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pediatric hematopoietic stem cell transplant

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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/28991134/isolated-testicular-recurrence-of-aml-in-patients-with-chronic-gvhd-1-year-following-allogeneic-stem-cell-transplant
#2
Brian N Dang, Satiro De Oliveira, LaVette Bowles, Theodore B Moore
BACKGROUND: Patients with chronic graft-versus-host disease (cGVHD) following allogeneic transplant for myeloid leukemias seem to experience a reduced risk of relapse than comparable patients without cGVHD. It is unclear to what extent extramedullary sites are impacted by a graft-versus-leukemia effect. DESIGN/METHOD: Case Series and review of the literature. RESULTS: We present 2 cases of pediatric patients with Acute Myelogenous Leukemia who developed isolated testicular relapse more than a year following hematopoietic stem cell transplantation despite having had extensive cGVHD...
October 4, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28988422/comparison-of-two-cytoreductive-regimens-for-%C3%AE-%C3%AE-t-cell-depleted-haploidentical-hsct-in-pediatric-malignancies-improved-engraftment-and-outcome-with-tbi-based-regimen
#3
Elad Jacoby, Nira Varda-Bloom, Gal Goldstein, Daphna Hutt, Chaim Churi, Helly Vernitsky, Amos Toren, Bella Bielorai
BACKGROUND: Graft manipulation using selective depletion of αβ-T cells provides a source of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) enriched in effector cells. We report our experience implementing this haplo-HSCT for high-risk malignancies in pediatric patients focusing on the conditioning regimen. PROCEDURE: We performed a retrospective study of patients who underwent T-cell receptor αβ-depleted haplo-HSCT for high-risk pediatric malignancies...
October 8, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28985203/cell-therapy-for-diverse-central-nervous-system-disorders-inherited-metabolic-diseases-and-autism
#4
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/28984677/advances-in-the-understanding-and-management-of-mucositis-during-stem-cell-transplantation
#5
Joanne M Bowen, Hannah R Wardill
PURPOSE OF REVIEW: Mucositis is a severe and common side effect of anticancer treatments, with an incidence of between 40 and 80% depending on the cytotoxic regimen used. The most profound mucositis burden is experienced during conditioning regimens for hematopoietic stem cell transplant (HSCT), where the use of highly mucotoxic agents with or without total body irradiation leads to serious damage throughout the alimentary tract. Currently, the assessment and management of both oral and gastrointestinal mucositis lack authoritative guideline, with recommendations only achieved in narrow clinical scenarios...
October 3, 2017: Current Opinion in Supportive and Palliative Care
https://www.readbyqxmd.com/read/28975382/population-pharmacokinetics-of-treosulfan-and-development-of-a-limited-sampling-strategy-in-children-prior-to-hematopoietic-stem-cell-transplantation
#6
Dorota Danielak, Jadwiga Twardosz, Anna Kasprzyk, Jacek Wachowiak, Krzysztof Kałwak, Franciszek Główka
PURPOSE: There is an increasing interest in use of treosulfan (TREO), a structural analogue of busulfan, as an agent in conditioning regimens prior to hematopoietic stem cell transplantation (HSCT), both in pediatric and adult populations. The aim of this study was to develop a population pharmacokinetic model and to establish limited sampling strategies (LSSs) enabling accurate estimation of exposure to this drug. METHODS: The study included 15 pediatric patients with malignant and non-malignant diseases, undergoing conditioning regimens prior to HSCT including TREO administered as a 1 h or 2 h infusion at daily doses of 10, 12, or 14 g/m(2)...
October 3, 2017: European Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/28973362/cd20-positivity-and-white-blood-cell-count-predict-treatment-outcomes-in-philadelphia-chromosome-negative-acute-lymphoblastic-leukemia-patients-ineligible-for-pediatric-inspired-chemotherapy
#7
Yusuke Isshiki, Chikako Ohwada, Emiko Sakaida, Masahiro Onoda, Nobuyuki Aotsuka, Hiroaki Tanaka, Motoharu Fukazawa, Ryuko Cho, Takeaki Sugawara, Takeharu Kawaguchi, Satoru Hara, Akira Yokota
Background: The efficacy of conventional chemotherapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been controversial as post-remission therapies for adult Philadelphia chromosome-negative acute lymphoblastic leukemia patients. Methods: We retrospectively analyzed 96 adolescent and adult cases of Philadelphia chromosome-negative acute lymphoblastic leukemia to evaluate whether allo-HSCT should be performed after first complete remission (1CR)...
