Persistence to growth hormone treatment and clinical characteristics of pediatric patients with growth hormone deficiency: A retrospective cohort study of data from the Japan Medical Data Center claims database.

A total of 1,020 patients with pediatric growth hormone deficiency treated with growth hormone replacement therapy were identified from the Japan Medical Data Center claims database.

• Mean age at initial diagnosis was 7.5 ± 3.8 years
• A bimodal pattern in age at diagnosis was found, with peaks at 3 years and 12 to 13 years of age
• The bimodal peaks were more pronounced in male patients
• Data source was a retrospective cohort using claims database

The median time to initial treatment discontinuation (first prescription-free period of 182 days or more) for 50% of patients was 2,526 days, similar to the time to treatment completion of 2,626 days.

• 1,016 patients were eligible for persistence analysis after excluding patients with early withdrawal
• Treatment discontinuation was defined as a prescription-free period of 182 days (6 months) or more
• The time to discontinuation (2,526 days) was similar to treatment completion time (2,626 days), suggesting most patients persisted until completion
• Only 24 out of 1,016 patients (2.4%) had a treatment discontinuation

The mean proportion of days covered by growth hormone replacement therapy was 89.8%.

• Proportion of days covered was used as a measure of treatment adherence
• This metric reflects the overall adherence among the 1,016 patients eligible for persistence analysis

Being female and older age at diagnosis were independently associated with shorter time to treatment discontinuation.

• Female sex was associated with a hazard ratio of 1.85 (95% CI: 1.36–2.51) for shorter time to discontinuation
• Older age at diagnosis was associated with a hazard ratio of 1.50 (95% CI: 1.41–1.60) for shorter time to discontinuation
• These findings suggest that some patients, particularly females and those diagnosed at older ages, complete GHRT before puberty

Most pediatric patients with growth hormone deficiency in Japan persist with growth hormone replacement therapy until puberty, but some complete treatment before puberty.

• The similarity between median time to discontinuation and time to treatment completion indicates most discontinuations represent planned treatment completion rather than premature dropout
• The authors note there are patients diagnosed and starting treatment just before puberty, supporting continued efforts toward early referral and diagnosis
• Only 2.4% of patients experienced true treatment discontinuation (prescription gap ≥182 days)