Abstract
Introduction Growth hormone (GH) treatment is approved for cancer survivors with GH deficiency. Alongside daily GH, long-acting GH (LAGH) has been recently approved for the treatment of GHD. Given its different mechanistic action to daily GH, the safety of LAGH in this group needs further study. Methods The Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (https://globe-reg.net/) was initiated in 2022 to collect real world data on safety and effectiveness of GH therapy. In late 2025, a non-interventional, retrospective and prospective study, SAFE-Survivors was launched with the aim of using GloBE-Reg to study cases of all ages who receive daily GH or LAGH following a cancer diagnosis; longitudinal data shall be analysed at 12, 24, 60, and 120 months after initiation of GH. The study will recruit for 10 years and this report presents the preliminary analysis of the first data tranche. Results Since its launch in late 2022, 44 centres from 24 countries in 5 continents have enrolled 5075 cases with a median current age of 12.2 (10th, 90th centile: 6.3, 18.1). Of these, 2928 (58%) had growth hormone deficiency (GHD). Among this cohort, 92 (3%) cases from 16 centres received GH following a cancer diagnosis. Of these, 10 (11%) were receiving LAGH, 2 had stopped GH and the remainder were on daily GH. In the first data tranche at study launch, data was available for 74 (80%) cases from 11 centres. The median age at start of therapy and the current age was 11 yrs (6.4, 14.8) and 14.5 yrs (9.8, 19.2), respectively. This cohort consisted of 64 reports of intracranial tumours including craniopharyngiomas (n,23) and pituitary tumours (n,8). Therapy data was available in 64 cases and the broad groups were surgery only (10), radiation only (3), chemotherapy only (3) and combined (48). Median follow-up after GH initiation was 28 months(4.5, 96) with data at 12, 24, 60 and 120 months in 23, 19, 9 and 3 cases respectively. Conclusions This initial analysis offers an early view of the long-term safety of LAGH in comparison to daily GH treatment in cancer survivors with GHD. No new cancers or recurrences were found in this cohort. However, this is based on a small sample and short follow up time and requires further data clarification. Increased patient recruitment and data collection of the GloBE-Reg over time will be crucial to fully assess safety and guide clinical practice.