Pharmacotherapy review: Emerging treatment modalities in transthyretin cardiac amyloidosis

2021 ◽  
Author(s):  
Emily Plumadore ◽  
Lindsay Lombardo ◽  
Katherine P Cabral
Keyword(s):  
Treatment Options ◽  
Disease State ◽  
Treatment Modalities ◽  
Limited Data ◽  
Limited Evidence ◽  
Agent Selection ◽  
Selection For ◽  
Tea Extract

Abstract Disclaimer In an effort to expedite the publication of articles , AJHP is posting manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time. Purpose This review aims to summarize the evidence and pharmacological characteristics of treatment options for transthyretin amyloid cardiomyopathy (ATTR-CM). Additionally, this review highlights the role of clinical pharmacists in helping to secure newly introduced therapies. Summary ATTR-CM, a disease characterized by misfolded protein that is deposited in the myocardium and disrupts cardiac functioning, has historically been underdiagnosed due to the need for invasive biopsy and an illusion of rarity. Once diagnosed, limited treatment modalities for ATTR-CM have led providers to rely on nonpharmacological remedies or off-label use of medications with limited evidence of benefit. However, recent noninvasive diagnostic advancements and heightened disease state awareness have revealed increased prevalence of ATTR-CM. This has led to the introduction of several first-in-class pharmaceuticals with actions targeted at inhibiting the various phases of amyloidosis: TTR stabilizers include diflunisal and first-in-class, Food and Drug Administration (FDA)-approved tafamidis; TTR silencers include patisiran and inotersen; fibril disrupters include doxycycline with tauroursodeoxycholic acid; and alternative agents include green tea extract and curcumin. Conclusion ATTR-CM treatments have emerged and, despite current limited data, are continuing to evolve. Tafamidis, the only agent approved by FDA for ATTR-CM, shows promise to improve survival and quality of life in patients with ATTR-CM. Pharmacists can play a key role in assisting with agent selection for this disease state, as well as providing knowledge about current and future clinical trials evaluating the safety and efficacy of the available treatment modalities.

Optimal Management of Small Renal Masses

2006 ◽  
Vol 4 (10) ◽  
pp. 1083-1088 ◽  
Author(s):  
Ralph Hauke
Keyword(s):  
Treatment Options ◽  
Treatment Modalities ◽  
Optimal Management ◽  
Limited Data ◽  
Invasive Procedures ◽  
Older Patient ◽  
Invasive Treatment ◽  
Small Renal Masses ◽  
Renal Masses

The incidence of small renal masses is increasing. Limited data suggest that although most are malignant, they are also likely to show indolent behavior. The tendency of these tumors to occur in older patient populations with multiple comorbidities raises important issues. Retrospective and prospective studies suggest that a substantial proportion of these small renal masses can be observed without compromising the opportunity for cure or subjecting patients to invasive procedures that might impact negatively on quality of life. Less-invasive treatment modalities, including partial nephrectomy and energy ablation techniques, provide multiple options for some patients. This article reviews selected aspects of the management of small renal masses, including their natural history, imaging, diagnostic biopsies, and treatment options. (

scholarly journals GH/IGF-1 Abnormalities and Muscle Impairment: From Basic Research to Clinical Practice

2021 ◽  
Vol 22 (1) ◽  
pp. 415
Author(s):  
Betina Biagetti ◽  
Rafael Simó
Keyword(s):  
Treatment Options ◽  
Basic Research ◽  
Poor Quality ◽  
Limited Evidence ◽  
Skeletal Muscle Function ◽  
Co Morbidity ◽  
Underlying Mechanisms ◽  
Muscle Impairment

The impairment of skeletal muscle function is one of the most debilitating least understood co-morbidity that accompanies acromegaly (ACRO). Despite being one of the major determinants of these patients’ poor quality of life, there is limited evidence related to the underlying mechanisms and treatment options. Although growth hormone (GH) and insulin-like growth factor-1 (IGF-1) levels are associated, albeit not indisputable, with the presence and severity of ACRO myopathies the precise effects attributed to increased GH or IGF-1 levels are still unclear. Yet, cell lines and animal models can help us bridge these gaps. This review aims to describe the evidence regarding the role of GH and IGF-1 in muscle anabolism, from the basic to the clinical setting with special emphasis on ACRO. We also pinpoint future perspectives and research lines that should be considered for improving our knowledge in the field.

