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Refractory Cytopenia

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https://www.readbyqxmd.com/read/29873072/evaluation-of-230-patients-with-relapsed-refractory-deletion-17p-chronic-lymphocytic-leukaemia-treated-with-ibrutinib-from-3-clinical-trials
#1
Jeffrey Jones, Anthony Mato, Steven Coutre, John C Byrd, Richard R Furman, Peter Hillmen, Anders Osterborg, Constantine Tam, Stephan Stilgenbauer, William G Wierda, Nyla A Heerema, Karl Eckert, Fong Clow, Cathy Zhou, Alvina D Chu, Danelle F James, Susan M O'Brien
Patients with chronic lymphocytic leukaemia/small lymphocytic lymphoma (CLL/SLL) with deletion 17p [del(17p)] have poor outcomes with chemoimmunotherapy. Ibrutinib is indicated for the treatment of CLL/SLL, including del(17p) CLL/SLL, and allows for treatment without chemotherapy. This integrated analysis was performed to evaluate outcomes in 230 patients with relapsed/refractory del(17p) CLL/SLL from three ibrutinib studies. With a median of 2 prior therapies (range, 1-12), 18% and 79% of evaluable patients had del(11q) or unmutated IGHV, respectively...
June 5, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29843647/haemophagocytic-lymphohistiocytosis-occurred-during-induction-chemotherapy-in-an-acute-monocytic-leukemia-patient-with-flt3-itd-and-dnmt3a-mutations
#2
Fei Li, Xiaojie Zhang, Yunyun Wang, Ailin Yang, Zhanglin Zhang, Weiping Tang, Nan Zhong, Huidong Shi
BACKGROUND: Haemophagocytic lymphohistiocytosis (HLH) is considered to be a large challenge for clinicians due to the variable overlaps of symptoms with other severe diseases and a high rate of mortality. Prompt diagnosis and treatment are crucial to avoid a fatal outcome. However, very limited reports have focused on HLH during chemotherapy (Ch-HLH) due to a low incidence and insufficient knowledge. CASE PRESENTATION: A 22-year-old male was diagnosed with acute monocytic leukemia with FLT3-ITD and DNMT3A mutations and pulmonary infection...
May 29, 2018: BMC Cancer
https://www.readbyqxmd.com/read/29779353/-using-target-next-generation-sequencing-assay-in-diagnosing-of-46-patients-with-suspected-congenital-anemias
#3
Y Li, G X Peng, Q Y Gao, Y Li, L Ye, J P Li, L Song, H H Fan, Y Yang, Y Z Xiong, Z J Wu, W R Yang, K Zhou, X Zhao, L P Jing, F K Zhang, L Zhang
Objective: To evaluate the impact of the targeted next-generation sequencing (NGS) assay for difficult congenital anemias. Methods: Blood Disease Hospital Anemia Panel 2014 (BDHAP-2014) including 217 known genes of congenital anemias was developed. NGS and parental verification were performed for patients who were suspected diagnosed with congenital anaemia from August 2014 to July 2017. Results: A total of 46 patients were enrolled in this study, the clinical suspection were 11 cases Fanconi anemia (FA), 8 cases congenital dyserythropoietic anemia (CDA), 6 cases congenital sideroblast anemia (CSA), 12 cases congenital hemolytic anemia (CHA), 1 case dyskeratosis congenital (DC), 4 cases iron-refractory iron deficiency anemia and 4 cases unexplained cytopenia (Uc), respectively...
May 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29773429/real-life-experience-with-ponatinib-in-chronic-myeloid-leukemia-a-multicenter-observational-study
#4
Adi Shacham-Abulafia, Pia Raanani, David Lavie, Yulia Volchek, Ron Ram, Ilana Helman, Liat Shargian, Anna Gourevitch, Evgeni Chubar, Roy Ratzon, Uri Rozovski
BACKGROUND: The strict recruitment criteria of patients for clinical trials often lead to reduced generalizability of the findings. We studied how ponatinib is used outside clinical trials in patients with chronic myeloid leukemia (CML). PATIENTS AND METHODS: The present retrospective study included all patients with a diagnosis of CML who had received ponatinib in 7 medical centers in Israel. RESULTS: From 2011 to 2016, we identified 37 patients with CML who had received ponatinib, 21 in the chronic phase and 16 in the advanced phase...
