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https://www.readbyqxmd.com/read/28651310/clinicopathological-characteristics-of-extremely-young-korean-multiple-myeloma-patients-therapeutic-implications
#1
Junghoon Shin, Youngil Koh, Jeonghwan Youk, Miso Kim, Byung Soo Kim, Chul Won Choi, Hwa Jung Sung, Yong Park, Sung-Soo Yoon, Inho Kim
Background/Aims: Although multiple myeloma (MM) is typically a disease of the elderly, a certain subset of extremely young patients exists. It is necessary to establish clinicopathological characteristics for this population. Methods: We reviewed the medical records of MM patients whose age was 40 years or younger at diagnosis. Results: A total of 32 patients were analyzed (male to female ratio 19:13, median age 37 years). According to International Staging System, 29%, 48%, and 16% were in stage I, II, and III, respectively...
June 26, 2017: Korean Journal of Internal Medicine
https://www.readbyqxmd.com/read/28651066/thalassemia-major-transfusion-and-chelation-or-transplantation
#2
REVIEW
Said Yousuf Mohamed
Thalassemia is the most common monogenic hematologic disease that affects millions in the world and kills thousands of patients every year. Without transfusion or transplantation, patients with thalassemia major are expected to die within months of diagnosis. However, long-term transfusion and chelation therapy is highly challenging for many developing countries where the disease is prevalent, representing a major and unsustainable health burden. Stem cell transplantation is the only cure for thalassemia. It has witnessed major developments that have made it less toxic, more successful, and feasible for a larger number of patients with diverse comorbidities and from a wider range of donors...
June 20, 2017: Hematology/oncology and Stem Cell Therapy
https://www.readbyqxmd.com/read/28650520/immature-midbrain-dopaminergic-neurons-derived-from-floor-plate-method-improve-cell-transplantation-therapy-efficacy-for-parkinson-s-disease
#3
Lifeng Qiu, Mei-Chih Liao, Allen K Chen, Shunhui Wei, Shaoping Xie, Shaul Reuveny, Zhi Dong Zhou, Walter Hunziker, Eng King Tan, Steve K W Oh, Li Zeng
Recent reports have indicated human embryonic stem cells-derived midbrain dopamine (mDA) neurons as proper cell resources for use in Parkinson's disease (PD) therapy. Nevertheless, no detailed and systematic study has been conducted to identify which differentiation stages of mDA cells are most suitable for transplantation in PD therapy. Here, we transplanted three types of mDA cells, DA progenitors (differentiated in vitro for 16 days [D16]), immature DA neurons (D25), and DA neurons (D35), into PD mice and found that all three types of cells showed high viability and strong neuronal differentiation in vivo...
June 26, 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28650455/clinical-impact-of-nk-cell-reconstitution-after-reduced-intensity-conditioned-unrelated-cord-blood-transplantation-in-patients-with-acute-myeloid-leukemia-analysis-of-a-prospective-phase-ii-multicenter-trial-on-behalf-of-the-soci%C3%A3-t%C3%A3-fran%C3%A3-aise-de-greffe-de
#4
S Nguyen, A Achour, L Souchet, S Vigouroux, P Chevallier, S Furst, A Sirvent, J-O Bay, G Socié, P Ceballos, A Huynh, J Cornillon, S Francois, F Legrand, I Yakoub-Agha, G Michel, N Maillard, G Margueritte, S Maury, M Uzunov, C-E Bulabois, M Michallet, L Clement, C Dauriac, K Bilger, J Lejeune, V Béziat, V Rocha, B Rio, S Chevret, V Vieillard
Unrelated cord blood transplantation (UCBT) after a reduced intensity conditioning regimen (RIC) has extended the use of UCB in elderly patients and those with co-morbidities without an HLA-identical donor, although post-transplant relapse remains a concern in high-risk acute myeloid leukemia (AML) patients. HLA incompatibilities between donor and recipient might enhance the alloreactivity of natural killer (NK) cells after allogeneic hematopoietic stem-cell transplantation (HSCT). We studied the reconstitution of NK cells and KIR-L mismatch in 54 patients who underwent a RIC-UCBT for AML in CR in a prospective phase II clinical trial...
June 26, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28650452/development-of-quality-of-life-in-patients-undergoing-allogeneic-stem-cell-transplantation-with-regard-to-anxiety-depression-and-resilience
#5
A Schumacher, C Sauerland, G Silling, W E Berdel, M Stelljes, R Koch
No abstract text is available yet for this article.
