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Myelodysplasia

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https://www.readbyqxmd.com/read/29051180/midostaurin-enasidenib-cpx-351-gemtuzumab-ozogomycin-and-venetoclax-bring-new-hope-to-aml
#1
Andrew H Wei, Ing S Tiong
In 2017, four new drugs have so far received FDA marketing approval for the treatment of acute myeloid leukemia (AML), including targeted therapies for mutant FLT3 (midostaurin) and IDH2 (enasidenib), a novel liposomal cytarabine-daunorubicin formulation (CPX-351) for therapy-related AML and AML with myelodysplasia-related changes and finally, resurgence of an antibody-drug conjugate designed to target CD33 (gemtuzumab ozogomycin; GO). Promising results also emerged for the BCL-2 inhibitor venetoclax combined with low-intensity therapy in older patients with AML unfit for intensive chemotherapy...
October 19, 2017: Blood
https://www.readbyqxmd.com/read/29046759/copper-deficiency-a-new-triad-anemia-leucopenia-and-myeloneuropathy
#2
Shoaib M Wazir, Ibrahim Ghobrial
Clinical copper deficiency is now more frequently recognized. Hematologically, it can present as anemia (microcytic, normocytic, or macrocytic) and neutropenia. Thrombocytopenia is relatively rare. Neurologically, it can manifest as myelopathy and peripheral neuropathy simulating subacute combined degeneration. Bone marrow findings can mimic myelodysplasia resulting in occasional inappropriate referral for bone marrow transplantation. Other conditions with similar presentations include infections, drug toxicity, autoimmunity, B12 deficiency, folate deficiency, myelodysplastic syndrome, aplastic anemia, and lymphoma with bone marrow involvement...
October 2017: Journal of Community Hospital Internal Medicine Perspectives
https://www.readbyqxmd.com/read/29033451/dasatinib-and-azacitidine-followed-by-haploidentical-stem-cell-transplant-for-chronic-myeloid-leukemia-with-evolving-myelodysplasia-a-case-report-and-review-of-treatment-options
#3
Fabian Lang, Lydia Wunderle, Heike Pfeifer, Susanne Schnittger, Gesine Bug, Oliver G Ottmann
BACKGROUND CML presenting with a variant Philadelphia translocation, atypical BCR-ABL transcript, additional chromosomal aberrations, and evolving MDS is uncommon and therapeutically challenging. The prognostic significance of these genetic findings is uncertain, even as singular aberrations, with nearly no data on management and outcome when they coexist. MDS evolving during the course of CML may be either treatment-associated or an independently coexisting disease, and is generally considered to have an inferior prognosis...
October 16, 2017: American Journal of Case Reports
https://www.readbyqxmd.com/read/29025719/aml-with-myelodysplasia-related-changes-masquerades-as-acute-panmyelosis-with-myelofibrosis
#4
Zhaodong Xu
No abstract text is available yet for this article.
October 12, 2017: Blood
https://www.readbyqxmd.com/read/29025286/-thalidomide-therapy-in-relapsed-diffuse-large-b-cell-lymphoma-in-elderly-patients-three-cases
#5
Nikolett Wohner, Gergely Varga, Péter Szloboda, Péter Farkas, András Masszi, Laura Horváth, Gergely Szombath, Judit Várkonyi, Szabolcs Benedek, Tamás Masszi
Diffuse large B-cell lymphoma (DLBCL), a high-grade lymphoproliferative disease, is the most common lymphoma in adults, representing 31% of non-Hodgkin lymphomas (NHL). In elderly patients treatment is problematic because of the high toxicity of standard chemotherapy protocols, especially in relapsed cases, where high-dose chemotherapy and haematopoietic stem cell transplantation would be the best choice. More and more data is becoming available on alternative treatment of refractory/relapsed NHL, including studies on the positive effect of thalidomide and second generation IMiDs in DLBCL, which are already part of the standard treatment protocol in myeloma multiplex and myelodysplasia...