May 7, 2018: Clinical Lymphoma, Myeloma & Leukemia
https://www.readbyqxmd.com/read/29766398/hepatosplenic-t-cell-lymphoma-a-unifying-entity-in-a-patient-with-hemolytic-anemia-massive-splenomegaly-and-liver-dysfunction
#5
Marianna Mavilia, Agnes McAuliffe, Safina Hafeez, Haleh Vaziri
Hepatosplenic T cell lymphoma (HSTCL) is a rare subtype of peripheral non-Hodgkin lymphoma, of which 20% of cases are associated with chronic immunosuppression. It often occurs in patients with inflammatory bowel disease treated with immunomodulating medications such a thiopurines or TNF-alpha inhibitors. Cytopenias are commonly seen but autoimmune hemolytic anemia (AIHA) is rare. Here we present a young male with longstanding ulcerative colitis on chronic azathioprine, exhibiting several rare features of HSTCL...
May 15, 2018: Clinical Journal of Gastroenterology
https://www.readbyqxmd.com/read/29743397/-transplantation-associated-thrombotic-microangiopathy-confirmed-by-renal-biopsy
#6
Satoshi Miyamoto, Shunsuke Kimura, Yosuke Hosoya, Daisuke Hasegawa, Hisashi Ishida, Atsuro Daida, Toshihiro Matsui, Yuri Yoshimoto, Shinsuke Hirabayashi, Takuya Fujimaru, Tadashi Kumamoto, Shin-Ichiro Mori, Koyu Suzuki, Atsushi Manabe
An eight-year-old girl with myelodysplastic syndrome (refractory cytopenia) received a bone marrow transplant (BMT) from an unrelated donor because of immunosuppressive therapy failure. Following administration of foscarnet for cytomegalovirus reactivation at day40 post-BMT, serum creatinine increased, and proteinuria, hematuria, and hypertension gradually exacerbated and became prolonged. However, neither schistocytosis nor other organ damage was evident. At six months post-BMT, renal biopsy revealed diffuse glomerular damage with glomerular lobulation, a double contour of the glomerular basement membrane, erythrocyte congestion and thrombi in the glomerular endocapillaries, and mesangiolysis, confirming the diagnosis of transplantation-associated thrombotic microangiopathy (TA-TMA)...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/29741506/recent-advances-in-the-cellular-and-molecular-understanding-of-myelodysplastic-syndromes-implications-for-new-therapeutic-approaches
#7
Andrew M Brunner, David P Steensma
It has been more than 10 years since any new disease-modifying therapies have received regulatory approval for indications related to myelodysplastic syndromes (MDS). Advances in our collective biological understanding of MDS in the last decade, however, have made it possible to hope that effective therapeutics can be designed to improve MDS-associated cytopenias and patients' quality of life, and perhaps even delay clonal progression and extend survival. Classes of MDS-associated mutations and disordered biological pathways targeted by developmental therapeutics include the following: aberrant messenger RNA splicing, neomorphic enzymes in the citric acid cycle with oncogenic activity, overactivated tyrosine and serine-threonine kinases, epigenetic and chromatin remodeling alterations, abnormal telomere dynamics, and failed protection of DNA integrity...
January 2018: Clinical Advances in Hematology & Oncology: H&O
https://www.readbyqxmd.com/read/29732477/effectiveness-of-rituximab-containing-treatment-regimens-in-idiopathic-multicentric-castleman-disease
#8
Yujun Dong, Lu Zhang, Lin Nong, Lihong Wang, Zeyin Liang, Daobin Zhou, David C Fajgenbaum, Hanyun Ren, Jian Li
Human herpes virus type 8 (HHV-8)-negative, idiopathic multicentric Castleman disease (iMCD) is a rare lymphoproliferative disease often involving constitutional symptoms, cytopenias, and multiple organ system dysfunction. In China, the majority of MCD cases are HHV-8 negative. Given that siltuximab, the only FDA-approved treatment for iMCD is not available in China; rituximab- and cyclophosphamide-containing regimens are often used in the treatment of Chinese iMCD patients. To evaluate the efficacy of rituximab in this rare and heterogeneous disease, clinical and pathological data from 27 cases of iMCD were retrospectively analyzed from two large medical centers in China...
