In this cross-sectional study, we compared the quality of life (QOL) in transfusion-dependent thalassemic patients who survived matched sibling hematopoietic SCT (HSCT, n=24) with patients treated conventionally with transfusion and iron chelation (n=74). WHOQOL-BREF(HK) and PedsQL questionnaires were administered to patients aged >18 years and 5–12 years, respectively. Patients aged 12–18 years received both questionnaires. WHOQOL-BREF(HK) revealed post transplant patients rated overall health better than those treated conventionally (score 3.67 vs 3.06, P=0.01). They are less dependent on medical aids (3.87 vs 2.96, P=0.006), having higher activity level (4.00 vs 3.36, P=0.026) and better personal relationships (4.13 vs 3.69, P=0.014). Physical health domain score was better (75.20 vs 63.94, P=0.007). These differences remained significant after adjustment for comorbidities. PedsQL revealed post transplant patients rated better for running (3.53 vs 2.72, P=0.001) and sports (3.20 vs 2.64, P=0.038), even after adjustment for comorbidities, but were less satisfied for school absence to attend hospital (2.53 vs 3.29, P=0.03). Post transplant patients were significantly more likely to consider marriage (100 vs 75.7%, P=0.033), but not childbearing (66.7 vs 51.4%, P=0.28). In conclusion, transplanted thalassemic patients enjoy better QOL, mainly in physical health, compared with conventionally treated patients. This information is important to patients considering HSCT.
The use of regular blood transfusion has improved the survival of patients with thalassemia major. However, regular blood transfusion leads to iron overload that can result in progressive organ damage. Iron-chelation therapy with desferrioxamine has become the standard treatment for most patients.1, 2 However, desferrioxamine has to be administered by s.c. or i.v. infusion for a prolonged period (8–12 h per day) regularly (4–7 times per week), which is cumbersome, difficult to comply with and painful. Deferiprone, an orally administered drug, also has its own adverse effects like gastrointestinal upset and arthropathy. The most recently available orally administered iron-chelating agent, deferasirox, has limited availability due to its high cost although its adverse effects appear fewer and its once daily administration is more convenient. Patients who cannot comply with iron-chelation therapy often develop various complications including cardiomyopathy and endocrinopathies. All these secondary problems may seriously impair the quality of life (QOL) and functional status of the patients. A study in Malaysian children with thalassemia found that they had significantly lower QOL than healthy controls by 10–24% in many aspects.3 Studies in the United Kingdom and Cyprus also found impaired QOL with problems in organization of transfusion, chelation therapy and communication.4 In addition, other studies have found that thalassemic patients are distressed about their disease and treatments and may have many different psychosocial and behavioral problems.5, 6, 7, 8
Allogeneic hematopoietic SCT (HSCT) from an HLA-matched donor remains the only proven curative therapy for transfusion-dependent thalassemia at present. Observations over more than 20 years have confirmed the durability of this cure. Although the overall 3-year survival of Lucarelli class I patients reached 94%, the overall survivals in classes II and III patients were only 80 and 61%, respectively.9 HSCT is also associated with possible long-term morbidity, most notably chronic GVHD (cGVHD) that occurs in 18–33% of patients and can cause damage to any organs.10, 11, 12 Although HSCT survivors with stable engraftment can stop regular transfusion and desferrioxamine injection, they might need to take multiple medications including immunosuppressants with the accompanying risk of infections and adverse drug effects. All these long-term complications might impair the QOL of HSCT survivors. A previous survey involving 31 children who received HSCT, including 1 patient with thalassemia, found that their QOL was significantly lower than healthy children in all domains except school functioning.13 However, there has been only one study dedicated to transfusion-dependent thalassemic patients after HSCT, without comparing with conventionally treated patients.14 Whether post transplant thalassemic patients can eventually enjoy a better QOL than patients receiving conventional therapy is currently uncertain. Therefore, we sought to investigate whether the QOL of transfusion-dependent thalassemic patients after HSCT was better compared to patients treated conventionally with regular transfusion and iron-chelation therapy. As secondary aims, we took the opportunity to evaluate factors that might influence QOL of the two groups of patients, such as comorbidities associated with iron overload, different types of chelation therapies used in conventionally treated patients, Pesaro risk class for transplanted patients, time lapse after HSCT and the need of long-term medications after HSCT.
