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  • 1
    Online-Ressource
    Online-Ressource
    Wiley ; 2023
    In:  British Journal of Haematology Vol. 201, No. 2 ( 2023-04), p. 199-214
    In: British Journal of Haematology, Wiley, Vol. 201, No. 2 ( 2023-04), p. 199-214
    Kurzfassung: The thalassaemias are a group of genetic disorders of haemoglobin which are endemic in the tropics but are now found worldwide due to migration. Basic standard of care therapy includes regular transfusions to maintain a haemoglobin level of around 10 g/dL, together with iron chelation therapy to prevent iron overload. Novel therapies, bone marrow transplantation, and gene therapy are treatment options that are unavailable in many countries with stressed economies. This Wider Perspectives article presents the strategies for management of an adolescent refugee patient with beta thalassaemia, as it would be performed by expert haematologists in six countries: Italy, Lebanon, Oman, the Sudan, Thailand and the United States. The experienced clinicians in each country have adapted their practice according to the resources available, which vary greatly. Even in the current modern era, providing adequate transfusions and chelation is problematic in many countries. On the other hand, ensuring adherence to therapy, particularly during adolescence, is a similar challenge seen in all countries. The concluding section highlights the disparities in available therapies and puts the role of novel therapies into a societal context.
    Materialart: Online-Ressource
    ISSN: 0007-1048 , 1365-2141
    URL: Issue
    RVK:
    Sprache: Englisch
    Verlag: Wiley
    Publikationsdatum: 2023
    ZDB Id: 1475751-5
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 2
    In: Journal of Blood Medicine, Informa UK Limited, Vol. Volume 12 ( 2021-10), p. 875-881
    Materialart: Online-Ressource
    ISSN: 1179-2736
    Sprache: Englisch
    Verlag: Informa UK Limited
    Publikationsdatum: 2021
    ZDB Id: 2587464-0
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 3
    In: Blood, American Society of Hematology, Vol. 130, No. Suppl_1 ( 2017-12-07), p. 952-952
    Kurzfassung: Background Thalassemia syndromes are the most common hereditary hemolytic anemia worldwide. Since 2013, Thalassemia International Federation (TIF) has launched new standard clinical practice guidelines (CPG) for non-transfusion dependent thalassemia (NTDT) and transfusion dependent thalassemia (TDT). Based on these guidelines, several measures should be routinely implemented such as monitoring and surveillance of thalassemia related complications in order to early detect such complications for a proper clinical management. At present, there is no data in Thailand to show that how physicians are treating thalassemia patients especially on early detection of all complications and whether their real life practice has changed to follow those of both guidelines. Objectives To evaluate the performance of routine care, in terms of surveillance for thalassemia related complications and their prevalence in adult thalassemia patients during 2 periods; before and after 2 CPG were published. Methods In this retrospective study, we analyzed data from 3,233 adult thalassemia patients who were diagnosed and treated at our clinics at Department of Medicine, Siriraj hospital during 1994-2017. We divided them into 2 groups; those who have been treated and followed-up for 3 consecutive years during 2012-2014 and 2015-2017 (a period before and after the implement of CPG, respectively). Clinical data and laboratory results were collected. Complications were recorded including iron overload (IOL) (ferritin), transaminitis (ALT & gt;3x upper normal limit), diabetes mellitus (DM) or impaired fasting glucose (IFG)(fasting blood sugar), hypothyroidism (thyroid function test), low morning cortisol, vitamin D abnormality, viral infection (hepatitis B and C), osteopenia/osteoporosis (bone mineral density, BMD) and gallstones (ultrasonography or CT scan). To compare the performance of routine care from different treating physician groups, patients were categorized into 3 groups; those who attended thalassemia clinic (Thal) treated by mainly staffs and residents in hematology, those in private hematology clinic (Private) treated by mainly attending hematology staffs and residents in hematology, and those in internal medicine clinic (Non-hem) treated by internists and GP. Prevalence and surveillance rates for each complication between groups were analyzed by Independent t test (Chi-square or Fisher's exact test). This study was approved by local ethical committee. Results Total available 459 NTDT and 65 TDT adult patients were