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
Lille University Hospital, Endocrinology and Metabolism, Lille, France.
Orphanet J Rare Dis. 2014 Oct 29;9:162. doi: 10.1186/s13023-014-0162-0.
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.
异基因造血干细胞移植主要适用于与血液疾病相关的骨髓功能障碍，也适用于一些罕见疾病（肾上腺脑白质营养不良、线粒体神经胃肠性脑病或MNGIE……）。几十年后，这种治疗已被证明是有效的，但其代价是出现许多早期和延迟的副作用，如感染、移植物抗宿主病、心血管并发症和继发性恶性肿瘤。这些并发症主要与预处理有关，预处理需要强效化疗联合全身照射（清髓）或免疫抑制（非清髓）。在副作用中，内分泌并发症可分为：1）激素内分泌缺乏（特别是性腺和生长激素缺乏），与化疗尤其是放疗的延迟后果有关，及其对生长、青春期、骨骼和生育能力的影响；2）自身免疫性疾病，特别是甲状腺功能障碍；3）继发性肿瘤，累及内分泌腺（甲状腺癌）或依赖激素状态（乳腺癌、脑膜瘤），因免疫失调和放疗而更易发生；4）代谢并发症，尤其是类固醇诱导的糖尿病和血脂异常，它们会增加心血管风险。这些并发症错综复杂。此外，激素替代疗法可以调节患者的心血管或肿瘤风险，而放疗和化疗，尤其是类固醇和蒽环类药物已经增加了这些风险……因此，应该告知患者及其家属这些副作用，以及采用多学科方法进行长期随访的重要性。
