Osteoporosis is a prominent cause of morbidity in patients with thalassaemia major (TM) with a complex pathophysiology. Patients with TM and osteoporosis have elevated markers of bone resorption. This increased osteoclast activity seems to be at least partially due to an imbalance in the receptor–activator of nuclear factor-kappa B ligand (RANKL)/osteoprotegerin (OPG) system, which is of great importance for the regulation of osteoclast differentiation and function. Denosumab is a fully human monoclonal antibody that binds to RANKL and thereby inhibits the activation of osteoclasts by RANKL. By blocking RANKL, denosumab inhibits osteoclast formation, function and survival, thereby decreasing bone resorption and increasing bone mass in postmenopausal women and patients with thalassaemia-induced osteoporosis.
{"title":"Osteoporosis in thalassaemia","authors":"E. Voskaridou, M. Dimopoulou, E. Terpos","doi":"10.4081/THAL.2018.7487","DOIUrl":"https://doi.org/10.4081/THAL.2018.7487","url":null,"abstract":"Osteoporosis is a prominent cause of morbidity in patients with thalassaemia major (TM) with a complex pathophysiology. Patients with TM and osteoporosis have elevated markers of bone resorption. This increased osteoclast activity seems to be at least partially due to an imbalance in the receptor–activator of nuclear factor-kappa B ligand (RANKL)/osteoprotegerin (OPG) system, which is of great importance for the regulation of osteoclast differentiation and function. Denosumab is a fully human monoclonal antibody that binds to RANKL and thereby inhibits the activation of osteoclasts by RANKL. By blocking RANKL, denosumab inhibits osteoclast formation, function and survival, thereby decreasing bone resorption and increasing bone mass in postmenopausal women and patients with thalassaemia-induced osteoporosis.","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/THAL.2018.7487","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48485115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Introducing the first mobile app designed specifically for the Thalassemia community; Thalime, your personalized private community. Thalime is a free app that connects patients and caregivers of Thalassemia to others who know what you’re going through. Learn about your condition from a trusted source. Improve your well-being with health-tracking tools. Get support from others just like you. With personalized disease management tools designed to make life easier every day, Thalime is your allin-one health resource that empowers you to be in control of your health. Build your private peer community to learn, share and receive support. Follow programs and set goals with our personalized recommendations and virtual coaching. Track your progress with our visual health tracker for blood transfusions and medication tracker. Additional health tracker tools allow you to monitor and share your mood, energy, pain and more.
{"title":"Thalime: A mobile app designed just for patients and their families","authors":"H. McMicking","doi":"10.4081/thal.2018.7496","DOIUrl":"https://doi.org/10.4081/thal.2018.7496","url":null,"abstract":"Introducing the first mobile app designed specifically for the Thalassemia community; Thalime, your personalized private community. Thalime is a free app that connects patients and caregivers of Thalassemia to others who know what you’re going through. Learn about your condition from a trusted source. Improve your well-being with health-tracking tools. Get support from others just like you. With personalized disease management tools designed to make life easier every day, Thalime is your allin-one health resource that empowers you to be in control of your health. Build your private peer community to learn, share and receive support. Follow programs and set goals with our personalized recommendations and virtual coaching. Track your progress with our visual health tracker for blood transfusions and medication tracker. Additional health tracker tools allow you to monitor and share your mood, energy, pain and more.","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/thal.2018.7496","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48344138","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Renal complications in thalassemia","authors":"Joseph Sleiman, A. Tarhini, A. Taher","doi":"10.4081/THAL.2018.7481","DOIUrl":"https://doi.org/10.4081/THAL.2018.7481","url":null,"abstract":"","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/THAL.2018.7481","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48328546","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Heart disease in patients with haemoglobinopathies","authors":"D. Farmakis, George Papingiotis","doi":"10.4081/THAL.2018.7480","DOIUrl":"https://doi.org/10.4081/THAL.2018.7480","url":null,"abstract":"","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/THAL.2018.7480","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48143577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Knowledge and autonomy are key aspects of informed choice; it is important to define what is important for participants to understand, when accepting or declining screening and for individuals to understand that screening is optional and their own personal choice There are no agreed thresholds or minimum standards for the knowledge an individual is required to have to make an ‘informed’ choice. It is time that minimum agreed standards are developed for practitioners who provide genetic information. There is no standard for evaluating good knowledge or informed choice in population reproductive genetic screening, however measuring people’s choices is a good indicator of informed choice. Informed choice in a multicultural world will be explored and an overview of the different levels of informed choice, practiced in the pathway from genetic screening to identifying at risk couples discussed.
