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https://read.qxmd.com/read/30706625/pediatric-acute-myeloid-leukemia-with-t-7-21-p22-q22
#1
Prabakaran Paulraj, Steven Diamond, Faisal Razzaqi, Dan Ozeran, Maria Longhurst, Erica F Andersen, Reha M Toydemir, Bo Hong
The t(7;21)(p22;q22) resulting in RUNX1-USP42 fusion, is a rare but recurrent cytogenetic abnormality associated with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The prognostic significance of this translocation has not been well established due to the limited number of patients. Herein, we report three pediatric AML patients with t(7;21)(p22;q22). All three patients presented with pancytopenia or leukopenia at diagnosis, accompanied by abnormal immunophenotypic expression of CD7 and CD56 on leukemic blasts...
January 31, 2019: Genes, Chromosomes & Cancer
https://read.qxmd.com/read/30655602/class-i-and-ii-human-leukocyte-antibodies-in-pediatric-haploidentical-allograft-candidates-prevalence-and-risk-factors
#2
Meng Lv, Shu-Zhen Zhai, Yu Wang, Lan-Ping Xu, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Yi-Fei Cheng, Chen-Hua Yan, Xiao-Dong Mo, Kai-Yan Liu, Ying-Jun Chang, Xiao-Jun Huang, Xiang-Yu Zhao
Donor-specific anti-human leukocyte antigen (HLA) antibodies (DSAs) were associated with graft failure (GF) following haploidentical stem cell transplantation (Haplo-HSCT). The prevalence and risk factors of DSAs in pediatric candidates remain to be determined. In a prospective trial (ChiCTR-OPC-15006672), 486 children with hematological diseases were enrolled to screen for the presence of anti-HLA class I and II antibodies of immunoglobulin G type. Fifty two patients (10.7%) demonstrated positive panel-reactive antibody (PRA) for class I; 24 (4...
January 17, 2019: Bone Marrow Transplantation
https://read.qxmd.com/read/30649224/bloodstream-infection-bsi-due-to-vancomycin-resistant-enterococcus-vre-is-associated-with-increased-mortality-after-hematopoietic-cell-transplantation-for-acute-leukemia-and-myelodysplastic-syndrome-a-multicenter-retrospective-cohort-study
#3
Genovefa A Papanicolaou, Celalettin Ustun, Jo-Anne H Young, Min Chen, Soyoung Kim, Kwang Woo Ahn, Krishna Komanduri, Caroline Lindemans, Jeffery J Auletta, Marcie L Riches
Background: We examined the impact of Enterococcus (VRE) bloodstream infection (BSI) on outcomes of allogeneic hematopoietic cell transplantation (HCT) utilizing the Center for International Blood and Marrow Transplant Research (CIBMTR) database. Methods: Adult and pediatric patients (n=7,128), who underwent first HCT for acute leukemia or myelodysplastic syndrome from 2008 through 2012, were analyzed as 3 groups-VRE-BSI, non-VRE-BSI, without BSI-according to BSI status at 100 days (D100) after allogeneic HCT...
January 14, 2019: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
https://read.qxmd.com/read/30625391/increased-health-care-utilization-and-costs-during-allogeneic-hematopoietic-cell-transplant-for-acute-leukemia-and-myelodysplastic-syndromes-in-adolescents-and-young-adults-compared-to-children-a-multicenter-study
#4
Hemalatha G Rangarajan, Lauren C Smith, Joseph R Stanek, Matthew Hall, Rolla Abu-Arja, Jeffery J Auletta, Sarah H O'Brien
Allogeneic hematopoietic cell transplant (HCT) is a curative option for patients with acute leukemia and myelodysplastic syndromes (MDS), but is associated with significant cost. When compared to children (age <15 years), adolescents and young adults (AYA, ages 15-39 years) receiving HCT have an increased risk for transplant-related complications. Whether such complications translate into increased resource utilization and costs during HCT remains unknown. Therefore, we conducted a multicenter database study using the Pediatric Health Information System database, an administrative database containing resource utilization data from 49 tertiary children's hospitals in the United States, to compare inpatient costs and resource utilization in children and AYA undergoing HCT for acute leukemia and MDS...
