A Longitudinal Examination of the Quality of Life of Persons with Hemophilia a Switching from Intravenous Recombinant Factor VIII Concentrate to Subcutaneous Non-FVIII Replacement Therapy
Bibliographic record
Abstract
Background. In Canada, ‘standard of care’ for persons with hemophilia A (PWH-A) has been regular intravenous infusions of factor VIII (FVIII) concentrates started early in life. Recently, novel non FVIII therapies that do not require intravenous infusion, and are administered infrequently and subcutaneously, have become clinically available. These therapies may impact health-related quality of life (HR-QoL) of PWH-A. This patient-reported outcome can be assessed using the Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLATv3.0). Aim. To compare the long-term HR-QoL of PWH-A pre-/post-switch to emicizumab. Methods. PWH-A on FVIII prophylaxis, and their caregivers, completed questionnaires at baseline (day of switch to emicizumab), 3-, 6-, and 12-months post-switch. This study was approved by the institutional research ethics board. Informed consent was obtained from participants. PWH-A aged 7-18 years completed the self-report CHO-KLAT, and caregivers of PWH-A aged 0-18 years completed the caregiver-report CHO-KLAT. The CHO-KLAT is scored 0- 100, with 100 representing best HR-QoL. The CHO-KLAT is also scored within 7 domains: activities, autonomy, bleeding, emotional health, hemophilia knowledge, social functioning, and treatment. Results. PWH-A (N=26; Mage=12.4 years, SD=4.2; 100% male; 97% infusing prophylaxis >twice/week pre-switch) and their caregivers (N=34) completed the questionnaires. Mean CHO KLAT scores increased at 3-months and plateaued by 12-months. Child and caregiver CHO-KLAT scores were significantly correlated at baseline (ρ=.53**, CI=.16-.77) and 12-months (ρ=.92**, CI=.66-.98), indicating similar caregiver versus child-perceived HR-QoL longitudinally. There was a significant improvement in caregiver-reported CHO-KLAT scores from baseline to 3- months, by an average of 8.65 (t18=-3.36, CI=-14.05–3.25). There was a significant improvement in caregiver-reported autonomy from baseline to 12-months (Z=-3.26, p<.001). Conclusion. PWH-A and their caregivers reported greatest HR-QoL at 3- and 6-months post switch. The HR-QoL findings align with the literature on other chronic conditions, where the greatest changes are observed immediately following a treatment switch, followed by a plateau over time.
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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 | 0.001 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.001 | 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".