scholarly journals Treating ITP: What are the options in the era of new guidelines and new drugs?

2020 ◽  
Vol 71 (4) ◽  
pp. 40-46
Author(s):  
Nikola Pantić ◽  
Nada Suvajdžić-Vuković

Primary immune thrombocytopenia (ITP) is an autoimmune disease characterized by isolated thrombocytopenia. Treating ITP may be challenging since different treatment modalities are available. While choosing the suitable option for every patient, a physician should take into account both patient's medical characteristics and wishes. The first line treatment options include: corticosteroids, intravenous immunoglobulins and intravenous anti-D immunoglobulin. Second line treatment options comprise medical (thrombopoietin receptor agonists, rituximab, fostamatinib, azathioprine, cyclophosphamide, cyclosporin A, hydroxychloroquine, mycophenolate mofetil, danazol, dapsone, vinca-alcaloids) and surgical (splenectomy) approach. However, there are some treatment gaps which remain uncovered with existing treatment modalities. Therefore, development of novel therapeutic strategies is required. The aim of this review is to provide an illustrative overview of novel treatments for adult ITP.

2018 ◽  
Vol 24 (5) ◽  
pp. 549-558 ◽  
Author(s):  
Vanessa Henriques ◽  
Teresa Martins ◽  
Wolfgang Link ◽  
Bibiana I. Ferreira

Melanoma is the deadliest form of skin cancer being responsible for 80% of skin cancer deaths. Furthermore, the incidence of metastatic melanoma has increased over the past three decades with a mortality rate that continues to rise faster than most of all other cancers. The last few years have witnessed an unparalleled change in treatment options for patients with metastatic melanoma by the development of new therapeutic strategies like targeted therapies and immunotherapies that highly improved the patient’s prognosis. Despite the paradigm- shifting success of these novel treatments, their effectiveness is still limited by intrinsic or acquired resistance. The objective of this review is to provide an overview of the new available treatment modalities, criteria to select patients who might benefit from a specific therapy, mechanisms of innate and acquired resistance to these treatments and to discuss strategies to overcome drug resistance.


1994 ◽  
Vol 80 (5) ◽  
pp. 315-326 ◽  
Author(s):  
Marco Colleoni ◽  
Fernando Gaion ◽  
Guido Liessi ◽  
Giorgio Mastropasqua ◽  
Patrizia Nelli ◽  
...  

Background Hepatocellular carcinoma (HCC) remains one of the most common neoplasms worldwide. Curative treatment options include liver transplantation or resection. Unfortunately, most patients still have unresectable or untransplantable HCC due to disease extension or comorbid factors and are therefore candidate only for palliative treatments. Methods In this review we have analyzed the different medical approaches employed in the treatment of HCC in an attempt to better define their roles. Results Palliative medical treatments including systemic chemotherapy, immunotherapy or hormonal manipulation rarely influence survival of the patients. Although a high response rate is often reported with new local therapies such as transcatheter arterial embolization, intraarterial chemotherapy or percutaneous ethanol injection, the real impact of these treatment modalities on patient survival remains to be determined. Conclusion One way to improve the diagnosis of HCC patients would be an appropriate approach to evaluate new drugs or treatment modalities. To answer all the open questions, further trials, possibly randomized, should be conducted on a substantial number of patients with homogeneous prognostic factors.


