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anemia aplastic in children

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https://www.readbyqxmd.com/read/28211781/post-therapeutic-recovery-of-serum-interleukin-35-level-might-predict-positive-response-to-immunosuppressive-therapy-in-pediatric-aplastic-anemia
#1
Zhen Huang, Hongfei Tong, Yuan Li, Haixia Zhou, Jiangchao Qian, Juxiang Wang, Jichen Ruan
BACKGROUND: The predictive value of interleukin-35 (IL-35) on efficacy of immunosuppressive therapy (IST) in aplastic anemia (AA) has not been well investigated. The aim of the study was to evaluate the association between serum IL-35 level and response to IST in pediatric AA. METHODS: A total of 154 children with AA and 154 controls were included between January 2012 and December 2013. Blood and bone marrow fluid specimens were collected. Serum level of IL-35 was determined by enzyme-linked immunosorbent assay...
February 17, 2017: Hematology (Amsterdam, Netherlands)
https://www.readbyqxmd.com/read/28100318/-clinical-characteristics-of-clonal-evolution-after-immunosuppressive-therapy-in-children-with-severe-very-severe-aplastic-anemia
#2
Jing-Liao Zhang, Tian-Feng Liu, Li-Xian Chang, Xia Chen, Yuan-Yuan Ren, Cong-Cong Sun, Chao Liu, Wen-Bin An, Yang Wan, Xiao-Juan Chen, Wen-Yu Yang, Shu-Chun Wang, Ye Guo, Yao Zou, Yu-Mei Chen, Xiao-Fan Zhu
OBJECTIVE: To evaluate the clinical characteristics and risk factors of clonal evolution after immunosuppressive therapy (IST) in children with severe/very severe aplastic anemia (SAA/VSAA). METHODS: The clinical data of 231 children with newly-diagnosed SAA/VSAA who received IST were retrospectively studied. The incidence and risk factors of clonal evolution after IST were analyzed. RESULTS: The 5-year overall survival rate of the 231 patients was 82...
January 2017: Zhongguo Dang Dai Er Ke za Zhi, Chinese Journal of Contemporary Pediatrics
https://www.readbyqxmd.com/read/28060106/interleukin-6-and-interleukin-8-levels-correlate-with-the-severity-of-aplastic-anemia-in-children
#3
Vineeta Gupta, Sushil Kumar, Rimjhim Sonowal, Surya K Singh
AIM: The aim of this study was to evaluate the levels of interleukin (IL)-6 and IL-8 in patients with aplastic anemia and its correlation with severity of the disease. MATERIALS AND METHODS: IL-6 and IL-8 levels were measured in 40 patients with aplastic anemia in the age group of 4 to 14 years. A total of 40 healthy children served as controls. Quantitative estimation of IL-6 and IL-8 was performed using a solid-phase sandwich ELISA kit. Results were presented as IL-6 and IL-8 concentrations in pg/mL...
April 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/27941773/haploidentical-transplantation-for-pediatric-patients-with-acquired-severe-aplastic-anemia
#4
L P Xu, X H Zhang, F R Wang, X D Mo, T T Han, W Han, Y H Chen, Y Y Zhang, J Z Wang, C H Yan, Y Q Sun, S N Zuo, X J Huang
Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1...
March 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27916046/-a-comparative-study-of-unrelated-donor-and-matched-sibling-donor-allogeneic-hematopoietic-stem-cell-transplantation-in-children-and-adolescents-with-acquired-severe-aplastic-anemia
#5
J Zhou, Y W Fu, L J Liang, Q Wang, L J Han, Y L Zu, Yanli Zhang, X H Zhu, F K Yu, B J Fang, X D Wei, Y P Song
Objective: To evaluate the efficacy of unrelated donor allogeneic hematopoietic stem cell transplantation(URD allo-HSCT) for children and adolescents with severe aplastic anemia (SAA). Methods: Clinical data of 34 SAA children and adolescents undergoing allo-HSCT were retrospectively analyzed from October 2001 to October 2015. According to the source of donor, the patients were divided into matched sibling donor allo-HSCT group (MSD group) and unrelated donor group (URD group). The clinical outcome of SAA children and adolescents receiving URD allo-HSCT was assessed, and patients in MSD allo-HSCT group were enrolled as control at the same period...