June 26, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28650451/inability-to-work-and-need-for-disability-pension-among-long-term-survivors-of-hematopoietic-stem-cell-transplantation
#6
A Tichelli, S Gerull, A Holbro, A Buser, G Nair, M Medinger, D Heim, J P Halter, J R Passweg
Return to work is critical goal following HSCT. However, late effects may impede return to normal activity after HSCT. In the case of inability to work, patients may need a work disability pension to ensure a reasonable livelihood. This study evaluated inability to work and need for disability pension among long-term survivors and analyzed possible determinants of need for social support. This retrospective, single-center study included all HSCT patients surviving ⩾5 years seen at the outpatient clinic between January 2013 and August 2015...
June 26, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28650448/transplant-associated-thrombotic-microangiopathy-is-an-endothelial-complication-associated-with-refractoriness-of-acute-gvhd
#7
M Zeisbrich, N Becker, A Benner, A Radujkovic, K Schmitt, J Beimler, A D Ho, M Zeier, P Dreger, T Luft
There is increasing evidence that endothelial dysfunction is involved in refractoriness of acute GvHD (aGvHD). Here we investigated the hypothesis that another endothelial complication, transplant-associated thrombotic microangiopathy (TMA), contributes to the pathogenesis of aGvHD refractoriness. TMA was retrospectively assessed in 771 patients after allogeneic stem cell transplantation (alloSCT). Incidences of TMA and refractory aGvHD were correlated with biomarkers of endothelial damage obtained before alloSCT for patients receiving or not receiving statin-based endothelial prophylaxis (SEP)...
June 26, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28649784/allogeneic-hematopoietic-stem-cell-transplantation-in-congenital-disorders-a-single-center-experience
#8
Maura Faraci, Stefano Giardino, Francesca Bagnasco, Giuseppe Morreale, M Paola Terranova, Daniela Di Martino, Edoardo Lanino
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the treatment of choice for a variety of congenital disorders. We report the experience of children affected by congenital diseases other than bone marrow failure syndromes who received allo-HSCT over a period of 25 years at G. Gaslini Paediatric Research Institute. HSCTs were performed in 57 children with congenital diseases (25 with congenital immunodeficiencies, 10 with severe combined immunodeficiencies, and 22 with metabolic diseases). Overall survival rate at 3 years in the whole group of patients was 76...
June 26, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/28649656/lgr5-is-a-marker-for-fetal-mammary-stem-cells-but-is-not-essential-for-stem-cell-activity-or-tumorigenesis
#9
Christy L Trejo, Gidsela Luna, Christopher Dravis, Benjamin T Spike, Geoffrey M Wahl
The search for the bipotent mammary stem cells that drive mammary development requires markers to enable their prospective isolation. There is general agreement that bipotent mouse mammary stem cells are abundant in late fetal development, but their existence in the adult is vigorously debated. Among markers useful for mammary stem cell identification, the Wnt co-receptor Lgr5 has been suggested by some to be both "necessary and sufficient" for bipotency and transplantation of adult mammary stem cell activity, though other studies disagree...
2017: NPJ Breast Cancer
https://www.readbyqxmd.com/read/28649514/early-hematopoietic-stem-cell-transplantation-in-a-patient-with-severe-mucopolysaccharidosis-ii-a-7%C3%A2-years-follow-up
#10
Anneliese L Barth, Tatiana S P C de Magalhães, Ana Beatriz R Reis, Maria Lucia de Oliveira, Fernanda B Scalco, Nicolette C Cavalcanti, Daniel S E Silva, Danielle A Torres, Alessandra A P Costa, Carmem Bonfim, Roberto Giugliani, Juan C Llerena, Dafne D G Horovitz
Mucopolysaccharidosis type II (MPS II - Hunter syndrome) is an X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2 sulfatase (I2S), leading to the accumulation of the glycosaminoglycans, affecting multiple organs and systems. Enzyme replacement therapy does not cross the blood brain barrier, limiting results in neurological forms of the disease. Another option of treatment for severe MPS, hematopoietic stem cell transplantation (HSCT) has become the treatment of choice for the severe form of MPS type I, since it can preserve neurocognition when performed early in the course of the disease...
September 2017: Molecular Genetics and Metabolism Reports
https://www.readbyqxmd.com/read/28649419/identification-of-transcription-factors-that-promote-the-differentiation-of-human-pluripotent-stem-cells-into-lacrimal-gland-epithelium-like-cells
#11
Masatoshi Hirayama, Shigeru B H Ko, Tetsuya Kawakita, Tomohiko Akiyama, Sravan K Goparaju, Atsumi Soma, Yuhki Nakatake, Miki Sakota, Nana Chikazawa-Nohtomi, Shigeto Shimmura, Kazuo Tsubota, Minoru S H Ko
Dry eye disease is the most prevalent pathological condition in aging eyes. One potential therapeutic strategy is the transplantation of lacrimal glands, generated in vitro from pluripotent stem cells such as human embryonic stem cells, into patients. One of the preceding requirements is a method to differentiate human embryonic stem cells into lacrimal gland epithelium cells. As the first step for this approach, this study aims to identify a set of transcription factors whose overexpression can promote the differentiation of human embryonic stem cells into lacrimal gland epithelium-like cells...
