Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial.

Background: Sickle cell disease, a debilitating, inherited haemolytic anaemia with premature morbidity and mortality, affects millions globally. Mitapivat, a first-in-class, oral, allosteric activator of pyruvate kinase, improves red blood cell survival by increasing ATP and diminishes sickling by decreasing 2,3-diphosphoglycerate. We aimed to evaluate the efficacy and safety of mitapivat in patients with sickle cell disease.

Clinical and Radiological Characteristics of Classical and Variant Type of Posterior Reversible Encephalopathy Syndrome on Prognosis Following Hematopoietic Stem Cell Transplantation in Pediatric Patients: A Single-Center Experience.

Objectives: Hematopoietic stem cell transplant is increasingly being used as a curative therapeutic option for patients with hematologic malignancies and nonmalignant diseases. Here, we aimed to determine the frequency and features of typical and atypical central variants of posterior reversible encephalopathy syndrome in children who had undergone an allogeneic hematopoietic stem cell transplant procedure for any indication at a single center.

Materials And Methods: We retrospectively analyzed 101 pediatric patients between aged 6 months and 18 years who had undergone hematopoietic stem cell transplant for any indication between 2010 and 2023.

Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options.

Background: Hemophilia B (HB), an X-linked recessive inherited bleeding disorder, exhibits a high prevalence among males.

Objectives: To present the first national cohort of persons with HB to define the demographics, clinical characteristics, and treatment patterns in Turkey.

Methods: This multicenter, retrospective study included 433 alive persons with HB registered in 35 centers between 1961 and 2018.

The outcome of relapsed childhood acute lymphoblastic leukemia after allogeneic hematopoietic stem-cell transplantations: A single-center experience.

In children with high-risk childhood acute leukemia who undergo allogeneic hematopoietic stem-cell transplantation (allo-HSCT), relapse is still the leading cause of treatment failure. The prognosis is poor, yet prospective studies have only limited data on risk factors and outcomes. We aimed to understand the outcomes and prognostic factors for patients with acute lymphoblastic leukemia (ALL) who relapsed following allo-HSCT.

Pharmacokinetics, Safety, and Efficacy of Letermovir for Cytomegalovirus Prophylaxis in Adolescent Hematopoietic Cell Transplantation Recipients.

Introduction: Letermovir is a cytomegalovirus (CMV) terminase complex inhibitor approved for prophylaxis of CMV infection and disease in adult CMV-seropositive allogeneic hematopoietic cell transplantation (allo-HCT) recipients (R+). We report pharmacokinetics (PK), safety, and efficacy of letermovir in adolescent (12-18 years) allogeneic HCT recipients from an ongoing clinical study.

Methods: In this phase 2b, multicenter, open-label study (NCT03940586), 28 adolescents received 480 mg letermovir [240 mg with cyclosporin A (CsA)] once daily orally or intravenously.

Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective, multicenter, open-label study.

Aim: To determine the immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients (PUPs) with severe hemophilia A (HA).

Methods: This prospective, phase 3 study (NCT02615691) was conducted in PUPs, or patients with ≤2 exposure days (EDs) prior to screening, aged <6 years with severe HA. The primary endpoint was incidence of factor VIII (FVIII) inhibitor development.

Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience.

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.

Effects of a supervised therapeutic exercise program on musculoskeletal health and gait in patients with haemophilia: A pilot study.

Introduction: Episodes of bleeding in patients with haemophilia (PwH) are associated with haemophilic arthropathy, limitations in physical performance, reduced quality of life (QoL), and gait disorders.

Aim: This non-randomized, controlled, interventional, prospective, single-centre pilot study aimed to assess the effects of an 8-week supervised therapeutic exercise program on musculoskeletal health, gait kinematic parameters (GKP), functional capacity, and QoL in adult PwH.

Methods: Nineteen PwH were allocated to an exercise group (n = 10) or a control group (n = 9).

The effects of manual therapy and exercises on pain, muscle strength, joint health, functionality and quality of life in haemophilic arthropathy of the elbow joint: A randomized controlled pilot study.

Introduction: Effective physiotherapy interventions are required for haemophilic arthropathy (HA) of the elbow due to its biomechanical differences and contribution to upper limb functionality.

Aim: To investigate the effects of manual therapy & exercises on bleeding frequency, pain, range of motion (ROM), strength, joint health, functionality and quality of life (QoL) in HA of the elbow.

Methods: Seventeen participants with HA of the elbow were randomized as Manual Therapy & Exercises Group (MTEG = 9) and Home Exercises Group (HEG = 8).

Impact of the HEAD-US Scoring System for Observing the Protective Effect of Prophylaxis in Hemophilia Patients: A Prospective, Multicenter, Observational Study.

Objective: This study aimed to observe the preventive effect of prophylactic treatment on joint health in people with hemophilia (PwH) and to investigate the importance of integration of ultrasonographic examination into clinical and radiological evaluation of the joints.

Materials And Methods: This national, multicenter, prospective, observational study included male patients aged ≥6 years with the diagnosis of moderate or severe hemophilia A or B from 8 centers across Turkey between January 2017 and March 2019. Patients were followed for 1 year with 5 visits (baseline and 3, 6, 9, and 12 month visits).

Umbilical Cord Management in Late Preterm and Term Infants: A Randomized Controlled Trial.

Objective: The study aimed to compare the effects of three different methods of umbilical cord management on hematological parameters in term and late-preterm infants.

Study Design: A randomized controlled trial comparing intact-umbilical cord milking (I-UCM) with cut-umbilical cord milking (C-UCM) and immediate cord clamping (ICC) in neonates born >35 weeks' gestation.

Results: A total of 587 infants were evaluated.

The effects of manual therapy on musculoskeletal system, functional level, joint health and kinesiophobia in young adults with severe haemophilia: A randomized pilot study.

Introduction: The effects of manual therapy (MT) on joint health and functional level in haemophilic arthropathy (HA) have relatively under-explored.

Aim: To investigate the effects of MT in HA of lower limb joints on musculoskeletal system, functional level, Functional Independence Score in Hemophilia (FISH), Hemophilia Joint Health Score (HJHS) and kinesiophobia.

Methods: Seventeen patients were randomized to the control group (CG = 9) and manual therapy group (MTG = 8).