October 2017: Orvosi Hetilap
https://www.readbyqxmd.com/read/28971906/favorable-impact-of-allogeneic-stem-cell-transplantation-in-patients-with-therapy-related-myelodysplasia-regardless-of-tp53-mutational-status
#6
Ibrahim Aldoss, Anh Pham, Sierra Min Li, Ketevan Gendzekhadze, Michelle Afkhami, Mihan Telatar, Hao Hong, Abbas Padeganeh, Victoria Bedell, Thai Cao, Samer K Khaled, Monzr M Al Malki, Amandeep Salhotra, Haris Ali, Ahmed Aribi, Joycelynne Palmer, Patricia Aoun, Ricardo Spielberger, Anthony S Stein, David Snyder, Margaret R O'Donnell, Joyce Murata-Collins, David Senitzer, Dennis Weisenburger, Stephen J Forman, Vinod Pullarkat, Guido Marcucci, Raju Pillai, Ryotaro Nakamura
Therapy-related myelodysplastic syndrome (t-MDS) is a long-term complication of cancer treatment for patients receiving cytotoxic therapy, characterized by high-risk genetics and poor outcomes. Allogeneic hematopoietic cell transplantation represents the only curative modality for t-MDS, but the prognostic impact of pre-transplant genetics and clinical features has not yet been fully characterized. We report here the genetic, clinical characteristics and outcomes of a relatively large cohort of t-MDS patients (n=67) who received alloHCT compared to similarly treated patients with de novo MDS (n=199)...
September 29, 2017: Haematologica
https://www.readbyqxmd.com/read/28958291/therapeutic-targeting-of-rna-splicing-in-myelodysplasia
#7
REVIEW
Young Joon Kim, Omar Abdel-Wahab
Genomic analysis of patients with myelodysplastic syndromes (MDS) has identified that mutations within genes encoding RNA splicing factors represent the most common class of genetic alterations in MDS. These mutations primarily affect SF3B1, SRSF2, U2AF1, and ZRSR2. Current data suggest that these mutations perturb RNA splicing catalysis in a manner distinct from loss of function but how exactly the global changes in RNA splicing imparted by these mutations result in MDS is not well delineated. At the same time, cells bearing mutations in RNA splicing factors are exquisitely dependent on the presence of the remaining wild-type (WT) allele to maintain residual normal splicing for cell survival...
July 2017: Seminars in Hematology
https://www.readbyqxmd.com/read/28943369/clinical-and-urodynamic-results-of-repeated-intradetrusor-onabotulinum-toxin-a-injections-in-refractory-neurogenic-detrusor-overactivity-up-to-five-injections-in-a-cohort-of-children-with-myelodysplasia
#8
Cagri Akin Sekerci, Yiloren Tanidir, Asgar Garayev, Cem Akbal, Tufan Tarcan, Ferruh Simsek
OBJECTIVE: To determine the efficacy and safety of repeated intradetrusor onabotulinum toxin A (onaBoNT-A) injections in children with neurogenic detrusor overactivity due to myelodysplasia. METHODS: The study group consisted of 19 children (4 boys, 15 girls), with a mean age of 10.3±3.1 years old, who had received at least two injections of 10 U/kg onaBoNT-A for the treatment of urinary incontinence resistant to anticholinergic treatment and clean intermittent catheterization in our clinic, between 2010 and 2015...
September 21, 2017: Urology
https://www.readbyqxmd.com/read/28939453/bone-marrow-wt1-levels-in-allogeneic-hematopoietic-stem-cell-transplantation-for-acute-myelogenous-leukemia-and-myelodysplasia-clinically-relevant-time-points-and-100-copies-threshold-value
#9
Josep F Nomdedéu, Albert Esquirol, Maite Carricondo, Marta Pratcorona, Montserrat Hoyos, Ana Garrido, Miguel Rubio, Elena Bussaglia, Irene García-Cadenas, Camino Estivill, Salut Brunet, Rodrigo Martino, Jorge Sierra
The outcome of allogeneic hematopoietic stem cell transplantation (HCT) in patients with myeloid malignancies is better in those without minimal residual disease (MRD) than in those with MRD+, as assessed by multiparametric flow cytometry (MPFC). WT1 quantitation also has been used to assess the probability of relapse in acute myelogenous leukemia (AML) treated with chemotherapy. We analyzed the clinical value of normalized bone marrow WT1 levels as a measure of the expanded myeloid progenitor compartment in a consecutive series of 193 adult patients with myeloid malignancies who underwent HCT...
September 20, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28928957/update-on-umbilical-cord-blood-transplantation
#10
REVIEW
Karen Ballen
Allogeneic hematopoietic cell transplant is a curative procedure for many patients with leukemia, lymphoma, myelodysplasia, myeloproliferative neoplasms, and genetic disorders. Umbilical cord blood transplantation is a graft source for patients who do not have a matched donor in their family or in the unrelated registry. It is particularly difficult for Black, Hispanic, and White patients of non-Western European background to find fully matched adult volunteer donors. An estimated 700,000 umbilical cord blood units have been donated for public use, and over 40,000 umbilical cord blood transplantations have been performed...
