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Haploidentical stem cell transplantation

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https://www.readbyqxmd.com/read/29456531/first-occurrence-of-plasmablastic-lymphoma-in-adenosine-deaminase-deficient-severe-combined-immunodeficiency-disease-patient-and-review-of-the-literature
#1
Maddalena Migliavacca, Andrea Assanelli, Maurilio Ponzoni, Roberta Pajno, Federica Barzaghi, Fabio Giglio, Francesca Ferrua, Marta Frittoli, Immacolata Brigida, Francesca Dionisio, Roberto Nicoletti, Miriam Casiraghi, Maria Grazia Roncarolo, Claudio Doglioni, Jacopo Peccatori, Fabio Ciceri, Maria Pia Cicalese, Alessandro Aiuti
Adenosine deaminase-deficient severe combined immunodeficiency disease (ADA-SCID) is a primary immune deficiency characterized by mutations in the ADA gene resulting in accumulation of toxic compounds affecting multiple districts. Hematopoietic stem cell transplantation (HSCT) from a matched donor and hematopoietic stem cell gene therapy are the preferred options for definitive treatment. Enzyme replacement therapy (ERT) is used to manage the disease in the short term, while a decreased efficacy is reported in the medium-long term...
2018: Frontiers in Immunology
https://www.readbyqxmd.com/read/29448057/hla-matched-sibling-vs-unrelated-vs-haploidentical-related-donor-allogeneic-hematopoietic-stem-cell-transplantation-for-patients-aged-over-60-years-with-acute-myeloid-leukemia-a-single-center-donor-comparison
#2
Raynier Devillier, Faezeh Legrand, Jérôme Rey, Luca Castagna, Sabine Fürst, Angela Granata, Aude Charbonnier, Samia Harbi, Evelyne d'Incan, Thomas Pagliardini, Catherine Faucher, Claude Lemarie, Colombe Saillard, Boris Calmels, Bilal Mohty, Valerio Maisano, Pierre-Jean Weiller, Christian Chabannon, Norbert Vey, Didier Blaise
Haploidentical related donor (HRD) allogeneic hematopoietic stem cell transplantation (Allo-HSCT) was developed as a valid option for the treatment of acute myeloid leukemia (AML) in the absence of a matched donor. However, many investigators are reluctant to consider the use of this alternative in elderly patients, anticipating high morbidity. Here, we report a single-center comparison of HRD vs. matched sibling donor (MSD) and unrelated donor (UD) Allo-HSCT for patients with AML aged ≥60 years. Ninety-four patients (MSD: n = 31; UD: n = 30; HRD: n = 33) were analyzed...
February 12, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29444931/complete-remission-with-reduction-of-high-risk-clones-following-haploidentical-nk-cell-therapy-against-mds-and-aml
#3
Andreas T Björklund, Mattias Carlsten, Ebba Sohlberg, Lisa L Liu, Trevor Clancy, Mohsen Karimi, Sarah Cooley, Jeffrey S Miller, Monika Klimkowska, Marie Schaffer, Emma Watz, Kristina I Wikstrom, Pontus Blomberg, Bjorn E Wahlin, Marzia Palma, Lotta Hansson, Per Ljungman, Eva Hellström-Lindberg, Hans-Gustaf Ljunggren, Karl-Johan Malmberg
PURPOSE: To evaluate the safety, efficacy and immunobiological correlates of allogeneic NK cell-based therapy in primary chemotherapy-refractory or relapsed high-risk myelodysplastic syndrome (MDS), secondary AML (MDS/AML), and de novo AML patients. EXPERIMENTAL DESIGN: Sixteen patients received fludarabine/cyclophosphamide conditioning combined with total lymphoid irradiation followed by adoptive immunotherapy with IL-2-activated haploidentical NK cells. RESULTS: NK cell infusions were well tolerated with only transient adverse events observed in the 16 patients...
