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??Abstract??Allogeneic hematopoietic stem cell transplantation ??HSCT?? is the only curative therapy for severe beta-thalassemia major. Patients with an available human leukocyte antigen ??HLA?? identical sibling donor should be offered HSCT as soon as possible before development of iron overload and transfusion associated complications?? and the cure rate was up to 80%??90%. High-resolution HLA typing matched unrelated donor transplants also achieved good results. HSCT from HLA-mismatched relatives or HLA-phenotypically-identical donor is an option to be performed in expert centers. Optimization of conditioning regimen and techniques to control transplant-related complications will further improve outcomes.     

造血干细胞移植治疗儿童白血病若干问题

该文涉及各类儿童造血干细胞移植(HSCT),如骨髓移植(BMT)、外周血造血干细胞移植(PBSCT)和脐血移植(UCBT)治疗白血病的优缺点及HSCT在儿童白血病治疗中的地位。绝大多数儿童白血病可通过正规联合化疗根治,仅少数(约20%)高危、难治及复发的白血病是异基因HSCT的适应证,无适合的同胞供体时,可选择HLA全相合非血缘相关BMT或PBSCT,UD-UCBT更适合于儿童患者。     

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??Objective??To analyze the effectiveness and the practicability of Nordic Society for Pediatric Hematology and Oncology??NOPHO?? AML 2004 protocol combined with hematopoietic stem cell transplantation??HSCT?? in treating childhood acute myeloid leukemia??AML?? in China. Methods??The clinical data of 53 children??aged under 14?? with AML??not including acute promyelocytic leukemia?? treated with NOPHO AML 2004 protocol??some children followed by HSCT?? between Dec. 2009 and Dec. 2015 was retrospectively analyzed. Survival was evaluated by Kaplan Meier analysis and Log-Rank test. Results??Complete remission??CR?? rate reached 86.8%. Totally 36 patients??67.9%?? finished 6 courses of chemotherapy??14 patients??26.4%??underwent HSCT after 1-3 courses of consolidation chemotherapy??3 patients gave up treatment after 1-3 courses of chemotherapy. The proleptic overall survival??pOS?? at 5 years and leukemia-free survival??pLFS?? was 72% and 56% respectively during a follow-up period of 12 to 82 months??median 29 months??. For standard-risk patients??the pLFS at 5 years reached 58%?? while high-risk patients had 46%??P??0.779??. The pLFS at 5 years in patients who only underwent chemotherapy and those with additional hematopoietic stem cell transplantation was 48% and 77% respectively??P??0.1268??. The pLFS in patients with good response??intermediate response and poor response on day 15 bone marrow examination was 75%??29% and 53% respectively??P??0.0071??. Conclusion??NOPHO AML 2004 protocol is effective and feasible for Chinese patients with childhood AML. The evaluation of blast in bone marrow on day 15 can be used as an important prognostic indicator.     

Iron overload in survivors of childhood leukemia after allogeneic hematopoietic stem cell transplantation

Abstract:  Iron overload has not been studied extensively and prospectively in pediatric survivors of allogeneic hematopoietic stem cell transplantation (HSCT); therefore, we conducted a prospective long-term study of 133 survivors of childhood leukemia to assess the incidence of and risk factors for iron overload and to investigate its association with organ dysfunction. One yr after HSCT, the mean serum ferritin level was 1158 ng/mL (range, 22–3264 ng/mL), with 124 patients (93.2%) having a serum ferritin level that exceeded the upper limit of the normal range (110 ng/mL). Thereafter, the serum ferritin level declined over time. There was a positive correlation between the level of serum ferritin and that of total bilirubin (r   =   0.21, p < 0.001) and glutamate pyruvate transaminase (r   =   0.17, p < 0.001). A high concentration of serum ferritin was associated with low cardiac fractional shortening (r   = −0.15, p  =  0.047). In addition, patients with hypothyroidism and GH deficiency had a higher level of serum ferritin than those without (p < 0.02). We conclude that iron overload is common after HSCT and is associated with hepatic, cardiac, and endocrine dysfunction.     