Author(s):  
Alyssa Bujnak ◽  
Carly A. Crowder ◽  
Michael L. Krychman

Abstract Vaginal rejuvenation is a marketing term that encompasses surgical and medical treatments for functional vaginal/vulvar problems including but not limited to genitourinary syndrome of menopause (GSM), sexual dysfunction, vaginal laxity, and stress urinary incontinence (SUI) and for esthetic concerns including dissatisfaction with vulvovaginal appearance. Multiple treatment options have become available for indications of functional vaginal problems. Noninvasive management options including the use of more novel treatments such as energy-based devices have gained interest. Previously, studies regarding the efficacy and safety of the energy-based devices for functional vaginal problems were mostly limited to cohort studies without sham treatment, control groups, randomization, or double blinding. As a result of this insufficient data in 2018, the FDA released a statement of warning against the use of energy-based devices in the treatment of functional vaginal problems or vaginal cosmetic procedures (Https://Www.Fda.Gov/Medical-Devices/Safety-Communications/Fda-Warns-Against-Use-Energy-Based-Devices-Perform-Vaginal-Rejuvenation-or-Vaginal-Cosmetic. 1–4, 2018). Purpose of Review This article reviews the most current treatment modalities in the realm of vaginal rejuvenation therapy with an emphasis on the efficacy and safety of the energy-based devices. Recent Findings In the most recent literature, there have been studies with improvements in study design that support the efficacy and the short-term safety of the energy-based devices. Summary More recent studies with improved study design evidence that the use of energy-based devices results in improvements in functional vaginal problems and that serious adverse events appear to be rare. The availability of these devices as treatment options for functional vaginal problems has the potential to impact patient by improving their symptoms and quality of life. Caution still remains however regarding their safety following a longer period of time after their use.


Author(s):  
Irushna Antonette Perera ◽  
Jebananthy Anandaselvam Pradeepan ◽  
Uruthirapasupathy Mayorathan ◽  
Thirunavukarasu Kumanan Thirunavukarasu Kum ◽  
Kopalasuntharam Muhunthan ◽  
...  

AFLP, though rare, is an obstetric emergency which carries a high incidence of maternal and perinatal mortality, despite optimal care. We report a case of a 23-year-old primi mother, who presented to us with vague symptoms of feeling unwell and abdominal pain, was diagnosed with AFLP, and was managed accordingly with the highest level of care available but succumbed a few days into her illness though timely delivery was able to save the baby. By this case report we once again wish to emphasize the value of a timely diagnosis with a high level of clinical suspicion and supportive laboratory investigations including imaging; the need for early termination of pregnancy; and adequate supportive care as the key management options for AFLP. Further, we wish to explore the current treatment options available for AFLP and discuss a few novel therapeutic strategies such as plasma exchange in treating such cases and the pros and cons associated with these treatment modalities.


2021 ◽  
Vol 14 (6) ◽  
pp. 520
Author(s):  
Ashli M. Heustess ◽  
Melissa A. Allard ◽  
Dorothea K. Thompson ◽  
Pius S. Fasinu

Since the outbreak and subsequent declaration of COVID-19 as a global pandemic in March 2020, concerted efforts have been applied by the scientific community to curtail the spread of the disease and find a cure. While vaccines constitute a vital part of the public health strategy to reduce the burden of COVID-19, the management of this disease will continue to rely heavily on pharmacotherapy. This study aims to provide an updated review of pharmacological agents that have been developed and/or repurposed for the treatment of COVID-19. To this end, a comprehensive literature search was conducted using the PubMed, Google Scholar, and LitCovid databases. Relevant clinical studies on drugs used in the management of COVID-19 were identified and evaluated in terms of evidence of efficacy and safety. To date, the FDA has approved three therapies for the treatment of COVID-19 Emergency Use Authorization: convalescent plasma, remdesivir, and casirivimab/imdevimab (REGN-COV2). Drugs such as lopinavir/ritonavir, umifenovir, favipiravir, anakinra, chloroquine, hydroxychloroquine, tocilizumab, interferons, tissue plasminogen activator, intravenous immunoglobulins, and nafamosat have been used off-label with mixed therapeutic results. Adjunctive administration of corticosteroids is also very common. The clinical experience with these approved and repurposed drugs is limited, and data on efficacy for the new indication are not strong. Overall, the response of the global scientific community to the COVID-19 pandemic has been impressive, as evident from the volume of scientific literature elucidating the molecular biology and pathophysiology of SARS-CoV-2 and the approval of three new drugs for clinical management. Reviewed studies have shown mixed data on efficacy and safety of the currently utilized drugs. The lack of standard treatment for COVID-19 has made it difficult to interpret results from most of the published studies due to the risk of attribution error. The long-term effects of drugs can only be assessed after several years of clinical experience; therefore, the efficacy and safety of current COVID-19 therapeutics should continue to be rigorously monitored as part of post-marketing studies.


