstop). Mean costs per patient in the GHcont group equaled EUR11,928 per year ( EUR27,868 from mid-puberty until NAH). For the GHstop group, the total costs per patient equaled EUR222 per year and EUR692 from mid-puberty until NAH. Earlier discontinuation of rhGH could potentially reduce costs in the Netherlands by approximately EUR2 million annually.

Conclusion

Withdrawing rhGH treatment 2-3 years earlier in GH-sufficient adolescents with transient IIGHD significantly decreases medical consumption and reduces healthcare costs.