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https://www.readbyqxmd.com/read/28288951/haploidentical-related-donor-hematopoietic-stem-cell-transplantation-for-dock8-deficiency-using-post-transplantation-cyclophosphamide
#1
Nirali N Shah, Alexandra F Freeman, Helen Su, Kristen Cole, Mark Parta, Niki M Moutsopoulos, Safa Baris, Elif Karakoc-Aydiner, Thomas E Hughes, Heidi H Kong, Steve M Holland, Dennis D Hickstein
Dedicator-of-Cytokinesis-8 (DOCK8) deficiency, a primary immunodeficiency disease, can be reversed by allogeneic hematopoietic stem cell transplant (HSCT); however, there are few reports describing the use of alternative donor sources for HSCT in DOCK8 deficiency. We describe HSCT for patients with DOCK8 deficiency who lack a matched related or unrelated donor using bone marrow from haploidentical related donors and post-transplantation cyclophosphamide (PT/CY) for GVHD prophylaxis. Seven patients with DOCK8 deficiency (median age 20 years, range 7-25 years) received a haploidentical related donor HSCT...
March 10, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28286617/allogeneic-hematopoietic-stem-cell-transplantation-for-adult-acute-lymphoblastic-leukemia-results-from-a-single-center-1993-2011
#2
Ipek Yonal-Hindilerden, Sevgi Kalayoglu-Besisik, Nuray Gurses-Koc, Fehmi Hindilerden, Deniz Sargin
Background: For adult ALL patients, the indications and appropriate timing of allogeneic hematopoietic stem cell transplantation (AHSCT) continue to be debated. The primary aim of this single-institution study was to compare the results of our adult ALL patients that had been allografted with those reported in the current literature. Subjects and Methods: This study included 53 consecutive adults with acute lymphoblastic leukemia (ALL) who underwent allogeneic hematopoietic stem cell transplantation (AHSCT) with myeloablative (92%) and reduced-intensity (8%) conditioning between 1993 and 2011...
January 1, 2017: International Journal of Hematology-oncology and Stem Cell Research
https://www.readbyqxmd.com/read/28214159/is-surveillance-for-colonization-of-carbapenem-resistant-gram-negative-bacteria-important-in-adult-bone-marrow-transplantation-units
#3
Hayati Demiraslan, Fatma Cevahir, Elife Berk, Gokhan Metan, Mustafa Cetin, Emine Alp
BACKGROUND: The aim of this study was to investigate the rate of carbapenem-resistant gram-negative bacilli (CRGNB) colonization and to analyze the risk factors associated with CRGNB colonization. METHODS: This prospective study was conducted in adult patients hospitalized in hematopoietic stem cell transplantation (HSCT) units over a period of 8 months. Rectal swab samples were obtained from each participant every Monday, and patients CRGNB positive on admission were excluded...
February 15, 2017: American Journal of Infection Control
https://www.readbyqxmd.com/read/28199755/incidence-and-risk-factor-of-hemorrhagic-cystitis-after-allogeneic-transplantation-with-fludarabine-busulfan-and-anti-thymocyte-globulin-myeloablative-conditioning
#4
Wilson Lam, Jan Storek, Haocheng Li, Michelle Geddes, Andrew Daly
BACKGROUND: Hemorrhagic cystitis (HC) is a complication of allogeneic stem cell transplantation (SCT), associated with factors such as BK polyomavirus reactivation, age, conditioning regimen, and presence of graft-versus-host disease (GVHD). The incidence and impact of HC in patients receiving fludarabine (Flu), busulfan (Bu), and anti-thymocyte globulin (ATG) conditioning is unknown. METHODS: We conducted a case-control study of patients undergoing SCT at our center between January 1, 2003 and Dec 31, 2012, to determine the incidence of HC and its effect on patient outcomes including overall survival (OS), relapse, non-relapse mortality, GVHD, and healthcare resource use...
February 15, 2017: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/28179273/radiation-free-alternative-donor-hct-for-fanconi-anemia-patients-results-from-a-prospective-multi-institutional-study
#5
Parinda A Mehta, Stella M Davies, Thomas Leemhuis, Kasiani Myers, Nancy A Kernan, Susan E Prockop, Andromachi Scaradavou, Richard J O'Reilly, David A Williams, Leslie Lehmann, Eva Guinan, David Margolis, K Scott Baker, Adam Lane, Farid Boulad
Fanconi anemia (FA) is an inherited bone marrow failure syndrome characterized by chromosomal fragility, progressive marrow failure and cancer predisposition. Hematopoietic cell transplantation (HCT) is curative for FA-related marrow failure or leukemia, but both radiation exposure during transplant and graft-versus host disease (GVHD) may increase risk of later malignancies of the head and neck and anogenital area. In this study we tested a radiation-free conditioning regimen with a T-cell depleted graft to eliminate radiation exposure and minimize early as well as late toxicities of transplant...
