Cohen A, Békássy A N, Gaiero A, Faraci M, Zecca S, Tichelli A, Dini G
Department of Pediatrics, University of Genova, San Paolo Hospital, Savona, Italy.
Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S43-8. doi: 10.1038/bmt.2008.54.
The main challenge for a pediatric hemato-oncologist today is to obtain a cure for the sick child with the minimum of treatment-related complications. Children on their way to achieving adulthood face many risks after hematopoietic SCT (HSCT). Continuous follow-up includes assessment of organ function, focus on vaccinations and screening for secondary malignancies. Updated treatment protocols are already adjusted according to the knowledge obtained on late effects, and the potential risks for complications are well balanced with expected benefits hopefully resulting in decreased potential risk for organ damage but still maintaining an unchanged or improved survival rate. Recent developments on pre-HSCT regimens, such as the introduction of new anticancer regimens and immunosuppressive agents will hopefully contribute to minimize the frequency and the severity of late complications. Knowledge about increased risk for long-term complications due to cancer therapy and pre-HSCT preparative regimens should encourage each caring physician to stick to follow-up protocols and treatment guidelines not only to improve the survival rate of transplanted children but also to improve their quality of life. To achieve adulthood by maintaining cognitive ability and psychosocial skills is the highest goal for an individual to become a competent member of a society. This review of late endocrine complications after HSCT focuses on growth, pubertal development, thyroid disorders and glucose metabolism in long-term survivors.
如今，儿科血液肿瘤学家面临的主要挑战是在将与治疗相关的并发症降至最低的情况下治愈患病儿童。接受造血干细胞移植（HSCT）的儿童在迈向成年的过程中面临许多风险。持续随访包括评估器官功能、关注疫苗接种以及筛查继发性恶性肿瘤。最新的治疗方案已根据对远期效应的认识进行了调整，并发症的潜在风险与预期益处得到了很好的平衡，有望降低器官损伤的潜在风险，但仍保持生存率不变或提高。HSCT前方案的最新进展，如引入新的抗癌方案和免疫抑制剂，有望有助于减少远期并发症的发生频率和严重程度。了解癌症治疗和HSCT预处理方案导致长期并发症风险增加的情况，应促使每位关爱患儿的医生坚持随访方案和治疗指南，不仅要提高移植儿童的生存率，还要改善他们的生活质量。通过保持认知能力和社会心理技能来长大成人，是个人成为社会合格成员的最高目标。这篇关于HSCT后晚期内分泌并发症的综述聚焦于长期存活者的生长发育、青春期发育、甲状腺疾病和糖代谢。
