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https://www.readbyqxmd.com/read/29745014/posttransplant-cyclophosphamide-for-haploidentical-stem-cell-transplantation-in-children-with-wiskott-aldrich-syndrome
#1
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/29731447/aryl-sulfonate-based-anticancer-alkylating-agents
#2
Hamdullah Khadim Sheikh, Tanzila Arshad, Ghazala Kanwal
This research work revolves around synthesis of antineoplastic alkylating sulfonate esters with dual alkylating sites for crosslinking of the DNA strands. These molecules were evaluated as potential antineoplastic cross linking alkylating agents by reaction with the nucleoside of Guanine DNA nucleobase at both ends of the synthesized molecule. Synthesis of the alkylating molecules and the crosslinking with the guanosine nucleoside was monitored by MALDITOF mass spectroscopy. The synthesized molecule's crosslinking or adduct forming rate with the nucleoside was compared with that of 1,4 butane disulfonate (busulfan), in form of time taken for the appearance of [M+H]+ ...
May 2018: Pakistan Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/29721346/hsct-gave-as-a-manifestation-of-chronic-graft-versus-host-disease-a-case-report-and-review-of-the-existing-literature
#3
Michael J Grant, Mitchell E Horwitz
Gastric antral vascular ectasia or "watermelon stomach" is a significant cause of nonvariceal upper GI bleeding and is characterized by red, tortuous ectatic vessels along longitudinal folds in the gastric antrum. The existing literature links GAVE to patients with cirrhosis, scleroderma, bone marrow transplantation, and chronic renal failure among other associations, but its pathophysiology remains ill-defined. Over 30 cases of hematopoietic stem cell transplant-related GAVE (HSCT-GAVE) have been reported in the literature to date and there are likely many more that go undiagnosed or are attributed to another cause of upper gastrointestinal bleeding...
2018: Case Reports in Transplantation
https://www.readbyqxmd.com/read/29721205/treosulfan-induces-distinctive-gonadal-toxicity-compared-with-busulfan
#4
Mattan Levi, Salomon M Stemmer, Jerry Stein, Ruth Shalgi, Irit Ben-Aharon
Treosulfan (L-treitol-1,4-bis-methanesulfonate) has been increasingly incorporated as a main conditioning protocol for hematopoietic stem cell transplantation in pediatric malignant and non-malignant diseases. Treosulfan presents lower toxicity profile than other conventional alkylating agents containing myeloablative and immunosuppressive traits such as busulfan. Yet, whereas busulfan is considered highly gonadotoxic, the gonadal toxicity profile of treosulfan remains to be elucidated. To study the gonadotoxicity of treosulfan, pubertal and prepubertal male and female mice were injected with treosulfan or busulfan and sacrificed one week, one month or six months later...
April 10, 2018: Oncotarget
https://www.readbyqxmd.com/read/29705567/intravenous-compared-to-oral-busulfan-with-cyclophosphamide-for-autologous-hematopoietic-cell-transplant-conditioning-for-plasma-cell-myeloma
#5
Moaath Mustafa Ali, Donna M Corrigan, Lisa A Rybicki, Melissa A Yurch, Kelly Cherni, Hien Liu, Robert Dean, Betty Hamilton, Brad Pohlman, Steven Andresen, Matt Kalaycio, Brian J Bolwell, Navneet S Majhail, Ronald Sobecks
No abstract text is available yet for this article.
April 24, 2018: Hematology/oncology and Stem Cell Therapy
https://www.readbyqxmd.com/read/29705215/relationship-between-exposure-to-treosulfan-and-its-monoepoxytransformer-an-insight-from-population-pharmacokinetic-study-in-pediatric-patients-before-hematopoietic-stem-cell-transplantation
#6
Dorota Danielak, Anna Kasprzyk, Tomasz Wróbel, Jacek Wachowiak, Krzysztof Kałwak, Franciszek Główka
Treosulfan (TREO), a structural analog of busulfan, is currently studied as a myeloablative agent in conditioning regimens before hematopoietic stem cell transplantation in pediatric patients. High exposure to TREO (>1650 mg∗h/mL) might be related to early toxicity, especially skin toxicity and mucositis. The aim of the present study was to investigate a potential relationship between exposure to TREO and its monoepoxytransformer (S,S-EBDM), as well as variability of the pharmacokinetics of these entities by means of a population pharmacokinetic approach with a non-linear mixed-effects analysis...
