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Refractory Cytopenia

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https://www.readbyqxmd.com/read/27902998/autoimmune-cytopenias-diagnosis-management
#1
Christian P Nixon, Joseph D Sweeney
The autoimmune cytopenias are a related group of disorders in which differentiated hematopoietic cells are destroyed by the immune system. Single lineage disease is characterized by the production of autoantibodies against red cells (autoimmune hemolytic anemia [AIHA]), platelets (autoimmune thrombocytopenia [ITP]) and neutrophils (autoimmune neutropenia [AIN]) whereas multilineage disease may include various combinations of these conditions. Central to the genesis of this disease is the breakdown of central and/or peripheral tolerance, and the subsequent production of autoantibodies by both tissue and circulating self-reactive B lymphocytes with support from T helper lymphocytes...
December 1, 2016: Rhode Island Medical Journal
https://www.readbyqxmd.com/read/27830539/primary-autoimmune-myelofibrosis-a-case-report-and-review-of-the-literature
#2
Yasmin Abaza, C Cameron Yin, Carlos E Bueso-Ramos, Sa A Wang, Srdan Verstovsek
Autoimmune myelofibrosis is a rare, distinct clinicopathological entity that can occur in isolation (primary) or in association with systemic autoimmune disorders (secondary), such as systemic lupus erythematosus and Sjogren's syndrome. This disease is characterized by isolated or combined chronic cytopenias associated with autoimmune phenomena and bone-marrow fibrosis. Due to the rarity of this disease, patients are frequently misdiagnosed as having primary myelofibrosis, the most common form of bone-marrow fibrosis...
November 9, 2016: International Journal of Hematology
https://www.readbyqxmd.com/read/27804150/progressive-refractory-light-chain-amyloidosis-and-multiple-myeloma-patients-are-responsive-to-the-addition-of-clarithromycin-to-imid-based-therapy
#3
Adir Shaulov, Chezi Ganzel, Noam Benyamini, Yossef Barshay, Neta Goldschmidt, David Lavie, Diana Libster, Alex Gural, Batia Avni, Moshe E Gatt
Multiple myeloma (MM) and primary systemic light chain amyloidosis (AL) are both chronic plasma cell dyscrasias with different clinical expression but limited treatment options for relapsed refractory disease. We report the effect of the addition of clarithromycin on 31 MM and 17 AL with relapsed or refractory disease who had an insufficient response or disease progression while on an IMiD based therapy. In this high risk population, hematological response was reported in 48% of MM patients and 94% of AL patients...
November 2, 2016: American Journal of Hematology
https://www.readbyqxmd.com/read/27801319/-analysis-of-the-impact-of-decitabine-treatment-cycles-on-efficacy-and-safety-in-patients-of-myelodysplastic-syndrome-refractory-anemia-with-excess-blasts
#4
X P Luo, Z F Xu, T J Qin, Y Zhang, H L Zhang, L W Fang, L J Pan, N B Hu, S Q Qu, B Li, Z J Xiao
Objective: To explore the impact of decitabine treatment cycles on efficacy and adverse events(AEs)in patients of myelodysplastic syndrome-refractory anemia with excess blasts(MDSRAEB). Methods: A total of fifty-six patients with MDS-RAEB who received decitabine 20 mg·m(-2)·d(-1)by IV infusion daily for 5 consecutive days every 4 weeks at a single institute in China were enrolled from December 2008 to March 2016. Their clinical features, efficacy, predictors of efficacy and AEs were analyzed retrospectively...
October 14, 2016: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/27761260/hemophagocytic-lymphohistiocytosis-as-an-unusual-cause-of-prolonged-bicytopenia-after-chemotherapy
#5
Enrico Schalk, Thomas Fischer
Hemophagocytic lymphohistiocytosis (HLH) is a rare cause of cytopenia and is often associated with lymphoma. The occurrence of HLH in the course of lymphoma treatment could be an indicator for refractory disease.
October 2016: Clinical Case Reports
https://www.readbyqxmd.com/read/27748021/characteristics-of-the-phenotypic-abnormalities-of-bone-marrow-cells-in-childhood-myelodysplastic-syndromes-and-juvenile-myelomonocytic-leukemia
#6
Anita F Oliveira, Aline Tansini, Daniel O Vidal, Luiz F Lopes, Konradin Metze, Irene Lorand-Metze
BACKGROUND: Immunophenotyping of bone marrow (BM) hemopoietic precursors is useful for diagnosis of adult myelodysplastic syndrome (MDS), but data concerning pediatric patients are limited. We analyzed immunophenotypic features of BM cells at diagnosis of children who were referred to the Brazilian Pediatric Cooperative Group of Myelodysplastic Syndromes. METHODS: Diagnosis was based on clinical information, peripheral blood counts, BM cytology and cytogenetics...
