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bone marrow transplant pediatric

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https://www.readbyqxmd.com/read/28817182/centralized-patient-reported-outcome-data-collection-in-transplantation-is-feasible-and-clinically-meaningful
#1
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
#2
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 (alloBMT) platform using a reduced intensity conditioning (RIC) and partially HLA-mismatched (haploidentical) related donors for this population of pediatric and AYA solid tumor patients. Sixteen patients received a fludarabine, cyclophosphamide (Cy), melphalan and low dose total body irradiation (TBI) RIC haploidentical BMT (haploBMT) followed by post-transplantation cyclophosphamide (PTCy), mycophenolate mofetil (MMF) and sirolimus...
August 11, 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-disorders-a-report-from-eurocord-cord-blood-committee-cbc-ctiwp-and-severe-aplastic-anemia-working-party-saawp-of-the-european-society-of-blood
#3
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 sibling is an attractive option for patients with bone-marrow-failures syndromes (BMF), due to 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, who received CBT from a related HLA-identical donor in EBMT centers, between 1988 and 2014. Ninety-seven patients had inherited and 20 patients acquired BMF. Eighty-two patients received a single cord blood (CB) unit, whereas 35 patients received a combination of CB and bone marrow (BM) cells from the same donor...
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
#4
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
#5
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
#6
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
#7
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
#8
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...
July 24, 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
#9
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
#10
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
#11
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
#12
Aslı Sürer Adanir, Gülseren Taşkiran, O Alphan Küpesiz, Esin Özatalay
BACKGROUND: Bone Marrow Transplantation(BMT) is used to treat children with various hematologic, oncologic and metabolic diseases. While the treatment is lifesaving, it is also found to be related with anxiety, posttraumatic stress disorder, depression and psychosocial problems both in children and parents. METHODS: The aim of this study was to investigate psychopathology in pediatric BMT survivors and their mothers compared to the healthy controls. All children were interviewed using Kiddie Schedule for Affective Disorders and Schizophrenia for assessing the life-long psychopathology...
June 20, 2017: Pediatrics International: Official Journal of the Japan Pediatric Society
https://www.readbyqxmd.com/read/28620914/post-traumatic-stress-disorder-and-health-related-quality-of-life-in-the-siblings-of-the-pediatric-bone-marrow-transplantation-survivors-and-post-traumatic-stress-disorder-in-their-mothers
#13
Özge Gizli Çoban, Aslı Sürer Adanır, Esin Özatalay
Although BMT is lifesaving in many childhood diseases, it was found to be related to anxiety, depression, and PTSD in parents, and PTSD, anxiety and overall low self-esteem in siblings. Research on siblings' HRQoL is limited. The aim of this study was to investigate PTSD and HRQoL in siblings (donor and non-donor) of pediatric BMT survivors and PTSD in their mothers, compared to the healthy controls. Thirty-five siblings and their mothers and 35 healthy peers and their mothers were recruited as the study group and as the comparison group, respectively...
June 15, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28609016/multiparametric-whole-blood-dissection-a-one-shot-comprehensive-picture-of-the-human-hematopoietic-system
#14
Luca Basso-Ricci, Serena Scala, Raffaella Milani, Maddalena Migliavacca, Attilio Rovelli, Maria Ester Bernardo, Fabio Ciceri, Alessandro Aiuti, Luca Biasco
Human hematopoiesis is a complex and dynamic system where morphologically and functionally diverse mature cell types are generated and maintained throughout life by bone marrow (BM) Hematopoietic Stem/Progenitor Cells (HSPC). Congenital and acquired hematopoietic disorders are often diagnosed through the detection of aberrant frequency or composition of hematopoietic cell populations. We here describe a novel protocol, called "Whole Blood Dissection" (WBD), capable of analyzing in a single test-tube, hematopoietic progenitors and all major mature cell lineages composing either BM or peripheral blood (PB) through a multiparametric flow-cytometry analysis...
June 13, 2017: Cytometry. Part A: the Journal of the International Society for Analytical Cytology
https://www.readbyqxmd.com/read/28605290/early-response-based-therapy-stratification-improves-survival-in-adult-early-thymic-precursor-acute-lymphoblastic-leukemia-a-group-for-research-on-adult-acute-lymphoblastic-leukemia-study
#15
Jonathan Bond, Carlos Graux, Ludovic Lhermitte, Diane Lara, Thomas Cluzeau, Thibaut Leguay, Agata Cieslak, Amélie Trinquand, Cedric Pastoret, Mohamed Belhocine, Salvatore Spicuglia, Véronique Lheritier, Stéphane Leprêtre, Xavier Thomas, Françoise Huguet, Norbert Ifrah, Hervé Dombret, Elizabeth Macintyre, Nicolas Boissel, Vahid Asnafi
Purpose Early thymic precursor (ETP) acute lymphoblastic leukemia (ALL) is an immunophenotypically defined subgroup of T-cell ALL (T-ALL) associated with high rates of intrinsic treatment resistance. Studies in children have shown that the negative prognostic impact of chemotherapy resistance is abrogated by the implementation of early response-based intensification strategies. Comparable data in adults are lacking. Patients and Methods We performed comprehensive clinicobiologic, genetic, and survival analyses of a large cohort of 213 adult patients with T-ALL, including 47 patients with ETP-ALL, treated in the GRAALL (Group for Research on Adult Acute Lymphoblastic Leukemia) -2003 and -2005 studies...
