Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation
Abstract
:1. Introduction
2. Material and Methods
2.1. Patients and Clinical Procedures
2.2. Health-Related Quality of Life Evaluation
2.3. Statistical Analysis
3. Results
3.1. Clinical Outcomes
3.2. HRQoL of β-TM Transplanted Patients Compared with Those Conventionally Treated
4. Discussion
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Conventional Therapy, N = 18 | Transplant, N = 25 | p-Value | |
---|---|---|---|
Median age at survey (range) | 10.1 (5–15) | 9.6 (5–15) | 0.6 |
Sex, male, n (%) | 9 (50) | 9 (36) | 0.52 |
Type of transplant, n (%) | |||
Sibling | 15 (60) | ||
Unrelated | 10 (40) | ||
Presence of comorbidities, n (%) | 3 (16.7) | 2 (8) | <0.001 |
CV diseases, n (%) | 1 (5.6) | 1 (4) | 0.78 |
Splenectomy, n (%) | 0 (0) | 2 (8) | <0.001 |
Venesection, n (%) | 0 (0) | 16 (64) | <0.001 |
RBC unit, n (%) | <0.001 | ||
none | 0 (0) | 25 (100) | |
0–3 RBC units | 10 (55.6) | 0 (0) | |
≥4 RBC units | 8 (44.4) | 0 (0) | |
Ferritin level (mcg/L), mean (SD) | 1293.55 (801.5) | 1448.8 (527.9) | <0.001 |
IC, n (%) | 18 (100) | 3 (12) | <0.001 |
Oral IC | 18 (100) | 0 (0) | |
SubC IC | 1 (5.6) | 3 (12) |
PedsQL | Conventional Therapy, Mean (SD) | Transplant, Mean (SD) | Mean Difference | 95% CI | p-Value |
---|---|---|---|---|---|
Child | |||||
Physical functioning | 76.7 (21.1) | 92.1 (9.6) | −15.4 | −26.4; −4.3 | 0.009 |
Emotional functioning | 65.0 (22.6) | 80.2 (20.2) | −15.2 | −28.4; −1.9 | 0.026 |
Social functioning | 86.7 (14.3) | 88.4 (18.9) | −1.7 | −12.4; 8.9 | 0.745 |
School functioning | 59.4 (29.7) | 84.4 (20.6) | −25.0 | −41.6; −8.3 | 0.005 |
Total score | 72.6 (17.9) | 87.4 (12.9) | −14.5 | −23.9; −4.9 | 0.004 |
Parents | |||||
Physical functioning | 82.6 (13.5) | 93.9 (8.5) | −11.3 | −18.2; −4.2 | 0.002 |
Emotional functioning | 65.6 (25.0) | 80.8 (21.2) | −15.2 | −30.0; −0.3 | 0.045 |
Social functioning | 85.6 (17.5) | 90.4 (17.7) | −4.8 | −16.3; 6.7 | 0.406 |
School functioning | 63.8 (29.1) | 86.3 (17.8) | −22.5 | −39.3; −5.6 | 0.011 |
Total score | 75.5 (15.6) | 88.6 (12.2) | −13.2 | −22.0; −4.2 | 0.005 |
PedsQL | HSCT Sibling, Mean (SD) | HSCT Unrelated, Mean (SD) | Mean Difference | 95% CI | p-Value |
---|---|---|---|---|---|
Child | |||||
Physical functioning | 93.7 (9.1) | 89.6 (10.2) | +4.0 | −4.0; 12.1 | 0.310 |
Emotional functioning | 83.0 (17.4) | 76.0 (24.2) | +7.0 | −10.1; 24.1 | 0.408 |
Social functioning | 93.3 (11.1) | 81.0 (25.5) | +12.3 | −6.5; 31.1 | 0.178 |
School functioning | 91.3 (10.6) | 74.0 (27.4) | +17.3 | −2.7; 37.4 | 0.084 |
Total score | 90.7 (8.5) | 81.4 (16.5) | +9.3 | −2.9; 21.7 | 0.124 |
Parents | |||||
Physical functioning | 95.2 (8.3) | 91.6 (8.8) | +3.5 | −3.91; 10.9 | 0.335 |
Emotional functioning | 85.3 (16.8) | 73.3 (26.4) | +12.0 | −6.2; 30.2 | 0.186 |
Social functioning | 92.3 (16.1) | 87.2 (20.7) | +5.1 | −10.5; 20.8 | 0.507 |
School functioning | 91.0 (10.7) | 78.3 (24.4) | +12.6 | −2.2; 27.5 | 0.092 |
Total score | 91.5 (9.5) | 83.8 (15.1) | +7.7 | −2.7; 18.1 | 0.139 |
