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haematopoietic stem cell transplant

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https://www.readbyqxmd.com/read/28920953/lifelong-haematopoiesis-is-established-by-hundreds-of-precursors-throughout-mammalian-ontogeny
#1
Miguel Ganuza, Trent Hall, David Finkelstein, Ashley Chabot, Guolian Kang, Shannon McKinney-Freeman
Current dogma asserts that mammalian lifelong blood production is established by a small number of blood progenitors. However, this model is based on assays that require the disruption, transplantation and/or culture of embryonic tissues. Here, we used the sample-to-sample variance of a multicoloured lineage trace reporter to assess the frequency of emerging lifelong blood progenitors while avoiding the disruption, culture or transplantation of embryos. We find that approximately 719 Flk1(+) mesodermal precursors, 633 VE-cadherin(+) endothelial precursors and 545 Vav1(+) nascent blood stem and progenitor cells emerge to establish the haematopoietic system at embryonic days (E)7-E8...
September 18, 2017: Nature Cell Biology
https://www.readbyqxmd.com/read/28906550/efficacy-and-safety-of-autologous-haematopoietic-stem-cell-transplantation-in-systemic-sclerosis-a-systematic-review-of-literature
#2
A Eyraud, L Scouppe, T Barnetche, E Forcade, E Lazaro, P Duffau, C Richez, J Seneschal, M-E Truchetet
We aimed at assessing the efficacy of autologous haematopoietic stem cell transplantation (HSCT) for skin sclerosis and lung function in systemic sclerosis (SSc). We performed a systematic literature review in the Pubmed and Scopus databases from the earliest records to March 2016. We assessed quality using the Cochrane tool for randomized studies, Newcastle-Ottawa scale for controlled-cohort studies and an 18-item quality appraisal checklist for case series. The primary outcome was the improvement of skin thickening using the modified Rodnan Skin Score (mRSS)...
September 14, 2017: British Journal of Dermatology
https://www.readbyqxmd.com/read/28874263/second-neoplasms-in-adult-patients-submitted-to-haematopoietic-stem-cell-transplantation
#3
Anna Torrent, Christelle Ferrá, Mireia Morgades, María-José Jiménez, Juan-Manuel Sancho, Susana Vives, Montserrat Batlle, Miriam Moreno, Blanca Xicoy, Albert Oriol, Gladys Ibarra, Josep-Maria Ribera
BACKGROUND AND OBJECTIVE: Patients submitted to haematopoietic stem cell transplantation (HSCT) are at increased risk of late complications, such as second neoplasm (SN). The incidence and risk factors of SN in patients receiving HSCT at a single centre were analysed. PATIENTS AND METHODS: The follow-up of adult patients who received a first HSCT (autologous [auto-HSCT] or allogeneic [allo-HSCT]) between January 2000 and December 2015 was reviewed. We collected their demographic characteristics, the primary disease and type of HSCT, and analysed the cumulative incidence of SN and their risk factors...
September 2, 2017: Medicina Clínica
https://www.readbyqxmd.com/read/28868142/recent-advances-in-understanding-and-treating-chronic-granulomatous-disease
#4
REVIEW
Andrew Gennery
A number of recent advances have been made in the epidemiology and treatment of chronic granulomatous disease. Several reports from developing regions describe the presentations and progress of local populations, highlighting complications due to Bacillus Calmette-Guérin vaccination. A number of new reports describe complications of chronic granulomatous disease in adult patients, as more survivors reach adulthood. The complications experienced by X-linked carriers are particularly highlighted in three new reports, confirming that infection and inflammatory or autoimmune conditions are more common and severe than previously recognised...
2017: F1000Research
https://www.readbyqxmd.com/read/28866625/autologous-haematopoietic-stem-cell-transplantation-for-neurological-diseases
#5
REVIEW
Joachim Burman, Andreas Tolf, Hans Hägglund, Håkan Askmark
Neuroinflammatory diseases such as multiple sclerosis, neuromyelitis optica, chronic inflammatory demyelinating polyneuropathy and myasthenia gravis are leading causes of physical disability in people of working age. In the last decades significant therapeutic advances have been made that can ameliorate the disease course. Nevertheless, many affected will continue to deteriorate despite treatment, and the costs associated with disease-modifying drugs constitute a significant fiscal burden on healthcare in developed countries...
