Department of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, California, USA.
Department of Pediatrics, Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, North Carolina, USA.
Pediatr Blood Cancer. 2024 Aug;71(8):e31070. doi: 10.1002/pbc.31070. Epub 2024 May 16.
Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with newly diagnosed SAA.
重型再生障碍性贫血(SAA)是一种罕见的潜在致命血液疾病。尽管治疗的总体效果很好,但在管理方法上存在差异,包括成人和儿科患者之间的治疗差异。某些治疗方面正在临床试验中积极研究。由于疾病的罕见性,一些儿科血液学家在 SAA 的复杂管理方面可能经验相对有限。以下建议反映了最新的基于证据的方法,用于治疗新诊断为 SAA 的儿童。