May 7, 2018: Annals of Hematology
https://www.readbyqxmd.com/read/29684566/eltrombopag-for-the-treatment-of-refractory-pure-rbc-aplasia-after-major-abo-incompatible-hematopoietic-stem-cell-transplantation
#9
Alessandro Busca, Chiara Dellacasa, Luisa Giaccone, Sara Manetta, Lucia Biale, Laura Godio, Semra Aydin, Moreno Festuccia, Lucia Brunello, Benedetto Bruno
Pure RBC aplasia (PRCS) is a well-recognized complication after allogeneic hematopoietic stem cell transplantation (HSCT). Many therapeutic options are available to treat this condition, including erythropoietin, rituximab, bortezomib, plasma exchange, immunoadsorption, donor lymphocyte infusion, mesenchymal stem cells, antithymocyte globulin, and high-dose steroids; however, treatment outcomes are often variable and can sometimes lead to disappointing results. In this brief article we report our experience with 2 patients with PRCA after major ABO-incompatible HSCT who were resistant to multiple therapeutic interventions and who eventually benefited from treatment with eltrombopag, a thrombopoietin mimetic approved by the US Food and Drug Administration for the treatment of patients with immune thrombocytopenic purpura or severe aplastic anemia refractory to immunosuppressive agents or not eligible for HSCT...
April 21, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29618691/-monomac-syndrome-patient-developing-myelodysplastic-syndrome-following-persistent-ebv-infection
#10
Hideyuki Yamamoto, Hikaru Hattori, Erina Takagi, Takanobu Morishita, Yuichi Ishikawa, Seitaro Terakura, Tetsuya Nishida, Yoshinori Ito, Makoto Murata, Hitoshi Kiyoi
An 18-year-old man was diagnosed with Epstein-Barr virus (EBV) -associated hemophagocytic syndrome (HPS) and treated with prednisolone (PSL) at a previous hospital. During PSL tapering, the HPS symptoms reappeared, and the patient was referred to our hospital. Increased PSL improved the symptoms, but the EBV infection remained unresolved. At age 20, he was admitted to our hospital for newly developed pneumonia and diagnosed with myelodysplastic syndrome (refractory cytopenia with multilineage dysplasia) (MDS-RCMD; normal karyotype, IPSS: Int-1) by bone marrow examination...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/29500545/effect-of-qinghuang-powder-combined-with-bupi-yishen-decoction-in-treating-patients-with-refractory-cytopenia-with-multilineage-dysplasia-through-regulating-dna-methylation
#11
Qing-Bing Zhou, Xiao-Hong Yang, Hong-Zhi Wang, De-Xiu Wang, Yong-Gang Xu, Xiao-Mei Hu, Feng-Qin Xu, Rou Ma
OBJECTIVE: To explore the effect of Qinghuang Powder (QHP, ) combined with Bupi Yishen Decoction (BPYS, ) on myelodysplastic syndromes (MDS) patients with refractory cytopenia with multilineage dysplasia (RCMD) and determine the change of DNA methylation in MDS-RCMD patients after the treatment of Chinese medicine formula. METHODS: All 308 MDS-RCMD patients were treated with QHP combined with BPYS for 2 months at least, absolute neutrophil count (ANC), hemoglobin (Hb), platelets (PLT), primitive bone marrow cells and chromosome karyotype were chosen as the main evaluation indexes to analyze the treatment effect according to criteria from the MDS International Working Group...
March 2, 2018: Chinese Journal of Integrative Medicine
https://www.readbyqxmd.com/read/29471300/yttrium-90-ibritumomab-tiuxetan-zevalin%C3%A2-radioimmunotherapy-after-cytoreduction-with-eshap-chemotherapy-in-patients-with-relapsed-follicular-non-hodgkin-lymphoma-final-results-of-a-phase-ii-study
#12
Soham D Puvvada, José M Guillén-Rodríguez, Jessica Yan, Lora Inclán, Kara Heard, Xavier I Rivera, Faiz Anwer, Daruka Mahadevan, Jonathan H Schatz, Daniel O Persky
BACKGROUND: Radioimmunotherapy (RIT) is effective in treating relapsed/refractory follicular lymphoma (FL), with durable remissions in first-line consolidation. We hypothesized that RIT with ibritumomab tiuxetan (Zevalin®) would result in durable remissions by eliminating minimal residual disease after cytoreduction. METHODS: Patients with FL received 2 cycles of ESHAP (etoposide, methylprednisolone, cytarabine, cisplatin) every 28 days, followed by Zevalin 4-6 weeks later if there was no disease progression and bone marrow biopsy showed < 25% involvement...
