Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine

Autores
Carmona, Rocío Guadalupe; Montivero, Malena; Quattrocchi, Georgina; Zarelli, Valeria; Giai, Constanza; Quintero, Cristián Andrés
Año de publicación
2025
Idioma
inglés
Tipo de recurso
artículo
Estado
versión publicada
Descripción
Fil: Carmona, Rocío Guadalupe. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Montivero, Malena. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Quattrocchi, Georgina. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Zarelli, Valeria. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Zarelli, Valeria. Universidad Juan Agustín Maza. Laboratorio de Genética, Ambiente y Reproducción; Argentina
Fil: Giai, Constanza. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Giai, Constanza. Universidad Juan Agustín Maza. Laboratorio de Biología Molecular y Celular; Argentina
Fil: Quintero, Cristián Andrés. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Quintero, Cristián Andrés. Universidad Juan Agustín Maza. Laboratorio de Biología Molecular y Celular; Argentina
Background: Acute lymphoblastic leukemia (ALL) is a heterogeneous hematological malignancy predominantly affecting individuals under 20 years of age. Traditional chemotherapy, such as clofarabine, has shown efficacy; however, novel immunotherapeutic strategies like tisagenlecleucel (Kymriah®) have significantly altered the treatment paradigm. Aim: This study aimed to perform a comparative analysis of tisagenlecleucel, a CAR-T cell therapy, and clofarabine, a second-generation purine nucleoside analog, evaluating their mechanisms of action, therapeutic benefits, limitations, and clinical applicability across diverse patient populations. Methods: A systematic comparative evaluation was conducted, encompassing pharmacological characteristics, mechanisms of action, treatment protocols, efficacy, safety profiles, and clinical indications of both agents. The análisis considered pharmacokinetic and pharmacodynamic data and included patient demographic variables. Results: Tisagenlecleucel demonstrated high efficacy in refractory B-cell ALL, with durable responses and a blood half-life of 128 days, but with notable immune-related adverse effects such as cytokine release syndrome. Clofarabine, effective across a broader patient population, acts via multiple antitumor mechanisms but carries significant toxicity risks, including infection and sepsis. Discussion: The therapies present distinct clinical profiles: tisagenlecleucel offers targeted immunotherapy with high specificity but requires specialized infrastructure and management of immune toxicities. Clofarabine is more widely accessible and applicable, but is associated with conventional chemotherapy-related side effects. Treatment accessibility and cost differ markedly between the two. Conclusions: Therapy selection should be personalized based on patient-specific factors and institutional resources. Tisagenlecleucel is ideal for pediatric and young adult patients with relapsed/refractory B-cell ALL in CAR-T-capable centers, while clofarabine remains a viable option for broader ALL populations, particularly when genetic therapies are not feasible. Further research is needed to optimize therapeutic strategies and improve access to advanced treatments.
Materia
tisagenlecleucel
clofarabina
leucemia linfoblástica aguda
Nivel de accesibilidad
acceso abierto
Condiciones de uso
https://creativecommons.org/licenses/by-nc-sa/4.0/
Repositorio
UMaza Digital
Institución
Universidad Maza
OAI Identificador
oai:repositorio.umaza.edu.ar:00261/3607

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network_name_str UMaza Digital
spelling Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and ClofarabineCarmona, Rocío GuadalupeMontivero, MalenaQuattrocchi, GeorginaZarelli, ValeriaGiai, ConstanzaQuintero, Cristián Andréstisagenlecleucelclofarabinaleucemia linfoblástica agudaFil: Carmona, Rocío Guadalupe. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; ArgentinaFil: Montivero, Malena. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; ArgentinaFil: Quattrocchi, Georgina. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; ArgentinaFil: Zarelli, Valeria. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; ArgentinaFil: Zarelli, Valeria. Universidad Juan Agustín Maza. Laboratorio de Genética, Ambiente y Reproducción; ArgentinaFil: Giai, Constanza. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; ArgentinaFil: Giai, Constanza. Universidad Juan Agustín Maza. Laboratorio de Biología Molecular y Celular; ArgentinaFil: Quintero, Cristián Andrés. