Türk toplumunda gözlenen ilk hemoglobin g-waimanalo ve hemoglobin fontainebleau olguları]

[No abstract available

Ferric Carboxymaltose as Treatment in Women with Iron-Deficiency Anemia

Objective. To evaluate safety and efficacy of intravenous ferric carboxymaltose (FCM) versus standard medical care (SMC) for iron-deficiency anemia (IDA) in postpartum women and women with heavy menstrual bleeding. Study Design. This open-label, multicenter study randomized women with IDA (hemoglobin ≤ 11.0 g/dL) to single doses of FCM (15 mg/kg [maximum 1000 mg]) or SMC (this treatment was determined by the investigator and there may have been no treatment). Safety data (primary outcome) were collected for 30 days. Results. Of 2045 subjects enrolled (FCM: = 1023; SMC: = 1022), 996 received FCM and 1022 received SMC. At least 1 serious adverse event (AE) was reported by 0.6% and 2.2% of subjects in the FCM and SMC groups, respectively; none were considered treatment related. The difference in serious AEs was primarily due to higher rates of uterine leiomyoma, uterine hemorrhage, and menorrhagia in SMC subjects with heavy menstrual bleeding. Common AEs were generally predictable, with higher rates of infusion site reactions in FCM subjects and gastrointestinal AEs in SMC subjects. Mean hemoglobin increases were greater in the FCM group than the SMC group. Conclusion. FCM was well tolerated and effectively increased mean hemoglobin levels in postpartum women or women with heavy menstrual bleeding and IDA. This trial is registered with ClinicalTrials.gov, NCT00548860

Health related quality of life in Malaysian children with thalassaemia

BACKGROUND: Health Related Quality of Life (HRQoL) studies on children with chronic illness such as thalassaemia are limited. We conducted the first study to investigate if children with thalassaemia have a lower quality of life in the four dimensions as measured using the PedsQL 4.0 generic Scale Score: physical, emotional, social and role (school) functioning compared to the healthy controls allowing for age, gender, ethnicity and household income. METHODS: The PedsQL 4.0 was administered to children receiving blood transfusions and treatments at Hospital Kuala Lumpur, Malaysia using PedsQL 4.0 generic Scale Score. Accordingly, the questionnaire was also administered to a control group of healthy school children. Socio-demographic data were also collected from patients and controls using an interview schedule designed for the study. RESULTS: Of the 96 thalassaemia patients approached, 78 gave consent to be interviewed giving a response rate of 81.3%. Out of 235 healthy controls approached, all agreed to participate giving a response rate of 100%. The mean age for the patients and schoolchildren is 11.9 and 13.2 years respectively. The age range for the patients and the schoolchildren is between 5 to 18 years and 7 to 18 years respectively. After controlling for age and demographic background, the thalassaemia patients reported having significantly lower quality of life than the healthy controls. CONCLUSION: Thalassaemia has a negative impact on perceived physical, emotional, social and school functioning in thalassaemia patients which was also found to be worse than the children's healthy counterparts. Continuing support of free desferal from the Ministry of Health should be given to these patients. More understanding and support especially from health authorities, school authorities and the society is essential to enhance their quality of life

Preliminary data on COVID-19 in patients with hemoglobinopathies : A multicentre ICET-A study

Objectives: This study aims to investigate, retrospectively, the epidemiological and clinical characteristics, laboratory results, radiologic findings, and outcomes of COVID-19 in patients with transfusion-dependent β thalassemia major (TM), β-thalassemia intermedia (TI) and sickle cell disease (SCD). Design: A total of 17 Centers, from 10 countries, following 9,499 patients with hemoglobinopathies, participated in the survey. Main outcome data: Clinical, laboratory, and radiologic findings and outcomes of patients with COVID-19 were collected from medical records and summarized. Results: A total of 13 patients, 7 with TM, 3 with TI, and 3 with SCD, with confirmed COVID-19, were identified in 6 Centers from different countries. The overall mean age of patients was 33.7±12.3 years (range:13-66); 9/13 (69.2%) patients were females. Six patients had pneumonia, and 4 needed oxygen therapy. Increased C-reactive protein (6/10), high serum lactate dehydrogenase (LDH; 6/10), and erythrocyte sedimentation rate (ESR; 6/10) were the most common laboratory findings. 6/10 patients had an exacerbation of anemia (2 with SCD). In the majority of patients, the course of COVID-19 was moderate (6/10) and severe in 3/10 patients. A 30-year-old female with TM, developed a critical SARS-CoV-2 infection, followed by death in an Intensive Care Unit. In one Center (Oman), the majority of suspected cases were observed in patients with SCD between the age of 21 and 40 years. A rapid clinical improvement of tachypnea/dyspnea and oxygen saturation was observed, after red blood cell exchange transfusion, in a young girl with SCD and worsening of anemia (Hb level from 9.2 g/dl to 6.1g/dl). Conclusions: The data presented in this survey permit an early assessment of the clinical characteristics of COVID 19 in different countries. 70% of symptomatic patients with COVID-19 required hospitalization. The presence of associated co-morbidities can aggravate the severity of COVID- 19, leading to a poorer prognosis irrespective of age

