Children’s Hospital at TriStar Centennial

Recent papers from Children’s Hospital at TriStar Centennial

1. Is the use of GCSF to mobilize peripheral blood stem cells in sickle cell disease the answer to poor mobilization? Not so fastBritish Journal of Haematology · 2026
2. Risks and benefits of gene therapy and transplantation in sickle cell disease. Comment on: ,HLA-haploidentical hematopoietic stem cell transplantation in patients with sickle cell disease: results from the phase II DREP-HAPLO trial-Haematologica · 2026
3. Implementing a First-in-Human Clinical Trial of Prime-Editing Gene Therapy in p47-CGD: Clinical Data and Site PerspectivesJournal of Human Immunity · 2026
4. Patient and Caregiver Perceptions of Acute Seizure Medications and the Rapid and Early Seizure Termination (REST) Approach: Qualitative Interviews (P4-11.003)Neurology · 2026
5. Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell DiseaseNew England Journal of Medicine · 2026
6. Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion‐Dependent β‐ThalassemiaAmerican Journal of Hematology · 2026
7. Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemiaBlood · 2026
8. Consensus Recommendations for CAR T-Cell Administration in Adult Acute Lymphoblastic Leukemia: a Modified Delphi StudyBlood · 2026
9. Addressing Barriers to Transitioning Pediatric Patients With Epilepsy to Adult Health Care in the United StatesNeurology Clinical Practice · 2026
10. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-ThalassemiaNew England Journal of Medicine · 2026
11. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell DiseaseNew England Journal of Medicine · 2026
12. Base Editing of HBG1 and HBG2 Promoters for Sickle Cell DiseaseNew England Journal of Medicine · 2026
13. Granulomatosis With Polyangiitis Presenting With Initially Isolated Cutaneous Facial Ulceration Without Early Other Characteristic ChangesAnnals of Internal Medicine Clinical Cases · 2026
14. Tiered Fixed-Dose Plerixafor Resulted in Enhanced CD34+ Cell Mobilization and Collection with a Comparable Safety Profile Versus Weight-Based Plerixafor in Patients with Sickle Cell Disease: Results from the Ongoing BEACON Study of Autologous CD34+ Base-Edited Hematopoietic Stem Cells (BEAM-101)Transplantation and Cellular Therapy · 2026
15. Blinatumomab Nonresponse Correlates with Poor Survival After BrexucabtageneBlood · 2026
16. Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121Blood · 2025
17. Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON studyBlood · 2025
18. Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (BEAM-101) in the ongoing BEACON studyBlood · 2025
19. First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101Blood · 2025
20. Prime Editing for p47 phox -Deficient Chronic Granulomatous DiseaseNew England Journal of Medicine · 2025