粒细胞集落刺激因子在儿童急性髓性白血病治疗中的应用

为了增加强化疗的安全性,15例次初治及32例次巩固强化治疗的急性髓性白血病患儿化疗中应用粒细胞集落刺激因子,中性粒细胞绝对值小于0.5×10~9/L的持续时间分别为5.3天、5.0天,与化疗方案相同的对照组相比分别缩短了5.6天、3.6天;感染发生率为40％、25％,较对照组下降了26.7％,19.4％,具显著差异(P<0.05);输血量亦随之减少,副作用少,提高了病人的生活质量,为强化疗治疗小儿急性髓性白血病提供了保障。     

儿童急性粒细胞性白血病治疗进展

急性粒细胞性白血产现在儿童急性白血病中约占２０％。在接受化疗过程中，人们认为ＡＭＬ较急性淋巴细胞白血病更易产一耐药。但从近１５年的整体治疗效果分析，ＡＭＬ的预后较ＡＬＬ更好，这归功于强化治疗手实施，骨髓移植的增多和不断改进的支持疗法。     

小儿白血病骨髓移植疗法的进展

小儿白血病骨髓移植疗法的进展张建忠，吕善根首例骨髓移植（ＢＭＴ）成功迄今已有２５年了，Ｔｈｏｍａｓ医生以其在异基因骨髓移植（Ａｌｌｏ－ＢＭＴ）中的先驱作用而获得了１９９０年诺贝尔医学奖。据国际骨髓移植登记处（ＩＢＭＴＲ）资料，１９６５～１９９０年大约...     

粒细胞集落刺激因子治疗急性淋巴细胞白血病疗效观察

我们对急性淋巴细胞白血病 (ＡＬＬ)患儿在化疗基础上应用重组人粒细胞集落刺激因子 (ｒｈＧ ＣＳＦ) ,观察化疗后骨髓抑制时对血白细胞 (ＷＢＣ)和中性粒细胞绝对值 (ＡＮＣ)的影响 ,现报告如下。对象与方法一、对象　 1998～ 2 0 0 1年新乡医学院第一、二附属医院住院的ＡＬＬ患儿 5 2例 ,均为初治患儿 ,年龄 1～ 12岁 ,平均6 .2岁 ,均符合血液病诊断及疗效标准[1] 。二、化疗方案及分组　均采用ＶＤＰ(长春新碱、柔红霉素、泼尼松 )或ＣＯＤＰ +Ｌ(环磷酰胺、长春新碱、柔红霉素、泼尼松、天门冬酰胺酶 )方案。其中 30例应用ｒｈＧ ＣＳ…     

非亲缘异基因骨髓移植治疗儿童白血病

目的　评价非亲缘异基因骨髓移植 (URD BMT)治疗儿童急性和慢性白血病的临床疗效。方法　 6例白血病患儿 ,其中慢性髓系白血病 2例 ,急性淋巴细胞白血病 3例 (第 1次完全缓解 ) ,急性早幼粒细胞白血病 1例 (第 2次完全缓解 ) ,由台湾慈济骨髓捐赠中心提供无关供者骨髓。预处理方案为马利兰 环磷酰胺 (Bu/Cy2 )方案 ,急性移植物抗宿主病 (aGVHD)预防为霉酚酸酯(MMF)、环孢菌素A(CsA)加氨甲喋呤 (MTX)联合方案 ;以前列素E1预防肝静脉闭塞病 (VOD) ,以巨细胞病毒 (CMV)抗原血症监测和更昔洛韦预防CMV病。供、受者间HLA基因位点型全相合 3例 ,1个基因位点型不合 2例 ,2个基因位点型不合 1例。结果　 6例患儿经DNA短串联重复序列多态性分析证明为供髓植入 ,中性粒细胞 >0 5× 10 9/L的中位天数为 14 5 (13～ 18)d ,血小板 >2 0×10 9/L的中位天数为 16 (11～ 2 3)d。发生Ⅱ～Ⅳ度aGVHD 2例 (33% ) ,局限性慢性移植物抗宿主病(cGVHD) 3例 ,未发生广泛性cGVHD。中位随访时间 4 12 (187～ 1338)d ,全部患儿均无病生存。结论非亲缘异基因骨髓移植是治疗儿童急性和慢性白血病的有效方法。     

