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low-risk myelodysplastic syndromes

Dawn Lee, Nic Brereton, Sujith Dhanasiri, Austin Kulasekararaj
BACKGROUND: Uncertainty within cost-effectiveness analysis, often driven by lack of mature data from large clinical trials, plays a key role in decisions made by the National Institute for Health and Care Excellence (NICE), particularly for early access medicines and orphan drugs. OBJECTIVES: In this case study, we used real-world evidence to address the uncertainty in the cost-effectiveness case for lenalidomide in transfusion-dependent low- and intermediate-1-risk myelodysplastic syndrome (MDS) deletion 5q [del(5q)], affecting a small but unique subpopulation with an orphan disease...
December 14, 2018: PharmacoEconomics Open
Sophie Park, Peter Greenberg, Aylin Yucel, Caroline Farmer, Frank O'Neill, Cisio De Oliveira Brandao, Pierre Fenaux
Many patients with lower-risk myelodysplastic syndrome (MDS) experience anaemia, which has negative consequences. Erythropoiesis-stimulating agents (ESAs) and their biosimilars are used to treat anaemia in MDS and, currently, epoetin alfa and darbepoetin alfa are commonly used and recommended by clinical guidelines. To better understand the evidence available on the use of ESAs for anaemia in lower-risk MDS, we conducted a systematic literature review to identify randomized and nonrandomized prospective studies reporting on clinical efficacy/effectiveness, patient-reported quality of life (QoL), and safety...
December 13, 2018: British Journal of Haematology
Melita Kenealy, Mark Hertzberg, Warwick Benson, Kerry Taylor, Ilona Cunningham, Will Stevenson, Devendra Hiwase, Richard Eek, Daniela Zantomio, Steve Jong, Meaghan Wall, Piers Blombery, Tracey Gerber, Marlyse Debrincat, Diana Zannino, John F Seymour
Standard treatment for higher risk myelodysplastic syndromes, chronic myelomonocytic leukemia and low blast acute myeloid leukemia is azacitidine. In single arm studies, adding lenalidomide had been suggested to improve outcomes. The ALLG MDS4 phase II trial randomized such patients to standard azacitidine or combination azacitidine (75mg/m2/d days 1 to 5) with lenalidomide (10mg days 1-21 of 28-day cycle from cycle 3) to assess clinical benefit (alive without progressive disease) at 12 months. A total 160 patients were enrolled; median age 70...
December 13, 2018: Haematologica
Awachana Jiamsakul, Mark Polizzotto, Stephane Wen-Wei Ku, Junko Tanuma, Eugenie Hui, Romanee Chaiwarith, Sasisopin Kiertiburanakul, Anchalee Avihingasanon, Evy Yunihastuti, Nagalingeswaran Kumarasamy, Penh Sun Ly, Sanjay Pujari, Rossana Ditangco, Cuong Duy Do, Tuti Parwati Merati, Pacharee Kantipong, Fujie Zhang, Kinh Van Nguyen, Adeeba Kamarulzaman, Jun Yong Choi, Benedict Lh Sim, Oon Tek Ng, Jeremy Ross, Wingwai Wong
BackgroundHaematological malignancies have continued to be highly prevalent among people living with HIV (PLHIV). This study assessed the occurrence, risk factors for, and outcomes of haematological and non-haematological malignancies in PLHIV in Asia.MethodsIncidence of malignancy after cohort enrolment was evaluated. Factors associated with development of haematological and non-haematological malignancy were analysed using competing risk regression and survival time using Kaplan-Meier.ResultsOf 7455 patients, 107 patients (1%) developed a malignancy: 34 (0...
November 29, 2018: Journal of Acquired Immune Deficiency Syndromes: JAIDS
Beatrice U Mueller, Katja Seipel, Thomas Pabst
Most patients above 60 years with acute myeloid leukemia (AML) will die from their disease. Nevertheless, the treatment concepts in elderly patients with myelodysplastic syndromes (MDS) and AML are rapidly evolving. A number of recent reports have identified better survival rates with intensive induction chemotherapy for patients up to 80 years, with the exception of patients with unfavorable genomic risk abnormalities or with major co-morbidities. Gemtuzumab ozogamicin is increasingly added to induction therapy for AML patients up to 70 years with favorable or intermediate risk profile, and Midostaurin for patients with a FLT3 mutation...
December 2018: European Journal of Internal Medicine
Ming-Jie Tian, Song-Ying Zhao, Hua Xue, Hui-Mei Guo, Luo-Ming Hua, Li-Xia Fan, Yun Wang, Yue Bian, Yan-Bin Pang
OBJECTIVE: To investigate a reliable clinical indication for predicting the therapeutic response of decitabine therapy in the patients with myelodysplastic syndromes (MDS). METHODS: The clinical efficacy of decitabine for 55 cases of MDS was analyzed retrospectively. According to the lymphocyte level at d28 after the first time treatment with decitabine, the patients were divided into high lymphocyte level group (H-Lym≥1.2×109 /L) and low lymphocyte level group (L-Lym<1...
