Health-Related Quality of Life following Sirolimus Treatment for Vascular Anomalies
Bibliographic record
Abstract
Objective: Sirolimus is a safe and effective pharmaceutical for the treatment of vascular anomalies (VAs); however, there are limited analyses of health-related quality of life (HRQoL) following sirolimus use. This study documents the indications, HRQoL outcomes, and complications of sirolimus in a cohort of patients with VAs. Methods: This was a cross-sectional study with retrospective chart review. Children and adults with VAs treated with sirolimus from 2010 to 2024 were invited to complete the Glasgow Children’s Benefit Index (GCBI) HRQoL survey. Demographics, diagnoses, treatment indications, additional treatments, and complications were collected from medical records. Results: Twenty-one of 24 patients completed the survey (88% response rate, 38% female). Diagnoses encompassed capillary, lymphatic, venous, and combined vascular malformations, as well as complex VAs associated with other disorders. Indications for sirolimus were bulk, pain, appearance, function, hematologic abnormalities, frequent infections, and lymphocutaneous fistula. Median age at treatment initiation was 12 years (range: 11 months–21 years), and median treatment duration was 28.5 months (range: 6.1 months–10.3 years). HRQoL outcomes varied (median: GCBI +8.0 [IQR: −4 to +20], range: −20.0 to +80.0), and 57% of participants experienced overall improvement. Sirolimus resulted in a positive median change in GCBI across all domains apart from emotion: learning: +16.7 (0.0–29.20), vitality: +15.0 (−5.0 to 30.0), physical health: +6.3 (−12.5 to 18.8), and emotion: 0.0 (0–16.7). About 71% of participants reported one or more complications, with mucositis being most common. Conclusion: Sirolimus improved HRQoL in 57% of participants with diverse VA diagnoses and varied indications for treatment. HRQoL was highly varied (GCBI range: −20.0 to +80.0). These results may help inform clinician and patient expectations for sirolimus HRQoL outcomes. Level of evidence: Level 4.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
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 | 0.004 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.002 |
| Bibliometrics | 0.001 | 0.000 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| 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".