短程应用rhG-CSF佐治小儿急性白血病疗效观察

目的　探讨短程应用重组人粒细胞集落刺激因子（ｒｈＧ－ ＣＳＦ） 佐治急性白血病强化疗所致骨髓抑制期的疗效。方法　将４５ 例急性白血病随机分为两组。对照组予常规治疗,治疗组在常规治疗同时加用ｒｈＧ－ ＣＳＦ,短程应用,共计３ ｄ。结果　治疗组骨髓抑制期恢复时间（７．７ ±１ ．２２ ｄ） ,较对照组（１０ ．７ ±４ ．５６ ｄ） 显著缩短（ Ｐ＜ ０．０５）,继发感染发生率（６０ ．８ ％）与对照组（６６ ．７ ％） 相比差异无显著意义（Ｐ＞０ ．０５）,但感染控制时间治疗组（４．３７ ±５ ．６１ ｄ） 较对照组（７ ．０８ ±３．６１ ｄ） 明显缩短（Ｐ＜０ ．０５）,成分输血量（ 均值３４０ ｍｌ） 较对照组（均值５４０ ｍｌ）显著减少（ Ｐ＜０ ．０１）,缓解率及缓解后无病生存期无明显差异（Ｐ＞ ０．０５）。结论　ｒｈＧ－ ＣＳＦ短程应用佐治急性白血病对强化疗后缓解骨髓抑制,顺利完成强化疗方案,疗效肯定,费用亦降低,值得临床推广应用。     

粒细胞集落刺激因子在小儿急性白血病治疗中的应用

小儿急性白血病在强化疗时白血病细胞被杀灭，正常造血尚未重建，由此导致感染、出血、贫血、在化疗后使用重组人粒细胞集落刺激因子(rhg-csf)减轻强烈化疗的骨髓抑制，缩短和减轻中性粒细胞减少期，使化疗强度加大，达到强化疗目的。现将我院应用rhg-csf的15例临床疗效报道如下。     

Haploidentical bone marrow transplantation for osteopetrosis

HLA-matched bone marrow transplantation is an effective form of treatment for some patients with malignant osteopetrosis, a defect of osteoclast function. Following transplant, normal osteoclasts differentiate from donor-derived marrow stem cells and can function normally in some of these patients. For patients without an HLA-matched marrow donor, pharmacologic treatments have not yet proved effective. This article demonstrates that normal osteoclast function can be obtained following the transplantation of HLA-nonidentical marrow that has been purged of T lymphocytes in vitro.     

Haploidentical bone marrow transplantation in Mexico

Haploidentical hematopoietic cell transplantation using CD34⁺ cells depleted of T lymphocytes by the CliniMACS is a treatment for hematological malignancy. We report on four Mexican children, three with acute lymphocytic leukemia and one with chronic myelocytic leukemia, who was transplanted with 12 × 10⁶ CD34⁺ stem cells/kg body weight (98% of purity) with a follow‐up of years. The engraftment was successful in three of the four children. All showed cytomegalovirus reactivation, and one died because of graft rejection and infectious complication. The risk of infections was a major problem. Pediatr Blood Cancer 2012; 59: 950–952. © 2012 Wiley Periodicals, Inc.     

Evans syndrome after unrelated bone marrow transplantation for refractory cytopenia of childhood

Post‐transplant ES, which is often resistant to therapies, has seldom been described. This report describes a case of ES after UBMT for RCC. A five‐yr‐old boy developed RCC with no evidence of monosomy 7. Because no matching family donors were available for SCT and immunosuppressive therapy was ineffective, UBMT was performed when he was six yr old. The conditioning regimen included TAI (3 Gy) and administration of FLU, CY, and rabbit antithymocyte globulin. The recovery of blood cells was good. He displayed grade II acute GVHD involving only the skin. ES developed on day 66, with positive results for Epstein–Barr virus DNA and HHV 6. Cytopenia was resolved with treatment with RTX, GCV, an escalated dose of steroids, high‐dose gammaglobulin, and romiplostim. No relapse has occurred since discontinuing steroids on day 177 and romiplostim on day 268. Post‐SCT ES after UBMT is rare, and the risk factors and therapies are unclear. Prospective analysis and collection of cases from multiple centers are required for clarification.     