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; ArgentinaFil: Quintero, Cristián Andrés. Universidad Juan Agustín Maza. Laboratorio de Biología Molecular y Celular; ArgentinaBackground: Acute lymphoblastic leukemia (ALL) is a heterogeneous hematological malignancy predominantly affecting individuals under 20 years of age. Traditional chemotherapy, such as clofarabine, has shown efficacy; however, novel immunotherapeutic strategies like tisagenlecleucel (Kymriah®) have significantly altered the treatment paradigm. Aim: This study aimed to perform a comparative analysis of tisagenlecleucel, a CAR-T cell therapy, and clofarabine, a second-generation purine nucleoside analog, evaluating their mechanisms of action, therapeutic benefits, limitations, and clinical applicability across diverse patient populations. Methods: A systematic comparative evaluation was conducted, encompassing pharmacological characteristics, mechanisms of action, treatment protocols, efficacy, safety profiles, and clinical indications of both agents. The análisis considered pharmacokinetic and pharmacodynamic data and included patient demographic variables. Results: Tisagenlecleucel demonstrated high efficacy in refractory B-cell ALL, with durable responses and a blood half-life of 128 days, but with notable immune-related adverse effects such as cytokine release syndrome. Clofarabine, effective across a broader patient population, acts via multiple antitumor mechanisms but carries significant toxicity risks, including infection and sepsis. Discussion: The therapies present distinct clinical profiles: tisagenlecleucel offers targeted immunotherapy with high specificity but requires specialized infrastructure and management of immune toxicities. Clofarabine is more widely accessible and applicable, but is associated with conventional chemotherapy-related side effects. Treatment accessibility and cost differ markedly between the two. Conclusions: Therapy selection should be personalized based on patient-specific factors and institutional resources. Tisagenlecleucel is ideal for pediatric and young adult patients with relapsed/refractory B-cell ALL in CAR-T-capable centers, while clofarabine remains a viable option for broader ALL populations, particularly when genetic therapies are not feasible. Further research is needed to optimize therapeutic strategies and improve access to advanced treatments.2025info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttp://purl.org/coar/resource_type/c_6501info:ar-repo/semantics/articuloapplication/pdfapplication/pdfCarmona, R. G., Montivero, M., Quattrocchi, G., Zarelli, V., Giai, C., & Quintero, C. A. (2025). Treatments for acute lymphoblastic leukemia: A comparison between tisagenlecleucel and clofarabine. Southern Journal of Sciences, 33(39), 25–31. https://doi.org/10.48141/2764-5959.04.v33.n39.2025_QUINTEROS_pgs_25_31https://repositorio.umaza.edu.ar/handle/00261/3607enginfo:eu-repo/semantics/altIdentifier/url/10.48141/2764-5959.04.v33.n39.2025_QUINTEROS_pgs_25_31.pdfinfo:eu-repo/semantics/openAccesshttps://creativecommons.org/licenses/by-nc-sa/4.0/reponame:UMaza Digitalinstname:Universidad Maza2025-09-29T14:29:18Zoai:repositorio.umaza.edu.ar:00261/3607instacron:UMAZAInstitucionalhttp://repositorio.umaza.edu.ar/Universidad privadaNo correspondehttp://repositorio.umaza.edu.ar/oaicienciaytecnica@umaza.edu.arArgentinaNo correspondeNo correspondeNo correspondeopendoar:44192025-09-29 14:29:18.495UMaza Digital - Universidad Mazafalse
dc.title.none.fl_str_mv Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
title Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
spellingShingle Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
Carmona, Rocío Guadalupe
tisagenlecleucel
clofarabina
leucemia linfoblástica aguda
title_short Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
title_full Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
title_fullStr Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
title_full_unstemmed Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
title_sort Treatments for acute lymphoblastic leukemia: A comparison between Tisagenlecleucel and Clofarabine
dc.creator.none.fl_str_mv Carmona, Rocío Guadalupe
Montivero, Malena
Quattrocchi, Georgina
Zarelli, Valeria
Giai, Constanza
Quintero, Cristián Andrés
author Carmona, Rocío Guadalupe
author_facet Carmona, Rocío Guadalupe
Montivero, Malena
Quattrocchi, Georgina
Zarelli, Valeria
Giai, Constanza
Quintero, Cristián Andrés
author_role author
author2 Montivero, Malena
Quattrocchi, Georgina
Zarelli, Valeria
Giai, Constanza
Quintero, Cristián Andrés