{"title":"Informed choice in a multicultural world","authors":"M. Petrou","doi":"10.4081/THAL.2018.7475","DOIUrl":"https://doi.org/10.4081/THAL.2018.7475","url":null,"abstract":"Knowledge and autonomy are key aspects of informed choice; it is important to define what is important for participants to understand, when accepting or declining screening and for individuals to understand that screening is optional and their own personal choice There are no agreed thresholds or minimum standards for the knowledge an individual is required to have to make an ‘informed’ choice. It is time that minimum agreed standards are developed for practitioners who provide genetic information. There is no standard for evaluating good knowledge or informed choice in population reproductive genetic screening, however measuring people’s choices is a good indicator of informed choice. Informed choice in a multicultural world will be explored and an overview of the different levels of informed choice, practiced in the pathway from genetic screening to identifying at risk couples discussed.","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/THAL.2018.7475","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48269258","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Although the improvements in the treatment and management of thalassemia patients in new years lead to the improved survival and quality of life (QOL) in this group of patients, QOL is still is an important dimension of care in thalassemic patients (1). WHO defines QOL as “an individual’s perception of their position in life in the context of the culture and value systems in which they live and in relation to their goals, expectations, standards, and concerns” (2). Thalassemia is a chronic disease needs life-long care with multiple physical, mental and social complications that affect QOL in patients. The most important factors which affect QOL in thalassemia are: effects of the disease on family, skeletal and face changes, frequent blood transfusion and drug infusion, sexual impairment and infertility, heart and liver disease as well as endocrine disorders, anxiety and low life expectancy (3).
{"title":"Quality of life: Transfusion dependent thalassemia vs non-transfusion dependent thalassemia","authors":"M. Karimi, N. Cohan","doi":"10.4081/thal.2018.7489","DOIUrl":"https://doi.org/10.4081/thal.2018.7489","url":null,"abstract":"Although the improvements in the treatment and management of thalassemia patients in new years lead to the improved survival and quality of life (QOL) in this group of patients, QOL is still is an important dimension of care in thalassemic patients (1). WHO defines QOL as “an individual’s perception of their position in life in the context of the culture and value systems in which they live and in relation to their goals, expectations, standards, and concerns” (2). Thalassemia is a chronic disease needs life-long care with multiple physical, mental and social complications that affect QOL in patients. The most important factors which affect QOL in thalassemia are: effects of the disease on family, skeletal and face changes, frequent blood transfusion and drug infusion, sexual impairment and infertility, heart and liver disease as well as endocrine disorders, anxiety and low life expectancy (3).","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/thal.2018.7489","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42115717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Access to the essential medicines is an important challenge in the developing countries. To have access to the quality and affordable medicines, the pharmaceutical decision makers try different strategies. The production of generic and copy medicines is one of the strategies that if adopted based on the recognized standards and norms can be effective in raising the health status in the developing countries. According to US Food and drug Administration, “a generic drug is a medication created to be the same as an already marketed brand-name drug in dosage form, safety, strength, route of administration, quality, performance characteristics, and intended use. These similarities help to demonstrate bioequivalence, which means that a generic medicine works in the same way and provides the same clinical benefit as its brand-name version”. To make it more understandable, you can define a generic medicine as an equal substitute for its brand-name if it has been produced based on standard norms. However, shakable regulation impairs the quality of generic and copy medicines and harms the health of consumers. NGOs including advocacy groups and scientific groups play effective and undeniable role to ensure quality of the health services which patients receive. Therefore, building a network between activists and scientists is the first step towards better quality. Since we are living in a global market and pharmaceutical active ingredients of pharmaceutical finished products can be found in different regions in the market, the second step of the battle against substandard is to make an international network between advocacy groups. The international network assists to prevent menaces of substandard medicines faster and with reliance on a scientific approach. Furthermore, in the lecture, we aim to reflect over the role of different beneficiaries including international organizations, governments, and pharmaceutical companies in ensuring the feasible and sustainable access of citizens to the essential medicines.
