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https://www.readbyqxmd.com/read/29790457/comparison-of-different-conditioning-regimens-of-haploidentical-hematopoietic-stem-cell-transplant-in-patients-with-acute-myeloid-leukemia
#1
Yujie Jiang, Xiaosheng Fang, Xiaohui Sui, Xin Liu, Ying Li, Xianghua Wang, Hongzhi Xu, Lingyan Zhang, Xin Wang
OBJECTIVES: We evaluated the safety and efficacy of 2 conditioning regimens (busulfan/fludarabine vs modified busulfan/cyclophosphamide) in patients with acute myeloid leukemia undergoing haploidentical hematopoietic stem cell transplant. MATERAILS AND METHODS: Twenty patients with primary acute myeloid leukemia had been randomized into busulfan/fludarabine and modified busulfan/cyclophosphamide groups. We retrospectively compared hematopoietic engraftment, regimen-related toxicity, graft-versus-host disease, transplant-related mortality, leukemia-free survival, and overall survival between the groups...
May 23, 2018: Experimental and Clinical Transplantation
https://www.readbyqxmd.com/read/29745014/posttransplant-cyclophosphamide-for-haploidentical-stem-cell-transplantation-in-children-with-wiskott-aldrich-syndrome
#2
Yan Yue, Xiaodong Shi, Zeliang Song, Jiayue Qin, Junhui Li, Shunqiao Feng, Rong Liu
BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is the curative treatment for Wiskott-Aldrich syndrome (WAS). However, it is difficult to find a matched donor for patients. Therefore, haploidentical donors should be considered for patients lacking a suitable donor. Our pilot study evaluated whether HSCT with posttransplantation cyclophosphamide (PTCy) is an effective treatment for WAS. METHODS: Haploidentical family donors were selected as donor sources for a total of five patients without a suitable donor between March 2015 and March 2017...
May 10, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29621063/posttransplant-cyclophosphamide-for-hla-haploidentical-transplantation-in-patients-with-mucopolysaccharidosis
#3
Yan Yue, Zeliang Song, Junhui Li, Shunqiao Feng, Rong Liu, Xiaodong Shi
We successfully used a haploidentical transplantation protocol with posttransplant cyclophosphamide (CY) (50 mg/kg/d on days +3 and +4) for in vivo T-cell depletion in patients with mucopolysaccharidosis using reduced-intensive conditioning regimens, followed by a busulfan-based conditioning regimen, which included busulfan (12 to 16 mg/kg) and fludarabine(150 to 200 mg/m)+rabbit antihuman thymocyte globulin (7.5 to 10 mg/kg) as a conditioning regimen. Cyclosporine or tacrolimus, methotrexate, mycophenolate mofetil, and methylprednisolone were administered to prevent graft-versus-host disease (GVHD)...
April 4, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29605545/a-comparison-of-intrauterine-haemopoietic-cell-transplantation-and-lentiviral-gene-transfer-for-the-correction-of-severe-%C3%AE-thalassaemia-in-a-hbbth3-murine-model
#4
Niraja M Dighe, Kang Wei Tan, Lay Geok Tan, Steven S W Shaw, Suzanne M K Buckley, Dedy Sandikin, Nuryanti Johana, Yi-Wan Tan, Arijit Biswas, Mahesh Choolani, Simon N Waddington, Michael N Antoniou, Jerry K Y Chan, Citra N Z Mattar
Major haemoglobinopathies place tremendous strain on global resources. Intrauterine haemopoietic cell (IUHCT) and gene (IUGT) therapies can potentially reduce perinatal morbidities with greater efficacy than postnatal therapy alone. We performed both procedures in the thalassaemic HbbTh3/+ murine model. Intraperitoneal delivery of coisogenic cells at E13-14 produced dose-dependent chimerism. High-dose adult bone marrow (BM) cells maintained 0.2-3.1% chimerism over ~24 weeks and treated heterozygotes demonstrated higher chimerism than wild-type pups (1...