February 8, 2017: Blood
https://www.readbyqxmd.com/read/28118857/impact-of-in-vivo-t-cell-depletion-in-hla-identical-allogeneic-stem-cell-transplantation-for-acute-myeloid-leukemia-in-first-complete-remission-conditioned-with-a-fludarabine-iv-busulfan-myeloablative-regimen-a-report-from-the-ebmt-acute-leukemia-working-party
#6
Marie Thérèse Rubio, Maud D'Aveni-Piney, Myriam Labopin, Rose-Marie Hamladji, Miguel A Sanz, Didier Blaise, Hakan Ozdogu, Etienne Daguindeau, Carlos Richard, Stella Santarone, Giuseppe Irrera, Ibrahim Yakoub-Agha, Moshe Yeshurun, Jose L Diez-Martin, Mohamad Mohty, Bipin N Savani, Arnon Nagler
BACKGROUND: The impact of the use of anti-thymocyte globulin (ATG) in allogeneic stem cell transplantation performed with HLA-identical sibling donors following fludarabine and 4 days intravenous busulfan myeloablative conditioning regimen has been poorly explored. METHODS: We retrospectively analyzed 566 patients who underwent a first HLA-identical allogeneic stem cell transplantation with this conditioning regimen for acute myeloid leukemia in first complete remission between 2006 and 2013 and compared the outcomes of 145 (25...
January 24, 2017: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/28108271/comparative-effectiveness-of-busulfan-and-fludarabine-versus-fludarabine-and-400%C3%A2-cgy-total-body-irradiation-conditioning-regimens-for-acute-myeloid-leukemia-myelodysplastic-syndrome
#7
Moaath Mustafa Ali, Donna M Abounader, Lisa A Rybicki, Melissa A Yurch, Jamie Starn, Christina Ferraro, Victoria Winslow, Betty K Hamilton, Aaron T Gerds, Hien Liu, Robert Dean, Brian T Hill, Brad Pohlman, Steven Andresen, Rabi Hanna, Matt Kalaycio, Brian J Bolwell, Navneet S Majhail, Ronald M Sobecks
Allogeneic hematopoietic cell transplantation conditioning regimen intensity has varied for patients with acute myeloid leukemia and myelodysplastic syndrome. A comparative effectiveness analysis was performed to assess outcomes of busulfan and fludarabine (BuFlu) versus those of fludarabine and 400 cGy total body irradiation (FluTBI) conditioning. Thirty-three subjects received BuFlu and 38 received FluTBI. The BuFlu group received more red blood cell transfusions (P = .02) and had a longer time to platelet recovery (P = ...
January 17, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28049637/low-immunosuppressive-burden-after-hla-matched-related-or-unrelated-bmt-using-posttransplantation-cyclophosphamide
#8
Christopher G Kanakry, Javier Bolaños-Meade, Yvette L Kasamon, Marianna Zahurak, Nadira Durakovic, Terry Furlong, Marco Mielcarek, Marta Medeot, Ivana Gojo, B Douglas Smith, Jennifer A Kanakry, Ivan M Borrello, Robert A Brodsky, Douglas E Gladstone, Carol Ann Huff, William H Matsui, Lode J Swinnen, Kenneth R Cooke, Richard F Ambinder, Ephraim J Fuchs, Marcos J de Lima, Borje S Andersson, Ravi Varadhan, Paul V O'Donnell, Richard J Jones, Leo Luznik
The intensive and prolonged immunosuppressive therapy required to prevent or treat graft-versus-host disease (GVHD) after allogeneic blood or marrow transplantation (alloBMT) puts patients at substantial risk for life-threatening infections, organ toxicity, and disease relapse. Posttransplantation cyclophosphamide (PTCy) can function as single-agent GVHD prophylaxis after myeloablative, HLA-matched related (MRD), or HLA-matched unrelated (MUD) donor T-cell-replete bone marrow allografting, obviating the need for additional prophylactic immunosuppression...
