HLA association is different in children and adults with severe acquired aplastic anemia

BACKGROUND: Severe aplastic anemia (SAA) is defined as pancytopenia caused by bone marrow failure. The pathogenesis of SAA is thought to involve autoimmune processes. Increased susceptibility to autoimmunity has been shown to be associated with several different HLA alleles. In SAA, few large studies based on data mainly from adults describe a positive HLA correlation with HLA-DR2 (DRB1*15) and HLA-B14. PROCEDURE: This study explored the HLA constitution of 181 children with SAA who were enrolled in the prospective multi-center study SAA94 between January 1994 and January 2002. The control group consisted of 303 healthy individuals of comparable demographic background. Allelic frequencies between patients and controls are compared using Fisher's exact test. RESULTS: In our pediatric cohort, we describe a positive association with HLA-B14 (P = 0.0039), but no association of HLA-DR2 with SAA. CONCLUSION: HLA associations appear to be different in children and adults with SAA. This might point towards a difference in pathophysiology between at least part of the children and adults.     

Successful autologous cord blood transplantation in a child with acquired severe aplastic anemia

Over 400 cases of pediatric SAA occur annually in the United States. A growing number of children with SAA may have had their stem cells harvested through cord blood collection. We describe a nine‐yr‐old male with SAA treated successfully with an autologous cord blood transplant following immunoablative chemotherapy. With the increasing number of people cryopreserving autologous cord blood, the use of autologous cord blood in the treatment of SAA might be considered as initial therapy. This case serves to discuss approaches to preparative therapy as well as the potential complications in this growing cohort of patients.     

Remission of aplastic anemia induced by treatment for Graves disease in a pediatric patient

Aplastic anemia (AA) is mediated by T-cell autoimmunity in the majority of cases; it is rare and mostly idiopathic in children. We describe a child, who developed AA following Graves' disease which could not be attributed to antithyroid drugs. We hypothesized that both diseases were caused by similar autoimmune process. We monitored the blood counts and did not administer any conventional treatment for AA assuming that the existing anti- hematopoietic stem cell humoral and cellular immunity might subside with induction of remission of Grave's disease. The child went into complete remission with the treatment of the Graves' disease.     

Favorable outcome with allogeneic hematopoietic stem cell transplantation in pediatric acquired aplastic anemia patients

The data of allogeneic HSCT in nine children with acquired AA between June 1998 and July 2006 were analyzed retrospectively. The median duration of time to neutrophil and platelet engraftment was 18 and 25 days, respectively. None of the patients had primary graft failure. Two (22.2%) patients developed acute GVHD and of these, one (11.1%) was Grade 1, and the other (11.1%) was Grade 3. Although the study group was composed of higher risk patients, including six of nine resistant to previous immunosuppressive treatment, eight had multiple not irradiated or filtered transfusion histories and one of the cases was only 5/6 HLA-compatible with his donor, the five-yr overall and EFS was 100%, and all recipients are alive without any graft failure. This may be attributed to the dose adjusted use of ATG according to individual transfusion history and gradual tapering of CsA and cessation at least nine months after allogeneic HSCT.     

两种免疫抑制疗法治疗儿童严重型再生障碍性贫血的比较

目的 探讨提高儿童严重型再生障碍性贫血（ＳＡＡ）疗效的方法。方法 结合骨髓基质液体培养法及针对性应用重组人粒－巨噬细胞集落刺激因子,比较联合免疫抑制疗法（ＣＩＳＴ）与强化免疫抑制疗法（ⅡＳＴ）治疗ＳＡＡ（分别为１１例和１６例）的疗效。结果在适当应用ｒｈｕＧＭ＿ＣＳＦ的基础上,ＣＩＳＴ及ⅡＳＴ方案治疗儿童ＳＡＡＵ职效差异无显著性（有效率分别为８２％及８１％）。结论 采用ＣＩＳＴ及ⅡＳＴ方案联合适当     

儿童重型再生障碍性贫血ATG治疗相关急性肺损伤1例并文献复习

目的探讨使用抗胸腺细胞球蛋白(ATG)治疗后出现少见血清病反应—急性肺损伤(ALI)的临床特点,以提高对本病的认识。方法回顾性分析1例重型再生障碍性贫血患儿第二次使用ATG后合并ALI的临床资料,并对相关文献进行复习。结果本例患儿于第二次使用ATG后10 d,出现发热、关节痛、肌痛、皮疹等临床表现,伴随突发烦躁、呼吸困难、低氧血症,肺部CT提示双肺弥漫毛玻璃影,临床诊断为ALI,应用甲泼尼龙治疗好转。通过文献检索发现8例类似报道,病死率较高。结论早期诊断和及时应用糖皮质激素治疗对于ATG相关ALI至关重要。     

