Patient blood management (PBM) is an evidence-based, multidisciplinary approach aimed at optimizing the care of patients who might require transfusion. While PBM has been widely adopted in adult practice, its application in pediatric settings remains limited and inconsistent despite the unique physiological and clinical challenges of this population. This review highlights the current strategies and future directions of PBM in pediatric patients. Key elements of pediatric PBM include the preservation of red cell mass and the management of preoperative anemia, strategies to minimize iatrogenic and surgical blood loss, approaches to enhance patients’ physiological tolerance to anemia, and optimization of transfusion practices. Recent studies support the use of restrictive transfusion thresholds for critically ill children, neonates, and specific high-risk groups such as those with congenital heart disease, demonstrating no added benefit of liberal transfusion practices. Looking ahead, advances in precision medicine, artificial intelligence, and non-invasive monitoring technologies are expected to further individualize transfusion practices and strengthen PBM implementation in pediatric care. However, high-quality pediatric-specific research remains essential to establish standardized protocols and ensure safe, effective, and patient-centered blood management.

Objective: Concerns about excessive non-relapse mortality (NRM) are a major issue following allogeneic hematopoietic cell transplantation (HCT). Although the HCT-Specific Comorbidity Index (HCT-CI) was established as a stratification model for NRM following allogeneic HCT, the Composite Health Risk Assessment Model (CHARM) score was also developed to predict the risk of NRM and overall mortality following allogeneic HCT from adult donors, particularly in older patients. The CHARM score has been shown to predict these outcomes better than the HCT-CI alone. However, the prognostic value of the CHARM score has not been validated in adult patients undergoing unrelated singleunit cord blood transplantation (CBT). This study aimed to address that gap in the research.
Materials and Methods: We retrospectively validated the impact of the CHARM score on transplant outcomes in 321 adults who underwent unrelated single-unit CBT at our institution.
Results: In univariate analysis, a higher CHARM score was significantly associated with worse overall mortality (p<0.001), higher relapse (p=0.007), and NRM (p=0.048). In multivariate analysis, the rates of overall mortality (hazard ratio [HR]: 1.56, 95% confidence interval [CI]: 1.06-2.29, p=0.022) and relapse (HR: 1.71, 95% CI: 1.09-2.69, p=0.020) were significantly higher in patients with higher CHARM scores, but NRM was not (HR: 1.17, 95% CI: 0.68-1.99, p=0.560). The detrimental effects of higher CHARM scores on overall mortality and relapse compared to lower CHARM scores were observed in subgroups of patients with high and very high risk, as defined by the refined Disease Risk Index.
Conclusion: In contrast to previous research, this study revealed that the CHARM score was able to predict overall mortality and relapse, but not NRM, in adults undergoing single-unit CBT.

Objective: Transfusion therapy is critical for many patients with β-thalassemia or sickle cell disease (SCD). We aimed to review current practices and document chronic transfusion therapy for patients with hemoglobinopathies in the transfusion service centers of Türkiye.
Materials and Methods: A survey with 16 structured questions was distributed electronically to adult and pediatric hematologists in Türkiye. Responses were received from 37 centers across 18 cities, representing 1449 patients diagnosed with β-thalassemia major, β-thalassemia intermedia, and SCD.
Results: Although 79% of centers reported performing extended red cell antigen typing prior to the first transfusion, adherence to national transfusion guidelines was inconsistent. Only 16% of centers routinely performed indirect antiglobulin testing before each transfusion despite guideline recommendations. Antibody identification capabilities varied, with 26% of centers lacking the capability onsite. Elution and adsorption testing were always performed at 13% of centers only, predominantly including university hospitals. Nearly half of the centers were always able to provide D, C, E, c, e, and Kell compatible red cell units, but one-fourth reported that they were unable to consistently provide compatible units due to limited supply. There was no access to red cell genotyping in the country.
Conclusion: Our survey revealed disparities in transfusion practices and transfusion service laboratory infrastructure across Türkiye. There is a need for national policy initiatives to mandate adherence to national and international guidelines, expand immunohematology testing capabilities, and ensure the equitable distribution of phenotypematched red cell units. These findings will contribute to discussions on establishing a centralized immunohematology reference laboratory and enabling red cell genotyping within the country to improve transfusion safety and health equity in hemoglobinopathy care.

