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Pediatric Bone marrow transplantation

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https://www.readbyqxmd.com/read/29021921/post-transplant-lymphoproliferative-disorder-ptld-single-institutional-experience-of-141-patients
#1
Rohit Bishnoi, Ravneet Bajwa, Aaron J Franke, William Paul Skelton, Yu Wang, Niraj M Patel, William Birdsall Slayton, Fei Zou, Nam H Dang
BACKGROUND: Post-transplant lymphoproliferative disorder is a well-recognized but rare complication of hematopoietic stem cell and solid organ transplant. Due to rarity of this disease, retrospective studies from major transplant centers has been the main source to provide treatment guidelines, which are still in evolution. The sample size of this study is among one of the largest study on PTLD till date reported throughout the world. METHODS: This study was performed at University of Florida which is one of the largest transplant center in South East United States...
2017: Experimental Hematology & Oncology
https://www.readbyqxmd.com/read/28985203/cell-therapy-for-diverse-central-nervous-system-disorders-inherited-metabolic-diseases-and-autism
#2
REVIEW
Jessica M Sun, Joanne Kurtzberg
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplantation (HSCT) began with the development of bone marrow transplantation for hematologic malignancies in the mid-1900s and is now standard of care for many hematologic disorders. In the past few decades, HSCT has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products...
October 6, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28985199/renal-lineage-cells-as-a-source-for-renal-regeneration
#3
REVIEW
Oren Pleniceanu, Dorit Omer, Orit Harari-Steinberg, Benjamin Dekel
The mammalian kidney is a highly complex organ, composed of various cell types within a unique structural framework. Nonetheless, in recent years, giant leaps in our understanding of nephrogenesis and the origin of new cells in the adult kidney, have resulted in novel routes to regenerate damaged nephrons. While several strategies can be envisioned to achieve this aim, one common theme is the reliance on renal lineage cells, since extra-renal cells, such as bone-marrow derived cells, have been shown to be devoid of renal differentiation capacity...
October 6, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28945662/incidence-and-outcome-of-pericardial-effusion-in-pediatric-patients-after-hematopoietic-stem-cell-transplant-a-single-institution-experience
#4
Melissa Diamond, Catalina Ruiz-Mesa, Fernando F Corrales-Medina, Leonardo J Tamariz, Edward Ziga, Sethuraman Swaminathan
BACKGROUND: Pericardial effusion (PE) is a known complication after hematopoietic stem cell transplant (HSCT). Limited data is currently available regarding the incidence and outcomes of PE in pediatric HSCT. METHODS: We conducted a retrospective study on a cohort of patients who underwent HSCT between 2004 and 2015. Risk factors associated with development of PE were evaluated. RESULTS: In 111 HSCT, stem cell source was bone marrow in 37 (33...
September 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28932646/reconstitution-of-multifunctional-cd56-low-cd16-low-natural-killer-cell-subset-in-children-with-acute-leukemia-given-%C3%AE-%C3%AE-t-cell-depleted-hla-haploidentical-haematopoietic-stem-cell-transplantation
#5
Stabile Helena, Nisti Paolo, Peruzzi Giovanna, Fionda Cinzia, Pagliara Daria, Pomonia Brescia Letizia, Merli Pietro, Locatelli Franco, Angela Santoni, Angela Gismondi
We recently described the CD56(low)CD16(low) subset of Natural Killer (NK) cells that both mediate cytotoxic activity and produce IFNγ, being more abundant in bone marrow (BM) than in peripheral blood (PB) of pediatric normal subjects. Given the multifunctional properties of this subset, we examined its development and functional recovery in a cohort of children undergoing α/β T-cell depleted HLA-haploidentical haematopoietic stem cell transplantation (HSCT). The results obtained indicate that CD56(low)CD16(low) NK cells are present in both PB and BM already at one month post-HSCT, with an increased frequency in BM of graft recipients as compared with normal subjects...
2017: Oncoimmunology
https://www.readbyqxmd.com/read/28899741/small-bowel-stenosis-a-manifestation-of-chronic-graft-versus-host-disease-in-children
#6
Mickael Tordjman, Marie Ouachee, Arnaud Bonnard, Bogdana Tilea, Karima Yakouben, Jerome Viala, Michel Peuchmaur, Dominique Berrebi
Digestive graft versus host disease (GVHD) is a frequent complication after bone marrow transplantation (BMT), but small bowel obstruction is an extremely rare event. We present herein the first pediatric series of 4 cases of small bowel obstruction after BMT with detailed gross, histological data and their genetic status of the NOD2 gene. All patients had a history of severe acute GVHD treated by immunosuppressive agents and/or infliximab (in 3 cases). Acute or progressively worsening abdominal pain accompanied by small bowel occlusion occurred 5-16months after graft and CT-scan revealed multiple small intestinal stenosis...
