Summary of study findings in patients with SCD following HSCT
| Author, y (control) . | Main conclusion . |
|---|---|
| Arnold et al,54 2015 (Patients on chronic SCD treatment and healthy siblings) | • No statistically significant difference in HRQOL between HSCT recipients and controls |
| • HSCT recipients had an overall lower health care utilization compared with SCD patients without HSCT | |
| Bhatia et al,55 2015 (No control) | • HRQOL domains improved over time until 1 y posttransplant |
| • Social domain did not improve significantly by 1 y, probably because patients were not enrolled in school | |
| • Parents and patients reported lower HRQOL than population mean for chronically ill children at baseline | |
| • Parents reported that physical domain did not significantly improve by 1 y | |
| • Improvement in QOL may be due to fewer inpatient visits by year 1 compared with SCD patients without HSCT | |
| Gallo et al,56 2019 (No control) | • Recipients with successful HSCT reported the highest HRQOL scores were pursuing their personal life goals |
| • HSCT recipients with avascular necrosis had lower HRQOL scores, yet still pursuing their personal goals | |
| • Lowest HRQOL scores were reported among patients with failed HSCT who continued to have SCD-related complications | |
| • HSCT success was associated with improvement an improvement in HRQOL scores and recipients’ ability to pursue goals | |
| Green et al,57 2017 (No control) | • HSCT recipients (pretransplant) were below at-risk cutoff, 1 standard deviation lower than healthy population (69.7) |
| • HRQOL improved as reported by patients and parents alike | |
| • Persistent brain abnormalities may contribute to HRQOL changes posttransplantation | |
| Kelly et al,53 2012 (Patients with acquired disorders including malignancies or severe aplastic anemia) | • No difference in the proportion of HSCT recipients recovering to baseline as compared with the control group |
| • As per child report, majority of HSCT recipients had not recovered emotional function | |
| • More parents reported their children recovered their general health when compared with the control group | |
| • HSCT recipients reported higher physical function than control group | |
| • Physical function improved over 3 months from baseline among HSCT recipients | |
| • Three-month scores for emotional function were not statistically different from baseline as per child report | |
| • Parents' ratings hit lowest HRQOL scores at 45 d posttransplant and returned to baseline at 3 months | |
| • Parents of HSCT recipients scored 18 points higher compared with the control group for general health | |
| • HSCT recipients rated higher for physical, emotional, and role function compared with their parents | |
| Krishnamurti et al,58 2019 (No control) | • There was significant improvement in physical function from baseline at 1-y post-HSCT transplant |
| • Pain interference domain was also significantly reduced | |
| • Changes in fatigue, anxiety, depression, sleep disturbance, satisfaction with participation in social roles, and pain intensity domains were not statistically significant | |
| • Initial physical function and pain interference domains were significantly lower than the reference population | |
| Saraf et al,59 2016 (No control) | • There was an improvement in HRQOL with nonmyeloablative conditioning regimen as early as day 30 post-HSCT |
| • There was a significant increase in general health, bodily pain, and vitality scores at 1 y post-HSCT |
| Author, y (control) . | Main conclusion . |
|---|---|
| Arnold et al,54 2015 (Patients on chronic SCD treatment and healthy siblings) | • No statistically significant difference in HRQOL between HSCT recipients and controls |
| • HSCT recipients had an overall lower health care utilization compared with SCD patients without HSCT | |
| Bhatia et al,55 2015 (No control) | • HRQOL domains improved over time until 1 y posttransplant |
| • Social domain did not improve significantly by 1 y, probably because patients were not enrolled in school | |
| • Parents and patients reported lower HRQOL than population mean for chronically ill children at baseline | |
| • Parents reported that physical domain did not significantly improve by 1 y | |
| • Improvement in QOL may be due to fewer inpatient visits by year 1 compared with SCD patients without HSCT | |
| Gallo et al,56 2019 (No control) | • Recipients with successful HSCT reported the highest HRQOL scores were pursuing their personal life goals |
| • HSCT recipients with avascular necrosis had lower HRQOL scores, yet still pursuing their personal goals | |
| • Lowest HRQOL scores were reported among patients with failed HSCT who continued to have SCD-related complications | |
| • HSCT success was associated with improvement an improvement in HRQOL scores and recipients’ ability to pursue goals | |
| Green et al,57 2017 (No control) | • HSCT recipients (pretransplant) were below at-risk cutoff, 1 standard deviation lower than healthy population (69.7) |
| • HRQOL improved as reported by patients and parents alike | |
| • Persistent brain abnormalities may contribute to HRQOL changes posttransplantation | |
| Kelly et al,53 2012 (Patients with acquired disorders including malignancies or severe aplastic anemia) | • No difference in the proportion of HSCT recipients recovering to baseline as compared with the control group |
| • As per child report, majority of HSCT recipients had not recovered emotional function | |
| • More parents reported their children recovered their general health when compared with the control group | |
| • HSCT recipients reported higher physical function than control group | |
| • Physical function improved over 3 months from baseline among HSCT recipients | |
| • Three-month scores for emotional function were not statistically different from baseline as per child report | |
| • Parents' ratings hit lowest HRQOL scores at 45 d posttransplant and returned to baseline at 3 months | |
| • Parents of HSCT recipients scored 18 points higher compared with the control group for general health | |
| • HSCT recipients rated higher for physical, emotional, and role function compared with their parents | |
| Krishnamurti et al,58 2019 (No control) | • There was significant improvement in physical function from baseline at 1-y post-HSCT transplant |
| • Pain interference domain was also significantly reduced | |
| • Changes in fatigue, anxiety, depression, sleep disturbance, satisfaction with participation in social roles, and pain intensity domains were not statistically significant | |
| • Initial physical function and pain interference domains were significantly lower than the reference population | |
| Saraf et al,59 2016 (No control) | • There was an improvement in HRQOL with nonmyeloablative conditioning regimen as early as day 30 post-HSCT |
| • There was a significant increase in general health, bodily pain, and vitality scores at 1 y post-HSCT |