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Pediatric Bone marrow transplantation

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https://www.readbyqxmd.com/read/28174417/amkl-chimeric-transcription-factors-are-potent-inducers-of-leukemia
#1
J Dang, S Nance, J Ma, J Cheng, M P Walsh, P Vogel, J Easton, G Song, M Rusch, A L Gedman, C Koss, J R Downing, T A Gruber
Acute megakaryoblastic leukemia in patients without Down syndrome is a rare malignancy with a poor prognosis. RNA sequencing of fourteen pediatric cases previously identified novel fusion transcripts that are predicted to be pathologic including CBFA2T3-GLIS2, GATA2-HOXA9, MN1-FLI, and NIPBL-HOXB9. In contrast to CBFA2T3-GLIS2 which is insufficient to induce leukemia, we demonstrate that the introduction of GATA2-HOXA9, MN1-FLI1 or NIPBL-HOXB9 into murine bone marrow induces overt disease in syngeneic transplant models...
February 8, 2017: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/28162930/a-randomized-study-of-autologous-bone-marrow-derived-stem-cells-in-pediatric-cardiomyopathy
#2
E Sian Pincott, Deborah Ridout, Margaret Brocklesby, Angus McEwan, Vivek Muthurangu, Michael Burch
BACKGROUND: Bone marrow mononuclear cell fraction has been used as therapy for dilated cardiomyopathy in adults. Although case series are reported, there are no randomized controlled studies in children. METHODS: We designed a randomized, crossover, controlled pilot study to determine safety and feasibility of intracoronary stem cell therapy in children. The primary safety end-point was freedom from death and transplantation or any complication that could be considered related to bone marrow injection or anesthesia (e...
January 5, 2017: Journal of Heart and Lung Transplantation
https://www.readbyqxmd.com/read/28115275/current-knowledge-and-priorities-for-future-research-in-late-effects-after-hematopoietic-cell-transplantation-hct-for-inherited-bone-marrow-failure-syndromes-ibmfs-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international
#3
REVIEW
Andrew C Dietz, Parinda A Mehta, Adrianna Vlachos, Sharon A Savage, Dorine Bresters, Jakub Tolar, Farid Boulad, Jean Hugues Dalle, Carmem Bonfim, Josu de la Fuente, Christine N Duncan, K Scott Baker, Michael A Pulsipher, Jeffrey M Lipton, John E Wagner, Blanche P Alter
Fanconi anemia (FA), dyskeratosis congenita (DC), and Diamond Blackfan anemia (DBA) are three of the most common inherited bone marrow failure syndromes (IBMFS), in which the hematologic manifestations can be cured with hematopoietic cell transplantation (HCT). Later in life these patients face a variety of medical conditions which may be a manifestation of underlying disease, may be due to their pre-HCT therapy, may be due to their HCT, or may be a combination of all these elements. Very limited long-term follow-up data exist in these populations, with FA the only IBMFS that has specific published data...
January 20, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28112743/fertility-preservation-practices-in-pediatric-and-adolescent-cancer-patients-undergoing-hsct-in-europe-a-population-based-survey
#4
T Diesch, A Rovo, N von der Weid, M Faraci, M Pillon, A Dalissier, J-H Dalle, P Bader
Chemotherapy and irradiation can affect the gonads, leading to impairment of pubertal development and/or infertility. Fertility preservation (FP) is therefore a crucial endeavor in hematopoietic stem cell transplantation (HSCT) because of the severe impact of infertility on the quality of life of long-term survivors. Despite the existence of different international guidelines, FP counseling and procedures are not routinely implemented as part of patient care. We present herein a survey conducted by the Pediatric Working Party of the European Society for Blood and Marrow Transplantation (EBMT), which aims to analyze and compare different FP practices for children and adolescents across EBMT centers in 2013...
January 23, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28100970/sickle-cell-disease-with-cyanotic-congenital-heart-disease-long-term-outcomes-in-5-children
#5
Glen J Iannucci, Olufolake A Adisa, Matthew E Oster, Michael McConnell, William T Mahle
Sickle cell disease is a risk factor for cerebrovascular accidents in the pediatric population. This risk is compounded by hypoxemia. Cyanotic congenital heart disease can expose patients to prolonged hypoxemia. To our knowledge, the long-term outcome of patients who have combined sickle cell and cyanotic congenital heart disease has not been reported. We retrospectively reviewed patient records at our institution and identified 5 patients (3 girls and 2 boys) who had both conditions. Their outcomes were uniformly poor: 4 died (age range, 12 mo-17 yr); 3 had documented cerebrovascular accidents; and 3 developed ventricular dysfunction...
