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anemia aplastic in children

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https://www.readbyqxmd.com/read/29744996/hematopoietic-stem-cell-transplantation-without-in-vivo-t-cell-depletion-for-pediatric-aplastic-anemia-a-single-center-experience
#1
Sidan Li, Bin Wang, Lingling Fu, Yilin Pang, Guanghua Zhu, Xuan Zhou, Jie Ma, Yan Su, Maoquan Qin, Runhui Wu
For young patients, HLA-MRD HSCT is the first-line treatment of SAA. However, due to China's birth control policy, few patients could find suitable sibling donors and HLA-MUD. More and more transplantation centers have used Haplo-D as the donor source for young adult and pediatric patients. However, studies with larger amount of pediatric patients are rare. We retrospectively analyzed the data of children with AA who were treated with allogeneic HSCT and compared the therapeutic efficacy of Haplo-HSCT and MRD/MUD group...
May 10, 2018: Pediatric Transplantation
https://www.readbyqxmd.com/read/29724291/comparison-of-two-dosages-of-rabbit-antithymocyte-globulin-r-atg-in-treating-children-with-severe-aplastic-anemia
#2
Shao-Fen Lin, Su Liu, Hong-Man Xue, Jun-Bin Huang, Jian Wang, Qi-Hui Chen, Bi-Hong Zhang, Chun Chen
In this study, efficacy and safety of two different dosages of rabbit antithymocyte globulin (r-ATG) combined with cyclosporine (CsA) in treating children with severe aplastic anemia (SAA) were compared. The clinical data of 122 SAA children treated by r-ATG/CsA between Jan 2005 and Jan 2017 at Sun Yat-sen Memorial Hospital of Sun Yat-sen University were retrospectively analyzed. The r-ATG dose of 55 cases was 2.5mg/(kg·d, group 1), and in the other 67 cases it was 3.5 mg/(kg·d, group 2). r-ATG was continuously administered for 5 days...
May 1, 2018: Die Pharmazie
https://www.readbyqxmd.com/read/29722478/successful-reduced-intensity-conditioning-hematopoietic-stem-cell-transplantation-for-paroxysmal-nocturnal-hemoglobinuria-with-aplastic-anemia-in-two-children
#3
Jeffrey R Andolina, Ariel L Reinish, Razia Akhtar, Suzie Noronha, Jessica C Shand, Angela Girvin, David N Korones, Lauren B Bruckner, Craig A Mullen, Kevin J Curran, Farid Boulad
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare cause of bone marrow failure in children. We report two children who presented with pancytopenia, and were diagnosed with PNH with severe aplastic anemia. Both children underwent upfront, successful hematopoietic stem cell transplantation with reduced-intensity conditioning. One patient had a syngeneic donor, and one patient had a 10/10 matched unrelated donor. Neither patient developed graft versus host disease, infections, or recurrent PNH. Reduced-intensity conditioning hematopoietic stem cell transplantation is a reasonable therapy for PNH with marrow failure in children...
May 3, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29700810/refining-the-phenotype-associated-with-biallelic-dnajc21-mutations
#4
G D'Amours, F Lopes, J Gauthier, V Saillour, C Nassif, R Wynn, N Alos, T Leblanc, Y Capri, S Nizard, E Lemyre, J L Michaud, V-A Pelletier, Y D Pastore, J-F Soucy
Inherited bone marrow failure syndromes (IBMFS) are caused by mutations in genes involved in genomic stability. Although they may be recognized by the association of typical clinical features, variable penetrance and expressivity are common, and clinical diagnosis is often challenging. DNAJC21, which is involved in ribosome biogenesis, was recently linked to bone marrow failure. However, the specific phenotype and natural history remain to be defined. We correlate molecular data, phenotype, and clinical history of five unreported affected children and all individuals reported in the literature...
