Abstract

Objectives

Cost-effectiveness analysis guidelines emphasize including all relevant costs and effects but few studies include bereavement effects. This study evaluated health-related quality of life, work productivity, and mental health among parents of children who had died of spinal muscular atrophy (SMA) type 1 and how outcomes changed over time.

Methods

From April to June 2023, we partnered with a patient advocacy organization, Cure SMA, to recruit and survey US parents of children who had died of SMA type 1. Survey development included a literature review and qualitative interviews with bereaved parents of children with SMA type 1. The survey measured health-related quality of life (with the Short-Form 12-Item Survey-version 2), work productivity (Work Limitations Questionnaire), anxiety (General Anxiety Disorder-7), and depression (Patient Health Questionarie-9). We derived health utility values using the Short-Form 6-Dimension.

Results

Of 72 responses, 70 completed more than half the survey and were included in the analysis. Respondents were primarily mothers (79%); 89% were more than age 34, and 84% identified as White. Health utility values were significantly (P .05) lower, and mild to moderate anxiety rates were significantly higher than age- and gender-matched US population norms for all parents. Parents bereaved within the last 10 years reported significantly higher productivity loss due to presenteeism, compared with matched norms. Absenteeism and rates of major depression were similar to or lower than matched norms for all parents.

Conclusions

Bereaved parents face significant health and productivity limitations, particularly in the first 10 years after their child’s death. Cost-effectiveness analyses of life-extending therapies should explore the inclusion of bereavement effects.