Background. The aim of this study is to examine the thickness of choroidal, macular and peripapillary retinal nerve fiber layer by spectral-domain optical coherence tomography (SD-OCT) in pediatric patients with sickle cell anemia (SCA) without retinopathy. Methods. A total of 75 children (30 SCA patients (Group 1) and 45 healthy individuals (Group 2) were included in the study. Macular (central, superior, inferior, nasal, temporal), choroidal (subfoveal, at nasal distances from the central fovea of 1000 μm [N1], 2000 μm [N2], 3000 μm [N3], at temporal distances from the central fovea of 1000 μm [T1], 2000 μm [T2], 3000 μm [T3]) and RNFL (average, temporal, superotemporal, inferotemporal, nasal, inferonasal and superonasal) measurements were performed by SD-OCT. These parameters were compared with healthy children with similar demographic characteristics. Results. The mean age was 14.11±3.86 (11-18) in sickle cell anemia patients and 13.15± 2.69 (10-18) in the healthy control group. Of the patients, 56.6% (n=17) of Group 1 and 44.4% (n=20) of Group 2 were male. Choroidal measurements made in the subfoveal, N1, N2, N3, T1, T2 and T3 quadrants showed that the choroid was thinner in 6 quadrants in SCA patients compared to the healthy group (p = 0.003, p = 0.039, p = 0.035, p = 0.595, p = 0.006, p = 0.005, p = 0.047, respectively). In RNFL measurements, there was significant thinning in the temporal, inferotemporal, and nasal quadrants of SCA patients compared to the healthy group. Changes in other quadrants were not significant. Conclusions. SD-OCT is a useful imaging method in the diagnosis and screening in patients with SCA without retinopathy. Early diagnosis of retinopathy during subclinical disease will prevent visual loss in these patients.
Objective: The current iron chelation therapy regimens include deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX) in transfusion-dependent patients. Compliance with iron-chelating therapy is one of the significant determinants of mortality and morbidities related to iron loading in chronically transfused patients. This survey aims to compare the compliance and the satisfaction of DFO, DFP, and DFX formulations in patients who have iron-overloading in three hematology centers from Turkey. Moreover, bioequivalent (generic) formulations of dispersible DFX tablet compared with the original formulation in terms of taste and treatment compliance. Patients and Methods: A written questionnaire with a list of pre-set questions was applied to measure patient-reported outcomes to a total of 85 patients, where 77 had beta-thalassemia major, 7 had beta-thalassemia intermedia, and 1 had sickle cell anemia diagnoses. Results: The patients’ median age at enrollment was 15 years (range 7 – 42). The compliance was below 50% in 8 (18.6%), 4 (16%), and 5 (6.7%) in patients receiving DFO, DFP, and DFX, respectively. Additionally the compliance was below 80% in 16 (37.2%), 9 (36%), and 17 (22.6%) in patients receiving DFO, DFP, and DFX, respectively. It was found that 39 (47%) patients had compliance problems due to the dispersible DFX tablet formulations’ taste, except combination therapies. There was no difference between the currently used oral chelators in terms of taste and treatment compliance. Conclusion: This study draws attention to compliance problems in patients with iron-loading anemias, partly due to the unpleasant taste of DFX. Improving patient satisfaction and compliance with iron-chelator therapy may reduce complications of iron overload.
nor chimerism and transplantation-related complications were collected prospectively. Results: Data of 4 transplanted patients are described here and results of 3 recently transplanted patients will be available for presentation. All 4 patients had successful engraftment. Three patients reached a donor myeloid chimerism of 100% at 1 month and remained stable in the following months (Figure). Patient #4 reached a donor myeloid chimerism of 69% at 1 month which increased to 75% at 3 months. CD3 T-cell chimerism was 79%, 48%, 70% and 23% in patients #1, 2, 3 and 4 respectively, and increased further over time. The percentages of donor myeloid and T-cell chimerism are higher than previously reported with alemtuzumab/ TBI only. All patients had a corrected SCD phenotype with normalized Hb levels at 3 months (mean ± SD 9,7 ± 1,2 g/dL to 12,7 ± 1,3 g/dL) with no reports of VOC. Importantly, there was no evidence of GvHD. Treatment-related complications were mTOR inhibitor-associated stomatitis (n = 3) responding well to local steroids, mild arthralgia (n = 2), and progression of preexistent chronic kidney injury in patient #3. Summary/Conclusion:Azathioprine/hydroxyurea preconditioning prior to alemtuzumab/TBI is well tolerated and in this small series of patients resulted in excellent donor chimerism and resolution of SCD. Longer term results of a larger cohort need to be awaited to determine the place of azathioprine/ hydroxyurea preconditioning in the setting of non-myeloablative MSD transplantation.
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