Etanercept as a salvage treatment for refractory aplastic anemia

About 10–15% of patients with acquired aplastic anemia (AAA) have resistant/recurrent disease not eligible for standard treatment like hematopoietic stem cell transplantation and/or combined immunosuppression. We report a 17‐year‐old male with an 11 years history of AAA who, after two courses of immunosuppression, was red cell transfusion‐dependent, severely thrombocytopenic, refractory to platelet transfusion, had iron overload and post‐transfusion HCV infection. This patient achieved transfusion independence from platelets and normalized Hb after treatment with the anti‐TNF agent Etanercept. Over a 12 months follow‐up he experienced only transient increase of liver transaminases. Pediatr Blood Cancer 2009;52:522–525. © 2008 Wiley‐Liss, Inc.     

Cardiac mucormycosis in a child with severe aplastic anemia: a case report

Mucormycosis is an uncommon, severe, life-threatening fungal infection in the immunocompromised host. Mucormycosis with aplastic anemia is seen rarely. Only a few cases of cardiac mucormycosis with aplastic anemia have been reported in the literature. The authors present a case with severe aplastic anemia that did not respond to classic and immunosuppressive treatment for disease and developing invasive cardiac mucormycosis despite empiric treatment for febrile neutropenia.     

抗淋巴细胞球蛋白联合环胞霉素A治疗儿童重型再生障碍性贫血早期疗效观察

目的探讨提高儿童重型再生障碍性贫血(SAA)早期疗效的办法。方法采用随机对照方法,比较抗淋巴细胞球蛋白(ALG)和环胞霉素(CSA)联合治疗组与单用CSA组治疗儿童SAA的早期疗效(6个月)。结果联合治疗组和CSA组的总有效率分别为(84%)、(60%),P<0·05,联合治疗可降低早期死亡率,缩短脱离红细胞和血小板的输注时间。结论ALG联合CSA治疗儿童SAA是安全的且疗效佳,应作为一线方案。     

抗淋巴细胞球蛋白联合环胞霉素A治疗儿童重型再生障碍性贫血早期疗效观察

目的探讨提高儿童重型再生障碍性贫血（SAA）早期疗效的办法。方法采用随机对照方法，比较抗淋巴细胞球蛋白（ALG）和环胞霉素（CSA）联合治疗组与单用CSA组治疗儿童SAA的早期疗效（6个月）。结果联合治疗组和CSA组的总有效率分别为（84％）、（60％），P〈0．05，联合治疗可降低早期死亡率，缩短脱离红细胞和血小板的输注时间。结论ALG联合CSA治疗儿童SAA是安全的且疗效佳，应作为一线方案。     

Metamizole sodium-induced severe aplastic anemia and its recovery with a short-course steroid therapy

The authors treated a girl with severe aplastic anemia that developed following urinary tract infection. She was referred to our hospital for bone marrow transplantation. The detailed medical history of the patient revealed iatrogenic metamizole sodium overdose that was given during her infection. She responded to a 3-day, high-dose methylprednisolone therapy and consequent conventional dose steroid. The authors observed at first partial and subsequently complete remission of all the hematologic parameters of the patient within 30 days and 4 months, respectively. This case suggests that the history of drug use should be questioned carefully in the patients with aplastic features. Steroid treatment may be an appropiate alternative therapy for aplastic anemia if associated drug use is detected in the medical history.     

儿童特发性再生障碍性贫血的诊断与治疗

再生障碍性贫血(再障)以全血细胞减少和骨髓增生降低为显著特点。绝大多数病例为特发性,部分为继发性,与辐射、化学物和药物暴露、病毒感染有关。特发性再障发病机制不清,但免疫机制介导的造血干细胞损伤发挥着极为重要的作用。诊断主要依靠典型的临床表现和骨髓形态学检查,注意与其他可导致全血细胞减少的疾病鉴别,尤其是低增生性骨髓增生异常综合征。应综合患者年龄、临床严重程度、有无造血干细胞来源及其种类等因素,制定个体化治疗方案,合理选择联合免疫抑制治疗和造血干细胞移植的类型和时机。重视对症支持治疗措施。     

Parvovirus B19 infection associated with severe aplastic anemia in an immunocompetent patient

Human parvovirus B19 (PVB19) infection may cause mild pancytopenia characterized by transient and spontaneous recovery in healthy subjects. Severe aplastic anemia associated with PVB19 infection in patients without an underlying disease has been described in a number of reports. Here, a previous healthy, 10-year-old girl with severe aplastic anemia associated with PVB19 infection is described. The patient underwent bone marrow transplantation from her HLA-identical sibling resulting in complete recovery. PVB19 infection should be considered as one of the causes of aplastic anemia in patients without an underlying disease.