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pediatric hematopoietic stem cell transplant

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https://www.readbyqxmd.com/read/28089099/clinical-characteristics-and-outcome-of-invasive-fungal-infections-in-pediatric-acute-myeloid-leukemia-patients-in-a-medical-center-in-taiwan
#1
Gu-Lung Lin, Hsiu-Hao Chang, Chun-Yi Lu, Chung-Ming Chen, Meng-Yao Lu, Ping-Ing Lee, Shiann-Tarng Jou, Yung-Li Yang, Li-Min Huang, Luan-Yin Chang
BACKGROUND: Invasive fungal infection (IFI) causes significant morbidity and mortality in patients with hematological malignancies, especially those with acute myeloid leukemia (AML), recurrent acute leukemia, high-risk acute lymphoblastic leukemia, and after allogeneic hematopoietic stem cell transplantation. The study aimed to investigate the clinical characteristics and outcome of IFIs in pediatric AML patients in a medical center in Taiwan. METHODS: We performed retrospective chart reviews...
December 19, 2016: Journal of Microbiology, Immunology, and Infection, Wei Mian Yu Gan Ran za Zhi
https://www.readbyqxmd.com/read/28088906/epratuzumab-and-blinatumomab-as-therapeutic-antibodies-for-treatment-of-pediatric-acute-lymphoblastic-leukemia-current-status-and-future-perspectives
#2
Raffaella Franca, Diego Favretto, Marilena Granzotto, Giuliana Decorti, Marco Rabusin, Gabriele Stocco
BACKGROUND: More than 85% of children affected by acute lymphoblastic leukemia (ALL) are successfully treated; however relapse remains a remarkable clinical concern, with 50-60% of relapsing patients facing a fatal outcome. Management of relapsed patients includes standardized intensive risk-adapted regimens based on conventional drugs, and hematopoietic stem cells transplantation for patients with unfavourable features. Biological drugs, in particular the monoclonal antibody epratuzumab and the bi-functional recombinant single chain peptide blinatumomab, have been recently recognized as novel potential agents to be integrated in salvage ALL therapy to further improve rescue outcome...
January 12, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/28087884/the-role-of-immunoglobulin-prophylaxis-for-prevention-of-cytomegalovirus-infection-in-pediatric-hematopoietic-stem-cell-transplantation-recipients
#3
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/28087458/medication-adherence-in-hematopoietic-stem-cell-transplant-a-review-of-the-literature
#4
REVIEW
Caroline F Morrison, Donna M Martsolf, Nicole Wehrkamp, Rebecca Tehan, Ahna L H Pai
Adherence to oral medications has been repeatedly shown to fall below the recommended 80-95% in pediatric and adult cancer populations. The purpose of this review is to report the state of the science about oral medication adherence during the acute phase of hematopoietic stem cell transplant across the lifespan. An exhaustive search of the literature yielded five records for inclusion in the review. Two studies examined adherence in pediatrics, two in adults, and one included both pediatric and adult patients...
January 10, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28068510/current-knowledge-and-priorities-for-future-research-in-late-effects-after-hematopoietic-stem-cell-transplantation-hct-for-severe-combined-immunodeficiency-patients-a-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international
#5
REVIEW
Jennifer Heimall, Jennifer Puck, Rebecca Buckley, Thomas A Fleisher, Andrew R Gennery, Benedicte Neven, Mary Slatter, Elie Haddad, Luigi D Notarangelo, K Scott Baker, Andrew C Dietz, Christine Duncan, Michael A Pulsipher, Mort J Cowan
Severe combined immunodeficiency (SCID) is 1 of the most common indications for pediatric hematopoietic cell transplantation (HCT) in patients with primary immunodeficiency. Historically, SCID was diagnosed in infants who presented with opportunistic infections within the first year of life. With newborn screening (NBS) for SCID in most of the United States, the majority of infants with SCID are now diagnosed and treated in the first 3.5 months of life; however, in the rest of the world, the lack of NBS means that most infants with SCID still present with infections...
