Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project.
Background: The availability of multiple treatments for type 1 Gaucher disease increases the need for real-life studies to evaluate treatment efficacy and safety and provide clinicians with more information to choose the best personalized therapy for their patients. Aims: To determine whether treatm...
| Autores: | , , , , , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Tipo de recurso: | artículo |
| Fecha de publicación: | 2023 |
| País: | España |
| Institución: | Conselleria de Salut i Consum del Govern de les Illes Balears |
| Repositorio: | Docusalut |
| Idioma: | inglés |
| OAI Identifier: | oai:docusalut.com:20.500.13003/24957 |
| Acceso en línea: | https://hdl.handle.net/20.500.13003/24957 |
| Access Level: | acceso abierto |
| Palabra clave: | Biomarkers Lipocalin-2 Gaucher Disease YKL Biomarcadores Lipocalina 2 Enfermedad de Gaucher Eliglustat Glucosylsphingosine SF-36 Type 1 Gaucher disease YKL-40 |
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Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project.Serrano-Gonzalo, IreneLópez de Frutos, LauraLahoz-Gil, CarlosDelgado-Mateos, FranciscoFernández-Galán, María ÁngelesMorales-Conejo, MontserratCalle-Gordo, María VictoriaIbarretxe-Gerediaga, DaianaMadinaveitia-Ochoa, AndrésAlbarracin-Arraigosa, AntonioBalanzat-Muñoz, JoséCorrecher-Medina, PatriciaGarcía-Frade, Luis JavierHernández-Rivas, Jesús MaríaLabbadia, FrancescaLópez-Dupla, Jesus MiguelLozano-Almela, María LuisaMora-Casterá, ElviraNoya-Pereira, María SoledadRuíz-Guinaldo, María ÁngelesTormo-Díaz, María del MarVitoria-Miñana, IsidroArévalo-Vargas, IsidroAndrade-Campos, MarcioGiraldo, PilarBiomarkersLipocalin-2Gaucher DiseaseYKLBiomarcadoresLipocalina 2Enfermedad de GaucherBiomarkersEliglustatGlucosylsphingosineLipocalin-2SF-36Type 1 Gaucher diseaseYKL-40Background: The availability of multiple treatments for type 1 Gaucher disease increases the need for real-life studies to evaluate treatment efficacy and safety and provide clinicians with more information to choose the best personalized therapy for their patients. Aims: To determine whether treatment with eliglustat produces, in adult GD1 patients, ans optimal response in daily clinical practice. Methods: We designed a real-life study with 2 years of follow-up (TRAZELGA [GEE-ELI-2017-01]) to uniformly evaluate the response and adverse events to eliglustat treatment. This study, conducted in 30 patients across Spain and previously treated with other therapies, included the evaluation of safety and efficacy by assessing visceral enlargement, bone disease (DEXA and T and Z scores), concomitant treatments and adverse events, as well as a quality of life evaluation (SF-36). In addition, the quantification of classical biomarkers (chitotriosidase activity, CCL18/PARC and glucosylsphingosine (GluSph)) and new candidates for GD biomarkers (YKL-40, cathepsin S, hepcidin and lipocalin-2 determined by immunoassay) were also assessed. Non-parametric statistical analysis was performed and p < 0.05 was considered statistically significant. Main results: Thirty patients were enrolled in the study. The median age was 41.5 years and the male-female ratio was 1.1:1. 84% of the patients had received ERT and 16% SRT as previous treatment. The most common symptoms at baseline were fatigue (42%) and bone pain (38%), no patient had a bone crisis during the study, and two years after switching, 37% had reduced their use of analgesics. Patient-reported outcomes showed a significant increase in physical function scores (p = 0.027) and physical pain scores (p = 0.010). None of the enrolled patients discontinued treatment due to adverse events, which were mild and transient in nature, mainly gastrointestinal and skin dryness. None of the biomarkers show a significant increase or decompensation after switching. CCL18/PARC (p = 0.0012), YKL-40 (p = 0.00004) and lipocalin-2 (p = 0.0155) improved after two years and GluSph after