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pediatric hematopoietic 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/28407118/voriconazole-dosing-in-children-younger-than-3-years-undergoing-cancer-chemotherapy-or-hematopoietic-stem-cell-transplantation
#3
Shirley Qiong Yan, Brian Seyboth, Rachel Kobos, Anne Eaton, Susan K Seo, Nina Cohen
There are limited pediatric population pharmacokinetic data for voriconazole dosing, particularly in younger children. In a cohort of 34 patients younger than 3 years receiving voriconazole, the majority (n = 23, 68%) had a low initial serum concentration <1 mg/L. Among 23 children <2 years old, 19 (83%) had an initial trough <1 mg/L. There was large intra- and interindividual variability in trough levels. Dosing also varied from 3.3 to 19.6 mg/kg per dose. Only 2 of 34 patients had a documented adverse drug reaction attributable to voriconazole...
April 12, 2017: Journal of the Pediatric Infectious Diseases Society
https://www.readbyqxmd.com/read/28396164/dose-escalation-of-total-marrow-irradiation-in-high-risk-patients-undergoing-allogeneic-hematopoietic-stem-cell-transplantation
#4
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
#5
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
#6
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/28392150/factual-reflections-and-recommendations-on-extracorporeal-photopheresis-in-pediatrics
#7
REVIEW
Irena Sniecinski, Jerard Seghatchian
One of the biggest challenges in evaluating available literature on extracorporeal photopheresis (ECP) practices in pediatric patients is the marked heterogeneity of approaches to the patient evaluation, procedural aspects and apheresis product analysis. These issues are most relevant in ECP management in children with graft versus host disease (GVHD) after hematopoietic stem cell transplantation. Extracorporeal photopheresis in pediatric patients is considered relatively safe with few adverse effects reported from retrospective or observational studies...
March 22, 2017: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/28390986/pericardial-effusion-following-hematopoietic-cell-transplantation-in-children-is-associated-with-increased-risk-of-mortality
#8
Kelly Cox, Rajesh Punn, Elizabeth Schnorr, Benjamin A Pinsky, Sandhya Kharbanda
Hematopoietic cell transplantation (HCT) is curative for many pediatric malignant and non-malignant disorders, but is associated with significant morbidity and mortality, including development of pericardial effusion (PEF). We report the results of a retrospective chart review performed to assess the incidence, risk factors and prognostic effect of PEF in the pediatric HCT patients at Lucile Packard Children's Hospital (LPCH), Stanford University. One hundred and nineteen patients receiving HCT between January of 2010 through December of 2013 were selected through the LPCH Pediatric Stem Cell Transplant Program database...
April 5, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28387643/novel-gopc-fig-ros1-fusion-in-a-pediatric-high-grade-glioma-survivor
#9
Erin N Kiehna, Maxine R Arnush, Benita Tamrazi, Jennifer A Cotter, Debra Hawes, Nathan J Robison, Cindy Y Fong, Dolores B Estrine, Jennifer H Han, Jaclyn A Biegel
Pediatric high-grade glioma is a rare tumor characterized by high mortality. The authors report the first case of a high-grade glioma associated with a GOPC(FIG)-ROS1 fusion in a pediatric patient. The patient underwent gross-total resection at the age of 4 years, followed by adjuvant high-dose chemotherapy and autologous hematopoietic stem cell rescue. At 30 months after transplantation, she remains disease free.
April 7, 2017: Journal of Neurosurgery. Pediatrics
https://www.readbyqxmd.com/read/28380398/a-retrospective-study-of-paroxysmal-nocturnal-hemoglobinuria-in-pediatric-and-adolescent-patients
#10
Angela Mercuri, Piero Farruggia, Fabio Timeus, Laura Lombardi, Daniela Onofrillo, Maria Caterina Putti, Marta Pillon, Maria Elena Cantarini, Paola Corti, Gloria Tridello, Massimiliano De Bortoli, Anna Pegoraro, Simone Cesaro
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease, especially in children, characterized by intravascular hemolysis, thrombotic events, serious infections and bone marrow failure. We describe 16 patients who were diagnosed with PNH in childhood or adolescence. The time interval between the onset of symptoms and the PNH diagnosis and its treatment were compared in patients with classic PNH versus PNH associated with bone marrow disorder (PNH/BMD). A greater delay in diagnosis was observed in classic PNH compared to PNH/BMD patients...
