骨髓发育不全是一种罕见且严重的血液系统疾病。虽然是良性的,它是一种血液病,其预后可能较差,其自发发展可能是致命的。治疗时间长,困难和昂贵的。在发展中国家,由于治疗管理的困难,死亡率很高,既支持又具体。我们对在卡萨布兰卡20奥特大学医院儿科血液学和肿瘤科(2010年1月至2020年1月)的92例AM进行了回顾性研究。在这项工作中,我们概述了卡萨布兰卡大学医院小儿血液病和肿瘤科的情况,并重点介绍了AM管理中遇到的困难。在我们的研究中,平均年龄是19岁,从3个月到29年不等,在15-20岁年龄段达到高峰。性别比例(M/F)为2.06,男性占67%。在我们的系列中,只有35%的患者出现完全骨髓衰竭.92%的患者出现贫血综合征,70%和41%的患者出现出血性和感染性综合征,分别。从诊断到治疗的中位时间为82天。根据卡米塔的评分,31%的患者有轻度AM,41%有严重的AM,28%的人患有严重的AM。病因学评估后,我们得出的结论是90%的患者患有特发性骨髓发育不全,2%有宪法性骨髓发育不全,8%的患者被怀疑患有继发性骨髓发育不全:肝炎后(3例),有毒(2例),药物诱导(1例),和再生PNH(1例)。诊断后前三个月的死亡率为21%。69%的患者接受了特定的治疗:28例患者单独使用环孢菌素(CIS)作为一线治疗,20接受了抗淋巴细胞血清(ALS)和环孢菌素的组合,2例接受造血干细胞移植(HSCT),而3只单独用雄激素治疗。CIS的总有效率为30%,42%采用ALS+CIS,100%采用HSCT。在我们的研究中,总死亡率为44%,而一年生存率为40%。值得注意的是,脓毒性休克是导致死亡的主要原因(占死亡人数的53%)。其次是失血性休克(24%)。这突出了缺乏血流动力学复苏和对症治疗。我们的多变量研究将以下危险因素定义为生存较差的预测因素:年龄大于16岁(RR:3.28;CI:1.29-8.33;P=0.012),PNN小于200或非常严重的骨髓发育不全(RR:3.01;1.1-8.08;P=0.028),未能接受任何特定治疗(RR:4.07;1.77-9.35;P=0.0003)。我们系列中的高总死亡率是由于几个因素:无法获得有效的治疗,延迟诊断,未能启动特定治疗,不充分的对症治疗,以及地理和金融上的不便。
Bone marrow aplasia is a rare and serious hematologic disorder. Although benign, it is a hematologic disorder whose prognosis can be poor and whose spontaneous development can be fatal. Treatment is long, difficult and costly. In developing countries, the mortality rate is high due to the difficulties of therapeutic management, both supportive and specific. We conducted a retrospective study of 92 cases of AM identified in the Pediatric Hematology and Oncology Department of the 20 Août University Hospital in Casablanca over a 10-year period (January 2010-January 2020). In this work, we present an overview of the situation and highlight the difficulties encountered in the management of AM in the Pediatric Hematology and Oncology Department of the University Hospital of Casablanca. In our study, the mean age was 19 years, ranging from 3 months to 29 years, with a peak in the 15-20 age group. The sex ratio (M/F) was 2.06, with a male predominance of 67%. In our series, only 35% of patients had complete bone marrow failure. An anemic syndrome was present in 92% of patients, and hemorrhagic and infectious syndromes were present in 70% and 41% of patients, respectively. The median time from diagnosis to treatment was 82 days. According to the Camitta score, 31% of our patients had mild AM, 41% had severe AM, and 28% had very severe AM. After etiologic evaluation, we concluded that 90% of the patients had idiopathic bone marrow aplasia, 2% had constitutional bone marrow aplasia, and 8% of the patients were suspected to have secondary bone marrow aplasia: post-hepatitis (3 cases), toxic (2 cases), drug-induced (1 case), and aplastic PNH (1 case). Mortality in the first three months after diagnosis was 21%. Sixty-nine percent of our patients received specific treatment: 28 were treated with cyclosporin (CIS) alone as first-line therapy, 20 received a combination of antilymphocyte serum (ALS) and cyclosporin, 2 received hematopoietic stem cell transplantation (HSCT), while 3 were treated with androgens alone. The overall response rate was 30% with CIS, 42% with ALS+CIS and 100% with HSCT. In our study, the overall death rate was 44%, while the one-year survival rate was 40%. It is important to note that septic shock was the leading cause of death (53% of deaths), followed by hemorrhagic shock (24%). This highlights the lack of hemodynamic resuscitation and symptomatic treatment. Our multivariate study defined the following risk factors as predictive of worse survival: age greater than 16 years (RR: 3.28; CI: 1.29-8.33; P=0.012), PNN less than 200 or very severe bone marrow aplasia (RR: 3.01; 1.1-8.08; P=0.028), and failure to receive any specific treatment (RR: 4.07; 1.77-9.35; P=0.0003). The high overall mortality in our series was due to several factors: inaccessibility to effective therapies, delayed diagnosis, failure to initiate specific treatment, inadequate symptomatic treatment, and geographical and financial inaccessibility.