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https://www.readbyqxmd.com/read/28346418/first-experience-of-hematopoietic-stem-cell-transplantation-treatment-of-shwachman-diamond-syndrome-using-unaffected-hla-matched-sibling-donor-produced-through-preimplantation-hla-typing
#1
A A Isaev, R V Deev, A Kuliev, I L Plaxa, N V Stancheva, A S Borovkova, I V Potapov, E A Pomerantseva, A G Chogovadze, K Y Boyarsky, A E Semenenko, A V Mikhailov, K G Shevchenko, A V Prikhodko, S Rechitsky, O V Paina, I M Barchatov, L S Zubarovskaya, O Verlinsky, I Y Bozo, B V Afanasyev
The only proven cure for Shwachman-Diamond syndrome (SDS) bone marrow failure is allogeneic hematopoietic stem cell transplantation (HSCT). However HSCT with donors other than HLA-identical siblings is associated with high mortality and unfavorable prognosis. This paper presents the first experience of HSCT treatment of SDS using an unaffected HLA-identical sibling produced through preimplantation genetic diagnosis (PGD). The patient was a 6-year-old blood transfusion-dependent SDS baby girl with secondary myelodysplastic syndrome, for whom no HLA-identical donor was available...
March 27, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28346415/haplo-cord-transplantation-compared-to-haploidentical-transplantation-with-post-transplant-cyclophosphamide-in-patients-with-aml
#2
M Kwon, G Bautista, P Balsalobre, I Sánchez-Ortega, P Montesinos, A Bermúdez, A de Laiglesia, P Herrera, C Martin, K Humala, A Zabalza, M Torres, L Bento, L L Corral, I Heras, D Serrano, I Buño, J Anguita, C Regidor, R Duarte, R Cabrera, J Gayoso, J L Diez-Martin
For patients with AML, the best alternative donor remains to be defined. We analyze outcomes of patients who underwent myeloablative umbilical cord blood or haploidentical hemopoietic stem cell transplantation (HSCT) in Spain. Fifty-one patients underwent single umbilical cord blood transplantation supported by a third party donor (Haplo-Cord) between 1999 and 2012, and 36 patients received an haploidentical HSCT with post-transplant cyclophosphamide (PTCY-haplo) between 2012 and 2014 in GETH centers. The Haplo-Cord cohort included a higher proportion of patients with high disease risk index and use of TBI in the conditioning regimen, and hematopoietic cell transplantation-age Comorbidity Age Index was higher in PTCY-haplo patients...
March 27, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28345003/expansion-of-human-tregs-from-cryopreserved-umbilical-cord-blood-for-gmp-compliant-autologous-adoptive-cell-transfer-therapy
#3
Howard R Seay, Amy L Putnam, Judit Cserny, Amanda L Posgai, Emma H Rosenau, John R Wingard, Kate F Girard, Morey Kraus, Angela P Lares, Heather L Brown, Katherine S Brown, Kristi T Balavage, Leeana D Peters, Ashley N Bushdorf, Mark A Atkinson, Jeffrey A Bluestone, Michael J Haller, Todd M Brusko
Umbilical cord blood is a traditional and convenient source of cells for hematopoietic stem cell transplantation. Thymic regulatory T cells (Tregs) are also present in cord blood, and there is growing interest in the use of autologous Tregs to provide a low-risk, fully human leukocyte antigen (HLA)-matched cell product for treating autoimmune diseases, such as type 1 diabetes. Here, we describe a good manufacturing practice (GMP)-compatible Treg expansion protocol using fluorescence-activated cell sorting, resulting in a mean 2,092-fold expansion of Tregs over a 16-day culture for a median yield of 1...
March 17, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28341839/therapeutic-efficacy-of-neural-stem-cells-originating-from-umbilical-cord-derived-mesenchymal-stem-cells-in-diabetic-retinopathy
#4
Wei Zhang, Yuexin Wang, Jiahui Kong, Meng Dong, Hongtao Duan, Song Chen
The aim of this study was to evaluate the effects of intravitreal injection of neural stem cells (NSCs) originating from human umbilical cord-derived mesenchymal stem cells (UC-MSCs) on neurodegeneration of diabetic retinopathy (DR) in rats. UC-MSCs were isolated and passaged, followed by induction to NSCs in neural differentiation medium. Four weeks following NSC transplantation, treatment attenuated retinal vascular dysfunction compared with non-treated rats, and BDNF and Thy-1 expression was significantly higher in the treated group than in the control group...