Decitabine prior to salvaged unrelated cord blood transplantation for refractory or relapsed childhood acute leukemia

No clinical studies have investigated the role of decitabine as a part of the myeloablative conditioning regimen prior to UCBT for refractory or relapsed childhood AL in patients in NR status. The aim of this study was to identify the potential benefits of decitabine as a prior therapy before salvaged unrelated UCBT for refractory or relapsed childhood AL. Eight consecutive patients with childhood refractory/relapsed AL were enrolled in our study between 2013 and 2014. All patients were in NR status before the time of transplant and had features associated with poor outcomes, such as CNSL, MDS‐AML, high WBC count at diagnosis, and hypodiploid status (FLT3+/ITD+). Additionally, all patients had one of the following disease statuses: PIF, multiple relapse, or early relapse. All transplants were performed with decitabine as part of the myeloablative conditioning regimen, which was decitabine+Flu/Bu/CY±BCNU or decitabine+Ara‐c/BU/CY2±BCNU. A total of seven patients (7 of 8) achieved neutrophil engraftment and platelet engraftment, and one patient experienced primary graft failure. All eight patients (100%) developed PES at a median of 7 days. Three patients developed stage II‐IV acute GVHD at a median of 18 days. Additionally, three patients developed chronic GVHD, but it was not extensive in any of those three patients. The median follow‐up time after CBT was 19.9 months (range, 9.2–30.7 months). The estimated probability of OS was 75%. Two patients (2 of 8) experienced a testis relapse, and two patients (2 of 8) died. Our experience suggests that the additional application of decitabine as part of the myeloablative conditioning regimen prior to UCBT for refractory or relapsed childhood AL among patients who are not in remission is safe and might be an effective treatment option.     

Haploidentical bone marrow transplantation in a patient with sickle cell disease and acute myeloid leukemia

Myeloid neoplasms in children with sickle cell disease have been rarely reported, and the exact underlying connection between these two conditions is not clearly understood. Whether the acute myeloid leukemia in hydroxyurea‐treated sickle cell patients is co‐incidental or related to therapy remains an unanswered question. Herein, we report a 14‐year‐old girl of Haitian descent with sickle beta zero thalassemia on chronic hydroxyurea therapy who developed FMS‐like tyrosine kinase 3 (FLT3)‐mutated acute myeloid leukemia and underwent a complete disease remission following a combination chemotherapy with sorafenib and was subsequently treated using a T cell replete unmanipulated haploidentical bone marrow transplantation followed by post‐transplant cyclophosphamide.     

人类白细胞抗原不全相合非血缘脐血移植治疗儿童难治性复发急性白血病

脐血移植治疗血液系统恶性肿瘤的疗效仍需观察。我科于2001年4月～2003年3月应用非血缘脐血移植(UD-UCBT)治疗儿童难治性复发急性白血病患儿3例,现报告如下。     

Longitudinal gonadal function after bone marrow transplantation for acute lymphoblastic leukemia during childhood

Total body irradiation and high-dose chemotherapy, applied as a preparatory regimen for bone marrow transplantation (BMT) in children with acute lymphoblastic leukemia (ALL), are particularly hazardous to the gonads and, in addition, can impair hypothalamo pituitary-gonadal control. Longitudinal data on pubertal development and gonadal function in these patients are limited. Twenty-one ALL patients (15 males, 6 females) who had successfully undergone allogeneic BMT before puberty (age at BMT: 3.4-12.3 yr) were followed up in University Children's Hospital, Tübingen, Germany over 2 (minimum) to 14 (maximum) years. Tanner development scores, serum testosterone and estradiol, basal follicle-stimulating hormone (FSH) and luteinizing hormone (LH) were analyzed. During pubertal age, the levels of FSH and LH rose consecutively, resulting in noticeably elevated serum concentrations in 100% and 89%, respectively, of boys older than 14 years and in 75% and 75%, respectively, of girls older than 13 years. Nevertheless, pubertal development has been normal in all patients except in one boy and two girls who required substitution with sexual steroids, as timely puberty (i.e. boys < 14 years, girls < 13 years) did not start. In males with normal puberty, testosterone levels, however, were found to be low-normal. In conclusion, after BMT preceded by total body irradiation for childhood ALL, gonadal function is impaired. Even if normal pubertal development occurs, deficiencies in long-term endocrine function cannot be ruled out. In view of the high FSH levels, the prognosis for fertility is doubtful.     

Isolated breast relapse after allogeneic bone marrow transplantation for childhood acute lymphoblastic leukemia

Unusual sites of relapses following bone marrow transplantation (BMT) for childhood acute lymphoblastic leukemia (ALL) are rarely reported. We report the case of a 16-year-old girl who presented with an isolated right breast relapse 8 months after allogeneic BMT for ALL in second remission. Biopsy showed an ALL infiltrate. Bone marrow and CSF were normal. The girl never showed before extramedullary involvement. She was treated with local radiotherapy and mild systemic chemotherapy. Nine months after breast relapse, she presented an isolated central nervous system relapse. The treatment of isolated extramedullary relapses following BMT is still controversial.