2017: F1000Research
https://www.readbyqxmd.com/read/28904628/tibial-derotational-osteotomies-in-two-neuromuscular-populations-comparing-cerebral-palsy-with-myelomeningocele
#11
R M Thompson, S Ihnow, L Dias, V Swaroop
PURPOSE: To review the outcomes of tibial derotational osteotomies (TDOs) as a function of complication and revision surgery rates comparing a cohort of children with myelodysplasia to a cohort with cerebral palsy (CP). METHODS: A chart review was completed on TDOs performed in a tertiary referral centre on patients with myelodysplasia or CP between 1985 and 2013 in patients aged > 5 years with > 2 years follow-up. Charts were reviewed for demographics, direction/degree of derotation, complications and need for re-derotation...
August 1, 2017: Journal of Children's Orthopaedics
https://www.readbyqxmd.com/read/28890262/-relapsing-polychondritis-what-s-new-in-2017
#12
J Dion, G Leroux, L Mouthon, J-C Piette, N Costedoat-Chalumeau
Relapsing polychondritis (RP) is a rare condition characterized by recurrent inflammation of cartilaginous tissue and systemic manifestations. Data on pathophysiology are scarce and suggest an autoimmune mechanism. Recently, the possibility of dividing patients with RP into three distinct clinical phenotypes has been suggested: the hematological form representing less than 10% of patients, essentially older men with associated myelodysplasia and poor prognosis, the respiratory form representing about 25% of patients with predominant tracheobronchial involvement, and the mild and most frequent form, representing 65% of patients, with a good prognosis...
September 7, 2017: La Revue de Médecine Interne
https://www.readbyqxmd.com/read/28886412/re-evaluation-of-acute-erythroid-leukemia-according-to-the-2016-who-classification
#13
Yan Chen, Maryam Pourabdollah, Eshetu G Atenafu, Anne Tierens, Aaron Schimmer, Hong Chang
In the recent update of WHO classification, the definition of myeloid neoplasms with erythroid predominance has been modified shifting the main criteria for calculating blast percentage from non-erythroid cells (NEC) to all nucleated marrow cells (ANC). Thus, the cases previously classified as erythroid/myeloid subtype of acute erythroid leukemia (AEL) based on the 2008 WHO will now be categorized either as myelodysplastic syndrome with excess blasts (MDS-EB) or acute myeloid leukemia, not otherwise specified (AML-NOS)...
October 2017: Leukemia Research
https://www.readbyqxmd.com/read/28866328/splicing-dysfunction-and-disease-the-case-of-granulopoiesis
#14
REVIEW
Maria-Cristina Keightley, Graham J Lieschke
Splicing is a ubiquitous process in eukaryotic cells, long recognised as contributing to diversity of the transcriptome. More specifically, splicing fine-tunes the transcriptome output for highly individual outcomes at different stages of cell development, in specific timeframes, which when perturbed result in significant human diseases. Granulopoiesis provides a particularly well studied example of how splicing can be a highly flexible but tightly regulated process. Focusing on the specific case of granulopoiesis, this review surveys the contribution of cis-splicing variations in individual genes and the trans-regulation of global splicing outcomes during the normal development of neutrophils...
August 30, 2017: Seminars in Cell & Developmental Biology
https://www.readbyqxmd.com/read/28723704/trends-and-territorial-inequalities-of-incidence-and-survival-of-childhood-leukaemia-and-their-relations-to-socioeconomic-status-in-hungary-1971-2015
#15
Zsuzsanna Jakab, Attila Juhasz, Csilla Nagy, Dezso Schuler, Miklos Garami
The Hungarian Childhood Cancer Registry, a population-based national registry of the Hungarian Paediatric Haemato-Oncology Network founded in 1971, monitors the incidence and mortality of childhood cancer. Our aims were to carry out a longitudinal study to investigate the trends and spatial inequalities of incidence and survival of leukaemia, and the association between survival and deprivation in Hungary. All cases of childhood leukaemia and myelodysplasia were analysed (3157 cases, 1971-2015, age: 0-14 years)...
