Longitudinaler Verlauf der Lungenfunktion in einer Kohorte von Kindern mit Sichelzellerkrankung und Hydroxyurea Therapie
Bibliographic record
Abstract
Background Prior to the introduction of hydroxyurea (HU) therapy, the annual decline in pulmonary function in patients with the most common and severe form of sickle cell disease (SCD), HbSS, was comparable to cohorts with cystic fibrosis (2-3% ppFEV 1 / year). HU results in improved outcomes including a reduction in acute chest syndrome and pain crises. It is recommended for the treatment of individuals with HbSS for over a decade now but long-term effects on longitudinal lung function decline in large paediatric cohorts are scarce. Methods Children diagnosed with HbSS on HU treatment or children with the milder HbSC SCD and at least two clinic visits with pulmonary function testing (PFT) at SickKids hospital were eligible for inclusion in this retrospective study. PFTs obtained during chest exacerbations were excluded. Between-group comparisons were carried out using chi-square or two-sample unpaired t-test. The annual decline in PFTs was analysed using unadjusted linear regression. Results 407 children (259 HbSS and 148 HbSC) with a total of 1,341 visits were included; average observation period 3.5 years. 56% were male, mean age at inclusion was 11.2 years (SD 3.0), FEV 1 was 76.8 (SD 3.0) and TLC 82.0 (SD 10.6) of percent predicted (pp). No significant differences in baseline characteristics were found between HbSS and HbSC. In males with HbSS, the annual decline in ppFEV 1 was -1.0%/year (p<0.0001) and TLC was -1.2%/year (p<0.0001). No significant decline in FEV 1 or TLC was found in females with HbSS. Children with HbSC also had stable pulmonary function over the observation period. Conclusions HbSS and male gender are risk factors for a more rapid progression of sickle cell lung disease in children treated with HU. While reasons for the observed sex difference are unclear, our observations should be implemented in prognostic stratification, personalized therapeutic decisions, and the implementation of surveillance programs. Publication History Article published online: 21 September 2022 © 2022. Thieme. All rights reserved. Georg Thieme Verlag Rüdigerstraße 14, 70469 Stuttgart, Germany
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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 | 0.004 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 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".