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Sickle Cell Disease Subjects Have a Distinct Abnormal Autonomic Phenotype Characterized by Peripheral Vasoconstriction With Blunted Cardiac Response to Head-Up Tilt
Conclusion We have shown that SCD subjects are much more likely than non-SCD subjects to have impaired cardiac, but intact peripheral responses to orthostatic stress induced by HUT. These abnormal responses are associated with low baseline cardiac parasympathetic activity, independent of hemoglobin level. The classification of autonomic phenotypes based on HUT response may have potential use for predicting disease severity, guiding and targeting treatments/interventions to alleviate the risk of adverse outcomes in SCD. Ethics Statement All experiments were conducted at Children’s Hospital Los Angeles (CHLA). The ...
Source: Frontiers in Physiology - April 10, 2019 Category: Physiology Source Type: research

Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Learning Collaborative
Sickle cell disease (SCD) has evolved from a life-threatening condition in early childhood to a chronic disease in adults [1,2]. Improved supportive care [3], use of hydroxyurea therapy [4], and regular blood transfusion therapy for primary [5] and secondary [6] stroke prevention have resulted in improved childhood clinical outcomes and survival. Approximately 99% of all children born with SCD are expected to live to adulthood [7,8]. The most feared complication in children with SCD is stroke [9].
Source: Biology of Blood and Marrow Transplantation - November 27, 2018 Category: Hematology Authors: Josu de la Fuente, Nathalie Dhedin, Tatsuki Koyama, Francoise Bernaudin, Mathieu Kuentz, Leena Karnik, G érard Socié, Kathryn A. Culos, Robert A. Brodsky, Michael R. DeBaun, Adetola A. Kassim Source Type: research

Haploidentical bone marrow transplant with post-transplant cyclophosphamide plus thiotepa improves donor engraftment in patients with sickle cell anemia: results of an international learning collaborative
Sickle cell disease (SCD) has evolved from a life-threatening condition in early childhood, to a chronic disease in adults [1,2]. Improved supportive care [3], use of hydroxyurea therapy [4], and regular blood transfusion therapy for primary [5] and secondary [6] stroke prevention, have resulted in improved childhood clinical outcomes and survival. Approximately 99% of all children born in high resource countries with SCD are expected to live to adulthood [7,8]. The most feared complication in children with SCD is stroke [9].
Source: Biology of Blood and Marrow Transplantation - November 27, 2018 Category: Hematology Authors: Josu de la Fuente, Nathalie Dhedin, Tatsuki Koyama, Francoise Bernaudin, Mathieu Kuentz, Leena Karnik, G érard Socié, Kathryn A. Culos, Robert A. Brodsky, Michael R. DeBaun, Adetola A. Kassim Source Type: research