Long-term health-related quality of life in patients with β-thalassemia after unrelated hematopoietic stem cell transplantation

Mulas, Olga; Caocci, Giovanni; Efficace, Fabio; Piras, E; Targhetta, Clara; Frau, Veronica; Barella, Susanna; Piroddi, Antonio; Orofino, Maria Grazia; Vacca, Adriana; Nasa, Giorgio La

doi:10.1038/s41409-022-01823-2

Cited by 3 publications

(6 citation statements)

References 14 publications

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“…In one study that examined the pediatric beta‐thalassemia patient population in Italy, HSCT was associated with a higher total PedsQL score and emotional functioning, physical functioning, and school functioning domain scores 40 . In this study, there was no significant difference in HRQOL between patients who received sibling versus unrelated HLA‐matched HSCT 36,39,40 . However, another study showed QOL was higher when transplantation occurred from a voluntary donor compared to an HLA‐mismatched related donor 46 …”

Section: Resultsmentioning

confidence: 54%

“…40 In this study, there was no significant difference in HRQOL between patients who received sibling versus unrelated HLA-matched HSCT. 36,39,40 However, another study showed QOL was higher when transplantation occurred from a voluntary donor compared to an HLA-mismatched related donor. 46 When examining the impact of beti-cel gene therapy on patients with TDT enrolled in phase 3 studies who reached transfusion independence, patients reported improvement in HRQOL for physical and mental component summary scores using the SF-36 and the total score on the PedsQL.…”

Section: Curative Therapies In Tdtmentioning

confidence: 50%

“…Of these, 250 met the timeline of publication in the last 5 years. These articles were abstract and full‐text screened, ultimately yielding 31 included studies 18–48 . The results of this literature review are provided in the PRISMA flowchart in Figure 1.…”

Section: Resultsmentioning

confidence: 99%

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Health‐related quality of life with standard and curative therapies in thalassemia: A narrative literature review

Shah,

Badawy

2024

Annals of the New York Academy of Sciences

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Health‐related quality of life (HRQOL) is a patient‐reported outcome that assesses the impact of a disease or illness on different domains of a patient's life. Different general and disease‐specific measures can be used to evaluate HRQOL. This article aimed to summarize the evidence for HRQOL among patients with transfusion‐dependent (TDT) and non‐transfusion‐dependent thalassemia (NTDT). We included HRQOL data related to standard therapy with blood transfusions, iron chelation, and/or luspatercept in TDT and NTDT, as well as curative therapies for TDT, including hematopoietic stem cell transplant (HSCT) and gene therapy. Patients with thalassemia had worse HRQOL scores compared to the general population, and chronic pain was seen to increase in frequency and severity over time with age. NTDT patients reported worse physical health and functioning, mental health, general health, and vitality than TDT patients. However, TDT patients reported worse pain, change in health, and social support than NTDT. Most therapies improved overall HRQOL among thalassemia patients. Deferasirox, an oral iron chelator, was associated with more HRQOL benefits compared to deferoxamine, an intravenous iron chelator. Luspatercept showed clinically meaningful improvement in physical functioning among TDT and NTDT. Furthermore, HSCT and gene therapy were associated with better physical, emotional, and mental domains scores.

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Section: Resultsmentioning

confidence: 54%

Section: Curative Therapies In Tdtmentioning

confidence: 50%

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Health‐related quality of life with standard and curative therapies in thalassemia: A narrative literature review

Shah,

Badawy

2024

Annals of the New York Academy of Sciences

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“…A possible role of splenectomy in improving the allogeneic hematopoietic stem cell transplantation (HSCT) outcome has also been explored. Although faster neutrophil engraftment was observed, this did not produce significantly better overall survival rates after HSCT and did not provide any advantages [ 9 , 15 , 32 ].…”

Section: Discussionmentioning

confidence: 99%

“…A recent review of randomized and quasi-randomized controlled studies was unable to find good-quality evidence about the efficacy of splenectomy for treating β-TM [ 12 ]. Among the parameters used to evaluate the patient’s wellness in a chronic disease such as β-TM, health-related quality of life (HRQoL) plays a key role [ 13 , 14 , 15 ]. HRQoL is generally conceptualized as a multidimensional construct referring to patients’ perceptions of the impact of disease and treatment on their physical, psychological, and social functioning and well-being [ 16 ].…”

Section: Introductionmentioning

confidence: 99%

Long-Term Health-Related Quality of Life and Clinical Outcomes in Patients with β-Thalassemia after Splenectomy

Caocci

Mulas

Barella

et al. 2023

JCM

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Few data are available on the efficacy and safety of splenectomy in patients with transfusion-dependent Beta-Thalassemia Major (β-TM) and on its impact on a patient’s health-related quality of life (HRQoL). We examined the long-term HRQoL of adult patients with β-TM in comparison with those treated with medical therapy by using the Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36). We also evaluated the safety and efficacy of splenectomy. Overall, 114 patients with a median age of 41 years (range 18–62) were enrolled in this cross-sectional study. Twenty-nine patients underwent splenectomy (25.4%) at a median age of 12 years (range 1–32). The median follow-up after splenectomy was 42 years (range 6–55). No statistically significant differences were observed in any of the scales of the SF-36 between splenectomized and not-splenectomized patients. The majority of surgical procedures (96.6%) were approached with open splenectomy. Post-splenectomy complications were reported in eight patients (27.5%): four overwhelming infections, three with pulmonary hypertension, and one with thrombosis. A significantly higher prevalence of cardiovascular comorbidities (58.6 vs. 21.2%, p < 0.001) and diabetes (17.2 vs. 3.5%, p = 0.013) was observed in splenectomized patients. These patients, however, required fewer red blood cell units per month, with only 27.6% of them transfusing more than 1 unit per month, compared with 72.9% of the not-splenectomized group. Overall, our data suggest that physicians should carefully consider splenectomy as a possible treatment option in patients with β-TM.

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Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation

Mulas,

Efficace,

Orofino

et al. 2023

JCM

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Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (β-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 β-TM after HSCT compared to that of patients treated with blood transfusions and iron chelation. The health-related quality of life of 43 β-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, Δ = −15.4, p = 0.009 and Δ = −11.3, p = 0.002, respectively; children’s and parents’ emotional functioning, Δ = −15.2, p = 0.026 and Δ = −15.2, p = 0.045, respectively; child’s and parents’ school functioning, Δ = −25, p = 0.005 and Δ = −22.5, p = 0.011, respectively; total child and parents scores, Δ = −14.5, p = 0.004 and Δ = −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 β-TM is associated with a good overall HRQoL profile. This information further supports physicians when counseling patients and their parents before the HSCT procedure.

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Long-term health-related quality of life in patients with β-thalassemia after unrelated hematopoietic stem cell transplantation

Abstract: Long-term health-related quality of life in patients with β-thalassemia after unrelated hematopoietic stem cell transplantation. Bone marrow transplantation, 57(12), 1833-1836.

Cited by 3 publications

References 14 publications

Health‐related quality of life with standard and curative therapies in thalassemia: A narrative literature review

Health‐related quality of life with standard and curative therapies in thalassemia: A narrative literature review

Long-Term Health-Related Quality of Life and Clinical Outcomes in Patients with β-Thalassemia after Splenectomy

Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation

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