September 9, 2017: Human Pathology
https://www.readbyqxmd.com/read/28883284/pediatric-acute-myeloid-leukemia-with-genetic-alterations
#7
Akira Shimada
Annually, it is estimated that approximately 150-200 children aged 0-16 years are diagnosed with acute myeloid leukemia (AML). In Japan, clinical studies with ANLL91, AML99, CCLSG-AML9805, and JPLSG-AML05 protocols were performed historically, and the risk stratification with a combination of chemotherapy and hematopoietic stem cell transplantation resulted in the improvement of clinical outcomes. Regarding the onset of pediatric AML at the molecular level, mutations in FLT3-ITD or KIT (Class I mutation) showed a poor prognosis, but the ratio of mutations in Class III-V genes was smaller than that in adult AML...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28853528/-neuro-oncology-a-new-field-in-davidoff-cancer-center-at-rabin-medical-center
#8
REVIEW
Shlomit Yust-Katz, Dror Limon, Ramez Abu-Shkara, Tali Siegal
Neuro-oncology is a subspecialty attracting physicians from medical disciplines such as neurology, neurosurgery, pediatrics, oncology, and radiotherapy. It deals with diagnosis and management of primary brain tumors, as well as metastatic and non-metastatic neurological manifestations that frequently affect cancer patients including brain metastases, paraneoplastic syndromes and neurological complications of cancer treatment. A neuro-oncology unit was established in Davidoff Cancer Center at Rabin Medical Center...
August 2017: Harefuah
https://www.readbyqxmd.com/read/28817182/centralized-patient-reported-outcome-data-collection-in-transplantation-is-feasible-and-clinically-meaningful
#9
Bronwen E Shaw, Ruta Brazauskas, Heather R Millard, Rachel Fonstad, Kathryn E Flynn, Amy Abernethy, Jenny Vogel, Charney Petroske, Deborah Mattila, Rebecca Drexler, Stephanie J Lee, Mary M Horowitz, J Douglas Rizzo
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) cures many patients, but often with the risk of late effects and impaired quality of life. The value of quantifying patient-reported outcomes (PROs) is increasingly being recognized, but the routine collection of PROs is uncommon. This study evaluated the feasibility of prospective PRO collection by an outcome registry at multiple time points from unselected HCT patients undergoing transplantation at centers contributing clinical data to the Center for International Blood and Marrow Transplant Research (CIBMTR), and then it correlated the PRO data with clinical and demographic data...
August 17, 2017: Cancer
https://www.readbyqxmd.com/read/28807769/reduced-intensity-haploidentical-bone-marrow-transplantation-with-post-transplant-cyclophosphamide-for-solid-tumors-in-pediatric-and-young-adult-patients
#10
Nicolas J Llosa, Kenneth R Cooke, Allen Chen, Christopher Gamper, Orly Klein, Elias Zambidis, Brandon Luber, Gary Rosner, Nicholas Siegel, Mary Jo Holuba, Nancy Robey, Masanori Hayashi, Richard J Jones, Ephraim Fuchs, Matthias Holdhoff, David M Loeb, Heather J Symons
High-risk, recurrent, or refractory solid tumors in pediatric, adolescent, and young adult (AYA) patients have an extremely poor prognosis despite current intensive treatment regimens. We piloted an allogeneic bone marrow transplant platform using reduced-intensity conditioning (RIC) and partially HLA-mismatched (haploidentical) related donors for this population of pediatric and AYA solid tumor patients. Sixteen patients received fludarabine, cyclophosphamide, melphalan, and low-dose total body irradiation RIC haploidentical BMT (haploBMT) followed by post-transplantation cyclophosphamide (PTCy), mycophenolate mofetil, and sirolimus...
August 12, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28797779/long-term-outcomes-of-cord-blood-transplantation-from-an-hla-identical-sibling-for-patients-with-bone-marrow-failure-syndromes-a-report-from-eurocord-cord-blood-committee-and-severe-aplastic-anemia-working-party-of-the-european-society-for-blood-and-marrow
#11
Simona Pagliuca, Régis Peffault de Latour, Fernanda Volt, Franco Locatelli, Marco Zecca, Jean-Hugues Dalle, Patrizia Comoli, Kim Vettenranta, Miguel Angel Diaz, Or Reuven, Yves Bertrand, Cristina Diaz de Heredia, Arnon Nagler, Ardeshir Ghavamzadeh, Sabina Sufliarska, Sarah Lawson, Chantal Kenzey, Vanderson Rocha, Carlo Dufour, Eliane Gluckman, Jakob Passweg, Annalisa Ruggeri
Cord blood transplantation (CBT) from HLA-identical siblings is an attractive option for patients with bone marrow failure (BMF) syndrome because of the low risk of graft-versus-host disease (GVHD) and the absence of risk to the donor. We analyzed outcomes of 117 patients with inherited or acquired BMF syndrome who received CBT from a related HLA-identical donor in European Society for Blood and Marrow Transplantation centers between 1988 and 2014. Ninety-seven patients had inherited and 20 patients acquired BMF syndrome...