July 18, 2017: European Journal of Cancer Prevention
https://www.readbyqxmd.com/read/28698788/treatment-of-low-blast-count-aml-using-hypomethylating-agents
#16
REVIEW
Eleonora De Bellis, Luana Fianchi, Francesco Buccisano, Marianna Criscuolo, Luca Maurillo, Laura Cicconi, Mattia Brescini, Maria Ilaria Del Principe, Ambra Di Veroli, Adriano Venditti, Sergio Amadori, William Arcese, Francesco Lo-Coco, Maria Teresa Voso
In 2002, the WHO classification reduced the proportion of blasts in the bone marrow (BM) necessary for the diagnosis of acute myeloid leukemia (AML) from 30% to 20%, eliminating the RAEB-t subtype of myelodysplastic syndromes (MDS). However, this AML subtype, defined as low-blast count AML (LBC-AML, with 20-30% BM-blasts) is characterized by peculiar features, as increased frequency in elderly individuals and after cytotoxic treatment for a different primary disease (therapy-related), poor-risk cytogenetics, lower white blood cell counts, and less frequent mutations of NPM1 and FLT3 genes...
2017: Mediterranean Journal of Hematology and Infectious Diseases
https://www.readbyqxmd.com/read/28698733/chronic-renal-failure-secondary-to-unrecognized-neurogenic-bladder-in-a-child-with-myelodysplasia
#17
Shameem Ahmed, Siba Prosad Paul
Myelodysplasia includes a group of developmental anomalies resulting from defects that occur during neural tube closure. Urological morbidity in patients with myelodysplasia is significant and if not treated appropriately in a timely manner can potentially lead to progressive renal failure, requiring dialysis or transplantation. We report the case of a 13-year old girl with neurogenic bladder who presented chronic renal failure secondary to lipomyelomeningocele with retethering of cord. She was managed with urinary indwelling catheterization until optimization of renal function and then underwent detethering of cord with excision and repair of residual lipomeningomyelocele...
2017: Iranian Journal of Child Neurology
https://www.readbyqxmd.com/read/28697759/the-distinct-biological-implications-of-asxl1-mutation-and-its-roles-in-leukemogenesis-revealed-by-a-knock-in-mouse-model
#18
Yueh-Chwen Hsu, Yu-Chiao Chiu, Chien-Chin Lin, Yuan-Yeh Kuo, Hsin-An Hou, Yi-Shiuan Tzeng, Chein-Jun Kao, Po-Han Chuang, Mei-Hsuan Tseng, Tzu-Hung Hsiao, Wen-Chien Chou, Hwei-Fang Tien
BACKGROUND: Additional sex combs-like 1 (ASXL1) is frequently mutated in myeloid malignancies. Recent studies showed that hematopoietic-specific deletion of Asxl1 or overexpression of mutant ASXL1 resulted in myelodysplasia-like disease in mice. However, actual effects of a "physiological" dose of mutant ASXL1 remain unexplored. METHODS: We established a knock-in mouse model bearing the most frequent Asxl1 mutation and studied its pathophysiological effects on mouse hematopoietic system...
July 11, 2017: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/28693140/acute-myeloid-leukemia-with-t-3-21-q26-2-q22-developing-following-low-dose-methotrexate-therapy-for-rheumatoid-arthritis-and-expressing-two-aml1-mds1-evi1-fusion-proteins-a-case-report
#19
Keisuke Tanaka, Gaku Oshikawa, Hiroki Akiyama, Shinya Ishida, Toshikage Nagao, Masahide Yamamoto, Osamu Miura
The t(3;21)(q26.2;q22) translocation is a rare chromosomal abnormality exhibited almost exclusively in therapy-related myelodysplastic syndrome/acute myeloid leukemia (t-MDS/AML) or in the blastic crisis phase of chronic myelogenous leukemia, which results in the fusion of the runt related transcription factor 1 (RUNX1, also called AML1) gene at 21q22 to the myelodysplasia syndrome 1 (MDS1)-ecotropic virus integration site 1 (EVI1) complex locus (MECOM) at 3q26.2, generating various fusion transcripts, including AML1/MDS1/EVI1 (AME)...
July 2017: Oncology Letters
https://www.readbyqxmd.com/read/28689244/changes-in-management-of-poorly-compliant-bladder-in-botulinum-toxin-a-era
#20
REVIEW
Nachiketh Soodana Prakash, Diana M Lopategui, Christopher Gomez
Bladder compliance is a measure of distensibility. Maladies such as myelodysplasia, myelomeningocele, spinal cord injury, multiple sclerosis and obstructive uropathy are known to decrease bladder compliance. Decrease in bladder compliance is a characteristic of neurogenic bladders. The pathophysiology of bladder compliance is complex but ultimately leads to high pressure during filling and storage phases. These high pressures lead to renal impairment, incontinence, and recurrent urinary tract infections. This review presents management of poorly compliant bladders with onabotulinumtoxinA...
August 2017: Current Urology Reports
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