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Patients with transfusion-dependent β-thalassemia were randomly assigned to receive luspatercept (a binder for TGF-β family member ligands) or placebo. During any 12-week period, a greater percentage of patients in the luspatercept group than in the placebo group had a reduction of at least 33% (70.5% vs. 29.5%) or at least 50% (40.2% vs. 6.3%) in the transfusion requirement.

Oxidative stress is a major mechanism contributing to the progression of β-thalassemia. To assess the effect of vitamin E and N-acetyl cysteine (NAC) as antioxidant agents on total oxidative stress (TOS) status and total antioxidant capacity (TAC) in patients with transfusion-dependent β-thalassemia (TDT). In this open-label randomized controlled trial, from May to August 2019, 78 eligible patients with TDT over the age of 18 were enrolled. All patients were registered at the Thalassemia Clinic of Shiraz University of Medical Sciences in Southern Iran. Patients were randomly allocated to the NAC group (10 mg/kg/day, orally), vitamin E group (10 U/kg/day, orally), and control group. The duration of the study was 3 months. The mean age of the participants was 28.5 ± 5.1 (range: 18–41) years. At the end of the study, TOS significantly decreased only in the vitamin E group (mean difference (MD), 95% confidence interval (CI): 0.27 (0.03–0.50), P = 0.026). TAC significantly decreased in both supplemented groups at the 3rd month of treatment (NAC group: MD (95% CI): 0.11 (0.04–0.18), P = 0.002 and vitamin E group: 0.09 (0.01–0.16), P = 0.022 respectively). Hemoglobin did not significantly change at the end of the study in each group (P > 0.05). Mild transient adverse events occurred in 4 patients of the NAC group and 5 patients of the vitamin E group with no need to discontinue the treatment. Vitamin E can be a safe and effective supplement in improving oxidative stress in patients with TDT. Moreover, it seems that a longer duration of using antioxidant supplements needs to make clinical hematologic improvement in TDT patients.

Non-adherence to treatment in patients with beta-thalassemia major (BTM) presents a significant challenge in effective disease management. This study aimed to assess the effect of a therapy reminder application (app) on treatment adherence in adult patients with BTM in Mashhad in 2024. A randomized clinical trial was conducted in 2024 at a thalassemia clinic affiliated with Mashhad University of Medical Sciences (Mashhad, Iran). Participants were randomly assigned to the intervention and control groups, using permuted block randomization, with concealed allocation. The intervention group used the ThalaMe therapy reminder app for 8 weeks (February-July 2024), while the control group received standard care. Medication adherence was measured using the Morisky Medication Adherence Scale (MMAS-8) and the Chronic Disease Treatment Adherence Questionnaire (CDTAQ) before and after the intervention. Statistical analysis was conducted using SPSS software, using paired tests or Wilcoxon signed-rank tests for within-group comparisons and independent tests or Mann-Whitney U tests for between-group comparisons. P<0.05 was considered statistically significant. The study included 76 adult patients with BTM, equally distributed between the intervention and control groups (n=38 each). Baseline measurements showed no significant differences between groups in either MMAS-8 scores (P=0.75) or CDTAQ scores (P=0.11). The MMAS-8 was inversely scored, with lower scores indicating higher adherence. Following the 8-week intervention period, the group using the ThalaMe app demonstrated significantly better adherence outcomes (1.05±0.78) than the controls (2.92±1.4, P<0.001). The intervention group had significantly higher CDTAQ scores (185.5±8.07) than the control group (151.79±27.08, P<0.001). The therapy reminder app significantly enhanced medication adherence and treatment management in patients with BTM, while simultaneously enhancing patient and family engagement through counseling. IRCT20240222061079N1.

A year-long, phase 3, randomized trial involving patients with sickle cell disease showed that the median number of pain crises was 25% lower and the median number of hospitalizations was 33% lower with l -glutamine supplementation than with placebo.

