Non-endocrine late complications in children after allogeneic haematopoietic SCT
M Faraci1, A N Békássy, V De Fazio
1Bone Marrow Transplantation Unit, Department of Haematology-Oncology, G Gaslini Children's Research Institute, Genova, Italy. maurafaraci@ospedale-gaslini.ge.it
Insights
Long-term survivors of paediatric haematopoietic stem cell transplant (HSCT) face non-endocrine complications. Vigilant surveillance and prompt intervention are crucial for managing late sequelae and improving outcomes.
Area of Science:
- Pediatric Hematology
- Transplant Immunology
- Oncology
Background:
- Children surviving hematopoietic stem cell transplant (HSCT) can experience diverse long-term non-endocrine complications.
- These late sequelae arise from preparative regimens (chemotherapy, radiotherapy), immune reconstitution, and chronic graft-versus-host disease (GvHD).
Purpose of the Study:
- To review the spectrum of late non-endocrine complications in pediatric HSCT survivors.
- To highlight the role of immune reconstitution and GvHD in late clinical abnormalities and infections.
- To discuss risks associated with novel transplant modalities and increased risk of second malignancies.
Main Methods:
- Literature review focusing on long-term complications after pediatric HSCT.
- Analysis of factors contributing to late sequelae, including preparative regimens and immune-related events.
- Examination of risks associated with advanced HSCT techniques and surveillance strategies.
Main Results:
- Preparative regimens can cause permanent organ damage.
- Immune reconstitution and chronic GvHD are key factors in late complications and infections.
- Novel transplant modalities may increase autoimmune syndromes; chemo/radiotherapy elevates second malignancy risk.
Conclusions:
- A proactive surveillance strategy is essential for vigilant post-transplant care in pediatric HSCT survivors.
- Paediatricians must be knowledgeable in monitoring and managing these potential complications.
- Effective management aims to minimize suffering and healthcare costs, representing a significant challenge in pediatric HSCT.
Abstract:
Non-endocrine events represent a heterogeneous group of complications occurring in children who survive long term after haematopoietic SCT. This review highlights the late sequel in a growing child. The preparative regimen itself with high-dose chemotherapy and/or radiotherapy (TBI) or the treatment given before the transplant procedure may cause organ damage with permanent sequel. Immune reconstitution and chronic GvHD have crucial role in occurrence of clinical abnormalities and late severe infections. Autoimmune syndromes may occur after use of novel transplant modalities (cord blood transplantation, reduced intensity conditioning regimen and haploidentical T-cell-depleted SCTs). Exposure to chemo- and/or radiotherapy increases the risk of second malignant neoplasms. Surveillance strategy focusing on each potential complication risk at continuous follow-up will allow vigilant post transplant care. Each paediatrician must be well versed in appropriate monitoring of these complications. Guidelines and recommendations are provided for serious problems occurring at follow-up, which must rapidly be identified so that appropriate intervention can be initiated. To achieve cure at a lowest possible price in terms of suffering and cost expenditures for health care is an extended frontier of paediatric haematopoietic SCT and biggest challenge for a paediatrician.
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