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Haematopoetic transplantation

N Williams, D Challoumas, D M Eastwood
PURPOSE: Mucopolysaccharidoses (MPS) are a group of rare lysosomal storage disorders associated with involvement of multiple organs along with a generalised skeletal dysplasia. Both haematopoetic stem cell transplant and enzyme replacement therapy have improved the outlook for patients while surgery remains high-risk and there is little information on clinical or functional outcome to justify many of the surgical procedures performed. This paper aims to summarise the orthopaedic surgical procedures in MPS patients for which quality of life (QoL) and functional data are available and to describe additional QoL and functional measurement tools of relevance to the assessment of orthopaedic outcomes in MPS...
August 1, 2017: Journal of Children's Orthopaedics
Lauren Host, Mandana Nikpour, Alicia Calderone, Paul Cannell, Janet Roddy
OBJECTIVES: Haematopoetic autologous stem cell transplantation (ASCT) has emerged as a treatment option for patients with refractory, severe autoimmune disease. This is a systematic review of the current literature on ASCT in adult patients with systemic sclerosis (SSc). METHODS: Original articles published between 2005 and 2016 that evaluated the use of ASCT in patients with SSc were reviewed with respect to the primary outcomes of overall and transplant related mortality (TRM) rates, and secondary outcomes of changes in modified Rodnan Skin Score (mRSS), forced vital capacity (FVC), progression/event free survival (P/EFS) and quality of life measures...
September 2017: Clinical and Experimental Rheumatology
Sara Anna Rowinski, Erik Christensen
INTRODUCTION: Refractory coeliac disease (RCD) is a rare and severe malabsorptive disease. The condition has two subtypes: RCDI and RCDII. Different treatments have been tested: and because RCD has a poor prognosis due to progress to enteropathy-associated T-cell lymphoma, the aim was to review the epidemiologic aspects and the therapeutic options for RCD. METHODS: A systematic literature search was performed in 18 databases, and 122 records were identified. Incidence, prevalence, treatment methods and their efficacy were evaluated...
December 2016: Danish Medical Journal
Geothy Chakupurakal, María Alejandra Garcia-Marquez, Alexander Shimabukuro-Vornhagen, Sandra Kluth, Hans Schlosser, Sebastian Theurich, Christof Scheid, Michael Hallek, Udo Holtick, Michael von Bergwelt-Baildon
OBJECTIVE: The role of B cells and the subgroup of IL-10 producing B cells, known to have a regulatory function, in patients following a haematopoietic stem cell transplant (alloSCT) has not been clearly understood to date. METHODS: We prospectively recruited 95 patients following an alloSCT and studied the B-cell reconstitution on days 30, 90 and 150. Regulatory B10 cells could be analysed in 22 consecutively recruited patients on day 30 post-transplant. RESULTS: The total B-cell percentages in transplant recipients (median 0...
February 2017: European Journal of Haematology
Rehan Khan, Sridevi Nair, Tullika Seth, Pravas Mishra, Manoranjan Mahapatra, Tushar Agarwal, Radhika Tandon, Murugesan Vanathi
BACKGROUND & OBJECTIVES: This study was aimed to report the occurrence of ocular graft versus host disease (oGVHD) in allogeneic haematopoietic stem cell transplantation (allo-HSCT) patients in a tertiary care hospital setting. METHODS: A cross-sectional study of ocular surface of allo-HSCT patients was done. Slit lamp biomicroscopy, symptom score, tear meniscus height, fluorescein tear break-up time, Schirmer's test I, ocular surface staining, dry eye severity, ocular surface disease index score were done...
November 2015: Indian Journal of Medical Research
Andrew J Innes, Dragana Milojkovic, Jane F Apperley
The management of chronic myeloid leukaemia (CML) has changed extensively over the past 15 years. Prior to the development of targeted therapies and in the absence of allogeneic haematopoetic stem-cell transplantation (HSCT), the median survival was 5-7 years. HSCT was quickly established as the standard of care for eligible patients through the 1980s and 1990s, when considerable advances were made in the optimization of conditioning regimens and supportive care. Exploiting a deeper understanding of the molecular basis of CML, the development of tyrosine kinase inhibitors (TKIs) in the late 1990s revolutionized the management of the disease...