March 29, 2018: Experimental Hematology
https://www.readbyqxmd.com/read/29579887/donor-selection-based-on-killer-cell-immunoglobulin-like-receptor-kir-genotype-may-improve-outcome-after-t-cell-replete-haploidentical-transplantation
#5
A Torío, M J Pascual, I Vidales, M Ortiz, A Caballero, A I Heiniger
BACKGROUND: In haploidentical stem cell transplantation (SCT), the "ideal donor" selection is not performed in a standardized way according to killer cell immunoglobulin-like receptor (KIR) genotype expressed by potential donors. The aim of this study was to evaluate the relevance of KIR genotype in a series of patients submitted to haploidentical SCT in our center. METHODS: We retrospectively analyzed 30 patients that were prepared with the use of a conditioning regimen including thiotepa-busulfan-fludarabine with high doses of post-transplantation cyclophosphamide (CyPT) and tacrolimus as graft-versus-host disease (GVHD) prophylaxis...
March 2018: Transplantation Proceedings
https://www.readbyqxmd.com/read/29562468/-outcomes-of-reduced-intensity-conditioning-allogeneic-hematopoietic-stem-cell-transplantation-for-10-patients-with-myelofibrosis
#6
H X Liu, J Yang, J L Jiang, Y Cai, L P Wan, L Wu, S Shao, C Wang
Objective: To evaluate the efficacy of reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation (RIC-allo-HSCT) for patients with myelofibrosis (MF). Methods: The clinical data of 10 patients with myelofibrosis (MF) who underwent RIC-allo-HSCT. Results: Of all 10 patients, 6 were male and 4 women, with a median age of 28.5 (22-54). Using fludarabine/busulfan plus total body irradiation (FB+TBI) pretreatment scheme based. Hematopoiesis reconstitution was achieved in 9 patients (90%)...
March 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29555313/a-comparison-of-the-myeloablative-conditioning-regimen-fludarabine-busulfan-with-cyclophosphamide-total-body-irradiation-for-allogeneic-stem-cell-transplantation-in-the-modern-era-a-cohort-analysis
#7
Mahasweta Gooptu, Haesook T Kim, Vincent T Ho, Edwin P Alyea, John Koreth, Philippe Armand, Jerome Ritz, Sarah Nikiforow, Brett E Glotzbecker, Prashant Nageshwar, Robert J Soiffer, Joseph H Antin, Corey S Cutler
With improvement in transplantation practices in the modern era, nonrelapse mortality (NRM) following allogeneic hematopoietic stem cell transplantation (HSCT) has improved, while disease relapse rates have remained unchanged. Survival outcomes are therefore driven by NRM in the modern era. Myeloablative conditioning (MAC) regimens are used to maximize disease control and facilitate engraftment; however, their use is often limited by toxicity. The commonly used MAC regimens incorporate either chemotherapy plus total body irradiation (TBI) or combination chemotherapy...
March 16, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29542029/clofarabine-followed-by-haploidentical-stem-cell-transplant-using-fludarabine-busulfan-and-total-body-irradiation-with-post-transplant-cyclophosphamide-in-non-remission-aml
#8
Kevin Rakszawski, Kosuke Miki, David Claxton, Henry Wagner, Hiroko Shike, Shin Mineishi, Seema Naik
Approximately 30-40% of patients with acute myeloid leukemia (AML) experience induction failures. In these patients who do not achieve remission with two cycles of standard induction therapies, the probability of achieving remission with subsequent inductions is very limited. Hematopoietic stem cell transplantation (HSCT) is the only curative option for these patients, but high relapse rate and transplant-related mortality often preclude them to proceed to transplant. Thus, AML not in remission at time of HSCT remains a huge unmet need in current HSCT practice, particularly if the patient does not have an HLA-matched donor identified by the time of two induction failures...
March 14, 2018: International Journal of Hematology
https://www.readbyqxmd.com/read/29528181/disease-burden-and-conditioning-regimens-in-asct1221-a-randomized-phase-ii-trial-in-children-with-juvenile-myelomonocytic-leukemia-a-children-s-oncology-group-study
#9
Christopher C Dvorak, Prakash Satwani, Elliot Stieglitz, Mitchell S Cairo, Ha Dang, Qinglin Pei, Yun Gao, Donna Wall, Tali Mazor, Adam B Olshen, Joel S Parker, Samir Kahwash, Betsy Hirsch, Susana Raimondi, Neil Patel, Micah Skeens, Todd Cooper, Parinda A Mehta, Stephan A Grupp, Mignon L Loh
BACKGROUND: Most patients with juvenile myelomonocytic leukemia (JMML) are curable only with allogeneic hematopoietic cell transplantation (HCT). However, the current standard conditioning regimen, busulfan-cyclophosphamide-melphalan (Bu-Cy-Mel), may be associated with higher risks of morbidity and mortality. ASCT1221 was designed to test whether the potentially less-toxic myeloablative conditioning regimen containing busulfan-fludarabine (Bu-Flu) would be associated with equivalent outcomes...
