Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia.
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| Title: | Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia. |
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| Alternate Title: | Trasplante haploidéntico de células progenitoras hematopoyéticas con ciclofosfamida después del trasplante en errores innatos de la inmunidad: experiencia en un centro de referencia en Colombia. |
| Authors: | Medina, Diego1,2, Orlando Castro, Jhonier2, Esteban Castro, David2, Beltrán, Estefanía3, Manzi, Eliana2,3, Antonio Franco, Alexis1,2, Olaya, Manuela2,4 |
| Source: | Biomédica: Revista del Instituto Nacional de Salud. 2024 Supplement, Vol. 44, p118-130. 13p. |
| Subjects: | HEMATOPOIETIC stem cell transplantation, SEVERE combined immunodeficiency, CHILD patients, T cells, B cells |
| Abstract (English): | Introduction. Inborn errors of immunity is a diverse group of rare diseases caused by over 400 genetic mutations affecting the immune system and increasing infection susceptibility, autoimmunity, and malignancy. Hematopoietic stem cell transplantation offers a curative option for some inborn errors of immunity, with haploidentical donors providing a viable alternative when identical donors are unavailable. Objective. To determine survival, usefulness of weekly chimerism monitoring, immune reconstitution, and complications in patients with inborn errors of immunity who underwent haploidentical hematopoietic stem cell transplantation at a reference center in Colombia. Materials and methods. We conducted a retrospective and observational study of a case series of pediatric patients who underwent haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and follow-up with weekly chimerism. Survival analysis was performed using the Kaplan-Meier method. Results. Sixteen patients with haploidentical familial donor transplantation were included. The most frequent diagnosis was severe combined immunodeficiency (n=5). Eleven out of seventeen patients received a non-myeloablative conditioning regimen. Twelve out of sixteen patients developed acute graft-versus-host disease. Out of these, 3 corresponded to grades III-IV. Post-transplant infections affected 14 of the subjects, predominating bacterial agents. Median T-cell chimerism was greater than 80% during the follow-up. Reconstitution of B and T lymphocytes was achieved in more than 80%. Overall survival at five years was 81%. Survival at 100 days was 94%. Conclusion. Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide is a viable alternative for inborn errors of immunity when an identical donor is unavailable. Serial chimerism monitoring is useful for graft follow-up. [ABSTRACT FROM AUTHOR] |
| Abstract (Spanish): | Introducción. Los errores innatos de la inmunidad son enfermedades raras causadas por más de 400 mutaciones genéticas que afectan al sistema inmunológico y aumentan la susceptibilidad a infecciones, la autoinmunidad y las neoplasias malignas. El trasplante de progenitores hematopoyéticos es una opción curativa para algunos errores innatos de la inmunidad y, específicamente, el trasplante haploidéntico es una alternativa viable en ausencia de un donante idéntico. Objetivo. Determinar la supervivencia, utilidad del monitoreo semanal del quimerismo, reconstitución inmunológica y complicaciones de los pacientes con errores innatos de la inmunidad sometidos a trasplante haploidéntico de progenitores hematopoyéticos en un centro de referencia en Colombia. Materiales y métodos. Se realizó un estudio observacional, retrospectivo, de serie de casos, con pacientes pediátricos sometidos a trasplante haploidéntico de progenitores hematopoyéticos, usando ciclofosfamida después del trasplante y haciendo seguimiento semanal de quimerismo. Se analizó la supervivencia con el método de Kaplan-Meier. Resultados. Se incluyeron 16 pacientes con trasplante de donante familiar haploidéntico. El diagnóstico más frecuente fue inmunodeficiencia combinada grave (n = 5). Once de diecisiete recibieron un esquema de acondicionamiento no mieloablativo. La enfermedad aguda de injerto contra huésped se desarrolló en 12 de 16 pacientes y en 3 