Current Therapy in Hematology/Oncology

Pathology ◽  
1986 ◽  
Vol 18 (4) ◽  
pp. 487
Author(s):  
Roger D. Scurr
Keyword(s):  
1985 ◽  
Vol 3 (1) ◽  
pp. 147-164 ◽  
Author(s):  
Michael E. Cohen ◽  
Patricia K. Duffner

1997 ◽  
Vol 77 (05) ◽  
pp. 0944-0948 ◽  
Author(s):  
Darla Liles ◽  
Charles N Landen ◽  
Dougald M Monroe ◽  
Celeste M Lindley ◽  
Marjorie s Read ◽  
...  

SummaryCurrent therapy for hemophilia B requires large intravenous doses of factor IX (F.IX) given in the clinic or at home. Although home therapy is possible for many patients, it is often complicated by factors such as the lack of good venous access. Very little is known about extravascular routes for administering proteins like F.IX (57 kD) or other vitamin K-dependent procoagulant factors into the circulation. Questions about the absorption rate from extravascular administration as well as plasma recovery and bioavailability have arisen recently with the growing availibility of highly purified procoagulant proteins and increased interest in gene therapy of hemophilia B. Therefore, a group of studies were undertaken to determine the absorption rate, plasma recovery, and bioavailability of high purity, human plasma-derived F.IX concentrates administered via extravascular routes in hemophilia B dogs and in one human hemophilia B subject. Five hemophilia B dogs were given human F.IX via either a subcutaneous (SC), intramuscular (IM), intra- peritoneal (IP) or intravenous (IV) route. In a subsequent study, a single SC administration of human F.IX was compared to an identical IV dose of F.IX in the human hemophilia B subject. All extravascular routes of F.IX administration in both the canine and human gave lower levels of circulating plasma F.IX than the IV route, however all routes resulted in measurable F.IX activity. Of the extravascular routes, the IM injection in the canine resulted in a bioavailibility of 82.8%, while the SC injection resulted in a bioavailability of 63.5%. F.IX reached the plasma compartment by all extravascular routes used, confirming that F.IX can be absorbed extravascularly. The duration of measurable F.IX activity following extravascular administration is prolonged beyond that typically seen with IV administration. These data show that significant levels of F.IX may be obtained via SC injection in canine and ‘ human hemophilia B subjects and further highlight the potential of extravascular routes of administration for future experimental and clinical uses of F.IX and other procoagulant proteins.


2019 ◽  
Vol 45 (1) ◽  
pp. 13
Author(s):  
Gladys Kusumowidagdo ◽  
Randy Sarayar ◽  
Kartika Rahayu ◽  
Gitalisa Andayani

Background: Diabetic macular edema (DME) is the main cause of visual impairment in diabetic retinopathy (DR). Current gold standard therapy of DME is macular laser photocoagulation (MPC). Growing evidences have shown benefits of intravitreal anti-VEGF agents (i.e bevacizumab) and intravitreal corticosteroids (i.e triamcinolone acetonide). Aim: To compare the visual acuity (VA) improvement of patients with DME, treated with intravitreal bevacizumab (IVB), a combination of IVB and intravitreal triamcinolone (IVB/IVT), and MPC. Method: A comprehensive PubMed® and Cochrane® databases search was conducted on May 4th, 2017 using appropriate keywords (diabetic macular edema, bevacizumab, triamcinolone, and laser photocoagulation using their MeSH terms). Studies were filtered using inclusion criterions (clinical trials, RCT, meta-analysis, systematic review, English, humans, and publication within 10 years) Results: Three studies (2 systematic reviews and 1 RCT) were found suitable. From these results, all studies showed favoring effects of IVB when compared to IVB/IVT combination and MPC in short term period (up to 6 months). However, there was no significant improvement of VA beyond this period in all groups. Conclusion: IVB appears to be superior to IVB/IVT and MPC in improving VA during 6 months follow- up period. Future systematic reviews and meta-analysis are required on the effect of IVB and MPC combination in cases of DME.


2020 ◽  
Vol 26 ◽  
Author(s):  
Ritu Mishra ◽  
Swati Gupta

Background: Rheumatoid arthritis (RA) is the most common occurring progressive, autoimmune disease, affecting 1% of the population and the ratio of affected women is three times as compared to men in most developing countries. Clinical manifestations of RA are the presence of anti-citrullinated protein antibody (ACPA) and rheumatoid factor (RF) in blood, tendered joints and soreness of the muscles. Some other factors which may lead to chronic inflammation are genetic and environmental factors as well as adaptive immune response. Several conventional drugs are available for the treatment of RA but have their own drawbacks which can be overcome by the use of novel drug delivery systems. : The objective of the present review is to focus on the molecular pathogenesis of the disease and its current conventional treatment with special reference to the role of novel drug delivery systems encapsulating anti rheumatic drugs and herbal drugs in passive and receptor mediated active targeting against RA. On reviewing the conventional and current therapeutics agains RA, we conclude that, although the current therapy for the treatment of RA is capable enough, yet more advances in the field of targeted drug delivery will sanguinely result in effective and appropriate treatment of this autoimmune disease.