异基因造血干细胞移植治疗儿童急性髓系白血病临床研究

目的评估应用异基因造血干细胞移植(allo-HSCT)治疗儿童急性髓系白血病(AML)的临床疗效及相关影响因素。方法回顾分析2002年1月至2017年11月49例确诊中、高危及复发AML行allo-HSCT患儿的临床资料,分析危险度分级、HLA分型、移植前状态、移植方式、干细胞来源及急慢性移植物抗宿主病(GVHD)等对allo-HSCT治疗效果的影响。结果 49例患儿中男35例、女14例,中位年龄9岁。三年总体存活率(OS)为(59.2±7.3)%,无白血病存活率(LFS)为(50.9±7.4)%。其中第1次缓解状态移植、非血缘移植、外周血干细胞移植、中危组移植的三年LFS分别为69.8%、69. 2%、73. 7%、65. 8%。19例死亡,分别为复发13例、严重感染5例、多器官衰竭1例。COX回归模型结果显示,急性GVHD是影响移植OS的独立危险因素(RR=3. 16,95%CI:1. 23～8. 09,P=0. 017),移植前状态为部分缓解及未缓解是影响移植LFS的独立危险因素(RR=4.76,95%CI:1.52～14.94,P=0.008;RR=5.28,95%CI:1.68～16.58,P=0.004)。结论移植前状态及急性GVHD是影响Allo-HSCT治疗儿童AML疗效的关键因素;白血病复发及感染是导致死亡的主要原因。     

Chronic neutrophilic leukemia,an extremely rare cause of neutrophilia in childhood: Cure with hematopoietic stem cell transplantation

CNL is a rare myeloproliferative disorder frequently seen in older adults. A significant proportion of patients show progression to AML. Here, we report the case of a patient with FA who was monitored for leukopenia but who developed leukocytosis during the follow‐up and was diagnosed with CNL probably after an acquired CSF3R mutation. Because the patient had FA, which could accelerate the progression to AML, an HSCT was performed, which resulted in cure. This patient (aged 12 years) is one of the youngest patients reported to develop CNL as well as the first FA patient with a diagnosis of CNL.     

造血干细胞移植后出血性膀胱炎的诊疗

出血性膀胱炎(HC)是异基因造血干细胞移植的常见并发症之一,是移植防治的重点,文章就HC病因、发病机制和防治现状作一介绍.HC的发生常与移植前大剂量放化疗毒性,药物代谢酶基因多态性,病毒感染,移植物抗宿主病,患者的年龄、性别,供者类型和移植方式等相关.根据相关病史、典型临床表现和必要的辅助检查可以诊断.有效的预防措施是...     

异基因造血干细胞移植治疗儿童慢性粒细胞性白血病的疗效分析

目的：研究异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）治疗儿童慢性粒细胞性白血病（chronic myelogenous leukemia, CML）的治疗效果,寻找可能的影响因素,以期改善患者预后。方法：对接受allo-HSCT治疗的20例儿童CML患者,分别从年龄、性别、诊断至移植间隔时间、供受体HLA配型相合情况、移植时患儿疾病状态以及急慢性移植物抗宿主病（gost-v-host disease, GVHD）等多种因素进行疗效分析。结果：截止至随访日期,20例患者中,13例无病存活,7例死亡,其中4例死于急性重度GVHD,2例死于慢性GVHD及其并发症,1例死于移植后复发,3年总无病生存率为（64.6±1.1%）。单因素分析显示年龄是影响儿童CML治疗预后最重要的因素之一（P0.05）。多因素logistic回归分析也进一步证明仅年龄是影响预后的因素（P<0.01）。各种严重急慢性 GVHD是引起患者死亡最重要的原因。选择10位点全相合的供体进行移植治疗预后好。结论：allo-HSCT能有效治疗儿童CML,对于年龄≥10岁的CML患儿宜早期行allo-HSCT移植治疗,且尽可能选择10位点全相合的供体进行移植,积极防治GVHD,改善CML患儿移植治疗后的转归。     

Bullous pemphigoid after allogeneic hematopoietic stem cell transplantation

Bullous pemphigoid (BP) is an autoimmune skin disorder characterized by subepidermal blisters due to deposit of autoantibody against dermal basement membrane protein. It has been reported that BP can occur after allogeneic hematopoietic stem cell transplantation (HSCT). We describe a patient with BP having autoantibody against BP180 after unrelated‐donor HSCT against T lymphoblastic leukemia. The patient was treated with steroid leading to complete resolution of BP, but T lymphoblastic leukemia progressed rapidly after steroid hormone treatment. Given that immunosuppressant may reduce graft‐versus‐tumor effect, immunomodulatory agents such as nicotinamide and tetracycline, erythromycin, and immunoglobulin may be appropriate as soon as typical blister lesions are seen after HSCT.