keyword
MENU ▼
Read by QxMD icon Read
search

Novel therapies hematologic

keyword
https://www.readbyqxmd.com/read/28323922/fibrogenesis-imperfecta-ossium-and-response-to-human-growth-hormone-a-potential-novel-therapy
#1
Sanjay Kumar Bhadada, Vandana Dhiman, Soham Mukherjee, Sameer Aggarwal, Amanjit Bal, Suja P Sukumar, Ashwani Sood, Dinesh Chandra Sharma, Niranjan Khandelwal, Anil Bhansali, Wim Van Hul, Sudhaker D Rao
Context: Fibrogenesis imperfecta ossium (FIO) is a rare bone disease manifested by generalized bone pain, fragility fractures, progressive disability, and extensive mineralization defect on bone biopsy. The pathogenesis of the disease is unknown and currently there is no effective treatment. Objective: To report on the effect of recombinant human growth hormone (rhGH) therapy in FIO. Design: An observational study in two patients. Setting: Endocrinology Clinic in an Academic Institution Patients or Other Participants: Two siblings with FIO Intervention(s): rhGH was administered subcutaneously at a dose of 1U daily for one year...
February 28, 2017: Journal of Clinical Endocrinology and Metabolism
https://www.readbyqxmd.com/read/28316129/cognitive-compensatory-processes-of-older-clinically-fit-patients-with-hematologic-malignancies-undergoing-chemotherapy-a-longitudinal-cohort-study
#2
Yves Libert, Cindy Borghgraef, Yves Beguin, Nicole Delvaux, Martine Devos, Chantal Doyen, Stéphanie Dubruille, Anne-Marie Etienne, Aurore Liénard, Isabelle Merckaert, Christine Reynaert, Jean-Louis Slachmuylder, Nicole Straetmans, Eric Van Den Neste, Dominique Bron, Darius Razavi
OBJECTIVE: Despite the well-known negative impacts of cancer and anticancer therapies on cognitive performance, little is known about the cognitive compensatory processes of older patients with cancer. This study was designed to investigate the cognitive compensatory processes of older, clinically-fit patients with hematologic malignancies undergoing chemotherapy. METHODS: We assessed 89 consecutive patients (age ≥ 65 years) without severe cognitive impairment and 89 age-, sex-, and education level-matched healthy controls...
March 18, 2017: Psycho-oncology
https://www.readbyqxmd.com/read/28315358/isocitrate-dehydrogenase-idh-inhibition-as-treatment-of-myeloid-malignancies-progress-and-future-directions
#3
REVIEW
Vivek A Upadhyay, Andrew M Brunner, Amir T Fathi
Isocitrate dehydrogenase (IDH) is an essential metabolic enzyme. Over the last two decades, there has been a growing focus on the metabolic derangements that occur with IDH1 and IDH2 mutations. The altered IDH protein leads to accumulation of 2-hydroxyglutarate (2-HG), a metabolite with oncogenic activity via epigenetic mechanisms. The advent of IDH inhibitors has engendered hope in novel and targeted therapies in IDH1/2 mutant myeloid malignancies. We here summarize the basic physiology of IDH, the metabolic and oncogenic consequences of mutant IDH1/2, and the clinical significance of IDH inhibition in hematologic malignancies...
March 14, 2017: Pharmacology & Therapeutics
https://www.readbyqxmd.com/read/28315032/novel-indications-for-fecal-microbial-transplantation-update-and-review-of-the-literature
#4
REVIEW
Nathaniel Aviv Cohen, Nitsan Maharshak
BACKGROUND AND AIMS: Fecal microbial transplantation (FMT) is an established successful treatment modality for recurrent Clostridium difficile infection (CDI). The safety profile and potential therapeutic advantages of FMT for diseases associated with dysbiosis and immune dysfunction have led to many publications, mainly case series, and while many studies and reviews have been published on the use of FMT for inflammatory bowel disease (IBD), its potential use for other disease conditions has not been thoroughly reviewed...
March 17, 2017: Digestive Diseases and Sciences
https://www.readbyqxmd.com/read/28314790/selinexor-kpt-330-induces-tumor-suppression-through-nuclear-sequestration-of-ikappab-and-down-regulation-of-survivin
#5
Jayasree S Nair, Elgilda Musi, Gary K Schwartz
PURPOSE: Selinexor, a small molecule that inhibits nuclear export protein XPO1 has demonstrated efficacy in solid tumors and hematologic malignancies with the evidence of clinical activity in sarcoma as a single agent. Treatment options available are very few and hence the need to identify novel targets and strategic therapies is of utmost importance. EXPERIMENTAL DESIGN: The mechanistic effects of selinexor in sarcomas as a monotherapy and in combination with proteasome inhibitor, carfilzomib, across a panel of cell lines in vitro and few in xenograft mouse models were investigated...