studied. Baseline characteristics were shown in Table 1. Three most common complications were osteopenia/osteoporosis (69.8%), gallstones (67.6%) and abnormal vitamin D level (67.6%). IOL is a complication that has been widely evaluated in all treatment groups (93.1%) followed by evaluation of liver function test (82.3%). However the rate of evaluation for other complications were significantly reduced and & lt;25% of patients were evaluated in several complications. This result suggests that the prevalence of thalassemia related complications might be underestimate the true prevalence and early detection of thalassemia related complications is still lacking. To test whether the implement of CPG has any impact on real-life clinical practice, we found that the rate of complication surveillance has increased significantly for several endocrine complications (DM/IFG, hypothyroid, adrenal insufficiency and low vitamin D) only in the group of patients treated at thalassemia clinic but not in others (Figure1). This suggested that CPG have yet to be implemented by those physicians and further endorsement is highly required. Conclusion This study was the first study that evaluated the real-world practical management of thalassemia patient in terms of complication surveillance. In our adult thalassemia population, thalassemia related complications were not uncommon and some occurred early in adulthood. Surveillance rates of these complications were low in all clinics. After implement of thalassemia CPG, the surveillance rates for complications were increased only in patients treated at thalassemia clinic but not in other two clinics. A two different standard of clinical practice even within the same tertiary care hospital such as Siriraj hospital has called for an immediate policy change to improve and standardize a care for thalassemia patients in Thailand. Disclosures No relevant conflicts of interest to declare.
    Materialart: Online-Ressource
    ISSN: 0006-4971 , 1528-0020
    RVK:
    RVK:
    Sprache: Englisch
    Verlag: American Society of Hematology
    Publikationsdatum: 2017
    ZDB Id: 1468538-3
    ZDB Id: 80069-7
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 4
    In: Health Science Reports, Wiley, Vol. 3, No. 1 ( 2020-03)
    Kurzfassung: Diagnosis of thrombotic microangiopathy (TMA) relies on microscopic schistocyte determination by an experienced microscopist. In addition, schistocytes can be found in non‐TMA–related disorders such as thalassaemia. We aimed to compare the accuracy of the automated haematology analyser Sysmex XN‐3000 for schistocyte detection, to that of the microscopy approach, in patients suspected of having schistocytosis. Methods Consecutive blood samples were collected between April 2016 and March 2017 at Siriraj Hospital, Mahidol University, Bangkok, Thailand. Specimens were collected from adults with suspected TMA or with thalassaemia trait and/or disease. All blood samples were examined by both microscopy and the analyser. Samples were considered to be positive for schistocytes (ie, schistocytosis) if they had a schistocyte count ≥1% by microscopy. The analyser's ability to determine schistocytosis was assessed by receiver operating characteristic (ROC) curve. Sensitivity, specificity, positive (PPV), and negative predictive value (NPV) of an appropriate cut‐off point were calculated, with manual microscopy as the standard. Quantitative agreement in schistocyte counts between the two approaches was assessed using 95% limits of agreement, Bland‐Altman plots, intraclass correlation coefficient, and concordance correlation coefficient. Results Ninety‐seven blood samples (62 suspected TMA and 35 thalassaemia) were collected. ROC curve analysis of the analyser for determining schistocytosis showed an area under the curve of 0.803 (95% confidence interval, 0.689‐0.917, P 〈 0.001). A cut‐off point of 0.6% yielded 86.1% sensitivity, 77.8% specificity, 94.4% PPV, and 56.0% NPV. The automated schistocyte count did not quantitatively agree with schistocyte counts by microscopy, neither in all blood specimens (mean of difference: −1.09; 95% limits of agreement, −11.9 to 9.7) nor in the subgroups (TMA, −0.88; 95% limits of agreement, −6.60 to 4.84; thalassaemia, −2.4; 95% limits of agreement, −14.10 to 9.30). The differences in the estimation of fragmented red blood cells between the methods tended to increase at higher schistocyte counts. Conclusion Sysmex XN‐3000 can be used for qualitative measurement of schistocytosis, but should not be used as a quantitative tool for schistocyte counting. Improvements are needed before this analyser's schistocyte detection feature can be recommended for use in clinical practice.