March 12, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29512932/modified-conditioning-regimen-improves-outcomes-of-unrelated-donor-peripheral-blood-stem-cell-transplantation-for-%C3%AE-thalassaemia-major-patients
#10
Ke Huang, Dun-Hua Zhou, Yang Li, Hong-Gui Xu, Li-Ping Que, Chun Chen, Hong-Man Xue, Hai-Xia Guo, Wen-Jun Weng, Shao-Liang Huang, Jian-Pei Fang
BACKGROUND: The objective of this study was to evaluate the feasibility of a modified conditioning regimen for the treatment of patients with β-thalassaemia major (TM), using unrelated donor peripheral blood stem cell transplantation (UD-PBSCT). METHODS: A modified conditioning regimen based on intravenous busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin was performed in 50 consecutive childhood patients with β-TM and a median age of 4.6 years (range, 2-12 years)...
March 7, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29490698/clinical-evaluation-of-haploidentical-hematopoietic-combined-with-human-umbilical-cord-derived-mesenchymal-stem-cells-in-severe-aplastic-anemia
#11
Lixin Xu, Zhouyang Liu, Yamei Wu, Xueliang Yang, Yongbin Cao, Xiaohong Li, Bei Yan, Songwei Li, Wanming Da, Xiaoxiong Wu
BACKGROUND: This study not only evaluated the clinical effects of treatment using haploidentical hematopoietic stem cells (haplo-HSCs) combined with human umbilical cord mesenchymal stem cells (UC-MSCs) in patients with severe aplastic anemia (SAA), but also investigated the factors related to graft versus host disease (GVHD). METHODS: Cotransplantation of haplo-HSCs and UC-MSCs was performed in 24 SAA patients. The conditioning regimens consisted of rabbit anti-human T-lymphocyte immunoglobulin (ATG), cyclophosphamide, and fludarabine with or without busulfan...
March 1, 2018: European Journal of Medical Research
https://www.readbyqxmd.com/read/29474820/effects-of-two-doses-of-anti-t-lymphocyte-globulin-fresenius-given-after-full-match-sibling-stem-cell-transplantation-in-acute-myeloblastic-leukemia-patients-who-underwent-myeloablative-fludarabine-busulfan-conditioning
#12
Can Boga, Mahmut Yeral, Ciğdem Gereklioglu, Suheyl Asma, Erkan Maytalman, Pelin Aytan, Ilknur Kozanoglu, Cagla Sariturk, Hakan Ozdogu
OBJECTIVE/BACKGROUND: Anti-T lymphocyte globulin Fresenius (rATG-F; ATG-Fresenius) and antithymocyte globulin (thymoglobulin), which are included in transplant protocols, are used to reduce the risk of chronic graft-versus-host disease (cGVHD) or suppress allograft rejection. Available clinical studies have been conducted in heterogenous patient populations and with different administration protocols including stem cell sources. Additionally, the pharmacokinetics of ATG is variable, and the clinically effective dose of rATG-F, in particular, is not exactly known...