March 17, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28297624/genomics-of-acute-myeloid-leukemia-diagnosis-and-pathways
#6
Lars Bullinger, Konstanze Döhner, Hartmut Döhner
In recent years, our understanding of the molecular pathogenesis of myeloid neoplasms, including acute myeloid leukemia (AML), has been greatly advanced by genomics discovery studies that use novel high-throughput sequencing techniques. AML, similar to most other cancers, is characterized by multiple somatically acquired mutations that affect genes of different functional categories, a complex clonal architecture, and disease evolution over time. Patterns of mutations seem to follow specific and temporally ordered trajectories...
March 20, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28290981/case-report-of-clinical-vignette-osteopetrosis
#7
John B Moore, Thanh D Hoang, Alfred F Shwayhat
INTRODUCTION: Osteopetrosis is a connective tissue disorder resulting from abnormally dense bone predisposing patients to fracture. The clinical pattern of fractures across time and space as well as suggestive radiographic findings usually raises diagnostic suspicion. Multiple genetic mutations resulting in dysfunctional osteoclasts have been implicated in the pathogenesis of osteopetrosis with variable inheritance patterns. In severe cases, usually inherited in an autosomal recessive pattern, the medullary cavity important in the production of normal blood cell progenitors is replaced by defective endochondral bone, leading to pancytopenia and consequential extramedullary hematopoiesis...
March 2017: Military Medicine
https://www.readbyqxmd.com/read/28273184/-how-can-we-treat-waldenstr%C3%A3-m-s-macroglobulinemia
#8
Anna Orsolya Mucsi, Zsolt Nagy
Waldenström's macroglobulinemia is a rare, low-grade non-Hodgkin lymphoma of B cell origin, most common in elderly male patients with a median age of 64 years at diagnosis. It accounts for approximately 2% of hematologic malignancies. The disease is incurable now with a median overall survival of 6.2 years. In the past decade growing evidence suggests the role of the complex signaling pathways and microenvironment as a potential target of the therapy in the lymphoproliferative disorders as well as Waldenström's macroglobulinemia...
March 8, 2017: Magyar Onkologia
https://www.readbyqxmd.com/read/28270368/clinical-outcomes-related-to-the-use-of-bendamustine-therapy-for-multiple-myeloma-patients-relapsed-refractory-to-imids-and-proteasome-inhibitors
#9
Fevzi Fırat Yalnız, Nihan Akkoç, Ayşe Salihoğlu, M Cem Ar, Seniz Aydın, A Emre Eşkazan, Teoman Soysal, Yıldız Aydın
Multiple myeloma (MM) patients who are relapsed or refractory to both proteasome inhibitory (PIs) and immunomodulatory drugs (IMiDs) have been reported to have poor outcome. Bendamustine has been reported to have an antitumor effect in newly diagnosed as well as relapsed refractory multiple myeloma (RRMM). The aim of this retrospective study was to evaluate the efficacy of bendamustine therapy in heavily pretreated MM patients, who were refractory to PIs and IMiDs. Nineteen RRMM patients treated either with bendamustine and steroid (n=13) or a combination of bendamustine in with novel drugs (n=6) were included...
March 8, 2017: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
https://www.readbyqxmd.com/read/28263217/peptide-receptor-radionuclide-therapy-with-177-lu-octreotate-in-patients-with-somatostatin-receptor-expressing-neuroendocrine-tumors-six-years-assessment
#10
Mohammadali Hamiditabar, Muzammil Ali, Joseph Roys, Edward M Wolin, Thomas M OʼDorisio, David Ranganathan, Izabela Tworowska, Jonathan R Strosberg, Ebrahim S Delpassand
OBJECTIVES: Peptide receptor radionuclide therapy (PRRT) with radiolabeled somatostatin analogues is a promising treatment for patients with inoperable, well to moderately differentiated metastatic neuroendocrine tumors (NETs). In continuation of our novel study with the radionuclide lutetium Lu, we now present further results of Lu DOTATATE therapy in managing NETs and other somatostatin receptor-expressing tumors in a larger and more diverse patient group. PATIENTS AND METHODS: One hundred forty-four consecutive patients (85 men and 59 women; age range, 11-87 years; mean age, 58...
