Thalassemic syndromes are characterized by clinical heterogenicity. For severe disease forms, lifelong blood transfusions remain the mainstay of therapy, while iron overload monitoring and adequate chelation treatment are required in order to ensure effective disease management. Compared to previous chelators, the new deferasirox film-coated tablet (DFX FCT) is considered to offer a more convenient and well-tolerated treatment scheme, aiming at better treatment-related and patient-related outcomes. The present study’s objective was to prospectively evaluate the safety and efficacy of DFX FCT in children and adolescents with transfusion-dependent thalassemia. Data collected included patient demographics, hematology and biochemistry laboratory work up, magnetic resonance imaging of heart and liver for iron load, as well as ophthalmological and audiological examination prior to and a year following drug administration. Study results confirmed DFX FCT safety in older children in a manner similar to adults, but demonstrated increased frequency of adverse events in younger patients, mainly, involving liver function. With regards to efficacy, study results confirmed the preventive role of DFX FCT in iron loading of liver and heart, however, higher doses than generally recommended were required in order to ensure adequate chelation.

Thalassemia treatment still relies on supportive care, mainly including blood transfusion and iron chelation therapy. Iron chelation is considered the main factor responsible for the marked improvement in survival rates of thalassemic patients. Hemosiderosis may be prevented if appropriate chelation therapy is offered from early childhood, with timely dose adjustments according to changing body weight and close monitoring of organ iron load. With three iron chelators currently available, the choice of appropriate chelation, either as monotherapy or combined therapy, should be individualized depending on the iron overload of target organs, patient’s age, presence of adverse events and compliance issues, given known limitations related to each agent’s administration.

Hemophilia is characterized by bleeding diathesis, primarily affecting the joints. Prophylactic use of missing factor aims at limiting the number of bleeds and, in the long term, the risk of permanent joint damage. However, standard prophylactic regimens are usually applied empirically, not adjusting for variations in bleeding phenotype or drug metabolism. Aim of the present study was to evaluate the need for individualizing prophylaxis, with guidance of pharmacokinetic (PK) studies and joint ultrasound in a setting of everyday clinical practice. To evaluate adequacy of applied regimens, joint status was assessed using the Hemophilia Joint Health Score as well as ultrasound imaging, while PK studies were performed using the Web-Accessible Population Pharmacokinetic Service—Hemophilia. Imaging results were consistent with early joint damage in a large proportion of pediatric patients, whereas PK measures were indicative of inadequate prophylaxis in many cases—despite the limited number of bleeds reported by patients. The study revealed the need for prophylaxis adjustment in the majority of patients. Real world data confirm that traditional prophylaxis is often unable to achieve therapeutic goals, while an individualized approach, guided by the use of novel modalities, may be of great benefit to young hemophilia patients.

Thromboembolism (TE) complicates the course of pediatric cancer in a considerable number of cases. Cancer-related TE is attributed to an interaction of the underlying malignancy, the effects of therapy, and a possible thrombophilia predisposition. More specifically, recognized risk factors include a very young age and adolescence, non-O blood group, type and site of cancer, inherited thrombophilia, presence of central venous catheter, and type of chemotherapy. TE in children with cancer most commonly occurs in their extremities. In the absence of evidence-based guidelines for the management of thrombotic complications in pediatric oncology patients, TE management follows general recommendations for the management of pediatric TEs. Given the limitations of conventional anticoagulant therapy, direct oral anticoagulants could provide an alternative; however, their safety and efficacy in children with cancer remain to be seen. As for thromboprophylaxis, numerous studies have been conducted, albeit with conflicting results. Although the survival of pediatric oncology patients has significantly improved in recent years, morbidity due to cancer-related TE remains, underlying the need for large multicenter trials investigating both TE management with currently available agents and primary prevention.

Hemophilia is no longer regarded solely as a male disorder; female carriers are increasingly attracting the attention of the scientific community due to the common, albeit overlooked, bleeding tendency associated with their condition. Moreover, women can, in rare cases, experience disease severity comparable to that of hemophilic males. The overall bleeding phenotype in females is complicated by gynecological and obstetric bleedings, which do not always correlate directly to their factor levels. As a patient population, they remain significantly underdiagnosed and inadequately treated due to a range of contributing factors. In genetic terms, a carrier exhibits no clinical or laboratory pathology. In that context, the term “hemophilia carrier” has historically led to the presumption of normal hemostasis, thereby impeding the provision of appropriate healthcare services to females in need. Females are not represented in clinical trials and even hemophilia centers lack sufficient experience and appropriate infrastructure to effectively manage and monitor females with hemophilia. Emerging data regarding the needs of this population should be enriched by larger studies, and previously established management practices should be reevaluated, potentially incorporating newer therapeutic options.

Introduction: Extended half-life (EHL) factor VIII (FVIII) products aim to reduce treatment burden and improve bleeding control in hemophilia A. Real-world evidence remains essential to complement clinical trials. Aim: To evaluate clinical outcomes following switching from standard half-life (SHL) rFVIII to efmoroctocog alfa in routine clinical practice in Greece. Methods: Multicenter observational pre–poststudy including patients with moderate to severe hemophilia A. Outcomes were assessed during the 12 months before and after switching. The primary endpoint was change in annualized bleeding rate (ABR). Secondary endpoints included annualized joint bleeding rate (AjBR), infusion frequency, joint health, pain, and FVIII consumption. Results: Sixty patients were included. Following switching, ABR decreased from 6.8 to 3.2 (53%), and AjBR from 6.4 to 2.9 (55%), p < 0.001. Reductions were more pronounced in patients switching from on-demand treatment, while more modest improvements were observed among patients already on prophylaxis. HJHS significantly decreased from 17.9 to 11.5 (p < 0.007), accompanied by a decrease in pain scores (p < 0.001), in available paired subsets. Weekly infusion frequency decreased (3.2 to 2.2; p < 0.001), while mean dose per infusion increased, resulting in no consistent reduction in total annual FVIII consumption. No inhibitor or treatment-related adverse events have been observed. Conclusions: Switching to efmoroctocog alfa in routine practice was associated with improved bleeding outcomes, reduced infusion frequency, and better joint-related parameters. These findings support the real world feasibility and clinical utility οf EHL FVIII therapy, while further controlled studies are needed to better define the independent effect of product switching from changes in treatment regimen and other potential confounders.