The effect of oral chelators on major thalassemia patients life quality

AMAÇ: Talasemi major'da yaşam kalitesini düşüren en önemli etkenlerden biri deferoksaminin uzun süreli subkutan uygulanmasının getirdiği zorluklardır. Bu kesitsel çalışmada, 8-18 yaş arası beta talasemı major'lu okul çocuklarında oral şelatörlerın yaşam kalitesine etkisi değerlendirilmiştir. GEREÇ ve YÖNTEM: Aynı merkezde en az 1 yıldır izlemde olan, talasemi major tanısı almış 32 hasta değerlendirildi. Deferıprone 40-75 mg/kg/g ve deferasıroks 20-40 mg/kg/g dozunda alınırken, deferıpron günde 3 dozda ve deferasıroks sabah tek dozda oral alındı. Yaşam kalıtesı değerlendırmesınde PedsQL anketı hastalara ve ebeveynlerıne ayrı olarak uygulandı. istatıstıksel değerlendırmede, ıkı gruba aıt nıcel değışkenlerın karşılaştırılması amacıyla Student's t testı veya Mann-Whıtney U testı kullanılmıştır. istatıstıksel olarak anlamlılık ıçın p<0.05 kabul edılmıştır. BULGULAR: Oral şelatör kullanımının, çocukların kendılerıne göre yaşam kalıtesı toplam puanına etkısı olmamakla beraber ebeveynlere göre deferasıroks alan hastaların deferıprone alanlara göre yaşam kalıtesı puan ortalamasının anlamlı derecede yüksek olduğu saptandı (p=0.019). Transfüzyon sıklığının artışı, ıstatıstıksel olarak anlamlı olmasa da, yaşam kalitesini düşürdüğü gözlendı. SONUÇ: Yaşam boyu tedavı uygulanmasının talasemı majordakı yaşam kalıtesıne etkısı nedenıyle hastalara olabıldığınce az sıklıkta hastane vızıtı ve tedavı uygulanması gerekmektedır. Çalışmamızda daha az doz sıklığında uygulanan deferasıroks'un yaşam kalitesini deferıpron'a göre arttırdığı gözlenmiştir.OBJECTIVE: One of the most important factors which decrease the quality of life of thalassemia major patients is the discomfort of long term subcutanous administration of deferoxamine. In this cross sectional study, we analysed the effect of oral chelator usage on quality of life of beta thalassemia major patients who are 8-18 years old school children. MATERIALS and METHODS: We studied 32 thalassemia major patients who were under observation for at least one year at the same center. Deferiprone and deferasirox were used in a dose of 40-75 mg/kg/d thrice daily and 10-40 mg/kg/d once daily in the morning, respectively. PedsQL was used separately for patients and their parents to assess the quality of life. Statistical analyses were perfomd to compare the two groups´ quantitative variables using the Student t-test or Mann-Whitney U test. Statistically signi? cant meant a p-value of less than 0.05. RESULTS: We determined that oral chelator usage did not affect the overall score of quality of life according to the self -reports of children, however according to the parents, the patients using deferasirox had significantly better quality of life than the patients using deferiprone (p=0.019). Higher frequency of transfusion decreased the quality of life scores, however it was not significant. CONCLUSION: Because of the major effect of lifelong treatment on quality of life in thalassemia major, hospital visits and treatments should be in lower frequency. We determined that deferasirox with a lower frequency of dosage application improved quality of life better than deferiprone in users

Hematopoietic Stem Cell Transplantation Activity and Trends at a Pediatric Transplantation Center in Turkey During 1998-2008

OBJECTIVE: The aim of this study was to document hematopoietic stem cell transplantation (HSCT) activity and trends at our treatment center. METHODS: Data collected over a 10-year period were retrospectively analyzed, concentrating primarily on types of HSCT, transplant-related mortality (TRM), stem cell sources, indications for HSCT, and causes of death following HSCT. RESULTS: In total, 222 allogeneic (allo)-HSCT (87.4%) and 32 autologous (auto)-HSCT (12.6%) procedures were performed between 1998 and 2008. Stem cells obtained from unrelated donors were used in 22.6% (50/222) of the allo- HSCTs. Cord blood was the source of hematopoietic stem cells (HSC) in 12.2% of all transplants. The most common indication for allo-HSCT was hemoglobinopathy (43.2%), versus neuroblastoma (53.1%) for auto-HSCT. The TRM rate 1 year post transplantation was 18.3% ± 2.5% for all transplants, but differed according to transplantation type (23.5% ± 7.9% for auto-HSCT and 17.5% ± 2.6% for allo-HSCT). The most common cause of death 1 year post HSCT was infection (35.9%). CONCLUSION: The TRM rate in the patients that underwent allo-HSCT was similar to that which has been previously reported; however, the TRM rate in the patients that underwent auto-HSCT was higher than previously reported in developed countries. The selection of these patients to be transplanted must be made attentively

