ArticleMolecular genetics & genomic medicine2018
Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1.
Article in Molecular genetics & genomic medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 2 of them syntheses that pooled it.
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8 citing papers in PubMed, 2 syntheses or guidelines pooled it, 17 citations in OpenAlex.
- Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups.Orphanet journal of rare diseases · 2026Pooled it
- Patient-reported outcomes in Gaucher's disease: a systematic review.Orphanet journal of rare diseases · 2023Pooled it
- Effects of cisplatin, paclitaxel combined with high-dose methotrexate as adjuvant therapy on survival rates in osteosarcoma patients, and analysis of influencing factors.American journal of cancer research · 2025Article
- Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials.Orphanet journal of rare diseases · 2022Article
- Short-term and mid-term effects of thoracoscopic repair of esophageal atresia: No anastomotic leaks or conversions to open technique.Frontiers in surgery · 2022Article
- Assessment of quality of life in Gaucher disease: A methodological approach.Molecular genetics & genomic medicine · 2021Article
- Liver involvement in patients with Gaucher disease types I and III.Molecular genetics and metabolism reports · 2020Article
- Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1.Molecular genetics & genomic medicine · 2018Article
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5 authors.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundGaucher disease type 1 (GD1, OMIM# 230800), is a condition with high impact in patient's quality of life (QoL). We report the improvement in QoL of children with GD1 measured by Lansky play-performance scale (LS) after enzymatic replacement therapy (ERT) and to describe our experience in the treatment of children with GD1.
methodsFive children with diagnosis of GD1 received imiglucerase 60 mg/kg every two weeks. LS, hepatomegaly, splenomegaly, hemoglobin, platelets, and growth rate were measured every 6 months after beginning ERT for 30 months.
resultsAfter ERT, LS increased significantly from 28 ± 16.48 points before ERT to 70 ± 10 (P = 0.0046) and 95 ± 10 (P = 0.0022) points after 6 and 30 months of ERT, respectively; hemoglobin and platelets changed significantly from 9.28 ± 0.61 to 12.40 ± 0.85 (P = 0.0198) and from 71.50 ± 14.89 to 205.00 ± 65.34 (P = 0.0428) after 30 months of ERT, respectively. All patients demonstrated decreased hepatic and splenic size with mean reductions of 66% and 80% at 30 months of treatment and the USG longitudinal axis was reduced in both liver and spleen after ERT.
conclusionThe use of ERT with imiglucerase 60 mg/kg every two weeks has substantial benefits and significantly improves QoL, assessed with Lansky Score, of the five children with GD1 studied.
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