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https://www.readbyqxmd.com/read/27931749/-hereditary-epidermolysis-bullosa-french-national-guidelines-pnds-for-diagnosis-and-treatment
#1
C Chiaverini, E Bourrat, J Mazereeuw-Hautier, S Hadj-Rabia, C Bodemer, J-P Lacour
Hereditary epidermolysis bullosa (EB) is a heterogeneous group of rare genetic diseases characterized by fragile skin and/or mucous membrane, and it may be either local or generalized. It is caused by mutations in genes encoding different proteins involved mainly in the structure and function of the dermal-epidermal junction. Nineteen genes have so far been identified. They are classified by level of skin cleavage (from top to bottom) into four groups: EB simplex, junctional EB, dystrophic EB and Kindler syndrome...
December 5, 2016: Annales de Dermatologie et de Vénéréologie
https://www.readbyqxmd.com/read/27931505/phase-iii-clinical-trial-assessing-safety-and-efficacy-of-umbilical-cord-blood-mononuclear-cell-transplant-therapy-of-chronic-complete-spinal-cord-injury
#2
Hui Zhu, Waisang Poon, Yansheng Liu, Gilberto Ka-Kit Leung, Yatwa Wong, Yaping Feng, Stephanie C P Ng, Kam Sze Tsang, David T F Sun, David K Yeung, Caihong Shen, Fang Niu, Zhexi Xu, Pengju Tan, Shaofeng Tang, Hongkun Gao, Yun Cha, Kwok-Fai So, Robert Fleischaker, Dongming Sun, John Chen, Jan Lai, Wendy Cheng, Wise Young
Umbilical cord blood-derived mononuclear cell (UCB-MNC) transplants improve recovery in animal spinal cord injury (SCI) models. We transplanted UCB-MNCs into 28 patients with chronic complete SCI in Hong Kong (HK) and Kunming (KM). Stemcyte Inc. donated UCB-MNCs isolated from human leukocyte antigen (HLA 4:6)-matched UCB units. In HK, four patients received four 4-l injections (1.6 million cells) into dorsal entry zones above and below the injury site, and another four received 8-l injections (3.2 million cells)...
November 2016: Cell Transplantation
https://www.readbyqxmd.com/read/27930388/should-patients-with-acute-myeloid-leukemia-and-measurable-residual-disease-be-transplanted-in-first-complete-remission
#3
Francesco Buccisano, Roland B Walter
PURPOSE OF REVIEW: Measurable ('minimal') residual disease in acute myeloid leukemia during first complete morphologic remission (MRD CR1) identifies patients with particularly high relapse risk and short survival. Here, we examine the evidence regarding optimal postremission treatment strategy for such patients. RECENT FINDINGS: With chemotherapy alone or chemotherapy/autologous hematopoietic cell transplantation (HCT), disease recurrence appears inevitable in patients with MRD CR1...
December 7, 2016: Current Opinion in Hematology
https://www.readbyqxmd.com/read/27927231/identification-of-a-potent-small-molecule-capable-of-regulating-polyploidization-megakaryocyte-maturation-and-platelet-production
#4
Nick Huang, Mabel Lou, Hua Liu, Cecilia Avila, Yupo Ma
BACKGROUND: Megakaryocytic cell maturation involves polyploidization, and megakaryocyte (MK) ploidy correlates with their maturation and platelet production. Retardation of MK maturation is closely associated with poor MK engraftment after cord blood transplantation and neonatal thrombocytopenia. Despite the high prevalence of thrombocytopenia in a range of setting that affect infants to adults, there are still very limited modalities of treatment. METHODS: Human CD34(+) cells were isolated from cord blood or bone marrow samples acquired from consenting patients...
December 8, 2016: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/27927023/novel-strategies-for-improving-hematopoietic-reconstruction-after-allogeneic-hematopoietic-stem-cell-transplantation-or-intensive-chemotherapy
#5
Frédéric Baron, Arnon Nagler
High-dose conditioning regimens for allogeneic hematopoietic cell transplantation (allo-HCT) as well as intensive poly-chemotherapy for acute myeloid leukemia (AML) induce prolonged periods of neutropenia. The duration of the neutropenia is particularly long following umbilical cord blood transplantation (UCBT). Areas covered: After briefly reviewing the impact of hematopoietic growth factors administration to hasten hematologic reconstitution after allo-HCT or intensive AML chemotherapy, this article summarizes recent approaches that have been investigated to prompt hematologic reconstruction after UCBT or intensive AML chemotherapy...
