METHODS: A cross-sectional survey using the EQ-5D-3L instrument was conducted between May to September 2018 across various public hospitals in Malaysia. Using a multi-stage sampling, patients diagnosed with TDT and receiving iron chelating therapy were sampled. The findings on the EQ-5D-3L survey were converted into utility values using local tariff values. A two-part model was used to examine and derive the HSUVs associated with the treatment and complications of iron overload in TDT.
RESULTS: A total of 585 patients were surveyed. The unadjusted mean (SD) EQ-5D-3L utility value for TDT patients were 0.893 (0.167) while mean (SD) EQ VAS score was 81.22 (16.92). Patients who had more than two iron overload complications had a significant decline in HRQoL. Patients who were on oral monotherapy had a higher utility value of 0.9180 compared to other regimen combinations.
CONCLUSION: Lower EQ-5D-3L utility values were associated with patients who developed iron overload complications and were on multiple iron chelating agents. Emphasizing compliance to iron chelating therapy to prevent the development of complications is crucial in the effort to preserve the HRQoL of TDT patients.
OBJECTIVES: To assess the effectiveness, safety and appropriate dose regimen of hydroxyurea in people with non-transfusion dependent beta thalassaemia (haemoglobin E combined with beta thalassaemia and beta thalassaemia intermedia).
SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Haemoglobinopathies Trials Register, compiled from electronic database searches and handsearching of relevant journals. We also searched ongoing trials registries and the reference lists of relevant articles and reviews.Date of last search: 30 April 2016.
SELECTION CRITERIA: Randomised or quasi-randomised controlled trials of hydroxyurea in people with non-transfusion dependent beta thalassaemia comparing hydroxyurea with placebo or standard treatment or comparing different doses of hydroxyurea.
DATA COLLECTION AND ANALYSIS: Two authors independently applied the inclusion criteria in order to select trials for inclusion. Both authors assessed the risk of bias of trials and extracted the data. A third author verified these assessments.
MAIN RESULTS: No trials comparing hydroxyurea with placebo or standard care were found. However, we included one randomised controlled trial (n = 61) comparing 20 mg/kg/day with 10 mg/kg/day of hydroxyurea for 24 weeks.Both haemoglobin and foetal haemoglobin levels were lower at 24 weeks in the 20 mg group compared with the 10 mg group, mean difference -2.39 (95% confidence interval - 2.8 to -1.98) and mean difference -1.5 (95% confidence interval -1.83 to -1.17), respectively. Major adverse effects were significantly more common in the 20 mg group, for neutropenia risk ratio 9.93 (95% confidence interval 1.34 to 73.97) and for thrombocytopenia risk ratio 3.68 (95% confidence interval 1.13 to 12.07). No difference was reported for minor adverse effects (gastrointestinal disturbances and raised liver enzymes). The effect of hydroxyurea on transfusion frequency was not reported.The overall quality for the outcomes reported was graded as very low mainly because the outcomes were derived from only one small study with an unclear method of allocation concealment.
AUTHORS' CONCLUSIONS: There is no evidence from randomised controlled trials to show whether hydroxyurea has any effect compared with controls on the need for blood transfusion. Administration of 10 mg/kg/day compared to 20 mg/kg/day of hydroxyurea resulted in higher haemoglobin levels and seems safer with fewer adverse effects. It has not been reported whether hydroxyurea is capable of reducing the need for blood transfusion. Large well-designed randomised controlled trials with sufficient duration of follow up are recommended.
METHODS: Lenke 1 and 2 adolescent idiopathic scoliosis (AIS) patients who underwent instrumented posterior spinal fusion (PSF) surgery from two centres between June 2014 and December 2015 were prospectively recruited into this study. The patients were grouped into Group 1 (single surgeon) and Group 2 (two surgeons). One to one matching using 'prospective propensity score-matched cohort patient sampling method' was done. The surgery was divided into six stages: stage 1-exposure, stage 2-screw insertion, stage 3-release, stage 4-correction, stage 5-corticotomies and bone grafting and stage 6-closure.
RESULTS: A total of 116 patients were recruited. Of 86 patients who were operated by the two surgeons, 30 patients were matched with 30 patients that were operated by a single surgeon. Operation duration was significantly longer in Group 1 (257.3 ± 51.4 min) compared to Group 2 (164.0 ± 25.7 min). The total blood loss was significantly higher in Group 1 (1254.7 ± 521.5 mL) compared to Group 2 (893.7 ± 518.4 mL). Total blood loss/level fused was significantly higher in Group 1 (117.5 ± 42.8 mL/level) compared to Group 2 (82.6 ± 39.4 mL/level). Group 1 had significantly higher blood loss and blood loss/level fused for stages 1, 2 and 3. Group 2 had lower incidence of allogenic blood transfusion.
CONCLUSIONS: In PSF surgery for AIS patients, two-surgeon strategy was associated with shorter operation duration, lesser blood loss and lower incidence of allogenic blood transfusion.