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https://www.readbyqxmd.com/read/29238707/drug-repurposing-for-the-treatment-of-acute-myeloid-leukemia
#1
REVIEW
Vibeke Andresen, Bjørn T Gjertsen
Acute myeloid leukemia (AML) is a heterogeneous disease characterized by the accumulation of immature myeloid progenitor cells in the bone marrow, compromising of normal blood cell production and ultimately resulting in bone marrow failure. With a 20% overall survival rate at 5 years and 50% in the 18- to 65-year-old age group, new medicines are needed. It is proposed that development of repurposed drugs may be a part of the new therapy needed. AML is subdivided into recurrent molecular entities based on molecular genetics increasingly accessible for precision medicine...
2017: Frontiers in Medicine
https://www.readbyqxmd.com/read/29230944/improved-insulin-sensitivity-appears-to-remain-for-a-mean-of-four-years-following-islet-cell-transplant-for-type-1-diabetes
#2
Brett Rydzon, Rebecca S Monson, Jose Oberholzer, Krista A Varady, Melena D Bellin, Kirstie K Danielson
BACKGROUND: Impaired insulin sensitivity (IS) predicts complications and mortality in type 1 diabetes (T1D). IS improves shortly after islet cell transplant for type 1 diabetes, yet long-term changes in IS, and associated factors such as patient characteristics, transplant factors, clinical management, and IS-related biomarkers are unknown. METHODS: Up to nine years (mean four) of longitudinal data were available on 22 adults (18 female) with T1D who received 1-3 transplants in Phase 1/2 or 3 clinical trials (2004-2014)...
December 12, 2017: Diabetes/metabolism Research and Reviews
https://www.readbyqxmd.com/read/29228600/non-invasive-in-vivo-molecular-imaging-of-intra-articularly-transplanted-immortalized-bone-marrow-stem-cells-for-osteoarthritis-treatment
#3
Bou-Yue Peng, Chi-Sheng Chiou, Navneet Kumar Dubey, Sung-Hsun Yu, Yue-Hua Deng, Feng-Chou Tsai, Han-Sun Chiang, Ying-Hua Shieh, Wei-Hong Chen, Win-Ping Deng
Pathophysiology of osteoarthritis (OA) is characterized by progressive loss of articular cartilage in the knee-joints. To impart regenerative ability in lowly metabolizing chondrocytes, the bone marrow stem cells (BMSCs) has recently been recognized as a superior alternative treatment for OA. However, study of primary BMSCs-mediated chondrogenesis is difficult due to progressive cellular aging and replicative senescence. To obtain a therapeutic cell population for OA, BMSCs were immortalized by human papilloma virus (HPV)-16 E6/E7 along with mCherry luciferase (mCL), a gene marker for non-invasive imaging, and designated as iBMSCs-mCL...
November 14, 2017: Oncotarget
https://www.readbyqxmd.com/read/29214523/haematopoietic-stem-cell-transplantation-arrests-the-progression-of-neurodegenerative-disease-in-late-onset-tay-sachs-disease
#4
Karolina M Stepien, Su Han Lum, J Edmond Wraith, Christian J Hendriksz, Heather J Church, David Priestman, Frances M Platt, Simon Jones, Ana Jovanovic, Robert Wynn
Tay-Sachs disease is a rare metabolic disease caused by a deficiency of hexosaminidase A that leads to accumulation of GM2 gangliosides predominantly in neural tissue. Late-onset Tay-Sachs disease variant is associated with a higher level of residual HexA activity. Treatment options are limited, and there are a few described cases who have undergone haematopoietic stem cell transplantation (HSCT) with variable outcome.We describe a case of a 23-year-old male patient who presented with a long-standing tremor since 7 years of age...
December 7, 2017: JIMD Reports
https://www.readbyqxmd.com/read/29198627/analytic-morphomics-predict-outcomes-after-lung-transplantation
#5
Michael J Pienta, Peng Zhang, Brian A Derstine, Binu Enchakalody, William B Weir, Tyler Grenda, Rebecca Goulson, Rishindra M Reddy, Andrew C Chang, Stewart C Wang, Jules Lin
BACKGROUND: The purpose of this study was to identify morphomic factors on standard, pretransplantation computed tomography (CT) scans associated with outcomes after lung transplantation. METHODS: A retrospective review of 200 patients undergoing lung transplantation at a single institution from 2003 to 2014 was performed. CT scans obtained within 1 year before transplantation underwent morphomic analysis. Morphomic characteristics included lung, dorsal muscle group, bone, and subcutaneous and visceral fat area and density...
