Patient experience with familial chylomicronemia syndrome before and after olezarsen treatment: Qualitative interviews with clinical trial participants
Bibliographic record
Abstract
BACKGROUND: Familial chylomicronemia syndrome (FCS), a rare genetic disorder, markedly increases plasma triglycerides and the risk of acute pancreatitis. FCS symptoms can profoundly impact patients' quality of life. OBJECTIVE: To explore the FCS patient experience during olezarsen treatment, including perceptions of meaningful changes in FCS symptoms and impacts. METHODS: Patients with FCS continuing olezarsen treatment in an open-label extension (OLE) (NCT05130450) of the randomized, placebo-controlled phase 3 Balance study (NCT04568434) participated in 1-hour qualitative interviews. Thematic analysis was conducted. RESULTS: Among 18 OLE participants who completed interviews (55.6% female; mean age, 43.5 years), 17 reported a history of pancreatitis, including 14 with pancreatitis events within 10 years prior to enrollment in the Balance study (13/14 requiring hospitalization). All participants reported having experienced FCS-related symptoms before trial enrollment (most commonly, abdominal pain [94.4%], physical fatigue [66.7%], diarrhea [55.6%], vomiting [50.0%], nausea [33.3%], and difficulty thinking [27.8%]) and impacts (most commonly, dietary restrictions [100%], mood/emotions [94.4%], hospitalizations [77.8%], and social activities [77.8%]). Fifteen of 18 participants (83.3%) reported improvements with olezarsen treatment, including reductions in FCS-related symptoms (abdominal pain [n = 14/17; 82.4%], physical fatigue [n = 7/12; 58.3%], diarrhea [n = 6/10; 60.0%], vomiting [n = 7/8; 87.5%], nausea [n = 3/5; 60.0%], and difficulty thinking [n = 3/5; 60.0%]) and impacts (relationships [n = 6/7; 85.7.0%], hospital admittances [n = 11/14; 78.6%], finances [n = 3/4; 75.0%], and mood/emotions [n = 12/17; 70.6%]). Most participants (15/18; 83.3%) reported meaningful improvements and indicated they were satisfied with olezarsen treatment. CONCLUSION: Results of this qualitative study underscore the significant burden of FCS and support the effectiveness of olezarsen from the patient perspective.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".