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bone marrow transplant pediatric

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https://www.readbyqxmd.com/read/29116687/erythropoietic-protoporphyria-in-an-adult-with-sequential-liver-and-hematopoietic-stem-cell-transplantation-a-case-report
#1
Annika L Windon, Rashmi Tondon, Nathan Singh, Samir Abu-Gazala, David L Porter, J Eric Russell, Colleen Cook, Elaine Lander, Georgeine Smith, Kim M Olthoff, Abraham Shaked, Maarouf Hoteit, Emma E Furth, Marina Serper
Erythropoieitic protoporphyria (EPP) is a rare inherited disorder of the heme biosynthesis pathway resulting in the accumulation of protoporphyrins in the blood, erythrocytes, and other tissues. Because of a gene mutation in the FECH gene, ferrochelatase, the enzyme involved in the final step of heme synthesis, is deficient in these patients. While the major symptom of this disorder is photosensitivity, rarely, it can cause progressive liver disease requiring liver transplantation (LT). However, LT is not curative and only bone marrow transplantation (BMT) can correct the underlying enzymatic defect...
November 8, 2017: American Journal of Transplantation
https://www.readbyqxmd.com/read/29115715/infusion-hemolysis-after-pediatric-major-abo-mismatched-bone-marrow-transplant-comparison-of-two-red-blood-cell-depletion-techniques
#2
Robert Sheppard Nickel, Muna Qayed, Diana Worthington-White, Sean R Stowell, Kuang-Yueh Chiang
BACKGROUND: During major ABO-mismatched bone marrow transplant (BMT), the infusion of incompatible red blood cells (RBCs) that are present in the bone marrow graft can cause adverse events from hemolysis. RBC depletion of the bone marrow graft can decrease this risk, but the optimal method to prevent hemolysis is unclear. PROCEDURE: We conducted a retrospective cohort study of patients who underwent major ABO-mismatched BMT at a pediatric center and had RBC depletion with either hydroxyethyl starch (HES) sedimentation or Ficoll density gradient separation...
November 8, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29099537/use-of-peripherally-inserted-central-venous-catheters-piccs-in-children-receiving-autologous-or-allogeneic-stem-cell-transplantation
#3
Stefano Benvenuti, Rosanna Ceresoli, Giovanni Boroni, Filippo Parolini, Fulvio Porta, Daniele Alberti
INTRODUCTION: The aim of our study was to present our experience with the use of peripherally inserted central catheters (PICCs) in pediatric patients receiving autologous or allogenic blood stem-cell transplantation. The insertion of the device in older children does not require general anesthesia and does not require a surgical procedure. METHODS: From January 2014 to January 2017, 13 PICCs were inserted as a central venous device in 11 pediatric patients submitted to 14 autologous or allogeneic stem-cell transplantation, at the Bone Marrow Transplant Unit of the Children's Hospital of Brescia...
October 31, 2017: Journal of Vascular Access
https://www.readbyqxmd.com/read/29050112/-clinical-pathologic-characteristics-and-treatment-outcomes-of-19-relapsed-pediatric-b-cell-lymphoma
#4
S Huang, L Jin, J Yang, Y L Duan, M Zhang, C J Zhou, X L Ma, Y H Zhang
Objective: To review the clinical-pathology characteristics of 19 relapsed pediatric mature B cell lymphoma and to find the risk factors for recurrence and the feasible treatment after relapse. Method: Data of 212 pediatric B cell lymphomas cases in Beijing Children's Hospital from January 2006 to June 2015 were collected retrospectively. All the patients were treated according to the B cell lymphoma regimen of Beijing Children's Hospital. During the study period, 19 of 212 cases were relapsed; the clinio-pathological characteristics of relapsed patients before treatment and after relapse were analyzed retrospectively, the treatment outcomes after relapse were summarized and the patients were followed-up...
October 2, 2017: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/29050111/-a-long-term-follow-up-report-of-pediatric-relapsed-wilms-tumor-after-retreatment
#5
T Y Wang, C Pan, Y J Gao, W T Hu, Q D Ye, M Zhou, J Y Tang
Objective: To investigate the long-term efficacy and prognostic factors of pediatric relapsed Wilms tumor (WT) after retreatment. Method: Sixteen children in Shanghai Children's Medical Center with relapsed Wilms tumor were enrolled consecutively in this study between April 2006 and June 2016. All patients were diagnosed according to pathology, imaging and medical and surgical oncologist's assistance. Relapse treatment included surgical excision, chemotherapy and selective radiation therapy. The clinical features, long-term outcomes and prognostic factors of patients were analyzed retrospectively...
October 2, 2017: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/29021921/post-transplant-lymphoproliferative-disorder-ptld-single-institutional-experience-of-141-patients
#6
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
#7
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
#8
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
#9
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
#10
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
#11
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
#12
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
#13
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
#14
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
#15
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
#16
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
#17
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...
November 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28771903/fludarabine-cytarabine-granulocyte-colony-stimulating-factor-and-idarubicin-for-relapsed-childhood-acute-myeloid-leukemia
#18
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; FLAG), with anthracyclines has become 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 the Japanese Pediatric Leukemia/Lymphoma Study Group. METHODS: Patients with AML aged between 2 and 20 years old, who had the first bone marrow (BM) relapse or induction failure, were enrolled...
October 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
#19
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
#20
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
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