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Hematopietic cell transplantation

Selami Demirci, Naoya Uchida, John F Tisdale
Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates, but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies. Ex vivo gene therapy through globin gene addition has been investigated extensively and is currently being tested in clinical trials that have begun reporting encouraging data...
May 30, 2018: Cytotherapy
Araci M Sakashita, Andrea T Kondo, Ana Paula H Yokoyama, Sanny M C Lira, Carolina B Bub, Aline M Souza, Andrea N F Cipolletta, Kelen C Alvarez, Nelson Hamerschlak, Jose M Kutner, Carlos S Chiattone
BACKGROUND: Autologous peripheral blood hematopoietic stem cell (PBSC) collection efficiency (CE) is reportedly affected by the patient's blood properties; however, studies to identify factors correlated with CE have shown inconsistent results. Additionally, variables such as stem cell graft granulocyte content and patient age, sex, and underlying disease, may be associated with hematopietic stem cell (HSC) infusion-related adverse reactions. In this study, we evaluated the correlation of preleukapheresis PB granulocyte count and PBSC harvest variables with CD34+ collection yield and efficiency, and thawed HSC infusion side effect occurrence...
January 19, 2018: Journal of Clinical Apheresis
Xiaoli Wang, David Haylock, Cing Siang Hu, Wioleta Kowalczyk, Tianbo Jiang, Jiajing Qiu, Goar Mosoyan, Wu He, Netonia Marshall, John Mascarenhas, Anna Tarasova, Joshua Brody, David Winkler, Ronald Hoffman
Recently, interactions between thrombopoietin (TPO) and its receptor, the myeloproliferative leukemia (MPL) virus oncogene, have been shown to play a role in the development and progression of myeloproliferative neoplasms including myelofibrosis (MF). These observations have led to the development of strategies to disrupt the association of TPO with its receptor as a means of targeting MF hematopoietic stem cells (HSCs) and hematopoietic progenitor cells (HPCs). In this report, we show that although both splenic and peripheral blood MF CD34(+) cells expressed lower levels of MPL than normal CD34(+) cells, TPO promoted the proliferation of MF CD34(+) cells and HPCs in a dose-dependent fashion...
June 30, 2016: Blood
Silvia Gimondi, Matteo Dugo, Antonio Vendramin, Anisa Bermema, Giulia Biancon, Alessandra Cavané, Paolo Corradini, Cristiana Carniti
Acute graft-versus-host disease (aGVHD) results in significant morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Noninvasive diagnostic and prognostic tests for aGVHD are currently lacking, but would be beneficial in predicting aGVHD and improving the safety of allo-HSCT. Circulating microRNAs exhibit marked stability and may serve as biomarkers in several clinical settings. Here, we evaluated the use of circulating microRNAs as predictive biomarkers of aGVHD in lymphoma patients after allo-HSCT from matched unrelated donors (MUDs)...
July 2016: Experimental Hematology
Alisa Damnernsawad, Guangyao Kong, Zhi Wen, Yangang Liu, Adhithi Rajagopalan, Xiaona You, Jinyong Wang, Yun Zhou, Erik A Ranheim, Hongbo R Luo, Qiang Chang, Jing Zhang
Previous studies indicate that Kras is dispensable for fetal liver hematopoiesis, but its role in adult hematopoiesis remains unclear. Here, we generated a Kras conditional knockout allele to address this question. Deletion of Kras in adult bone marrow (BM) is mediated by Vav-Cre or inducible Mx1-Cre. We find that loss of Kras leads to greatly reduced thrombopoietin (TPO) signaling in hematopoietic stem cells (HSCs) and multipotent progenitors (MPPs), while stem cell factor-evoked ERK1/2 activation is not affected...
July 2016: Stem Cells
Eva de Berranger, Charlotte Jubert, Gérard Michel
Under the long-term monitoring of patients treated in childhood or adolescence for cancer, we present in this article the long-term monitoring and therefore possible effects of patients who underwent allergenic hematopoietic stem cell transplantation. This article is based on a collaborative effort organized by the French Society of Bone Marrow Transplantation and Cell Therapy (SFGM-TC), which took place during the 4th day of allograft harmonization practices. Patients affected are children and young adults (0-25 years)...
July 2015: Bulletin du Cancer
Xiao-Yun Chen, Yong-Liang Zheng, Yi-Jian Chen
This study was aimed to evaluate the efficacy and safety of imatinib in the treatment of patients with adult Ph chromosome-positive acute lymphoblastic leukemia (Ph(+)ALL). A total of 32 diagnosed adult Ph(+)ALL patients from July 2007 to February 2014 in our hospital were retrospectively analyzed and were divided into two groups: imatinib plus chemotherapy group and traditional chemotherapy group. The differences between two groups were analysed in disease-free survival time (DFS), overall survival time (OS) and toxicity...