    Materialart: Online-Ressource
    ISSN: 2398-8835 , 2398-8835
    URL: Issue
    Sprache: Englisch
    Verlag: Wiley
    Publikationsdatum: 2020
    ZDB Id: 2927182-4
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 5
    In: Hematology, Informa UK Limited, Vol. 28, No. 1 ( 2023-12-31)
    Materialart: Online-Ressource
    ISSN: 1607-8454
    Sprache: Englisch
    Verlag: Informa UK Limited
    Publikationsdatum: 2023
    ZDB Id: 2035573-7
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 6
    In: Scientific Reports, Springer Science and Business Media LLC, Vol. 11, No. 1 ( 2021-06-24)
    Kurzfassung: Based on Thalassemia International Federation clinical practice guidelines (CPG) for non-transfusion dependent and transfusion dependent thalassemia, several measures should be routinely implemented such as monitoring and surveillance of thalassemia related complications for early detection and proper clinical management. To evaluate the prevalence and the performance of routine surveillance for thalassemia related complications during 2 periods; before and after published CPGs (2012–2014 vs 2015–2017), data from 524 adult thalassemia patients attended at Siriraj hospital were compared among different treating physician groups; thalassemia, private hematology, and internal medicine clinics. Three most common complications were osteopenia/osteoporosis (69.8%), gallstones (67.6%) and abnormal vitamin D level (67.6%). Iron overload has been widely evaluated (93.1%) followed by liver function test (82.3%). However, the rate of evaluation for other complications were significantly reduced and  〈  25% of patients were evaluated in several complications. Comparing among clinics, the surveillance rate has increased significantly for several endocrine complications only in patients treated at thalassemia clinic but not in others. This study was the first study that evaluated real-world practical management of thalassemia patient in terms of complication surveillance. This different clinical practice has called for an immediate policy change to improve and standardize a care for thalassemia patients in Thailand.
    Materialart: Online-Ressource
    ISSN: 2045-2322
    Sprache: Englisch
    Verlag: Springer Science and Business Media LLC
    Publikationsdatum: 2021
    ZDB Id: 2615211-3
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 7
    In: Transfusion, Wiley, Vol. 62, No. 10 ( 2022-10), p. 2039-2047
    Kurzfassung: Thalassemia is a common genetic disease in Southeast Asia. Red blood cell (RBC) transfusion is an essential treatment for severe forms of thalassemia. We performed a study to demonstrate RBC alloimmunization and other transfusion‐related complications in patients with transfusion‐dependent thalassemia (TDT). Study Design and Methods A multi‐center web‐based registry of TDT was conducted in eight medical centers across Thailand. Thalassemia information, transfusion therapy, and transfusion‐related complications were collected. Factors associated with each complication were demonstrated using the logistic regression analysis. Results Of 1000 patients recruited for the study, 449 were males (44.9%). The mean age was 23.9 ± 15.4 years. The majority of patients, 738 (73.8%) had hemoglobin E/beta‐thalassemia. In the study, 421 transfusion‐related complications were reported from 357 patients (35.7%). Alloimmunization was the most common complication which was found in 156 patients (15.6%) with 284 positive antibody tests. The most frequent antibodies against RBC were anti‐E (80/284, 28.2%) followed by anti‐Mi a (45/284, 15.8%) and anti‐c (32/284, 11.3%). Age ≥3 years at initial blood transfusion, splenomegaly, higher frequencies, and volumes of transfusion were significant factors associated with alloimmunization. None of the patients had to terminate blood transfusion due to multiple alloantibodies. Other commonly seen complications were allergic reactions (130, 13.0%), autoimmune hemolytic anemia (70, 7.0%) and febrile non‐hemolytic transfusion reaction (54, 5.4%). Conclusions Transfusion‐related complications, especially alloimmunization, were common among Thai patients with TDT. Extended RBC antigen‐matching for the Rh system and Mi a should be implemented to prevent the development of alloantibodies in multi‐transfused patients.
