Sickle cell disease (SCD) is a disorder that causes red blood cells to become sticky and rigid. Sickle cells can block blood flow in small blood vessels depriving the eye of oxygen and cause damage. This is called sickle retinopathy that can progress to severe proliferative sickle cell retinopathy, bleeding into the eye, detachment of the retina or even loss of vision. To assess ocular manifestations and detect frequency of retinopathy in patients with SCD. Cross-sectional study was conducted on 32 patients with SCD. They were 22 males and 10 females with mean age of 12 years. Routine investigations as well as ophthalmological examination including visual acuity, fluorescein angiography and optical coherence tomography were done. We found that 8 patients (25%) suffered from proliferative retinopathy, 10 patients (31%) showed tortuous retinal veins, while 14 patients (44%) were normal. All patients showed macular thinning on optical coherence tomography examination. We concluded that frequency of retinopathy in patients with SCD is more than expected and it was higher in patients who started transfusion at a later age. More attention should be paid for this problem and close observations and follow up is strongly needed.

Background: Iron overload is inevitable but a manageable complication of frequent blood transfusion, the effectiveness and safety of two iron chelation therapy in young children with transfusion-dependent B thalassemia (TDB-T) using either deferiprone (DFP) or deferasirox (DFX) were well studied in clinical trials. The primary objectives were; reporting efficacy and frequency of all adverse events (AEs) as compared with published data. Secondary objective was reporting deviation from guidelines, The analysis of clinical data is the first step to design new strategies Methodology: An investigator initiated 2-year multicenter prospective study enrolling over 6 months period; 302 children with TDB-T from 11 centers; keeping pre-transfusion Hb >8gm/dl. When serum ferritin (SF) reached around 1000 ng/ml; they had received according to availability; either DFP liquid solution in a dose; 75 mg/kg/day (n=62) or DFX (n=240); (dispersed tablet; 25% or film-coated tablet 75% in a dose; 20-25 mg/kg/d or its equivalent). CBC was done biweekly in DFP and pre-transfusion in DFX group. Liver transaminases, serum creatinine and (SF) were done /3 months. Changes in growth velocity over time were documented. Results: 66% were males, age at enrolment was; (11-19 and 14-26 months) in DFP and DFX respectively; they had received (6-8) and (7-11) transfusions prior to chelation in both groups respectively in addition to (16-21) during the study. 20% of patients did not achieve the target pre-transfusion Hb level. At initiation of DFP; SF median; 960 (IQR; 122 ng/ml) while those on DFX had SF; median 1070 (IQR 138 ng/ml) both groups had comparable transfusion iron input during the study. At 24 months of regular transfusion-chelation; SF < 1000 ng/ml was noticed in 65% and 60% of the patients respectively with good growth velocity in children using either chelator. Unnecessary over-dosing of DFX > 40 mg/kg was observed in 5%, meanwhile 3.2% had received DFP in a dose >75-100 mg/kg while SF was <1500 ng/ml. Under-reporting of adverse events in both groups was observed; 5% GIT upset, neither arthralgia nor agranulocytosis, neutropenia (3.2%) in DFP group and CBC was not checked biweekly; but only prior to transfusion. 4-5 % had elevated transaminases in both chelators, while patients on DFX reported; GIT upset in 4.2% and rash in 1.6%. Elevated serum creatinine > 33% from baseline on 2 successive visits was observed in 5% in DFX without reducing the dose or re-check. No unexpected, serious severe adverse events were reported in both groups. Poorly controlled patients with SF > 1500 ng /ml at 12 or 24 months were 12, 8% in DFP group; in contrast to 15%, 10% in DFX group respectively; with no statistical difference; (compliance was comparable). The total DFP and DFX discontinuation ratio was 5% in each group. Conclusion: infants and young children with TDB-T naïve to chelation were put on mono-therapy at younger age than reported before. Both chelators were effective, well tolerated and with no severe safety concerns; however deviation from guidelines was observed as; unnecessary over-dosing of DFX and under-reporting of AEs in both groups in real life practice. Disclosures Hamdy: ApoPharma: Honoraria; Amgen: Honoraria; Bayer: Honoraria; Novartis: Honoraria; NovoNordisk: Honoraria; Roche: Honoraria; Takeda: Honoraria.

Background Thalassemia is a genetic disorder of hemoglobin synthesis. Beta-thalassemia is particularly prevalent among Mediterranean people. Long-term transfusion therapy and iron chelating agents are the mainstay treatments of thalassemia patients. Aim The aim of this study was to identify the demographic, clinical, laboratory profile, and therapeutic features of B-thalassemia patients attending the Pediatric Hematology Clinic at Damanhour Medical Institute. Patients and methods This study included 74 thalassemia patients in the age range of 2–16 years, mostly from rural areas (el Behera Governorate). The total number of thalassemia patients followed up in the Pediatric Hematology Unit at Damanhour Medical Institute is up to 400 cases; the patients of this study were selected randomly from among them. Results Dysmorphic features were demonstrated in 44% of cases; 74% of cases were receiving packed red blood cells every month and 26% were receiving packed red blood cells more than once a month. Oral iron chelator (Deferasirox) was the main chelator used. Most of the patients had serum ferritin greater than 1000 µg/ml; thyroid dysfunction was evident in 21% of cases, with no definite clinical manifestations.