February 20, 2018: Hematology/oncology and Stem Cell Therapy
https://www.readbyqxmd.com/read/29473150/better-outcomes-of-modified-myeloablative-conditioning-without-antithymocyte-globulin-versus-myeloablative-conditioning-in-cord-blood-transplantation-for-hematological-malignancies-a-retrospective-development-and-a-prospective-validation-study
#13
Zimin Sun, Huilan Liu, Chenhui Luo, Liangquan Geng, Changcheng Zheng, Baolin Tang, Xiaoyu Zhu, Juan Tong, Xingbing Wang, Kaiyang Ding, Xiang Wan, Lei Zhang, Wen Yao, Kaiding Song, Xuhan Zhang, Yue Wu, Huizhi Yang, Yongsheng Han, Xin Liu, Weibo Zhu, Jingsheng Wu, Zuyi Wang
Cord blood transplantation (CBT) is an effective option for treating hematological malignancies, but graft failure (GF) remains the primary cause of therapy failure. Thus, based on myeloablative conditioning (MAC) of busulfan with cyclophosphamide (Bu/Cy) or total body irradiation with Cy (TBI/Cy), fludarabine (Flu) was added to Bu/Cy and cytarabine (CA) to TBI/Cy for a modified myeloablative conditioning (MMAC). To compare the prognosis of MMAC with MAC, we conducted a retrospective study including 58 patients who underwent CBT with MAC or MMAC from 2000 to 2011...
February 23, 2018: International Journal of Cancer. Journal International du Cancer
https://www.readbyqxmd.com/read/29431621/haploidentical-hsct-for-hemoglobinopathies-improved-outcomes-with-tcr%C3%AE-%C3%AE-cd19-depleted-grafts
#14
Javid Gaziev, Antonella Isgrò, Pietro Sodani, Katia Paciaroni, Gioia De Angelis, Marco Marziali, Michela Ribersani, Cecilia Alfieri, Alessandro Lanti, Tiziana Galluccio, Gaspare Adorno, Marco Andreani
We examined outcomes of haploidentical hematopoietic cell transplantation (haplo-HCT) using T-cell receptor αβ+ (TCRαβ+ )/CD19+ -depleted grafts (TCR group, 14 patients) in children with hemoglobinopathies. Patients received a preparative regimen consisting of busulfan, thiotepa, cyclophosphamide, and antithymocyte globulin preceded by fludarabine, hydroxyurea, and azathioprine. The median follow-up among surviving patients was 3.9 years. The 5-year probabilities of overall survival (OS) and disease-free survival (DFS) were 84% and 69%, respectively...
February 13, 2018: Blood Advances
https://www.readbyqxmd.com/read/29423053/thiotepa-busulfan-fludarabine-compared-to-busulfan-fludarabine-for-sibling-and-unrelated-donor-transplant-in-acute-myeloid-leukemia-in-first-remission
#15
Francesco Saraceni, Myriam Labopin, Rose-Marie Hamladji, Ghulam Mufti, Gerard Socié, Avichai Shimoni, Jeremy Delage, Eric Deconinck, Patrice Chevallier, Didier Blaise, Jaime Sanz, Anne Huynh, Edouard Forcade, Bipin N Savani, Mohamad Mohty, Arnon Nagler
Background: A preparatory regimen consisting of thiotepa-busulfan-fludarabine (TBF) has been associated with reduced relapse in patients with haematological malignancies after haploidentical and cord blood transplants; however, few data exist regarding TBF conditioning in sibling (MSD) and unrelated donor (URD) transplants for AML. Results: Among patients receiving a myeloablative (MAC) regimen, TBF-MAC was associated with significantly lower relapse (HR 0.47, p = 0...
January 9, 2018: Oncotarget
https://www.readbyqxmd.com/read/29412158/allogeneic-hematopoietic-stem-cell-transplantation-for-gata2-deficiency-using-a-busulfan-based-regimen
#16
Mark Parta, Nirali N Shah, Kristin Baird, Hind Rafei, Katherine R Calvo, Thomas Hughes, Kristen Cole, Meg Kenyon, Bazetta Blacklock Schuver, Jennifer Cuellar-Rodriguez, Christa S Zerbe, Steven M Holland, Dennis D Hickstein
Allogeneic hematopoietic stem cell transplantation (HSCT) reverses the bone marrow failure syndrome due to GATA2 deficiency. The intensity of conditioning required to achieve reliable engraftment and prevent relapse remains unclear. Here, we describe the results of a prospective study of HSCT in 22 patients with GATA2 deficiency using a busulfan-based conditioning regimen. The study included 2 matched related donor (MRD) recipients, 13 matched unrelated donor (URD) recipients, and 7 haploidentical related donor (HRD) recipients...