{"title":"Safety and efficacy of drugs: What do I need to know?","authors":"M. Dehshal","doi":"10.4081/thal.2018.7494","DOIUrl":"https://doi.org/10.4081/thal.2018.7494","url":null,"abstract":"Access to the essential medicines is an important challenge in the developing countries. To have access to the quality and affordable medicines, the pharmaceutical decision makers try different strategies. The production of generic and copy medicines is one of the strategies that if adopted based on the recognized standards and norms can be effective in raising the health status in the developing countries. According to US Food and drug Administration, “a generic drug is a medication created to be the same as an already marketed brand-name drug in dosage form, safety, strength, route of administration, quality, performance characteristics, and intended use. These similarities help to demonstrate bioequivalence, which means that a generic medicine works in the same way and provides the same clinical benefit as its brand-name version”. To make it more understandable, you can define a generic medicine as an equal substitute for its brand-name if it has been produced based on standard norms. However, shakable regulation impairs the quality of generic and copy medicines and harms the health of consumers. NGOs including advocacy groups and scientific groups play effective and undeniable role to ensure quality of the health services which patients receive. Therefore, building a network between activists and scientists is the first step towards better quality. Since we are living in a global market and pharmaceutical active ingredients of pharmaceutical finished products can be found in different regions in the market, the second step of the battle against substandard is to make an international network between advocacy groups. The international network assists to prevent menaces of substandard medicines faster and with reliance on a scientific approach. Furthermore, in the lecture, we aim to reflect over the role of different beneficiaries including international organizations, governments, and pharmaceutical companies in ensuring the feasible and sustainable access of citizens to the essential medicines.","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/thal.2018.7494","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46394515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"TIF 2.0: The Thal e-Course and TIF expert patients’ programme for disease-related education and self-management skills in thalassaemia","authors":"V. Antoniadou, M. Angastiniotis, A. Eleftheriou","doi":"10.4081/THAL.2018.7495","DOIUrl":"https://doi.org/10.4081/THAL.2018.7495","url":null,"abstract":"","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":"23 1","pages":""},"PeriodicalIF":0.3,"publicationDate":"2018-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/THAL.2018.7495","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70310998","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
N. Iacovidou, M. Kollia, E. Nana, T. Boutsikou, C. Savvidis, A. Kattamis, D. Kyriakopoulou, V. Ladis