Thrombopoietin Receptor Agonists in Children with Immune Thrombocytopenia: A New Therapeutic Era

2020 ◽  
Vol 20 ◽  
Author(s):  
Giuseppe Lassandro ◽  
Valentina Palladino ◽  
Giovanni Carlo Del Vecchioa ◽  
Viviana Valeria Palmieri ◽  
Paola Carmela Corallo ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Treatment Options ◽  
Thrombopoietin Receptor Agonists ◽  
Receptor Agonists ◽  
Thrombopoietin Receptor ◽  
Related Quality ◽  
Health Related

Background and Objective: Immune thrombocytopenia (ITP) is a common bleeding disorder in childhood. The management of ITP in children is controversial, requiring personalized assessment of patients and therapeutic choices. Thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, have been shown to be safety and effective for the treatment of pediatric ITP. The aim of our research is defining the role of thrombopoietin receptor agonists in the management of pediatric ITP. Method: This review focuses on the use of TPO-RAs in pediatric ITP, in randomized trials and in clinical routine, highlighting their key role in management of the disease. Results: Eltrombopag and romiplostim appear effective treatment options for children with ITP. Several clinical studies have assessed that the use of TPO-RAs increases platelet count, decreases bleeding symptoms and improves health-related quality of life. Moreover, TPO-RAs are well tolerated with minor side effects. Conclusion: Although TPO-RAs long term efficacy and safety still require further investigations, their use is gradually expanding in clinical practice of children with ITP.

Role of Erlotinib in Influencing the Quality of Life of Cancer Patients

2020 ◽  
Vol 07 ◽  
Author(s):  
Deepika Purohit ◽  
Parijat Pandey
Keyword(s):  
Quality Of Life ◽  
Lung Cancer ◽  
Cancer Patients ◽  
Kinase Inhibitors ◽  
Treatment Modalities ◽  
Pancreatic Cancers ◽  
Review Reports ◽  
Causes Of Deaths

Background:: Cancer is one of the significant causes of morbidity and mortality in patients globally. Lung cancer, among other cancers, remains to be one of the principal causes of deaths in both men and women. The most common type of lung cancer is the non-small-cell lung cancer (NSCLC). Apart from lung cancer, pancreatic cancer is also one of the common cancers currently. Objective:: The assessment of QoL in erlotinib-treated patients can also prove to be very useful in the establishment of this drug as the main treatment option for the patients with pancreatic and lung cancer. Methods:: Therapies that target EGFR-mediated signalling are the latest keystones for treating these two types of cancers. They comprise of two main treatment modalities: firstly, against the extracellular fields, that include monoclonal antibodies and secondly, mechanisms that create interferences in the signalling pathways, primarily the small molecule tyrosine kinase inhibitors. Results:: Quality of life (QoL) is one of the key advantages in erlotinib therapy over chemotherapy. Conclusion:: The present review reports the role of erlotinib in improving the quality of life of cancer patients especially in NSCLC and pancreatic cancers. The studies or trials establishing the relations between erlotinib and QoL are discussed in detail in this review.

scholarly journals Association Between Diabetes Medications and the Risk of Parkinson's Disease: A Systematic Review and Meta-Analysis

2021 ◽  
Vol 12 ◽  
Author(s):  
Xiaocui Qin ◽  
Xia Zhang ◽  
Pinyu Li ◽  
Min Wang ◽  
Li Yan ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Meta Analysis ◽  
Limited Data ◽  
Dipeptidyl Peptidase 4 ◽  
Single Study ◽  
Dipeptidyl Peptidase 4 Inhibitors ◽  
Glucagon Like Peptide 1