2018: Oncology
https://www.readbyqxmd.com/read/29427826/tocilizumab-and-refractory-takayasu-disease-four-case-reports-and-systematic-review
#13
REVIEW
Paul Decker, Pierre Olivier, Jessie Risse, Stéphane Zuily, Denis Wahl
BACKGROUND: Relapses upon corticosteroids tapering and immunosuppressive agents are frequent in Takayasu arteritis (TA). Interleukin-6 is highly involved in physiopathology of TA. Many reports showed efficacy of tocilizumab (TCZ) in refractory TA cases. We report four cases and an updated literature review on the TCZ efficacy and safety in patients with TA. METHODS: Patients with TA defined by ACR 1990 criteria were included. Clinical, biological and imaging data were retrospectively reported...
April 2018: Autoimmunity Reviews
https://www.readbyqxmd.com/read/29415943/-relationship-between-clinical-features-and-somatic-gene-mutations-in-myelodysplastic-syndrome
#14
Nobuhiro Hirasawa, Mamiko Sakata-Yanagimoto, Yasuhito Nannya, Keiichiro Hattori, Yasuhito Suehara, Takayasu Kato, Yasuhisa Yokoyama, Naoki Kurita, Naoshi Obara, Seishi Ogawa, Yuichi Hasegawa, Shigeru Chiba
Recent progress in sequencing studies has suggested that somatic mutations can be used in clinical sequencing for predicting prognosis and selecting treatment options in myelodysplastic syndrome (MDS). A 48-year-old man was diagnosed with refractory cytopenia with multilineage dysplasia that is classified as a subtype of high-risk MDS based on both revised International Prognostic Scoring System and refined WHO classification based Prognostic Scoring System. He received a bone marrow transplant from an HLA-matched sibling donor at X+87 months because of disease progression...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/29330964/spectrum-of-bone-marrow-pathology-and-hematological-abnormalities-in-methylmalonic-acidemia
#15
Nasir A Bakshi, Talal Al-Anzi, Said Y Mohamed, Zuhair Rahbeeni, Moeen AlSayed, Mohammed Al-Owain, Raashda A Sulaiman
Patients with isolated methylmalonic acidemia (MMA) may present with a wide range of hematological complications including anemia, leukopenia, thrombocytopenia, and pancytopenia. However, there are very limited data on the development of hemophagocytosis or myelodysplasia in these patients. We report three patients with isolated MUT related MMA who presented with severe refractory pancytopenia during acute illness. Their bone marrow examination revealed a wide spectrum of pathology varying from bone marrow hypoplasia, hemophagocytosis to myelodysplasia with ring sideroblasts...
March 2018: American Journal of Medical Genetics. Part A
https://www.readbyqxmd.com/read/29317997/experience-with-ibrutinib-for-first-line-use-in-patients-with-chronic-lymphocytic-leukemia
#16
REVIEW
Gilad Itchaki, Jennifer R Brown
Ibrutinib is the first in-class, orally administered, Bruton's tyrosine kinase (BTK) inhibitor that abrogates the critical signaling downstream of the B-cell receptor (BCR). This signaling is required for B-cell survival, proliferation and interaction with the microenvironment. Ibrutinib proved active in preclinical models of lymphoproliferative diseases and achieved impressive response rates in heavily pretreated relapsed and refractory (R/R) patients with chronic lymphocytic leukemia (CLL). Ibrutinib prolonged survival compared to standard therapy and mitigated the effect of most poor prognostic factors in CLL, thus becoming the main therapeutic option in high-risk populations...
