Extracorporeal photopheresis did not prevent the development of an autoimmune disease: myasthenia gravis

WOS: 000390578300026PubMed ID: 27612294BACKGROUNDMyasthenia gravis (MG) is a neuromuscular disorder characterized by an autoimmune defect in the neuromuscular junction. In most patients, the autoimmune attack is mediated by antibodies against the acetylcholine receptor (AChR) on the postsynaptic membrane. Deficient immunoregulation, including regulatory T cells, is consistently observed. Extracorporeal photopheresis (ECP) leads to the induction of regulatory T cells that mediate immunologic tolerance in autoimmune diseases; however, the data regarding MG are very limited. CASE REPORTHere, we report a patient who, during ongoing ECP therapy for his severe, refractory, chronic graft-versus-host disease (cGVHD), developed MG, although he responded very well to ECP, as indicated by the lowering of his chronic cGVHD severity grade to moderate. RESULTSDespite receiving ECP, our patient developed MG, which was resistant to treatment and required intensive care unit support. CONCLUSIONSClose surveillance is required when ECP is planned as one of the treatment alternatives in myasthenia gravis that develop in cGVHD

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

Outcomes of high-grade gastrointestinal graft-versus-host disease posthematopoietic stem cell transplantation in children

WOS: 000388566200044PubMed ID: 27858879We explored the clinical course of acute high-grade gastrointestinal graft-versus-host disease in children in a single center. This was a retrospective analysis of 28 pediatric patients who presented with a clinical diagnosis of stage III and IV acute graft-versus-host disease (aGVHD) of the gastrointestinal system (GIS). Generally, skin involvement was the initial manifestation of aGVHD that began in the first 3 weeks of hematopoietic stem cell transplantation (HSCT); on the other hand, GIS involvement predominated after the second week of HSCT. Reported adult data show a survival rate of only 25%; however, our study showed more favorable outcomes in children with a survival rate of 55%. We monitored levels of albumin and immunoglobulin G and observed low levels overall during treatment of unresponsive patients, although only albumin levels were shown to be significantly different. We observed a significant increase in mortality with the use of antithymocyte globulin in GIS aGVHD, although antithymocyte globulin used for graft-versus-host disease prophylaxis had no demonstrable effect on GIS aGVHD mortality. Whether the significantly lower GIS aGVHD mortality among the children recruited in our study than among their historical adult counterparts is a primary result of the specific attributes of the pediatric GIS, or whether it originated from HSCT kinetics remains to be determined by future studies

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

Higher incidence of hypertension with intravenous busulfan compared to oral busulfan in thalassaemic children who underwent haematopoietic stem cell transplantation

Bu çalışma, 03-06 Nisan 2011 tarihleri arasında Paris[Fransa]’da düzenlenen 37. European Group for Bone and Marrow Transplantation/27. Meeting of the EBMT Nurses Group/10th Meeting of the EBMT Management Group’da bildiri olarak sunulmuştur.European Grp Bone & Marrow TransplantatEuropean Grp Bone & Marrow Transplantat Nurses GrpEuropean Grp Bone & Marrow Transplantat Management Gr

Outcomes of high grade gastrointestinal GVHD POST-HSCT in children

21st Congress of the European-Hematology-Association -- JUN 09-12, 2016 -- Copenhagen, DENMARKWOS: 000379484603291…The European Hematology Associatio

Extracorporeal photopheresis ın steroid refractory pediatric gvhd patients: A single center experience

...European Society for Blood and Marrow Transplantatio