October 17, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27721370/ruxolitinib-treatment-for-gvhd-in-patients-with-myelofibrosis
#7
Y Mori, K Ikeda, T Inomata, G Yoshimoto, N Fujii, H Ago, T Teshima
Jak1/2 inhibitor ruxolitinib is a promising agent for treating steroid-refractory GvHD after allogeneic hematopoietic stem cell transplantation (SCT) to produce quick and durable responses. However, optimal dose and tapering schedule of ruxolitinib remain to be determined. Discontinuation of ruxolitinib in myelofibrosis often induces 'withdrawal syndrome' characterized by acute relapse of the disease, but this issue is not well addressed in the treatment of GvHD. Four patients with GvHD (one acute and three chronic) after SCT for myelofibrosis were treated with ruxolitinib...
October 10, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27718324/childhood-onset-autoimmune-cytopenia-as-the-presenting-feature-of-biallelic-acp5-mutations
#8
Anne-Sylvia Sacri, Annelyse Bruwier, Geneviève Baujat, Sylvain Breton, Stéphane Blanche, Tracy A Briggs, Brigitte Bader-Meunier
Childhood-onset chronic and refractory cytopenias are rare and may be genetic in etiology. We report three pediatric cases of severe autoimmune thrombocytopenia or anemia associated with growth retardation and spastic diplegia with intracranial calcification. The identification of platyspondyly and metaphyseal lesions suggested a potential diagnosis of spondyloenchondrodysplasia (SPENCD), which was confirmed with the identification of biallelic ACP5 mutations. Two patients demonstrated elevated serum interferon alpha levels...
October 8, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27715070/-clinically-relevant-possibilities-and-limits-of-differential-diagnosis-of-megaloblastic-anemia-and-myelodysplastic-syndrome%C3%A2-refractory-anemia-type-in-bone-marrow-biopsies
#9
Petra Vašeková, Peter Szépe, Ján Marcinek, Tomáš Balhárek, Lukáš Plank
INTRODUCTION: Megaloblastic anemia (MA) represents a subtype of macrocytic anemia caused by impaired DNA synthesis, mostly due to folate and vitamin B12 deficiency. Its mildest forms lead to macrocytosis without concomitant anemia, but more severe forms to thrombocytopenia and/or leucopenia as well. In majority of the cases, the diagnosis of MA dose not represent a serious clinical problem, however, other causes of macrocytosis including myelodysplastic syndrome (MDS) must be excluded...
2016: Vnitr̆ní Lékar̆ství
https://www.readbyqxmd.com/read/27699872/are-myelodysplastic-syndromes-underdiagnosed-in-poland-a-report-by-the-polish-adult-leukaemia-group
#10
Joanna E Drozd-Sokołowska, Krzysztof Mądry, Anna Waszczuk-Gajda, Tomasz Żółtak, Anna Sikorska, Andrzej Mital, Jarosław Wajs, Grażyna Semeńczuk, Anna Szmigielska-Kapłon, Magdalena Szczepańska, Ewa Wasilewska, Paweł Szwedyk, Jadwiga Hołojda, Marzena Wątek, Beata Stella-Hołowiecka, Maria Soroka-Wojtaszko, Wojciech Homenda, Mirosław Polak, Renata Guzicka-Kazimierska, Agnieszka Porowska, Wiesław Wiktor-Jędrzejczak, Jadwiga Dwilewicz-Trojaczek
OBJECTIVES: The epidemiology of myelodysplastic syndromes (MDS) differs among countries. Here, we present the first epidemiological indices determined for Poland. METHODS: Twenty-one haematological centres participated in the study. Patients diagnosed with MDS and acute myeloid leukaemia (AML) with 20-29% blasts were enrolled. Data collection was conducted for strictly predefined period. RESULTS: The overall crude incidence rate for all MDS subtypes was 1...
October 4, 2016: European Journal of Haematology
https://www.readbyqxmd.com/read/27695288/eltrombopag-for-the-treatment-of-aplastic-anemia-current-perspectives
#11
Su Han Lum, John D Grainger
Aplastic anemia (AA) is a potential life-threatening hematopoietic stem cell (HSC) disorder resulting in cytopenia. The mainstays of treatment for AA are definitive therapy to restore HSCs and supportive measures to ameliorate cytopenia-related complications. The standard definitive therapy is HSC transplantation for young and medically fit patients with suitable donors and immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporine for the remaining patients. A significant proportion of patients are refractory to IST or relapse after IST...