Hematopoietic stem cell transplantation for hemophagocytic lymphohistiocytosis: A single center experience

...European Society for Blood and Marrow Transplantatio

Effect of central nervous system complications and radiologic findings on patients' survival after hematopoietic stem cell transplantation

...European Society for Blood and Marrow Transplantatio

A National Registry of Thalassemia in Turkey: Demographic and Disease Characteristics of Patients, Achievements, and Challenges in Prevention

Objective: The Turkish Society of Pediatric Hematology set up a National Hemoglobinopathy Registry to demonstrate the demographic and disease characteristics of patients and assess the efficacy of a hemoglobinopathy control program (HCP) over 10 years in Turkey. Materials and Methods: A total of 2046 patients from 27 thalassemia centers were registered, of which 1988 were eligible for analysis. This cohort mainly comprised patients with &#946;-thalassemia major (n=1658, 83.4%) and intermedia (n=215, 10.8%). Results: The majority of patients were from the coastal areas of Turkey. The high number of patients in Southeastern Anatolia was due to that area having the highest rates of consanguineous marriage and fertility. The most common 11 mutations represented 90% of all &#946;-thalassemia alleles and 47% of those were IVS1-110(G-&gt;A) mutations. The probability of undergoing splenectomy within the first 10 years of life was 20%, a rate unchanged since the 1980s. Iron chelators were administered as monotherapy regimens in 95% of patients and deferasirox was prescribed in 81.3% of those cases. Deferasirox administration was the highest (93.6%) in patients aged &lt;10 years. Of the thalassemia major patients, 5.8% had match-related hemopoietic stem cell transplantation with a success rate of 77%. Cardiac disease was detected as a major cause of death and did not show a decreasing trend in 5-year cohorts since 1999. Conclusion: While the HCP has been implemented since 2003, the affected births have shown a consistent decrease only after 2009, being at lowest 34 cases per year. This program failure resulted from a lack of premarital screening in the majority of cases. Additional problems were unawareness of the risk and misinformation of the at-risk couples. In addition, prenatal diagnosis was either not offered to or was not accepted by the at-risk families. This study indicated that a continuous effort is needed for optimizing the management of thalassemia and the development of strategies is essential for further achievements in the HCP in Turkey

A National Registry of Thalassemia in Turkey: Demographic and Disease Characteristics of Patients, Achievements, and Challenges in Prevention

WOS: 000426572200002PubMed ID: 28404539Objective: The Turkish Society of Pediatric Hematology set up a National Hemoglobinopathy Registry to demonstrate the demographic and disease characteristics of patients and assess the efficacy of a hemoglobinopathy control program (HCP) over 10 years in Turkey. Materials and Methods: A total of 2046 patients from 27 thalassemia centers were registered, of which 1988 were eligible for analysis. This cohort mainly comprised patients with beta-thalassemia major (n = 1658, 83.4%) and intermedia (n = 215, 10.8%). Results: The majority of patients were from the coastal areas of Turkey. The high number of patients in Southeastern Anatolia was due to that area having the highest rates of consanguineous marriage and fertility. The most common 11 mutations represented 90% of all beta-thalassemia alleles and 47% of those were IVS1-110(G->A) mutations. The probability of undergoing splenectomy within the first 10 years of life was 20%, a rate unchanged since the 1980s. Iron chelators were administered as monotherapy regimens in 95% of patients and deferasirox was prescribed in 81.3% of those cases. Deferasirox administration was the highest (93.6%) in patients aged <10 years. Of the thalassemia major patients, 5.8% had match-related hemopoietic stem cell transplantation with a success rate of 77%. Cardiac disease was detected as a major cause of death and did not show a decreasing trend in 5-year cohorts since 1999. Conclusion: While the HCP has been implemented since 2003, the affected births have shown a consistent decrease only after 2009, being at lowest 34 cases per year. This program failure resulted from a lack of premarital screening in the majority of cases. Additional problems were unawareness of the risk and misinformation of the at-risk couples. In addition, prenatal diagnosis was either not offered to or was not accepted by the at-risk families. This study indicated that a continuous effort is needed for optimizing the management of thalassemia and the development of strategies is essential for further achievements in the HCP in Turkey.Ege Children's Foundation; Novartis Pharmaceuticals CorporationNovartisThe authors thank Caglar Serdar, Aylin Gokduman, and Tolga Turgay of Plexus Information Technologies for their website support. The current study and the work presented here are from an Investigator Initiated Trial, which was sponsored by the Ege Children's Foundation and funded by Novartis Pharmaceuticals Corporation