August 10, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28598593/intravenous-palivizumab-in-respiratory-syncytial-virus-infection-after-hematopoietic-stem-cell-transplant-in-children
#16
Juan Pablo Torres, Lorena I Tapia, Paula Catalán, Verónica De la Maza, Asunción Mejías
Respiratory syncytial virus (RSV) infection can cause lower respiratory tract disease and mortality in pediatric hematopoietic stem cell transplant (HSCT) recipients. We report two children who underwent HSCT and developed RSV infection simultaneously at the Bone Marrow Transplant Unit. The treatment with intravenous palivizumab was provided and sequential viral loads were measured in nasopharyngeal (NP) and whole blood samples. To our knowledge, this is the first report where RSV loads were measured in parallel (NP and blood), before and after palivizumab, in correlation with a favorable clinical outcome in both cases...
June 9, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28584756/disseminated-cutaneous-trichosporonosis-in-an-adult-bone-marrow-transplant-patient
#17
A M Y Yong, S S Yang, K B Tan, S A Ho
The Trichosporon species are yeast-like opportunistic pathogens in immunocompromised patients. Trichosporon asahii infections have been reported in pediatric bone marrow transplant (BMT) patients. However, its incidence is low in the adult literature. A 52-year-old Chinese woman who was diagnosed with acute myeloid leukemia received induction chemotherapy and underwent allogenic bone marrow transplant, which was complicated by a relapse and required salvage chemotherapy. She developed persistent non-neutropenic fever secondary to presumed hepatosplenic candidiasis...
May 2017: Indian Dermatology Online Journal
https://www.readbyqxmd.com/read/28572263/recommendations-for-surveillance-for-children-with-leukemia-predisposing-conditions
#18
REVIEW
Christopher C Porter, Todd E Druley, Ayelet Erez, Roland P Kuiper, Kenan Onel, Joshua D Schiffman, Kami Wolfe Schneider, Sarah R Scollon, Hamish S Scott, Louise C Strong, Michael F Walsh, Kim E Nichols
Leukemia, the most common childhood cancer, has long been recognized to occasionally run in families. The first clues about the genetic mechanisms underlying familial leukemia emerged in 1990 when Li-Fraumeni syndrome was linked to TP53 mutations. Since this discovery, many other genes associated with hereditary predisposition to leukemia have been identified. Although several of these disorders also predispose individuals to solid tumors, certain conditions exist in which individuals are specifically at increased risk to develop myelodysplastic syndrome (MDS) and/or acute leukemia...
June 1, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28571522/risk-factors-predicting-the-survival-of-pediatric-patients-with-relapsed-refractory-non-hodgkin-lymphoma-who-underwent-hematopoietic-stem-cell-transplantation-a-retrospective-study-from-the-turkish-pediatric-bone-marrow-transplantation-registry
#19
Volkan Hazar, Vural Kesik, Gülsün Tezcan Karasu, Gülyüz Öztürk, Alphan Küpesiz, Suar Çakı Kılıç, Erman Ataş, Vedat Uygun, Nurşah Eker, Fatih Erbey, Şebnem Yılmaz Bengoa, Suna Emir, Sema Anak, Haldun Öniz, Hayriye Daloğlu, Serap Aksoylar, Ülker Koçak, Musa Karakükçü, Murat Elli, Nilgün Kurucu, Akif Yeşilipek
We examined outcomes of 62 pediatric patients with relapsed or refractory non-Hodgkin lymphoma (rr-NHL) who underwent hematopoietic stem cell transplantation (HSCT). The overall survival (OS) and event-free survival (EFS) rates were 65% and 48%, respectively. Survival rates for patients with chemosensitive disease at the time of HSCT were significantly higher than those of patients with chemoresistant disease (69% vs. 37%, p = .019 for OS; 54% vs. 12%, p < .001 for EFS; respectively). A chemoresistant disease at transplantation was the only factor that predicted a limited OS (hazard ratio = 10...
June 2, 2017: Leukemia & Lymphoma
https://www.readbyqxmd.com/read/28533057/late-effects-screening-guidelines-after-hematopoietic-cell-transplantation-for-inherited-bone-marrow-failure-syndromes-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international-conference-on-late-effects-after-pediatric
#20
REVIEW
Andrew C Dietz, Sharon A Savage, Adrianna Vlachos, Parinda A Mehta, Dorine Bresters, Jakub Tolar, Carmem Bonfim, Jean Hugues Dalle, Josu de la Fuente, Roderick Skinner, Farid Boulad, Christine N Duncan, K Scott Baker, Michael A Pulsipher, Jeffrey M Lipton, John E Wagner, Blanche P Alter
Patients with inherited bone marrow failure syndromes (IBMFS), such as Fanconi anemia (FA), dyskeratosis congenita (DC), or Diamond Blackfan anemia (DBA), can have hematologic manifestations cured through hematopoietic cell transplantation (HCT). Subsequent late effects seen in these patients arise from a combination of the underlying disease, the pre-HCT therapy, and the HCT process. During the international consensus conference sponsored by the Pediatric Blood and Marrow Transplant Consortium on late effects screening and recommendations following allogeneic hematopoietic cell transplantation for immune deficiency and nonmalignant hematologic diseases held in Minneapolis, Minnesota in May 2016, a half-day session was focused specifically on the unmet needs for these patients with IBMFS...
May 19, 2017: Biology of Blood and Marrow Transplantation
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