March 3, 2017: Clinical Nuclear Medicine
https://www.readbyqxmd.com/read/28259887/hiv-associated-lung-cancer
#11
Til R Kiderlen, Jan Siehl, Marcus Hentrich
Lung cancer (LC) is one of the most common non-AIDS (acquired immune deficiency syndrome)-defining malignancies. It occurs more frequently in persons living with human immunodeficiency virus (PLWHIV) than in the HIV-negative population. Compared to their HIV-negative counterparts, patients are usually younger and diagnosed at more advanced stages. The pathogenesis of LC in PLWHIV is not fully understood, but immunosuppression in combination with chronic infection and the oncogenic effects of smoking and HIV itself all seem to play a role...
2017: Oncology Research and Treatment
https://www.readbyqxmd.com/read/28256053/making-inroads-to-the-cure-barriers-to-clinical-trial-enrollment-in-hematopoietic-cell-transplantation
#12
REVIEW
Michael Byrne, Nathalie Danielson, Amanda Harris, Bipin N Savani
A significant barrier to advancing the standard of care for patients with hematologic malignancies undergoing stem cell transplantation is access and willingness to participate in clinical trials. The importance of clinical trial enrollment is magnified in an era of targeted therapies, accelerated drug discovery, and investment by the pharmaceutical industry. As disease targets are identified, novel therapies are being evaluated in efforts to reduce treatment-related toxicity and improve progression-free and overall survival...
March 3, 2017: Clinical Transplantation
https://www.readbyqxmd.com/read/28254430/anti-tumor-activity-of-pi3k-%C3%AE-inhibitor-in-hematologic-malignant-cells-shedding-new-light-on-resistance-to-idelalisib
#13
Davood Bashash, Ava Safaroghli-Azar, Maryam Dadashi, Majid Safa, Majid Momeny, Seyed H Ghaffari
Genetic and laboratory experiments have brought remarkable advances in management of human malignancies, which not only revolutionized the understanding of the disease, but also led to development of novel and effective targeted therapies against specific deregulated pathways. This study aimed to investigate anti-cancer effects of Idelalisib, a potent PI3K-δ inhibitor, in a panel of hematological cell lines. The resulting data showed that Idelalisib decreased cell survival in all the tested cell lines; however, as compared to NB4, viability of other cell lines, irrespective of their molecular characteristics or even the compensatory activation of MEK/ERK pathway, was inhibited at higher concentrations...
February 22, 2017: International Journal of Biochemistry & Cell Biology
https://www.readbyqxmd.com/read/28247057/novel-hematological-parameters-for-the-evaluation-of-patients-with-myeloproliferative-neoplasms-the-immature-platelet-and-reticulocyte-fractions
#14
Paolo Strati, Prithviraj Bose, Lindsey Lyle, Katie Gaw, Lingsha Zhou, Sherry A Pierce, Julie Huynh-Lu, Cheryl F Hirsch-Ginsberg, Daniel E Bueso-Mendoza, Carlos E Bueso-Ramos, Srdan Verstovsek
New automated hematology analyzers have led to the availability of novel hematological parameters, including the immature platelet fraction (IPF) and the immature reticulocyte fraction (IRF), both of potential interest in patients with myeloproliferative neoplasms (MPNs). We performed a prospective analysis of 217 patients with MPN, including 32 (15%) with essential thrombocythemia (ET), 43 (20%) with polycythemia vera (PV), and 142 (65%) with myelofibrosis (MF); the IPF and IRF were measured by the Sysmex XN analyzer...
February 28, 2017: Annals of Hematology
https://www.readbyqxmd.com/read/28246194/optimized-depletion-of-chimeric-antigen-receptor-t-cells-in-murine-xenograft-models-of-human-acute-myeloid-leukemia
#15
Sarah K Tasian, Saad S Kenderian, Feng Shen, Marco Ruella, Olga Shestova, Miroslaw Kozlowski, Yong Li, April Schrank-Hacker, Jennifer J D Morrissette, Martin Carroll, Carl H June, Stephan A Grupp, Saar Gill
Others and we previously reported potent anti-leukemia efficacy of CD123-redirected chimeric antigen receptor (CAR) T-cells in preclinical human acute myeloid leukemia (AML) models at cost of severe hematologic toxicity. This observation raises concern for potential myeloablation in patients with AML treated with CD123-redirected CAR T-cells and mandates novel approaches for toxicity mitigation. We hypothesized that CAR T-cell depletion with optimal timing after AML eradication would preserve leukemia remission and allow subsequent hematopoietic stem cell transplantation...
