journal
MENU ▼
Read by QxMD icon Read
search

Therapeutic Advances in Hematology

journal
https://www.readbyqxmd.com/read/30210757/practical-aspects-of-extended-half-life-products-for-the-treatment-of-haemophilia
#1
REVIEW
Thierry Lambert, Gary Benson, Gerry Dolan, Cedric Hermans, Victor Jiménez-Yuste, Rolf Ljung, Massimo Morfini, Silva Zupančić-Šalek, Elena Santagostino
Haemophilia A and haemophilia B are congenital X-linked bleeding disorders caused by deficiency of coagulation factor VIII (FVIII) and IX (FIX), respectively. The preferred treatment option for patients with haemophilia is replacement therapy. For patients with severe disease, prophylactic replacement of coagulation factor is the treatment of choice; this has been shown to reduce arthropathy significantly, reduce the frequency of bleeds and improve patients' quality of life. Prophylaxis with standard recombinant factor requires regular intravenous infusion at least two (FIX) to three (FVIII) times a week...
September 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30210756/gene-therapy-for-hemophilia-what-does-the-future-hold
#2
REVIEW
Bhavya S Doshi, Valder R Arruda
Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have reported remarkable success in ameliorating disease phenotype in hemophilia A and B. These trials, which highlight the challenges overcome through decades of preclinical and first in human clinical studies, have generated considerable excitement for patients and caregivers alike. Optimization of vector and transgene expression has significantly improved the ability to achieve therapeutic factor levels in these subjects...
September 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30210755/update-on-the-role-of-brentuximab-vedotin-in-classical-hodgkin-lymphoma
#3
REVIEW
Sarah Tomassetti, Alex F Herrera
Brentuximab vedotin (BV) is an effective and well-tolerated treatment for patients with classical Hodgkin lymphoma (HL). It was initially approved by the US FDA for the treatment of HL after failure of autologous hematopoietic stem cell transplant (autoHSCT) or after failure of at least two prior lines of multiagent chemotherapy in patients who are not transplant candidates, and then subsequently, as consolidation therapy after autoHSCT in patients who are at high risk for relapse. However, the role of BV in the treatment of HL is evolving...
September 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30210754/leveraging-jak-stat-regulation-in-myelofibrosis-to-improve-outcomes-with-allogeneic-hematopoietic-stem-cell-transplant
#4
REVIEW
Michael Byrne, Bipin Savani, Michael R Savona
Primary myelofibrosis (PMF) is a disease characterized by bone marrow fibrosis, extramedullary hematopoiesis, risk of transformation to acute myeloid leukemia, and a substantial symptom burden with diminished quality of life. Allogeneic hematopoietic cell transplantation (HCT) is the only curative option; however, disease relapse and graft versus host disease (GVHD) are significant barriers to long-term survival. The discovery of the JAK2 V617F mutation, and subsequent development of JAK inhibitors, resulted in improved survival and significant improvements in spleen volumes and symptom scores...
September 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30181844/the-role-of-patient-and-healthcare-professionals-in-the-era-of-new-hemophilia-treatments-in-developed-and-developing-countries
#5
REVIEW
Fadi Nossair, Courtney D Thornburg
Medical decisions in hemophilia care are primarily related to the type of factor replacement and treatment regimen. With the growing number of treatment options for patients with hemophilia, decision making is more complex and requires careful consideration of benefits, risks, and patient goals. Shared decision making and decision-aid tools facilitate patient and healthcare provider communication. In this review, the overall role of shared decision making in medicine is outlined, with special emphasis on models for practical implementation...
August 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30181843/recent-advances-in-musculoskeletal-physiotherapy-for-haemophilia
#6
REVIEW
David Stephensen, Melanie Bladen, Paul McLaughlin
Physiotherapy is directed towards the movement needs and potential of individuals, providing treatment and rehabilitation to develop, maintain and restore maximum movement and functional ability throughout the lifespan. Recent systematic reviews and randomized controlled trials have extended evidence for the clinical efficacy of physiotherapy interventions and rehabilitation for people with haemophilia. This narrative review synthesizes recent evidence to discuss; differentiating musculoskeletal bleeding and haemophilic arthropathy, efficacy of physiotherapy and rehabilitation for acute musculoskeletal bleeding and arthropathy, as well as monitoring musculoskeletal health...