December 2014: Zhongguo Shi Yan Xue Ye Xue za Zhi
Ana C T Mercadante, Suelen M Perobelli, Ana P G Alves, Triciana Gonçalves-Silva, Wallace Mello, Ana C Gomes-Santos, Anderson Miyoshi, Vasco Azevedo, Ana M C Faria, Adriana Bonomo
Allogeneic hematopietic stem cell transplantation (aHSCT) is widely used for the treatment of hematologic malignancies. Although aHSCT provides a good response against the malignant cells (graft-versus-leukemia [GVL]), it also leads to the development of graft-versus-host disease (GVHD), a severe disease with high mortality and morbidity rates. Therapy for GVHD is commonly based on nonspecific immunosupression of the transplanted recipient, resulting in the concomitant inhibition of the GVL effect. In this study, we propose an alternative approach to specifically suppress GVHD while sparing the GVL, based on oral treatment of transplant donors with recipient Ags, associated with the intake of probiotic Lactococcus lactis as tolerogenic adjuvant (combined therapy)...
February 15, 2014: Journal of Immunology: Official Journal of the American Association of Immunologists
Francesco Saglio, Patrick J Hanley, Catherine M Bollard
Adoptive immunotherapy-in particular, T-cell therapy-has recently emerged as a useful strategy with the potential to overcome many of the limitations of antiviral drugs for the treatment of viral complications after hematopietic stem cell transplantation. In this review, we briefly summarize the current methods for virus-specific T-cell isolation or selection and we report results from clinical trials that have used these techniques, focusing specifically on the strategies aimed to broaden the application of this technology...
February 2014: Cytotherapy
G Sambuceti, M Massollo, C Marini, M Podestà, C Cassanelli, S Morbelli, F Fiz, A Buschiazzo, S Capitanio, C Augeri, G Curti, M Piana, F Frassoni
AIM: Despite its enormous relevance, homing of hematopoietic stem cells (SCs) remains relatively uncertain due to the limitations of measuring small number of systemically administered cells in the different organs. Despite its high sensitivity, radionuclide detection has been relatively underutilized to this purpose since it cannot differentiate hematopietic SCs recruited by target tissues from those circulating in the blood pool. Our study aims to verify the potential of tracer kinetic approaches in estimating the recruitment of labeled SCs after their systemic administration...
June 2013: Quarterly Journal of Nuclear Medicine and Molecular Imaging
Khalid Ahmed Al-Anazi, Baha Abdalhamid, Zeyad Alshibani, Khalid Awad, Abdullah Alzayed, Hoda Hassan, Mohammed Alsayiegh
Acinetobacter baumannii is a gram-negative, nonfermentative coccobacillus that causes infections in immunocompromised and chronically ill patients and is associated with multidrug resistance. Two days before receiving her nonmyeloablative stem cell allograft, a patient with acute myeloid leukemia developed Acinetobacter baumannii bacteremia that caused septic shock which was successfully treated with imipenem and removal of the central venous catheter. To our knowledge, this is the first report of Acinetobacter baumannii septicemia in a hematopietic stem cell transplantation recipient...
2012: Case Reports in Transplantation
Yixuan Wang, Chen-Guang Zheng, Yonghua Jiang, Jiqin Zhang, Jiayu Chen, Chao Yao, Qingguo Zhao, Sheng Liu, Ke Chen, Juan Du, Ze Yang, Shaorong Gao
The generation of induced pluripotent stem cells (iPSCs) from differentiated somatic cells by over-expression of several transcription factors has the potential to cure many genetic and degenerative diseases currently recalcitrant to traditional clinical approaches. One such genetic disease is β-thalassemia major (Cooley's anemia). This disease is caused by either a point mutation or the deletion of several nucleotides in the β-globin gene, and it threatens the lives of millions of people in China. In the present study, we successfully generated iPSCs from fibroblasts collected from a 2-year-old patient who was diagnosed with a homozygous 41/42 deletion in his β-globin gene...
April 2012: Cell Research
Emanuele Angelucci, Federica Pilo, Clara Targhetta, Martina Pettinau, Cristina Depau, Claudia Cogoni, Sara Usai, Mario Pani, Laura Dessì, Donatella Baronciani
The basis of allogeneic hemopoietic stem cell (HSC) transplantation in thalassemia consists in substituting the ineffective thalassemic erythropoiesis with and allogeneic effective one. This cellular replacement therapy is an efficient way to obtain a long lasting, probably permanent, clinical effective correction of the anaemia avoiding transfusion requirement and subsequent complications like iron overload. The first HSC transplant for thalassemia was performed in Seattle on Dec 2, 1981. In the early eighties transplantation procedure was limited to very few centres worldwide...