February 14, 2018: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/29442161/allogeneic-hematopoietic-stem-cell-transplantation-in-primary-cutaneous-t-cell-lymphoma
#4
Laura Cudillo, Raffaella Cerretti, Alessandra Picardi, Benedetta Mariotti, Gottardo De Angelis, Maria Cantonetti, Massimiliano Postorino, Eleonora Ceresoli, Giovanna De Santis, Daniela Nasso, Francesco Pisani, Enrico Scala, Fabio Di Piazza, Alessandro Lanti
In our retrospective study, 16 patients affected by advanced cutaneous T cell lymphoma (CTCL) underwent allogeneic hematopoietic stem cell transplantation (HSCT). Two patients (12.5%) were in complete remission (CR), nine (56.3%) in partial remission (PR), and five (31.2%) with active disease. The patients were transplanted from an HLA-identical (n = 7) from a mismatched (n = 1) or haploidentical (n = 1) sibling, from matched unrelated donor (n = 5), or from a single cord blood unit (n = 2). Conditioning regimen was standard myeloablative in 6 patients and at reduced intensity in 10...
February 13, 2018: Annals of Hematology
https://www.readbyqxmd.com/read/29423758/impact-of-pre-transplantation-minimal-residual-disease-determined-by-multiparameter-flow-cytometry-on-the-outcome-of-aml-patients-with-flt3-itd-after-allogeneic-stem-cell-transplantation
#5
Xiaosu Zhao, Zhidong Wang, Guorui Ruan, Yanrong Liu, Yu Wang, Xiaohui Zhang, Lanping Xu, Xiaojun Huang, Yingjun Chang
In this study, using multiparameter flow cytometry (FCM), we investigate the impact of minimal residual disease prior to transplantation (pre-MRD) on the transplant outcomes of AML patients with fms-related tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) mutation. A total of 20 patients who received HLA-matched sibling donor transplantation (MSDT) and 63 patients who received unmanipulated haploidentical hematopoietic stem cell transplantation (haplo-HSCT) were enrolled. Patients were classified into four groups based on the status of pre-FCM: group 1 with positive pre-FCM before MSDT, group 2 with negative pre-FCM before MSDT, group 3 with positive pre-FCM before haplo-HSCT, and group 4 with positive pre-FCM before haplo-HSCT...
February 8, 2018: Annals of Hematology
https://www.readbyqxmd.com/read/29416633/allogeneic-hematopoietic-stem-cell-transplantation-for-relapsed-acute-myeloid-leukemia-in-eto-positive-with-reduced-intensity-conditioning
#6
Zhi Guo, Chen Xu, Hu Chen
Objective: This research is conducted under the intention of exploring the efficacy and safety of reduced-intensity conditioning for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of relapsed ETO positive acute myeloid leukemia (AML). Materials and Methods: Treatment of 15 cases referring to recurrent ETO positive acute myeloid leukemia in an army hospital from January 2010 to January 2013 through allo-HSCT with reduced-intensity conditioning...
January 2, 2018: Oncotarget
https://www.readbyqxmd.com/read/29412158/allogeneic-hematopoietic-stem-cell-transplantation-for-gata2-deficiency-using-a-busulfan-based-regimen
#7
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 includes 2 matched related donor (MRD) recipients, 13 matched unrelated donor (URD) recipients, and 7 haploidentical related donor (HRD) recipients...
February 2, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29410500/optimal-donor-for-severe-aplastic-anemia-patient-requiring-allogeneic-hematopoietic-stem-cell-transplantation-a-large-sample-study-from-china
#8
Yunjing Zeng, Sanbin Wang, Jishi Wang, Li Liu, Yi Su, Zhixiang Lu, Xuemei Zhang, Yanqi Zhang, Jiang Fan Zhong, Lihui Peng, Qiang Liu, Yinghao Lu, Lei Gao, Xi Zhang
HLA-haploidentical hematopoietic stem cell transplantation (HSCT) may be an option for severe aplastic anemia (SAA) patients. However, to date, no large-sample studies have been performed to determine which types of SAA patients are suitable for HLA-haploidentical HSCT. We retrospectively studied 189 consecutive patients with SAA who underwent HLA-identical or HLA-haploidentical HSCT at seven transplant centers in China. Propensity score matching (PSM) was applied in this study to reduce the influence of potential confounders...
