Evidence map›Paper›PMID 30145746›Full record

SynthesisEndocrine2019

Effectiveness and safety of pegvisomant: a systematic review and meta-analysis of observational longitudinal studies.

Letícia P Leonart, Fernanda S Tonin, Vinicius L Ferreira, Fernando Fernandez-Llimos, Roberto Pontarolo

Abstract readMeta-AnalysisSystematic Review
PubMed Publisher
In one paragraph

Synthesis in Endocrine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed, 1 pooled it
3.1field-weighted citation impact, top 7% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

19 citing papers in PubMed, 1 synthesis or guideline pooled it, 49 citations in OpenAlex.

  1. Pooled it
  2. Current treatment landscape of acromegaly.The Journal of clinical endocrinology and metabolism · 2026
    Review
  3. Article
  4. Pegvisomant in managing comorbidities of acromegaly: a review.Reviews in endocrine & metabolic disorders · 2026
    Review
  5. Article
  6. Review
  7. Acromegaly: diagnostic challenges and individualized treatment.Expert review of endocrinology & metabolism · 2025
    Review
  8. Article
  9. Review
  10. Article
  11. Review
  12. Review
  13. Article
  14. Article
  15. Review
  16. Article
  17. Multidisciplinary management of acromegaly: A consensus.Reviews in endocrine & metabolic disorders · 2020
    Review
  18. [Pituitary adenomas].Der Nervenarzt · 2019
    Review
  19. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 2 institutions in 2 countries.

Letícia P LeonartUniversidade Federal do Paraná, Graduate Program in Pharmaceutical Sciences, Curitiba, Paraná, Brazil.
Fernanda S ToninUniversidade Federal do Paraná, Graduate Program in Pharmaceutical Sciences, Curitiba, Paraná, Brazil.
Vinicius L FerreiraUniversidade Federal do Paraná, Graduate Program in Pharmaceutical Sciences, Curitiba, Paraná, Brazil.
Fernando Fernandez-LlimosResearch Institute for Medicines (iMed.ULisboa), Department of Social Pharmacy, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.
Roberto PontaroloUniversidade Federal do Paraná, Graduate Program in Pharmaceutical Sciences, Curitiba, Paraná, Brazil. pontarolo@ufpr.br.
Universidade Federal do Paraná · BRUniversity of Lisbon · PT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeAcromegaly is a rare disease that often requires drug treatment to achieve control, with pegvisomant being one of the most widely used therapies. In the present paper, we aimed to obtain evidence regarding the effectiveness and safety of pegvisomant by reviewing real-world observational longitudinal studies.

methodsA systematic review was performed with a meta-analysis of event rates (95% confidence interval (CI)) using a random effects model. Sensitivity and subgroup analyses were performed (comprehensive meta-analysis 2.0). The systematic review was performed in accordance to preferred reporting items for systematic reviews and meta-analyses, meta-analysis of observational studies in epidemiology, and Cochrane recommendations (PROSPERO register CRD 42017059880). PubMed, Scopus, Web of Science, and SciELO were used to search for literature. Observational studies in patients using pegvisomant for the treatment of acromegaly were included.

resultsInitially, 552 papers were retrieved from the databases; and 31 articles were included in the qualitative analysis and 14 in the quantitative analysis. Eight primary meta-analyses were performed. The overall rate of patients with disease control was of 60.9% (51.8-69.3%; 95% CI). When considering patients under monotherapy, the control rate was 71.7% (64.0-78.4%; 95% CI). Tumor growth was estimated in 7.3% (4.7-11.1%; 95% CI) and elevation of transaminases in 3.0% (1.7-5.2%; 95% CI).

conclusionsThe real-world data showed that the effectiveness of pegvisomant is not as high as reported in interventional studies. Acromegaly appears to be better controlled when pegvisomant is used as a monotherapy. No serious adverse events were associated with the use of pegvisomant; however, given the high cost of this drug, further studies are required.

Indexed as

AcromegalyHuman Growth HormoneHumansLongitudinal StudiesObservational Studies as TopicHuman Growth HormonepegvisomantAcromegalyMeta-analysisObservational studyPegvisomantPituitary gland

Identifiers

PMID30145746
OpenAlexW2888323704

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.