Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report
BackgroundWith recent advances in clinical practice, including the use of reduced-toxicity conditioning regimens and innovative approaches such as ex vivo TCRαβ/CD19 depletion of haploidentical donor stem cells or post-transplant cyclophosphamide (PTCY), hematopoietic stem cell transplantation (HSCT...
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Frontiers Media S.A.
2025-01-01
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| Series: | Frontiers in Immunology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fimmu.2024.1475448/full |
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| author | Azadeh Kiumarsi Amirarsalan Alaei Mohsen Nikbakht Saeed Mohammadi Mohammad Reza Ostad-Ali Zahra Rasti Tahereh Rostami |
| author_facet | Azadeh Kiumarsi Amirarsalan Alaei Mohsen Nikbakht Saeed Mohammadi Mohammad Reza Ostad-Ali Zahra Rasti Tahereh Rostami |
| author_sort | Azadeh Kiumarsi |
| collection | DOAJ |
| description | BackgroundWith recent advances in clinical practice, including the use of reduced-toxicity conditioning regimens and innovative approaches such as ex vivo TCRαβ/CD19 depletion of haploidentical donor stem cells or post-transplant cyclophosphamide (PTCY), hematopoietic stem cell transplantation (HSCT) has emerged as a curative treatment option for a growing population of patients with inborn errors of immunity (IEI). However, despite these promising developments, graft failure (GF) remains a significant concern associated with HSCT in these patients. Although a second HSCT is the only established salvage therapy for patients who experience GF, there are no uniform, standardized strategies for performing these second transplants. Furthermore, even less data is available regarding the outcomes and best practices for a third HSCT as a salvage measure when a second HSCT fails to achieve engraftment.Case presentationA 6-year-old boy with leukocyte adhesion deficiency type I (LAD-I) experienced GF after the first and second HSCT from a matched unrelated donor. As a salvage measure, the patient received a dual in vivo T-cell depleted haploidentical HSCT. The conditioning regimen for this third HSCT included anti-thymocyte globulin (ATG) and PTCY. Complete donor chimerism was assessed using the short tandem repeat (STR) PCR technique. By day +28 after the transplant, the expression of the leukocyte adhesion molecules CD18, CD11b, and CD11c on the patient’s peripheral blood neutrophils had recovered to over 99%. It remained stable throughout the 18-month follow-up period.ConclusionT-cell replete haploidentical HSCT with ATG and PTCY may be a viable salvage option for LAD patients who have rejected prior HSCT. |
| format | Article |
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| institution | Kabale University |
| issn | 1664-3224 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Immunology |
| spelling | doaj-art-5bdd2d0df0a545e88a69e57ba8dd2df92025-01-08T06:12:06ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-01-011510.3389/fimmu.2024.14754481475448Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case reportAzadeh Kiumarsi0Amirarsalan Alaei1Mohsen Nikbakht2Saeed Mohammadi3Mohammad Reza Ostad-Ali4Zahra Rasti5Tahereh Rostami6Department of Pediatrics, School of Medicine, Children’s Medical Center, Tehran University of Medical Sciences, Tehran, IranImmunology Research Center, Tabriz University of Medical Sciences, Tabriz, IranCell Therapy and Hematopoietic Stem Cell Transplantation Research Center, Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, Tehran University of Medical Sciences, Tehran, IranHematologic Malignancies Research Center, Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, Tehran University of Medical Sciences, Tehran, IranCell Therapy and Hematopoietic Stem Cell Transplantation Research Center, Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, Tehran University of Medical Sciences, Tehran, IranDepartment of Hematology, School of Allied Medical Sciences, Tehran University of Medical Sciences, Tehran, IranCell Therapy and Hematopoietic Stem Cell Transplantation Research Center, Research Institute for Oncology, Hematology and Cell Therapy, Shariati Hospital, Tehran University of Medical Sciences, Tehran, IranBackgroundWith recent advances in clinical practice, including the use of reduced-toxicity conditioning regimens and innovative approaches such as ex vivo TCRαβ/CD19 depletion of haploidentical donor stem cells or post-transplant cyclophosphamide (PTCY), hematopoietic stem cell transplantation (HSCT) has emerged as a curative treatment option for a growing population of patients with inborn errors of immunity (IEI). However, despite these promising developments, graft failure (GF) remains a significant concern associated with HSCT in these patients. Although a second HSCT is the only established salvage therapy for patients who experience GF, there are no uniform, standardized strategies for performing these second transplants. Furthermore, even less data is available regarding the outcomes and best practices for a third HSCT as a salvage measure when a second HSCT fails to achieve engraftment.Case presentationA 6-year-old boy with leukocyte adhesion deficiency type I (LAD-I) experienced GF after the first and second HSCT from a matched unrelated donor. As a salvage measure, the patient received a dual in vivo T-cell depleted haploidentical HSCT. The conditioning regimen for this third HSCT included anti-thymocyte globulin (ATG) and PTCY. Complete donor chimerism was assessed using the short tandem repeat (STR) PCR technique. By day +28 after the transplant, the expression of the leukocyte adhesion molecules CD18, CD11b, and CD11c on the patient’s peripheral blood neutrophils had recovered to over 99%. It remained stable throughout the 18-month follow-up period.ConclusionT-cell replete haploidentical HSCT with ATG and PTCY may be a viable salvage option for LAD patients who have rejected prior HSCT.https://www.frontiersin.org/articles/10.3389/fimmu.2024.1475448/fullinborn errors of immunity (IEI)leukocyte adhesion deficiency (LAD)hematopoietic stem cell transplantation (HSCT)haploidenticalgraft failure (GF) |
| spellingShingle | Azadeh Kiumarsi Amirarsalan Alaei Mohsen Nikbakht Saeed Mohammadi Mohammad Reza Ostad-Ali Zahra Rasti Tahereh Rostami Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report Frontiers in Immunology inborn errors of immunity (IEI) leukocyte adhesion deficiency (LAD) hematopoietic stem cell transplantation (HSCT) haploidentical graft failure (GF) |
| title | Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report |
| title_full | Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report |
| title_fullStr | Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report |
| title_full_unstemmed | Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report |
| title_short | Dual in vivo T cell depleted haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and anti-thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure: a case report |
| title_sort | dual in vivo t cell depleted haploidentical hematopoietic stem cell transplantation with post transplant cyclophosphamide and anti thymocyte globulin as a third salvage transplant for leukocyte adhesion deficiency with graft failure a case report |
| topic | inborn errors of immunity (IEI) leukocyte adhesion deficiency (LAD) hematopoietic stem cell transplantation (HSCT) haploidentical graft failure (GF) |
| url | https://www.frontiersin.org/articles/10.3389/fimmu.2024.1475448/full |
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