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Author(s):  
JOST Maud ◽  
Jeffrey T Jensen ◽  
Andrew M Kaunitz ◽  
Melissa Chen ◽  
Jean-Michel Foidart ◽  
...  

Author(s):  
Kazuomi Kario ◽  
Sadayoshi Ito ◽  
Hiroshi Itoh ◽  
Hiromi Rakugi ◽  
Yasuyuki Okuda ◽  
...  

AbstractThere are limited data on the nighttime blood pressure (BP)-lowering effect of esaxerenone and its effect on N-terminal pro b-type natriuretic peptide (NT-proBNP), a predictor of cardiovascular risk, according to different dipping patterns of nocturnal BP. This was a post hoc analysis of a multicenter, open-label, long-term phase 3 study of esaxerenone, a new highly selective mineralocorticoid receptor blocker, in patients with essential hypertension. Patients were classified by dipping pattern (extreme dippers, dippers, non-dippers, risers). Mean changes in BP, changes in dipping pattern, mean NT-proBNP levels, and percentage of patients with normal NT-proBNP levels (<55 pg/mL) at baseline and Weeks 12 and 28 were evaluated. Nighttime systolic BP decreased in all dipping pattern groups at Week 28, with the riser group showing the greatest change (−25.5 mmHg). A significant shift in dipping pattern and riser/non-dipper pattern changes to dipper/extreme dipper pattern were found from baseline to Week 28 (p < 0.0001). The prevalence of the riser pattern decreased from 14.4% to 9.8%, and that of the non-dipper pattern from 44.7% to 39.2%. The decrease in NT-proBNP from baseline to Week 28 was statistically significant in risers, non-dippers, dippers, and extreme dippers (p < 0.001, respectively). At baseline, the proportion of patients with NT-proBNP <55 pg/mL was lowest in risers versus the other dipping pattern types, but after reductions in NT-proBNP in all groups to Week 28, these differences disappeared. Long-term administration of esaxerenone may be a useful treatment option for nocturnal hypertension, especially in patients with a riser pattern.


Author(s):  
JOST Maud ◽  
Andrew M Kaunitz ◽  
Sharon L Achilles ◽  
Jean-Michel Foidart ◽  
Mitchell Creinin, MD

Haematologica ◽  
2021 ◽  
Author(s):  
Marcelo Capra ◽  
Thomas Martin ◽  
Philippe Moreau ◽  
Ross Baker ◽  
Ludek Pour ◽  
...  

Renal impairment (RI) is common in patients with multiple myeloma (MM) and new therapies that can improve renal function are needed. The Phase 3 IKEMA study (NCT03275285) investigated isatuximab (Isa) with carfilzomib and dexamethasone (Kd) vs Kd in relapsed MM. This subgroup analysis examined results from patients with RI, defined as estimated glomerular filtration rate


2021 ◽  
Vol 12 ◽  
Author(s):  
Katarzyna Kotulska ◽  
Aviva Fattal-Valevski ◽  
Jana Haberlova

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletion or mutation of the SMN1 gene. It is characterized by a progressive loss of motor neurons resulting in muscle weakness. The disease affects 1 in 11,000 live births and before the era of treatment SMA was a leading genetic cause of mortality in infants. Recently, disease modifying therapies have been introduced in clinical practice. They include intrathecal and oral antisense oligonucleotides binding to pre-mRNA of SMN2 gene and increasing the translation of fully functional SMN protein as well as SMN1 gene replacement therapy. Onasemnogene abeparvovec uses the adeno-associated virus 9 (AAV9) vector to deliver the SMN1 gene. Phase 1 and phase 3 clinical trials showed that a single administration of onasemnogene abeparvovec resulted in improvement of motor functions in the majority of infants with SMA. Currently, phase 3 trials in SMA1 and SMA2 patients, as well as presymptomatic infants diagnosed with SMA, are ongoing. The drug was approved for medical use in the US in 2019, and in Japan and the European Union in 2020. Thus, first real-world data on efficacy and safety of onasemnogene abeparvovec in SMA patients are available.


Author(s):  
Lisa Stockdale ◽  
Basil Sambou ◽  
Muhamed Sissoko ◽  
Uzochukwu Egere ◽  
Abdou K. Sillah ◽  
...  

AbstractUsing a matched case control design conducted at MRC Gambia in 2015, we measured vitamin D levels in pairs of asymptomatic children with discordant tuberculin skin test status despite the same sleeping proximity to the same adult TB index case. Median ages of groups (infected; 10.0 years, uninfected 8.8 years) were not significantly different (p = 0.13). Mean vitamin D levels were 2.05 ng/mL (95% CI − 0.288 to 4.38) higher in 24 highly TB-exposed uninfected children compared with 24 matched highly TB-exposed infected children (p = 0.08). The findings warrant further investigation in larger studies to understand the implications and significance. Conclusion: Vitamin D levels were higher in TB-uninfected children compared with TB-infected despite equal high exposure to a TB case. What is Known:• Paediatrics TB represents one of the leading causes of child death globally.• Current literature shows an inconsistent relationship between vitamin D deficiency and increased risk of TB disease however a large Phase 3 trial of vitamin D supplementation in (largely vitamin D deficient) Mongolian children did not find any association with TB infection rates. What is New:• This study adds to the literature in a vitamin D sufficient paediatric population whereby children with equal exposure to a household TB case with no evidence of TB infection have higher levels of vitamin D compared with matched children with TB infection.