September 8, 2017: Japanese Journal of Clinical Oncology
https://www.readbyqxmd.com/read/28968980/safety-and-tolerability-of-deferasirox-in-pediatric-hematopoietic-stem-cell-transplant-recipients-one-facility-s-five-years-experience-of-chelation-treatment
#8
Natalia Maximova, Massimo Gregori, Roberto Simeone, Aurelio Sonzogni, Giulia Boz, Carmen Fucile, Valeria Marini, Antonietta Martelli, Francesca Mattioli
42 pediatric patients with iron overload, who underwent liver biopsy and DFX treatment after hematopoietic stem cell transplantation were included in the study group. The patients were divided into two groups diversified according to deferasirox trough plasma concentrations (DFX Ctrough) with cut-off equal to10 mcg/mL. The average dose of DFX was 25.9 mg/kg in the DFX Ctrough < 10 mcg/mL group versus 19.2 mg/kg in the DFX Ctrough > 10 mcg/mL group (p=0,0003). The mean duration of DFX treatment was 135...
September 8, 2017: Oncotarget
https://www.readbyqxmd.com/read/28960811/vitamin-d-deficiency-and-outcomes-in-pediatric-hematopoietic-stem-cell-transplantation
#9
Kristen Beebe, Kyrie Magee, Annmarie McNulty, Jennifer Stahlecker, Dana Salzberg, Holly Miller, Lucia Mirea, Roberta Adams, Alexander Ngwube
BACKGROUND: Pediatric patients undergoing hematopoietic stem cell transplantation (HSCT) are frequently diagnosed with vitamin D deficiency, which may impact outcomes. OBJECTIVES: To estimate the prevalence of vitamin D deficiency and examine its association with short-term survival in pediatric HSCT patients. METHODS: Patients undergoing HSCT at Phoenix Children's Hospital were retrospectively identified. Routine serum 25-hydroxyvitamin D measurements were described prior to transplant and at 100 days and 1-year post-HSCT...
September 27, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28959694/use-of-rapamycin-in-a-patient-with-juvenile-myelomonocytic-leukemia-a-case-report
#10
Shivani Y Upadhyay, Satiro N De Oliveira, Theodore B Moore
The relapse rate for children with juvenile myelomonocytic leukemia (JMML) status post hematopoietic stem cell transplantation (HSCT) approaches 50% within 5 years. Graft-versus-leukemia (GVL) is thought to play important role in the treatment of JMML. For this reason, careful management of immunosuppressive drugs after HSCT is crucial. This case report demonstrates that rapamycin and GVL represent a viable medical strategy for the management of pediatric patients with JMML who relapse following status post-HSCT...
July 2017: Journal of Investigative Medicine High Impact Case Reports
https://www.readbyqxmd.com/read/28958895/young-female-donors-do-not-increase-risk-of-gvhd-or-impact-overall-outcomes-in-pediatric-hla-matched-sibling-hsct
#11
Paola Friedrich, Pilar Guerra-Garcia, Alyssa Stetson, Christine Duncan, Leslie Lehmann
Optimal donor selection is critical in hematopoietic stem-cell transplantation (HSCT). Donor/recipient sex-mismatch, donor age and female donor/donor parity are known to impact graft-versus-host disease (GVHD) and outcomes in adults. Minor histocompatibility antigens (mHCA) encoded by the human Y-chromosome (H-Y) can result in specific antibody formation in some female donors, may increase in frequency with increasing donor age and may be contributory to the increased incidence of GVHD. To better understand the role of donor age/sex and sex matching in HSCT outcomes, we conducted a retrospective study of pediatric patients receiving their first myeloablative sibling-donor HSCT (n=244) from 1998 to 2012...
September 25, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28958195/pulmonary-veno-occlusive-disease-in-a-pediatric-hematopoietic-stem-cell-transplant-patient-a-cautionary-tale
#12
M S Zinter, A Melton, A J Sabnis, C C Dvorak, B M Elicker, H M Nawaytou, R J Kameny, J R Fineman
No abstract text is available yet for this article.
September 28, 2017: Leukemia & Lymphoma
https://www.readbyqxmd.com/read/28951259/chimerism-in-pediatric-hematopoietic-stem-cell-transplantation-and-its-correlation-with-the-clinical-outcome
#13
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/28945662/incidence-and-outcome-of-pericardial-effusion-in-pediatric-patients-after-hematopoietic-stem-cell-transplant-a-single-institution-experience
#14
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/28945115/advances-in-immunotherapy-for-pediatric-acute-myeloid-leukemia
#15
Challice L Bonifant, Mireya Paulina Velasquez, Stephen Gottschalk
Achieving better disease control in patients diagnosed with acute myeloid leukemia (AML) has proven challenging. Overall survival has been impacted by addressing treatment related mortality with focused supportive care measures. Despite this improvement, it remains difficult to induce durable leukemia remissions despite aggressive chemotherapeutic regimens. The addition of hematopoietic stem cell transplants (HSCT) has allowed further treatment intensification and provided the benefit of graft-versus-leukemia (GVL) effect...