January 6, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28065838/current-results-and-future-research-priorities-in-late-effects-after-hematopoietic-stem-cell-transplantation-hct-for-children-with-sickle-cell-disease-and-thalassemia-a-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international
#6
Shalini Shenoy, Emanuele Angelucci, Staci D Arnold, K Scott Baker, Monica Bhatia, Dorine Bresters, Andrew C Dietz, Josu De La Fuente, Christine Duncan, Javid Gaziev, Allison A King, Michael A Pulsipher, Angela Smith, Mark C Walters
Sustained donor engraftment after allogeneic hematopoietic cell transplantation (HCT) converts to healthy donor hemoglobin synthesis and halts disease symptoms in patients with sickle cell disease (SCD) and thalassemia major. A disease free survival probability that exceeds 90% has been reported when HCT using an HLA-matched sibling donor is performed in young patients with low risk disease or treatment related risk factors. Alternate donor HCT and HCT in adults is performed infrequently due to a higher risk profile...
January 5, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28060118/cytomegalovirus-treatment-in-pediatric-hematopoietic-stem-cell-transplant-patients
#7
Bethany A Wattles, Abby J Kim, Alexandra C Cheerva, Kenneth G Lucas, Joshua J Elder
Cytomegalovirus (CMV) is a frequent complication of hematopoietic stem cell transplant in pediatric patients, with significant morbidity and mortality. Antiviral drugs are used as prophylactic, preemptive or therapeutic medicines; however, no uniform guidelines exist for the best strategy to prevent CMV disease. Resistance to standard antiviral therapies can lead to further difficulty in managing CMV disease. Studies for investigational therapies are underway and could provide options for treatment of resistant CMV, while limiting toxicities associated with currently used antiviral therapies...
January 5, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28052585/diagnostic-yield-of-bronchoalveolar-lavage-in-immunocompromised-children-with-malignant-and-non-malignant-disorders
#8
Sruti Nadimpalli, Marc Foca, Prakash Satwani, Maria Luisa Sulis, Andrei Constantinescu, Lisa Saiman
BACKGROUND: The diagnostic yield of bronchoalveolar lavage (BAL) in the Immunocompromised pediatric population has ranged from 28% to 68%. We hypothesized that the diagnostic yield of BALs would be higher in more recent years due to new diagnostic assays. METHODS: A retrospective case series was performed among immunocompromised children ≤18 years old who underwent BALs from 2001 to 2012, to assess the yield of microbiologic diagnostic studies and to determine the impact of BAL findings on antimicrobial management...
January 3, 2017: Pediatric Pulmonology
https://www.readbyqxmd.com/read/28039955/mycobacterium-genavense-induced-spindle-cell-pseudotumor-in-a-pediatric-hematopoietic-stem-cell-transplant-recipient-case-report-and-review-of-the-literature
#9
Ritika Coelho, Rabi Hanna, Aron Flagg, Lisa M Stempak, Sarah Ondrejka, Gary W Procop, Susan Harrington, Anthony Zembillas, Karissa Kusick, Blanca E Gonzalez
We describe the first reported pediatric patient to our knowledge with a spindle cell pseudotumor caused by Mycobacterium genavense in a hematopoietic stem cell transplant recipient, and review the literature of such an entity in the transplant population. This article is protected by copyright. All rights reserved.
December 31, 2016: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/28039914/the-metabolic-syndrome-and-its-components-in-pediatric-survivors-of-allogeneic-hematopoietic-stem-cell-transplantation
#10
Bella Bielorai, Yael Weintraub, Daphna Hutt, Rina Hemi, Hannah Kanety, Dalit Modan-Moses, Gal Goldstein, Dana Hadar, Liat Lerner-Geva, Amos Toren, Orit Pinhas-Hamiel
MetS is a known complication after HSCT that contributes to long-term morbidity. We assessed the prevalence of components of the MetS in pediatric survivors of allogeneic HSCT, and identified associated risk factors. Thirty-eight patients, median age at HSCT, 8.5 years, were evaluated at a median of 3.9 years post HSCT. Overweight or obesity were seen in 23.7% of the patients, 15.8% had hypertension, 15.8% had hypertriglyceridemia and 13% had low HDL-C levels according to age and gender. Four (10.5%) met the criteria of MetS, all were transplanted for malignant disease...
December 31, 2016: Clinical Transplantation
https://www.readbyqxmd.com/read/28039080/risk-factors-for-and-the-clinical-impact-of-cytomegalovirus-and-epstein-barr-virus-infections-in-pediatric-recipients-of-tcr-%C3%AE-%C3%AE-and-cd19-depleted-grafts
#11
Alexandra Laberko, Anna Bogoyavlenskaya, Larisa Shelikhova, Zhanna Shekhovtsova, Dmitriy Balashov, Kirill Voronin, Elena Kurnikova, Elena Boyakova, Elena Raykina, Varvara Brilliantova, Valentina Pirumova, Galina Novichkova, Alexei Maschan, Michael Maschan
Both α/β T cell and CD19 depletion are used to improve the outcomes of hematopoietic stem cell transplantation (HSCT). We evaluated the burden of cytomegalovirus (CMV) and Epstein-Barr virus (EBV) in pediatric patients after this HSCT type. A cohort of 182 patients with malignant (n = 114) or nonmalignant (n = 68) disorders was transplanted from either matched unrelated (n = 124) or haploidentical (n = 58) donors. The cumulative incidence of CMV and EBV viremia were 51% and 33%, respectively...
