A systematic review of cost-effectiveness analyses of gene therapy for hemophilia type A and B.

Background: In 2022-2023, the US Food and Drug Administration approved 2 novel gene therapies, valoctocogene roxaparvovec and etranacogene dezaparavovec, for hemophilia A and B, respectively. These one-time-administered gene therapies have been marketed at prices that create financial challenges for payers and patients. Understanding the magnitude and uncertainties around the long-term value of these therapies and how they can potentially relate to managed care practices is of high interest to the payer and patient community.

Valoctocogene Roxaparvovec and Etranacogene Dezaparavovec: Novel Gene Therapies for Hemophilia A and B.

Objective: To review efficacy and safety data of valoctocogene roxaparvovec (Roctavian) and etranacogene dezaparavovec (Hemgenix), novel gene therapies for the treatment of the life-threatening bleeding disorders hemophilia A and B, respectively.

Data Sources: A PubMed/Google Scholar search from inception through August 11, 2023 was conducted using the following keywords: , , , , ,

Study Selection And Data Extraction: Data, including phase 1 to 3 clinical trials (non-comparator), were obtained from primary literature and package inserts. These reports evaluated clinical pharmacology, efficacy, safety, adverse events, warnings, and precautions.

Antibiotic Approvals in the Last Decade: Are We Keeping Up With Resistance?

Objective: To review the spectrum of activity, efficacy, safety, and role in therapy of all antibiotics and related biologics approved by the Food and Drug Administration (FDA) in the last decade.

Data Sources: A literature search was performed using PubMed and Google Scholar (2010 to end May 2021) with the search terms' name of the antibiotic or the biologic. Data were also obtained from the prescribing information, FDA, and ClinicalTrials.

Managing Multiple Myeloma in the Face of Drug-Induced Adverse Drug Reaction.

Drug-induced liver injury has been reported to cause up to 10% of adverse drug reactions in the United States. Risk factors for druginduced liver injury include female gender, older age, interacting medications and drugs that are metabolized by the liver. This case report describes a patient who was newly initiated on tizanidine, an alpha2 adrenergic agonist used for muscle spasm and musculoskeletal pain, and bortezomib, a proteasome inhibitor used for multiple myeloma.

Osilodrostat: A Novel Steroidogenesis Inhibitor to Treat Cushing's Disease.

Objective: To review data on efficacy and safety of osilodrostat (Isturisa), a novel oral steroidogenesis inhibitor for treatment of Cushing's disease (CD), a life-threatening endocrine disorder.

Data Sources: A PubMed/CINAHL search from inception to September 25, 2020, was performed using the following keywords: , and .

Study Selection And Data Extraction: Phase 2 and 3 clinical trials and supplementary documents investigating osilodrostat were obtained from a primary literature search, the manufacturer's website, and the Food and Drug Administration website.

Peanut Allergen Powder-dnfp: A Novel Oral Immunotherapy to Mitigate Peanut Allergy.

Objective: To review data on efficacy and safety of peanut allergen powder-dnfp (PAP; Palforzia), a novel oral immunotherapy for peanut allergy, a common food allergy.

Data Sources: A PubMed/CINAHL search in English was performed from inception to June 30, 2020, using the search words , and .

Study Selection And Data Extraction Quantification: Published phase 2 and 3 clinical trials, documents presented to the Food and Drug Administration, and supplemental study documentation were reviewed.

Intravenous Immune Globulin (IVIG) for Treatment of Autoimmune Heparin-Induced Thrombocytopenia: A Systematic Review.

Objective: To evaluate intravenous immune globulin (IVIG) for autoimmune heparin-induced thrombocytopenia (aHIT), including platelet recovery, IVIG dose, dosing weight, IVIG product used, and complications reported.

Data Sources: PubMed and EMBASE were searched from inception through June 21, 2020. Search terms included , , , , , , and .