December 8, 2016: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/27923274/frequency-of-fetal-maternal-microchimerism-an-analysis-of-the-hla-drb1-gene-in-cord-blood-and-maternal-sample-pairs
#6
Eun Youn Roh, Jong Hyun Yoon, Sue Shin, Eun Young Song, Hye Yoon Chung, Myoung Hee Park
PURPOSE: We aimed to investigate the frequency of fetal-maternal microchimerism among cord blood (CB) from a Korean population. MATERIALS AND METHODS: We previously developed a nested polymerase chain reaction-single-strand conformation polymorphism method for microchimerism detection that is highly sensitive (0.01-0.001%) and specific. We used this method to investigate the frequency of fetal-maternal HLA-DRB1 microchimerism among 153 maternal and 152 CB samples...
December 7, 2016: Journal of Maternal-fetal & Neonatal Medicine
https://www.readbyqxmd.com/read/27919572/non-fucosylated-cb-cd34-cells-represent-a-good-target-for-enforced-fucosylation-to-improve-engraftment-following-cord-blood-transplantation
#7
Simon N Robinson, Michael W Thomas, Paul J Simmons, Junjun Lu, Hong Yang, Jeannie A Javni, Elizabeth J Shpall, Patrick A Zweidler-Mckay
BACKGROUND AIMS: Despite ethnic diversity and ready availability of cryopreserved, human leukocyte antigen-typed cord blood (CB), delayed engraftment remains a significant hurdle to successful CB transplantation. Suboptimal homing of CB hematopoietic stem and progenitor cells (HSPCs) to the hematopoietic microenvironment (HM) is thought to be responsible and due to low levels of HSPC fucosylation. Fucosylation (decoration with sialyl-Lewis(X)) may improve HSPC homing to HM by increasing the strength of HSPC/E-selectin interactions, where E-selectin is constitutively expressed by HM microvasculature...
December 2, 2016: Cytotherapy
https://www.readbyqxmd.com/read/27917700/posterior-reversible-encephalopathy-syndrome-following-elevated-mean-arterial-pressures-for-cervical-spinal-cord-injury
#8
Jeffrey H Zimering, Addisu Mesfin
BACKGROUND: Increasing the mean arterial pressure (MAP) is an accepted treatment modality to minimize the risk for irreversible neurologic damage secondary to spinal cord ischemia. Posterior reversible encephalopathy syndrome (PRES) is a rare complication occurring after transplantation surgery, in persons having an autoimmune disorder or after abrupt increases in blood pressure of various etiologies. STUDY DESIGN: Case report. METHODS: Retrospective evaluation of medical records...
December 5, 2016: Journal of Spinal Cord Medicine
https://www.readbyqxmd.com/read/27913467/transplantation-for-bone-marrow-failure-current-issues
#9
Régis Peffault de Latour
The preferred treatment of idiopathic aplastic anemia (AA) is allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-identical sibling donor. Transplantation from a well-matched unrelated donor (MUD) may be considered for patients without a sibling donor after failure of immunosuppressive therapy, as may alternative transplantation (mismatched, cord blood or haplo-identical HSCT) for patients without a MUD. HSCT may also be contemplated for congenital disorders in cases of pancytopenia or severe isolated cytopenia...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27909435/immune-reconstitution-after-allogeneic-hematopoietic-stem-cell-transplantation
#10
REVIEW
Justyna Ogonek, Mateja Kralj Juric, Sakhila Ghimire, Pavankumar Reddy Varanasi, Ernst Holler, Hildegard Greinix, Eva Weissinger
The timely reconstitution and regain of function of a donor-derived immune system is of utmost importance for the recovery and long-term survival of patients after allogeneic hematopoietic stem cell transplantation (HSCT). Of note, new developments such as umbilical cord blood or haploidentical grafts were associated with prolonged immunodeficiency due to delayed immune reconstitution, raising the need for better understanding and enhancing the process of immune reconstitution and finding strategies to further optimize these transplant procedures...
2016: Frontiers in Immunology
https://www.readbyqxmd.com/read/27906795/factors-determining-the-outcome-of-hematopoietic-stem-cell-transplantation-in-patients-with-acute-lymphoblastic-leukemia-at-king-faisal-specialist-hospital-and-research-center-riyadh-saudi-arabia
#11
Suleimman Al-Sweedan, Amal Al-Seraihy, Ali Al-Ahmari, Abdullah Al-Jefri, Viqaruddin Mohammed, Rafat Jafri, Khawar Siddiqui, Mouhab Ayas
Medical records of 82 patients with acute lymphoblastic leukemia (ALL) who underwent hematopoietic cell transplantation (HCT) at our institution from 2005 to 2011 were reviewed. Forty-five patients were male (54.8%). The median age at HCT was 7.46 years (range, 0.98 to 14.31 y), the median time to HCT after diagnosis was 12.56 months. Ten patients were below the age of 1 year (12%). All patients were in complete remission at the time of HCT. In 83 transplants, 64 patients received HCT from human leukocyte antigen-identical-related donors and 19 from other donors...
