scholarly journals Recent advances in the management of Thalassaemia: A Review Update

2017 ◽  
Vol 6 (1) ◽  
pp. 31-37
Author(s):  
Nihar Ranjan Sarker ◽  
Ashis Kumar Ghosh ◽  
Santosh Kumar Saha ◽  
Abdullah Shahriar
Keyword(s):  
Cord Blood Transplantation ◽  
Fetal Hemoglobin ◽  
B Cell Lymphoma ◽  
Cardiac Complications ◽  
Thalassemia Patient ◽  
Transfusion Therapy ◽  
Hematopoietic Stem ◽  
Blood Transplantation ◽  
Hemoglobin Switching ◽  
Successful Experiment

The discussion of disease management focuses on the use of transfusion therapy and the newly developed oral iron chelators, deferiprone and deferasirox, especially combination of the chelator drugs. It has been also discussed on splenectomy and pediatrician management of endocrinopathies and cardiac complications. In addition, the use of hematopoietic stem cell transplantation has produced cure rates as high as 97%, and the use of cord blood transplantation as well. Major advances have being made in the discovery of critical modifier genes, such as Myb and especially BCL11A (B cell lymphoma 11A), a master regulator of HbF (fetal hemoglobin) and hemoglobin switching. Finally, the year 2010 has brought in the first successful experiment of gene therapy in a ß-thalassemia patient, opening up the perspective of a generalized cure for all ß- Thalassaemia patients.J Shaheed Suhrawardy Med Coll, June 2014, Vol.6(1); 31-37

scholarly journals How I treat thalassemia

Blood ◽  
2011 ◽  
Vol 118 (13) ◽  
pp. 3479-3488 ◽  
Author(s):  
Eliezer A. Rachmilewitz ◽  
Patricia J. Giardina
Keyword(s):  
Single Point ◽  
Cord Blood Transplantation ◽  
Point Mutations ◽  
Clinical Manifestations ◽  
Treatment Strategies ◽  
Thalassemia Major ◽  
Diagnostic Tools ◽  
Cardiac Complications ◽  
Transfusion Therapy ◽  
Hematopoietic Stem

Abstract The purpose of this article is to set forth our approach to diagnosing and managing the thalassemias, including β-thalassemia intermedia and β-thalassemia major. The article begins by briefly describing recent advances in our understanding of the pathophysiology of thalassemia. In the discussion on diagnosing the condition, we cover the development of improved diagnostic tools, including the use of very small fetal DNA samples to detect single point mutations with great reliability for prenatal diagnosis of homozygous thalassemia. In our description of treatment strategies, we focus on how we deal with clinical manifestations and long-term complications using the most effective current treatment methods for β-thalassemia. The discussion of disease management focuses on our use of transfusion therapy and the newly developed oral iron chelators, deferiprone and deferasirox. We also deal with splenectomy and how we manage endocrinopathies and cardiac complications. In addition, we describe our use of hematopoietic stem cell transplantation, which has produced cure rates as high as 97%, and the use of cord blood transplantation. Finally, we briefly touch on therapies that might be effective in the near future, including new fetal hemoglobin inducers and gene therapy.

scholarly journals Minimal change glomerulopathy after unrelated umbilical cord blood transplantation for the treatment of children：case report

2021 ◽  
Author(s):  
Liangliang Ren ◽  
Ling Li ◽  
Lei Zhang ◽  
Xin Li ◽  
xiaorui Fu ◽  
...  
Keyword(s):  
Cord Blood ◽  
Umbilical Cord ◽  
Umbilical Cord Blood ◽  
Cell Lymphoma ◽  
Cord Blood Transplantation ◽  
Blood System ◽  
Minimal Change ◽  
Hematopoietic Stem ◽  
Blood Transplantation ◽  
Child Patient

Umbilical cord blood allogeneic hematopoietic stem cell transplantation(UCBT) has been gradually applied in the treatment of patients with blood system diseases. This paper reports a case of a child patient with highly invasive T-cell lymphoma who underwent UCBT after chemotherapy and developed minimal change glomerulopathy after transplantation.

scholarly journals THE STUDY OF TRANSFUSION THERAPY IN CORD BLOOD TRANSPLANTATION: COMPARISON OF CONVENTIONAL CORD BLOOD TRANSPLANTATION AND MINI-CORD BLOOD TRANSPLANTAITON

2011 ◽  
Vol 57 (3) ◽  
pp. 169-172
Author(s):  
Yasuji Kouzai ◽  
Tamae Hamaki ◽  
Kazunari Yamada ◽  
Katsuhiro Kogure ◽  
Kouichi Kajiwara ◽  
...  
Keyword(s):  
Cord Blood ◽  
Cord Blood Transplantation ◽  
Transfusion Therapy ◽  
Blood Transplantation

scholarly journals The replication rate of human hematopoietic stem cells in vivo

