Health-Related Quality of Life of Adults and Adolescents with Sickle Cell Disease after Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation
Bibliographic record
Abstract
Sickle cell disease (SCD) is a group of inherited blood disorders that leads to lifelong multisystem complications that significantly influence health-related quality of life (HRQOL). Allogeneic hematopoietic stem cell transplantation (HSCT) is a well-established curative therapy for patients with severe SCD. The primary objective of this study was to compare the HRQOL of adolescent and adult SCD patients who underwent non-myeloablative (NMA) HSCT (recipients) versus a group of HSCT candidates who did not undergo transplant (candidates). 296 SCD patients ≥ 14 years of age (111 candidates and 185 recipients) were invited to complete a one-time online survey that contained sociodemographic questions, a multimorbidity scale, and an HRQOL questionnaire (SF-36). Clinical data was collected from the electronic medical records of enrolled participants. Multiple linear regression models were conducted to analyze the difference in the HRQOL domains between recipients and candidates while adjusting for covariates. A total of 181 SCD patients (73 candidates and 108 recipients) completed the online survey between January 2023 and June 2023. Recipients reported significantly higher HRQOL mean scores in all SF-36 domains (p<.05) in the univariate analysis compared to candidates except for Mental Health (p=.892). Likewise, while controlling for demographic and clinical characteristics, the multiple regression analysis showed a significant difference in all domains (p<.05) and in the physical health component score (p<.001), but not in the Mental Health domain (p=.823) and mental health component score (p=.158). In this study, adolescents and adults with SCD who underwent NMA HSCT reported better HRQOL compared to similar groups of patients who were considered candidates for HSCT. Overall, the findings suggest that NMA HSCT has a positive impact on HRQOL. These results may assist providers and patients/families when considering HSCT as a treatment for SCD.
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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 | 0.000 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| 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".