December 27, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28030535/development-of-a-limited-sampling-strategy-for-the-estimation-of-exposure-to-high-dose-etoposide-after-intravenous-infusion-in-pediatric-patients
#12
Dorota Danielak, Joanna Sobiak, Jacek Wachowiak, Franciszek Główka, Maria Chrzanowska
BACKGROUND: Etoposide (VP-16), a podophylotoxin derivative, is used in conditioning regimens prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with acute lymphoblastic leukemia (ALL). The aim of this study was to develop a limited sampling strategy (LSS) suitable for the prediction of exposure to VP-16 defined as area-under-time-concentration curve (AUC). METHODS: The study included 28 pediatric patients with ALL, who were administered a 4h infusion of 60 mg/kg VP-16...
December 20, 2016: Therapeutic Drug Monitoring
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
#13
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/27991893/cord-blood-transplantation-rewind-to-fast-forward
#14
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/27987201/treatment-of-acute-lymphoblastic-leukemia-in-adults-applying-lessons-learned-in-children
#15
REVIEW
Ibrahim T Aldoss, Guido Marcucci, Vinod Pullarkat
Although pediatric acute lymphoblastic leukemia (ALL) has cure rates of over 90%, adult ALL remains a challenging disease to treat, with cure rates roughly half those seen in children. The inferior outcomes in adults can be attributed mainly to adverse genetic features, as well as the inability-particularly of older adults-to tolerate chemotherapy. Modest improvements have been seen in outcomes for adolescents and young adults; these can largely be attributed to the use of pediatric-type combination chemotherapy regimens in patients aged 50 years or younger...
December 15, 2016: Oncology (Williston Park, NY)
https://www.readbyqxmd.com/read/27960128/effect-of-donor-stat4-polymorphism-rs7574865-on-clinical-outcomes-of-pediatric-acute-leukemia-patients-after-hematopoietic-stem-cell-transplant
#16
Cheng Mun Wun, Zhe Piao, Kyung Taek Hong, Jung Yoon Choi, Che Ry Hong, June Dong Park, Kyung Duk Park, Hee Young Shin, Hyoung Jin Kang
STAT4 polymorphism, rs7574865 is linked to various autoimmune diseases such as systemic lupus erythematosus and rheumatoid arthritis. Its T minor allele is associated with higher STAT4 mRNA and protein expression, indicating a stronger skewed immune response than the norm. Although widely studied in autoimmune disease patients and the general population, its effect on immunocompromised subjects is still unknown. Especially in situations, i.e. post-hematopoietic stem cell transplantation (post-HSCT), where control of the immune response is crucial...
February 2017: International Immunopharmacology
https://www.readbyqxmd.com/read/27941778/cytogenetics-and-outcome-of-allogeneic-transplantation-in-first-remission-of-acute-myeloid-leukemia-the-french-pediatric-experience
#17
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
#18
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
#19
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
https://www.readbyqxmd.com/read/27932175/first-attempt-of-sequential-living-donor-liver-and-hematopoietic-stem-cell-transplantation-in-a-child-with-advanced-hepatocellular-carcinoma-case-report
#20
J A Picoraro, N Ovchinsky, M Martinez, S J Lobritto, P Satwani, R Ramphal, M S Cairo, T Kato
Effective therapeutic options for advanced hepatocellular carcinoma are limited. Hematopoietic stem cell transplantation may offer a graft-versus-tumor effect. Combined liver and hematopoietic stem cell transplantation from the same donor with preparatory conditioning may promote tolerogenicity to the liver allograft and offers the potential for immunosuppression withdrawal. We report our experience with the use of this approach in a pediatric patient with invasive hepatocellular carcinoma and pulmonary metastases who underwent a living-donor liver transplantation followed by reduced-toxicity myeloablative conditioning and hematopoietic stem cell transplant from the same parental donor...
November 2016: Transplantation Proceedings
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