March 18, 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28377772/treatment-of-pediatric-acute-graft-versus-host-disease-lessons-from-primary-immunodeficiency
#11
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
#12
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/28369555/understanding-the-relationship-between-child-health-related-quality-of-life-and-parent-emotional-functioning-in-pediatric-hematopoietic-stem-cell-transplant
#13
Angie Mae Rodday, Norma Terrin, Laurel K Leslie, Robert J Graham, Susan K Parsons
 Explore interrelationships between domains of child health-related quality of life (HRQL) and parent emotional functioning using parent-proxy and child report in the context of hematopoietic stem cell transplant (HSCT).  Data on 258 parent-child dyads were used from two longitudinal studies. Domains of HRQL included physical, emotional, and role functioning, and HSCT-related worry. We used structural equation modeling to model the outcome of parent emotional functioning using primary and alternative conceptual models...
March 20, 2017: Journal of Pediatric Psychology
https://www.readbyqxmd.com/read/28368380/risk-of-melanocytic-nevi-and-nonmelanoma-skin-cancer-in-children-after-allogeneic-hematopoietic-stem-cell-transplantation
#14
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-following-pediatric-allogeneic-stem-cell-transplantation
#15
H Pichler, K Horner, G Engstler, U Poetschger, E Glogova, S Karlhuber, M Martin, W Eibler, V Witt, W Holter, S 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 transplanted between 2001 and 2013 in our hospital, 138 of which 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/28350290/hla-genotypes-in-turkish-hematopoietic-cell-recipients-and-likelihood-of-finding-a-matched-donor-through-family-searches
#16
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
#17
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/28326208/a-child-as-a-donor-for-hematopoietic-stem-cell-transplantation-bioethical-justification-a-case-study-on-sickle-cell-disease
#18
Andrea Z Pereira, Ricardo Hellman, Nelson Hamerschlak, Andrea Kondo, Polianna Mara Rodrigues de Souza, Wilson Leite Pedreira, Luiz Fernando Alves Lima Mantovani, Eduardo Juan Troster, Henrique Grunspun, Marco Aurélio Scarpinella Bueno
Hematopoietic stem cell transplantation (HSCT) is an important treatment option for children with severe and refractory sickle cell disease (SCD) with debilitating clinical complications. HSCT with cells from the bone marrow of a HLA-identical sibling used in SCD has a low mortality risk, high cure rate, and high event-free survival rate after a median follow-up of 5-6 years. However, matched donors are found in only about 20% of the patients. A boy aged 8 years with SCD had a sister, <2 years old, a fully compatible donor...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/28319075/autologous-hematopoietic-stem-cell-transplantation-for-pediatric-multiple-sclerosis-a-registry-based-study-of-the-autoimmune-diseases-working-party-adwp-and-pediatric-diseases-working-party-pdwp-of-the-european-society-for-blood-and-marrow-transplantation-ebmt
#19
J Burman, K Kirgizov, K Carlson, M Badoglio, G L Mancardi, G De Luca, B Casanova, J Ouyang, R Bembeeva, J Haas, P Bader, J Snowden, D Farge
Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse...
March 20, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28306183/flavivirus-cross-reactivity-in-serological-tests-and-guillain-barr%C3%A3-syndrome-in-a-hematopoietic-stem-cell-transplant-patient-a-case-report
#20
Sonia M Raboni, Carmem Bonfim, Bernardo M Almeida, Camila Zanluca, Andrea C Koishi, Paula R V P Rodrigues, Claudia K Kay, Lisandro L Ribeiro, Rosana H Scola, Claudia N Duarte Dos Santos
Serological diagnosis of flavivirus infection is a challenge, particularly in the context of a disease associated with immune response enhancement in a transplant patient, where aspects such as previous flavivirus infections may be involved with the outcome. We report a case of a pediatric patient who developed Guillain-Barré syndrome (GBS) after matched-unrelated hematopoietic stem cell transplantation (HSCT). The patient lives in a Brazilian region that is experiencing an epidemic of Zika virus (ZIKV) and dengue virus (DENV)...
March 17, 2017: Transplant Infectious Disease: An Official Journal of the Transplantation Society
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