Objective: This study aimed to investigate the clinical manifestations, treatment patterns, and clinical outcomes of patients with immune thrombotic thrombocytopenic purpura (iTTP) across Türkiye via an interim analysis of the Turkish iTTP Registry.
Materials and Methods: A total of 215 patients with iTTP (median age at diagnosis: 41 years; 58.6% female) diagnosed between 2001 and 2023 were retrospectively analyzed in the interim analysis of a prospective non-interventional observational multicenter iTTP registry study (ClinicalTrials.gov Identifier: NCT05950750) conducted at 19 tertiary hematology centers. Data on patient demographics, disease characteristics at initial admission, treatment characteristics and responses, exacerbations/relapses, and survival outcome were obtained from electronic case report forms.
Results: Infection (15.0%), new drug initiation (9.7%), and pregnancy/ postpartum period (6.3%) within 3 weeks before diagnosis were the most prevalent potential triggers. Patients presented most commonly with systemic/constitutional (fatigue: 68.8%; fever: 18.1%) and neurological (headache: 40.0%; vertigo: 32.1%) symptoms, followed by hemorrhagic, gastrointestinal, renal, and cardiovascular manifestations. Based on PLASMIC risk scoring, 77.8% of patients were initially at high risk for TTP. The initial treatment was begun within the first 48 hours of hospital admission for 64.1% of patients (36.2% on the day of admission). Treatment was mainly based on therapeutic plasma exchange (92.1%) and steroids (63.7%), while rituximab was used in 15.8% of patients. The clinical response rate was 79.9% and clinical remission was achieved by 68.2% of patients. Regarding a thrombospondin type 1 motif member (ADAMTS13) 13 levels, partial and complete responses were achieved by 17.7% and 14.6%, respectively. During a median of 30 months (range: 0.1-262.4 months) of follow-up, 35 patients experienced exacerbations/relapses. Mortality occurred in 11 (5.5%) patients and was found to be diseaserelated in 6 cases (3.0%).
Conclusion: This interim analysis of the nationwide Turkish iTTP Registry study provides valuable data on real-world clinical practices in the diagnosis and management of iTTP at different hematology clinics across the country.

Objective: Eltrombopag (EPAG) added to standard immunosuppressive therapy (IST) has been associated with higher overall response (OR) and complete response (CR) rates in adult patients with treatmentnaïve severe aplastic anemia (SAA), but clinical evidence on the efficacy of EPAG in children with acquired aplastic anemia is limited and controversial. This retrospective study aimed to determine the efficacy and safety of EPAG combined with IST in pediatric patients with SAA compared to a standard IST group.
Materials and Methods: We compared the efficacy and safety of EPAG combined with IST (n=38) versus IST alone (n=57) as frontline treatment for pediatric patients with SAA.
Results: The EPAG+IST group had higher CR and OR rates at 3 and 6 months, although the 1-year OR, CR, and partial response rates showed no significant difference between the two groups. Older age at diagnosis (>8.95 years) was associated with a higher OR rate at 6 months and 1 year in the EPAG+IST group (p=0.007 and p=0.005, respectively). The addition of EPAG to IST did not achieve superiority over IST alone in terms of overall survival (OS) and event-free survival (EFS) in this study, with 1-year EFS of 81.1% for EPAG+IST and 71.3% for IST, and 1-year OS of 89.2% versus 80.4%, respectively.
Conclusion: EPAG+IST induced a faster response compared to IST alone without increasing toxic effects, but EPAG did not confer additional benefits regarding OS or relapse rates in children. Notably, older age at diagnosis was significantly associated with improved response rates in the EPAG+IST group.