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Given that appropriate long-term iron chelation is essential in the treatment of transfusion-dependent anaemias,14,15 deferiprone b.i.d. has the potential to improve treatment adherence and health outcomes in patients with transfusional iron overload. Twenty-seven patients had a primary diagnosis of thalassaemia major, one patient had -thalassaemia/haemoglobin H disease and one patient had sickle -thalassaemia. Fourteen patients reported mild AEs, 13 patients reported moderate AEs and one patient reported a severe AE. Lifelong iron chelation therapy is critical for patients with transfusion-dependent anaemias, and full adherence to therapy is essential to optimise long-term patient outcomes.1,2 Deferiprone is an oral iron chelator with high efficiency in binding and removing excess intracellular and extracellular iron.3,4 Owing to its elimination half-life of approximately 2 h, deferiprone is administered three times daily (t.i.d.) to promote longer extent of exposure and better control of labile iron.5-7 Clinical trial data show adherence rates with deferiprone t.i.d. from 79% to 98%8,9; however, real-world adherence is generally lower than in clinical trials, as the t.i.d. regimen may be inconvenient and the midday dose may often be missed.10-12 The United States Food and Drug Administration recently approved a twice-a-day modified-release formulation of deferiprone (Ferriprox TAD 1000 mg tablet; manufactured by Apotex Inc., Toronto, Ontario, Canada) for the treatment of patients with transfusional iron overload due to thalassaemia syndromes, sickle cell disease or other anaemias.13 In single-dose and multiple-dose pharmacokinetic studies of deferiprone in healthy volunteers (Tables S1 and S2), we found that the twice-daily (b.i.d.) formulation had equivalent 24-h drug exposure to the original immediate-release tablet administered t.i.d. (Figure S1), and exposure was not affected by administration with food (Table S3). [Extracted from the article]

Control haemoglobinopathy blood safety project Keywords: sickle cell disease; thalassaemia; vaccines; iron overload; hepatitis EN sickle cell disease thalassaemia vaccines iron overload hepatitis e160 e164 5 11/30/21 20211201 NES 211201 Non-concordance with preventive recommendations is common among patients with chronic conditions, including sickle cell disease (SCD) and transfusion-dependent thalassaemia (TDT).1,2 Iron overload is common among chronically transfused patients with SCD and TDT leading to increased morbidity, mortality and cost of care, especially as they live longer.3-5 The Centers for Disease Control and Prevention (CDC) Advisory Committee on Immunization Practices (ACIP) recommendations support completion of hepatitis A (HepA) and hepatitis B (HepB) vaccinations, including SCD and TDT.6,7 Cardiac iron overload is the leading cause of mortality in TDT,8 while it is rare in SCD.9 Guidelines recommend liver iron overload assessment every 1-2 years for patients with SCD receiving chronic transfusions.10,11 The recommendation for TDT is annual assessment of liver and cardiac iron concentration.3,8 However, there are limited data on concordance with these recommendations. Child/adolescent patients with TDT who were concordant with HepA vaccination recommendations were concordant with the other two preventive service recommendations (Fig 1B). [Extracted from the article]

Summary: Depressive symptoms are prevalent in individuals living with sickle cell disease (SCD) and may exacerbate pain. This study examines whether higher depressive symptoms are associated with pain outcomes, pain catastrophizing, interference and potential opioid misuse in a large cohort of adults with SCD. The study utilized baseline data from the 'CaRISMA' trial, which involved 357 SCD adults with chronic pain. Baseline assessments included pain intensity, daily mood, the Patient Health Questionnaire (PHQ), the Generalized Anxiety Disorders scale, PROMIS Pain Interference, Pain Catastrophizing Scale, the Adult Sickle Cell Quality of Life Measurement Information System and the Current Opioid Misuse Measure. Participants were categorized into 'high' or 'low' depression groups based on PHQ scores. Higher depressive symptoms were significantly associated with increased daily pain intensity, negative daily mood, higher pain interference and catastrophizing, poorer quality of life and a higher likelihood of opioid misuse (all p < 0.01). SCD patients with more severe depressive symptoms experienced poorer pain outcomes, lower quality of life and increased risk of opioid misuse. Longitudinal data from this trial will determine whether addressing depressive symptoms may potentially reduce pain frequency and severity in SCD. [ABSTRACT FROM AUTHOR]

Summary: Sickle cell disease (SCD) is an inherited red blood cell disorder associated with frequent painful events and organ damage. Hydroxyurea (HU) is the recommended evidence‐based treatment of SCD. However, among patients eligible for HU, prescription rates are low. Utilizing a scoping review approach, we summarized and synthesized relevant findings regarding provider barriers and facilitators to the prescription of HU in youth and adults with SCD and provided suggestions for future implementation strategies to improve prescription rates. Relevant databases were searched using specified search terms. Articles reporting provider barriers and/or facilitators to prescribing HU were included. A total of 10 studies met the inclusion criteria. Common barriers to the prescription of HU identified by providers included: doubts around patients' adherence to HU and their engaging in required testing, concerns about side effects, lack of knowledge, cost and patient concerns about side effects. Facilitators to the prescription of HU included beliefs in the effectiveness of HU, provider demographics and knowledge. Findings suggest significant provider biases exist, particularly in the form of negative perceptions towards patients' ability to adhere to taking HU and engaging in the required follow‐up. Improving provider knowledge and attitudes towards HU and SCD may help improve low prescription rates. [ABSTRACT FROM AUTHOR]

Summary: It is not known whether obesity has a differential effect on allogeneic haematopoietic cell transplantation outcomes with alternative donor types. We report the results of a retrospective registry study examining the effect of obesity [body mass index (BMI) > 30] on outcomes with alternative donors (haploidentical related donor with two or more mismatches and receiving post‐transplant cyclophosphamide [haplo] and cord blood (CBU)] versus matched unrelated donor (MUD). Adult patients receiving haematopoietic cell transplantation for haematologic malignancy (2013–2017) (N = 16 182) using MUD (n = 11 801), haplo (n = 2894) and CBU (n = 1487) were included. The primary outcome was non‐relapse mortality (NRM). The analysis demonstrated a significant, non‐linear interaction between pretransplant BMI and the three donor groups for NRM: NRM risk was significantly higher with CBU compared to haplo at BMI 25–30 [hazard ratio (HR) 1.66–1.71, p < 0.05] and MUD transplants at a BMI of 25–45 (HR, 1.61–3.47, p < 0.05). The results demonstrated that NRM and survival outcomes are worse in overweight and obese transplant recipients (BMI ≥ 25) with one alternative donor type over MUD, although obesity does not appear to confer a uniform differential mortality risk with one donor type over the other. BMI may serve as a criterion for selecting a donor among the three (MUD, haplo and CBU) options, if matched sibling donor is not available. [ABSTRACT FROM AUTHOR]