Development and assessment of educational materials for spinal muscular atrophy carrier screening in the Plain community.

Spinal muscular atrophy (SMA) has been reported in both Amish and Mennonite (Plain) communities, and a higher incidence has been observed in certain Mennonite communities compared to the general population. There are several therapies for SMA, but all are most effective in pre-symptomatic newborns. To identify couples from the Wisconsin Plain community who are most likely to have a child with SMA, carrier screening is offered via mailed kits with at-home specimen collection.

Lifetime Costs for Treated Follicular Lymphoma Patients in the US.

Background And Objective: The objective of this study was to estimate the lifetime costs of patients receiving treatment for follicular lymphoma (FL) in the United States.

Methods: A Markov model was programmed in hēRo3 with a 6-month cycle length, 35-year time horizon (lifetime projection), and health states for line of treatment, response, receipt of maintenance therapy among responders, transformation to diffuse large B-cell lymphoma (DLBCL), development of second primary malignancy (SPM), and death. The model was used to estimate the expected lifetime costs of FL (in 2019 USD), including costs of drug acquisition and administration, transplant procedures, radiotherapy, adverse events, follow-up, DLBCL, SPM, end-of-life care, and indirect costs.

Preferences of Canadian Patients and Physicians for Treatment of HR+/HER2- Advanced Breast Cancer.

(1) Background: Past research suggests that patients with advanced breast cancer prefer treatments with improved clinical outcomes and lower risk of side effects. Evidence on preferences of Canadian patients and physicians for treatments for advanced breast cancer is limited. (2) Methods: Patients' and physicians' preferences for treatments for HR+/HER2-, pre-/peri-menopausal advanced breast cancer were assessed by an online discrete choice experiment (DCE).

Pediatric Vaccines and Cost-Effectiveness Thresholds: How Much is Too Much to Pay for Prevention?

Cost-effectiveness evaluations play an important role in recommendations for use of pediatric vaccines that are set forth by the US Advisory Committee on Immunization Practices (ACIP). The fact that these evaluations are undertaken and accorded weight suggests that a critical value for designating pediatric vaccines as cost-effective (or not) must exist. For recommended pediatric vaccines, however, reported incremental cost-effectiveness ratios (ICERs) have varied greatly, and there does not appear to be an explicit threshold used by the ACIP to define how much is too much to pay for the prevention of communicable diseases in children.

Preferences of Canadian patients and physicians for adjuvant treatments for melanoma.

Background: Past research suggests that patients with early- and late-stage melanoma will endure adverse events and inconvenient treatment regimens for improved survival. Evidence about the preferences of Canadian patients and physicians for novel adjuvant treatments for melanoma is unavailable.

Methods: Patient and physician preferences for adjuvant treatments for melanoma were assessed in an online discrete choice experiment (dce).