January 6, 2019: Biology of Blood and Marrow Transplantation
https://read.qxmd.com/read/30537106/a-single-institutional-review-of-pediatric-bacillus-spp-bloodstream-infections-demonstrates-increased-incidence-among-children-with-cancer
#5
David S Shulman, Preeti Mehrotra, Traci M Blonquist, Andrew Capraro, Leslie E Lehmann, Lewis B Silverman, Neeraj K Surana, Andrew E Place
BACKGROUND: Bacillus species are known to cause severe infection in immunocompromised hosts. The incidence of Bacillus bloodstream infections and characteristics of infection among children with cancer or indication for hematopoietic cell transplant (HCT) is unknown. METHODS: We performed a retrospective medical record review of all cases of Bacillus bacteremia between January 1, 2005, and December 31, 2014, at Boston Children's Hospital. We report average incidences from 2012 to 2014...
December 11, 2018: Pediatric Blood & Cancer
https://read.qxmd.com/read/30536675/population-pharmacokinetic-analysis-of-decitabine-in-pediatric-patients-with-acute-myeloid-leukemia
#6
Wangda Zhou, Dolly A Parasrampuria, Sepideh Nemat, Susumu Nakahara, Italo Poggesi, Joseph Massarella, Liping Zhang, Carlos Appiani
Dacogen, the formulated product of the pharmaceutically active agent decitabine (5 aza-2'-deoxycytidine), is approved for treatment of myelodysplastic syndromes (MDSs) and acute myeloid leukemia (AML). The current analysis was performed to characterize the pharmacokinetics of decitabine in pediatric patients with AML and evaluate their consistency with the PK in adult patients. A population pharmacokinetic model was developed by pooling decitabine concentration-time data from 5 adult (AML and MDS) and 2 pediatric (AML) studies...
December 11, 2018: Journal of Clinical Pharmacology
https://read.qxmd.com/read/30501706/-clinical-charcteristics-and-prognostic-analysis-of-28-cases-of-pediatric-myelodysplastic-syndrome
#7
Jian Wang, Shao-Fen Lin, Qi-Hui Chen, Kun-Yin Qiu, Hong-Gui Xu, Ke Huang, Yang Li, Jian-Pei Fang, Zhou Dun-Hua Zhou
OBJECTIVE: To analyze the clinical features and prognosis of 28 children with myelodysplastic syndrome (MDS) and to screen the high risk factors affecting the prognosis so as to provide the new ideas for standard of clinical diagnosis and therapy. METHODS: The clinical data of 28 children with newly diagnosed MDS treated in our hospital from March 1994 to July 2016 were analyzed retrospectively, the features of disease onset and the results of laboratory examination were summarized, all MDS children were followed up, the prognosis and the high risk factors affecting the prognosis were evaluated...
December 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://read.qxmd.com/read/30450160/knock-in-of-the-wt1-r394w-mutation-causes-mds-and-cooperates-with-flt3-itd-to-drive-aggressive-myeloid-neoplasms-in-mice
#8
Colleen E Annesley, Cara Rabik, Amy S Duffield, Rachel E Rau, Daniel Magoon, Li Li, Vicki Huff, Donald Small, David M Loeb, Patrick Brown
Wilms tumor 1 (WT1) is a zinc finger transcriptional regulator, and has been implicated as both a tumor suppressor and oncogene in various malignancies. Mutations in the DNA-binding domain of the WT1 gene are described in 10-15% of normal-karyotype AML (NK-AML) in pediatric and adult patients. Similar WT1 mutations have been reported in adult patients with myelodysplastic syndrome (MDS). WT1 mutations have been independently associated with treatment failure and poor prognosis in NK-AML. Internal tandem duplication (ITD) mutations of FMS-like tyrosine kinase 3 ( FLT3 ) commonly co-occur with WT1 -mutant AML, suggesting a cooperative role in leukemogenesis...