September 2, 2017: Journal of Neurology, Neurosurgery, and Psychiatry
https://www.readbyqxmd.com/read/28863969/soluble-interleukin-7-receptor-levels-and-risk-of-acute-graft-versus-disease-after-allogeneic-haematopoietic-stem-cell-transplantation
#6
Katrine Kielsen, Zaiba Shamim, Stephanie Thiant, Sylvie Faucher, Wendy Decker, Ib Jarle Christensen, Lars Peter Ryder, Ibrahim Yakoub-Agha, Klaus Müller
Interleukin-7 is a cytokine essential for T cell homeostasis. IL-7 binds to cellular IL-7 receptors in competition with a soluble form of the receptor (sIL-7Rα). We hypothesized that altered sIL-7Rα levels may cause adverse outcomes in patients undergoing HSCT. In parallel, we investigated the impact of the IL-7Rα SNP rs6897932, which has been associated with release of IL-7R. The sIL-7Rα levels decreased during HSCT (from 114ng/ml before to 48ng/ml at day +14 (P<0.0001)). This pattern was inversely mirrored by IL-7...
August 29, 2017: Clinical Immunology: the Official Journal of the Clinical Immunology Society
https://www.readbyqxmd.com/read/28842642/neonatal-umbilical-cord-blood-transplantation-halts-skeletal-disease-progression-in-the-murine-model-of-mps-i
#7
Isabella Azario, Alice Pievani, Federica Del Priore, Laura Antolini, Ludovica Santi, Alessandro Corsi, Lucia Cardinale, Kazuki Sawamoto, Francyne Kubaski, Bernhard Gentner, Maria Ester Bernardo, Maria Grazia Valsecchi, Mara Riminucci, Shunji Tomatsu, Alessandro Aiuti, Andrea Biondi, Marta Serafini
Umbilical cord blood (UCB) is a promising source of stem cells to use in early haematopoietic stem cell transplantation (HSCT) approaches for several genetic diseases that can be diagnosed at birth. Mucopolysaccharidosis type I (MPS-I) is a progressive multi-system disorder caused by deficiency of lysosomal enzyme α-L-iduronidase, and patients treated with allogeneic HSCT at the onset have improved outcome, suggesting to administer such therapy as early as possible. Given that the best characterized MPS-I murine model is an immunocompetent mouse, we here developed a transplantation system based on murine UCB...
August 25, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28840597/retinoic-acid-postconsolidation-therapy-for-high-risk-neuroblastoma-patients-treated-with-autologous-haematopoietic-stem-cell-transplantation
#8
REVIEW
Frank Peinemann, Elvira C van Dalen, Heike Enk, Frank Berthold
BACKGROUND: Neuroblastoma is a rare malignant disease and mainly affects infants and very young children. The tumours mainly develop in the adrenal medullary tissue, with an abdominal mass as the most common presentation. About 50% of patients have metastatic disease at diagnosis. The high-risk group is characterised by metastasis and other features that increase the risk of an adverse outcome. High-risk patients have a five-year event-free survival of less than 50%. Retinoic acid has been shown to inhibit growth of human neuroblastoma cells and has been considered as a potential candidate for improving the outcome of patients with high-risk neuroblastoma...
August 25, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28837858/risk-factors-for-herpes-simplex-virus-1-2-viremia-and-clinical-outcomes-following-unmanipulated-haploidentical-haematopoietic-stem-cell-transplantation
#9
Fei-Fei Tang, Xiao-Su Zhao, Lan-Ping Xu, Xiao-Hui Zhang, Yu-Hong Chen, Xiao-Dong Mo, Yu-Qian Sun, Kai-Yan Liu, Xiao-Jun Huang
BACKGROUND: Herpes simplex virus (HSV)-1/2 can still be reactivated after allogeneic haematopoietic stem cell transplantation (allo-HSCT) even when the prophylactic acyclovir is used. However, the risk factors for HSV-1/2 viremia and the clinical outcomes following unmanipulated haploidentical HSCT remain unknown. OBJECTIVES AND STUDY DESIGN: Nineteen patients with HSV-1/2 viremia and fifty-seven patients without HSV-1/2 viremia which were selected using the case-pair method after undergoing haploidentical HSCT were enrolled...
August 4, 2017: Journal of Clinical Virology: the Official Publication of the Pan American Society for Clinical Virology
https://www.readbyqxmd.com/read/28833474/short-term-side-effects-and-attitudes-towards-second-donation-a-comparison-of-related-and-unrelated-haematopoietic-stem-cell-donors
#10
Simon Pahnke, Gunnar Larfors, Ulla Axdorph-Nygell, Anne Fischer-Nielsen, Eva Haastrup, Dag Heldal, Maija Itälä-Remes, Jan-Erik Johansson, Marjut Kauppila, Stig Lenhoff, Per Ljungman, Riita Niittyvuopio, Anna Sandstedt, Hans Hägglund
The Nordic Register of Haematopoietic Stem Cell Donors (NRHSD) has registered related and unrelated donors from 10 transplant centres in Sweden, Norway, Finland and Denmark since 1998. We present a prospective, observational study of 1,957 donors, focusing mainly on the differences between related and unrelated donors. Related donors are reported to have more comorbidities, but similar side effects compared with unrelated donors. Side effects after BM or PBSC donation are generally of short duration and in this study no deaths, myocardial infarctions, splenic ruptures, or thromboembolic events are reported...
