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 machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.027 | 0.052 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.005 | 0.005 |
| Scholarly communication | 0.003 | 0.003 |
| Open science | 0.002 | 0.005 |
| Research integrity | 0.002 | 0.004 |
| Insufficient payload (model declined to judge) | 0.003 | 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 source (direct Gemma or distilled Codex), 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".