August 7, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28783147/marrow-ablative-chemotherapy-followed-by-tandem-autologous-hematopoietic-cell-transplantation-in-pediatric-patients-with-malignant-brain-tumors
#12
J A Guerra, G Dhall, A Marachelian, E Castillo, J Malvar, K Wong, R Sposto, J L Finlay
To improve survival in young children with malignant brain tumors, irradiation-avoiding or -minimizing marrow-ablative chemotherapy (HDCx) with autologous hematopoietic cell transplantation (AuHCT) has been investigated. We evaluated the outcome of 44 children with malignant brain tumors treated with HDCx and tandem AuHCT at Children's Hospital Los Angeles between June 1999 and July 2012. Forty-four children with malignant brain tumors were studied. Twenty-one had medulloblastoma/primitive neuro-ectodermal tumor, eight atypical teratoid/rhabdoid tumor (ATRT), five high-grade glioma, four malignant germ cell tumor, three ependymoma and three choroid plexus carcinoma...
August 7, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28771903/fludarabine-cytarabine-g-csf-and-idarubicin-for-children-with-relapsed-aml
#13
Hideki Nakayama, Daisuke Tomizawa, Shiro Tanaka, Shotaro Iwamoto, Akira Shimada, Akiko M Saito, Yuka Yamashita, Hiroshi Moritake, Kiminori Terui, Takashi Taga, Hidemasa Matsuo, Yoshiyuki Kosaka, Katsuyoshi Koh, Hajime Hosoi, Hidemitsu Kurosawa, Keiichi Isoyama, Keizo Horibe, Shuki Mizutani, Souichi Adachi
BACKGROUND: The combination of fludarabine (Flu), high dose cytarabine (Ara-C) and granulocyte-colony stimulating factor (G-CSF) called "FLAG" with anthracyclines has become a standard chemotherapy for refractory acute myeloid leukemia (AML) in European children and adults. To clarify the efficacy and the safety of FLAG-idarubicin (IDA) for children prospectively, we planned a multicenter phase II study (AML-R11) by Japanese Pediatric Leukemia/Lymphoma Study Group. METHODS: Patients with AML at the age between 2 and 20 years old, who had the first bone marrow (BM) relapse or induction failure, were enrolled...
August 3, 2017: Pediatrics International: Official Journal of the Japan Pediatric Society
https://www.readbyqxmd.com/read/28759025/diagnosis-and-severity-criteria-for-sinusoidal-obstruction-syndrome-veno-occlusive-disease-in-pediatric-patients-a-new-classification-from-the-european-society-for-blood-and-marrow-transplantation
#14
S Corbacioglu, E Carreras, M Ansari, A Balduzzi, S Cesaro, J-H Dalle, F Dignan, B Gibson, T Guengoer, B Gruhn, A Lankester, F Locatelli, A Pagliuca, C Peters, P G Richardson, A S Schulz, P Sedlacek, J Stein, K-W Sykora, J Toporski, E Trigoso, K Vetteranta, J Wachowiak, E Wallhult, R Wynn, I Yaniv, A Yesilipek, M Mohty, P Bader
The advances in hematopoietic cell transplantation (HCT) over the last decade have led to a transplant-related mortality below 15%. Hepatic sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is a life-threatening complication of HCT that belongs to a group of diseases increasingly identified as transplant-related, systemic endothelial diseases. In most cases, SOS/VOD resolves within weeks; however, severe SOS/VOD results in multi-organ dysfunction/failure with a mortality rate >80%. A timely diagnosis of SOS/VOD is of critical importance, given the availability of therapeutic options with favorable tolerability...
July 31, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28741344/anidulafungin-in-children-experience-in-a-tertiary-care-children-s-hospital-in-argentina
#15
María T Rosanova, Claudia Sarkis, Florencia Escarra, Carolina Epelbaum, Norma Sberna, Susana Carnovale, Carlos Figueroa, Rosa Bologna, Roberto Lede
The experience using anidulafungin for the treatment of invasive fungal infections in pediatrics is limited. In this article, we describe our experience in 55 children. Anidulafungin was administered intravenously at a loading dose of 3 mg/kg once daily, followed by 1.5 mg/kg every 24 hours over a mean period of 14 days (range: 7-22 days). Patients' median age was 114 months old (interquartile range: 32-168 months old). All patients had underlying diseases. Among patients with bone marrow transplant, the difference in white blood cell count, transaminase levels, and renal function at baseline and at the end of anidulafungin administration was not significant...