2019 ◽  
Vol 16 (3) ◽  
pp. 251-258 ◽  
Author(s):  
Javad Behravan ◽  
Atefeh Razazan ◽  
Ghazal Behravan

Breast cancer is the second leading cause of cancer death among women. National cancer institute of the US estimates that one in eight women will be diagnosed with breast cancer during their lifetime. Considering the devastating effects of the disease and the alarming numbers many scientists and research groups have devoted their research to fight breast cancer. Several recommendations are to be considered as preventing measures which include living a healthy lifestyle, regular physical activity, weight control and smoking cessation. Early detection of the disease by annual and regular mammography after the age of 40 is recommended by many healthcare institutions. This would help the diagnosis of the disease at an earlier stage and the start of the treatment before it is spread to other parts of the body. Current therapy for breast cancer includes surgical ablation, radiotherapy and chemotherapy which is often associated with adverse effects and even may lead to a relapse of the disease at a later stage. In order to achieve a long-lasting anticancer response with minimal adverse effects, development of breast cancer vaccines is under investigation by many laboratories. The immune system can be stimulated by a vaccine against breast cancer. This approach has attracted a great enthusiasm in recent years. No breast cancer vaccines have been approved for clinical use today. One breast cancer vaccine (NeuVax) has now completed clinical trial phase III and a few preventive and therapeutic breast cancer vaccines are at different steps of development. We think that with the recent advancements in immunotherapy, a breast cancer vaccine is not far from reach.


2021 ◽  
Vol 18 (4) ◽  
pp. 398-418
Author(s):  
Vinícius Guimarães da Paixão ◽  
Samuel Silva da Rocha Pita

Background: Leishmania infantum causes the most lethal form of Leishmaniasis: Visceral leishmaniasis. Current therapy for this disease is related to the development of drug-resistant species and toxicity. Trypanothione Reductase (LiTR), a validated target for the drug discovery process, is involved with parasites' thiol-redox metabolism. Objective: In this study, through Virtual Screening employing two distinct Natural Products Brazilian databases, we aimed to identify novel inhibitor scaffolds against LiTR. Results: Thus, the “top 10” LiTR-ligand energies have been selected and their interaction profiles into LiTR sites through the AuPosSOM server have been verified. Finally, Pred-hERG, Aggregator Advisor, FAF-DRUGS, pkCSM and DataWarrior were employed and their results allowed us to evaluate, respectively, the cardiotoxicity, aggregation capacity, presence of false-positive compounds (PAINS) and their toxicities. Conclusion: Three molecules that overcame the in silico pharmacokinetic analysis and have a good interaction with LiTR, were chosen to use in vitro assays hoping that our computational results reported here would aid the development of new anti-leishmanial compounds.


Cardiology ◽  
2000 ◽  
Vol 93 (1-2) ◽  
pp. 56-69 ◽  
Author(s):  
Carl V. Leier ◽  
Rene J. Alvarez ◽  
Philip F. Binkley

Author(s):  
Michał Nowicki ◽  
Stanisława Bazan-Socha ◽  
Mariusz Kłopotowski ◽  
Beata Błażejewska-Hyżorek ◽  
Mariusz Kusztal ◽  
...  

Current therapy for Anderson–Fabry disease in Poland includes hospital or clinic-based intravenous enzyme replacement therapy with recombinant agalsidase alpha or beta, or oral pharmacological chaperone therapy with migalastat. Some countries around the world offer such treatment to patients in the comfort of their own homes. The 2020–2021 COVID-19 pandemic has pushed global healthcare providers to evolve their services so as to minimize the risk of COVID-19 exposure to both patients and providers; this has led to advances in telemedicine services and the increasing availability of at-home treatment for various procedures including parenteral drug administration. A total of 80% of surveyed Anderson–Fabry disease patients in Poland would prefer home-based treatment, which would be a safe and convenient alternative to clinic-based treatment if patient selection is based on our proposed algorithm. Our recommendations for home-based treatments appear feasible for the long term care of Anderson–Fabry disease patients during the COVID-19 pandemic and beyond. This may also serve as a basis for home-based treatment programs in other rare and ultra-rare genetic diseases.


2020 ◽  
Vol 22 (Supplement_3) ◽  
pp. iii406-iii406
Author(s):  
Kübra Taban ◽  
David Pauck ◽  
Mara Maue ◽  
Viktoria Marquardt ◽  
Hua Yu ◽  
...  

