Cataract Formation due to use of Deferiprone in a Patient with Thalassemia Major

Talasemiler; otozomal resesif kalıtım gösteren, hemoglobin zincirlerinden birinin veya bir kaçının hasarlı sentezi sonucu ortaya çıkan hipokrom mikroster anemi ile karakterize heterojen bir grup hastalıktır. Hastaların sık transfüzyona maruz kalması sonucunda aşırı demir birikimine bağlı yaşamı tehdit eden önemli klinik bulgular ortaya çıkar. Talasemili hastalarda hastalığın kendisine ya da kullanılan şelatör tedavisine bağlı olarak oküler değişikliklere çok sık olmasa da rastlanmaktadır. Bu değişiklikler genellikle katarakt, optik nöropati, retinal pigment epitelinde (RPE) dejenerasyon, RPE mottling, retinal venous tortuosity, vitreoretinal hemorrhages and obliteration of iris patern şeklindedir. İlk defa demir bağlayıcı şelatör olarak kullanılmaya başlanan deferoxamin optik nöropati ve retinal toksisite gibi iyi bilinen komplikasyonlara sahiptir. Ancak deferoxaminin yerini alan ve yakın dönemde sık kullanılan deferipronun oküler toksisitesi ile ilgili literatürde daha önce bildirilmiş az sayıda bilgi vardır. Bu olgu deferipron kullanımına bağlı gelişen katarakt oluşumuna dikkat çekmek amacıyla bildirilmiştir.Thalassemias are a heterogeneous group of autosomal recessive diseases characterized by hypochromic microcytic anemia and occur as a result of defective synthesis of one or more hemoglobin chains. In patients, life-threatening clinical manifestations may occur because of severe iron overload due to frequent blood transfusions. Ocular changes in patients with thalassemia are to be encountered depending on the disease itself or chelator used in the treatment, but not very often. These changes are usually cataracts, optic neuropathy, retinal pigment epithelium (RPE) degeneration, RPE mottling, retinal venous tortuosity, vitreoretinal hemorrhages and obliteration of the iris pattern. Desferrioxamine that is used as the first iron-binding chelating has well-known complications such as optic neuropathy and retinal toxicity. However, Deferiprone that used more common recently has replaced the Desferrioxamine but, there is very little information in the literature about the ocular toxicity of deferiprone. In this case report, we have reported a patient with deferiprone-induced cataract formation in order to draw attention to a little-known complication of the drug

Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group (p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes

Thalassemia-free and graft-versus-host-free survival: Outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group (p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes.Turkish Society of Pediatric Hematolog