Patient-Centered Research

Feb 27, 2024 | link.springer.com | Patient-Centered Research

AbstractAcid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B.

Dec 6, 2023 | frontiersin.org | Patient-Centered Research |Jean Cheikh |Catarina Geraldes |Jack Khouri

1 IntroductionThe incidence of multiple myeloma (MM) is highly variable across countries but has increased uniformly since 1990 (1, 2). This hematologic cancer disproportionately affects older patients (median age of diagnosis >70 years) in whom the burden of adverse events and decline in health-related quality of life (HRQoL) can be particularly high (3, 4).

Aug 2, 2023 | frontiersin.org | Patient-Centered Research

Caitlin Thomas1* Sikander Ailawadhi2 Rakesh Popat3 David Kleinman4 Melissa M. Ross5 Boris Gorsh6 Sarah Mulnick5 Alicia O'Neill6 Prani Paka6 Maya Hanna6 Nicolas Krucien1 Alexa Molinari6, 7 Heather L.