March 24, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28331055/reticular-dysgenesis-international-survey-on-clinical-presentation-transplantation-and-outcome
#5
Manfred Hoenig, Chantal Lagresle-Peyrou, Ulrich Pannicke, Luigi D Notarangelo, Fulvio Porta, Andrew R Gennery, Mary Slatter, Morton J Cowan, Polina Stepensky, Hamoud Al-Mousa, Daifulah Al-Zahrani, Sung-Yun Pai, Waleed Al Herz, Hubert B Gaspar, Paul Veys, Koichi Oshima, Kohsuke Imai, Hiromasa Yabe, Lenora M Noroski, Nico M Wulffraat, Karl-Walter Sykora, Pere Soler-Palacin, Hideki Muramatsu, Mariam Al Hilali, Despina Moshous, Klaus-Michael Debatin, Catharina Schuetz, Eva-Maria Jacobsen, Ansgar S Schulz, Klaus Schwarz, Alain Fischer, Wilhelm Friedrich, Marina Cavazzana
Reticular Dysgenesis (RD) is a rare congenital disorder defined clinically by the combination of severe combined immunodeficiency (SCID), agranulocytosis and sensorineural deafness. Mutations in the gene encoding Adenylate Kinase 2 (AK2) were identified to cause the disorder. Hematopoietic stem cell transplantation (HSCT) is the only option to cure this otherwise fatal disease. Retrospective data on clinical presentation, genetics and outcome of HSCT were collected from centers in Europe, Asia and North America for a total of 32 patients born between 1982 and 2011...
March 22, 2017: Blood
https://www.readbyqxmd.com/read/28330501/intraarterial-transplantation-of-human-umbilical-cord-blood-mononuclear-cells-in-hyperacute-stroke-improves-vascular-function
#6
Lei Huang, Yichu Liu, Jianfei Lu, Bianca Cerqueira, Vivek Misra, Timothy Q Duong
BACKGROUND: Human umbilical cord blood (hUCB) cell therapy is a promising treatment for ischemic stroke. The effects of hyperacute stem cell transplantation on cerebrovascular function in ischemic stroke are, however, not well understood. This study evaluated the effects of hyperacute intraarterial transplantation of hUCB mononuclear cells (MNCs) on cerebrovascular function in stroke rats using serial magnetic resonance imaging (MRI). METHODS: HUCB MNCs or vehicle were administered to stroke rats via the internal carotid artery immediately after reperfusion at 60 min following ischemia onset...
March 22, 2017: Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/28322752/neuroprotector-effect-of-stem-cells-from-human-exfoliated-deciduous-teeth-transplanted-after-traumatic-spinal-cord-injury-involves-inhibition-of-early-neuronal-apoptosis
#7
Fabrício do Couto Nicola, Marília Rossato Marques, Felipe Odorcyk, Danusa Mar Arcego, Letícia Petenuzzo, Dirceu Aristimunha, Adriana Vizuete, Eduardo Farias Sanches, Daniela Pavulack Pereira, Natasha Maurmann, Carla Dalmaz, Patricia Pranke, Carlos A Netto
Stem cells from human exfoliated deciduous teeth (SHED) transplants have been investigated as a possible treatment strategy for spinal cord injuries (SCI) due to their potential for promoting functional recovery. The aim of present study was to investigate the effects of SHED on neuronal death after an experimental model of SCI. METHODS: Wistar rats were spinalized using NYU impactor®. Animals were randomly distributed into 4 groups: Control (Naive) or Surgical control, Sham (laminectomy with no SCI); SCI (laminectomy followed by SCI, treated with vehicle); SHED (SCI treated with intraspinal transplantation of 3×10(5) SHED, 1 h after SCI)...