Materials and methods
Study design and participants
This was a cross-sectional comparative study on the QOL of transfusion-dependent thalassemic patients treated with regular blood transfusion and iron-chelation therapy, or have undergone HSCT in Hong Kong. All patients had diagnosis of thalassemia confirmed by hemoglobin pattern analyses and genotype studies. The severity of thalassemia necessitates regular blood transfusion. The patients were recruited from Queen Mary Hospital (QMH) that is a university-affiliated quaternary referral center in Hong Kong performing HSCT, and Tuen Mun Hospital (TMH) that is a large regional hospital providing regular blood transfusion, iron-chelation therapies and follow-up care for thalassemic patients. Patients with HSCT were recruited from QMH whereas conventionally treated patients were recruited from both QMH and TMH. All patients who were treated in QMH and TMH were invited to participate in the study. QMH was situated in the South of Hong Kong and TMH was situated in the northwest. The two large hospitals receive all patients in their regions, which constitute more than one-quarter of all transfusion-dependent thalassemic patients in Hong Kong. On the other hand, QMH performed about half of all HSCT in Hong Kong and patients from about half of the territory were referred to QMH for HSCT. Therefore, the patient cohort in this study should be fairly representative of all transfusion-dependent thalassemic patients treated with HSCT or conventionally in Hong Kong.
All HSCT used matched siblings as donors. All patients were counseled about the treatment option of HSCT and offered HSCT if they had HLA-matched siblings and gave fully informed consent, regardless of the age or complications from iron overload or Pesaro risk class. Pretransplant conditioning regimens and GVHD prophylaxis followed that described by Lucarelli.9, 15 After discharge, the patients would be followed up once to twice weekly with physical examination and routine blood tests within the first 3–6 months post transplant, then once every 2–4 weeks till 1-year post transplant. Afterward, iron-overloaded patients would receive 4-weekly venesection until serum ferritin decreased to below 1000 ng/ml. The patients would then be followed up once every 2–3 months in the subsequent years. After 5 years, the patients would be followed up once every 6 months usually.
Conventional treatments of transfusion-dependent thalassemic patients consisted of regular 4-weekly transfusion to maintain Hb at about 10–14 g per 100 ml, and iron-chelation therapy with desferrioxamine, deferiprone or a combination of desferrioxamine and deferiprone, or deferasirox alone, depending on the serum ferritin levels and the cardiac iron overload status and the preference of the patient. The transfusion was mostly scheduled during weekends to minimize disruption of work schedules and schooling of the patients.
The patients' data on demographic and clinical characteristics and complications were extracted from clinical records and computerized Clinical Management System (CMS) of the Hong Kong Hospital Authority Server, as well as the BMT database of our Departmental Server.
Quality of life assessments
Two QOL measuring instruments, the WHOQOL-BREF(HK) and the PedsQL questionnaires, were used in the current study. Both questionnaires are self-administered with written instructions. An investigator would provide assistance to the patients and read each item word for word to young children. Patients aged above 18 years were administered the WHOQOL-BREF(HK) questionnaire and patients aged 5–12 years were administered the PedsQL questionnaire. Patients aged 12–18 years were administered both questionnaires.
The WHOQOL-BREF(HK) questionnaire is a simplified instrument based on the original WHOQOL-100 questionnaire. It consists of 24 global questions and 2 national questions validated in the Chinese language for patients above 12 years. It assesses a patient's QOL in four domains: physical health, psychological, social relationships and environment, with 4–6 questions in each domain. All questions are based on the self-evaluated status in the past 2 weeks and are rated on a five-point Likert scale. The raw item score therefore ranges from 1 to 5. This is transformed by multiplying by 4 to give a scale from 4 to 20. The sum of item scores in each domain yields the domain score which is transformed to a scale of 0–100. Higher score represents better self-rated QOL. It is acceptable for a missing score on a question to be replaced by the mean of the rest of the questions in that domain. However, if more than one question in either the physical, psychological or social relationships domains are missing, the domain score for that particular domain cannot be calculated as the domain score would become invalid and unreliable. If more than two questions in the environment domain are missing, the environment domain score cannot be calculated. Since a previous local study found that the question on sexual satisfaction caused some discomfort for the patients and most patients did not answer that question, we have omitted that particular question in the current study. This would cause one missing component in the social relationships domain but the domain score can still be calculated unless more missing values are present.