August 18, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/28813413/polylox-barcoding-reveals-haematopoietic-stem-cell-fates-realized-in-vivo
#11
Weike Pei, Thorsten B Feyerabend, Jens Rössler, Xi Wang, Daniel Postrach, Katrin Busch, Immanuel Rode, Kay Klapproth, Nikolaus Dietlein, Claudia Quedenau, Wei Chen, Sascha Sauer, Stephan Wolf, Thomas Höfer, Hans-Reimer Rodewald
Developmental deconvolution of complex organs and tissues at the level of individual cells remains challenging. Non-invasive genetic fate mapping has been widely used, but the low number of distinct fluorescent marker proteins limits its resolution. Much higher numbers of cell markers have been generated using viral integration sites, viral barcodes, and strategies based on transposons and CRISPR-Cas9 genome editing; however, temporal and tissue-specific induction of barcodes in situ has not been achieved. Here we report the development of an artificial DNA recombination locus (termed Polylox) that enables broadly applicable endogenous barcoding based on the Cre-loxP recombination system...
August 16, 2017: Nature
https://www.readbyqxmd.com/read/28807768/european-group-for-blood-and-marrow-transplantation-risk-score-predicts-the-outcome-of-patients-with-acute-leukemia-receiving-single-umbilical-cord-blood-transplantation
#12
Xiaoyu Zhu, Lulu Huang, Changcheng Zheng, Baolin Tang, Huilan Liu, Liangquan Geng, Juan Tong, Lei Zhang, Xuhan Zhang, Wen Yao, Kaidi Song, Xingbing Wang, Kaiyang Ding, Zimin Sun
The European Group for Blood and Marrow Transplantation (EBMT) risk score has been implemented as an important tool to predict patient outcomes after allogeneic hematopoietic stem cell transplantation. However, to our knowledge, this score has never been applied in cases of single umbilical cord blood transplantation (sUCBT). We retrospectively analyzed 207 consecutive patients with acute leukemia who received sUCBT at our center between February 2011 and December 2015. The probabilities of 3-year overall survival (OS) and leukemia-free survival (LFS) of the entire cohort were 65...
August 12, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28806922/hepatitis-b-reactivation-characterized-by-hbsag-negativity-and-anti-hbsag-antibodies-persistence-in-haematopoietic-stem-cell-transplanted-patient-after-lamivudine-withdrawal
#13
C Cerva, G Maffongelli, V Svicher, R Salpini, L Colagrossi, A Battisti, B Mariotti, R Cerretti, L Cudillo, L Sarmati
BACKGROUND: HBV reactivation is associated with high mortality rates in hematopoietic stem cell transplantation (HSCT) and prophylactic lamivudine (LMV) treatment is suggested to prevent this phenomenon. However, the duration of LMV treatment in HSCT patients is not fully defined and the time of immune recovery is considered the best parameter for a drug to be safely interrupted. In patients undergoing allogeneic HSCT, the time of immune recovery is not easy to define and may take years after transplantation and prolonged LMV treatments, which can lead to drug-resistant viral strains...
August 15, 2017: BMC Infectious Diseases
https://www.readbyqxmd.com/read/28796639/development-and-validation-of-a-multivariable-prediction-rule-for-detecting-a-severe-acquired-adamts13-activity-deficiency-in-patients-with-thrombotic-microangiopathies
#14
Jorge M Nieto, Félix De La Fuente-Gonzalo, Fernando A González, Ana Villegas, Rafael Martínez, Manuel E Fuentes, Paloma Ropero
BACKGROUND: Thrombotic microangiopathies (TMAs) are a group of diseases that have different aetiologies and treatments, but a clinical differential diagnosis remains difficult. Among TMAs, thrombotic thrombocytopenic purpura (TTP) is characterised by a severe ADAMTS13 functional deficiency. However, assays exploring ADAMTS13 activity are limited to some specialised laboratories. Our objective was to develop and validate a diagnostic method for TTP in adult patients with TMA. METHODS: We generated a multivariable model (four predictors) on a cohort of 174 TMA patients in order to predict an ADAMTS13 activity deficiency (AUC of 0...