December 1, 2016: Zhonghua Nei Ke za Zhi [Chinese Journal of Internal Medicine]
https://www.readbyqxmd.com/read/27913534/myelodysplastic-and-myeloproliferative-disorders-of-childhood
#6
Henrik Hasle
Myelodysplastic syndrome (MDS) and myeloproliferative disorders are rare in children; they are divided into low-grade MDS (refractory cytopenia of childhood [RCC]), advanced MDS (refractory anemia with excess blasts in transformation), and juvenile myelomonocytic leukemia (JMML), each with different characteristics and management strategies. Underlying genetic predisposition is recognized in an increasing number of patients. Germ line GATA2 mutation is found in 70% of adolescents with MDS and monosomy 7. It is challenging to distinguish RCC from aplastic anemia, inherited bone marrow failure, and reactive conditions...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27808465/long-term-outcomes-of-95-children-with-moderate-aplastic-anemia-treated-with-horse-antithymocyte-globulin-and-cyclosporine
#7
Eri Nishikawa, Hiroshi Yagasaki, Asahito Hama, Hiromasa Yabe, Akira Ohara, Yoshiyuki Kosaka, Kazuko Kudo, Ryoji Kobayashi, Shouichi Ohga, Akira Morimoto, Ken-Ichiro Watanabe, Nao Yoshida, Hideki Muramatsu, Yoshiyuki Takahashi, Seiji Kojima
BACKGROUND: Currently, the standard management of moderate aplastic anemia (MAA) has not been well described, although the superiority of the combination of antithymocyte globulin (ATG) and cyclosporine (CyA) over CyA alone has been demonstrated in terms of hematological responses and failure-free survival (FFS). PROCEDURE: We adopted this therapeutic strategy and treated 95 children with MAA who were enrolled in two consecutive prospective studies between October 1992 and August 2009...
May 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27806787/-analysis-of-clinical-characteristics-and-prognosis-of-non-severe-aplastic-anemia-children-with-chromosomal-abnormalities
#8
S Zhu, W B An, Y Wan, Y Y Ren, R R Zhang, J L Zhang, C Liu, C C Sun, L X Chang, X J Chen, W Y Yang, Y Guo, Y M Chen, Y Zou, X F Zhu
Objective: To analyze the clinical characteristics and prognosis of non-severe aplastic anemia (NSAA) with chromosomal abnormalities in children. Method: A retrospective analysis of 304 cases with NSAA with successful karyotyping from 2001 to 2014 in the Institute of Hematology & Blood Disease Hospital was carried out. The treatment response, condition of blood transfusion were analyzed using χ(2) test, the cumulative survival was estimated by the Kaplan-Meier method. Result: Out of 304 patients, 28 patients had chromosomal abnormalities with trisomy 8 (7 cases, 25...
November 2, 2016: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/27668766/immunosuppressive-therapy-versus-alternative-donor-hematopoietic-stem-cell-transplantation-for-children-with-severe-aplastic-anemia-who-lack-an-hla-matched-familial-donor
#9
Y B Choi, E S Yi, J W Lee, K W Sung, H H Koo, K H Yoo
We compared the outcomes of immunosuppressive treatment (IST) with those of alternative donor hematopoietic stem cell transplantation (HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 42 patients with SAA who received frontline IST (N=19) or frontline HSCT with an alternative donor (N=23) between 1998 and 2012 were analyzed retrospectively. Six patients responded in the frontline IST group, whereas 11 underwent salvage HSCT after IST failure. Twenty-one of 23 patients who underwent frontline HSCT survived without treatment failure...
January 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27548335/hemophagocytic-lymphohistiocytosis-in-a-child-with-very-severe-aplastic-anemia-double-jeopardy-resulting-in-fatality
#10
Rumesh Chandar, Venkatesh Chandrasekaran, Barath Jagadisan, Delhi Kumar, Niranjan Biswal
A 7-year-old male child with very severe aplastic anemia developed refractory disease, which was attributed to febrile hemolytic transfusion reactions and was treated with immunosuppressants, which lead to a transient improvement. However, the child worsened subsequently and succumbed to an underlying hemophagocytic lymphohistiocytosis that was recognized late due to an overlap of clinical and biochemical parameters in both the conditions. Hemophagocytic lymphohistiocytosis should be an early suspicion in children with refractory very severe aplastic anemia and the detection of underlying gene mutations can predict disease severity...
