Evidence, with context

The research library.

Clinical trials, pharmacometric research, and emerging findings on Pegpesen. Each entry identifies the evidence type and links to its source.

Recent additions

Reviewed 28 September 2026
2026Network meta-analysisMethodological correspondence ↓

Long-acting growth hormones in East Asia: comparative efficacy and safety

Liu Q, Wu C, Peng D, et al. · Endocrine Practice. 32(8):1303–1310. · Online 1 April; issue August 2026.

This five-study network analysis included Pegpesen, Jintrolong, and somapacitan. For height velocity, the Pegpesen estimate versus daily GH was −0.10 cm/year (95% CI −0.53 to 0.32). The authors reported more favorable growth estimates for Jintrolong. Differences in adverse-event and serious-adverse-event rates were not statistically significant.

Interpretation: the figures above are the original authors’ reported results, not independently reproduced estimates. Alongside the limits of indirect comparisons, a forthcoming letter raises specific questions about study identification and safety-data handling. Read the methodological correspondence below before interpreting comparative rankings.

FORTHCOMING LETTERAdded 28 September 2026

Data provenance and safety analysis: three questions requiring reassessment

Robin Wan. Request for Editorial Review of Data Provenance and Outcome-Altering Analytical Issues. Letter to the Editor, Endocrine Practice.

Acceptance reported by the author; journal publication details and DOI are pending. This summary is based on the author-supplied revised manuscript.

The letter requests an editorial reassessment of the populations and safety outcomes used in Liu et al.’s analysis. It identifies the following issues and calls for access to the extraction data and executable model code.

  1. Trial identity and the analyzed population. Wan reports that the study-characteristics table labels Luo 2017 as Phase II (108 participants), while the extracted Jintrolong efficacy and adverse-event values match Phase III (343 participants). If that Phase III cohort supplied the modeled data, the corresponding total would be 1,087 rather than 852 (852 − 108 + 343). The original Luo report confirms the two distinct trial populations; the network’s actual input file is needed to resolve which cohort was analyzed.
  2. Exclusion of the 391-participant Pegpesen Phase III safety dataset. According to the letter, this trial was included for efficacy but excluded for safety on the basis that adverse events were insufficiently differentiated. Its published Table 4 does report treatment-emergent adverse events, drug-related events, discontinuations, and serious adverse events over 52 weeks. Wan argues that relying on the smaller, 12-week Phase II dataset warrants reassessment with aligned event definitions and follow-up periods.
  3. Severe versus serious events, and a double-zero comparison. The letter questions whether the source statement about no severe events was recoded as no serious adverse events (0/228 versus 0/115), then used to produce a relative risk and SUCRA ranking. Severity and seriousness are different concepts. As the letter emphasizes, a comparison with no observed events in either arm does not, by itself, establish a direction of relative safety; any rank requires scrutiny of coding and model assumptions.
What the Pegpesen Phase III source actually reports

Participants with at least one event, n/N (%), over 52 weeks. These are participant counts, not the total number of event episodes.

Selected outcomes from Liang et al., Table 4 (safety analysis set)
OutcomePegpesen (N = 261)Daily rhGH (N = 130)
Any treatment-emergent adverse event229/261 (87.7%)123/130 (94.6%)
Drug-related treatment-emergent adverse event52/261 (19.9%)26/130 (20.0%)
Any serious adverse event17/261 (6.5%)8/130 (6.2%)

This single-trial table establishes that differentiated safety data are available. It is not a reanalysis of the network and does not, by itself, establish a comparative safety advantage.

Requested next steps: verify study-by-outcome extraction, reproduce the model independently, reconcile study identities and denominators, reassess inclusion of Phase III safety data, and remove rare-event rankings that lack support.

The concerns are attributed to Wan’s letter. They should not be represented as an issued journal correction, a retraction, or a finding of misconduct. The letter itself states that the discrepancies do not establish misconduct.

2026Conference abstract

Pegpesen in non-GHD short stature: a Phase II basket trial

Liang Y and colleagues · ENDO 2026, oral abstract ORF47-04. · 15 June 2026.

The randomized, open-label, 52-week study enrolled 78 children with idiopathic short stature (ISS), small for gestational age (SGA), or Turner syndrome (TS). The abstract reports dose-response modeling and growth findings for weekly Pegpesen versus daily rhGH, with similar reported safety profiles between groups.

Preliminary conference evidence, not a full journal paper. These populations and investigational regimens should not be treated as additional approved indications or dosing recommendations.

2026Population PK/PD

Optimizing Pegpesen dosing through population PK/PD modeling

Zhao Y, Zou F, Gu J, He R, Yin Y. · Journal of Endocrinological Investigation. 49:599–607. · Online 5 November 2025; issue March 2026.

Data from the Phase I–III program informed simulations in 292 children with GHD. The model explored quarterly dose escalation and weight-banded dosing; modeled growth responses converged by month 24.

This is a modeling study. Its simulated titration and weight bands are research proposals, not replacements for approved dosing instructions.

The core clinical program

Product-specific studies
2025Randomized Phase III

Weekly Y-shaped PEGylated rhGH in children with GHD

Liang Y, Wei H, Yang F, et al. · The Journal of Clinical Endocrinology & Metabolism. 110:e2605–e2613. · Online 28 November 2024; 2025 issue.

The pivotal, active-controlled trial randomized 391 treatment-naïve children with GHD to weekly Pegpesen or daily somatropin. The 52-week analysis met the prespecified growth-velocity non-inferiority criterion.

Read efficacy together with adverse events, immunogenicity, the study population, and the duration of follow-up.

2022Randomized Phase II

Dose-ranging, PK/PD, and short-term growth outcomes

Liang Y, Zhang C, Wei H, et al. · Frontiers in Endocrinology. 13:922304. · 11 August 2022.

A multicenter study in 43 children evaluated three once-weekly PEGylated rhGH doses against daily rhGH over 12 weeks, characterizing exposure, IGF-1 response, and short-term growth.

The short treatment period and small groups limit conclusions about long-term outcomes.

Wider GH context

Background reading · not Pegpesen-specific evidence
2025Consensus

Long-acting GH therapy in pediatric GHD: international consensus

The Journal of Clinical Endocrinology & Metabolism · PMID 39672599.

Field-level guidance addressing long-acting GH treatment, monitoring, adherence, and remaining evidence gaps.

Class-level guidance should not be used to assume that different GH products have interchangeable doses or identical evidence.

Search scope: Pegpesen, inpegsomatropin, and Y-shaped PEGylated recombinant human growth hormone. Sources reviewed include PubMed, journal publishers, and the Endocrine Society’s ENDO 2026 program. Online-publication and journal-issue dates are shown separately where relevant. This is a curated reference list, not a systematic review. Local PDFs retained from the existing library.