April 26, 2018: Clinical Genetics
https://www.readbyqxmd.com/read/29620676/hepatitis-associated-aplastic-anemia-treated-successfully-with-antithymocyte-globulin
#5
Hongyan Ji, Li Chen, Xiaojun Sun, Xiuli Li, Wei Chen, Ping Zhao, Yulin Wang
Hepatitis-associated aplastic anemia (HAAA) is a variant of acquired aplastic anemia in which bone marrow failure follows the development of an acute episode of seronegative hepatitis. HAAA occurs most frequently in male children and is lethal if left untreated. Antilymphocyte globulin, antithymocyte globulin, and allogeneic bone marrow transplantation have been used in the treatment of this disease. In this work, we report the case of a 3-year-old boy with HAAA treated successfully with immunosuppressive therapy...
April 3, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29562461/-comparison-of-different-styles-of-allogeneic-hematopoietic-stem-cell-transplantation-as-first-line-treatment-treated-with-severe-aplastic-anemia-in-children-and-adolescents
#6
S W Yang, R J Ma, J J Zhao, H F Zhong, X L Yuan, L Jiang, J Yang, P C Lei, Y Zhang, Y W Fu, D M Wan, Z M Zhu
Objective: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) from different donors as first-line treatment for children and adolescents with severe aplastic anemia (SAA) . Methods: The clinical data of 79 children and adolescents with SAA diagnosed from January 2013 to December 2016 in Henan Province were retrospectively analyzed. There were 50 males and 29 females, with a median age of 14(4-18) years. 40 cases received matched sibling transplantation (MSD-HSCT), 17 with unrelated donor transplantation (UD-HSCT), and 22 with haploidentical transplantation (haplo-HSCT)...
March 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29549173/the-clinical-spectrum-of-pyruvate-kinase-deficiency-data-from-the-pyruvate-kinase-deficiency-natural-history-study
#7
Rachael F Grace, Paola Bianchi, Eduard J van Beers, Stefan W Eber, Bertil Glader, Hassan M Yaish, Jenny M Despotovic, Jennifer A Rothman, Mukta Sharma, Melissa M McNaull, Elisa Fermo, Kimberly Lezon-Geyda, D Holmes Morton, Ellis J Neufeld, Satheesh Chonat, Nina Kollmar, Christine M Knoll, Kevin Kuo, Janet L Kwiatkowski, Dagmar Pospíŝilová, Yves D Pastore, Alexis A Thompson, Peter E Newburger, Yaddanapudi Ravindranath, Winfred C Wang, Marcin W Wlodarski, Heng Wang, Susanne Holzhauer, Vicky R Breakey, Joachim Kunz, Sujit Sheth, Melissa J Rose, Heather A Bradeen, Nolan Neu, Dongjing Guo, Hasan Al-Sayegh, Wendy B London, Patrick G Gallagher, Alberto Zanella, Wilma Barcellini
An international, multicenter registry was established to collect retrospective and prospective clinical data on patients with pyruvate kinase (PK) deficiency, the most common glycolytic defect causing congenital non-spherocytic hemolytic anemia. Medical history and laboratory and radiologic data were retrospectively collected at enrollment in 254 patients with molecularly confirmed PK deficiency. Perinatal complications were common, including anemia requiring transfusions, hyperbilirubinemia, hydrops, and prematurity...
March 16, 2018: Blood
https://www.readbyqxmd.com/read/29487060/eltrombopag-for-use-in-children-with-immune-thrombocytopenia
#8
REVIEW
Taylor Olmsted Kim, Jenny Despotovic, Michele P Lambert
Eltrombopag is currently the only US Food and Drug Administration-approved thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia (ITP) in children. This oral, once-per-day therapy has shown favorable efficacy and adverse effect profiles in children. Two multicenter, double-blind, placebo controlled clinical trials (PETIT [Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP)] and PETIT2 [Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body]) demonstrated efficacy in raising platelet counts, reducing bleeding, and reducing the need for concomitant ITP therapies with relatively few adverse effects...
