Clinical

Early diagnosis of Fabry disease is essential and that since symptoms depend on the type of disease and sex and age of the patient, a high-risk screening system should account for the age of the target population, the researchers said.

Oncology drugs are a huge driver of cost, but there’s a lot of innovation happening in the space to improve care and outcomes for patients, said Michelle Booth, PharmD, director of specialty clinical solutions at Magellan Rx Management.

There are a number of trastuzumab biosimilars available, but so far there have not been any patient characteristics that have played a role in which ones are prescribed, said Cate Lockhart, PhD, PharmD, MS, program, director, Biologics and Biosimilars Collective Intelligence Consortium.

Risankizumab, a biologic, is used for psoriasis, but whether it can improve asthma control has been unclear.

For 3 categories of evaluation, elevated cardiac troponin I levels most influenced the development of myocardial injury among persons at higher lifetime weight totals.

Use of comprehensive genomic profiling in patients with advanced non–small cell lung cancer (NSCLC) was linked with significant improvements in progression-free survival and overall survival.

Ryan Haumschild, PharmD, MS, MBA, discusses optimizing the transition to biosimilars, covering from what patients to look for as candidates to the process of switching a patient from a brand to a biosimilar to the importance of patient education.

Ryan Haumschild, PharmD, MS, MBA, continues focusing on transition optimization, explaining how to efficiently create your inventory, how his use of biosimilars will change in the future, and offering advice to those looking to transition to biosimilars.

A new review article highlights some of the newest therapies available to treat graft-versus-host disease (GVHD), but the authors say there is insufficient data to draw broad conclusions.

A pan-cancer reference atlas provides a framework for an immune-based patient stratification that study authors expect to be predictive for patient prognosis and immunotherapy response at an elevated level after further studies.

In patients refractory to conventional therapy, the combination resulted in a 70% reduction in a key biomarker compared with placebo.

Posters presented at AMCP Nexus 2021 evaluated the health care resource utilization (HRU) and costs associated with the chimeric antigen receptor T-cell therapies tisagenlecleucel and axicabtagene ciloleucel.

The report suggests factors besides molecular profiling can be helpful in patient stratification.

Two abstracts presented at the CHEST Annual Meeting 2021 showed that patients with narcolepsy treated with once-nightly sodium oxybate, FT218, vs placebo exhibited significant improvements in sleep latency and cataplexy.

Angelman syndrome affects roughly 1 in every 20,000 children and it has no approved treatment.

A report suggests that people with chronic obstructive pulmonary disease (COPD) and asthma have a protein in their lungs that leaks a small molecule into their bloodstream that restricts their breathing instead of relaxing their airways.

Mortality and liver-related complications increased with fibrosis stage in patients with nonalcoholic fatty liver disease (NAFLD).

Study findings suggest that the unique genomic variations of cerebrospinal fluid can be leveraged as a liquid biopsy to effectively and safely improve decision-making regarding treatment of patients with non–small cell lung cancer (NSCLC) with leptomeningeal metastasis.

Posters presented at the AMCP Nexus 2021 meeting reviewed the cost-effectiveness and cost per response for zanubrutinib vs ibrutinib.

A review of current molecular and electrophysiological biomarkers in spinal muscular atrophy (SMA) concluded that more exploration is necessary to find noninvasive, yet accurate, measures of disease progression and therapy response.

Autologous tumor lysate particle-loaded dendritic cell (TLPLDC) vaccination is well-tolerated in combination with other immunotherapies, but further research is needed to confirm its efficacy.

The benefit of real-world evidence is that it provides more data on subpopulations and diverse populations, said Christina Barrington, vice president of pharmacy programs at Priority Health.

A new study from Turkey shows, among other results, that delayed initiation of treatment with growth hormone among pediatric patients may lead to minimal height improvements despite adherence to the therapy.

Adalimumab biosimilar AVT02 exhibited comparative efficacy, safety, tolerability, and immunogenicity to the reference drug Humira in patients with moderate-to-severe chronic plaque psoriasis.

There are currently no FDA-approved treatments for refractory chronic cough or unexplained chronic cough. At the CHEST Annual Meeting 2021, attendees heard a readout from a durability study as well as pooled analyses from 2 phase 3 trials for gefapixant.