Patients with thalassemia major who received allogeneic hematopoietic cell transplantation are at increased risk of gonadal insufficiency and reduced fertility due to the toxicity of both the transfusional iron overload and the gonadotoxic effects of drugs used in the conditioning regimen. We present a case of an ex-thalassemic patient with spontaneous recovery of spermatogenesis that fathered a healthy, term male neonate. Maternal hemoglobin electrophoresis was within normal limits. At the age of 9.5 years the patient underwent hematopoietic cell transplantation. The conditioning therapy included busulfan (16 mg/kg) and cyclophosphamide (200 mg/kg). No irradiation was administered. Thirty-two days after the hematopoietic cell transplantation the patient developed acute graft-versus-host disease needing long-term treatment with methylprednisolone, cyclosporine and immunoglobulin. Although consecutive semen analyses after the hematopoietic cell transplantation revealed azoospermia, the last semen analysis before conception, at the age of 33 years, was improved and normal follicle stimulating hormone (FSH), luteinizing hormone (LH) and testosterone (Te) levels were detected. The current pregnancy was the result of physical conception. In this case, it seems that thalassemia major along with the respective treatment prior to- and posthematopoietic cell transplantation did not irreparably impair spermatogenesis, probably due to the pre-pubertal time frame they were implemented. 对于接受异基因造血细胞移植的重型地中海贫血患者，由于输注性铁过载的毒性和预处理方案中所用药物性腺毒性作用这两方面的原因，都使其面临更大的性腺功能不全风险和更低的生育力。本文报道一例精子发生出现自然恢复的原重型地中海贫血患者，他成功孕育出一个健康的足月男婴。母体血红蛋白电泳在正常范围内。患者在9岁半时接受了造血细胞移植。预处理治疗包括白消安（16 mg/kg）和环磷酰胺（200 mg/kg）。未给予照射。造血细胞移植32天后，患者出现急性移植物抗宿主病，需要长期使用甲基强的松龙、环孢素和免疫球蛋白治疗。虽然造血细胞移植后连续的精液分析显示无精子症，但在33岁时受精前的最后一次精液分析有所改善，经检测发现卵泡刺激素（FSH）、黄体生成素（LH）和睾酮（Te）水平正常。目前的怀孕是自然受孕的结果。在这个病例中，看来重型地中海贫血以及造血细胞移植前后相应的治疗并没有对精子发生造成不可恢复的破坏，这可能是由于移植时处于青春发育期前时间段的原因。
Patients with Thalassemia major who received allogenic hematological cell transplantation are at an increased risk of governmental inefficiency and reduced utility due to the toxicity of both the transitional iron overload and the Gonadoxic effects of drugs used in the conditioning region We present a case of an ex-thalasemic patient with sparse recovery of spermatogenes that fathered a health, term male neonate Material hemoglobin electrophoresis was within normal limits At the age of 9.5 years the patient underneath hematological cell transmission The conditioning therapy includes busulfan (16 mg/kg) and cyclophosphamide (200 mg/kg) No iration was administered Third two days after the hematopoietic cell transmission of the patient developed acute gradient Versus host disease needing long term treatment with methylprednisolone, cyclosporine, and immunoglobal Although consecutive semen analyses after the hematopoietic cell transmission revised azoospermia, the last semen analysis before concept, at the age of 33 years, was improved and normal follicular stimulating hormone (FSH), luteinizing hormone (LH) and testosterone (Te) levels were detected The current reservation was the result of physical concept In this case, it appears that that assembly major along with the prospective treatment prior to - and postdoctoral cell transplantation did not irreparably improve sperm genes, possibly due to the pre public time frame they were implemented For patients with severe thalassemia undergoing allogeneic hematopoietic cell transplantation, due to the toxicity of iron overload during infusion and the gonadal toxicity of the drugs used in the pre-treatment regimen, they face a greater risk of gonadal dysfunction and lower fertility. This article reports a case of a patient with severe thalassemia who experienced natural recovery after spermatogenesis. He successfully gave birth to a healthy full-term male infant. The maternal hemoglobin electrophoresis is within the