Haemoglobinopathy prevention program in Turkey

Thalassemia and abnormal haemoglobins are a serious health problem in Turkey. Very important steps for toward preventing thalassemia have been taken in Turkey by Ministry of Health (MOH), Turkish National Haemoglobinopathy Council (TNHC) and Thalassemia Federation of Turkey (TFT) since 2000. In 1993, a law was issued called Fight Against Hereditary Blood Disease especially for thalassemia and haemoglobinopathies. The law commends to prevent haemoglobinopathies and to treat all patients with haemoglobinopathy and thalassemia. A pilot project was started and centres were created in the MOH Hospitals in the southern provinces of Turkey. In 2000, TNHC was installed to combine all centres, foundations, and associations into one organization controlled by the MOH. In 2001, the MOH and the TNHC made an inventory of all recorded patients with thalassemia and abnormal hemoglobins in Turkey, registering at least 4513 patients. In 2002, written regulations for the Fight Against Hereditary Blood Disease were published. MOH and TNHC selected 33 provinces situated in the Thrace, Marmara, Aegean, Mediterranean and South Eastern regions with high birth prevalence of severe haemoglobinopathies. In 2003, the haemoglobinopathy scientific committee was set-up, a guidebook was published and a national Hemoglobinopaty Prevention Program (HPP) was started in these high risk provinces . This program is running in these provinces successfully. In 2005, TFT was established as a secular society organization instead of TNHC. In 2007, National Thalassemia Prevention Campaign (NTPC) was organized for public education by TFT. This campaign contributed very important supporting to HPP in Turkey, because totally 62.682 people such as health workers, students, teachers, demarches, religion officers and the other many people were educated for preventing thalassemia and haemoglobinopathies. In 2009, National Thalassemia Education Seminars (NTES) for health personnel have been planned in 26 cities by MOH and TFT. A total 3.600 health persons were educated on thalassemia prevention and therapy with NTES in 18 centres in 2009 and 2010. In conclusion, according to reports of MOH, 46 first level haemoglobinopathy diagnosis centres, 5 second level diagnosis and therapy centre and 5 third level prenatal diagnosis centre were setup and licenced in 30 cities between 2003 and 2009. While premarital screening tests were 30% of all couples in 2003, it increased continuously during 6 years and it reached 81% in 2008. The number of new born with thalassemias and hemoglobinopathies was 272 in 2002, it was decreased to 23 in 2008, as a result there has been an 90% reduction in new affected births.&nbsp;地中海贫血和异常血红蛋白是土耳其国内的两大严重的健康问题。 从2000年起，土耳其卫生部(MOH)、土耳其国家血红蛋白病委员会（TNHC）和土耳其地中海贫血联合会（TFT）采取了多种预防地中海贫血的重要措施。1993年，土耳其颁布了名为&ldquo;抵抗遗传性血色病&rdquo;（主要针对地中海贫血和异常血红蛋白）的法律， 旨在防止血红蛋白病和治疗所有血红蛋白病患者和地中海贫血患者。 在土耳其南部各省的卫生部所属医院启动了试点项目并成立了多个卫生中心。 2000年，土耳其国家血红蛋白病委员会成立。该委员会和所有卫生中心、基金会和协会组成一个庞大组织，归属卫生部管理。 2001年，MOH和TNHC登记了所有记录在案的地中海贫血患者和异常血红蛋白患者，共计4513人。 2002年，颁布了抵抗遗传性血色病的书面法规。 MOH和TNHC确定了位于色雷斯、马尔马拉、爱琴海、地中海和东南地区等血红蛋白病出生高发地区的33个省。 2003年，成立血红蛋白病科学委员会，出版了一本指导手册并在高危地区启动了血红蛋白病预防项目（HPP）。 该项目在这些省成功执行。 2005年，社会安全组织&ldquo;土耳其地中海贫血联合会&rdquo;（TFT）成立，并取代TNHC。 2007年，组织了国家血红蛋白病预防运动（NTPC）以便TFT实施公众教育。 该项运动对实施血红蛋白病预防项目（HPP）起到非常重要的支持作用，因为共计62682民众（包括卫生工作者、学生、教师、市长、宗教官员和其他很多身份的人民）都受到预防地中海贫血和异常血红蛋白的教育。 2009年，MOH和TFT为卫生工作者规划在26个城市召开国家地中海贫血教育研讨会（NTES）。 2009年和2010年，总共3600名正常人在18个卫生中心受到NTES开展的地中海贫血预防教育。总结，据MOH报告， 2003年到2009年期间，土耳其在30个城市总共成立了46第个一级血红蛋白病诊断中心、5个第二级诊断和治疗中心和5个第三级产前诊断中心，并全部颁发执照。婚前筛选检查在2003年占所有夫妻的30%， 之后6年持续升高，并在2008年达到81%。2002年，新生地中海贫血和血红蛋白病患儿人数为272名，2008年下降到23人，也就是说，受感染新生患儿减少了90%。</p

Preface

During the last two decades there has been a rapid and significant increase of knowledge and success in the control of Hb disorders. This was the result of a marked revival of interest by the international medical community and of the tremendous progress achieved in the empowerment of patients all over the world. Ingredients that contributed to literally changing the natural history of this disease from a fatal one to a well managed chronic disease, with survival and quality of life of patients to near normal standards..

TT virus infection in transfusion-dependent thalassemia

WOS: 00016924340054