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Bone marrow engraftment

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https://www.readbyqxmd.com/read/28092353/non-myeloablative-conditioning-for-second-hematopoietic-cell-transplantation-for-graft-failure-in-patients-with-non-malignant-disorders-a-prospective-study-and-review-of-the-literature
#1
K Mallhi, P J Orchard, W P Miller, Q Cao, J Tolar, T C Lund
Allogeneic hematopoietic cell transplantation (HCT) effectively treats several non-malignant disorders such as selected lysosomal disorders, cerebral adrenoleukodystrophy and hemoglobinopathies. However, rates of graft failure (GF) in non-malignant populations exceed those of patients with malignant indications for HCT. Salvage conditioning regimens and outcomes for second HCT for GF vary immensely in the literature. We report 17 consecutive pediatric patients with non-malignant disorders who underwent a second allogenic HCT for GF using a non-myeloablative, low-dose busulfan-based regimen...
January 16, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28079980/induction-of-mesenchymal-stem-cell-differentiation-in-the-absence-of-soluble-inducer-for-cutaneous-wound-regeneration-by-a-chitin-nanofibers-based-hydrogel
#2
Kangquan Shou, Yao Huang, Baiwen Qi, Xiang Hu, Zhanjun Ma, Ang Lu, Chao Jian, Lina Zhang, Aixi Yu
Transplantation of bone marrow mesenchymal stem cells (BMSCs) has been considered as a promising strategy for wound healing. However, poor viability of engrafted BMSCs and limited capabilities of differentiation into the desired cell types in wounds often hinder their application. Few studies report the induction of BMSCs differentiation into the skin regeneration related cell types using natural biopolymer, e.g., chitin and its derivative. Here we utilized a chitin nanofibers(CNFs) hydrogel as a directive cue to induce BMSCs differentiation for enhancing cutaneous wound regeneration in the absence of cell-differentiating factors...
January 12, 2017: Journal of Tissue Engineering and Regenerative Medicine
https://www.readbyqxmd.com/read/28067886/dose-adapted-post-transplant-cyclophosphamide-for-hla-haploidentical-transplantation-in-fanconi-anemia
#3
M S Thakar, C Bonfim, M C Walters, R Storb, R Pasquini, L Burroughs, B M Sandmaier, A Woolfrey, H-P Kiem
We developed a haploidentical transplantation protocol with post-transplant cyclophosphamide (CY) for in vivo T-cell depletion (TCD) using a novel adapted-dosing schedule (25 mg/kg on days +3 and +4) for Fanconi anemia (FA). With median follow-up of 3 years (range, 37 days to 6.2 years), all six patients engrafted. Two patients with multiple pre-transplant comorbidities died, one from sepsis and one from sepsis with associated chronic GVHD. Four patients without preexisting comorbidities and early transplant referrals are alive with 100% donor chimerism and excellent performance status...
January 9, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28067880/a-phase-i-ii-trial-of-intrabone-marrow-cord-blood-transplantation-and-comparison-of-the-hematological-recovery-with-the-japanese-nationwide-database
#4
N Kurita, M Gosho, Y Yokoyama, T Kato, N Obara, M Sakata-Yanagimoto, Y Hasegawa, N Uchida, S Takahashi, Y Kouzai, Y Atsuta, M Kurata, T Ichinohe, S Chiba
Intrabone marrow cord blood transplantation (IB-CBT) was proposed as a promising treatment modality to improve hematological recovery. However, clinical advantages of IB-CBT over conventional IV CBT have been unclear. We conducted a prospective single-center trial of IB-CBT to evaluate its safety and superiority in terms of hematological recovery. Fifteen adults with hematological malignancies were enrolled. A thawed and unwashed single cord blood unit was injected into the bilateral superior-posterior iliac crests under local anesthesia...
January 9, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28067873/cotransplantation-of-bone-marrow-derived-mesenchymal-stem-cells-in-haploidentical-hematopoietic-stem-cell-transplantation-in-patients-with-severe-aplastic-anemia-an-interim-summary-for-a-multicenter-phase-ii-trial-results
#5
Z Liu, Y Zhang, H Xiao, Z Yao, H Zhang, Q Liu, B Wu, D Nie, Y Li, Y Pang, Z Fan, L Li, Z Jiang, F Duan, H Li, P Zhang, Y Gao, L Ouyang, C Yue, M Xie, C Shi, Y Xiao, S Wang
Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for severe aplastic anemia (SAA) is mainly limited by the high incidence of graft failure and GvHD. Mesenchymal stem cells (MSCs) have been shown to support hematopoiesis in vivo and to display potent immunosuppressive effects to prevent or treat GvHD after HSCT. In a multicenter phase II trial, we developed an approach with co-transplantation of MSCs in patients undergoing haplo-HSCT. Forty-four patients with SAA were included. The conditioning regimen included busulfan, cyclophosphamide and thymoglobulin (ATG)...
