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Management of endocrino-metabolic dysfunctions after allogeneic hematopoietic stem cell transplantation

Allogeneic hematopoietic stem cell transplantation is mainly indicated in bone marrow dysfunction related to blood diseases, but also in some rare diseases (adrenoleucodystrophy, mitochondrial neurogastrointestinal encephalomyopathy or MNGIE...). After decades, this treatment has proven to be effici...

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Published in:Orphanet journal of rare diseases 2014-10, Vol.9 (1), p.162-162, Article 162
Main Authors: Vantyghem, Marie-Christine, Cornillon, Jérôme, Decanter, Christine, Defrance, Frédérique, Karrouz, Wassila, Leroy, Clara, Le Mapihan, Kristell, Couturier, Marie-Anne, De Berranger, Eva, Hermet, Eric, Maillard, Natacha, Marcais, Ambroise, Francois, Sylvie, Tabrizi, Reza, Yakoub-Agha, Ibrahim
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Language:English
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Summary:Allogeneic hematopoietic stem cell transplantation is mainly indicated in bone marrow dysfunction related to blood diseases, but also in some rare diseases (adrenoleucodystrophy, mitochondrial neurogastrointestinal encephalomyopathy or MNGIE...). After decades, this treatment has proven to be efficient at the cost of numerous early and delayed side effects such as infection, graft-versus-host disease, cardiovascular complications and secondary malignancies. These complications are mainly related to the conditioning, which requires a powerful chemotherapy associated to total body irradiation (myelo-ablation) or immunosuppression (non myelo-ablation). Among side effects, the endocrine complications may be classified as 1) hormonal endocrine deficiencies (particularly gonado- and somatotropic) related to delayed consequences of chemo- and above all radiotherapy, with their consequences on growth, puberty, bone and fertility); 2) auto-immune diseases, particularly dysthyroidism; 3) secondary tumors involving either endocrine glands (thyroid carcinoma) or dependent on hormonal status (breast cancer, meningioma), favored by immune dysregulation and radiotherapy; 4) metabolic complications, especially steroid-induced diabetes and dyslipidemia with their increased cardio-vascular risk. These complications are intricate. Moreover, hormone replacement therapy can modulate the cardio-vascular or the tumoral risk of patients, already increased by radiotherapy and chemotherapy, especially steroids and anthracyclins... Therefore, patients and families should be informed of these side effects and of the importance of a long-term follow-up requiring a multidisciplinary approach.
ISSN:1750-1172
1750-1172
DOI:10.1186/s13023-014-0162-0