Pereira Andrea Z, Hellman Ricardo, Hamerschlak Nelson, Kondo Andrea, de Souza Polianna Mara Rodrigues, Pedreira Wilson Leite, Mantovani Luiz Fernando Alves Lima, Troster Eduardo Juan, Grunspun Henrique, Bueno Marco Aurélio Scarpinella
Oncology, Hematology, and Bioethical Committee, Hospital Israelita Albert Einstein, São Paulo, SP, Brazil.
Oncology and Hematology, Hospital Israelita Albert Einstein, São Paulo, SP, Brazil.
Case Rep Hematol. 2017;2017:8394732. doi: 10.1155/2017/8394732. Epub 2017 Feb 23.
Hematopoietic stem cell transplantation (HSCT) is an important treatment option for children with severe and refractory sickle cell disease (SCD) with debilitating clinical complications. HSCT with cells from the bone marrow of a HLA-identical sibling used in SCD has a low mortality risk, high cure rate, and high event-free survival rate after a median follow-up of 5-6 years. However, matched donors are found in only about 20% of the patients. A boy aged 8 years with SCD had a sister, <2 years old, a fully compatible donor. The boy met all eligibility criteria to undergo HSCT, and he was suffering from cognitive and neurologic impairment due to ischemic events. A Bioethical Committee jointly discussed the ethical issues on this case after a pediatric evaluation released the very young sister for donation. The justification was that the sister would benefit from the donation too because of the greater likelihood of survival and cure and less suffering of her brother. The parents were informed about the risks and benefits for both children, and the family was psychologically evaluated. After their consent, HSCT was performed and the patient is cured from SCD. The complication for the donor was the need for blood transfusion.
造血干细胞移植(HSCT)是患有严重难治性镰状细胞病(SCD)并伴有使人衰弱的临床并发症的儿童的重要治疗选择。使用来自 HLA 相同同胞骨髓的细胞进行的 HSCT 用于 SCD 时,在中位随访 5 - 6 年后,具有低死亡风险、高治愈率和高无事件生存率。然而,仅约 20%的患者能找到匹配的供体。一名 8 岁患有 SCD 的男孩有一个不到 2 岁的妹妹,妹妹是完全匹配的供体。该男孩符合接受 HSCT 的所有资格标准,且由于缺血事件正遭受认知和神经功能损害。在儿科评估后,一个生物伦理委员会共同讨论了该病例的伦理问题,之后同意让年幼的妹妹进行捐赠。理由是妹妹也会从捐赠中受益,因为她哥哥存活和治愈的可能性更大,痛苦更少。已向父母告知了两个孩子的风险和益处,并对家庭进行了心理评估。在他们同意后,进行了 HSCT,患者已从 SCD 中治愈。供体的并发症是需要输血。