

Transfusion independence occurred in 20 of 22 patients who could be evaluated (91%), including 6 of 7 patients (86%) who were younger than 12 years of age. ResultsĪ total of 23 patients were enrolled and received treatment, with a median follow-up of 29.5 months (range, 13.0 to 48.2). The primary end point was transfusion independence (i.e., a weighted average hemoglobin level of ≥9 g per deciliter without red-cell transfusions for ≥12 months). Patients underwent myeloablation with busulfan (with doses adjusted on the basis of pharmacokinetic analysis) and received beti-cel intravenously. In this open-label, phase 3 study, we evaluated the efficacy and safety of beti-cel in adult and pediatric patients with transfusion-dependent β-thalassemia and a non–β 0/β 0 genotype.


The most trusted, influential source of new medical knowledge and clinical best practices in the world.īetibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (β A-T87Q) gene.
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Information and tools for librarians about site license offerings. Valuable tools for building a rewarding career in health care. The authorized source of trusted medical research and education for the Chinese-language medical community. The most advanced way to teach, practice, and assess clinical reasoning skills. Information, resources, and support needed to approach rotations - and life as a resident. The most effective and engaging way for clinicians to learn, improve their practice, and prepare for board exams. NEW! Peer-reviewed journal featuring in-depth articles to accelerate the transformation of health care delivery.Ĭoncise summaries and expert physician commentary that busy clinicians need to enhance patient care.
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It encourages a particular focus on client’s responses to unwanted body sensations.NEW! A digital journal for innovative original research and fresh, bold ideas in clinical trial design and clinical decision-making. The Therapy Blueprint For Panic is a relapse prevention worksheet tailored for clients who are completing a course of therapy for panic attacks or panic disorder. Therapists will find it helpful to introduce the therapy blueprint prior to the final session, and as early as the client is willing.

They act as a way of promoting resilience by reinforcing what has been learned. Therapy blueprints are one way to help clients reflect on what they have learned during therapy. Patients can be encouraged to think of it as ‘the first session of the rest of your life’. A therapy blueprint is CBT tool which summarizes the work a therapist and patient have completed together.
