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Hematopoietic stem cell transplantation for children with sickle cell diseaseOringanje C, Nemecek E, Oniyangi O
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SummaryTransplantation of blood-forming stem cells for children with sickle cell diseaseSickle cell disease is a genetic disorder mainly characterized by the presence of deformed, sickle-shaped red blood cells in the blood stream. These cells deprive tissues of blood and oxygen resulting in periodic and recurrent painful attacks. Complications include acute chest syndrome and stroke. Although sickle cell disease is responsive to preventive and supportive measures such as the use of prophylactic antibodies and periodic blood transfusion, these do not provide a cure. The use of hematopoietic (blood forming) stem cell transplantation involves replacing the deformed red blood cells and the cells that produced these hematopoietic stem cells with normal cells from a healthy donor thereby producing normal red blood cells. Hematopoietic stem cell transplantation involves the stem cells from another person. These can be derived from either the bone marrow or blood (umbilical cord blood or peripheral blood). Before the transplantation, the immune system is suppressed to prevent rejection of the new stem cells. There are no randomized controlled trials assessing the benefits and risks; the most appropriate source of stem cells; or the most eligible participants (those who have experience severe complication or those who have not) of the procedure in children with sickle cell disease.
This is a Cochrane review abstract and plain language summary, prepared and maintained by The Cochrane Collaboration, currently published in The Cochrane Database of Systematic Reviews 2010 Issue 1, Copyright © 2010 The Cochrane Collaboration. Published by John Wiley and Sons, Ltd.. The full text of the review is available in The Cochrane Library (ISSN 1464-780X).
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January 21. 2009 AbstractBackgroundSickle cell disease is a genetic disorder involving a defect in the red blood cells due to its sickled hemoglobin. The main therapeutic interventions include preventive and supportive measures. Hematopoietic stem cell transplantations are carried out with the aim of replacing the defective cells and their progenitors (hematopoietic (i.e. blood forming) stem cells) in order to correct the disorder. ObjectivesTo determine whether stem cell transplantation can improve survival and prevent symptoms and complications associated with sickle cell disease. To examine the risks of stem cell transplantation against the potential long-term gain for people with sickle cell disease. Search strategyWe searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Group's Haemoglobinopathies Trials Register complied from electronic searches of the Cochrane Central Register of Controlled Trials (Clinical Trials) (updated each new issue of The Cochrane Library) and quarterly searches of MEDLINE. Unpublished work was identified by searching the abstract books of major conference proceedings and we conducted a search of the website: www.ClinicalTrials.gov. Date of the most recent search of the Group's Haemoglobinopathies Trials Register: 26th August 2009. Selection criteriaRandomized controlled and quasi-randomized studies that compared any method of stem cell transplantation with either each other or with any of the preventive or supportive interventions (e.g. periodic blood transfusion, use of hydroxyurea, antibiotics, pain relievers, supplemental oxygen) in children under 16 years of age irrespective of the type of sickle cell disease, gender and setting. Data collection and analysisNo relevant trials have been identified. Main resultsTen trials were identified by the initial search and none for the update. None of the trials were suitable for inclusion in this review. Authors' conclusionsReports on the use of hematopoietic stem cell transplantation improving survival and preventing symptoms and complications associated with sickle cell disease are currently limited to observational and other less robust studies. No randomized controlled trial has assessed the benefit or risk of different types of hematopoietic stem cell transplantations in children. Thus, this systematic review identifies the need for a multicentre randomized controlled trial assessing the benefits and possible risks of different types of hematopoietic stem cell transplantations comparing sickle status and severity of disease in children. |