Summary of study findings in patients with thalassemia following HSCT
| Author, y (control) . | Main results . |
|---|---|
| Caocci et al,45 2006 (No control) | • All HRQOL domains were found to be worse in HSCT recipients with GVHD |
| • No statistically significant difference between GVHD and non-GVHD patients for global QOL | |
| Caocci et al,44 2011 (No control) | • Physical function worsened from baseline to 3 mo post-HSCT, then improved at 6 to 18 mo |
| • Total PedsQL scores were stable from baseline to 3 mo post-HSCT, then improved at 6 and 18 mo | |
| • Social, emotional, and psychosocial scores did not differ significantly following HSCT | |
| • Parents slightly underestimated their children's QOL | |
| Caocci et al,46 2016 (Sibling donors and patients treated with conventional treatments) | • Physical scores were not significantly different in HSCT recipients, compared with their sibling donors, but were overall better than patients with thalassemia on conventional treatment (chronic transfusions and iron chelation therapy) |
| • No significant difference between the groups for mental component summary scores | |
| Cheuk et al,47 2008 (Thalassemia patients with chronic blood transfusion and iron chelation) | • Overall health in HSCT recipients was better than transfusion-dependent patients |
| • HSCT recipients had less fatigue and better relationships | |
| • Physical domain scores were better (higher) in HSCT recipients | |
| • Psychological, social relationships, and environment domains were similar between the 2 groups | |
| • Physical, emotional, social, school, and psychosocial PedsQL domains were not significantly different among the 2 groups | |
| Javanbakht et al,48 2015 (Thalassemia patients with chronic blood transfusion and iron chelation) | • HSCT recipients reported better QOL scores compared with controls |
| • Patients living in rural areas had better EQ-VAS scores | |
| • Patients who underwent HSCT had better score in the physical component scale | |
| • No significant difference between HSCT recipients and controls in self-care, mobility, and usual activities | |
| • HSCT recipients had significantly better pain, discomfort, anxiety, and depression domains | |
| • No significant difference between the groups for EQ-5D-3L index scores | |
| Kelly et al,53 2012 (Patients with acquired disorders including malignancies or severe aplastic anemia) | • No difference in the proportion of patients with HSCT recipients recovering to baseline, compared with the control group |
| • Per child report, majority of HSCT recipients had not recovered emotional function | |
| • More parents reported that their children recovered their general health as compared with the control group | |
| • HSCT recipients physical function improved over 3 months from baseline, which was also better compared with controls | |
| • Three-month scores for emotional function were not statistically different from baseline among groups, per child report | |
| • Parents' report of HRQOL was worst at 45 d posttransplant and returned to baseline at 3 months | |
| • Parents of HSCT recipients scored 18 points higher (better) compared with the control for general health | |
| • Children rated higher for physical, emotional, and role function, compared with their parents | |
| Kisecik Sengul et al,49 2017 (No control) | • Child total scale score and psychosocial health score are slightly and moderately associated with parental Beck's Depression Index score, respectively |
| • No significant association between parent's depression score and child's physical health score | |
| • According to parents’ HRQOL survey, there was no significant association between parental depression and children's total score or physical health score, but there was a significant association with child's psychosocial health score | |
| • Depressed parents scored their children’s HRQOL lower than parents who were not depressed | |
| La Nasa et al,50 2013 (General population and thalassemia patients on chronic blood transfusion and iron chelation) | • HSCT recipients had similar HRQOL profiles compared with the general population in the long term and had a better HRQOL compared with conventionally treated β-thalassemia patients |
| • All other physical and mental health domains had no significant difference | |
| • Patients with comorbidities at the time of survey scored their general health lower than patients without comorbidities | |
| • HSCT recipients scored better physical function, bodily pain, and emotional function, compared with nontransplant patients | |
| • Patients who developed GVHD had significantly worse general health perception scores | |
| • Patients without GVHD scored significantly better than the general population in the mental health, role emotion function, and mental component summary scores | |
| • Patients transplanted after the age of 15 years had worse scores in general health and physical component summary scores | |
| Patel et al,51 2018 (Children and adults on regular transfusion with iron chelation therapy) | • HSCT improved overall quality of life in thalassemia major patients |
| • HSCT recipients had a significantly higher score in all domains except for social functioning compared with controls | |
| • The type of transplant and age at which transplant was done had no significant effect on HRQOL | |
| Uygun at al,52 2012 (Thalassemia patients with chronic blood transfusion and iron chelation) | • HRQOL scores were overall better in HSCT recipients compared with controls |
| • No significant difference among 2- to 4-y-old children and adult HSCT recipients when compared with controls | |
| • HSCT recipients (5 to 18 y old) had a significantly better HRQOL score compared with the control, with the 8- to 12-y-old group being the most prominent of the subgroups | |
| • HRQOL scores were not related to gender, welfare status, and time since HSCT | |
| • HSCT recipients (2 to 4 y old) also did not have any significant difference in the individual domains compared with control | |
| • HSCT recipients (5 to 7 y old) had significantly better emotional and physical scores compared with controls | |
| • HSCT recipients (8 to 12 y old) had significantly better physical and school domain scores compared with control | |
| • HSCT recipients (13 to 18 y old) patients had better physical and emotional domain scores | |
| • Among adult patients, only physical domain had better scores when compared with the control | |
| • HSCT recipients perceived that their QOL was unaffected by transplant | |
| • HSCT recipients’ overall health perception was significantly better than the control | |
| • Drug independence and sleep satisfaction were significantly better in adult HSCT recipients compared with control |