Author(s):  
Priya Kathuria

A clinical decision report using: Orkin C, Molina JM, Negredo E, et al. Efficacy and safety of switching from boosted protease inhibitors plus emtricitabine and tenofovir disoproxil fumarate regimens to single-tablet darunavir, cobicistat, emtricitabine, and tenofovir alafenamide at 48 weeks in adults with virologically suppressed HIV-1 (EMERALD): a phase 3, randomised, non-inferiority trial. Lancet HIV. 2018;5(1):e23-e34. https://doi.org/10.1016/S2352-3018(17)30179-0 for a patient with viriologically suppressed HIV-1.


Cephalalgia ◽  
2021 ◽  
pp. 033310242110485
Author(s):  
Uwe Reuter ◽  
John H Krege ◽  
Louise Lombard ◽  
Elisa Gomez Valderas ◽  
Judith Krikke-Workel ◽  
...  

Background A significant proportion of triptan users exhibit an insufficient response or inadequate tolerability to a triptan, and some may develop a contraindication. Lasmiditan, a selective 5-HT1F receptor agonist, may be an option for these individuals. We assessed lasmiditan efficacy in a subgroup of patients in CENTURION (Phase 3 migraine consistency study) who exhibited an insufficient response to triptans, including a subgroup with insufficient response due to efficacy only. Methods Patients were randomized to lasmiditan 200 mg for four attacks, lasmiditan 100 mg for four attacks, or placebo for three and lasmiditan 50 mg for one attack. Triptan insufficient responders were pre-defined as patients with insufficient efficacy or tolerability, or who developed a contraindication. Results In triptan insufficient responders, lasmiditan was superior to placebo ( p < 0.05) for pain freedom beginning at 1 h (both doses); pain relief beginning at 0.5 (200 mg) or 1 h (100 mg); migraine-related disability freedom, much/very much better on the Patient Global Impression of Change, and most bothersome symptom freedom at 2 h; sustained pain freedom; and need for rescue medication. Lasmiditan showed benefit for consistency of effect across attacks for 2-h pain freedom and pain relief. Findings were similar in triptan responders and triptan naïve patients and when the triptan insufficient response definition was based on efficacy only. Conclusions Lasmiditan was efficacious across multiple clinically relevant endpoints in the acute treatment of migraine independent of prior response to triptans. Trial Registration: CENTURION (NCT03670810); SAMURAI (NCT02439320); SPARTAN (NCT02605174)


2021 ◽  
Vol 11 ◽  
Author(s):  
Shanshan Deng ◽  
Antonio Solinas ◽  
Diego F. Calvisi

Hepatocellular carcinoma (HCC) is the fourth leading cause of cancer-related mortality worldwide. Patients with early-stage HCC can be treated successfully with surgical resection or liver transplantation. However, the usual late diagnosis of HCC precludes curative treatments, and systemic therapies are the only viable option for inoperable patients. Sorafenib, an orally available multikinase inhibitor, is a systemic therapy approved for treating patients with advanced HCC yet providing limited benefits. Consequently, new drugs have been developed to overcome sorafenib resistance and improve patients’ prognoses. A new promising strategy is using c-MET inhibitors, such as cabozantinib, as activation of c-MET occurs in up to 40% of HCC patients. In particular, cabozantinib, in combination with the checkpoint inhibitor atezolizumab, is currently in phase 3 clinical trial for HCC, and the results are eagerly awaited. Herein, we summarize and review the drugs approved for the treatment of advanced HCC, mainly focusing on the clinical and preclinical efficacy evaluation of cabozantinib. Also, we report the available preclinical data on cabozantinib-based combination therapies for HCC, current obstacles for cabozantinib therapy, and the future directions for cabozantinib-based treatment for HCC.


2021 ◽  
Author(s):  
Chunli Yang ◽  
Chunxi Fu ◽  
Huawei Weng ◽  
Liqun Zou

Abstract Background Glucocorticoids-containing regimens are the standard first line treatment for most lymphoma patients, however, as the improvement of overall survival in these cases, glucocorticoids related osteopenia or osteoporosis attracted attention in clinical administration. We aim to investigate the efficacy of bisphosphonates in prevention bone mineral loss in glucocorticoids-treated lymphoma patients. Methods This is a prospective, randomized controlled phase 3 trial. Eligible lymphoma adults from China with first line glucocorticoids involved treatment, were randomly assigned 1:1 to receive either Zoledronic acid (ZA) for twice infusion or not. All patients received daily oral calcium and vitamin D3 for one year. The primary endpoint, alternations of bone mineral loss from enrollment to the twelfth month at the lumbar spine (L1-L4), left hip and left femoral neck, measured by the dual-energy, x-ray absorptiometry scanners, recorded as T score; and new bone fractures during the first year, were analyzed by intent-to-treat. This trial was registered with www.Chictr.org, number ChiCTR-INR-17010771. Results Between May, 2016 to July, 2019, 84 patients were randomly assigned to both Zoledronic acid and control groups, 29 patients completed the study and were on follow-up. We found a marked improvement of T score in ZA group compared with the control in a year at lumbar spine, T score changes of L1-4 in ZA group and control group were as following: L1, 0.14±0.61 vs. -0.33±0.40, p=0.009; L2, 0.28±0.41 vs. -0.35±0.54, p=0.003; L3, 0.23±0.42 vs. -0.23±0.66, p=0.020; L4, 0.28±0.44 vs. -0.35±0.54, p=0.020. Meanwhile, no severe adverse event was observed on both groups. Conclusions Glucocorticoids-treatment lymphoma patients that prophylactic ZA infusion could relieve BMD loss to prevent osteoporosis without increasing severe adverse effects in Chinese population, providing reference for clinicians to consider ZA application in this situation.


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