Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years
IntroductionAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is a treatment method for a wide range of malignant and non-malignant diseases. Infants constitute a distinct patient group, especially due to their organ immaturity and differences in drug metabolism. The present paper aims...
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Frontiers Media S.A.
2022-08-01
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| Series: | Frontiers in Pediatrics |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fped.2022.956108/full |
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| author | Justyna Miśkiewicz-Bujna Izabella Miśkiewicz-Migoń Zofia Szmit Dawid Przystupski Monika Rosa Anna Król Krzysztof Kałwak Marek Ussowicz Ewa Gorczyńska |
| author_facet | Justyna Miśkiewicz-Bujna Izabella Miśkiewicz-Migoń Zofia Szmit Dawid Przystupski Monika Rosa Anna Król Krzysztof Kałwak Marek Ussowicz Ewa Gorczyńska |
| author_sort | Justyna Miśkiewicz-Bujna |
| collection | DOAJ |
| description | IntroductionAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is a treatment method for a wide range of malignant and non-malignant diseases. Infants constitute a distinct patient group, especially due to their organ immaturity and differences in drug metabolism. The present paper aims to analyse the short- and long-term outcomes after allo-HSCT in infants.Material and methodsIn the study period, 67 patients under 12 months of age underwent allo-HSCT. This study is a retrospective analysis of patient medical records, in the form of paper and electronic documentation.ResultsThe probability of 5-year OS was 69% and 72% in patients with malignant and non-malignant diseases, respectively. The allo-HSCT from a matched donor was associated with improved OS in comparison to haploidentical donor (0.8 vs. 0.58%, p = 0.0425). The overall incidence of acute graft-vs.-host disease (aGVHD) was 59.3%, and grade III–IV aGVHD was diagnosed in 23% of patients. The 100-day non-relapse mortality (NRM) in the study cohort was 17.9%, while the 5-year NRM was 26.9%. Among the causes of NRM, infections occurred in 83.3% of patients, and aGVHD in 16.3% of individuals. Twenty-two children (32.8%) required hospitalization in the pediatric intensive care unit (PICU). The median length of PICU hospitalization was 6 days (range 1 to 12 days). Late sequelae diagnosed during post-transplant surveillance included ocular disorders in 26.8% of patients, cardiac complications in 4.4%, as well as endocrinopathy with short stature (<3rd percentile) in 37.2% and overt hypothyroidism in 35.4%. In the long-term perspective, 83.3% of survivors were able to attend a regular school.ConclusionsImprovements in unrelated donor availability, and better supportive care resulted in better outcomes. Management of infant allo-HSCT recipients requires the formation of multi-disciplinary specialist teams. In addition, the role of parental empowerment must be acknowledged; for example, in speech therapy and rehabilitation. |
| first_indexed | 2024-04-14T03:18:09Z |
| format | Article |
| id | doaj.art-7c7d451e6f5d4646850eac60a8cc6ef2 |
| institution | Directory Open Access Journal |
| issn | 2296-2360 |
| language | English |
| last_indexed | 2024-04-14T03:18:09Z |
| publishDate | 2022-08-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pediatrics |
| spelling | doaj.art-7c7d451e6f5d4646850eac60a8cc6ef22022-12-22T02:15:23ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602022-08-011010.3389/fped.2022.956108956108Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 yearsJustyna Miśkiewicz-Bujna0Izabella Miśkiewicz-Migoń1Zofia Szmit2Dawid Przystupski3Monika Rosa4Anna Król5Krzysztof Kałwak6Marek Ussowicz7Ewa Gorczyńska8Department of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Paediatric Anaesthesiology and Intensive Care, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandDepartment of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, PolandIntroductionAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is a treatment method for a wide range of malignant and non-malignant diseases. Infants constitute a distinct patient group, especially due to their organ immaturity and differences in drug metabolism. The present paper aims to analyse the short- and long-term outcomes after allo-HSCT in infants.Material and methodsIn the study period, 67 patients under 12 months of age underwent allo-HSCT. This study is a retrospective analysis of patient medical records, in the form of paper and electronic documentation.ResultsThe probability of 5-year OS was 69% and 72% in patients with malignant and non-malignant diseases, respectively. The allo-HSCT from a matched donor was associated with improved OS in comparison to haploidentical donor (0.8 vs. 0.58%, p = 0.0425). The overall incidence of acute graft-vs.-host disease (aGVHD) was 59.3%, and grade III–IV aGVHD was diagnosed in 23% of patients. The 100-day non-relapse mortality (NRM) in the study cohort was 17.9%, while the 5-year NRM was 26.9%. Among the causes of NRM, infections occurred in 83.3% of patients, and aGVHD in 16.3% of individuals. Twenty-two children (32.8%) required hospitalization in the pediatric intensive care unit (PICU). The median length of PICU hospitalization was 6 days (range 1 to 12 days). Late sequelae diagnosed during post-transplant surveillance included ocular disorders in 26.8% of patients, cardiac complications in 4.4%, as well as endocrinopathy with short stature (<3rd percentile) in 37.2% and overt hypothyroidism in 35.4%. In the long-term perspective, 83.3% of survivors were able to attend a regular school.ConclusionsImprovements in unrelated donor availability, and better supportive care resulted in better outcomes. Management of infant allo-HSCT recipients requires the formation of multi-disciplinary specialist teams. In addition, the role of parental empowerment must be acknowledged; for example, in speech therapy and rehabilitation.https://www.frontiersin.org/articles/10.3389/fped.2022.956108/fullallogeneichematopoietic (stem) cell transplantation (HCT)infant-ageneurological complicationssequelae |
| spellingShingle | Justyna Miśkiewicz-Bujna Izabella Miśkiewicz-Migoń Zofia Szmit Dawid Przystupski Monika Rosa Anna Król Krzysztof Kałwak Marek Ussowicz Ewa Gorczyńska Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years Frontiers in Pediatrics allogeneic hematopoietic (stem) cell transplantation (HCT) infant-age neurological complications sequelae |
| title | Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years |
| title_full | Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years |
| title_fullStr | Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years |
| title_full_unstemmed | Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years |
| title_short | Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years |
| title_sort | short and long term outcome of allogeneic stem cell transplantation in infants a single center experience over 20 years |
| topic | allogeneic hematopoietic (stem) cell transplantation (HCT) infant-age neurological complications sequelae |
| url | https://www.frontiersin.org/articles/10.3389/fped.2022.956108/full |
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