Background: Diabetes mellitus (DM) increases the risk of Parkinson's disease (PD). However, whether DM medications play a part on that increased PD risk is unclear. We designed this meta-analysis to assess the influence of different oral DM medications on the PD risk in patients with DM.Methods: We searched PubMed, Embase, and CENTRAL databases for relevant studies up until January 2021. We pooled adjusted outcomes to assess the PD risk in patients using different DM medications including sulfonylurea, metformin, glitazones (GTZ), dipeptidyl peptidase-4 inhibitors (DPP4i), and glucagon-like peptide-1 agonists (GLP1a).Results: We included 10 studies in our analysis. Our results indicate a lack of significant association between the PD risk and the use of sulfonylureas (three studies; HR, 1.26; 95% CI, 0.95 to 1.66; I2, 70%; p = 0.11), DPP4i (three studies; HR, 0.69; 95% CI, 0.35 to 1.38; I2, 88%; p = 0.30), metformin (five studies; HR, 1.23; 95% CI, 0.98 to 1.78; I2, 84%; p = 0.13), and GTZ (six studies; HR, 0.88; 95% CI, 0.66 to 1.16; I2, 92%; p = 0.35). After exclusion of a single study in the GTZ analysis, our results indicate a significantly reduced PD risk with GTZ use (HR, 0.78; 95% CI, 0.65 to 0.93; I2, 59%; p = 0.06). Similarly, after the exclusion of a single study, our results indicate a significantly increased PD risk with the use of metformin (HR, 1.50; 95% CI, 1.11 to 2.02; I2, 80%; p = 0.008). We also found a significantly reduced PD risk with the use of GLP1a (two studies; HR, 0.41; 95% CI, 0.19 to 0.87; I2, 0%; p = 0.02).Conclusion: The role of different DM medications on the PD risk remains unclear, and the quality of studies is low. While our analysis suggests a lack of association between the use of metformin, GTZ, DPP4i, and sulfonylureas and the PD risk, metformin (to a higher degree) and GTZ may still increase the risk. Limited data suggest a protective effect of GLP1a on the PD risk.

scholarly journals Nasobiliary Drain Diverted through a Percutaneous Endoscopic Gastrostomy Tube: A Novel Approach to Nasobiliary Drainage

2021 ◽  
pp. 891-897
Author(s):  
Nikolaos Dimitrios Pantzaris ◽  
Tim Lord ◽  
Robyn Sotheran ◽  
John Hutchinson ◽  
Charles Millson
Keyword(s):  
Percutaneous Endoscopic Gastrostomy ◽  
Treatment Options ◽  
Primary Biliary Cholangitis ◽  
Percutaneous Endoscopic Gastrostomy Tube ◽  
Limited Data ◽  
Endoscopic Gastrostomy ◽  
Novel Approach ◽  
Nasobiliary Drainage ◽  
Uvb Phototherapy

Intractable pruritus is a common, debilitating symptom and a well-defined entity occurring in chronic cholestatic disorders. Treatment options include cholestyramine, rifampicin, naltrexone, gabapentin, and sertraline, as well as more interventional measures, such as plasmapheresis, extracorporeal albumin dialysis, nasobiliary drains (NBDs), and UVB phototherapy in patients who fail to respond to medical therapy. Despite the limited data, NBD seems to be a highly effective treatment in the relief of refractory cholestatic pruritus. In this article, we present the case of a 73-year-old woman with primary biliary cholangitis and intractable pruritus, refractory to medical treatment. The patient had a complete resolution of her symptoms following an NBD placement, in which, with a novel approach, the nasal end was redirected and exited through a percutaneous endoscopic gastrostomy port, significantly improving her quality of life.

The Role of Nursing in Caring for Patients Receiving Palliative Surgery or Chemotherapy

2019 ◽  
pp. 660-669
Author(s):  
Virginia Sun ◽  
Tami Tittelfitz ◽  
Marjorie J. Hein
Keyword(s):  
Palliative Treatment ◽  
Symptom Control ◽  
Palliative Surgery ◽  
Treatment Modalities ◽  
Indications For Surgery ◽  
Role Of Nursing ◽  
Definition Of ◽  
Treatment Intent

Surgery and chemotherapy are common treatment modalities used to manage disease and symptoms in palliative settings where the disease is incurable. These treatment modalities can lead to deteriorations in a patient’s quality of life (QOL). The benefits of palliative surgery and chemotherapy should always focus on QOL, symptom control, and symptom prevention. The purpose of this chapter is to provide an overview of the definition of palliative surgery and chemotherapy, describe common indications for surgery and chemotherapy for palliative treatment intent, and discuss the role of nursing in caring for patients who are receiving palliative surgery and chemotherapy.