January 9, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28067165/gene-therapy-in-fanconi-anemia-a-matter-of-time-safety-and-gene-transfer-tool-efficiency
#6
Verhoeyen Els, Francisco José Román Rodríguez, François-Loïc Cosset, Camille Lévy, Paula Rio
Fanconi anemia (FA) is a rare genetic syndrome characterized by progressive marrow failure. Gene therapy by infusion of FA-corrected autologous hematopoietic stem cells (HSCs) may offer a potential cure since it is a monogenetic disease with mutations in the FANC genes, coding for DNA repair enzymes (See review[1]). However, the collection of hCD34 +-cells in FA patients implies particular challenges because of the reduced numbers of progenitor cells present in their bone marrow (BM)[2] or mobilized peripheral blood[3-5]...
January 9, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28062216/post-transplant-cyclophosphamide-after-bone-marrow-transplantation-is-not-associated-with-an-increased-risk-of-donor-derived-malignancy
#7
Robbie G Majzner, Huzefa Mogri, Ravi Varadhan, Patrick Brown, Kenneth R Cooke, Javier Bolaños-Meade, Lode Swinnen, Jennifer Kanakry, Leo Luznik, Rick Jones, Ephraim Fuchs, Rich Ambinder, Yvette Kasamon, Heather J Symons
Post-transplant cyclophosphamide (PTCy) can be used for graft versus host disease (GVHD) prophylaxis alone or in combination with other agents and is associated with excellent rates of engraftment, acute and chronic GVHD, and an absence of post-transplant lymphoproliferative disease. No study has previously evaluated the risk for developing donor derived malignancy (DDM) in patients who receive PTCy. Giving chemotherapy in the immediate post-transplant period carries with it a theoretic risk of disturbing the graft at a time of increased hematopoietic stress and causing or accelerating the development of malignancy...
January 3, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28053635/relapse-of-biphenotypic-acute-leukemia-as-a-breast-mass
#8
Hee-Chul Shin
In acute leukemia, leukemic infiltration of the breast is extremely rare. We report a case of biphenotypic acute leukemia (BAL) that presented as a breast mass. A 30-year-old woman presented with a 4-month history of a right breast mass with nipple discharge and easy fatigue. She had received chemotherapy and peripheral blood stem cell transplantation for BAL and had been in complete remission for the last 2 years. Core needle biopsy of the breast mass revealed monomorphous infiltrates of blast cells with round nuclei and fine chromatin, consistent with leukemic infiltration...
December 2016: Journal of Breast Cancer
https://www.readbyqxmd.com/read/28041853/stem-cells-in-male-sexual-dysfunction-are-we-getting-somewhere
#9
REVIEW
Mohammad Ayodhia Soebadi, Uros Milenkovic, Emmanuel Weyne, Fabio Castiglione, Maarten Albersen
INTRODUCTION: Stem cells for sexual disorders are steadily being introduced into clinical trials. Two conditions of importance are the main target for this line of treatment, especially when regarding the wide array of translational and basic science highlighting the potential advantages of regenerative therapy: erectile dysfunction (ED) and more recently Peyronie disease (PD). Cellular therapy offers a treatment modality that might reverse disease progression. It would be used in a curative setting, in contrast to other pharmaceutical agents that are currently available...
December 29, 2016: Sexual Medicine Reviews
https://www.readbyqxmd.com/read/28026131/sphingosine-1-phosphate-receptor-3-supports-hematopoietic-stem-and-progenitor-cell-residence-within-the-bone-marrow-niche
#10
Molly E Ogle, Claire E Olingy, Anthony O Awojoodu, Anusuya Das, Rafael A Ortiz, Hoi Yin Cheung, Edward A Botchwey
Hematopoietic stem and progenitor cells (HSPCs) egress from bone marrow during homeostasis and at increased rates during stress; however, the mechanisms regulating their trafficking remain incompletely understood. Here we describe a novel role for lipid receptor, sphingosine-1-phosphate receptor 3 (S1PR3), in HSPC residence within the bone marrow niche. HSPCs expressed increased levels of S1PR3 compared to differentiated bone marrow cells. Pharmacological antagonism or knockout of S1PR3 mobilized HSPCs into blood circulation, suggesting that S1PR3 influences niche localization...