A Systematic Review of Newer Antidiabetic Agents in the Treatment of Nonalcoholic Fatty Liver Disease.

Objective: To evaluate glucagon-like peptide 1 receptor agonists (GLP-1 RAs), dipeptidyl-peptidase IV (DPP-4) inhibitors, and sodium-glucose cotransporter 2 (SGLT) inhibitors to treat nondiabetic and type 2 diabetes mellitus (T2DM) nonalcoholic fatty liver disease (NAFLD) as it relates to improvement in hepatosteatosis (HS) or steatohepatitis (SH).

Data Sources: MEDLINE and CINAHL were searched from inception through May 1, 2020. Search terms included , , , , , and .

Omadacycline: A New Tetracycline Antibiotic.

Objective: To review the chemistry, pharmacology, microbiology, pharmacokinetics, pharmacodynamics, clinical efficacy, tolerability, dosage, and administration of omadacycline, a new tetracycline antibiotic.

Data Sources: A literature search through PubMed, Google Scholar, and clinicaltrials.gov was conducted (2008 to October 2018) using the search terms omadacycline and PTK-0796.

Providing Additional "Muscle" for Older Adults Through Optimal Influenza Vaccine Selection.

Immunization is the best strategy to protect individuals from influenza; however, older adults tend to respond less favorably to vaccines because of immunosenescence. The Centers for Disease Control and Prevention states that any licensed, recommended, and age-appropriate influenza vaccine may be used in older adults despite reasonable evidence suggesting that the high-dose and, to a lesser extent, the adjuvanted and recombinant influenza vaccines provide better protection than the standard-dose vaccines in this vulnerable population. In this era of precision medicine, clinicians can preferentially recommend these contemporary vaccines to equip their older patients with the best possible protection against influenza.

Reducing HIV and partner violence risk among women with criminal justice system involvement: a randomized controlled trial of two motivational interviewing-based interventions.

Women with histories of incarceration show high levels of risk for HIV and intimate partner violence (IPV). This randomized controlled trial with women at risk for HIV who had recent criminal justice system involvement (n = 530) evaluated two interventions based on Motivational Interviewing to reduce either HIV risk or HIV and IPV risk. Baseline and 3, 6, and 9-month follow-up assessments measured unprotected intercourse, needle sharing, and IPV.

Heparin-induced thrombocytopenia: treatment options and special considerations.

Heparin-induced thrombocytopenia (HIT) is an immune-mediated adverse effect that typically manifests several days after the start of heparin therapy, although both rapid- and delayed-onset HIT have been described. Its most serious complication is thrombosis. Although not all patients develop thrombosis, it can be life threatening.

Venous thromboembolism prevention in acutely ill nonsurgical patients.

Objective: To review recent advances in the prevention of venous thromboembolism (VTE) in acutely ill nonsurgical inpatients.

Data Sources: A MEDLINE search (1966-March 2005) was done to identify relevant articles relating to prevention of VTE in acutely ill nonsurgical inpatients.

Study Selection And Data Extraction: Four major prophylaxis trials, one registry, one guideline, and supporting articles representative of the subject matter from the last few years were included.

Long-acting inhaled beta 2-agonists for stable COPD.

Objective: To describe the pathogenesis of chronic obstructive pulmonary disease (COPD) and mechanisms of benefit, formulations available, drug costs, pharmacokinetic profiles, and pertinent clinical studies for long-acting beta(2)-agonists.

Data Sources: A MEDLINE search was conducted from July 1966 through October 2002.

Study Selection And Data Extraction: Pertinent articles related to COPD and long-acting beta(2)-agonists.

Risedronate for the prevention and treatment of corticosteroid-induced osteoporosis.

Objective: To evaluate the role of risedronate in corticosteroid-induced osteoporosis.

Data Sources: Clinical literature was accessed through MEDLINE (1966-February 2001). Key search terms included risedronate, corticosteroid, osteoporosis, and bisphosphonate.