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Pediatric allogenic stem cell transplant

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https://www.readbyqxmd.com/read/28429246/comparison-of-efficacy-and-safety-of-caspofungin-versus-micafungin-in-pediatric-allogeneic-stem-cell-transplant-recipients-a-retrospective-analysis
#1
Natalia Maximova, Giulia Schillani, Roberto Simeone, Alessandra Maestro, Davide Zanon
INTRODUCTION: The high morbidity and mortality associated with invasive fungal infections (IFIs) provide the rationale for antifungal prophylaxis in immuno-compromised pediatric patients undergoing hematopoietic stem cell transplantation (HSCT). Caspofungin and micafungin are antifungal agents of interest for prophylaxis of IFIs because of their potency against Candida and minimal toxicity or interactions with other drugs. Few studies have demonstrated the safety and efficacy of such echinocandins as prophylaxis for IFIs in patients undergoing HSCT...
April 20, 2017: Advances in Therapy
https://www.readbyqxmd.com/read/28424633/psychopathological-aspects-in-childhood-hematopoietic-stem-cell-transplantation-hsct-the-perception-of-parents-and-adolescents
#2
Silvia Zanato, Annalisa Traverso, Marta Tremolada, Francesco Sinatora, Alessio Porreca, Giorgio Pozziani, Nicoletta Di Florio, Fabia Capello, Antonio Marzollo, Manuela Tumino, Chiara Cattelan, Giuseppe Basso, Chiara Messina
Background: Data about psychosocial sequelae of childhood Hematopoietic Stem Cell Transplantation (HSCT) are limited and the association with a specific donor type or other medical factors is largely unknown (Chang et al., 2012). The aim of the present study was to compare the psychological aspects of pediatric HSCT survivors with healthy peers. A secondary aim was to detect whether parents and children differed in the perception of mental health status. The influence of medical factors on psychological status was also examined...
2017: Frontiers in Psychology
https://www.readbyqxmd.com/read/28396164/dose-escalation-of-total-marrow-irradiation-in-high-risk-patients-undergoing-allogeneic-hematopoietic-stem-cell-transplantation
#3
Susanta Hui, Claudio Brunstein, Yutaka Takahashi, Todd DeFor, Shernan G Holtan, Veronika Bachanova, Christopher Wilke, Darren Zuro, Celalettin Ustun, Daniel Weisdorf, Kathryn Dusenbery, Michael R Verneris
Patients with refractory leukemia or minimal residual disease (MRD) at transplant have increased risk of relapse. Augmentation of irradiation, especially to sites of disease (i.e., bone marrow) is one potential strategy to overcome this risk. We studied the feasibility of radiation dose escalation in high risk patients using total marrow irradiation (TMI) in a phase I dose-escalation trial. Four pediatric and 8 adult patients received conditioning with cyclophosphamide and fludarabine in conjunction with image-guided radiation to the bone marrow at 15 Gy and 18 Gy (in 3 Gy/fractions), while maintaining the total body irradiation (TBI) dose to the vital organs (lungs, hearts, eyes, liver, kidneys) at <13...
April 7, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28396162/phase-i-study-of-clofarabine-and-2-gy-total-body-irradiation-as-a-non-myeloablative-preparative-regimen-for-hematopoietic-stem-cell-transplantation-hsct-in-pediatric-patients-with-hematologic-malignancies-a-therapeutic-advances-in-childhood-leukemia-tacl-consortium
#4
Sandeep Soni, Hisham Abdel-Azim, Meghann McManus, Eneida Nemecek, Richard Sposto, Ann Woolfrey, Haydar Frangoul
Clofarabine is a purine nucleoside analog with immunosuppressive and anti-leukemic activity and its inclusion in reduced intensity regimens could potentially improve outcomes. We performed a prospective Phase I study of clofarabine combined with 2 Gy total body irradiation (TBI) as a non-myeloablative (NMA) preparative regimen for allogeneic stem cell transplantation in pediatric patients, who were considered at high risk of mortality from standard myeloablative regimens. The main goal of the study was to delineate the maximum feasible dose (MFD) of clofarabine in combination with 2 Gy TBI...
April 7, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28395444/-prognostic-value-of-dynamic-monitoring-of-runx1-runx1t1-transcript-in-pediatric-acute-myeloid-leukemia
#5
H T Gao, Y Zhang, K Sun, J M Guo, Y Q Chen, X L Chen, J Shi, X N Niu, F Wang, L Huo
Objective: To investigate the prognostic value of dynamic monitoring of RUNX1-RUNX1T1 transcript in pediatric patients with t (8;21) acute myeloid leukemia (AML) . Methods: The clinical features and RUNX1-RUNX1T1 transcript levels of 55 pediatric t (8;21) AML patients, newly diagnosed from Jan. 2010 to Apr. 2016, were analyzed retrospectively. The relationship between the minimal residual disease (MRD) and prognosis was analysed by dynamic monitoring of RUNX1-RUNX1T1 transcript levels using real-time quantitative PCR (RQ-PCR) technology...