March 16, 2017: Brain Research
https://www.readbyqxmd.com/read/28321921/insights-into-cell-free-therapeutic-approach-role-of-stem-cell-soup-ernatant
#8
REVIEW
Shalini Raik, Ajay Kumar, Shalmoli Bhattacharyya
Current advances in medicine have revolutionized the field of regenerative medicine dramatically with newly evolved therapies for repair or replacement of degenerating or injured tissues. Stem cells can be harvested from different sources for clinical therapeutics which include fetal tissues, umbilical cord blood, embryos and adult tissues. Stem cells can be isolated and differentiated into desired lineages for tissue regeneration and cell replacement therapy. However, several loopholes need to be addressed properly before this can be extended for large scale therapeutic application...
March 21, 2017: Biotechnology and Applied Biochemistry
https://www.readbyqxmd.com/read/28320105/anti-fibrotic-potential-of-human-umbilical-cord-mononuclear-cells-and-mouse-bone-marrow-cells-in-ccl4-induced-liver-fibrosis-in-mice
#9
Nageh Ahmed Elmahdy, Samia Salem Sokar, Mohamed Labib Salem, Naglaa Ibrahim Sarhan, Sherin Hamed Abou-Elela
Liver fibrosis is the consequence of hepatocyte injury that leads to the activation of hepatic stellate cells (HSC). The treatment of choice is Liver transplantation; however, it has many problems such as surgery-related complications, immunological rejection and high costs associated with the procedure. Stem cell-based therapy would be a potential alternative, so the aim of this study is to investigate the therapeutic potential of human umbilical cord mononuclear cells (MNC) and mouse bone marrow cells (BMC) against carbon tetrachloride (CCl4) induced liver fibrosis in mice and compare it with that of silymarin...
March 18, 2017: Biomedicine & Pharmacotherapy, Biomédecine & Pharmacothérapie
https://www.readbyqxmd.com/read/28315229/primary-central-nervous-system-lymphoma-essential-points-in-diagnosis-and-management
#10
REVIEW
Semra Paydas
Primary central nervous system lymphoma (PCNSL) is an extra-nodal non-Hodgkin lymphoma. PCNSL is defined as lymphoma involving the brain, leptomeninges, eyes, or spinal cord without evidence of lymphoma outside the CNS. Treatment includes induction with chemotherapy and consolidation with whole-brain radiotherapy or high-dose chemotherapy supported by autologous stem cell transplantation. High-dose methotrexate is the most important drug in cases with PCNSL, and this drug will be used in combination with small molecules, BTK inhibitors, new monoclonal antibodies, and checkpoint blockers...
April 2017: Medical Oncology
https://www.readbyqxmd.com/read/28302713/family-cord-blood-banking-for-sickle-cell-disease-a-twenty-year-experience-in-two-dedicated-public-cord-blood-banks
#11
Hanadi Rafii, Françoise Bernaudin, Helene Rouard, Valérie Vanneaux, Annalisa Ruggeri, Marina Cavazzana, Valerie Gauthereau, Aurélie Stanislas, Malika Benkerrou, Mariane De Montalembert, Christele Ferry, Robert Girot, Cecile Arnaud, Annie Kamdem, Joelle Gour, Claudine Touboul, Audrey Cras, Mathieu Kuentz, Claire Rieux, Fernanda Volt, Barbara Cappelli, Karina T Maio, Annalisa Paviglianiti, Chantal Kenzey, Jerome Larghero, Eliane Gluckman
Efforts to implement family cord blood banking have been developed in the past decades for siblings requiring stem cell transplantation for conditions such as sickle cell disease. However, public banks are faced with challenging decisions about the units to be stored, discarded, or used for other endeavors. We report here 20 years of experience in family cord blood banking for sickle cell disease in two dedicated public banks. Participants were pregnant women who had previous child diagnosed with homozygous sickle cell disease...