2017: NPJ Aging and Mechanisms of Disease
https://www.readbyqxmd.com/read/28648947/hematopoietic-stem-cell-transplantation-in-the-sultanate-of-oman
#12
REVIEW
Murtadha Al-Khabori, Mohammed Al-Huneini
The Sultanate of Oman is one of the Arabian Gulf countries with a total population of 4,414,051 as of mid 2016, of which 2,427,825 are Omanis. The gross national income per capita was 7327.7 RO (Omani rial; equivalent to US$19,033) in 2014. There are two hematopoietic stem cell transplantation (HSCT) centers in Oman: the Sultan Qaboos University Hospital (SQUH; allogeneic and autologous) and the Royal Hospital (RH; autologous). HSCT activity in Oman started in 1995 at the SQUH center, which had only one bed, and four cases were performed in that year...
June 17, 2017: Hematology/oncology and Stem Cell Therapy
https://www.readbyqxmd.com/read/28648733/-is-transplantation-an-alternative-to-the-transfusional-impasse-in-sickle-cell-disease
#13
F Bernaudin, M Kuentz
Sickle cell disease is the most frequent genetic disease in France, concerning 400 newborns each year. The management of these Afro-Caribbean patients requires frequent transfusions from Caucasian donors. Due to important erythroid antigenic differences between Caucasian and African, the prevalence of allo-immunization is high in this population with a risk of transfusional impasse. Allogeneic stem cell transplantation is the only curative treatment for this disease and the replacement of red cells and lymphocytes of the sickle cell patient by those of the donor can also resolve the transfusional impasse...
June 22, 2017: Transfusion Clinique et Biologique: Journal de la Société Française de Transfusion Sanguine
https://www.readbyqxmd.com/read/28648672/prognostic-significance-of-interventricular-septal-thickness-in-patients-with-al-amyloidosis
#14
Hyunsoo Cho, Soo-Jeong Kim, Chi Young Shim, Geu-Ru Hong, Jong-Won Ha, Yu Ri Kim, Woo Ick Yang, Haerim Chung, Ji Eun Jang, June-Won Cheong, Yoo Hong Min, Jin Seok Kim
The major prognostic determinant of immunoglobulin light chain (AL) amyloidosis is cardiac involvement. However, the role of interventricular septal thickness (IVST), which reflects the extent of cardiac involvement, remains unclear. Therefore, we analyzed 77 patients with newly diagnosed AL amyloidosis and evaluated the prognostic role of IVST. Fifty patients (64.9%) had cardiac involvement and 17 patients (22.1%) showed IVST >15mm. Among all patients, the revised Mayo Clinic Stage III-IV and IVST >15mm were independently associated with inferior overall survival (OS) in a multivariable analysis...
June 16, 2017: Leukemia Research
https://www.readbyqxmd.com/read/28648365/direct-reprogramming-of-fibroblasts-via-a-chemically-induced-xen-like-state
#15
Xiang Li, Defang Liu, Yantao Ma, Xiaomin Du, Junzhan Jing, Lipeng Wang, Bingqing Xie, Da Sun, Shaoqiang Sun, Xueqin Jin, Xu Zhang, Ting Zhao, Jingyang Guan, Zexuan Yi, Weifeng Lai, Ping Zheng, Zhuo Huang, Yanzhong Chang, Zhen Chai, Jun Xu, Hongkui Deng
Direct lineage reprogramming, including with small molecules, has emerged as a promising approach for generating desired cell types. We recently found that during chemical induction of induced pluripotent stem cells (iPSCs) from mouse fibroblasts, cells pass through an extra-embryonic endoderm (XEN)-like state. Here, we show that these chemically induced XEN-like cells can also be induced to directly reprogram into functional neurons, bypassing the pluripotent state. The induced neurons possess neuron-specific expression profiles, form functional synapses in culture, and further mature after transplantation into the adult mouse brain...