one year (p = 0.0008) and two years (p = 0.0245) of oral therapy. Conclusion: In summary, this real-life study, showed that eliglustat maintains stability and can improve quality of life with few side effects. Significant reductions in classic and other novel biomarkers were observed after two years of therapy.BMC20232023-01-0120232023-01-01research articlehttp://purl.org/coar/resource_type/c_2df8fbb1info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/20.500.13003/24957reponame:Docusalutinstname:Conselleria de Salut i Consum del Govern de les Illes BalearsInglésengopen accesshttp://purl.org/coar/access_right/c_abf2info:eu-repo/semantics/openAccessoai:docusalut.com:20.500.13003/249572026-06-22T12:44:07Z |
| dc.title.none.fl_str_mv |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| title |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| spellingShingle |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. Serrano-Gonzalo, Irene Biomarkers Lipocalin-2 Gaucher Disease YKL Biomarcadores Lipocalina 2 Enfermedad de Gaucher Biomarkers Eliglustat Glucosylsphingosine Lipocalin-2 SF-36 Type 1 Gaucher disease YKL-40 |
| title_short |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| title_full |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| title_fullStr |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| title_full_unstemmed |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| title_sort |
Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project. |
| dc.creator.none.fl_str_mv |
Serrano-Gonzalo, Irene López de Frutos, Laura Lahoz-Gil, Carlos Delgado-Mateos, Francisco Fernández-Galán, María Ángeles Morales-Conejo, Montserrat Calle-Gordo, María Victoria Ibarretxe-Gerediaga, Daiana Madinaveitia-Ochoa, Andrés Albarracin-Arraigosa, Antonio Balanzat-Muñoz, José Correcher-Medina, Patricia García-Frade, Luis Javier Hernández-Rivas, Jesús María Labbadia, Francesca López-Dupla, Jesus Miguel Lozano-Almela, María Luisa Mora-Casterá, Elvira Noya-Pereira, María Soledad Ruíz-Guinaldo, María Ángeles Tormo-Díaz, María del Mar Vitoria-Miñana, Isidro Arévalo-Vargas, Isidro Andrade-Campos, Marcio Giraldo, Pilar |
| author |
Serrano-Gonzalo, Irene |
| author_facet |
Serrano-Gonzalo, Irene López de Frutos, Laura Lahoz-Gil, Carlos Delgado-Mateos, Francisco Fernández-Galán, María Ángeles Morales-Conejo, Montserrat Calle-Gordo, María Victoria Ibarretxe-Gerediaga, Daiana Madinaveitia-Ochoa, Andrés Albarracin-Arraigosa, Antonio Balanzat-Muñoz, José Correcher-Medina, Patricia García-Frade, Luis Javier Hernández-Rivas, Jesús María Labbadia, Francesca López-Dupla, Jesus Miguel Lozano-Almela, María Luisa Mora-Casterá, Elvira Noya-Pereira, María Soledad Ruíz-Guinaldo, María Ángeles Tormo-Díaz, María del Mar Vitoria-Miñana, Isidro Arévalo-Vargas, Isidro Andrade-Campos, Marcio Giraldo, Pilar |
| author_role |
author |
| author2 |
López de Frutos, Laura Lahoz-Gil, Carlos Delgado-Mateos, Francisco Fernández-Galán, María Ángeles Morales-Conejo, Montserrat Calle-Gordo, María Victoria Ibarretxe-Gerediaga, Daiana Madinaveitia-Ochoa, Andrés Albarracin-Arraigosa, Antonio Balanzat-Muñoz, José Correcher-Medina, Patricia García-Frade, Luis Javier Hernández-Rivas, Jesús María Labbadia, Francesca López-Dupla, Jesus Miguel Lozano-Almela, María Luisa Mora-Casterá, Elvira Noya-Pereira, María Soledad Ruíz-Guinaldo, María Ángeles Tormo-Díaz, María del Mar Vitoria-Miñana, Isidro Arévalo-Vargas, Isidro Andrade-Campos, Marcio Giraldo, Pilar |
| author2_role |
author author author author author author author author author author author author author author author author author author author author author author author author |
| dc.contributor.none.fl_str_mv |
|
| dc.subject.none.fl_str_mv |
Biomarkers Lipocalin-2 Gaucher Disease YKL Biomarcadores Lipocalina 2 Enfermedad de Gaucher Biomarkers Eliglustat Glucosylsphingosine Lipocalin-2 SF-36 Type 1 Gaucher disease YKL-40 |
| topic |