normal range. The patient received a hematopoietic cell transplant at the age of 9 and a half. Preconditioning treatment includes Baixiaoan (16 mg/kg) and cyclophosphamide (200 mg/kg). No irradiation was given. After 32 days of hematopoietic cell transplantation, the patient developed acute graft versus host disease and required long-term treatment with methylprednisolone, cyclosporine, and immunoglobulin. Although continuous semen analysis after hematopoietic cell transplantation showed azoospermia, the last semen analysis before fertilization improved at the age of 33, and normal levels of follicle stimulating hormone (FSH), luteinizing hormone (LH), and testosterone (Te) were detected. The current pregnancy is the result of natural conception. In this case, it appears that severe thalassemia and the corresponding treatment before and after hematopoietic cell transplantation did not cause irreparable damage to spermatogenesis, which may be due to the pre pubertal period during transplantati
{"title":"Spontaneous fertility in a male thalassemic patient after allogeneic hematopoietic cell transplantation","authors":"N. Iacovidou, M. Kollia, E. Nana, T. Boutsikou, C. Savvidis, A. Kattamis, D. Kyriakopoulou, V. Ladis","doi":"10.4081/THAL.2017.7090","DOIUrl":"https://doi.org/10.4081/THAL.2017.7090","url":null,"abstract":"Patients with thalassemia major who received allogeneic hematopoietic cell transplantation are at increased risk of gonadal insufficiency and reduced fertility due to the toxicity of both the transfusional iron overload and the gonadotoxic effects of drugs used in the conditioning regimen. We present a case of an ex-thalassemic patient with spontaneous recovery of spermatogenesis that fathered a healthy, term male neonate. Maternal hemoglobin electrophoresis was within normal limits. At the age of 9.5 years the patient underwent hematopoietic cell transplantation. The conditioning therapy included busulfan (16 mg/kg) and cyclophosphamide (200 mg/kg). No irradiation was administered. Thirty-two days after the hematopoietic cell transplantation the patient developed acute graft-versus-host disease needing long-term treatment with methylprednisolone, cyclosporine and immunoglobulin. Although consecutive semen analyses after the hematopoietic cell transplantation revealed azoospermia, the last semen analysis before conception, at the age of 33 years, was improved and normal follicle stimulating hormone (FSH), luteinizing hormone (LH) and testosterone (Te) levels were detected. The current pregnancy was the result of physical conception. In this case, it seems that thalassemia major along with the respective treatment prior to- and posthematopoietic cell transplantation did not irreparably impair spermatogenesis, probably due to the pre-pubertal time frame they were implemented. 对于接受异基因造血细胞移植的重型地中海贫血患者，由于输注性铁过载的毒性和预处理方案中所用药物性腺毒性作用这两方面的原因，都使其面临更大的性腺功能不全风险和更低的生育力。本文报道一例精子发生出现自然恢复的原重型地中海贫血患者，他成功孕育出一个健康的足月男婴。母体血红蛋白电泳在正常范围内。患者在9岁半时接受了造血细胞移植。预处理治疗包括白消安（16 mg/kg）和环磷酰胺（200 mg/kg）。未给予照射。造血细胞移植32天后，患者出现急性移植物抗宿主病，需要长期使用甲基强的松龙、环孢素和免疫球蛋白治疗。虽然造血细胞移植后连续的精液分析显示无精子症，但在33岁时受精前的最后一次精液分析有所改善，经检测发现卵泡刺激素（FSH）、黄体生成素（LH）和睾酮（Te）水平正常。目前的怀孕是自然受孕的结果。在这个病例中，看来重型地中海贫血以及造血细胞移植前后相应的治疗并没有对精子发生造成不可恢复的破坏，这可能是由于移植时处于青春发育期前时间段的原因。","PeriodicalId":22261,"journal":{"name":"Thalassemia Reports","volume":" ","pages":""},"PeriodicalIF":0.3,"publicationDate":"2017-11-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4081/THAL.2017.7090","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45216194","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Chathupa Wickremaarachchi, E. McGill, A. Bosco, G. Kidson-Gerber