June 20, 2017: Cell Stem Cell
https://www.readbyqxmd.com/read/28648364/differentiation-of-human-pluripotent-stem-cells-into-colonic-organoids-via-transient-activation-of-bmp-signaling
#16
Jorge O Múnera, Nambirajan Sundaram, Scott A Rankin, David Hill, Carey Watson, Maxime Mahe, Jefferson E Vallance, Noah F Shroyer, Katie L Sinagoga, Adrian Zarzoso-Lacoste, Jonathan R Hudson, Jonathan C Howell, Praneet Chatuvedi, Jason R Spence, John M Shannon, Aaron M Zorn, Michael A Helmrath, James M Wells
Gastric and small intestinal organoids differentiated from human pluripotent stem cells (hPSCs) have revolutionized the study of gastrointestinal development and disease. Distal gut tissues such as cecum and colon, however, have proved considerably more challenging to derive in vitro. Here we report the differentiation of human colonic organoids (HCOs) from hPSCs. We found that BMP signaling is required to establish a posterior SATB2+ domain in developing and postnatal intestinal epithelium. Brief activation of BMP signaling is sufficient to activate a posterior HOX code and direct hPSC-derived gut tube cultures into HCOs...
June 20, 2017: Cell Stem Cell
https://www.readbyqxmd.com/read/28647567/stearoyl-coa-desaturase-regulates-sorafenib-resistance-via-modulation-of-er-stress-induced-differentiation
#17
Mark Kin Fai Ma, Eunice Yuen Ting Lau, Doris Hoi Wing Leung, Jessica Lo, Nicole Pui Yu Ho, Lily Kwan Wai Cheng, Stephanie Ma, Chi Ho Lin, John A Copland, Jin Ding, Regina Cheuk Lam Lo, Irene Oi Lin Ng, Terence Kin Wah Lee
We investigated the functional role and clinical significance of Stearoyl CoA desaturase-1 (SCD1) mediated endoplasmic reticulum (ER) stress in regulation of liver tumor-initiating cells (T-ICs) and sorafenib resistance, aiming to develop a novel therapeutic strategy against hepatocellular carcinomas (HCCs) METHODS We evaluated the clinic-pathological relevance of SCD1 and its correlation with sorafenib resistance in large cohorts of HCC clinical samples by qPCR and immunohistochemical analyses. Lentiviral-based overexpression and knockdown approaches were performed to characterize functional roles of SCD1 in regulation of liver T-ICs and sorafenib resistance...
June 21, 2017: Journal of Hepatology
https://www.readbyqxmd.com/read/28647558/durable-chimerism-and-long-term-survival-following-unrelated-umbilical-cord-blood-transplantation-for-pediatric-hemophagocytic-lymphohistiocytosis-a-single-center-experience
#18
Sachit A Patel, Heather A Allewelt, Jesse D Troy, Paul L Martin, Timothy A Driscoll, Vinod K Prasad, Joanne Kurtzberg, Kristin M Page, Suhag H Parikh
Hemophagocytic Lymphohistiocytosis (HLH) is a life-threatening disorder of immune dysregulation characterized by fever, hepatosplenomegaly, cytopenias, CNS disease, increased inflammatory markers and hemophagocytosis. Currently, allogeneic hematopoietic stem cell transplantation is the only curative approach for patients with HLH with reported survival ranging from 50-70% with myeloablative conditioning (MAC) regimens. However, donor availability and transplant related mortality associated with conventional MAC are major barriers to success...
June 21, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28647402/autologous-stem-cell-transplantation-in-patients-with-multiple-myeloma-an-activity-based-costing-analysis-comparing-a-total-inpatient-model-versus-an-early-discharge-model
#19
Massimo Martino, Giuseppe Console, Letteria Russo, Antonella Meliado', Nicola Meliambro, Tiziana Moscato, Giuseppe Irrera, Giuseppe Messina, Antonella Pontari, Fortunato Morabito
BACKGROUND: Activity-based costing (ABC) was developed and advocated as a means of overcoming the systematic distortions of traditional cost accounting. MATERIALS AND METHODS: We calculated the cost of high-dose chemotherapy and autologous stem cell transplantation (ASCT) in patients with multiple myeloma using the ABC method, through 2 different care models: the total inpatient model (TIM) and the early-discharge outpatient model (EDOM) and compared this with the approved diagnosis related-groups (DRG) Italian tariffs...
June 6, 2017: Clinical Lymphoma, Myeloma & Leukemia
https://www.readbyqxmd.com/read/28647301/factors-affecting-hematopoietic-stem-cell-mobilization-and-apheresis-in-allogeneic-donors-the-role-of-iron-status
#20
Zübeyde Nur Özkurt, Leyla Batmaz, Zeynep Arzu Yeğin, Çiğdem İlhan
Infused CD34 cell count has a significant impact on transplant outcome. In this retrospective study, we aimed to analyze the impact of donor iron parameters on peripheral blood stem cell (PBSC) collection. A total of 303 related donors were included in the study. The mobilization regimen, recombinant G-CSF, was given for four consecutive days. A CD34(+) cell count below 2×10(6)/kg was defined as mobilization failure which was demonstrated in 23 donors (7.6%). Mobilization failure was more frequent in female donors than male donors (13...
June 3, 2017: Transfusion and Apheresis Science
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