Mental Health, Quality of Life, and Work Functioning of Parents of Children Who Have Died from Spinal Muscular Atrophy.

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

Methods: From April-June 2023, we partnered with a patient advocacy organization, Cure SMA, to recruit and survey U.S. parents of children who had died from SMA type 1. Survey development included a literature review and qualitative interviews with bereaved SMA type 1 parents. The survey measured HRQOL (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 Six-Dimension.

Results: Of 72 responses, 70 completed over half the survey and were included in the analysis. Respondents were primarily mothers (79%); 89% were over age 34, and 84% identified as White. Health utility values were significantly (p<0.05) lower, and mild to moderate anxiety rates were significantly higher, than age- and gender-matched U.S. population norms for all parents. Parents bereaved within the last 10 years reported significantly higher productivity loss due to presenteeism, compared to 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 following their child's death. CEAs of life-extending therapies should explore the inclusion of bereavement effects.