Clinical

Patients with myelofibrosis (MF) who achieved a spleen response while taking ruxolitinib and lost it had survival outcomes similar to patients who did not respond, according to results from a real-world study.

A different type of test may improve diagnosis for some patients with myasthenia gravis (MG).

The second half of 2022 brought the first gene therapies for hemophilia to the market in both the United States and abroad.

Relugolix was frequently used in combination with other medications for prostate cancer in patients both new to androgen-deprivation therapy and continuing androgen-deprivation therapy in a real-world study.

Investigators note that although the overall cost for implementing next-generation sequencing (NGS) testing would be more than implementing current polymerase chain reaction (PCR)–based testing, the assay may be more cost effective overall because it screens for multiple conditions.

The results of the futility analysis also suggest the therapy leads to significant clinical benefits.

Despite coming at a high cost, an analysis found that in some cases, chimeric antigen receptor T-cell therapy may be cost-effective compared with the standard of care in the second line or later for relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL).

Geographic atrophy is a leading cause of blindness. The new approval will help to fill an unmet need for patients.

Patients with heart failure and high comorbidity burdens who received postdischarge noninvasive telemonitoring and nurse telephone coaching showed better survival outcomes than those who received standard care.

The authors say newer treatment options for small cell lung cancer (SCLC) may lead to improvement in this area of high unmet need.

Dr Sheth discusses his insights on anti-VEGF therapy that he has noticed in the treatment of wet AMD over the last decade, and Caesar Luo, MD, FASRS, FACS shares his insight on new treatment options and their impact on patient access and cost considerations.

Dr Coney and Veeral Sheth, MD, MBA, FACS, FASRS, discuss treatment selection, treatment goals, and unmet needs for patients diagnosed with wet AMD and DME.

A novel algorithm based on patient-reported outcome questionnaires stratified patients by disease complexity and effectively identified those at a higher risk of having an acute care visit.

Callie Coombs, MD, reviews how patients develop resistance to Bruton Tyrosine Kinase inhibitor (BTKi) therapy and how BTKi resistance is managed.

Roy Beveridge, MD, discusses how payers and health systems are managing Bruton tyrosine kinase inhibitors and how they are chosen for formulary and pathway coverage.

The phase 3 PhALLCON study found ponatinib plus chemotherapy more effective than imatinib with chemotherapy in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia.

Maria Lopes, MD, MS, reviews some challenges surrounding access to care, patient education, and financial burdens associated with psoriasis across skin phototypes.

Early initiation of prophylaxis regimens and joint status monitoring over time are the most relevant aspects of evaluating treatment efficacy in this patient population, study authors concluded.

Amy McMichael, MD, discusses ways to ensure patients of all races and ethnicities are experiencing the same level of quality face-to-face interactions with their providers.

Evidence suggests as many as 29% of people with myeloproliferative neoplasms (MPN) or myelodysplastic syndrome (MDS)/MPN have chronic kidney disease.

Data show that patients who have factor VIII (FVIII) inhibitor titers of 20 or higher BU/mL took longer to achieve complete responses (CR) and had lower CR rates vs patients with lower titers.

The treatment landscape of HoFH is discussed by Eliot Brinton, MD.

Key opinion leaders discuss drivers in health care utilization for patients diagnosed with HoFH.

While the current therapies are effective for treating branch retinal vein occlusion (BRVO) and central retinal vein occlusion (CRVO), they are inadequate for long-term treatment in clinical practice, according to an analysis of real-world data.

Two studies looking at a higher dose of aflibercept found that an 8-mg dose can be maintained at longer dosing intervals with similar benefits and no additional safety signals compared with the 2-mg dose.

Much of the evidence that has been used to base current standard of care for diabetic eye diseases stems from studies performed by the DRCR Retina Network over the years.

The BUTTERFLEYE trial sought to determine if aflibercept, an anti–vascular endothelial growth factor (anti-VEGF) therapy, was equivalent to laser photocoagulation, the gold standard to treat retinopathy of prematurity (ROP) in preterm babies.

Patients with wet age-related macular degeneration (AMD) who are responsive to anti–vascular endothelial growth factor (anti-VEGF) therapy experienced visual gains with no additional injections in the 6 months after treatment with the gene therapy.

Home optical coherence tomography (OCT) has shown there is a wide degree of heterogeneity in fluid dynamics and treatment response that may not be clear during regular office visits and scans. Presenters reviewed the latest data in home OCT to manage wet age-related macular degeneration (AMD).

A review of data on 2 mitochondrial membrane stabilizers—risuteganib and elamipretide—has highlighted the potential to not just slow disease progression but actually reverse vision loss in patients with intermediate dry age-related macular degeneration (AMD).