March 16, 2017: Haematologica
https://www.readbyqxmd.com/read/28298913/cathepsin-e-deficiency-ameliorates-graft-versus-host-disease-and-modifies-dendritic-cell-motility
#12
Jörg Mengwasser, Liane Babes, Steffen Cordes, Sarah Mertlitz, Katarina Riesner, Yu Shi, Aleixandria McGearey, Martina Kalupa, Thomas Reinheckel, Olaf Penack
Microbial products influence immunity after allogeneic hematopoietic stem cell transplantation (allo-SCT). In this context, the role of cathepsin E (Ctse), an aspartate protease known to cleave bacterial peptides for antigen presentation in dendritic cells (DCs), has not been studied. During experimental acute graft-versus-host disease (GVHD), we found infiltration by Ctse-positive immune cells leading to higher Ctse RNA- and protein levels in target organs. In Ctse-deficient allo-SCT recipients, we found ameliorated GVHD, improved survival, and lower numbers of tissue-infiltrating DCs...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/28297787/-umbilical-cord-mesenchymal-stem-cells-and-their-association-with-liver-fibrosis
#13
L Tuo, W Z Zeng, H L Xue, X L Wu
At present, the most effective therapeutic method for end-stage liver fibrosis is liver transplantation. However, the application of liver transplantation is limited by a shortage of liver donors, a high incidence rate of surgical complications, graft-versus-host disease, and high medical costs. Umbilical cord mesenchymal stem cell (UC-MSC) transplantation may become a promising method for the treatment of liver diseases. UC-MSCs are adult stem cells which exhibit multipotential differentiation and can differentiate into hepatic parenchymal cells...
January 20, 2017: Zhonghua Gan Zang Bing za Zhi, Zhonghua Ganzangbing Zazhi, Chinese Journal of Hepatology
https://www.readbyqxmd.com/read/28297572/safety-and-efficacy-of-megakaryocytes-induced-from-hematopoietic-stem-cells-in-murine-and-nonhuman-primate-models
#14
Xin Guan, Meng Qin, Yu Zhang, Yanan Wang, Bin Shen, Zhihua Ren, Xinxin Ding, Wei Dai, Yongping Jiang
Because of a lack of platelet supply and a U.S. Food and Drug Administration-approved platelet growth factor, megakaryocytes have emerged as an effective substitute for alleviating thrombocytopenia. Here, we report the development of an efficient two-stage culture system that is free of stroma, animal components, and genetic manipulations for the production of functional megakaryocytes from hematopoietic stem cells. Safety and functional studies were performed in murine and nonhuman primate models. One human cryopreserved cord blood CD34(+) cell could be induced ex vivo to produce up to 1...
March 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28296144/aligned-poly-%C3%AE%C2%B5-caprolactone-nanofibers-guide-the-orientation-and-migration-of-human-pluripotent-stem-cell-derived-neurons-astrocytes-and-oligodendrocyte-precursor-cells-in-vitro
#15
Anu Hyysalo, Mervi Ristola, Tiina Joki, Mari Honkanen, Minnamari Vippola, Susanna Narkilahti
Stem cell transplantations for spinal cord injury (SCI) have been studied extensively for the past decade in order to replace the damaged tissue with human pluripotent stem cell (hPSC)-derived neural cells. Transplanted cells may, however, benefit from supporting and guiding structures or scaffolds in order to remain viable and integrate into the host tissue. Biomaterials can be used as supporting scaffolds, as they mimic the characteristics of the natural cellular environment. In this study, hPSC-derived neurons, astrocytes, and oligodendrocyte precursor cells (OPCs) are cultured on aligned poly(ε-caprolactone) nanofiber platforms, which guide cell orientation to resemble that of spinal cord in vivo...
March 15, 2017: Macromolecular Bioscience
https://www.readbyqxmd.com/read/28295190/phase-i-study-of-cord-blood-derived-natural-killer-cells-combined-with-autologous-stem-cell-transplantation-in-multiple-myeloma
#16
Nina Shah, Li Li, Jessica McCarty, Indreshpal Kaur, Eric Yvon, Hila Shaim, Muharrem Muftuoglu, Enli Liu, Robert Z Orlowski, Laurence Cooper, Dean Lee, Simrit Parmar, Kai Cao, Catherine Sobieiski, Rima Saliba, Chitra Hosing, Sairah Ahmed, Yago Nieto, Qaiser Bashir, Krina Patel, Catherine Bollard, Muzaffar Qazilbash, Richard Champlin, Katy Rezvani, Elizabeth J Shpall
Multiple myeloma (MM) is a disease with known immune dysregulation. Natural killer (NK) cells have shown preclinical activity in MM. We conducted a first-in-human study of umbilical cord blood-derived (CB) NK cells for MM patients undergoing high dose chemotherapy and autologous haematopoietic stem cell transplantation (auto-HCT). Patients received lenalidomide (10 mg) on days -8 to -2, melphalan 200 mg/m(2) on day -7, CB-NK cells on day -5 and auto-HCT on day 0. Twelve patients were enrolled, three on each of four CB-NK cell dose levels: 5 × 10(6) , 1 × 10(7) , 5 × 10(7) and 1 × 10(8) CB-NK cells/kg...