December 2016: Texas Heart Institute Journal
https://www.readbyqxmd.com/read/28092353/non-myeloablative-conditioning-for-second-hematopoietic-cell-transplantation-for-graft-failure-in-patients-with-non-malignant-disorders-a-prospective-study-and-review-of-the-literature
#6
K Mallhi, P J Orchard, W P Miller, Q Cao, J Tolar, T C Lund
Allogeneic hematopoietic cell transplantation (HCT) effectively treats several non-malignant disorders such as selected lysosomal disorders, cerebral adrenoleukodystrophy and hemoglobinopathies. However, rates of graft failure (GF) in non-malignant populations exceed those of patients with malignant indications for HCT. Salvage conditioning regimens and outcomes for second HCT for GF vary immensely in the literature. We report 17 consecutive pediatric patients with non-malignant disorders who underwent a second allogenic HCT for GF using a non-myeloablative, low-dose busulfan-based regimen...
January 16, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28087884/the-role-of-immunoglobulin-prophylaxis-for-prevention-of-cytomegalovirus-infection-in-pediatric-hematopoietic-stem-cell-transplantation-recipients
#7
Gal Goldstein, Tal Frenkel Rutenberg, Sarina Levy Mendelovich, Daphna Hutt, Michal Teperberg Oikawa, Amos Toren, Bella Bielorai
BACKGROUND: Following cessation of intravenous immunoglobulin (IVIg) administration for allogeneic hematopoietic stem cell transplantation (HSCT) recipients at our unit, we observed a sharp decline in the incidence of cytomegalovirus (CMV) infection. PROCEDURE: We conducted a retrospective study of the role of IVIg in the prevention of CMV infection in children and young adults who underwent HSCT from matched related donor. RESULTS: We included 109 patients (IVIg+/IVIg- ratio 82/27)...
January 14, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28078809/three-year-analysis-of-repeated-laboratory-tests-for-the-markers-total-cholesterol-ferritin-vitamin-d-vitamin-b12-and-folate-in-a-large-research-and-teaching-hospital-in-italy
#8
Monica Lanzoni, Marco Fornili, Irene Felicetta, Rita Maiavacca, Elia Biganzoli, Silvana Castaldi
RATIONALE, AIMS AND OBJECTIVES: The increasing number of diagnostic tests requests all over the world is a problem that can partially be explained by inappropriate testing. Impact on the total costs of health systems becomes relevant when tests are performed in a large amount. In this paper, retesting of total cholesterol, ferritin, vitamin B12 , vitamin D, and folate is assessed. METHODS: The Quality Unit of Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano (Fondazione) decided to perform a first assessment of the appropriate use of the laboratory tests cholesterol, ferritin, vitamin B12 , vitamin D, and folate focusing on the retesting interval for the same patient in the time period January 1, 2012, to December 31, 2014, in every care setting...
January 12, 2017: Journal of Evaluation in Clinical Practice
https://www.readbyqxmd.com/read/28063963/timing-and-utility-of-relapse-surveillance-after-allogeneic-hematopoietic-cell-transplantation-in-children-with-leukemia
#9
Anya Levinson, Staci Arnold, Zhezhen Jin, Monica Bhatia, Diane George, James H Garvin, Prakash Satwani
The utility and optimal timing of routine bone marrow (BM) and cerebrospinal fluid (CSF) surveillance after allogeneic hematopoietic cell transplantation (alloHCT) in children with leukemia have not been previously studied. To examine the current practice concerning relapse surveillance in this population, we conducted a national survey of pediatric bone marrow transplant physicians. Sixty-two of 152 potential participants (41%) completed the survey. For acute lymphoblastic leukemia (ALL) patients, 41 physicians (66%) reported performing routine BM analysis in all such patients, 15 (24%) in some patients and 6 (10%) in no patients...
January 4, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28033481/fatal-scopulariopsis-brumptii-in-a-pediatric-immunocompromised-host
#10
Louise Helander, Matthew Stark
Scopulariopsis species cause a broad range of disease, from superficial skin infections to often fatal disseminated disease in the immunocompromised that is refractory to standard antifungal treatment. This report describes the first case of fatal disseminated Scopulariopsis brumptii in a pediatric patient with hyper-IgM syndrome status post bone marrow transplant.