October 11, 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/28939456/combination-of-high-dose-methylprednisolone-and-defibrotide-for-veno-occlusive-disease-in-pediatric-hematopoietic-stem-cell-transplant-recipients
#16
Nicholas J Gloude, Sonata Jodele, Ashley Teusink-Cross, Michael Grimley, Stella M Davies, Adam Lane, Kasiani C Myers
Veno-occlusive disease (VOD) is a serious complication of hematopoietic stem cell transplant (HSCT), with high mortality in severe cases, and until recently very limited therapeutic options, consisting largely of supportive care. Defibrotide was recently approved in the United States for the treatment of severe VOD in patients with renal or pulmonary dysfunction following HSCT. Our group previously published on the use of high-dose methylprednisolone (500 mg/m(2)/dose every 12 hours for 6 doses) in patients with VOD showing good success...
September 19, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28934891/quality-of-life-among-boys-with-adrenoleukodystrophy-following-hematopoietic-stem-cell-transplant
#17
Nicole B Beckmann, Weston P Miller, Mary S Dietrich, Paul J Orchard
Hematopoietic stem cell transplant (HSCT) is the only accepted treatment capable of halting the progression of X-linked cerebral adrenoleukodystrophy (CALD). While survival and neurological outcomes have been described, there is little information regarding the quality of life (QoL) of transplanted patients with CALD. This analysis is a cross-sectional study of QoL in 16 males diagnosed with CALD who underwent HSCT at a single institution. Each child or parent proxy completed subscales from the Neuro-QoL and the PROMIS Pediatric Profile Instrument representing physical, mental, and social health domains...
September 21, 2017: Child Neuropsychology: a Journal on Normal and Abnormal Development in Childhood and Adolescence
https://www.readbyqxmd.com/read/28927202/role-of-molecular-biomarkers-in-the-diagnosis-of-invasive-fungal-diseases-in-children
#18
Anna R Huppler, Brian T Fisher, Thomas Lehrnbecher, Thomas J Walsh, William J Steinbach
Invasive fungal diseases are important clinical problems that are often complicated by severe illness and therefore the inability to use invasive measures to definitively diagnose the disease. Tests for a range of fungal biomarkers that do not require an invasive sample-collection procedure have been incorporated into adult clinical practice, but pediatric data and pediatric-specific recommendations for some of these diagnostic tools are lacking. In this review, we summarize the published literature and contemporary strategies for using the biomarkers galactomannan, (1→3)-β-d-glucan, Candida mannan antigen and anti-mannan antibody, and fungal polymerase chain reaction for diagnosing invasive fungal disease in children...
September 1, 2017: Journal of the Pediatric Infectious Diseases Society
https://www.readbyqxmd.com/read/28927200/epidemiology-of-invasive-fungal-disease-in-children
#19
Zoi Dorothea Pana, Emmanuel Roilides, Adilia Warris, Andreas H Groll, Theoklis Zaoutis
Considerable progress has been made in the prevention, diagnosis, and management of pediatric patients with invasive fungal disease (IFD). The reported decreasing trend in the incidence of invasive candidiasis (IC) over the past 15 years in both neonates and children has been encouraging. Nevertheless, due to the growing number of immunocompromised children at risk for IFD, this disease continues to be associated with significant morbidity and death and with increased financial burden to the health care system...
September 1, 2017: Journal of the Pediatric Infectious Diseases Society
https://www.readbyqxmd.com/read/28921804/early-blood-stream-infection-following-allogeneic-hematopoietic-stem-cell-transplantation-is-a-risk-factor-for-acute-grade-iii-iv-gvhd-in-children-and-adolescents
#20
Hirozumi Sano, Joseph A Hilinski, Muna Qayed, Kristy Applegate, Joanna G Newton, Benjamin Watkins, Kuang-Yueh Chiang, John Horan
BACKGROUND: Graft-versus-host disease (GVHD) remains a major cause of mortality and morbidity in allogeneic hematopoietic stem cell transplantation (HSCT). In adults, early blood stream infection (BSI) and acute GVHD (AGVHD) have been reported to be related. The impact of BSI on risk for AGVHD, however, has not been assessed in pediatric patients. PROCEDURE: We conducted a retrospective analysis to test the hypothesis that early BSI (before day +30) predisposes allogeneic pediatric transplant patients to severe AGVHD...
September 17, 2017: Pediatric Blood & Cancer
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