March 14, 2017: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/28377772/treatment-of-pediatric-acute-graft-versus-host-disease-lessons-from-primary-immunodeficiency
#6
REVIEW
Aisling M Flinn, Andrew R Gennery
Allogeneic hematopoietic stem cell transplant (HSCT) is used to treat increasing numbers of malignant and non-malignant disorders. Despite significant advances in improved human leukocyte antigens-typing techniques, less toxic conditioning regimens and better supportive care, resulting in improved clinical outcomes, acute graft-versus-host disease (aGvHD) continues to be a major obstacle and, although it principally involves the skin, gastrointestinal tract, and liver, the thymus is also a primary target. An important aim following HSCT is to achieve complete and durable immunoreconstitution with a diverse T-cell receptor (TCR) repertoire to recognize a broad range of pathogens providing adequate long-term adaptive T-lymphocyte immunity, essential to reduce the risk of infection, disease relapse, and secondary malignancies...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/28370238/pediatric-patients-undergoing-hematopoietic-stem-cell-transplantation-can-greatly-benefit-from-a-novel-once-daily-intravenous-busulfan-dosing-nomogram
#7
Su-Jin Rhee, Ji Won Lee, Kyung-Sang Yu, Kyung Taek Hong, Jung Yoon Choi, Che Ry Hong, Kyung Duk Park, Hee Young Shin, Sang Hoon Song, Hyoung Jin Kang, Howard Lee
Busulfan, a bifunctional alkylating agent, has been used as a conditioning regimen prior to allogeneic hematopoietic stem cell transplantation (HSCT). The aims of this study were to derive a novel once-daily intravenous (IV) busulfan dosing nomogram for pediatric patients undergoing HSCT using a population pharmacokinetic (PK) model. A population PK analysis was performed using 2,183 busulfan concentrations in 137 pediatric patients (age: 0.6 - 22.2 years), who received IV busulfan once-daily for 4 days before undergoing HSCT...
March 28, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28368380/risk-of-melanocytic-nevi-and-nonmelanoma-skin-cancer-in-children-after-allogeneic-hematopoietic-stem-cell-transplantation
#8
J S Song, W B London, E B Hawryluk, D Guo, M Sridharan, D E Fisher, L E Lehmann, C N Duncan, J T Huang
There is a known increased risk of skin cancer in the adult population after hematopoietic stem cell transplantation (HSCT). However, late dermatologic effects that children may experience after HSCT have not been well described. The primary objective of this study was to characterize nevi and skin cancers affecting children after allogeneic HSCT. A cross-sectional cohort study of 85 pediatric HSCT recipients and 85 controls matched for age, sex and skin phototype was performed at a single institution. All participants underwent a full skin examination...
April 3, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28359910/cost-effectiveness-of-defibrotide-in-the-prophylaxis-of-veno-occlusive-disease-after-pediatric-allogeneic-stem-cell-transplantation
#9
Herbert Pichler, Karolina Horner, Gernot Engstler, Ulrike Poetschger, Evgenia Glogova, Susanne Karlhuber, Manuel Martin, Werner Eibler, Volker Witt, Wolfgang Holter, Susanne Matthes-Martin
Veno-occlusive disease (VOD) remains a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT). Prophylactic use of defibrotide (DF) might further reduce VOD rates but has no impact on the incidence of severe VOD or VOD-associated mortality. We investigated the cost-effectiveness of prophylactic DF according to the BCSH/BSBMT guidelines in 348 children who underwent transplantation between 2001 and 2013 in our hospital, 138 of whom were at risk for VOD. The VOD incidence was 7.4% for the total cohort...
March 27, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28353017/treatment-of-young-adults-with-acute-lymphoblastic-leukemia
#10
REVIEW
Ankit Kansagra, Mark Litzow
Young adults with acute lymphoblastic leukemia are a distinctive category of patients, with substantial difference in disease biology and response to therapy; hence, they pose unique challenges and issues beyond those faced by children and older adults. Despite inferior survival compared to children, there is growing evidence to suggest that young adults have improved outcomes when treated with pediatric-based approaches. With better supportive care and toxicity management and multidisciplinary team and approach, we have made great improvement in outcomes of young adults with ALL...