December 29, 2016: Fetal and Pediatric Pathology
https://www.readbyqxmd.com/read/28027285/acute-rhinosinusitis-in-immunosuppressed-children-diagnostic-and-therapeutic-management
#11
Amit Ritter, Salvador Fischer, Eitan Yaniv, Tuvia Hadar, Ethan Soudry, Daniel Shai, Dror Gilony, Gideon Bachar
BACKGROUND: Immunosuppressive therapy places pediatric patients at risk of developing life-threatening sinonasal infections. Diagnosis and treatment is challenging owing to nonspecific signs and symptoms. The aim of the study was to present our department's experience with the surgical management of acute rhinosinusitis in immunosuppressed children. METHODS: The records of all children with a hematologic or oncologic disease who underwent endoscopic sinus surgery (ESS) for acute rhinosinusitis from January 2005 to May 2014 were reviewed...
December 23, 2016: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/28008770/the-budget-impact-and-cost-effectiveness-of-defibrotide-for-treatment-of-veno-occlusive-disease-with-multi-organ-dysfunction-in-patients-post-hematopoietic-stem-cell-transplant
#12
David L Veenstra, Gregory F Guzauskas, Kathleen F Villa, Denise M Boudreau
BACKGROUND: A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD). AIM: To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center. METHODS: We developed a budget impact model from the perspective of a bone-marrow transplant center...
December 23, 2016: Journal of Medical Economics
https://www.readbyqxmd.com/read/28007665/cd25-blockade-delays-regulatory-t-cell-reconstitution-and-does-not-prevent-graft-versus-host-disease-after-allogeneic-hematopoietic-cell-transplantation
#13
Frederick L Locke, Joseph Pidala, Barry Storer, Paul J Martin, Michael A Pulsipher, Thomas R Chauncey, Niels Jacobsen, Nicolaus Kröger, Irwin Walker, Susan Light, Bronwen E Shaw, Francisca Beato, Ginna G Laport, Auayporn Nademanee, Armand Keating, Gerard Socie, Claudio Anasetti
Daclizumab, a humanized monoclonal antibody, binds CD25 and blocks formation of the IL-2 receptor on T cells. A study of daclizumab as acute graft-versus-host disease (GVHD) prophylaxis after unrelated bone marrow transplantation was conducted before the importance of CD25(+)FOXP3(+) regulatory T cells (Tregs) was recognized. Tregs can abrogate the onset of GVHD. The relation between Tregs and a graft-versus-malignancy effect is not fully understood. An international, multicenter, double-blind clinical trial randomized 210 adult or pediatric patients to receive 5 weekly doses of daclizumab at 0...
December 19, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27994717/evaluation-and-management-of-osteochondral-lesions-of-the-talus
#14
REVIEW
Christopher A Looze, Jason Capo, Michael K Ryan, John P Begly, Cary Chapman, David Swanson, Brian C Singh, Eric J Strauss
Osteochondral lesions of the talus are common injuries that affect a wide variety of active patients. The majority of these lesions are associated with ankle sprains and fractures though several nontraumatic etiologies have also been recognized. Patients normally present with a history of prior ankle injury and/or instability. In addition to standard ankle radiographs, magnetic resonance imaging and computed tomography are used to characterize the extent of the lesion and involvement of the subchondral bone...
January 2017: Cartilage
https://www.readbyqxmd.com/read/27991893/cord-blood-transplantation-rewind-to-fast-forward
#15
REVIEW
A Dahlberg, F Milano
The utilization of cord blood as a source of stem cells for transplantation has decreased in recent years. Although cord blood transplantation (CBT) is an established practice for the treatment of adult and pediatric patients with hematological malignancies, the high acquisition cost of CB units along with high transplant-related mortality due to delayed hematopoietic recovery and immune reconstitution have contributed to the slowing in widespread adoption of CBT. Strategies aimed to enhance speed of engraftment and ongoing clinical trials are investigating ways to make CBT more widely available...