Abstract Medulloblastoma (MB) is the most common malignant brain tumor in children and is frequently metastatic at diagnosis. Treatment with surgery, radiation and multi-agent chemotherapy may leave survivors of these brain tumors with long-term deficits as a consequence. One of the four consensus molecular subgroups of MB is the MYC-driven group 3 MB, which is the most malignant type and has a poor prognosis under current therapy. Thus, it is important to discover more effective targeted therapeutic approaches. We conducted a high-throughput drug screening to identify novel compounds showing efficiency in group 3 MB using both clinically established inhibitors (n=196) and clinically-applicable compounds (n=464). More than 20 compounds demonstrated a significantly higher anti-tumoral effect in MYChigh (n=7) compared to MYClow (n=4) MB cell models. Among these compounds, Navitoclax and Clofarabine showed the strongest effect in inducing cell cycle arrest and apoptosis in MYChigh MB models. Furthermore, we show that Navitoclax, an orally bioavailable and blood-brain barrier passing anti-cancer drug, inhibits specifically Bcl-xL proteins. In line, we found a significant correlation between BCL-xL and MYC mRNA levels in 763 primary MB patient samples (Data source: “R2 https://hgserver1.amc.nl”). In addition, Navitoclax and Clofarabine have been tested in cells obtained from MB patient-derived-xenografts, which confirmed their specific efficacy in MYChigh versus MYClow MB. In summary, our approach has identified promising new drugs that significantly reduce cell viability in MYChigh compared to MYClow MB cell models. Our findings point to novel therapeutic vulnerabilities for MB that need to be further validated in vitro and in vivo.


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1774.2-1774
Author(s):  
N. Jain ◽  
N. Reddy ◽  
A. Moorthy

Background:Cannabinoids has recently gained popularity for use in chronic pain. There is a lot of inquisitiveness among our patients wherein health care professionals are asked about its efficacy, side effects and sometimes even ask for a prescription! As there is paucity of data and research about its use in rheumatology, patient reported outcome(PROM) can guide ahead in expanding our knowledge and experience.Objectives:To study usage of cannabinoids by rheumatology patientsTo study awareness among primary physicians regarding Cannabinoid usage in rheumatology.Methods:Cross sectional survey with two arms. Arm 1 Information from patients attending tertiary rheumatology clinic,including perception regarding the use of Cannabinoids.Arm 2 consisted of collecting data via web-based survey with20-question from 100 GPs of Leicestershire. Questions on demographics, perspectives on and knowledge of cannabinoid use. Statistical analysis SPSS software.Results:Arm1 Total 102 rheumatology patients with 60%were females and 45% secondary education. 48% were unemployed. 75% Caucasians, 18% Asians. RA most common diagnosis followed by OA and FMS. 40 % depression and anxiety in addition to Rheumatic disease. 94% reported ongoing pain with 6-8 on a VAS scale. 79% were satisfied with their current therapy. 65% had heard about complementary medicine and 15% reported using cannabinoids.Most common form Cannabinoids oil 60% followed by smoking 20%. 56% reported using >3 months and majority 72% use daily. Median age 55 years. 88% users Caucasians. Mean disease duration 6.25 years among users indicates chronicity of disease has a direct proportion in usage. All users had ongoing pain of 7 on VAS. 87% believed it helps them managing pain effectively with a pain free state. On an average spends between 50-100 pounds per week. More than half believe cannabinoids should be available as a prescription drug in NHS and 30% interested to know more about it.In Arm 2 consisting of Primary care physicians, response rate 50%. Average clinical experience 5 years. Only 20% heard about usage of complementary medicine by rheumatology patient. Most replied that 10% of their patients use Cannabinoids for pain management. Most did not believe use of cannabinoids benefited the patients. Only 4% recommend its usage. 25% think it should be available as prescription. 40% experienced patients asking about cannabinoids during appointment. 88% of respondents did not know much about cannabinoid usage in rheumatology and have never prescribed it in their practice.Conclusion:Cannabinoids widely used by the rheumatology patients with PROM favouring its efficacy for control of chronic pain. Preclinical data suggest that cannabinoids might have a therapeutic potential RA1, OA, FMS2. Clinical data regarding cannabinoid treatment for rheumatic diseases are scarce, therefore, recommendations concerning cannabinoid treatment cannot be made. All patients who reported using it suffered from moderate to severe chronic pain. Thus main indication of usage was pain rather than recreational purpose. Although a small survey it clearly highlights lack of knowledge among primary physicians. These results emphasise the need for further research regarding the benefits and risks of cannabinoids in rheumatology.References:[1]RichardsonD. etal Characterisation ofthe cannabinoid receptor system in synovial tissue andfluid in patients with OA and RA Arthritis Res.Ther. 10, R43 (2008).[2]Walitt, B etal Cannabinoids for fibromyalgia. Cochrane DatabaseSyst. Rev. 7, CD011694 (2016).Disclosure of Interests:Nibha Jain: None declared, Neelima Reddy: None declared, Arumugam Moorthy Speakers bureau: Abbvie, Novartis,UCB,MSD


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