Sabarense Alessandra Palhoni, Silva Célia Maria, Muniz Maristela Braga de Sousa Rodrigues, Viana Marcos Borato
Faculdade de Medicina da Universidade Federal de Minas Gerais (FM UFMG), Belo Horizonte, MG, Brazil.
Fundação Hemominas, Belo Horizonte, MG, Brazil.
Hematol Transfus Cell Ther. 2022 Oct-Dec;44(4):478-484. doi: 10.1016/j.htct.2021.05.001. Epub 2021 Jun 18.
Stroke is a serious complication of sickle cell anemia (SCA). The transcranial Doppler (TCD) is the risk-screening tool for ischemic strokes. The objective of the study was to describe the clinical progression of children with SCA who presented with high risk for stroke by TCD or relevant changes by magnetic resonance angiography (MRA) and underwent the regular transfusion program (RTP) and/or hydroxyurea (HU) treatment between 2007 and 2018.
This was a neonatal retrospective/prospective cohort study with children born between 1999 and 2014 with the homozygotic form (HbSS) or Sβ-thalassemia who underwent TCD at least once.
Of the 718 children screened during this period, 675 had HbSS and 43 Sβ-thalassemia. In 54 children (7.5%), all with HbSS, a high-risk TCD (n = 45) or, when the TCD was inconclusive, an MRA with cerebral vasculopathy (n = 9) was used for detection. Of these, 51 started the RTP and the families of three refused treatment. Of the 43 children with a high-risk TCD who initiated the RTP, 29 (67.4%) reverted to low risk. In 18 of them (62%), HU was started at the maximum tolerated dose (MTD) before transfusion discontinuation. None of these 29 patients had a stroke. Eight children (18.6%) maintained a high-risk TCD, even using the RTP/HU and two had a stroke.
The TCD was confirmed as a viable tool for tracking patients with a risk for stroke. The RTP was effective in preventing the primary event. New strategies are necessary to prevent stroke using HU and new drugs, in addition to bone marrow transplantation.
中风是镰状细胞贫血（SCA）的一种严重并发症。经颅多普勒（TCD）是缺血性中风的风险筛查工具。本研究的目的是描述2007年至2018年间通过TCD显示有中风高风险或磁共振血管造影（MRA）有相关变化并接受常规输血方案（RTP）和/或羟基脲（HU）治疗的SCA儿童的临床进展。
这是一项针对1999年至2014年间出生的患有纯合形式（HbSS）或Sβ地中海贫血且至少接受过一次TCD检查的儿童的新生儿回顾性/前瞻性队列研究。
在此期间筛查的718名儿童中，675名患有HbSS，43名患有Sβ地中海贫血。在54名儿童（7.5%）中，均为HbSS患儿，通过高风险TCD（n = 45）或当TCD结果不明确时通过显示脑血管病变的MRA（n = 9）进行检测。其中，51名开始接受RTP治疗，3名患儿的家属拒绝治疗。在43名开始接受RTP治疗的高风险TCD患儿中，29名（67.4%）恢复为低风险。其中18名（62%）在停止输血前开始以最大耐受剂量（MTD）使用HU。这29名患者均未发生中风。8名儿童（18.6%）即使使用RTP/HU仍维持高风险TCD，其中2名发生了中风。
TCD被确认为追踪中风风险患者的可行工具。RTP在预防主要事件方面有效。除骨髓移植外，还需要新的策略来使用HU和新药预防中风。