No consensus exists on whether preoperative blood transfusions are beneficial in patients with sickle-cell disease. We assessed whether perioperative complication rates would be altered by preoperative transfusion. We did a multicentre, randomised trial. Eligible patients were aged at least 1 year, had haemoglobin SS or Sβ0thalassaemia sickle-cell-disease subtypes, and were scheduled for low-risk or medium-risk operations. Patients were randomly assigned no transfusion or transfusion no more than 10 days before surgery. The primary outcome was the proportion of clinically important complications between randomisation and 30 days after surgery. Analysis was by intention to treat. 67 (96%) of 70 enrolled patients—33 no preoperative transfusion and 34 preoperative transfusion—were assessed. 65 (97%) of 67 patients had the haemoglobin SS subtype and 54 (81%) were scheduled to undergo medium-risk surgery. 13 (39%) of 33 patients in the no-preoperative-transfusion group had clinically important complications, compared with five (15%) in the preoperative-transfusion group (p=0·023). Of these, 10 (30%) and one (3%), respectively, had serious adverse events. The unadjusted odds ratio of clinically important complications was 3·8 (95% CI 1·2–12·2, p=0·027). 10 (91%) of 11 serious adverse events were acute chest syndrome (nine in the no-preoperative-transfusion group and one in the preoperative-transfusion group). Duration of hospital stay and readmission rates did not differ between study groups. Preoperative transfusion was associated with decreased perioperative complications in patients with sickle-cell disease in this trial. This approach could, therefore, be beneficial for patients with the haemoglobin SS subtype who are scheduled to undergo low-risk and medium-risk surgeries. NHS Blood and Transplant.

Background Thalassemia is one of the most common genetic disorders. Patients with beta-thalassemia major confront serious clinical and psychosocial challenges in their all lives, which require coping strategies. It appears that psychological interventions are necessary to improve their coping skills. The aim of this study was to determine the effect of applying emotional intelligence components on coping strategies in adolescents with beta- thalassemia major. Methods This randomized clinical trial study involved 60 teenagers with beta- thalassemia major who were divided equally into intervention and control groups. The experimental group participated in 9 sessions of an emotional intelligence program consisting of 90 min, held both virtually and in person, two sessions per week. We investigated problem-focused and emotion-focused (including positive emotion-focused and negative emotion-focused) coping strategies of both groups of adolescents using the Billings and Moos questionnaire before the intervention, immediately after the intervention, and one month after the intervention. Data were analyzed using SPSS 21. Then, according to the research objectives, independent t-tests, Chi-square, Mann–Whitney, repeated measures Analysis of Variance (ANOVA) and Bonferroni test were used. Results In experimental group, the mean score of problem-focused (problem-solving, cognitive evaluation) and positive emotion-focused (social support) coping increased from ( 14.2  ±  2.6) and (5.0  ±  0.5) before the intervention to ( 29.6  ±  3.1) and (10.9  ±  1.3) one month after the intervention, respectively ( P  < 0.001). However, the mean score of emotional inhibition and somatic inhibition (negative emotion-focused) decreased from ( 13.8  ±  1.7 ) and ( 6.7  ±  1.5 ) before the intervention to ( 8.6  ±  2.0 ) and ( 3.8  ±  1.8 ) one month after the intervention, respectively ( P  < 0.001). While the mean score of problem-focused and emotion-focused coping strategies before and one month after the intervention remained stable in the control group. Conclusions Adolescents with beta-thalassemia suffer from psychosocial disorders and they also cope maladaptive with their illness. Applying emotional intelligence has improved their coping strategies. Caregivers should be encouraged to assess coping skills in teenagers with beta-thalassemia major and use methods such as emotional intelligence to improve them. Therefore, it can help these adolescents to deal effectively with stress and complications of the disease. Trial registration number IRCT20210521051356N1 (17/06/2021).