December 1, 2017: Annals of Thoracic Surgery
https://www.readbyqxmd.com/read/29145251/renal-osteodystrophy-presenting-as-a-metabolic-superscan-on-f-18-fdg-pet-ct-a-case-report
#6
Ismaheel Lawal, Alfred Ankrah, Kehinde Ololade, Moshe Modiselle, Mike Sathekge
RATIONALE: F-18 Fluoro Deoxyglucose positron emission tomography and computed tomography (F-18 FDG PET/CT) is a useful modality in the evaluation of patients with malignancies. Increased incidence of lympho-proliferative disorders has been reported in individuals with long-standing end-stage renal disorders treated with renal replacement therapy. PATIENT CONCERNS: A 30-year-old male on peritoneal dialysis on account of end-stage renal disease. He had acute rejection of an earlier transplanted renal allograft...
November 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29131150/neurologic-complications-after-allogeneic-hematopoietic-stem-cell-transplantation-risk-factors-and-impact
#7
M R Dowling, S Li, B R Dey, S L McAfee, H R Hock, T R Spitzer, Y-B Chen, K K Ballen
Neurologic complications (NCs) may be a significant source of morbidity and mortality after hematopoietic cell transplantation (HCT). We performed a retrospective study of 263 consecutive patients undergoing allogeneic HCT for hematological malignancies to determine the incidence, risk factors and clinical impact of NCs in the first 5 years after HCT. We determined the incidence of central nervous system (CNS) infection, intracranial hemorrhage, ischemic stroke, metabolic encephalopathy, posterior reversal encephalopathy syndrome, seizure and peripheral neuropathy...
November 13, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29109103/hepatocyte-nuclear-factor-1a-deficiency-causes-hemolytic-anemia-in-mice-by-altering-erythrocyte-sphingolipid-homeostasis
#8
Karin von Wnuck Lipinski, Sarah Weske, Petra Keul, Susann Peters, Hideo A Baba, Gerd Heusch, Markus H Gräler, Bodo Levkau
The Hepatocyte Nuclear Factor (HNF) family regulates complex networks of metabolism and organ development. Human mutations in its prototypical member HNF1A cause maturity-onset of diabetes of the young (MODY) type 3. In this study, we identified an important role for HNF1A in the preservation of erythrocyte membrane integrity, calcium homeostasis and osmotic resistance through a yet unrecognized link of HNF1A to sphingolipid homeostasis. HNF1A(-/-) mice displayed microcytic hypochromic anemia with reticulocytosis that was partially compensated by avid extramedullary erythropoiesis at all erythroid stages in the spleen thereby excluding erythroid differentiation defects...
November 6, 2017: Blood
https://www.readbyqxmd.com/read/29108345/mri-based-evaluation-of-multiorgan-iron-overload-is-a-predictor-of-adverse-outcomes-in-pediatric-patients-undergoing-allogeneic-hematopoietic-stem-cell-transplantation
#9
Natalia Maximova, Massimo Gregori, Giulia Boz, Roberto Simeone, Davide Zanon, Giulia Schillani, Floriana Zennaro
The medical records of 44 pediatric patients who underwent allogeneic transplantation from 2011 to 2015 were retrospectively reviewed. Magnetic resonance imaging was used to measure iron concentrations in the liver, spleen, pancreas and bone. These patients were divided into two groups, 18 with non-elevated (< 100 μmol/g; Group 1) liver iron concentration before transplantation and 26 with elevated (> 100 μmol/g; Group 2) concentration . We compared transplant-related outcomes in the two groups. Iron overload was a negative prognostic risk factor for sinusoidal obstruction syndrome (OR = 17), osteoporosis (OR = 6...
October 3, 2017: Oncotarget
https://www.readbyqxmd.com/read/29105864/study-of-bone-metabolism-and-angiogenesis-in-patients-undergoing-high-dose-chemotherapy-autologous-hematopoietic-stem-cell-transplantation
#10
Georgios Boutsikas, Evangelos Terpos, Athanasios Papatheodorou, Pantelis Tsirkinidis, Panayiotis Tsirigotis, Anastasia Meletiou, Eleni Lalou, Vasileios Telonis, Anna Zannou, Alexander Kanellopoulos, Zacharoula Galani, Angeliki Stefanou, Panayiotis Tsaftaridis, Nora-Athina Viniou, Panayiotis Panayiotidis, Marie-Christine Kyrtsonis, John Meletis, Theodoros P Vassilakopoulos, Maria K Angelopoulou
OBJECTIVES: Since the interaction between Hematopoietic Stem Cells (HSCs) and endosteal and endothelial niches in HSCs homing is essential, we aimed to study bone turnover and angiogenesis in 29 patients with lymphoma/multiple myeloma undergoing Hematopoietic Stem Cell Transplantation (HSCT). METHODS: Serum samples were collected before High Dose Chemotherapy (HDT), at the end of HDT, after HSC infusion, at the nadir of myelotoxicity and at engraftment. Bone metabolism (CTX, TRACP-5b, bALP, OC, DKK1, RANKL, OPG) and angiogenesis (Ang1, Ang2) markers were measured...