January 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29296758/inotuzumab-ozogamicin-in-adults-with-relapsed-or-refractory-cd22-positive-acute-lymphoblastic-leukemia-a-phase-1-2-study
#17
Daniel J DeAngelo, Wendy Stock, Anthony S Stein, Andrei Shustov, Michaela Liedtke, Charles A Schiffer, Erik Vandendries, Katherine Liau, Revathi Ananthakrishnan, Joseph Boni, A Douglas Laird, Luke Fostvedt, Hagop M Kantarjian, Anjali S Advani
This study evaluated the safety, antitumor activity, pharmacokinetics, and pharmacodynamics of inotuzumab ozogamicin (InO) for CD22-positive relapsed/refractory acute lymphoblastic leukemia. In phase 1, patients received InO 1.2 (n = 3), 1.6 (n = 12), or 1.8 (n = 9) mg/m2 per cycle on days 1, 8, and 15 over a 28-day cycle (≤6 cycles). The recommended phase 2 dose (RP2D) was confirmed (expansion cohort; n = 13); safety and activity of InO were assessed in patients receiving the RP2D in phase 2 (n = 35) and in all treated patients (n = 72)...
June 27, 2017: Blood Advances
https://www.readbyqxmd.com/read/29285268/reactive-oxygen-species-levels-control-nf-%C3%AE%C2%BAb-activation-by-low-dose-deferasirox-in-erythroid-progenitors-of-low-risk-myelodysplastic-syndromes
#18
Mathieu Meunier, Sarah Ancelet, Christine Lefebvre, Josiane Arnaud, Catherine Garrel, Mylène Pezet, Yan Wang, Patrice Faure, Gautier Szymanski, Nicolas Duployez, Claude Preudhomme, Denis Biard, Benoit Polack, Jean-Yves Cahn, Jean Marc Moulis, Sophie Park
Anemia is a frequent cytopenia in myelodysplastic syndromes (MDS) and most patients require red blood cell transfusion resulting in iron overload (IO). Deferasirox (DFX) has become the standard treatment of IO in MDS and it displays positive effects on erythropoiesis. In low risk MDS samples, mechanisms improving erythropoiesis after DFX treatment remain unclear. Herein, we addressed this question by using liquid cultures with iron overload of erythroid precursors treated with low dose of DFX (3μM), which corresponds to DFX 5 mg/kg/day, an unusual dose used for iron chelation...
December 1, 2017: Oncotarget
https://www.readbyqxmd.com/read/29281873/safety-results-and-analysis-of-eribulin-efficacy-according-to-previous-microtubules-inhibitors-sensitivity-in-the-french-prospective-expanded-access-program-for-heavily-pre-treated-metastatic-breast-cancer
#19
Renaud Sabatier, Véronique Diéras, Xavier Pivot, Etienne Brain, Henri Roché, Jean-Marc Extra, Audrey Monneur, Magali Provansal, Carole Tarpin, François Bertucci, Patrice Viens, Christophe Zemmour, Anthony Gonçalves
Purpose: Eribulin is approved for advanced breast cancers refractory to anthracyclines and taxanes. Efficacy according to sensitivity to previous therapies has been poorly explored. Materials and Methods: Safety data were collected prospectively and we retrospectively collected efficacy data from the five French centres that participated in the Eribulin E7389-G000-398 expanded access program. Our main objectives were exploration of safety and analysis of eribulin efficacy (progression-free survival [PFS] and overall survival [OS]) according to sensitivity to the last microtubule-inhibiting agent administered...
December 28, 2017: Cancer Research and Treatment: Official Journal of Korean Cancer Association
https://www.readbyqxmd.com/read/29275390/hhv-8-associated-haemophagocytic-lymphohistiocytosis-in-a-patient-with-advanced-aids
#20
Saroja Bangaru, Amanda Strickland, Dominick Cavuoti, Nainesh Shah
We present a patient with advanced AIDS admitted with recurrent shock of unclear aetiology, fevers, altered mental status and refractory cytopenias. His case posed a diagnostic challenge because evaluation of septic shock in the setting of advanced AIDS requires a time-consuming work-up for broad infectious aetiologies that can delay consideration of other diagnoses, including primary or secondary haemophagocytic lymphohistiocytosis (HLH). After this patient did not improve with supportive care and empiric antimicrobials, there was concern for HLH given that he met ≥5 of the HLH consortium criteria...
December 22, 2017: BMJ Case Reports
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