February 3, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29410181/outcomes-of-children-with-hemophagocytic-lymphohistiocytosis-given-allogeneic-hematopoietic-stem-cell-transplantation-in-italy
#17
Chiara Messina, Marco Zecca, Franca Fagioli, Attilio Rovelli, Stefano Giardino, Pietro Merli, Fulvio Porta, Maurizio Aricò, Elena Sieni, Giuseppe Basso, Mimmo Ripaldi, Claudio Favre, Marta Pillon, Antonio Marzollo, Marco Rabusin, Simone Cesaro, Mattia Algeri, Maurizio Caniglia, Paolo Di Bartolomeo, Ottavio Ziino, Francesco Saglio, Arcangelo Prete, Franco Locatelli
We report on 109 patients with hemophagocytic lymphohistiocytosis (HLH) undergoing 126 procedures of allogeneic hematopoietic stem cell transplantation (HSCT) between 2000 and 2014 in centers associated with the Italian Pediatric Hematology Oncology Association. Genetic diagnosis was FHL2 (32%), FHL3 (33%), or other defined disorders known to cause HLH (15%); in the remaining patients no genetic abnormality was found. Donor for first transplant was an HLA-matched sibling for 25 patients (23%), an unrelated donor for 73 (67%), and an HLA-partially matched family donor for 11 children (10%)...
February 2, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29403023/allogenic-peripheral-stem-cell-transplantation-from-hla-matched-related-donors-for-adult-sickle-cell-disease-remarkable-outcomes-from-a-single-center-trial
#18
H Ozdogu, C Boga, M Yeral, I Kozanoglu, C Gereklioglu, P Aytan, M Kasar, S Asma, N Buyukkurt, S Solmaz, A Korur, C Sariturk
Adult patients with sickle cell disease (SCD) are highly susceptible to stem cell transplant complications, including drug toxicity, graft versus host disease (GVHD), and graft rejection due to SCD-related tissue damage, endothelial activation, and inflammation. The scarcity of compatible stem cells for transplantation further limits treatment options, with only 43 cases of adult allogeneic peripheral blood stem cell transplantation (allo-PSCT) from human leukocyte antigen (HLA)-identical sibling donors reported in the international registry for the period 1986-2013...
February 5, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29337223/sequential-conditioning-with-thiotepa-in-t-cell-replete-hematopoietic-stem-cell-transplantation-for-the-treatment-of-refractory-hematologic-malignancies-comparison-with-matched-related-haplo-mismatched-and-unrelated-donors
#19
Rémy Duléry, Anne-Lise Ménard, Sylvain Chantepie, Jean El-Cheikh, Sylvie François, Jérémy Delage, Federica Giannotti, Annalisa Ruggeri, Eolia Brissot, Giorgia Battipaglia, Florent Malard, Ramdane Belhocine, Simona Sestili, Anne Vekhoff, François Delhommeau, Oumédaly Reman, Ollivier Legrand, Myriam Labopin, Marie-Thérèse Rubio, Mohamad Mohty
The results of conventional allogeneic stem cell transplantation (SCT) in refractory hematologic malignancies are poor. Sequential strategies have shown promising results in refractory acute myelogenous leukemia (AML), but have not been validated in a haploidentical (Haplo) transplant setting. We have developed a new sequential approach combining chemotherapy with broad antitumor activity (thiotepa 10 mg/kg, etoposide 400 mg/m2 , and cyclophosphamide 1600 mg/m2 from day -15 to day -10), followed after 3 days of rest by a reduced-intensity conditioning regimen (fludarabine 150 mg/m2 , i...
May 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29278934/impact-of-oral-and-gastrointestinal-mucositis-on-body-weight-alterations-during-hematopoietic-stem-cell-transplantation
#20
Fernanda de Paula Eduardo, Leticia Mello Bezinelli, Marcella Ferreira Gobbi, Andrea Z Pereira, Cristina Vogel, Nelson Hamerschlak, Luciana Corrêa
The aim of this study was to evaluate whether digestive tract mucositis is a predictive factor for body weight (BW) alterations during hematopoietic stem cell transplantation (HSCT). Data about characteristics of transplantation, initial nutritional conditions and gastrointestinal mucositis were collected from adult patients (n = 105) who underwent autologous and allogeneic HSCT. Oral mucositis (OM) was not a predictive factor for BW loss, but it was an independent factor for BW gain in autologous HSCT (β = 0...
February 2018: Nutrition and Cancer
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