November 6, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/29069247/reversal-of-uremic-tumoral-calcinosis-by-optimization-of-clinical-treatment-of-bone-and-mineral-metabolism-disorder
#11
Mariana Espiga Maioli, Vinicius Daher Alvares Delfino, Amanda Carolina Damasceno Zanuto Guerra, Luiz Fernando Kunii, Raquel Ferreira Nassar Frange
Tumoral calcinosis is an uncommon type of extraosseous calcification characterized by large rubbery or cystic masses containing calcium-phosphate deposits. The condition prevails in the periarticular tissue with preservation of osteoarticular structures. Elevated calcium-phosphorus products and severe secondary hyperparathyroidism are present in most patients with uremic tumoral calcionosis (UTC). Case report of an obese secondary to chronic glomerulonephritis, undergoing continuous ambulatory peritoneal dialysis (CAPD) reported the appearance of painless tumors in the medial surface of fifth finger and left arm...
April 2017: Jornal Brasileiro de Nefrologia: ʹorgão Oficial de Sociedades Brasileira e Latino-Americana de Nefrologia
https://www.readbyqxmd.com/read/29051055/skeletal-impairment-in-pierson-syndrome-is-there-a-role-for-laminin%C3%AE-2-in-bone-physiology
#12
Camille Beaufils, Delphine Farlay, Irma Machuca-Gayet, Alice Fassier, Martin Zenker, Caroline Freychet, Edith Bonnelye, Aurélia Bertholet-Thomas, Bruno Ranchin, Justine Bacchetta
INTRODUCTION: Pierson syndrome is caused by a mutation of LAMB2, encoding for laminin β2. Clinical phenotype is variable but usually associates congenital nephrotic syndrome (CNS) and ocular abnormalities. Neuromuscular impairment has also been described. METHODS: We report on a 15-year old girl, suffering from Pierson Syndrome, who developed severe bone deformations during puberty. This patient initially displayed CNS and microcoria, leading to the clinical diagnosis of Pierson syndrome...
October 16, 2017: Bone
https://www.readbyqxmd.com/read/29035781/lack-of-myeloid-fatp1-increases-atherosclerotic-lesion-size-in-ldlr-mice
#13
Liyang Zhao, Alyssa J Cozzo, Amy R Johnson, Taylor Christensen, Alex J Freemerman, James E Bear, Jeremy D Rotty, Brian J Bennett, Liza Makowski
BACKGROUND AND AIMS: Altered metabolism is an important regulator of macrophage (MΦ) phenotype, which contributes to inflammatory diseases such as atherosclerosis. Broadly, pro-inflammatory, classically-activated MΦs (CAM) are glycolytic while alternatively-activated MΦs (AAM) oxidize fatty acids, although overlap exists. We previously demonstrated that MΦ fatty acid transport protein 1 (FATP1, Slc27a1) was necessary to maintain the oxidative and anti-inflammatory AAM phenotype in vivo in a model of diet-induced obesity...
November 2017: Atherosclerosis
https://www.readbyqxmd.com/read/28987258/an-update-on-the-treatment-and-follow-up-of-patients-with-primary-biliary-cholangitis
#14
REVIEW
Blaire E Burman, Manan A Jhaveri, Kris V Kowdley
Primary biliary cholangitis (PBC) is an autoimmune liver disease characterized by chronic granulomatous lymphocytic cholangitis of the small bile ducts. PBC was a leading indication for liver transplant in the United States; with early diagnosis and treatment, the majority of patients with PBC have a normal life expectancy. Pathogenesis involves inflammatory damage of bile duct epithelium secondary to innate and adaptive immune responses, and toxicity from accumulated bile acids. Cholestasis and disease progression can lead to cirrhosis...
November 2017: Clinics in Liver Disease
https://www.readbyqxmd.com/read/28985203/cell-therapy-for-diverse-central-nervous-system-disorders-inherited-metabolic-diseases-and-autism
#15
REVIEW
Jessica M Sun, Joanne Kurtzberg
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplantation (HSCT) began with the development of bone marrow transplantation for hematologic malignancies in the mid-1900s and is now standard of care for many hematologic disorders. In the past few decades, HSCT has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products...