February 28, 2017: Blood
https://www.readbyqxmd.com/read/28243938/hemorrhagic-fever-of-bunyavirus-etiology-disease-models-and-progress-towards-new-therapies
#16
REVIEW
Brian B Gowen, Brady T Hickerson
A growing number of bunyaviruses are known to cause viral hemorrhagic fever (VHF), a severe febrile illness which can progress to hypovolemic shock and multi-organ failure and is characterized by hematologic abnormalities and vascular leak. At present, there are no approved vaccines or antiviral therapies to effectively prevent or treat VHF caused by pathogenic bunyaviruses. Advances in the modeling of bunyaviral infections have facilitated efforts towards the development of novel post-exposure prophylactic and therapeutic countermeasures, several of which may some day be approved for human use...
March 2017: Journal of Microbiology / the Microbiological Society of Korea
https://www.readbyqxmd.com/read/28222220/meaningful-endpoints-for-therapies-approved-for-hematologic-malignancies
#17
B Douglas Smith, Amy E DeZern, Alex W Bastian, Brian G M Durie
BACKGROUND: Overall survival (OS) is considered the gold standard for determining treatment efficacy in oncology trials, but the relation between treatment and OS can be challenging to assess because of long study durations and the impact of subsequent therapies on outcome. Using OS can be particularly difficult for new therapies in hematologic malignancies (HMs). METHODS: This retrospective analysis was conducted to characterize the primary endpoints used to support US Food and Drug Administration (FDA) approvals for new drug or novel HM indications between January 2002 and July 2015...
February 21, 2017: Cancer
https://www.readbyqxmd.com/read/28210842/uric-acid-as-a-novel-biomarker-for-bone-marrow-function-and-incipient-hematopoietic-reconstitution-after-aplasia-in-patients-with-hematologic-malignancies
#18
Sebastian P Haen, Vicky Eyb, Nora Mirza, Aline Naumann, Andreas Peter, Markus W Löffler, Christoph Faul, Wichard Vogel, Wolfgang A Bethge, Hans-Georg Rammensee, Lothar Kanz, Martin Heni
PURPOSE: Prolonged aplasia and graft failure (GF) represent life-threatening complications after hematopoietic cell transplantation (HCT) requiring suitable biomarkers for early detection and differentiation between GF and poor graft function (PGF). Uric acid (UA) is a strong immunological danger signal. METHODS: Laboratory results were analyzed from patients undergoing either allogeneic or autologous HCT or induction chemotherapy for acute leukemia (n = 50 per group, n = 150 total)...
February 16, 2017: Journal of Cancer Research and Clinical Oncology
https://www.readbyqxmd.com/read/28205194/novel-and-expanded-oncology-drug-approvals-of-2016-part-2-new-options-in-the-management-of-hematologic-malignancies
#19
REVIEW
Todd C Knepper, James Saller, Christine M Walko
The recent past has brought pharmacotherapeutic advances that benefit patients with hematologic malignancies. In 2016, two novel hematology drugs were approved and four previously approved hematology drugs were granted expanded use for the treatment of appropriate patient populations by the US Food and Drug Administration. These new approvals and indications represent significant steps forward in patient management: they include the first-in-class B-cell lymphoma 2 inhibitor, venetoclax, the newest targeted therapy available for the treatment of hematologic malignancies; and nivolumab, the first immune checkpoint inhibitor to be approved for treatment of a hematologic malignancy...
February 15, 2017: Oncology (Williston Park, NY)
https://www.readbyqxmd.com/read/28203342/optimizing-current-and-emerging-therapies-in-multiple-myeloma-a-guide-for-the-hematologist
#20
REVIEW
Shahzad Raza, Rachael A Safyan, Evan Rosenbaum, Alex S Bowman, Suzanne Lentzsch
Multiple myeloma (MM) is the second most common hematologic malignancy. The diagnosis of MM requires ⩾10% clonal plasma cells in the bone marrow or biopsy-proven plasmacytoma, plus evidence of end-organ damage (hypercalcemia, renal failure, anemia, and lytic bone lesions). The definition of MM has recently been expanded to include a ⩾60% clonal plasma cell burden in the bone marrow, serum involved/uninvolved light chain ratio of ⩾100, or more than one focal lesion on magnetic resonance imaging ⩾5 mm in the absence of end-organ damage...
February 2017: Therapeutic Advances in Hematology
keyword
keyword
95259
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"