February 6, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29410343/single-antigen-mismatched-unrelated-hematopoietic-stem-cell-transplantation-using-high-dose-post-transplantation-cyclophosphamide-is-a-suitable-alternative-for-patients-lacking-hla-matched-donors
#9
Ana Sofia Jorge, María Suárez-Lledó, Arturo Pereira, Gonzalo Gutierrez, Francesc Fernández-Avilés, Laura Rosiñol, Noemí Llobet, Teresa Solano, Álvaro Urbano-Ispízua, Montserrat Rovira, Carmen Martínez
The optimal prophylaxis regimen for graft-versus-host disease (GVHD) in the setting of mismatched unrelated donor (MMUD) allogeneic hematopoietic stem cell transplantation (alloSCT) is not defined. The use of high-dose post-transplant cyclophosphamide (PTCy) in haploidentical transplantation has proven feasible and effective in overcoming the negative impact of HLA-disparity on survival. We hypothesized that PTCy could also be effective in the setting of MMUD transplantation. We retrospectively analyzed 86 consecutive adult recipients of alloHSCT in our institution, comparing two contemporaneous groups: PTCyMMUD (n=26) vs...
February 1, 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
#10
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 1, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29407318/treatment-of-high-risk-acute-myeloid-leukemia-with-cladribine-cytarabine-mitoxantrone-and-granulocyte-colony-stimulating-factor-then-subsequent-bridging-to-myeloablative-allogeneic-hematopoietic-stem-cell-transplantation-a-case-series
#11
J Zhang, Y Sun, X Zhang, B Long, Y Lu, X Li
This report preliminarily evaluates the efficacy and safety of cladribine, cytarabine, mitoxantrone, and granulocyte colony-stimulating factor (CLAG-M) as bridging therapy to myeloablative allogeneic hematopoietic cell transplantation (allo-HCT) in the treatment of patients with refractory or relapsed acute myeloid leukemia. Five patients with high-risk refractory or relapsed acute myeloid leukemia received the CLAG-M regimen and subsequent bridging to myeloablative allo-HCT between December 2014 and August 2015 in our hospital...
January 2018: Transplantation Proceedings
https://www.readbyqxmd.com/read/29406585/erratum-chang-y-j-wang-y-mo-x-d-et-al-optimal-dose-of-rabbit-thymoglobulin-in-conditioning-regimens-for-unmanipulated-haploidentical-hematopoietic-stem-cell-transplantation-long-term-outcomes-of-a-prospective-randomized-trial-cancer-2017-123-2881-2892
#12
https://www.readbyqxmd.com/read/29398342/source-of-stem-cells-in-haploidentical-transplantation
#13
Manjulika Das
No abstract text is available yet for this article.
February 1, 2018: Lancet Oncology
https://www.readbyqxmd.com/read/29383623/nk-cell-therapy-for-hematologic-malignancies
#14
REVIEW
Rohtesh S Mehta, Brion Randolph, May Daher, Katayoun Rezvani
Natural killer (NK) cells are part of the innate immune system and represent the first line of defense against infections and tumors. In contrast to T cells, NK cells do not require prior antigen sensitization to induce cytotoxicity and do not cause graft-versus-host disease. These, along with other advantages, make NK cells an attractive candidate for adoptive cellular therapy. Herein, we describe the mechanisms of NK cell cytotoxicity, which is governed by an intricate balance between various activating and inhibitory receptors, including the killer cell immunoglobulin-like receptors (KIRs)...
January 27, 2018: International Journal of Hematology
https://www.readbyqxmd.com/read/29383224/haploidentical-hematopoietic-stem-cell-transplantation-in-a-myelofibrosis-patient-with-primary-graft-failure
#15
Cristina Tecchio, Angelo Andreini, Claudio Costantini, Alberto Zamò, Donata de Sabata, Fiorenza Aprili, Roberta Galavotti, Emanuele Guardalben, Fabio Benedetti
No abstract text is available yet for this article.