October 19, 2018: Oncotarget
https://read.qxmd.com/read/30334483/-change-in-paradigm-in-the-treatment-of-pediatric-acquired-bone-marrow-failure-syndromes-in-hungary
#9
Krisztián Kállay, Judit Csomor, Emma Ádám, Csaba Bödör, Csaba Kassa, Réka Simon, Gábor Kovács, György Péter, Gábor Ottóffy, Katalin Bartyik, Csongor Kiss, Péter Masát, Marienn Réti, Blanka Tóth, Gergely Kriván
INTRODUCTION: Acquired bone marrow failures are rare but fatal diseases in childhood. Since 2013, Hungary has been participating as a full member in the work of the European Working Group on uniform diagnostics and therapy in patients with acquired bone marrow failure syndromes. Hypocellular refractory cytopenia of childhood has been emphasized as a frequent entity, transplanted by reduced intensity conditioning with excellent outcomes. AIM: To analyse and compare the results of treatment before and after our joining...
October 2018: Orvosi Hetilap
https://read.qxmd.com/read/30305537/-progress-in-research-of-the-pathogenesis-of-childhood-mds-mpn
#10
Kenichi Yoshida
Recent research reveals novel insights into the pathogenesis of childhood myelodysplastic syndromes (MDS) in addition to that of juvenile myelomonocytic leukemia (JMML). In pediatric MDS, the genetic characteristics of which have been barely elucidated previously, germline mutations, particularly those in GATA2, SAMD9, and SAML9L, have been frequently identified, indicating the importance of germline predisposition in childhood MDS compared with adult MDS. In JMML, in addition to the known Ras-pathway mutations, novel secondary mutations and causative fusion genes have been reported...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://read.qxmd.com/read/30303870/acute-megakaryoblastic-leukemia-with-diffuse-periosteal-reaction-of-bilateral-lower-extremities
#11
Erin E Doherty, Erica K Schallert, Alexandra M Stevens, Jyotinder N Punia, Jennifer H Foster
We report the case of a 3-year-old girl diagnosed with acute megakaryoblastic leukemia, who presented after >1 year of bilateral leg pain. At times the pain was severe enough to prevent ambulation, prompting visits to her primary care provider. However, it was not until acute respiratory failure occurred with subsequent hospitalization in the pediatric intensive care unit that severe anemia and thrombocytopenia were discovered and the diagnosis of acute myeloid leukemia was made. Bilateral lower extremity swelling was noted on admission and radiographs showed diffusely abnormal appearance of the long bones of her lower extremities with periosteal reaction and echogenic debris in the subperiosteal space, thought to represent leukemic cells...
October 9, 2018: Journal of Pediatric Hematology/oncology
https://read.qxmd.com/read/30088135/long-term-pediatric-hematological-morbidity-of-the-early-term-newborn
#12
Gil Gutvirtz, Tamar Wainstock, Eyal Sheiner, Daniella Landau, Alon Slutzky, Asnat Walfisch
Children born at early term (37 0/7 to 38 6/7 weeks' gestation) are at an increased risk for long-term respiratory, cardiovascular, neurological, and developmental morbidities as compared with children born at full term (39 0/7 to 40 6/7 weeks' gestation). In this population-based cohort analysis, we sought to evaluate the long-term hematological morbidity of early-term born children. The cohort consisted of 223,242 term singleton deliveries. Hospitalizations of the offspring up to 18 years of age involving hematological morbidity were evaluated, including hereditary and acquired anemias, immunodeficiency disorders, coagulation disorders, white blood cell disorders, cytopenias, polycythemia, and myelodysplastic syndrome...