PedsQL | Child, Mean (SD) | Parents, Mean (SD) | Correlation Coefficient | p-Value |
---|---|---|---|---|
Physical functioning | 85.6 (17) | 89.3 (12.0) | 0.81 | <0.01 |
Emotional functioning | 73.8 (22.3) | 74.7 (23.7) | 0.95 | <0.01 |
Social functioning | 87.6 (16.9) | 88.5 (17.5) | 0.83 | <0.01 |
School functioning | 73.9 (27.4) | 77.2 (25.2) | 0.93 | <0.01 |
Total score | 80.9 (16.5) | 83.3 (19.94) | 0.94 | <0.01 |
Author | Age of HSCT Cohort (Median, Range in Years) | Patients and Parents (No.) | Control Cohort of Conventional Treated Patients (No.) | Years of Follow Up from HSCT (Median, Range) | HRQoL Survey | Principal Findings |
---|---|---|---|---|---|---|
Cheuk et al., 2008 [20] | 15.2 (5.3–18) | 15/not reported | 25 | 6.5 (1.1–13.5) | PedsQL | Emotional, social, and school domains did not differ; physical domain similar, but better scores in running and exercise in transplanted patients. |
Caocci et al., 2011 [21] | 10 (5–17) | 28/28 | longitudinal study | 18 months | PedsQL | Physical function improved from 3 to 18 months after HSCT; parents underestimated their children’s HRQoL. |
Uygun et al., 2012 [22] | 11.6 (3.5–18) | 40/40 | 28 | 4.4 (2–12) | PedsQL | Better emotional domain in 5–7-year-old and physical/school domains in 8–12-year-old transplanted patients; strong correlation between children’s and parents’ HRQoL evaluations. |
Patel et al., 2017 [19] | 10 (5–18) | 40/40 | 60 | 5 (2–10) | PedsQL | Transplanted patients reported significantly higher scores in all domains except for social functioning. |
This report | 9.6 (5–15) | 25/25 | 18 | 5 (1–13) | PedsQL | Transplanted patients reported significantly higher score in all domains except for social functioning; strong correlation between children’s and parents’ HRQoL evaluation. |
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Mulas, O.; Efficace, F.; Orofino, M.G.; Piroddi, A.; Piras, E.; Vacca, A.; Barella, S.; Costa, A.; Giesinger, J.M.; La Nasa, G.; et al. Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation. J. Clin. Med. 2023, 12, 6047. https://doi.org/10.3390/jcm12186047
Mulas O, Efficace F, Orofino MG, Piroddi A, Piras E, Vacca A, Barella S, Costa A, Giesinger JM, La Nasa G, et al. Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation. Journal of Clinical Medicine. 2023; 12(18):6047. https://doi.org/10.3390/jcm12186047
Chicago/Turabian StyleMulas, Olga, Fabio Efficace, Maria Grazia Orofino, Antonio Piroddi, Eugenia Piras, Adriana Vacca, Susanna Barella, Alessandro Costa, Johannes M. Giesinger, Giorgio La Nasa, and et al. 2023. "Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation" Journal of Clinical Medicine 12, no. 18: 6047. https://doi.org/10.3390/jcm12186047
APA StyleMulas, O., Efficace, F., Orofino, M. G., Piroddi, A., Piras, E., Vacca, A., Barella, S., Costa, A., Giesinger, J. M., La Nasa, G., & Caocci, G. (2023). Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation. Journal of Clinical Medicine, 12(18), 6047. https://doi.org/10.3390/jcm12186047