March 9, 2017: Blood
https://www.readbyqxmd.com/read/28018447/targeted-busulfan-and-fludarabine-based-conditioning-for-bone-marrow-transplantation-in-chronic-granulomatous-disease
#9
Hee Young Ju, Hyoung Jin Kang, Che Ry Hong, Ji Won Lee, Hyery Kim, Sang Hoon Song, Kyung-Sang Yu, In-Jin Jang, June Dong Park, Kyung Duk Park, Hee Young Shin, Joong-Gon Kim, Hyo Seop Ahn
Chronic granulomatous disease (CGD) is a primary immunodeficiency disease caused by impaired phagocytic function. Hematopoietic stem cell transplantation (HSCT) is a definitive cure for CGD; however, the use of HSCT is limited because of associated problems, including transplantation-related mortality and engraftment failure. We report a case of a patient with CGD who underwent successful HSCT following a targeted busulfan and fludarabine reduced-toxicity myeloablative conditioning. Intravenous busulfan was administered once daily for 4 consecutive days (days -8 to -5), and the target area under the curve was 75,000 µg·hr/L...
November 2016: Korean Journal of Pediatrics
https://www.readbyqxmd.com/read/27991894/fludarabine-with-pharmacokinetically-guided-iv-busulfan-is-superior-to-fixed-dose-delivery-in-pretransplant-conditioning-of-aml-mds-patients
#10
B S Andersson, P F Thall, B C Valdez, D R Milton, G Al-Atrash, J Chen, A Gulbis, D Chu, C Martinez, S Parmar, U Popat, Y Nieto, P Kebriaei, A Alousi, M de Lima, G Rondon, Q H Meng, A Myers, J Kawedia, L L Worth, M Fernandez-Vina, T Madden, E J Shpall, R B Jones, R E Champlin
We hypothesized that IV busulfan (Bu) dosing could be safely intensified through pharmacokinetic (PK-) dose guidance to minimize the inter-patient variability in systemic exposure (SE) associated with body-sized dosing, and that this should improve outcome of AML/MDS patients undergoing allogeneic stem cell transplantation. To test this hypothesis, we treated 218 patients (median age 50.7 years, male/female 50/50%) with fludarabine 40 mg/m(2) once daily x4, each dose followed by IV Bu, randomized to 130 mg/m(2) (N=107) or PK-guided to average daily SE, AUC of 6000 μM min (N=111), stratified for remission status and allo-grafting from HLA-matched donors...
December 19, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27914967/survival-advantage-and-comparable-toxicity-in-reduced-toxicity-treosulfan-based-versus-reduced-intensity-busulfan-based-conditioning-regimen-in-myelodysplastic-syndrome-and-acute-myeloid-leukemia-patients-after-allogeneic-hematopoietic-cell-transplantation
#11
Ioanna Sakellari, Despina Mallouri, Eleni Gavriilaki, Ioannis Batsis, Maria Kaliou, Varnavas Constantinou, Apostolia Papalexandri, Chrysavgi Lalayanni, Chrysanthi Vadikolia, Anastasia Athanasiadou, Evangelia Yannaki, Damianos Sotiropoulos, Christos Smias, Achilles Anagnostopoulos
Treosulfan has been incorporated in conditioning regimens for sustained remission without substantial toxicity and treatment-related mortality (TRM). We aimed to analyze the safety and efficacy of a fludarabine 150 mg/m(2) and treosulfan 42 g/m(2) (FluTreo) conditioning regimen in medically infirm patients. Outcomes were compared with those of a similar historical group treated with fludarabine 150 mg/m(2) to 180 mg/m(2), busulfan 6.4 mg/kg, and antithymocyte globulin (ATG) 5 mg/kg to 7.5 mg/kg (FluBuATG)...
November 30, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27843940/conditioning-with-fludarabine-busulfan-versus-busulfan-cyclophosphamide-is-associated-with-lower-agvhd-and-higher-survival-but-more-extensive-and-long-standing-bone-marrow-damage
#12
Xin He, YongBin Ye, XiaoJun Xu, Jing Wang, YuXian Huang, GuangYang Weng, MingWan Zhang, KunYuan Guo
Acute graft-versus-host disease (aGVHD) is a major complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) and a major cause of nonrelapse mortality after allo-HSCT. A conditioning regimen plays a pivotal role in the development of aGVHD. To provide a platform for studying aGVHD and evaluating the impact of different conditioning regimens, we established a murine aGVHD model that simulates the clinical situation and can be conditioned with Busulfan-Cyclophosphamide (Bu-Cy) and Fludarabine-Busulfan (Flu-Bu)...