January 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/27530658/brca2-functions-from-dna-repair-to-replication-fork-stabilization
#11
Amélie Fradet-Turcotte, Justine Sitz, Damien Grapton, Alexandre Orthwein
Maintaining genomic integrity is essential to preserve normal cellular physiology and to prevent the emergence of several human pathologies including cancer. The breast cancer susceptibility gene 2 (BRCA2, also known as the Fanconi anemia (FA) complementation group D1 (FANCD1)) is a potent tumor suppressor that has been extensively studied in DNA double-stranded break (DSB) repair by homologous recombination (HR). However, BRCA2 participates in numerous other processes central to maintaining genome stability, including DNA replication, telomere homeostasis and cell cycle progression...
October 2016: Endocrine-related Cancer
https://www.readbyqxmd.com/read/27467371/autoimmune-hepatitis-and-seronegative-hepatitis-associated-with-myelodysplastic-syndrome-in-children
#12
Line K Rasmussen, Elisabeth V Stenbøg, Gitte B Kerndrup, Henrik Hasle
An association between hepatitis and aplastic anemia (AA) is known as hepatitis-associated AA, and is characterized by an acute attack of hepatitis followed by the development of AA. We report 2 clinical cases of acute seronegative hepatitis in which pancytopenia with mild dysplasia developed after 3 months; however, neither of our cases fulfilled the histological criteria of AA, but rather myelodysplastic syndrome. This novel association bears considerable resemblance to hepatitis-associated AA, and raises the question of whether hepatitis-associated dysmyelopoiesis should be included in the spectrum of hypocellular myelodysplastic syndrome...
November 2016: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/27467367/high-dose-cyclophosphamide-is-effective-therapy-for-pediatric-severe-aplastic-anemia
#13
Christopher J Gamper, Clifford M Takemoto, Allen R Chen, Heather J Symons, David M Loeb, James F Casella, Amy E Dezern, Karen E King, Andrea M McGonigle, Richard J Jones, Robert A Brodsky
OBJECTIVE: Use of high-dose cyclophosphamide without hematopoietic stem cell transplant to treat severe aplastic anemia (SAA) has been controversial due to concern for increased infectious toxicity as compared with antithymocyte globulin and cyclosporine A. As children often tolerate dose-intensive therapy better than adults, we sought to perform a detailed retrospective analysis of both treatment response and toxicity in 28 patients younger than 22 years of age treated with 29 courses of high-dose cyclophosphamide as the sole form of immunosuppression...
November 2016: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/27431066/-efficacy-and-security-of-matched-unrelated-donor-hematopoietic-stem-cell-transplant-with-transfusion-of-multipotent-mesenchymal-cells-in-pediatric-severe-aplastic-anemia
#14
L Wang, H X Wang, L Zhu, X L Zheng, Z D Wang, H M Yan, L Ding, D M Han
OBJECTIVE: To observe the efficacy of matched unrelated donor hematopoietic stem cell transplant (HSCT) with transfusion of multipotent mesenchymal cells (MSC) in pediatric severe aplastic anemia (SAA). METHODS: 19 children with SAA received matched unrelated donor HSCT with MSC, and the hematopoietic recovery and transplant-associated complications of these children were monitored. RESULTS: All patients achieved rapid hematopoietic reconstruction after HSCT, and the median durations to neutrophil and platelet recovery were 12 (9-21) days and 14 (8-24) days respectively, but delayed rejection occurred in one case four months after HSCT...
June 14, 2016: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/27418648/genetic-features-of-myelodysplastic-syndrome-and-aplastic-anemia-in-pediatric-and-young-adult-patients
#15
Siobán B Keel, Angela Scott, Marilyn Sanchez-Bonilla, Phoenix A Ho, Suleyman Gulsuner, Colin C Pritchard, Janis L Abkowitz, Mary-Claire King, Tom Walsh, Akiko Shimamura
The clinical and histopathological distinctions between inherited versus acquired bone marrow failure and myelodysplastic syndromes are challenging. The identification of inherited bone marrow failure/myelodysplastic syndromes is critical to inform appropriate clinical management. To investigate whether a subset of pediatric and young adults undergoing transplant for aplastic anemia or myelodysplastic syndrome have germline mutations in bone marrow failure/myelodysplastic syndrome genes, we performed a targeted genetic screen of samples obtained between 1990-2012 from children and young adults with aplastic anemia or myelodysplastic syndrome transplanted at the Fred Hutchinson Cancer Research Center...