March 28, 2017: Current Hematologic Malignancy Reports
https://www.readbyqxmd.com/read/28350290/hla-genotypes-in-turkish-hematopoietic-cell-recipients-and-likelihood-of-finding-a-matched-donor-through-family-searches
#11
Zühre Kaya, Sevim Gönen, Burcu Çalışkan, Zeynep Kemer, Ayşe Betül Ünal, Enes Değirmenci
OBJECTIVES: Allogeneic hematopoietic stem cell transplant is a life-saving treatment, but donor numbers in Turkey do not meet the increasing demand for this procedure. Here, our objectives were (1) to assess the frequency of HLA-matched related donors in the Turkish population and (2) to identify the HLA antigens and haplotypes that are most frequent in Turkey. MATERIALS AND METHODS: The HLA genotypes of 841 consecutive recipients and 3071 family members were retrospectively reviewed...
March 28, 2017: Experimental and Clinical Transplantation
https://www.readbyqxmd.com/read/28344057/ruxolitinib-as-salvage-therapy-in-steroid-refractory-acute-graft-versus-host-disease-in-pediatric-hematopoietic-stem-cell-transplant-patients
#12
Pooja Khandelwal, Ashley Teusink-Cross, Adam S Nelson, Christopher E Dandoy, Javier El-Bietar, Rebecca A Marsh, Ashish R Kumar, Michael S Grimley, Stella M Davies, Sonata Jodele, Kasiani C Myers
We describe our retrospective clinical experience with ruxolitinib for steroid-refractory acute graft versus host disease (GVHD) in pediatric allogeneic hematopoietic stem cell transplant (HSCT) patients. Ruxolitinib was administered orally at 5 mg twice daily for children ≥ 25 kg or 2.5 mg twice daily if < 25 kg. We excluded patients who received new immune suppressive agents within two weeks before initiation of ruxolitinib from response analysis. Patients were called a treatment failure if ruxolitinib was stopped before completion of 4 weeks of therapy due to adverse effects and not because of progression of acute GVHD...
March 23, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28295914/development-of-a-gastric-carcinoid-tumor-following-allogeneic-hematopoietic-stem-cell-transplantation-for-early-t-cell-precursor-acute-lymphoblastic-leukemia
#13
Tsung-Yen Chang, Jin-Yao Lai, Chao-Jan Wang, Shih-Yen Chen, Tang-Her Jaing, Chuen Hsueh, Lee-Yung Shih, Shih-Hsiang Chen
Gastric carcinoid tumor is rarely diagnosed in children. We report a case of gastric carcinoid tumor that occurred after allogeneic HSCT. A 13-year-old girl with ETP acute lymphoblastic leukemia underwent allogeneic HSCT from a 7/8 HLA-matched unrelated donor. She presented with rashes, abdominal pain, and diarrhea, which were suggestive of GVHD, 7 months after HSCT. Immunosuppressive agents failed to resolve these symptoms well. After a series of evaluations, carcinoid syndrome caused by a gastric carcinoid tumor was diagnosed...
March 11, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28271997/a-retrospective-study-of-clinical-profile-and-long-term-outcome-to-imatinib-mesylate-alone-in-childhood-chronic-myeloid-leukemia-in-chronic-phase
#14
(no author information available yet)
OBJECTIVE: Chronic myeloid leukemia (CML) is relatively rare malignancy in childhood. There are limited studies of use of Imatinib Mesylate (IM) alone in management of CML in this age group. METHOD: We retrospectively analyzed the outcome of 30 consecutive children with CML chronic phase treated with IM alone. RESULTS: The median age at the time of diagnosis was 11 years with male preponderance. Asthenia and abdominal discomfort due to splenomegaly were the most common presenting features and splenomegaly a dominant sign...
January 2017: Gulf Journal of Oncology
https://www.readbyqxmd.com/read/28271813/immune-response-to-exercise-during-growth
#15
Shlomit Radom-Aizik
Two papers were selected for this commentary. The first paper (Citation 1) suggests that a 10-week, moderate-intensity exercise program performed early after allogeneic hematopoietic stem cell transplantation is feasible in this fragile population, and might improve cell cytotoxicity by redistributing subpopulations of NK cells. This study adds to the growing evidence that enhancing immune cell surveillance (e.g., NK cells) in response to exercise could benefit cancer patients. The second paper (Citation 2) studied neutrophil-related mediators of oxidative stress and inflammatory cytokines in response to exercise in children compared with adults...