October 6, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28964571/fractures-in-patients-with-ckd-diagnosis-treatment-and-prevention-a-review-by-members-of-the-european-calcified-tissue-society-and-the-european-renal-association-of-nephrology-dialysis-and-transplantation
#16
REVIEW
Ana Pimentel, Pablo Ureña-Torres, M Carola Zillikens, Jordi Bover, Martine Cohen-Solal
Mineral and bone disease is omnipresent in patients with chronic kidney disease (CKD) and leads to a diverse range of clinical manifestations, including bone pain and fractures. The accumulation of traditional clinical risk factors, in addition to those related to CKD, enhances the risk of comorbidity and mortality. Despite significant advances in understanding bone disease in CKD, most clinical and biochemical targets used in clinical practice remain controversial, resulting in an undermanagement of bone fragility...
December 2017: Kidney International
https://www.readbyqxmd.com/read/28942400/extreme-bony-pelvic-deformity-in-a-renal-transplant-patient
#17
Alexander Henry Moffat, Priyesh Chauhan, Surabhi Choudhary, Tarekegn Geberhiwot
A 35-year-old female renal transplant recipient was referred to the metabolic bone clinic because of a 15 cm loss of height. She was noted to have thoracic kyphosis and vertebral X-ray confirmed a wedge fracture of the eighth thoracic vertebra. She was managed accordingly but on subsequent follow-up mentioned in passing that her gynaecologist had been unable to obtain a cervical smear for routine screening. This led to further questioning, and the patient reported intermittent urinary stress incontinence, feelings of vaginal fullness and severe dyspareunia...
September 23, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28940569/weekly-oral-bisphosphonates-over-2%C3%A2-years-prevent-bone-loss-in-cardiac-transplant-patients
#18
Uwe Lange, Katharina Classen, Ulf Müller-Ladner, Manfred Richter
BACKGROUND: Heart transplantation (HTX) is an established therapy for end-stage heart disease. The aim of this study was to determine whether application of oral bisphosphonates is effective in preventing osseous complications after HTX. METHODS: Thirty-three cardiac transplant recipients were treated with alendronate 70 mg/wk or risedronate 35 mg/wk in combination with 1000 mg calcium and 800 IU vitamin D. Markers of bone metabolism and dual-energy X-ray absorptiometry (DXA) were determined directly after HTX and 2 years later...
September 23, 2017: Clinical Transplantation
https://www.readbyqxmd.com/read/28926770/1-25-oh-2d3-induces-regulatory-t-cell-differentiation-by-influencing-the-vdr-plc-%C3%AE-1-tgf-%C3%AE-1-pathway
#19
Qiang Zhou, Shengying Qin, Jinyan Zhang, Lin Zhon, Zhihai Pen, Tonghai Xing
Vitamin D has been recommended as an immune modulator in recent years, in addition to regulating calcium-phosphorous-bone metabolism. Clinical studies on organ transplantation found that vitamin D sufficiency patients were less likely to develop acute cellular rejection within one year after transplantation compared to those with vitamin D deficiency. Thus, a high percentage of regulatory T cells might play a key role in preventing acute cellular rejection (ACR). In this report, we studied the specific effects of 1,25(OH)2D3 on human T cell diff ;erentiation, and determined the potential molecule mechanism behind...
November 2017: Molecular Immunology
https://www.readbyqxmd.com/read/28901391/protective-effects-of-heme-oxygenase-1-transduced-bone-marrow-derived-mesenchymal-stem-cells-on-reduced%C3%A2-size-liver-transplantation-role-of-autophagy-regulated-by-the-erk-mtor-signaling-pathway
#20
Raorao Wang, Zhongyang Shen, Liu Yang, Mingli Yin, Weiping Zheng, Bin Wu, Tao Liu, Hongli Song
Autophagy is a critical lysosomal pathway that degrades cytoplasmic components to maintain cell homeostasis and provide substrates for energy metabolism. A study revealed that heme oxygenase-1 (HO-1)-transduced bone marrow-derived mesenchymal stem cells (BM-MSCs) could protect 50% reduced-size liver transplantation (RSLT) in a rat model. However, the mechanisms remain mostly unknown. The aim of the present study was to explore the effects and related mechanism of autophagy on the protection conferred by HO-1-transduced BM-MSCs (HO-1/BM-MSCs) on 50% RSLT in a rat model...
November 2017: International Journal of Molecular Medicine
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