April 26, 2018: European Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/29702970/a-new-intensive-conditioning-regimen-for-allogeneic-hematopoietic-stem-cell-transplantation-in-patients-with-refractory-or-relapsed-acute-myeloid-leukemia
#7
Jingbo Wang, Jie Zhao, Xinhong Fei, Yuming Yin, Haoyu Cheng, Weijie Zhang, Jiangying Gu, Fan Yang, Yixin Yang, Song Xue, Zhengqin Tian, Junbao He, Shuqin Zhang, Xiaocan Wang
To explore the efficacy, and safety of the intensive conditioning regimen consisting of cladribine, cytarabine (Ara-C), and granulocyte colony-stimulating factor (G-CSF) plus modified busulfan (Bu) combined with cytoxan (Cy) (BuCy), prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with refractory, or relapsed acute myeloid leukemia (R/R AML).Thirty-Six R/R AML patients scheduled to receive allo-HSCT were consecutively, enrolled in this prospective study, and treated using intensive conditioning regimen consisting of CLAG plus modified BuCy...
April 2018: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29684562/bmt-following-non-myeloablative-treosulfan-conditioning-is-curative-in-a-murine-model-of-sickle-cell-disease
#8
Divya Devadasan, Chiao-Wang Sun, Erik R Westin, Li-Chen Wu, Kevin M Pawlik, Tim M Townes, Frederick D Goldman
Allogeneic hematopoietic stem cell transplantation (HSCT) can be curative for patients with sickle cell disease (SCD). However, morbidity associated with myeloablative conditioning and graft versus host disease has limited its utility. To this end, autologous HSCT for SCD using lentiviral gene-modified bone marrow (BM) or peripheral blood stem cells has been undertaken, though toxicities of fully ablative conditioning with busulfan and incomplete engraftment have been encountered. Treosulfan, a busulfan analog with a low extra-medullary toxicity profile, has been employed successfully as part of a myeloablative conditioning regimen in the allogeneic setting in SCD...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29680515/safety-and-efficacy-of-once-daily-intravenous-busulfan-in-allogeneic-transplantation-a-matched-pair-analysis
#9
Shinichi Kako, Shinichiro Fujiwara, Miki Sato, Shun-Ichi Kimura, Hideki Nakasone, Kazuteru Ohashi, Toshiro Kawakita, Tetsuo Maeda, Takanobu Morishita, Ritsuro Suzuki, Takahiro Fukuda, Tatsuo Ichinohe, Mio Kurata, Yoshiko Atsuta, Yoshinobu Kanda
Compared with 4-times-daily infusion of intravenous busulfan (ivBU4), the safety and efficacy of once-daily infusion of ivBU (ivBU1) has not been fully clarified. We have been routinely using ivBU1 in a conditioning regimen in adult patients with myeloid malignancy who undergo allogeneic hematopoietic stem cell transplantation. In this study, a total of 91 patients who received ivBU1 for 2 days (n = 18) or 4 days (n = 73) in our institutions were compared with 273 control patients who received ivBU4, who were matched for age, sex, performance status, disease risk, conditioning regimen, and donor type, selected from the database of the Japanese Society for Hematopoietic Cell Transplantation using optimal matching algorithms...
April 19, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29679773/distinctive-infectious-complications-in-patients-with-central-nervous-system-lymphoma-undergoing-thiotepa-busulfan-and-cyclophosphamide-tbc-conditioned-autologous-stem-cell-transplantation
#10
Michael Scordo, Sejal M Morjaria, Eric R Littmann, Ankush Bhatia, Helen H Chung, Molly Maloy, Lisa M DeAngelis, Sergio A Giralt, Ying Taur, Craig S Sauter
We investigated the incidence of viral, fungal, bacterial, and parasitic infections observed in 57 patients with central nervous system lymphoma (CNSL) after TBC-ASCT and 79 patients with systemic NHL after traditional BEAM-ASCT. Twenty of 57 (35%) TBC-ASCT patients had detectable viremia with human herpesvirus 6 (HHV-6), cytomegalovirus (CMV), adenovirus (ADV), or BK virus (BKV), versus 9 of 79 (11%) BEAM-ASCT patients. Nine TBC-ASCT patients had clinically relevant viral infections (4 HHV-6, 2 CMV, 1 ADV, 2 BKV), versus 0 in the BEAM-ASCT group...
