Applicability of and potential barriers preventing allogeneic stem cell transplant in sickle cell patients treated outside a sickle cell program

American Journal of Hematology, EarlyView.
Source: American Journal of Hematology - Category: Hematology Source Type: research

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ckman E, Poirot C, Socié G, Société Française de Greffe de Moelle et de Thérapie Cellulaire Abstract Allogeneic stem cell transplantation remains the only curative treatment for sickle-cell anemia, but the place of myeloablative conditioning remains to be defined. The aim of the present study was to analyze long-term outcomes, including chimerism, sickle-cell anemia-related events and biological data (hemoglobin, reticulocytes, HbS%), and fertility, in a French series of 234 SCA-patients younger than 30 years who received (1988-2012) a matched-sibling-donor stem cell transplantat...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
Discussion This case demonstrates successful cure of pre-B-ALL complicating XLA by alloSCT with restoration of B-cell development and functional antibody response. We are aware of only one previous case of pre-B-ALL in an XLA patient (21), which suggests that human BTK deficiency in itself does not predispose to pre-B-ALL. However, there are data to suggest that BTK may act as a tumor suppressor, and BTK deficiency may predispose to tumor development following a “second hit.” Mice with a genetic deficiency in Slp65, a gene encoding an adaptor protein that functions together with BTK, have a block in progenito...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Publication date: 2019Source: European Journal of Radiology Open, Volume 6Author(s): Eliseo Picchi, Francesca Di Giuliano, Simone Marziali, Silvia Minosse, Valentina Ferrazzoli, Valerio Da Ros, Javid Gaziev, Chiara Adriana Pistolese, Roberto Floris, Francesco GaraciAbstractTo evaluate, by Magnetic Resonance Imaging, if there is a typical pattern or severity of PRES in transplanted children for hemoglobinopathy. Secondary point was to investigate the pattern and severity of PRES in children with thalassemia-THAL and sickle-cell disease-SCD after autologous hematopoietic stem cell transplantation (aHSCT). Finally, we evaluat...
Source: European Journal of Radiology Open - Category: Radiology Source Type: research
This study was done at Johns Hopkins Hospital (Baltimore, MD, USA). Patients aged 2–70 years receiving their first bone marrow transplant were eligible for inclusion in the study. Patients received rabbit-derived intravenous anti-thymocyte globulin 0·5 mg/kg on day −9 and 2 mg/kg on days −8 and −7, intravenous fludarabine 30 mg/m2 on days −6 to −2, intravenous cyclophosphamide 14·5 mg/kg on days −6 and −5, and total body irradiation 400 cGy administered as a single fraction on day −1. We collected unmanipulated bone marrow and infused on day 0. GVHD prophy...
Source: The Lancet Haematology - Category: Hematology Source Type: research
β-globin gene transfer may reduce or eliminate complications in patients with sickle cell disease (SCD). LentiGlobin gene therapy (GT) comprises drug product (DP) made from autologous hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV) encoding β-globin with an anti-s ickling T87Q substitution (HbAT87Q). The safety and efficacy of LentiGlobin GT in adults with SCD is being evaluated in a phase 1 study, HGB-206 (NCT02140554). Patients were initially treated with DP made from bone marrow harvested (BMH) HSCs (Group [Grp] A, fully enrolled), then from DP made from B MH HSCs but using a ...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 81 Source Type: research
It is unknown if improvements in supportive care have altered the risk of calcineurin inhibitor (CNI) mediated posterior reversible encephalopathy syndrome (PRES) in patients with hemoglobinopathies such as sickle cell disease (SCD) and β-thalassemia major. We conducted a multicenter retrospective study to determine prevalence of PRES and associated risk factors in a contemporary cohort of patients with hemoglobinopathies transplanted at US children's hospitals.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 208 Source Type: research
Pre-existing endothelial dysfunction and vascular injury sustained during allogeneic hematopoietic stem cell transplantation (HCT) increases risk for endothelial injury-related complications like posterior reversible encephalopathy (PRES) and transplant-associated thrombotic microangiopathy (TA-TMA) in patients with sickle cell disease (SCD). Herein, we report two SCD HCT recipients in whom PRES-related symptoms resolved only after eculizumab therapy was initiated for underlying TA-TMA.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 189 Source Type: research
In 1984, the first successful allogeneic hematopoietic stem cell transplantation (HSCT) was performed to manage sickle cell disease (SCD). Allogeneic hematopoietic stem cell transplantation remains the only curative treatment for sickle cell disease. Although allogeneic HSCT offers the potential for cure, it can cause a host of complications. These complications include infection, narcotic dependency, alloimmunization, sudden death, psychosocial complications, and transfusion dependency.Given these comorbidities and other underlying conditions, the transplant process poses unique difficulties and represents a heavy psychos...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 662 Source Type: research
Hematopoietic stem cell transplant (HSCT) is the only curative option available for patients living with sickle cell disease (SCD). Stable mixed chimerism is sufficient to reverse the sickle phenotype. The current conditioning regimen of post-transplant cyclophosphamide (PT-Cy) and sirolimus (Sir) has proven to synergistically induce stable mixed chimerism.1 The mechanism of tolerance is not fully understood. Previous studies demonstrated that a combination of Sir and abatacept (CTLA4-Ig) promotes tolerance and prolongs allograft survival.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 266 Source Type: research
We and others have previously reported the safety of allogeneic stem cell transplantation in children with SCD without sibling HLA matched bone marrow donors who commonly have SCDT. The majority of the sibling donors donated bone marrow and received G-CSF mobilized PBSCs (Bhatia/Cairo et al, BMT 2014; Gluckman et al, Blood 2017). Furthermore, only 14-18% of siblings can serve as donors based on HLA match and presence of SCD (Mentzer, Am J Ped Hem/Onc 1994; Walters, BBMT 1996). Unrelated cord blood as a donor source for SCD patients was associated with unacceptable primary graft failure rates (Radhakrishnan/Cairo, BBMT 2013).
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 457 Source Type: research
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