The PedsQL questionnaire is another instrument validated in the Chinese language for local use in children aged 5–18 years. The questionnaire consists of 23 items encompassing four domains: physical, emotional, social and school. The physical domain contains eight questions whereas the other three domains contain five questions each. Each question is also scored on a five-point Likert scale and transformed to a scale of 0–100. Higher score represents better self-rated QOL. The mean score of the questions in each domain represents the domain score. Apart from the four individual domain scores, a psychosocial summary score is formed by the mean of the emotional, social and school domains. If more than 50% of the individual item scores in a domain are missing, the particular domain score is not calculated. Again missing values are replaced by the mean score of the remaining items in the same domain provided that less than 50% are missing.
In addition to the two standard questionnaires, we also included two questions on self-perception of general health for all patients: (1) do you think that you are healthy or have disease? (a) healthy, (b) disease under control, (c) diseased; (2) what do you think about your current health? (a) very bad, (b) bad, (c) not bad or good, (d) good, (e) very good. For patients above 12 years old, we added two more questions about future marriage and childbearing: (1) will you consider getting married in the future? (a) already married or will consider, (b) will not consider; (2) will you consider having children in the future? (a) already had children or will consider; (b) will not consider.
The mean item scores and the mean domain scores in each questionnaire were calculated separately for the transplant and nontransplant groups of patients. The mean scores of the two groups were compared by independent sample t-tests in univariate analyses. Multiple linear regression was used to adjust for the presence of age and gender and comorbidities including cardiomyopathy, diabetes mellitus, hypothyroidism, hypoparathyroidism, hypogonadism and osteoporosis. The dependent variables were item or domain scores and the independent variables were group status (transplant vs nontransplant) and comorbidities coded as binary variables, that is, presence or absence. A P-value less than 0.05 was considered statistically significant. All statistical analyses were carried out by the SPSS 11.0 software (SPSS Inc., 2003). This study has been approved by the Institutional Review Board of the Hospital Authority of Hong Kong, which complies with the Declaration of Helsinki.
A total of 111 patients were invited to fill in the questionnaires, including 27 patients who had received matched sibling HSCT and 84 patients who had not. A total of 98 patients participated, including 24 patients in the transplant group and 74 patients in the nontransplant group. The response rate was 88.9% in the transplant group and 88.1% in the nontransplant group. All patients had β-thalassemia major except one patient who had Hemoglobin Bart's disease and received HSCT. The median age was 15.2 years (range, 5.3–30.4 years) for the transplant group and 21.0 years (range, 5.0–32.4 years) for the nontransplant group. For the transplant group, the median time interval between HSCT and the administration of the questionnaire was 6.5 years (range, 1.1–13.5 years). The other demographic and clinical characteristics of the two groups are shown in Tables 1 and 2.
QOL by WHOQOL-BREF(HK)
A total of 85 patients aged above 12 years completed the WHOQOL-BREF(HK) questionnaires, including 15 patients in the transplant group and 70 patients in the nontransplant group. The age and gender distribution, as well as scores in all items and domains are shown in Table 3.
There were significant differences between the transplant group and the nontransplant group in several items. Post transplant patients rated their overall health significantly better than patients on transfusion and chelation therapy (score 3.67 vs 3.06, P=0.01). They also considered themselves less dependent on medical substances and medical aids (score 3.87 vs 2.96, P=0.006), having more energy and less fatigue (score 4.00 vs 3.36, P=0.026) and having better personal relationships (score 4.13 vs 3.69, P=0.014). The difference in these scores between the transplant and the nontransplant groups remained significant even after adjustment for age and gender and the presence of other complications including cardiomyopathy, diabetes mellitus, hypothyroidism, hypoparathyroidism, hypogonadism and osteoporosis. Overall score in the physical health domain was also better in the transplant group than in the nontransplant group (score 75.20 vs 63.94, P=0.007), even after adjustment for other complications. However, scores in psychological, social relationships and environment domains were similar between the two groups.
QOL by PedsQL
A total of 40 patients aged below 18 years completed the PedsQL questionnaires, including 15 patients in the transplant group and 25 patients in the nontransplant group. The age and gender distribution, as well as scores in all items and domains are shown in Table 4.