December 22, 2017: Hematology Reports
https://www.readbyqxmd.com/read/29381772/comparison-of-outcomes-in-hematological-malignancies-treated-with-haploidentical-or-hla-identical-sibling-hematopoietic-stem-cell-transplantation-following-myeloablative-conditioning-a-meta-analysis
#16
Dangui Chen, Di Zhou, Dan Guo, Peipei Xu, Bing Chen
PURPOSE: Haploidentical and human leukocyte antigen (HLA)-identical sibling hematopoietic stem transplantation are two main ways used in allogeneic hematopoietic stem cell transplantation (allo-HSCT). In recent years, remarkable progress has been made in haploidentical allo-HSCT (HID-SCT), and some institutions found HID-SCT had similar outcomes as HLA-identical sibling allo-HSCT (ISD-SCT). To clarify if HID-SCT has equal effects to ISD-SCT in hematologic malignancies, we performed this meta-analysis...
2018: PloS One
https://www.readbyqxmd.com/read/29378303/superior-survival-of-black-versus-white-patients-following-post-transplant-cyclophosphamide-based-haploidentical-transplantation-for-adults-with-hematologic-malignancy
#17
Scott R Solomon, Xu Zhang, H Kent Holland, Lawrence E Morris, Melhem Solh, Asad Bashey
Available evidence from large registry studies has shown inferior survival for black adult patients following both unrelated donor and cord blood transplantation. Post-transplant cyclophosphamide (PTCy)-based haploidentical donor transplantation (HIDT) is being increasingly used in ethnic minorities. However, no studies of the impact of race on outcomes following HIDT have been reported. We analyzed 203 consecutive patients (123 white, 80 black) who underwent first HIDT using PTCy for hematologic malignancy at a single institution...
January 25, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29373243/haploidentical-stem-cell-transplantation-in-a-boy-with-chronic-granulomatous-disease
#18
A Regueiro-García, S Fariña-Nogueira, J Á Porto-Arceo, J M Couselo-Sánchez
Chronic granulomatous disease is a primary immunodeficiency caused by mutations in any one of the five components of the NADPH oxidase in phagocytic leucocytes. This causes impaired microbial killing, which leads to severe life-threatening bacterial and fungal infections. Currently, allogenic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for chronic granulomatous disease, although gene therapy may provide a new therapeutic option for the treatment of patients with CGD. Haploidentical HSCT provides a potentially curative treatment option for patients who lack a suitably HLA-matched donor, but only a few cases have been reported in the literature...
January 16, 2018: Allergologia et Immunopathologia
https://www.readbyqxmd.com/read/29371943/tumorablative-conditioning-regimen-for-haploidentical-stem-cell-transplantation-in-102-children-with-hematologic-malignancies-a-single-center-experience
#19
Luo Rongmu, Zhang Xiaomei, Du Zhenlan, Wang Ya, Chen Wei, Si Yingjian, Gu Wenjing, Xing Guosheng, Wang Yang, Da Wanming
Haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) is widely carried out in China, and transplantation related complications decreased gradually with the transplant technology improving, and the overall survival(OS) increased year by year. However, relapse after transplantation is still one of the main causes of death in patients with hematological malignancy. In order to reduce the recurrence after HSCT, we set a tumorablative conditioning regimen (TAC ) regimen; the aim is as much as possible to eliminate the malignant clone to reduce the recurrence without increasing the conditioning toxicity...
December 26, 2017: Oncotarget
https://www.readbyqxmd.com/read/29360162/bone-marrow-versus-mobilized-peripheral-blood-stem-cells-in-haploidentical-transplants-using-posttransplantation-cyclophosphamide
#20
Annalisa Ruggeri, Myriam Labopin, Andrea Bacigalupo, Zafer Gülbas, Yener Koc, Didier Blaise, Benedetto Bruno, Giuseppe Irrera, Johanna Tischer, Jose Luiz Diez-Martin, Luca Castagna, Fabio Ciceri, Mohamad Mohty, Arnon Nagler
BACKGROUND: Incidence of graft-versus-host disease (GVHD) in haploidentical bone marrow (BM) transplants using posttransplantion cyclophosphamide (PT-Cy) is low, whereas GVHD using mobilized peripheral blood stem cells (PBSC) ranges between 30% and 40%. METHODS: To evaluate the effect of stem cell source in haploidentical transplantation with PT-Cy, we analyzed 451 patients transplanted for acute myeloid leukemia or acute lymphoblastic leukemia reported to the European Society for Blood and Marrow Transplantation...
January 23, 2018: Cancer
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