scholarly journals Update on the Treatment of Idiopathic Hypersomnia

2019 ◽  
Vol 5 (4) ◽  
pp. 207-214
Author(s):  
M. S. Schinkelshoek ◽  
R. Fronczek ◽  
G. J. Lammers
Keyword(s):  
Treatment Options ◽  
Pharmacological Therapy ◽  
Small Sample ◽  
Treatment Modalities ◽  
Lifestyle Advice ◽  
Pharmacological Interventions ◽  
First Choice ◽  
Idiopathic Hypersomnia ◽  
Small Sample Sizes

Abstract Purpose of Review Idiopathic hypersomnia is an incapacitating disorder with a profound impact on daytime performance and quality of life. The most commonly used treatment modalities are lifestyle advice and pharmacological therapy. We present an update on the evidence concerning treatment options for idiopathic hypersomnia. Recent Findings Evidence for non-pharmacological interventions is lacking; improvement in symptoms on introducing these interventions is often less pronounced than in narcolepsy. Additional pharmacological treatment is therefore usually initiated. The few treatment studies that have been performed are hampered by small sample sizes and the use of variable and often insufficiently validated outcome parameters for the whole spectrum of idiopathic hypersomnia symptoms. Conclusion Evidence on treatment is scarce. Since the efficacy of modafinil is consistently described and there is much experience with this substance, it is reasonable to start with modafinil as a first choice treatment. Methylphenidate and dexamphetamine are good alternatives. In the future, newer drugs such as sodium oxybate, pitolisant, and solriamfetol might be authorized for use in idiopathic hypersomnia.

scholarly journals Pressure ulcers: Current understanding and newer modalities of treatment

2015 ◽  
Vol 48 (01) ◽  
pp. 004-016 ◽  
Author(s):  
Surajit Bhattacharya ◽  
R. K. Mishra
Keyword(s):  
Pressure Ulcers ◽  
Treatment Options ◽  
Healing Process ◽  
Treatment Algorithm ◽  
Wound Debridement ◽  
Advantages And Disadvantages ◽  
Prone Area ◽  
Long Time

ABSTRACTThis article reviews the mechanism, symptoms, causes, severity, diagnosis, prevention and present recommendations for surgical as well as non-surgical management of pressure ulcers. Particular focus has been placed on the current understandings and the newer modalities for the treatment of pressure ulcers. The paper also covers the role of nutrition and pressure-release devices such as cushions and mattresses as a part of the treatment algorithm for preventing and quick healing process of these wounds. Pressure ulcers develop primarily from pressure and shear; are progressive in nature and most frequently found in bedridden, chair bound or immobile people. They often develop in people who have been hospitalised for a long time generally for a different problem and increase the overall time as well as cost of hospitalisation that have detrimental effects on patient’s quality of life. Loss of sensation compounds the problem manifold, and failure of reactive hyperaemia cycle of the pressure prone area remains the most important aetiopathology. Pressure ulcers are largely preventable in nature, and their management depends on their severity. The available literature about severity of pressure ulcers, their classification and medical care protocols have been described in this paper. The present treatment options include various approaches of cleaning the wound, debridement, optimised dressings, role of antibiotics and reconstructive surgery. The newer treatment options such as negative pressure wound therapy, hyperbaric oxygen therapy, cell therapy have been discussed, and the advantages and disadvantages of current and newer methods have also been described.

Locus of Control, Locus of Control Differences, and Quality of Relationship in Married Dyads

1986 ◽  
Vol 58 (3) ◽  
pp. 939-945 ◽  
Author(s):  
Ronald M. Sabatelli
Keyword(s):  
Locus Of Control ◽  
Marital Satisfaction ◽  
Interpersonal Relationships ◽  
Additional Research ◽  
Limited Data ◽  
Control Locus ◽  
The Relationship ◽  
Mismatch Effect

This study represents a replication and extension of the work focusing on the relationship between spouses' locus of control expectancies and their level of marital complaints. In the present study, spouses' locus of control orientations were examined with reference to their own and their partners' marital complaints. It was predicted that internality would be positively correlated with one's own marital satisfaction and the satisfaction experienced by one's partner. In addition, it was expected that personality complementarity of a particular type, an external wife/internal husband configuration, would be associated with more complaints. The most consistent result of the study suggests the tendency for wives to have more complaints when paired with a relatively external husband. In addition, the analysis did not support the presence of a personality match or mismatch effect on marital complaints. Noting some of the inconsistencies in the limited data amassed on the role of locus of control in the domain of intimate interpersonal relationships, additional research is indicated before further conclusions are advanced.

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