Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia
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...
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Instituto Nacional de Salud
2024-12-01
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| Series: | Biomédica: revista del Instituto Nacional de Salud |
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| Online Access: | https://revistabiomedica.org/index.php/biomedica/article/view/7560 |
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| author | Diego Medina Jhonier Orlando Castro David Esteban Castro Estefanía Beltrán Eliana Manzi Alexis Antonio Franco Manuela Olaya |
| author_facet | Diego Medina Jhonier Orlando Castro David Esteban Castro Estefanía Beltrán Eliana Manzi Alexis Antonio Franco Manuela Olaya |
| author_sort | Diego Medina |
| collection | DOAJ |
| description | 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. |
| format | Article |
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| institution | Kabale University |
| issn | 0120-4157 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Instituto Nacional de Salud |
| record_format | Article |
| series | Biomédica: revista del Instituto Nacional de Salud |
| spelling | doaj-art-cc38658388b14b6296b5542258e9ffff2025-01-21T18:56:10ZengInstituto Nacional de SaludBiomédica: revista del Instituto Nacional de Salud0120-41572024-12-0144Sp. 211813010.7705/biomedica.75609015Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in ColombiaDiego Medina0https://orcid.org/0000-0002-1465-7957Jhonier Orlando Castro1https://orcid.org/0000-0002-8155-5985David Esteban Castro2https://orcid.org/0000-0001-9177-3698Estefanía Beltrán3https://orcid.org/0000-0003-2438-8356Eliana Manzi4https://orcid.org/0000-0001-5577-970XAlexis Antonio Franco5https://orcid.org/0000-0002-3173-6059Manuela Olaya6https://orcid.org/0000-0002-3239-8928Unidad de Trasplante de Progenitores Hematopoyéticos, Servicio de Hemato-Oncología Pediátrica, Departamento Materno Infantil, Fundación Valle del Lili, Cali, Colombia; Facultad de Ciencias de la Salud, Universidad ICESI, Cali, ColombiaFacultad de Ciencias de la Salud, Universidad ICESI, Cali, ColombiaFacultad de Ciencias de la Salud, Universidad ICESI, Cali, ColombiaCentro de Investigaciones Clínicas, Fundación Valle del Lili, Cali, ColombiaFacultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia; Centro de Investigaciones Clínicas, Fundación Valle del Lili, Cali, ColombiaUnidad de Trasplante de Progenitores Hematopoyéticos, Servicio de Hemato-Oncología Pediátrica, Departamento Materno Infantil, Fundación Valle del Lili, Cali, Colombia; Facultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombiaacultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia; Servicio de Alergología e Inmunología Pediátrica, Departamento Materno-Infantil, Fundación Valle del Lili, Cali, ColombiaIntroduction. 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.https://revistabiomedica.org/index.php/biomedica/article/view/7560hematopoietic stem cell transplantationimmune reconstitution |
| spellingShingle | Diego Medina Jhonier Orlando Castro David Esteban Castro Estefanía Beltrán Eliana Manzi Alexis Antonio Franco Manuela Olaya Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia Biomédica: revista del Instituto Nacional de Salud hematopoietic stem cell transplantation immune reconstitution |
| title | Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia |
| title_full | Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia |
| title_fullStr | Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia |
| title_full_unstemmed | Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia |
| title_short | Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia |
| title_sort | haploidentical hematopoietic stem cell transplantation using post transplant cyclophosphamide in patients with inborn errors of immunity experience in a reference center in colombia |
| topic | hematopoietic stem cell transplantation immune reconstitution |
| url | https://revistabiomedica.org/index.php/biomedica/article/view/7560 |
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