Current medical research and opinion
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We have conducted a narrative review based on a structured search strategy, focusing on the effects of metformin on the progression of non-diabetic hyperglycemia to clinical type 2 diabetes mellitus. The principal trials that demonstrated a significantly lower incidence of diabetes in at-risk populations randomized to metformin (mostly with impaired glucose tolerance [IGT]) were published mainly from 1999 to 2012. Metformin reduced the 3-year risk of diabetes by -31% in the randomized phase of the Diabetes Prevention Program (DPP), vs. -58% for intensive lifestyle intervention (ILI). ⋯ Multiple health economic analyses suggest that either metformin or ILI is cost-effective in a community setting. Long-term diabetes prevention with metformin is feasible and is supported in influential guidelines for selected groups of subjects. Future research will demonstrate whether intervention with metformin in people with non-diabetic hyperglycemia will improve long-term clinical outcomes.
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Onasemnogene abeparvovec, a one-time intravenous gene replacement therapy, and nusinersen, an antisense oligonucleotide that requires ongoing intrathecal administration, have been evaluated as treatments for spinal muscular atrophy (SMA) type 1 in separate Phase III trials, but no head-to-head comparison studies have been conducted. Onasemnogene abeparvovec was compared with nusinersen using a matching-adjusted indirect comparison (MAIC) to estimate the treatment effect of onasemnogene abeparvovec relative to nusinersen for the treatment of symptomatic patients with SMA type 1 for up to 24 months of follow-up. ⋯ Despite limitations of the current MAIC analysis (mainly a small sample size for statistical testing, even for the pooled onasemnogene abeparvovec trials, and potential differences in prognostic and predictive factors between studies), the relative treatment effects in EFS, OS, and motor milestone achievement indicate that onasemnogene abeparvovec may offer continued benefit compared with nusinersen through 24 months of follow-up.
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This study estimated the comparative efficacy of ciltacabtagene autoleucel (cilta-cel) versus the approved idecabtagene vicleucel (ide-cel) dose range of 300-460 × 106 CAR-positive T-cells for the treatment of patients with relapsed or refractory multiple myeloma (RRMM) who were previously treated with a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody (i.e. triple-class exposed) using matching-adjusted indirect treatment comparisons (MAICs). ⋯ These analyses demonstrate improved efficacy with cilta-cel versus ide-cel for all outcomes, highlighting its therapeutic potential in patients with triple-class exposed RRMM.
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Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare neurological condition, associated with a wide range of symptoms and functional issues, such as profound motor impairment and learning disability. Most individuals with AADC deficiency are completely dependent on their caregivers. This study explored the impact of caring for an individual with AADC deficiency. ⋯ This is the first qualitative study to report on the experience of caring for an individual with AADC deficiency. Caregivers reported that caring had a substantial multifaceted impact on their lives. These findings highlight the importance of considering the caregiver experience when evaluating the burden of AADC deficiency.
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Generalized pustular psoriasis (GPP) is a rare and severe, inflammatory skin disease. GPP is characterized by recurrent flares that consist of disseminated erythematous skin rash with sterile neutrophil-filled pustules that can result in an emergency department (ED) visit or hospital stay due to systemic complications. This study characterizes hospitalizations, ED visits, and inpatient treatment due to GPP in the United States (US). ⋯ The presence of concurrent immune-mediated conditions, and frequent prescribing of analgesics, including opioids, illustrate the burden of GPP in patients requiring acute and inpatient care.