Dipartimento di Oncologia ed Ematologia, U.O.C. Ematologia per le Malattie Rare del Sangue e degli Organi Ematopoietici, A.O. Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Am J Hematol. 2013 Nov;88(11):E261-4. doi: 10.1002/ajh.23531. Epub 2013 Aug 30.
Stroke is a common cause of morbidity and mortality in sickle cell disease (SCD) and silent cerebral infarction is the most common form of neurologic injury. The frequency and risk factors for new silent cerebral infarction are incompletely understood. Moreover, no recommended treatment has been established. Although hydroxyurea (HU) is recommended for SCD, concerns remain regarding its role in the prevention of cerebrovascular events, including silent cerebral infarction. A single center population of 104 Italian patients with HbS-ß thalassemia treated with HU has been followed for a mean of 11 years. Clinical evaluation and brain imaging by Magnetic Resonance Imaging were done before and during HU treatment. During follow-up, the number of sickle cell crises (86%, 7.8 ± 6.9 vs. 1.2 ± 0.5 per year, P < 0.0001), hospitalizations (2.5 ± 2.9 vs. 0.3 ± 1.5 per year, P < 0.0001), and days in the hospital (22.4 ± 21.9 vs. 0.3±1.5 per year, P < 0.0001) decreased significantly and HbF increased from a mean of 8-20.8%. Cerebral infarcts occurred in 37.5% of patients. Among these, 6.7% had overt strokes, while 30% had new or progressive silent cerebral infarction. Stroke and silent cerebral infarction were not related to clinical hematologic or HbF response to HU. These findings suggest that in adults, HU treatment does not prevent new cerebrovascular events or the progression of existent silent cerebral infarcts in HbS-β thalassemia. A major benefit of HU is the increase in HbF; the association of high HbF and reduced cerebrovascular disease has been weak. New treatment strategies should be developed for the prevention of sickle cerebrovascular disease.
中风是镰状细胞病（SCD）发病率和死亡率的常见原因，而无症状性脑梗死是最常见的神经损伤形式。新发生的无症状性脑梗死的频率和危险因素尚未完全清楚。此外，也尚未确定推荐的治疗方法。尽管羟基脲（HU）被推荐用于 SCD，但人们仍对其在预防包括无症状性脑梗死在内的脑血管事件中的作用存在担忧。意大利的一个中心对 104 名接受 HU 治疗的 HbS-β地中海贫血患者进行了为期 11 年的随访。在 HU 治疗前后进行临床评估和磁共振成像脑成像。在随访期间，镰状细胞危象的数量明显减少（86%，7.8±6.9 比 1.2±0.5 次/年，P<0.0001），住院次数（2.5±2.9 比 0.3±1.5 次/年，P<0.0001）和住院天数（22.4±21.9 比 0.3±1.5 天/年，P<0.0001）减少，HbF 从平均 8-20.8%增加。37.5%的患者发生脑梗死。其中，6.7%有显性中风，而 30%有新的或进展性无症状性脑梗死。中风和无症状性脑梗死与 HU 对临床血液学或 HbF 的反应无关。这些发现表明，在成人中，HU 治疗不能预防新的脑血管事件或 HbS-β地中海贫血中已有无症状性脑梗死的进展。HU 的主要益处是增加 HbF；HbF 与减少脑血管疾病之间的关联一直很弱。应制定新的治疗策略来预防镰状细胞脑血管病。