December 19, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27981925/comparison-of-presentation-and-outcome-in-100-pediatric-hodgkin-lymphoma-patients-treated-at-children-hospital-lahore-pakistan-and-royal-marsden-hospital-uk
#16
Mahwish Faizan, Mary M Taj, Saadia Anwar, Nyla Asghar, Alia Ahmad, Donna Lancaster, Ayad Atra, Agha Shabbir Ali
OBJECTIVE: To compare differences in demographics and outcomes in childhood Hodgkin lymphoma (HL) presenting at the Children's Hospital Lahore (CHL), and Royal Marsden Hospital (RMH), UK. STUDY DESIGN: An observational comparative study. PLACE AND DURATION OF STUDY: From January 2011 to February 2012 at CH, Lahore and from October 2008 to February 2012 at RMH, UK. METHODOLOGY: Consecutive HL patients (50 from each hospital) were inducted...
November 2016: Journal of the College of Physicians and Surgeons—Pakistan: JCPSP
https://www.readbyqxmd.com/read/27966803/yield-of-screening-echocardiograms-during-pediatric-follow-up-in-survivors-treated-with-anthracyclines-and-cardiotoxic-radiation
#17
Michael B Spewak, Rebecca S Williamson, Ann C Mertens, William L Border, Lillian R Meacham, Karen J Wasilewski-Masker
BACKGROUND: Guidelines published by the Children's Oncology Group recommend screening echocardiograms for childhood cancer survivors exposed to anthracyclines and/or cardiotoxic radiation. This study aims to assess risk factors for cardiac late effects while evaluating the overall yield of screening echocardiograms. PROCEDURE: Demographics, exposures, and echocardiogram results were abstracted from the medical records of survivors diagnosed at ≤ 21 years old and ≥ 2 years off therapy who were exposed to anthracyclines and/or potentially cardiotoxic radiotherapy...
December 14, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27941778/cytogenetics-and-outcome-of-allogeneic-transplantation-in-first-remission-of-acute-myeloid-leukemia-the-french-pediatric-experience
#18
A-L Alloin, G Leverger, J-H Dalle, C Galambrun, Y Bertrand, A Baruchel, A Auvrignon, V Gandemer, C Ragu, A Loundou, C Bilhou-Nabera, M Lafage-Pochitaloff, N Dastugue, B Nelken, C Jubert, F Rialland, G Plat, C Pochon, J-P Vannier, P-S Rohrlich, J Kanold, P Lutz, A Sirvent, C Oudin, W Cuccuini, G Michel
We analyzed the impact of cytogenetics on 193 children enrolled in two successive French trials (LAME89/91 and ELAM02), who received hematopoietic stem cell transplantation during CR1. Detailed karyotype was available for 66/74 (89%) in LAME89/91 and 118/119 (99%) in ELAM02. Several karyotype and transplant characteristics differed according to therapeutic protocol: unfavorable karyotypes were more frequent in ELAM02 (36% vs 18%), pretransplant chemotherapy included high-dose cytarabine in ELAM02 and not in LAME89/91, IV replaced oral busulfan in the conditioning regimen, methotrexate was removed from post-transplant immunosuppression, and matched unrelated donor and cord blood transplantation were introduced...
December 12, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27941773/haploidentical-transplantation-for-pediatric-patients-with-acquired-severe-aplastic-anemia
#19
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...
December 12, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27934561/various-regimens-of-total-body-irradiation-for-hematopoietic-stem-cell-transplant
#20
Naoya Ishibashi, Toshiya Maebayashi, Takuya Aizawa, Masakuni Sakaguchi, Osamu Abe, Kazuyoshi Sakanishi, Yuji Endoh, Tsutomu Saito, Jiro Kawamori, Yoshiaki Tanaka
OBJECTIVES: We aimed to evaluate the safety of total body irradiation before bone marrow transplant. MATERIALS AND METHODS: We analyzed 110 patients (65 male, 45 female) who underwent total body irradiation for hematopoietic stem cell transplant between May 1998 and March 2013. Median age at total body irradiation was 17 years (range, 1-62 y). Median observation time was 777 days (range, 31-5494 d). Initial diagnoses were acute lymphoblastic leukemia (24 patients), acute myeloid leukemia (26 patients), chronic myeloid leukemia (7 patients), myelodysplastic syndrome (8 patients), malignant lymphoma (13 patients), mucopolysaccharidosis (12 patients), neuroblastoma (10 patients), and other diseases (10 patients)...
December 2016: Experimental and Clinical Transplantation
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