Session: FP20-Growth: Clinical Trials & Observational Studies
Room 122 (Moscone Center)
Poster Board SUN-628
Newly diagnosed PHDs were identified during follow-up of 716 pediatric pts with organic IGHD in the GeNeSIS prospective, multinational observational study (510 with congenital, 204 with acquired and 2 with unknown GHD cause). PHDs were ascertained by check box on case report forms, reports of adverse events, or initiation of replacement therapy.
During mean±SD follow-up of 3.3±2.9 years (yr), ≥1 additional PHD was reported for 71 of the 716 pts (10%). Limiting the time window of observation to pts with at least 3.5 yr follow-up or development of MPHD within 4.5yr (“4 yr cohort”), resulted in 290 pts (mean±SD follow-up of 5.8±2.3 yr), of whom 60 (21%) were reported to develop ≥1 additional PHD. Of 80 pts with acquired GHD in the 4 yr cohort, 27 (34%) developed MPHD (21/50 [42%] of those with GHD due to intracranial tumor), compared with 33/210 (16%) with GHD due to congenital causes (22/121 [18%] of those with abnormal pituitary development). Comparison of pts in the 4 yr cohort who developed MPHD vs. those who did not, showed the following significant differences (p by ANOVA): age at diagnosis of GHD: 8.2±4.4 vs. 6.8±3.7 yr, p=0.017; age at start of GH: 9.0±4.3 vs. 7.4±3.6 yr, p=0.005; baseline IGF-I SDS: -4.4±2.1 vs. -2.9±2.1), p=0.007; additionally median (Q1;Q3) maximum stimulated GH peak was 1.6 (0.6;3.3) vs. 4.9 (2.7;7.9) μg/L, p<0.001 (with GH peak ≤5 µg/L: 87% vs. 54%, p<0.001). Interestingly, 4 pts who developed MPHD had peak GH >10 µg/L. Deficiency of TSH was the most frequent (73%), followed by LH/FSH (23%), ACTH (15%), and ADH (10%); 80% of MPHD pts had 1 additional PHD diagnosed during 4.5yr follow-up, 18% had 2 and 2% had 3. In the entire population the time (yr) from diagnosis of GHD to the first additional PHD [median (Q1;Q3)] was 1.0 (0.9;2.3) for ADH, 1.9 (1.7;2.8) for ACTH, 2.4 (1.0;3.6) for TSH, and 6.0 (2.5;7.3) for LH/FSH. Multivariable logistic regression modeling identified the following significant (p<0.05) predictors for development of MPHD: female gender, older baseline age, longer follow-up time, and lower stimulated GH peak.
Pts with organic IGHD, especially those with more severe GHD, and those with acquired causes, are at high risk of developing MPHD. However, even after 4 yr of observation the majority (79%) of pts had not (yet) manifested additional PHDs, and as signs and symptoms of additional PHDs may be delayed, long-term monitoring of pituitary function is recommended.
Disclosure: CJC: Employee, Eli Lilly & Company, Employee, Eli Lilly & Company. CLD: Medical Advisory Board Member, Eli Lilly & Company. AGZ: Employee, Eli Lilly & Company, Employee, Eli Lilly & Company. CAQ: Employee, Eli Lilly & Company, Employee, Eli Lilly & Company. RGR: Medical Advisory Board Member, Eli Lilly & Company. SLD: Medical Advisory Board Member, Eli Lilly & Company. GBC Jr.: , Eli Lilly & Company, , Eli Lilly & Company. WFB: Employee, Eli Lilly & Company, Employee, Eli Lilly & Company.
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