November 2018: European Journal of Pediatrics
https://read.qxmd.com/read/30047423/monosomy-7-in-pediatric-myelodysplastic-syndromes
#13
REVIEW
Marcin W Wlodarski, Sushree S Sahoo, Charlotte M Niemeyer
Myelodysplastic syndromes (MDS) in children and adolescents are a rare heterogeneous group of clonal stem cell disorders. Complete or partial loss of chromosome 7 constitutes the most common cytogenetic abnormality encountered in any type of childhood MDS, is associated with more advanced disease, and usually requires a timely allogeneic stem cell transplantation. This article provides insights into the current understanding of the genotype, phenotype, and clonal evolution patterns in pediatric MDS associated with loss of chromosome 7...
August 2018: Hematology/oncology Clinics of North America
https://read.qxmd.com/read/30020493/radiation-exposure-from-pediatric-ct-scans-and-subsequent-cancer-risk-in-the-netherlands
#14
Johanna M Meulepas, Cécile M Ronckers, Anne M J B Smets, Rutger A J Nievelstein, Patrycja Gradowska, Choonsik Lee, Andreas Jahnen, Marcel van Straten, Marie-Claire Y de Wit, Bernard Zonnenberg, Willemijn M Klein, Johannes H Merks, Otto Visser, Flora E van Leeuwen, Michael Hauptmann
Background: Computed tomography (CT), a strong diagnostic tool, delivers higher radiation doses than most imaging modalities. As CT use has increased rapidly, radiation protection is important, particularly among children. We evaluate leukemia and brain tumor risk following exposure to low-dose ionizing radiation from CT scans in childhood. Methods: For a nationwide retrospective cohort of 168 394 children who received one or more CT scans in a Dutch hospital between 1979 and 2012 who were younger than age 18 years, we obtained cancer incidence, vital status, and confounder information by record linkage with external registries...
July 18, 2018: Journal of the National Cancer Institute
https://read.qxmd.com/read/30008805/an-uncommon-t-9-11-p24-q22-with-monoallelic-loss-of-atm-and-kmt2a-genes-in-a-child-with-myelodysplastic-syndrome-acute-myeloid-leukemia-who-evolved-from-fanconi-anemia
#15
Viviane Lamim Lovatel, Daiane Corrêa de Souza, Tatiana Fonseca Alvarenga, Roberto R Capela de Matos, Claudia Diniz, Marcia Trindade Schramm, Juan Clinton Llerena Júnior, Maria Luiza Macedo Silva, Eliana Abdelhay, Teresa de Souza Fernandez
Background: Myelodysplastic syndrome (MDS) is rare in the pediatric age group and it may be associated with inheritable bone marrow failure (BMF) such as Fanconi anemia (FA). FA is a rare multi-system genetic disorder, characterized by congenital malformations and progressive BMF. Patients with FA usually present chromosomal aberrations when evolving to MDS or acute myeloid leukemia (AML). Thus, the cytogenetic studies in the bone marrow (BM) of these patients have an important role in the therapeutic decision, mainly in the indication for hematopoietic stem cell transplantation (HSCT)...
2018: Molecular Cytogenetics
https://read.qxmd.com/read/29891567/clonal-dynamics-of-donor-derived-myelodysplastic-syndrome-after-unrelated-hematopoietic-cell-transplantation-for-high-risk-pediatric-b-lymphoblastic-leukemia
#16
Jason R Schwartz, Michael P Walsh, Jing Ma, Tamara Lamprecht, Shuoguo Wang, Gang Wu, Susana Raimondi, Brandon M Triplett, Jeffery M Klco
Donor-derived hematologic malignancies are rare complications of hematopoietic cell transplantation (HCT). Although these are commonly either a myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), in general, they are a heterogeneous group of diseases, and a unified mechanism for their development has remained elusive. Here we report next-generation sequencing, including whole-exome sequencing (WES), whole-genome sequencing (WGS), and targeted sequencing, of a case of donor-derived MDS (dMDS) following HCT for high-risk B-lymphoblastic leukemia (B-ALL) in an adolescent...
