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Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: State of the Science
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Source: European Journal of Haematology - September 1, 2014 Category: Hematology Authors: Julie‐An Talano, Mitchell S. Cairo Tags: Review Article Source Type: research

Smoothing the crescent curve: sickle cell disease.
Authors: Talano JA, Cairo MS Abstract Sickle cell disease (SCD) is an inherited disorder secondary to a point mutation at the sixth position of the beta chain of human hemoglobin that results in the replacement of valine for glutamic acid. This recessive genetic abnormality precipitates the polymerization of the deoxygenated form of hemoglobin S that induces a major distortion of red blood cells (sickle red blood cells), which decreases sickle red blood cell deformability, leading to chronic hemolysis and vasoocclusion. These processes can result in severe complications, including chronic pain, end organ dysfunctio...
Source: Hematology ASH Education Program - February 20, 2015 Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research

Outcomes of Unrelated Donor Stem Cell Transplant with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease
Sickle cell disease (SCD) is a hemoglobinopathy affecting 1 in every 500 African American newborns and an estimated 100,000 persons in the United States [1]. Vascular occlusion caused by the sickled RBCs leads to decreased perfusion in target organs and manifests clinically as vaso-occlusive pain crisis, stroke (cerebral infarction), pulmonary restrictive disease, and pulmonary hypertension [2]. Despite advances in supportive care, including the use of hydroxyurea (HU), RBC transfusion, and iron chelation therapy, patients with SCD continue to experience considerable morbidity and mortality in their lifetime.
Source: Biology of Blood and Marrow Transplantation - October 20, 2017 Category: Hematology Authors: Hemalatha G. Rangarajan, Rolla Abu-Arja, Vinita Pai, Gregory M.T. Guilcher, Sandeep Soni Source Type: research

Outcomes of Unrelated Donor Stem Cell Transplantion with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease
Sickle cell disease (SCD) is a hemoglobinopathy affecting 1 in every 500 African American newborns and an estimated 100,000 persons in the United States [1]. Vascular occlusion caused by the sickled RBCs leads to decreased perfusion in target organs and manifests clinically as vaso-occlusive pain crisis, stroke (cerebral infarction), pulmonary restrictive disease, and pulmonary hypertension [2]. Despite advances in supportive care, including the use of hydroxyurea (HU), RBC transfusion, and iron chelation therapy, patients with SCD continue to experience considerable morbidity and mortality in their lifetime.
Source: Biology of Blood and Marrow Transplantation - October 20, 2017 Category: Hematology Authors: Hemalatha G. Rangarajan, Rolla Abu-Arja, Vinita Pai, Gregory M.T. Guilcher, Sandeep Soni Source Type: research

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Backgroundβ-globin gene transfer has the potential for substantial clinical benefit in patients with sickle cell disease (SCD). LentiGlobin Drug Product (DP) contains autologous CD34+ hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV), encoding β-globin with an anti-sickling substitution (T87Q). The safety and efficacy of LentiGlobin gene therapy is being evaluated in the ongoing Phase 1 HGB-206 study (NCT02140554). Results in the initial 7 patients treated with LentiGlobin DP from steady state bone marrow harvested (BMH) HSCs using original DP manufacturing process (Group A) demons...
Source: Blood - November 21, 2018 Category: Hematology Authors: Tisdale, J. F., Kanter, J., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Shi, W., Ribeil, J.-A., Asmal, M., Thompson, A. A., Walters, M. C. Tags: 801. Gene Therapy and Transfer: Gene Therapy for Blood Cell Disorders Source Type: research

Outcome of Age-Adapted Approach to HLA-Identical Related Hematopoietic Stem Cell Transplantation in Severe Sickle Cell Disease: Saudi Experience
In this study, we reviewed the outcome of SCD patients who underwent transplant at our institution using standard protocols (NMA regimen in patients ≥14 years and myeloablative regimen in < 14 years) to address whether age remains a risk factor that influences HSCT outcome in SCD.Children (<14 years) with severe SCD received myeloablative conditioning using one of two regimens: first regimen was cyclophosphamide (Cy) 200mg/kg, busulfan (Bu) 16mg/kg, and thymoglobulin (ATG) 10mg/kg and recently we use thiotepa 8mg/kg, Bu 16mg/kg, and fludarabine (Flu) 160mg/m2. Bu pharmacokinetics was performed to target AUC of 900...
Source: Blood - November 21, 2018 Category: Hematology Authors: Alzahrani, M., Damlaj, M., Essa, M., Alahmari, B., Alaskar, A., Hejazi, A., Basher, E., Abujoub, R., Ghazi, S., Abuelgasim, K., Salama, H., Gmati, G., Alsultan, A. Tags: 732. Clinical Allogeneic Transplantation: Results: Poster II Source Type: research

Outcomes of Hospitalization for Stem Cell Transplant in Sickle Cell Disease: Are We There Yet?
Conclusions: The rate of inpatient mortality with SCT in sickle cell disease is lower than the overall inpatient mortality rate for allogeneic SCT (7%; Godara et al bbmt 2018), indicating a favorable outcome for these patients. Infections do occur commonly during the course of hospitalization, especially in association with GVHD. Length of stay is adversely impacted by occurrence of GVHD, bacterial sepsis, C.difficile infection and viral infections. While we are limited by duration of follow up in our study, these patterns suggest some essential modifiers for inpatient morbidity and mortality, therefore require validation ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Godara, A., Siddiqui, N., Afzal, A., Khan, M., Yared, J., Kansagra, A., Dahiya, S. Tags: 732. Clinical Allogeneic Transplantation: Results: Poster III Source Type: research

Allogenic Hematopoietic Stem Cell Transplantation in Sickle Cell Disease
Sickle cell disease (SCD) is one of the most common monogenic disorders worldwide and affects approximately 100,000 people in the United States alone. SCD can cause numerous complications, including anemia, pain, stroke, and organ failure, which can lead to death. Although there are a few disease-modifying treatments available to patients with SCD, the only current curative option is a hematopoietic stem cell transplant (HSCT). In this review, we will discuss the different approaches to allogeneic HSCT in the treatment of SCD and the outcomes of these approaches.
Source: Transfusion and Apheresis Science - January 10, 2021 Category: Hematology Authors: Dana K. Furstenau, John F. Tisdale Source Type: research