August 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30181842/clinicopathological-parameters-influencing-inhibitor-development-in-patients-with-hemophilia-a-receiving-on-demand-therapy
#7
Sanya Arshad, Anshima Singh, Namrata Punit Awasthi, Swati Kumari, Nuzhat Husain
Background: Development of inhibitors to transfused factor VIII in patients with hemophilia A continues to be a challenge for professionals involved in hemophilia care. The majority of patients in India receive 'on-demand' rather than prophylactic therapy. The present study was done to assess the prevalence of factor VIII inhibitors in patients with hemophilia A (PWHA) receiving 'on-demand' therapy in a North Indian population and to study the clinicopathological parameters influencing the development of inhibitors...
August 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30013767/a-case-series-of-kimura-s-disease-a-diagnostic-challenge
#8
Vivek Kumar, Navneet Mittal, Yiwu Huang, Jasminka Balderracchi, Huo Xiang Zheng, Zujin Li, Yiqing Xu
Kimura's disease (KD) is a rare, benign disorder characterized by subcutaneous masses with regional lymph-node enlargement. It is considered to be due to chronic inflammation of unclear etiology. Most cases have been reported in young, 20-30-year-old men of Asian descent. The diagnosis of KD is based on pathological features and elevated immunoglobulin E levels. Characteristic pathological features include intact lymph-node architecture, florid germinal center hyperplasia, extensive eosinophilic infiltrates, and proliferation of postcapillary venules...
July 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30013766/bay-81-8973-a-full-length-recombinant-factor-viii-for-the-treatment-of-hemophilia-a-product-review
#9
REVIEW
Johnny N Mahlangu, Sanjay P Ahuja, Jerzy Windyga, Nikki Church, Anita Shah, Lawrence Schwartz
BAY 81-8973 (Kovaltry® ) is an unmodified, full-length recombinant factor VIII (rFVIII) approved for the prevention and treatment of bleeding episodes in patients with hemophilia A. The amino acid sequence for BAY 81-8973 is identical to that of sucrose-formulated rFVIII (rFVIII-FS; Kogenate® FS/KOGENATE® , Bayer), but the two products differ in their manufacturing approaches. The manufacture of BAY 81-8973 includes several modifications and enhancements, such as the introduction of the gene for human heat shock protein 70, a molecular chaperone protein that facilitates folding of proteins; no addition of human- or animal-derived proteins in the cell culture, purification process, or final formulation; and use of a 20-nm filter to remove any potential aggregates and pathogens...
July 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30013765/update-on-the-role-of-lenalidomide-in-patients-with-multiple-myeloma
#10
REVIEW
Sarah A Holstein, Vera J Suman, Philip L McCarthy
Lenalidomide is a derivative of thalidomide and belongs to the class of drugs known as the immunomodulatory drugs (IMiDs). The IMiDs have played a large role in improving the survival outcomes of patients with multiple myeloma. In particular, lenalidomide is currently standard of care in the newly diagnosed setting, in the maintenance setting post-autologous stem cell transplant, as well as in the relapsed/refractory setting. While the combination of lenalidomide and various proteasome inhibitors has proven particularly effective, there are emerging data demonstrating the effectiveness of lenalidomide in combination with other important classes of drugs including the monoclonal antibodies...
