Real-world IGF-1 Variations & Its Management in Children on Recombinant Human Growth Hormone (rhGH) Therapy (RIGHT Study)

Chen Suet Ching; Malika Alimussina; Sanhita Koley; Sheila Shepherd; Al-Agha Abdulmoein Eid; Nadia Amin; Navoda Atapattu; JiaJia Chen; Yana Deyanova; Antony Fu; Michael Højby; de Lima Jorge Alexander Augusto; Violeta Iotova; Dominika Januś; Renata Markosyan; Miller Bradley S.; Lars Savendahl; Seneviratne Sumudu Nimali; Shaikh M Guftar; Savitha Shenoy; W Tack Lloyd J; Malgorzata Wasniewska; Ahmed S Faisal

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ESPE Abstracts (2024) 98 P2-357

ESPE2024 Poster Category 2 Late Breaking (107 abstracts)

Real-world IGF-1 Variations & Its Management in Children on Recombinant Human Growth Hormone (rhGH) Therapy (RIGHT Study)

Suet Ching Chen ^1,2 , Malika Alimussina ^1,2 , Sanhita Koley ^1,2 , Sheila Shepherd ¹ , Abdulmoein Eid Al-Agha ³ , Nadia Amin ⁴ , Navoda Atapattu ⁵ , JiaJia Chen ⁶ , Yana Deyanova ⁷ , Antony Fu ⁸ , Michael Højby ⁹ , Alexander Augusto de Lima Jorge ¹⁰ , Violeta Iotova ⁷ , Dominika Januś ¹¹ , Renata Markosyan ¹² , Bradley S. Miller ¹³ , Lars Savendahl ¹⁴ , Sumudu Nimali Seneviratne ¹⁵ , M Guftar Shaikh ¹ , Savitha Shenoy ¹⁶ , Lloyd J W Tack ¹⁷ , Malgorzata Wasniewska ¹⁸ & S Faisal Ahmed ^1,2

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Author affiliations

¹Developmental Endocrinology Research Group, Royal Hospital for Children, University of Glasgow, Glasgow, United Kingdom. ²Office for Rare Conditions Registries, University of Glasgow, Glasgow, United Kingdom. ³King Abdulaziz University Hospital, Jeddah, Saudi Arabia. ⁴Leeds Children’s Hospital, Leeds, United Kingdom. ⁵Lady Ridgeway Hospital for Children, Colombo, Sri Lanka. ⁶Beijing Children’s Hospita, Beijing, China. ⁷Medical University UMHAT “Sv. Marina”, Varna, Bulgaria. ⁸Princess Margaret Hospital, Hong Kong, China. ⁹Novo Nordisk, Denmark, Netherlands. ¹⁰Endocrinology Division of Hospital das Clinicas of University of Sao Paulo School of Medicine, Sao Paolo, Brazil. ¹¹Jagiellonian University and University Children Hospital, Krakow, Poland. ¹²Yerevan State Medical University, Wigmore Women & Children’s Hospital, Yerevan, Armenia. ¹³M Health Fairview Masonic Children’s Hospital, University of Minnesota, Minnesota, USA. ¹⁴Karolinska Institutet, Stockholm, Sweden. ¹⁵University of Colombo, Colombo, Sri Lanka. ¹⁶Leicester Children’s Hospital, Leicester, United Kingdom. ¹⁷Ghent University Hospital, Ghent, Belgium. ¹⁸Department of Human Pathology of Adulthood and Childhood University of Messina, Messina, Italy

Aim: Serum IGF-1 is widely advocated as a tool for monitoring adherence, safety and effectiveness of recombinant human growth hormone (rhGH). However, there is a need to understand the real-world variations in IGF-1 levels in children on rhGH and the management of abnormal IGF-1 levels in routine clinical practice.

Method: Centres participating in the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (www.GloBE-Reg.net) were invited to complete the minimum dataset for rhGH therapy. Information on the indications for rhGH therapy, gender, age at diagnosis and rhGH initiation, rhGH doses and serum IGF-1 on therapy were collected for analysis. Results are shown in median (range).

Results: Since study launch in Jan 2024, 1,716 IGF-1 values from 262 cases were available from 14 centres in 10 countries. For a subset of 351 IGF-1 values from 79 cases (54M: 25F) in 2 centres, reference data were available for analysis. The main indications for rhGH treatment in these 79 cases included GHD in 43 (54%), Prader Willi syndrome (PWS) in 11 (14%), small for gestational age (SGA) in 11 (14%) with a few other indications. All the patients were on daily rhGH, except one on LAGH. Seventy-five (21%) of the IGF-1 values were 2 standard deviations (SD) outside the mean for age and sex (SDS). Of the 75, the IGF-1 values were below –2SDS in 28 (37%) [median, –2.68 (range, -3.9, -2.25)] and above +2SDS in 47 (63%) [median, +2.62 (+2.08, +4.49)]. The rhGH doses for the group with low and high IGF-1 values were 29 (17,43) and 27 (11,44) mcg/kg/day. Children with PWS (P <0.001) and SGA (P <0.02) were more likely to have high IGF-1 levels compared to GHD. Data on clinical management were available for 23/28 (82%) low IGF-1 values. In 14 (61%), a dose increment of 17% (7,100) was recorded; no dose change was recorded on 9 occasions. Adherence information was only available in 2/28 (7%) of the low IGF-1 values. Of the 47 high results, 8 (17%) had dose reduction by 15% (4,47), with no change in 23 (49%) and a further dose increase by 15% (7,50) in 16 (34%).

Conclusions: A fifth of serum IGF-1 values in children on rhGH treatment are outside the assay reference range and this is more likely in those with SGA and PWS. Further studies are required to better understand the value of measuring serum IGF-1 in routine clinical practice.