Post transplant patients had significantly higher scores for running (3.53 vs 2.72, P=0.001), and sports or exercise (3.20 vs 2.64, P=0.038) compared with patients on conventional treatment, even after adjustment for age and gender and the presence of complications including cardiomyopathy, diabetes mellitus, hypothyroidism, hypoparathyroidism, hypogonadism and osteoporosis. However, the two groups were similar in the score for walking (3.07 vs 3.12, P=0.88); and post transplant patients were less satisfied that they had more school absence to go to the doctor or the hospital (score 2.53 vs 3.29, P=0.03). The overall scores in the physical, emotional, social, school and psychosocial domains were not significantly different between the transplant and the nontransplant groups.
Perception on general health
Post transplant patients tended to have better perception of their general health compared to conventionally treated patients, which was consistent for the two additional questions (P for trend <0.001 for the first question, P for trend=0.001 for the second questions). Significantly more post transplant patients considered themselves to be healthy (45.8 vs 4.1%, P<0.001), and judged their current health condition as good or very good (75 vs 31.1%, P<0.001).
Marriage and childbearing considerations
There were significantly more post transplant patients who would consider marriage in the future compared to conventionally treated patients (100 vs 75.7%, P=0.033). On the other hand, there was no significant difference in the proportions of patients who would consider having children in the transplant and the nontransplant groups (66.7 vs 51.4%, P=0.28).
The time lapse after transplant was found to correlate significantly with certain aspects of QOL in transplanted patients. The longer the time after transplant, the less was the dependence on medical care (r=0.63, P=0.029) and the more secure and safe the patients felt (r=0.68, P=0.015) as evaluated by the WHOQOL-BREF(HK); and the higher were the scores in the emotional domain (r=0.54, P=0.037) and the psychosocial domain (r=0.52, P=0.049) of the PedsQL.
Within the transplant group, class III patients had significantly lower scores in the psychological domain of the WHOQOL-BREF(HK) than class II patients (56.2 vs 72.6, P=0.042). Patients who required two or more medications rated significantly poorer in physical safety and security (3.50 vs 4.40, P=0.048) compared with patients who required no or just one medication. Patients who required long-term medications also rated significantly poorer in the physical domain of the PedsQL compared to patients who did not require medications (66.1 vs 78.5, P=0.043). These medications included sex hormone or thyroid hormone replacement or calcium supplement. None of the patients required immunosuppressants.
Within the transplant group, patients with hypogonadism had lower scores in the physical health (73.2 vs 88.0, P=0.001) and the social relationships domains (72.2 vs 87.5, P=0.024) of the WHOQOL-BREF(HK), even though all patients with hypogonadism were receiving hormonal replacement therapy at the time of the questionnaire survey. In addition, patients with hypothyroidism also had significantly lower scores in the physical domain (58.3 vs 76.3, P=0.016) of the PedsQL, although all patients with hypothyroidism were receiving thyroid hormone replacement at the time of the questionnaire survey. On the other hand, the presence of hypothyroidism or hypogonadism did not significantly affect the QOL in patients on conventional treatment. Instead, conventionally treated patients with cardiomyopathy had significantly lower scores in the physical health (57.3 vs 65.6, P=0.044), the psychological (54.2 vs 66.2, P=0.016) and the environment domains (57.8 vs 66.9, P=0.036) of the WHOQOL-BREF(HK). Nontransplant patients on deferasirox had higher scores in all domains compared with patients on desferrioxamine or deferiprone (Table 5), and all domain scores of patients on deferasirox were similar to post transplant patients.
Matched sibling HSCT for thalassemia major has resulted in overall survival of 82–90% at 25 years, which is lower compared to the 99% overall survival in compliant patients treated conventionally.16 Although currently HSCT remains the only cure for thalassemia major, it is associated with significant risk of mortality and short-term complications of infection, bleeding, organ failure and GVHD. Late adverse effects are also not uncommon. The long and complex peritransplant process could be extremely traumatic to patients and parents.17 However, the current study demonstrates that the QOL in patients surviving HSCT is not worse than conventionally treated patients. Post transplant patients may even enjoy better QOL in some aspects in the long term, which may compensate for more than the risk of mortality and morbidity in the early post transplant period.