July 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30013764/the-role-of-enasidenib-in-the-treatment-of-mutant-idh2-acute-myeloid-leukemia
#11
REVIEW
Iman Abou Dalle, Courtney D DiNardo
Recurrent mutations affecting cellular metabolism and epigenetic regulation are implicated in the pathogenesis of acute myeloid leukemia (AML). Isocitrate dehydrogenase 2 ( IDH2 ) gene mutations are described in 12% of patients with AML and 5% of patients with myelodysplastic syndromes. IDH2 enzyme is involved in the Krebs cycle, catalyzing α-ketoglutarate from isocitrate. Mutant IDH2 enzymes acquire a neomorphic enzymatic activity with the ability to produce 2-hydroxyglutarate from α-ketoglutarate, inhibiting multiple α-ketoglutarate-dependent dioxygenase reactions; leading to aberrant DNA hypermethylation and differentiation block in myeloid precursors and ultimately promoting leukemogenesis...
July 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29899890/pharmacokinetic-based-prediction-of-real-life-dosing-of-extended-half-life-clotting-factor-concentrates-on-hemophilia
#12
REVIEW
Massimo Morfini, Stefano Gherardini
The improvement of clotting factor concentrates (CFCs) has undergone an impressive boost during the last six years. Since 2010, several new recombinant factor (rF)VIII/IX concentrates entered phase I/II/III clinical trials. The improvements are related to the culture of human embryonic kidney (HEK) cells, post-translational glycosylation, PEGylation, and co-expression of the fragment crystallizable (Fc) region of immunoglobulin (Ig)G1 or albumin genes in the manufacturing procedures. The extended half-life (EHL) CFCs allow an increase of the interval between bolus administrations during prophylaxis, a very important advantage for patients with difficulties in venous access...
June 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29899889/acute-myeloid-leukemia-chimeric-antigen-receptor-t-cell-immunotherapy-how-far-up-the-road-have-we-traveled
#13
REVIEW
Sarah K Tasian
Chemotherapy resistance and relapse remain significant sources of mortality for children and adults with acute myeloid leukemia (AML). Further intensification of conventional cytotoxic chemotherapy is likely not feasible due to the severity of acute and long-term side effects upon normal tissues commonly induced by these drugs. Successful development and implementation of new precision medicine treatment approaches for patients with AML, which may improve leukemia remission and diminish toxicity, is thus a major priority...
June 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29713445/the-evolution-of-stem-cell-transplantation-in-multiple-myeloma
#14
REVIEW
Sarakshi Mahajan, Nidhi Tandon, Shaji Kumar
Autologous stem-cell transplantation (ASCT) remains an integral part of treatment for previously untreated, and may have value in the treatment of relapsed patients with, multiple myeloma (MM). The addition of novel agents like immunomodulators and proteasome inhibitors as induction therapy before and as consolidation/maintenance therapy after ASCT has led to an improvement in complete response (CR) rates, progression-free survival (PFS) and overall survival (OS). With advances in supportive care, older patients and patients with renal insufficiency are now able to safely undergo the procedure...
May 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29713444/personalizing-initial-therapy-in-acute-myeloid-leukemia-incorporating-novel-agents-into-clinical-practice
#15
REVIEW
Christin B DeStefano, Christopher S Hourigan
While the past decade has seen a revolution in understanding of the genetic and molecular etiology of the disease, in clinical practice, initial therapy for acute myeloid leukemia (AML) patients has been a relatively straightforward choice between intensive combination cytotoxic induction therapy as used for decades or less-intensive hypomethylating therapy. The year 2017, however, witnessed US Food and Drug Administration approvals of midostaurin, enasidenib, gemtuzumab ozogamicin and CPX-351 for AML patients, with many other promising agents currently in clinical trials...
May 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29623180/pembrolizumab-and-its-role-in-relapsed-refractory-classical-hodgkin-s-lymphoma-evidence-to-date-and-clinical-utility
#16
REVIEW
Polina Shindiapina, Lapo Alinari
Immune evasion is a critical mechanism of malignant cell survival, and relies in part on molecular signaling through the programmed cell death 1 (PD-1)/PD-1 ligand (PD-L1) axis that contributes to T cell exhaustion. Immune modulatory therapy with monoclonal antibodies against PD-1 designed to enhance antitumor immune response have shown promise in the treatment of advanced solid tumors and hematologic malignancies. Classical Hodgkin's lymphoma (cHL), a unique B cell malignancy characterized by an extensive but ineffective immune cell infiltrate surrounding a small number of tumor cells, has shown significant response to anti-PD-1 directed therapy...