February 2016: Nature Reviews. Clinical Oncology
Björn M van Geel, Bwee Tien Poll-The, Aad Verrips, Jaap-Jan Boelens, Stephan Kemp, Marc Engelen
BACKGROUND: X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal metabolic disorder. Male patients develop adrenocortical insufficiency (80 % before 18 years), a chronic myelopathy (adrenomyeloneuropathy (AMN); all in adulthood), or progressive cerebral demyelination (cerebral ALD; 40 % before 18 years). Cerebral ALD is treated with haematopoetic cell transplantation (HCT). It is unknown if AMN still develops in patients with X-ALD that underwent HCT for cerebral ALD in childhood. PATIENTS AND METHODS: A retrospective observational study was performed by selecting all adult patients with X-ALD in our cohort that underwent HCT in childhood...
March 2015: Journal of Inherited Metabolic Disease
Chirag Shah
No abstract text is available yet for this article.
2014: Molecular Cytogenetics
Djordje Atanackovic, Georgia Schilling
High-dose chemotherapy followed by autologous haematopoetic stem cell transplantation (ASCT) is a standard frontline therapy for multiple myeloma (MM). Unfortunately, there are no randomized clinical studies examining the role of a second ASCT in patients who relapse after the initial autotransplant. Analysing all available retrospective studies, it seems that salvage ASCT can safely be performed in most patients with an overall treatment-related mortality rate <5%. Approximately 65% of patients will achieve an objective response and progression-free and overall survival will be around 12 months and 32 months, respectively...
December 2013: British Journal of Haematology
A Liu, E Meyer, L Johnston, J Brown, L B Gerson
BACKGROUND: There is lack of consensus regarding whether both upper and lower endoscopic examinations are required for diagnosis of gastrointestinal acute graft versus host disease (GI-AGVHD). AIM: To evaluate the impact of endoscopic procedures on the diagnosis of GI-AGVHD. METHODS: We performed a retrospective case-control study of recipients of allogeneic haematopoetic cell transplant (HCT) from 2000 to 2011, who presented with GI symptoms between 20 and 125 days post-HCT...
October 2013: Alimentary Pharmacology & Therapeutics
Heiner Falkenberg, Teja Falk Radke, Gesine Kögler, Kai Stühler
Ex vivo expansion of haematopoetic cells by application of specific cytokines is one approach to overcome boundaries in cord blood transplantation due to limited numbers of haematopoetic stem cells. While many protocols describe an effective increase of total cell numbers and the amount of CD34-positive cells, it still remains unclear if and how the procedure actually affects the cells' properties. In the presented publications, CD34-positive cells were isolated from cord blood and expanded for up to 7 days in media supplemented with stem cell factor (SCF), thrombopoietin (THPO), interleukin 6 (IL-6), and fms-related tyrosine kinase 3 ligand (FLT3lg)...
2013: Stem Cells International
Adam Denes, Fiona Wilkinson, Brian Bigger, Michael Chu, Nancy J Rothwell, Stuart M Allan
Interleukin-1 (IL-1) is a key regulator of inflammation and ischaemic brain injury, but the contribution of central and peripheral sources of IL-1 to brain injury is not well understood. Here we show that haematopoietic-derived IL-1 is a key driver of ischaemic brain injury. Wild type (WT) mice transplanted with IL-1αβ-deficient bone marrow displayed a significant (40%) reduction in brain injury induced by focal cerebral ischaemia compared with WT mice transplanted with WT bone marrow. This was paralleled by improved neurological outcome and the almost complete absence of splenic-derived, but not liver-derived, IL-1α after stroke in WT mice lacking haematopoietic-derived IL-1...
July 2013: Disease Models & Mechanisms
Michiel G H Betjes
Uraemia causes inflammation and reduces immune system function as evidenced by an increased risk of viral-associated cancers, increased susceptibility to infections and decreased vaccination responses in patients with end-stage renal disease (ESRD). The substantially increased risk of atherosclerosis in these patients is also probably related to uraemia-associated inflammation. Uraemia is associated with a reduction in the number and function of lymphoid cells, whereas numbers of myeloid cells in uraemic patients are normal or increased with increased production of inflammatory cytokines and reactive oxygen species...
