Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions
Sickle cell disease-related organ injuries cannot be prevented despite hydroxyurea use, infection prophylaxis and supportive therapies. As a consequence, disease-related mortality reaches 14% in adolescents and young adults. Hematopoietic stem cell transplantation is a unique curative therapeutic ap...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Galenos Publishing House
2015-07-01
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| Series: | Turkish Journal of Hematology |
| Subjects: | |
| Online Access: | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-32967 |
| _version_ | 1797921019246149632 |
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| author | Hakan Özdoğu Can Boğa |
| author_facet | Hakan Özdoğu Can Boğa |
| author_sort | Hakan Özdoğu |
| collection | DOAJ |
| description | Sickle cell disease-related organ injuries cannot be prevented despite hydroxyurea use, infection prophylaxis and supportive therapies. As a consequence, disease-related mortality reaches 14% in adolescents and young adults. Hematopoietic stem cell transplantation is a unique curative therapeutic approach for sickle cell disease. Myeloablative allogeneic hematopoietic stem cell transplantation is curative for children with sickle cell disease. Current data indicate that long term disease-free survival is about 90% and overall survival about 95% after transplantation. However, it is toxic in adults due to organ injuries. In addition, this curative treatment approach has several limitations, such as difficulties finding donors, transplant-related mortality, graft loss, graft-versus-host disease (GVHD) and infertility. Engraftment effectivity and toxicity for transplantations performed with nonmyeloablative reduced-intensity regimens in adults are being investigated in phase I/II trials at many centers. Preliminary data indicate that GVHD could be prevented in transplantations performed using reduced-intensity regimens. It is necessary to develop novel regimens to prevent graft loss and reduce the risk of GVHD. |
| first_indexed | 2024-04-10T14:10:00Z |
| format | Article |
| id | doaj.art-7c92cda9c1f2408da5174e1b14f1eaf7 |
| institution | Directory Open Access Journal |
| issn | 1308-5263 |
| language | English |
| last_indexed | 2024-04-10T14:10:00Z |
| publishDate | 2015-07-01 |
| publisher | Galenos Publishing House |
| record_format | Article |
| series | Turkish Journal of Hematology |
| spelling | doaj.art-7c92cda9c1f2408da5174e1b14f1eaf72023-02-15T16:09:48ZengGalenos Publishing HouseTurkish Journal of Hematology1308-52632015-07-0132319520510.4274/tjh.2014.0311TJH-32967Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and SolutionsHakan Özdoğu0Can Boğa1Başkent University Faculty Of Medicine, Department Of Internal Medicine, Division Of Hematology, Adana, TurkeyBaşkent University Faculty Of Medicine, Department Of Internal Medicine, Division Of Hematology, Adana, TurkeySickle cell disease-related organ injuries cannot be prevented despite hydroxyurea use, infection prophylaxis and supportive therapies. As a consequence, disease-related mortality reaches 14% in adolescents and young adults. Hematopoietic stem cell transplantation is a unique curative therapeutic approach for sickle cell disease. Myeloablative allogeneic hematopoietic stem cell transplantation is curative for children with sickle cell disease. Current data indicate that long term disease-free survival is about 90% and overall survival about 95% after transplantation. However, it is toxic in adults due to organ injuries. In addition, this curative treatment approach has several limitations, such as difficulties finding donors, transplant-related mortality, graft loss, graft-versus-host disease (GVHD) and infertility. Engraftment effectivity and toxicity for transplantations performed with nonmyeloablative reduced-intensity regimens in adults are being investigated in phase I/II trials at many centers. Preliminary data indicate that GVHD could be prevented in transplantations performed using reduced-intensity regimens. It is necessary to develop novel regimens to prevent graft loss and reduce the risk of GVHD.https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-32967sickle cell diseasehematopoietic stem cell transplantationgraft-versus-host diseasegraft rejectionconditioning |
| spellingShingle | Hakan Özdoğu Can Boğa Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions Turkish Journal of Hematology sickle cell disease hematopoietic stem cell transplantation graft-versus-host disease graft rejection conditioning |
| title | Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions |
| title_full | Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions |
| title_fullStr | Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions |
| title_full_unstemmed | Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions |
| title_short | Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions |
| title_sort | hematopoietic stem cell transplantation in adult sickle cell disease problems and solutions |
| topic | sickle cell disease hematopoietic stem cell transplantation graft-versus-host disease graft rejection conditioning |
| url | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-32967 |
| work_keys_str_mv | AT hakanozdogu hematopoieticstemcelltransplantationinadultsicklecelldiseaseproblemsandsolutions AT canboga hematopoieticstemcelltransplantationinadultsicklecelldiseaseproblemsandsolutions |