February 2017: Pediatric Exercise Science
https://www.readbyqxmd.com/read/28267083/toxic-epidermal-necrolysis-like-reaction-after-hematopoietic-stem-cell-transplantation-in-children
#16
Maura Faraci, Stefano Giardino, Edoardo Lanino, Giuseppe Morreale, Emilia Ghibaudo, Monica Francesia Berta, Marco Risso, Elio Castagnola, Mimmo Ripaldi, Andrea Moscatelli, Giovanni Ghigliotti
This study report clinical course, etiology, management, and long-term outcome of children who developed toxic epidermal necrolysis-like reaction (TEN-LR) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). We retrospectively collected children with TEN-LR occurring after allo-HSCT performed in 2 pediatric bone marrow units between 2005 and 2014. We identified 6 cases of TEN-LR of 322 patients (1.8%). Possible triggers of TEN included antibiotics, antiepileptics, antimycotics, and Mycoplasma infection...
May 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28239380/development-of-three-different-nk-cell-subpopulations-during-immune-reconstitution-after-pediatric-allogeneic-hematopoietic-stem-cell-transplantation-prognostic-markers-in-gvhd-and-viral-infections
#17
Sabine Huenecke, Claudia Cappel, Ruth Esser, Verena Pfirrmann, Emilia Salzmann-Manrique, Sibille Betz, Eileen Keitl, Julia Banisharif-Dehkordi, Shahrzad Bakhtiar, Christoph Königs, Andrea Jarisch, Jan Soerensen, Evelyn Ullrich, Thomas Klingebiel, Peter Bader, Melanie Bremm
Natural killer (NK) cells play an important role following allogeneic hematopoietic stem cell transplantation (HSCT) exerting graft-versus-leukemia/tumor effect and mediating pathogen-specific immunity. Although NK cells are the first donor-derived lymphocytes reconstituting post-HSCT, their distribution of CD56(++)CD16(-) (CD56(bright)), CD56(++)CD16(+) (CD56(intermediate=int)), and CD56(+)CD16(++) (CD56(dim)) NK cells is explicitly divergent from healthy adults, but to some extent comparable to the NK cell development in early childhood...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/28224776/clinical-usefulness-of-monitoring-cytomegalovirus-specific-immunity-by-quantiferon-cmv-in-pediatric-allogeneic-hematopoietic-stem-cell-transplantation-recipients
#18
Sae Mi Lee, Yae Jean Kim, Keon Hee Yoo, Ki Woong Sung, Hong Hoe Koo, Eun Suk Kang
Cytomegalovirus (CMV) is a well-established cause of morbidity and mortality in pediatric recipients of allogeneic hematopoietic stem cell transplantation (allo-HSCT). CD8⁺ T-cells are important for controlling CMV infection. We conducted a prospective pilot study to investigate the clinical utility of measuring the CMV-specific T-cell immune response using the QuantiFERON-CMV assay (QF-CMV) in pediatric allo-HSCT recipients. Overall, 16 of 25 (64%) patients developed CMV infection. QF-CMV was evaluated in these 16 patients during the early and late phases of the first CMV infection post allo-HSCT...
May 2017: Annals of Laboratory Medicine
https://www.readbyqxmd.com/read/28218757/allogeneic-stem-cell-transplantation-for-refractory-acute-myeloid-leukemia-in-pediatric-patients-the-uk-experience
#19
P O'Hare, G Lucchini, M Cummins, P Veys, M Potter, S Lawson, A Vora, R Wynn, A Peniket, K Kirkland, R Pearce, J Perry, P J Amrolia
We report outcomes for 44 children who underwent stem cell transplantation (SCT) for refractory AML in the UK between 2000 and 2012. Median age at SCT was 11.5 years. Twenty-three patients had primary refractory and 21 relapsed refractory AML. Refractory disease was confirmed by cytogenetics/molecular genetics in 24 cases. Median follow-up of the whole cohort is 6.8 years (2.1-14.9 years). Thirty patients (68%) achieved a CR following SCT. Transplant-related mortality at 1 year was 18%. Acute GVHD incidence was 52% (grade ⩾III 19%), chronic 7%...
February 20, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28212370/the-evolution-of-allogeneic-stem-cell-transplant-for-children-and-adolescents-with-acute-myeloid-leukemia
#20
Allyson Flower, Mitchell S Cairo
Survival rates in subsets of pediatric patients who have acute myeloid leukemia (AML) with favorable risk features are now greater than 90%. However, outcomes for patients with high-risk (HR) features remain unacceptably poor. As novel technologies for the identification of HR biomarkers and the detection of residual disease are developed, risk stratification and the application of allogeneic hematopoietic stem cell transplant (HSCT) are evolving. HSCT has been shown to benefit subpopulations of pediatric patients with AML, including those with HR cytogenetic translocations, genetic mutations, and/or residual disease after induction...
January 2017: Clinical Advances in Hematology & Oncology: H&O
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