Kitonyi G W, Macharia W M, Mwanda O W, Pamnani R
Haematology and Blood Transfusion Unit, Department of Human Pathology, College of Health Sciences, University of Nairobi, P.O. Box 19676-00202, Nairobi, Kenya.
East Afr Med J. 2009 Dec;86(12 Suppl):S39-46. doi: 10.4314/eamj.v86i12.62899.
To determine clinical-pathologic characteristics, treatment modalities and treatment outcomes of children diagnosed with neuroblastoma.
Cross-sectional descriptive study based on secondary data from patient records.
Records department of Kenyatta National Hospital (KNH), a tertiary teaching and referral hospital based in Nairobi.
Children aged 15 years and below, admitted with the diagnosis of neuroblastoma, between January 1997 and December 2005.
Presenting clinical features, diagnostic modalities including laboratory and imaging data, treatment modalities, response to treatment and patient survival.
Twenty six patients were eligible for the study; 13 males and 13 females giving a M:F ratio of 1:1. The age range was 5 days to 12 years, with a median age of five years. Abdominal swelling (53.8%), inability to walk due to bone pains, (50%), and cranial or periorbital swelling, (38.5%) were the commonest presenting features. Diagnosis of neuroblastoma was based on tissue biopsy in 50% (95% CI 40.6-79.8%) of the patients, and on fine needle aspiration cytology of mass or bone marrow in the rest. Bone marrow involvement was present in 16, (75%). Anaemia, was common with 72.7% patients having a haemoglobin (HB) <8 g/dl at presentation. Immunohistochemistry and cytological grading were done in two, (8%), patients. Urinary vanilly 1 mandelic acid (VMA), screening was positive in 50% (95% CI 29.9%-70.1%). The most frequently involved organs were abdomen (88.9%), and skeleton, (84.6%). Majority of patients, (92.3%), presented with advanced stage IV disease. Three patients died before commencement of treatment. All treated patients (100%), received cytotoxic therapy. Only two patients (8.6%) had surgery as part of treatment while one, (4.3%) was treated with radiotherapy. The initial treatment regimen was similar for all the patients. Although most patients had a complete initial response to treatment, early relapse, treatment failure, death or loss to follow up of patients with progressive disease were common. Overall survival (OS) at one year and two years were 19.2% (95% CI 6.6-39.4%) and 7.7% (95% CI 0.9%-25.1%) respectively. Only one patient was alive, (also free of disease), five years after diagnosis.
Although other clinical-pathologic findings of the patients were similar to those reported elsewhere, virtually all study patients presented with advanced stage IV disease, which would be associated with poor prognosis irrespective of quality of care. Priority must therefore be on ensuring early diagnosis and referral of patients with neuroblastoma before any other interventions can be expected to positively impact on outcome. The limited role of surgery and radiotherapy observed over the study period may be attributed to late presentation of the patients. Pathologic evaluation of important information could have been availed at minimal extra cost. To be at par with current internationally accepted treatment approaches that have been associated with improved survival, there is need to base choice of regimens for individual patients on clinical and readily accessible pathologic markers.
确定诊断为神经母细胞瘤的儿童的临床病理特征、治疗方式及治疗结果。
基于患者记录的二手数据进行横断面描述性研究。
位于内罗毕的三级教学及转诊医院——肯雅塔国家医院的记录科室。
1997年1月至2005年12月期间收治的诊断为神经母细胞瘤的15岁及以下儿童。
呈现的临床特征、包括实验室及影像学数据在内的诊断方式、治疗方式、对治疗的反应及患者生存情况。
26例患者符合研究条件；男13例，女13例，男女比例为1:1。年龄范围为5天至12岁，中位年龄为5岁。腹部肿胀（53.8%）、因骨痛无法行走（50%）及头颅或眶周肿胀（38.5%）是最常见的临床表现。50%（95%可信区间40.6 - 79.8%）的患者基于组织活检诊断为神经母细胞瘤，其余患者基于肿块或骨髓的细针穿刺细胞学检查诊断。16例（75%）存在骨髓受累。贫血常见，72.7%的患者就诊时血红蛋白（HB）<8 g/dl。2例（8%）患者进行了免疫组化及细胞学分级。尿香草扁桃酸（VMA）筛查50%呈阳性（95%可信区间29.9% - 70.1%）。最常受累的器官是腹部（88.9%）和骨骼（84.6%）。大多数患者（92.3%）表现为IV期晚期疾病。3例患者在开始治疗前死亡。所有接受治疗的患者（100%）均接受了细胞毒性治疗。仅2例患者（8.6%）接受了手术作为治疗的一部分，1例（4.3%）接受了放疗。所有患者的初始治疗方案相似。尽管大多数患者对初始治疗有完全反应，但疾病进展的患者早期复发、治疗失败、死亡或失访很常见。1年和2年的总生存率（OS）分别为19.2%（95%可信区间6.6 - 39.4%）和7.7%（95%可信区间0.9% - 25.1%）。诊断后5年仅有1例患者存活（且无疾病）。
尽管患者的其他临床病理表现与其他地方报道的相似，但几乎所有研究患者均表现为IV期晚期疾病，无论医疗质量如何，这都预示着预后不良。因此，在期望任何其他干预措施对结果产生积极影响之前，必须优先确保神经母细胞瘤患者的早期诊断和转诊。研究期间观察到的手术和放疗作用有限可能归因于患者就诊较晚。以最低的额外成本即可获取重要信息的病理评估。为了与当前国际上公认的与生存率提高相关的治疗方法接轨，需要根据临床及易于获得的病理标志物为个体患者选择治疗方案。