The aim of this study was to improve current transfusion practice in transfusiondependent thalassaemia patients by determining whether safe transition from triplewashed red cells (TWRC) to leucodepleted red cells (LDRC), increasing transfusion rates, reducing the use of frusemide and creating uniform practice across patients is possible. In patients receiving regular transfusions (50), triple-washed red blood cells were changed to LDRC, transfusion rates were increased to 5 mL/kg/h (in line with the Cooley’s Foundation guidelines) to a maximum of 300 mL/h and frusemide was ceased. Medical review occurred at completion of the transfusion. Of the 20 patients on TWRC, 18 were transitioned to leucodepleted red cells (90%). Recurrent allergic reactions in 2 patients required re-institution of TWRC. 7 of the 8 patients on regular frusemide ceased this practice with no documented transfusion-related fluid overload. One patient refused. Of the eligible 50 patients, 20 patients (40%) were increased to the maximum transfusion rate of 300 mLs/h; 6 (12%) increased rate but refused to go to the maximum; 9 (18%) refused a change in practice and 15 (30%) were already at the maximum rate. There was only one documented transfusion reaction (palpitations) however this patient was able to tolerate a higher transfusion rate on subsequent transfusions. Thalassemia patients on TWRC were safely transitioned to LDRC. Transfusion rates were safely increased, with a calculated reduction in day-stay bed time of 17.45 h per month. This confirms a guideline of 5 mL/kg/h for transfusion-dependant thalassaemia patients with preserved cardiac function is well tolerated and may be translated to other centres worldwide. 本研究的目的是通过确定是否有可能进行从三洗红细胞（TWRC）到去白细胞红细胞（LDRC）的安全过渡，提高输血速率，减少速尿的使用，并在患者中实施统一规则，从而改进输血依赖型地中海贫血患者中现有的输血实践。在接受定期输血的患者（50例）中，将三洗红细胞改为 LDRC，输血速率提高至5 mL/kg/h（符合库利氏贫血基金会的指引），最高可达到300 mL/h，并停止使用速尿。输血完成后进行体检。在使用TWRC的20例患者中，18例转为去白细胞红细胞（90%）。2例患者产生的复发性过敏反应需要重新输以TWRC。8名定期使用速尿的患者中，7名中止了使用该药物，并且没有输血相关液体超负荷的相关记录。一名病人拒绝。在符合条件的50例患者中，20例（40%）增加至300 mL/h的最大输血速率；6例（12%）输血速率提高但拒绝增加至最大；9例（18%）拒绝做出改变，15例（30%）已经达到了最大速率。只产生一例有记录的输血反应（心悸），但是该患者在随后的输血中能够耐受更高的输血速率。使用TWRC的地中海贫血患者安全转用LDRC。输血速率安全地得到提高，计算出减少的白天卧床时间为每月17.45小时。这证实了5 mL/kg/h的指引在心功能得到保护的输血依赖型地中海贫血患者中有良好的耐受性，可以推广至全球其他中心。
本研究的目的是通过确定是否可以安全地从三洗红细胞(TWRC)转移到贫白细胞(LDRC)，增加输血率，减少氟塞米的使用，并在患者之间建立统一的输血实践，来改善输血依赖性地中海贫血患者目前的输血实践。在接受常规输血的患者中(50例)，三洗红细胞改为LDRC，输血率增加到5ml /kg/h(符合Cooley基金会指南)至最高300 mL/h，并停止使用氟塞胺。输血结束后进行了医学检查。在接受TWRC治疗的20例患者中，18例(90%)转变为白细胞减少的红细胞。2例患者出现复发性过敏反应，需重新接受TWRC治疗。8例常规使用氟塞胺的患者中有7例停止了这种做法，没有记录与输血相关的液体超载。一位病人拒绝了。在符合条件的50例患者中，20例患者(40%)增加到最大输血率300 ml /h;6个(12%)增加速率但未达到最大值;9家(18%)拒绝改变做法，15家(30%)已经达到最高比率。只有一例记录在案的输血反应(心悸)，但该患者在随后的输血中能够耐受更高的输液率。使用TWRC的地中海贫血患者被安全地转移到LDRC。输血率安全增加，计算减少每月17.45小时的日间住院时间。这证实了5ml /kg/h的输血依赖型地中海贫血患者心脏功能保持良好的耐受性，并可推广到世界其他中心。本研究的目的是通过确定是否有可能进行从三洗红细胞(TWRC)到去白细胞红细胞(LDRC)的安全过渡,提高输血速率,减少速尿的使用,并在患者中实施统一规则,从而改进输血依赖型地中海贫血患者中现有的输血实践。在接受定期输血的患者(50例)中,将三洗红细胞改为LDRC,输血速率提高至5 mL / kg / h(符合库利氏贫血基金会的指引),最高可达到300 mL / h,并停止使用速尿。输血完成后进行体检。在使用TWRC的20例患者中,18例转为去白细胞红细胞。2例(90%)患者产生的复发性过敏反应需要重新输以TWRC.8名定期使用速尿的患者中,7名中止了使用该药物,并且没有输血相关液体超负荷的相关记录。一名病人拒绝。在符合条件的50例患者中,20例(40%)增加至300毫升/小时的最大输血速率;6例(12%)输血速率提高但拒绝增加至最大;9例(18%)拒绝做出改变,15例(30%)已经达到了最大速率。只产生一例有记录的输血反应（心悸），但是该患者在随后的输血中能够耐受更高的输血速率。中国日报网2016-10-20。这证实了5 mL / kg / h的指引在心功能得到保护的输血依赖型地中海贫血患者中有良好的耐受性,可以推广至全球其他中心。
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