Evidence map›Paper›PMID 38464899›Full record

ArticleFrontiers in pediatrics2024

Taliglucerase alfa in the longterm treatment of children and adolescents with type 1 Gaucher disease: the Albanian experience.

Paskal Cullufi, Sonila Tomori, Virtut Velmishi, Agim Gjikopulli, Ilir Akshija, Aferdita Tako, Ermira Dervishi, Gladiola Hoxha, Marjeta Tanka, Erjon Troja and 1 more

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Article in Frontiers in pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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

1 citing paper in PubMed.

  1. Article
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

11 authors.

Paskal Cullufi *Pediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Sonila Tomori *Pediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Virtut VelmishiPediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Agim GjikopulliPediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Ilir AkshijaStatistics Department, University Hospital Center Mother Teresa, Tirana, Albania.
Aferdita TakoPediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Ermira DervishiPediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Gladiola HoxhaPediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.
Marjeta TankaRadiology Department, University Hospital Center Mother Teresa, Tirana, Albania.
Erjon TrojaPharmacy Department, University Hospital Center Mother Teresa, Tirana, Albania.
Mirela TabakuPediatric Department, University Hospital Center Mother Teresa, Tirana, Albania.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: Enzyme replacement therapy is already recognized as the gold standard of care for patients with Gaucher disease. Taliglucerase alfa is one of the three alternatives recommended for treatment of Gaucher disease in children and adults. Aim: This study aims to evaluate the long-term efficacy and safety of Taliglucerase alfa in children and adolescents with Type 1 Gaucher disease. Patients and methods: Over a six-year period, we monitored the efficacy of continuous treatment in 10 patients by assessing various parameters, including hemoglobin concentration, platelet count, liver and spleen volume, bone mineral density, glucosylsphingosine level, chitotriosidase activity, and growth parameters Results: The mean age of patients was 13.4 ± 3.6 years and the treatment duration was 60.24 ± 13.4 months. From baseline to end line the parameters change as follows: hemoglobin concentration improved from 12.7 (±1.3) to 14.6 (±1.5) and platelet count from 180 (±74) to 198 (±79). The spleen volume, was reduced by 46% ( Conclusion: Taliglucerase alfa exhibits good efficacy and a safe profile in the treatment of children and adolescents with Type 1 Gaucher disease.

Indexed as

adolescentchildrenefficacyenzyme replacement therapyGaucher diseasesafetyTaliglucerase alfa

Identifiers

PMID38464899
PMCPMC10920268

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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.