Survival Among Children and Adults With Sickle Cell Disease in Belgium: Benefit From Hydroxyurea Treatment
Survival Among Children and Adults With Sickle Cell Disease in Belgium: Benefit From Hydroxyurea Treatment
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DOI：
10.1002/pbc.25608
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发表时间：
2015-11-01
影响因子：
3.2
通讯作者：
Ferster, Alina
中科院分区：
文献类型：
--
作者：
Le, Phu Quoc;Gulbis, Beatrice;Ferster, Alina
Objective.评价比利时镰状细胞病（SCD）登记处记录的SCD患者的生存率，并评估疾病缓解治疗（DMT）的影响。法2008年创建的登记研究包括8个中心的患者。2008年的所有可用数据都在数据库中进行了追溯编码。2008年之后至2012年，前瞻性记录了已登记患者以及新诊断受试者的所有数据。数据登记从新生儿筛查或从诊断（首次接触）到末次随访或死亡。数据包括诊断、人口统计学和结局数据。结果我们在5，110患者年（PY）随访期内收集了469例患者的数据。尽管有13例患者死亡（2.8%），但总体死亡率较低（0.25/100 PY），儿童、青少年（10-18岁）和年轻成人之间的死亡率相似（P = 0.76）。在该队列中，185例患者在末次随访时接受了羟基脲治疗（中位治疗持续时间：10.3年），90例接受了造血干细胞移植（HSCT），24例长期输血，170例从未接受过任何DMT。与移植个体或无DMT的人相比，羟基脲对患者结局显示出显著的益处，这反映在死亡率较低（分别为0.14、0.36和0.38/100 PY）和15年生存率的Kaplan-Meier估计值较高与HSCT组（93.8%; P = 0.01）或无DMT组（95.4%; P = 0.04）相比，结论比利时的SCD死亡率较低，在年轻成人中未观察到增加。与未接受DMT或移植的患者相比，接受羟基脲治疗的患者在生存方面表现出显著的益处。(C)2015 Wiley Periodicals，Inc.
Objective. To evaluate the survival of patients with sickle cell disease (SCD) recorded in the Belgian SCD Registry and to assess the impact of disease-modifying treatments (DMT). Method. The Registry created in 2008 included patients of eight centers. All available data in 2008 were retrospectively encoded in the database. After 2008 and until 2012, all data were recorded prospectively for already registered patients as well as newly diagnosed subjects. Data were registered from neonatal screening or from diagnosis (first contact) until last follow-up or death. Data included diagnosis, demography, and outcome data. Results. We collected data from 469 patients over a 5,110 patient years (PY) follow-up period. The global mortality rate was low (0.25/100 PY), although 13 patients died (2.8%) and was similar between children, adolescents (10-18 years), and young adults (P = 0.76). Out of the cohort, 185 patients received hydroxyurea at last follow-up (median duration of treatment: 10.3 years), 90 underwent hematopoietic stem cell transplantation (HSCT), 24 were chronically transfused, and 170 had never had any DMT. Hydroxyurea showed significant benefit on patients outcome as reflected by a lower mortality rate compared to transplanted individuals or people without DMT (0.14, 0.36, and 0.38 per 100 PY, respectively) and by higher Kaplan-Meier estimates of 15 year survival (99.4%) compared to HSCT (93.8%; P = 0.01) or no DMT groups (95.4%; P = 0.04). Conclusion. SCD mortality in Belgium is low with no increase observed in young adults. Patients treated with hydroxyurea demonstrate a significant benefit in survival when compared to those without DMT or transplanted. (C) 2015 Wiley Periodicals, Inc.