April 18, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29679380/a-non-myeloablative-chimeric-mouse-model-accurately-defines-microglia-and-macrophage-contribution-in-glioma
#11
Kenny Yu, A Saam Youshani, Fiona L Wilkinson, Claire O'Leary, Peter Cook, Liisi Laaniste, Aiyin Liao, Dominic Mosses, Christopher Waugh, Hannah Shorrock, Omar Pathmanaban, Andrew Macdonald, Ian Kamaly-Asl, Federico Roncaroli, Brian W Bigger
AIMS: Resident and peripherally-derived glioma associated microglia/macrophages (GAMM) play a key role in driving tumour progression, angiogenesis, invasion, and attenuating host immune responses. Differentiating these cells' origins is challenging and current pre-clinical models such as irradiation-based adoptive transfer, parabiosis and transgenic mice have limitations. We aimed to develop a novel non-myeloablative transplantation (NMT) mouse model that permits high levels of peripheral chimerism without blood-brain barrier (BBB) damage or brain infiltration prior to tumour implantation...
April 20, 2018: Neuropathology and Applied Neurobiology
https://www.readbyqxmd.com/read/29674658/high-dose-carboplatin-etoposide-melphalan-increases-risk-of-thrombotic-microangiopathy-and-organ-injury-after-autologous-stem-cell-transplantation-in-patients-with-neuroblastoma
#12
Sonata Jodele, Christopher E Dandoy, Kasiani Myers, Gregory Wallace, Adam Lane, Ashley Teusink-Cross, Brian Weiss, Stella M Davies
Transplant-associated thrombotic microangiopathy (TA-TMA) is an increasingly recognized complication of hematopoietic cell transplant that can result in multi-organ failure (MOF). Patients undergoing high-dose chemotherapy with autologous stem cell transplant (aHCT) for neuroblastoma require good organ function to receive post-transplant radiation and immunotherapy. We examined TA-TMA incidence and transplant outcomes in patients with neuroblastoma receiving different transplant preparative regimens. Sixty patients underwent aHCT using high-dose chemotherapy: 41 patients received carboplatin/etoposide/melphalan (CEM), 13 patients busulfan/melphalan (Bu/Mel) and six patients received tandem transplant (cyclophosphamide/thiotepa and CEM)...
April 19, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29673920/-sinusoidal-obstruction-syndrome-after-beam-conditioning-regiment-for-autologous-stem-cell-transplantation-imputability-of-bendamustine-report-of-two-cases-and-literature-review
#13
A Meyer, L-M Fornecker, A Guffroy, A-S Korganow, M Martin
INTRODUCTION: Sinusoidal obstruction syndrome is a rare complication of autologous hematopoietic stem cell transplantation. This syndrome is mainly described following conditioning regiment with busulfan, cyclophosphamide and/or total body irradiation. CASE REPORTS: We report for the first time two cases of sinusoidal obstruction syndrome occurring lately after BeAM conditioning regiment (bendamustine, etoposide, aracytine, melphalan) for autologous stem cell transplantation in patients treated for malignant lymphoma...
April 16, 2018: La Revue de Médecine Interne
https://www.readbyqxmd.com/read/29669226/gene-therapy-in-patients-with-transfusion-dependent-%C3%AE-thalassemia
#14
Alexis A Thompson, Mark C Walters, Janet Kwiatkowski, John E J Rasko, Jean-Antoine Ribeil, Suradej Hongeng, Elisa Magrin, Gary J Schiller, Emmanuel Payen, Michaela Semeraro, Despina Moshous, Francois Lefrere, Hervé Puy, Philippe Bourget, Alessandra Magnani, Laure Caccavelli, Jean-Sébastien Diana, Felipe Suarez, Fabrice Monpoux, Valentine Brousse, Catherine Poirot, Chantal Brouzes, Jean-François Meritet, Corinne Pondarré, Yves Beuzard, Stany Chrétien, Thibaud Lefebvre, David T Teachey, Usanarat Anurathapan, P Joy Ho, Christof von Kalle, Morris Kletzel, Elliott Vichinsky, Sandeep Soni, Gabor Veres, Olivier Negre, Robert W Ross, David Davidson, Alexandria Petrusich, Laura Sandler, Mohammed Asmal, Olivier Hermine, Mariane De Montalembert, Salima Hacein-Bey-Abina, Stéphane Blanche, Philippe Leboulch, Marina Cavazzana
BACKGROUND: Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer of a marked β-globin (βA-T87Q ) gene could substitute for long-term red-cell transfusions in a patient with β-thalassemia, we wanted to evaluate the safety and efficacy of such gene therapy in patients with transfusion-dependent β-thalassemia...