    Materialart: Online-Ressource
    ISSN: 0041-1132 , 1537-2995
    URL: Issue
    Sprache: Englisch
    Verlag: Wiley
    Publikationsdatum: 2022
    ZDB Id: 2018415-3
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 8
    In: Hematology, Informa UK Limited, Vol. 24, No. 1 ( 2019-01-01), p. 720-726
    Materialart: Online-Ressource
    ISSN: 1607-8454
    Sprache: Englisch
    Verlag: Informa UK Limited
    Publikationsdatum: 2019
    ZDB Id: 2035573-7
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 9
    In: Pediatric Blood & Cancer, Wiley, Vol. 70, No. 10 ( 2023-10)
    Kurzfassung: Management of transfusion‐dependent thalassemia (TDT) can be challenging due to numerous potential disease‐related complications and comorbidities in particular age groups. The objective of this study was to report thalassemia‐related complications and risk factors in pediatric, adolescent, and young adult patients with TDT. Methods A multicenter web‐based registry was conducted in patients with TDT aged 25 years and younger from eight university hospitals covering all parts of Thailand. Factors significantly associated with each complication were analyzed by logistic regression methods. Results Of 605 patients, 267 thalassemia‐related complications were reported from 231 pediatric, adolescent, and young adult patients with TDT patients (38.2%). The most common complications were infections, followed by cholelithiasis and growth failure. Splenectomy and elevated pre‐transfusion hemoglobin were statistically significant risk factors for infections (adjusted odds ratio [AOR] = 2.3, 95% confidence interval [CI] : 1.2–4.5, p ‐value = .01 and AOR = 1.5, 95% CI: 1.2–1.7, p ‐value  〈  .005, respectively). There were two statistically significant risk factors conferred endocrinopathies, including older age (AOR = 1.06, 95% CI: 1.01–1.1, p ‐value = .01) and being male (AOR = 2.4, 95% CI: 1.4–4.0, p ‐value = .002). Conclusion Nearly 40% of the patients in this cohort had thalassemia‐related complications. Periodic surveillance and optimal care for respective complications may minimize comorbidities in pediatric, adolescent, and young adult patients with TDT.
    Materialart: Online-Ressource
    ISSN: 1545-5009 , 1545-5017
    URL: Issue
    Sprache: Englisch
    Verlag: Wiley
    Publikationsdatum: 2023
    ZDB Id: 2130978-4
    Standort Signatur Einschränkungen Verfügbarkeit
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  • 10
    Online-Ressource
    Online-Ressource
    Springer Science and Business Media LLC ; 2022
    In:  Annals of Hematology Vol. 101, No. 10 ( 2022-10), p. 2149-2157
    In: Annals of Hematology, Springer Science and Business Media LLC, Vol. 101, No. 10 ( 2022-10), p. 2149-2157
    Kurzfassung: In heterozygous females, X-inactivation causes a change in glucose-6-phosphate dehydrogenase (G6PD) activity from normal to deficient. Most G6PD screening tests are used to accurately diagnose hemizygous males, but they are less reliable for diagnosing heterozygous females. This study established flow cytometric cut-off values for screening of G6PD deficiency in hemizygous males and heterozygous or homozygous females. We studied 205 (125 females, 80 males) leftover blood samples from quantitative methemoglobin reduction (MR) screening. G6PD gene mutations determined by multiplex amplification refractory mutation system-polymerase chain reaction and direct DNA sequencing were used as the gold standard reference. Accuracy of the test, including the sensitivity, specificity, and positive and negative predictive values, was analyzed using MedCalc software. The optimal cut-off values for classification of %red blood cells with normal G6PD activity or %bright cells into homozygous normal, heterozygous, and homozygous deficiency in females were 85.4–100%, 6.3–85.3%, and 0–6.2%, respectively (sensitivity 93.2%, specificity 100%). The cut-offs for classification into hemizygous normal and hemizygous deficiency in males were 76.5–100% and 0–76.4%, respectively (sensitivity 100%, specificity 96.5%). Flow cytometry can be used to differentiate heterozygous females with intermediate phenotype from homozygous females, but cannot distinguish between heterozygous females with extreme phenotype and homozygous females. By flow cytometry, heterozygous and homozygous deficiency was detected in 29.6% and 3.2% of females, respectively. Among males, hemizygous deficiency was found in 31.3%. Flow cytometry can be used to screen patients with G6PD deficiency, and reliably and efficiently identify heterozygous and homozygous females, and hemizygous males based on cellular G6PD activity.
    Materialart: Online-Ressource
    ISSN: 0939-5555 , 1432-0584
    Sprache: Englisch
    Verlag: Springer Science and Business Media LLC
    Publikationsdatum: 2022
    ZDB Id: 1458429-3
    Standort Signatur Einschränkungen Verfügbarkeit
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