2016: BioMed Research International
https://www.readbyqxmd.com/read/27816650/sequential-intensified-conditioning-regimen-allogeneic-hematopoietic-stem-cell-transplantation-in-adult-patients-with-intermediate-or-high-risk-acute-myeloid-leukemia-in-complete-remission-a-study-from-the-acute-leukemia-working-party-of-the-european-group
#13
Florent Malard, Myriam Labopin, Gernot Stuhler, Jörg Bittenbring, Arnold Ganser, Johanna Tischer, Mauricette Michallet, Nicolaus Kröger, Christoph Schmid, Anne Huynh, Michael Hallek, Bipin N Savani, Mohamad Mohty, Arnon Nagler
Post-transplant relapse is the leading cause of treatment failure in acute myeloid leukemia (AML) patients after reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation (allo-HSCT). To improve their outcome, we evaluated the outcome of a sequential intermediate-intensity conditioning regimen combining fludarabine, cytosine arabinoside, amsacrine, cyclophosphamide, and either total body irradiation or busulfan (FLAMSA) in patients with intermediate or high-risk AML in first or second complete remission (CR)...
February 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27748046/clofarabine-versus-fludarabine-based-reduced-intensity-conditioning-regimen-prior-to-allogeneic-transplantation-in-adults-with-aml-mds
#14
Patrice Chevallier, Myriam Labopin, Regis Peffault de La Tour, Bruno Lioure, Claude-Eric Bulabois, Anne Huynh, Didier Blaise, Pascal Turlure, Etienne Daguindau, Natacha Maillard, Ibrahim Yakoub-Agha, Gaelle Guillerm, Jeremy Delage, Nathalie Contentin, Jacques-Olivier Bay, Florence Beckerich, Jean-Henri Bourhis, Marie Detrait, Stéphane Vigouroux, Sylvie François, Faezeh Legrand, Thierry Guillaume, Mohamad Mohty
We have retrospectively compared survivals between acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS) patients who received either a clofarabine/busulfan (CloB2A2) or a fludarabine/busulfan (FB2A2) RIC regimen for allogeneic stem cell transplantation. Between 2009 and 2014, 355 allotransplanted cases were identified from the SFGM-TC registry as having received either the FB2A2 (n = 316, 56% males, median age: 59.2 years, AML 78.5%, first complete remission [CR1] 72%, median follow-up: 20 months) or the CloB2A2 (n = 39, 62% males, median age: 60...
November 2016: Cancer Medicine
https://www.readbyqxmd.com/read/27715384/allogeneic-hematopoietic-stem-cell-transplantation-for-nonmalignant-hematologic-disorders-using-chemotherapy-only-cytoreductive-regimens-and-t-cell-depleted-grafts-from-human-leukocyte-antigen-matched-or-mismatched-donors
#15
Alberto Mussetti, Nancy A Kernan, Susan E Prockop, Andromachi Scaradavou, Rachel Lehrman, Julianne M Ruggiero, Kevin Curran, Rachel Kobos, Richard O'Reilly, Farid Boulad
Nonmalignant hematologic disorders (NMHD) of childhood comprise a variety of disorders, including acquired severe aplastic anemia and inherited marrow failure syndromes. Patients with high-risk NMHD without matched related donors fare poorly with allogeneic hematopoietic alternative donor stem cell transplantation (allo-HSCT) and are at high risk for developing graft-versus-host disease following unmodified grafts. The authors retrospectively analyzed data on 18 patients affected by NMHD, lacking a human leukocyte antigen (HLA)-identical sibling donor, who underwent an alternative donor allo-HSCT at their institution between April 2005 and May 2013...
September 2016: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/27705929/a-5-day-cytoreductive-chemotherapy-followed-by-haplo-identical-hsct-fa5-bucy-as-a-tumor-ablative-regimen-improved-the-survival-of-patients-with-advanced-hematological-malignancies
#16
Ting Yang, Qiaoxian Lin, Jinhua Ren, Ping Chen, Xiaohong Yuan, Xiaofeng Luo, Tingbo Liu, Jing Zheng, Zhihong Zheng, Xiaoyun Zheng, Xinji Chen, Langhui Zhang, Hao Zheng, Zaisheng Chen, Xueling Hua, Shaohua Le, Jian Li, Zhizhe Chen, Jianda Hu
Haplo-HSCT has been used when HLA-matched siblings are not available. Conditioning regimens aim to reduce tumor burden prior to HSCT and provide sufficient immunoablation. We report the outcome of haplo-HSCT in 63 consecutive patients from 2/2013 to 12/2015 (19 females/44 males) with high-risk or relapsed/refractory hematological malignancies (n=29-AML; 8-sAML; 19-ALL; 5-advanced-MDS; 2-CML-BC). Median age was 20 years (range: 1.1-49). Twenty-one patients achieved remission prior to transplant, while 42 did not...
