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Allogeneic hematopoietic stem cell transplantation (HSCT) is a combination of replacement medicine (rather than surgery) and immunotherapy. Whatever the transplanted stem cell source (i.e. bone marrow, peripheral blood stem cells or cord blood stem cells), the graft provide both new hematopoiesis (erythrocyte, white blood cell and platelet-production) and new "immunopoiesis". This new infused immunologic system is able to participate in the underlying malignant disease control in association with previous chemotherapy and/or radiotherapy by providing graft-versus-leukelia effect. The main steps of HSCT are 1. Administration of a conditioning regimen, mainly myelo-ablative and then toxic in the frame of acute leukemia in children and adolescents; 2. Management of prolonged and severe neutropenia and of chemotherapy-related toxicities; 3. Management of possible immunological conflict between competent T-cells from the graft and recipient tissues (namely graft versus host disease); 4. Relapse occurrence monitoring and management; 5. Management of HSCT related late-effects and sequelae. [ABSTRACT FROM AUTHOR]

Background: The only French center for pediatric oncology and hematology outside of the metropolitan territory is in the Indian Ocean, in Saint Denis, on Reunion Island. It welcomes children from Reunion Island but also from Mayotte and neighboring countries. A quarter of them requires a secondary medical transfer to metropolitan France for specific technic care., Method: We conducted a retrospective single-center study of all pediatric medical evacuations that occurred between 2015 and 2019 from the pediatric oncology and hematology department of Reunion Island. The purpose of this study is to describe these transfers and the consequences of these care pathways for families and care teams., Results: A total of 189 transfers took place for 105 children: 66 from Reunion Island, 17 from Mayotte and 22 were foreigners. In total, 92 % of the children received the medical care for which they were transferred to metropolitan France. Difficulties were reported: family for 26 % of them, social in 11 % of cases and medical in 10 % of medical records., Conclusions: This organization allows children in the Indian Ocean to benefit from similar care than metropolitan children. Many difficulties arise in connection with family and societal breakdowns caused by these transfers. These differences and difficulties are important to know to better accompany patients, families and caregivers in this stage of their medical pathways., (Copyright © 2022 Société Française du Cancer. Published by Elsevier Masson SAS. All rights reserved.)

Inherited Metabolic Diseases (IMD) are rare genetic diseases, including both lysosomal and peroxisomal diseases. Lysosomal diseases are related to the deficiency of one or more lysosomal enzymes or transporter. Lysosomal diseases are progressive and involve several tissues with most often neurological damage. Among peroxisomal diseases, X-linked adrenoleukodystrophy (ALD) is a neurodegenerative disease combining neurological and adrenal damage. For these diseases, enzyme replacement therapy (ERT), allogeneic hematopoietic cell transplantation (allo-HCT) and gene therapy represent various possible treatment options, used alone or in combination. The purpose of this workshop is to describe the indications, modalities, and follow-up of allo-HCT as well as the use of ERT peri-transplant. All indications for transplant in these rare diseases are associated with comorbidities and are subject to criteria that must be discussed in a dedicated national multidisciplinary consultation meeting. There are some consensual indications in type I-H mucopolysaccharidosis (MPS-IH) and in the cerebral form of ALD. For other IMDs, no clear benefit from the transplant has been demonstrated. The ideal donor is a non-heterozygous HLA-identical sibling. The recommended conditioning is myeloablative combining fludarabine and busulfan. In MPS-IH, ERT has to be started at diagnosis and continued until complete chimerism and normal enzyme assay are achieved. The pre-transplant assessment and post-transplant follow-up are made according to the published recommendations (PNDS). Standard follow-up is carried out jointly by the transplant and referral teams., (Copyright © 2022 Société Française du Cancer. Published by Elsevier Masson SAS. All rights reserved.)

The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 7th allogeneic hematopoietic stem cell transplantation clinical practices harmonization workshop series in September 2016 in Lille, France. The objective of our workshop is to provide a discussion on the conservation and congelation of hematopoietic stem cells in a pediatric setting as well as our recommendations for this technique., (Copyright © 2017 Société Française du Cancer. Published by Elsevier Masson SAS. All rights reserved.)

Success of allogeneic hematopoietic stem cells transplantation requires both the underlying disease eradication and satisfying reconstitution of hematopoiesis from donor cells. However, reconstitution delays, secondary development or persistence of cytopenia are regularly observed and are potential causes of failure after allogeneic transplantation. These graft dysfunctions should be distinguished from non-engraftment/engraftment failure. Although these situations are relatively common, there is no consensus in the literature for their management. During the workshop of the SFGM-TC, the working group proposed recommendations from an analysis of the literature., (Copyright © 2016. Published by Elsevier Masson SAS.)

Better understanding of the antitumor effect of allogeneic transplant and the need to reduce the toxicity of the procedure, particularly in elderly patients have spurred the development of reduced-intensity conditioning regimens (RIC). These regimens allow fast engraftment with very low chemotherapy-induced toxicity. They are widely used in adults and there are numerous studies to demonstrate their feasibility and efficiency, but in pediatrics, the place of RIC remains to be determined. They can be proposed in two pediatric populations. First, solid tumors or hematological malignancies remaining unresponsive to the reference strategies according to best practices in pediatrics. Second, in children presenting malignancies for which allografting is the only recognized curative indication but is contraindicated with myeloablative conditioning regimens. More than 100 pediatrics cases have been reported in various pathologies, including blood diseases, acute leukemia, Hodgkin's lymphoma and solid tumors, and promising results published recently underline how RIC warrants further investigation in prospective comparative multicentric trials. The use of new post-graft treatment modalities is expected to pave the way to the development of RIC in pediatric patients.