There has been only one report in the literature specifically addressing the QOL of thalassemic patients who received HSCT.14 In that small study of 19, class III adult thalassemic patients who underwent matched unrelated donor HSCT, the QOL after 300 days post transplant was found to be satisfactory by the European Organisation for Research and Treatment of Cancer QLC-C30 questionnaire.14 Very good scores were obtained for the physical, emotional, cognitive, role and social function domains. However, there were no conventionally treated thalassemic patients for comparison in that study. In the current study, we found that post transplant patients rated their overall health significantly better than those on transfusion and iron-chelation therapy. The differences were mainly found in perceived physical health that they considered themselves less dependent on medical substances and medical aids, having more energy, less fatigue and doing better in running, sports and exercise. These are easily understandable as most of our post transplant patients were free from long-term morbidity, thus being able to function relatively normally in their daily activities, work and leisure activities, compared to conventionally treated patients who have a regular period of mild anemia before the scheduled transfusion which might limit their exercise capacity. This is supported by a study which found that approximately 15% of conventionally treated thalassemia major patients had their activities very often stopped due to thalassemia, its complications or desferrioxamine treatment; and 20% had their physical activities limited at least a bit.18
In addition to physical health, post transplant patients also rated better in personal relationships. In patients on conventional treatments, personal relationships may be affected because of limitations in participation of social activities due to limited exercise capacity or burden of regular treatments. The stigmatization of chronic illness might also be lower in post transplant patients who were cured of their disease.
On the other hand, we found that the scores in the psychological domain were similar in the transplant group and the nontransplant group. This might indicate that the conventionally treated patients adapted relatively well psychologically to the burden of chronic illness and treatments. There was also no significant difference in the scores of the environment domain between the transplant and the nontransplant groups. This is not unexpected as patients' satisfaction of the environment does not depend on individual physical health, but largely depends on service provision and social, financial and political situations. In our local setting, patients received all forms of medical treatments with a uniform minimal fee and the government sponsors majority of the expenses. Therefore, under our health-care system, patients should not display systematic difference in the environment domain.
We found one aspect where the post transplant patients rated worse than conventionally treated school-aged patients, that is, school absence to attend medical care. This may be because post transplant patients need to attend regular follow-up in the post transplant period. At the time of the study, all patients had undergone HSCT for more than 1 year and did not require more than once monthly follow-up, which is less frequent than conventionally treated patients who required at least monthly transfusion. In fact, having to go to hospital for blood transfusion and missing school is one of the most important factors affecting the QOL of conventionally treated thalassemic patients.3, 19 However, we found that post transplant patients were even less satisfied in this respect. This might be due to their higher expectation, as they might think that they were cured after HSCT and should not have as much school absence as before transplant; or due to the fact that in our setting, they were required to attend follow-up clinic during office hours rather than receiving blood transfusions during weekends.
Among the 24 post transplant patients, 3 had graft failure and reverted back to the original transfusion-dependent state and received standard transfusion and chelation therapies when the questionnaires were administered. These 3 patients were still included with the remaining 21 transfusion-independent patients in the post transplant group in the current study so that the overall QOL results in this group could fully represent the transplant outcomes. We found that even with a certain degree of transplant failure, the average QOL in post transplant patients was superior to patients on conventional treatment, although the QOL of the three patients with graft failure appeared to resemble that of the nontransplant group.
Among the post transplant patients, we found that patients with hypogonadism rated worse in the social relationships domain. This might result from lower self-esteem and requires further investigations. On the other hand, patients with hypothyroidism rated poorer in the physical domain. As all patients with hypothyroidism had already received thyroxine replacement with normal thyroid function, the reason for the poorer self-rated physical health was not apparent and requires replication and exploration in future studies. In addition, as the number of patients in the transplant group is small, multivariate or subgroup analyses are limited by inadequate statistical power and further studies with larger sample size are needed to clarify the association between hypogonadism or hypothyroidism with QOL in post transplant patients.
Furthermore, we found that some aspects of QOL correlated significantly with the time lapse after transplant. Patients who had been transplanted for a longer period of time felt safer and more secure, considered themselves less dependent on medical care, and rated better in the emotional and the psychosocial domains. These results are encouraging as post transplant patients might enjoy better QOL with the passage of time, as most post transplant complications gradually settle and follow-up visits become less frequent.
We found that conventionally treated patients were less likely to consider marriage in the future compared to post transplant patients. This might indicate that conventionally treated patients are less capable or less willing to develop intimate personal relationship. Their chronic illness and reliance on the health-care system may deter them from starting their own family. However, they are not more likely to reject childbearing compared to post transplant patients. The proportion of conventionally treated patients not considering to have children is similar in the current study and the study by Caro et al. (48.6 vs 40.2%).18 The attitudes and expectations of thalassemic patients toward their own future family certainly warrant further investigations.
