Hematopoietic stem cell transplantation for children with sickle cell disease

Background Sickle 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. Objectives To 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 strategy We 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 Unpublished work was identified by searching the abstract books of major conference proceedings and we conducted a search of the website:www.ClinicalTrials.gov.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: September 2008. Selection criteria Randomized 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 analysis No relevant trials have been identified. Main results Ten trials were identified by the initial search of which none were suitable for inclusion in this review. Authors' conclusions Reports 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.