2019 ◽  
Vol 24 (3) ◽  
pp. 242-246
Author(s):  
Mary Soliman ◽  
Elissa Engel ◽  
Juan Rico ◽  
Carina Rodriguez

Thrombocytopenia and other hematologic manifestations related to HIV are not uncommon. Treatment of HIV-related thrombocytopenia is challenging: treatment options are not effective in all patients, or less well studied, particularly in the pediatric population. We aim to present and discuss the case of a 13-year-old with HIV and persistent thrombocytopenia who, after failing monthly IVIG infusions, showed normalization of platelet count on the novel thrombopoietin receptor agonist, eltrombopag. A retrospective chart review of the case patient's medical record was conducted. Additionally, a thorough literature review was performed on this topic, including the pathophysiology of underlying HIV-related thrombocytopenia and its treatment modalities. The patient was treated initially with monthly IVIG infusions for about 1 year but did not show a sustained response, particularly in between infusions. After initiation with eltrombopag 50 mg daily, the patient showed a sustained increase in his platelet count. During a brief lapse in eltrombopag treatment, his platelet count dropped, which then increased upon his reinitiation of therapy. He has continued to show a sustained platelet response and has not been symptomatic or required IVIG for more than 1 year. To our knowledge, this is the first report of a pediatric patient with HIV-related thrombocytopenia who has benefited from the use of eltrombopag.


Blood ◽  
2017 ◽  
Vol 129 (21) ◽  
pp. 2829-2835 ◽  
Author(s):  
Michele P. Lambert ◽  
Terry B. Gernsheimer

Abstract Immune thrombocytopenia (ITP) occurs in 2 to 4/100 000 adults and results in variable bleeding symptoms and thrombocytopenia. In the last decade, changes in our understanding of the pathophysiology of the disorder have led to the publication of new guidelines for the diagnosis and management of ITP and standards for terminology. Current evidence supports alternatives to splenectomy for second-line management of patients with persistently low platelet counts and bleeding. Long-term follow-up data suggest both efficacy and safety, in particular, for the thrombopoietin receptor agonists and the occurrence of late remissions. Follow-up of patients who have undergone splenectomy for ITP reveals significant potential risks that should be discussed with patients and may influence clinician and patient choice of second-line therapy. Novel therapeutics are in development to address ongoing treatment gaps.


VASA ◽  
2012 ◽  
Vol 41 (3) ◽  
pp. 163-176 ◽  
Author(s):  
Weidenhagen ◽  
Bombien ◽  
Meimarakis ◽  
Geisler ◽  
A. Koeppel

Open surgical repair of lesions of the descending thoracic aorta, such as aneurysm, dissection and traumatic rupture, has been the “state-of-the-art” treatment for many decades. However, in specialized cardiovascular centers, thoracic endovascular aortic repair and hybrid aortic procedures have been implemented as novel treatment options. The current clinical results show that these procedures can be performed with low morbidity and mortality rates. However, due to a lack of randomized trials, the level of reliability of these new treatment modalities remains a matter of discussion. Clinical decision-making is generally based on the experience of the vascular center as well as on individual factors, such as life expectancy, comorbidity, aneurysm aetiology, aortic diameter and morphology. This article will review and discuss recent publications of open surgical, hybrid thoracic aortic (in case of aortic arch involvement) and endovascular repair in complex pathologies of the descending thoracic aorta.


2011 ◽  
Vol 7 (1) ◽  
pp. 51 ◽  
Author(s):  
Frederic Baumann ◽  
Nicolas Diehm ◽  
◽  

Patients with critical limb ischaemia (CLI) constitute a subgroup of patients with particularly severe peripheral arterial occlusive disease (PAD). Treatment modalities for these patients that often exhibit multilevel lesions and severe vascular calcifications are complicated due to multiple comorbidities, i.e. of cardiac and vascular but also of renal origin. These need to be taken into consideration while planning treatment options. Although CLI is associated with considerably high morbidity and mortality rates, the clinical outcome of patients being subjected to revascularisation has improved substantially in recent years. This is mainly due to improved secondary prevention strategies as well as dedicated endovascular innovations for this most challenging patient cohort. The aim of this article is to provide a discussion of the contemporary treatment concepts for CLI patients with a focus on arterial revascularisation.


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