author2_role author
author
author
author
author
dc.subject.none.fl_str_mv tisagenlecleucel
clofarabina
leucemia linfoblástica aguda
topic tisagenlecleucel
clofarabina
leucemia linfoblástica aguda
dc.description.none.fl_txt_mv Fil: Carmona, Rocío Guadalupe. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Montivero, Malena. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Quattrocchi, Georgina. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Zarelli, Valeria. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Zarelli, Valeria. Universidad Juan Agustín Maza. Laboratorio de Genética, Ambiente y Reproducción; Argentina
Fil: Giai, Constanza. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Giai, Constanza. Universidad Juan Agustín Maza. Laboratorio de Biología Molecular y Celular; Argentina
Fil: Quintero, Cristián Andrés. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
Fil: Quintero, Cristián Andrés. Universidad Juan Agustín Maza. Laboratorio de Biología Molecular y Celular; Argentina
Background: Acute lymphoblastic leukemia (ALL) is a heterogeneous hematological malignancy predominantly affecting individuals under 20 years of age. Traditional chemotherapy, such as clofarabine, has shown efficacy; however, novel immunotherapeutic strategies like tisagenlecleucel (Kymriah®) have significantly altered the treatment paradigm. Aim: This study aimed to perform a comparative analysis of tisagenlecleucel, a CAR-T cell therapy, and clofarabine, a second-generation purine nucleoside analog, evaluating their mechanisms of action, therapeutic benefits, limitations, and clinical applicability across diverse patient populations. Methods: A systematic comparative evaluation was conducted, encompassing pharmacological characteristics, mechanisms of action, treatment protocols, efficacy, safety profiles, and clinical indications of both agents. The análisis considered pharmacokinetic and pharmacodynamic data and included patient demographic variables. Results: Tisagenlecleucel demonstrated high efficacy in refractory B-cell ALL, with durable responses and a blood half-life of 128 days, but with notable immune-related adverse effects such as cytokine release syndrome. Clofarabine, effective across a broader patient population, acts via multiple antitumor mechanisms but carries significant toxicity risks, including infection and sepsis. Discussion: The therapies present distinct clinical profiles: tisagenlecleucel offers targeted immunotherapy with high specificity but requires specialized infrastructure and management of immune toxicities. Clofarabine is more widely accessible and applicable, but is associated with conventional chemotherapy-related side effects. Treatment accessibility and cost differ markedly between the two. Conclusions: Therapy selection should be personalized based on patient-specific factors and institutional resources. Tisagenlecleucel is ideal for pediatric and young adult patients with relapsed/refractory B-cell ALL in CAR-T-capable centers, while clofarabine remains a viable option for broader ALL populations, particularly when genetic therapies are not feasible. Further research is needed to optimize therapeutic strategies and improve access to advanced treatments.
description Fil: Carmona, Rocío Guadalupe. Universidad Juan Agustín Maza. Facultad de Farmacia y Bioquímica; Argentina
publishDate 2025
dc.date.none.fl_str_mv 2025
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info:eu-repo/semantics/publishedVersion
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info:ar-repo/semantics/articulo
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv Carmona, R. G., Montivero, M., Quattrocchi, G., Zarelli, V., Giai, C., & Quintero, C. A. (2025). Treatments for acute lymphoblastic leukemia: A comparison between tisagenlecleucel and clofarabine. Southern Journal of Sciences, 33(39), 25–31. https://doi.org/10.48141/2764-5959.04.v33.n39.2025_QUINTEROS_pgs_25_31
https://repositorio.umaza.edu.ar/handle/00261/3607
identifier_str_mv Carmona, R. G., Montivero, M., Quattrocchi, G., Zarelli, V., Giai, C., & Quintero, C. A. (2025). Treatments for acute lymphoblastic leukemia: A comparison between tisagenlecleucel and clofarabine. Southern Journal of Sciences, 33(39), 25–31. https://doi.org/10.48141/2764-5959.04.v33.n39.2025_QUINTEROS_pgs_25_31
url https://repositorio.umaza.edu.ar/handle/00261/3607
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language eng
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https://creativecommons.org/licenses/by-nc-sa/4.0/
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