| Author, y (control) . | Main results . |
|---|---|
| Caocci et al,45 2006 (No control) | • All HRQOL domains were found to be worse in HSCT recipients with GVHD |
| • No statistically significant difference between GVHD and non-GVHD patients for global QOL | |
| Caocci et al,44 2011 (No control) | • Physical function worsened from baseline to 3 mo post-HSCT, then improved at 6 to 18 mo |
| • Total PedsQL scores were stable from baseline to 3 mo post-HSCT, then improved at 6 and 18 mo | |
| • Social, emotional, and psychosocial scores did not differ significantly following HSCT | |
| • Parents slightly underestimated their children's QOL | |
| Caocci et al,46 2016 (Sibling donors and patients treated with conventional treatments) | • Physical scores were not significantly different in HSCT recipients, compared with their sibling donors, but were overall better than patients with thalassemia on conventional treatment (chronic transfusions and iron chelation therapy) |
| • No significant difference between the groups for mental component summary scores | |
| Cheuk et al,47 2008 (Thalassemia patients with chronic blood transfusion and iron chelation) | • Overall health in HSCT recipients was better than transfusion-dependent patients |
| • HSCT recipients had less fatigue and better relationships | |
| • Physical domain scores were better (higher) in HSCT recipients | |
| • Psychological, social relationships, and environment domains were similar between the 2 groups | |
| • Physical, emotional, social, school, and psychosocial PedsQL domains were not significantly different among the 2 groups | |
| Javanbakht et al,48 2015 (Thalassemia patients with chronic blood transfusion and iron chelation) | • HSCT recipients reported better QOL scores compared with controls |
| • Patients living in rural areas had better EQ-VAS scores | |
| • Patients who underwent HSCT had better score in the physical component scale | |
| • No significant difference between HSCT recipients and controls in self-care, mobility, and usual activities | |
| • HSCT recipients had significantly better pain, discomfort, anxiety, and depression domains | |
| • No significant difference between the groups for EQ-5D-3L index scores | |
| Kelly et al,53 2012 (Patients with acquired disorders including malignancies or severe aplastic anemia) | • No difference in the proportion of patients with HSCT recipients recovering to baseline, compared with the control group |
| • Per child report, majority of HSCT recipients had not recovered emotional function | |
| • More parents reported that their children recovered their general health as compared with the control group | |
| • HSCT recipients physical function improved over 3 months from baseline, which was also better compared with controls | |
| • Three-month scores for emotional function were not statistically different from baseline among groups, per child report | |
| • Parents' report of HRQOL was worst at 45 d posttransplant and returned to baseline at 3 months | |
| • Parents of HSCT recipients scored 18 points higher (better) compared with the control for general health | |
| • Children rated higher for physical, emotional, and role function, compared with their parents | |
| Kisecik Sengul et al,49 2017 (No control) | • Child total scale score and psychosocial health score are slightly and moderately associated with parental Beck's Depression Index score, respectively |
| • No significant association between parent's depression score and child's physical health score | |
| • According to parents’ HRQOL survey, there was no significant association between parental depression and children's total score or physical health score, but there was a significant association with child's psychosocial health score | |
| • Depressed parents scored their children’s HRQOL lower than parents who were not depressed | |
| La Nasa et al,50 2013 (General population and thalassemia patients on chronic blood transfusion and iron chelation) | • HSCT recipients had similar HRQOL profiles compared with the general population in the long term and had a better HRQOL compared with conventionally treated β-thalassemia patients |
| • All other physical and mental health domains had no significant difference | |
| • Patients with comorbidities at the time of survey scored their general health lower than patients without comorbidities | |
| • HSCT recipients scored better physical function, bodily pain, and emotional function, compared with nontransplant patients | |
| • Patients who developed GVHD had significantly worse general health perception scores | |
| • Patients without GVHD scored significantly better than the general population in the mental health, role emotion function, and mental component summary scores | |
| • Patients transplanted after the age of 15 years had worse scores in general health and physical component summary scores | |
| Patel et al,51 2018 (Children and adults on regular transfusion with iron chelation therapy) | • HSCT improved overall quality of life in thalassemia major patients |
| • HSCT recipients had a significantly higher score in all domains except for social functioning compared with controls | |
| • The type of transplant and age at which transplant was done had no significant effect on HRQOL | |
| Uygun at al,52 2012 (Thalassemia patients with chronic blood transfusion and iron chelation) | • HRQOL scores were overall better in HSCT recipients compared with controls |
| • No significant difference among 2- to 4-y-old children and adult HSCT recipients when compared with controls | |
| • HSCT recipients (5 to 18 y old) had a significantly better HRQOL score compared with the control, with the 8- to 12-y-old group being the most prominent of the subgroups | |
| • HRQOL scores were not related to gender, welfare status, and time since HSCT | |
| • HSCT recipients (2 to 4 y old) also did not have any significant difference in the individual domains compared with control | |
| • HSCT recipients (5 to 7 y old) had significantly better emotional and physical scores compared with controls | |
| • HSCT recipients (8 to 12 y old) had significantly better physical and school domain scores compared with control | |
| • HSCT recipients (13 to 18 y old) patients had better physical and emotional domain scores | |
| • Among adult patients, only physical domain had better scores when compared with the control | |
| • HSCT recipients perceived that their QOL was unaffected by transplant | |
| • HSCT recipients’ overall health perception was significantly better than the control | |
| • Drug independence and sleep satisfaction were significantly better in adult HSCT recipients compared with control |