December 27, 2016: Stem Cells
https://www.readbyqxmd.com/read/28024490/-allogeneic-hematopoietic-stem-cell-transplantation-for-treatment-of-t-cell-lymphoblastic-lymphoma-clinical-observation-of-10-cases
#11
Fang Gong, Heng Chen, Lin-Yan Zhao, Tong Wang, Ri Zhang, De-Pei Wu
OBJECTIVE: To analyze the result of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for treatment of patients with T cell Lymphoblastic lymphoma(T-LBL). METHODS: The engraftment, graft versus host disease (GVHD), infection, relapse and survival of 10 T-LBL patients received allo-HSCT was observed. The clinical outcome of allo-HSCT for T-LBL patients was analyzed. RESULTS: The median age of patients was 25 years old, 10 (6 males and 4 females) T-LBL patients received allo-HSCT including 3 from HLA-matched unrelated donors, 3 from HLA-matched sibling donors, 2 from HLA haploidentical sibling donors, and 2 from haploidentical related donors...
December 2016: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/28018447/targeted-busulfan-and-fludarabine-based-conditioning-for-bone-marrow-transplantation-in-chronic-granulomatous-disease
#12
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/28013015/alternative-donor-transplantation-with-high-dose-post-transplantation-cyclophosphamide-for-refractory-severe-aplastic-anemia
#13
Amy E DeZern, Marianna Zahurak, Heather Symons, Kenneth Cooke, Richard J Jones, Robert A Brodsky
Severe aplastic anemia (SAA) is a life-threatening hematopoietic stem cell disorder that is treated with bone marrow transplantation (BMT) or immunosuppressive therapy (IST). The management of patients with refractory SAA after IST is a major challenge. Alternative donor BMT is best chance for cure in refractory SAA, but morbidity and mortality from graft failure and complications of graft-versus-host disease (GVHD) have limited enthusiasm for this approach. Here, we employed post-transplantation high-dose cyclophosphamide in an effort to safely expand the donor pool in 16 consecutive patients with refractory SAA who did not have a matched sibling donor...
December 21, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28009288/engineered-murine-hscs-reconstitute-multi-lineage-hematopoiesis-and-adaptive-immunity
#14
Yi-Fen Lu, Patrick Cahan, Samantha Ross, Julie Sahalie, Patricia M Sousa, Brandon K Hadland, Wenqing Cai, Erik Serrao, Alan N Engelman, Irwin D Bernstein, George Q Daley
Hematopoietic stem cell (HSC) transplantation is curative for malignant and genetic blood disorders, but is limited by donor availability and immune-mismatch. Deriving HSCs from patient-matched embryonic/induced-pluripotent stem cells (ESCs/iPSCs) could address these limitations. Prior efforts in murine models exploited ectopic HoxB4 expression to drive self-renewal and enable multi-lineage reconstitution, yet fell short in delivering robust lymphoid engraftment. Here, by titrating exposure of HoxB4-ESC-HSC to Notch ligands, we report derivation of engineered HSCs that self-renew, repopulate multi-lineage hematopoiesis in primary and secondary engrafted mice, and endow adaptive immunity in immune-deficient recipients...
December 20, 2016: Cell Reports
https://www.readbyqxmd.com/read/28008146/autologous-reconstitution-of-human-cancer-and-immune-system-in-vivo
#15
Juan Fu, Rupashree Sen, David L Masica, Rachel Karchin, Drew Pardoll, Vonn Walter, D Neil Hayes, Christine H Chung, Young J Kim
Correlative studies from checkpoint inhibitor trials have indicated that better understanding of human leukocytic trafficking into the human tumor microenvironment can expedite the translation of future immune-oncologic agents. In order to directly characterize signaling pathways that can regulate human leukocytic trafficking into the tumor, we have developed a completely autologous xenotransplantation method to reconstitute the human tumor immune microenvironment in vivo. We were able to genetically mark the engrafted CD34+ bone marrow cells as well as the tumor cells, and follow the endogenous leukocytic infiltration into the autologous tumor...
