1.
An Evaluation of Bone Health Parameters in Regularly Transfused Beta-Thalassemia Major Patients.
Kothimira, VK, Kumar, A, Richhele, LR, Sood, N, Gulati, A
Journal of pediatric hematology/oncology. 2020;(6):381-385
Abstract
As beta-thalassemia major patients need regular blood transfusions due to the severe hemoglobin deficiency, the occurrence of related bone defects with simultaneous fluctuations in the biochemical and hematologic parameters is seen. The hospital-based cross-sectional observational study was done to determine and correlate the bone mineral density (BMD) with biochemical parameters and hematologic parameters in 50 regularly transfused beta-thalassemia major patients of older than 6 years of age. Descriptive statistics were analyzed with SPSS version 20.0 software. A P<0.05 was considered as statistically significant. The prevalence of suboptimal BMD at lumbar spine was 86% and at femur neck was 74%. A statistically significant correlation of BMD was found with mean pretransfusion hemoglobin values, serum calcium levels, and serum vitamin D levels (P<0.05). It was concluded that continuous monitoring of the BMD, biochemical, and hematologic parameters in regularly transfused beta-thalassemia major patients may help assess the ongoing deficiencies; helping to maintain timely and regular blood transfusions with supplementation of calcium, vitamin D to ensure good bone health.
2.
Efficacy and Safety of Combined Oral Chelation With Deferiprone and Deferasirox in Children With β-Thalassemia Major: An Experience From North India.
Parakh, N, Chandra, J, Sharma, S, Dhingra, B, Jain, R, Mahto, D
Journal of pediatric hematology/oncology. 2017;(3):209-213
Abstract
OBJECTIVE A combination of desferrioxamine with either deferiprone (DFP) or deferasirox (DFX) for patients with β-thalassemia major who do not achieve negative iron balance with monotherapy has been studied widely. However, poor compliance resulting from the need for parentral administration of desferrioxamine and its cost necessicitates combining 2 oral chelators. METHODS A prospective study was conducted in patients with transfusion-dependent β-thalassemia major in a tertiary care center over 2 years. Patients on either DFP or DFX who were not improving on monotherapy over a long period and persistently maintaining serum ferritin >2500 µg/L were enrolled. Efficacy was assessed by serum ferritin levels assessed at 12 months and 2 years. Complete blood counts and liver and kidney function tests were monitored to assess the safety of the combination of drugs. RESULTS In total, 33 patients with a mean age of 12.67 years (7.5 to 17.5 y) and a mean ferritin of 4835.2394±1443.85 µg/L formed the study cohort.In total, 28 patients completed the 1-year study period; and 12 patients completed 2 years. Mean serum ferritin reduction at 1 and 2 years was 34.99%±18.13% (range, -34.36% to 56.17%) and 44.67%±13.78% (range, 22.17% to 62.74%), respectively. The combination therapy was well tolerated. CONCLUSIONS Combined oral chelation with DFP and DFX has better efficacy than either drug used alone. The combination of drugs was well tolerated and no new adverse effects were observed.