May 2013: Nature Reviews. Nephrology
Louise Henningsson, Tove Eneljung, Pernilla Jirholt, Sara Tengvall, Ulf Lidberg, Wim B van den Berg, Fons A van de Loo, Inger Gjertsson
Rheumatoid arthritis (RA) is a chronic destructive autoimmune disease characterised by periods of flare and remission. Today's treatment is based on continuous immunosuppression irrespective of the patient's inflammatory status. When the disease is in remission the therapy is withdrawn but withdrawal attempts often results in inflammatory flares, and re-start of the therapy is commenced when the inflammation again is prominent which leads both to suffering and increased risk of tissue destruction. An attractive alternative treatment would provide a disease-regulated therapy that offers increased anti-inflammatory effect during flares and is inactive during periods of remission...
2012: PloS One
Andreas Pircher, Eva Maria Gassner, Michael Steurer, Dominik Wolf
The optimal management of patients with diffuse large B cell lymphoma relapsing after autologous haematopoetic stem cell transplantation (HCT) is difficult and no standard treatment has been defined. The authors here report the case of a rituximab-naive patient who relapsed after HCT and was cured with rituximab monotherapy.
2012: BMJ Case Reports
Caterina Garone, Saba Tadesse, Michio Hirano
Mitochondrial neurogastrointestinal encephalomyopathy is a rare multisystemic autosomic recessive disorder characterized by: onset typically before the age of 30 years; ptosis; progressive external ophthalmoplegia; gastrointestinal dysmotility; cachexia; peripheral neuropathy; and leucoencephalopathy. The disease is caused by mutations in the TYMP gene encoding thymidine phosphorylasethymine phosphorylase. Anecdotal reports suggest that allogeneic haematopoetic stem cell transplantation may be beneficial for mitochondrial neurogastrointestinal encephalomyopathy, but is associated with a high mortality...
November 2011: Brain: a Journal of Neurology
S Genadieva-Stavrik, B Georgievski, Z Stojanoski, S Krstveska-Balkanov, A Pivkova, M Trajkova, M Genadieva-Dimitrova, V Serafimoski
The myelodisplastic syndrome is a heterogeneous group of diseases, characterised by ineffective and dysplastic haematopoesis and pancytopenia in the peripheral blood, followed by progressive disturbance of differentiation of the haematopoetic stem cell, resulting in evolution of the disease towards acute leukaemia. According to the latest WHO classification, the term myelodisplastic syndrome includes diseases with an indolent course, as well as diseases with a fast evolution towards acute leukaemia. Because of this diversity, haematologists base their therapeutic decisions on prognostic scoring systems which incorporate all the significant factors with an influence on survival in this group of patients with myelodisplastic syndrome...
2011: Prilozi
Tina Dalianis, Per Ljungman
BK virus (BKV)-associated hemorrhagic cystitis (HC), varying from mild hematuria with or without dysuria to life-threating bleeding and clots that may cause urinary obstruction and renal failure, causes significant morbidity and mortality in haematopoetic stem cell transplanted (HSCT) patients. Unfortunately, its development is difficult to predict since BK viruria is very common after HSCT and can be present in patients with and without HC. There is therefore the need to identify risk factors that may increase the risk of developing HC after HSCT...
March 2011: Anticancer Research
Hanne Baekgaard Larsen, Carsten Heilmann, Christoffer Johansen, Lis Adamsen
AIM: The aim of this study was to investigate the parents' experiences and reflections on their parental role while taking care of their child. BACKGROUND: Parents of children undergoing treatment with allogeneic haematopoetic stem cell transplantation for life threatening diseases in protective isolation, experience distress related to the child's care. Parents struggle to cope with the stress related to the child's disease, treatment and survival possibilities...
July 2011: Journal of Advanced Nursing
Lidia Sobkow, F Philipp Seib, Ljupco Prodanov, Ina Kurth, Juliane Drichel, Martin Bornhäuser, Carsten Werner
The therapeutic success of haematopoetic stem and progenitor cell (HSPC) transplantation is critically dependent on HSPC engraftment in the bone marrow. Gradients of stromal cell-derived factor 1 (SDF1) direct HSPC homing, both in vitro and in vivo. Potentially, regulating the delivery levels of exogenous SDF1 applied to the bone marrow could augment HSPC engraftment. Thus, the aim of the present study was to revise the ability of biocompatible hydrogels to direct HSPC migration in vitro. The delivery system of choice is based on heparin cross-linked with collagen1...
August 2011: Annals of Hematology
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