The regimen of iron chelation probably has significant influence on the QOL of conventionally treated patients. Arboretti et al.20 reported that 21% of thalassemic patients had great discomfort with the use of desferrioxamine, which was a strong predictor for negative perception on multivariate analysis. Telfer et al.4 reported that nearly half of thalassemic patients disliked desferrioxamine. Other studies also reported that the use of desferrioxamine was a strong impediment of QOL, and effective oral chelation therapy might improve QOL.18, 21, 22, 23, 24, 25 Combination chelation therapy with desferrioxamine and deferiprone might be more acceptable to patients.4 In the current study, we found that patients on deferasirox had significantly better self-rated QOL than patients on other iron-chelation regimen, and was comparable to post transplant patients. This indicates a significant advantage of deferasirox over desferrioxamine or deferiprone, which might stem from its convenient once daily oral dosing with few adverse effects. However, as the comparison was not based on randomization of the treatment arms, the results were subjected to selection bias and confounding by other factors. Future randomized controlled trials are required to verify the superiority of deferasirox in terms of QOL, which may justify its substantially higher cost. A recent cost-effectiveness analysis based on patients' preference found that deferasirox resulted in a gain of 4.5 quality-adjusted life years (QALY) per patient at an additional expected lifetime cost of US$126 018 per patient, and the cost per QALY gained was US$28 255, which might be considered cost effective from a US health-care perspective.26
The current study has several limitations. First, there was no concurrent control group of healthy subjects for comparison. We do not know whether the QOL of post transplant thalassemic patients were comparable to that of healthy people. Further studies are required to clarify this. Second, QOL assessments were carried out at different time points for post transplant patients, who might have different spectrum of complications and morbidities in different post transplant periods. However, all patients were assessed at least 1 year after HSCT, with most if not all of the acute complications resolved. Therefore, the assessments should be reasonably representative of the long-term QOL. Third, this was a cross-sectional study without longitudinal follow-up. Therefore, we could not assess the evolution of QOL in post transplant patients and conventionally treated patients. However, the current study has evaluated post transplant patients at different time points and suggested that longer time lapse after transplant correlated with better QOL in several aspects. Fourth, the comparison groups were not randomized and therefore selection bias or volunteer bias might have occurred, as patients who had undergone HSCT might have been healthier; and we could not ascertain whether the clinical condition and QOL of patients before HSCT is comparable to conventionally treated patients at similar age. Fifth, we have not evaluated the causes of the impaired QOL in conventionally treated patients and the proportional contribution of different potential causes such as transfusion vs chelation, and discomfort vs toxicity from different chelation regimen. Finally, some of the observed difference in QOL between the two groups of patients may be due to perception bias. For example, the QOL scores in post transplant patients may be boosted by a perception that they are disease free. Further studies are needed to address this issue.
In conclusion, patients with transfusion-dependent thalassemia who survive a matched sibling HSCT enjoy better QOL, mainly in the physical health domain, compared to those treated with transfusion and chelation therapy, although this may not apply to all subgroups of patients. This information is helpful to both physicians and patients/parents during their counseling before transplant. They should not rely only on the information related to the risk of mortality and morbidity of HSCT, but also consider the potential improvement in QOL in the future. Therefore, the current study provides further insights for both physicians and patients/parents in weighing the pros and cons of HSCT. However, whether the improvement in QOL applies to unrelated donor transplantation, which might carry higher risk of therapy-related morbidity such as cGVHD, requires further study. Future studies should also consider incorporating the construct of illness intrusiveness because many of the factors identified as stressful for transplant and nontransplant patients entail disease- or treatment-induced interference with important aspects of life; and illness intrusiveness, as measured by the Illness Intrusiveness Ratings Scale boasts strong psychometric properties including sensitivity to change.
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This project was supported by research fund from Hong Kong Children's Thalassemia Foundation.
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Cheuk, D., Mok, A., Lee, A. et al. Quality of life in patients with transfusion-dependent thalassemia after hematopoietic SCT. Bone Marrow Transplant 42, 319–327 (2008). https://doi.org/10.1038/bmt.2008.165
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A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?
Blood Reviews (2018)
Health-related quality of life in pediatric and adolescent patients with transfusion-dependent ß-thalassemia in upper Egypt (single center study)
Health and Quality of Life Outcomes (2018)
The Indian Journal of Pediatrics (2018)
Endocrine complications in patients with transfusion-dependent thalassaemia after haemopoietic stem cell transplantation
Bone Marrow Transplantation (2018)