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Real-world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center.
Pediatr Investig
December 2024
Hematology Department, Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, Key Laboratory of Major Diseases in Children, National Center for Children's Health National Key Discipline of Pediatrics (Capital Medical University), Ministry of Education, Beijing Children's Hospital, Capital Medical University Beijing China.
Importance: Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China.
Objective: To explore the possibility of using reduced-dosage EMI in Chinese HA children.
Vitamin D in patients with low tumor-burden indolent non-Hodgkin lymphoma treated with rituximab therapy (ILyAD): a randomized, phase 3 clinical trial.
EClinicalMedicine
December 2024
Department of Lymphoma and Myeloma, MD Anderson Cancer Center, Houston, TX, USA.
Background: There is a significant association between low vitamin D levels at diagnosis of indolent B-cell lymphomas and inferior overall survival (OS). To determine whether supplemental vitamin D improves event-free survival (EFS) in these patients, we conducted a comparative double-blind study of vitamin D vs. placebo.
View Article and Find Full Text PDFBAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.
Eur J Haematol
December 2024
Sheba Medical Center, The Israeli National Hemophilia Center, The Amalia Biron Thrombosis Research Institute and The Sackler Medical School, Tel Aviv University, Tel-Hashomer, Israel.
Objectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.
Methods: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82).
ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B.
Eur J Haematol
December 2024
CRH, CRC-MHC (Centre de Référence de l'Hémophilie, Centre de Ressource et de Compétence Des Maladies Hémorragiques Constitutionnelles), University Hospital, Rennes, France.
Objectives: To assess the real-world efficacy and safety of recombinant factor IX albumin fusion protein (rIX-FP) in patients with hemophilia B (HB) in France.
Methods: Data on dosing frequency, weekly consumption, and bleeds before-and-after switching to rIX-FP, were collected from December 2021 to February 2024. Annualized (spontaneous) bleeding rates [A(s)BRs] were calculated only in patients on prophylaxis with a follow-up ≥ 6 months.
Background: To evaluate the safety and efficacy of low-dose roxadustat combined with low-dose recombinant human erythropoietin (rhEPO) for the treatment of renal anemia in hemodialysis patients.
Materials And Methods: We retrospectively reviewed the medical records of hemodialysis patients with moderate renal anemia between December 2019 and July 2023 from two medical centers. Patients were classified into 3 groups: rhEPO (150 - 300 IU/kg/week), roxadustat (1.
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