Hydroxyurea is an antimetabolite drug that induces fetal haemoglobin in sickle cell disease. However, its clinical usefulness in β-thalassaemia is unproven. We conducted a randomised, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of hydroxyurea in transfusion-dependent β-thalassaemia. Sixty patients were assigned 1:1 to oral hydroxyurea 10–20 mg/kg/day or placebo for 6 months by stratified block randomisation. Hydroxyurea treatment did not alter the blood transfusion volume overall. However, a significantly higher proportion of patients on hydroxyurea showed increases in fetal haemoglobin percentage (89% vs. 59%; p  < 0.05) and reductions in erythropoietic stress as measured by soluble transferrin receptor concentration (79% vs. 40%; p  < 0.05). Based on fetal haemoglobin induction (> 1.5%), 44% of patients were identified as hydroxyurea-responders. Hydroxyurea-responders, required significantly lower blood volume (77 ± SD27ml/kg) compared to hydroxyurea-non-responders (108 ± SD24ml/kg; p  < 0.01) and placebo-receivers (102 ± 28ml/kg; p  < 0.05). Response to hydroxyurea was significantly higher in patients with HbE β-thalassaemia genotype (50% vs. 0%; p  < 0.01) and Xmn1 polymorphism of the γ-globin gene (67% vs. 27%; p  < 0.05). We conclude that oral hydroxyurea increased fetal haemoglobin percentage and reduced erythropoietic stress of ineffective erythropoiesis in patients with transfusion-dependent β-thalassaemia. Hydroxyurea reduced the transfusion burden in approximately 40% of patients. Response to hydroxyurea was higher in patients with HbE β-thalassaemia genotype and Xmn 1 polymorphism of the γ-globin gene.

ObjectivesThe non-transfusion-dependent beta-thalassaemia-patient-reported outcome (NTDT-PRO) questionnaire was developed for assessing anaemia-related tiredness/weakness (T/W) and shortness of breath (SoB) among patients with NTDT. Psychometric properties were evaluated using blinded data from the BEYOND trial (NCT03342404).DesignAnalysis of a phase 2, double-blind, randomised, placebo-controlled trial.SettingUSA, Greece, Italy, Lebanon, Thailand and the UK.ParticipantsAdults (≥18 years) (N=145) with NTDT who had not received a red blood cell transfusion within 8 weeks prior to randomisation, with mean baseline haemoglobin level ≤100 g/L.MeasuresNTDT-PRO daily scores from baseline until week 24, and scores at select time points for the 36-Item Short Form Health Survey version 2 (SF-36v2), Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-F) and Patient Global Impression of Severity (PGI-S).ResultsCronbach’s alpha at weeks 13–24 was 0.95 and 0.84 for the T/W and SoB domains, respectively, indicating acceptable internal consistency reliability. Among participants self-reporting no change in thalassaemia symptoms via the PGI-S between baseline and week 1, intraclass correlation coefficients were 0.94 and 0.92 for the T/W and SoB domains, respectively, indicating excellent test–retest reliability. In a known-groups validity analysis, least-squares mean T/W and SoB scores at weeks 13–24 were worse in participants with worse scores for the FACIT-F Fatigue Subscale (FS), SF-36v2 vitality or PGI-S. Indicating responsiveness, changes in T/W and SoB domain scores were moderately correlated with changes in haemoglobin levels, and strongly correlated with changes in SF-36v2 vitality, FACIT-F FS, select FACIT-F items and the PGI-S. Improvements in least-squares mean T/W and SoB scores were higher in participants with greater improvements in scores on other PROs measuring similar constructs.ConclusionsThe NTDT-PRO demonstrated adequate psychometric properties to assess anaemia-related symptoms in adults with NTDT and can be used to evaluate treatment efficacy in clinical trials.