November 30, 2016: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/27905684/the-effect-of-maternal-and-infant-factors-on-cord-blood-yield
#12
REVIEW
Seyed Hadi Mousavi, Saeid Abroun, Morteza Zarrabi, Mona Ahmadipanah
Umbilical cord blood (CB) can be used as an alternative hematopoietic stem cell source for transplantation in hematological malignancy and blood disorders. The success of transplantation is highly related to the levels of total nucleated cell and CD34(+) cell counts. The evaluation of optimal conditions can decrease the rate of graft rejection due to low cell count and increases the quality of CB units (CBUs) in the blood bank and the success rate of engraftment. To this end, we review the maternal and infant parameters affecting the quality and quantity of CBUs...
December 1, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27904117/successful-cord-blood-stem-cell-transplantation-for-an-adult-case-of-chronic-active-epstein-barr-virus-infection
#13
Masuho Saburi, Masao Ogata, Takako Satou, Natsumi Yoshida, Kentaro Nagamatsu, Yuko Nashimoto, Yui Moroga, Kuniko Takano, Kazuhiro Kohno, Kuniaki Shirao
A 41-year-old man was referred to our hospital for treatment of anaplastic lymphoma kinase (ALK)-negative anaplastic large cell lymphoma. Chronic active Epstein-Barr virus (CAEBV) was diagnosed based on the findings of elevated EBV antibody titers and positive EBV-DNA in the peripheral blood, and cord blood stem cell transplantation (CBT) was performed. The EBV-DNA levels in the blood fell below the limit of detection. His lymphoma relapsed on Day 165 with the appearance of eruptions, which disappeared after the withdrawal of tacrolimus...
2016: Internal Medicine
https://www.readbyqxmd.com/read/27899358/cytohesin-1-regulates-homing-and-engraftment-of-human-hematopoietic-stem-and-progenitor-cells
#14
Justyna Rak, Katie Foster, Katarzyna Potrzebowska, Mehrnaz Safaee Talkhoncheh, Natsumi Miharada, Karolina Komorowska, Therese Torngren, Anders Kvist, Åke Borg, Lena Svensson, Dominique Bonnet, Jonas Larsson
Adhesion is a key component of hematopoietic stem cell regulation mediating homing and retention to the niche in the bone marrow. Here, using an RNA interference screen, we identify cytohesin 1 (CYTH1) as a critical mediator of adhesive properties in primary human cord-blood derived hematopoietic stem and progenitor cells (HSPCs). Knockdown of CYTH1 disrupted adhesion of HSPC to primary human mesenchymal stroma cells (MSCs). Attachment to fibronectin and ICAM1, two integrin ligands, was severely impaired, and CYTH1 deficient cells showed a reduced integrin β1 activation response, suggesting that CYTH1 mediates integrin-dependent functions...
November 29, 2016: Blood
https://www.readbyqxmd.com/read/27888015/early-and-long-term-impaired-t-lymphocyte-immune-reconstitution-after-cord-blood-transplantation-with-antithymocyte-globulin
#15
Nerea Castillo, Irene García-Cadenas, Pere Barba, Carme Canals, Cristina Díaz-Heredia, Rodrigo Martino, Christelle Ferrà, Isabel Badell, Izaskun Elorza, Jorge Sierra, David Valcárcel, Sergio Querol
Immune reconstitution is crucial to the success of allogeneic hematopoietic stem cell transplantation. Umbilical cord blood transplantation (UCBT) has been associated with delayed immune reconstitution. We characterized the kinetics and investigated the risk variables affecting the main lymphocyte subsets recovery in 225 consecutive pediatric and adult patients [males, n=126, median age=15, range 0.3-60, interquartile range, 4-35] who underwent myeloablative single-UCBT between 2005 and 2015 for malignant and non-malignant disorders...
November 22, 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27887864/optimization-of-cgmp-purification-and-expansion-of-umbilical-cord-blood-derived-t-regulatory-cells-in-support-of-first-in-human-clinical-trials
#16
David H McKenna, Darin Sumstad, Diane M Kadidlo, Bjorn Batdorf, Colin J Lord, Sarah C Merkel, Christine M Koellner, Julie M Curtsinger, Carl H June, James L Riley, Bruce L Levine, Jeffrey S Miller, Claudio G Brunstein, John E Wagner, Bruce R Blazar, Keli L Hippen
BACKGROUND AIMS: Thymic-derived regulatory T cells (tTreg) are critical regulators of the immune system. Adoptive tTreg transfer is a curative therapy for murine models of autoimmunity, graft rejection, and graft-versus-host disease (GVHD). We previously completed a "first-in-human" clinical trial using in vitro expanded umbilical cord blood (UCB)-derived tTreg to prevent GVHD in patients undergoing UCB hematopoietic stem cell transplantation (HSCT). tTreg were safe and demonstrated clinical efficacy, but low yield prevented further dose escalation...