December 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
Zhen-Hua Bao, Hong-Guo Zhao, Hong-E Yu
OBJECTIVE: To explore the clinical efficacy of decitabine for treatment of patients with myelodysplastic syndrome (MDS) and factors predicting the prognosis. METHODS: The clinical data of 87 patients with MDS treated with decitabine were analyzed retrospectively. The hENT1 mRNA expression and TP53 gene mutation were detected by Q-PCR and gene target sequencing, respectively. The relationship of clinical characteristics and molecular indicators with the clinical response to decitabine was analyzed...
December 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
Jian Wang, Shao-Fen Lin, Qi-Hui Chen, Kun-Yin Qiu, Hong-Gui Xu, Ke Huang, Yang Li, Jian-Pei Fang, Zhou Dun-Hua Zhou
OBJECTIVE: To analyze the clinical features and prognosis of 28 children with myelodysplastic syndrome (MDS) and to screen the high risk factors affecting the prognosis so as to provide the new ideas for standard of clinical diagnosis and therapy. METHODS: The clinical data of 28 children with newly diagnosed MDS treated in our hospital from March 1994 to July 2016 were analyzed retrospectively, the features of disease onset and the results of laboratory examination were summarized, all MDS children were followed up, the prognosis and the high risk factors affecting the prognosis were evaluated...
December 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
Julia Montoro, Helena Pomares, Guillermo Villacampa, Brayan Merchán, Antonieta Molero, Esther Alonso, Laura Gallur, Javier Grau, Olga Salamero, Elisa Roldán, Silvia Saumell, Margarita Ortega, Anna Sureda, Francesc Bosch, Montserrat Arnan, David Valcárcel
In clinical practice, patients with myelodysplastic syndromes (MDS) are usually classified in low or high-risk groups to take therapeutic decisions, conservative for low-risk, whereas active for high-risk. Nevertheless, in the Revised International Prognostic Scoring System (IPSS-R) is not well stated which patients are low or high-risk. This study was aimed to ascertain in 364 MDS patients which IPSS-R threshold better dichotomized in low vs. high-risk. The best dichotomization was obtained with an IPSS-R cut-point of 3...
November 30, 2018: Leukemia & Lymphoma
Daniel Heudobler, Sebastian Klobuch, Simone Thomas, Joachim Hahn, Wolfgang Herr, Albrecht Reichle
Cutaneous manifestations in hematologic malignancies, especially in leukemia, are not common and may be very variable. Here we report a very unusual case of a patient (female, 70 years old) who was admitted to the hospital in 2016 because of skin lesions on the face, the trunk of the body and the extremities. She had a history of breast cancer in the year 2004 (pT1b, pN0, cM0, L0, V0, R0) which had been resected and treated with adjuvant radiation and chemotherapy (cyclophosphamide, methotrexate, 5-fluorouracile) as well as psoriasis treated with methotrexate and cyclosporine...
2018: Frontiers in Pharmacology
Ali T Taher, Raffaella Origa, Silverio Perrotta, Alexandra Kouraklis, Giovan Battista Ruffo, Antonis Kattamis, Ai-Sim Goh, Vicky Huang, Aiesha Zia, Raquel Merino Herranz, John B Porter
BACKGROUND: Adherence to long-term chelation therapy in transfusion-dependent patients is critical to prevent iron overload-related complications. Once-daily deferasirox dispersible tablets (DT) have proven long-term efficacy and safety in patients ≥2 years old with chronic transfusional iron overload. However, barriers to optimal adherence remain, including palatability, preparation time, and requirements for fasting state. A new film-coated tablet (FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light meal...
November 19, 2018: Health and Quality of Life Outcomes
Marie Warny, Jens Helby, Henrik Sengeløv, Børge G Nordestgaard, Henrik Birgens, Stig E Bojesen
OBJECTIVE: Short telomere length is a known risk factor for developing clonal hematopoietic stem cell disorders, probably due to chromosomal instability. We tested the hypotheses that bone marrow mononuclear cell telomere length change from diagnosis through chemotherapy-induced remission and relapse, and that long telomere length is associated with low risk of relapse and all-cause mortality in patients with acute myeloid leukaemia or high-risk myelodysplastic syndrome. METHODS: We measured telomere length in bone marrow mononuclear cells from 233 patients at diagnosis, 112 patients at chemotherapy-induced remission and 58 patients at relapse of disease...
November 14, 2018: European Journal of Haematology
Julie Seguier, Véronique Gelsi-Boyer, Mikael Ebbo, Zeinab Hamidou, Aude Charbonnier, Emmanuelle Bernit, Jean-Marc Durand, Jean-Robert Harlé, Norbert Vey, Nicolas Schleinitz
BACKGROUND: We conducted a monocentric retrospective study of patients with myelodysplastic syndromes (MDS) and autoimmune or inflammatory disorders (AIMs) and a literature review. We analyzed the association with subgroups of the WHO 2016 MDS classification and patient's survival in a case control study. Risk factors associated with survival were analyzed by uni- and multivariate analysis. RESULTS: From all MDS patients 11% presented with AIMs. These were heterogeneous and the most frequent where polyarthritis (25%) and autoimmune cytopenias (17%)...