Biomarkers Lipocalin-2 Gaucher Disease YKL Biomarcadores Lipocalina 2 Enfermedad de Gaucher Biomarkers Eliglustat Glucosylsphingosine Lipocalin-2 SF-36 Type 1 Gaucher disease YKL-40 |
| description |
Background: The availability of multiple treatments for type 1 Gaucher disease increases the need for real-life studies to evaluate treatment efficacy and safety and provide clinicians with more information to choose the best personalized therapy for their patients. Aims: To determine whether treatment with eliglustat produces, in adult GD1 patients, ans optimal response in daily clinical practice. Methods: We designed a real-life study with 2 years of follow-up (TRAZELGA [GEE-ELI-2017-01]) to uniformly evaluate the response and adverse events to eliglustat treatment. This study, conducted in 30 patients across Spain and previously treated with other therapies, included the evaluation of safety and efficacy by assessing visceral enlargement, bone disease (DEXA and T and Z scores), concomitant treatments and adverse events, as well as a quality of life evaluation (SF-36). In addition, the quantification of classical biomarkers (chitotriosidase activity, CCL18/PARC and glucosylsphingosine (GluSph)) and new candidates for GD biomarkers (YKL-40, cathepsin S, hepcidin and lipocalin-2 determined by immunoassay) were also assessed. Non-parametric statistical analysis was performed and p < 0.05 was considered statistically significant. Main results: Thirty patients were enrolled in the study. The median age was 41.5 years and the male-female ratio was 1.1:1. 84% of the patients had received ERT and 16% SRT as previous treatment. The most common symptoms at baseline were fatigue (42%) and bone pain (38%), no patient had a bone crisis during the study, and two years after switching, 37% had reduced their use of analgesics. Patient-reported outcomes showed a significant increase in physical function scores (p = 0.027) and physical pain scores (p = 0.010). None of the enrolled patients discontinued treatment due to adverse events, which were mild and transient in nature, mainly gastrointestinal and skin dryness. None of the biomarkers show a significant increase or decompensation after switching. CCL18/PARC (p = 0.0012), YKL-40 (p = 0.00004) and lipocalin-2 (p = 0.0155) improved after two years and GluSph after one year (p = 0.0008) and two years (p = 0.0245) of oral therapy. Conclusion: In summary, this real-life study, showed that eliglustat maintains stability and can improve quality of life with few side effects. Significant reductions in classic and other novel biomarkers were observed after two years of therapy. |
| publishDate |
2023 |
| dc.date.none.fl_str_mv |
2023 2023-01-01 2023 2023-01-01 |
| dc.type.none.fl_str_mv |
research article http://purl.org/coar/resource_type/c_2df8fbb1 |
| dc.type.openaire.fl_str_mv |
info:eu-repo/semantics/article |
| format |
article |
| dc.identifier.none.fl_str_mv |
https://hdl.handle.net/20.500.13003/24957 |
| url |
https://hdl.handle.net/20.500.13003/24957 |
| dc.language.none.fl_str_mv |
Inglés eng |
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Inglés |
| language |
eng |
| dc.rights.none.fl_str_mv |
open access http://purl.org/coar/access_right/c_abf2 |
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info:eu-repo/semantics/openAccess |
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open access http://purl.org/coar/access_right/c_abf2 |
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openAccess |
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application/pdf |
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BMC |
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BMC |
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reponame:Docusalut instname:Conselleria de Salut i Consum del Govern de les Illes Balears |
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Conselleria de Salut i Consum del Govern de les Illes Balears |
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Docusalut |
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15.811543 |