2016: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/27693133/changes-in-the-updated-2016-who-classification-of-the-myelodysplastic-syndromes-and-related-myeloid-neoplasms
#12
John M Bennett
In comparison with the 2008 World Health Organization "Blue Book" on hematopoietic neoplasms, a small number of changes have been made in the classification. In the lower-risk patients, Refractory Cytopenias with Multilineage Dysplasia and Ring Sideroblasts (RCMD-RS) has been separated from RCMD to recognize the importance of the SF3B1 mutation. Often there has been confusion as to the degree of morphologic dysplasia and/or cytopenias to define some of the lower-risk subtypes. In addition, the type of dysplasia or cytopenias is not always concordant...
November 2016: Clinical Lymphoma, Myeloma & Leukemia
https://www.readbyqxmd.com/read/27638045/phase-1-study-evaluating-the-safety-and-pharmacokinetics-of-pralatrexate-in-relapsed-refractory-advanced-solid-tumors-and-lymphoma-patients-with-mild-moderate-and-severe-renal-impairment
#13
Kevin R Kelly, Nashat Gabrail, Steven Weitman, John Sarantopoulos, Anthony J Olszanski, William Edenfield, Jurgen Venitz, Guru Reddy, Allen Yang, Steven J Hasal, A Craig Lockhart
PURPOSE: Pralatrexate is a folate analogue indicated for the treatment of relapsed or refractory peripheral T-cell lymphoma. It has not been formally tested in patients with renal impairment. This study evaluated the pharmacokinetic (PK) profile of pralatrexate in patients with renal impairment and with relapsed/refractory advanced solid tumors and lymphoma. METHODS: This was an open-label, nonrandomized, phase 1 study. Eligible patients received pralatrexate administered as an IV push over 3-5 min once weekly for 6 weeks in 7-week cycles until progressive disease or intolerable toxicity...
September 16, 2016: Cancer Chemotherapy and Pharmacology
https://www.readbyqxmd.com/read/27637985/ibrutinib-for-patients-with-relapsed-or-refractory-chronic-lymphocytic-leukaemia-with-17p-deletion-resonate-17-a-phase-2-open-label-multicentre-study
#14
Susan O'Brien, Jeffrey A Jones, Steven E Coutre, Anthony R Mato, Peter Hillmen, Constantine Tam, Anders Österborg, Tanya Siddiqi, Michael J Thirman, Richard R Furman, Osman Ilhan, Michael J Keating, Timothy G Call, Jennifer R Brown, Michelle Stevens-Brogan, Yunfeng Li, Fong Clow, Danelle F James, Alvina D Chu, Michael Hallek, Stephan Stilgenbauer
BACKGROUND: The TP53 gene, encoding tumour suppressor protein p53, is located on the short arm of chromosome 17 (17p). Patients with 17p deletion (del17p) chronic lymphocytic leukaemia have poor responses and survival after chemoimmunotherapy. We assessed the activity and safety of ibrutinib, an oral covalent inhibitor of Bruton's tyrosine kinase, in relapsed or refractory patients with del17p chronic lymphocytic leukaemia or small lymphocytic lymphoma. METHODS: We did a multicentre, international, open-label, single-arm study at 40 sites in the USA, Canada, Europe, Australia, and New Zealand...
October 2016: Lancet Oncology
https://www.readbyqxmd.com/read/27615037/a-novel-immunomodulatory-treatment-involving-mycophenolate-mofetil-and-corticosteroids-for-pediatric-autoimmune-cytopenias
#15
Arun Panigrahi, Amy Clark, John Myers, Ashok Raj
BACKGROUND: Successful treatment of both pediatric autoimmune hemolytic anemia (AIHA) and immune thrombocytopenic purpura (ITP), specifically those that are refractory to first-line therapies, remains unsatisfactory in terms of long-term remission and medication side effects. Here, we propose a novel combination therapy of mycophenolate mofetil (MMF), an adjunct immunosuppressive, and short-term corticosteroids for the treatment of persistent or chronic autoimmune cytopenias in children...