October 1, 2016: Oncotarget
https://www.readbyqxmd.com/read/27623172/impact-of-atg-dose-on-the-outcome-of-patients-undergoing-reduced-intensity-conditioning-followed-by-allogeneic-hematopoietic-stem-cell-transplantation-for-hematological-malignancies
#17
COMMENT
Jérôme Cornillon, Marie Balsat, Aurélie Cabrespine, Emmanuelle Tavernier-Tardy, Eric Hermet, Aurélien Mulliez, Karine Augeul-Meunier, Denis Guyotat, Jacques-Olivier Bay
Reduced intensity conditioning for allogeneic hematopoietic stem cell transplantation (allo-HSCT) is often proposed for patients with comorbidities. To enhance engraftment and limit graft-versus-host disease (GVHD), antithymoglobulin (ATG) is usually used. However, the dose needed remains unclear unlike myeloablative conditioning. In order to clarify this point, we conducted a retrospective study on patients who received a reduced intensity conditioning allo-HSCT based on a 2-day fludarabine and busulfan treatment with either 1 or 2 days of ATG treatment...
2016: Acta Haematologica
https://www.readbyqxmd.com/read/27596129/myeloablative-versus-reduced-intensity-conditioning-in-patients-with-myeloid-malignancies-a-propensity-score-matched-analysis
#18
Hassan Sibai, Umberto Falcone, Uday Deotare, Fotios V Michelis, Jieun Uhm, Vikas Gupta, John Kuruvilla, Jeffrey H Lipton, Matthew D Seftel, Hans A Messner, Dennis Dong Hwan Kim
Reduced-intensity conditioning (RIC) has been shown to have similar overall survival (OS) but higher relapse rates compared with myeloablative (MAC) regimens in patients with myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Using propensity score matching (PSM) analysis, well-balanced pairs of different variables can be compared effectively. We retrospectively compared allo-HSCT recipients with acute myeloid leukemia or myelodysplasia receiving a RIC regimen (FBT200; fludarabine 30 mg/m(2)/day for 4 days, busulfan 3...
December 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27543157/a-single-center-experience-comparing-alemtuzumab-fludarabine-and-melphalan-reduced-intensity-conditioning-with-myeloablative-busulfan-cyclophosphamide-and-antithymocyte-globulin-for-chronic-granulomatous-disease
#19
Pooja Khandelwal, Jacob J Bleesing, Stella M Davies, Rebecca A Marsh
Myeloablative conditioning (MAC) regimens are commonly used in transplantation for chronic granulomatous disease (CGD) but are associated with toxicity. Reduced-intensity conditioning (RIC) regimens have lower toxicity but may fail to achieve stable donor chimerism. We report a comparison between 4 patients who received a RIC regimen consisting of alemtuzumab (1 mg/kg), fludarabine (150 mg/m(2)), and melphalan (140 mg/m(2)) and 14 patients who received a MAC regimen consisting of busulfan (area under the curve, 1800 to 2000 µMol/min twice daily × 4 days), cyclophosphamide (50 mg/kg/day × 4), and antithymocyte globulin (15 mg/kg twice daily on days -2 and -1, then daily on days +1 and +2)...
November 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27518930/matched-sibling-donor-hematopoietic-stem-cell-transplantation-for-thalassemia
#20
Surapol Issaragrisil, Chutima Kunacheewa
PURPOSE OF REVIEW: Bone marrow transplantation is the only curative treatment for severe thalassemia. Since its successful first report in 1981, more than 4000 patients with this disease worldwide underwent bone marrow transplantation. The purpose of this review is to update the most recent reports of matched sibling donor hematopoietic stem cell transplantation in thalassemia. RECENT FINDINGS: Advanced and improved transplant techniques result in the improved outcomes in those transplants from a matched sibling donor with transplant-related mortality less than 5%...
November 2016: Current Opinion in Hematology
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