PMID- 37762987
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20231003
IS  - 2077-0383 (Print)
IS  - 2077-0383 (Electronic)
IS  - 2077-0383 (Linking)
VI  - 12
IP  - 18
DP  - 2023 Sep 19
TI  - Health-Related Quality-of-Life Profile of Pediatric Patients with beta Thalassemia 
      after Hematopoietic Stem Cell Transplantation.
LID - 10.3390/jcm12186047 [doi]
LID - 6047
AB  - Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative 
      treatment in pediatric patients with beta thalassemia major (beta-TM). However, 
      little data are available regarding patients and their parents' health-related 
      quality of life (HRQoL) after the procedure. As such, we investigated the HRQoL 
      of pediatric patients with beta-TM after HSCT compared to that of patients treated 
      with blood transfusions and iron chelation. The health-related quality of life of 
      43 beta-TM pediatric patients and 43 parents were evaluated using the Pediatric 
      Quality of Life Inventory (PedsQL). A total of 25 patients underwent HSCT: 15 
      from a sibling and 10 from an HLA-matched donor. The median follow-up time from 
      HSCT was 5 years (range 1-13 years). The mean ages at the survey were 10.1 years 
      (range 5-15) and 9.6 years (range 5-15) for transfused and transplanted patients, 
      respectively. A significant reduction in HRQoL was reported in the group of 
      transfused patients compared with that of patients transplanted in the following 
      PedsQL domains: children's and parents' physical functions, Delta = -15.4, p = 0.009 
      and Delta = -11.3, p = 0.002, respectively; children's and parents' emotional 
      functioning, Delta = -15.2, p = 0.026 and Delta = -15.2, p = 0.045, respectively; child's 
      and parents' school functioning, Delta = -25, p = 0.005 and Delta = -22.5, p = 0.011, 
      respectively; total child and parents scores, Delta = -14.5, p = 0.004 and Delta = -13.2, 
      p = 0.005, respectively. The results of a multivariable analysis showed that the 
      HSCT procedure was significantly associated with a higher total child PedsQL 
      score (adjusted mean difference = 15.3, p = 0.001) and a higher total parent 
      PedsQL score (adjusted mean difference = 14.1, p = 0.006). We found no 
      significant difference in the HRQoL measured after sibling or unrelated human 
      leukocyte antigen (HLA)-matched HSCT. Finally, a significant positive correlation 
      across all the PedsQL domains was found between the scores reported by the 
      children and those reported by their parents. In conclusion, our study shows that 
      HSCT in pediatric patients with beta-TM is associated with a good overall HRQoL 
      profile. This information further supports physicians when counseling patients 
      and their parents before the HSCT procedure.
FAU - Mulas, Olga
AU  - Mulas O
AD  - Hematology Unit, Businco Hospital, Department of Medical Sciences and Public 
      Health, University of Cagliari, 09124 Cagliari, Italy.
FAU - Efficace, Fabio
AU  - Efficace F
AD  - Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases 
      (GIMEMA) Data Center, 00161 Rome, Italy.
FAU - Orofino, Maria Grazia
AU  - Orofino MG
AD  - Bone Marrow Transplant Center, Pediatric Hospital "Microcitemico A. Cao", 09121 
      Cagliari, Italy.
FAU - Piroddi, Antonio
AU  - Piroddi A
AD  - Bone Marrow Transplant Center, Pediatric Hospital "Microcitemico A. Cao", 09121 
      Cagliari, Italy.
FAU - Piras, Eugenia
AU  - Piras E
AD  - Hematology Unit, Businco Hospital, Department of Medical Sciences and Public 
      Health, University of Cagliari, 09124 Cagliari, Italy.
FAU - Vacca, Adriana
AU  - Vacca A
AD  - Hematology Unit, Businco Hospital, Department of Medical Sciences and Public 
      Health, University of Cagliari, 09124 Cagliari, Italy.
FAU - Barella, Susanna
AU  - Barella S
AD  - Pediatric Clinic, Thalassemia and Rare Diseases, Pediatric Hospital 
      "Microcitemico A. Cao", 09121 Cagliari, Italy.
FAU - Costa, Alessandro
AU  - Costa A
AUID- ORCID: 0000-0002-7726-2936
AD  - Hematology Unit, Businco Hospital, Department of Medical Sciences and Public 
      Health, University of Cagliari, 09124 Cagliari, Italy.
FAU - Giesinger, Johannes M
AU  - Giesinger JM
AD  - University Hospital of Psychiatry II, Medical University of Innsbruck, 6020 
      Innsbruck, Austria.
FAU - La Nasa, Giorgio
AU  - La Nasa G
AD  - Hematology Unit, Businco Hospital, Department of Medical Sciences and Public 
      Health, University of Cagliari, 09124 Cagliari, Italy.
FAU - Caocci, Giovanni
AU  - Caocci G
AUID- ORCID: 0000-0002-6585-5187
AD  - Hematology Unit, Businco Hospital, Department of Medical Sciences and Public 
      Health, University of Cagliari, 09124 Cagliari, Italy.
LA  - eng
PT  - Journal Article
DEP - 20230919
PL  - Switzerland
TA  - J Clin Med
JT  - Journal of clinical medicine
JID - 101606588
PMC - PMC10532003
OTO - NOTNLM
OT  - bone marrow transplantation
OT  - health-related quality of life
OT  - thalassemia
COIS- All authors have no conflict of interest to disclose regarding this study.
EDAT- 2023/09/28 06:42
MHDA- 2023/09/28 06:43
PMCR- 2023/09/19
CRDT- 2023/09/28 01:23
PHST- 2023/08/03 00:00 [received]
PHST- 2023/08/29 00:00 [revised]
PHST- 2023/09/06 00:00 [accepted]
PHST- 2023/09/28 06:43 [medline]
PHST- 2023/09/28 06:42 [pubmed]
PHST- 2023/09/28 01:23 [entrez]
PHST- 2023/09/19 00:00 [pmc-release]
AID - jcm12186047 [pii]
AID - jcm-12-06047 [pii]
AID - 10.3390/jcm12186047 [doi]
PST - epublish
SO  - J Clin Med. 2023 Sep 19;12(18):6047. doi: 10.3390/jcm12186047.