Blood ◽  
2011 ◽  
Vol 117 (17) ◽  
pp. 4460-4466 ◽  
Author(s):  
Sandra N. Catlin ◽  
Lambert Busque ◽  
Rosemary E. Gale ◽  
Peter Guttorp ◽  
Janis L. Abkowitz
Keyword(s):  
Stem Cells ◽  
Hematopoietic Stem Cells ◽  
Cord Blood Transplantation ◽  
Cell Labeling ◽  
Replication Rate ◽  
Hematopoietic Stem ◽  
Daughter Cells ◽  
Blood Transplantation ◽  
Human Hscs

Abstract Hematopoietic stem cells (HSCs) replicate (self-renew) to create 2 daughter cells with capabilities equivalent to their parent, as well as differentiate, and thus can both maintain and restore blood cell production. Cell labeling with division-sensitive markers and competitive transplantation studies have been used to estimate the replication rate of murine HSCs in vivo. However, these methods are not feasible in humans and surrogate assays are required. In this report, we analyze the changing ratio with age of maternal/paternal X-chromosome phenotypes in blood cells from females and infer that human HSCs replicate on average once every 40 weeks (range, 25-50 weeks). We then confirm this estimate with 2 independent approaches, use the estimate to simulate human hematopoiesis, and show that the simulations accurately reproduce marrow transplantation data. Our simulations also provide evidence that the number of human HSCs increases from birth until adolescence and then plateaus, and that the ratio of contributing to quiescent HSCs in humans significantly differs from mouse. In addition, they suggest that human marrow failure, such as the marrow failure that occurs after umbilical cord blood transplantation and with aplastic anemia, results from insufficient numbers of early progenitor cells, and not the absence of HSCs.

Successful engraftment after reduced-intensity umbilical cord blood transplantation for myelofibrosis

Blood ◽  
2010 ◽  
Vol 116 (4) ◽  
pp. 649-652 ◽  
Author(s):  
Shinsuke Takagi ◽  
Yasunori Ota ◽  
Naoyuki Uchida ◽  
Koichi Takahashi ◽  
Kazuya Ishiwata ◽  
...  
Keyword(s):  
Cord Blood ◽  
Umbilical Cord ◽  
Umbilical Cord Blood ◽  
Cord Blood Transplantation ◽  
Donor Cell ◽  
Umbilical Cord Blood Transplantation ◽  
Hematopoietic Stem ◽  
Blood Transplantation ◽  
Marrow Fibrosis ◽  
Reduced Intensity

Abstract Although allogeneic hematopoietic stem cell transplantation has recently been applied to patients with myelofibrosis with reproducible engraftment and resolution of marrow fibrosis, no data describe the outcomes of umbilical cord blood transplantation. We describe 14 patients with primary (n = 1) and secondary myelofibrosis (n = 13) who underwent reduced-intensity umbilical cord blood transplantation. Conditioning regimens included fludarabine and graft-versus-host disease prophylaxis composed cyclosporine/tacrolimus alone (n = 6) or a combination of tacrolimus and mycophenolate mofetil (n = 8). Thirteen patients achieved neutrophil engraftment at a median of 23 days. The cumulative incidence of neutrophil and platelet engraftment was 92.9% at day 60 and 42.9% at day 100, respectively. Posttransplantation chimerism analysis showed full donor type in all patients at a median of 14 days. The use of umbilical cord blood could be feasible even for patients with severe marrow fibrosis, from the viewpoint of donor cell engraftment.

scholarly journals INFECTIOUS COMPLICATIONS AFTER UMBILICAL CORD-BLOOD TRANSPLANTATION FROM UNRELATED DONORS

2016 ◽  
Vol 8 ◽  
pp. 2016051 ◽  
Author(s):  
Juan Montoro ◽  
Jaime Sanz
Keyword(s):  
Stem Cell ◽  
Cord Blood ◽  
Umbilical Cord ◽  
Umbilical Cord Blood ◽  
Cord Blood Transplantation ◽  
Infectious Complications ◽  
Unrelated Donor ◽  
Alternative Source ◽  
Hematopoietic Stem ◽  
Blood Transplantation

Umbilical cord-blood (UCB) is a well-recognized alternative source of stem cells for unrelated donor hematopoietic stem cell transplantation (HSCT). As compared with other stem cell sources from adult donors, it has the advantages of immediate availability of cells, absence of risk to the donor and reduced risk of graft-versus-host disease despite donor-recipient HLA disparity. However, the use of UCB is limited by the delayed post-transplant hematologic recovery due, at least in part, to the reduced number of hematopoietic cells in the graft and the delayed or incomplete immune reconstitution. As a result, severe infectious complications continue to be a leading cause of morbidity and mortality following UCB transplantation (UCBT). We will address the complex differences in the immune properties of UCB and review the incidence, characteristics, risk factors, and severity of bacterial, fungal and viral infectious complications in patients undergoing UCBT.

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