February 27, 2018: Blood Advances
https://www.readbyqxmd.com/read/29359844/hepatitis-a-cardiomyopathy-aplastic-anemia-and-acute-liver-failure-a-devastating-scenario
#9
Verónica Botero, Víctor H García, Ana M Aristizabal, Catalina Gomez, Paola Perez, Luis A Caicedo, Gabriel J Echeverri
Hepatitis A virus (HAV) causes an acute infection and is usually asymptomatic in children. When clinical manifestations appear, these include choluria, jaundice, and abdominal pain. Although infrequent, extra-hepatic manifestations related to HAV have been described, affecting the heart, bone marrow, blood vessels, and other tissues.A 10-year-old boy from a rural area presented with a 15-day history of malaise, fever, and jaundice; laboratory examinations were compatible with HAV infection. The patient turned encephalopathic and was remitted to our center, where laboratory examinations showed a medullary aplasia and fulminant hepatitis requiring a liver transplant that was performed 72 hours after admission...
April 2018: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/29297952/first-line-choice-for-severe-aplastic-anemia-in-children-transplantation-from-a-haploidentical-donor-vs-immunosuppressive-therapy
#10
Yifei Cheng, Zhengli Xu, Yuanyuan Zhang, Jun Wu, Fengrong Wang, Xiaodong Mo, Yuhong Chen, Wei Han, Jinsong Jia, Yu Wang, Xiaohui Zhang, Xiaojun Huang, Leping Zhang, Lanping Xu
We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first-line treatment. The estimated 10-year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID-HSCT (P = ...
February 2018: Clinical Transplantation
https://www.readbyqxmd.com/read/29244176/parvovirus-b19-is-associated-with-a-significant-decrease-in-hemoglobin-level-among-children-5-years-of-age-with-anemia-in-northwestern-tanzania
#11
Yustina A Tizeba, Mariam M Mirambo, Neema Kayange, Tumaini Mhada, Emmanuela E Ambrose, Luke R Smart, Stephen E Mshana
Parvovirus B19 (B19) can cause transient aplastic crisis and lead to acute severe anemia. This study investigated the relationship between B19 and anemia among children <5 years old in the city of Mwanza, Tanzania. An enzyme immunoassay was used to detect B19 IgM- and IgG-specific antibodies among children with various categories of anemia according to the World Health Organization (WHO) guidelines. A total of 265 children with median age of 28.5 months (interquartile range 18-39.5) were investigated. Eighty-six children (32...
December 13, 2017: Journal of Tropical Pediatrics
https://www.readbyqxmd.com/read/29203412/comparable-outcomes-after-hla-matched-sibling-and-alternative-donor-hematopoietic-cell-transplantation-for-children-with-fanconi-anemia-and-severe-aplastic-anemia
#12
Christen L Ebens, Todd E DeFor, Rebecca Tryon, John E Wagner, Margaret L MacMillan
Fanconi anemia (FA)-associated severe aplastic anemia (SAA) requires allogeneic hematopoietic cell transplantation (HCT) for cure. With the evolution of conditioning regimens over time, outcomes of alternative donor HCT (AD-HCT) have improved dramatically. We compared outcomes of HLA-matched sibling donor HCT (MSD-HCT; n = 17) and AD-HCT (n = 57) performed for FA-associated SAA at a single institution between 2001 and 2016. Overall survival at 5 years was 94% for MSD-HCT versus 86% for AD-HCT, neutrophil engraftment was 100% versus 95%, platelet recovery was 100% versus 89%, grade II-IV acute graft-versus-host disease (GVHD) was 6% versus 12%, grade III-IV acute GVHD was 6% versus 4%, and chronic GVHD was 0 versus 7%, with no statistically significant differences by type of transplant...