April 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29623179/co-expression-of-myc-and-bcl2-predicts-poorer-outcomes-for-relapsed-refractory-diffuse-large-b-cell-lymphoma-with-r-ice-and-intent-to-transplant
#17
Joshua Allen, Ana Lucia Ruano Mendez, Lisa Rybicki, Yazeed Sawalha, Deepa Jagadeesh, Robert Dean, Brad Pohlman, Mitchell R Smith, Eric D Hsi, Brian T Hill
Diffuse large B-cell lymphoma (DLBCL) with co-expression of MYC and BCL2 protein by immunohistochemistry (IHC) - that is, double-expresser lymphoma (DEL) - is associated with poor outcomes after standard frontline therapy. Less is known about the prognostic impact of DEL in patients with relapsed/refractory disease treated with salvage therapy and autologous stem cell transplantation (ASCT). We analyzed the outcomes of 167 patients with relapsed/refractory DLBCL treated with R-ICE (rituximab, ifosfamide, carboplatin and etoposide), of whom 111 patients (66%) underwent ASCT...
April 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29531660/safety-and-efficacy-of-nilotinib-in-routine-clinical-practice-in-patients-with-chronic-myeloid-leukemia-in-chronic-or-accelerated-phase-with-resistance-or-intolerance-to-imatinib-results-from-the-novel-study
#18
Ching-Yuan Kuo, Po-Nan Wang, Wen-Li Hwang, Cheng-Hwai Tzeng, Li-Yaun Bai, Jih-Luh Tang, Ming-Chih Chang, Sheng-Fung Lin, Tsai-Yun Chen, Yeu-Chin Chen, Tran-Der Tan, Chih-Yi Hsieh, Chinjune Lin, Clinton Lai, Darko Miljkovic, Cheng-Shyong Chang
Background: Nilotinib, a second-generation tyrosine kinase inhibitor (TKI), is approved for the treatment of patients with chronic myeloid leukemia (CML) in many countries, including Taiwan. Though a number of controlled clinical trials have demonstrated the safety and efficacy of nilotinib, studies assessing the safety and efficacy of nilotinib in routine clinical practice are limited. Methods: The current study was an open-label, single-arm study conducted across 12 centers in Taiwan in adult patients with CML in chronic or accelerated phase with confirmed Ph+ chromosome (or BCR-ABL) and resistant or intolerant to one or more previous TKIs...
March 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29387330/novel-therapies-and-current-clinical-progress-in-hemophilia-a
#19
REVIEW
Pauline Balkaransingh, Guy Young
The evolution of hemophilia treatment and care is a fascinating one but has been fraught with many challenges at every turn. Over the last 50 years or so patients with hemophilia and providers have witnessed great advances in the treatment of this disease. With these advances, there has been a dramatic decrease in the mortality and morbidity associated with hemophilia. Even with the remarkable advancements in treatment, however, new and old challenges continue to plague the hemophilia community. The cost of factor replacement and the frequency of infusions, especially in patients with severe hemophilia on prophylaxis, remains a significant challenge for this population...
February 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29317998/new-and-emerging-therapies-for-acute-and-chronic-graft-versus-host-disease
#20
REVIEW
LaQuisa Hill, Amin Alousi, Partow Kebriaei, Rohtesh Mehta, Katayoun Rezvani, Elizabeth Shpall
Graft versus host disease (GVHD) remains a major cause of morbidity and mortality following allogeneic hematopoietic stem-cell transplantation (HSCT). Despite the use of prophylactic GVHD regimens, a significant proportion of transplant recipients will develop acute or chronic GVHD following HSCT. Corticosteroids are standard first-line therapy, but are only effective in roughly half of all cases with ~50% of patients going on to develop steroid-refractory disease, which increases the risk of nonrelapse mortality...
January 2018: Therapeutic Advances in Hematology
journal
journal
43357
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"