2009: Mediterranean Journal of Hematology and Infectious Diseases
Roberta Rizzo, Giacomo Lanzoni, Marina Stignani, Diana Campioni, Francesco Alviano, Francesca Ricci, Pier Luigi Tazzari, Loredana Melchiorri, Sergio Zaccaria Scalinci, Antonio Cuneo, Laura Bonsi, Francesco Lanza, Gian Paolo Bagnara, Olavio R Baricordi
BACKGROUND AIMS: The beneficial activity of mesenchymal stromal cells (MSC) in allogeneic hematopietic stem cell transplantation requires correct use in terms of cell dose and timing of infusion and the identification of biomarkers for selection. The immunosuppressive bone marrow (BM)-derived MSC (BM-MSC) functions have been associated with the production of soluble HLA-G molecules (sHLA-G) via interleukin (IL)-10. We have established a reliable method for evaluating BM-MSC HLA-G expression without the influence of peripheral blood mononuclear cells (PBMC)...
May 2011: Cytotherapy
Yukiko Komeno, Jiro Kitaura, Toshio Kitamura
Myelodysplastic syndrome (MDS) is a clonal disorder of hematopietic stem cells characterized by ineffective hematopoiesis, peripheral blood cytopenia, morphologic dysplasia, and susceptibility to acute myeloid leukemia. Several mechanisms have been suggested as causes of MDS: unbalanced chromosomal abnormalities reflecting a gain or loss of chromosomal material, point mutations of transcription factors, and inactivation of p53. However, appropriate animal models that mimic MDS have long been lacking. We recently reported a novel murine model of MDS that recapitulates trilineage dysplasia and transformation to AML...
June 2009: Journal of Cellular Physiology
Anna Korycka, Ewa Lech-Marańda, Tadeusz Robak
Recently, the search for more effective and safer antineoplastic agents has led to synthesis and introduction into preclinical and clinical studies of a few new purine nucleoside analogues (PNA). Three of them: clofarabine (CAFdA), nelarabine, and forodesine (immucillin H, BCX-1777), despite belonging to the same group of drugs such as PNA, have shown some differences concerning their active forms, metabolic properties and mechanism of action. However, all these drugs have demonstrated promising activity in patients with relapsed and refractory acute lymphoblastic leukemia (ALL)...
June 2008: Recent Patents on Anti-cancer Drug Discovery
Kai-Xun Hu, Shi-Fu Zhao, Mei Guo, Hui-Sheng Ai
The aim of this study was to investigate the effect of mesenchymal stem cells (MSCs) on cell cycle and apoptosis of thymus, spleen and bone marrow cells in mice totally irradiated with sublethal dose, and to explore its mechanisms. BALB/c mice irradiated with 5.5 Gy 60Co gamma-ray were randomly divided into control group and MSC group. Mice in MSC group were infused with 0.4 ml containing 2.5x10(7)/kg of MSCs through tail vein at 1 hour after irradiation. Mice in control group were infused with 0.4 ml normal saline...
December 2007: Zhongguo Shi Yan Xue Ye Xue za Zhi
Yoshihiko Nakamura, Takashi Yahata, Yukari Muguruma, Tomoko Uno, Tadayuki Sato, Hideyuki Matsuzawa, Shunichi Kato, Yukari Shirasugi, Tomomitsu Hotta, Kiyoshi Ando
OBJECTIVE: Hematopoietic stem cells (HSCs) consist of heterogenous subpopulations, one of which is CD34(-) HSCs. Recent development of successful engraftment by intra-bone marrow transplantation revealed severe combined immunodeficiency (scid) mouse-repopulating cell (SRC) activity in human CD34(-) cord blood (CB) cells. On the other hand, CD34(-) cells from bone marrow (BM) cells remain relatively undefined. Here, we investigated pre-SRC populations in human BM CD34(-) cells and the effect of the niche-related factor, angiopoietin-1, on them...
December 2007: Experimental Hematology
Ute Koch, Anne Wilson, Monica Cobas, Rolf Kemler, H Robson Macdonald, Freddy Radtke
Hematopietic stem cells (HSCs) maintain life-long hematopoiesis in the bone marrow via their ability to self-renew and to differentiate into all blood lineages. Although a central role for the canonical wnt signaling pathway has been suggested in HSC self-renewal as well as in the development of B and T cells, conditional deletion of beta-catenin (which is considered to be essential for Wnt signaling) has no effect on hematopoiesis or lymphopoiesis. Here, we address whether this discrepancy can be explained by a redundant and compensatory function of gamma-catenin, a close homolog of beta-catenin...
January 1, 2008: Blood
Y Blanco, A Saiz, M Costa, J F Torres-Peraza, E Carreras, J Alberch, D Jaraquemada, F Graus
A neuroprotective role of inflammation has been suggested based on that immune cells are the main source of brain-derived neurotrophic factor (BDNF). We investigated the 3-year evolution of BDNF levels in serum, CSF and culture supernatant of peripheral blood mononuclear cells (PBMC), unstimulated and stimulated with anti-CD3 and soluble anti-CD28 antibodies, in 14 multiple sclerosis patients who underwent an autologous hematopoietic stem cell transplantation (AHSCT). BDNF levels were correlated with previously reported MRI measures that showed a reduction of T2 lesion load and increased brain atrophy, mainly at first year post-transplant...
May 20, 2005: Neuroscience Letters
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