Non-transfusion-dependent (NTD) thalassaemia is characterised by ineffective erythropoiesis and haemolytic anaemia, leading to long-term complications, poor quality of life, and early mortality. No oral disease-modifying therapies are approved for β-thalassaemia and no agents are approved for α-thalassaemia. The objective of this study was to evaluate the efficacy and safety of mitapivat, an oral activator of pyruvate kinase, in adults with NTD α-thalassaemia or NTD β-thalassaemia. ENERGIZE is a phase 3, double-blind, randomised, placebo-controlled trial followed by an open-label extension conducted at 70 hospitals in 18 countries globally. Participants had to be aged 18 years or older with NTD α-thalassaemia or NTD β-thalassaemia and haemoglobin concentrations of 10 g/dL or lower. Participants were randomly assigned 2:1 to mitapivat or placebo (100 mg orally twice a day for 24 weeks) via a central interactive response technology system using block randomisation, stratified by baseline haemoglobin concentration and thalassaemia genotype. Everyone was masked to the patients' treatment assignment until the study was unblinded for the analysis of the primary endpoint. The primary endpoint was haemoglobin response (≥1·0 g/dL increase from baseline in mean haemoglobin concentration from week 12 through week 24), analysed in all patients who were randomly assigned. Safety was analysed in all patients who received at least one dose of study treatment. This study is registered with ClinicalTrials.gov, number NCT04770753, and is active but not recruiting. Between Nov 8, 2021, and March 31, 2023, 235 patients were screened, of whom 194 were enrolled (123 [63%] were female and 71 [37%] were male). 130 patients were randomly assigned to mitapivat and 64 patients to placebo and formed the full analysis set. One patient in each group was randomly assigned but not given treatment and was therefore excluded from the safety analysis set (mitapivat 129 patients and placebo 63 patients). Seven patients in the mitapivat group and one patient in the placebo group discontinued treatment before the end of the 24-week double-blind period. 55 (42%) of 130 patients in the mitapivat group had a haemoglobin response versus one (2%) of 64 in the placebo group (least-squares mean difference 41% [95% CI 32–50], two-sided p<0·0001). Adverse events were reported in 107 (83%) of 129 patients who received mitapivat and 50 (79%) of 63 patients who received placebo. The most commonly reported adverse events with mitapivat were headache (29 [22%] of 129 patients in the mitapivat group vs six [10%] of 63 in the placebo group), initial insomnia (18 [14%] vs three [5%]), nausea (15 [12%] vs five [8%]), and upper respiratory tract infection (14 [11%] vs four [6%]). No deaths were reported. Mitapivat could be a new oral treatment for adults with NTD α-thalassaemia or NTD β-thalassaemia by increasing haemoglobin concentration and improving fatigue. Agios Pharmaceuticals.

Transfusion-Dependent Thalassemia (TDT) causes severe anemia requiring chronic transfusions, leading to iron overload and endocrine complications, including hypothyroidism. Iron chelation therapy (ICT) mitigates iron toxicity, but its effects on thyroid function remain understudied in resource-limited settings like Pakistan. This cross-sectional study compared 100 TDT patients receiving ICT (deferasirox, deferoxamine, or deferiprone) for ≥ 6 months with 100 Control group who have not taken ICT. Thyroid function tests (free T3, free T4, TSH), serum ferritin, and hepatic/renal biomarkers were analyzed. Statistical analyses included t-tests, ANOVA, Pearson correlation, and multivariate regression (SPSS v25). ICT-treated patients had significantly lower TSH (3.10 ± 0.62 vs. 9.95 ± 3.19 μIU/mL, p  < 0.001) and higher free T3/T4 levels than controls ( p  < 0.001). Deferasirox users exhibited the lowest TSH (2.43 ± 0.06 μIU/mL) among chelators ( p  < 0.001). Ferritin strongly correlated with TSH (r = 0.94, p  < 0.001) and independently predicted TSH levels (coefficient = 0.0010, p  = 0.048). ICT also preserved hepatic (ALT: 50.55 ± 41.87 vs. 291.36 ± 161.99 U/L, p  < 0.001) and renal function (creatinine: 0.97 ± 0.22 vs. 1.65 ± 0.34 mg/dL, p  < 0.001). ICT, particularly deferasirox, protects against thyroid dysfunction in TDT, with ferritin as a key predictor. These findings support personalized chelation strategies and routine endocrine monitoring in thalassemia management.