We performed a systematic review and meta‐analysis of randomized controlled trials to assess the efficacy and safety of the novel, ultra‐rapid‐acting insulins aspart and lispro in adults with type 1 or type 2 diabetes. Our primary outcome was change in HbA1c from baseline. We additionally assessed eight efficacy and six safety endpoints. We calculated weighted mean differences (WMD) for continuous outcomes and odds ratios (ORs) for dichotomous outcomes, alongside 95% confidence intervals (CIs). We additionally assessed statistical heterogeneity among studies with the I ² statistic, considering values greater than 60% as indicative of substantial heterogeneity. Nine studies comprising 5931 patients were included in the systematic review; eight active‐controlled studies could be synthesized in terms of a meta‐analysis. Treatment with ultra‐rapid‐acting insulins had a similar effect on change in HbA1c compared with rapid‐acting insulins (WMD −0.02%, 95% CI −0.08 to 0.05, I ² = 61% for patients with type 1 diabetes and −0.02%, 95% CI −0.09 to 0.04, I ² = 19% for patients with type 2 diabetes). Similarly, no difference was evident in terms of change in fasting plasma glucose, self‐measured plasma glucose, body weight, basal or bolus insulin dose, incidence of serious adverse events and hypoglycaemia. Compared with rapid‐acting insulins, ultra‐rapid‐acting insulins reduced 1‐ and 2‐hour postprandial glucose (PPG) increment based on a liquid meal test, both in patients with type 1 and type 2 diabetes (WMD −0.94 mmol/L, 95% CI −1.17 to −0.72, I ² = 0% and −0.56 mmol/L, 95% CI −0.79 to −0.32, I ² = 0%, respectively, for change in 1‐hour PPG increment). In conclusion, ultra‐rapid‐acting insulins were as efficacious and safe as rapid‐acting insulins, showing a favourable effect solely on PPG control.

Summary: Luspatercept has been approved for the treatment of anaemia in transfusion‐dependent (TD) patients with lower risk (LR) myelodysplastic syndromes (MDS) after erythroid‐stimulated agent (ESA) failure, according to the results of the MEDALIST trial. In this multicentre retrospective study, we report efficacy and safety data of luspatercept administered in 98 TD LR‐MDS patients after ESA failure. The percentage of patients that stopped luspatercept due to adverse events was comparable to that reported in the MEDALIST study. Furthermore, we observed that 44.3% patients who had completed 24 weeks of follow‐up achieved transfusion independence lasting longer than 8 weeks, compared to 38% in the MEDALIST trial. These positive results may be attributed to the inclusion of patients with lower transfusion needs in our study. All responses were observed within 8 months since luspatercept onset and many were long‐lasting, even in the high‐transfusion burden patient group. In addition, response to luspatercept and the presence of less than two mutations independently predicted for longer overall survival. Overall, our results confirm luspatercept's safety and efficacy in TD LR‐MDS patients who have experienced ESA failure in a real‐world setting.

The assessment of health-related quality of life (HRQoL) in thalassemia offers a holistic approach to the disease and facilitates better communication between physicians and patients. This study aimed to evaluate the HRQoL of transfusion-dependent thalassemia (TDT) patients in Greece. This was a multicentric, cross-sectional study conducted in 2017 involving 283 adult TDT patients. All participants completed a set of two QoL questionnaires, the generic SF-36v2 and the disease-specific TranQol. Demographic and clinical characteristics were used to predefine patient subgroups. Significant factors identified in the univariate analysis were entered into a multivariate analysis to assess their effect on HRQoL. The SF-36 scores of TDT patients were consistently lower compared to the general population in Greece. The mean summary score of TranQol was relatively high (71 ± 14%), exceeding levels observed in national surveys in other countries. Employment emerged as the most significant independent factor associated with better HRQoL, whereas age had the most significant negative effect. This study represents the first comprehensive QoL assessment of a representative sample of the TDT population in Greece. The implementation of TranQol allowed for the quantification of HRQoL in Greece, establishing a baseline for future follow-up, and identifying more vulnerable patient subgroups.