November 5, 2018: Autoimmunity Reviews
Chao Liu, Wen-Bin An, Jing-Liao Zhang, Ran-Ran Zhang, Cong-Cong Sun, Li-Xian Chang, Tian-Feng Liu, Yao Zou, Hui-Jun Wang, Xiao-Fan Zhu
OBJECTIVE: To investigate the value of multiparameter flow cytometry (MFC) and flow cytometric scoring system (FCSS) in the diagnosis and prognostic evaluation of childhood myelodysplastic syndrome (MDS). METHODS: A retrospective analysis was performed for the clinical data of 42 children who were diagnosed with MDS. MFC was performed to investigate the phenotype and proportion of each lineage of bone marrow cells. The correlations of FCSS score with MDS type, International Prognostic Scoring System (IPSS) score, and revised IPSS (IPSS-R) score were analyzed...
October 2018: Zhongguo Dang Dai Er Ke za Zhi, Chinese Journal of Contemporary Pediatrics
Simone Feurstein, Kathrin Thomay, Winfried Hofmann, Guntram Buesche, Hans Kreipe, Felicitas Thol, Michael Heuser, Arnold Ganser, Brigitte Schlegelberger, Gudrun Göhring
Myelodysplastic syndrome (MDS) can easily transform into acute myeloid leukemia (AML), a process which is often associated with clonal evolution and development of complex karyotypes. Deletion of 5q (del(5q)) is the most frequent aberration in complex karyotypes. This prompted us to analyze clonal evolution in MDS patients with del(5q). There were 1684 patients with low and intermediate-risk MDS and del(5q) with or without one additional cytogenetic abnormality, who were investigated cytogenetically in our department, involving standard karyotyping, fluorescence in situ hybridization (FISH) and multicolor FISH...
October 21, 2018: International Journal of Molecular Sciences
Nikolaos Gardikas, Myrofora Vikentiou, Evgenia Konsta, Christos K Kontos, Sotirios G Papageorgiou, Aris Spathis, Efthimia Bazani, Anthi Bouchla, Violetta Kapsimali, Katherina Psarra, Periklis Foukas, George Dimitriadis, Vasiliki Pappa
BACKGROUND: Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders with unknown aetiology. Multiparameter flow cytometry (MFC) is being evaluated for the diagnosis and prognosis of MDS. METHODS: In the present study, five-color MFC was performed on bone marrow aspirates of 50 untreated patients, newly diagnosed with MDS and 27 age matched control samples. Patients were classified according to World Health Organization 2016, International Prognostic Scoring System (IPSS), and Revised IPSS (IPSS-R)...
October 17, 2018: Cytometry. Part B, Clinical Cytometry
Zhen Ping, Si Chen, Sjoerd J F Hermans, Keane J G Kenswil, Jacqueline Feyen, Claire van Dijk, Eric M J Bindels, Athina M Mylona, Niken M Adisty, Remco M Hoogenboezem, Mathijs A Sanders, Eline M P Cremers, Dicky J Lindenbergh-Kortleve, Janneke N Samsom, Arjan A van de Loosdrecht, Marc H G P Raaijmakers
No abstract text is available yet for this article.
October 12, 2018: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
Yasuhito Nannya
Recent advances in drug treatment for higher-risk myelodysplastic syndromes (MDS) have focused on DNA hypomethylating agents (HMAs). Azacitidine (AZA), a representative HMA available in Japan, has demonstrated a survival benefit over conventional treatment. However, unsatisfactory treatment profiles of AZA exemplified by a low response rate of <20% complete remission (CR), a short duration of response (usually <1 year), and dismal outcomes after the failure to fulfil unmet needs in AZA treatments have highlighted the urgent need for the development of novel therapeutic modalities...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Yasuyoshi Morita
Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders characterized by cytopenia and leukemic transformation. Allogeneic hematopoietic stem cell transplantation is the only potential curative therapy for MDS; however, the first treatment option for transfusion-independent patients with low-risk MDS remains limited. Recently, lenalidomide has become available for patients with low-risk MDS and symptomatic anemia with del (5q) (5q-syndrome), darbepoetin (DA) for those with anemia without del (5q), and azacitidine for those with DA-resistant anemia or anemia with thrombocytopenia...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Chen Mei, Yanling Ren, Xinping Zhou, Li Ye, Liya Ma, Yingwan Luo, Peipei Lin, Weilai Xu, Chenxi Lu, Haiyang Yang, Wenjuan Yu, Liping Mao, Juying Wei, Jie Jin, Hongyan Tong
It is controversial whether acute myeloid leukemia (AML) patients with 20-29% bone marrow (BM) blasts should be considered AML or myelodysplastic syndromes (MDS). We retrospectively studied 382 patients, including 108 AML with 20-29% BM blasts (AML20-29), 210 AML with ≥30% BM blasts (AML ≥ 30), and 64 MDS with 10-19% BM blasts (MDS-EB2). We found that AML20-29 were more similar to MDS-EB2 in terms of advanced age, less blood count, the increased presence of poor-risk cytogenetics. The frequency of mutated genes in AML20-29 had both the characters of AML and MDS...
October 10, 2018: Leukemia & Lymphoma
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