December 2, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29146883/a-landscape-of-germ-line-mutations-in-a-cohort-of-inherited-bone-marrow-failure-patients
#13
Olivier Bluteau, Marie Sebert, Thierry Leblanc, Régis Peffault de Latour, Samuel Quentin, Elodie Lainey, Lucie Hernandez, Jean-Hugues Dalle, Flore Sicre de Fontbrune, Etienne Lengline, Raphael Itzykson, Emmanuelle Clappier, Nicolas Boissel, Nadia Vasquez, Mélanie Da Costa, Julien Masliah-Planchon, Wendy Cuccuini, Anna Raimbault, Louis De Jaegere, Lionel Adès, Pierre Fenaux, Sébastien Maury, Claudine Schmitt, Marc Muller, Carine Domenech, Nicolas Blin, Bénédicte Bruno, Isabelle Pellier, Mathilde Hunault, Stéphane Blanche, Arnaud Petit, Guy Leverger, Gérard Michel, Yves Bertrand, André Baruchel, Gérard Socié, Jean Soulier
Bone marrow (BM) failure (BMF) in children and young adults is often suspected to be inherited, but in many cases diagnosis remains uncertain. We studied a cohort of 179 patients (from 173 families) with BMF of suspected inherited origin but unresolved diagnosis after medical evaluation and Fanconi anemia exclusion. All patients had cytopenias, and 12.0% presented ≥5% BM blast cells. Median age at genetic evaluation was 11 years; 20.7% of patients were aged ≤2 years and 36.9% were ≥18 years. We analyzed genomic DNA from skin fibroblasts using whole-exome sequencing, and were able to assign a causal or likely causal germ line mutation in 86 patients (48...
February 15, 2018: Blood
https://www.readbyqxmd.com/read/29140934/response-of-symptomatic-persistent-chronic-disseminated-candidiasis-to-corticosteroid-therapy-in-immunosuppressed-pediatric-patients-case-study-and-review-of-the-literature
#14
Vered Shkalim-Zemer, Itzhak Levi, Salvador Fischer, Hannah Tamary, Joanne Yakobovich, Gali Avrahami, Gil Gilad, Sara Elitzur, Isaac Yaniv, Ronit Elhasid, Michal Manistersky, Itamar Shalit
BACKGROUND: Chronic disseminated candidiasis (CDC) is a severe invasive fungal infection principally observed during neutrophil recovery in patients with acute leukemia treated with intensive chemotherapy. Its pathophysiology remains unclear. We describe the management of six children with symptomatic CDC who did not respond to antifungal therapy. METHODS: The databases of the hematology-oncology departments of two tertiary pediatric medical centers were searched for all patients diagnosed with CDC from 2003 to 2015 who responded to corticosteroids after failing antifungal therapy...
November 14, 2017: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/29125443/neurosurgical-management-in-children-with-bleeding-diathesis-auditing-neurological-outcome
#15
Zaitun Zakaria, Chandrasekaran Kaliaperumal, Darach Crimmins, John Caird
OBJECTIVE The aim of this study was to assess the outcome of neurosurgical treatment in children with bleeding diathesis and also to evaluate the current management plan applied in the authors' service. METHODS The authors retrospectively analyzed all cases in which neurosurgical procedures were performed in pediatric patients presenting with intracranial hematoma due to an underlying bleeding tendency over a 5-year period at their institution. They evaluated the patients' neurological symptoms from the initial referral, hematological abnormalities, surgical treatment, neurological outcome, and scores on the Pediatric Glasgow Outcome Scale-Extended (GOS-E Peds) obtained 1 year after the last operation...
January 2018: Journal of Neurosurgery. Pediatrics
https://www.readbyqxmd.com/read/29068868/elevated-serum-interleukin-6-predicts-favorable-response-to-immunosuppressive-therapy-in-children-with-aplastic-anemia
#16
Shuanglong Lu, Xiaohong Qiao, Xiaotian Xie
BACKGROUND: Immunosuppressive therapy (IST) is the standard treatment for aplastic anemia (AA) children who lack a sibling donor, but the clinical response rate to IST varies. Predictors of response to IST are valuable for stratifying AA patients and making clinical decisions. METHODS: The serum interleukin (IL)-6 levels of 41 AA patients were measured at the time of diagnosis and the response rate of the patients to IST was evaluated at 3, 6, and 12 months after IST...