March 14, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28293049/microrna-modulation-during-the-in-vitro-culture-of-hematopoietic-stem-cells-prior-to-transplantation
#17
Saeid Shahrabi, Saeid Kaviani, Masoud Soleimani, Ali Akbar Pourfathollah, Behnaz Bakhshandeh, Saeideh Hajizamani, Najmaldin Saki
BACKGROUND: Human umbilical cord blood (HUCB) is an acceptable and readily accessible source of stem cells. There is an ongoing interest in cord blood stem cell therapies; however, little is known about the possible unfavorable effects of laboratory modifications on the isolated HUCB cells. The involvement of miRNAs in several biological processes has been shown. The aim of this study was to evaluate the possible changes in miRNA expression profiles in CD133(+) hematopoietic cells after in vitro culture...
January 2017: Iranian Journal of Medical Sciences
https://www.readbyqxmd.com/read/28292847/human-umbilical-cord-blood-borne-fibroblasts-contain-marrow-niche-precursors-that-form-a-bone-marrow-organoid-in-vivo
#18
Alice Pievani, Benedetto Sacchetti, Alessandro Corsi, Benedetta Rambaldi, Samantha Donsante, Valeria Scagliotti, Patrizia Vergani, Cristina Remoli, Andrea Biondi, Pamela G Robey, Mara Riminucci, Marta Serafini
Human umbilical cord blood (CB) has attracted much attention as a reservoir for functional hematopoietic stem and progenitor cells, and, recently, as a source of blood-borne fibroblasts (CB-BFs). Previously, we demonstrated that bone marrow stromal cell (BMSC) and CB-BF pellet cultures make cartilage in vitro Furthermore, upon in vivo transplantation, BMSC pellets remodelled into miniature bone/marrow organoids. Using this in vivo model, we asked whether CB-BF populations that express characteristics of the hematopoietic stem cell (HSC) niche contain precursors that reform the niche...
March 15, 2017: Development
https://www.readbyqxmd.com/read/28291470/comparative-efficacy-of-multiple-variables-of-mesenchymal-stem-cell-transplantation-for-the-treatment-of-neuropathic-pain-in-rats
#19
LiPing Liu, Zhen Hua, Jun Shen, Yan Yin, Jing Yang, Kathleen Cheng, Aijun Liu, Lina Wang, Jianguo Cheng
OBJECTIVES: The current treatment options for neuropathic pain due to nerve injuries are limited and largely unsatisfactory. Mesenchymal stem cell transplantation (MSC) has shown promise as an emerging therapy for neuropathic pain. However, a number of critical parameters, including the sources of cells, the number of cells, and routes of transplantation, need to be elucidated before it can be tested clinically. METHODS: MSCs were isolated from rat bone marrow (rBM-MSCs) and adipose tissue (rAD-MSCs) and characterized by flow cytometry and functional differentiation...
March 2017: Military Medicine
https://www.readbyqxmd.com/read/28281429/single-centre-experience-of-allogeneic-haemopoietic-stem-cell-transplant-in-paediatric-patients-in-cape-town-south-africa
#20
A Van Eyssen, N Novitsky, P De Wit, T Schlaphoff, V Thomas, D Pillay, M Hendricks, A Davidson
BACKGROUND: Allogeneic haemopoietic stem cell transplant (Allo-HSCT) is a specialised and costly intervention, associated with significant morbidity and mortality. It is used to treat a broad range of paediatric conditions. South Africa (SA) is an upper middle-income country with limitations on healthcare spending. The role of paediatric Allo-HSCT in this setting is reviewed. OBJECTIVES: To review paediatric patients who underwent Allo-HSCT at the Groote Schuur Hospital/University of Cape Town Private Academic Hospital transplant unit in Cape Town, South Africa, and received post-transplant care at Red Cross War Memorial Children's Hospital, over the period January 2006 - December 2014 in respect of indications for the transplant, donor sources, conditioning regimens, treatment-related morbidity and overall survival (OS)...
February 27, 2017: South African Medical Journal, Suid-Afrikaanse Tydskrif Vir Geneeskunde
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