April 19, 2018: New England Journal of Medicine
https://www.readbyqxmd.com/read/29663421/population-pharmacokinetic-analysis-of-busulfan-in-japanese-pediatric-and-adult-hct-patients
#15
Atsuko Kawazoe, Tomoo Funaki, Seongryul Kim
Busulfan is the most common chemotherapy agent used in allogeneic hematopoietic cell transplant (HCT) conditioning regimens. As narrow therapeutic index and interpatient variability exists in the effectiveness and toxicity of conditioning regimens, personalizing intravenous busulfan therapy is desirable. Population pharmacokinetic-based approaches have been applied to therapeutic drug monitoring for the purpose of personalizing therapy. A population pharmacokinetic analysis with the objective of personalizing therapy in Japanese patients was conducted by integrating pediatric patient data with adult patient data...
April 17, 2018: Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/29660493/effect-of-absolute-monocyte-count-post-transplant-on-the-outcome-of-patients-with-acute-myeloid-leukemia-undergoing-myeloablative-allogeneic-hematopoietic-stem-cell-transplant-with-busulfan-and-cyclophosphamide-conditioning
#16
Liyuan Tang, Na Wang, Chongyun Xing, Qiang Zhuang, Bin Liang, Lan Sun, Yi Chen, Yan Qian, Zhijian Shen, Songfu Jiang, Kang Yu, Jianhua Feng
Peripheral monocytes have recently been evaluated as a prognostic factor in different types of hematological malignancies. This study assessed the prognostic value of absolute monocyte count (AMC) post-transplant on the clinical outcomes of 59 patients with acute myeloid leukemia (AML) who had undergone myeloablative conditioning (MAC) allogeneic hematopoietic stem cell transplant (allo-HSCT) with busulfan and cyclophosphamide (Bu/Cy). Kaplan-Meier analysis showed that patients with a high AMC (≥ 0.57 × 109 /L) on post-transplant day (PTD) 15 had a significantly worse overall survival (OS) compared to patients with a low AMC (< 0...
April 11, 2018: Leukemia Research
https://www.readbyqxmd.com/read/29644709/optimizing-the-pretransplant-regimen-for-autologous-stem-cell-transplantation-in-acute-myelogenous-leukemia-better-outcomes-with-busulfan-and-melphalan-compared-with-busulfan-and-cyclophosphamide-in-high-risk-patients-autografted-in-first-complete-remission
#17
Norbert Claude Gorin, Myriam Labopin, Didier Blaise, Pierre-Yves Dumas, Thomas Pabst, Silvia Maria Trisolini, William Arcese, Mohamed Houhou, Mohamad Mohty, Arnon Nagler
Autologous stem cell transplantation (ASCT) remains a clinical option to consolidate some adult patients with acute myelogenous leukemia (AML) in first complete remission (CR1). In a small cohort of patients, we have previously shown better outcomes following Busulfan and Melphalan (BUMEL) over Busulfan and Cyclophosphamide (BUCY). To identify the subpopulations that might get the highest benefit with BUMEL, we designed a larger study. All adult patients with primary AML and available cytogenetics, autografted from January 2000 to December 2016 in CR1, were included: 1137 patients received BUCY and 512 BUMEL...
April 12, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29621063/posttransplant-cyclophosphamide-for-hla-haploidentical-transplantation-in-patients-with-mucopolysaccharidosis
#18
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/29608607/pharmacokinetics-adapted-busulfan-based-myeloablative-conditioning-before-unrelated-umbilical-cord-blood-transplantation-for-myeloid-malignancies-in-children
#19
Joy Benadiba, Marc Ansari, Maja Krajinovic, Marie-France Vachon, Michel Duval, Pierre Teira, Sonia Cellot, Henrique Bittencourt
Unrelated umbilical cord blood transplantation (UCBT) is an alternative to provide transplants in children with acute leukemia or myelodysplastic syndrome who lack a related donor. Intravenous Busulfan (Bu) combined with therapeutic drug monitoring-guided dosing has been increasingly used, with more predictable bioavailability and better outcomes comparing to oral Bu. There is still an important variation in Bu pharmacokinetic between patients that is associated with an increased risk of toxicity and graft failure...
2018: PloS One
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
#20
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
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