December 19, 2016: Oncotarget
https://www.readbyqxmd.com/read/27998927/niche-wnt5a-regulates-the-actin-cytoskeleton-during-regeneration-of-hematopoietic-stem-cells
#16
Christina Schreck, Rouzanna Istvánffy, Christoph Ziegenhain, Theresa Sippenauer, Franziska Ruf, Lynette Henkel, Florian Gärtner, Beate Vieth, M Carolina Florian, Nicole Mende, Anna Taubenberger, Áine Prendergast, Alina Wagner, Charlotta Pagel, Sandra Grziwok, Katharina S Götze, Jochen Guck, Douglas C Dean, Steffen Massberg, Marieke Essers, Claudia Waskow, Hartmut Geiger, Mathias Schiemann, Christian Peschel, Wolfgang Enard, Robert A J Oostendorp
Here, we show that the Wnt5a-haploinsufficient niche regenerates dysfunctional HSCs, which do not successfully engraft in secondary recipients. RNA sequencing of the regenerated donor Lin(-) SCA-1(+) KIT(+) (LSK) cells shows dysregulated expression of ZEB1-associated genes involved in the small GTPase-dependent actin polymerization pathway. Misexpression of DOCK2, WAVE2, and activation of CDC42 results in apolar F-actin localization, leading to defects in adhesion, migration and homing of HSCs regenerated in a Wnt5a-haploinsufficient microenvironment...
January 2017: Journal of Experimental Medicine
https://www.readbyqxmd.com/read/27991893/cord-blood-transplantation-rewind-to-fast-forward
#17
REVIEW
A Dahlberg, F Milano
The utilization of cord blood as a source of stem cells for transplantation has decreased in recent years. Although cord blood transplantation (CBT) is an established practice for the treatment of adult and pediatric patients with hematological malignancies, the high acquisition cost of CB units along with high transplant-related mortality due to delayed hematopoietic recovery and immune reconstitution have contributed to the slowing in widespread adoption of CBT. Strategies aimed to enhance speed of engraftment and ongoing clinical trials are investigating ways to make CBT more widely available...
December 19, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27982316/mobilization-of-hematopoietic-progenitor-cells-for-autologous-transportation-consensus-recommendations
#18
Fernando Barroso Duarte, Benedito de Pina Almeida Prado, Garles Miller Matias Vieira, Luciano J Costa
Selected patients with certain hematological malignancies and solid tumors have the potential to achieve long-term survival with autologous hematopoietic progenitor cell transplant. The collection of these cells in peripheral blood avoids multiple bone marrow aspirations, results in faster engraftment and allows treatment of patients with infection, fibrosis, or bone marrow hypocellularity. However, for the procedure to be successful, it is essential to mobilize a sufficient number of progenitor cells from the bone marrow into the blood circulation...
October 2016: Revista da Associação Médica Brasileira
https://www.readbyqxmd.com/read/27980777/improved-b-cell-development-in-humanized-nod-scid-il2r%C3%AE-null-mice-transgenically-expressing-human-stem-cell-factor-granulocyte-macrophage-colony-stimulating-factor-and-interleukin-3
#19
Sonal Jangalwe, Leonard D Shultz, Anuja Mathew, Michael A Brehm
INTRODUCTION: Immunodeficient mice engrafted with human immune systems support studies of human hematopoiesis and the immune response to human-specific pathogens. A significant limitation of these humanized mouse models is, however, a severely restricted ability of human B cells to undergo class switching and produce antigen-specific IgG after infection or immunization. METHODS: In this study, we have characterized the development and function of human B cells in NOD-scid IL2Rγ(null) (NSG) mice transgenically expressing human stem cell factor (SCF), granulocyte macrophage colony-stimulating factor (GM-CSF), and IL-3 (NSG-SGM3) following engraftment with human hematopoietic stem cells, autologous fetal liver, and thymic tissues (bone marrow, liver, thymus or BLT model)...
December 2016: Immunity, Inflammation and Disease
https://www.readbyqxmd.com/read/27965305/bone-marrow-cell-trafficking-analyzed-by-89zr-oxine-positron-emission-tomography-in-a-murine-transplantation-model
#20
Kingsley O Asiedu, Sho Koyasu, Lawrence P Szajek, Peter L Choyke, Noriko Sato
PURPOSE: The success of hematopoietic stem cell transplantation (HSCT) depends on donor cell homing to the bone marrow (BM). However, there is no reliable method of noninvasively monitoring the kinetics and distribution of transferred cells. Using Zirconium-89 ((89)Zr)-oxine cell labeling combined with positron emission tomography (PET) imaging, we sought to visualize and quantify donor cell homing in a mouse BM transplantation model. EXPERIMENTAL DESIGN: The effect of (89)Zr-oxine labeling on BM cell viability and differentiation was evaluated in vitro...
December 13, 2016: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
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