August 10, 2017: Clinical Chemistry and Laboratory Medicine: CCLM
https://www.readbyqxmd.com/read/28791443/outcomes-of-shoulder-arthroplasty-in-haematopoietic-stem-cell-transplant-patients
#15
Joseph M Statz, Brian P Chalmers, Cameron K Ledford, Joaquin Sanchez-Sotelo, John W Sperling, Mark E Morrey
BACKGROUND: With the growing population of patients undergoing haematopoietic stem cell transplants (HSCTs), the demand for shoulder arthroplasty (SA) in this population can be expected to increase. No studies in the literature have examined the outcomes of SA in HSCT patients. PATIENTS AND METHODS: A retrospective review of 11 SAs in 10 patients with previous HSCT was performed. We characterized the clinical outcomes, survivorship, and peri-operative complication rates in patients who underwent SA after receiving a HSCT...
August 9, 2017: International Orthopaedics
https://www.readbyqxmd.com/read/28783141/haematopoietic-stem-cell-transplantation-induces-severe-dysbiosis-in-intestinal-microbiota-of-paediatric-all-patients
#16
K Lähteenmäki, P Wacklin, M Taskinen, E Tuovinen, O Lohi, J Partanen, J Mättö, K Vettenranta
No abstract text is available yet for this article.
August 7, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28782391/in-vitro-expansion-of-cd-133-cells-derived-from-umbilical-cord-blood-in-poly-l-lactic-acid-plla-scaffold-coated-with-fibronectin-and-collagen
#17
Maryam Islami, Yousef Mortazavi, Masoud Soleimani, Samad Nadri
CONTEXT: Due to their renewal and potency, umbilical cord blood (UCB) stem cells have the ability to proliferate and serve as an attractive alternative source for bone marrow transplantation. However, insufficient number of haematopoietic stem cells (HSCs) in UCB is still a major constraint in clinical applications. OBJECTIVE: In vitro expansion of stem cells on fibronectin (Fn)-coated poly-L-lactic acid (PLLA) scaffold can be a proper way to overcome this limitation...
August 6, 2017: Artificial Cells, Nanomedicine, and Biotechnology
https://www.readbyqxmd.com/read/28765176/transient-clinical-improvement-of-a-mitochondrial-neurogastrointestinal-encephalomyopathy-like-syndrome-after-allogeneic-haematopoietic-stem-cell-transplantation
#18
Malcolm Kevin Baker, Clara Maria Schutte, Neelay Ranchhod, David Brittain, J E van Rensburg
Mitochondrial neurogastrointestinal encephalopathy (MNGIE), usually an autosomal-recessive inherited condition, causes gastrointestinal dysmotility, ophthalmoplegia, ptosis, leukoencephalopathy and neuropathy. The chromosome 22 disorder, due to mutations in the nuclear gene TYMP encoding thymidine phosphorylase (TP), leads to the accumulation of thymidine and deoxyuridine, with mitochondrial dysfunction.This report describes a patient with an MNGIE-like syndrome with a heterozygous TYMP mutation who showed marked, but transient improvement postallogeneic haematopoietic stem cell transplantation (HSCT)...
August 1, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28763349/late-respiratory-effects-of-cancer-treatment
#19
Clare Harris, Clare R Sander
PURPOSE OF REVIEW: The aim of this article is to examine significant advances in our understanding of the late respiratory effects of cancer treatment, including surgery, radiotherapy, chemotherapy, biological therapies and haematopoietic stem cell transplant, and to provide a framework for assessing such patients. RECENT FINDINGS: Oncology therapies have advanced considerably over recent years but pulmonary toxicity remains a concern. Advances have been made in our understanding of the risk factors, including genetic ones that lead to toxicity from radiotherapy and chemotherapy and risk stratification models are being developed to aid treatment planning...
September 2017: Current Opinion in Supportive and Palliative Care
https://www.readbyqxmd.com/read/28752571/haematopoietic-stem-cell-transplantation-for-primary-immunodeficiency-syndromes-a-5-year-single-centre-experience
#20
Melissa Norman, Clementine David, Brynn Wainstein, John B Ziegler, Richard Cohn, Richard Mitchell, Tracey O'Brien, Susan Russell, Toby Trahair, Annette Trickett, Katie Frith, Paul Gray
AIM: Haematopoietic stem cell transplantation (HSCT) is a central therapy in the treatment of primary immunodeficiency diseases (PIDs). Over the past 5 years, outcomes have been greatly improved due to earlier diagnosis, improved donor availability, advancements in graft manipulation and the use of less toxic preparative regimens. We present a 5-year audit of HSCT for PID at a single Australian tertiary hospital. METHODS: Retrospective case note review identified diagnosis, pre-transplant medical morbidity, transplant protocol, engraftment, adverse events, post-transplant immune reconstitution and general health...
July 28, 2017: Journal of Paediatrics and Child Health
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