August 1, 2017: Archivos Argentinos de Pediatría
https://www.readbyqxmd.com/read/28737775/fertility-preservation-issues-in-pediatric-hematopoietic-stem-cell-transplantation-practical-approaches-from-the-consensus-of-the-pediatric-diseases-working-party-of-the-ebmt-and-the-international-bfm-study-group
#16
A Balduzzi, J-H Dalle, K Jahnukainen, M von Wolff, G Lucchini, M Ifversen, K T Macklon, C Poirot, T Diesch, A Jarisch, D Bresters, I Yaniv, B Gibson, A M Willasch, R Fadini, L Ferrari, A Lawitschka, A Ahler, N Sänger, S Corbacioglu, M Ansari, R Moffat, A Dalissier, E Beohou, P Sedlacek, A Lankester, C D De Heredia Rubio, K Vettenranta, J Wachowiak, A Yesilipek, E Trigoso, T Klingebiel, C Peters, P Bader
Fertility preservation is an urgent challenge in the transplant setting. A panel of transplanters and fertility specialists within the Pediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation (EBMT) and the International BFM Study Group provides specific guidelines. Patients and families should be informed of possible gender- and age-specific cryopreservation strategies that should be tailored according to the underlying disease, clinical condition and previous exposure to chemotherapy...
October 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28708333/conversion-from-calcineurin-inhibitors-to-mtor-inhibitors-as-primary-immunosuppressive-drugs-in-pediatric-heart-transplantation
#17
Alfred Asante-Korang, Jennifer Carapellucci, Diane Krasnopero, Abigail Doyle, Brian Brown, Ernest Amankwah
There are only a few reports of successful use of mammalian target of rapamycin (mTORI) as primary immunosuppression in pediatric heart transplantation. Compared to calcineurin inhibitors, mTORI have less side effects, especially nephrotoxicity, infections, and malignancies. A retrospective study was conducted at our institution of all 170 heart transplants from 1995 to 2015. Nineteen patients were switched from tacrolimus (n=15) or cyclosporin (n=4) to everolimus (n=4) or sirolimus (n=15) due to nephrotoxicity (n=5), malignancy (n=8), EBV viremia/reactive plasmacytic changes (n=5), and immune hemolytic anemia (n=1)...
July 14, 2017: Clinical Transplantation
https://www.readbyqxmd.com/read/28696061/outcomes-and-healthcare-utilization-in-children-and-young-adults-with-aplastic-anemia-a-multiinstitutional-analysis
#18
Ashish Gupta, Pingfu Fu, Hasan Hashem, Anant Vatsayan, Steven Shein, Jignesh Dalal
BACKGROUND: Aplastic anemia is a bone marrow failure syndrome with high mortality affecting children and young adults. Although current treatment guidelines recommend hematopoietic stem cell transplant (HCT) for patients with matched sibling donors, outcomes with alternate donor options have been improving. PROCEDURE: We analyzed a validated multiinstitutional pediatric cohort using one of the largest pediatric and young adult database, the Pediatric Health Information System, for patients diagnosed with aplastic anemia (AA) from 2006 to 2015...
July 11, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28696051/unexpected-acute-renal-injury-after-high-dose-etoposide-phosphate-and-total-body-irradiation-in-children-undergoing-hematopoietic-stem-cell-transplantation
#19
C Cordero, C Loboda, I Clerc-Urmès, L Clément, C Pochon, P Chastagner
High-dose etoposide phosphate, a water-soluble prodrug of etoposide, may be used after total body irradiation (TBI) in pediatric allogeneic bone marrow transplantation for lymphoblastic leukemia. In a retrospective study of 21 children treated at the Nancy University Hospital (2000-2014), we identified unprecedentedly an unexpectedly high incidence (57%) of acute renal injury following etoposide phosphate infusion. Patients who developed renal function impairment experienced more severe mucositis but had outcomes similar to those who did not...
July 11, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28631412/psychopathology-in-pediatric-bone-marrow-transplantation-survivors-and-their-mothers
#20
Aslı Sürer Adanir, Gülseren Taşkiran, Osman Alphan Küpesiz, Esin Özatalay
BACKGROUND: Bone marrow transplantation (BMT) is used to treat various hematologic, oncologic and metabolic diseases. While the treatment is lifesaving, it is also associated with anxiety, post-traumatic stress disorder, depression and psychosocial problems both in children and parents. METHODS: The aim of this study was to investigate the psychopathology in pediatric BMT survivors and their mothers compared with healthy controls. All children were interviewed using Kiddie Schedule for Affective Disorders and Schizophrenia to assess lifelong psychopathology...
June 20, 2017: Pediatrics International: Official Journal of the Japan Pediatric Society
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