November 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29032585/infection-surveillance-in-pediatric-hematopoietic-stem-cell-transplantation-recipients
#17
Daniela Caldas Teixeira, Lilian Martins Oliveira Diniz, Paulo Henrique Orlandi Mourão, Fabiana Maria Kakehashi, Antonio Vaz de Macedo, Helena Duani, Wanessa Trindade Clemente, Karla Emília de Sá Rodrigues, Roberta Maia de Castro Romanelli
OBJECTIVE: To describe the profile of reported healthcare-associated infections (HAIs) in pediatric patients submitted to hematopoietic stem cell transplantation (HSCT) at a reference center. METHODS: Retrospective cohort of pediatric patients who were submitted to HSCT from 2008 to 2016. The criteria for HAI were based on those established by the National Healthcare Safety Network. Data were collected by active surveillance performed daily by professionals. This study was approved by the institutional research ethics committee...
January 2018: European Journal of Haematology
https://www.readbyqxmd.com/read/28951259/chimerism-in-pediatric-hematopoietic-stem-cell-transplantation-and-its-correlation-with-the-clinical-outcome
#18
Hala Gabr, Ilham Youssry, Yasmin El-Ansary, Ghada Mosallam, Nermine Magdi Riad, Mariam Onsy F Hanna
Hematopoietic stem cell transplantation (HSCT) is the only hope to cure many inherited and acquired hematological disorders in children. Monitoring of chimerism helps to predict the post-transplantation events, with the intention to enhance the long-term disease free survival (DFS). The study aimed to investigate the importance of early chimerism detection to predict the clinical outcome following HSCT. The study included nine recipients (six β-thalassemia and three severe aplastic anemia patients) and their 10/10 HLA identical sibling donors...
September 23, 2017: Transplant Immunology
https://www.readbyqxmd.com/read/28900860/modified-immunosuppressive-therapy-with-porcine-antilymphocyte-globulin-plus-delayed-cyclosporine-a-in-children-with-severe-aplastic-anemia
#19
Qingya Cui, Pingping Sha, Haifei Chen, Hongshi Shen, Longmei Qin, Zhengyang Li, Tianqin Wu, Zhaoyue Wang
Immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine (CsA) is the standard treatment for children with severe aplastic anemia (SAA) with no human leukocyte antigen-matched siblings. Due to the unavailability of horse ATG in China, porcine antilymphocyte globulin (p-ALG), which is less expensive and more effective than rabbit ATG, is widely used. We sought to evaluate the efficacy and safety profile of modified IST with p-ALG plus delayed CsA at day 21 in 50 SAA children. Eighteen SAA patients who progressed from nonsevere aplastic anemia (NSAA) were classified as SAA-II; the other 32 patients were classified as SAA-I...
January 2018: International Journal of Hematology
https://www.readbyqxmd.com/read/28859041/case-report-clinical-variation-in-children-with-thrombopoietin-receptor-c-mpl-mutations-report-of-2-cases
#20
Clara Lo, Elysia Alvarez, Robert S Ohgami, Michael Jeng
Congenital amegakaryocytic thrombocytopenia (CAMT, MIM# 604498) is a rare congenital bone marrow failure syndrome which presents early in life with abnormal bleeding because of thrombocytopenia. Classically, megakaryocytes are decreased to absent in the bone marrow. The development of aplastic anemia early in childhood has led to the recommendation for early stem cell transplantation. Quantitative or loss-of-function mutations in the myeloproliferative leukemia gene (c-mpl), whose gene product functions as the thrombopoietin receptor, have been identified as causative for CAMT...
January 2018: Journal of Pediatric Hematology/oncology
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