November 2016: Haematologica
https://www.readbyqxmd.com/read/27265631/invasive-fungal-diseases-in-children-with-hematologic-disorders
#16
Birol Baytan, Adalet Meral Güneş, Solmaz Çelebi, Ünsal Günay
OBJECTIVE: Fungal infection is a significant problem, causing of infective deaths of leukemic patients. The situation in developing countries is not well documented. The purpose of this study was characterizing IFD by analyzing data retrospectively to determine the incidence, predisposing factors, diagnostic methods, efficacy of treatment, and the outcome in pediatric patients with hematological disorders. METHODS: There were 160 children with leukemia (22 AML, 129 ALL) and 9 with aplastic anemia (AA)...
December 5, 2009: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
https://www.readbyqxmd.com/read/27263601/posterior-reversible-leukoencephalopathy-syndrome-in-children-with-hematologic-disorders
#17
Barış Malbora, Zekai Avcı, Fulden Dönmez, Bülent Alioğlu, Esra Baskın, Füsun Alehan, Namık Özbek
OBJECTIVE: Posterior reversible leukoencephalopathy syndrome (PRES) is characterized by headache, altered mental status, cortical blindness, and seizures associated with neuroradiological findings. It involves predominantly white matter of the parieto-occipital lobes. Several medications and disorders play a role in the etiology of PRES. In this study, we aimed to show how the prognosis of PRES in hematological diseases of childhood might be according to the etiological factors. METHODS: Here, we report PRES in six patients, aged 4 to 14 years, with diagnoses of leukemia and aplastic anemia...
September 5, 2010: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
https://www.readbyqxmd.com/read/27186230/subclinical-left-ventricular-dysfunction-in-children-after-hematopoietic-stem-cell-transplantation-for-severe-aplastic-anemia-a-case-control-study-using-speckle-tracking-echocardiography
#18
Beom Joon Kim, Kyung Pil Moon, Ji-Hong Yoon, Eun-Jung Lee, Jae Young Lee, Seong Koo Kim, Jae Wook Lee, Nack Gyun Chung, Bin Cho, Hack Ki Kim
PURPOSE: Severe aplastic anemia (SAA), a fatal disease, requires multiple transfusion, immunosuppressive therapy, and finally, hematopoietic stem cell transplantation (HSCT) as the definitive treatment. We hypothesized that iron overloading associated with multiple transfusions and HSCTrelated complications may adversely affect cardiac function. Left ventricular (LV) function was assessed in children after HSCT for SAA. METHODS: Forty-six consecutive patients with a median age of 9...
April 2016: Korean Journal of Pediatrics
https://www.readbyqxmd.com/read/27173422/transient-hyperphosphatasemia-in-three-pediatric-patients-treated-with-cyclosporine
#19
Takeshi Mori, Ryojiro Tanaka, Kosuke Nishida, Nobuyuki Yamamoto, Akira Hayakawa, Noriyuki Nishimura, Kandai Nozu, Kazumoto Iijima
Transient hyperphosphatasemia (TH) is defined as marked elevation of serum alkaline phosphatase (ALP), predominantly its bone or liver isoform. It is a rare condition and is usually detected on laboratory examination in patients without any clinical symptoms. In typical patients with TH, ALP spontaneously normalizes, but no apparent cause of TH has been identified. Some drugs are suspected triggers of TH, but no clear evidence of their association with TH has been shown to date. We herein report three cases of TH in pediatric patients...
May 2016: Pediatrics International: Official Journal of the Japan Pediatric Society
https://www.readbyqxmd.com/read/27107757/immunosuppressive-therapy-for-patients-with-down-syndrome-and-idiopathic-aplastic-anemia
#20
Kyogo Suzuki, Hideki Muramatsu, Yusuke Okuno, Atsushi Narita, Asahito Hama, Yoshiyuki Takahashi, Makoto Yoshida, Yasuo Horikoshi, Ken-Ichiro Watanabe, Kazuko Kudo, Seiji Kojima
Idiopathic aplastic anemia (AA) is a rare hematological complication of Down syndrome (DS). The safety and efficacy of immunosuppressive therapy (IST) in individuals with DS remain unknown. We used a standard regimen of IST, comprising antithymocyte globulin and cyclosporine A, to treat three children with DS and idiopathic acquired AA. Two patients achieved a hematological (complete or partial) response and became transfusion independent at the final follow-up. The third patient failed to respond to IST and underwent bone marrow transplantation from a human leukocyte antigen (HLA)-mismatched unrelated donor...
July 2016: International Journal of Hematology
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