November 22, 2016: Cytotherapy
https://www.readbyqxmd.com/read/27882356/intra-osseous-co-transplantation-of-cd34-selected-umbilical-cord-blood-and-mesenchymal-stromal-cells
#17
Leland Metheny, Saada Eid, Karen Lingas, Jane Reese, Howard Meyerson, Alexander Tong, Marcos de Lima, Alex Y Huang
Human mesenchymal stromal cells (MSC) have been shown to support the growth and differentiation of hematopoietic stem cells (HSC). We hypothesized that intra-osseous (IO) co-transplantation of MSC and umbilical cord blood (UCB) may be effective in improving early HSC engraftment, as IO transplantation has been demonstrated to enhance UCB engraftment in NOD SCID-gamma (NSG) mice. Following non-lethal irradiation (300rads), 6 groups of NSG mice were studied: 1) intravenous (IV) UCB CD34(+) cells, 2) IV UCB CD34(+) cells and MSC, 3) IO UCB CD34(+) cells, 4) IO UCB CD34(+) cells and IO MSC, 5) IO UCB CD34(+) cells and IV MSC, and 6) IV UCB CD34(+) and IO MSC...
2016: Hematol Med Oncol
https://www.readbyqxmd.com/read/27881982/milestones-of-hematopoietic-stem-cell-transplantation-from-first-human-studies-to-current-developments
#18
REVIEW
Mateja Kralj Juric, Sakhila Ghimire, Justyna Ogonek, Eva M Weissinger, Ernst Holler, Jon J van Rood, Machteld Oudshoorn, Anne Dickinson, Hildegard T Greinix
Since the early beginnings, in the 1950s, hematopoietic stem cell transplantation (HSCT) has become an established curative treatment for an increasing number of patients with life-threatening hematological, oncological, hereditary, and immunological diseases. This has become possible due to worldwide efforts of preclinical and clinical research focusing on issues of transplant immunology, reduction of transplant-associated morbidity, and mortality and efficient malignant disease eradication. The latter has been accomplished by potent graft-versus-leukemia (GvL) effector cells contained in the stem cell graft...
2016: Frontiers in Immunology
https://www.readbyqxmd.com/read/27880980/impact-of-total-body-irradiation-on-successful-neutrophil-engraftment-in-unrelated-bone-marrow-or-cord-blood-transplantation
#19
Hideki Nakasone, Fuji Shigeo, Kimikazu Yakushijin, Makoto Onizuka, Akihito Shinohara, Kazuteru Ohashi, Koichi Miyamura, Naoyuki Uchida, Minoko Takanashi, Tatsuo Ichinohe, Yoshiko Atsuta, Takahiro Fukuda, Masao Ogata
Total body irradiation (TBI) has been thought to promote donor cell engraftment in allogeneic hematopoietic cell transplantation (HCT) from alternative donors. However, recent progress in HCT strategies may affect the clinical significance of TBI on neutrophil engraftment. With the use of a Japanese transplant registry database, we analyzed 3933 adult recipients (>15 y.o.) who underwent HCT between 2006 and 2013 from an 8/8 HLA-matched unrelated bone marrow donor (MUD, n=1367), an HLA-mismatched unrelated bone marrow donor (MMUD, n=1102), or unrelated cord blood (CBT, n=1464)...
November 23, 2016: American Journal of Hematology
https://www.readbyqxmd.com/read/27876094/nitric-oxide-has-contrasting-age-dependent-effects-on-the-functionality-of-murine-hematopoietic-stem-cells
#20
Sapana Jalnapurkar, Shweta Singh, Moirangthem Ranjita Devi, Lalita Limaye, Vaijayanti Kale
BACKGROUND: The success of hematopoietic stem cell (HSC) transplantation is dependent on the quality of the donor HSCs. Some sources of HSCs display reduced engraftment efficiency either because of inadequate number (e.g., fetal liver and cord blood), or age-related dysfunction (e.g. in older individuals). Therefore, use of pharmacological compounds to improve functionality of HSCs is a forefront research area in hematology. METHODS: Lineage negative (Lin(-)) cells isolated from murine bone marrow or sort-purified Lin(-)Sca-1(+)c-Kit(+)CD34(-) (LSK-CD34(-)) were treated with a nitric oxide donor, sodium nitroprusside (SNP)...
November 22, 2016: Stem Cell Research & Therapy
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