October 2018: Cold Spring Harbor Molecular Case Studies
https://read.qxmd.com/read/29888021/sacral-myeloid-sarcoma-manifesting-as-radiculopathy-in-a-pediatric-patient-an-unusual-form-of-myeloid-leukemia-relapse
#17
Joana Ruivo Rodrigues, Manuel João Brito, Rui Pedro Faria Pais, Sílvia Carvalho
Myeloid sarcoma (MS), granulocytic sarcoma or chloroma, is defined as a localized extramedullary mass of blasts of granulocytic lineage with or without maturation, occurring outside the bone marrow. MS can be diagnosed concurrently with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The authors report a case of sacral MS occurring as a relapse of myeloid leukemia in a 5-year-old girl who was taken to the emergency department with radiculopathy symptoms.
2018: Case Reports in Radiology
https://read.qxmd.com/read/29803285/acquired-aplastic-anemia-what-have-we-learned-and-what-is-in-the-horizon
#18
REVIEW
Süreyya Savaşan
Acquired aplastic anemia (aAA) characterized by peripheral pancytopenia and bone marrow aplasia is a rare and serious disorder. Differential diagnosis includes constitutional bone marrow failure syndromes and myelodysplastic disorders. Autoimmune reaction to altered hematopoietic stem cells highlights the underlying mechanism. Matched related donor allogeneic hematopoietic stem cell transplantation is the ideal pediatric treatment; alternative approaches include immunosuppressive therapy and use of eltrombopag...
June 2018: Pediatric Clinics of North America
https://read.qxmd.com/read/29753157/treosulfan-fludarabine-and-low-dose-total-body-irradiation-for-children-and-young-adults-with-acute-myeloid-leukemia-or-myelodysplastic-syndrome-undergoing-allogeneic-hematopoietic-cell-transplantation-prospective-phase-ii-trial-of-the-pediatric-blood-and-marrow
#19
Eneida R Nemecek, Ralf A Hilger, Alexia Adams, Bronwen E Shaw, Deidre Kiefer, Jennifer Le-Rademacher, John E Levine, Gregory Yanik, Wing Leung, Julie-An Talano, Paul Haut, David Delgado, Neena Kapoor, Aleksandra Petrovic, Roberta Adams, Rabi Hanna, Hemalatha Rangarajan, Jignesh Dalal, Joseph Chewning, Michael R Verneris, Stacy Epstein, Lauri Burroughs, Evelio D Perez-Albuerne, Michael A Pulsipher, Colleen Delaney
This multicenter study evaluated a treosulfan-based regimen in children and young adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplant (HCT). Forty patients with median age 11 years (range, 1 to 19) underwent allogeneic HCT for AML in first (n = 18), second (n = 11), and third or greater remission (n = 3) or MDS (n = 8) using bone marrow (n = 25), peripheral blood stem cells (n = 5), or cord blood (n = 9). The regimen consisted of body surface area (BSA)-based treosulfan 10 g/m2 /day (BSA ≤ ...
August 2018: Biology of Blood and Marrow Transplantation
https://read.qxmd.com/read/29742616/spontaneous-remission-of-monosomy-7-six-years-after-diagnosis
#20
Holly J Edington, Eric J Lowe
Monosomy 7 may be a poor prognostic indicator in pediatric myelodysplastic syndrome. There are case reports of children with monosomy 7 who undergo spontaneous remission 2 to 24 months after diagnosis. We report a case of a child with bone marrow failure and monosomy 7 who underwent spontaneous remission 75 months after diagnosis. The patient had no exposure to chemotherapeutic or immunosuppressive agents. The patient did not receive chemotherapy or other treatment during the 75 months. Despite remaining positive for monosomy 7, he never developed myelodysplasia or acute myeloid leukemia...
May 8, 2018: Journal of Pediatric Hematology/oncology
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