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. 2021 Jan 26;5(2):570–583. doi: 10.1182/bloodadvances.2020002948

Table 5.

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