September 12, 2016: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/27596146/-allogeneic-stem-cell-transplantation-an-option-for-autoimmune-diseases
#16
S Wirths, W Bethge, J C Henes
According to experimental animal models and experiences of patients with coexisting autoimmune diseases, allogeneic stem cell transplantation has the potential to reestablish and maintain immunological tolerance. On the other hand, it is associated with significant treatment related mortality and may induce diverse immunological diseases by graft-versus-host reaction. Other than with severe aplastic anemia, it is not an established therapy for autoimmune diseases; it is under investigation in clinical trials and might be considered in severe, refractory immune cytopenia...
October 2016: Zeitschrift Für Rheumatologie
https://www.readbyqxmd.com/read/27544076/a-phase-i-trial-of-the-human-double-minute-2-inhibitor-mk-8242-in-patients-with-refractory-recurrent-acute-myelogenous-leukemia-aml
#17
Farhad Ravandi, Ivana Gojo, Mrinal M Patnaik, Mark D Minden, Hagop Kantarjian, Amy O Johnson-Levonas, Craig Fancourt, Raymond Lam, Mary Beth Jones, Clayton D Knox, Shelonitda Rose, Payal Shah Patel, Raoul Tibes
OBJECTIVE: Evaluate safety/tolerability/efficacy of MK-8242 in subjects with refractory/recurrent AML. METHODS: MK-8242 was dosed p.o. QD (30-250mg) or BID (120-250mg) for 7on/7off in 28-day cycle. Dosing was modified to 7on/14off, in 21-day cycle (210 or 300mg BID). RESULTS: 26 subjects enrolled (24 evaluable for response); 5/26 discontinued due to AEs. There were 7 deaths; 1 (fungal pneumonia due to marrow aplasia) possibly drug-related...
September 2016: Leukemia Research
https://www.readbyqxmd.com/read/27515671/the-use-of-cyclosporine-a-in-rheumatology-a-2016-comprehensive-review
#18
Cecilia Beatrice Chighizola, Voon H Ong, Pier Luigi Meroni
Cyclosporine A, an inhibitor of calcineurin, exerts an immunomodulator action interfering with T cell activation. Even though novel therapeutic tools have emerged, CyA still represents a suitable option in several clinical rheumatology settings. This is the case of refractory nephritis and cytopenias associated with systemic lupus erythematosus. Furthermore, CyA is a valued therapeutic tool in the management of uveitis and thrombophlebitis in course of Behçet's disease. Topical CyA has been proven to be beneficial in the dry eye of Sjogren's syndrome, whereas oral treatment with CyA can be considered for the severe complications of adult onset Still's disease...
August 11, 2016: Clinical Reviews in Allergy & Immunology
https://www.readbyqxmd.com/read/27454522/busulfan-is-effective-second-line-therapy-for-older-patients-with-philadelphia-negative-myeloproliferative-neoplasms-intolerant-of-or-unresponsive-to-hydroxyurea
#19
Genevieve Douglas, Claire Harrison, Cecily Forsyth, Michael Bennett, William Stevenson, John Hounsell, Sumita Ratnasingam, David Ritchie, David M Ross, Andrew Grigg
Hydroxyurea (Hu) is widely used as first-line cytoreductive therapy for patients with high-risk Philadelphia-negative myeloproliferative neoplasms (Ph-neg MPN), but a small proportion of patients have refractory disease or experience adverse effects. Studies have demonstrated busulfan (Bu) to be an active first-line agent, but data on its role as second-line or later therapy are minimal. To evaluate its efficacy and safety in this context, we undertook a multicenter audit of Ph-neg MPN patients who had received Bu as therapy for Hu intolerance or failure...
January 2017: Leukemia & Lymphoma
https://www.readbyqxmd.com/read/27412026/multiple-colonic-ulcers-associated-with-trisomy-8-serial-changes-in-colonoscopic-findings
#20
Shunichi Yanai, Shotaro Nakamura, Keisuke Kawasaki, Shigeki Ito, Tamotsu Sugai, Takayuki Matsumoto
We report a 54-year-old female patient with myelodysplastic syndrome (MDS) associated with trisomy 8, who had multiple colonic ulcers. The patient had been diagnosed as having MDS of refractory cytopenia with trisomy 8 10 years previously. She underwent colonoscopy for abdominal pain, which revealed severe circumferential stenosis with multiple ulcers in the ileocecal region and a discrete excavating ulcer in the transverse colon. The patient had been free from any dermatological, oral, genital or ocular symptoms suggestive of Behçet's disease (BD)...
October 2016: Clinical Journal of Gastroenterology
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