fue de grado III-IV. Las infecciones posteriores al trasplante afectaron a 14 pacientes, con predominio de agentes bacterianos. La mediana de quimerismo de células T fue mayor al 80 % durante el seguimiento. La reconstitución de linfocitos B y T se logró en más de 11 pacientes La supervivencia global a cinco años fue del 81 % y, a 100 días, fue del 91 %. Conclusión. El trasplante haploidéntico de progenitores hematopoyéticos utilizando ciclofosfamida postrasplante, es una alternativa factible en pacientes con errores innatos de la inmunidad en ausencia de un donante idéntico. El monitoreo seriado del quimerismo es útil para el seguimiento del injerto. [ABSTRACT FROM AUTHOR] |
| Copyright of Biomédica: Revista del Instituto Nacional de Salud is the property of Instituto Nacional de Salud of Colombia and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.) | |
| Database: | MedicLatina |
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| Items | – Name: Title Label: Title Group: Ti Data: Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia. – Name: TitleAlt Label: Alternate Title Group: TiAlt Data: Trasplante haploidéntico de células progenitoras hematopoyéticas con ciclofosfamida después del trasplante en errores innatos de la inmunidad: experiencia en un centro de referencia en Colombia. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AR" term="%22Medina%2C+Diego%22">Medina, Diego</searchLink><relatesTo>1,2</relatesTo><br /><searchLink fieldCode="AR" term="%22Orlando+Castro%2C+Jhonier%22">Orlando Castro, Jhonier</searchLink><relatesTo>2</relatesTo><br /><searchLink fieldCode="AR" term="%22Esteban+Castro%2C+David%22">Esteban Castro, David</searchLink><relatesTo>2</relatesTo><br /><searchLink fieldCode="AR" term="%22Beltrán%2C+Estefanía%22">Beltrán, Estefanía</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Manzi%2C+Eliana%22">Manzi, Eliana</searchLink><relatesTo>2,3</relatesTo><br /><searchLink fieldCode="AR" term="%22Antonio+Franco%2C+Alexis%22">Antonio Franco, Alexis</searchLink><relatesTo>1,2</relatesTo><br /><searchLink fieldCode="AR" term="%22Olaya%2C+Manuela%22">Olaya, Manuela</searchLink><relatesTo>2,4</relatesTo> – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22Biomédica%3A+Revista+del+Instituto+Nacional+de+Salud%22">Biomédica: Revista del Instituto Nacional de Salud</searchLink>. 2024 Supplement, Vol. 44, p118-130. 13p. – Name: Subject Label: Subjects Group: Su Data: <searchLink fieldCode="DE" term="%22HEMATOPOIETIC+stem+cell+transplantation%22">HEMATOPOIETIC stem cell transplantation</searchLink><br /><searchLink fieldCode="DE" term="%22SEVERE+combined+immunodeficiency%22">SEVERE combined immunodeficiency</searchLink><br /><searchLink fieldCode="DE" term="%22CHILD+patients%22">CHILD patients</searchLink><br /><searchLink fieldCode="DE" term="%22T+cells%22">T cells</searchLink><br /><searchLink fieldCode="DE" term="%22B+cells%22">B cells</searchLink> – Name: Abstract Label: Abstract (English) Group: Ab Data: Introduction. Inborn errors of immunity is a diverse group of rare diseases caused by over 400 genetic mutations affecting the immune system and increasing infection susceptibility, autoimmunity, and malignancy. Hematopoietic stem cell transplantation offers a curative option for some inborn errors of immunity, with haploidentical donors providing a viable alternative when identical donors are unavailable. Objective. To determine survival, usefulness of weekly chimerism monitoring, immune reconstitution, and complications in patients with inborn errors of immunity who underwent haploidentical hematopoietic stem cell transplantation at a reference center in Colombia. Materials and methods. We conducted a retrospective and observational study of a case series of pediatric patients who underwent haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and follow-up with weekly chimerism. Survival analysis was performed using the Kaplan-Meier method. Results. Sixteen patients with haploidentical familial donor transplantation were included. The most frequent diagnosis was severe combined immunodeficiency (n=5). Eleven out of seventeen patients received a non-myeloablative conditioning regimen. Twelve out of sixteen patients developed acute graft-versus-host disease. Out of these, 3 corresponded to grades III-IV. Post-transplant infections affected 14 of the subjects, predominating bacterial agents. Median T-cell chimerism was greater than 80% during the follow-up. Reconstitution of B and T lymphocytes was achieved in more than 80%. Overall survival at five years was 81%. Survival at 100 days was 94%. Conclusion. Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide is a viable alternative for inborn errors of immunity when an identical donor is unavailable. Serial chimerism monitoring is useful for graft follow-up. [ABSTRACT FROM AUTHOR] – Name: Abstract Label: Abstract (Spanish) Group: Ab Data: Introducción. Los errores innatos de la inmunidad son enfermedades raras causadas por más de 400 mutaciones genéticas que afectan al sistema inmunológico y aumentan la susceptibilidad a infecciones, la autoinmunidad y las neoplasias malignas. El trasplante de progenitores hematopoyéticos es una opción curativa para algunos errores innatos de la inmunidad y, específicamente, el trasplante haploidéntico es una alternativa viable en ausencia de un donante idéntico. Objetivo. Determinar la supervivencia, utilidad del monitoreo semanal del quimerismo, reconstitución inmunológica y complicaciones de los pacientes con errores innatos de la inmunidad sometidos a trasplante haploidéntico de progenitores hematopoyéticos en un centro de referencia en Colombia. Materiales y métodos. Se realizó un estudio observacional, retrospectivo, de serie de casos, con pacientes pediátricos sometidos a trasplante haploidéntico de progenitores hematopoyéticos, usando ciclofosfamida después del trasplante y haciendo seguimiento semanal de quimerismo. Se analizó la supervivencia con el método de Kaplan-Meier. Resultados. Se incluyeron 16 pacientes con trasplante de donante familiar haploidéntico. El diagnóstico más frecuente fue inmunodeficiencia combinada grave (n = 5). Once de diecisiete recibieron un esquema de acondicionamiento no mieloablativo. La enfermedad aguda de injerto contra huésped se desarrolló en 12 de 16 pacientes y en 3 fue de grado III-IV. Las infecciones posteriores al trasplante afectaron a 14 pacientes, con predominio de agentes bacterianos. La mediana de quimerismo de células T fue mayor al 80 % durante el seguimiento. La reconstitución de linfocitos B y T se logró en más de 11 pacientes La supervivencia global a cinco años fue del 81 % y, a 100 días, fue del 91 %. Conclusión. El trasplante haploidéntico de progenitores hematopoyéticos utilizando ciclofosfamida postrasplante, es una alternativa factible en pacientes con errores innatos de la inmunidad en ausencia de un donante idéntico. El monitoreo seriado del quimerismo es útil para el seguimiento del injerto. [ABSTRACT FROM AUTHOR] – Name: AbstractSuppliedCopyright Label: Group: Ab Data: <i>Copyright of Biomédica: Revista del Instituto Nacional de Salud is the property of Instituto Nacional de Salud of Colombia and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.) |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.7705/biomedica.7560 Languages: – Code: eng Text: English PhysicalDescription: Pagination: PageCount: 13 StartPage: 118 Subjects: – SubjectFull: HEMATOPOIETIC stem cell transplantation Type: general – SubjectFull: SEVERE combined immunodeficiency Type: general – SubjectFull: CHILD patients Type: general – SubjectFull: T cells Type: general – SubjectFull: B cells Type: general Titles: – TitleFull: Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Medina, Diego – PersonEntity: Name: NameFull: Orlando Castro, Jhonier – PersonEntity: Name: NameFull: Esteban Castro, David – PersonEntity: Name: NameFull: Beltrán, Estefanía – PersonEntity: Name: NameFull: Manzi, Eliana – PersonEntity: Name: NameFull: Antonio Franco, Alexis – PersonEntity: Name: NameFull: Olaya, Manuela IsPartOfRelationships: – BibEntity: Dates: – D: 02 M: 12 Text: 2024 Supplement Type: published Y: 2024 Identifiers: – Type: issn-print Value: 01204157 Numbering: – Type: volume Value: 44 Titles: – TitleFull: Biomédica: Revista del Instituto Nacional de Salud Type: main |
| ResultId | 1 |
